Winterer 1985.
| Methods | RCT. Cross‐over design. Location: single tertiary centre in Brazil. Duration: 4 to 6 weeks of each dose schedule. |
|
| Participants | 8 participants randomised. Age, range: 21OH deficiency, 9 to 20 years; 11OH deficiency 6 to 12 years. Gender split: 7 males, 1 female. Diagnosis: 6 participants with 21‐hydroxylase deficiency, 2 participants with 11‐hydroxylase deficiency. |
|
| Interventions | HC (+2 participants were on FC). HC split across different timings (morning, noon, evening) and combined where necessary with placebo (it was not stated was the placebo was). Total dose per day was 12.5 mg/m². Group 1: morning dose: full daily dose of HC; noon dose: placebo; evening dose: placebo. Group 2: morning dose: 2/3 daily dose HC; noon dose: placebo; evening dose: 1/3 daily dose HC. Group 3: morning dose: 1/3 daily dose HC: noon dose: 1/3 daily dose HC; evening dose: 1/3 daily dose HC Group 4: morning dose: 1/3 daily dose HC; noon dose: placebo; evening dose: 2/3 daily dose HC. Group 5: morning dose: placebo; noon dose: placebo; evening dose: full daily dose of HC. |
|
| Outcomes | Plasma 17 OHP, cortisol or cortisol and 11‐deoxycortisol‐increase urine 17‐ketosteroids, 17‐hydroxysteroids, pregnanetriol. | |
| Notes | Outcomes measured but not presented in the review: cortisol or cortisol and 11‐deoxycortisol increase, urine 17‐ketosteroids, 17‐hydroxysteroids and pregnanetriol. | |
| Risk of bias | ||
| Bias | Authors' judgement | Support for judgement |
| Random sequence generation (selection bias) | Unclear risk | Treatment randomly assigned, but paper does not state how sequence was generated. |
| Allocation concealment (selection bias) | Unclear risk | No mention of allocation of concealment. |
| Blinding of participants and personnel (performance bias) All outcomes | Low risk | Reported the trial was double blinded, but it was not clear who was blinded. |
| Blinding of outcome assessment (detection bias) All outcomes | Low risk | Reported the trial was double blinded, but it was not clear who was blinded. |
| Incomplete outcome data (attrition bias) All outcomes | High risk | Attrition rate was not reported |
| Selective reporting (reporting bias) | High risk | We did not have any access to the original trial protocols to definitely confirm whether this occurred and we attempted to contact the investigators but have not received a response. |
| Other bias | Unclear risk | We did not have any access to the original trial protocols to definitely confirm this but there was no washout period described in the paper for this cross‐over trial. |
17 OHP: 17‐hydroxyprogesterone ACTH: adrenocorticotrophic hormone BSA: body surface area CAH: congenital adrenal hyperplasia FC: fludrocortisone GH: growth hormone HC: hydroxycortisone IV: intravenous OH: hydroxylase PD: prednisolone RCT: randomised controlled trial SNP: single nucleotide polymorphism