RESUMEN
OBJECTIVE: The objective was to assess the efficacy and safety of growth hormone (GH) treatment in severely growth retarded children with nephropathic cystinosis during conservative treatment and during renal replacement therapy. STUDY DESIGN: The design was an open-labeled prospective trial with a run-in period of 1 year. RESULTS: A total of 74 children with cystinosis (age 3.0 to 18 years) were treated with GH over a mean period of 3.1 years (range 1 to 10 years); 52 patients were receiving conservative treatment (mean age 7.1 years), 7 were receiving dialysis (12.5 years), and 15 had received a renal transplant (14.8 years). The mean standardized height (SD score) was -4.0 in the conservative treatment group, -4.4 in the dialysis group, and -4.9 in the renal transplant group. During the first treatment year, height velocity doubled in the conservative treatment group, increased by 80% in the dialysis group, and increased by 45% in renal transplant group. Within 3 years the height SD score increased by +1.6 (P <.001) in prepubertal patients receiving conservative treatment, and percentile parallel growth was maintained thereafter. These effects of GH were less expressed in peripubertal patients receiving renal replacement therapy. No major side effects were observed. CONCLUSION: Long-term GH treatment is safe and effective in young children with nephropathic cystinosis. GH treatment should be started early in the course of the disease if adequate nutrition and cysteamine treatment do not prevent growth retardation.
Asunto(s)
Cistinosis/terapia , Trastornos del Crecimiento/terapia , Hormona del Crecimiento/uso terapéutico , Enfermedades Renales/terapia , Adolescente , Estatura/efectos de los fármacos , Niño , Preescolar , Femenino , Humanos , Trasplante de Riñón , Cuidados a Largo Plazo , Masculino , Estudios Multicéntricos como Asunto , Estudios Prospectivos , Diálisis RenalRESUMEN
OBJECTIVE: Assessment of growth after renal transplantation in children of pubertal age by analyzing the annual increment in height standard deviation score (Ht SDS) in all girls > or =10 years and boys > or =11 years of age at the time of transplantation until latest follow-up (minimum 2 years). PATIENTS: A total of 59 grafts were placed in 54 recipients (30 boys) between December 1984 and January 1995. Mean (range) age at transplantation was 13.6 years (10.1 to 17.7 years). Fifty-one percent had congenital renal disease, 36% acquired renal disease, and 13% had hereditary nephropathies. Eighty-seven percent were first grafts; of these, 29% were performed pre-emptively, and 23% were from living related donors. RESULTS: Mean (SD) Ht SDS at transplantation was -1.8 (0.2) and increased significantly thereafter, such that it was -1.6 (0.2) at 1 year, n = 52; -1.5 (0.2) at 2 years, n = 47; -1.0 (0.2) at 3 years, n = 27; -0.7 (0.3) at 4 years, n = 19; and -0.6 (0.3), n = 13, at 5 years after transplantation (analysis of variance, P < .001). The greatest improvement in Ht SDS in the first year was seen in children with the highest glomerular filtration rate (r = 0.429, P = .002) and in those who were shortest at the time of transplantation (r = -0.356, P = .009). CONCLUSION: Catch-up growth occurs in children receiving renal transplants during the expected time of puberty.