Table 3.
Patient demographic and clinical characteristics pre- and postmatching (GL cohort)
| Treated (n = 62) | Pre-match Metreleptin-naïvea (n = 81) | Matched Metreleptin-naïve (n = 62) | |
|---|---|---|---|
| Age at first symptoms in years, mean (SD) | 9.0 (7.3) | 9.2 (11.9) | 9.8 (12.5) |
| Age at start of treatment or index observation date in years, mean (SD) | 17.7 (11.7) | 14.4* (4.9) | 16.9 (14.3) |
| Male, % | 22.6 | 40.7* | 32.3 |
| GL subtype,b % | |||
| AGL | 21.0 | 8.6 | 6.5* |
| CGL | 71.0 | 88.9* | 91.9** |
| Generalized progeroid lipodystrophy | 8.1 | 2.5 | 1.6 |
| Clinical characteristics at start of treatment or index observation date | |||
| Elevated HbA1c (≥6.5%), % | 79.0 | 34.6** | 48.4** |
| Triglyceride levels in mg/dL,b mean (SD) | 1354 (2260) | 363** (462) | 473** (687) |
| Experienced ≥1 episode of pancreatitis,b % | 33.9 | 2.47** | 6.5** |
| Number of organs among heart, liver and kidneys with observed abnormalities, mean (SD) | 2.177 (0.859) | 0.975** (0.987) | 1.516** (0.971) |
| Heart, % | 56.5 | 16** | 24.2** |
| Liver, % | 90.3 | 55.6** | 77.4 |
| Kidneys, % | 71.0 | 25.9** | 50.0* |
| Patients with record of triglyceride levels, n | 61 | 46c | 46c |
| Patients with record of HbA1c levels, n | 62 | 36c | 42c |
* P < 0.05.
** P < 0.01 compared to metreleptin-treated cohort.
Abbreviations: AGL, acquired generalized lipodystrophy; CGL, congenital generalized lipodystrophy; GL, generalized lipodystrophy; HbA1c, hemoglobin A1c; NA, not applicable; SD, standard deviation.
a Index observation date for the pre-match metreleptin-naïve cohort was defined as the time at which metreleptin-naïve patients achieved the mean age at the start of treatment of the treated sample (17.7 years) or the date of their last available observation, whichever comes first.
b GL subtype, triglyceride levels and pancreatitis were not used as matching parameters for the metreleptin-naïve cohort.
c Counts only include patients who have lab measurements taken on or after their index observation date.