Table 3.

Outcomes in the control and metreleptin groups at 0 and 12 months

	Metreleptin				Control
	Baseline (N = 9)	12 months (N = 9)	Delta (N = 9)	P for 0-12 month difference	Baseline (N = 7)	12 months (N = 6)	Delta (N = 6)	P for 0-12 month difference	P for baseline between groups	P for deltas; Tx across all time points
Age (years)	13.8 ± 5.0	14.8 ± 5.0		<0.0001	11.4 ± 5.3	12.8 ± 5.7		<0.0001	0.67	-
A1c (%)	10.7 ± 1.5	9.3 ± 1.7	-1.4 ± 1.1	0.006	8.8 ± 2.2	9.4 ± 2.2	0.2 ± 0.7	0.43	0.03	0.007
Fasting glucose (mg/dL)	168 ± 72	125 ± 75	-43 ± 54	0.04	134 ± 72	132 ± 62	-14 ± 70	0.64	0.23	0.19
24-hour urine glucose excretion (g/24 h)	146.5 ± 82.6	86.9 ± 42.5b	-62.1 ± 71.2d	0.12	55.6 ± 38.9	72.4 ± 43.2e	3.7 ± 38.2e	0.86	0.04	0.93
OGTT
Glucose AUC (mg/dL 190 min)	62 536 ± 13 231	52 163 ± 19 242	-10 373 ± 8036	0.005	46 397 ± 19 231c	52 865 ± 24 911f	673 ± 4413f	0.82	0.08	0.07
Insulin AUC (mcU/mL 190 min)	90 665 [42 488, 134 420]	90 383 [4 5931, 145 903]	-568 [-10 424,28 473]	0.99	81 070 [43 226, 286 888]d	183 480 [85 585, 309 775]f	-14 625 [-163 790, 102 410] f	0.86	0.85	0.70
C-peptide AUC (ng/mL 190 min)	1879 ± 1512	1847 ± 1457	-32 ± 850	0.91	1862 ± 81f	3114 ± 1036g	1283 ± 948g	0.31	0.98	0.20
Fasting insulin (U/mL)	321 [269,531]	232 [161,742]	-17 [-158,15]	0.25	289 [214,1000]	360 [229,699]d	-63 [-405,-22]d	0.15	0.76	0.42
HOMA-IR	147 [91, 219]	82 [33, 229]	-43 [-73,34]	0.08	108 [32,506]	153 [69,186]d	-26 [-382,9]d	0.27	0.30	0.68
Weight SDS	-2.8 ± 1.8	-3.8 ± 1.9	-1.1 ± 0.5	0.0001	-2.5 ± 1.4	-2.7 ± 1.2	0.2 ± 1.0	0.67	0.30	0.03
Height SDS	-2.9 ± 1.5d	-3.0 ± 1.8d	-0.08 ± 0.4d	0.70	-2.5 ± 1.5c	-3.0 ± 1.2d	-0.1 ± 0.2d	0.23	0.13	0.65
BMI SDS	-1.0 ± 1.5	-2.3 ± 1.6	-1.3 ± 0.6	0.0001	-1.2 ± 1.2	-1.0 ± 0.7	0.4 ± 1.2	0.48	0.77	0.01
IGF-1 SDS	-3.6 ± 0.5	-3.4 ± 0.7a	0.2 ± 0.5a	0.43	-3.3 ± 0.8c	-3.6 ± 1.0e	-0.4 ± 0.3f	0.18	0.38	0.16
Growth hormone (ng/mL)	2.28 ± 2.0b	7.29 ± 7.56d	7.59 ± 6.56e	0.10	2.82 ± 2.72 c	1.07 ± 0.87e	-2.60 ± 3.55e	0.24	0.79	0.04
24-hour urine protein excretion (mg/24 h)	194.2 ± 158.2	138 ± 107b	-107.1 ± 193.9b	0.35	204.0 ± 203.9e	151.3 ± 21.6g	-162.3h	N/A	0.49	0.21
24-hour urine albumin excretion (mg/24 h)	26.1 [15.6,54.7]	35.7 [30.4,59.5] b	9.1 [-25.2,16.9] b	0.80	17.5 [8.2,69.2]c	31.1 [26.6,257.4] e	9.8 [-2.0,11.2] f	0.26	0.42	0.69
eGFR (mL/min/1.73 m 2 )	253 [177,289]	218 [169,339]	-11 [-55,11]	0.49	215 [161,254]c	152 [124,229]	-16 [-91,45] d	0.55	0.34	0.08

Glycemia, anthropometrics and kidney function for the metreleptin-treated group and control group at 0 and 12 months. P for deltas represents the linear mixed effects models to determine a difference in change from baseline between the control and metreleptin groups across all time points. Data are presented as mean ± SD or median [IQR: 25^th, 75^th]. Height SDS only includes patients who had achieved ≤ 90% of adult height, based on review of growth charts.

^aN = 8;

^bN = 7;

^cN = 6;

^dN = 5;

^eN = 4;

^fN = 3;

^gN = 2;

^hN = 1.