Table 2

Characteristics of the non-randomised studies that evaluated the use of growth hormone in patients with Prader-Willi syndrome

AuthorTypeAgeN rhGHDose of rhGHPeriod (years)CountryOutcome
Donze SH et al 15 BEFORE AND AFTER6 months-3 years291 mg/m2/day3NetherlandsPsychomotor development (BSID-II - mental and motor development), head circumference
Dykens EM et al 13 COHORT
(three separated studies)
 4–21 years96;
127;
168
1 mg/m2/day6, 2USABMI-SDS. Cognition: KBIT-2 (verbal IQ, non-verbal IQ, composite IQ) VABS-II (communication and daily live skills, socialisation, adaptive composite). Hyperphagia Questionnaire; Repetitive Behaviour Scale
Bakker NE et al
10
BEFORE AND AFTER6±4.3 years15660.23 mg/kg/week3KIGS database -worldwideZ-stature, BMI-SDS, adverse effects
Bakker NE et al 18 BEFORE AND AFTER6–14 years761 mg/m2/day11NetherlandsQuality of life (DUX25 and DUXPW questionnaires)
Scheermeyer E et al 47 BEFORE AND AFTER13–24 months 2–12 years721 mg/m2/day3AustraliaZ-stature, BMI-SDS and adverse effects
Lo ST 201534 COHORT3.5–14 years241 mg/m2/day2;8NetherlandsBehaviour. Developmental Behaviour Checklist of Children with intellectual disability (DBC) and Children's Social Behaviour Questionnaire (CSBQ)
Lo ST et al 36 COHORT3.5–14 years531 mg/m2/day2;7NetherlandsVABS-II, Cognitive function: Bayley Scales of Infants II- NL (BSID-II), Wechsler Preschool and Primary Scale of Intelligence-Revised Dutch version
Bakker NE et al 12 BEFORE AND AFTER 3–7 years (mean: 5.49)601 mg/m2/day8NetherlandsZ-stature, BMI-SDS, lean body mass-SDS, % fat mass-SDS, head circumference
Meinhardt U et al 42 COHORT 0.4–12.2 years (mean:3.8)410.03 mg/kg/day1; 6Switzerland, Denmark, GermanyZ-stature, body composition and adverse effects
Siemensma EPC et al 14 COHORT3.5–14 years291 mg/m2/day2; 4NetherlandsWISC-R (vocabulary, similarities, block design and picture arrangement) and Wechsler Preschool and Primary Scale of Intelligence-Revised Dutch version - less 7 years old
Colmenares A et al 48 COHORT1–15 years (mean: 6±3.7)360.03 mg/kg/day3FranceZ-stature, BMI-SDS and body composition- lean mass (kg)/ %fat mass
Sipilä I et al 39 BEFORE AND AFTER 2.0–10.3 years (mean: 6.6)20 0.033 mg⁄kg⁄ day1; 10FinlandZ-stature, BMI-SDS, body composition (%fat), and quality of life (HRQOL)
Carrel AL et al 8 COHORT4–32 months/. 6–9 years461 mg/m2/day6USAZ-stature, body composition and muscular strength
de Lind van Wijngaarden RFA et al 9 BEFORE AND AFTER5.9±3.2 years551 mg/m2/day4NetherlandsZ-stature, BMI-SDS, body composition (%fat-SDS and lean body mass- SDS), head circumference
Nyunt O et al 49 COHORT<3 years and >3 years540,6 mg/m2/day4AustraliaZ-stature and BMI-SDS
Lindgren AC et al 50 BEFORE AND AFTER 4.9–11.3 years (mean 6.9)220.03 mg/kg/day10SwedenZ-stature, BMI-SDS and body composition
Lin H-Y et al 51 BEFORE AND AFTER1.3–13.5 years460.01 U/kg/day5TaiwanZ-stature, BMI-SDS and adverse effects
Angulo MA et al 43 COHORT8.3±2.7 years210.25 mg/kg/week7.9USAZ-stature and BMI-SDS
Carrel AL et al 40 BEFORE AND AFTER12 years481 mg/m2/day4USAZ-stature, body composition, muscular strength, adverse effects
Tauber M et al 52 BEFORE AND AFTER8.4±3.2 years140.5 U/kg/week3.6FranceZ- stature SDS, BMI-SDS, growth velocity
  • BMI, body mass index; rhGH, recombinant human GH; SDS, SD scores.