Table 3.
Benefits of the metreleptin therapy.
| Mutation—case | Initial metreleptin dose | Adjustments/reason? | Duration | Outcomes | Ref. |
|---|---|---|---|---|---|
| ΔG133—Ob1 | 0.028 mg/kg LBW | Yes. Changes in the body weight |
12 months | (1) Loss of 16.4 kg of fat mass; (2) sustained reduction of energy consumption; (3) increase of physical activity; and (4) increase of serum gonadotropin. | (23) |
| ΔG133—Ob2 | 0.017 mg/kg LBW | Yes. Changes in the body weight/metreleptin neutralization by antibodies |
36 months | (1) Loss of 10.7 kg of fat mass; (2) reduction of energy consumption; (3) decrease of plasma insulin level, serum cholesterol, triglycerides, and LDL and increase of serum HDL cholesterol; and (4) normalization of the immunophenotype | (2) |
| ΔG133—Ob3 | 0.014 mg/kg LBW | Yes. Changes in the body weight/metreleptin neutralization by antibodies |
6 months | (1) Loss of 2.2 kg of fat mass; (2) Reduction of energy consumption; (3) decrease of plasma insulin level, serum cholesterol, triglycerides, and LDL and increase of serum HDL cholesterol; and (4) normalization of the immunophenotype. | (2) |
| ΔG133—Ob4 | 0.019 mg/kg LBW | Yes. Changes in the body weight |
48 months | (1) Loss of 15.9 kg of fat mass; (2) normalization of plasma triglycerides, insulin, and TSH and increase of HDL cholesterol; (3) increase of white blood cells count, with amelioration of perineal dermatitis and asthma. | (3) |
| p.Arg105Trp—male | 0.01–0.04 mg/kg | Yes. Changes in the body weight |
18 months | (1) Loss of 52.1 kg of fat mass; (2) resolution of the hypogonadism; and (3) neuroplasticity. | (24–26) |
| p.Arg105Trp—female | 0.01–0.04 mg/kg | Yes. Changes in the body weight |
18 months | (1) Loss of 37.6 kg of fat mass; (2) resolution amenorrhea; and (3) neuroplasticity. | (24–26) |
| p.Arg105Trp—eldest female | 0.01–0.04 mg/kg | Yes. Changes in the body weight |
18 months | (1) Loss of 39.1 kg of fat mass. (2) resolution of the type 2 diabetes mellitus and amenorrhea; and (3) neuroplasticity. | (24–26) |
| Leptin-deficient—male | N.A. | N.A. | 7 days | (1) Decrease of hyperphagia and (2) increase of satiety. | (27) |
| Leptin-deficient—female | N.A. | N.A. | 7 days | (1) Decrease of hyperphagia and (2) increase of satiety. | (27) |
| p.Arg105Trp—7-year-old boy | 1.36 mg/day | Yes. Changes in the body weight |
28 months | (1) Weight loss; (2) amelioration of hypertension, dyslipidemia and hyperinsulinemia; and (3) increase of patient’s general cognitive ability and neuropsychological function. | (12) |
| p.Asn103Lys | 0.03 mg/kg LBW | No | 2 months | (1) Amelioration of hyperphagia and satiety and (2) weight loss of 6.2 kg. | (14) |
| p.Asn103Lys | 0.03 mg/kg LBW | No | 2 months | (1) Amelioration of hyperphagia and satiety and (2) weight loss of 3.5 kg. | (14) |
LWB, lean body mass.