High response rate and improved exercise capacity and quality of life with a new regimen of darbepoetin alfa with or without filgrastim in lower-risk myelodysplastic syndromes: a phase II study by the GFM

Ann Hematol. 2013 May;92(5):621-31. doi: 10.1007/s00277-013-1686-4. Epub 2013 Jan 29.

Abstract

Darbepoetin (DAR), with or without granulocyte colony-stimulating factor (G-CSF), has proved effective in treating anemia in patients with lower-risk myelodysplastic syndrome (MDS), but its effects on quality of life (QoL) and exercise functioning are less well established. In this phase II study (no. NCT00443339), lower-risk MDS patients with anemia and endogenous erythropoietin (EPO) level <500 IU/L received DAR 500 μg once every 2 weeks for 12 weeks, with G-CSF added at week 12 in non-responders. Physical performance was assessed with the 6-min walking test and, for fit patients, maximal oxygen consumption (VO2max). QoL was evaluated using SF-36 and FACT-An tests. In 99 patients, erythroid response rate according to IWG 2006 criteria was 48 and 56 % at 12 and 24 weeks, respectively. Addition of G-CSF rescued 22 % of non-responders. In 48 % of the responders, interval between darbepoetin injections could be increased for maintenance treatment. Serum EPO level was the only independent predictive factor of response at 12 weeks, and its most discriminant cutoff value was 100 IU/L. QoL and VO2max showed improvement over time in responders, compared with non-responders. With a median follow-up of 52 months, median response duration was not reached, and 3-year cumulative incidence of acute myeloid leukemia and overall survival (OS) was 14.5 and 70 %, respectively. Baseline transfusion dependence, International Prognostic Score System (IPSS), and Revised IPSS accurately predicted OS from treatment onset. Tolerance of darbepoetin was good. In conclusion, this regimen of darbepoetin every 2 weeks yielded high response rates and prolonged response duration. Objective improvement in exercise testing and in patient-reported QoL confirms the clinical relevance of anemia correction with erythropoiesis-stimulating agents.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study

MeSH terms

  • Aged
  • Anemia / complications
  • Anemia / drug therapy
  • Anemia / mortality
  • Anemia / physiopathology
  • Darbepoetin alfa
  • Erythropoietin / administration & dosage
  • Erythropoietin / adverse effects
  • Erythropoietin / analogs & derivatives*
  • Exercise / physiology
  • Exercise Tolerance / drug effects*
  • Female
  • Filgrastim
  • Granulocyte Colony-Stimulating Factor / administration & dosage*
  • Granulocyte Colony-Stimulating Factor / adverse effects
  • Hematinics / administration & dosage
  • Hematinics / adverse effects
  • Humans
  • Male
  • Myelodysplastic Syndromes / complications
  • Myelodysplastic Syndromes / drug therapy*
  • Myelodysplastic Syndromes / mortality
  • Myelodysplastic Syndromes / physiopathology
  • Quality of Life*
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / adverse effects
  • Risk
  • Survival Analysis
  • Treatment Outcome

Substances

  • Hematinics
  • Recombinant Proteins
  • Erythropoietin
  • Granulocyte Colony-Stimulating Factor
  • Darbepoetin alfa
  • Filgrastim

Associated data

  • ClinicalTrials.gov/NCT00443339