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Responses to pomalidomide and placebo in myelofibrosis-related anaemia

Leukemia volume 31, pages 532–533 (2017)Cite this article

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Anaemia related to myeloproliferative myelofibrosis (MF) is a major problem for which existing therapies (other than transplantation) have limited efficacy. In the cohort of 525 primary MF patients studied by the International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) anaemia (haemoglobin <100 g/l) was identified as an adverse prognostic factor and was present in 36% of patients at diagnosis, rising to almost 85% during follow-up.1 MF-related anaemia is an area of need in which potential new therapies are greeted with enthusiasm.

Thalidomide (with or without corticosteroids) was reported to give anaemia response rates of around 30% in MF patients (reviewed Gowin et al.2). Subsequent studies have used the more potent thalidomide-related immunomodulatory drugs (IMIDs), lenalidomide and pomalidomide. Three Phase 2 studies with pomalidomide have shown anaemia response rates of 20–40% in MF patients, and response rates with lenalidomide are similar.2 Higher rates of pomalidomide response were reported in JAK2-positive patients, and in those with higher levels of inflammatory cytokines.3

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  1. Haematology Directorate, SA Pathology, Adelaide, South Australia, Australia

    D M Ross

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Correspondence to D M Ross.

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DMR reports clinical trial or research funding from Celgene, Gilead, and Novartis to his institution. He has received honoraria from Novartis.

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Ross, D. Responses to pomalidomide and placebo in myelofibrosis-related anaemia. Leukemia 31, 532–533 (2017). https://doi.org/10.1038/leu.2016.348

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