Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia.

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

SUBMITTER: Taher AT 

PROVIDER: S-EPMC10040027 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature