Table 2.
Patient demographic and clinical characteristics pre- and postmatching (overall cohort)
| Treated (n = 103) | Pre-match Metreleptin-naïvea (n = 230) | Matched Metreleptin-naïve (n = 103) | |
|---|---|---|---|
| Age at first symptoms in years, mean (SD) | 13.8 (11.5) | 19.2 (16.5)** | 15.0 (14) |
| Age at start of treatment or index observation date in years, mean (SD) | 24.7 (15.7) | 21.2* (5.94) | 25.3 (17.1) |
| Male, % | 15.5 | 30.4** | 21.4 |
| Diagnosis of GL, % | 60.2 | 35.2** | 60.2 |
| GL/PL subtype,b % | |||
| AGL | 12.6 | 3.0** | 3.9* |
| CGLc | 42.7 | 31.3 | 55.3 |
| Generalized progeroid lipodystrophy | 4.9 | 0.9 | 1.0 |
| APL | 2.9 | 12.2* | 2.9 |
| FPLDd | 36.9 | 52.6* | 36.9 |
| Clinical characteristics at start of treatment or index observation date | |||
| Elevated HbA1c (≥6.5%), % | 78.6 | 24.3** | 60.2** |
| Triglyceride levels in mg/dL,b mean (SD) | 1304 (2180) | 472** (785) | 486** (592) |
| Experienced ≥1 episode of pancreatitis,b % | 40.8 | 3.91** | 10.7** |
| Number of organs among heart, liver and kidneys with observed abnormalities, mean (SD) | 2.049 (0.797) | 0.613** (0.893) | 1.650** (0.871) |
| Heart, % | 46.6 | 8.26** | 29.1** |
| Liver, % | 92.2 | 35.7** | 83.5 |
| Kidneys, % | 66.0 | 17.4** | 52.4* |
| Patients with record of triglyceride levels, n | 102 | 103e | 82e |
| Patients with record of HbA1c levels, n | 103 | 118e | 77e |
* P < 0.05.
** P < 0.01 compared with metreleptin-treated cohort.
Abbreviations: AGL, acquired generalized lipodystrophy; CGL, congenital generalized lipodystrophy; FPLD, familial partial 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 (24.7 years) or the date of their last available observation, whichever comes first.
b GL/PL subtypes, triglyceride levels and pancreatitis were not used as matching parameters for the metreleptin-naïve cohort.
c Patients with mutations in AGPAT2 were the most common (n = 26 in treated cohort; n = 22 in matched metreleptin-naïve cohort), followed by those with mutations in BSCL2 (n = 15 in treated cohort; n = 15 in matched metreleptin-naïve cohort). The treated cohort also had 2 patients with CGL who had other mutations and 1 patient with CGL where genetic testing data were either missing or a mutation could not be confirmed. The matched metreleptin-naïve cohort also had 2 patients with PTRF4 mutations, 2 patients with CGL who had other mutations, and 16 patients with CGL where genetic testing data were either missing or a mutation could not be confirmed.
d Patients with mutations in LMNA were the most common (n = 20 in treated cohort; n = 25 in matched metreleptin-naïve cohort), followed by those with mutations in PPARG (n = 8 in treated cohort; n = 3 in matched metreleptin-naïve cohort). The treated cohort also had 1 patient with FPLD who had a PCYT1A mutation and 9 patients with FPLD where genetic testing data were either missing or a mutation could not be confirmed. The matched metreleptin-naïve cohort also had 3 patients with Köbberling type FPLD; 5 patients with FPLD who had other mutations, and 2 patients with FPLD where genetic testing data were either missing or a mutation could not be confirmed.
e Counts only include patients who have lab measurements taken on or after their index observation date.