The first 60 days: Primary myelofibrosis
Primary myelofibrosis is a blood cancer in which the marrow scars over, the spleen swells and patients become anaemic and exhausted. JAK inhibitors, ruxolitinib first (COMFORT) and then fedratinib, pacritinib and momelotinib (MOMENTUM), shrink the spleen and relieve symptoms; only a donor stem cell transplant can cure it. Below, week by week, is what OnCo's record of Primary myelofibrosis says about the first two months: the order is typical, the timing is yours to ask about. Sections appear only where the record has something to say. Orientation, not medical advice.
What happens now
Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.
Ask which of these were tested and what the results were; see the report reader for what each value means.
These specialties appear in the standard of care for this cancer. In most centres they meet weekly as a tumour board to agree each plan; you can ask when yours was discussed and what was decided.
- Medical oncologistNamed in the standard of care for: Lower-risk, asymptomatic, Symptomatic splenomegaly or constitutional symptoms, Anaemia of myelofibrosis, Higher-risk, fit for transplant and 1 more.
- Transplant and cell therapy teamNamed in the standard of care for: Symptomatic splenomegaly or constitutional symptoms, Higher-risk, fit for transplant.
- Palliative and supportive care teamNamed in the standard of care for: Anaemia of myelofibrosis, Ruxolitinib failure and trials.
A second opinion from a centre that treats many similar cases is normal, not rude; the standard of care tells you what to compare it against.
Each row is a setting from the standard of care, in the order it usually arises. Not all will apply to you; your stage and biomarkers decide which do. The guideline grade, where recorded, says how strong the evidence is.
Observation; aspirin for thrombosis risk where platelets are high; hydroxyurea or interferon for proliferative features.
Ruxolitinib (COMFORT-I and II) first; fedratinib after ruxolitinib failure or intolerance; pacritinib if platelets are below 50 x 10^9/L; momelotinib if anaemic.
Momelotinib (MOMENTUM), erythropoiesis-stimulating agents where erythropoietin is low, danazol, transfusion with iron chelation; luspatercept in trials (INDEPENDENCE).
MomelotinibEpoetin alfaTransfusion support and anaemia managementLuspaterceptAn Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on ConcomiStudy of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and AnemiaAllogeneic stem cell transplant, usually under 70 and at MIPSS70 high or DIPSS intermediate-2 or higher, after JAK inhibitor to reduce spleen size.
Switch JAK inhibitor; combination trials of ruxolitinib with navitoclax, pelabresib, selinexor or imetelstat; imetelstat versus best available therapy in IMpactMF.
NavitoclaxPelabresibSelinexorImetelstatStudy of Oral Navitoclax Tablet in Combination With Oral Ruxolitinib Tablet Versus Best Available Therapy to Assess Change in Spleen Volume in Adult Participants With Relapsed/Refractory MyelofibrosisStudy of Selinexor in Combination With Ruxolitinib in MyelofibrosisA Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor TreatmentExtended Access of Momelotinib in Adults With Myelofibrosis
Generated from this cancer's standard of care, biomarkers and open problems. Take the group that matches where you are. To tick, add your own and print, open the one-page appointment sheet.
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example JAK2 V617F, CALR and MPL driver mutations, High-molecular-risk mutations: ASXL1, SRSF2, EZH2, IDH1/2, U2AF1, DIPSS-plus and MIPSS70-plus v2 scores, Marrow fibrosis grade, Spleen volume by imaging), and what were the results?These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Recognised subtypes for this cancer include Prefibrotic primary myelofibrosis, Overt primary myelofibrosis, lower risk, Overt primary myelofibrosis, higher risk.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Lower-risk, asymptomatic
- For my situation (lower-risk, asymptomatic), which of the standard options do you recommend and why?Guideline options include: Observation; aspirin for thrombosis risk where platelets are high; hydroxyurea or interferon for proliferative features.
- Am I a candidate for Aspirin, Hydroxyurea (hydroxycarbamide), Ropeginterferon alfa-2b or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Symptomatic splenomegaly or constitutional symptoms
- For my situation (symptomatic splenomegaly or constitutional symptoms), which of the standard options do you recommend and why?Guideline options include: Ruxolitinib (COMFORT-I and II) first; fedratinib after ruxolitinib failure or intolerance; pacritinib if platelets are below 50 x 10^9/L; momelotinib if anaemic.
- Am I a candidate for Ruxolitinib, Fedratinib, Pacritinib or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Anaemia of myelofibrosis
- For my situation (anaemia of myelofibrosis), which of the standard options do you recommend and why?Guideline options include: Momelotinib (MOMENTUM), erythropoiesis-stimulating agents where erythropoietin is low, danazol, transfusion with iron chelation; luspatercept in trials (INDEPENDENCE).
- Am I a candidate for Momelotinib, Epoetin alfa, Luspatercept, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomi and Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Higher-risk, fit for transplant
- For my situation (higher-risk, fit for transplant), which of the standard options do you recommend and why?Guideline options include: Allogeneic stem cell transplant, usually under 70 and at MIPSS70 high or DIPSS intermediate-2 or higher, after JAK inhibitor to reduce spleen size.
- Am I a candidate for Ruxolitinib, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Ruxolitinib failure and trials
- For my situation (ruxolitinib failure and trials), which of the standard options do you recommend and why?Guideline options include: Switch JAK inhibitor; combination trials of ruxolitinib with navitoclax, pelabresib, selinexor or imetelstat; imetelstat versus best available therapy in IMpactMF.
- Am I a candidate for Navitoclax, Pelabresib, Selinexor or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of Study of Oral Navitoclax Tablet in Combination With Oral Ruxolitinib Tablet Versus Best Available Therapy to Assess Change in Spleen Volume in Adult Participants With Relapsed/Refractory Myelofibrosis and Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Any stage
- Are there clinical trials I could join, for example of Momelotinib, Pacritinib, Navitoclax, Pelabresib?Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Supportive care improves quality of life and helps patients complete treatment.
- I read that “No drug has been shown to change the course of the disease rather than its symptoms”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Transplant timing: too early risks a fatal procedure in someone with years to live, too late loses the window”. How does that affect my plan?Open problems are where trials and second opinions matter most.
Trials open now for this cancer in OnCo, largest phase first. Joining a trial is a decision like any other: ask what the comparison arm is, whether a placebo is used, and what happens if you leave. The cancer page searches ClinicalTrials.gov live for more.
- A Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor TreatmentPhase 3 · active · NCT04576156A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients With Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK) Inhibitor
- An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on ConcomiPhase 3 · active · NCT04717414A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK Inhibitor Therapy and Who Require Red Blood Cell Transfusions
- Study of Oral Navitoclax Tablet in Combination With Oral Ruxolitinib Tablet Versus Best Available Therapy to Assess Change in Spleen Volume in Adult Participants With Relapsed/Refractory MyelofibrosisPhase 3 · active · NCT04468984Randomized, Open-Label, Phase 3 Study Evaluating Efficacy and Safety of Navitoclax in Combination With Ruxolitinib Versus Best Available Therapy in Subjects With Relapsed/Refractory Myelofibrosis (TRANSFORM-2), Incorporating Extension Arm C - Continued Access for Navitoclax to Roll Over Subjects From Studies M10-166, M16-109, M16-191, and M19-753
- Study of Selinexor in Combination With Ruxolitinib in MyelofibrosisPhase 3 · active · NCT04562389A Phase 1/3 Study to Evaluate Efficacy and Safety of Selinexor, a Selective Inhibitor of Nuclear Export, in Combination With Ruxolitinib in Treatment-naïve Patients With Myelofibrosis
- Extended Access of Momelotinib in Adults With MyelofibrosisPhase 2 · active · NCT03441113Extended Access of Momelotinib for Subjects With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- Primary myelofibrosis: the full pagePrimary myelofibrosis is a blood cancer in which the marrow scars over, the spleen swells and patients become anaemic and exhausted. JAK inhibitors, ruxolitinib first (COMFORT) and then fedratinib, pacritinib and momelotinib (MOMENTUM), shrink the spleen and relieve symptoms; only a donor stem cell transplant can cure it.
- One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
- Treatment sequencingWhich treatment tends to follow which, line by line.
- NavigatorStandard of care for your stage, what you have tried, and trials near you.
- Anaemia: A shortage of red blood cells or haemoglobin, causing tiredness and breathlessness.
- Conditioning regimen (myeloablative, reduced-intensity): The chemotherapy (with or without whole-body radiation) given in the days before a stem cell transplant to destroy the diseased marrow and, for donor transplants, suppress the patient's immune system so the graft is not rejected.
- JAK2 V617F: A single letter change in the JAK2 gene that jams the growth signal for blood cells in the on position.
- Post-PV myelofibrosis (spent phase): The late stage some people with polycythaemia vera reach after many years, when the marrow scars over, the red count falls and the spleen swells.
- Allogeneic stem cell transplant (allo-SCT): Replacing a patient's blood system with a donor's: chemotherapy wipes out the marrow, donor stem cells rebuild it, and the donor's immune cells hunt down leftover leukaemia.
Every term links to the glossary.