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慢性肾功能不全儿童停用重组人生长激素治疗后的生长情况。基因泰克合作研究小组。

Growth after discontinuation of recombinant human growth hormone therapy in children with chronic renal insufficiency. The Genentech Cooperative Study Group.

作者信息

Fine R N, Brown D F, Kuntze J, Wooster P, Kohaut E E

机构信息

Department of Pediatrics, State University of New York at Stony Brook 11794-8111, USA.

出版信息

J Pediatr. 1996 Dec;129(6):883-91. doi: 10.1016/s0022-3476(96)70033-1.

Abstract

OBJECTIVES

The impact of a pause in treatment with recombinant human growth hormone (rhGH) during the course of chronic renal insufficiency (CRI) once target height (50th percentile for mid-parental height) is reached and the impact of cessation of rhGH treatment after successful renal transplantation were evaluated. Prepubertal patients with CRI included in a multicenter, randomized, double-blind, placebo-controlled trial who either reached target height or received a renal transplant, or both, were included in this report. Patients in the placebo group may have initiated pubertal development at the time rhGH treatment was begun.

STUDY DESIGN

Growth velocity (measured in centimeters per year) and standardized height score (SDS) in 22 patients who had a pause in rhGH therapy after attainment of target height were evaluated serially before and after the pause, and 30 patients, 4 of whom were also included in the pause group, who discontinued rhGH therapy at the time of transplantation were followed as long as 68 months after transplantation.

RESULTS

Six of twenty-two patients (two of whom subsequently underwent transplantation) continued with the pause in treatment for a mean (+/-SD) duration of 25.5 +/- 26.9 months (group 1), and 16 of 22 resumed rhGH therapy after pausing for a mean (+/-SD) of 9.0 +/- 4.6 months (group 2). The mean (+/-SD) growth velocity during the pause in group 1 was 5.1 +/- 1.8 cm/yr and in group 22.7 +/- 1.7 cm/yr. After reinstitution of rhGH in group 2, the mean (+/-SD) growth velocity increased to 7.2 +/- 1.7 cm/yr. The mean (+/-SD) height SDS in the 30 patients who discontinued rhGH therapy at the time of transplantation was -2.8 +/- 0.9 at baseline (initiation of rhGH therapy), -1.6 +/- 1.3 at the time of transplantation, and -1.7 +/- 1.2 at last follow-up. The mean (+/-SD) growth velocity was 5.1 +/- 4.7 cm/yr after transplantation, and the mean (+/-SD) delta (delta) height SDS was -0.07 +/- 0.5 at last follow-up.

CONCLUSIONS

A pause in rhGH treatment in children with CRI after attainment of target height leads to maintenance of height SDS in 27% and a marked reduction in growth velocity, requiring reinstitution of rhGH therapy, in 73%; discontinuing rhGH treatment at the time of transplantation does not result in substantive posttransplantation "catch down" growth.

摘要

目的

评估慢性肾功能不全(CRI)病程中,一旦达到目标身高(父母平均身高的第50百分位数)后停用重组人生长激素(rhGH)的影响,以及肾移植成功后停止rhGH治疗的影响。纳入一项多中心、随机、双盲、安慰剂对照试验的青春期前CRI患者,这些患者达到了目标身高或接受了肾移植,或两者兼而有之,均纳入本报告。安慰剂组的患者可能在rhGH治疗开始时已开始青春期发育。

研究设计

对22例达到目标身高后暂停rhGH治疗的患者,在暂停前后连续评估生长速度(以每年厘米数衡量)和标准化身高评分(SDS);对30例在移植时停止rhGH治疗的患者进行随访,随访时间长达移植后68个月,其中4例也纳入了暂停组。

结果

22例患者中有6例(其中2例随后接受了移植)继续暂停治疗,平均(±标准差)持续时间为25.5±26.9个月(第1组),22例中的16例在暂停平均(±标准差)9.0±4.6个月后恢复rhGH治疗(第2组)。第1组暂停治疗期间的平均(±标准差)生长速度为5.1±1.8厘米/年,第2组为2.7±1.7厘米/年。第2组重新使用rhGH后,平均(±标准差)生长速度增至7.2±1.7厘米/年。30例在移植时停止rhGH治疗的患者,基线(rhGH治疗开始时)的平均(±标准差)身高SDS为-2.8±0.9,移植时为-1.6±1.3,末次随访时为-1.7±1.2。移植后的平均(±标准差)生长速度为5.1±4.7厘米/年,末次随访时平均(±标准差)身高SDS的变化(Δ)为-0.07±0.5。

结论

CRI患儿达到目标身高后暂停rhGH治疗,27%的患儿身高SDS得以维持,73%的患儿生长速度显著降低,需要重新使用rhGH治疗;移植时停止rhGH治疗不会导致移植后出现实质性的“追赶生长”。

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