A prospective study involving patients with newly diagnosed, high-risk (HR) myelodysplastic syndrome (MDS) suggests that those who received active therapy soon after being diagnosed had improved outcomes. Results of the study were recently published in the journal Leukemia & Lymphoma.

“This study highlights the importance of initiating active treatment in newly diagnosed intermediate-2/high risk MDS patients,” the study investigators wrote in their report.

The study was the ERASME study (CEL-SMD-2012-01), which was conducted across 53 hospitals in Spain. The study investigators had a goal of assessing clinical outcomes in patients with newly diagnosed MDS or chronic myelomonocytic leukemia (CMML) based on the timing of initiation of active treatment. Event-free survival (EFS) was the primary end point of the study, and multiple secondary end points were also evaluated. Patients had a minimum follow-up duration of 36 months, and the reported analysis focused on patients who had HR-MDS.

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The full analysis set included 204 patients with HR-MDS, with 175 patients included in safety analyses. Among therapeutic strategies patients received, 52.0% of patients had active treatment, intention to pursue hematopoietic stem cell transplantation (HSCT) was the strategy followed for 25.5%, supportive care was used with 19.1%, and watchful waiting (WW) was the strategy for 3.4% of patients.

Patients in the HSCT group had a lower median age (59.0 years) than patients receiving active treatment did (75.0 years; P <.0001). Patients treated with a supportive care/WW strategy had a median age of 80.0 years (P =.0010) in comparison with patients on active treatment.

The median EFS for the study population overall was 7.9 months (95% CI, 6.1-9.5). By treatment group, the median EFS was 9.1 months (95% CI, 7.2-12.1) for the group receiving active treatment, compared with 8.3 months (95% CI, 5.5-12.3) for the HSCT group and 4.3 months (95% CI, 3.2-6.9) for patients in the supportive care/WW group. Patients in the supportive care/WW group who underwent a switch to active treatment had a median EFS of 4.0 months (95% CI, 2.2-6.9).

The median progression-free survival (PFS) for the study population overall was 10.1 months (95% CI, 8.7-12.8). Median PFS was shortest for the supportive care/WW group, and overall significantly differed between treatment groups (P =.0004). Differences also were seen for overall survival across treatment groups (P =.0119), with the supportive care/WW group showing the shortest overall survival.

Reported grade 3 or higher adverse events occurred in 72.6% of the safety population, with 60.6% of patients having serious adverse events and 33.1% of patients having fatal adverse events. Patients in the HSCT group had the lowest rates of comorbidity worsening, new symptomology, and new comorbidity.

“Ultimately, selecting an active treatment for HR-MDS patients had a protective effect against the risk of events, disease progression, and death,” the study investigators concluded in their report.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Montoro MJ, Pomares H, Coll R, et al; on behalf of ERASME study group. Evaluation of the outcomes of newly diagnosed patients with high-risk myelodysplastic syndrome according to the initial therapeutical strategies chosen in usual clinical practice. Leuk Lymphoma. Published online December 28, 2022. doi:10.1080/10428194.2022.2154604.

This article originally appeared on Hematology Advisor