Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia
Issued Date
2023-03-22
Resource Type
eISSN
20446055
Scopus ID
2-s2.0-85150806850
Pubmed ID
36948565
Journal Title
BMJ Open
Volume
13
Issue
3
Rights Holder(s)
SCOPUS
Bibliographic Citation
BMJ Open Vol.13 No.3 (2023)
Suggested Citation
Taher A.T., Musallam K.M., Viprakasit V., Kattamis A., Lord-Bessen J., Yucel A., Guo S., Pelligra C., Shields A.L., Shetty J.K., Miteva D., Bueno L.M., Cappellini M.D. Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia. BMJ Open Vol.13 No.3 (2023). doi:10.1136/bmjopen-2022-066683 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/82358
Title
Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia
Other Contributor(s)
Abstract
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.