Fabio Efficace1, Valeria Santini2, Giorgio La Nasa3, Francesco Cottone1, Carlo Finelli4, Lorenza Borin5, Giulia Quaresmini6, Anna Angela Di Tucci7, Antonio Volpe8, Daniela Cilloni9, Giovanni Quarta10, Grazia Sanpaolo11, Flavia Rivellini12, Flavia Salvi13, Alfredo Molteni14, Maria Teresa Voso15, Giuliana Alimena16, Susanna Fenu17, Franco Mandelli1, Emanuele Angelucci7. 1. Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy. 2. Functional Unit of Haematology, Azienda Ospedaliero Universitaria Careggi, University of Florence, Florence, Italy. 3. Bone Marrow Transplantation Center, R. Binaghi Hospital, Cagliari, Italy. 4. Department of Hematology, Policlinico S. Orsola-Malpighi, Bologna, Italy. 5. Department of Hematology, San Gerardo Hospital, Monza, Italy. 6. Hematology and Bone Marrow Transplant Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy. 7. Hematology and Bone Marrow Transplantation Unit, Ospedale Oncologico di Riferimento Regionale "Armando Businco", Cagliari, Italy. 8. Hematology Division, A.O.R.N. San G. Moscati, Avellino, Italy. 9. Department of Clinical and Biological Sciences, University of Turin, Turin, Italy. 10. Division of Hematology, Ospedale A. Perrino, Brindisi, Italy. 11. Department of Hematology, IRCCS 'Casa Sollievo della Sofferenza' Hospital, San Giovanni Rotondo, Italy. 12. UO Medicina Interna e Onco-Ematologica P.O. "Umberto I", Nocera Inferiore, Italy. 13. Department of Hematology, A.O. Nazionale Santi Antonio e Biagio e C. Arrigo, Alessandria, Italy. 14. Department of Hematology, Niguarda Ca' Granda Hospital, Milan, Italy. 15. Department of Hematology, Università Cattolica del Sacro Cuore, Rome, Italy. 16. Department of Hematology, "Sapienza" University of Rome, Rome, Italy. 17. Department of Hematology, San Giovanni Hospital, Rome, Italy.
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. 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. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/
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. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/
Authors: Gregory A Abel; Fabio Efficace; Rena J Buckstein; Sara Tinsley; Joseph G Jurcic; Yolanda Martins; David P Steensma; Corey D Watts; Azra Raza; Stephanie J Lee; Alan F List; Robert J Klaassen Journal: Haematologica Date: 2016-03-04 Impact factor: 9.941
Authors: Naser Mobarra; Mehrnoosh Shanaki; Hassan Ehteram; Hajar Nasiri; Mehdi Sahmani; Mohsen Saeidi; Mehdi Goudarzi; Hoda Pourkarim; Mehdi Azad Journal: Int J Hematol Oncol Stem Cell Res Date: 2016-10-01
Authors: Carmen P Escalante; Stephanie Chisolm; Juhee Song; Marsha Richardson; Ellen Salkeld; Etsuko Aoki; Guillermo Garcia-Manero Journal: Cancer Med Date: 2019-01-11 Impact factor: 4.452