Scholarlink

Aims <p>Nontuberculous mycobacterial pulmonary disease (NTM-PD) is a chronic infection often necessitating long-term, multidrug treatment. Here we report the development of a novel disease-specific patient-reported outcome measure (PROM) for use as a clinical trial endpoint.</p> Methods <p>We performed a literature review and conducted semi-structured interviews with adult subjects with NTM-PD recruited from four U.S. clinical sites. Interviews were recorded, transcribed, and consensus coded. NTM Symptom Scale (NTM-SS) items were selected and tested in cognitive interviews. We refined the NTM-SS using theoretical relevance/face validity, exploratory factor analysis (factorial validity), item response theory (IRT) and traditional reliability estimation, and convergent validity testing.</p> Results <p>The concept elicitation interviews identified key symptoms of cough (100%), shortness of breath (98%), fatigue (95%), sputum production (90%), throat clearing (80%), mental fogging (75%), sensitivity to cold (62%), night sweats (55%), poor appetite (53%), chills/feverishness (50%/43%), weight loss (48%), and chest congestion (45%). Within the conceptual framework of Respiratory Symptoms, Fatigue, and other NTM Symptoms, we selected an initial set of 44 items, editing 4 items after cognitive testing. Psychometric testing resulted in 7 subscales (final: 32 items). Scale reliabilities ranged from 0.85 to 0.95. Test–retest reliability was high (r = 0.73–0.88), and subscales showed strong convergent validity with existing measures (r = 0.64–0.87).</p> Conclusion <p>We identified a 32-item disease-specific PROM with a highly reliable set of 7 subscales. Separately scored NTM-SS subscales allow for selection of appropriate clinical trial endpoints. Further validation, including responsiveness to change, is needed.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Development of the novel nontuberculous mycobacterial pulmonary disease symptoms scale (NTM-SS)

  • 期刊论文

  • 发表日期：

    2026年5月3日

查看全文
  • E. Henkle,
  • N. F. Dieckmann,
  • H. Franklin,
  • D. Cella,
  • G. Megson,
  • C. L. Daley,
  • P. A. Flume,
  • P. J. McShane,
  • K. L. Winthrop,
  • A. L. Quittner

摘要

Aims

Nontuberculous mycobacterial pulmonary disease (NTM-PD) is a chronic infection often necessitating long-term, multidrug treatment. Here we report the development of a novel disease-specific patient-reported outcome measure (PROM) for use as a clinical trial endpoint.

Methods

We performed a literature review and conducted semi-structured interviews with adult subjects with NTM-PD recruited from four U.S. clinical sites. Interviews were recorded, transcribed, and consensus coded. NTM Symptom Scale (NTM-SS) items were selected and tested in cognitive interviews. We refined the NTM-SS using theoretical relevance/face validity, exploratory factor analysis (factorial validity), item response theory (IRT) and traditional reliability estimation, and convergent validity testing.

Results

The concept elicitation interviews identified key symptoms of cough (100%), shortness of breath (98%), fatigue (95%), sputum production (90%), throat clearing (80%), mental fogging (75%), sensitivity to cold (62%), night sweats (55%), poor appetite (53%), chills/feverishness (50%/43%), weight loss (48%), and chest congestion (45%). Within the conceptual framework of Respiratory Symptoms, Fatigue, and other NTM Symptoms, we selected an initial set of 44 items, editing 4 items after cognitive testing. Psychometric testing resulted in 7 subscales (final: 32 items). Scale reliabilities ranged from 0.85 to 0.95. Test–retest reliability was high (r = 0.73–0.88), and subscales showed strong convergent validity with existing measures (r = 0.64–0.87).

Conclusion

We identified a 32-item disease-specific PROM with a highly reliable set of 7 subscales. Separately scored NTM-SS subscales allow for selection of appropriate clinical trial endpoints. Further validation, including responsiveness to change, is needed.