Myelofibrosis
Myelofibrosis (MF) is a rare blood cancer that leads to scarring in the bone marrow, which prevents the normal production of blood cells. The condition is a primary subtype of myeloproliferative neoplasms (MPNs) and, in some cases, starts as another MPN. Risk factors for primary MF are unclear, although a history of polycythemia vera (PV) or essential thrombocythemia (ET) are risk factors for development of secondary MF. The disease is stratified as low, intermediate, or high risk using various International Prognostic Scoring System scales, and the prognosis depends on individual risk factors including age, comorbidities, and treatment response.
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A real-world retrospective study evaluated transfusion dependent or nondependent patients at ruxolitinib initiation.
Management of asymptomatic, clinically stable disease is not necessarily improved by implementing routine hemostasis tests.
Investigational agent DISC-0974 produced anemia response independent of patient transfusion dependency or JAK inhibitor use.
A study observed that addition of pelabresib to ruxolitinib led to bone marrow microenvironment improvement.
An expert on myelofibrosis comments on promising results from current trials and on the need for useful clinical biomarkers.
History of thrombotic events, hematocrit ≤0.45 L/L, and JAK2 p.V617F were identified as risk factors for progression.
LOXL2 upregulation is associated with key inflammatory signaling pathways in primary myelofibrosis.
Treatment with selinexor reduced hepcidin and pro-inflammatory cytokines in patients with myelofibrosis.
The rates of baseline comorbidities and constitutional symptoms were higher in patients with anemia versus those without.
New or worsening anemia following initiation of ruxolitinib does not appear to diminish clinical benefit.
Hemoglobin improvement at week 24 after transfusion is associated with improved HRQOL in myelofibrosis and anemia.
Prospective analysis suggest that 4.3% of patients with ET progressed to myelofibrosis over five years of follow up.
Patients with MF or ET with CALR or JAK2 mutations carried similar symptom burdens according to prospective data.
The phase III MANIFEST-2 trial compared pelabresib plus ruxolitinib with placebo plus ruxolitinib in JAKi-naive patients.
The findings were from a retrospective study of patient data from the US Flatiron Health electronic health record database.
A retrospective analysis covered two phase III trials of the JAK2 inhibitor in patients with and without monocytosis.
A study evaluated progression according to hemoglobin levels, platelet counts, constitutional symptoms, and other markers.
CTLA-4 blockade increased the cytotoxic T-cell–mediated immune response against myelofibrosis cells in human xenografts.
A Markov model has compared delayed with immediate performance of allogeneic HSCT following ruxolitinib failure in PMF.
Using mass spectrometry-based proteomics, chemokine platelet factor 4 (PF4) was identified as a driver of myelofibrosis.
New therapies, such as TP-3654, are currently being explored in combination studies.
Three of four patients with myelofibrosis had objective responses at one year, and no dose-limiting toxicities were observed.
No significant differences in HSCT were observed, suggesting haploidentical HSCT as an acceptable treatment approach.
AI-driven quantitative analysis was compared with manual evaluation of bone marrow trephine slides in a phase II trial.
Julie Braish, MBBCh, discusses her study on JAK2 allele burden in myelofibrosis presented at ASCO 2024.
Dr. Rampal, of Memorial Sloan Kettering Cancer Center, discusses his abstract at the EHA 2024 Congress in Madrid, Spain.
MPN depend on mutated JAK signaling, and targeting the mutations suppressed disease features and improved overall survival.
Pelabresib plus ruxolitinib improved spleen and symptom responses in JAKi-naïve patients with myelofibrosis.
Treatment with luspatercept improved transfusion burden and anemia across four cohorts of patients with myelofibrosis.
Prefibrotic PMF is an MPN with distinct characteristics comprising histopathological, clinical, and biological parameters.
Prefibrotic myelofibrosis is the focus of “SOHO State of the Art Updates and Next Questions.”
Momelotinib achieved higher rates of transfusion independence compared with ruxolitinib.
Splenic irradiation before HSCT is associated with reduced spleen size and lower incidence of relapse in patients with MF.
Sotatercept monotherapy, and combined with ruxolitinib, is a safe and effective treatment for patients with PMF and anemia.
The primary endpoint was overall best response after blast-reduction therapy.
Momelotinib and ruxolitinib improved bone marrow fibrosis, but changes were not associated with improved outcomes in MF.
Nicolaus Kröger, MD, discusses a study on GVHD and its impact on relapse in patients with myelofibrosis undergoing HSCT.
Myelofibrosis, a rare blood cancer, affects four to six per 100,000 individuals in the United States.
An international working group collaborating with the EBMT and ELN updated the 2015 guidelines for HSCT in myelofibrosis.
Ruxolitinib plus pelabresib was well tolerated and improved spleen and symptom burden in patients with myelofibrosis.
Parsaclisib plus ruxolitinib can improve symptoms and spleen volume in certain patients with myelofibrosis.
FREEDOM2 is a phase III study comparing fedratinib with best available therapy as a second-line treatment for myelofibrosis.
“Among the high-risk splicing mutations, SRSF2 has the worst prognostic role,” Dr. Braish and colleagues wrote.
The REVEAL study identified five risk factors, including leukocytosis, duration of time with PV, and more.
The treatment led to reduced hepcidin levels when used alone or in combination with ruxolitinib.
Venugopal highlights myelofibrosis research at ASH 2023.
Dr. Al-Ali covers data on combined BET and JAK inhibition for patients with myelofibrosis presented at the 2023 ASH Meeting.
SRSF2 and SF3B1 were the most common splicing mutations, followed by U2AF1 and ZRSR2.
CALR and MPL mutations, which are associated with better outcomes, showed earlier mutation clearance than JAK2.
Mass spectrometry–based proteomics were used to learn more about how MKs drive bone marrow fibrosis.
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Section Editor
Douglas Tremblay, MDAssistant Professor of Medicine | Icahn School of Medicine at Mount Sinai
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