Table 1

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

Author/yearAgeN rhGH/controlDose of rhGHPeriod (years)CountryOutcome
Bakker NE et al 45 6 months to 14 years12/161 mg/m2/day2NetherlandsZ-stature and body composition (%fat mass and LBM-SDS).
Bakker NE et al 18 6–14 years11/81 mg/m2/day2NetherlandsQuality of life (Dutch generic questionnaire-DUX25 and PWS-Dutch specific questionnaire-DUXPW questionnaires)
de Lind van Wijngaarden RFA et al 41 4, 9±3.0 years (<3 years and >3 years)29/211 mg/m2/day1;2NetherlandsZ-stature, BMI-SDS, body composition (%fat)
Böhm B et al 33 3–11 years190033 mg/kg/day2SwedenBehaviour (specific test for the study) and cognition (Raven's Standard Progressive Matrices test, Arthur's adaptation of Leiter's Performance Scale; verbal comprehension test (SPIQ test), Terman’s Scale of Intelligence, Bender Gestalt test, Harris Draw-a-Man)
Lo ST et al 34 3, 5–14 years24/181 mg/m2/day1;2NetherlandsBehaviour (Developmental Behaviour Checklist of Children with intellectual disability and Children Social Behaviour Questionnaire)
Lo ST et al 36 3, 5–14 years41/341 mg/m2/day1;2NetherlandsVABS-II, cognitive function: Bayley Scales of Infants II- NL (BSID II), Weschsler Preschool and Primary Scale of Inteligence-Revised Dutch version
Reus L et al 37 12.9±7.1 months10/121 mg/m2/day2NetherlandsMuscular thickness with ultrasound, muscle strength (Infant Muscle Strength metre) and motor performance (Gross Motor Function Measure)
Reus L et al 35 12.9±7.1 months10/121 mg/m2/day2NetherlandsCognition (BSID-II) and motor function (Gross Motor Function)
Siemensma EPC et al 14 3, 5–14 (mean 7,4 years)29/211 mg/m2/day2NetherlandsWISC-R (vocabulary, similarities, block design and picture arrangement) and Wechsler Preschool and Primary Scale of Intelligence-Revised Dutch version
Festen DAM et al 7 3–14 years25/251 mg/m2/day2NetherlandsZ-stature, body composition (%fat, LBM-SDS), head circumference (Z)
Festen DAM et al 16 Grupo I: 2–3 years C: 1–5 years20/221 mg/m2/day1NetherlandsMotor and mental development (BSID-II).
Myers SE et al 44 4–37 months12/111 mg/m2/day1USAZ-stature, head circumference, body composition (%fat, LBM-kg), language/cognition (age of first spoken word and Capute scales of infant’s language), motor development (age of walking and Toddler and Infant Motor Evaluation)
Carrel AL et al 38 4–37 months15/141 mg/m2/day1USAGrowth velocity, body composition and motor development (Toddler Infant Motor Evaluation)
Whitman BY et al 17 4–16 years35/191 mg/m2/day2USABehaviour (Offord Survey Diagnostic Instrument and Family Inventory of Life Events)
Carrel AL et al 5 4–16 years35/191 mg/m2/day1USABody composition (%fat, LBM-kg), growth velocity, BMI-kg, muscular strength and agility (agility run, broad jump, sit-ups in 20 secs and upper extremity strength)
Lindgren AC et al 6 3–12 years15/120.1 IU/kg/day1Sweden
Denmark
Z-stature, BMI-SDS, body composition (%fat, LBM-kg), growth velocity.
  • BMI, body mass index; LBM, lean body mass; rhGH, recombinant human GH; SDS, SD scores.