RT Journal Article
SR Electronic
T1 Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia
JF BMJ Open
JO BMJ Open
FD British Medical Journal Publishing Group
SP e066683
DO 10.1136/bmjopen-2022-066683
VO 13
IS 3
A1 Taher, Ali T
A1 Musallam, Khaled M
A1 Viprakasit, Vip
A1 Kattamis, Antonis
A1 Lord-Bessen, Jennifer
A1 Yucel, Aylin
A1 Guo, Shien
A1 Pelligra, Christopher
A1 Shields, Alan L
A1 Shetty, Jeevan K
A1 Miteva, Dimana
A1 Bueno, Luciana Moro
A1 Cappellini, Maria Domenica
YR 2023
UL http://bmjopen.bmj.com/content/13/3/e066683.abstract
AB Objectives The non-transfusion-dependent beta-thalassaemia-patient-reported outcome (NTDT-PRO) questionnaire was developed for assessing anaemia-related tiredness/weakness (T/W) and shortness of breath (SoB) among patients with NTDT. Psychometric properties were evaluated using blinded data from the BEYOND trial (NCT03342404).Design Analysis of a phase 2, double-blind, randomised, placebo-controlled trial.Setting USA, Greece, Italy, Lebanon, Thailand and the UK.Participants Adults (≥18 years) (N=145) with NTDT who had not received a red blood cell transfusion within 8 weeks prior to randomisation, with mean baseline haemoglobin level ≤100 g/L.Measures NTDT-PRO daily scores from baseline until week 24, and scores at select time points for the 36-Item Short Form Health Survey version 2 (SF-36v2), Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT-F) and Patient Global Impression of Severity (PGI-S).Results Cronbach’s alpha at weeks 13–24 was 0.95 and 0.84 for the T/W and SoB domains, respectively, indicating acceptable internal consistency reliability. Among participants self-reporting no change in thalassaemia symptoms via the PGI-S between baseline and week 1, intraclass correlation coefficients were 0.94 and 0.92 for the T/W and SoB domains, respectively, indicating excellent test–retest reliability. In a known-groups validity analysis, least-squares mean T/W and SoB scores at weeks 13–24 were worse in participants with worse scores for the FACIT-F Fatigue Subscale (FS), SF-36v2 vitality or PGI-S. Indicating responsiveness, changes in T/W and SoB domain scores were moderately correlated with changes in haemoglobin levels, and strongly correlated with changes in SF-36v2 vitality, FACIT-F FS, select FACIT-F items and the PGI-S. Improvements in least-squares mean T/W and SoB scores were higher in participants with greater improvements in scores on other PROs measuring similar constructs.Conclusions The NTDT-PRO demonstrated adequate psychometric properties to assess anaemia-related symptoms in adults with NTDT and can be used to evaluate treatment efficacy in clinical trials.Data are available upon reasonable request. All data relevant to the study are included in the article or uploaded as supplemental information.