Materiały źródłowe

• #1 Myelofibrosis management in routine clinical practice with a focus on patients with cytopenias: recommendations from a global consensus group | Leukemia

  https://www.nature.com/articles/s41375-024-02330-7

  Myelofibrosis (MF) is a Philadelphia chromosome (BCR::ABL1)-negative myeloproliferative neoplasm, a hallmark of which is progressive deposition of fibrotic tissue in bone marrow. Clinical manifestations of MF often include splenomegaly, cytopenias (such as severe anemia), and extramedullary hematopoiesis. The Janus kinase inhibitor (JAKi) therapies ruxolitinib (RUX) and fedratinib (FED) have demonstrated significant clinical efficacy in splenic volume reduction and symptom improvement, but they may induce treatment-related anemia and thrombocytopenia. Other JAKi options include pacritinib (PAC), which received FDA approval in 2022 for patients with MF and severe thrombocytopenia (platelet count 50109/l), and momelotinib (MMB), which received FDA and EMA approval in 2023/2024, respectively, for patients with MF and anemia. National and international guidelines exist for the management of MF; however, a need remains for practical guidance applicable in everyday clinical practice, especially for patients experiencing cytopenias or potential failure of current therapy. The landscape is further complicated by the availability of multiple prognostic tools for MF; as such, clinicians may find disease prognostication challenging and confusing. Additionally, to maximize clinical applicability of trial data, inclusivity of eligibility criteria in the context of the real-world MF patient population should be considered.

• #2 Myelofibrosis | GSK US

  https://us.gsk.com/en-us/company/oncology/myelofibrosis/

  Myelofibrosis can lead to severe low blood counts, including anemia and thrombocytopenia (low platelet count), as well as constitutional symptoms, such as weight loss, fatigue, and splenomegaly (enlarged spleen).1,3 […] Some currently approved treatments for myelofibrosis exacerbate anemia, leading to a more than 30% discontinuation rate.4 […] There remains a significant medical need for myelofibrosis patients with anemia, which is associated with a negative impact on overall survival and quality of life.4 […] The goal of treatment for people with myelofibrosis is to reduce symptom burden and minimize the risk of developing acute leukemia, a cancer that progresses rapidly. Based on the symptoms you’re experiencing, your doctor might recommend treatment for anemia (i.e., blood transfusions, medications), enlarged spleen (i.e., chemotherapy, removal of the spleen, radiation therapy) and/or thrombocytopenia. […] In addition to these treatments, some patients are eligible for a bone marrow transplant, also known as an allogenic stem cell transplant. This procedure involves replacing disease bone marrow with healthy blood stem cells from a donor. This treatment has the potential to cure myelofibrosis.

• #3 Myelofibrosis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/myelofibrosis/diagnosis-treatment/drc-20355062

  Treatments for myelofibrosis include blood transfusions and medicines, such as chemotherapy and targeted therapy. Other treatments include surgery to remove the spleen, radiation therapy and bone marrow transplant, also called stem cell transplant. […] The goal of treatment for most people with myelofibrosis is to provide relief from symptoms of the disease. For some, a bone marrow transplant, also called a stem cell transplant, may provide a chance for a cure. This treatment can be very hard on the body, and it might not be an option for many people. […] To find out which myelofibrosis treatments are most likely to benefit you, your healthcare professional may use one or more formulas to assess your condition. These formulas take into account many aspects of your cancer and your overall health. A healthcare professional may assign a risk category that indicates how serious your condition is.

• #4 Myelofibrosis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/myelofibrosis/diagnosis-treatment/drc-20355062

  A lower risk myelofibrosis might grow slowly. Treatment might not be needed right away, or treatment might focus on controlling any symptoms. A higher risk myelofibrosis might be getting worse quickly. People with higher risk myelofibrosis might consider stronger treatments, such as a bone marrow transplant. […] Myelofibrosis treatment might not be needed right away if you don’t have any symptoms. Rather than giving you treatment, your healthcare professional may monitor your health closely through regular checkups and exams. Some people remain symptom-free for years. […] If myelofibrosis is causing severe anemia, your healthcare professional may recommend treatments such as blood transfusions and medicines that increase blood cell production. […] If an enlarged spleen is causing complications, your healthcare professional may recommend treatment. Your options may include targeted therapy, chemotherapy, surgery to remove the spleen, and radiation therapy.

• #5 Myelofibrosis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/myelofibrosis/diagnosis-treatment/drc-20355062

  Treatments for myelofibrosis include blood transfusions and medicines, such as chemotherapy and targeted therapy. Other treatments include surgery to remove the spleen, radiation therapy and bone marrow transplant, also called stem cell transplant. […] The goal of treatment for most people with myelofibrosis is to provide relief from symptoms of the disease. For some, a bone marrow transplant, also called a stem cell transplant, may provide a chance for a cure. This treatment can be very hard on the body, and it might not be an option for many people. […] To find out which myelofibrosis treatments are most likely to benefit you, your healthcare professional may use one or more formulas to assess your condition. These formulas take into account many aspects of your cancer and your overall health. A healthcare professional may assign a risk category that indicates how serious your condition is.

• #6 Myelofibrosis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/myelofibrosis/diagnosis-treatment/drc-20355062

  A lower risk myelofibrosis might grow slowly. Treatment might not be needed right away, or treatment might focus on controlling any symptoms. A higher risk myelofibrosis might be getting worse quickly. People with higher risk myelofibrosis might consider stronger treatments, such as a bone marrow transplant. […] Myelofibrosis treatment might not be needed right away if you don’t have any symptoms. Rather than giving you treatment, your healthcare professional may monitor your health closely through regular checkups and exams. Some people remain symptom-free for years. […] If myelofibrosis is causing severe anemia, your healthcare professional may recommend treatments such as blood transfusions and medicines that increase blood cell production. […] If an enlarged spleen is causing complications, your healthcare professional may recommend treatment. Your options may include targeted therapy, chemotherapy, surgery to remove the spleen, and radiation therapy.

• #7 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  There is not one treatment that is effective for all myelofibrosis (MF) patients. Patients have varying symptoms and circumstances that require different treatment options. […] The only potential cure for MF is allogeneic stem cell transplantation. But this procedure is risky for older patients and those with other health problems. […] For most people with MF, treatment remains aimed at controlling disease symptoms and complications, enhancing quality of life and extending survival. […] The treatment approach for low-risk category patients who have symptoms of MF may be observation only. In certain circumstances, the doctor may prescribe cytoreductive treatment (a medication to lower blood cell counts) to help relieve the patients symptoms. […] Treatment options include: Participation in a clinical trial, Ruxolitinib (Jakafi), Peginterferon alfa-2a (Pegasys), Hydroxyurea (Hydrea) to relieve symptoms caused by high blood counts, Pacritinib (Vonjo) if the platelet count is less than 50,000, Momelotinib (Ojjaara), Fedratinib (Inrebic).

• #8 Myelofibrosis Treatments, Side Effects, and Alternative Therapies

  https://www.webmd.com/cancer/myelofibrosis-treatments

  Myelofibrosis (MF) treatment is based on your symptoms. If you don’t feel bad right now, you may be able to just watch and wait without treating your cancer. Regular checkups and blood tests will make sure you don’t have complications like anemia or an enlarged spleen. […] The drugs fedratinib (Inrebic), pacritinib (Vonjo), and ruxolitinib (Jakafi) are approved to treat MF. Most people with MF have a mutation, or change, in one of their genes that tell their body how to make blood cells. These inhibitors are used to block the processes of those faulty genes. The medications can ease some MF symptoms like an enlarged spleen, bone pain, itching, and night sweats. But they can have side effects, like a decrease in platelets or making your anemia worse. […] Only through a process called allogeneic stem cell transplantation. In MF, your stem cells don’t work like they should. This causes scar tissue to build up in your bone marrow. During this treatment, you’re given healthy stem cells from a donor to replace your faulty ones.

• #9 Myelofibrosis Treatment | What Is Jakafi® (ruxolitinib) | Jakafi.com

  https://www.jakafi.com/myelofibrosis/mf-treatment

  Jakafi (ruxolitinib) is a prescription medicine available as a pill. Jakafi is used to treat adults with intermediate or high-risk myelofibrosis (MF). […] Jakafi is the first medicine approved by the Food and Drug Administration (FDA) for the treatment of these patients. […] Jakafi works by targeting JAKs, which control the production of blood cells. In doing so, Jakafi helps to reduce overactive JAK signaling to help keep the production of blood cells under control. […] Jakafi is used to treat adults with certain types of myelofibrosis. […] Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had low white or red blood cell counts, have or had tuberculosis (TB) or have been in close contact with someone who has TB, had shingles (herpes zoster), have or had hepatitis B, have or had liver or kidney problems, are on dialysis, have high cholesterol or triglycerides, had cancer, are a current or past smoker, had a blood clot, heart attack, other heart problems or stroke, or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change your dose or stop taking Jakafi without first talking to your healthcare provider. […] Women should not take Jakafi while pregnant or planning to become pregnant. Do not breastfeed during treatment with Jakafi and for 2 weeks after the final dose.

• #10 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. […] The oral Janus kinase (JAK) 1/JAK2 inhibitor ruxolitinib was initially approved by the US Food and Drug Administration in 2011 for the treatment of patients with intermediate or high-risk MF, including primary MF, post-polycythemia vera MF, and post-essential thrombocythemia MF, based on efficacy and safety findings from the randomized, controlled, phase 3 COMFORT trials. […] Over a decade later, ruxolitinib continues to be the standard of care in higher-risk MF, and dose optimization and management remain crucial for safely maximizing clinical benefits of ruxolitinib. […] Ruxolitinib has been shown to not only improve splenomegaly and the burdensome symptoms associated with MF but also to improve overall survival (OS).

• #11 Treatment Strategies Used in Treating Myelofibrosis: State of the Art

  https://www.mdpi.com/2038-8330/16/4/67

  In the COMFORT-I trial, 41.9% of patients in the RUXO arm achieved the primary endpoint of a ≥35% reduction in spleen volume from baseline at week 24. […] Additional JAK inhibitors have been studied for the treatment of MF. Fedratinib (FEDRA) is a JAK2-selective inhibitor, approved in the United States as a therapy for patients with intermediate-2 or high-risk primary or secondary MF after the results of the JAKARTA study. […] Pacritinib (PACRI) is a selective inhibitor of JAK2, JAK2 V617F, and FLT3 and does not inhibit JAK1. […] Momelotinib (MMB) is an inhibitor of JAK1 and JAK2, an emerging treatment for MF patients. […] Despite the development of JAK inhibitors, the number of MF patients undergoing allo-SCT is steadily increasing, and the procedure remains the only curative treatment for these patients.

• #12 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. […] The oral Janus kinase (JAK) 1/JAK2 inhibitor ruxolitinib was initially approved by the US Food and Drug Administration in 2011 for the treatment of patients with intermediate or high-risk MF, including primary MF, post-polycythemia vera MF, and post-essential thrombocythemia MF, based on efficacy and safety findings from the randomized, controlled, phase 3 COMFORT trials. […] Over a decade later, ruxolitinib continues to be the standard of care in higher-risk MF, and dose optimization and management remain crucial for safely maximizing clinical benefits of ruxolitinib. […] Ruxolitinib has been shown to not only improve splenomegaly and the burdensome symptoms associated with MF but also to improve overall survival (OS).

• #13 Myelofibrosis – MPN Research Foundation

  https://mpnresearchfoundation.org/primary-myelofibrosis-pmf/

  There is no single treatment that is effective for all MF patients. Each patient has a unique set of symptoms and circumstances that require different treatment options, as prescribed by a doctor. Also, some patients with MF remain symptom-free for many years and may not require immediate treatment. However, anyone who has been diagnosed with MF needs to be monitored over time for signs or symptoms that suggest the disease has worsened. […] Available treatments and therapies for MF include: […] Jakafi (ruxolitinib) is the first drug approved by the FDA for treating MF patients. Jakafi is indicated for the treatment of patients with intermediate or high-risk myelofibrosis (MF), including primary MF, postpolycythemia vera MF and postessential thrombocythemia MF. […] INREBIC (fedratinib) is indicated for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF).

• #14 Myelofibrosis: Symptoms, Types, Prognosis & Treatment

  https://my.clevelandclinic.org/health/diseases/15672-myelofibrosis

  Myelofibrosis Treatment Find a Doctor and Specialists Make an Appointment […] You dont need treatment unless youre experiencing symptoms. Still, your healthcare provider will monitor your condition even if you dont require immediate treatment. […] Jakafi (ruxolitinib), Inrebic (fedratinib) and Vonjo (pacritinib) are U.S. Food and Drug Administration (FDA)-approved drugs for treating intermediate or high-risk myelofibrosis. All three drugs are JAK inhibitors that reduce overactive Janus-associated kinase (JAK) signaling. They help relieve some of the symptoms associated with myelofibrosis, including an enlarged spleen, night sweats, itching, weight loss and fever. […] For most people, treatment goals are to manage conditions associated with myelofibrosis, including anemia and splenomegaly.

• #15 Next Generation Therapeutics for the Treatment of Myelofibrosis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8146033/

  Myelofibrosis is a myeloproliferative neoplasm characterized by splenomegaly, constitutional symptoms, bone marrow fibrosis, and a propensity towards transformation to acute leukemia. JAK inhibitors are the only approved therapy for myelofibrosis and have been successful in reducing spleen and symptom burden. However, they do not significantly impact disease progression and many patients are ineligible due to coexisting cytopenias. Patients who are refractory to JAK inhibition also have a dismal survival. Therefore, non-JAK inhibitor-based therapies are being explored in pre-clinical and clinical settings. […] Currently, there are two United States Food and Drug Administration (FDA) approved therapies for MF: ruxolitinib (Jakafi, Incyte, Wilmington, DE, USA) and fedratinib (Inrebic, Bristol-Myers Squibb, New York, NY, USA). Both are JAK inhibitors (JAKi), with ruxolitinib demonstrating equipotent inhibition of both JAK1 and JAK2 while fedratinib demonstrates selectivity inhibition for JAK2 but also FLT3.

• #16 Treatment Strategies Used in Treating Myelofibrosis: State of the Art

  https://www.mdpi.com/2038-8330/16/4/67

  In the COMFORT-I trial, 41.9% of patients in the RUXO arm achieved the primary endpoint of a ≥35% reduction in spleen volume from baseline at week 24. […] Additional JAK inhibitors have been studied for the treatment of MF. Fedratinib (FEDRA) is a JAK2-selective inhibitor, approved in the United States as a therapy for patients with intermediate-2 or high-risk primary or secondary MF after the results of the JAKARTA study. […] Pacritinib (PACRI) is a selective inhibitor of JAK2, JAK2 V617F, and FLT3 and does not inhibit JAK1. […] Momelotinib (MMB) is an inhibitor of JAK1 and JAK2, an emerging treatment for MF patients. […] Despite the development of JAK inhibitors, the number of MF patients undergoing allo-SCT is steadily increasing, and the procedure remains the only curative treatment for these patients.

• #17 Primary Myelofibrosis Diagnosis & Disease Information

  https://www.cancertherapyadvisor.com/ddi/primary-myelofibrosis/

  Fedratinib is a JAK2/FLT3 inhibitor. It can be used as a first-line therapy or as an option for patients with intermediate- or high-risk primary myelofibrosis who do not respond to or cannot tolerate ruxolitinib. Similar to ruxolitinib, fedratinib reduces splenomegaly and disease-related symptoms, but it may be associated with increased gastrointestinal (GI) toxicity compared with ruxolitinib. […] Momelotinib is a JAK1/2 inhibitor that is indicated for treating patients with intermediate- or high-risk primary myelofibrosis who are anemic. Its effects on the general symptoms and splenomegaly of primary myelofibrosis are similar to those of ruxolitinib. Its main advantage is its ability to alleviate anemia. […] Pacritinib is a JAK2, FLT3, and IRAK1 inhibitor. It is indicated for patients with patients with intermediate- or high-risk primary myelofibrosis and a platelet count less than 50 x 109/L. Pacritinib may be a preferred option for patients with severe thrombocytopenia and/or anemia, though it is associated with increased GI toxicity compared with ruxolitinib.

• #18 Primary Myelofibrosis Diagnosis & Disease Information

  https://www.cancertherapyadvisor.com/ddi/primary-myelofibrosis/

  Fedratinib is a JAK2/FLT3 inhibitor. It can be used as a first-line therapy or as an option for patients with intermediate- or high-risk primary myelofibrosis who do not respond to or cannot tolerate ruxolitinib. Similar to ruxolitinib, fedratinib reduces splenomegaly and disease-related symptoms, but it may be associated with increased gastrointestinal (GI) toxicity compared with ruxolitinib. […] Momelotinib is a JAK1/2 inhibitor that is indicated for treating patients with intermediate- or high-risk primary myelofibrosis who are anemic. Its effects on the general symptoms and splenomegaly of primary myelofibrosis are similar to those of ruxolitinib. Its main advantage is its ability to alleviate anemia. […] Pacritinib is a JAK2, FLT3, and IRAK1 inhibitor. It is indicated for patients with patients with intermediate- or high-risk primary myelofibrosis and a platelet count less than 50 x 109/L. Pacritinib may be a preferred option for patients with severe thrombocytopenia and/or anemia, though it is associated with increased GI toxicity compared with ruxolitinib.

• #19 Primary Myelofibrosis Treatment & Management: Approach Considerations, JAK Inhibitors, FGFR Inhibitors

  https://emedicine.medscape.com/article/197954-treatment

  The following Janus-associated kinase (JAK) inhibitors are approved for the treatment of myelofibrosis: Ruxolitinib (Jakafi), Fedratinib (Inrebic), Pacritinib (Vonjo), Momelotinib (Ojjaara). […] Ruxolitinib (Jakafi), a JAK1/JAK2 inhibitor, is the first chemotherapeutic agent to be approved by the US Food and Drug Administration (FDA) for the treatment of myelofibrosis. […] Another JAK inhibitor, fedratinib (Inrebic), was approved in 2019 for adults with intermediate-2 or high-risk primary myelofibrosis. […] In March 2022, the FDA approved pacritinib (Vonjo), the first treatment for adults with myelofibrosis and severe thrombocytopenia (platelet count 50 109/L). […] In September 2023, the FDA approved momelotinib (Ojjaara) for the treatment of intermediate- or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (postpolycythemia vera and postessential thrombocythemia), in adults with anemia. […] Splenectomy may be considered for patients with massive splenomegaly. […] Allogeneic stem cell transplantation (SCT) is a potentially curative therapy in patients with primary myelofibrosis.

• #20 Next-Generation JAK Inhibitors Signal the Future of Myelofibrosis Treatment Advances

  https://www.onclive.com/view/next-generation-jak-inhibitors-signal-the-future-of-myelofibrosis-treatment-advances

  The major issue in treating myelofibrosis is symptom control. Patients will develop extreme fatigue due to cytokines. […] Ruxolitinib was the first FDA-approved JAK inhibitor. […] However, one of the problems with [ruxolitinib] is that it causes significant myelosuppression, so it could worsen anemia, which is [a symptom] that many patients with myelofibrosis suffer from. […] Where fedratinib fits best [into the myelofibrosis treatment paradigm] is for patients who have been on ruxolitinib and have lost their response, because fedratinib has also been shown to recapture those responses. […] Pacritinib doesn’t seem to worsen thrombocytopenia, so for patients with significant thrombocytopenia, I reach for pacritinib.

• #21 Next-Generation JAK Inhibitors Signal the Future of Myelofibrosis Treatment Advances

  https://www.onclive.com/view/next-generation-jak-inhibitors-signal-the-future-of-myelofibrosis-treatment-advances

  Newer-generation JAK inhibitors are increasingly adept at controlling symptoms in patients with myelofibrosis and may recapture treatment response in patients who have progressed on prior ruxolitinib (Jakafi), according to Joseph G. Jurcic, MD. […] Jurcic discussed the benefits and limitations of several JAK inhibitors for patients with myelofibrosis, highlighting the treatment advances that have been made since the introduction of ruxolitinib to the treatment paradigm, considerations for the use of fedratinib (Inrebic), and the potential advantages of pacritinib (Vonjo) for patients with anemia. […] Pacritinib received FDA accelerated approval in 2022 for the treatment of patients with intermediate- or high-risk myelofibrosis with a platelet count below 50 109/L based on findings from PERSIST-1, the pivotal phase 3 PERSIST-2 trial (NCT02055781), and the phase 2 PAC203 trial (NCT04884191), in which the agent generated a SVR of at least 35% in 29% of patients vs 3% of those who received best available therapy (BAT), including ruxolitinib.

• #22 Next-Generation JAK Inhibitors Signal the Future of Myelofibrosis Treatment Advances

  https://www.onclive.com/view/next-generation-jak-inhibitors-signal-the-future-of-myelofibrosis-treatment-advances

  The major issue in treating myelofibrosis is symptom control. Patients will develop extreme fatigue due to cytokines. […] Ruxolitinib was the first FDA-approved JAK inhibitor. […] However, one of the problems with [ruxolitinib] is that it causes significant myelosuppression, so it could worsen anemia, which is [a symptom] that many patients with myelofibrosis suffer from. […] Where fedratinib fits best [into the myelofibrosis treatment paradigm] is for patients who have been on ruxolitinib and have lost their response, because fedratinib has also been shown to recapture those responses. […] Pacritinib doesn’t seem to worsen thrombocytopenia, so for patients with significant thrombocytopenia, I reach for pacritinib.

• #23 Myelofibrosis Treatment in 2024: New Horizons and Innovations

  https://www.delveinsight.com/blog/myelofibrosis-treatment-landscape

  Myelofibrosis is a rare disease with a high unmet need, with no disease-modifying treatments. Before 2011, treatment options for myelofibrosis were limited to either allogeneic transplant or palliation. Currently, Janus kinase (JAK) inhibitors have become the mainstay of pharmacologic therapy for myelofibrosis patients. […] Over a decade later, JAKAFI continues to be the standard of care in higher-risk myelofibrosis. […] The second JAK inhibitor approved for the treatment of myelofibrosis was INREBIC (fedratinib), approved in 2019. […] The third approved JAK inhibitor for myelofibrosis was VONJO (pacritinib). […] The fourth and most recently approved JAK inhibitor was OJJAARA (momelotinib) by GSK. In September 2023, the US FDA approved OJJAARA for the treatment of intermediate or high-risk myelofibrosis, including primary or secondary myelofibrosis, in adults with anemia.

• #24 Myelofibrosis management in routine clinical practice with a focus on patients with cytopenias: recommendations from a global consensus group | Leukemia

  https://www.nature.com/articles/s41375-024-02330-7

  Recognizing these significant challenges, an international expert consensus group was established to provide best practice recommendations for healthcare professionals, intending to supplement, but not replace, existing guidelines. The consensus program was conducted between March and September 2023, led by a Steering Committee (SC) of nine international hematology experts. The SC identified 25 key clinical questions across five consensus themes: […] Theme 1 (CR1CR4) focused on optimizing anemia management in MF. Diagnosing and addressing anemia in patients with MF is complex, as it is frequently multifactorial and may be disease and/or treatment related. A comprehensive workup, accurate diagnosis, and appropriate treatment are of critical importance. All current and emerging treatment options for MF- and treatment-related anemia should be considered and are outlined in Table 2. Importantly, only MMB is approved specifically for MF-related anemia. In addition to JAKi monotherapy, existing data support the efficacy of RUX-based combination therapies in managing MF with anemia; clinical trials featuring RUX-based combination therapies are summarized in reference.

• #25 FDA Approves Treatment for Myelofibrosis and Anemia | Leukemia and Lymphoma Society

  https://www.lls.org/news/fda-approves-treatment-myelofibrosis-and-anemia

  The U.S. Food and Drug Administration (FDA) recently approved momelotinib (Ojjaara) for use in adults with intermediate or high-risk myelofibrosis, including primary or secondary myelofibrosis, with anemia. […] Treatment with momelotinib resulted in better symptom reduction versus ruxolitinib (Jakafi) in one trial and danazol in another, which the FDA used to support this approval. […] This approval gives patients with myelofibrosis a once-daily treatment to better help manage some of the most common symptoms associated with this disease, says LLSs Chief Scientific Officer Lee Greenberger, Ph.D. […] Research into additional treatment options to elicit long-term responses and symptom control is ongoing, says Dr. Greenberger. […] Continued work is necessary to ensure patients not only live longer and see durable remissions, but that they can return to what they once considered normal daily routines, he concluded.

• #26 Primary Myelofibrosis Diagnosis & Disease Information

  https://www.cancertherapyadvisor.com/ddi/primary-myelofibrosis/

  Fedratinib is a JAK2/FLT3 inhibitor. It can be used as a first-line therapy or as an option for patients with intermediate- or high-risk primary myelofibrosis who do not respond to or cannot tolerate ruxolitinib. Similar to ruxolitinib, fedratinib reduces splenomegaly and disease-related symptoms, but it may be associated with increased gastrointestinal (GI) toxicity compared with ruxolitinib. […] Momelotinib is a JAK1/2 inhibitor that is indicated for treating patients with intermediate- or high-risk primary myelofibrosis who are anemic. Its effects on the general symptoms and splenomegaly of primary myelofibrosis are similar to those of ruxolitinib. Its main advantage is its ability to alleviate anemia. […] Pacritinib is a JAK2, FLT3, and IRAK1 inhibitor. It is indicated for patients with patients with intermediate- or high-risk primary myelofibrosis and a platelet count less than 50 x 109/L. Pacritinib may be a preferred option for patients with severe thrombocytopenia and/or anemia, though it is associated with increased GI toxicity compared with ruxolitinib.

• #27 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  Treatment options include: Allogeneic stem cell transplant, Participation in a clinical trial, Pacritinib (Vonjo) (preferred regimen) or momelotinib (Ojjaara). […] Treatment options include: Allogeneic stem cell transplant, Participation in a clinical trial, Ruxolitinib (Jakafi), fedratinib (Inrebic), momelotinib (Ojjaara), pacritinib (Vonjo). […] For patients with anemia, the treatment approach depends on whether MF symptoms are present and, if present, on how well drug therapy is controlling these symptoms. […] Treatment may include: Participation in a clinical trial, Luspatercept-aamt (Reblozyl), Erythropoiesis (EPO)-stimulating agents (ESA) if EPO level is lower than 500 mU/mL, Momelotinib (Ojjaara), Pacritinib (Vonjo), Receive red blood cell transfusions. […] Allogeneic stem cell transplantation is the only current treatment with the potential to cure MF, but it also carries a high risk of life-threatening side effects.

• #28 Tests and treatment for myelofibrosis | Cancer Research UK

  https://www.cancerresearchuk.org/about-cancer/myelofibrosis/tests-treatment

  Your treatment depends on a number of factors including: […] Treatments might include one or more of the following: […] Blood transfusions increase the number of red blood cells. This helps to control tiredness, breathlessness and weakness. […] You take aspirin as a tablet. It can help to reduce the risk of your blood clotting if you are at risk of developing them. […] Allopurinol lowers the amount of uric acid in the blood and so helps to reduce the symptoms of gout. […] Folic acid is a vitamin that helps the body make red blood cells. You may have this if you have anaemia. […] JAK2 inhibitors work by slowing or stopping the growth of cancer cells. […] Ruxolitinib is used to treat an enlarged spleen and other symptoms. […] Some people might have fedratinib to treat an enlarged spleen or other symptoms.

• #29 Myelofibrosis – Blood Disorders – Merck Manual Consumer Version

  https://www.merckmanuals.com/home/blood-disorders/myeloproliferative-disorders/myelofibrosis

  The combination of an androgen (a medication with the effects of male sex hormones) and prednisone temporarily lessens the severity of the anemia in about one third of people with myelofibrosis, but this treatment is not often used. […] Erythropoietin, a hormone that stimulates red blood cell production, may improve anemia but also causes enlargement of the spleen. […] Sometimes erythropoietin is used together with a JAK inhibitor such as ruxolitinib to prevent enlargement of the spleen. […] Some people with severe anemia require red blood cell transfusions. […] Doctors sometimes also give thalidomide or lenalidomide together with prednisone, which can help maintain the platelet count and also reduce the size of the spleen. […] Other medications are being developed to treat myelofibrosis, so people may want to search for a clinical trial.

• #30 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  Treatment options include: Allogeneic stem cell transplant, Participation in a clinical trial, Pacritinib (Vonjo) (preferred regimen) or momelotinib (Ojjaara). […] Treatment options include: Allogeneic stem cell transplant, Participation in a clinical trial, Ruxolitinib (Jakafi), fedratinib (Inrebic), momelotinib (Ojjaara), pacritinib (Vonjo). […] For patients with anemia, the treatment approach depends on whether MF symptoms are present and, if present, on how well drug therapy is controlling these symptoms. […] Treatment may include: Participation in a clinical trial, Luspatercept-aamt (Reblozyl), Erythropoiesis (EPO)-stimulating agents (ESA) if EPO level is lower than 500 mU/mL, Momelotinib (Ojjaara), Pacritinib (Vonjo), Receive red blood cell transfusions. […] Allogeneic stem cell transplantation is the only current treatment with the potential to cure MF, but it also carries a high risk of life-threatening side effects.

• #31 Myelofibrosis – Blood Disorders – Merck Manual Consumer Version

  https://www.merckmanuals.com/home/blood-disorders/myeloproliferative-disorders/myelofibrosis

  The combination of an androgen (a medication with the effects of male sex hormones) and prednisone temporarily lessens the severity of the anemia in about one third of people with myelofibrosis, but this treatment is not often used. […] Erythropoietin, a hormone that stimulates red blood cell production, may improve anemia but also causes enlargement of the spleen. […] Sometimes erythropoietin is used together with a JAK inhibitor such as ruxolitinib to prevent enlargement of the spleen. […] Some people with severe anemia require red blood cell transfusions. […] Doctors sometimes also give thalidomide or lenalidomide together with prednisone, which can help maintain the platelet count and also reduce the size of the spleen. […] Other medications are being developed to treat myelofibrosis, so people may want to search for a clinical trial.

• #32 Tests and treatment for myelofibrosis | Cancer Research UK

  https://www.cancerresearchuk.org/about-cancer/myelofibrosis/tests-treatment

  You might have momelotinib to treat an enlarged spleen or symptoms of anaemia. […] Chemotherapy uses cytotoxic drugs to destroy the immature cells. […] Doctors might recommend that you have one of the following: […] Pegylated interferon is a type of immunotherapy. It helps to reduce the rate at which blood cells are made. […] A donor stem cell transplant is usually only suitable for people who: […] Your doctor may suggest you take danazol. […] Radiotherapy is the use of radiation, usually x-rays, to kill cells. […] You might have an operation to remove your spleen (splenectomy) if it is enlarged and other treatments haven’t been able to reduce the size. […] You cant have some treatments for myelofibrosis if you are pregnant. […] Your doctor might ask you to take part in a clinical trial if one is available for your situation. […] Your healthcare team might discuss ways to maintain a healthy lifestyle and help with stopping smoking if you need it. […] We have information on support organisations that can help you cope with the diagnosis and treatment of myelofibrosis.

• #33 Myelofibrosis Treatments, Side Effects, and Alternative Therapies

  https://www.webmd.com/cancer/myelofibrosis-treatments

  With anemia, your body has too few red blood cells. These treatments can help: Androgen therapy. Your doctor might give you a man-made version of the male hormone androgen to help raise red blood cell counts and ease anemia symptoms. It can cause side effects like liver damage, and in women, changes such as body hair growth or hair loss. […] Steroid medications treat severe anemia, but long-term use might cause your bones to thin or raise your blood sugar and blood pressure. […] If MF causes your spleen to swell, you might feel severe pain and bloating. There are a few ways to treat this: Interferon alfa-2a. Injections may help with this condition. […] Yes. Natural approaches could ease your symptoms or just help you manage your disease. While they won’t treat or cure your cancer, they could help you feel better overall. Talk to your doctor before you try any of them.

• #34 Primary Myelofibrosis Diagnosis & Disease Information

  https://www.cancertherapyadvisor.com/ddi/primary-myelofibrosis/

  Other medications used to address the anemia that is frequently associated with primary myelofibrosis (particularly for patients without splenomegaly or constitutional symptoms) include prednisone; danazol; and thalidomide or lenalidomide, with or without prednisone. For patients with severe anemia, erythropoiesis-stimulating agents such as erythropoietin can be used to stimulate the production of red blood cells. However, these medications are typically not recommended as a primary treatment for primary myelofibrosis. […] Interferon-alpha has demonstrated efficacy, particularly in patients with low-risk primary myelofibrosis, but is generally not recommended for those with higher-risk disease. Hydroxyurea may be an option for patients with lower-risk primary myelofibrosis and splenomegaly who do not have anemia or constitutional symptoms.

• #35 Myelofibrosis – Blood Disorders – Merck Manual Consumer Version

  https://www.merckmanuals.com/home/blood-disorders/myeloproliferative-disorders/myelofibrosis

  The combination of an androgen (a medication with the effects of male sex hormones) and prednisone temporarily lessens the severity of the anemia in about one third of people with myelofibrosis, but this treatment is not often used. […] Erythropoietin, a hormone that stimulates red blood cell production, may improve anemia but also causes enlargement of the spleen. […] Sometimes erythropoietin is used together with a JAK inhibitor such as ruxolitinib to prevent enlargement of the spleen. […] Some people with severe anemia require red blood cell transfusions. […] Doctors sometimes also give thalidomide or lenalidomide together with prednisone, which can help maintain the platelet count and also reduce the size of the spleen. […] Other medications are being developed to treat myelofibrosis, so people may want to search for a clinical trial.

• #36 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  Treatment options include: Allogeneic stem cell transplant, Participation in a clinical trial, Pacritinib (Vonjo) (preferred regimen) or momelotinib (Ojjaara). […] Treatment options include: Allogeneic stem cell transplant, Participation in a clinical trial, Ruxolitinib (Jakafi), fedratinib (Inrebic), momelotinib (Ojjaara), pacritinib (Vonjo). […] For patients with anemia, the treatment approach depends on whether MF symptoms are present and, if present, on how well drug therapy is controlling these symptoms. […] Treatment may include: Participation in a clinical trial, Luspatercept-aamt (Reblozyl), Erythropoiesis (EPO)-stimulating agents (ESA) if EPO level is lower than 500 mU/mL, Momelotinib (Ojjaara), Pacritinib (Vonjo), Receive red blood cell transfusions. […] Allogeneic stem cell transplantation is the only current treatment with the potential to cure MF, but it also carries a high risk of life-threatening side effects.

• #37 Primary Myelofibrosis (PMF) – Hematology and Oncology – Merck Manual Professional Edition

  https://www.merckmanuals.com/professional/hematology-and-oncology/myeloproliferative-disorders/primary-myelofibrosis-pmf

  A third JAK-2 inhibitor, pacritinib is available for patients whose platelets counts are too low to initiate ruxolitinib. […] Momelotinib, another JAK-2 inhibitor, may be protective against anemia. […] For patients with advanced disease, allogeneic stem cell transplantation may be beneficial and is the only potentially curative treatment. […] Among agents that are under investigation for treatment of myelofibrosis is luspatercept, an activin receptor ligand trap for treatment of anemia. Many other agents are being evaluated singly or in combination with JAK-2 inhibitors such as anti-Bcl-xL compounds, and phosphatidylinositol-3-kinase inhibitors. New JAK inhibitors are also being tested as first-line therapies or in patients who progress on or do not tolerate currently available JAK inhibitors.

• #39 Tests and treatment for myelofibrosis | Cancer Research UK

  https://www.cancerresearchuk.org/about-cancer/myelofibrosis/tests-treatment

  Your treatment depends on a number of factors including: […] Treatments might include one or more of the following: […] Blood transfusions increase the number of red blood cells. This helps to control tiredness, breathlessness and weakness. […] You take aspirin as a tablet. It can help to reduce the risk of your blood clotting if you are at risk of developing them. […] Allopurinol lowers the amount of uric acid in the blood and so helps to reduce the symptoms of gout. […] Folic acid is a vitamin that helps the body make red blood cells. You may have this if you have anaemia. […] JAK2 inhibitors work by slowing or stopping the growth of cancer cells. […] Ruxolitinib is used to treat an enlarged spleen and other symptoms. […] Some people might have fedratinib to treat an enlarged spleen or other symptoms.

• #40 Treatment Goals in Intermediate or High-Risk Myelofibrosis (MF) | Jakafi® (ruxolitinib)

  https://hcp.jakafi.com/myelofibrosis/treatment-goals

  MF management should focus on achieving 3 key treatment goals, including overall survival. […] When managing patients with MF, my goals include control of the SPLEEN and SYMPTOMS, with an ultimate goal of OVERALL SURVIVAL. […] Jakafi is indicated for treatment of intermediate or high-risk myelofibrosis (MF), including primary MF, postpolycythemia vera MF and postessential thrombocythemia MF in adults. […] When my adult patients with intermediate- to high-risk myelofibrosis are experiencing any degree of splenomegaly at diagnosis, that is a sign that I need to intervene with Jakafi. […] A post hoc pooled analysis of OS with ruxolitinib was performed using data from 2 phase 3 studies: COMFORT-I, a randomized, double-blind, placebo-controlled study with 309 patients with intermediate-2-risk or high-risk MF, and COMFORT-II, a randomized, open-label study with 219 patients with intermediate-2-risk or high-risk MF. The primary endpoint in both studies was the proportion of patients achieving a 35% reduction in spleen volume (measured by CT or MRI) at week 24 in COMFORT-I and at week 48 in COMFORT-II.

• #41 Myelofibrosis – Blood Disorders – Merck Manual Consumer Version

  https://www.merckmanuals.com/home/blood-disorders/myeloproliferative-disorders/myelofibrosis

  Fedratinib, pacritinib, and momelotinib also inhibit the mutations because they also activate JAK2 enzyme. It is effective in reducing spleen size and controlling symptoms and may increase overall survival and slow the rate of fibrosis, but it does not reverse existing fibrosis in most cases and can lead to anemia and low platelet counts. […] Reducing the size of the spleen tends to relieve symptoms. In addition to ruxolitinib, hydroxyurea, a chemotherapy drug, may decrease the size of the liver or spleen but may worsen anemia and lower the platelet count. […] Radiation therapy can decrease the size of the spleen but has only a temporary effect and can cause very low white blood cell counts and infection. […] Rarely, the spleen becomes extremely large and painful and may have to be removed, but spleen removal is recommended only after all other measures have been tried.

• #42 Primary myelofibrosis: current therapeutic options

  https://pmc.ncbi.nlm.nih.gov/articles/PMC4997889/

  Hydroxyurea (HU) is a non-alkylating antineoplastic agent used for cytoreduction in myeloproliferative neoplasms. […] When HU resistance/refractoriness is documented in the PMF proliferative phase, switching from HU to a molecular targeted therapy (i.e., JAK1/2 inhibitor) should promptly be considered. […] Anemia is a frequent manifestation of PMF that might be caused by different interacting factors, such as bone marrow insufficiency (fibrosis), hypersplenism, bleeding, iron deficiency, vitamin B12 or folate deficiency, or autoimmune hemolysis. […] In PMF patients with an unsatisfactory response to pharmacological treatment, splenectomy and splenic radiation may be treatment options, as discussed below. […] Allo-HSCT is currently the only curative treatment option for myelofibrosis patients.

• #43 Myelofibrosis; Treatment, Prevention & Management | Massive Bio

  https://massivebio.com/myelofibrosis-treatment-prevention-management/

  Treatment for myelofibrosis can be done in a variety of ways, depending on the patients symptoms and circumstances. […] Jakafi (ruxolitinib) is the first medicine to be approved by the FDA for the treatment of intermediate or high-risk myelofibrosis. […] For most people with myelofibrosis, the goal of treatment is to alleviate the diseases signs and symptoms. Treatment options include: […] Splenectomy: If your spleen becomes so large that it causes you pain and begins to cause harmful complications and if you dont respond to other forms of therapy you may benefit from having your spleen surgically removed. […] Chemotherapy: Chemotherapy uses powerful drugs to kill cancer cells. Chemotherapy drugs may reduce the size of an enlarged spleen and relieve related symptoms, such as pain. […] Bone Marrow Transplant: Also called a stem cell transplant, is a procedure to replace your diseased bone marrow using healthy blood stem cells. For myelofibrosis, the procedure uses stem cells from a donor (allogeneic stem cell transplant). […] Myelofibrosis cannot be avoided, although allogeneic hematopoietic cell transplantation (HCT) may offer a treatment. The treatment entails replacing a patients immune system with that of an appropriate donor.

• #44 Myelofibrosis | Symptoms and Treatment

  https://patient.info/allergies-blood-immune/polycythaemia-rubra-vera-high-red-blood-cell-count/myelofibrosis

  If myelofibrosis is causing severe anaemia then you may need regular blood transfusions. Taking a medicine similar to the male hormones (androgens) may help your body to make red blood cells and so improve anaemia. […] If an enlarged spleen is causing any complications, you may need surgical removal of your spleen (splenectomy). Splenectomy does have some risks, including infections, excessive bleeding and blood clot formation leading to a stroke or pulmonary embolism (a blood clot in the lung). Chemotherapy or radiotherapy can also be used to reduce the size of the spleen, when surgical removal isn’t an option. […] Stem cell transplant from a suitable donor (allogeneic stem cell transplantation) is the only treatment that has the potential to cure myelofibrosis. Stem cell transplant is also called a bone marrow transplant. But it also has a high risk of life-threatening side-effects. Most people with myelofibrosis, because of age, stability of the disease or other health problems, don’t qualify for this treatment.

• #45 Myelofibrosis – Blood Disorders – Merck Manual Consumer Version

  https://www.merckmanuals.com/home/blood-disorders/myeloproliferative-disorders/myelofibrosis

  Fedratinib, pacritinib, and momelotinib also inhibit the mutations because they also activate JAK2 enzyme. It is effective in reducing spleen size and controlling symptoms and may increase overall survival and slow the rate of fibrosis, but it does not reverse existing fibrosis in most cases and can lead to anemia and low platelet counts. […] Reducing the size of the spleen tends to relieve symptoms. In addition to ruxolitinib, hydroxyurea, a chemotherapy drug, may decrease the size of the liver or spleen but may worsen anemia and lower the platelet count. […] Radiation therapy can decrease the size of the spleen but has only a temporary effect and can cause very low white blood cell counts and infection. […] Rarely, the spleen becomes extremely large and painful and may have to be removed, but spleen removal is recommended only after all other measures have been tried.

• #46 Treatment Options for Myelofibrosis – Massive Bio

  https://massivebio.com/treatment-options-for-myelofibrosis/

  Recombinant erythropoietin: This treatment may be used if a patient has anemia related to low levels of erythropoietin, a hormone that’s necessary for production of red blood cells. […] Anagrelide hydrochloride: If a patient with myelofibrosis has a related condition called thrombocytosis, he or she produces too many blood platelets, which can cause dangerous clots in blood vessels. This drug reduces platelet count. […] This treatment, also called radiotherapy, uses beams of intense energy to shrink tumors and relieve symptoms in many forms of cancer. In myelofibrosis, radiation therapy may be recommended to shrink an enlarged spleen, though some doctors suggest that it should be reserved for patients who can’t undergo surgery to treat the problem. […] Surgical removal of the spleen, or splenectomy, is often considered for patients who develop severe symptoms related to myelofibrosis, which can cause this organ to become enlarged.

• #47 Primary myelofibrosis: current therapeutic options

  https://pmc.ncbi.nlm.nih.gov/articles/PMC4997889/

  Primary myelofibrosis is a Philadelphia-negative myeloproliferative neoplasm characterized by clonal myeloid expansion, followed by progressive fibrous connective tissue deposition in the bone marrow, resulting in bone marrow failure. […] Herein the main current treatment options for primary myelofibrosis patients are discussed, contemplating disease-modifying therapeutics in addition to palliative measures, in an individualized patient-based approach. […] Despite the recent advances in the development of targeted therapies, allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative option available for PMF. […] Evidence on the main therapeutic options for PMF will be discussed in this article. […] The decision regarding the best treatment combination in PMF must be individualized, taking the symptoms, risks and life expectation of each patient into account.

• #48 Myelofibrosis: Symptoms, Types, Prognosis & Treatment

  https://my.clevelandclinic.org/health/diseases/15672-myelofibrosis

  Treatments for anemia include: […] Treatments used to manage an enlarged spleen include JAK inhibitors, immunomodulators and chemotherapy drugs. In severe cases, you may need to have your spleen removed (splenectomy), or you may need radiation therapy to your spleen. […] You may need radiation therapy to treat extramedullary hematopoiesis (abnormal growth of stem cells outside of your bone marrow). […] Allogeneic hematopoietic cell transplantation (HCT) is a potential cure, but its a risky procedure that may not be an option for everyone. […] The procedure involves replacing abnormal blood cells with healthy cells from a donor. Before the transplant, youll receive chemotherapy or radiation therapy to destroy your diseased cells so the new cells from the donor will take over. […] HCT carries a high risk of complications and is only suitable for certain people. The risk of a complication is higher for people with other medical conditions. Various factors will determine whether youre a candidate, including your age, the severity of your symptoms and your likelihood of success.

• #49 Myelofibrosis: Symptoms, Types, Prognosis & Treatment

  https://my.clevelandclinic.org/health/diseases/15672-myelofibrosis

  Treatments for anemia include: […] Treatments used to manage an enlarged spleen include JAK inhibitors, immunomodulators and chemotherapy drugs. In severe cases, you may need to have your spleen removed (splenectomy), or you may need radiation therapy to your spleen. […] You may need radiation therapy to treat extramedullary hematopoiesis (abnormal growth of stem cells outside of your bone marrow). […] Allogeneic hematopoietic cell transplantation (HCT) is a potential cure, but its a risky procedure that may not be an option for everyone. […] The procedure involves replacing abnormal blood cells with healthy cells from a donor. Before the transplant, youll receive chemotherapy or radiation therapy to destroy your diseased cells so the new cells from the donor will take over. […] HCT carries a high risk of complications and is only suitable for certain people. The risk of a complication is higher for people with other medical conditions. Various factors will determine whether youre a candidate, including your age, the severity of your symptoms and your likelihood of success.

• #50 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  Reduced-intensity or nonmyeloablative allogeneic stem cell transplantation is a type of transplant that is being used to treat some patients with MF. […] Patients should talk with their doctors about whether stem cell transplantation is a treatment option for them. […] Taking part in a clinical trial may be the best treatment choice for some myelofibrosis (MF) patients. […] Certain factors can affect a patients prognosis (a persons chance of recovery or the likely outcome of their disease). […] These factors help doctors plan the most appropriate treatment for each patient. […] It is important for patients and the members of their medical team to discuss all treatment options, including treatments being studied in clinical trials.

• #51 Myelofibrosis: Causes, Symptoms, Treatment, and More

  https://www.healthline.com/health/myelofibrosis

  Researchers are working on developing new treatments for MF. While many of these require further study to make sure they’re safe, doctors have started using two new treatments in certain cases: Stem cell transplant: Stem cell transplants have the potential to cure MF and restore bone marrow function. However, the procedure can cause life-threatening complications, so it’s usually only done when nothing else works. Interferon-alpha: Interferon-alfa has delayed the formation of scar tissue in the bone marrow of people who receive treatment early on, but more research is needed to determine its long-term safety. […] There isn’t one single treatment that works the same for every person or every type of MF. The only true cure involves a stem cell transplant, but this can also cause complications and needs more research on its safety.

• #52 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  Reduced-intensity or nonmyeloablative allogeneic stem cell transplantation is a type of transplant that is being used to treat some patients with MF. […] Patients should talk with their doctors about whether stem cell transplantation is a treatment option for them. […] Taking part in a clinical trial may be the best treatment choice for some myelofibrosis (MF) patients. […] Certain factors can affect a patients prognosis (a persons chance of recovery or the likely outcome of their disease). […] These factors help doctors plan the most appropriate treatment for each patient. […] It is important for patients and the members of their medical team to discuss all treatment options, including treatments being studied in clinical trials.

• #53 Next Generation Therapeutics for the Treatment of Myelofibrosis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8146033/

  Despite the undeniable improvements in patient quality of life with the advent of JAKi, there remains several unmet needs for patients with MF. For one, JAK inhibition leads to on-target myelosuppression, as the erythropoietin and thrombopoietin receptors are JAK-STAT dependent. […] A major limitation to JAKi therapy in MF is hematologic TEAE including anemia and thrombocytopenia. However, there are novel JAKi in development which may be useful in these challenging scenarios. […] Therapeutic options after ruxolitinib failure are unfortunately limited. […] Therefore, novel therapies are being explored that act on pathways outside the JAK-STAT pathway. […] In this review, we describe novel therapies for MF outside of JAKi. […] CPI-0610 (Constellation Pharmaceuticals, Cambridge, MA) is a potent BET inhibitor in development for MF.

• #54 The Latest in Myelofibrosis: Understanding Promising Treatment Options

  https://thepatientstory.com/patient-events/myelofibrosis-patient-events/myelofibrosis-treatments-ash-2023-update/

  The myelofibrosis panelists discuss momelotinib, fedratinib, selinexor, pelabresib, navitoclax, and other myelofibrosis treatments. […] It’s exciting. I can’t remember participating in an ASH conference where there were two phase 3 studies presented about myelofibrosis, the navitoclax and pelabresib studies. That in and of itself was a reflection of how far the field has come. We’re now presenting phase 3 studies on drugs that may get approved. […] The combination (ruxolitinib and navitoclax) led to a very dramatic improvement in spleen volume reduction in patients compared to patients who were treated with ruxolitinib alone. […] About pelabresib, this is a study of the combination of pelabresib, which is a BET inhibitor, plus ruxolitinib versus ruxolitinib alone. The study looked at whether patients had a greater degree of spleen shrinkage with the combination or ruxolitinib alone. The answer was unequivocally yes. Patients who got the combination had a much greater spleen response, 65% versus 30%.

• #55 Next Generation Therapeutics for the Treatment of Myelofibrosis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8146033/

  Bomedemstat (IMG-7289, Imago Biosciences, San Francisco, CA, USA) is an irreversible inhibitor of LSD1. […] PRM-151 (Roche, Basel, Switzerland) is a recombinant human pentraxin-2 which functions as an anti-fibrotic agent in preclinical models of fibrosis and has been in clinical testing for idiopathic pulmonary fibrosis. […] AVID200 (Forbius, Austin, TX, USA) is a TGF1 trap which binds TGF1 and TGF3, but not TGF2 (which is a positive regulator of hematopoiesis). […] Imetelstat (Geron, Forest City, CA, USA) is an oligonucleotide complimentary to the template region of telomerase and binds with high affinity, resulting in competitive inhibition. […] Given these encouraging results, the multicenter, randomized phase 2 study (Imbark) was performed with two dose levels of imetelstat. […] JAKi have undoubtedly improved the lives of patients with MF. However, most patients will discontinue ruxolitinib. […] As a result, current therapeutic development in MF is focused on targets outside the JAK-STAT pathway.

• #56 How We Treat Myeloproliferative Neoplasms (MPNs) | Dana-Farber Cancer Institute

  https://www.dana-farber.org/cancer-care/types/myeloproliferative-neoplasms/treatment

  Treatment for Myelofibrosis varies based on how aggressive the disease is, and the symptoms or complications the patient experiences: […] Patients with intermediate- and high-risk myelofibrosis who experience symptoms are usually treated with JAK2 inhibitors, medications that aim to stop overproduction of blood cells. […] Allogeneic stem cell transplantation is also a treatment option for intermediate- and high-risk patients. […] We may offer patients with myelofibrosis treatment through a clinical trial that is evaluating new therapies for the disorder. […] Current trials explore the use of drugs that cause cell death (apoptosis) to treat patients with myelofibrosis. […] Jacqueline Garcia, MD, is leading trials of navitoclax, an oral, small molecule inhibitor targeting BCL-XL/BCL-2, in combination with ruxolitinib for patients with myelofibrosis. […] Identifying novel biomarkers of myelofibrosis to understand the key mechanisms that drive myelofibrosis.

• #57 The Latest in Myelofibrosis: Understanding Promising Treatment Options

  https://thepatientstory.com/patient-events/myelofibrosis-patient-events/myelofibrosis-treatments-ash-2023-update/

  The myelofibrosis panelists discuss momelotinib, fedratinib, selinexor, pelabresib, navitoclax, and other myelofibrosis treatments. […] It’s exciting. I can’t remember participating in an ASH conference where there were two phase 3 studies presented about myelofibrosis, the navitoclax and pelabresib studies. That in and of itself was a reflection of how far the field has come. We’re now presenting phase 3 studies on drugs that may get approved. […] The combination (ruxolitinib and navitoclax) led to a very dramatic improvement in spleen volume reduction in patients compared to patients who were treated with ruxolitinib alone. […] About pelabresib, this is a study of the combination of pelabresib, which is a BET inhibitor, plus ruxolitinib versus ruxolitinib alone. The study looked at whether patients had a greater degree of spleen shrinkage with the combination or ruxolitinib alone. The answer was unequivocally yes. Patients who got the combination had a much greater spleen response, 65% versus 30%.

• #58 Next Generation Therapeutics for the Treatment of Myelofibrosis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8146033/

  Bomedemstat (IMG-7289, Imago Biosciences, San Francisco, CA, USA) is an irreversible inhibitor of LSD1. […] PRM-151 (Roche, Basel, Switzerland) is a recombinant human pentraxin-2 which functions as an anti-fibrotic agent in preclinical models of fibrosis and has been in clinical testing for idiopathic pulmonary fibrosis. […] AVID200 (Forbius, Austin, TX, USA) is a TGF1 trap which binds TGF1 and TGF3, but not TGF2 (which is a positive regulator of hematopoiesis). […] Imetelstat (Geron, Forest City, CA, USA) is an oligonucleotide complimentary to the template region of telomerase and binds with high affinity, resulting in competitive inhibition. […] Given these encouraging results, the multicenter, randomized phase 2 study (Imbark) was performed with two dose levels of imetelstat. […] JAKi have undoubtedly improved the lives of patients with MF. However, most patients will discontinue ruxolitinib. […] As a result, current therapeutic development in MF is focused on targets outside the JAK-STAT pathway.

• #59 Next Generation Therapeutics for the Treatment of Myelofibrosis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8146033/

  Bomedemstat (IMG-7289, Imago Biosciences, San Francisco, CA, USA) is an irreversible inhibitor of LSD1. […] PRM-151 (Roche, Basel, Switzerland) is a recombinant human pentraxin-2 which functions as an anti-fibrotic agent in preclinical models of fibrosis and has been in clinical testing for idiopathic pulmonary fibrosis. […] AVID200 (Forbius, Austin, TX, USA) is a TGF1 trap which binds TGF1 and TGF3, but not TGF2 (which is a positive regulator of hematopoiesis). […] Imetelstat (Geron, Forest City, CA, USA) is an oligonucleotide complimentary to the template region of telomerase and binds with high affinity, resulting in competitive inhibition. […] Given these encouraging results, the multicenter, randomized phase 2 study (Imbark) was performed with two dose levels of imetelstat. […] JAKi have undoubtedly improved the lives of patients with MF. However, most patients will discontinue ruxolitinib. […] As a result, current therapeutic development in MF is focused on targets outside the JAK-STAT pathway.

• #60

  https://link.springer.com/article/10.1007/s11899-024-00729-8

  Combination treatment with ruxolitinib and pegylated interferon alfa-2a can also be used for MF, mainly in patients without splenomegaly or symptoms. […] The clinical benefit of initiating treatment with ruxolitinib in patients with int-2 or high-risk MF as soon as possible after diagnosis, rather than delaying treatment, has been demonstrated in the COMFORT studies as well as in other clinical trials and real-world studies. […] Taken together, there is little to no benefit to delaying active treatment in patients with symptomatic lower-risk or int-2/high-risk MF.

• #61 Next Generation Therapeutics for the Treatment of Myelofibrosis

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8146033/

  Despite the undeniable improvements in patient quality of life with the advent of JAKi, there remains several unmet needs for patients with MF. For one, JAK inhibition leads to on-target myelosuppression, as the erythropoietin and thrombopoietin receptors are JAK-STAT dependent. […] A major limitation to JAKi therapy in MF is hematologic TEAE including anemia and thrombocytopenia. However, there are novel JAKi in development which may be useful in these challenging scenarios. […] Therapeutic options after ruxolitinib failure are unfortunately limited. […] Therefore, novel therapies are being explored that act on pathways outside the JAK-STAT pathway. […] In this review, we describe novel therapies for MF outside of JAKi. […] CPI-0610 (Constellation Pharmaceuticals, Cambridge, MA) is a potent BET inhibitor in development for MF.

• #62 The Latest in Myelofibrosis: Understanding Promising Treatment Options

  https://thepatientstory.com/patient-events/myelofibrosis-patient-events/myelofibrosis-treatments-ash-2023-update/

  The myelofibrosis panelists discuss momelotinib, fedratinib, selinexor, pelabresib, navitoclax, and other myelofibrosis treatments. […] It’s exciting. I can’t remember participating in an ASH conference where there were two phase 3 studies presented about myelofibrosis, the navitoclax and pelabresib studies. That in and of itself was a reflection of how far the field has come. We’re now presenting phase 3 studies on drugs that may get approved. […] The combination (ruxolitinib and navitoclax) led to a very dramatic improvement in spleen volume reduction in patients compared to patients who were treated with ruxolitinib alone. […] About pelabresib, this is a study of the combination of pelabresib, which is a BET inhibitor, plus ruxolitinib versus ruxolitinib alone. The study looked at whether patients had a greater degree of spleen shrinkage with the combination or ruxolitinib alone. The answer was unequivocally yes. Patients who got the combination had a much greater spleen response, 65% versus 30%.

• #63 The Latest in Myelofibrosis: Understanding Promising Treatment Options

  https://thepatientstory.com/patient-events/myelofibrosis-patient-events/myelofibrosis-treatments-ash-2023-update/

  The myelofibrosis panelists discuss momelotinib, fedratinib, selinexor, pelabresib, navitoclax, and other myelofibrosis treatments. […] It’s exciting. I can’t remember participating in an ASH conference where there were two phase 3 studies presented about myelofibrosis, the navitoclax and pelabresib studies. That in and of itself was a reflection of how far the field has come. We’re now presenting phase 3 studies on drugs that may get approved. […] The combination (ruxolitinib and navitoclax) led to a very dramatic improvement in spleen volume reduction in patients compared to patients who were treated with ruxolitinib alone. […] About pelabresib, this is a study of the combination of pelabresib, which is a BET inhibitor, plus ruxolitinib versus ruxolitinib alone. The study looked at whether patients had a greater degree of spleen shrinkage with the combination or ruxolitinib alone. The answer was unequivocally yes. Patients who got the combination had a much greater spleen response, 65% versus 30%.

• #64 Myelofibrosis Treatment in 2024: New Horizons and Innovations

  https://www.delveinsight.com/blog/myelofibrosis-treatment-landscape

  Currently approved therapies are associated with significant adverse effects. […] JAK inhibitors have minimal effects on achieving complete hematological remission and normalizing blood counts without strong evidence of disease-modifying effects. These obstacles underscore the necessity for alternative therapies for myelofibrosis beyond JAK inhibitors. […] Although existing JAK inhibitors prescribed for myelofibrosis treatment have demonstrated considerable clinical advantages by reducing symptoms and reducing spleen enlargement, they have not exhibited definitive disease-modifying effects, leading to most patients discontinuing the medication within one to two years. […] Different mechanisms other than JAK inhibitors under investigation are telomerase inhibitor (imetelstat), BCL-XL/BCL-2 inhibitor (navitoclax), MDM2 protein inhibitor (navtemadlin), BET inhibitor (pelabresib), and others which may cover the patient need and provide an alternative myelofibrosis treatment for the patients.

• #65 Tests and treatment for myelofibrosis | Cancer Research UK

  https://www.cancerresearchuk.org/about-cancer/myelofibrosis/tests-treatment

  Your treatment depends on a number of factors including: […] Treatments might include one or more of the following: […] Blood transfusions increase the number of red blood cells. This helps to control tiredness, breathlessness and weakness. […] You take aspirin as a tablet. It can help to reduce the risk of your blood clotting if you are at risk of developing them. […] Allopurinol lowers the amount of uric acid in the blood and so helps to reduce the symptoms of gout. […] Folic acid is a vitamin that helps the body make red blood cells. You may have this if you have anaemia. […] JAK2 inhibitors work by slowing or stopping the growth of cancer cells. […] Ruxolitinib is used to treat an enlarged spleen and other symptoms. […] Some people might have fedratinib to treat an enlarged spleen or other symptoms.

• #66 Tests and treatment for myelofibrosis | Cancer Research UK

  https://www.cancerresearchuk.org/about-cancer/myelofibrosis/tests-treatment

  Your treatment depends on a number of factors including: […] Treatments might include one or more of the following: […] Blood transfusions increase the number of red blood cells. This helps to control tiredness, breathlessness and weakness. […] You take aspirin as a tablet. It can help to reduce the risk of your blood clotting if you are at risk of developing them. […] Allopurinol lowers the amount of uric acid in the blood and so helps to reduce the symptoms of gout. […] Folic acid is a vitamin that helps the body make red blood cells. You may have this if you have anaemia. […] JAK2 inhibitors work by slowing or stopping the growth of cancer cells. […] Ruxolitinib is used to treat an enlarged spleen and other symptoms. […] Some people might have fedratinib to treat an enlarged spleen or other symptoms.

• #67 Tests and treatment for myelofibrosis | Cancer Research UK

  https://www.cancerresearchuk.org/about-cancer/myelofibrosis/tests-treatment

  Your treatment depends on a number of factors including: […] Treatments might include one or more of the following: […] Blood transfusions increase the number of red blood cells. This helps to control tiredness, breathlessness and weakness. […] You take aspirin as a tablet. It can help to reduce the risk of your blood clotting if you are at risk of developing them. […] Allopurinol lowers the amount of uric acid in the blood and so helps to reduce the symptoms of gout. […] Folic acid is a vitamin that helps the body make red blood cells. You may have this if you have anaemia. […] JAK2 inhibitors work by slowing or stopping the growth of cancer cells. […] Ruxolitinib is used to treat an enlarged spleen and other symptoms. […] Some people might have fedratinib to treat an enlarged spleen or other symptoms.

• #68 Myelofibrosis | Canadian Cancer Society

  https://cancer.ca/en/cancer-information/cancer-types/leukemia/what-is-leukemia/myeloproliferative-neoplasms/myelofibrosis

  Anagrelide (Agrylin) may be used to lower a high platelet count. […] Androgen therapy, such as danazol (Cyclomen), is treatment with male hormones that may be used to increase the number of red blood cells. […] Peginterferon alfa (Pegasys) is a cytokine used to boost the immune system to fight the disease. […] Growth factors, such as epoetin alfa (Eprex), may be used to increase the number of red blood cells. […] Bisphosphonates stop the body from breaking down bone, strengthen bone and help manage bone pain. You may be given zoledronic acid (Zometa) if you have myelofibrosis. […] You may be offered blood transfusions to treat severe anemia. […] Surgery to remove the spleen (called a splenectomy) is rarely done. It could be considered if an enlarged spleen is causing pain and drug therapy has not worked. […] An allogeneic stem cell transplant may be a treatment option for younger people who have advanced myelofibrosis.

• #69 Myelofibrosis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/myelofibrosis/diagnosis-treatment/drc-20355062

  A bone marrow transplant, also called a bone marrow stem cell transplant, involves putting healthy bone marrow stem cells into the body. These cells replace cells hurt by chemotherapy and other treatments. […] This treatment has the potential to cure myelofibrosis. It also has a high risk of life-threatening side effects. This treatment isn’t an option for everyone with myelofibrosis. […] Palliative care is a special type of healthcare that helps people with serious illnesses feel better. If you have cancer, palliative care can help relieve pain and other symptoms. […] When palliative care is used along with all of the other treatments, people with cancer may feel better and live longer.

• #70 https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  https://www.lls.org/myeloproliferative-neoplasms/myelofibrosis/treatment

  Reduced-intensity or nonmyeloablative allogeneic stem cell transplantation is a type of transplant that is being used to treat some patients with MF. […] Patients should talk with their doctors about whether stem cell transplantation is a treatment option for them. […] Taking part in a clinical trial may be the best treatment choice for some myelofibrosis (MF) patients. […] Certain factors can affect a patients prognosis (a persons chance of recovery or the likely outcome of their disease). […] These factors help doctors plan the most appropriate treatment for each patient. […] It is important for patients and the members of their medical team to discuss all treatment options, including treatments being studied in clinical trials.

• #71 Myelofibrosis Treatment in 2024: New Horizons and Innovations

  https://www.delveinsight.com/blog/myelofibrosis-treatment-landscape

  Currently approved therapies are associated with significant adverse effects. […] JAK inhibitors have minimal effects on achieving complete hematological remission and normalizing blood counts without strong evidence of disease-modifying effects. These obstacles underscore the necessity for alternative therapies for myelofibrosis beyond JAK inhibitors. […] Although existing JAK inhibitors prescribed for myelofibrosis treatment have demonstrated considerable clinical advantages by reducing symptoms and reducing spleen enlargement, they have not exhibited definitive disease-modifying effects, leading to most patients discontinuing the medication within one to two years. […] Different mechanisms other than JAK inhibitors under investigation are telomerase inhibitor (imetelstat), BCL-XL/BCL-2 inhibitor (navitoclax), MDM2 protein inhibitor (navtemadlin), BET inhibitor (pelabresib), and others which may cover the patient need and provide an alternative myelofibrosis treatment for the patients.

• #72 When to Initiate Treatment in Myelofibrosis – Hematology & Oncology

  https://www.hematologyandoncology.net/archives/november-2016/when-to-initiate-treatment-in-myelofibrosis/

  Given that ruxolitinib is a JAK1/JAK2 inhibitor, it might be expected that only patients with the JAK mutation would respond to treatment. Studies show, however, that patients respond regardless of their phenotypic driver mutation; no particular group responds better or worse. […] There is no evidence that patients with low-risk disease benefit from earlier intervention with a specific treatment. For patients with intermediate-2 and high-risk disease, data from the COMFORT studies suggest that earlier treatment with ruxolitinib might be beneficial. […] Myelofibrosis is a rapidly growing field, with new data appearing all the time. There is increasing interest in the JAK inhibitors and in therapies that target other facets of the disease.

• #73 Myelofibrosis – MPN Research Foundation

  https://mpnresearchfoundation.org/primary-myelofibrosis-pmf/

  The risk of thrombosis may be managed with low-dose aspirin therapy or hydroxyurea. […] Non-liver and spleen extramedullary hematopoiesis may be treated with radiation therapy. […] Constitutional symptoms, such as night sweats, pruritus, weight loss, and fever may be treated with Jakafi. […] For many patients with MF, available treatment approaches may not be effective, and experimental treatments (which involve receiving a novel drug or treatment on a clinical trial), may be an appropriate option. […] There are many novel therapies currently in clinical trial, including multiple mechanisms of action: […] A number of other drugs that inhibit JAK2 (JAK inhibitors) are currently in clinical trials. […] Epigenetic drugs change the way genes are organized to make them more or less accessible for use by the cell. […] Pomalidomide has been shown to effectively treat anemia in early studies. […] Everolimus (also known as RAD001) is an inhibitor of the mTOR/AKT pathway, which is highly active in MF blood producing cells and appears to contribute to abnormal cell growth.

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