RESUMO
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.
Assuntos
Cistinose/terapia , Transtornos do Crescimento/terapia , Hormônio do Crescimento/uso terapêutico , Nefropatias/terapia , Adolescente , Estatura/efeitos dos fármacos , Criança , Pré-Escolar , Feminino , Humanos , Transplante de Rim , Assistência de Longa Duração , Masculino , Estudos Multicêntricos como Assunto , Estudos Prospectivos , Diálise RenalRESUMO
Collecting pertinent information is first step in assessing the use of living-related kidneys for transplantation. Current bioethics legislation in France limits kidney donation to first-degree family members and spouses in emergency situations. Severe penalties are inflicted for use of other donors or sale of organs. Further valuable information can be obtained from reports in the literature on complications in donors and on the advantages of living donor organs. The proportion of live donors in France is small (3.5% from 1984 through 1993) indicating that transplantation teams prefer cadaver organs except in pediatric cases. The proportion of live donor organs transplanted in northern Europe and North America is much higher. A quick survey of French teams show that opinions and practices vary. Questions still under debate include how to guarantee freedom to refuse or accept, a freedom directly related to correct information. Several propositions have been made in an attempt to harmonize management. First, an information sheet could be distributed during the early discussions, outlining the advantages and disadvantages of live organ donation. A list of complementary examinations could also be established to identify possible contraindications for nephrectomy and define exclusion criteria. A similar procedure adopted by all transplantation teams could be based on these propositions presented in the appendix. Potential donors could then benefit from uniform protection.
Assuntos
Transplante de Rim , Doadores de Tecidos , Família , França , Humanos , Transplante de Rim/estatística & dados numéricos , Fatores de Risco , Doadores de Tecidos/legislação & jurisprudência , Doadores de Tecidos/estatística & dados numéricosRESUMO
The long-term follow-up of 28 patients with congenital primary tubular acidosis is described. Ten patients had affected siblings but no history of similar symptoms in the preceding generation. Deafness was associated in 14 patients and developed before 12 years of age. Deafness was present in all familial cases, and patients without deafness showed no familial incidence, suggesting the existence of two different entities. All patients had growth retardation, which was more severe in the older patients and was always markedly improved by alkaline therapy. Rickets was found in some patients but seemed related to vitamin D deficiency. Catch-up growth was limited to the first 2 years of therapy in patients treated before 2 years of age, but sometimes lasted longer in older patients. Of the 12 patients who reached adulthood, those without rickets achieved a normal height but the others did not. We believe that therapy should be continued throughout life because of the risk of nephrocalcinosis.
Assuntos
Acidose Tubular Renal , Acidose Tubular Renal/congênito , Acidose Tubular Renal/tratamento farmacológico , Acidose Tubular Renal/fisiopatologia , Estatura , Desenvolvimento Ósseo , Criança , Pré-Escolar , Feminino , Seguimentos , Crescimento , Humanos , Lactente , Recém-Nascido , Masculino , PuberdadeRESUMO
A controlled study of growth in children receiving alternate-day versus daily corticosteroid regimens was performed with 60 children and adolescents with kidney grafts. The study started 14 to 27 months after transplantation in patients with normal graft function, after a graft biopsy. Thirty-five patients were available for the growth study; 17 were randomly allocated to receive alternate-day therapy and 18 remained on a daily regimen. The cumulative dose was similar in the two groups, as were bone age and renal function. After 1 year of follow-up, the mean statural growth, expressed as change in SD score, was significantly better in those on the alternate-day regimen (+0.49 +/- 0.42 SD/yr) than in those on the daily regimen (-0.12 +/- 0.53 SD/yr; p less than 0.005). The difference was also significant when prepubertal and pubertal children were analyzed separately. During the second year of the study most children who were receiving daily treatment were given alternate-day therapy; their mean growth velocity increased to +0.29 +/- 0.35 SD/yr (p less than 0.05 vs the first year); children who had been on the alternate-day regimen since the outset of the study continued to have similar positive SD scores (0.52 +/- 0.37 SD/yr). Renal function remained stable throughout the study regardless of corticosteroid regimen, except in the case of one patient undergoing daily therapy who had a rejection crisis. We conclude that in children with a kidney graft a given cumulative dose of corticosteroid has a significantly lesser inhibitory effect on growth velocity when given on alternate days.
Assuntos
Crescimento/efeitos dos fármacos , Transplante de Rim , Prednisona/administração & dosagem , Adolescente , Determinação da Idade pelo Esqueleto , Estatura/efeitos dos fármacos , Criança , Esquema de Medicação , Feminino , Seguimentos , Humanos , Imunossupressores/uso terapêutico , Masculino , Prednisona/uso terapêutico , Fatores de TempoRESUMO
Renal biopsy, the introduction of immunohistologic methods and electron microscopy have allowed the differentiation of clinicopathologic entities associated to nephrotic syndrome. Two main categories must be differentiated: in the first, diffuse lesions of the glomerulus, including those secondary to specific diseases the same as those that are apparently primary, are responsible for the increased permeability of glomerular capillaries. Any one of the following clinical signs suggests this category: acute onset with nephritic syndrome, moderate nephritic syndrome, moderate nephrotic syndrome, gross hematuria, persistent hypertension and/or renal failure, poor selectivity of proteinuria and drop in complement serum levels (C3). In the second category, known as idiopathic nephrotic syndrome, the mechanism of disorder of the glomerular capillary is unknown and the nephrotic syndrome is more marked. In most cases with idiopathic nephrotic syndrome, minimal glomerular lesions (MGL) are present. The clinicopathologic correlation among these three types of lesions shows that the type with MGL is characterized by selective proteinuria, absence of hematuria, good response to corticosteroids and good outlook; whereas in types with diffuse mesangial proliferation (DMP) and segmentary sclerosis, proteinuria is frequently non selective, hematuria shows in 50 to 75% of the patients; prognosis is poor. However, MGL, DMP and focal segmentary glomerular sclerosis are not different entities, but represent variants of the same disease. Considering that corticosensitive nephrosis to this moment is the most common cause of the nephrotic syndrome, especially in children under 8 years, renal biopsy should be done only under two circumstances: a) when the clinical symptoms suggest diffuse glomerular lesions and b), when resistance to corticosteroids becomes evident.
Assuntos
Rim/patologia , Síndrome Nefrótica/patologia , Corticosteroides/uso terapêutico , Biópsia , Criança , Pré-Escolar , Clorambucila/uso terapêutico , Resistência a Medicamentos , Feminino , Glomerulonefrite/patologia , Humanos , Lactente , Glomérulos Renais/patologia , Masculino , Nefrose/patologia , Nefrose Lipoide , Síndrome Nefrótica/tratamento farmacológico , PrognósticoRESUMO
Of 33 children with membranous nephropathy screened for HBs Ag, 14 were found to be HBs Ag carriers, whereas HBs Ag was detected in 3 of 170 and 4 of 100 children with glomerular and nonglomerular kidney diseases, respectively. HBs Ag was often associated with acute hepatitis at onset (five patients) or with elevated transminases values. This high incidence and the prevalence of an unusual subtype (ayw2) suggest a relationship between HBs Ag and the glomerular lesions. Using immunofluorescence, however, HBs Ag could not be detected within the deposits, so that the nature of the relationship cannot be considered as established. The clinical outcome (50% remission), the plasma complement component disturbances, and findings by immunofluorescence did not differ from those observed in children with MGN without detectable HBs Ag.
Assuntos
Glomerulonefrite/imunologia , Antígenos de Superfície da Hepatite B/isolamento & purificação , Adolescente , Portador Sadio , Criança , Pré-Escolar , Proteínas do Sistema Complemento/isolamento & purificação , Feminino , Glomerulonefrite/complicações , Glomerulonefrite/patologia , Hepatite B/imunologia , Anticorpos Anti-Hepatite B/isolamento & purificação , Humanos , Técnicas Imunológicas , Lactente , Rim/patologia , Masculino , Síndrome Nefrótica/complicações , Fatores SexuaisRESUMO
To assess the gonadal effects of chlorambucil given to prepubertal and pubertal boys, testicular function of 21 adolescents or young men, treated with chlorambucil for nephrotic syndrome before or during puberty, have been evaluated. Evaluations have been based on penis and testicular size, serum FSH, LH and testosterone values, and sperm count. Nine patients have testicular hypotrophy, 13 have significantly elevated FSH, 17 have azoospermia, two have severe oligospermia, and repetitive sperm counts show no improvement. A quiescent state of the epithelium of the seminiferous tubules does not protect against chlorambucil toxicity, which seems to be related to the cumulative dose. Chlorambucil should be used with caution, even in children.