Thrombocytopenia in Patients With Myelofibrosis: A Practical Management Guide

Tremblay, Douglas; Baine, Ian; Mascarenhas, John

doi:10.1016/j.clml.2022.08.011

Cited by 4 publications

(4 citation statements)

References 65 publications

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“…Further, none of the approved JAK inhibitors have shown substantial clinical improvement in anemia in a prospective randomized clinical trial with prespecified anemia end points. Alternative options for the management of thrombocytopenia in patients with MF include platelet transfusions, splenectomy, immunomodulatory drugs with or without corticosteroids, androgens, or hypomethylating agents; however, each of these approaches have shown limited benefit 36 . Thus, effective and safe therapies for patients with anemia and thrombocytopenia are needed.…”

Section: Discussionmentioning

confidence: 99%

“…Alternative options for the management of thrombocytopenia in patients with MF include platelet transfusions, splenectomy, immunomodulatory drugs with or without corticosteroids, androgens, or hypomethylating agents; however, each of these approaches have shown limited benefit. 36 Thus, effective and safe therapies for patients with anemia and thrombocytopenia are needed. Through inhibition of ACVR1, JAK1, and JAK2 and demonstrated symptom, spleen, and anemia benefits in patients with MF, momelotinib is uniquely positioned to fill this unmet need in MF.…”

Section: Discussionmentioning

confidence: 99%

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Momelotinib in Myelofibrosis Patients With Thrombocytopenia: Post Hoc Analysis From Three Randomized Phase 3 Trials

Kiladjian,

Vannucchi,

Gerds

et al. 2023

HemaSphere

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The oral activin A receptor type I, Janus kinase 1 (JAK1), and JAK2 inhibitor momelotinib demonstrated symptom, spleen, and anemia benefits in intermediate- and high-risk myelofibrosis (MF). Post hoc analyses herein evaluated the efficacy and safety of momelotinib in patients with MF and thrombocytopenia (platelet counts <100 × 109/L) from randomized phase 3 studies: MOMENTUM (momelotinib versus danazol; JAK inhibitor experienced); SIMPLIFY-1 (momelotinib versus ruxolitinib; JAK inhibitor naïve); and SIMPLIFY-2 (momelotinib versus best available therapy; JAK inhibitor experienced); these studies were not statistically powered to assess differences in thrombocytopenic subgroups, and these analyses are descriptive. The treatment effect of momelotinib versus ruxolitinib on week 24 response rates (spleen volume reduction ≥35%/Total Symptom Score reduction ≥50%/transfusion independence) was numerically comparable or better in thrombocytopenic patients versus the overall JAK inhibitor naive population; rates were preserved with momelotinib in thrombocytopenic patients but attenuated with ruxolitinib (momelotinib: 27%/28%/67% overall versus 39%/35%/61% in thrombocytopenic group; ruxolitinib: 29%/42%/49% overall versus 0%/22%/39% in thrombocytopenic group, respectively). In contrast to ruxolitinib, momelotinib maintained high dose intensity throughout the treatment. In the JAK inhibitor experienced population, thrombocytopenic patients had the following: (1) numerically higher symptom and transfusion independence response rates with momelotinib than in control arms; and (2) preserved spleen, symptom, and transfusion independence response rates with momelotinib relative to the overall study populations. The safety profile of momelotinib in thrombocytopenic patients was also consistent with the overall study population. In summary, momelotinib represents a safe and effective treatment option for patients with MF and moderate-to-severe thrombocytopenia.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Momelotinib in Myelofibrosis Patients With Thrombocytopenia: Post Hoc Analysis From Three Randomized Phase 3 Trials

Kiladjian,

Vannucchi,

Gerds

et al. 2023

HemaSphere

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“…However, patients with moderate thrombocytopenia are treated with low doses, whereas those with platelet count below 50 × 10 9 /L are excluded from treatment 15 . Ruxolitinib is also burdened by on‐target hematological toxicity, with many dose reductions and treatment discontinuations caused by anemia and/or thrombocytopenia 16,17 . New JAK2 inhibitors, namely fedratinib, pacritinib, and momelotinib, are becoming available for the treatment of MF and may have a role in patients with cytopenia.…”

Section: Introductionmentioning

confidence: 99%

“…15 Ruxolitinib is also burdened by on-target hematological toxicity, with many dose reductions and treatment discontinuations caused by anemia and/or thrombocytopenia. 16,17 New JAK2 inhibitors, namely fedratinib, pacritinib, and momelotinib, are becoming available for the treatment of MF and may have a role in patients with cytopenia.…”

Section: Introductionmentioning

confidence: 99%

Ruxolitinib in cytopenic myelofibrosis: Response, toxicity, drug discontinuation, and outcome

Palandri

Breccia²,

Mazzoni

et al. 2023

Cancer

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Background: Patients with cytopenic myelofibrosis (MF) have more limited therapeutic options and poorer prognoses compared with patients with the myeloproliferative phenotype.Aims and Methods: Prognostic correlates of cytopenic phenotype were explored in 886 ruxolitinib-treated patients with primary/secondary MF (PMF/SMF) included in the RUX-MF retrospective study. Cytopenia was defined as: leukocyte count <4 � 10 9 /L and/or hemoglobin <11/<10 g/dL (males/females) and/or platelets <100 � 10 9 /L.Results: Overall, 407 (45.9%) patients had a cytopenic MF, including 249 (52.4%) with PMF. In multivariable analysis, high molecular risk mutations (p = .04), intermediate 2/high Dynamic International Prognostic Score System (p < .001) and intermediate 2/high Myelofibrosis Secondary to Polycythemia Vera and Essential Thrombocythemia Prognostic Model (p < .001) remained associated with cytopenicThe first two and last two authors contributed equally to the manuscript.This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

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Danazol/pomalidomide/prednisone

2023

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Thrombocytopenia in Patients With Myelofibrosis: A Practical Management Guide

Cited by 4 publications

References 65 publications

Momelotinib in Myelofibrosis Patients With Thrombocytopenia: Post Hoc Analysis From Three Randomized Phase 3 Trials

Momelotinib in Myelofibrosis Patients With Thrombocytopenia: Post Hoc Analysis From Three Randomized Phase 3 Trials

Ruxolitinib in cytopenic myelofibrosis: Response, toxicity, drug discontinuation, and outcome

Danazol/pomalidomide/prednisone

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