title: Pomalidomide and Low-Dose Dexamethasone Improves Health-Related Quality of Life and Prolongs Time to Worsening in Relapsed/Refractory Patients With Multiple Myeloma Enrolled in the MM-003 Randomized Phase III Trial.
abstract: BACKGROUND
  Health-related quality of life (HRQoL) is an important element for consideration in treatment decisions in patients with relapsed/refractory multiple myeloma (RRMM). The pivotal MM-003 (A Phase 3, Multicenter, Randomized, Open-Label Study to Compare the Efficacy and Safety of Pomalidomide in Combination With Low-Dose Dexamethasone vs. High-Dose Dexamethasone in Patients With Refractory Multiple Myeloma or Relapsed and Refractory Multiple Myeloma and Companion Study [NIMBUS]) randomized, open-label, multicenter, phase III trial demonstrated improved progression-free survival (PFS) and prolonged overall survival (OS) with pomalidomide (POM) plus low-dose dexamethasone (POM + LoDEX) versus high-dose dexamethasone (HiDEX) in patients with RRMM in whom lenalidomide (LEN) and bortezomib (BORT) had failed. MM-003 also investigated HRQoL as a predefined secondary end point.
  
  
  METHODS
  Recruited patients (n = 455) were refractory to their last treatment and had failed LEN and BORT after ≥ 2 consecutive cycles of each (alone or in combination). Eight clinically relevant and validated HRQoL domains from the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30, EORTC QLQ-MY20, and EQ-5D questionnaires were selected for analysis. Time to symptom worsening based on minimally important differences (MIDs) was calculated.
  
  
  RESULTS
  Clinically meaningful improvements in HRQoL as determined by MIDs, regression analyses, and best response analyses were observed more frequently in patients receiving POM + LoDEX than in those receiving HiDEX. POM + LoDEX significantly extended median time to clinically meaningful worsening in HRQoL versus HiDEX in 4 HRQoL domains and demonstrated a trend in an additional 3 domains. Patients in the HiDEX arm experienced earlier HRQoL deterioration compared with those in the POM + LoDEX arm in each domain analyzed.
  
  
  CONCLUSIONS
  POM + LoDEX offer good clinical outcomes that lead to improved and prolonged HRQoL compared with HiDEX in patients with RRMM and end-stage disease.
authors.affiliation: University Hospital Tübingen, Tübingen, Germany. Electronic address: katja.weisel@med.uni-tuebingen.de.
authors.forename: Katja
authors.initials: K
authors.lastname: Weisel
authors.affiliation: Alexandra Hospital, Athens, Greece.
authors.forename: Meletios
authors.initials: M
authors.lastname: Dimopoulos
authors.affiliation: Vancouver General Hospital, Vancouver, BC, Canada.
authors.forename: Kevin W
authors.initials: KW
authors.lastname: Song
authors.affiliation: University Hospital Hotel-Dieu, Nantes, France.
authors.forename: Philippe
authors.initials: P
authors.lastname: Moreau
authors.affiliation: University Hospital and Health City of Science of Torino, Torino, Italy.
authors.forename: Antonio
authors.initials: A
authors.lastname: Palumbo
authors.affiliation: University of Alberta, Edmonton, Canada.
authors.forename: Andrew
authors.initials: A
authors.lastname: Belch
authors.affiliation: King's College Hospital and King's College London, London, UK.
authors.forename: Stephen
authors.initials: S
authors.lastname: Schey
authors.affiliation: Erasmus Medical Center Rotterdam, Rotterdam, The Netherlands.
authors.forename: Pieter
authors.initials: P
authors.lastname: Sonneveld
authors.affiliation: Celgene Corporation, Summit, NJ.
authors.forename: Lars
authors.initials: L
authors.lastname: Sternas
authors.affiliation: Celgene Corporation, Summit, NJ.
authors.forename: Xin
authors.initials: X
authors.lastname: Yu
authors.affiliation: Adelphi Values, Boston, MA.
authors.forename: Ramesh
authors.initials: R
authors.lastname: Amatya
authors.affiliation: Celgene Corporation, Summit, NJ.
authors.forename: Craig J
authors.initials: CJ
authors.lastname: Gibson
authors.affiliation: Celgene Corporation, Summit, NJ.
authors.forename: Mohamed
authors.initials: M
authors.lastname: Zaki
authors.affiliation: Celgene Corporation, Summit, NJ.
authors.forename: Christian
authors.initials: C
authors.lastname: Jacques
authors.affiliation: Hospital Universitario de Salamanca, Salamanca, Spain.
authors.forename: Jesus
authors.initials: J
authors.lastname: San Miguel
mesh_terms: D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D000069286:Bortezomib / Q000627:therapeutic use; D003907:Dexamethasone / Q000627:therapeutic use*; D018450:Disease Progression; D018572:Disease-Free Survival; D019008:Drug Resistance, Neoplasm / Q000187:drug effects; D005260:Female; D006801:Humans; D000077269:Lenalidomide; D008297:Male; D009101:Multiple Myeloma / Q000188:drug therapy*; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy*; D011788:Quality of Life; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D013792:Thalidomide; D000069286:Bortezomib; D003907:Dexamethasone; C467566:pomalidomide; D000077269:Lenalidomide
keywords: Low dose dexamethasone; Multiple myeloma; Pomalidomide; Quality of life; Relapsed/Refractory