Patient Quality of Life and Benefits of Leptin Replacement Therapy (LRT) in Generalized and Partial Lipodystrophy (GL, PL)

Abstract

Effects of LRT on metabolic disease in GL and PL have been studied; however, little is known about consequences of GL and PL, or the effect of LRT, on quality of life (QoL). To characterize health utility consequences of GL and PL attributes on patients, a discrete choice experiment (following a tutorial on the disease) was conducted with 1,000 members of the general population. Multinomial logit regression was used to estimate utility decrements associated with each attribute using an approach similar to Bansback et al. (2012). Results were combined with data on prevalence of attributes before and after 1 year of LRT, derived from a retrospective chart review, to assess overall QoL consequences of GL, PL, and the impact of LRT in Quality-Adjusted Life Years (QALYs). Overall, QALY gains associated with LRT were estimated at 0.423 across all patients. Impaired work/school ability, hyperphagia and organ damage were the largest contributing attributes. A subgroup analysis for GL and PL found total QALY gains associated with LRT were 0.569 and 0.199, respectively. This study indicates that lipodystrophy is associated with a large QoL impairment and that the benefits of LRT may be substantial in these patients. Attribute utility decrements appear to be consistent with those reported in other research (e.g., for diabetes), yet the cumulative impact of attributes makes the burden of GL and PL high. Disclosure O.A. Ali: Consultant; Self; Aegerion Pharmaceuticals. K. Cook: Consultant; Self; Aegerion Pharmaceuticals. K.S. Shampanier: Consultant; Self; Aegerion Pharmaceuticals. E. Tuttle: Consultant; Self; Aegerion Pharmaceuticals. C. Gerrits: None. R. Brown: None.