title: Health-related quality of life in transfusion-dependent patients with myelodysplastic syndromes: a prospective study to assess the impact of iron chelation therapy.
abstract: OBJECTIVE
  The primary objective of this study was to evaluate the health-related quality of life (HRQOL) in lower-risk, transfusion-dependent patients with myelodysplastic syndromes (MDS) treated with deferasirox. A secondary objective was to investigate the relationship between HRQOL, serum ferritin levels and transfusion dependency.
  
  
  METHODS
  This was a prospective multicentre study enrolling 159 patients, of whom 152 received at least one dose of deferasirox. HRQOL was assessed with the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) at baseline and then at 3, 6, 9 and 12 months. Primary analysis was performed estimating mean HRQOL scores over time by a linear mixed model on selected scales.
  
  
  RESULTS
  The median age of treated patients was 72 years (range 24-87 years). No statistically significant changes over time were found in mean scores for global health status/quality of life (p=0.564), physical functioning (p=0.409) and fatigue (p=0.471) scales. Also, no significant changes were found for constipation (p=0.292), diarrhoea (p=0.815) and nausea and vomiting (p=0.643). Serum ferritin levels were not associated with HRQOL outcomes. A higher patient-reported baseline pain severity was an independent predictive factor of an earlier achievement of transfusion independence with a HR of 1.032 (99% CI 1.004 to 1.060; p=0.003).
  
  
  CONCLUSIONS
  HRQOL of transfusion-dependent patients with MDS receiving deferasirox therapy remains stable over time. HRQOL assessment might also provide important predictive information on treatment outcomes.
  
  
  BACKGROUND
  NCT00469560.
authors.affiliation: Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
authors.forename: Fabio
authors.initials: F
authors.lastname: Efficace
authors.affiliation: Functional Unit of Haematology, Azienda Ospedaliero Universitaria Careggi, University of Florence, Florence, Italy.
authors.forename: Valeria
authors.initials: V
authors.lastname: Santini
authors.affiliation: Bone Marrow Transplantation Center, R. Binaghi Hospital, Cagliari, Italy.
authors.forename: Giorgio
authors.initials: G
authors.lastname: La Nasa
authors.affiliation: Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Cottone
authors.affiliation: Department of Hematology, Policlinico S. Orsola-Malpighi, Bologna, Italy.
authors.forename: Carlo
authors.initials: C
authors.lastname: Finelli
authors.affiliation: Department of Hematology, San Gerardo Hospital, Monza, Italy.
authors.forename: Lorenza
authors.initials: L
authors.lastname: Borin
authors.affiliation: Hematology and Bone Marrow Transplant Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy.
authors.forename: Giulia
authors.initials: G
authors.lastname: Quaresmini
authors.affiliation: Hematology and Bone Marrow Transplantation Unit, Ospedale Oncologico di Riferimento Regionale "Armando Businco", Cagliari, Italy.
authors.forename: Anna Angela
authors.initials: AA
authors.lastname: Di Tucci
authors.affiliation: Hematology Division, A.O.R.N. San G. Moscati, Avellino, Italy.
authors.forename: Antonio
authors.initials: A
authors.lastname: Volpe
authors.affiliation: Department of Clinical and Biological Sciences, University of Turin, Turin, Italy.
authors.forename: Daniela
authors.initials: D
authors.lastname: Cilloni
authors.affiliation: Division of Hematology, Ospedale A. Perrino, Brindisi, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Quarta
authors.affiliation: Department of Hematology, IRCCS 'Casa Sollievo della Sofferenza' Hospital, San Giovanni Rotondo, Italy.
authors.forename: Grazia
authors.initials: G
authors.lastname: Sanpaolo
authors.affiliation: UO Medicina Interna e Onco-Ematologica P.O. "Umberto I", Nocera Inferiore, Italy.
authors.forename: Flavia
authors.initials: F
authors.lastname: Rivellini
authors.affiliation: Department of Hematology, A.O. Nazionale Santi Antonio e Biagio e C. Arrigo, Alessandria, Italy.
authors.forename: Flavia
authors.initials: F
authors.lastname: Salvi
authors.affiliation: Department of Hematology, Niguarda Ca' Granda Hospital, Milan, Italy.
authors.forename: Alfredo
authors.initials: A
authors.lastname: Molteni
authors.affiliation: Department of Hematology, Università Cattolica del Sacro Cuore, Rome, Italy.
authors.forename: Maria Teresa
authors.initials: MT
authors.lastname: Voso
authors.affiliation: Department of Hematology, "Sapienza" University of Rome, Rome, Italy.
authors.forename: Giuliana
authors.initials: G
authors.lastname: Alimena
authors.affiliation: Department of Hematology, San Giovanni Hospital, Rome, Italy.
authors.forename: Susanna
authors.initials: S
authors.lastname: Fenu
authors.affiliation: Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
authors.forename: Franco
authors.initials: F
authors.lastname: Mandelli
authors.affiliation: Hematology and Bone Marrow Transplantation Unit, Ospedale Oncologico di Riferimento Regionale "Armando Businco", Cagliari, Italy.
authors.forename: Emanuele
authors.initials: E
authors.lastname: Angelucci
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001565:Benzoates / Q000008:administration & dosage / Q000627:therapeutic use*; D000077588:Deferasirox; D005260:Female; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000378:metabolism / Q000523:psychology*; D011446:Prospective Studies; D011788:Quality of Life; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox
keywords: Chelation Therapy; Myelodysplastic Syndromes; Quality of life; Symptom Burden; Transfusions