Summary of Study and Population Characteristics

Manufacturer’s Pooled Clinical Trial Data (Safety Set)a
1 mg/kg per day (n = 200)3 mg/kg per day (n = 235)All (N = 435)Placebo (N = 55)Literature Data (N = 3766)CUP (N = 1661)
Number of study types included3831
 RCT1 pivotal RCT4
 Prospective2 open-label studies35Yes
Patients treated, n2002354355537661661
Age at initiation of treatment, moN = 1628
 Mean (SD)3.39 (1.04)3.57 (1.33)3.49 (1.21)3.41 (1.02)7.0 (n = 56)b5.6
 Range1.2–5.11.1–13.01.1–13.01.2–5.0−1.1c to 228 (n = 70)1 d–6.4 y
Sex, n (%)N = 3407N = 1628
 Male62 (31.0)59 (25.1)121 (27.8)17 (30.9)971 (28.5)401 (24.6)
 Female138 (69.0)176 (74.9)314 (72.2)38 (69.1)2436 (71.5)1227 (75.4)
IH characteristicsNon–high risk (studies 102, 201, 301) and high risk100VariousHigh riskd
  • RCT, randomized controlled trial.

  • a Manufacturer’s pooled clinical trial data are presented by dose of propranolol or placebo (whatever the assigned regimen) and for all patients treated with propranolol whatever the dose. Therefore, for those patients assigned to one of the two 3-mo propranolol regimens (followed by 3 mo taking placebo) data are presented within both the propranolol and placebo groups according to the period during which the event occurred. Patients in study 301 who received 2 mg/kg per day of propranolol were analyzed with the 3-mg/kg-per-day group for data described by dose and in the “all propranolol” column describing all patients treated with propranolol whatever the dose.

  • b Estimated mean age for articles presenting mean-age data (n = 56).

  • c Age corrected for prematurity.

  • d Seventy-two percent functional impairment, 40% severe ulceration, 16% at vital risk.