Mielofibroza
Objawy

Mielofibroza (MF) to rzadki nowotwór hematologiczny charakteryzujący się włóknieniem szpiku kostnego, prowadzącym do zaburzeń hematopoezy i objawów takich jak anemia, leukopenia, małopłytkowość oraz splenomegalia, obecna u niemal wszystkich pacjentów w momencie diagnozy. Objawy kliniczne obejmują zmęczenie, duszność, ból brzucha związany z powiększoną śledzioną, nocne poty, utratę masy ciała oraz świąd skóry. Przebieg choroby jest zmienny, z możliwością stabilnego przebiegu lub progresji do ciężkiej anemii, powiększenia narządów, infekcji i transformacji w ostrą białaczkę szpikową (AML) u 10-20% pacjentów w ciągu 10 lat. Średni czas przeżycia wynosi około 6 lat, z podziałem na grupy ryzyka prognostycznego, gdzie niskie ryzyko wiąże się ze średnim przeżyciem około 11 lat, a wysokie ryzyko około 2 lat. Kluczowe czynniki prognostyczne to wiek, obecność objawów ogólnoustrojowych, stopień anemii, liczba blastów, leukocytoza lub leukopenia, małopłytkowość oraz mutacje genetyczne (JAK2, CALR, MPL).

Objawy mielofibrozyi jej przebieg

Mielofibroza (MF) to rzadki nowotwór krwi charakteryzujący się nieprawidłowym rozrostem włókien oraz blizn w szpiku kostnym, co prowadzi do zaburzeń produkcji komórek krwi. Ten proces wpływa na zdolność szpiku do wytwarzania prawidłowych komórek krwi, prowadząc do wielu charakterystycznych objawów. Przebieg choroby jest zazwyczaj powolny, a niektórzy pacjenci mogą pozostawać bezobjawowi przez lata123.

Objawy we wczesnym stadium

Około jedna trzecia pacjentów z mielofibrozą nie wykazuje żadnych objawów we wczesnych stadiach choroby12. U wielu osób choroba jest wykrywana przypadkowo podczas rutynowych badań krwi12. Warto podkreślić, że nie zawsze istnieje bezpośredni związek między nasileniem objawów a stadium choroby – zależy to od indywidualnych czynników, poziomu komórek krwi oraz zmian zachodzących w szpiku kostnym1.

Typowe objawy mielofibrozyi

Wraz z postępem choroby, pacjenci mogą doświadczać następujących objawów123:

  • Uczucie zmęczenia, osłabienia lub duszności (wynikające z anemii)
  • Ból lub uczucie pełności poniżej żeber po lewej stronie (spowodowane powiększoną śledzioną)
  • Łatwe siniaczenie się
  • Zwiększona skłonność do krwawień
  • Nadmierne pocenie się podczas snu (nocne poty)
  • Gorączka
  • Ból kości i stawów
  • Uczucie pełności po zjedzeniu niewielkiej ilości pożywienia
  • Utrata wagi i spadek apetytu
  • Świąd skóry (pruritis), często nasilający się po ciepłej kąpieli lub prysznicu
  • Częste infekcje
  • Blada skóra

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Objawy związane z nieprawidłowościami w morfologii krwi

Objawy mielofibrozyi są często związane z zaburzeniami w produkcji komórek krwi12:

  • Anemia (niedobór czerwonych krwinek) – powoduje zmęczenie, osłabienie, duszność, zawroty głowy
  • Leukopenia (niedobór białych krwinek) – prowadzi do częstszych i poważniejszych infekcji, takich jak przeziębienia, grypa, zapalenie płuc czy zakażenia układu moczowego
  • Małopłytkowość (trombocytopenia, niedobór płytek krwi) – skutkuje łatwym siniaczeniem się, nietypowymi krwawieniami, takimi jak krwawienia z nosa czy nadmierne krwawienia miesiączkowe

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Powiększona śledziona i wątroba

Praktycznie wszyscy pacjenci z pierwotną mielofibrozą mają powiększoną śledzionę (splenomegalia) w momencie diagnozy1. W około jednej trzeciej przypadków śledziona jest znacznie powiększona. Częste objawy związane z powiększoną śledzioną obejmują123:

  • Uczucie dyskomfortu, bólu lub pełności w górnej lewej części brzucha
  • Nadciśnienie wrotne (zwiększone ciśnienie krwi w żyle wrotnej)
  • Krwawienie z żylaków przełyku
  • Uczucie szybkiego nasycenia podczas jedzenia

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Powiększenie wątroby (hepatomegalia) występuje u około 60-70% pacjentów z mielofibrozą1 i może przyczyniać się do podobnych objawów jak powiększenie śledziony.

Progresja choroby

Przebieg naturalny mielofibrozyi może znacznie różnić się między pacjentami1. U niektórych osób choroba pozostaje stabilna przez długi czas, umożliwiając prowadzenie normalnego życia z minimalnymi przerwami spowodowanymi chorobą lub jej leczeniem. U innych mielofibroza postępuje szybciej, powodując objawy wymagające leczenia12.

Wraz z postępem choroby mogą pojawić się lub nasilić następujące objawy123:

  • Ciężka anemia – rozległe bliznowacenie w szpiku kostnym prowadzi do ciężkiej anemii, powodującej osłabienie, zmęczenie i duszność
  • Bardziej powiększona śledziona i wątroba – organy te powiększają się, próbując przejąć część funkcji produkcji komórek krwi. Może to powodować ból brzucha i nadciśnienie wrotne
  • Utrata masy ciała – zaawansowana mielofibroza może powodować niezamierzoną utratę wagi
  • Ból kości – postępujące bliznowacenie szpiku kostnego może prowadzić do bólu kości, wpływając na mobilność i jakość życia
  • Częstsze infekcje bakteryjne lub grzybicze – wskazujące na progresję choroby

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Transformacja do ostrej białaczki szpikowej

U około 10-20% pacjentów z mielofibrozą choroba przekształca się w ostrą białaczkę szpikową (acute myeloid leukemia, AML) w ciągu 10 lat od diagnozy123. Jest to poważna postać nowotworu krwi i szpiku kostnego, która postępuje szybko i może być trudna do leczenia12.

Objawy wskazujące na transformację do AML mogą obejmować1:

  • Bóle głowy
  • Łatwe siniaczenie się
  • Nadmierne krwawienia
  • Nasilona anemia (słabość i zmęczenie)
  • Częste infekcje
  • Zwiększony odsetek komórek blastycznych (co najmniej 20%) w szpiku kostnym i krwi

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Monitorowanie i ocena progresji choroby

Regularne monitorowanie przebiegu mielofibrozyi jest kluczowe dla optymalnego dostosowania strategii leczenia i skutecznego zarządzania powikłaniami12. Oznaki wskazujące na progresję choroby obejmują12:

  • Postępujące powiększenie śledziony (splenomegalia) pomimo leczenia
  • Wzrost liczby krążących blastów we krwi obwodowej
  • Pogłębiająca się anemia i małopłytkowość
  • Rozwój dodatkowych nieprawidłowości cytogenetycznych
  • Pogorszenie stanu funkcjonalnego pacjenta
  • Zdarzenia zakrzepowe, takie jak zakrzepica żył głębokich czy zatorowość płucna

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Ważne jest regularne śledzenie objawów i informowanie o wszelkich zmianach swojego lekarza prowadzącego12. Nawet jeśli objawy wydają się niezwiązane z mielofibrozą, mogą dostarczyć cennych informacji na temat progresji choroby1.

Czynniki prognostyczne i rokowanie

Rokowanie w mielofibrozyi jest zróżnicowane i zależy od wielu czynników12. Średni czas przeżycia wynosi około 6 lat od diagnozy, ale może wahać się od 2 do 15 lat, a w niektórych przypadkach nawet dłużej12.

Do czynników prognostycznych wpływających na przebieg choroby należą123:

  • Wiek (powyżej 65 roku życia wiąże się z gorszym rokowaniem)
  • Objawy ogólnoustrojowe (gorączka, nocne poty, utrata masy ciała)
  • Anemia
  • Poziom blastów we krwi obwodowej
  • Liczba leukocytów (leukocytoza lub leukopenia)
  • Małopłytkowość
  • Obecność specyficznych mutacji genetycznych (JAK2, CALR, MPL)
  • Przypadki potrójnie negatywne (bez mutacji JAK2, CALR i MPL) związane są z gorszym rokowaniem

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W zależności od czynników ryzyka, pacjentów można podzielić na cztery grupy prognostyczne1:

  • Niskie ryzyko – średni czas przeżycia około 11 lat
  • Pośrednie 1 – średni czas przeżycia około 8 lat
  • Pośrednie 2 – średni czas przeżycia około 4 lat
  • Wysokie ryzyko – średni czas przeżycia około 2 lat

1

Powikłania mielofibrozyi

Wraz z postępem mielofibrozyi mogą wystąpić różne powikłania, które znacząco wpływają na jakość życia pacjentów12:

Nadciśnienie wrotne i powikłania żylakowe

Nadciśnienie wrotne występuje u około 7% pacjentów z pierwotną mielofibrozą i może być związane ze zwiększonym przepływem wrotnym wynikającym z znacznej splenomegalii oraz z wewnątrzwątrobowej obstrukcji wynikającej z zakrzepowego zamknięcia małych żył wrotnych1. Może prowadzić do12:

  • Wodobrzusza
  • Żylaków przełyku i żołądka
  • Krwawień z przewodu pokarmowego
  • Encefalopatii wątrobowej

1

Pozaszpikowa hematopoeza

W miarę postępu choroby, produkcja komórek krwi może przenieść się do miejsc poza szpikiem kostnym (hematopoeza pozaszpikowa), szczególnie do śledziony i wątroby1. Może to prowadzić do różnych objawów w zależności od zajętego organu1:

  • Krwawienia z przewodu pokarmowego
  • Ucisk rdzenia kręgowego
  • Napady padaczkowe ogniskowe
  • Objawy związane z guzami mózgu
  • Wodobrzusze
  • Krwiomocz
  • Wysięk osierdziowy
  • Wysięk opłucnowy
  • Krwioplucie
  • Niewydolność oddechowa

1

Zaburzenia krzepnięcia i krwawienia

Krwawienia obserwuje się u jednej czwartej pacjentów z pierwotną mielofibrozą i mogą one różnić się pod względem nasilenia – od nieistotnych wybroczyn skórnych do ciężkich, zagrażających życiu krwawień z przewodu pokarmowego1. Przyczyniają się do tego1:

  • Dysfunkcja płytek krwi
  • Nabyte niedobory czynników krzepnięcia
  • Małopłytkowość
  • Rozsiane wykrzepianie wewnątrznaczyniowe (DIC)
  • Żylaki przełyku
  • Choroba wrzodowa żołądka i dwunastnicy

1

Zaburzenia odporności i infekcje

Połowa pacjentów z pierwotną mielofibrozą ma nieprawidłowości odporności humoralnej1. Może to prowadzić do1:

  • Zwiększonej podatności na infekcje, szczególnie zapalenie płuc
  • Obecności różnych autoprzeciwciał i krążących kompleksów immunologicznych
  • Rozwoju amyloidozy

1

Znaczenie monitorowania objawów

Ważne jest dokładne monitorowanie objawów mielofibrozyi, ponieważ ich zmniejszenie jest kluczowym celem leczenia1. Staranne śledzenie objawów może pomóc pacjentowi i lekarzowi lepiej zrozumieć, jak zarządzać i modyfikować opiekę w czasie12.

Regularne monitorowanie objawów dostarcza cennych informacji o1:

  • Potencjalnej progresji choroby
  • Skuteczności leczenia
  • Potrzebie dostosowania dawki leków
  • Ogólnym stanie zdrowia

1

Szybkie zgłoszenie się do lekarza w przypadku pojawienia się lub nasilenia objawów mielofibrozyi jest niezwykle ważne1. Wcześnie rozpoczęte leczenie jest kluczowe dla osiągnięcia najlepszych możliwych wyników. Opóźnienie leczenia może pozwolić na progresję mielofibrozyi, utrudniając osiągnięcie remisji1.

Wpływ na jakość życia

Objawy mielofibrozyi mogą mieć znaczący wpływ na jakość życia pacjentów12. Według badania MPN Landmark Survey, 81% pacjentów z mielofibrozą zgłaszało, że ich objawy zmniejszyły jakość ich życia1.

Obciążenie objawami może być znaczące nawet u pacjentów z niskim ryzykiem prognostycznym i z najniższym nasileniem objawów1. Dlatego ważne jest, aby leczenie było ukierunkowane nie tylko na spowolnienie progresji choroby, ale także na łagodzenie objawów i poprawę jakości życia1.

Podsumowanie przebiegu mielofibrozyi

Mielofibroza to postępująca choroba, która może rozwijać się w różnym tempie u poszczególnych pacjentów12. Podczas gdy niektórzy mogą pozostawać bezobjawowi przez lata, u innych choroba może szybko postępować, prowadząc do poważnych powikłań, w tym transformacji do ostrej białaczki szpikowej12.

Kluczowe jest ścisłe monitorowanie objawów i regularne wizyty lekarskie, aby ocenić progresję choroby i dostosować leczenie. Wczesne rozpoznanie pogorszenia stanu zdrowia i odpowiednie interwencje mogą pomóc w kontrolowaniu objawów i potencjalnie poprawić rokowanie12.

Wielodyscyplinarne podejście do leczenia, uwzględniające zarówno fizyczne, jak i psychologiczne aspekty choroby, jest niezbędne do skutecznego zarządzania mielofibrozą i utrzymania jak najlepszej jakości życia pacjentów1.

Kolejne rozdziały

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  1. 12.04.2026
  2. www.leksykon.com.pl

Materiały źródłowe

  • #1 Myelofibrosis – Symptoms and causes – Mayo Clinic
    https://www.mayoclinic.org/diseases-conditions/myelofibrosis/symptoms-causes/syc-20355057
    Myelofibrosis signs and symptoms may include: […] Feeling tired, weak or short of breath. […] Pain or fullness below the ribs on the left side. […] Easy bruising. […] Easy bleeding. […] Sweating a lot while sleeping, so that you wake feeling covered in sweat. […] Fever. […] Bone pain. […] Feeling full after eating a small amount of food. […] Some people with myelofibrosis have no symptoms and might not need treatment right away. Others with more-serious forms of the disease might need treatment. Treatment for myelofibrosis typically focuses on relieving symptoms. […] The extra blood cells that don’t work right and the bone marrow scarring both can cause too few healthy blood cells in the body. This leads to the symptoms of myelofibrosis. For example, feeling weak and tired can happen if there are too few red blood cells to carry oxygen in the blood.
  • #1 Myelofibrosis: Symptoms, Types, Prognosis & Treatment
    https://my.clevelandclinic.org/health/diseases/15672-myelofibrosis
    Myelofibrosis progresses slowly, so you may not have symptoms for many years. About one-third of people dont show symptoms during the disorders early stages. […] When they arise, the most common symptoms of myelofibrosis are severe fatigue (resulting from anemia) and an enlarged spleen. Symptoms may include: Fatigue. Fever. Itching. Pale skin. Weight loss. Night sweats. Bone or joint pain. Frequent infections. Enlarged spleen or liver. Unexplained blood clots. Abnormal bleeding or bruising. Enlarged veins in your stomach and esophagus. (These veins may rupture and cause bleeding.) […] In about 12% of all cases, primary myelofibrosis progresses to acute myeloid leukemia, a very aggressive form of blood cancer. […] Myelofibrosis is aggressive cancer with a median survival rate of six years. A median is a midpoint, which means that some people live less than six years, and about the same number of people live longer than six years.
  • #1 Myelofibrosis – MPN Research Foundation
    https://mpnresearchfoundation.org/primary-myelofibrosis-pmf/
    Myelofibrosis symptoms are often caused by an enlarged spleen and/or insufficient numbers of normal blood cells and chronic inflammation. […] Common MF symptoms and signs may include: Tiredness, weakness, or shortness of breath with mild exertion; Fullness, discomfort or pain in the left upper area of the abdomen; Fever; Night sweats; Weight loss or malnutrition; Bone pain; Itching (pruritus); Easy bleeding or bruising; Susceptibility to infection; Joint pain or gout; Abdominal distension/fluid retention (if portal hypertension or increased blood pressure within the portal vein is present); Compromised liver function; Abnormal growth of blood forming cells outside of the bone marrow. […] Routine medical examinations including complete blood counts (CBCs) are important for diagnosing MF and other MPNs, since some MF patients exhibit no symptoms (especially during the early course of the disease).
  • #1 Myelofibrosis (MF) symptoms | Blood Cancer UK
    https://bloodcancer.org.uk/understanding-blood-cancer/myelofibrosis/myelofibrosis-symptoms/
    The symptoms of myelofibrosis vary depending on what is happening in the bone marrow, the spongy material inside our larger bones where blood cells are made. […] Some people dont have any symptoms at all when they are diagnosed with myelofibrosis (MF). Others will have more obvious symptoms. […] Its important to realise that there isnt always a direct link between the level of symptoms and the stage of the disease. It depends on you, your blood counts, and whats happening in your bone marrow. […] Common symptoms include: extreme tiredness (fatigue), losing weight without trying, drenching night sweats, unexplained fever, itching, sometimes particularly after a hot bath or shower, general weakness, feeling full up very quickly when trying to eat. […] Low levels of blood cells can lead to a range of symptoms: Too few red blood cells can make you feel tired, breathless and dizzy. The medical name for this is anaemia. Too few white blood cells can mean you get more infections like colds and flu, chest and urinary tract infections. These may last longer and be more serious than usual. Too few platelets can lead to bruising and unusual bleeding such as nose bleeds and heavy periods. […] A few people may have bone pain. This can happen at any stage but is more likely at a later stage of myelofibrosis (MF) when there is more scarring (fibrosis) and more inflammation in the bone marrow.
  • #1 Primary myelofibrosis – Leukaemia Foundation
    https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/myeloproliferative-neoplasms/primary-myelofibrosis/
    Primary myelofibrosis (also called chronic idiopathic myelofibrosis, agnogenic myeloid metaplasia) is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. […] Symptoms of anaemia are common and include unexplained tiredness, weakness, shortness of breath and palpitations. Other nonspecific symptoms include fever, unintended weight loss, pruritus (generalised itching) and excess sweating, especially at night. […] Virtually all patients with primary myelofibrosis have an enlarged spleen (splenomegaly) when they are first diagnosed. In around a third of cases the spleen is very enlarged. Common symptoms include feelings of discomfort, pain or fullness in the upper left-side of the abdomen.
  • #1 Myelofibrosis – Blood Disorders – Merck Manual Consumer Version
    https://www.merckmanuals.com/home/blood-disorders/myeloproliferative-disorders/myelofibrosis
    The liver and spleen enlarge as they try to take over some of the job of making blood cells, but blood cell production is ineffective in these organs and instead just contributes to the spleen enlargement. Enlargement of the liver and spleen may cause pain in the abdomen and may lead to abnormally high blood pressure in certain veins, such as a vein supplying blood to the liver (portal hypertension) and bleeding from varicose veins in the esophagus (esophageal varices). […] Malignant myelofibrosis (sometimes called acute myelofibrosis) is a rare type of myelofibrosis in which levels of red blood cells, white blood cells, and platelets are all decreased. The number of certain immature white blood cells (blast cells) in the bone marrow is increased. Malignant myelofibrosis is considered to be a type of acute leukemia.
  • #1 Primary Myelofibrosis Clinical Presentation: History, Physical Examination
    https://emedicine.medscape.com/article/197954-clinical
    Portal hypertension may occur as a result of markedly increased splenoportal blood flow and decreased hepatic vascular compliance. Ascites, esophageal and gastric varices, GI tract bleeding, and hepatic encephalopathy may occur. Hepatic or portal vein thrombosis may also arise as complications. […] Patients with primary myelofibrosis develop osteosclerosis. This may cause severe joint and bone pain. […] One half of patients with primary myelofibrosis have abnormalities of humoral immunity. A variety of autoantibodies and circulating immune complexes may be detected, and amyloidosis may develop. Infections, commonly pneumonia, may occur as a result of immune deficiency. […] Splenomegaly is the most common finding in patients with primary myelofibrosis, and it is present in approximately 90% of patients. Spleen size may vary from barely palpable to massive (observed in 35% of patients). […] Hepatomegaly is found in 60-70% of patients with primary myelofibrosis, and pallor is observed in 60% of patients. Other physical findings include petechiae and ecchymosis (20%), lymphadenopathy (10-20%), signs of portal hypertension (10-18%), and gout (6%).
  • #1 Primary myelofibrosis – Leukaemia Foundation
    https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/myeloproliferative-neoplasms/primary-myelofibrosis/
    The natural course of the disease can vary considerably between individuals. In some people their disease remains stable for long periods and they are free to live a normal life with minimal interruptions from their disease or its treatment. For others, myelofibrosis progresses more quickly and people require treatment to help relieve symptoms of their disease. Transformation to a type of leukaemia called acute myeloid leukaemia occurs in between 10 and 20 per cent of cases.
  • #1 Myelofibrosis: How It Progresses
    https://www.webmd.com/cancer/lymphoma/stages-myelofibrosis
    As the disease gets worse or progresses, you may have new symptoms. These could include: […] Myelofibrosis is not curable, but different things can affect how long you live and your quality of life. Some patients live for 6 years or longer after their diagnosis. Speak with your doctor to see which treatments would be best for you.
  • #1 Myelofibrosis prognosis: Life expectancy, treatment options & more
    https://www.medicalnewstoday.com/articles/myelofibrosis-prognosis
    The outlook for someone with myelofibrosis depends on factors such as age, blood cell counts, and symptoms. For some people, the condition progresses more rapidly than others. […] Because it is a progressive disease, it worsens over time but affects each person differently. While some people have severe symptoms that progress quickly, others may live without symptoms for years. […] MF may cause no symptoms for years. However, late stage MF can present with symptoms that include: […] Severe anemia: The extensive scarring in the bone marrow can lead to severe anemia. This can cause weakness, fatigue, and shortness of breath. […] Enlarged spleen and liver: These organs enlarge as they attempt to take over some of the job of making blood cells. This can cause abdominal pain and high blood pressure in certain veins, which doctors call portal hypertension.
  • #1 Myelofibrosis: Transformation to acute myeloid leukemia
    https://www.medicalnewstoday.com/articles/myelofibrosis-transformation-to-acute-myeloid-leukemia
    Myelofibrosis belongs to a group of rare diseases called myeloproliferative neoplasms, which are types of blood cancer. These neoplasms can progress to acute myeloid leukemia (AML). […] Myelofibrosis, also known as primary myelofibrosis, is the most common type of myeloproliferative neoplasm to progress to AML. […] According to a 2021 study, AML will occur in about 5-10% of people with a myeloproliferative neoplasm. This progression is most common in people with myelofibrosis. Within a 10-year period, approximately 20% of people with myelofibrosis will develop AML. […] The NCI explains that there is no general staging system for the progression of myeloproliferative neoplasms, as experts do not know exactly how myelofibrosis progresses into AML. […] Most myeloproliferative neoplasms progress to AML gradually, possibly over the course of several years. However, the progression can be much quicker for some people. One sign of progression is a higher percentage of blast cells, meaning at least 20%, in the bone marrow and blood. Frequent medical visits are necessary to monitor the percentage of blast cells.
  • #1 Myelofibrosis – MPN Research Foundation
    https://mpnresearchfoundation.org/primary-myelofibrosis-pmf/
    While some individuals live for many years without developing major symptoms, others find that the disease progresses more quickly. […] For most patients, an MF prognosis requires the management of several symptoms and signs, including: Anemia (not enough red blood cells to carry oxygen); Splenomegaly (enlarged spleen); Extramedullary hematopoiesis (production of blood cells in organs outside the bone marrow, such as the spleen and liver); Constitutional and systemic (whole body) symptoms (e.g., fatigue, night sweats, weight loss, pruritus, fever, bone and joint pain). […] For a small number of patients, MF can transform to acute myeloid leukemia (AML), a serious blood and bone marrow cancer. When AML does arise from MF, it progresses quickly and can be difficult to treat.
  • #1 Myelofibrosis: Transformation to acute myeloid leukemia
    https://www.medicalnewstoday.com/articles/myelofibrosis-transformation-to-acute-myeloid-leukemia
    If someone has myelofibrosis without AML, their outlook will largely depend on the severity of any symptoms they are experiencing. The symptoms of myelofibrosis include: night sweats, fever, fatigue, unintentional weight loss, fullness or discomfort in the abdomen, quickly feeling full, pain while urinating, blood in the urine, gastrointestinal bleeding, pain in the bones. […] Should myelofibrosis progress to AML, a person may also experience some common AML symptoms, including: headaches, easy bruising, excessive bleeding, anemia, which may involve feelings of weakness and fatigue, frequent infections. […] Myelofibrosis can progress to AML, which is a very severe condition, through a complex process of genetic mutation. Some treatments can reduce the chances of myelofibrosis progressing to AML. […] Several factors will determine the outlook of people with these conditions, including age, symptom severity, and the effectiveness of treatment.
  • #1 Myelofibrosis: Causes and Treatments
    https://www.drkarunhematology.com/blog/myelofibrosis-causes-and-treatments/
    Monitoring the progression of myelofibrosis is essential for optimizing treatment strategies and managing complications effectively. Certain signs that myelofibrosis is progressing, warranting close medical supervision and intervention, are: […] Progression to acute myeloid leukaemia (AML) or blast-phase myelofibrosis is a sign of advanced disease and carries a poor prognosis. […] Progressive enlargement of the spleen (splenomegaly) despite treatment may indicate worsening fibrosis and disease progression. […] The development of additional cytogenetic abnormalities, such as complex karyotype or high-risk mutations, may signify disease progression and treatment resistance. […] Deterioration in functional status may indicate advanced disease and poor prognosis. This deterioration is characterized by decreased performance status or worsening Eastern Cooperative Oncology Group (ECOG) score. […] Progressive thrombocytopenia and thrombotic events, like deep vein thrombosis (DVT) or pulmonary embolism (PE), may occur as the disease advances.
  • #1 Understanding Drivers of Progression in Myelofibrosis
    https://www.onclive.com/view/understanding-drivers-of-progression-in-myelofibrosis
    Some of the molecular markers have been shown to be prognostic. […] In addition, the clinical characteristics that demarcate a patient who is likely to have more aggressive or more advanced disease include patients with rapidly enlarging spleen size, patients who have increased circulating blasts. […] The progression of the disease is a problem. About 20% of the patients with myelofibrosis do change to acute myeloid leukemia. […] Apart from the transformation from acute myeloid leukemia, which happens in 20% of patients, other patients die from the myelofibrosis itself. […] The cause of that may be liver failure because of liver infiltration, pulmonary hypertension because of pulmonary infiltration, or cardiac failure because cardiac output goes up, bleeding from the GI tract in relation to portal hypertension, cachexia, bleeding, thrombosis, infections, and multiple different factors.
  • #1 Myelofibrosis (MF) Signs and Symptoms
    https://www.voicesofmpn.com/myelofibrosis-symptoms
    Myelofibrosis (MF) can cause a variety of symptoms, and each persons experience with MF symptoms can change over time. […] Because MF is a progressive disease, its important to keep an eye out for any new or worsening symptoms that could indicate the disease is getting worse. […] In addition to the symptoms caused by an enlarged spleen, MF can affect the body in other ways. […] According to the MPN Landmark Survey, * 81% of patients with MF reported that their symptoms reduced their quality of life. […] Because MF is a progressive condition, it has the potential to change over time. […] This is why its important to take an active role in monitoring your condition over time by tracking your MF on a regular basis and sharing any changes with your Healthcare Professional. […] Regular symptom tracking provides a number of valuable insights.
  • #1 New Markers of Disease Progression in Myelofibrosis
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8582535/
    The prognosis of PMF is variable and the median survival is estimated around 6 years from diagnosis, ranging from 2 to 15 years, and in some cases even more. […] The prognosis of PMF is variable and the median survival is estimated around 6 years from diagnosis, ranging from 2 to 15 years, and in some cases even more. […] Elevated concentrations of sIL-2R have been observed in cancers and autoimmune, inflammatory, and infectious diseases. […] In MPNs, sIL-2R was shown to correlate with disease progression, poorer survival, and blast transformation-free survival. […] The data evidenced no correlation between eNAMPT levels and disease duration, spleen size, circulating CD34+ cells, and cholesterol level in serum; moreover, there was no difference between eNAMPT concentration and different genotypes.
  • #1 Primary Myelofibrosis Diagnosis & Disease Information
    https://www.cancertherapyadvisor.com/ddi/primary-myelofibrosis/
    The prognosis of primary myelofibrosis varies substantially. One study that included 1282 patients with primary myelofibrosis documented a median overall survival rate of 4.4 years. Age, illness severity, and thrombotic history are important prognostic factors. Advanced age, especially older than 65 years, carries a worse prognosis due to decreased tolerance of treatment and higher risks of comorbidities. More severe illness as reflected by extensive bone marrow fibrosis, a greatly enlarged spleen, and extensive symptoms are associated with a worse prognosis. […] Primary myelofibrosis can result in a range of complications secondary to its effects on bone marrow and systemic involvement. These include chronic anemia, which can lead to profound fatigue, weakness, and diminished quality of life; splenomegaly and hepatomegaly, which can cause abdominal discomfort, early satiety, and pain; thrombocytopenia, which increases the risk of bleeding and bruising; increased risk of opportunistic infections, particularly in patients receiving immunosuppressive medications; and cardiovascular events such as thrombosis and hypertension. […] Primary myelofibrosis can transform into acute myeloid leukemia, especially in late stages of the disease. This transformation, which is called blast phase myelofibrosis, affects 10% to 20% of patients, requires aggressive treatment, and is associated with a poor prognosis.
  • #1 Understanding Drivers of Progression in Myelofibrosis
    https://www.onclive.com/view/understanding-drivers-of-progression-in-myelofibrosis
    The primary reason for assessment of risk of dying is to see if transplant is a possibility and to refer patients for the transplant. […] We can divide patients into four different groups: low-risk, have none of the five; intermediate 1, one of the five; intermediate 2: risk of dying, two out of the five; and then high risk of dying earlier on if they have three, four, or five of these factors. […] Now, the years of life that we are talking about range from 11 years for low-risk, 8 years for an intermediate 12, 4 years for intermediate 2, and 2 years for high-risk.
  • #1 Primary Myelofibrosis: Practice Essentials, Pathophysiology, Etiology
    https://emedicine.medscape.com/article/197954-overview
    Primary myelofibrosis is categorized as a chronic myeloproliferative disorder, along with chronic myelogenous leukemia (CML), polycythemia vera, and essential thrombocytosis. […] The disorder is characterized by the following: Anemia, Bone marrow fibrosis (myelofibrosis), Extramedullary hematopoiesis, Leukoerythroblastosis and teardrop-shaped red blood cells (RBCs) in peripheral blood, Hepatosplenomegaly. […] Portal hypertension occurs in approximately 7% of patients with primary myelofibrosis and may be related to increased portal flow resulting from marked splenomegaly and to intrahepatic obstruction resulting from thrombotic obliteration of small portal veins. […] Splenic infarction may occur and results in an acute or subacute onset of severe pain in the left upper quadrant that may be associated with nausea, fever, and referred left shoulder discomfort.
  • #1 Primary myelofibrosis – Wikipedia
    https://en.wikipedia.org/wiki/Primary_myelofibrosis
    In primary myelofibrosis, progressive scarring, or fibrosis, of the bone marrow occurs, for the reasons outlined above. The result is extramedullary hematopoiesis, i.e. blood cell formation occurring in sites other than the bone marrow, as the hemopoietic cells are forced to migrate to other areas, particularly the liver and spleen. This causes an enlargement of these organs. In the liver, the abnormal size is called hepatomegaly. Enlargement of the spleen is called splenomegaly, which also contributes to causing pancytopenia, particularly thrombocytopenia and anemia. Another complication of extramedullary hematopoiesis is poikilocytosis, or the presence of abnormally shaped red blood cells. […] Epidemiologically, the disorder usually develops slowly and is mainly observed in people over the age of 50.
  • #1 Primary Myelofibrosis Clinical Presentation: History, Physical Examination
    https://emedicine.medscape.com/article/197954-clinical
    A hypermetabolic state occurs and can result in weight loss, night sweats, and low-grade fever. Gout and urate kidney stones may develop. […] Bleeding is observed in one fourth of patients with primary myelofibrosis and varies in severity from insignificant cutaneous petechiae to severe, life-threatening gastrointestinal (GI) tract bleeding. Platelet dysfunction, acquired factor V deficiency, thrombocytopenia, disseminated intravascular coagulation (DIC), esophageal varices, and peptic ulcer disease may occur, contributing to bleeding. […] Extramedullary hematopoiesis may cause symptoms, depending on the organ or site of involvement. The condition may result in GI tract hemorrhage, spinal cord compression, focal seizures, symptoms related to brain tumors, ascites, hematuria, pericardial effusion, pleural effusion, hemoptysis, and respiratory failure.
  • #1 Myelofibrosis: Causes and Treatments
    https://www.drkarunhematology.com/blog/myelofibrosis-causes-and-treatments/
    Recognizing the myelofibrosis symptoms is crucial for early diagnosis and intervention. While the presentation of symptoms may vary among individuals, certain common signs indicate the presence of this condition. […] Fatigue, weakness, and shortness of breath are common symptoms associated with anemia in myelofibrosis. […] Enlarged spleen (splenomegaly), abdominal pain or discomfort, early satiety, and portal hypertension-related complications such as ascites and varices. […] Fever, night sweats, and unintended weight loss may occur due to the inflammatory nature of myelofibrosis. […] Bone pain, especially in long bones, ribs, and spine, may result from fibrotic tissue expanding in the bone marrow. […] Easy bruising, bleeding gums, petechiae, and prolonged bleeding from minor injuries may occur due to decreased platelet counts.
  • #1 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/signs-and-symptoms
    https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/signs-and-symptoms
    MF usually develops slowly. MF often does not cause early symptoms and may be found during a routine blood test. When fibrosis develops in the bone marrow, the bone marrow is unable to produce enough normal blood cells. The lack of blood cells causes many of the signs and symptoms of MF. These include: […] When MF causes symptoms, they can be troublesome. Reducing symptoms is a key goal of treatment. Therefore, it is important to take an active role in monitoring your MF symptoms. Careful tracking of your symptoms can help you and your doctor better understand how to manage and modify your care over time.
  • #1 6 Signs Your Myelofibrosis May Be Progressing | myMPNteam
    https://www.mympnteam.com/resources/signs-your-myelofibrosis-may-be-progressing
    Symptoms of thrombocytopenia include: Bruising or pooling of blood underneath your skin, Frequent nosebleeds, Bleeding or swollen gums, Blood in your urine or stool, Heavy menstrual periods, Petechiae (tiny, rashlike red or purple dots on your lower legs). […] When living with PMF, this symptom is cause for concern. The likely culprit is splenomegaly an enlarged spleen. […] Splenomegaly is most common in PMF, but it can also occur in people with essential thrombocythemia and polycythemia vera, other types of MPNs. […] Excessive night sweats are another sign of MPNs, and theyre common in people with PMF. This disease causes body-wide inflammation that leads to fevers and night sweats. […] Frequent bacterial or fungal infections indicate that your PMF is progressing. […] PMF affects your bone marrow, so it makes sense that you may experience bone or joint pain as your disease progresses. […] If youve started experiencing one or more of these PMF symptoms, its time to check with your doctor. They can run extra tests and determine if your disease is progressing. Its vital to start treatment as soon as possible if your PMF is transforming into acute myeloid leukemia.
  • #1 What are the Signs and Symptoms of Myelofibrosis? – HealthTree for Myelofibrosis
    https://healthtree.org/myelofibrosis/community/what-are-the-signs-and-symptoms-of-myelofibrosis
    Fever: This can be a sign of infection, which can occur because the disease weakens the immune system […] Bone pain: This is a less common symptom, but some people with myelofibrosis experience pain in their bones or joints […] Night sweats: These can be a result of the body trying to fight off an infection or due to hormonal imbalances caused by the disease […] Enlarged spleen (splenomegaly): When the bone marrow can’t make enough normal cells, the spleen helps out by starting to produce them, causing the spleen to grow larger. This can result in pain or a feeling of fullness below the ribs on the left side […] Seeking medical attention promptly if you are experiencing symptoms related to your myelofibrosis is of utmost importance. Prompt initiation of treatment is essential to achieve the best possible outcomes. Delaying treatment can allow the myelofibrosis to progress, making it more challenging to achieve remission. […] Medical attention can help manage and alleviate your symptoms and side effects and allow doctors to tailor a myelofibrosis treatment plan that allows you to live as comfortably as possible.
  • #1 Myelofibrosis (MF) Symptoms | MPN Connect
    https://www.mpnconnect.com/myelofibrosis-mf/symptoms
    In myelofibrosis (MF), symptoms can gradually develop over time and thus may not be fully appreciated by patients as they have sometimes compensated for them. […] MF carries a symptom burden that affects the majority of patients. […] The MPN Landmark Survey was a large-scale survey of 813 patients with myeloproliferative neoplasms (MPNs) (MF, n = 207) and assessed perceptions of overall disease burden, quality of life (QOL), activities of daily living, and work productivity. […] 81% of patients reported reduced QOL related to their condition; this was true even for patients with low prognostic risk scores and with their lowest symptom severity. […] Common symptoms and clinical manifestations include: Fatigue, Fever, Weight loss, Anemia, Leukoerythroblastosis, Pruritus, Night Sweats, Splenomegaly, Increased or decreased white blood cell levels, Bone marrow fibrosis. […] Other possible clinical presentations of MF are: Hepatomegaly, Lymphadenopathy, Pleural effusion, Osteosclerosis, Periostitis, Portal hypertension, Ascites, Nerve or spinal cord compression, Hypertrophic osteoarthropathy, Megakaryocytic dysplasia.
  • #1 Myelofibrosis – Diagnosis and treatment – Mayo Clinic
    https://www.mayoclinic.org/diseases-conditions/myelofibrosis/diagnosis-treatment/drc-20355062
    Myelofibrosis diagnosis often starts with a physical exam. […] A healthcare professional may take a sample of your bone marrow for testing. […] In people with myelofibrosis, this test might find signs of scarring in the bone marrow. […] The goal of treatment for most people with myelofibrosis is to provide relief from symptoms of the disease. […] Myelofibrosis treatment might not be needed right away if you don’t have any symptoms. […] If myelofibrosis is causing severe anemia, your healthcare professional may recommend treatments such as blood transfusions. […] If an enlarged spleen is causing complications, your healthcare professional may recommend treatment. […] A bone marrow transplant, also called a bone marrow stem cell transplant, involves putting healthy bone marrow stem cells into the body. […] This treatment has the potential to cure myelofibrosis. […] Palliative care is a special type of healthcare that helps people with serious illnesses feel better. […] When palliative care is used along with all of the other treatments, people with cancer may feel better and live longer.
  • #2 Myelofibrosis: Symptoms, Types, Prognosis & Treatment
    https://my.clevelandclinic.org/health/diseases/15672-myelofibrosis
    Myelofibrosis progresses slowly, so you may not have symptoms for many years. About one-third of people dont show symptoms during the disorders early stages. […] When they arise, the most common symptoms of myelofibrosis are severe fatigue (resulting from anemia) and an enlarged spleen. Symptoms may include: Fatigue. Fever. Itching. Pale skin. Weight loss. Night sweats. Bone or joint pain. Frequent infections. Enlarged spleen or liver. Unexplained blood clots. Abnormal bleeding or bruising. Enlarged veins in your stomach and esophagus. (These veins may rupture and cause bleeding.) […] In about 12% of all cases, primary myelofibrosis progresses to acute myeloid leukemia, a very aggressive form of blood cancer. […] Myelofibrosis is aggressive cancer with a median survival rate of six years. A median is a midpoint, which means that some people live less than six years, and about the same number of people live longer than six years.
  • #2 Myelofibrosis (MF) symptoms | Blood Cancer UK
    https://bloodcancer.org.uk/understanding-blood-cancer/myelofibrosis/myelofibrosis-symptoms/
    The symptoms of myelofibrosis vary depending on what is happening in the bone marrow, the spongy material inside our larger bones where blood cells are made. […] Some people dont have any symptoms at all when they are diagnosed with myelofibrosis (MF). Others will have more obvious symptoms. […] Its important to realise that there isnt always a direct link between the level of symptoms and the stage of the disease. It depends on you, your blood counts, and whats happening in your bone marrow. […] Common symptoms include: extreme tiredness (fatigue), losing weight without trying, drenching night sweats, unexplained fever, itching, sometimes particularly after a hot bath or shower, general weakness, feeling full up very quickly when trying to eat. […] Low levels of blood cells can lead to a range of symptoms: Too few red blood cells can make you feel tired, breathless and dizzy. The medical name for this is anaemia. Too few white blood cells can mean you get more infections like colds and flu, chest and urinary tract infections. These may last longer and be more serious than usual. Too few platelets can lead to bruising and unusual bleeding such as nose bleeds and heavy periods. […] A few people may have bone pain. This can happen at any stage but is more likely at a later stage of myelofibrosis (MF) when there is more scarring (fibrosis) and more inflammation in the bone marrow.
  • #2 What is myelofibrosis? | Cancer Research UK
    https://www.cancerresearchuk.org/about-cancer/myelofibrosis/what-is-myelofibrosis
    Myelofibrosis usually develops slowly and doesnt cause symptoms at first. Some people find out about their myelofibrosis when having a blood test for something else. […] As the condition progresses it causes symptoms. These might include: tiredness and shortness of breath – due to low numbers of red blood cells, bleeding and bruising easily – due to low numbers of platelets, pain and discomfort in the tummy (abdomen) due to enlarged spleen and liver, bone pain, gout – you might have painful, stiff or swollen joints, loss of appetite and weight loss, fever, night sweats, very itchy skin (pruritus). […] Myelofibrosis is rare, so if you have any of these symptoms it is more likely to be something else. But it is still important to contact your doctor to find out what is causing them.
  • #2 6 Signs Your Myelofibrosis May Be Progressing | myMPNteam
    https://www.mympnteam.com/resources/signs-your-myelofibrosis-may-be-progressing
    When youre first diagnosed with primary myelofibrosis (PMF), you may not experience any symptoms. […] In this article, well go over six signs that may mean your PMF is progressing, so youll be ready to make an appointment with your doctor or hematologist as soon as possible for testing. Fast treatment is the best way to slow the progression and give you the best chance for a good outcome. […] Most PMF cases arent treated until symptoms appear. Its important to know what signs to look for so you can start treatment as quickly as possible. Here are six signs that may mean your PMF is progressing. […] The most common symptoms of anemia are weakness and fatigue. You may find yourself struggling to get through the day with low energy levels. […] This may suggest that your PMF is progressing. In addition to causing low red blood cell counts, PMF can lead to thrombocytopenia a low platelet count.
  • #2 Myelofibrosis – Blood Disorders – Merck Manual Consumer Version
    https://www.merckmanuals.com/home/blood-disorders/myeloproliferative-disorders/myelofibrosis
    The liver and spleen enlarge as they try to take over some of the job of making blood cells, but blood cell production is ineffective in these organs and instead just contributes to the spleen enlargement. Enlargement of the liver and spleen may cause pain in the abdomen and may lead to abnormally high blood pressure in certain veins, such as a vein supplying blood to the liver (portal hypertension) and bleeding from varicose veins in the esophagus (esophageal varices). […] Malignant myelofibrosis (sometimes called acute myelofibrosis) is a rare type of myelofibrosis in which levels of red blood cells, white blood cells, and platelets are all decreased. The number of certain immature white blood cells (blast cells) in the bone marrow is increased. Malignant myelofibrosis is considered to be a type of acute leukemia.
  • #2 6 Signs Your Myelofibrosis May Be Progressing | myMPNteam
    https://www.mympnteam.com/resources/signs-your-myelofibrosis-may-be-progressing
    Symptoms of thrombocytopenia include: Bruising or pooling of blood underneath your skin, Frequent nosebleeds, Bleeding or swollen gums, Blood in your urine or stool, Heavy menstrual periods, Petechiae (tiny, rashlike red or purple dots on your lower legs). […] When living with PMF, this symptom is cause for concern. The likely culprit is splenomegaly an enlarged spleen. […] Splenomegaly is most common in PMF, but it can also occur in people with essential thrombocythemia and polycythemia vera, other types of MPNs. […] Excessive night sweats are another sign of MPNs, and theyre common in people with PMF. This disease causes body-wide inflammation that leads to fevers and night sweats. […] Frequent bacterial or fungal infections indicate that your PMF is progressing. […] PMF affects your bone marrow, so it makes sense that you may experience bone or joint pain as your disease progresses. […] If youve started experiencing one or more of these PMF symptoms, its time to check with your doctor. They can run extra tests and determine if your disease is progressing. Its vital to start treatment as soon as possible if your PMF is transforming into acute myeloid leukemia.
  • #2 Myelofibrosis prognosis: Life expectancy, treatment options & more
    https://www.medicalnewstoday.com/articles/myelofibrosis-prognosis
    The outlook for someone with myelofibrosis depends on factors such as age, blood cell counts, and symptoms. For some people, the condition progresses more rapidly than others. […] Because it is a progressive disease, it worsens over time but affects each person differently. While some people have severe symptoms that progress quickly, others may live without symptoms for years. […] MF may cause no symptoms for years. However, late stage MF can present with symptoms that include: […] Severe anemia: The extensive scarring in the bone marrow can lead to severe anemia. This can cause weakness, fatigue, and shortness of breath. […] Enlarged spleen and liver: These organs enlarge as they attempt to take over some of the job of making blood cells. This can cause abdominal pain and high blood pressure in certain veins, which doctors call portal hypertension.
  • #2 Myelofibrosis prognosis: Life expectancy, treatment options & more
    https://www.medicalnewstoday.com/articles/myelofibrosis-prognosis
    Weight loss: Late stage myelofibrosis may cause unintentional weight loss. […] Bone pain: Progressive bone marrow scarring can lead to bone pain, affecting mobility and quality of life. […] A persons myelofibrosis prognosis depends on various factors, and some people remain symptom-free for many years. […] However, for those with risk factors such as being over 65 years old, having whole-body symptoms or anemia, and having specific genetic abnormalities, the outlook may be less favorable.
  • #2 Primary myelofibrosis – Leukaemia Foundation
    https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/myeloproliferative-neoplasms/primary-myelofibrosis/
    The natural course of the disease can vary considerably between individuals. In some people their disease remains stable for long periods and they are free to live a normal life with minimal interruptions from their disease or its treatment. For others, myelofibrosis progresses more quickly and people require treatment to help relieve symptoms of their disease. Transformation to a type of leukaemia called acute myeloid leukaemia occurs in between 10 and 20 per cent of cases.
  • #2 Myelofibrosis – Symptoms and causes – Mayo Clinic
    https://www.mayoclinic.org/diseases-conditions/myelofibrosis/symptoms-causes/syc-20355057
    Myelofibrosis makes it hard for the body to make healthy platelets. Platelets are blood cells that help stop bleeding. If you have too few healthy platelets, you might bruise more easily. You also could have an increased risk of dangerous bleeding. […] Some people with myelofibrosis develop acute myelogenous leukemia. This is a type of blood and bone marrow cancer that often gets worse quickly.
  • #2 Myelofibrosis: Transformation to acute myeloid leukemia
    https://www.medicalnewstoday.com/articles/myelofibrosis-transformation-to-acute-myeloid-leukemia
    If someone has myelofibrosis without AML, their outlook will largely depend on the severity of any symptoms they are experiencing. The symptoms of myelofibrosis include: night sweats, fever, fatigue, unintentional weight loss, fullness or discomfort in the abdomen, quickly feeling full, pain while urinating, blood in the urine, gastrointestinal bleeding, pain in the bones. […] Should myelofibrosis progress to AML, a person may also experience some common AML symptoms, including: headaches, easy bruising, excessive bleeding, anemia, which may involve feelings of weakness and fatigue, frequent infections. […] Myelofibrosis can progress to AML, which is a very severe condition, through a complex process of genetic mutation. Some treatments can reduce the chances of myelofibrosis progressing to AML. […] Several factors will determine the outlook of people with these conditions, including age, symptom severity, and the effectiveness of treatment.
  • #2 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/signs-and-symptoms
    https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/signs-and-symptoms
    MF usually develops slowly. MF often does not cause early symptoms and may be found during a routine blood test. When fibrosis develops in the bone marrow, the bone marrow is unable to produce enough normal blood cells. The lack of blood cells causes many of the signs and symptoms of MF. These include: […] When MF causes symptoms, they can be troublesome. Reducing symptoms is a key goal of treatment. Therefore, it is important to take an active role in monitoring your MF symptoms. Careful tracking of your symptoms can help you and your doctor better understand how to manage and modify your care over time.
  • #2 New Markers of Disease Progression in Myelofibrosis
    https://pmc.ncbi.nlm.nih.gov/articles/PMC8582535/
    Disease progression and prognosis in PMF are usually associated with worsening of anemia, increase of circulating blasts, and, more recently, with the presence, in addition to the classical driver mutations, of JAK2, MPL, and CALR genes, as well as of cytogenetic and molecular abnormalities that have been incorporated into new genetically based prognostic scoring systems. […] Patients display variable clinical and laboratoristic features, including anemia, thrombocytopenia, splenomegaly, thrombotic complications, systemic symptoms, and curtailed survival due to infections, thrombo-hemorrhagic events, or progression to leukemic transformation. […] Disease evolution is characterized by cytopenia(s), in particular anemia, marrow failure, and thrombo-hemorrhagic complications, and 15-20% of patients progress toward a leukemic transformation.
  • #2 Understanding Drivers of Progression in Myelofibrosis
    https://www.onclive.com/view/understanding-drivers-of-progression-in-myelofibrosis
    Some of the molecular markers have been shown to be prognostic. […] In addition, the clinical characteristics that demarcate a patient who is likely to have more aggressive or more advanced disease include patients with rapidly enlarging spleen size, patients who have increased circulating blasts. […] The progression of the disease is a problem. About 20% of the patients with myelofibrosis do change to acute myeloid leukemia. […] Apart from the transformation from acute myeloid leukemia, which happens in 20% of patients, other patients die from the myelofibrosis itself. […] The cause of that may be liver failure because of liver infiltration, pulmonary hypertension because of pulmonary infiltration, or cardiac failure because cardiac output goes up, bleeding from the GI tract in relation to portal hypertension, cachexia, bleeding, thrombosis, infections, and multiple different factors.
  • #2 Myelofibrosis (MF) Signs and Symptoms
    https://www.voicesofmpn.com/myelofibrosis-symptoms
    It is important to keep track of any symptoms you have, even if you are not sure they are related to your MF. […] Symptoms of an enlarged spleen can include pain or discomfort in the abdomen or under the left ribs and feeling full when you haven’t eaten or have eaten very little. […] With MF, scar tissue can build up in the bone marrow, preventing it from making enough red blood cells, resulting in anemia. Anemia is common in people living with MF.
  • #2 Myelofibrosis (MF) – MPN Voice
    https://www.mpnvoice.org.uk/about-mpns/questions/myelofibrosis-mf/
    The prognosis depends on your age, the results of your blood and bone marrow tests and your symptoms. […] The progression of MF is very variable. People in a good prognostic group can have up to at least fifteen years of being well. Those in the poor prognosis group may have a survival of about twelve to eighteen months. Between 10 to 20% of MF patients may develop acute myeloid leukaemia (AML), a type of blood and bone marrow cancer that progresses rapidly. […] MF is a progressive disorder which is irreversible in most cases.
  • #2 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/diagnosis
    https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/diagnosis
    About 10 percent of MF patients do not have a JAK2, MPL or CALR gene mutation. In these cases, the disease is referred to as triple-negative MF, and it is associated with a worse prognosis (outcome). […] Scientists are investigating the role that these and other mutations may have in the onset and progression of MF.
  • #2 What Are the Complications of Myelofibrosis?
    https://www.healthline.com/health/myelofibrosis/symptoms-and-complications-of-myelofibrosis
    As MF progresses, you may have more severe symptoms. As bone marrow continues to turn to scar tissue and blood cell production becomes more abnormal, you also may begin to experience more serious side effects or complications. […] Symptoms you may feel as the disease progresses include fever and increased fatigue. […] The median survival rate for a person with myelofibrosis is approximately 5 years. […] The 4 most common hallmarks or symptoms of myelofibrosis are: enlarged spleen, anemia, bone marrow scarring, symptoms associated with the disease, such as: pale skin, easy bruising or bleeding, excessive sweating while sleeping, fever. […] MF may not cause symptoms in its early stages, but it can lead to serious complications. Being aware of possible symptoms can help you have an informed discussion with your doctor, so MF is not mistaken for other conditions. […] Symptoms to look for include: pale skin, easy bruising or bleeding, excessive sweating while sleeping, fever, fatigue.
  • #2 Primary Myelofibrosis Clinical Presentation: History, Physical Examination
    https://emedicine.medscape.com/article/197954-clinical
    Portal hypertension may occur as a result of markedly increased splenoportal blood flow and decreased hepatic vascular compliance. Ascites, esophageal and gastric varices, GI tract bleeding, and hepatic encephalopathy may occur. Hepatic or portal vein thrombosis may also arise as complications. […] Patients with primary myelofibrosis develop osteosclerosis. This may cause severe joint and bone pain. […] One half of patients with primary myelofibrosis have abnormalities of humoral immunity. A variety of autoantibodies and circulating immune complexes may be detected, and amyloidosis may develop. Infections, commonly pneumonia, may occur as a result of immune deficiency. […] Splenomegaly is the most common finding in patients with primary myelofibrosis, and it is present in approximately 90% of patients. Spleen size may vary from barely palpable to massive (observed in 35% of patients). […] Hepatomegaly is found in 60-70% of patients with primary myelofibrosis, and pallor is observed in 60% of patients. Other physical findings include petechiae and ecchymosis (20%), lymphadenopathy (10-20%), signs of portal hypertension (10-18%), and gout (6%).
  • #2 Myelofibrosis (MF) Signs and Symptoms
    https://www.voicesofmpn.com/myelofibrosis-symptoms
    Myelofibrosis (MF) can cause a variety of symptoms, and each persons experience with MF symptoms can change over time. […] Because MF is a progressive disease, its important to keep an eye out for any new or worsening symptoms that could indicate the disease is getting worse. […] In addition to the symptoms caused by an enlarged spleen, MF can affect the body in other ways. […] According to the MPN Landmark Survey, * 81% of patients with MF reported that their symptoms reduced their quality of life. […] Because MF is a progressive condition, it has the potential to change over time. […] This is why its important to take an active role in monitoring your condition over time by tracking your MF on a regular basis and sharing any changes with your Healthcare Professional. […] Regular symptom tracking provides a number of valuable insights.
  • #2 Myelofibrosis (MF) Symptoms | MPN Connect
    https://www.mpnconnect.com/myelofibrosis-mf/symptoms
    In myelofibrosis (MF), symptoms can gradually develop over time and thus may not be fully appreciated by patients as they have sometimes compensated for them. […] MF carries a symptom burden that affects the majority of patients. […] The MPN Landmark Survey was a large-scale survey of 813 patients with myeloproliferative neoplasms (MPNs) (MF, n = 207) and assessed perceptions of overall disease burden, quality of life (QOL), activities of daily living, and work productivity. […] 81% of patients reported reduced QOL related to their condition; this was true even for patients with low prognostic risk scores and with their lowest symptom severity. […] Common symptoms and clinical manifestations include: Fatigue, Fever, Weight loss, Anemia, Leukoerythroblastosis, Pruritus, Night Sweats, Splenomegaly, Increased or decreased white blood cell levels, Bone marrow fibrosis. […] Other possible clinical presentations of MF are: Hepatomegaly, Lymphadenopathy, Pleural effusion, Osteosclerosis, Periostitis, Portal hypertension, Ascites, Nerve or spinal cord compression, Hypertrophic osteoarthropathy, Megakaryocytic dysplasia.
  • #3 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/signs-and-symptoms
    https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/signs-and-symptoms
    MF usually develops slowly. MF often does not cause early symptoms and may be found during a routine blood test. When fibrosis develops in the bone marrow, the bone marrow is unable to produce enough normal blood cells. The lack of blood cells causes many of the signs and symptoms of MF. These include: […] When MF causes symptoms, they can be troublesome. Reducing symptoms is a key goal of treatment. Therefore, it is important to take an active role in monitoring your MF symptoms. Careful tracking of your symptoms can help you and your doctor better understand how to manage and modify your care over time.
  • #3 Myelofibrosis – MPN Research Foundation
    https://mpnresearchfoundation.org/primary-myelofibrosis-pmf/
    Myelofibrosis symptoms are often caused by an enlarged spleen and/or insufficient numbers of normal blood cells and chronic inflammation. […] Common MF symptoms and signs may include: Tiredness, weakness, or shortness of breath with mild exertion; Fullness, discomfort or pain in the left upper area of the abdomen; Fever; Night sweats; Weight loss or malnutrition; Bone pain; Itching (pruritus); Easy bleeding or bruising; Susceptibility to infection; Joint pain or gout; Abdominal distension/fluid retention (if portal hypertension or increased blood pressure within the portal vein is present); Compromised liver function; Abnormal growth of blood forming cells outside of the bone marrow. […] Routine medical examinations including complete blood counts (CBCs) are important for diagnosing MF and other MPNs, since some MF patients exhibit no symptoms (especially during the early course of the disease).
  • #3 6 Signs Your Myelofibrosis May Be Progressing | myMPNteam
    https://www.mympnteam.com/resources/signs-your-myelofibrosis-may-be-progressing
    Symptoms of thrombocytopenia include: Bruising or pooling of blood underneath your skin, Frequent nosebleeds, Bleeding or swollen gums, Blood in your urine or stool, Heavy menstrual periods, Petechiae (tiny, rashlike red or purple dots on your lower legs). […] When living with PMF, this symptom is cause for concern. The likely culprit is splenomegaly an enlarged spleen. […] Splenomegaly is most common in PMF, but it can also occur in people with essential thrombocythemia and polycythemia vera, other types of MPNs. […] Excessive night sweats are another sign of MPNs, and theyre common in people with PMF. This disease causes body-wide inflammation that leads to fevers and night sweats. […] Frequent bacterial or fungal infections indicate that your PMF is progressing. […] PMF affects your bone marrow, so it makes sense that you may experience bone or joint pain as your disease progresses. […] If youve started experiencing one or more of these PMF symptoms, its time to check with your doctor. They can run extra tests and determine if your disease is progressing. Its vital to start treatment as soon as possible if your PMF is transforming into acute myeloid leukemia.
  • #3 6 Signs Your Myelofibrosis May Be Progressing | myMPNteam
    https://www.mympnteam.com/resources/signs-your-myelofibrosis-may-be-progressing
    When youre first diagnosed with primary myelofibrosis (PMF), you may not experience any symptoms. […] In this article, well go over six signs that may mean your PMF is progressing, so youll be ready to make an appointment with your doctor or hematologist as soon as possible for testing. Fast treatment is the best way to slow the progression and give you the best chance for a good outcome. […] Most PMF cases arent treated until symptoms appear. Its important to know what signs to look for so you can start treatment as quickly as possible. Here are six signs that may mean your PMF is progressing. […] The most common symptoms of anemia are weakness and fatigue. You may find yourself struggling to get through the day with low energy levels. […] This may suggest that your PMF is progressing. In addition to causing low red blood cell counts, PMF can lead to thrombocytopenia a low platelet count.
  • #3 Myelofibrosis: Symptoms, Types, Prognosis & Treatment
    https://my.clevelandclinic.org/health/diseases/15672-myelofibrosis
    Myelofibrosis progresses slowly, so you may not have symptoms for many years. About one-third of people dont show symptoms during the disorders early stages. […] When they arise, the most common symptoms of myelofibrosis are severe fatigue (resulting from anemia) and an enlarged spleen. Symptoms may include: Fatigue. Fever. Itching. Pale skin. Weight loss. Night sweats. Bone or joint pain. Frequent infections. Enlarged spleen or liver. Unexplained blood clots. Abnormal bleeding or bruising. Enlarged veins in your stomach and esophagus. (These veins may rupture and cause bleeding.) […] In about 12% of all cases, primary myelofibrosis progresses to acute myeloid leukemia, a very aggressive form of blood cancer. […] Myelofibrosis is aggressive cancer with a median survival rate of six years. A median is a midpoint, which means that some people live less than six years, and about the same number of people live longer than six years.
  • #3 Myelofibrosis prognosis: Life expectancy, treatment options & more
    https://www.medicalnewstoday.com/articles/myelofibrosis-prognosis
    Weight loss: Late stage myelofibrosis may cause unintentional weight loss. […] Bone pain: Progressive bone marrow scarring can lead to bone pain, affecting mobility and quality of life. […] A persons myelofibrosis prognosis depends on various factors, and some people remain symptom-free for many years. […] However, for those with risk factors such as being over 65 years old, having whole-body symptoms or anemia, and having specific genetic abnormalities, the outlook may be less favorable.
  • #3 Primary Myelofibrosis: Practice Essentials, Pathophysiology, Etiology
    https://emedicine.medscape.com/article/197954-overview
    Extramedullary hematopoiesis may involve any organ; symptoms depend on the organ or site of involvement. […] Patients with primary myelofibrosis are also prone to developing infectious complications because of defects in humoral immunity. […] Osteosclerosis, hypertrophic osteoarthropathy, and periostitis may occur, resulting in significant pain and discomfort. […] The median length of survival for patients with primary myelofibrosis is 3.5-5.5 years. […] The common causes of death in patients with primary myelofibrosis are infections, hemorrhage, cardiac failure, postsplenectomy mortality, and transformation into acute leukemia. […] Advanced age and anemia are associated with shorter survival. […] Other poor prognostic factors include hypercatabolic symptoms, leukocytosis, leukopenia, circulating blasts, increased numbers of granulocyte precursors, thrombocytopenia, and karyotype abnormalities.
  • #4 Myelofibrosis (MF) symptoms | Blood Cancer UK
    https://bloodcancer.org.uk/understanding-blood-cancer/myelofibrosis/myelofibrosis-symptoms/
    The symptoms of myelofibrosis vary depending on what is happening in the bone marrow, the spongy material inside our larger bones where blood cells are made. […] Some people dont have any symptoms at all when they are diagnosed with myelofibrosis (MF). Others will have more obvious symptoms. […] Its important to realise that there isnt always a direct link between the level of symptoms and the stage of the disease. It depends on you, your blood counts, and whats happening in your bone marrow. […] Common symptoms include: extreme tiredness (fatigue), losing weight without trying, drenching night sweats, unexplained fever, itching, sometimes particularly after a hot bath or shower, general weakness, feeling full up very quickly when trying to eat. […] Low levels of blood cells can lead to a range of symptoms: Too few red blood cells can make you feel tired, breathless and dizzy. The medical name for this is anaemia. Too few white blood cells can mean you get more infections like colds and flu, chest and urinary tract infections. These may last longer and be more serious than usual. Too few platelets can lead to bruising and unusual bleeding such as nose bleeds and heavy periods. […] A few people may have bone pain. This can happen at any stage but is more likely at a later stage of myelofibrosis (MF) when there is more scarring (fibrosis) and more inflammation in the bone marrow.
  • #5 Primary myelofibrosis – Leukaemia Foundation
    https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/myeloproliferative-neoplasms/primary-myelofibrosis/
    Primary myelofibrosis (also called chronic idiopathic myelofibrosis, agnogenic myeloid metaplasia) is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. […] Symptoms of anaemia are common and include unexplained tiredness, weakness, shortness of breath and palpitations. Other nonspecific symptoms include fever, unintended weight loss, pruritus (generalised itching) and excess sweating, especially at night. […] Virtually all patients with primary myelofibrosis have an enlarged spleen (splenomegaly) when they are first diagnosed. In around a third of cases the spleen is very enlarged. Common symptoms include feelings of discomfort, pain or fullness in the upper left-side of the abdomen.