BACKGROUND: Chronic liver insufficiency in children is frequently associated with growth retardation. Growth resumes after successful orthotopic liver transplantation in the majority of children with previous chronic liver failure. However, a subgroup of children demonstrates stunted growth even after orthotopic liver transplantation. The current study was conducted to determine whether administration of recombinant human growth hormone might benefit these patients. METHODS: Ten children were identified who met the criteria of growth failure despite normal transplant function in a cohort of 60 transplantation patients: height standard deviation score (hSDS) for chronological age less than -2, and growth velocity SDS (gvSDS) for chronological age equaling 0. Seven of these patients were treated with subcutaneous injections of recombinant human growth hormone at 4.0 U/m2 body surface area per day for at least 1 year. Two patients in this group showed insufficient growth hormone response to stimulation (arginine, clonidine) before therapy. Treatment was begun after a median time of 4.6 years after liver transplantation (2.55-8.4 years). Five children were treated with cyclosporin A and prednisolone and two with tacrolimus and prednisolone for maintenance immunosuppression. RESULTS: Within 3 months of treatment, median serum levels of insulin-like growth factor (IGF)-I increased from 0.05 to 0.71 (P < 0.02). Within 1 year, median hSDS improved from -2.7 (range, -5.6 to -2.3) to -2.1 (-4.5 to -1.4; P < 0.03). Median annual growth rate increased from 3.9 cm/year (range, 3-6) in the year before treatment to 8.2 cm/year (range, 6.1-10.4; P < 0.02) after the beginning of recombinant human growth hormone therapy. All patients tolerated treatment without side effects. During the cumulative treatment time of 14 years no rejection episode was observed. CONCLUSIONS: Short-statured prepubertal liver transplant recipients who do not show sufficient compensatory growth after transplantation benefit from treatment with recombinant human growth hormone. Treatment with the hormone was safe without any side effects.
BACKGROUND: Chronic liver insufficiency in children is frequently associated with growth retardation. Growth resumes after successful orthotopic liver transplantation in the majority of children with previous chronic liver failure. However, a subgroup of children demonstrates stunted growth even after orthotopic liver transplantation. The current study was conducted to determine whether administration of recombinant humangrowth hormone might benefit these patients. METHODS: Ten children were identified who met the criteria of growth failure despite normal transplant function in a cohort of 60 transplantation patients: height standard deviation score (hSDS) for chronological age less than -2, and growth velocity SDS (gvSDS) for chronological age equaling 0. Seven of these patients were treated with subcutaneous injections of recombinant humangrowth hormone at 4.0 U/m2 body surface area per day for at least 1 year. Two patients in this group showed insufficientgrowth hormone response to stimulation (arginine, clonidine) before therapy. Treatment was begun after a median time of 4.6 years after liver transplantation (2.55-8.4 years). Five children were treated with cyclosporin A and prednisolone and two with tacrolimus and prednisolone for maintenance immunosuppression. RESULTS: Within 3 months of treatment, median serum levels of insulin-like growth factor (IGF)-I increased from 0.05 to 0.71 (P < 0.02). Within 1 year, median hSDS improved from -2.7 (range, -5.6 to -2.3) to -2.1 (-4.5 to -1.4; P < 0.03). Median annual growth rate increased from 3.9 cm/year (range, 3-6) in the year before treatment to 8.2 cm/year (range, 6.1-10.4; P < 0.02) after the beginning of recombinant humangrowth hormone therapy. All patients tolerated treatment without side effects. During the cumulative treatment time of 14 years no rejection episode was observed. CONCLUSIONS: Short-statured prepubertal liver transplant recipients who do not show sufficient compensatory growth after transplantation benefit from treatment with recombinant humangrowth hormone. Treatment with the hormone was safe without any side effects.