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Treatment Algorithm of CMML and Other Adult MDS/MPN Subtypes

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Pathogenesis and Treatment of Leukemia

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Abstract

Myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) overlap syndromes are now recognized as distinct entities in recent World Health Organization classifications. Aside from juvenile myelomonocytic leukemia, MDS/MPNs including chronic myelomonocytic leukemias and rare subtypes such as atypical chronic myeloid leukemia or MDS/MPN with ring sideroblasts and thrombocytosis occur in older adults. Each entity harbors a distinct clinical presentation and molecular profile, but their prognosis remains overall poor. Different risk-scoring systems have been established which are yet to be integrated with therapeutic algorithms. The only curative therapy remains allogeneic hematopoietic stem cell transplantation (HSCT), but few patients are eligible due to their age and comorbidities. Because of their low incidence, few clinical trials have been conducted in MDS/MPNs, and aside from azacitidine in a subset of CMMLs, no drug is labeled for these entities. Therapeutic decisions in MDS/MPNs thus often rely on small retrospective series or case reports and aim to alleviate symptoms, with limited hope to alter the disease’s natural history. Thus, MDS/MPNs remain an unmet medical need. In this chapter, we review the epidemiology, diagnostic, and prognostic criteria of each MDS/MPN entity and propose therapeutic algorithms to guide the management of these rare but high-risk patients.

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  1. Service Hématologie Adolescents et Jeunes Adultes, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France

    Florence Rabian

  2. Service Hématologie Adultes, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France

    Raphael Itzykson

  3. Université de Paris, Génomes, biologie cellulaire et thérapeutique U944, INSERM, CNRS, Paris, France

    Raphael Itzykson

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    Harinder Gill

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    Yok-Lam Kwong

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Rabian, F., Itzykson, R. (2023). Treatment Algorithm of CMML and Other Adult MDS/MPN Subtypes. In: Gill, H., Kwong, YL. (eds) Pathogenesis and Treatment of Leukemia. Springer, Singapore. https://doi.org/10.1007/978-981-99-3810-0_32

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