Prognosis of lower-risk (International Prognostic Scoring System [IPSS] low/intermediate-1) myelodysplastic syndrome (MDS) is heterogeneous and relies on steady-state assessment of cytopenias. We analyzed relative drops in neutrophil and platelet counts during the first 6 months of follow-up of lower-risk MDS patients. We performed a landmark analysis of overall survival (OS) of lower-risk MDS patients prospectively included in the European LeukaemiaNet MDS registry having a visit at 6 ± 1 month from inclusion to assess the prognostic relevance of relative drops in neutrophils and platelets, defined as (count at landmark - count at inclusion)/count at inclusion. Of 2102 patients, 807 were eligible for the stringent 6-month landmark analysis. Median age was 73 years. Revised IPSS was very low, low, and intermediate/higher in 26%, 43%, and 31% of patients, respectively. A relative drop in platelets >25% at landmark predicted shorter OS (5-year OS, 21.9% vs 48.6% with platelet drop ≤25%, P < 10-4), regardless of baseline IPSS-revised or absolute platelet counts. Relative neutrophil drop >25% had no significant impact on OS. We built a classifier based on red blood cell transfusion dependence (RBC-TD) and relative platelet drop >25% at landmark. Patients with none (62%), either (27%), or both criteria (11%) had 5-year OS of 53.3%, 32.7%, and 9.0%, respectively (P < 10-4). This classifier was validated in an independent cohort of 335 patients. Combining relative platelet drop >25% and RBC-TD at 6 months from diagnosis provides an inexpensive and noninvasive way to predict outcome in lower-risk MDS. This study was registered at www.clinicaltrials.gov as #NCT00600860.

Center of Hematology and Bone Marrow Transplantation Fundeni Clinical Institute Bucharest Romania

Centro de Investigación Biomédica en Red Cáncer Instituto Carlos 3 Madrid Spain

Clinic of Hematology Clinical Center of Vojvodina Faculty of Medicine University of Novi Sad Novi Sad Serbia

Département d'Hématologie Hôpital Saint Louis Assistance Publique des Hôpitaux de Paris and Université Paris Diderot Paris France

Department of Clinical Hematology Institute of Hematology and Blood Transfusion Praha Czech Republic

Department of Haematology Aarhus University Hospital Aarhus Denmark

Department of Haematology Aberdeen Royal Infirmary Aberdeen United Kingdom

Department of Haematology Oncology and Clinical Immunology Universitätsklinik Düsseldorf Düsseldorf Germany

Department of Haematology Oncology and Internal Medicine Warsaw Medical University Warsaw Poland

Department of Hematology Hospital da Luz Lisbon Portugal

Department of Hematology Hospital Universitario y Politécnico La Fe Valencia Spain

Department of Hematology Oncology Fondazione IRCCS Policlinico San Matteo University of Pavia Pavia Italy

Department of Hematology Radboud University Medical Center Nijmegen The Netherlands

Department of Internal Medicine 5 Innsbruck Medical University Innsbruck Austria

Department of Medicine A Tel Aviv Sourasky Medical Center Sackler Medical Faculty Tel Aviv University Tel Aviv Israel

Department of Tumor Immunology Nijmegen Center for Molecular Life Sciences Radboud University Medical Center Nijmegen The Netherlands

Division Hematology Department of Internal Medicine University of Patras Medical School Patras Greece

Division Hematology Department of Medicine Karolinska Institute Stockholm Sweden

Division of Hematology Department of Internal Medicine Merkur University Hospital Zagreb Croatia

Epidemiology and Cancer Statistics Group Department of Health Sciences University of York York United Kingdom

Service d'Hématologie Centre Hospitalier Universtaire Brabois Vandoeuvre Nancy France

Servicio d'Hematología Hospital Universitario Central de Asturias Oviedo Spain

St James's Institute of Oncology Leeds Teaching Hospitals Leeds United Kingdom; and

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Somatic mutations and DNA methylation identify a subgroup of poor prognosis within lower-risk myelodysplastic syndromes

Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome; the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective

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ClinicalTrials.gov
NCT00600860