Validation of a patient ‐reported outcomes symptom measure for patients with nontransfusion‐dependent thalassemia (NTDT‐PRO©)
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 compri ses 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; ...
Source: American Journal of Hematology - Category: Hematology 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 Tags: RESEARCH ARTICLE Source Type: research
More News: Anemia | Cancer | Cancer & Oncology | Chad Health | Greece Health | Hematology | Italy Health | Lebanon Health | Middle East Health | Study | Thailand Health