Last update 01 Nov 2024

Anemia, Myelophthisic

Last update 01 Nov 2024

Basic Info

Synonyms

ANAEMIA LEUKOERYTHROBLASTIC, ANEMIA LEUKOERYTHROBLASTIC, ANEMIA, LEUKOERYTHROBLASTIC

+ [48]

Introduction

Anemia characterized by appearance of immature myeloid and nucleated erythrocytes in the peripheral blood, resulting from infiltration of the bone marrow by foreign or abnormal tissue.

Drugs associated with Anemia, Myelophthisic

Momelotinib Dihydrochloride

Target

ALK2 x JAK1 x JAK2

Mechanism

ALK2 inhibitors [+2]

Active Org.

GSK Plc [+4]

Originator Org.

Gilead Sciences, Inc.

Active Indication

Anemia [+8]

Inactive Indication

EGFR-mutated non-small Cell Lung Cancer [+6]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

First Approval Date15 Sep 2023

Zilurgisertib

Target

ALK2

Mechanism

ALK2 inhibitors

Active Org.

Incyte Corp. [+1]

Originator Org.

Incyte Corp.

Active Indication

Myositis Ossificans [+8]

Inactive Indication-

Drug Highest PhasePhase 2

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

100 Clinical Results associated with Anemia, Myelophthisic

100 Translational Medicine associated with Anemia, Myelophthisic

0 Patents (Medical) associated with Anemia, Myelophthisic

301

Literatures (Medical) associated with Anemia, Myelophthisic

01 Sep 2024·Annals of Hematology

Treatment of myelofibrosis with refractory anemia with luspatercept: a multicenter Chinese study

Article

Author: Guo, Shuxia ; Liu, Yinghui ; Shi, Hongxia ; Liu, Ziwei ; Han, Bing ; Fang, Liwei ; Yang, Xiuli ; Hu, Qinglin ; Long, Chan ; Wang, Leyu ; Chen, Miao ; Yang, Chen

Myelofibrosis is a rare and often fatal hematological neoplasm, and the treatment of myelofibrosis-associated anemia remains suboptimal, with no improved therapies. Luspatercept was shown to display some efficacy in a phase 2 clinical trial for Myelofibrosis with anemia, yet relevant research are limited. Threrfore, data from patients diagnosed with refractory anemic primary or post-essential thrombocythemia/polycythemia vera myelofibrosis, who were treated with luspatercept for at least 9 weeks, were retrospectively collected. Eighteen patients with myelofibrosis treated with luspatercept were enrolled. Median age was 68 years (range, 44-80 years), and 27.8% were males. Ten (55.6%) were transfusion-dependent. Ten (55.6%) were Dynamic International Prognostic Scoring System intermediate-1, and eight (44.4%) were intermediate-2. The median follow-up was 7 (4-16) months. Erythroid response occurred in eight patients (44.4%) at week 12, four patients (30.8%) at week 24, and nine (50%) at the end of follow-up. Patients who were transfusion-dependent and not transfusion-dependent had similar HI-E responses, at different time points (P > 0.05). Patients had a significantly higher hemoglobin level at 12 weeks, 24 weeks, and at the end of follow-up, than at baseline (P = 0.001, P = 0.021, and P = 0.005, respectively). Treatment-related adverse events occurred in five (16.7%) patients, with no serious adverse events. Two (11.1%) patients relapsed at weeks 15 and 31. One patient progressed to acute myeloid leukemia. No patients had died by the end of follow-up. Luspatercept induced a good response in patients with anemic myelofibrosis, with a low relapse rate and good tolerance.

02 Jan 2024·Expert Opinion on Investigational Drugs

Treatment of anemia in myelofibrosis: focusing on novel therapeutic options

Review

Author: Arslan Davulcu, Eren ; Eşkazan, Ahmet Emre ; Kılıç, Emre ; Oğuz, Merve Beyza

INTRODUCTIONMyelofibrosis is a clonal myeloproliferative neoplasm associated with the proliferation of hematopoietic stem cells, increased bone marrow fibrosis, extramedullary hematopoiesis, hepatosplenomegaly, abnormal cytokine production, and constitutional symptoms. These and many other factors contribute to the development of anemia in myelofibrosis patients.AREAS COVEREDThis review summarizes novel and promising treatments for anemia in myelofibrosis including transforming growth factor-β inhibitors luspatercept and KER-050, JAK inhibitors momelotinib, pacritinib, and jaktinib, BET inhibitors pelabresib and ABBV-744, antifibrotic PRM-151, BCL2/BCL-XL inhibitor navitoclax, and telomerase inhibitor imetelstat.EXPERT OPINIONStandard approaches to treat myelofibrosis-related anemia have limited efficacy and are associated with toxicity. New drugs have shown positive results in myelofibrosis-associated anemia when used alone or in combination.

01 Jan 2024·[Rinsho ketsueki] The Japanese journal of clinical hematology

[Primary myelofibrosis with double mutation in U2AF1].

Article

Author: Ino, Kazuko ; Maeyama, Keiko ; Tawara, Isao ; Kawakami, Keiki ; Sugimoto, Yuka ; Nagaharu, Keiki

A 47-year-old woman presented with subcutaneous hemorrhage. Blood tests revealed leukoerythroblastosis, anemia, and thrombocytopenia. Bone marrow biopsy led to a diagnosis of primary myelofibrosis (aaDIPSS, DIPSS-plus: intermediate-II risk). JAK2, CALR, and MPL mutations were not detected in peripheral blood, but targeted sequencing of bone marrow specimens revealed a double mutation (Q157R, S34F) in U2AF1. Allo-PBSCT was performed using an HLA-matched related donor, and post-transplantation bone marrow examination showed complete donor chimerism on day 55. Two years after allogeneic transplantation, the patient remains relapse-free. Although U2AF1 gene abnormality is known as a poor prognostic factor in primary myelofibrosis, this patient had a favorable long-term prognosis due to prompt transplantation therapy. This case highlights the importance of detailed gene mutation analysis in patients with triple-negative MF.

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Anemia, Myelophthisic

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