Mielofibroza – Epidemiologia

Mielofibroza
Epidemiologia

Mielofibroza (MF) to rzadki nowotwór mieloproliferacyjny BCR-ABL-ujemny, charakteryzujący się włóknieniem szpiku, niedokrwistością, pozaszpikową hematopoezą i splenomegalią. Roczna zapadalność waha się od 0,1 do 1,5 na 100 000 osób, z wyższymi wartościami w krajach rozwiniętych (do 1,9 na 100 000 w UE, 1,6 na 100 000 w Niemczech w 2021 r.). Chorobowość globalnie wynosi około 1 na 100 000, z wyższymi wskaźnikami w USA i Europie (do 11,0 na 100 000 w Niemczech). Mediana wieku diagnozy to 65-68 lat, z przewagą mężczyzn (stosunek 1,5:1) i częstszym występowaniem u osób białych. Pierwotna mielofibroza (PMF) stanowi około 75% przypadków, a mutacje JAK2 występują u 50-60% pacjentów, co podkreśla znaczenie genetycznej stratyfikacji ryzyka. Przeżycie mediana wynosi 3-7 lat w stadium włóknienia, a 5-letnie przeżycie to 50-58%. Powikłania obejmują zakrzepicę (do 10%), nadciśnienie wrotne (7%) oraz ryzyko progresji do ostrej białaczki szpikowej.

Epidemiologia Mielofibrozy

Zapadalność na Mielofibroze
Chorobowość Mielofibrozy
Czynniki Demograficzne i Rozkład Populacji
Podtypy Mielofibrozy i Stratyfikacja Ryzyka
Przeżycie i Powikłania
Trendy i Zmiany w Epidemiologii
Wyzwania w Badaniach Epidemiologicznych

Nadzór Epidemiologiczny Mielofibrozy

Nadzór nad Objawami i Powikłaniami
Programy i Rejestry Nadzoru
Przyszłe Trendy i Potrzeby Nadzorcze
Kolejne rozdziały

Epidemiologia Mielofibrozy

Mielofibroza (MF) jest rzadkim nowotworem mieloproliferacyjnym charakteryzującym się włóknieniem szpiku kostnego, niedokrwistością, pozaszpikową hematopoezą i splenomegalią. Należy do grupy nowotworów mieloproliferacyjnych (MPN) BCR-ABL-ujemnych, a jej epidemiologia jest zróżnicowana w zależności od regionu geograficznego oraz metod diagnostycznych stosowanych w różnych krajach.¹²³

Zapadalność na Mielofibroze

Roczna zapadalność na mielofibroze według różnych źródeł wynosi od 0,1 do 1,5 przypadków na 100 000 osób. Metaanaliza badań epidemiologicznych dotyczących MPN opublikowanych przed 2014 rokiem, obejmująca głównie populacje z Europy i Ameryki Północnej, wykazała, że roczne wskaźniki zapadalności na pierwotną mielofibroze (PMF) wahały się od 0,22 do 0,99 na 100 000.¹² W Stanach Zjednoczonych roczna zapadalność szacowana jest na około 0,5-1,5 przypadków na 100 000 osób.¹²

W Europie wskaźniki zapadalności na mielofibroze różnią się w zależności od kraju. W przeglądzie europejskich źródeł danych, zapadalność na mielofibroze wahała się od 0,1 do 1 przypadku na 100 000 osób rocznie.¹² W krajach Unii Europejskiej zapadalność waha się od niskiego poziomu 0,3 na 100 000 osób do wysokiego 1,9 na 100 000 osób, ze średnią zapadalnością wynoszącą 1,1 na 100 000 osób.¹

Interesujące badanie przeprowadzone w Norwegii wykazało stopniowy wzrost wskaźnika zapadalności na mielofibroze w czasie. Według rejestru nowotworów w Norwegii, standaryzowany według wieku roczny wskaźnik zapadalności wzrósł z 0,2 na 100 000 w latach 1995-1997 do 0,5 na 100 000 w latach 2010-2012.¹

Najnowsze dane z Niemiec, oparte na analizie roszczeń ubezpieczeniowych, wykazały, że skumulowana zapadalność na mielofibroze w 2021 roku wynosiła 1,6 na 100 000 osób, z zakresem od 1,2 do 1,8 na 100 000 w zależności od przyjętych scenariuszy wrażliwości.¹

Chorobowość Mielofibrozy

Chorobowość mielofibrozy jest zróżnicowana globalnie. Ogólnie szacuje się, że wynosi ona około 1 na 100 000 osób na całym świecie.¹² W krajach rozwiniętych chorobowość waha się od 0,5 na 100 000 rocznie do 9 na 100 000 rocznie.¹

Badania przeprowadzone w Europie i USA wskazują, że chorobowość waha się od 2,5 do 5,7 na 100 000 osób. Jednakże najnowsze dane z Niemiec pokazują znacznie wyższy wskaźnik chorobowości – standaryzowana chorobowość punktowa na dzień 31.12.2021 r. wynosiła 11,0 na 100 000 osób.¹²

Według raportu epidemiologicznego obejmującego siedem głównych rynków (7MM: Stany Zjednoczone, Niemcy, Francja, Włochy, Hiszpania, Wielka Brytania i Japonia), całkowita liczba przypadków chorobowości mielofibrozy wynosiła około 55 900 w 2024 roku i prognozuje się jej wzrost w okresie badania (2020-2034).¹ Inne źródło podaje, że w 2023 roku w 7MM było prawie 56 700 przypadków chorobowości mielofibrozy.¹

W samych Stanach Zjednoczonych całkowita liczba przypadków chorobowości mielofibrozy wynosiła prawie 19 300 w 2023 roku, a przewiduje się, że liczba ta wzrośnie w okresie prognozy (2024-2034).¹ Szacuje się, że w USA żyje około 13 000 osób z mielofibroza.¹

Warto zauważyć, że w rozszerzonym zestawie ośmiu głównych rynków (8MM), który obejmuje również Chiny, zdiagnozowane przypadki chorobowości mielofibrozy wynosiły 69 236 w 2021 roku i przewiduje się, że wzrosną do 84 311 przypadków w 2031 roku, przy rocznym wskaźniku wzrostu (AGR) wynoszącym 2,18%.¹²

Czynniki Demograficzne i Rozkład Populacji

Mielofibroza jest chorobą, która zwykle dotyka osoby w średnim i starszym wieku. Mediana wieku w momencie diagnozy wynosi około 65-68 lat, przy czym średni wiek zachorowania to 60-70 lat.¹²³ Chociaż choroba może wystąpić u młodszych osób, jest rzadka wśród osób poniżej 50 roku życia. Około 22% dotkniętych pacjentów jest młodszych niż 56 lat.¹²

Mielofibroza u dzieci zwykle występuje w pierwszych 3 latach życia, choć jest to bardzo rzadkie.¹²

Rozkład płci

Większość badań wskazuje na przewagę zachorowań wśród mężczyzn niż kobiet. Stosunek mężczyzn do kobiet wynosi około 1,5:1.¹¹ Jednakże u młodszych dzieci dziewczynki są dotknięte chorobą dwa razy częściej niż chłopcy.¹²

Rozkład płci może różnić się w zależności od podtypu choroby – pierwotna mielofibroza (PMF) jest częstsza u mężczyzn, natomiast wtórna mielofibroza po nadpłytkowości samoistnej jest częstsza u kobiet.¹

Należy zauważyć, że niektóre źródła nie wykazują przewagi płci, sugerując brak dominacji mężczyzn lub kobiet.¹ Również według niektórych raportów mielofibroza jest równie częsta u mężczyzn i kobiet.¹

Rozkład rasowy i etniczny

Mielofibroza wydaje się być częstsza u osób białych niż u osób innych ras. Ponadto, zwiększona częstość występowania tego zaburzenia została zauważona u Żydów aszkenazyjskich.¹²³

Afroamerykanie, Latynosi i Azjaci są mniej narażeni na rozwój mielofibrozy.¹ Jednakże interesujące badanie przeprowadzone w Malezji wykazało, że Chińczycy mieli stosunkowo wysoki ważony odsetek zachorowań (43,2%), następnie Hindusi (23,8%), Malajowie (15,8%) i inne grupy etniczne (17,2%).¹

Podtypy Mielofibrozy i Stratyfikacja Ryzyka

Mielofibroza może być dalej podzielona na pierwotną mielofibroze (PMF) i wtórną mielofibroze. W 2024 roku pierwotna mielofibroza stanowiła około 75% wszystkich przypadków w USA.¹¹

Rejestr ERNEST (European Registry for Myeloproliferative Neoplasms; towards a better understanding of Epidemiology, Survival and Treatment) obejmował dane 1209 pacjentów z mielofibroze (mediana wieku 66 lat), z czego 61% miało PMF, 20% miało wtórną mielofibroze po nadpłytkowości samoistnej (post-ET MF), a 19% miało wtórną mielofibroze po czerwienicy prawdziwej (post-PV MF).¹

Stratyfikacja ryzyka

Na podstawie ryzyka, przypadki mielofibrozy są stratyfikowane jako niskie ryzyko, pośrednie-1 ryzyko, pośrednie-2 ryzyko i wysokie ryzyko. W 2024 roku w USA największa liczba pacjentów była sklasyfikowana jako grupa wysokiego ryzyka.¹¹

Stratyfikacja ryzyka jest kluczowym elementem w podejmowaniu decyzji dotyczących leczenia, szczególnie przy rozważaniu roli transplantacji.¹

Alteracje molekularne

W USA przypadki mutacji JAK2 stanowią około 60% wszystkich przypadków w 2024 roku.¹¹ Ostatnie badania wykazują, że około 50-60% osób z mielofibroze ma mutację w białku JAK2, które reguluje produkcję komórek krwi.¹

U wielu pacjentów z mielofibroze choroba jest napędzana przez mutacje w genie JAK2.¹ Wysoka częstość mutacji JAK2 wskazuje na potrzebę migracji z konwencjonalnych systemów prognostycznych opartych głównie na cechach klinicznych, takich jak Międzynarodowy System Punktacji Prognostycznej (IPSS) i Dynamiczny Międzynarodowy System Punktacji Prognostycznej (DIPSS), w kierunku nowych systemów, które stratyfikują ryzyko pacjentów w oparciu o ich profil genetyczny.¹

Przeżycie i Powikłania

Pacjenci z mielofibroze mają podwyższone ryzyko zmniejszonego przeżycia w porównaniu z populacją ogólną. Przeżycie wynosi 3-7 lat dla pacjentów zdiagnozowanych w stadium włóknienia oraz 10-15 lat dla pacjentów zdiagnozowanych we wczesnej fazie przedwłóknieniowej.¹

Wskaźniki przeżycia 5-letniego od diagnozy zawierają się między 50% a 58%.¹ Według jednego ze źródeł, 39% pacjentów z mielofibroze osiąga 5-letnie przeżycie całkowite.¹

Mediana przeżycia wynosi około 5,2-5,9 lat.¹ Rokowanie jest gorsze u pacjentów z cytopeniami i podwyższonym ryzykiem transformacji białaczkowej.¹

Powikłania i komorbidowosc

Mielofibroza wiąże się z różnymi powikłaniami, które mogą wpływać na przebieg choroby i przeżycie pacjentów:

Do 10% pacjentów doświadcza zdarzenia zakrzepowo-zatorowego, najczęściej żylnej choroby zakrzepowo-zatorowej¹
Splenomegalia jest nieuniknionym wynikiem i może prowadzić do zawału śledziony, krwotoku, zakrzepicy żył trzewnych, nadciśnienia wrotnego lub objawów efektu masy¹
Istnieje również niewielkie ryzyko progresji do ostrej białaczki szpikowej¹²
MPN są obecnie uznawane za wiodącą przyczynę zakrzepicy żył trzewnych, dotykając młodszą grupę wiekową (mediana wieku 48 lat)¹
Nadciśnienie wrotne dotyka 7% pacjentów, z powodu zwiększonego przepływu krwi w wątrobie, wewnątrzwątrobowej niedrożności żylnej i zastoju ze splenomegalią¹

Pacjenci z mielofibroze leczeni ruksolitynibem mogą mieć zwiększone ryzyko wystąpienia drugiego pierwotnego nowotworu złośliwego; ryzyko to wydaje się być jeszcze większe u mężczyzn i pacjentów z historią zakrzepicy tętniczej oraz długotrwałej ekspozycji na hydroksykarbamid.¹

Trendy i Zmiany w Epidemiologii

Epidemiologia mielofibrozy podlega ciągłym zmianom. Wskaźniki chorobowości są generalnie wyższe niż wcześniej raportowano, co może być wyjaśnione przez ulepszoną diagnostykę, zwiększoną ofertę badań profilaktycznych i zmianę klasyfikacji przedwłóknieniowej mielofibrozy przez Światową Organizację Zdrowia (WHO) w 2016 roku.¹

Epidemiolodzy przypisują wzrost zdiagnozowanych przypadków zachorowań i chorobowości mielofibrozy zmianom w zdiagnozowanej zapadalności i wskaźnikach przeżycia, a także dynamice populacji na każdym rynku.¹²

W Chinach przewiduje się największą liczbę zdiagnozowanych przypadków zachorowań na mielofibroze w 8MM w 2031 roku, z 9 302 przypadkami, podczas gdy Hiszpania będzie miała najmniejszą liczbę zdiagnozowanych przypadków zachorowań na mielofibroze z 413 przypadkami.¹

Wyzwania w Badaniach Epidemiologicznych

Istnieje szeroka zmienność zarówno w szacunkach chorobowości, jak i zapadalności obserwowanych w europejskich źródłach danych.¹ Badania epidemiologiczne dotyczące mielofibrozy napotykają na różne wyzwania:

Ograniczone dane epidemiologiczne dla mielofibrozy i brak w wielu krajach znormalizowanego raportowania danych¹
Trudności w porównywaniu badań dotyczących chorobowości, ponieważ różne rodzaje chorobowości są obliczane (chorobowość punktowa a chorobowość okresowa) i standaryzacja jest dokonywana według różnych populacji (np. populacja światowa i populacja europejska)¹
Brak danych epidemiologicznych dotyczących mielofibrozy w niektórych krajach, np. we Włoszech¹

Potrzebne są starannie zaprojektowane badania ze znormalizowanymi definicjami MPN i pełnym ustaleniem pacjentów, w tym zarówno pierwotnej, jak i wtórnej mielofibrozy, aby lepiej zrozumieć populację docelową dla nowych terapii.¹

Nadzór Epidemiologiczny Mielofibrozy

Ruxolitinib (Jakavi) jest doustnym inhibitorem JAK1/JAK2, który został po raz pierwszy zatwierdzony przez amerykańską Agencję ds. Żywności i Leków (FDA) w 2011 roku do leczenia dorosłych z mielofibroze o pośrednim lub wysokim ryzyku, w tym pierwotną mielofibroze, mielofibroze po czerwienicy prawdziwej i mielofibroze po nadpłytkowości samoistnej.¹

Dane z badań nadzoru pomarketingowego ruksolitinibu były ogólnie zgodne z danymi raportowanymi w badaniach klinicznych. Łącznie 14 445 pacjentów otrzymało leczenie ruksolitinibem od Międzynarodowej Daty Urodzenia Rozwoju (29 lutego 2008 r.) w interwencyjnych badaniach klinicznych sponsorowanych przez firmy Novartis i Incyte oraz w programach dostępu zarządzanego.¹

W Korei Południowej prowadzone jest badanie obserwacyjne posprzedażowe w celu oceny bezpieczeństwa fedratinibu w rzeczywistych warunkach u dorosłych pacjentów z pierwotną mielofibroze, mielofibroze po czerwienicy prawdziwej lub mielofibroze po nadpłytkowości samoistnej, którzy byli wcześniej leczeni ruksolitinibem.¹

W Europie prowadzone jest wieloośrodkowe retrospektywne i prospektywne badanie obserwacyjne, którego celem jest uzyskanie informacji na temat cytopenii związanych z mielofibroze, które stanowią znaczące wyzwanie u współczesnych pacjentów z mielofibroze.¹

Nadzór nad Objawami i Powikłaniami

Ocena objawów u pacjentów w momencie diagnozy jest ważna. Według retrospektywnego przeglądu dokumentacji medycznej 180 pacjentów z mielofibroze, 95% pacjentów zgłosiło 2 lub więcej objawów związanych z mielofibroze w momencie diagnozy.¹

Wytyczne NCCN zalecają monitorowanie pacjentów z mielofibroze, w tym ocenę obciążenia objawami, określanie, czy pacjent jest kandydatem do przeszczepu, monitorowanie odpowiedzi na terapię cytoredukcyjną oraz monitorowanie pod kątem oznak i objawów progresji choroby.¹

Zaleca się aktywny nadzór skórny dla pacjentów z mielofibroze leczonych ruksolitinibem, zwłaszcza dla tych, którzy są również narażeni na długotrwałe działanie hydroksymocznika. Pacjenci z historią zakrzepicy tętniczej powinni być badani pod kątem drugich nowotworów, a klinicyści powinni uwzględniać płeć pacjenta podczas oceny ryzyka.¹

Programy i Rejestry Nadzoru

Istnieje kilka programów i rejestrów, które monitorują epidemiologię, leczenie i wyniki mielofibrozy:

ERNEST (European Registry for Myeloproliferative Neoplasms; towards a better understanding of Epidemiology, Survival and Treatment) – rejestr obejmujący dane ponad 1200 pacjentów z mielofibroze¹
Badanie rejestru ERNEST badające wpływ ruksolitinibu na ogólne przeżycie, z wykorzystaniem danych zebranych prospektywnie¹
Pierwsze badanie MPN Landmark u pacjentów z MPN pod leczeniem w USA, dotyczące percepcji pacjentów na temat obciążenia chorobą i zarządzania leczeniem¹
Badania nadzoru pomarketingowego dla leków takich jak ruksolitinib i fedratinib¹²

Programy te są kluczowe dla lepszego zrozumienia rzeczywistego obciążenia chorobą, skuteczności i bezpieczeństwa leczenia oraz niezaspokojonych potrzeb medycznych w mielofibrozie.

Przyszłe Trendy i Potrzeby Nadzorcze

W miarę ewolucji wiedzy patofizjologicznej i klinicznej w MPNs, istnieje potrzeba rewizji kryteriów diagnostycznych, prognozowania i zaleceń terapeutycznych.¹

Rutyny kodowania diagnostycznego w Niemczech powinny odzwierciedlać podtypy mielofibrozy (przedwłóknieniowa, po-ET, po-PV, pierwotna MF) zgodnie z aktualną klasyfikacją WHO.¹

Nadzór nad cytopeniami związanymi z mielofibroze stanowi znaczące wyzwanie u współczesnych pacjentów z mielofibroze.¹ Potrzebne są dalsze badania nad MPN i ich związkiem z COVID-19, ponieważ nie ma wystarczających dowodów na to, że COVID-19 przyspiesza progresję mielofibrozy.¹

Potrzeba więcej danych na temat terapii ruksolitinibem u pacjentów stratyfikowanych według płci, co prawdopodobnie przyczyni się do opracowania spersonalizowanych strategii zarządzania powikłaniami związanymi z terapią i chorobą.¹

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Materiały źródłowe

#1 Epidemiology and disease characteristics of myelofibrosis: a comparative analysis between Italy and global perspectives
https://pmc.ncbi.nlm.nih.gov/articles/PMC11303153/
Myelofibrosis (MF) is a rare condition. According to a meta-analysis of epidemiologic studies of MPNs published before 2014, and comprising mostly populations from Europe and North America, annual incidence rates of PMF ranged from 0.22 to 0.99 per 100,000. […] Overall, the findings of these studies suggest that age-standardized incidence rates of PMF range from 0.3 to 0.8 per 100,000 person-years, the incidence is higher in men than women, and survival rates at 5 years from diagnosis are included between 50% and 58%. […] On the whole, this overview highlights the paucity of epidemiological data for MF and the lack in many countries of standardized data reporting, as already pointed out by Moulard et al. in their comprehensive review of the epidemiologic literature published in 2014. Epidemiologic data about MF in Italy are lacking.
#1 Primary Myelofibrosis: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/197954-overview
Primary myelofibrosis is an uncommon disease, with an annual incidence of approximately 0.5-1.5 cases per 100,000 individuals in the United States. In a review of European data sources, the incidence rate of myelofibrosis ranged from 0.1 to 1 cases per 100,000 per year. […] […] Primary myelofibrosis appears to be more common in Whites than in individuals of other races. In addition, an increased prevalence rate of the disorder has been noted in Ashkenazi Jews. […] A slight male preponderance appears to exist for primary myelofibrosis; however, in younger children, girls are affected twice as frequently as boys. […] Primary myelofibrosis characteristically occurs in individuals over age 50 years, with the median age at diagnosis being approximately 65 years. However, the disease has been reported in persons in all phases of life, from neonates to octogenarians. […] Approximately 22% of affected patients are younger than 56 years. Primary myelofibrosis in children usually occurs in the first 3 years of life.
#1 Epidemiology of myelofibrosis, essential thrombocythemia, and polycythemia vera in the European Union – PubMed
https://pubmed.ncbi.nlm.nih.gov/24372927/
Results: Eleven articles identified from EMBASE, three online hematology or oncology registries, and two Web-based databases or reports were used to summarize epidemiological estimates for MF, PV, and ET. The incidence rate of MF ranged from 0.1 per 100,000 per year to 1 per 100,000 per year. Among the various registries, the incidence of PV ranged from 0.4 per 100,000 per year to 2.8 per 100,000 per year, while the literature estimated the range of PV incidence to be 0.68 per 100,000 to 2.6 per 100,000 per year. The estimated incidence of ET was between 0.38 per 100,000 per year and 1.7 per 100,000 per year. While a few studies reported on the MPNs’ prevalences, it is difficult to compare them as various types of prevalence were calculated (point prevalence vs. period prevalence) and standardization was made according to different populations (e.g., the world population and the European population).
#1 Myelofibrosis epidemiology and demographics – wikidoc
https://www.wikidoc.org/index.php/Myelofibrosis_epidemiology_and_demographics
Myelofibrosis is a disease that tends to affect the middle-aged and elderly population with a mean age of 60 years at diagnosis. Males are more commonly affected than females. The male to female ratio is approximately 1.5 to 1. Myelofibrosis usually affects individuals of the Ashkenazi Jews race. African American, Latin American, and Asian individuals are less likely to develop myelofibrosis. […] The prevalence of myelofibrosis is approximately 1 per 100,000 individuals worldwide. […] In the countries of the European Union, the incidence of myelofibrosis ranges from a low of 0.3 per 100,000 persons to a high of 1.9 per 100,000 persons with an average incidence of 1.1 per 100,000 persons. […] In developed countries, the prevalence of myelofibrosis is ranged from 0.5 per 100,000 per year to 9 per 100,000 per year. […] Myelofibrosis is more prevalent in the Ashkenazi Jews with an evidence of genetic transmission. […] Gender distribution can differ by the subtype of the disease with primary myelofibrosis (PMF) being more prevalent in males and post-essential thrombocythemia being more common in females.
#1 Epidemiology and disease characteristics of myelofibrosis: a comparative analysis between Italy and global perspectives
https://pmc.ncbi.nlm.nih.gov/articles/PMC11303153/
The study based on the Cancer Registry of Norway revealed a gradual increase in the age-adjusted annual incidence rate of MF, which progressively increased from 0.2 per 100,000, in the period 1995-1997, to 0.5 per 100,000 in the period 2010-2012. […] Several studies have shown that the incidence of MF increases with age. The study by Hultcrantz et al. on data from the Swedish Cancer Register and the Swedish Blood Cancer Register has highlighted a substantially higher crude incidence rate of PMF in older versus younger individuals. […] The first MPN Landmark survey in patients with MPN under treatment in the US addressed patient perception of disease burden and treatment management. Of the 813 respondents, 207 (25.5%) had MF; their median age was 66 years and the median duration of disease was 4 years.
#1
https://journals.lww.com/hemasphere/fulltext/2023/08003/pb2203__incidence_and_prevalence_of_myelofibrosis.2065.aspx
Myelofibrosis (MF) is a rare type of hematologic neoplasia belonging to the group of myeloproliferative neoplasms that includes primary de novo disease and disease secondary to essential thrombocythemia (ET) or polycythemia vera (PV). Prevalence from studies conducted in Europe and the US ranges from 2.5-5.7/100,000 persons. To date, robust data on the epidemiology of MF in Germany are lacking. […] This study investigated the epidemiology of MF in Germany, including prevalence, incidence, and patient characteristics. […] In the base case scenario, 371 patients with MF were identified during the study period (median age, 68 years; female, 49.9%). Standardized point prevalence on 12/31/2021 based on the total German population was 11.0/100,000 persons. […] Cumulative incidence of MF in 2021 was estimated at 1.6/100,000 persons, with a range based on sensitivity scenarios of 1.2-1.8/100,000.
#1 Orphanet: Primary myelofibrosis
https://www.orpha.net/en/disease/detail/824
The annual incidence of primary myelofibrosis (PMF) is approximately 1 case per 100,000 individuals, although an increased prevalence is noted in Ashkenazi Jews. […] Age at diagnosis is usually in adulthood, around the sixth decade of life. […] The pathology is not inherited although it is linked with important gene mutations which occur at any time in life. […] Epidemiological studies have indicated the presence of common susceptibility alleles.
#1 Myelofibrosis (MF) Market Epidemiology and Forecast 2034:
https://www.globenewswire.com/news-release/2025/02/19/3028524/0/en/Myelofibrosis-MF-Market-Epidemiology-and-Forecast-2034-JAKAFI-Leads-Myelofibrosis-Market-But-New-Therapies-Poised-to-Disrupt-by-2034.html
The total prevalent cases of myelofibrosis in the 7MM were nearly ~55,900 in 2024 and is projected to increase during the study period (2020-2034). […] Among the EU4 and the UK, Germany accounted for the highest number of myelofibrosis diagnosed prevalent cases, followed by the Spain, whereas the UK accounted for the lowest number of cases in 2024. […] Based on risk, myelofibrosis cases are stratified as low risk, intermediate-1 risk, intermediate-2, and high risk. The high-risk accounted for the highest number of patients in 2024 in the US. […] Myelofibrosis can be further categorized into primary myelofibrosis and secondary myelofibrosis. In 2024, primary myelofibrosis accounted for ~75% of all cases in the US. […] In the US, based on age, myelofibrosis cases are stratified in the age group =49 years, 40-69 years, and =70 years. =70 years of age group accounted for the highest number of patients i.e. nearly ~12,100 in 2024 in the US. […] In the US, the cases of JAK2 mutations account for approximately ~60% in 2024.
#1 Myelofibrosis Epidemiology Forecast Research 2020-2034 – ResearchAndMarkets.com
https://www.businesswire.com/news/home/20250212300969/en/Myelofibrosis-Epidemiology-Forecast-Research-2020-2034—ResearchAndMarkets.com
The total prevalent cases of myelofibrosis in the 7MM were nearly 56,700 in 2023 and is projected to increase during the study period (2020-2034). […] The total prevalent cases of myelofibrosis in the United States were nearly 19,300 in 2023. These cases are expected to rise during the forecast period (2024-2034).
#1 Targeting a Rare Blood Cancer: Myelofibrosis | The AACR
https://www.aacr.org/patients-caregivers/progress-against-cancer/targeting-a-rare-blood-cancer-myelofibrosis/
Myelofibrosis is one of a group of blood cancers called chronic myeloproliferative neoplasms. […] It is estimated that there are about 13,000 people living with myelofibrosis in the United States. […] Fedratinib was approved for the treatment of adults with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. […] In many patients with myelofibrosis, the disease is driven by mutations in the JAK2 gene.
#1 Myelofibrosis Epidemiology Analysis and Forecast 2021-2022 & 2031: Focus on United States, France, Germany, Italy, Spain, UK, Japan, and China
https://www.prnewswire.com/news-releases/myelofibrosis-epidemiology-analysis-and-forecast-2021-2022–2031-focus-on-united-states-france-germany-italy-spain-uk-japan-and-china-301661129.html
In the 8MM, diagnosed incident cases of MF (sum of the diagnosed incident cases of PMF and SMF) are expected to increase from 12,276 cases in 2021 to 15,009 cases in 2031, at an Annual Growth Rate (AGR) of 2.23%. […] In 2031, China will have the highest number of diagnosed incident cases of MF in the 8MM, with 9,302 cases, whereas Spain will have the fewest diagnosed incident cases of MF with 413 cases. […] In the 8MM, the diagnosed prevalent cases of MF (sum of the diagnosed prevalent cases of PMF and SMF) are expected to increase from 69,236 cases in 2021 to 84,311 cases in 2031, at an AGR of 2.18%. […] The publisher’s epidemiologists attribute the increase in the diagnosed incident cases and diagnosed prevalent cases of MF to changes in the diagnosed incidence and survival rates, and population dynamics in each market.
#1 Myelofibrosis overview – wikidoc
https://www.wikidoc.org/index.php/Myelofibrosis_overview
The prevalence of myelofibrosis is approximately 1 per 100,000 individuals worldwide. […] Myelofibrosis is a disease that tends to affect the middle-aged and elderly population. The mean age at diagnosis is 60 years. […] Males are more commonly affected with myelofibrosis than females. The male to female ratio is approximately 1.5 to 1. […] Myelofibrosis usually affects individuals of the Ashkenazi Jews race. African American, Latin American, and Asian individuals are less likely to develop myelofibrosis. […] Common risk factors in the development of myelofibrosis may be age, other myeloproliferative disorders, radiation, or industrial chemical exposure.
#1 SEER Hematopoietic and Lymphoid Neoplasm Database
https://seer.cancer.gov/seertools/hemelymph/51f6cf57e3e27c3994bd5381/
Age: 60-70’s years median age Incidence: 0.5-1.5 per 100,000 persons per year Sex: no male or female predominance Survival: 3-7 years for patients diagnosed in fibrotic stage; 10-15 years for patients diagnosed in early prefibrotic phase
#1 Myelofibrosis (MF) – MPN Voice
https://www.mpnvoice.org.uk/about-mpns/questions/myelofibrosis-mf/
Myelofibrosis (MF) is considered to be a rare blood cancer. The number of people diagnosed each year with MF will be two to three cases per 100,000 and it is equally common in men and women. It primarily affects middle-aged and elderly people and is hardly ever diagnosed in children. […] Recent research shows that about 50-60% of people with MF have a mutation (or change) in a protein called JAK2, a protein that regulates blood cell production. […] We are learning more but additional research needs to be done to determine the causes of MF.
#1 The epidemiology and clinical characteristics of myeloproliferative neoplasms in Malaysia | Experimental Hematology & Oncology | Full Text
https://ehoonline.biomedcentral.com/articles/10.1186/s40164-018-0124-7
The evolution of molecular studies in myeloproliferative neoplasms (MPN) has enlightened us the understanding of this complex disease consisting of polycythaemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The epidemiology is well described in the western world but not in Asian countries like Malaysia. […] A total of 1010 patients were registered over a period of 5 years. The mean age was 54 years with male predominance. The ethnic distribution revealed that Chinese had a relatively high weighted incidence proportion (43.2%), followed by Indian (23.8%), Malay (15.8%) and other ethnic groups (17.2%). […] Chinese ethnicity is associated with higher rates of MPN. The history of thrombosis, age 60 years and hypertension are risk factors that can be correlated to JAK2 V617F mutation. […] In terms of annual incidence rate, it was reported as 0.84, 1.03 and 0.47 per 100,000 for PV, ET and PMF respectively based on meta-analysis of 34 studies which were highly heterogenous. […] The presence of JAK2 V617F mutation is associated with higher risk of arterial thrombosis.
#1 Myelofibrosis (MF) – Market Insight, Epidemiology, and Market Forecast – 2034
https://www.giiresearch.com/report/del1544152-myelofibrosis-mf-market-insight-epidemiology.html
The total prevalent cases of myelofibrosis in the 7MM were nearly ~55,900 in 2024 and is projected to increase during the study period (2020-2034). […] Among the EU4 and the UK, Germany accounted for the highest number of myelofibrosis diagnosed prevalent cases, followed by the Spain, whereas the UK accounted for the lowest number of cases in 2024. […] Based on risk, myelofibrosis cases are stratified as low risk, intermediate-1 risk, intermediate-2, and high risk. The high-risk accounted for the highest number of patients in 2024 in the US. […] Myelofibrosis can be further categorized into primary myelofibrosis and secondary myelofibrosis. In 2024, primary myelofibrosis accounted for ~75% of all cases in the US. […] In the US, based on age, myelofibrosis cases are stratified in the age group =49 years, 40-69 years, and =70 years. =70 years of age group accounted for the highest number of patients i.e. nearly ~12,100 in 2024 in the US. […] In the US, the cases of JAK2 mutations account for approximately ~60% in 2024.
#1 Epidemiology and disease characteristics of myelofibrosis: a comparative analysis between Italy and global perspectives
https://pmc.ncbi.nlm.nih.gov/articles/PMC11303153/
The ERNEST registry (European Registry for Myeloproliferative Neoplasms; towards a better understanding of Epidemiology, Survival and Treatment) included the data of 1209 patients with MF (median age 66 years), 61% with PMF, 20% with post-ET MF, and 19% with post-PV MF. […] A further study of the ERNEST registry involving 1010 patients with MF investigated the impact of ruxolitinib on overall survival using prospectively collected data. Age, male sex, high-risk category based on the Dynamic International Prognostic Scoring System (DIPSS) were identified as factors that negatively affected overall survival.
#1 Biological drivers of clinical phenotype in myelofibrosis | Leukemia
https://www.nature.com/articles/s41375-022-01767-y
Myelofibrosis (MF) is a myeloproliferative disorder that exhibits considerable biological and clinical heterogeneity. […] The incidence of MF is 0.44 per 100,000 person-years, with a median age at time of diagnosis of approximately 68 years and a median survival of 5.25.9 years. […] Prognostication for MF has evolved over the years and originated with the Lille classification which included leukocytosis or leukopenia and anemia to define three prognostic risk categories. […] The current benefit of utilizing any of these prognostic models is either in the context of determining clinical trial eligibility or risk-adapted treatment decision making and particularly when considering the role of transplantation. […] The cytopenic MF patient population is enriched for the PMF subtype, clinically typified by cytopenias and less extensive splenomegaly, and molecularly characterized by wild-type JAK2 or low JAK2 V617F VAF frequently accompanied by somatic mutations involving the spliceosome, epigenome, and apoptotic pathways. […] Due to inferior OS, higher risk of leukemic transformation and increased resistance to ruxolitinib therapy, cytopenic MF patients pose a therapeutic challenge and represent an unmet medical need.
#1 Meta-analysis of the frequency of JAK2 in primary myelofibrosis according to the detection method used
http://www.scielo.org.co/scielo.php?script=sci_arttext&pid=S0120-24482020000400041
Primary myelofibrosis (PMF) is a Philadelphia-negative chronic myeloproliferative neoplasm (CMPN). The estimated annual incidence for this disease is 0.5-1.5 cases per 100,000 persons; its prevalence is increasing due to improved diagnosis and survival 1,2. […] The high frequencies of JAK2 mutation found in this study evidence the need to migrate from the conventional prognostic systems based mainly on clinical characteristics, such as the International Prognostic Scoring System (IPSS) and Dynamic International Prognostic Scoring System (DIPSS), towards new systems which stratify patients’ risk based on their genetic profile, mainly the detection of the JAK2 mutation 40-45. […] The prevalence of JAK2 mutations showed no statistically significant difference according to the technique used. These results could be affected by a low statistical power (77.5%) caused by a low number of studies and patients analyzed per each of the techniques. […] A high frequency of JAK2 mutations was seen, showing that the diagnosis of PMF should not be made only by clinical and hematological characteristics, but also by the search for specific molecular markers.
#1 Quality Initiatives for Myelofibrosis | MPN Quality Initiatives
https://www.incytempnqualityinitiatives.com/myelofibrosis-quality-initiatives
Myelofibrosis (MF) is a serious hematologic malignancy. […] 39% of patients with MF reach 5-year overall survival. […] Most patients with MF have intermediate or high-risk disease, which is associated with shortened survival. […] Assessing patient symptoms at diagnosis is important. […] 95% of patients reported 2+ MF-related symptoms at diagnosis based on a retrospective chart review of 180 patients with MF. […] The majority of patients with MF reported that symptoms impact quality of life. […] The NCCN Guidelines recommend monitoring patients with MF. […] Recommended monitoring includes assessing symptom burden in patients with MF, determining whether they are a transplant candidate, monitoring response to cytoreductive therapy, and monitoring for signs and symptoms of disease progression.
#1 Primary myelofibrosis: spectrum of imaging features and disease-related complications | Insights into Imaging | Full Text
https://insightsimaging.springeropen.com/articles/10.1186/s13244-019-0758-y
The annual incidence of primary myelofibrosis (PMF) is 0.41.4 per 100,000 population and shows a predilection for older males, although younger patients can be affected. […] Of the MPNs, PMF is the least common but is associated with poorer survival, approximately 2 to 5 years upon diagnosis and symptom onset. […] Up to 10% of patients experience a thromboembolic event, most commonly venous thromboembolism. […] Splenomegaly is an inevitable outcome and may lead to splenic infarction, haemorrhage, splanchnic vein thrombosis, portal hypertension, or mass effect symptoms. […] There is also a small risk of progression to acute myeloid leukaemia. […] MPNs are now recognised as the leading systemic cause of splanchnic vein thrombosis and affecting a younger age group in general. […] Some of the largest studies of MPN-associated splanchnic vein thrombosis studies reported a prevalence of underlying MPN in splanchnic vein thrombosis of up to 50% with median age of 48 years.
#1 Primary myelofibrosis: spectrum of imaging features and disease-related complications | Insights into Imaging | Full Text
https://insightsimaging.springeropen.com/articles/10.1186/s13244-019-0758-y
The clinical management of splanchnic vein thrombosis in PMF is particularly challenging with a typically younger age of patient at diagnosis, severity of short- and long-term outcomes of inadequate treatment, and balance against the risks of haemorrhage with treatment. […] Portal hypertension affects 7% of patients, due to increased hepatic blood flow, intrahepatic venous obstruction, and stasis with splenomegaly.
#1 Surveillance for Second Primary Malignancy Recommended in Select Patients With Myelofibrosis Treated With Ruxolitinib
https://www.targetedonc.com/view/surveillance-for-second-primary-malignancy-recommended-in-select-patients-with-myelofibrosis-treated-with-ruxolitinib
Patients with myelofibrosis who receive ruxolitinib appear to face a higher risk of second primary malignancy; these risks are suggested to be even higher in men and patients with a history of arterial thrombosis and prolonged hydroxycarbamide exposure. […] Patients with MF, like patients with all types of myeloproliferative neoplasms (MPNs), face a higher-than-average risk of SPM, and existing research shows SPM is a major cause of morbidity and mortality in patients with MF. […] The authors said active skin surveillance is recommended for patients with MF treated with ruxolitinib. […] In terms of clinical practice, the authors said careful skin surveillance is essential for all patients with MF who undergo ruxolitinib therapy, and especially for those who also are exposed to hydroxyurea for long periods of time. Patients with a history of arterial thrombosis should be screened for second cancers, and clinicians should consider the patients gender when assessing risk. […] Further collection of data on [ruxolitinib] therapy in patients stratified by sex will probably contribute to the development of personalized strategies for the management of therapy- and disease-related complications.
#1
https://journals.lww.com/hemasphere/fulltext/2023/08003/pb2203__incidence_and_prevalence_of_myelofibrosis.2065.aspx
To our knowledge, this is the first study investigating the epidemiology of MF based on German claims data. Prevalence rates of MF were generally higher than previously reported, which may be explained by improved diagnostics, increased offerings for preventive checkups, and the classification change of prefibrotic MF by the World Health Organization (WHO) in 2016. Diagnostic coding routines in Germany should reflect subtypes of MF (prefibrotic, post-ET, post-PV, primary MF) per the current WHO classification.
#1 Epidemiology of myelofibrosis, essential thrombocythemia, and polycythemia vera in the European Union – PubMed
https://pubmed.ncbi.nlm.nih.gov/24372927/
Conclusion: There is a wide variation in both prevalence and incidence estimates observed across European data sources. Carefully designed studies, with standardized definitions of MPNs and complete ascertainment of patients including both primary and secondary MFs, should be conducted so that estimates of the population aimed to receive novel treatments for these neoplasms are better understood assist public health planning and provide valuable information about the burden of illness to policy makers, funding agencies, resource planners, healthcare insurers, and pharmaceutical manufacturers.
#1 Ten years of treatment with ruxolitinib for myelofibrosis: a review of safety | Journal of Hematology & Oncology | Full Text
https://jhoonline.biomedcentral.com/articles/10.1186/s13045-023-01471-z
Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by bone marrow fibrosis, anemia, extramedullary hematopoiesis, and splenomegaly. Patients with MF are at risk for reduced survival versus the general population and often experience burdensome signs and symptoms that reduce quality of life. […] Ruxolitinib is an oral Janus kinase (JAK) 1/JAK2 inhibitor first approved by the US Food and Drug Administration (FDA) in 2011 for the treatment of adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera (PV) MF, and post-essential thrombocythemia MF. […] Consequently, more than a decade since its initial approval, ruxolitinib continues to be the standard of care in patients with higher-risk MF. […] The approval of ruxolitinib was based on efficacy and safety demonstrated in the randomized, placebo-controlled phase 3 COMFORT trials.
#1 Ten years of treatment with ruxolitinib for myelofibrosis: a review of safety | Journal of Hematology & Oncology | Full Text
https://jhoonline.biomedcentral.com/articles/10.1186/s13045-023-01471-z
Here we review the safety profile of ruxolitinib, taking into account patients with MF who participated in clinical trials […] as well as those evaluated in postmarketing studies. […] In general, data from ruxolitinib postmarketing surveillance studies have been consistent with those reported in clinical trials. […] Overall, 14,445 patients received ruxolitinib treatment cumulatively since the Development International Birth Date (February 29, 2008) in Novartis- and Incyte-sponsored interventional clinical trials and managed access programs. […] The safety profile of ruxolitinib in MF has been established in the COMFORT trials and during the 10 years since its approval by the FDA, including in a series of clinical trials, pooled analyses, expanded-access studies, and postmarketing analyses.
#1 A Post-Marketing Surveillance Study to Assess the Safety of Fedratinib in Korean Patients With Myelofibrosis
https://www.careacross.com/clinical-trials/trial/NCT06073847
Trial Title: A Post-Marketing Surveillance Study to Assess the Safety of Fedratinib in Korean Patients With Myelofibrosis […] The purpose of this study is to assess the real-world safety of fedratinib for the treatment of adult participants with primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), or post essential thrombocythemia myelofibrosis (post-ET MF) who were previously treated with ruxolitinib. […] Adult participants with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis who were previously treated with ruxolitinib.
#1 Clinical Epidemiology in Contemporary Patients With Myelofibrosis. | Clinical Research Trial Listing
https://www.centerwatch.com/clinical-trials/listings/NCT06533813/clinical-epidemiology-in-contemporary-patients-with-myelofibrosis
Multicenter retrospective and prospective European observational study. […] This research was justified as the landscape of both pathophysiological and clinical knowledge in MPNs was rapidly evolving, prompting to revise diagnostic criteria, prognostication and therapy recommendations. […] The purpose of this study observational retrospective/prospective study is to gain information on MF associated cytopenias that represent a significant challenge in the contemporary patients with MF.
#1 Overt-stage primary myelofibrosis after COVID-19 infection: a case report – WCRJ
https://www.wcrj.net/article/2388
OBJECTIVE: Primary Myelofibrosis (PMF) is an uncommon hematological condition where the bone marrow becomes progressively fibrotic, resulting in anemia, extramedullary hematopoiesis, and splenomegaly. […] No sufficient evidence proves that COVID-19 accelerates the disease progression of primary myelofibrosis. More studies on the MPN and its relation to COVID-19 are needed.
#2 Epidemiology of myelofibrosis, essential thrombocythemia, and polycythemia vera in the European Union – PubMed
https://pubmed.ncbi.nlm.nih.gov/24372927/
Epidemiology of myelofibrosis, essential thrombocythemia, and polycythemia vera in the European Union […] Background: Primary myelofibrosis (PMF), essential thrombocythemia (ET), and polycythemia vera (PV) are BCR ABL-negative myeloproliferative neoplasms (MPN). Published epidemiology data are scarce, and multiple sources are needed to assess the disease burden. […] Methods: We assembled the most recent information available on the incidence and prevalence of myelofibrosis (MF), ET, and PV by conducting a structured and exhaustive literature review of the published peer-reviewed literature in EMBASE and by reviewing online documentation from disease registries and relevant health registries in European countries. The search was restricted to human studies written in English or French and published between January 1, 2000, and December 6, 2012.
#2 Primary Myelofibrosis: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/197954-overview
Primary myelofibrosis is an uncommon disease, with an annual incidence of approximately 0.5-1.5 cases per 100,000 individuals in the United States. In a review of European data sources, the incidence rate of myelofibrosis ranged from 0.1 to 1 cases per 100,000 per year. […] […] Primary myelofibrosis appears to be more common in Whites than in individuals of other races. In addition, an increased prevalence rate of the disorder has been noted in Ashkenazi Jews. […] A slight male preponderance appears to exist for primary myelofibrosis; however, in younger children, girls are affected twice as frequently as boys. […] Primary myelofibrosis characteristically occurs in individuals over age 50 years, with the median age at diagnosis being approximately 65 years. However, the disease has been reported in persons in all phases of life, from neonates to octogenarians. […] Approximately 22% of affected patients are younger than 56 years. Primary myelofibrosis in children usually occurs in the first 3 years of life.
#2 Primary Myelofibrosis Diagnosis & Disease Information
https://www.cancertherapyadvisor.com/ddi/primary-myelofibrosis/
Primary myelofibrosis is a rare cancer. In the United States (US), the annual incidence is approximately 0.3 cases per 100,000 individuals. The median age of patients with primary myelofibrosis is 65 to 67 years. […] Although primary myelofibrosis can occur in younger individuals, it is rare among those younger than age 50 years. Most studies have found that the incidence of primary myelofibrosis is higher in men than in women.
#2 Myelofibrosis epidemiology and demographics – wikidoc
https://www.wikidoc.org/index.php/Myelofibrosis_epidemiology_and_demographics
Myelofibrosis is a disease that tends to affect the middle-aged and elderly population with a mean age of 60 years at diagnosis. Males are more commonly affected than females. The male to female ratio is approximately 1.5 to 1. Myelofibrosis usually affects individuals of the Ashkenazi Jews race. African American, Latin American, and Asian individuals are less likely to develop myelofibrosis. […] The prevalence of myelofibrosis is approximately 1 per 100,000 individuals worldwide. […] In the countries of the European Union, the incidence of myelofibrosis ranges from a low of 0.3 per 100,000 persons to a high of 1.9 per 100,000 persons with an average incidence of 1.1 per 100,000 persons. […] In developed countries, the prevalence of myelofibrosis is ranged from 0.5 per 100,000 per year to 9 per 100,000 per year. […] Myelofibrosis is more prevalent in the Ashkenazi Jews with an evidence of genetic transmission. […] Gender distribution can differ by the subtype of the disease with primary myelofibrosis (PMF) being more prevalent in males and post-essential thrombocythemia being more common in females.
#2
https://journals.lww.com/hemasphere/fulltext/2023/08003/pb2203__incidence_and_prevalence_of_myelofibrosis.2065.aspx
To our knowledge, this is the first study investigating the epidemiology of MF based on German claims data. Prevalence rates of MF were generally higher than previously reported, which may be explained by improved diagnostics, increased offerings for preventive checkups, and the classification change of prefibrotic MF by the World Health Organization (WHO) in 2016. Diagnostic coding routines in Germany should reflect subtypes of MF (prefibrotic, post-ET, post-PV, primary MF) per the current WHO classification.
#2 Myelofibrosis Epidemiology Analysis and Forecast, 2021-2031
https://www.researchandmarkets.com/reports/5648207/myelofibrosis-epidemiology-analysis-and?srsltid=AfmBOoo35JLpIKq2iGWA9kCT7qGoMMtJY37Xq0eUoUGFHg_t9As8Wlx3
Myelofibrosis (MF) belongs to a group of diseases called myeloproliferative disorders. […] In the 8MM, diagnosed incident cases of MF (sum of the diagnosed incident cases of PMF and SMF) are expected to increase from 12,276 cases in 2021 to 15,009 cases in 2031, at an Annual Growth Rate (AGR) of 2.23%. […] In the 8MM, the diagnosed prevalent cases of MF (sum of the diagnosed prevalent cases of PMF and SMF) are expected to increase from 69,236 cases in 2021 to 84,311 cases in 2031, at an AGR of 2.18%. […] The publisher’s epidemiologists attribute the increase in the diagnosed incident cases and diagnosed prevalent cases of MF to changes in the diagnosed incidence and survival rates, and population dynamics in each market. […] The myelofibrosis epidemiology report is written and developed by Masters- and PhD-level epidemiologists. […] The Epidemiology Report is in-depth, high quality, transparent and market-driven, providing expert analysis of disease trends in the 8MM.
#2 SEER Hematopoietic and Lymphoid Neoplasm Database
https://seer.cancer.gov/seertools/hemelymph/51f6cf57e3e27c3994bd5381/
Age: 60-70’s years median age Incidence: 0.5-1.5 per 100,000 persons per year Sex: no male or female predominance Survival: 3-7 years for patients diagnosed in fibrotic stage; 10-15 years for patients diagnosed in early prefibrotic phase
#2 Primary Myelofibrosis: Practice Essentials, Pathophysiology, Etiology
https://emedicine.medscape.com/article/956806-overview
Primary myelofibrosis is an uncommon disease, with an annual incidence of approximately 0.5-1.5 cases per 100,000 individuals in the United States. In a review of European data sources, the incidence rate of myelofibrosis ranged from 0.1 to 1 cases per 100,000 per year. […] Primary myelofibrosis appears to be more common in Whites than in individuals of other races. In addition, an increased prevalence rate of the disorder has been noted in Ashkenazi Jews. […] A slight male preponderance appears to exist for primary myelofibrosis; however, in younger children, girls are affected twice as frequently as boys. […] Primary myelofibrosis characteristically occurs in individuals over age 50 years, with the median age at diagnosis being approximately 65 years. However, the disease has been reported in persons in all phases of life, from neonates to octogenarians. […] Approximately 22% of affected patients are younger than 56 years. Primary myelofibrosis in children usually occurs in the first 3 years of life.
#2 Treatment for Myeloproliferative Neoplasms (MPN) | Memorial Sloan Kettering Cancer Center
https://www.mskcc.org/cancer-care/types/leukemias/treatment/treatment-myeloproliferative-neoplasms
Some people with myelofibrosis develop acute myeloid leukemia. At MSK, we are committed to improving outcomes for people with myelofibrosis and people with acute myeloid leukemia that has developed after myelofibrosis. We are exploring new treatment options for people with these conditions and are working to translate new ideas from our research laboratories into innovative clinical trials.
#2 A Post-Marketing Surveillance Study to Assess the Safety of Fedratinib in Korean Patients With Myelofibrosis
https://www.careacross.com/clinical-trials/trial/NCT06073847
Trial Title: A Post-Marketing Surveillance Study to Assess the Safety of Fedratinib in Korean Patients With Myelofibrosis […] The purpose of this study is to assess the real-world safety of fedratinib for the treatment of adult participants with primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), or post essential thrombocythemia myelofibrosis (post-ET MF) who were previously treated with ruxolitinib. […] Adult participants with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis who were previously treated with ruxolitinib.
#3 Ten years of treatment with ruxolitinib for myelofibrosis: a review of safety | Journal of Hematology & Oncology | Full Text
https://jhoonline.biomedcentral.com/articles/10.1186/s13045-023-01471-z
Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by bone marrow fibrosis, anemia, extramedullary hematopoiesis, and splenomegaly. Patients with MF are at risk for reduced survival versus the general population and often experience burdensome signs and symptoms that reduce quality of life. […] Ruxolitinib is an oral Janus kinase (JAK) 1/JAK2 inhibitor first approved by the US Food and Drug Administration (FDA) in 2011 for the treatment of adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera (PV) MF, and post-essential thrombocythemia MF. […] Consequently, more than a decade since its initial approval, ruxolitinib continues to be the standard of care in patients with higher-risk MF. […] The approval of ruxolitinib was based on efficacy and safety demonstrated in the randomized, placebo-controlled phase 3 COMFORT trials.
#3 Epidemiology and disease characteristics of myelofibrosis: a comparative analysis between Italy and global perspectives
https://pmc.ncbi.nlm.nih.gov/articles/PMC11303153/
The ERNEST registry (European Registry for Myeloproliferative Neoplasms; towards a better understanding of Epidemiology, Survival and Treatment) included the data of 1209 patients with MF (median age 66 years), 61% with PMF, 20% with post-ET MF, and 19% with post-PV MF. […] A further study of the ERNEST registry involving 1010 patients with MF investigated the impact of ruxolitinib on overall survival using prospectively collected data. Age, male sex, high-risk category based on the Dynamic International Prognostic Scoring System (DIPSS) were identified as factors that negatively affected overall survival.
#3 Orphanet: Primary myelofibrosis
https://www.orpha.net/en/disease/detail/824
The annual incidence of primary myelofibrosis (PMF) is approximately 1 case per 100,000 individuals, although an increased prevalence is noted in Ashkenazi Jews. […] Age at diagnosis is usually in adulthood, around the sixth decade of life. […] The pathology is not inherited although it is linked with important gene mutations which occur at any time in life. […] Epidemiological studies have indicated the presence of common susceptibility alleles.