Article ; Online: Establishing how much improvement in lung function and distance walked is clinically important for adult patients with Pompe disease.
2024 Volume 31, Issue 5, Page(s) e16223
Abstract: Background and purpose: Pompe disease is a rare, inheritable, progressive metabolic myopathy. This study aimed to estimate the minimal clinically important difference (MCID) for an improvement in forced vital capacity in the upright seated position (FVC! ...
Abstract | Background and purpose: Pompe disease is a rare, inheritable, progressive metabolic myopathy. This study aimed to estimate the minimal clinically important difference (MCID) for an improvement in forced vital capacity in the upright seated position (FVC Methods: Data were obtained from two prospective follow-up studies. Between-group and within-group MCIDs were estimated using anchor-based methods. Additionally, a distribution-based method was used to generate supportive evidence. As anchors, self-reported change in health and in physical functioning, shortness of breath and a categorization of the Short-Form 36 Physical Component Summary score were used. Anchor appropriateness was assessed using Spearman correlations (absolute values ≥0.29) and a sufficient number of observations in each category. Results: In all, 102 patients had at least one FVC Conclusion: The MCIDs for FVC |
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MeSH term(s) | Male ; Adult ; Female ; Humans ; Middle Aged ; Glycogen Storage Disease Type II/drug therapy ; Prospective Studies ; Walk Test ; Follow-Up Studies ; Lung ; Treatment Outcome |
Language | English |
Publishing date | 2024-02-20 |
Publishing country | England |
Document type | Journal Article |
ZDB-ID | 1280785-0 |
ISSN | 1468-1331 ; 1351-5101 ; 1471-0552 |
ISSN (online) | 1468-1331 |
ISSN | 1351-5101 ; 1471-0552 |
DOI | 10.1111/ene.16223 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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