Outcomes in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with isolated deletion 5q treated with lenalidomide: a subset analysis from the MDS-004 study

Autores de IIS La Fe

• Guillermo Sanz Santillana

  Autor

Participantes ajenos a IIS La Fe

• Giagounidis A
• Mufti GJ
• Mittelman M
• Platzbecker U
• Muus P
• Selleslag D
• Beyne-Rauzy O
• te Boekhorst P
• del Cañizo C
• Guerci-Bresler A
• Nilsson L
• Lübbert M
• Quesnel B
• Ganser A
• Bowen D
• Schlegelberger B
• Göhring G
• Fu T
• Benettaib B
• Hellström-Lindberg E
• Fenaux P

Grupos

• Hematología y Hemoterapia

  

  Investigador Principal

  Javier De La Rubia Comos

Abstract

ObjectiveA subset analysis of the randomised, phase 3, MDS-004 study to evaluate outcomes in patients with International Prognostic Scoring System (IPSS)-defined Low-/Intermediate (Int)-1-risk myelodysplastic syndromes (MDS) with isolated del(5q). MethodsPatients received lenalidomide 10mg/d (days 1-21; n=47) or 5mg/d (days 1-28; n=43) on 28-d cycles or placebo (n=45). From the placebo and lenalidomide 5mg groups, 84% and 58% of patients, respectively, crossed over to lenalidomide 5 or 10mg at 16wk, respectively. ResultsRates of red blood cell-transfusion independence (RBC-TI) 182d were higher in the lenalidomide 10mg (57.4%; P<0.0001) and 5mg (37.2%; P=0.0001) groups vs. placebo (2.2%). Cytogenetic response rates (major+minor responses) were 56.8% (P<0.0001), 23.1% (P=0.0299) and 0%, respectively. Two-year cumulative risk of acute myeloid leukaemia progression was 12.6%, 17.4% and 16.7% in the lenalidomide 10mg, 5mg, and placebo groups, respectively. In a 6-month landmark analysis, overall survival was longer in lenalidomide-treated patients with RBC-TI 182d vs. non-responders (P=0.0072). The most common grade 3-4 adverse event was myelosuppression. ConclusionsThese data support the clinical benefits and acceptable safety profile of lenalidomide in transfusion-dependent patients with IPSS-defined Low-/Int-1-risk MDS with isolated del(5q).

Keywords

• acute myeloid leukaemia; del(5q); lenalidomide; myelodysplastic syndromes; transfusion independence