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Title: Validation of a patient-reported outcomes symptom measure for patients with nontransfusion-dependent thalassemia (NTDT-PRO <sup>©</sup> )
Authors: Ali Taher
Maria Domenica Cappellini
Vip Viprakasit
Pranee Sutcharitchan
Dalia Mahmoud
Abderrahmane Laadem
Anzalee Khan
Chad Gwaltney
Gale Harding
Kenneth Attie
Xiaosha Zhang
Jun Zou
Joseph Pariseau
X. Henry Hu
Antonis Kattamis
Evidera, USA
American University of Beirut Medical Center
Università degli Studi di Milano
Chulalongkorn University
Aghia Sophia Children's Hospital
Celgene Corporation
Faculty of Medicine, Siriraj Hospital, Mahidol University
Nathan S. Kline Institute for Psychiatric Research
NeuroCog Trials
Acceleron Pharma
Keywords: Medicine
Issue Date: 1-Feb-2019
Citation: American Journal of Hematology. Vol.94, No.2 (2019), 177-183
Abstract: © 2018 The Authors. American Journal of Hematology published by Wiley Periodicals, Inc. This study demonstrates the quantitative characteristics of the first patient-reported outcome (PRO) tool developed for patients with nontransfusion-dependent β-thalassemia (NTDT), the NTDT-PRO © . A multicenter validation study was performed over 24 weeks, involving 48 patients from Italy, Lebanon, Greece, and Thailand. Most patients were female (68.8%), with a median age of 34.5 years (range, 18-52); 66.7% were diagnosed with β-thalassemia intermedia, and median time since diagnosis was 22 years (range, 0-43). The NTDT-PRO comprises 6 items across 2 domains (Tiredness/Weakness and Shortness of Breath [SoB]), and was valid and reliable, with good consistency. At baseline, most patients reported symptoms as present via the NTDT-PRO, and were highly compliant, ≥90% completing the NTDT-PRO tool. In a pairwise correlation analysis, all items were positively correlated. Correlations between NTDT-PRO and existing tools—36-Item Short Form Health Survey version 2 (SF-36v2) and Functional Assessment of Cancer Therapy-Anemia (FACT-An)—were assessed at weeks 1, 3, and 12; robust correlations were seen between SoB and SF-36v2-Vitality (r s = −0.53), and between SoB and Fact-An-Fatigue Experience (r s = −0.66) at week 1. Internal consistency was high for both Tiredness/Weakness (Cronbach alpha, 0.91) and SoB (Spearman-Brown coefficient, 0.78); intraclass correlation coefficients were high (Tiredness/Weakness, 0.88 and 0.97; SoB, 0.92 and 0.98), demonstrating stability. Further studies are required to fully support the validity of this tool, this study demonstrated the usefulness of the NTDT-PRO in the clinical setting and for longitudinal clinical research, particularly in trials where patient health-related quality of life is expected to change.
ISSN: 10968652
Appears in Collections:Scopus 2019

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