Table 3.
Outcomes in Patients With FPLD With Pathogenic Variants in PPARG and LMNA Genes Before vs After 12 Months of Metreleptin
| PPARG Variant Group | LMNA Variant Group | |||||||
|---|---|---|---|---|---|---|---|---|
| Clinical Variables | Baseline (n = 7) | 12 Months (n = 7) | P Value | Baseline (n = 22) | 12 Months (n = 22) | P Value | P Value for Combined Group | P Value for Metreleptin × Genetic Interaction |
| Weight, kg | 64 ± 13 | 65 ± 14 | 0.5 | 70 ± 13 | 68 ± 13 | 0.006 | 0.036 | 0.06 |
| BMI, kg/m2 | 24.4 ± 2.2 | 25.2 ± 2.5 | 0.3 | 25.7 ± 3.4 | 24.9 ± 3.4 | 0.01 | 0.2 | 0.02 |
| Glycemic Parameters | ||||||||
|---|---|---|---|---|---|---|---|---|
| Fasting glucose, mg/dL | 177 ± 97 | 158 ± 81 | 0.9 | 180 ± 89 | 124 ± 49 | 0.0001 | 0.005 | 0.3 |
| Fasting insulin, µU/mLa | 70 [27, 144] | 76 [27, 105] | 1.0 | 15 [11, 42] | 18 [9, 56] | 0.6 | 0.7 | 0.9 |
| Fasting C-peptide, ng/mLa | 3.7 [0.9, 5.2] | 3.5 [1.6, 6.1] | 0.7 | 4.1 [3.2, 4.9] | 3.4 [2.2, 4.5] | 0.02 | 0.07 | 0.1 |
| Hemoglobin A1c, % | 9.2 ± 2.3 | 7.7 ± 2.4 | 0.02 | 7.8 ± 2.1 | 7.3 ± 1.7 | 0.01 | 0.0005 | 0.06 |
| Insulin dose, U/kg/da | 3.8 [2.7, 4.3] | 2.0 [1.6, 3.0] | 0.03 | 1.7 [1.3, 4.] | 1.2 [0.9, 2.3] | 0.02 | 0.0002 | 0.8 |
| Number of diabetic medications | 2.6 ± 0.8 | 2.6 ± 0.8 | 1.0 | 1.6 ± 0.9 | 1.4 ± 1.0 | 0.1 | 0.1 | 0.3 |
| Lipid Parameters | ||||||||
|---|---|---|---|---|---|---|---|---|
| Total cholesterol, mg/dLa | 321 [155, 415] | 202 [140, 209] | 0.08 | 205 [153, 243] | 175 [161, 218] | 0.1 | 0.01 | 0.01 |
| Triglycerides, mg/dLa | 1377 [278, 5577] | 680 [296, 783] | 0.2 | 332 [198, 562] | 293 [148, 406] | 0.001 | 0.002 | 0.1 |
| HDL-C, mg/dL | 27 ± 5 | 25 ± 5 | 1.0 | 34 ± 10 | 35 ± 10 | 0.3 | 0.3 | 0.3 |
| LDL-C, mg/dL | 60 ± 14 | 88 ± 58 | 0.4 | 98 ± 31 | 103 ± 35 | 0.7 | 0.5 | 0.9 |
| Number of lipid-lowering medications | 1.9 ± 0.7 | 1.6 ± 0.8 | 0.6 | 1.1 ± 0.8 | 1.1 ± 0.9 | 0.7 | 0.7 | 0.2 |
| Hepatic Parameters | ||||||||
|---|---|---|---|---|---|---|---|---|
| Alanine aminotransferase, U/La | 36 [20, 46] | 29 [23, 52] | 1.0 | 29 [23, 47] | 29 [19, 44] | 0.2 | 0.2 | 0.8 |
| Aspartate aminotransferase, U/La | 25 [18, 37] | 26 [17, 40] | 0.9 | 25 [19, 30] | 21 [18, 27] | 0.2 | 0.5 | 0.8 |
Data are presented as mean and ± SD. P < 0.05 is considered statistically significant.
Abbreviations: AUC, area under the curve; HDL-C, high-density lipoprotein cholesterol; LDL-C, low-density lipoprotein cholesterol.
Represents data as median [25th percentile, 75th percentile].