Lipodystrophy Severity Score to Assess Disease Burden in Lipodystrophy
Authors' conclusion
The LDS can reflect the severity of diverse manifestations of lipodystrophy and monitor changes following interventions.
Study context
- Population
- Patients with lipodystrophy syndromes; LDS rated by 28 clinicians and 9 patient representatives; 20 putative patient profiles at 2 time points
- Outcome
- LDS validity and reliability
Methodological detailsexpert consensus development, external validation in 2 metreleptin-treated cohorts, 8-domain LDS developed by 8 disease experts with patient organizations
- Expert consensus development
- External validation in 2 metreleptin-treated cohorts
- 8-domain LDS developed by 8 disease experts with patient organizations
Performance
As reported in the abstract
Reliability, intraclass correlation coefficients
>0.95
Correlation of global and domain-specific LDS and of score changes across visits with Clinical Global Impression (CGI)
R = 0.79-0.99, P < .001 for all
Further findings
LDS with metreleptin, generalized lipodystrophy · 12 months
- LDS reduced from 46 to 26
- P < 0.001
LDS with metreleptin, partial lipodystrophy · 12 months
- LDS reduced from 65 to 61
- P = 0.04
Original publication
Lipodystrophy Severity Score to Assess Disease Burden in Lipodystrophy. J Clin Endocrinol Metab. Published 19 February 2025. doi:10. 1210/ clinem/ dgaf103
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