title: 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.
abstract: OBJECTIVE
  A 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).
  
  
  METHODS
  Patients received lenalidomide 10 mg/d (days 1-21; n = 47) or 5 mg/d (days 1-28; n = 43) on 28-d cycles or placebo (n = 45). From the placebo and lenalidomide 5 mg groups, 84% and 58% of patients, respectively, crossed over to lenalidomide 5 or 10 mg at 16 wk, respectively.
  
  
  RESULTS
  Rates of red blood cell-transfusion independence (RBC-TI) ≥182 d were higher in the lenalidomide 10 mg (57.4%; P < 0.0001) and 5 mg (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 10 mg, 5 mg, and placebo groups, respectively. In a 6-month landmark analysis, overall survival was longer in lenalidomide-treated patients with RBC-TI ≥182 d vs. non-responders (P = 0.0072). The most common grade 3-4 adverse event was myelosuppression.
  
  
  CONCLUSIONS
  These 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).
authors.affiliation: Marien Hospital Düsseldorf, Düsseldorf, Germany.
authors.forename: Aristoteles
authors.initials: A
authors.lastname: Giagounidis
authors.forename: Ghulam J
authors.initials: GJ
authors.lastname: Mufti
authors.forename: Moshe
authors.initials: M
authors.lastname: Mittelman
authors.forename: Guillermo
authors.initials: G
authors.lastname: Sanz
authors.forename: Uwe
authors.initials: U
authors.lastname: Platzbecker
authors.forename: Petra
authors.initials: P
authors.lastname: Muus
authors.forename: Dominik
authors.initials: D
authors.lastname: Selleslag
authors.forename: Odile
authors.initials: O
authors.lastname: Beyne-Rauzy
authors.forename: Peter
authors.initials: P
authors.lastname: te Boekhorst
authors.forename: Consuelo
authors.initials: C
authors.lastname: del Cañizo
authors.forename: Agnès
authors.initials: A
authors.lastname: Guerci-Bresler
authors.forename: Lars
authors.initials: L
authors.lastname: Nilsson
authors.forename: Michael
authors.initials: M
authors.lastname: Lübbert
authors.forename: Bruno
authors.initials: B
authors.lastname: Quesnel
authors.forename: Arnold
authors.initials: A
authors.lastname: Ganser
authors.forename: David
authors.initials: D
authors.lastname: Bowen
authors.forename: Brigitte
authors.initials: B
authors.lastname: Schlegelberger
authors.forename: Gudrun
authors.initials: G
authors.lastname: Göhring
authors.forename: Tommy
authors.initials: T
authors.lastname: Fu
authors.forename: Bouchra
authors.initials: B
authors.lastname: Benettaib
authors.forename: Eva
authors.initials: E
authors.lastname: Hellström-Lindberg
authors.forename: Pierre
authors.initials: P
authors.lastname: Fenaux
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002872:Chromosome Deletion; D002895:Chromosomes, Human, Pair 5; D020732:Cytogenetic Analysis; D018450:Disease Progression; D004334:Drug Administration Schedule; D017707:Erythrocyte Transfusion; D005260:Female; D006801:Humans; D000077269:Lenalidomide; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000235:genetics / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D012189:Retrospective Studies; D012306:Risk; D016019:Survival Analysis; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D013792:Thalidomide; D000077269:Lenalidomide
keywords: acute myeloid leukaemia; del(5q); lenalidomide; myelodysplastic syndromes; transfusion independence