Treatment outcomes for patients with myelodysplastic syndrome/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis Journal Article


Authors: Komrokji, R.; Melody, M.; al Ali, N.; Chan, O.; Klimek, V.; Ball, B. J.; Sekeres, M. A.; Lucas, G.; Maciejewski, J. P.; Sallman, D. A.; Padron, E.; Kuykendall, A.; Lasho, T.; Al-Kali, A.; Naqvi, K.; Steensma, D. P.; Garcia-Manero, G.; Patnaik, M. M.
Article Title: Treatment outcomes for patients with myelodysplastic syndrome/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis
Abstract: Myelodysplastic syndrome/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) is characterized by anemia, ring sideroblast erythroid precursors, and persistent thrombocytosis. Case reports suggest lenalidomide may be effective in treating MDS/MPN-RS-T. We evaluated a large series of patients with MDS/MPN-RS-T to compare hematological improvement (HI) response rates among different drug therapies including lenalidomide. We identified 167 patients with MDS/MPN-RS-T. Among the patients tested, 84% had SF3B1 mutations and 43% had JAK2 V617F mutations. The median OS for the cohort was 81 months. Overall, 76 patients (46%) received erythropoiesis-stimulating agents (ESAs), 47 patients (28%) received lenalidomide, and 45 patients (27%) received hypomethylating agents (HMAs). The HI rates were 58%, 53%, and 24%, respectively. The median duration of treatment was 11 months for lenalidomide compared to 6 months for HMAs. Rates of HI improvement were higher in patients with MDS/MPN-RS-T treated with ESAs or lenalidomide, in comparison to those treated with HMAs. © 2021 Informa UK Limited, trading as Taylor & Francis Group.
Keywords: adult; cancer survival; controlled study; treatment outcome; treatment response; aged; gene mutation; human cell; major clinical study; overall survival; somatic mutation; antianemic agent; janus kinase 2; lenalidomide; drug efficacy; treatment duration; erythroid precursor cell; hemoglobin; retrospective study; wild type; myelodysplastic syndrome; azacitidine; myeloproliferative neoplasm; thrombocytosis; high throughput sequencing; decitabine; human; male; female; article; mixed myelodysplastic myeloproliferative disease; hematological improvement; mds/mpn-rs-t
Journal Title: Leukemia and Lymphoma
Volume: 63
Issue: 1
ISSN: 1042-8194
Publisher: Taylor & Francis Group  
Date Published: 2022-01-01
Start Page: 199
End Page: 204
Language: English
DOI: 10.1080/10428194.2021.1971217
PUBMED: 34448437
PROVIDER: scopus
DOI/URL:
Notes: Article -- Export Date: 1 February 2022 -- Source: Scopus
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  1. Virginia Klimek
    147 Klimek
  2. Brian John Ball
    17 Ball