Ali Taher 1 | Maria Domenica Cappellini 2 | Vip Viprakasit 3 | Pranee Sutcharitchan 4 |Dalia Mahmoud 5 | Abderrahmane Laadem 5 | Anzalee Khan 6,7 | Chad Gwaltney 8 |Gale Harding 9 | Kenneth Attie 10 | Xiaosha Zhang10 | Jun Zou 5 | Joseph Pariseau 5 |X. Henry Hu 5† | Antonis Kattamis 11

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 (rs = −0.53), and between SoB and Fact-An-Fatigue Experience (rs = −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.

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Taher_et_al-NTDT-PRO Validation-2018-American_Journal_of_Hematology