Rak mięśniakomięśniowy
Zapobieganie i profilaktyka

Rak mięśniakomięśniowy (RMS) jest najczęstszym mięsakiem tkanek miękkich u dzieci, głównie poniżej 10 roku życia, jednak brak jest potwierdzonych czynników środowiskowych lub stylu życia wpływających na jego rozwój, co ogranicza możliwości profilaktyki. Znane czynniki ryzyka, takie jak wiek, płeć i predyspozycje genetyczne, są niemodyfikowalne. Badania genetyczne wykazują potencjał w identyfikacji wariantów predysponujących (CPVs), szczególnie w rodzinach z zespołami genetycznymi, co umożliwia wczesny nadzór kliniczny i kaskadowe testowanie. W terapii RMS stosuje się profilaktykę infekcji, m.in. trimetoprim-sulfametoksazol do 6 miesięcy po chemioterapii, a także leki G-CSF, takie jak mekapegfilgrastym, wykazujące akceptowalny profil bezpieczeństwa i skuteczności u dzieci i młodzieży leczonych schematem VDC (winkrystyna, doksorubicyna, cyklofosfamid). Rola profilaktyki ośrodkowego układu nerwowego (OUN) w RMS głowy i szyi z zajęciem opon mózgowo-rdzeniowych pozostaje kontrowersyjna, a badania wskazują na wątpliwą skuteczność radioterapii mózgu (30 Gy) i dokanałowego metotreksatu (I.T. MTX) w tym kontekście.

Wprowadzenie do profilaktyki raka mięśniakomięśniowego

Rak mięśniakomięśniowy (rhabdomyosarcoma, RMS) jest rzadkim typem nowotworu tkanek miękkich, ale jednocześnie najczęstszym mięsakiem tkanek miękkich występującym u dzieci.1 Choroba ta dotyka najczęściej dzieci poniżej 10 roku życia.2 Aktualne dane wskazują, że nie istnieją potwierdzone czynniki środowiskowe ani związane ze stylem życia, które przyczyniają się do rozwoju RMS, co znacznie ogranicza możliwości opracowania skutecznych strategii profilaktycznych.34

Ograniczenia w profilaktyce pierwotnej

Obecnie nie istnieją sprawdzone metody zapobiegania rozwojowi raka mięśniakomięśniowego.56 W przeciwieństwie do wielu nowotworów występujących u dorosłych, gdzie zmiany stylu życia (takie jak utrzymywanie prawidłowej masy ciała czy rzucenie palenia) mogą zmniejszyć ryzyko zachorowania, w przypadku nowotworów dziecięcych nie opracowano skutecznych metod profilaktycznych.7

Znane czynniki ryzyka RMS, takie jak wiek, płeć oraz określone uwarunkowania genetyczne, nie podlegają modyfikacji.8 Ze względu na brak precyzyjnego rozpoznania przyczyn rozwoju RMS, nie istnieją zalecane modyfikacje stylu życia ani środowiska, które mogłyby zapobiec wystąpieniu tego nowotworu.91011

Rola badań genetycznych w profilaktyce

Mimo braku klasycznych metod profilaktyki, badania genetyczne mogą odgrywać istotną rolę w identyfikacji osób z podwyższonym ryzykiem zachorowania na RMS, zwłaszcza w rodzinach z określonymi zespołami genetycznymi.12

Predyspozycje genetyczne i testowanie

Warianty predysponujące do zachorowania na nowotwór (cancer-predisposition variants, CPVs) mogą być pomocne w przewidywaniu ryzyka w niektórych nowotworach dziecięcych, w tym w raku mięśniakomięśniowym.13 Wyniki badań wspierają przeprowadzanie testów genetycznych w kierunku wariantów predysponujących do rozwoju nowotworów u dzieci z rozpoznanym RMS, co może pomóc w opracowaniu wcześniejszych strategii nadzoru klinicznego dla pacjentów oraz w kaskadowym testowaniu członków rodziny.14

Rzadko RMS wiąże się z zespołami genetycznymi przekazywanymi z pokolenia na pokolenie.15 W przypadku pacjentów z rozpoznanymi zespołami predysponującymi do rozwoju nowotworów (np. zespół Li-Fraumeni) zalecane jest unikanie radioterapii, która mogłaby zwiększyć ryzyko rozwoju wtórnych nowotworów.1617

Istnieje jednak luka w zrozumieniu roli predyspozycji nowotworowych w pediatrycznym RMS, co ogranicza oddziaływanie translacyjne. Nie przeprowadzono dotychczas badań populacyjnych, które określiłyby rzeczywisty wpływ tych mutacji na rozwój RMS u dzieci, co z kolei ogranicza wytyczne dotyczące sekwencjonowania klinicznego i protokoły nadzoru u tych dzieci.18

Profilaktyka nawrotu choroby

Nawet po pomyślnym zakończeniu leczenia, regularny follow-up jest kluczowy dla zapobiegania wystąpieniu wtórnych nowotworów.19 Badania obrazowe, takie jak CT i MRI, są zwykle zalecane w celu wykrycia nawrotu guza. Fizjoterapia może pomóc pacjentowi odzyskać siłę.20

Nowe podejścia do zapobiegania nawrotom

Naukowcy z St. Jude Children’s Research Hospital przeprowadzili badania nad populacją komórek, które przetrwały terapię i powodują nawrót RMS. Odkryli, że te komórki przypominają wczesny stan rozwojowy, który odpowiada na inhibitory EGFR (receptora naskórkowego czynnika wzrostu).21

Badacze chcieli lepiej zrozumieć komórki, które dają początek nawrotowemu RMS, aby zidentyfikować nowe metody leczenia zapobiegające powrotowi nowotworu po terapii.22 Zespół odkrył, że ta populacja komórek zależy od sygnalizacji EGFR i jest wrażliwa na inhibitory EGFR. Inhibitory EGFR są rodzajem terapii celowanej stosowanej w leczeniu nowotworów z mutacjami w genie EGFR, takich jak rak płuc u dorosłych.23

Te odkrycia potwierdzają potrzebę zastosowania inhibitorów EGFR w leczeniu RMS:24

  • Istnieje dowód koncepcyjny, że jeśli połączy się leczenie tych rzadkich przetrwałych komórek inhibitorem EGFR z chemioterapią, uzyskuje się znacznie lepszy wynik
  • Takie podejście odzwierciedla inny sposób myślenia o terapii, który nie koncentruje się wyłącznie na początkowej odpowiedzi na leczenie

25

Naukowcy kontynuują badania nad inhibitorami EGFR przeciwko RMS i badają także możliwość zastosowania koniugatów przeciwciało-lek oraz możliwość opracowania komórek CAR-T dla tego celu.26 Konieczne jest jednak dalsze przeprowadzenie badań, zanim inhibitory EGFR będą mogły być testowane u pacjentów.27

Profilaktyka w trakcie leczenia

Profilaktyka zakażeń

W trakcie leczenia RMS stosuje się różne środki profilaktyczne zapobiegające powikłaniom infekcyjnym:28

29

Profilaktyka neutropenii i gorączki neutropenicznej

Obecnie leki z grupy czynników stymulujących wzrost kolonii granulocytów (G-CSF) są uznawane za jeden z najczęściej stosowanych schematów profilaktyki gorączki neutropenicznej (FN) i neutropenii wywołanej chemioterapią, jednak niewiele badań koncentrowało się na skuteczności i bezpieczeństwie leków G-CSF u pacjentów pediatrycznych i młodzieży.30

Przeprowadzono pilotażowe, jednoramienne badanie mające na celu zbadanie skuteczności i bezpieczeństwa mekapegfilgrastymu w profilaktyce FN u dzieci i młodzieży z RMS lub mięsakiem Ewinga (ES). Mekapegfilgrastym wykazał akceptowalny profil skuteczności i bezpieczeństwa u pediatrycznych i młodzieżowych pacjentów z RMS lub ES.31

Pacjenci leczeni schematem VDC (winkrystyna, doksorubicyna, cyklofosfamid) powinni być monitorowani z ostrożnością nawet po otrzymaniu długo działających rekombinowanych leków G-CSF.32 Potrzebne są dalsze randomizowane badania z większymi grupami pacjentów oraz danymi dotyczącymi profilaktyki wtórnej.33

Profilaktyka CUN w RMS głowy i szyi

W przypadku pacjentów z RMS głowy i szyi z zajęciem opon mózgowo-rdzeniowych, rola profilaktyki ośrodkowego układu nerwowego (OUN) pozostaje kontrowersyjna.

W badaniu porównującym dwie kolejne serie dzieci z RMS (RMSA) okolicy głowy i szyi z zajęciem opon mózgowo-rdzeniowych, druga seria pacjentów, oprócz miejscowej radioterapii i polichemioterapii, otrzymała radioterapię mózgu (30 Gy) i metotreksat dokanałowo (I.T. MTX) jako profilaktykę ośrodkowego układu nerwowego (OUN) w latach 1978-1980.34 Jednak ze względu na zastosowanie wyższych dawek radioterapii na guz pierwotny w drugiej serii, trudno jednoznacznie przypisać poprawę wyników wyłącznie dodaniu profilaktyki OUN.35

W innym badaniu serii 15 kolejnych dzieci z RMS głowy i szyi bez zajęcia oczodołu z zajęciem opon mózgowo-rdzeniowych prospektywnie leczono chemioterapią składającą się z Adriamycyny (doksorubicyna; Adria Laboratory, Columbus, OH) (ADM), winkrystyny (VCR), cyklofosfamidu (CPM) i daktynomycyny (DACT), a następnie radioterapii (60 Gy) na objętość guza pierwotnego, wraz z metotreksatem dokanałowo (IT MTX).36 Wyniki tego badania sugerują, że profilaktyka OUN z radioterapią jest wątpliwa w leczeniu dziecięcego RMSA z zajęciem opon mózgowo-rdzeniowych.37

Podsumowanie aktualnych badań nad profilaktyką

Potrzebne są dynamiczne podejścia integrujące badania populacyjne i biologię molekularną, aby wyjaśnić mechanizmy leżące u podstaw dziecięcego raka mięśniakomięśniowego i określić nowe strategie profilaktyczne.38 Zidentyfikowano potencjalne cele terapeutyczne, takie jak EGFR, które mogą być wykorzystane do zapobiegania nawrotom RMS.39

Naukowcy opracowują nowe strategie eliminacji wszystkich komórek nowotworowych (każdego klonu) w celu osiągnięcia wyleczenia.40 Obejmują one zastosowanie chemioterapii z użyciem leków celowanych (np. inhibitory EGFR), koniugaty przeciwciało-lek, a nawet rozważenie komórek CAR-T w zapobieganiu nawrotom RMS.4142

Obecnie znając mechanizmy molekularne i komórkowe nawrotu, badacze mogą skuteczniej eliminować wszystkie komórki nowotworowe i zapobiegać nawrotom.43 Te odkrycia stanowią podstawę do opracowania innowacyjnych strategii profilaktycznych, które mogą znacząco poprawić rokowanie w RMS.

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  1. 09.04.2026
  2. www.leksykon.com.pl

Materiały źródłowe

  • #1 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    Rhabdomyosarcoma is a rare type of soft tissue cancer but the most common type of soft tissue sarcoma in children. […] We are now applying this approach to all pediatric solid tumors to be more effective at preventing recurrence and improving outcomes. […] We need to eliminate all the tumor cells (each clone) to achieve a cure. […] We will employ chemotherapy using targeted agents (e.g., EGFR inhibitors) and antibody-drug conjugates and even consider CAR T-cells. […] We were pleased to see EGFR as a potential target within our data because there is a deep and long-standing track record of targeting that receptor. […] So, it seemed natural to test EGFR inhibitors against rhabdomyosarcoma. […] The results were exciting, but we still have a lot of work to do before we’re ready to test EGFR inhibitors in patients.
  • #2 Rhabdomyosarcoma – Symptoms and causes – Mayo Clinic
    https://www.mayoclinic.org/diseases-conditions/rhabdomyosarcoma/symptoms-causes/syc-20390962
    There is no way to prevent rhabdomyosarcoma. […] Factors that may increase the risk of rhabdomyosarcoma include: […] Rhabdomyosarcoma most often happens to children younger than 10. […] Rarely, rhabdomyosarcoma has been linked to genetic syndromes that are passed from parents to children.
  • #3 Rhabdomyosarcoma Causes, Risk Factors, and Prevention | American Cancer Society
    https://www.cancer.org/cancer/types/rhabdomyosarcoma/causes-risks-prevention.html
    There are no proven lifestyle-related or environmental causes of RMS, so at this time there is no way to protect against these cancers. […] Can Rhabdomyosarcoma Be Prevented?
  • #4 Can Rhabdomyosarcoma Be Prevented? | American Cancer Society
    https://www.cancer.org/cancer/types/rhabdomyosarcoma/causes-risks-prevention/prevention.html
    The risk of many cancers that typically occur in adults can be reduced with certain lifestyle changes (such as staying at a healthy weight or quitting smoking), but at this time there are no known ways to prevent most cancers in children. […] The only known risk factors for rhabdomyosarcoma (RMS) age, sex, and certain inherited conditions cant be changed. There are no proven lifestyle-related or environmental causes of RMS, so at this time there is no known way to protect against these cancers.
  • #5 Rhabdomyosarcoma primary prevention – wikidoc
    https://www.wikidoc.org/index.php/Rhabdomyosarcoma_primary_prevention
    There are no primary preventive measures available for rhabdomyosarcoma. […] There are no primary preventive measures available for childhood cancers such as rhabdomyosarcoma.
  • #6 Pediatric Rhabdomyosarcoma: Practice Essentials, Background, Pathophysiology
    https://emedicine.medscape.com/article/988803-overview
    No preventive measures are known for childhood cancers.
  • #7 Can Rhabdomyosarcoma Be Prevented? | American Cancer Society
    https://www.cancer.org/cancer/types/rhabdomyosarcoma/causes-risks-prevention/prevention.html
    The risk of many cancers that typically occur in adults can be reduced with certain lifestyle changes (such as staying at a healthy weight or quitting smoking), but at this time there are no known ways to prevent most cancers in children. […] The only known risk factors for rhabdomyosarcoma (RMS) age, sex, and certain inherited conditions cant be changed. There are no proven lifestyle-related or environmental causes of RMS, so at this time there is no known way to protect against these cancers.
  • #8 Can Rhabdomyosarcoma Be Prevented? | American Cancer Society
    https://www.cancer.org/cancer/types/rhabdomyosarcoma/causes-risks-prevention/prevention.html
    The risk of many cancers that typically occur in adults can be reduced with certain lifestyle changes (such as staying at a healthy weight or quitting smoking), but at this time there are no known ways to prevent most cancers in children. […] The only known risk factors for rhabdomyosarcoma (RMS) age, sex, and certain inherited conditions cant be changed. There are no proven lifestyle-related or environmental causes of RMS, so at this time there is no known way to protect against these cancers.
  • #9 Rhabdomyosarcoma Treatment in Mumbai, India | Nanavati Max Hospital
    https://www.nanavatimaxhospital.org/our-specialities/cancer-centre/conditions-treatments/rhabdomyosarcoma
    Because the exact cause of the tumor is unknown, there aren’t any measures to prevent rhabdomyosarcoma. Lifestyle changes or environmental modifications do not prevent rhabdomyosarcoma. […] Even after successfully completing the treatment, follow-up should be regularly done to prevent the onset of secondary tumors. CT scans and MRI scans are usually advised to detect the recurrence of the tumor. Physiotherapy can help the patient to regain strength.
  • #10 Rhabdomyosarcomas | EBSCO Research Starters
    https://www.ebsco.com/research-starters/health-and-medicine/rhabdomyosarcomas
    There are no known environmental or lifestyle risk factors associated with its development, although certain inherited conditions can increase susceptibility. […] While there are no preventive measures for rhabdomyosarcoma, advances in treatment have improved outcomes for affected children. […] There is no way to prevent rhabdomyosarcoma.
  • #11 Rhabdomyosarcoma Treatment in Delhi, India | Symptoms & Causes
    https://www.blkmaxhospital.com/our-specialities/sarcoma-and-bone-cancer-program/conditions-treatments/rhabdomyosarcoma
    As doctors and medical experts do not know why and how a child gets rhabdomyosarcoma, there is no way to prevent this cancer or reduce the risk.
  • #12 Rhabdomyosarcoma | Baptist Health Miami Cancer Institute
    https://baptisthealth.net/services/cancer-care/miami-cancer-institute/our-approach/pediatric-cancers/rhabdomyosarcoma
    Because most known risk factors are not preventable, there are no recommended preventive measures. […] Some children with risk factors may benefit from genetic testing to determine specific risk.
  • #13 Cancer-predisposition variants associated with adverse outcomes in rhabdomyosarcoma | BCM
    https://www.bcm.edu/news/cancer-predisposition-variants-associated-with-adverse-outcomes-in-rhabdomyosarcoma
    Germline cancer-predisposition variants (CPVs) can be helpful in predicting risk in some pediatric cancers. […] The results of our study support germline testing for cancer-predisposition variants among children with rhabdomyosarcoma, which could aid in early clinical surveillance strategies for patients and cascade testing of family members, Lupo said.
  • #14 Cancer-predisposition variants associated with adverse outcomes in rhabdomyosarcoma | BCM
    https://www.bcm.edu/news/cancer-predisposition-variants-associated-with-adverse-outcomes-in-rhabdomyosarcoma
    Germline cancer-predisposition variants (CPVs) can be helpful in predicting risk in some pediatric cancers. […] The results of our study support germline testing for cancer-predisposition variants among children with rhabdomyosarcoma, which could aid in early clinical surveillance strategies for patients and cascade testing of family members, Lupo said.
  • #15 Rhabdomyosarcoma – Symptoms and causes – Mayo Clinic
    https://www.mayoclinic.org/diseases-conditions/rhabdomyosarcoma/symptoms-causes/syc-20390962
    There is no way to prevent rhabdomyosarcoma. […] Factors that may increase the risk of rhabdomyosarcoma include: […] Rhabdomyosarcoma most often happens to children younger than 10. […] Rarely, rhabdomyosarcoma has been linked to genetic syndromes that are passed from parents to children.
  • #16 Rhabdomyosarcoma | 5-Minute Pediatric Consult
    https://peds.unboundmedicine.com/pedscentral/view/5-Minute-Pediatric-Consult/617335/all/Rhabdomyosarcoma?q=antiemetics
    No standard approach because usually sporadic […] Avoidance of radiation in patients with known predisposing syndromes (e.g., Li-Fraumeni syndrome)
  • #17 Rhabdomyosarcoma | 5-Minute Pediatric Consult
    https://peds.unboundmedicine.com/pedscentral/view/5-Minute-Pediatric-Consult/617335/all/Rhabdomyosarcoma?q=Cell+Germ+Tumors
    No standard approach because usually sporadic. […] Avoidance of radiation in patients with known predisposing syndromes (e.g., Li-Fraumeni syndrome).
  • #18 Germline and Somatic Genetic Landscape of Pediatric Rhabdomyosarcoma – EGA European Genome-Phenome Archive
    https://ega-archive.org/studies/phs002304
    Dynamic approaches that integrate population-based research and molecular biology are needed to explain the mechanisms underlying pediatric rhabdomyosarcoma (RMS) and to determine novel prevention strategies. […] Therefore, a major gap in our understanding of the role of cancer predisposition in pediatric RMS that limit translational impact is there have been no population-based assessments to determine the true impact of these mutations on pediatric RMS, which limits clinical sequencing guidelines and surveillance protocols in these children.
  • #19 Rhabdomyosarcoma Treatment in Mumbai, India | Nanavati Max Hospital
    https://www.nanavatimaxhospital.org/our-specialities/cancer-centre/conditions-treatments/rhabdomyosarcoma
    Because the exact cause of the tumor is unknown, there aren’t any measures to prevent rhabdomyosarcoma. Lifestyle changes or environmental modifications do not prevent rhabdomyosarcoma. […] Even after successfully completing the treatment, follow-up should be regularly done to prevent the onset of secondary tumors. CT scans and MRI scans are usually advised to detect the recurrence of the tumor. Physiotherapy can help the patient to regain strength.
  • #20 Rhabdomyosarcoma Treatment in Mumbai, India | Nanavati Max Hospital
    https://www.nanavatimaxhospital.org/our-specialities/cancer-centre/conditions-treatments/rhabdomyosarcoma
    Because the exact cause of the tumor is unknown, there aren’t any measures to prevent rhabdomyosarcoma. Lifestyle changes or environmental modifications do not prevent rhabdomyosarcoma. […] Even after successfully completing the treatment, follow-up should be regularly done to prevent the onset of secondary tumors. CT scans and MRI scans are usually advised to detect the recurrence of the tumor. Physiotherapy can help the patient to regain strength.
  • #21 Lab findings suggest EGFR inhibitors may prevent rhabdomyosarcoma recurrence – St. Jude Children’s Research Hospital
    https://www.stjude.org/media-resources/news-releases/2022-medicine-science-news/egfr-inhibitors-may-prevent-rhabdomyosarcoma-recurrence.html
    Rhabdomyosarcoma is a type of soft tissue cancer. Scientists at St. Jude Childrens Research Hospital studied the population of cells that persists after therapy, causing rhabdomyosarcoma recurrence. They found that these cells mirror an early developmental state that responds to EGFR inhibitors. […] The researchers wanted to better understand the cells that give rise to recurrent rhabdomyosarcoma to identify new treatments to prevent the cancer from returning after therapy. […] The team found that this population of cells depends on epidermal growth factor receptor (EGFR) signaling and is sensitive to EGFR inhibitors. EGFR inhibitors are a type of targeted therapy used to treat cancers with mutations in the EGFR gene, such as lung cancer in adults. The findings support a clinical trial strategy that includes EGFR inhibitors in rhabdomyosarcoma treatment.
  • #22 Lab findings suggest EGFR inhibitors may prevent rhabdomyosarcoma recurrence – St. Jude Children’s Research Hospital
    https://www.stjude.org/media-resources/news-releases/2022-medicine-science-news/egfr-inhibitors-may-prevent-rhabdomyosarcoma-recurrence.html
    Rhabdomyosarcoma is a type of soft tissue cancer. Scientists at St. Jude Childrens Research Hospital studied the population of cells that persists after therapy, causing rhabdomyosarcoma recurrence. They found that these cells mirror an early developmental state that responds to EGFR inhibitors. […] The researchers wanted to better understand the cells that give rise to recurrent rhabdomyosarcoma to identify new treatments to prevent the cancer from returning after therapy. […] The team found that this population of cells depends on epidermal growth factor receptor (EGFR) signaling and is sensitive to EGFR inhibitors. EGFR inhibitors are a type of targeted therapy used to treat cancers with mutations in the EGFR gene, such as lung cancer in adults. The findings support a clinical trial strategy that includes EGFR inhibitors in rhabdomyosarcoma treatment.
  • #23 Lab findings suggest EGFR inhibitors may prevent rhabdomyosarcoma recurrence – St. Jude Children’s Research Hospital
    https://www.stjude.org/media-resources/news-releases/2022-medicine-science-news/egfr-inhibitors-may-prevent-rhabdomyosarcoma-recurrence.html
    Rhabdomyosarcoma is a type of soft tissue cancer. Scientists at St. Jude Childrens Research Hospital studied the population of cells that persists after therapy, causing rhabdomyosarcoma recurrence. They found that these cells mirror an early developmental state that responds to EGFR inhibitors. […] The researchers wanted to better understand the cells that give rise to recurrent rhabdomyosarcoma to identify new treatments to prevent the cancer from returning after therapy. […] The team found that this population of cells depends on epidermal growth factor receptor (EGFR) signaling and is sensitive to EGFR inhibitors. EGFR inhibitors are a type of targeted therapy used to treat cancers with mutations in the EGFR gene, such as lung cancer in adults. The findings support a clinical trial strategy that includes EGFR inhibitors in rhabdomyosarcoma treatment.
  • #24 Lab findings suggest EGFR inhibitors may prevent rhabdomyosarcoma recurrence – St. Jude Children’s Research Hospital
    https://www.stjude.org/media-resources/news-releases/2022-medicine-science-news/egfr-inhibitors-may-prevent-rhabdomyosarcoma-recurrence.html
    We have a proof of concept that if you target those rare cells that persist with an EGFR inhibitor and combine that with chemotherapy you get a much better outcome because you’re treating the entire tumor. This reflects a different way of thinking about therapy that isn’t focused just on initial response.
  • #25 Lab findings suggest EGFR inhibitors may prevent rhabdomyosarcoma recurrence – St. Jude Children’s Research Hospital
    https://www.stjude.org/media-resources/news-releases/2022-medicine-science-news/egfr-inhibitors-may-prevent-rhabdomyosarcoma-recurrence.html
    We have a proof of concept that if you target those rare cells that persist with an EGFR inhibitor and combine that with chemotherapy you get a much better outcome because you’re treating the entire tumor. This reflects a different way of thinking about therapy that isn’t focused just on initial response.
  • #26 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    In addition to the EGFR inhibitors, we are also testing antibody-drug conjugates and hope to explore the possibility of developing a CAR T cell for this target. […] Now that we know the molecular and cellular mechanisms of recurrence, we can do a better job of killing all the tumor cells and preventing recurrence.
  • #27 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    Rhabdomyosarcoma is a rare type of soft tissue cancer but the most common type of soft tissue sarcoma in children. […] We are now applying this approach to all pediatric solid tumors to be more effective at preventing recurrence and improving outcomes. […] We need to eliminate all the tumor cells (each clone) to achieve a cure. […] We will employ chemotherapy using targeted agents (e.g., EGFR inhibitors) and antibody-drug conjugates and even consider CAR T-cells. […] We were pleased to see EGFR as a potential target within our data because there is a deep and long-standing track record of targeting that receptor. […] So, it seemed natural to test EGFR inhibitors against rhabdomyosarcoma. […] The results were exciting, but we still have a lot of work to do before we’re ready to test EGFR inhibitors in patients.
  • #28 Pediatric Rhabdomyosarcoma Follow-up: Further Outpatient Care, Further Inpatient Care, Inpatient & Outpatient Medications
    https://emedicine.medscape.com/article/988803-followup
    Trimethoprim-sulfamethoxazole: Prophylaxis against pneumocystic pneumonia should continue until 6 months after the end of chemotherapy. […] Fluconazole: Systemic fungal prophylaxis is not necessary. […] Clotrimazole: Prophylactic therapy for thrush may be discontinued after chemotherapy is completed. […] Chlorhexidine mouth rinse: Prophylaxis against gingivitis and other mouth infections may be discontinued after chemotherapy is completed.
  • #29 Pediatric Rhabdomyosarcoma Follow-up: Further Outpatient Care, Further Inpatient Care, Inpatient & Outpatient Medications
    https://emedicine.medscape.com/article/988803-followup
    Trimethoprim-sulfamethoxazole: Prophylaxis against pneumocystic pneumonia should continue until 6 months after the end of chemotherapy. […] Fluconazole: Systemic fungal prophylaxis is not necessary. […] Clotrimazole: Prophylactic therapy for thrush may be discontinued after chemotherapy is completed. […] Chlorhexidine mouth rinse: Prophylaxis against gingivitis and other mouth infections may be discontinued after chemotherapy is completed.
  • #30 Mecapegfilgrastim for prophylaxis of febrile neutropenia in children and adolescents with rhabdomyosarcoma or Ewing sarcoma: a prospective, single-arm, pilot study | BMC Cancer | Full Text
    https://bmccancer.biomedcentral.com/articles/10.1186/s12885-024-12766-w
    Mecapegfilgrastim showed acceptable efficacy and safety profile in pediatric and adolescent patients with RMS or ES. […] Our objective was to investigate the efficacy and safety of mecapegfilgrastim, a biosimilar of pegfilgrastim, in prophylaxis of FN for pediatric and adolescent patients with RMS or ES. […] Currently, granulocyte-colony stimulating factor (G-CSF) drugs have been recognized as one of the most commonly used regimens for prophylaxis of chemotherapy-induced FN and neutropenia, however, only a few studies have focused on the efficacy and safety of G-CSF drugs in pediatric and adolescent patients. […] On the basis of the above information, we conducted this pilot, single-arm study aimed to explore the efficacy and safety of mecapegfilgrastim for the prophylaxis of FN in pediatric and adolescent patients with RMS or ES. […] Patients who are treated with the VDC regimen should be monitored with caution even after receiving long-acting rhG-CSF drugs. […] Further randomized studies with large sample sizes and secondary prophylaxis data are warranted.
  • #31 Mecapegfilgrastim for prophylaxis of febrile neutropenia in children and adolescents with rhabdomyosarcoma or Ewing sarcoma: a prospective, single-arm, pilot study | BMC Cancer | Full Text
    https://bmccancer.biomedcentral.com/articles/10.1186/s12885-024-12766-w
    Mecapegfilgrastim showed acceptable efficacy and safety profile in pediatric and adolescent patients with RMS or ES. […] Our objective was to investigate the efficacy and safety of mecapegfilgrastim, a biosimilar of pegfilgrastim, in prophylaxis of FN for pediatric and adolescent patients with RMS or ES. […] Currently, granulocyte-colony stimulating factor (G-CSF) drugs have been recognized as one of the most commonly used regimens for prophylaxis of chemotherapy-induced FN and neutropenia, however, only a few studies have focused on the efficacy and safety of G-CSF drugs in pediatric and adolescent patients. […] On the basis of the above information, we conducted this pilot, single-arm study aimed to explore the efficacy and safety of mecapegfilgrastim for the prophylaxis of FN in pediatric and adolescent patients with RMS or ES. […] Patients who are treated with the VDC regimen should be monitored with caution even after receiving long-acting rhG-CSF drugs. […] Further randomized studies with large sample sizes and secondary prophylaxis data are warranted.
  • #32 Mecapegfilgrastim for prophylaxis of febrile neutropenia in children and adolescents with rhabdomyosarcoma or Ewing sarcoma: a prospective, single-arm, pilot study | BMC Cancer | Full Text
    https://bmccancer.biomedcentral.com/articles/10.1186/s12885-024-12766-w
    Mecapegfilgrastim showed acceptable efficacy and safety profile in pediatric and adolescent patients with RMS or ES. […] Our objective was to investigate the efficacy and safety of mecapegfilgrastim, a biosimilar of pegfilgrastim, in prophylaxis of FN for pediatric and adolescent patients with RMS or ES. […] Currently, granulocyte-colony stimulating factor (G-CSF) drugs have been recognized as one of the most commonly used regimens for prophylaxis of chemotherapy-induced FN and neutropenia, however, only a few studies have focused on the efficacy and safety of G-CSF drugs in pediatric and adolescent patients. […] On the basis of the above information, we conducted this pilot, single-arm study aimed to explore the efficacy and safety of mecapegfilgrastim for the prophylaxis of FN in pediatric and adolescent patients with RMS or ES. […] Patients who are treated with the VDC regimen should be monitored with caution even after receiving long-acting rhG-CSF drugs. […] Further randomized studies with large sample sizes and secondary prophylaxis data are warranted.
  • #33 Mecapegfilgrastim for prophylaxis of febrile neutropenia in children and adolescents with rhabdomyosarcoma or Ewing sarcoma: a prospective, single-arm, pilot study | BMC Cancer | Full Text
    https://bmccancer.biomedcentral.com/articles/10.1186/s12885-024-12766-w
    Mecapegfilgrastim showed acceptable efficacy and safety profile in pediatric and adolescent patients with RMS or ES. […] Our objective was to investigate the efficacy and safety of mecapegfilgrastim, a biosimilar of pegfilgrastim, in prophylaxis of FN for pediatric and adolescent patients with RMS or ES. […] Currently, granulocyte-colony stimulating factor (G-CSF) drugs have been recognized as one of the most commonly used regimens for prophylaxis of chemotherapy-induced FN and neutropenia, however, only a few studies have focused on the efficacy and safety of G-CSF drugs in pediatric and adolescent patients. […] On the basis of the above information, we conducted this pilot, single-arm study aimed to explore the efficacy and safety of mecapegfilgrastim for the prophylaxis of FN in pediatric and adolescent patients with RMS or ES. […] Patients who are treated with the VDC regimen should be monitored with caution even after receiving long-acting rhG-CSF drugs. […] Further randomized studies with large sample sizes and secondary prophylaxis data are warranted.
  • #34
    https://journals.lww.com/amjclinicaloncology/abstract/1983/08000/childhood_rhabdomyosarcoma_with_meningeal.2.aspx
    RESULTS ACHIEVED IN TWO CONSECUTIVE SERIES of children with rhabdomyosarcoma (RMSA) of the head and neck area, presenting with meningeal involvement, are compared. […] The second series consisted of 11 children who, besides local radiotherapy and polichemotherapy, received brain radiotherapy (30 Gy) and intrathecal (I.T.) methotrexate (MTX) as central nervous system (CNS) prophylaxis from 1978 to 1980. […] This observation and the higher doses of radiotherapy delivered to the primary tumor in the second series prevent the conclusion that the improved results are attributable entirely to the addition of CNS prophylaxis.
  • #35
    https://journals.lww.com/amjclinicaloncology/abstract/1983/08000/childhood_rhabdomyosarcoma_with_meningeal.2.aspx
    RESULTS ACHIEVED IN TWO CONSECUTIVE SERIES of children with rhabdomyosarcoma (RMSA) of the head and neck area, presenting with meningeal involvement, are compared. […] The second series consisted of 11 children who, besides local radiotherapy and polichemotherapy, received brain radiotherapy (30 Gy) and intrathecal (I.T.) methotrexate (MTX) as central nervous system (CNS) prophylaxis from 1978 to 1980. […] This observation and the higher doses of radiotherapy delivered to the primary tumor in the second series prevent the conclusion that the improved results are attributable entirely to the addition of CNS prophylaxis.
  • #36 Questionable role of CNS radioprophylaxis in the therapeutic management of childhood rhabdomyosarcoma with meningeal extension – PubMed
    https://pubmed.ncbi.nlm.nih.gov/2230872/
    A series of 15 consecutive children with head and neck nonorbital rhabdomyosarcoma (RMSA) with meningeal extension were prospectively treated with chemotherapy consisting of Adriamycin (doxorubicin; Adria Laboratory, Columbus, OH) (ADM), vincristine (VCR), cyclophosphamide (CPM), and dactinomycin (DACT) followed by radiotherapy (60 Gy) to the primary tumor volume, along with intrathecal methotrexate (IT MTX). […] It is concluded that CNS prophylaxis with radiotherapy is questionable in the management of childhood RMSA with meningeal extension.
  • #37 Questionable role of CNS radioprophylaxis in the therapeutic management of childhood rhabdomyosarcoma with meningeal extension – PubMed
    https://pubmed.ncbi.nlm.nih.gov/2230872/
    A series of 15 consecutive children with head and neck nonorbital rhabdomyosarcoma (RMSA) with meningeal extension were prospectively treated with chemotherapy consisting of Adriamycin (doxorubicin; Adria Laboratory, Columbus, OH) (ADM), vincristine (VCR), cyclophosphamide (CPM), and dactinomycin (DACT) followed by radiotherapy (60 Gy) to the primary tumor volume, along with intrathecal methotrexate (IT MTX). […] It is concluded that CNS prophylaxis with radiotherapy is questionable in the management of childhood RMSA with meningeal extension.
  • #38 Germline and Somatic Genetic Landscape of Pediatric Rhabdomyosarcoma – EGA European Genome-Phenome Archive
    https://ega-archive.org/studies/phs002304
    Dynamic approaches that integrate population-based research and molecular biology are needed to explain the mechanisms underlying pediatric rhabdomyosarcoma (RMS) and to determine novel prevention strategies. […] Therefore, a major gap in our understanding of the role of cancer predisposition in pediatric RMS that limit translational impact is there have been no population-based assessments to determine the true impact of these mutations on pediatric RMS, which limits clinical sequencing guidelines and surveillance protocols in these children.
  • #39 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    Rhabdomyosarcoma is a rare type of soft tissue cancer but the most common type of soft tissue sarcoma in children. […] We are now applying this approach to all pediatric solid tumors to be more effective at preventing recurrence and improving outcomes. […] We need to eliminate all the tumor cells (each clone) to achieve a cure. […] We will employ chemotherapy using targeted agents (e.g., EGFR inhibitors) and antibody-drug conjugates and even consider CAR T-cells. […] We were pleased to see EGFR as a potential target within our data because there is a deep and long-standing track record of targeting that receptor. […] So, it seemed natural to test EGFR inhibitors against rhabdomyosarcoma. […] The results were exciting, but we still have a lot of work to do before we’re ready to test EGFR inhibitors in patients.
  • #40 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    Rhabdomyosarcoma is a rare type of soft tissue cancer but the most common type of soft tissue sarcoma in children. […] We are now applying this approach to all pediatric solid tumors to be more effective at preventing recurrence and improving outcomes. […] We need to eliminate all the tumor cells (each clone) to achieve a cure. […] We will employ chemotherapy using targeted agents (e.g., EGFR inhibitors) and antibody-drug conjugates and even consider CAR T-cells. […] We were pleased to see EGFR as a potential target within our data because there is a deep and long-standing track record of targeting that receptor. […] So, it seemed natural to test EGFR inhibitors against rhabdomyosarcoma. […] The results were exciting, but we still have a lot of work to do before we’re ready to test EGFR inhibitors in patients.
  • #41 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    Rhabdomyosarcoma is a rare type of soft tissue cancer but the most common type of soft tissue sarcoma in children. […] We are now applying this approach to all pediatric solid tumors to be more effective at preventing recurrence and improving outcomes. […] We need to eliminate all the tumor cells (each clone) to achieve a cure. […] We will employ chemotherapy using targeted agents (e.g., EGFR inhibitors) and antibody-drug conjugates and even consider CAR T-cells. […] We were pleased to see EGFR as a potential target within our data because there is a deep and long-standing track record of targeting that receptor. […] So, it seemed natural to test EGFR inhibitors against rhabdomyosarcoma. […] The results were exciting, but we still have a lot of work to do before we’re ready to test EGFR inhibitors in patients.
  • #42 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    In addition to the EGFR inhibitors, we are also testing antibody-drug conjugates and hope to explore the possibility of developing a CAR T cell for this target. […] Now that we know the molecular and cellular mechanisms of recurrence, we can do a better job of killing all the tumor cells and preventing recurrence.
  • #43 Azthena logo with the word Azthena
    https://www.news-medical.net/news/20220512/Researchers-Identify-Potential-Target-for-Rhabdomyosarcoma-Recurrence-Prevention.aspx
    In addition to the EGFR inhibitors, we are also testing antibody-drug conjugates and hope to explore the possibility of developing a CAR T cell for this target. […] Now that we know the molecular and cellular mechanisms of recurrence, we can do a better job of killing all the tumor cells and preventing recurrence.