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Health-related quality of life in transfusion-dependent patients with myelodysplastic syndromes: a prospective study to assess the impact of iron chelation therapy
  1. Fabio Efficace1,
  2. Valeria Santini2,
  3. Giorgio La Nasa3,
  4. Francesco Cottone1,
  5. Carlo Finelli4,
  6. Lorenza Borin5,
  7. Giulia Quaresmini6,
  8. Anna Angela Di Tucci7,
  9. Antonio Volpe8,
  10. Daniela Cilloni9,
  11. Giovanni Quarta10,
  12. Grazia Sanpaolo11,
  13. Flavia Rivellini12,
  14. Flavia Salvi13,
  15. Alfredo Molteni14,
  16. Maria Teresa Voso15,
  17. Giuliana Alimena16,
  18. Susanna Fenu17,
  19. Franco Mandelli1 and
  20. Emanuele Angelucci7
  1. 1Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy
  2. 2Functional Unit of Haematology, Azienda Ospedaliero Universitaria Careggi, University of Florence, Florence, Italy
  3. 3Bone Marrow Transplantation Center, R. Binaghi Hospital, Cagliari, Italy
  4. 4Department of Hematology, Policlinico S. Orsola-Malpighi, Bologna, Italy
  5. 5Department of Hematology, San Gerardo Hospital, Monza, Italy
  6. 6Hematology and Bone Marrow Transplant Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy
  7. 7Hematology and Bone Marrow Transplantation Unit, Ospedale Oncologico di Riferimento Regionale “Armando Businco”, Cagliari, Italy
  8. 8Hematology Division, A.O.R.N. San G. Moscati, Avellino, Italy
  9. 9Department of Clinical and Biological Sciences, University of Turin, Turin, Italy
  10. 10Division of Hematology, Ospedale A. Perrino, Brindisi, Italy
  11. 11Department of Hematology, IRCCS ‘Casa Sollievo della Sofferenza’ Hospital, San Giovanni Rotondo, Italy
  12. 12UO Medicina Interna e Onco-Ematologica P.O. “Umberto I”, Nocera Inferiore, Italy
  13. 13Department of Hematology, A.O. Nazionale Santi Antonio e Biagio e C. Arrigo, Alessandria, Italy
  14. 14Department of Hematology, Niguarda Ca’ Granda Hospital, Milan, Italy
  15. 15Department of Hematology, Università Cattolica del Sacro Cuore, Rome, Italy
  16. 16Department of Hematology, “Sapienza” University of Rome, Rome, Italy
  17. 17Department of Hematology, San Giovanni Hospital, Rome, Italy
  1. Correspondence to Dr Fabio Efficace, Head, Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), GIMEMA Data Center, Via Benevento, 6, –Rome 00161, Italy; f.efficace{at}


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.

Patients and 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.

Trial registration number NCT00469560.

  • Chelation Therapy
  • Quality of life
  • Myelodysplastic Syndromes
  • Symptom Burden
  • Transfusions

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