Table 1.
Authors | Study design | Sample size (only boys) | Age of patientsa | Type of intervention | Follow-up | Duration of treatment | Outcomes |
---|---|---|---|---|---|---|---|
Laron & Sarel 1970 (12) | Observational | 6 | 1–11 | / | / | / | Penile and testicular size |
Laron et al. 1980 (14) | Observational | 7 | 0–11 (at enrollment) | / | 15.8 years (age at least examination: M: 15–25) | / | Age, testicular volume, penile length and circumference, breast development, bone age, state of development |
Guevara-Aguirre et al. 1993 (15) | Observational | 27 | NR | / | / | / | Auxological parameters, GH, IGF1, IGF2, IGFBP3 |
Pertzelan et al. 1993 (16) | Observational | 6 | NR | / | / | / | Puberty onset and duration |
Laron et al. 1993 (17) | Observational | 10 | NR | / | ~ 20 years (from infancy to puberty) | / | Pubertal development, growth curves |
Savage et al. 1993 (18) | Observational | 12 | 3.6–22 | / | / | / | Height, pubertal development, bone age, GH, IGF1, IGF2, IGF1BP1, IGFBP2, IGFBP3, GHBP |
Castilla-Cortazar et al. 2017 (19) | Case report | 1 | 14 and 10 months | Recombinant IGF1 (0.04–0.12 mg/kg twice daily) | 7 months | NR | Case description, including pubertal development, height, IGF1, glucose, shoe size increment |
Laron & Klinger 1998 (20) | Longitudinal interventional | 7 (4 younger than 5 years, 2 of pubertal age, 1 adult) | 0.5–28 | Long-term treatment with recombinant IGF1 (150–120 µg/kg daily) | NR | Depend on the onset of side effects or the auxological parameters | Serum gonadotropin, androgens, insulin, IGF1, testicular volume, penile size |
Ben-Amitai & Laron 2011 (21) | Longitudinal interventional | 10 (8 untreated, 2 treated) | 0–5 (at enrollment) | Long-term treatment with recombinant IGF1 (70–200 µg/Kg daily) | 28 years 8 years (age at least examination: M: 20–41) | M: 25.5–33.5 years | Serum gonadotropin, androgens, insulin, IGF1, acne |
Phanse-Gupte et al. 2014 (22) | Observational | 7 | 7.8 ± 4.1b | / | / | / | Presence of high-pitched voice, sparse hair, micropenis, blue sclera, hypoglycemia, pubertal development, GH, IGF1, IGF2, IGFBP1, IGFBP3, GHBP |
Burren et al. 2001 (23) | Observational | 24 | 8.6 ± 4.6b | / | / | / | Presence of high-pitched voice, sparse hair, micropenis, blue sclera, hypoglycemia, pubertal development, GH, IGF1, IGF2, IGFBP1, IGFBP3, GHBP |
Ioimo et al. 2018 (24) | Case report | 1 | 2.5 years | Recombinant IGF1 0.20 mg/kg/day | NR | NR | Case description, including auxological parameters, micropenis, GH, GH peak, IGF1, IGF1 peak, IGFBP3 |
Guleria et al. 2014 (27) | Case report | 2 | 7 years and 7 months 5.5 years |
NR | NR | NR | Case description, including auxological parameters, micropenis |
Hopp et al. 1996 (28) | Case report | 1 | 11 years 9 months | NR | NR | NR | Case description, including auxological parameters, micropenis |
Krzisnik et al. 1994 (25) | Case report | 1 | 6 | IGF1 treatment | NR | NR | Case description, including auxological parameters, and puberty onset |
Rosenbloom et al. 1995 (26) | Case report | 2 | 3.7 1.9 |
/ | / | / | Case description, including auxological parameters, micropenis, GH, GHBP, IGF1, IGFBP3, LH, FSH, T |
De Lima Jorge et al. 2008 (29) | Case report | 1 | 10.6 | / | / | / | Case description, including auxological parameters, micropenis, GH, GH peak, IGF1, IGF1 peak, IGFBP3 |
aWith the exception of the studies by Phanse-Gupte et al. 2014 and Burren et al. 2001, only the age of the male patients has been reported. bThe study reports the age of all (male and female) the included patients. The age of the male ones is not available.
FSH, follicle-stimulating hormone; GH, growth hormone; GHBP, GH binding protein; IGF1, insulin-like growth factor 1; IGF2, insulin-like growth factor 2; IGFBP1, IGF binding protein 1; IGFBP2, IGF binding protein 2; IGFBP3, IGF binding protein 3; LH, luteinizing hormone; T, testosterone.