Identifying thresholds for meaningful improvements in NTDT-PRO scores to support conclusions about treatment benefit in clinical studies of patients with non-transfusion-dependent beta-thalassaemia: analysis of pooled data from a phase 2, double-blind, placebo-controlled, randomised trial

Identifying thresholds for meaningful improvements in NTDT-PRO scores to support conclusions about treatment benefit in clinical studies of patients with non-transfusion-dependent beta-thalassaemia: analysis of pooled data from a phase 2, double-blind, placebo-controlled, randomised trial

Objectives To estimate thresholds for defining meaningful within-patient improvement from baseline to weeks 13–24 and interpreting meaningfulness of between-group difference for the non-transfusion-dependent beta-thalassaemia patient-reported outcome (NTDT-PRO) tiredness/weakness (T/W) and shortness...

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Main Authors: Ali T Taher, Maria Domenica Cappellini, Khaled M Musallam, Vip Viprakasit, Antonis Kattamis, Jennifer Lord-Bessen, Aylin Yucel, Shien Guo, Alan L Shields, Jeevan K Shetty, Luciana Moro Bueno, Christopher G Pelligra, Mrudula B Glassberg
Format: Article
Language:English
Published: BMJ Publishing Group 2024-11-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/14/11/e085234.full
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https://bmjopen.bmj.com/content/14/11/e085234.full

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