{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T01:37:08Z","timestamp":1787017028970,"version":"build-2736575974"},"reference-count":28,"publisher":"BMJ","issue":"3","license":[{"start":{"date-parts":[[2023,3,21]],"date-time":"2023-03-21T00:00:00Z","timestamp":1679356800000},"content-version":"unspecified","delay-in-days":20,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Bristol Myers Squibb","award":["N\/A"],"award-info":[{"award-number":["N\/A"]}]}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["BMJ Open"],"accepted":{"date-parts":[[2023,3,5]]},"published-print":{"date-parts":[[2023,3]]},"abstract":"<jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>\n                      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 (\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"clintrialgov\" xlink:href=\"NCT03342404\">NCT03342404<\/jats:ext-link>\n                      ).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>Analysis of a phase 2, double-blind, randomised, placebo-controlled trial.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Setting<\/jats:title>\n                    <jats:p>USA, Greece, Italy, Lebanon, Thailand and the UK.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Participants<\/jats:title>\n                    <jats:p>Adults (\u226518 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 \u2264100\u2009g\/L.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Measures<\/jats:title>\n                    <jats:p>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\u2013Fatigue (FACIT-F) and Patient Global Impression of Severity (PGI-S).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Cronbach\u2019s alpha at weeks 13\u201324 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\u2013retest reliability. In a known-groups validity analysis, least-squares mean T\/W and SoB scores at weeks 13\u201324 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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1136\/bmjopen-2022-066683","type":"journal-article","created":{"date-parts":[[2023,5,3]],"date-time":"2023-05-03T06:56:38Z","timestamp":1683096998000},"page":"e066683","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":6,"title":["Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia"],"prefix":"10.1136","volume":"13","clinical-trial-number":[{"clinical-trial-number":"nct03342404","registry":"10.18810\/clinical-trials-gov"},{"clinical-trial-number":"nct03342404","registry":"10.18810\/clinical-trials-gov"}],"author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8515-2238","authenticated-orcid":false,"given":"Ali T","family":"Taher","sequence":"first","affiliation":[{"name":"Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3935-903X","authenticated-orcid":false,"given":"Khaled M","family":"Musallam","sequence":"additional","affiliation":[{"name":"Thalassemia Center, Burjeel Medical City, Abu Dhabi, UAE"},{"name":"International Network of Hematology, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vip","family":"Viprakasit","sequence":"additional","affiliation":[{"name":"Division of Hematology & Oncology, Department of Pediatrics & Siriraj Thalassemia Center, Siriraj Research Hospital, Mahidol University, Bangkok, Thailand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonis","family":"Kattamis","sequence":"additional","affiliation":[{"name":"First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Lord-Bessen","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb, Princeton, New Jersey, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aylin","family":"Yucel","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb, Princeton, New Jersey, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shien","family":"Guo","sequence":"additional","affiliation":[{"name":"Evidera, Waltham, Massachusetts, 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