Nowotwory mózgu u dzieci
Rokowania, prognozy i postęp choroby

Nowotwory mózgu stanowią około 25% wszystkich nowotworów wieku dziecięcego i są główną przyczyną zgonów onkologicznych u pacjentów poniżej 19 roku życia. Wskaźnik 5-letniego przeżycia dla wszystkich pierwotnych nowotworów mózgu u dzieci wynosi około 83,1-83,9%, natomiast dla guzów złośliwych spada do około 75,6%. W Polsce obserwuje się niższe wartości: 5-letnie przeżycie wynosi około 60,9%, a 10-letnie 58,2%. Kluczowe czynniki prognostyczne to wiek w momencie diagnozy (najgorsze rokowanie u niemowląt, aHR 0,47 dla wieku 10-19 lat), rasa i pochodzenie etniczne (wyższe ryzyko zgonu u pacjentów afroamerykańskich i latynoamerykańskich, aHR 1,21), lokalizacja guza (najgorsze rokowanie dla guzów pnia mózgu, aHR 2,62) oraz stopień złośliwości histologicznej (grade III aHR 3,39, grade IV aHR 2,18). Glejaki wysokiego stopnia (HGG) mają 5-letni wskaźnik przeżycia poniżej 20%, a rozlane glejaki pnia mózgu (DIPG) cechują się wyjątkowo złym rokowaniem ze względu na brak możliwości resekcji.

Nowotwory mózgu u dzieci – Wprowadzenie

Nowotwory mózgu stanowią drugą najczęstszą grupę nowotworów u dzieci (po białaczkach), odpowiadając za około 25% wszystkich nowotworów wieku dziecięcego. Każdego roku w Stanach Zjednoczonych diagnozuje się około 4000-5000 nowych przypadków guzów mózgu i rdzenia kręgowego u dzieci i młodzieży.12 Co niepokojące, w ciągu ostatnich 20 lat obserwuje się wzrost liczby zachorowań o około 0,7% rocznie.3 Nowotwory mózgu są główną przyczyną zgonów związanych z chorobą nowotworową wśród dzieci i młodzieży poniżej 19 roku życia.45

Guzy ośrodkowego układu nerwowego (OUN) stanowią heterogenną grupę schorzeń o różnej biologii, lokalizacji, histologii oraz rokowaniu. Mimo znacznego postępu w terapii, jaki osiągnięto w ostatnich dekadach, choroby te pozostają ogromnym wyzwaniem dla współczesnej medycyny, zarówno pod względem diagnostyki, jak i efektywnego leczenia.67

Rokowanie w nowotworach mózgu u dzieci – perspektywa ogólna

Wskaźniki przeżycia w przypadku nowotworów mózgu u dzieci są zróżnicowane i zależą od wielu czynników. Ogólny 5-letni wskaźnik przeżycia dla wszystkich pierwotnych nowotworów mózgu u dzieci wynosi około 83,1-83,9%. W przypadku guzów złośliwych, wskaźnik ten spada do około 75,6%.8 Według badań prowadzonych w Polsce, całkowite 5-letnie przeżycie dla wszystkich typów nowotworów mózgu u dzieci wynosi około 60,9%, a 10-letnie około 58,2%.9

Pomimo stosunkowo wysokich wskaźników przeżycia, należy pamiętać, że u około 95% dzieci, które przeżyją nowotwór mózgu, rozwijają się przewlekłe problemy zdrowotne, wtórne nowotwory, zaburzenia poznawcze oraz obserwuje się skrócenie długości życia z powodu toksyczności obecnych opcji leczenia.10

Kluczowe czynniki prognostyczne

Na podstawie szeroko zakrojonych badań epidemiologicznych i klinicznych zidentyfikowano kilka kluczowych czynników wpływających na rokowanie w nowotworach mózgu u dzieci:11

  • Wiek w momencie diagnozy
  • Rasa i pochodzenie etniczne
  • Płeć
  • Stopień złośliwości guza (grade)
  • Podtyp histologiczny nowotworu
  • Lokalizacja anatomiczna guza
  • Status socjoekonomiczny
  • Region geograficzny

1112

Wpływ wieku na rokowanie

Wiek dziecka w momencie diagnozy jest jednym z najważniejszych czynników prognostycznych. Badania wykazały, że dzieci diagnozowane w starszym wieku mają lepsze rokowanie w porównaniu z młodszymi pacjentami, szczególnie niemowlętami. Według danych epidemiologicznych, skorygowany współczynnik ryzyka (aHR) dla dzieci w wieku 1-10 lat wynosi 0,60 (95% CI: 0,55-0,67), a dla dzieci w wieku 10-19 lat 0,47 (95% CI: 0,42-0,52) w porównaniu z niemowlętami.13 Wskaźniki przeżycia poprawiają się wraz z wiekiem w momencie diagnozy, przy czym najgorsze rokowanie obserwuje się u pacjentów poniżej pierwszego roku życia.14

Szczególnie niekorzystne rokowanie mają guzy wrodzone, gdzie całkowite przeżycie wynosi mniej niż 30%.15

Rasa i pochodzenie etniczne

Istnieją istotne różnice w przeżywalności związane z rasą i pochodzeniem etnicznym. Pacjenci pochodzenia kaukaskiego (nie-Latynoamerykanie) wykazują niższe ryzyko zgonu w porównaniu z pacjentami pochodzenia afroamerykańskiego (nie-Latynoamerykanie) i latynoamerykańskiego. Współczynnik ryzyka dla obu tych grup wynosi 1,21 (95% CI: 1,11-1,32) w porównaniu z populacją kaukaską.16

Lokalizacja guza

Lokalizacja anatomiczna nowotworu ma kluczowe znaczenie dla rokowania. Guzy umiejscowione w pniu mózgu wiążą się z najwyższym ryzykiem zgonu (aHR: 2,62; 95% CI: 2,41-2,85), następnie guzy mózgowia (aHR: 1,63; 95% CI: 1,46-1,81) w porównaniu z guzami zlokalizowanymi w płatach mózgu.17 Lokalizacja w pniu mózgu jest szczególnie niekorzystnym czynnikiem prognostycznym również według analiz jednozmiennych.18

Stopień złośliwości (grade)

Stopień złośliwości histologicznej według klasyfikacji WHO jest jednym z najsilniejszych czynników prognostycznych. Ryzyko zgonu wzrasta wraz z wyższym stopniem złośliwości:19

  • Grade II – aHR: 1,32 (95% CI: 1,07-1,62)
  • Grade III – aHR: 3,39 (95% CI: 2,74-4,19)
  • Grade IV – aHR: 2,18 (95% CI: 1,80-2,64)

19

Guzy wysokiego stopnia złośliwości (grade III-IV) są niezależnym czynnikiem niekorzystnego rokowania w analizach wieloczynnikowych.20

Podtypy histologiczne

Różne podtypy histologiczne nowotworów mózgu wiążą się z odmiennym rokowaniem. W porównaniu z glejakami niskiego stopnia złośliwości (low-grade glioma), inne typy histologiczne wykazują zwiększone ryzyko zgonu:21

  • Glejaki wysokiego stopnia (high-grade glioma) – aHR: 7,92 (95% CI: 7,09-8,85)
  • Prymitywne guzy neuroektodermalne (PNET) – aHR: 4,72 (95% CI: 4,15-5,37)
  • Rdzeniak (medulloblastoma) – aHR: 3,11 (95% CI: 2,79-3,47)
  • Inne glejaki – aHR: 3,38 (95% CI: 3,08-3,71)
  • Wyściółczaki (ependymal tumors) – aHR: 2,20 (95% CI: 1,95-2,48)

21

Kilka czynników związanych z leczeniem ma istotny wpływ na rokowanie:

  • Zakres resekcji chirurgicznej – niepełna resekcja chirurgiczna jest niezależnym czynnikiem niekorzystnego rokowania22
  • Powikłania po leczeniu chirurgicznym – istotnie pogarszają rokowanie23
  • Progresja choroby podczas terapii (chemioterapii lub radioterapii) – ma bardzo negatywny wpływ na przeżycie24

Rokowanie w konkretnych typach nowotworów mózgu u dzieci

Rokowanie znacząco różni się w zależności od typu histologicznego nowotworu:

Glejaki niskiego stopnia złośliwości (LGG)

Gwiaździak włosowatokomórkowy (pilocytic astrocytoma) ma najwyższe wskaźniki przeżycia po diagnozie.25 Ogólnie, glejaki niskiego stopnia złośliwości wykazują najlepsze rokowanie, z 5-letnim wskaźnikiem przeżycia wynoszącym około 83,3%.26 Diagnoza LGG jest jedynym czynnikiem przewidującym dobre rokowanie w analizach wieloczynnikowych.27

Glejaki wysokiego stopnia złośliwości (HGG)

Glejaki wysokiego stopnia złośliwości (WHO grade IV) mają najwyższy wskaźnik śmiertelności spośród wszystkich nowotworów mózgu u dzieci, z 5-letnim wskaźnikiem przeżycia poniżej 20% pomimo maksymalnych interwencji terapeutycznych.28 W badaniach prowadzonych w Polsce odnotowano 0% przeżycia w 72 miesiącu dla glejaków wysokiego stopnia.29 Glejak wielopostaciowy (glioblastoma) ma około 20% wskaźnik 5-letniego przeżycia.30

HGG powodują największy odsetek zgonów (44,2%) wśród wszystkich nowotworów mózgu u dzieci.31

Rozlane glejaki linii środkowej (DIPG)

Rozlane glejaki pnia mózgu (DIPG) mają wyjątkowo złe rokowanie ze względu na ich wewnętrzną naturę, która uniemożliwia przeprowadzenie resekcji. Te nowotwory często wykazują zmiany genomowe w białku p53, receptorze alfa płytkopochodnego czynnika wzrostu i receptorze typu I aktywiny A.32 Różnorodność mutacji przyczynia się do znacznej heterogenności międzyguzowej w DIPG, co komplikuje strategie leczenia.33

Rdzeniak (medulloblastoma)

Rdzeniak wykazuje 5-letnie przeżycie na poziomie około 48,1% według badań prowadzonych w Polsce.34 Jest to drugi, po glejakach wysokiego stopnia, nowotwór odpowiadający za znaczny odsetek zgonów wśród dzieci z nowotworami mózgu.35

Klinicznym wyzwaniem w przypadku rdzeniaka jest wysoce zróżnicowana odpowiedź pacjentów na terapię. Wysokie poziomy ekspresji receptora neurofropiliny-3 (TrkC) korelują z korzystnym rokowaniem w rdzeniaku, co sugeruje molekularne podłoże zmienności wyników leczenia.36

Wyściółczak (ependymoma)

Wyściółczaki stanowią około 5% nowotworów mózgu u dzieci i występują częściej w tylnym dole czaszki.37 Wskaźnik 5-letniego przeżycia dla wyściółczaków wynosi około 56,6% według badań polskich.38 Skuteczność chemioterapii po radioterapii w leczeniu wyściółczaków w różnych sytuacjach klinicznych jest nadal badana, a chemioterapia jest stosowana w przypadkach wysokiego ryzyka, niecałkowitej resekcji lub nawrotu choroby.39

Atypowy teratoidny/rabdoidny guz (AT/RT)

AT/RT wraz z glejakami wysokiego stopnia mają najniższe wskaźniki przeżycia względnego po diagnozie dla pacjentów w wieku 0-19 lat z guzem mózgu.40 AT/RT jest również jednym z głównych typów nowotworów odpowiadających za znaczną liczbę zgonów wśród dzieci z nowotworami mózgu.41

Molekularne czynniki prognostyczne

Postęp w dziedzinie badań molekularnych i genetycznych doprowadził do identyfikacji istotnych czynników prognostycznych na poziomie molekularnym:

Zmiany epigenetyczne

Profil epigenetyczny nowotworów mózgu może być użyteczny jako:42

  • Potencjalne cele dla leków
  • Markery prognostyczne
  • Narzędzia do klasyfikacji nowotworów
  • Podstawa do zrozumienia rozwoju nowotworów

42

Zmiany epigenetyczne są niezbędne dla transformacji nowotworowej, ponieważ tkanki guza wykazują nieprawidłowe wzorce metylacji w porównaniu ze zdrowymi tkankami.43 Komórki nowotworowe wykorzystują również mechanizmy epigenetyczne do ucieczki przed chemioterapią i nadzorem immunologicznym gospodarza, dlatego poprawa zrozumienia tych mechanizmów może poprawić wyniki leczenia.44

Mutacje histonowe

Mutacje histonowe stanowią nieco ponad połowę wszystkich przypadków dziecięcych glejaków wysokiego stopnia. Klasyfikacja pHGG opiera się na podgrupach molekularnych i istotnych korelacjach klinicznych (tj. wiek, lokalizacja anatomiczna i rokowanie).45

Największy jak dotąd zbiór danych pHGG został wykorzystany w badaniu retrospektywnym, które ujawniło dziesięć podgrup opartych na określonych genach i procesach oraz pokazało, że mutacje histonowe współwystępują z odrębnymi modyfikacjami i ścieżkami downstream.46

Profile ekspresji genów

Opracowano metody przewidywania klas fenotypowych guzów mózgu, takich jak typ guza mózgu lub wynik leczenia, dla próbek guzów mózgu na podstawie profili ekspresji genów.47 Wynik kliniczny pacjentów (np. dzieci) z rdzenikami jest wysoce przewidywalny w oparciu o profile ekspresji genów ich guzów w momencie diagnozy.48

Metody te pozwalają na przewidywanie skuteczności leczenia guza mózgu poprzez określenie profilu ekspresji genów co najmniej jednego informatywnego genu, przy czym profil ekspresji genów jest skorelowany z wynikiem leczenia, co umożliwia klasyfikację próbki pod względem wyniku leczenia.49

Rokowanie neuropsychologiczne

Deficyty neurologiczne, poznawcze i neuropsychologiczne są najbardziej upośledzającymi długoterminowymi skutkami guzów mózgu u dzieci.50 Problemy poznawcze i neuropsychologiczne występują częściej w tej populacji w porównaniu z osobami, które przeżyły inne nowotwory dziecięce.51

Deficyty poznawcze

Wielu pacjentów doświadcza zarówno znaczących globalnych deficytów z obniżonym ilorazem inteligencji (IQ), jak i specyficznych deficytów neuropsychologicznych, takich jak upośledzenie funkcji wykonawczych, pamięci i szybkości przetwarzania. Szacuje się, że duża część pacjentów leczonych z powodu guza OUN, w zakresie od 40% do 100%, doświadczy deficytu w domenie neurokognitywnej.52

Dysfunkcje poznawcze związane z rakiem dotykają około jednej trzeciej osób, które przeżyły raka dziecięcego w USA. Neurokognitywne późne efekty u dzieci, które przeżyły guz mózgu, są stosunkowo powszechne i odgrywają znaczącą rolę w modyfikowaniu jakości życia związanej ze zdrowiem (HR-QOL).53

Najczęstsze deficyty funkcjonalne

Najbardziej dotknięte funkcje obejmują uwagę, pamięć roboczą i szybkość przetwarzania. Funkcje wykonawcze (EF) obejmują hamowanie, elastyczność umysłową, planowanie i podejmowanie decyzji, rozumowanie abstrakcyjne, tworzenie pojęć, rozwiązywanie problemów i świadomość.54

Spadki IQ są widoczne u dzieci, które przeżyły nowotwór OUN, już w pierwszym roku po diagnozie i leczeniu, z potencjalną progresją przez następne 5-7 lat.55

Postęp w przewidywaniu rokowania

Ostatnie postępy w dziedzinie badań nad nowotworami mózgu u dzieci przynoszą nowe możliwości w zakresie przewidywania rokowania:

Uczenie głębokie i multimodalne modele predykcyjne

Wprowadzenie głębokiego uczenia zarówno w obrazowaniu, jak i genomice znacznie zaawansowało analizę danych biomedycznych. Opracowano ramy głębokiego uczenia (DL), które łączą te dwie modalności w celu przewidywania rokowania guzów mózgu.56

Wyniki badań pokazują, że proponowane strategie fuzji danych multimodalnych umożliwiają zintegrowanym modelom osiągnięcie lepszej dokładności predykcji niż przy użyciu histopatologii i danych genomowych w izolacji.57 Strategia wczesnej fuzji okazała się najlepiej działającym modelem w przeprowadzonych eksperymentach.58

Wykorzystując koncepcję uczenia transferowego, modele multimodalne dla glejaków dziecięcych mogą być bezpośrednio wykorzystywane do przewidywania przeżycia w innych podtypach z rozsądną wydajnością.59 To sugeruje, że podejście multimodalnego głębokiego uczenia jest obiecującym sposobem uchwycenia trajektorii choroby pacjentów z guzami mózgu.60

Markery MRI jako predyktory funkcji poznawczych

Istnieje potencjał wykorzystania markerów rezonansu magnetycznego (MRI) jako dobrych wskaźników obciążenia guzami mózgu i ich leczenia, a zatem prawdopodobnie jako predyktorów upośledzenia poznawczego na poziomie indywidualnego pacjenta.61

Przewidywanie wyników neurokognitywnych na poziomie indywidualnym umożliwiłoby terminowe i dostosowane wsparcie ze strony szkoły i usług zdrowotnych, promując lepsze wyniki dla tych dzieci, przy efektywnym priorytetowym traktowaniu ograniczonych zasobów opieki zdrowotnej dla osób najbardziej zagrożonych.62

Biorąc pod uwagę zwiększoną liczbę dorosłych, którzy przeżyli nowotwory mózgu w dzieciństwie, a także gorsze długoterminowe wyniki poznawcze, edukacyjne i zawodowe oraz znaczne obciążenie, jakie stanowi to dla pacjentów, rodzin i opieki zdrowotnej, konieczne jest przeprowadzenie dalszych badań w tym zakresie.63

Przyszłe kierunki poprawy rokowania

Aby poprawić wyniki leczenia dzieci z nowotworami mózgu, kluczowe jest lepsze zrozumienie choroby, znalezienie skuteczniejszych metod leczenia guzów mózgu oraz zmniejszenie toksyczności leczenia.64

Terapie celowane molekularnie

Zrozumienie mechanizmów molekularnych doprowadziło do zwiększonej dokładności i niezawodności diagnozy guza, ujawniło potencjalne cele terapeutyczne i dostarcza racjonalnej podstawy do wzmocnienia lub zmniejszenia intensywności terapii.65

Wiedza o genomowych czynnikach napędzających dziecięce glejaki wysokiego stopnia zaowocowała kilkoma badaniami klinicznymi testującymi leki ukierunkowane przeciwko tej grupie guzów. Szlak PI3K/AKT/mTOR, który promuje przeżycie i wzrost komórek i jest aktywowany w kilku nowotworach, stanowi jeden z celów terapeutycznych.66

Immunoterapia

Napędzany sukcesem w niektórych nowotworach u dorosłych, nastąpił wzrost liczby badań klinicznych wykorzystujących różne formy immunoterapii zarówno dla dorosłych, jak i dziecięcych guzów OUN.67 Inhibitory punktów kontrolnych układu immunologicznego są badane w guzach mózgu u dorosłych, a kilka badań klinicznych jest w toku, aby wykorzystać inhibitory anty-PD1 i CTLA-4 w dziecięcych glejakach wysokiego stopnia.68

Aktywność kliniczna blokady punktów kontrolnych koreluje z trzema głównymi zmiennymi:69

  • Liczba niesynonimicznych/przesunięcia ramki odczytu mutacji somatycznych w guzie, co skutkuje produkcją neoantygenów
  • Wysoka ekspresja ligandu PD-1 w komórkach nowotworowych
  • Częstotliwość aktywowanych limfocytów T CD8+ w krążeniu

69

Użyteczność i sukces terapii inhibitorami punktów kontrolnych w nowotworach mózgu u dzieci pozostają jeszcze do wykazania.70

Potrzeba finansowania badań

Mimo że nowotwory mózgu są obecnie główną przyczyną zgonów związanych z rakiem u dzieci i młodzieży w wieku poniżej 19 lat, badania nad dziecięcymi nowotworami mózgu są niedofinansowane. Tylko 4% federalnego finansowania badań nad rakiem jest przeznaczane na badania nad nowotworami wieku dziecięcego.71

Zwiększenie nakładów na badania w tej dziedzinie jest kluczowe dla poprawy rokowania i jakości życia dzieci z nowotworami mózgu.

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  1. 10.04.2026
  2. www.leksykon.com.pl

Materiały źródłowe

  • #1 Key Statistics for Brain and Spinal Cord Tumors in Children | American Cancer Society
    https://www.cancer.org/cancer/types/brain-spinal-cord-tumors-children/about/key-statistics.html
    Brain and spinal cord tumors are the second most common cancers in children (after leukemia). They account for about 1 out of 4 childhood cancers. More than 4,000 brain and spinal cord tumors are diagnosed each year in children and teens. The incidence rate (number of tumors per 100,000 children) has not changed much in recent years. […] About 3 out of 4 children with brain tumors (all types combined) survive at least 5 years after being diagnosed. But the outlook can vary a great deal based on the type of tumor, where it is, and other factors.
  • #2 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Pediatric brain tumors affect all races, both sexes, and can be diagnosed at any age. Over the last 20 years, there has been a 0.7% increase in cases each year. […] Close to 5,000 children and teens are diagnosed with brain and spinal cord tumors each year. […] Brain tumors are the 2nd most common cancers in children, after leukemia, and account for 15.9% of all new childhood cancer cases. Among all childhood cancers, brain tumors are the leading cause of death. […] Astrocytomas, including glioblastoma multiforme, are the most common type of glioma, making up half of all childhood brain tumors, according to Johns Hopkins Medicine. They have a survival rate of about 25%. […] Three out of four (75.5%) children diagnosed with a brain tumor will survive at least 5 years. […] Congenital brain tumors have a poor prognosis with overall survival of less than 30%.
  • #3 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Pediatric brain tumors affect all races, both sexes, and can be diagnosed at any age. Over the last 20 years, there has been a 0.7% increase in cases each year. […] Close to 5,000 children and teens are diagnosed with brain and spinal cord tumors each year. […] Brain tumors are the 2nd most common cancers in children, after leukemia, and account for 15.9% of all new childhood cancer cases. Among all childhood cancers, brain tumors are the leading cause of death. […] Astrocytomas, including glioblastoma multiforme, are the most common type of glioma, making up half of all childhood brain tumors, according to Johns Hopkins Medicine. They have a survival rate of about 25%. […] Three out of four (75.5%) children diagnosed with a brain tumor will survive at least 5 years. […] Congenital brain tumors have a poor prognosis with overall survival of less than 30%.
  • #4 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    Approximately 3.9% of all brain tumors cases diagnosed occur in children ages 0-14 years. […] An estimated 3,920 new cases of primary childhood brain tumors are expected to be diagnosed in 2023. […] Brain tumors are the most commonly diagnosed solid cancer in children ages 0-14 years, as well as the leading cause of childhood cancer-related death. […] The five-year relative survival rate for all primary childhood brain tumors is 83.1%. […] For primary malignant childhood tumors, the five-year relative survival rate is 75.6%. […] The five-year relative survival rate for all primary pediatric brain tumors (0-19) is 83.9%. […] For malignant tumors, the five-year relative survival rate is 75.6%. […] Pediatric brain tumors are the leading cause of cancer-related death among children and adolescents ages 0-19 years.
  • #5 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Pediatric brain tumors affect all races, both sexes, and can be diagnosed at any age. Over the last 20 years, there has been a 0.7% increase in cases each year. […] Close to 5,000 children and teens are diagnosed with brain and spinal cord tumors each year. […] Brain tumors are the 2nd most common cancers in children, after leukemia, and account for 15.9% of all new childhood cancer cases. Among all childhood cancers, brain tumors are the leading cause of death. […] Astrocytomas, including glioblastoma multiforme, are the most common type of glioma, making up half of all childhood brain tumors, according to Johns Hopkins Medicine. They have a survival rate of about 25%. […] Three out of four (75.5%) children diagnosed with a brain tumor will survive at least 5 years. […] Congenital brain tumors have a poor prognosis with overall survival of less than 30%.
  • #6 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    Pediatric brain tumors are the leading cause of cancer-related death in childhood. […] Advances in surgery, neuro-oncology, neuroradiology and radiation oncology have improved patient survival for some tumor types such as low-grade gliomas and medulloblastomas. However, the prognosis for patients with diffuse midline gliomas, other high-grade gliomas (HGGs), and most recurrent pediatric brain tumors remains poor since current therapeutic strategies are unable to extend survival by more than a few months in most patients. […] Improving survival rates in this patient cohort has been the primary focus of most pediatric-cancer treatment consortia such as the Children’s Oncology Group, but an equally important aim has been to minimize the immediate and long-term side effects of chemotherapy and ionizing radiation.
  • #7 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    Brain tumors are the leading cause of childhood cancer-related deaths. […] Recent research findings from molecular genetic analyses have revealed molecular and genetic changes in pediatric tumors that are necessary for appropriate classification to avoid misdiagnosis, the development of treatment modalities, and the clinical management of tumors. […] As many of the molecular-based therapies developed from clinical trials on adults are not always effective against pediatric brain tumors, recent advances have improved our understanding of the molecular profiles of pediatric brain tumors and have led to novel epigenetic and immunotherapeutic treatment approaches currently being evaluated in clinical trials. […] The comparison of pediatric and adult brain tumors highlights the need for treatments designed specifically for pediatric brain tumors.
  • #8 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    Approximately 3.9% of all brain tumors cases diagnosed occur in children ages 0-14 years. […] An estimated 3,920 new cases of primary childhood brain tumors are expected to be diagnosed in 2023. […] Brain tumors are the most commonly diagnosed solid cancer in children ages 0-14 years, as well as the leading cause of childhood cancer-related death. […] The five-year relative survival rate for all primary childhood brain tumors is 83.1%. […] For primary malignant childhood tumors, the five-year relative survival rate is 75.6%. […] The five-year relative survival rate for all primary pediatric brain tumors (0-19) is 83.9%. […] For malignant tumors, the five-year relative survival rate is 75.6%. […] Pediatric brain tumors are the leading cause of cancer-related death among children and adolescents ages 0-19 years.
  • #9 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Aim: Analysis of risk factors for survival in long-term follow-up of children treated at a single pediatric center in Poland. […] Overall 5-year survival in the whole cohort was 60.94.7%, while 10-year survival was 58.24.7%. […] Survival was 48.19.6% for patients with medulloblastoma and primitive neuroectodermal tumors; 83.36.2% for low-grade astrocytoma; 56.616.6% for ependymoma, while 0% at 72 months for high-grade glioma. […] Multivariate analysis showed that factors predicting poor outcome were: grade III-IV tumor, incomplete surgical resection, and complications after surgical resection, while diagnosis of low-grade glioma was the only factor predicting good outcome. […] Progression of the disease during therapy was an additional independent adverse risk factor for survival. […] Long-term survival was achieved by 58% of children with brain tumors.
  • #10 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Glioblastomas have a 20% 5-year survival rate. […] If a child survives, they have a 95 percent chance of developing chronic health problems, secondary cancers, cognitive impairments, and a shortened lifespan due to the harshness of current treatment options. […] Despite the 75% survival rates, cancer victims continue to suffer from the disease. Late effects can be long-term and progressive. This is especially for children who were young when they were diagnosed and who were treated with cranial radiation. […] To improve the outcomes of children who are diagnosed with brain tumors, it’s critical to improve our understanding of the disease, find ways to treat brain tumors more effectively, and reduce the toxicity of treatments. […] Researchers have found some changes in genes, chromosomes, and proteins inside brain tumor cells that can be used to help predict a child’s outlook or help guide treatment. […] However, pediatric brain tumor research is underfunded. Only 4% of federal funding for cancer research goes to studies on pediatric cancers. […] Pediatric brain tumors, which are now the leading cause of cancer-related death in children and adolescents ages 19 and younger.
  • #11 Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8142618/
    Age at diagnosis, race, sex, tumor grade, and tumor histologic subtypes and anatomic sites are the major identified prognostic factors. Studies have demonstrated that children diagnosed at an older age typically have better survival compared with those diagnosed at a younger age. Racial variations in pediatric brain cancer survival have also been observed in previous studies. Non-Hispanic African American (NH-AA) children are at greater risk of death compared with their non-Hispanic Caucasian (NH-Caucasian) counterparts. Another frequently assessed factor is the tumor anatomic site, where the location at the brain stem and cerebrum are identified as having the worst survival. […] The risk of mortality increased with increasing levels of grades in both adjusted and unadjusted models. However, the extent of the difference in risk among grades reduced after controlling for other factors. The risk of mortality by histology subtypes also varied substantially. Ependymal tumors (aHR (95% CI): 2.20 (1.95, 2.48), high-grade glioma (7.92 (7.09, 8.85)), medulloblastoma (3.11 (2.79, 3.47)), other gliomas (3.38 (3.08, 3.71)), and PNET (4.72 (4.15, 5.37)) showed worse likelihood of survival compared to the low-grade glioma.
  • #12 Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8142618/
    In conclusion, this study identified age at diagnosis, race-ethnicity, tumor grade, primary site, histologic subtypes, county-poverty level, and geographic regions as significant prognostic factors of pediatric brain tumor survival in the United States. Improvement toward existing survival variation in demographic and biological host factors, disease etiology, SES as well as environmental barriers to accessing treatment may further reduce the risk of mortality.
  • #13 Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8142618/
    Brain tumors, a group of heterogeneous diseases, are the second most common cancer and the leading cause of cancer-related deaths in children. Insight into the prognosis of pediatric brain tumor survival has led to improved outcomes and could be further advanced through precision in prognosis. Mortality risk declined with increased age at diagnosis, the adjusted hazard ratio (aHR) (95% confidence interval) was 0.60 (0.55, 0.67) and 0.47 (0.42, 0.52) for ages at diagnosis 1-10 years and 10-19 years, respectively, when compared with infants. Non-Hispanic Caucasian patients showed a lower risk of mortality than non-Hispanic African Americans (1.21 (1.11, 1.32)) and Hispanics (1.21 (1.11, 1.32)). Primary tumor sites, grades, and histology showed substantial heterogeneity in mortality risk. Brainstem (2.62 (2.41, 2.85)) and Cerebrum (1.63 (1.46, 1.81)) had an elevated risk of mortality than lobes. Similarly, Grade II (1.32 (1.07, 1.62)), Grade III (3.39 (2.74, 4.19)), and Grade IV (2.18 (1.80, 2.64)) showed an inflated risk of mortality than Grade I. Compared to low-grade glioma, high-grade glioma (7.92 (7.09, 8.85)), Primitive neuroectodermal tumors (4.72 (4.15, 5.37)), Medulloblastoma (3.11 (2.79, 3.47)), and Ependymal-tumors (2.20 (1.95, 2.48)) had increased risk of mortality. This large population-based comprehensive study confirmed identified prognostic factors of pediatric brain tumor survival and provided estimates as epidemiologic evidence with greater generalization.
  • #14 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    AT/RT and high-grade gliomas have the lowest relative survival after diagnosis for patients 0-19 years with a brain tumor. […] High-grade gliomas cause the greatest proportion of deaths (44.2%), followed by medulloblastoma and AT/RT. […] Pilocytic astrocytoma has the highest survival rates after diagnosis. […] Relative survival rates generally improve with increasing age at diagnosis in this population group, with poorest survival in those less than a year old at diagnosis.
  • #15 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Pediatric brain tumors affect all races, both sexes, and can be diagnosed at any age. Over the last 20 years, there has been a 0.7% increase in cases each year. […] Close to 5,000 children and teens are diagnosed with brain and spinal cord tumors each year. […] Brain tumors are the 2nd most common cancers in children, after leukemia, and account for 15.9% of all new childhood cancer cases. Among all childhood cancers, brain tumors are the leading cause of death. […] Astrocytomas, including glioblastoma multiforme, are the most common type of glioma, making up half of all childhood brain tumors, according to Johns Hopkins Medicine. They have a survival rate of about 25%. […] Three out of four (75.5%) children diagnosed with a brain tumor will survive at least 5 years. […] Congenital brain tumors have a poor prognosis with overall survival of less than 30%.
  • #16 Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8142618/
    Brain tumors, a group of heterogeneous diseases, are the second most common cancer and the leading cause of cancer-related deaths in children. Insight into the prognosis of pediatric brain tumor survival has led to improved outcomes and could be further advanced through precision in prognosis. Mortality risk declined with increased age at diagnosis, the adjusted hazard ratio (aHR) (95% confidence interval) was 0.60 (0.55, 0.67) and 0.47 (0.42, 0.52) for ages at diagnosis 1-10 years and 10-19 years, respectively, when compared with infants. Non-Hispanic Caucasian patients showed a lower risk of mortality than non-Hispanic African Americans (1.21 (1.11, 1.32)) and Hispanics (1.21 (1.11, 1.32)). Primary tumor sites, grades, and histology showed substantial heterogeneity in mortality risk. Brainstem (2.62 (2.41, 2.85)) and Cerebrum (1.63 (1.46, 1.81)) had an elevated risk of mortality than lobes. Similarly, Grade II (1.32 (1.07, 1.62)), Grade III (3.39 (2.74, 4.19)), and Grade IV (2.18 (1.80, 2.64)) showed an inflated risk of mortality than Grade I. Compared to low-grade glioma, high-grade glioma (7.92 (7.09, 8.85)), Primitive neuroectodermal tumors (4.72 (4.15, 5.37)), Medulloblastoma (3.11 (2.79, 3.47)), and Ependymal-tumors (2.20 (1.95, 2.48)) had increased risk of mortality. This large population-based comprehensive study confirmed identified prognostic factors of pediatric brain tumor survival and provided estimates as epidemiologic evidence with greater generalization.
  • #17 Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8142618/
    Brain tumors, a group of heterogeneous diseases, are the second most common cancer and the leading cause of cancer-related deaths in children. Insight into the prognosis of pediatric brain tumor survival has led to improved outcomes and could be further advanced through precision in prognosis. Mortality risk declined with increased age at diagnosis, the adjusted hazard ratio (aHR) (95% confidence interval) was 0.60 (0.55, 0.67) and 0.47 (0.42, 0.52) for ages at diagnosis 1-10 years and 10-19 years, respectively, when compared with infants. Non-Hispanic Caucasian patients showed a lower risk of mortality than non-Hispanic African Americans (1.21 (1.11, 1.32)) and Hispanics (1.21 (1.11, 1.32)). Primary tumor sites, grades, and histology showed substantial heterogeneity in mortality risk. Brainstem (2.62 (2.41, 2.85)) and Cerebrum (1.63 (1.46, 1.81)) had an elevated risk of mortality than lobes. Similarly, Grade II (1.32 (1.07, 1.62)), Grade III (3.39 (2.74, 4.19)), and Grade IV (2.18 (1.80, 2.64)) showed an inflated risk of mortality than Grade I. Compared to low-grade glioma, high-grade glioma (7.92 (7.09, 8.85)), Primitive neuroectodermal tumors (4.72 (4.15, 5.37)), Medulloblastoma (3.11 (2.79, 3.47)), and Ependymal-tumors (2.20 (1.95, 2.48)) had increased risk of mortality. This large population-based comprehensive study confirmed identified prognostic factors of pediatric brain tumor survival and provided estimates as epidemiologic evidence with greater generalization.
  • #18 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Advanced tumor stage, incomplete surgical resection, complications of surgical treatment, and progression of the disease during treatment predicted poor outcome. […] By univariate analysis, factors predicting poor outcome were: brain stem localization, HGG diagnosis, grade III-IV tumors according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome. […] By multivariate analysis, factors predicting poor outcome were: grade III-IV tumor according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome.
  • #19 Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8142618/
    Brain tumors, a group of heterogeneous diseases, are the second most common cancer and the leading cause of cancer-related deaths in children. Insight into the prognosis of pediatric brain tumor survival has led to improved outcomes and could be further advanced through precision in prognosis. Mortality risk declined with increased age at diagnosis, the adjusted hazard ratio (aHR) (95% confidence interval) was 0.60 (0.55, 0.67) and 0.47 (0.42, 0.52) for ages at diagnosis 1-10 years and 10-19 years, respectively, when compared with infants. Non-Hispanic Caucasian patients showed a lower risk of mortality than non-Hispanic African Americans (1.21 (1.11, 1.32)) and Hispanics (1.21 (1.11, 1.32)). Primary tumor sites, grades, and histology showed substantial heterogeneity in mortality risk. Brainstem (2.62 (2.41, 2.85)) and Cerebrum (1.63 (1.46, 1.81)) had an elevated risk of mortality than lobes. Similarly, Grade II (1.32 (1.07, 1.62)), Grade III (3.39 (2.74, 4.19)), and Grade IV (2.18 (1.80, 2.64)) showed an inflated risk of mortality than Grade I. Compared to low-grade glioma, high-grade glioma (7.92 (7.09, 8.85)), Primitive neuroectodermal tumors (4.72 (4.15, 5.37)), Medulloblastoma (3.11 (2.79, 3.47)), and Ependymal-tumors (2.20 (1.95, 2.48)) had increased risk of mortality. This large population-based comprehensive study confirmed identified prognostic factors of pediatric brain tumor survival and provided estimates as epidemiologic evidence with greater generalization.
  • #20 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Advanced tumor stage, incomplete surgical resection, complications of surgical treatment, and progression of the disease during treatment predicted poor outcome. […] By univariate analysis, factors predicting poor outcome were: brain stem localization, HGG diagnosis, grade III-IV tumors according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome. […] By multivariate analysis, factors predicting poor outcome were: grade III-IV tumor according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome.
  • #21 Epidemiology and prognostic factors of pediatric brain tumor survival in the US: Evidence from four decades of population data
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8142618/
    Age at diagnosis, race, sex, tumor grade, and tumor histologic subtypes and anatomic sites are the major identified prognostic factors. Studies have demonstrated that children diagnosed at an older age typically have better survival compared with those diagnosed at a younger age. Racial variations in pediatric brain cancer survival have also been observed in previous studies. Non-Hispanic African American (NH-AA) children are at greater risk of death compared with their non-Hispanic Caucasian (NH-Caucasian) counterparts. Another frequently assessed factor is the tumor anatomic site, where the location at the brain stem and cerebrum are identified as having the worst survival. […] The risk of mortality increased with increasing levels of grades in both adjusted and unadjusted models. However, the extent of the difference in risk among grades reduced after controlling for other factors. The risk of mortality by histology subtypes also varied substantially. Ependymal tumors (aHR (95% CI): 2.20 (1.95, 2.48), high-grade glioma (7.92 (7.09, 8.85)), medulloblastoma (3.11 (2.79, 3.47)), other gliomas (3.38 (3.08, 3.71)), and PNET (4.72 (4.15, 5.37)) showed worse likelihood of survival compared to the low-grade glioma.
  • #22 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Advanced tumor stage, incomplete surgical resection, complications of surgical treatment, and progression of the disease during treatment predicted poor outcome. […] By univariate analysis, factors predicting poor outcome were: brain stem localization, HGG diagnosis, grade III-IV tumors according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome. […] By multivariate analysis, factors predicting poor outcome were: grade III-IV tumor according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome.
  • #23 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Advanced tumor stage, incomplete surgical resection, complications of surgical treatment, and progression of the disease during treatment predicted poor outcome. […] By univariate analysis, factors predicting poor outcome were: brain stem localization, HGG diagnosis, grade III-IV tumors according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome. […] By multivariate analysis, factors predicting poor outcome were: grade III-IV tumor according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome.
  • #24 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Another finding of our study is that progression of the disease during therapy (chemotherapy or radiotherapy) had a highly negative impact on survival. […] In conclusion, we have shown that 10-year survival for children with brain tumors was 58%; it was the best for LGG, and the worst for HGG patients. […] We found that advanced tumor stage, incomplete surgical resection, complications of surgical treatment and progression of the disease during treatment predicted poor outcome.
  • #25 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    AT/RT and high-grade gliomas have the lowest relative survival after diagnosis for patients 0-19 years with a brain tumor. […] High-grade gliomas cause the greatest proportion of deaths (44.2%), followed by medulloblastoma and AT/RT. […] Pilocytic astrocytoma has the highest survival rates after diagnosis. […] Relative survival rates generally improve with increasing age at diagnosis in this population group, with poorest survival in those less than a year old at diagnosis.
  • #26 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Aim: Analysis of risk factors for survival in long-term follow-up of children treated at a single pediatric center in Poland. […] Overall 5-year survival in the whole cohort was 60.94.7%, while 10-year survival was 58.24.7%. […] Survival was 48.19.6% for patients with medulloblastoma and primitive neuroectodermal tumors; 83.36.2% for low-grade astrocytoma; 56.616.6% for ependymoma, while 0% at 72 months for high-grade glioma. […] Multivariate analysis showed that factors predicting poor outcome were: grade III-IV tumor, incomplete surgical resection, and complications after surgical resection, while diagnosis of low-grade glioma was the only factor predicting good outcome. […] Progression of the disease during therapy was an additional independent adverse risk factor for survival. […] Long-term survival was achieved by 58% of children with brain tumors.
  • #27 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Advanced tumor stage, incomplete surgical resection, complications of surgical treatment, and progression of the disease during treatment predicted poor outcome. […] By univariate analysis, factors predicting poor outcome were: brain stem localization, HGG diagnosis, grade III-IV tumors according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome. […] By multivariate analysis, factors predicting poor outcome were: grade III-IV tumor according to the WHO classification, incomplete surgical resection, complications after surgical resection, and progression of the disease during therapy, while diagnosis of LGG was the only factor predicting for good outcome.
  • #28 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    This insight into molecular mechanisms has resulted in increased accuracy and reliability of tumor diagnosis, has revealed potential therapeutic targets, and provides a rational basis to either augment or de-intensify therapy. […] High-grade gliomas (WHO Grade IV) have the highest mortality rate of all pediatric brain tumors, with five-year survival rates less than 20% despite maximal therapeutic interventions. […] The insight we have gained into the genomic drivers of pediatric high-grade gliomas has resulted in several clinical trials testing targeted agents against this group of tumors. […] The PI3K/AKT/mTOR pathway promotes cell survival and growth and is activated in several cancers. […] Ependymomas constitute about 5% of childhood brain tumors and occur more frequently in the posterior fossa.
  • #29 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Aim: Analysis of risk factors for survival in long-term follow-up of children treated at a single pediatric center in Poland. […] Overall 5-year survival in the whole cohort was 60.94.7%, while 10-year survival was 58.24.7%. […] Survival was 48.19.6% for patients with medulloblastoma and primitive neuroectodermal tumors; 83.36.2% for low-grade astrocytoma; 56.616.6% for ependymoma, while 0% at 72 months for high-grade glioma. […] Multivariate analysis showed that factors predicting poor outcome were: grade III-IV tumor, incomplete surgical resection, and complications after surgical resection, while diagnosis of low-grade glioma was the only factor predicting good outcome. […] Progression of the disease during therapy was an additional independent adverse risk factor for survival. […] Long-term survival was achieved by 58% of children with brain tumors.
  • #30 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Glioblastomas have a 20% 5-year survival rate. […] If a child survives, they have a 95 percent chance of developing chronic health problems, secondary cancers, cognitive impairments, and a shortened lifespan due to the harshness of current treatment options. […] Despite the 75% survival rates, cancer victims continue to suffer from the disease. Late effects can be long-term and progressive. This is especially for children who were young when they were diagnosed and who were treated with cranial radiation. […] To improve the outcomes of children who are diagnosed with brain tumors, it’s critical to improve our understanding of the disease, find ways to treat brain tumors more effectively, and reduce the toxicity of treatments. […] Researchers have found some changes in genes, chromosomes, and proteins inside brain tumor cells that can be used to help predict a child’s outlook or help guide treatment. […] However, pediatric brain tumor research is underfunded. Only 4% of federal funding for cancer research goes to studies on pediatric cancers. […] Pediatric brain tumors, which are now the leading cause of cancer-related death in children and adolescents ages 19 and younger.
  • #31 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    AT/RT and high-grade gliomas have the lowest relative survival after diagnosis for patients 0-19 years with a brain tumor. […] High-grade gliomas cause the greatest proportion of deaths (44.2%), followed by medulloblastoma and AT/RT. […] Pilocytic astrocytoma has the highest survival rates after diagnosis. […] Relative survival rates generally improve with increasing age at diagnosis in this population group, with poorest survival in those less than a year old at diagnosis.
  • #32 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    Most pediatric gliomas are grade 1 or 2 low-grade gliomas, but some progress rapidly to grade 3 or 4 as pHGG. […] While astrocytomas are more prevalent in adults as HGGs, in children, most of these tumors are low-grade. […] The majority of pPFE patients succumb to the disease, some survive with excellent functional outcomes. […] DIPG also has genomic alterations in tumor protein p53, platelet-derived growth factor receptor alpha, and activin A receptor type I, among other genes. […] These tumors have an extremely poor prognosis, as their intrinsic nature makes it impossible to perform resection. […] The diverse array of mutations contributes to significant intertumoral heterogeneity in DIPG, complicating treatment strategies. […] Profiling each tumor for epigenetic changes (in addition to gene modifications) will improve stratification in clinical trials and predictions of responses to therapy.
  • #33 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    Most pediatric gliomas are grade 1 or 2 low-grade gliomas, but some progress rapidly to grade 3 or 4 as pHGG. […] While astrocytomas are more prevalent in adults as HGGs, in children, most of these tumors are low-grade. […] The majority of pPFE patients succumb to the disease, some survive with excellent functional outcomes. […] DIPG also has genomic alterations in tumor protein p53, platelet-derived growth factor receptor alpha, and activin A receptor type I, among other genes. […] These tumors have an extremely poor prognosis, as their intrinsic nature makes it impossible to perform resection. […] The diverse array of mutations contributes to significant intertumoral heterogeneity in DIPG, complicating treatment strategies. […] Profiling each tumor for epigenetic changes (in addition to gene modifications) will improve stratification in clinical trials and predictions of responses to therapy.
  • #34 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Aim: Analysis of risk factors for survival in long-term follow-up of children treated at a single pediatric center in Poland. […] Overall 5-year survival in the whole cohort was 60.94.7%, while 10-year survival was 58.24.7%. […] Survival was 48.19.6% for patients with medulloblastoma and primitive neuroectodermal tumors; 83.36.2% for low-grade astrocytoma; 56.616.6% for ependymoma, while 0% at 72 months for high-grade glioma. […] Multivariate analysis showed that factors predicting poor outcome were: grade III-IV tumor, incomplete surgical resection, and complications after surgical resection, while diagnosis of low-grade glioma was the only factor predicting good outcome. […] Progression of the disease during therapy was an additional independent adverse risk factor for survival. […] Long-term survival was achieved by 58% of children with brain tumors.
  • #35 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    AT/RT and high-grade gliomas have the lowest relative survival after diagnosis for patients 0-19 years with a brain tumor. […] High-grade gliomas cause the greatest proportion of deaths (44.2%), followed by medulloblastoma and AT/RT. […] Pilocytic astrocytoma has the highest survival rates after diagnosis. […] Relative survival rates generally improve with increasing age at diagnosis in this population group, with poorest survival in those less than a year old at diagnosis.
  • #36 Family
    https://patents.google.com/patent/EP1356114A2/en
    The predicted treatment outcome can be, for example, survival after treatment. […] The present invention also provides methods for monitoring the effect of a treatment regimen in an individual by monitoring the gene expression profile for one or more informative genes. […] A clinical challenge concerning medulloblastoma is the highly variable response of patients to therapy. […] High levels of expression of the neurofroplin-3 receptor (TrkC), however, have been reported to correlate with a favorable medulloblastoma outcome, suggesting a molecular basis of medulloblastoma outcome variability. […] The present invention provides methods for determining a treatment plan for an individual. […] The present invention can be applied to screen potential drug candidates for their efficacy in treating brain tumors.
  • #37 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    This insight into molecular mechanisms has resulted in increased accuracy and reliability of tumor diagnosis, has revealed potential therapeutic targets, and provides a rational basis to either augment or de-intensify therapy. […] High-grade gliomas (WHO Grade IV) have the highest mortality rate of all pediatric brain tumors, with five-year survival rates less than 20% despite maximal therapeutic interventions. […] The insight we have gained into the genomic drivers of pediatric high-grade gliomas has resulted in several clinical trials testing targeted agents against this group of tumors. […] The PI3K/AKT/mTOR pathway promotes cell survival and growth and is activated in several cancers. […] Ependymomas constitute about 5% of childhood brain tumors and occur more frequently in the posterior fossa.
  • #38 Survival and Prognostic Factors in Children with Brain Tumors: Long-term Follow-up Single Center Study in Poland | Anticancer Research
    https://ar.iiarjournals.org/content/34/1/323
    Aim: Analysis of risk factors for survival in long-term follow-up of children treated at a single pediatric center in Poland. […] Overall 5-year survival in the whole cohort was 60.94.7%, while 10-year survival was 58.24.7%. […] Survival was 48.19.6% for patients with medulloblastoma and primitive neuroectodermal tumors; 83.36.2% for low-grade astrocytoma; 56.616.6% for ependymoma, while 0% at 72 months for high-grade glioma. […] Multivariate analysis showed that factors predicting poor outcome were: grade III-IV tumor, incomplete surgical resection, and complications after surgical resection, while diagnosis of low-grade glioma was the only factor predicting good outcome. […] Progression of the disease during therapy was an additional independent adverse risk factor for survival. […] Long-term survival was achieved by 58% of children with brain tumors.
  • #39 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    The efficacy of post-radiation chemotherapy in treating ependymoma in various settings is still being investigated, and chemotherapy is used for high-risk, subtotally resected, or recurrent disease. […] Driven by success in some adult cancers, there has been an increase in clinical trials utilizing various forms of immunotherapy for both adult and pediatric CNS tumors. […] Immune checkpoint inhibitors have been studied against adult brain tumors, and several clinical trials are underway to utilize anti-PD1 and CTLA-4 inhibitors in pediatric high-grade glioma. […] Pediatric brain tumors are the most common solid malignancies in children.
  • #40 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    AT/RT and high-grade gliomas have the lowest relative survival after diagnosis for patients 0-19 years with a brain tumor. […] High-grade gliomas cause the greatest proportion of deaths (44.2%), followed by medulloblastoma and AT/RT. […] Pilocytic astrocytoma has the highest survival rates after diagnosis. […] Relative survival rates generally improve with increasing age at diagnosis in this population group, with poorest survival in those less than a year old at diagnosis.
  • #41 Brain Tumor Facts
    https://braintumor.org/brain-tumors/about-brain-tumors/brain-tumor-facts/
    AT/RT and high-grade gliomas have the lowest relative survival after diagnosis for patients 0-19 years with a brain tumor. […] High-grade gliomas cause the greatest proportion of deaths (44.2%), followed by medulloblastoma and AT/RT. […] Pilocytic astrocytoma has the highest survival rates after diagnosis. […] Relative survival rates generally improve with increasing age at diagnosis in this population group, with poorest survival in those less than a year old at diagnosis.
  • #42 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    Tumor cells also use epigenetic mechanisms to escape chemotherapy and host immune surveillance, so improving our understanding of these mechanisms will improve treatment outcomes. […] Epigenetic changes are essential to malignant transformation, as tumor tissues exhibit abnormal patterns of methylation compared to healthy tissues. […] Epigenetic profiles may be useful for: (a) potential drug targets; (b) cancer prognostics; (c) tumor classification; and (d) the basic science of tumor development. […] The majority of patients do not benefit from immunotherapy, as tumors often fail to respond or develop resistance, presenting a significant hurdle to improving the efficacy of immunotherapy. […] The low mutational load (and corresponding neoantigen level) in pediatric brain tumors is a central issue for immunotherapy since appropriate tumor antigens are needed as targets to induce an immune response against tumors.
  • #43 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    Tumor cells also use epigenetic mechanisms to escape chemotherapy and host immune surveillance, so improving our understanding of these mechanisms will improve treatment outcomes. […] Epigenetic changes are essential to malignant transformation, as tumor tissues exhibit abnormal patterns of methylation compared to healthy tissues. […] Epigenetic profiles may be useful for: (a) potential drug targets; (b) cancer prognostics; (c) tumor classification; and (d) the basic science of tumor development. […] The majority of patients do not benefit from immunotherapy, as tumors often fail to respond or develop resistance, presenting a significant hurdle to improving the efficacy of immunotherapy. […] The low mutational load (and corresponding neoantigen level) in pediatric brain tumors is a central issue for immunotherapy since appropriate tumor antigens are needed as targets to induce an immune response against tumors.
  • #44 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    Tumor cells also use epigenetic mechanisms to escape chemotherapy and host immune surveillance, so improving our understanding of these mechanisms will improve treatment outcomes. […] Epigenetic changes are essential to malignant transformation, as tumor tissues exhibit abnormal patterns of methylation compared to healthy tissues. […] Epigenetic profiles may be useful for: (a) potential drug targets; (b) cancer prognostics; (c) tumor classification; and (d) the basic science of tumor development. […] The majority of patients do not benefit from immunotherapy, as tumors often fail to respond or develop resistance, presenting a significant hurdle to improving the efficacy of immunotherapy. […] The low mutational load (and corresponding neoantigen level) in pediatric brain tumors is a central issue for immunotherapy since appropriate tumor antigens are needed as targets to induce an immune response against tumors.
  • #45 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    We also discuss the advancements in novel molecularly targeted drugs and how they are being integrated with standard therapy to improve the classification and outcomes of pediatric brain tumors in the future. […] Current treatment options for pediatric brain tumors have limited efficacy due to the different responses to available therapies. […] Molecular profiling has revealed significant differences between adult and pediatric brain tumors, despite having similar histology. […] The classification of pHGGs is based on molecular subgroups and significant clinical correlations (i.e., age, anatomical location, and prognosis). […] Overall, histone mutations represent slightly more than half of all childhood pHGG cases. […] The largest pHGG dataset to date was used in a retrospective study that revealed ten subgroups based on specific genes and processes and showed that histone mutations cosegregate with distinct modifications and downstream pathways.
  • #46 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    We also discuss the advancements in novel molecularly targeted drugs and how they are being integrated with standard therapy to improve the classification and outcomes of pediatric brain tumors in the future. […] Current treatment options for pediatric brain tumors have limited efficacy due to the different responses to available therapies. […] Molecular profiling has revealed significant differences between adult and pediatric brain tumors, despite having similar histology. […] The classification of pHGGs is based on molecular subgroups and significant clinical correlations (i.e., age, anatomical location, and prognosis). […] Overall, histone mutations represent slightly more than half of all childhood pHGG cases. […] The largest pHGG dataset to date was used in a retrospective study that revealed ten subgroups based on specific genes and processes and showed that histone mutations cosegregate with distinct modifications and downstream pathways.
  • #47 Family
    https://patents.google.com/patent/EP1356114A2/en
    Methods for predicting phenotypic classes of brain tumors, such as brain tumor type or treatment outcome, for brain tumor samples based on gene expression profiles are described. […] The present invention relates to one or more sets of informative genes whose expression correlates with a class distinction among brain tumor samples. […] In another embodiment the class distinction is a treatment outcome or survival class distinction. […] Further, the clinical outcome of patients (e.g., children) with medulloblastomas is highly predictable based on the gene expression profiles of their tumors at diagnosis. […] The present invention is directed to a method of predicting the efficacy of treating a brain tumor comprising the steps of: obtaining a sample of cells derived from a brain tumor; isolating a gene expression product from at least one informative gene from one or more cells in said sample; and determining a gene expression profile of at least one informative gene, wherein the gene expression profile is correlated with a treatment outcome, thereby classifying the sample with respect to treatment outcome.
  • #48 Family
    https://patents.google.com/patent/EP1356114A2/en
    Methods for predicting phenotypic classes of brain tumors, such as brain tumor type or treatment outcome, for brain tumor samples based on gene expression profiles are described. […] The present invention relates to one or more sets of informative genes whose expression correlates with a class distinction among brain tumor samples. […] In another embodiment the class distinction is a treatment outcome or survival class distinction. […] Further, the clinical outcome of patients (e.g., children) with medulloblastomas is highly predictable based on the gene expression profiles of their tumors at diagnosis. […] The present invention is directed to a method of predicting the efficacy of treating a brain tumor comprising the steps of: obtaining a sample of cells derived from a brain tumor; isolating a gene expression product from at least one informative gene from one or more cells in said sample; and determining a gene expression profile of at least one informative gene, wherein the gene expression profile is correlated with a treatment outcome, thereby classifying the sample with respect to treatment outcome.
  • #49 Family
    https://patents.google.com/patent/EP1356114A2/en
    Methods for predicting phenotypic classes of brain tumors, such as brain tumor type or treatment outcome, for brain tumor samples based on gene expression profiles are described. […] The present invention relates to one or more sets of informative genes whose expression correlates with a class distinction among brain tumor samples. […] In another embodiment the class distinction is a treatment outcome or survival class distinction. […] Further, the clinical outcome of patients (e.g., children) with medulloblastomas is highly predictable based on the gene expression profiles of their tumors at diagnosis. […] The present invention is directed to a method of predicting the efficacy of treating a brain tumor comprising the steps of: obtaining a sample of cells derived from a brain tumor; isolating a gene expression product from at least one informative gene from one or more cells in said sample; and determining a gene expression profile of at least one informative gene, wherein the gene expression profile is correlated with a treatment outcome, thereby classifying the sample with respect to treatment outcome.
  • #50 Neuropsychological Outcomes of Children Treated for Brain Tumors
    https://pmc.ncbi.nlm.nih.gov/articles/PMC10046931/
    Central nervous system (CNS) neoplasms are the most common solid tumors diagnosed in children. CNS tumors represent the leading cause of cancer death and cancer-related morbidity for children less than 20 years of age, although there has been a moderate increase in survival rates over the past several decades. The average survival at 5 years now nearly reaches 75%, and for some, non-malignant histology approximates 97% at 20 years from diagnosis. Neurological, cognitive, and neuropsychological deficits are the most disabling long-term effects of brain tumors in children. […] Cognitive and neuropsychological problems are more frequent in this population compared to survivors of all other pediatric malignancies. It can happen not only because of the experience of a potentially lethal condition and its therapeutic pathway but also due to the involvement of specific cerebral areas, which hits the CNS in a phase of maximum development.
  • #51 Neuropsychological Outcomes of Children Treated for Brain Tumors
    https://pmc.ncbi.nlm.nih.gov/articles/PMC10046931/
    Central nervous system (CNS) neoplasms are the most common solid tumors diagnosed in children. CNS tumors represent the leading cause of cancer death and cancer-related morbidity for children less than 20 years of age, although there has been a moderate increase in survival rates over the past several decades. The average survival at 5 years now nearly reaches 75%, and for some, non-malignant histology approximates 97% at 20 years from diagnosis. Neurological, cognitive, and neuropsychological deficits are the most disabling long-term effects of brain tumors in children. […] Cognitive and neuropsychological problems are more frequent in this population compared to survivors of all other pediatric malignancies. It can happen not only because of the experience of a potentially lethal condition and its therapeutic pathway but also due to the involvement of specific cerebral areas, which hits the CNS in a phase of maximum development.
  • #52 Neuropsychological Outcomes of Children Treated for Brain Tumors
    https://pmc.ncbi.nlm.nih.gov/articles/PMC10046931/
    Many patients experience both significant global deficits with decreased intelligence quotients (IQ) and specific neuropsychological deficits such as impaired executive functioning, memory, and processing speed. It is estimated that a large proportion of patients treated for a CNS tumor, ranging from 40% to 100%, will experience a deficit in a neurocognitive domain. […] Cancer-related cognitive dysfunction affects about one-third of childhood cancer survivors in the US. Neurocognitive late effects in childhood brain tumor survivors are relatively common and play a significant role in modifying Health-related Quality of Life (HR-QOL). […] Declines in IQ are evident in pediatric CNS tumor survivors as early as the first year following diagnosis and treatment, with potential progression over the next 5 to 7 years.
  • #53 Neuropsychological Outcomes of Children Treated for Brain Tumors
    https://pmc.ncbi.nlm.nih.gov/articles/PMC10046931/
    Many patients experience both significant global deficits with decreased intelligence quotients (IQ) and specific neuropsychological deficits such as impaired executive functioning, memory, and processing speed. It is estimated that a large proportion of patients treated for a CNS tumor, ranging from 40% to 100%, will experience a deficit in a neurocognitive domain. […] Cancer-related cognitive dysfunction affects about one-third of childhood cancer survivors in the US. Neurocognitive late effects in childhood brain tumor survivors are relatively common and play a significant role in modifying Health-related Quality of Life (HR-QOL). […] Declines in IQ are evident in pediatric CNS tumor survivors as early as the first year following diagnosis and treatment, with potential progression over the next 5 to 7 years.
  • #54 Neuropsychological Outcomes of Children Treated for Brain Tumors
    https://pmc.ncbi.nlm.nih.gov/articles/PMC10046931/
    The most frequently impaired functions include attention, working memory, and processing speed. […] EFs involve inhibition, mental flexibility, planning and decision-making, abstract reasoning, concept formation, problem-solving, and awareness. […] Although survival rates in children diagnosed with brain tumors have significantly improved in the last decades, especially for some tumor types, it is now clear that the disease itself, together with aggressive treatments, put this population at substantial risk for functional deficits in multiple domains, reducing their quality of survival.
  • #55 Neuropsychological Outcomes of Children Treated for Brain Tumors
    https://pmc.ncbi.nlm.nih.gov/articles/PMC10046931/
    Many patients experience both significant global deficits with decreased intelligence quotients (IQ) and specific neuropsychological deficits such as impaired executive functioning, memory, and processing speed. It is estimated that a large proportion of patients treated for a CNS tumor, ranging from 40% to 100%, will experience a deficit in a neurocognitive domain. […] Cancer-related cognitive dysfunction affects about one-third of childhood cancer survivors in the US. Neurocognitive late effects in childhood brain tumor survivors are relatively common and play a significant role in modifying Health-related Quality of Life (HR-QOL). […] Declines in IQ are evident in pediatric CNS tumor survivors as early as the first year following diagnosis and treatment, with potential progression over the next 5 to 7 years.
  • #56 Multimodal deep learning to predict prognosis in adult and pediatric brain tumors | Communications Medicine
    https://www.nature.com/articles/s43856-023-00276-y
    The introduction of deep learning in both imaging and genomics has significantly advanced the analysis of biomedical data. […] Here, we propose a DL framework that combines these two modalities with the aim to predict brain tumor prognosis. […] Leveraging the concept of transfer learning, we demonstrate how our pediatric multimodal models can be used to predict prognosis for two more rare (less available samples) pediatric brain tumors. […] Our study illustrates that a multimodal data fusion approach can be successfully implemented and customized to model clinical outcome of adult and pediatric brain tumors. […] For adult, but especially for pediatric brain tumor patients, approaches are urgently needed to predict clinical outcome and assist physicians to make treatment decisions. […] Using a cross-validation approach, our results show that the suggested strategies for multimodal data fusion enable the integrated models to achieve improved prediction accuracy than using histopathology and genomic data in isolation.
  • #57 Multimodal deep learning to predict prognosis in adult and pediatric brain tumors | Communications Medicine
    https://www.nature.com/articles/s43856-023-00276-y
    The introduction of deep learning in both imaging and genomics has significantly advanced the analysis of biomedical data. […] Here, we propose a DL framework that combines these two modalities with the aim to predict brain tumor prognosis. […] Leveraging the concept of transfer learning, we demonstrate how our pediatric multimodal models can be used to predict prognosis for two more rare (less available samples) pediatric brain tumors. […] Our study illustrates that a multimodal data fusion approach can be successfully implemented and customized to model clinical outcome of adult and pediatric brain tumors. […] For adult, but especially for pediatric brain tumor patients, approaches are urgently needed to predict clinical outcome and assist physicians to make treatment decisions. […] Using a cross-validation approach, our results show that the suggested strategies for multimodal data fusion enable the integrated models to achieve improved prediction accuracy than using histopathology and genomic data in isolation.
  • #58 Multimodal deep learning to predict prognosis in adult and pediatric brain tumors | Communications Medicine
    https://www.nature.com/articles/s43856-023-00276-y
    Thus, also in transfer learning an approach using multiple modalities potentially adds value in predicting survival probabilities. […] In summary, we observe improved predictive performance in overall survival prediction from data fusion methods on both adult and pediatric multi-scale data cohorts, with the early fusion strategy the best-performing model in our experiments. […] Our results indeed show that pediatric glioma models can be directly used for survival prediction on the other subtypes with reasonable performance. […] Thus, also when using transfer learning the two modalities add value in predicting survival probabilities. […] This suggests that a multimodal deep learning approach is a promising way of capturing the disease trajectories of brain tumor patients.
  • #59 Multimodal deep learning to predict prognosis in adult and pediatric brain tumors | Communications Medicine
    https://www.nature.com/articles/s43856-023-00276-y
    Thus, also in transfer learning an approach using multiple modalities potentially adds value in predicting survival probabilities. […] In summary, we observe improved predictive performance in overall survival prediction from data fusion methods on both adult and pediatric multi-scale data cohorts, with the early fusion strategy the best-performing model in our experiments. […] Our results indeed show that pediatric glioma models can be directly used for survival prediction on the other subtypes with reasonable performance. […] Thus, also when using transfer learning the two modalities add value in predicting survival probabilities. […] This suggests that a multimodal deep learning approach is a promising way of capturing the disease trajectories of brain tumor patients.
  • #60 Multimodal deep learning to predict prognosis in adult and pediatric brain tumors | Communications Medicine
    https://www.nature.com/articles/s43856-023-00276-y
    Thus, also in transfer learning an approach using multiple modalities potentially adds value in predicting survival probabilities. […] In summary, we observe improved predictive performance in overall survival prediction from data fusion methods on both adult and pediatric multi-scale data cohorts, with the early fusion strategy the best-performing model in our experiments. […] Our results indeed show that pediatric glioma models can be directly used for survival prediction on the other subtypes with reasonable performance. […] Thus, also when using transfer learning the two modalities add value in predicting survival probabilities. […] This suggests that a multimodal deep learning approach is a promising way of capturing the disease trajectories of brain tumor patients.
  • #61 Limited Research Investigating the Value of MRI in Predicting Future Cognitive Morbidity in Survivors of Paediatric Brain Tumours: A Call to Action for Clinical Neuroimaging Researchers | medRxiv
    https://www.medrxiv.org/content/10.1101/2024.01.12.24301212v2.full-text
    This systematic review posits that magnetic resonance imaging (MRI) measures are likely to be a good proxy of the burden of brain tumours and their treatment thus, are likely to be predictive of cognitive impairment at the individual patient-level. […] A key consideration, however, is the timing of the MRI used to predict these outcomes. […] This systematic review specifically investigates existing literature using MRI scans, taken at any point in the disease course, to predict non-contemporaneous, later neuropsychological outcomes in survivors of pediatric brain tumours. […] Overall, there exists plenty clinical reasoning to prompt scientific enthusiasm and attention for this topic. […] In conclusion, Given the increased number of adult survivors of childhood brain tumours, the poorer long-term cognitive, educational and employment outcomes and the significant burden this represents to patients, families and healthcare, work now needs to be completed to integrate predictive data into these models, which will expand their explanatory value and utility to clinical practice.
  • #62 Limited Research Investigating the Value of MRI in Predicting Future Cognitive Morbidity in Survivors of Paediatric Brain Tumours: A Call to Action for Clinical Neuroimaging Researchers | medRxiv
    https://www.medrxiv.org/content/10.1101/2024.01.12.24301212v2.full-text
    Survivors of pediatric brain tumour patients are at high risk of cognitive morbidity. […] The current systematic review aims to answer the question Do MRI markers predict future cognitive functioning in pediatric brain tumour survivors?. […] Ultimately, the key finding of this review is that the current literature is limited. […] Therefore, current findings are at high risk of bias and thus the quality and conclusions are limited. […] While disease and treatment will inevitably place all pediatric brain tumour patients at some level of risk for poor cognitive outcomes, knowing individualised risk, an estimate of the severity of difficulties and specific domains likely to be impacted, will influence clinical practice. […] Prediction of individual-level neurocognitive outcomes would enable timely and tailored input from school and allied health services, promoting outcomes for these children, with limited healthcare resources being efficiently prioritised for those most at risk.
  • #63 Limited Research Investigating the Value of MRI in Predicting Future Cognitive Morbidity in Survivors of Paediatric Brain Tumours: A Call to Action for Clinical Neuroimaging Researchers | medRxiv
    https://www.medrxiv.org/content/10.1101/2024.01.12.24301212v2.full-text
    This systematic review posits that magnetic resonance imaging (MRI) measures are likely to be a good proxy of the burden of brain tumours and their treatment thus, are likely to be predictive of cognitive impairment at the individual patient-level. […] A key consideration, however, is the timing of the MRI used to predict these outcomes. […] This systematic review specifically investigates existing literature using MRI scans, taken at any point in the disease course, to predict non-contemporaneous, later neuropsychological outcomes in survivors of pediatric brain tumours. […] Overall, there exists plenty clinical reasoning to prompt scientific enthusiasm and attention for this topic. […] In conclusion, Given the increased number of adult survivors of childhood brain tumours, the poorer long-term cognitive, educational and employment outcomes and the significant burden this represents to patients, families and healthcare, work now needs to be completed to integrate predictive data into these models, which will expand their explanatory value and utility to clinical practice.
  • #64 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Glioblastomas have a 20% 5-year survival rate. […] If a child survives, they have a 95 percent chance of developing chronic health problems, secondary cancers, cognitive impairments, and a shortened lifespan due to the harshness of current treatment options. […] Despite the 75% survival rates, cancer victims continue to suffer from the disease. Late effects can be long-term and progressive. This is especially for children who were young when they were diagnosed and who were treated with cranial radiation. […] To improve the outcomes of children who are diagnosed with brain tumors, it’s critical to improve our understanding of the disease, find ways to treat brain tumors more effectively, and reduce the toxicity of treatments. […] Researchers have found some changes in genes, chromosomes, and proteins inside brain tumor cells that can be used to help predict a child’s outlook or help guide treatment. […] However, pediatric brain tumor research is underfunded. Only 4% of federal funding for cancer research goes to studies on pediatric cancers. […] Pediatric brain tumors, which are now the leading cause of cancer-related death in children and adolescents ages 19 and younger.
  • #65 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    This insight into molecular mechanisms has resulted in increased accuracy and reliability of tumor diagnosis, has revealed potential therapeutic targets, and provides a rational basis to either augment or de-intensify therapy. […] High-grade gliomas (WHO Grade IV) have the highest mortality rate of all pediatric brain tumors, with five-year survival rates less than 20% despite maximal therapeutic interventions. […] The insight we have gained into the genomic drivers of pediatric high-grade gliomas has resulted in several clinical trials testing targeted agents against this group of tumors. […] The PI3K/AKT/mTOR pathway promotes cell survival and growth and is activated in several cancers. […] Ependymomas constitute about 5% of childhood brain tumors and occur more frequently in the posterior fossa.
  • #66 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    This insight into molecular mechanisms has resulted in increased accuracy and reliability of tumor diagnosis, has revealed potential therapeutic targets, and provides a rational basis to either augment or de-intensify therapy. […] High-grade gliomas (WHO Grade IV) have the highest mortality rate of all pediatric brain tumors, with five-year survival rates less than 20% despite maximal therapeutic interventions. […] The insight we have gained into the genomic drivers of pediatric high-grade gliomas has resulted in several clinical trials testing targeted agents against this group of tumors. […] The PI3K/AKT/mTOR pathway promotes cell survival and growth and is activated in several cancers. […] Ependymomas constitute about 5% of childhood brain tumors and occur more frequently in the posterior fossa.
  • #67 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    The efficacy of post-radiation chemotherapy in treating ependymoma in various settings is still being investigated, and chemotherapy is used for high-risk, subtotally resected, or recurrent disease. […] Driven by success in some adult cancers, there has been an increase in clinical trials utilizing various forms of immunotherapy for both adult and pediatric CNS tumors. […] Immune checkpoint inhibitors have been studied against adult brain tumors, and several clinical trials are underway to utilize anti-PD1 and CTLA-4 inhibitors in pediatric high-grade glioma. […] Pediatric brain tumors are the most common solid malignancies in children.
  • #68 Advances in the Treatment of Pediatric Brain Tumors
    https://www.mdpi.com/2227-9067/10/1/62
    The efficacy of post-radiation chemotherapy in treating ependymoma in various settings is still being investigated, and chemotherapy is used for high-risk, subtotally resected, or recurrent disease. […] Driven by success in some adult cancers, there has been an increase in clinical trials utilizing various forms of immunotherapy for both adult and pediatric CNS tumors. […] Immune checkpoint inhibitors have been studied against adult brain tumors, and several clinical trials are underway to utilize anti-PD1 and CTLA-4 inhibitors in pediatric high-grade glioma. […] Pediatric brain tumors are the most common solid malignancies in children.
  • #69 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    The clinical activity of checkpoint blockade correlates with three main variables: (1) the number of nonsynonymous/frameshift somatic mutations in the tumor, which results in the production of neoantigens; (2) high expression of the PD-1 ligand in tumor cells; and (3) the frequency of activated CD8+ T cells in the circulation. […] The utility and success of checkpoint inhibition therapy for pediatric brain cancer remain to be demonstrated.
  • #70 Epigenomics and immunotherapeutic advances in pediatric brain tumors | npj Precision Oncology
    https://www.nature.com/articles/s41698-021-00173-4
    The clinical activity of checkpoint blockade correlates with three main variables: (1) the number of nonsynonymous/frameshift somatic mutations in the tumor, which results in the production of neoantigens; (2) high expression of the PD-1 ligand in tumor cells; and (3) the frequency of activated CD8+ T cells in the circulation. […] The utility and success of checkpoint inhibition therapy for pediatric brain cancer remain to be demonstrated.
  • #71 Pediatric Brain Tumor Statistics & The Critical Need for More Research – IronMatt
    https://ironmatt.org/pediatric-brain-tumor-statistics/
    Glioblastomas have a 20% 5-year survival rate. […] If a child survives, they have a 95 percent chance of developing chronic health problems, secondary cancers, cognitive impairments, and a shortened lifespan due to the harshness of current treatment options. […] Despite the 75% survival rates, cancer victims continue to suffer from the disease. Late effects can be long-term and progressive. This is especially for children who were young when they were diagnosed and who were treated with cranial radiation. […] To improve the outcomes of children who are diagnosed with brain tumors, it’s critical to improve our understanding of the disease, find ways to treat brain tumors more effectively, and reduce the toxicity of treatments. […] Researchers have found some changes in genes, chromosomes, and proteins inside brain tumor cells that can be used to help predict a child’s outlook or help guide treatment. […] However, pediatric brain tumor research is underfunded. Only 4% of federal funding for cancer research goes to studies on pediatric cancers. […] Pediatric brain tumors, which are now the leading cause of cancer-related death in children and adolescents ages 19 and younger.