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泼尼松龙与醋酸氢化可的松治疗21-羟化酶缺乏所致经典型先天性肾上腺皮质增生症患者的比较研究

[Comparative study of prednisolone versus hydrocortisone acetate for treatment of patients with the classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency].

作者信息

Leite Flavia M, Longui Carlos A, Kochi Cristiane, Faria Claudia D C, Borghi Mauro, Calliari Luis Eduardo P, Monte Osmar

机构信息

Unidade de Endocrinologia Pediátrica, Departamento de Pediatria e Puericultura, Irmandade da Santa Casa de Misericórdia de São Paulo, São Paulo, SP, Brazil.

出版信息

Arq Bras Endocrinol Metabol. 2008 Feb;52(1):101-8. doi: 10.1590/s0004-27302008000100014.

DOI:10.1590/s0004-27302008000100014
PMID:18345402
Abstract

Hydrocortisone acetate is usually employed in the treatment of classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. In Brazil, however, oral hydrocortisone acetate is only available from manipulation pharmacies. Prednisolone has stable oral pharmaceutical formulations commercially available, with the advantage of a single daily dose. The aim of this study was to compare the efficacy of oral prednisolone and oral hydrocortisone in the treatment of CAH due to 21-hydroxylase deficiency. Fifteen patients with mean (SD) chronological age of 7.2 (3.6) years, were evaluated in two consecutive 1-year periods. In the first year, hydrocortisone (17.5 mg/m2/day, divided in three doses) was used in the treatment, followed by the use of prednisolone (3 mg/m2/day, once in the morning) in the second year. The comparison between the two treatments was assessed after a one-year treatment period by: variation of height standard deviation score (SDS) (delta Height SDS), variation of height SDS according to bone age (delta BA SDS), variation of body mass SDS (delta BMI SDS) and serum levels of androstenedione. No significant difference was observed in relation to the delta Height SDS, delta BA SDS and delta BMI SDS. No significant difference was observed in the serum levels of androstenedione. We conclude that the efficacy of prednisolone administered once a day orally is comparable to the oral use of hydrocortisone three times a day. Oral prednisolone may be an option for patients with CAH due to 21-hydroxylase deficiency.

摘要

醋酸氢化可的松通常用于治疗因21-羟化酶缺乏引起的经典型先天性肾上腺皮质增生症(CAH)。然而,在巴西,口服醋酸氢化可的松仅可从调配药房获得。泼尼松龙有稳定的口服制剂可供商业销售,优点是每日只需服用一次。本研究的目的是比较口服泼尼松龙和口服醋酸氢化可的松治疗因21-羟化酶缺乏引起的CAH的疗效。15名患者的平均(标准差)实足年龄为7.2(3.6)岁,在两个连续的1年期内接受评估。第一年使用氢化可的松(17.5 mg/m²/天,分三次服用)进行治疗,第二年使用泼尼松龙(3 mg/m²/天,晨起一次服用)。在一年的治疗期后,通过以下指标评估两种治疗方法的差异:身高标准差评分(SDS)的变化(身高SDS差值)、根据骨龄计算的身高SDS变化(骨龄SDS差值)、体重SDS变化(BMI SDS差值)以及血清雄烯二酮水平。在身高SDS差值、骨龄SDS差值和BMI SDS差值方面未观察到显著差异。在血清雄烯二酮水平方面也未观察到显著差异。我们得出结论,每日口服一次泼尼松龙的疗效与每日口服三次醋酸氢化可的松相当。对于因21-羟化酶缺乏引起的CAH患者,口服泼尼松龙可能是一种选择。

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