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-blin
The thresholds proposed may support the use of NTDT-PRO in assessing and interpreting treatment effects in clinical studies and identifying patients with NTDT in need of symptom relief.
Authors
- Aylin Yücel (ORCID: https://orcid.org/0000-0002-7442-4549)
- Mrudula B. Glassberg (ORCID: https://orcid.org/0000-0003-1929-7775)
- Luciana Moro Bueno
- Alan L. Shields (ORCID: https://orcid.org/0000-0003-3362-3135)
- Jeevan K. Shetty (ORCID: https://orcid.org/0000-0001-9311-6675)
- Antonis Kattamis (ORCID: https://orcid.org/0000-0002-5178-0655)
- Khaled M. Musallam (ORCID: https://orcid.org/0000-0003-3935-903X)
- Maria Domenica Cappellini (ORCID: https://orcid.org/0000-0001-8676-6864)
- C. Pelligra (ORCID: https://orcid.org/0000-0002-5255-2777)
- Alì Taher (ORCID: https://orcid.org/0000-0001-8515-2238)
- Jennifer Lord‐Bessen (ORCID: https://orcid.org/0000-0001-8624-4556)
- Vip Viprakasit (ORCID: https://orcid.org/0000-0003-3162-1849)
- Shien Guo
Institutions
- Ithaka Harbors (US)
- Siriraj Hospital (TH)
- University of Milan (IT)
- National and Kapodistrian University of Athens (GR)
- Cornell University (US)
- Mahidol University (TH)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Bristol-Myers Squibb (United States) (US)
- Bristol-Myers Squibb (France) (FR)
- American University of Beirut Medical Center (LB)
- Adelphi Laboratory Center (US)
- Bristol-Myers Squibb (Switzerland) (CH)
Publication Details
- Journal
- BMJ Open
- Published
- 2024-11-01
- DOI
- https://doi.org/10.1136/bmjopen-2024-085234
- Citations
- 1
- Primary Topic
- Hemoglobinopathies and Related Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.44
Funders
- Bristol-Myers Squibb