Table 4.
Improvement of disease attributes after treatment with metreleptin
| All patients N = 112 (93F, 19M) | GL patients N = 68 (51F, 17M) | PL patients N = 44 (42F, 2M) | ||||
|---|---|---|---|---|---|---|
| Patients with evidence of issue pre-MET, n (%)a | Patients with evidence of issue who improved post-MET, n (%) | Patients with evidence of issue pre-MET, n (%)a | Patients with evidence of issue who improved post-MET, n (%) | Patients with evidence of issue pre-MET, n (%)a | Patients with evidence of issue who improved post-MET, n (%) | |
| Disruption to female reproductive functionb | 45 (40) | 20 (44) | 21 (31) | 12 (57) | 24 (55) | 8 (33) |
| HbA1c (>6.5%)c | 83 (75) | 66 (80) | 52 (78) | 46 (88) | 31 (70) | 20 (65) |
| Heart | ||||||
| Heart abnormalityd | 50 (45) | - | 36 (53) | - | 14 (32) | - |
| Hypertensione,f | 61 (54) | 36 (59) | 35 (51) | 27 (77) | 26 (59) | 9 (35) |
| Hyperphagiag | 74 (79) | 73 (99) | 51 (82) | 51 (100) | 23 (72) | 22 (96) |
| Impaired physical appearance | 86 (77) | 52 (60) | 56 (82) | 38 (68) | 30 (68) | 14 (47) |
| Kidney | ||||||
| Kidney abnormalitye | 71 (63) | 19 (27) | 46 (68) | 16 (35) | 25 (57) | 3 (12) |
| Elevated 24-hour protein excretionde | 46 (41) | 27 (59) | 34 (50) | 23 (68) | 12 (27) | 4 (33) |
| Liver | ||||||
| Liver abnormalitye | 105 (94) | 38 (36) | 63 (93) | 32 (51) | 42 (95) | 6 (14) |
| Elevated ALTe | 57 (51) | 48 (84) | 45 (66) | 41 (91) | 12 (27) | 7 (58) |
| Elevated ASTe | 48 (43) | 35 (73) | 38 (56) | 30 (79) | 10 (23) | 5 (50) |
| Pancreatitis | 44 (39) | 43 (98) | 21 (31) | 20 (95) | 23 (52) | 23 (100) |
| Triglycerides (>200 mg/dL)h | 89 (81) | 79 (89) | 52 (79) | 47 (90) | 37 (84) | 32 (86) |
| Inability to work/attend school | 48 (43) | 36 (75) | 39 (57) | 31 (79) | 9 (20) | 5 (56) |
Abbreviations: ALT, alanine transaminase; AST, aspartate transaminase; F, female; GL, generalized lipodystrophy; HbA1c, glycated hemoglobin; M, male; MET, metreleptin; PL, partial lipodystrophy.
aPatients with evidence of lipodystrophy characteristics at baseline as noted in Table 3.
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. Prevalence of disruption to reproductive function prior to treatment with metreleptin among female patients of reproductive capacity is 80%. Of 56 female patients of reproductive capacity (GL: 27; PL: 29), 45 presented with evidence of disruption to reproductive function prior to treatment with metreleptin.
cPercentages reported are of the patients with available baseline HbA1c values (GL: 67; PL: 44).
dBaseline data only available as the clinical resolution of heart abnormalities could not be determined with available data post-metreleptin.
eYear 1 post-metreleptin laboratory values are defined as the average of the specific laboratory measurements captured from 0.5 years post-metreleptin (inclusive) to 1.5 years post-metreleptin (exclusive).
fClinical improvement was defined as a 1-stage improvement in baseline hypertension categorization (as defined in Table 2) at year 1 post-metreleptin initiation (eg, moved from Stage 2 hypertension to Stage 1 hypertension, or prehypertensive to normal).
gData on hyperphagia were collected for first 94 patients (GL: 62, PL: 32).
hPercentages reported are of the patients with available baseline triglycerides values (GL: 66; PL: 44).