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The problem
Intervention: Growth hormone treatment at 1.4 mg/m² per day for at least one year
Who it was tested with: Six children (4 boys, 2 girls) with pycnodysostosis, all prepubertal, median age 10.4 years at treatment start.
Experience from people who deliver programmes: stories, case studies and lessons learned. Valuable for understanding how and why, and for spotting what to test next, but not a measure of effect.
"Six subjects (4 boys, 2 girls) presented with pycnodysostosis, treated with GH 1.4 mg/m2/day for ≥1 year...After 1 year of GH, median height gain was 7.6 cm...All children were prepubertal at start of GH." This is a descriptive case series following individual patients over time without a control group, representing practitioner-level evidence of treatment effects in a very rare condition.
Small sample size (n=6) limits generalizability. Three children still in treatment at time of writing, so final outcomes unknown. Different treatment durations and one child received additional GnRH analogue treatment, making it difficult to isolate the effect of GH alone. No comparison group to determine if outcomes differ from untreated children.
Across countries and designs
1 study · 1 country
