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. 2024 Sep;24(9):e283-e292.
doi: 10.1016/j.clml.2024.05.007. Epub 2024 May 7.

Clinical Outcomes and Healthcare Resource Utilization for Patients With Lower-Risk Myelodysplastic Syndromes Treated With Erythropoiesis-Stimulating Agents

Guillermo Garcia-Manero  1 , Rayna K Matsuno  2 , Ali McBride  3 , Hina Mohammed  4 , Danny Idryo  4 , Ronda Broome  4 , Autumn Herriman  4 , Tiffany Johnson  4 , Kristiana Wilkinson  4 , Andrew Schrag  4 , Colden Johanson  4 , Monika Izano  4 , Adeola Makinde  5 , Sudipto Mukherjee  6
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Free article

Clinical Outcomes and Healthcare Resource Utilization for Patients With Lower-Risk Myelodysplastic Syndromes Treated With Erythropoiesis-Stimulating Agents

Guillermo Garcia-Manero et al. Clin Lymphoma Myeloma Leuk. 2024 Sep.
Free article

Abstract

Introduction: Real-world studies of lower-risk myelodysplastic syndromes (LR-MDS) are limited. We evaluated treatment patterns, clinical outcomes, and healthcare resource utilization (HCRU) among patients with LR-MDS treated with erythropoiesis-stimulating agents (ESAs) in the United States.

Patients and methods: This retrospective study included patients with LR-MDS who initiated treatment with ESAs between January 1, 2016 and June 30, 2019. The primary analysis assessed patient demographic and clinical characteristics, treatment patterns, clinical outcomes (hematologic response, transfusion requirements, disease progression), and HCRU (medical encounters, laboratory tests, and medication use). Subgroup analyses of patients repeatedly treated with ESA therapy evaluated selected clinical outcomes and primary ESA failure by SF3B1 mutational status, per recently updated NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines©).

Results: A total of 142 patients were included with a median follow-up time of 17 months (interquartile range [IQR], 7-33). Median age at ESA initiation was 79 years (IQR, 73-85). Patients were predominantly male (54%), overweight or obese (32% and 23%, respectively), of White race (96%) and non-Hispanic ethnicity (89%). Overall, 57% patients were initially treated with darbepoetin alfa and 43% with epoetin alfa. Clinical outcomes were poor, and there was a significant burden on both the health system and individual patients treated with ESA therapies. Hematologic improvement- erythroid was only seen in 26% of 142 patients treated with ESAs, and 65% of 82 retreated patients experienced primary ESA failure.

Conclusion: Our results indicate that primary ESA failure is largely unrecognized and that many patients should be considered for alternative treatments.

Keywords: Clinical Outcomes; Erythropoiesis-Stimulating Agents; Healthcare Resource Utilization Outcomes; Lower-Risk Myelodysplastic Syndromes.

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Conflict of interest statement

Disclosure Guillermo Garcia-Manero received research support from Bristol Myers Squibb. Rayna K. Matsuno, Hina Mohammed, Danny Idryo, Ronda Broome, Autumn Herriman, Tiffany Johnson, Kristiana Wilkinson, Andrew Schrag, and Monika Izano are employees of Syapse. Rayna K. Matsuno, Hina Mohammed, Ronda Broome, Kristiana Wilkinson, and Monika Izano report divested equity in a private or publicly traded company in the past 24 months at Syapse. Rayna K. Matsuno, Hina Mohammed, Tiffany Johnson, and Colden Johanson also report research funding from Bristol Myers Squibb. Ali McBride and Adeola Makinde are employees of Bristol Myers Squibb. Sudipto Mukherjee participated in advisory boards for AbbVie, Blueprint Medicines, Bristol Myers Squibb, Celgene/Acceleron, EUSA Pharma, Genentech, and Novartis; received honoraria from the Aplastic Anemia and MDS International Foundation, Bristol Myers Squibb, Celgene, McGraw Hill Hematology Oncology Board Review, Partnership for Health Analytic Research, and EUSA Pharma; reports consultancy from BioPharm, Bristol Myers Squibb, Celgene, EUSA Pharma, and Novartis; and research funding to the institution from Celgene, Jazz Pharmaceuticals, and Novartis.