With the exception of allogeneic stem cell transplantation (alloSCT) and iron chelation therapy (ICT), many guideline treatments may not improve survival among patients with myelodysplastic syndrome (MDS), according to research published in the Annals of Hematology.

MDS represents a heterogeneous group of malignancies with a variable clinical course, which depends on both disease and patient factors. A number of guidelines help clinicians to recommend treatments based on these characteristics, including European LeukemiaNet (ELN) and the National Comprehensive Cancer Network (NCCN).

It is unknown whether adherence to guideline recommendations improves survival in this patient group. For this study, which had a retrospective cohort and prospective cohort, researchers evaluated whether treatment guideline adherence improves survival among patients with MDS.

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All data were obtained from the Duesseldorf MDS Registry. Cohort 1 included data from 1698 patients, to which the researchers applied ELN guidelines retrospectively. Cohort 2 included patients who were given expert-level guidance on MDS treatment.

Overall, in cohort 1, of the treatment options evaluated, adherence to lenalidomide, hypomethylating agents, low-dose chemotherapy, intensive chemotherapy, or best supportive care therapies did not improve patient survival; similar results were found in analyses of cohort 2.

Patients receiving ICT or alloSCT in line with expert guidelines did, however, appear to have improved survival compared with those who were eligible for such treatments but did not receive them. The median survival of patients who were recommended and received ICT was 70 months vs 32 months for eligible patients who did not receive ICT (P =.012). In addition, the median survival for alloSCT-eligible patients who received transplantation compared with patients who did not receive transplantation was 65 months vs 16 months, respectively (P <.0005).

“Altogether, our retrospective and prospective analyses imply that, with the exception of alloSCT, none of the currently available therapies is powerful enough to render deviation from guideline-based expert advice a major disadvantage in terms of prognosis,” the authors wrote. “We clearly need better treatment options, which can really make a change when correctly applied by an MDS expert.”

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


Kasprzak A, Nachtkamp K, Kondakci M, et al. Analysis of the impact of adherence to guidelines and expert advice in patients with myelodysplastic syndromes. Ann Hematol. Published online November 7, 2020. doi:10.1007/s00277-020-04325-7