Table 5.

Prevalence of disease attributes

	Rescaled utility decrement	GL Patients N = 68				PL Patients N = 44
		Prevalence at baselinea (%)	Post-MET prevalencea (%)	Change in prevalence (%)	Change in QALYs	Prevalence at baselinea (%)	Post-MET prevalencea (%)	Change in prevalence (%)	Change in QALYs
Disruption to female reproductive functionb	−0.026	31	18	−13	0.005	55	45	−10	0.003
HbA1c
>6.5% and ≤8%	−0.038	12	15	+3	−0.001	23	30	+7	−0.003
>8%	−0.082	66	20	−46	0.038	45	28	−17	0.015
Heart abnormality c	−0.096	53	57	+4	−0.004	32	39	+7	−0.007
Hyperphagiad	−0.071	82	11	−71	0.050	72	9	−63	0.044
Impaired physical appearance	−0.056	82	29	−53	0.030	68	41	−27	0.015
Kidney abnormality	−0.066	68	47	−21	0.014	57	61	+4	−0.003
Liver abnormality	−0.082	93	47	−46	0.038	95	84	−11	0.009
Pancreatitise	−0.060	31	2	−29	0.018	52	2	−50	0.030
Progression of organ damage
Slow	0.030	21	24	+3	0.001	82	26	−56	−0.017
Fast	−0.080	75	26	−49	0.039	18	11	−7	0.006
Triglycerides
>200 mg/dL and ≤500 mg/dL	−0.012	35	26	−9	0.001	45	45	0	<0.001
>500 mg/dL	−0.032	44	15	−29	0.009	39	28	−11	0.004
Inability to work/attend school	−0.167	57	12	−45	0.076	20	9	−11	0.019

Abbreviations: GL, generalized lipodystrophy; HbA1c, glycated hemoglobin; MET, metreleptin; PL, partial lipodystrophy; QALYs, quality-adjusted life years.

^aWhen calculating prevalence, it was assumed that a disease attribute is not present when data are not recorded.

^bOnly female patients of reproductive capacity (GL: 27; PL: 29) were eligible for impairment; all other patients were considered unimpaired. Reproductive capacity was defined as female patients who were postpubescent and premenopausal who had not undergone surgical removal of reproductive organs at the time of starting metreleptin.

^cPrevalence of heart abnormality can only increase as clinical resolution of abnormalities could not be determined with available data.

^dData on hyperphagia were collected for first 94 patients (GL: 62, PL: 32).

^eOne patient with GL and one patient with PL each experienced a single episode of pancreatitis while on treatment with metreleptin.