Lipodystrophy Severity Score to Assess Disease Burden in Lipodystrophy.
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Brown Rebecca J, Akinci Baris, Yosef Matheos, Phillips Helen, Khalatbari Shokoufeh, Sorkina Ekaterina, Santini Ferruccio, Vigouroux Corinne, Brush Maiah, Meral Rasimcan, Ceccarini Giovanni, Zeybel Mujdat, Prodam Flavia, von Schnurbein Julia, Sorice Gian P, Guler Merve C, Patni Nivedita, Tanrikulu Seher, Alyaarubi Saif, Ozgen Basak S, Foss-Freitas Maria C, Ozisik Secil, Segrestin Benerice, Ozcan Busra, Adiyaman Suleyman C, Musolino Gianluca, Sekizkardes Hilal, Musso Carla, Lebenthal Yael, Ozen Samim, Simha Vinaya, Simsir Ilgin Y, Stears Anna, Scherer Thomas, Gambineri Alessandra, Lima Josivan G, Semple Robert, Wabitsch Martin, Araujo-Vilar David, Hegele Robert A, Oral Elif A
Аннотация
OBJECTIVE: Our goal was to develop a lipodystrophy severity score (LDS) to holistically capture the diverse manifestations of lipodystrophy into a numerical score to aid in prediction of clinical outcomes and/or treatment impact.
DESIGN: An 8-domain LDS was developed by 8 disease experts in consultation with patient organizations. The LDS was rated for feasibility and content validity by 28 additional clinicians and 9 patient representatives. LDS was compared to the Clinical Global Impression (CGI) of severity for 20 putative patient profiles, each at 2 different time points, and by comparing change in LDS to global impression of change. For external validation, LDS was calculated in 2 cohorts of patients with lipodystrophy treated with metreleptin.
RESULTS: LDS domains include Diabetes/Insulin Resistance, Microvascular Complications of Diabetes, Lipids, Cardiovascular, Liver, Kidney, Reproductive, and Other. Each domain is assessed by 1 or more questions assessing both lifetime and recent complications of lipodystrophy. The LDS had high content validity and feasibility and high reliability by intraclass correlation coefficients (>0.95). Global and domain-specific LDS were strongly correlated with CGI, as were changes in scores across visits (R = 0.79-0.99, P < .001 for all). In generalized lipodystrophy, metreleptin significantly reduced LDS (from 46 to 26 at 12 months, P < .001). The reductions were smaller in partial lipodystrophy (from 65 to 61 at 12 months, P = .04).
CONCLUSION: The LDS can reflect the severity of diverse manifestations of lipodystrophy and monitor changes following interventions.