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- Title
Eltrombopag Use in Patients With Chronic Myelomonocytic Leukemia (CMML): A Cautionary Tale.
- Authors
Ramadan, Hanadi; Duong, Vu H.; Al Ali, Najla; Padron, Eric; Zhang, Ling; Lancet, Jeffrey E.; List, Alan F.; Komrokji, Rami S.
- Abstract
<bold>Introduction: </bold>Eltrombopag used for thrombocytopenia in myelodysplastic syndrome (MDS) patients is being explored in clinical trials including those with chronic myelomonocytic leukemia (CMML).<bold>Patients and Methods: </bold>We report our experience treating patients with CMML enrolled in a sequential 2-stage dose escalation study with eltrombopag in patients with MDS after hypomethylating agent failure. Patients with CMML were compared with patients with MDS in respect to response and adverse events.<bold>Results: </bold>Among 33 patients in the study, we treated 7 patients with CMML. Hematological improvement or better (by International Working Group 2006 criteria) was observed in 6 (23%) of 26 MDS patients and 1 (14%) of 7 patients with CMML. The responding patient with CMML had bilineage response. Two patients with CMML became platelet transfusion-independent. Five (71%) patients with CMML developed leukocytosis compared with 3 (12%) patients with MDS. Four (57%) patients with CMML showed peripheral myeloblasts after treatment compared with 11 (42%) in the MDS group. One (14%) patient with CMML developed grade 3 fibrosis from grade 0 to 1 at baseline compared with 3 (12%) patients with MDS. The rate of acute myeloid leukemia transformation was 39% and 29%, respectively, in patients with MDS and CMML.<bold>Conclusion: </bold>Further clinical studies are needed to identify patients with CMML who will benefit from this treatment.
- Subjects
ANTI-infective agents; CLINICAL trials; COMPARATIVE studies; HETEROCYCLIC compounds; RESEARCH methodology; MEDICAL cooperation; MYELODYSPLASTIC syndromes; ORGANIC compounds; PROGNOSIS; RESEARCH; RESEARCH funding; EVALUATION research; CHRONIC myeloid leukemia; TREATMENT effectiveness; DIAGNOSIS; THERAPEUTICS
- Publication
Clinical Lymphoma, Myeloma & Leukemia, 2016, Vol 16, pS64
- ISSN
2152-2650
- Publication type
journal article
- DOI
10.1016/j.clml.2016.02.009