Patient‐reported disease burden in the Accelerate Clinical Trials in Charcot–Marie–Tooth Disease Study.

Autor:	Rehbein, T., Purks, J., Dilek, N., Behrens‐Spraggins, S., Sowden, J. E., Eichinger, K. J., Creigh, Peter, Koopman, Nicole, Wood, Elizabeth, Donlevy, Gabrielle, Cornett, Kayla, Bray, Paula, Mandarakas, Melissa, Pisciotta, Chiara, Cavalca, Eleonora, Schirinzi, Giulia, Saveri, Paola, Crivellari, Luca, Laura, Matilde, Donnell, Luke O.
Předmět:	STATISTICAL correlation RESEARCH funding T-test (Statistics) CLINICAL trials SEX distribution GLOBAL burden of disease AGE distribution DESCRIPTIVE statistics LONGITUDINAL method QUALITY of life RESEARCH HEALTH outcome assessment COMPARATIVE studies CONFIDENCE intervals CHARCOT-Marie-Tooth disease EVALUATION DISEASE complications
Zdroj:	Journal of the Peripheral Nervous System; Dec2024, Vol. 29 Issue 4, p487-493, 7p
Abstrakt:	Background and Aims: The Charcot–Marie–Tooth Disease Health Index (CMT‐HI) is a disease‐specific, patient‐reported disease burden measure. As part of an international clinical trial readiness study, individuals with CMT1A (ages 18–75 years) underwent clinical outcome assessments (COAs), including the CMT‐HI, to capture their longitudinal perspective on the disease burden. Methods: Two hundred and fifteen participants underwent serial COAs including the CMT‐HI, CMT Functional Outcome Measure (CMT‐FOM), CMT Neuropathy Score (CMTNSv2R), and CMT Exam Score (CMTES/CMTES‐R). Correlations between the total and subscale scores for the CMT‐HI and other COAs were determined. Changes in the CMT‐HI scores over 12 months were assessed using paired t‐tests. The minimum clinically important difference (MCID) for the CMT‐HI and its subscales were calculated by anchoring to a participant global impression of change scale. Results: At baseline, CMT1A participants were 44.5 ± 15 years old (range: 18–75) and 58% were women. The mean CMT‐HI was 25.7 ± 18.8 (range: 0–91.9; 100 reflecting maximal disease burden). The CMT‐HI correlated with the CMT‐FOM (r =.54, p <.0001), CMTNSv2R (r =.48, p <.0001), and CMTES/CMTES‐R (r =.52/r =.54, p <.0001). Disease burden was greater in women than in men (CMT‐HI 29.1 ± 19.1 vs. 21.2 ± 17.3, p =.001). Over 12 months, there was a nonsignificant mean increase in CMT‐HI of 0.40 ± 10.0 (n = 189, p =.89). The MCID for the CMT‐HI total score was 3.8 points (95% CI: 1.7–5.9). Discussion: Patient‐reported disease burden in CMT1A as measured by the CMT‐HI is associated with measures of neurologic impairment and physical functioning. Women reported a higher disease burden than men. These data will inform the design of clinical trials in CMT1A. [ABSTRACT FROM AUTHOR]
Databáze:	Complementary Index
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