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Thalassemia is a chronic, inherited blood disorder, which, in its most severe form, causes life-threatening anemia. Advances in treatment have led to increased life expectancy however the need for chronic blood transfusions and chelation therapy remains a significant burden for patients. Our study compared health related quality of life (HRQOL) from the Thalassemia Clinical Research Network's (TCRNs) Thalassemia Longitudinal Cohort (TLC) study to US norms and assessed association with clinical variables. There were 264 patients over age 14 who completed the Medical Outcomes Study 36-Item Short Form Health Survey version 2 (SF36v2) baseline assessment. When compared to US norms, TLC patients had statistically significant (P 0.2, where 0.2–0.49 = “small,” 0.50–0.79 = “moderate” and >0.80 = “large” 7, 8. A difference in at least 2 points on the normalized (mean of 50) SF-36 scores was used as an approximation of a clinically relevant MCID. Descriptive statistics were calculated for demographic variables (age, gender, race, country), transfusion status (transfused versus nontransfused), chelation choice (deferoxamine, oral chelator, combination therapy or no chelation), complications (as a continuous measure), ferritin (log transformed) and measures of adherence, as well as the SF-36 component scores and summary scores. Because of the differences noted in the UK patients, analyses were re-run with North American patients only. Bivariate analysis was done on each of the above mentioned variables with the SF-36 scores to determine associations and for consideration of inclusion in the final multivariate model. Multivariate analysis was done including factors that were significant on bivariate screen (age, gender, country, complications, chelator choice, side effects, ferritin, transfusion status). Race was kept in the model despite not being significant on univariate screen because it was felt that it was important to include in this population. Since the only measure of adherence with a significant association with SF-36 scores was “side-effect,” it was the only adherence measure included in the final model. The final multivariate model included gender, race (Asian vs. non-Asian), chelator choice (only for patients on chelator), self-reported frequency of side effects from chelation, country, number of complications (as a continuous measure), ferritin (in log scale) and transfusion status (transfused versus not-transfused). Statistical analyses were performed with SAS 9.2 for Windows (SAS Institute Inc., Cary, NC). QualityMetric Health Outcomes™ Scoring Software 2.0 (QualityMetric Inc., Lincoln, RI) was used to score the SF36 data. The following institutions and researchers contributed to the Thalassemia Clinical Research Network Thalassemia Longitudinal Cohort data reported in this paper. Children's Hospital, Boston (N = 38): Ellis Neufeld, MD, PhD, Principal Investigator, Jennifer Braunstein, NP, Research Nurse, Amber Smith, Study Coordinator, Latoya Lashley, Study Coordinator; Satellite: University of Texas Southwestern Medical Center at Dallas (N = 12), Charles Quinn, MD, MS, Principal Investigator, Deborah Boger, RN, MSN, PNP, Study Coordinator, Leah Adix, Study Coordinator, Sandra Richardson, Study Coordinator; Children's Healthcare of Atlanta (N = 16), Jeanne Boudreaux, MD, Principal Investigator, Leann Hassen, Study Coordinator; Baylor College of Medicine (N = 6), Brigitta Mueller, MD, Principal Investigator, Bogden Dino, Study Coordinator. Weill Medical College of Cornell University (N = 59): Patricia Giardina, MD, Principal Investigator, Elizabeth Evans, Study Coordinator; Satellite: Winthrop University Hospital (N = 6), Mark Weinblatt, MD, Principal Investigator, Linda Skelly, Study Coordinator. The Children's Hospital of Philadelphia (N = 59): Janet Kwiatkowski, MD, Principal Investigator, Marie Martin, RN, Research Nurse, Owen Beams, Study Coordinator; Satellite: Children's Memorial Hospital, Chicago, IL (N = 39), Alexis Thompson, MD, Principal Investigator, Janice Beatty, RN, Research Nurse, Tiffany Drinkwater, Study Coordinator. Children's Hospital at Oakland (N = 52): Elliott Vichinsky, MD, Principal Investigator, Dru Foote, NP, Research Nurse, Nancy Sweeters, Study Coordinator, Olivia Vega, Study Coordinator; Satellites: Children's Hospital of Los Angeles (N = 12), Thomas Coates, MD, Principal Investigator, Susan Carson, RN, Research Nurse, Eun Ha Pang, Study Coordinator, Rachna Khanna, Study Coordinator; Stanford Hospital (N = 5), Michael Jeng, MD, Principal Investigator, Kokil Bakshi, Clinical Research Associate; Children's and Women's Health Center of British Columbia (N = 4), John Wu, Principal Investigator, Heather McCartney, RN, Research Nurse, Colleen Fitzgerald, Study Coordinator, Stephanie Badour, Study Coordinator. Toronto General Hospital, Toronto, Ontario, Canada (N = 5): Nancy F. Olivieri, MD, Principal Investigator, Vivek Thayalasuthan, Study Coordinator; Satellite: Hospital for Sick Children (N = 64), Isaac Odame, MD, Principal Investigator, Manuela Merelles-Pulcini, RN, Study Coordinator. University College London (N = 15), John Porter, MD, Principal Investigator, Cindy Bhagwandin, Study Coordinator; Satellite: Whittington Hospital (N = 24), Farrukh Shah, MD, Principal Investigator. NHLBI oversight, Kathryn Hassell, MD. Data Coordinating Center: New Research PhD, Principal Investigator, RN, MS, PhD,
Sobota et al. (Fri,) studied this question.