Use of immunosuppressive therapy for management of myelodysplastic syndromes: a systematic review and meta-analysis.

Maximilian Stahl, Jan Philipp Bewersdorf, Smith Giri, Rong Wang, Amer M Zeidan

Journal: Haematologica 2021;105(1):102-111

PMID: 31004015

Abstract

Immunosuppressive therapy (IST) is one therapy option for treatment of patients with lower-risk myelodysplastic syndromes (MDS). However, the use of several different immunosuppressive regimens, the lack of high-quality studies, and the absence of validated predictive biomarkers pose important challenges. We conducted a systematic review and meta-analysis according to the Meta-Analysis of Observational Studies in Epidemiology (MOOSE) guidelines and searched MEDLINE PubMed, Ovid EMBASE, COCHRANE registry of clinical trials (CENTRAL), and the Web of Science without language restriction from inception through September 2018, as well as relevant conference proceedings and abstracts, for prospective cohort studies or clinical trials investigating IST in MDS. Fixed and Random-effects models were used to pool response rates. We identified nine prospective cohort studies and 13 clinical trials with a total of 570 patients. Overall response rate was 42.5% [95% confidence interval (CI): 36.1-49.2%] including a complete remission rate of 12.5% (95%CI: 9.3-16.6%) and red blood cell transfusion independence rate of 33.4% (95% CI: 25.1-42.9%). The most commonly used forms of IST were anti-thymocyte globulin alone or in combination with cyclosporin A with a trend towards higher response rates with combination therapy. Progression rate to acute myeloid leukemia was 8.6% per patient year (95%CI: 3.3-13.9%). Overall survival and adverse events were only inconsistently reported. We were unable to validate any biomarkers predictive of a therapeutic response to IST. IST for treatment of lower-risk MDS patients can be successful to alleviate transfusion burden and associated sequelae.

Copyright© 2020 Ferrata Storti Foundation.

Address: Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, NY.; Department of Internal Medicine, Section of Hematology, Yale School of Medicine, New Haven, CT.; Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, CT.; Department of Chronic Disease Epidemiology, School of Public Health, Yale University, New Haven, CT, USA.; Department of Internal Medicine, Section of Hematology, Yale School of Medicine, New Haven, CT [email protected].; Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, CT.
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