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, start as another MPN, like polycythemia vera (PV) or essential thrombocythemia (ET), and later evolve into myelofibrosis.
Myelofibrosis Awareness Day is marked on September 20th each year.
Researchers presented updated data from the phase I XPORT-MF-034 trial at the Eleventh SOHO Annual Meeting.
The open-label ACE-536-MF-001 study included patient cohorts grouped by transfusion dependance and ruxolitinib therapy.
In PERSIST-2, treatment with pacritinib demonstrated a significant SVR benefit compared with the best available therapy, incl
Nearly all myelofibrosis patients are estimated to develop anemia over the course of the disease.
Ruxolitinib, an oral JAK1/JAK2 inhibitor, initially received FDA approval in 2011.
Researchers conducted the study because the mechanism by which pacritinib improves anemia has not been elucidated.
Researchers conducted an indirect comparison analysis of multiple clinical trials to address the question.
In June 2023, the manufacturer of the drug initiated XPORT-MF-034, a pivotal phase III clinical trial.
From Houston, Texas, to Beirut, Lebanon, the SOHO global community continues to grow thanks to its Ambassador Program.
At a median follow-up of 55 weeks, 90% of patients completed 24 weeks of treatment and 56% completed 48 weeks of treatment.
Initiation of ruxolitinib therapy within two years of diagnosis was associated with increased response rates in all patients.
Just under half (43%) of patients receiving ruxolitinib achieved a CR, while 26% achieved a CR on the best available therapy.
However, achieving a spleen volume reduction on the best available therapy was not linked with improved survival.
Dr. Mascarenhas and colleagues identified 11,371 patients with myelofibrosis, finding that 76.8% had concurrent anemia.
Spleen volume reduction predicts OS in patients with myelofibrosis who are taking pacritinib.
A new study is exploring add-on treatment of CK0804 in patients with myelofibrosis and suboptimal response to ruxolitinib.
The study indirectly compared safety outcomes from phase II and phase III trials of momelotinib and fedratinib.
The median time to achieving the first spleen volume reduction of at least 35% from baseline was 12 weeks.
Prithviraj Bose, MD, and colleagues presented results of the study during the 2023 ASCO Annual Meeting.
The authors concluded that treatment with INCB057643 monotherapy was generally well tolerated.
Researchers followed the patients for three years after their final dose of luspatercept.
Jakatinib may be a new effective treatment option for patients with myelofibrosis.
Pacritinib demonstrates consistent efficacy for spleen and symptom response in patients with MF regardless of blood counts.
Selinexor plus ruxolitinib was effective in certain subgroups of patients with myelofibrosis.
The pooled analysis set included patients from both arms of the intent-to-treat populations in SIMPLIFY-1 and SIMPLIFY-2.
The trial will enroll certain patients who had an "inadequate response” to ruxolitinib alone.
Dr. Tefferi discusses risk stratification systems for primary myelofibrosis, transplant considerations, and more.
They detected mutated TP53 in 49 (13%) patients, with 30 of those patients showing a multihit configuration.
The model was developed and validated using data from the CIBMTR and EBMT registries.
The study evaluated longitudinal symptom score changes to “complement the interpretation of the landmark symptom ...
Planning for a phase III trial of the combination is underway.
Jan Bewersdorf, MD, discusses a current ongoing phase I study investigating the combination of ruxolitinib and abemaciclib.
Hematopoietic stem/progenitor cells from patients with myelofibrosis are “enriched” for a CXCL8/CXCR2 gene signature.
A reduction in spleen volume reduction of ≥35% at 24 weeks occurred in 68% of patients.
The study’s investigators are continuing to monitor overall survival and conduct ongoing patient follow-up.
The overall risk of death from primary myelofibrosis declined by more than 50% after the U.S. FDA approved ruxolitinib.
Mutation of CALR in patients with myelofibrosis may be associated with a more anemic phenotype at diagnosis.
John Mascarenhas, MD, discusses the results of the PACIFICA trial at the 2022 American Society of Hematology Annual Meeting.
Parsaclisib plus ruxolitinib can improve symptoms and spleen volume in certain patients with myelofibrosis.
TP53 and complex karyotype are very high-risk factors in patients with myelofibrosis undergoing HSCT.
The combination of ruxolitinib and pegylated IFNα2a showed significant reductions in spleen length.
BMS-986158 combined with ruxolitinib or fedratinib reduced spleen volume in patients with myelofibrosis.
Selinexor plus ruxolitinib demonstrated promising clinical activity in patients with treatment-naïve myelofibrosis.
Momelotinib led to an increased likelihood of becoming transfusion-independent compared with danazol in myelofibrosis.
An analysis of patients with MF found that the combination of navitoclax and ruxolitinib reduced MF-associated splenomegaly.
Findings from a new study “underscore the limited therapeutic value of luspatercept” in anemia and myelofibrosis.
Those with MF with high molecular and cytogenetic risk do not benefit from higher intensity conditioning before transplant.
Transfusion independence was associated with improved survival in studies of momelotinib in patients with myelofibrosis.
Adding navitoclax to ruxolitinib led to durable spleen volume reductions and improved total symptoms in myelofibrosis.
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