Table 4:
Patient demographic and clinical characteristics pre- and postmatching (PL cohort)
| Treated (n = 41) | Pre-match Metreleptin-naïvea (n = 149) | Matched Metreleptin-naïve (n = 41) | |
|---|---|---|---|
| Age at first symptoms in years, mean (SD) | 20.7 (12.9) | 24.7 (16.1) | 22.7 (12.5) |
| Age at start of treatment or index observation date in years, mean (SD) | 35.2 (15.2) | 30.7 (7.2) | 38.0 (12.6) |
| Male, % | 4.9 | 24.8** | 4.9 |
| PL subtype,b % | |||
| APL | 7.3 | 18.8 | 7.3 |
| FPLD | 92.7 | 81.2 | 92.7 |
| Clinical characteristics at start of treatment or index observation date | |||
| Elevated HbA1c (≥6.5%), % | 78.0 | 30.9** | 78.0 |
| Triglyceride levels in mg/dL,b mean (SD) | 1230 (2090) | 483* (599) | 503* (452) |
| Experienced ≥1 episode of pancreatitis,b % | 51.2 | 6.7** | 17.1** |
| Number of organs among heart, liver and kidneys with observed abnormalities, mean (SD) | 1.854 (0.654) | 0.497** (0.794) | 1.854 (0.654) |
| Heart, % | 31.7 | 6.0** | 36.6 |
| Liver, % | 95.1 | 28.9** | 92.7 |
| Kidneys, % | 58.5 | 14.8** | 56.1 |
| Patients with record of triglyceride levels, n | 41 | 73c | 36c |
| Patients with record of HbA1c levels, n | 41 | 82c | 35c |
* P < 0.05.
** P < 0.01 compared to metreleptin-treated cohort.
Abbreviations: APL, acquired partial lipodystrophy; FPLD, familial partial lipodystrophy; PL, partial lipodystrophy; HbA1c, hemoglobin A1c; NA, not applicable; SD, standard deviation.
a Index observation date for the prematch 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 (35.2 years) or the date of their last available observation, whichever comes first.
b PL 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.