Immunosuppressive therapy (IST) may lead to durable objective responses in patients with lower-risk myelodysplastic syndromes (MDS), thereby emerging as a viable treatment option, according to data published in Blood Advances.

Patients with lower-risk MDS are typically treated with transfusions, lenalidomide, hypomethylating agents, or erythropoiesis-stimulating agents. However, patients being treated with these agents often experience treatment failure. IST has been used with patients with lower-risk MDS, but clinicians use IST sparingly due to the operational challenges in administering the drugs involved in IST as well as the inability to accurately predict whether patients will benefit from IST.

In this international, multicenter, retrospective study, researchers analyzed data collected from 207 patients (median age 65 years; 63% male) from 15 centers in the United States and Europe. Primary outcomes were overall response rate (ORR), overall survival (OS), predictors of response, and red blood cell transfusion independence (TI; defined as the ability to maintain a hemoglobin level of at least 8 g/dL for 6 weeks without red blood cell transfusions). 

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Of the 125 patients whose response data were recorded, 14 (11.2%) achieved complete response, 7 (5.6%) achieved partial response, and 40 (32%) achieved hematologic improvement, yielding an ORR of 48.8%. Red blood cell TI was achieved in 30% of previously transfusion-dependent patients, with a median time of 9.4 weeks from IST initiation to TI and a median TI duration of 19.9 months.

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Median OS for all patients after initiation of IST was 47.4 months. Patients who achieved a response to IST experienced OS of at least 52.1 months, whereas the OS for patients who achieved no response was 27.7 months. Similarly, patients who achieved TI had a minimum OS of 76.9 months, whereas patients who did not achieve TI had a median OS of 26.6 months.

No predictors of response were identified when multivariate analysis was conducted. However, in univariate analysis of predictors of OS, improved OS was predicted by lower-risk International Prognostic Scoring System (IPSS) score and low bone marrow blast percentage.

The authors noted that selection bias and missing response or predictor data are potential limitations of the study and may have inflated the benefit of IST.

Disclosures: Multiple authors disclosed relationships with industry. Please refer to the original study for a full list of disclosures.


1. Stahl M, DeVeaux M, de Witte T, et al. The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort [published online July 23, 2018]. Blood Advances. doi: 10.1182/bloodadvances.201819414