The burden of red blood cell transfusions in patients with lower-risk myelodysplastic syndromes and ring sideroblasts: an analysis of the prospective MDS-CAN registry

Abstract

Many patients with lower-risk myelodysplastic syndromes (LR MDS) require long-term red blood cell (RBC) transfusions to manage anemia. The consequences of RBC transfusions in LR MDS with ring sideroblasts (LR MDS-RS) are not well known. We estimated the association between cumulative RBC dose density and clinical and patient-reported outcomes using data from the MDS-CAN registry for patients enrolled between January 2008 and December 2018. Outcomes included overall survival, hospitalization, and health-related quality of life (HRQoL). A total of 145 enrolled patients with LR MDS and RS ≥5% had a median follow-up time of 27.1 months; 45 had no transfusions during follow-up, 51 had <1 transfusion per month, and 49 had ≥1 transfusion per month. The cumulative density of RBC transfusions was associated with significantly greater mortality, hospitalization, and inferior HRQoL, suggesting that exposure to RBC transfusion may constitute a significant treatment burden in patients with LR MDS-RS.

Keywords:

• Lower-risk MDS
• ring sideroblasts
• red blood cell transfusions
• overall survival
• Canadian MDS registry

Acknowledgements

The authors would like to thank all the patients and their families, nurses, study personnel, and investigators who participated in the MDS-CAN registry and made this study possible, the clinical study teams who participated in the study, and the protocol managers for this study.

Disclosure statement

PS and CC are paid employees of EVERSANA™, which was contracted by Celgene Inc. (Bristol-Myers Squibb) to work on this project; CC is also a shareholder of EVERSANA™. HAL served on the advisory board and received honoraria and research funding from AbbVie, Alexion Pharmaceuticals, AstraZeneca, Bristol Myers Squibb/Celgene, Janssen Pharmaceuticals, Novartis, and Taiho Oncology. JS received research support from AbbVie, Astellas Pharma, Bristol Myers Squibb/Celgene Canada, Novartis, and Janssen Pharmaceuticals; and served on the advisory board for AbbVie, Astellas Pharma, Bristol Myers Squibb/Celgene Canada, Janssen Pharmaceuticals, Novartis, and Taiho Oncology. BL served on the advisory board and received honoraria from AbbVie, Alexion Pharmaceuticals, AMGEN, Astellas Pharma, Astex Pharmaceuticals, Bristol Myers Squibb/Celgene, F. Hoffmann-La Roche, Janssen Pharmaceuticals, Jazz Pharmaceuticals, Novartis, Otsuka Pharmaceutical, Paladin Labs, Pfizer, and Treadwell Therapeutics; and was a consultant for AbbVie, Novartis, and Pfizer. KWLY was a consultant for Bristol Myers Squibb/Celgene, F. Hoffmann-La Roche, Jazz Pharmaceuticals, Novartis, Pfizer, Shattuck Labs, Taiho Oncology, and Takeda Pharmaceutical Company; received research funding from Astex Pharmaceuticals, F. Hoffmann-La Roche, Forma Therapeutics, Genentech, Geron Corporation, Gilead Sciences, Janssen Pharmaceuticals, Jazz Pharmaceuticals, Novartis, and Treadwell Therapeutics; and received honoraria from AbbVie and Novartis. TN served on the advisory board and received honoraria for AbbVie, Alexion Pharmaceuticals, Bristol Myers Squibb/Celgene, Jazz Pharmaceuticals, Novartis, and Taiho Oncology; and received research funding from Alexion Pharmaceuticals and Bristol Myers Squibb/Celgene. M-MK served on the advisory board for Celgene and Taiho Oncology. RB received research support from Bristol Myers Squibb/Celgene and Takeda Pharmaceutical Company; received funding for the national MDS registry from Bristol Myers Squibb and Taiho Pharma Canada; received honoraria from Bristol Myers Squibb, Taiho Oncology, and Takeda; served on the advisory board for Bristol Myers Squibb and Taiho Oncology; and had an unpaid role in the scientific advisory board of the MDS Foundation and the MDS guidelines committee of the Aplastic Anemia & MDS International Foundation. CW and DT are paid employees of Bristol Myers Squibb; DT is also a stockholder/shareholder of Bristol Myers Squibb. MG received research support (institutional clinical trial contract) from AbbVie, Amgen, Bristol Myers Squibb/Celgene, Geron Corporation, Gilead Sciences, Janssen Pharmaceuticals, and Novartis; received honoraria from Bristol Myers Squibb/Celgene; was an expert witness for Novartis and Taiho Oncology; and served on the advisory board for AbbVie, Amgen, Bristol Myers Squibb/Celgene, Jazz Pharmaceuticals, Novartis, Paladin Labs, Pfizer, and Taiho Oncology. VB had a role as content expert, paid or unpaid, in the CLL Society, CLL Canada, and The Leukemia & Lymphoma Society of Canada; was a consultant for AbbVie, AstraZeneca, BeiGene, and Janssen Pharmaceuticals; and received honoraria from AbbVie, AstraZeneca, Janssen Pharmaceuticals, Medicom, and Oncology Education and royalties or licenses from Biogen. ES-H served on the advisory board for Bristol Myers Squibb and Taiho Oncology. LC served on the advisory board for AbbVie. NF was a consultant for Alexion Pharmaceuticals, Amgen, AstraZeneca, Bristol Myers Squibb/Celgene, F. Hoffmann-La Roche, Ipsen, Janssen Pharmaceuticals, Lundbeck, Merck & Co., Novartis, Pfizer, Sanofi, Takeda Pharmaceutical Company; and received honoraria from AstraZeneca, Bristol Myers Squibb/Celgene, F. Hoffmann-La Roche, FORUS Therapeutics, Novartis, and Pfizer. GC, DK, AP, and AT have no disclosures to reports

Data availability statement

The data that support the findings of this study are available in the supplementary material of this article and from the corresponding author upon reasonable request.

Additional information

Funding

This study was funded by Bristol Myers Squibb. The authors would like to thank Celgene Inc., a Bristol-Myers Squibb Company, Canada for unrestricted funds that help support the registry operational costs. The authors received editorial support in the preparation of this manuscript from Patricia Fonseca, PhD, of Excerpta Medica, funded by Bristol Myers Squibb.