Erythropoiesis-stimulating agents are first choice for treating anemia in low-risk MDS. This double-blind, placebo-controlled study assessed the efficacy and safety of epoetin-α in IPSS low- or intermediate-1 risk (i.e., low-risk) MDS patients with Hb ≤ 10.0 g/dL, with no or moderate RBC transfusion dependence (≤4 RBC units/8 weeks). Patients were randomized, 2:1, to receive epoetin-α 450 IU/kg/week or placebo for 24 weeks, followed by treatment extension in responders. The primary endpoint was erythroid response (ER) through Week 24. Dose adjustments were driven by weekly Hb-levels and included increases, and dose reductions/discontinuation if Hb > 12 g/dL. An independent Response Review Committee (RRC) blindly reviewed all responses, applying IWG-2006 criteria but also considering dose adjustments, drug interruptions and longer periods of observation.A total of 130 patients were randomized (85 to epoetin-α and 45 to placebo). The ER by IWG-2006 criteria was 31.8% for epoetin-α vs 4.4% for placebo (p < 0.001); after RRC review, the ER was 45.9 vs 4.4% (p < 0.001), respectively. Epoetin-α reduced RBC transfusions and increased the time-to-first-transfusion compared with placebo.Thus, epoetin-α significantly improved anemia outcomes in low-risk MDS. IWG-2006 criteria for ER may require amendments to better apply to clinical studies.

A phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-α in anemic patients with low-risk MDS / Fenaux P.; Santini V.; Spiriti M.A.A.; Giagounidis A.; Schlag R.; Radinoff A.; Gercheva-Kyuchukova L.; Anagnostopoulos A.; Oliva E.N.; Symeonidis A.; Berger M.H.; Gotze K.S.; Potamianou A.; Haralampiev H.; Wapenaar R.; Milionis I.; Platzbecker U.. - In: LEUKEMIA. - ISSN 0887-6924. - STAMPA. - 32:(2018), pp. 2648-2658. [10.1038/s41375-018-0118-9]

A phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-α in anemic patients with low-risk MDS

Santini V.;
2018

Abstract

Erythropoiesis-stimulating agents are first choice for treating anemia in low-risk MDS. This double-blind, placebo-controlled study assessed the efficacy and safety of epoetin-α in IPSS low- or intermediate-1 risk (i.e., low-risk) MDS patients with Hb ≤ 10.0 g/dL, with no or moderate RBC transfusion dependence (≤4 RBC units/8 weeks). Patients were randomized, 2:1, to receive epoetin-α 450 IU/kg/week or placebo for 24 weeks, followed by treatment extension in responders. The primary endpoint was erythroid response (ER) through Week 24. Dose adjustments were driven by weekly Hb-levels and included increases, and dose reductions/discontinuation if Hb > 12 g/dL. An independent Response Review Committee (RRC) blindly reviewed all responses, applying IWG-2006 criteria but also considering dose adjustments, drug interruptions and longer periods of observation.A total of 130 patients were randomized (85 to epoetin-α and 45 to placebo). The ER by IWG-2006 criteria was 31.8% for epoetin-α vs 4.4% for placebo (p < 0.001); after RRC review, the ER was 45.9 vs 4.4% (p < 0.001), respectively. Epoetin-α reduced RBC transfusions and increased the time-to-first-transfusion compared with placebo.Thus, epoetin-α significantly improved anemia outcomes in low-risk MDS. IWG-2006 criteria for ER may require amendments to better apply to clinical studies.
2018
32
2648
2658
Fenaux P.; Santini V.; Spiriti M.A.A.; Giagounidis A.; Schlag R.; Radinoff A.; Gercheva-Kyuchukova L.; Anagnostopoulos A.; Oliva E.N.; Symeonidis A.; Berger M.H.; Gotze K.S.; Potamianou A.; Haralampiev H.; Wapenaar R.; Milionis I.; Platzbecker U.
File in questo prodotto:
File Dimensione Formato  
41375_2018_Article_118.pdf

accesso aperto

Descrizione: Articolo
Tipologia: Pdf editoriale (Version of record)
Licenza: Open Access
Dimensione 1.13 MB
Formato Adobe PDF
1.13 MB Adobe PDF

I documenti in FLORE sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificatore per citare o creare un link a questa risorsa: https://hdl.handle.net/2158/1175135
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 96
  • ???jsp.display-item.citation.isi??? 88
social impact