Division of Pediatric Endocrinology, Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana, USA.
Department of Biostatistics and Health Data Science, Indiana University School of Medicine and Richard M. Fairbanks School of Public Health, Indianapolis, Indiana, USA.
Horm Res Paediatr. 2022;95(5):461-464. doi: 10.1159/000525332. Epub 2022 Jun 1.
Three times daily (TID) hydrocortisone (HC) is recommended as the optimal glucocorticoid regimen in growing children with congenital adrenal hyperplasia (CAH). However, a variety of other treatment schemes are used in the clinical setting.
The aim of this study was to determine whether there are clinical differences between children being treated with TID HC versus those receiving other glucocorticoid regimens. Furthermore, we sought to determine whether there was evidence of a deleterious effect on growth in children receiving treatment with alternate regimens.
Medical records of children followed in our pediatric endocrinology outpatient clinic for classic CAH secondary to 21-hydroxylase deficiency during the last 10 years were reviewed. Variables analyzed included sex, age at the most recent visit, glucocorticoid type, frequency and dose (mg/m2/day), height z-score, BMI z-score, ethnicity, most recent bone age, growth velocity z-score, and provider's impression of compliance (good or poor).
Of 104 children (51% boys) with CAH, 50 (48%) were on TID HC, 43 (41%) were on prednisone or prednisolone, and 5 (5%) were on dexamethasone. An additional 6 (6%) were on HC administered either 2 or 4 times daily. No differences were seen between TID HC and alternate regimen groups with respect to sex, height z-score, BMI z-score, ethnicity, provider assessment of compliance, ratio of bone age to chronologic age, or growth velocity. The average height z-score was -0.40 ± 1.31 in the TID HC group compared to -0.87 ± 1.33 in the alternate regimen group (p = 0.075). Patients receiving TID HC were younger (p = 0.027) and on a lower glucocorticoid dose (p = 0.001) than those on alternate regimens.
Less than half of our patients with CAH were receiving TID HC. Reassuringly, growth parameters and other indices of disease control were equivalent between patients on conventional HC dosing and other therapeutic approaches. These results suggest that a range of glucocorticoid treatment regimens may be equally viable in children with CAH.
每日三次（TID）氢化可的松（HC）被推荐为患有先天性肾上腺增生症（CAH）的生长中儿童的最佳糖皮质激素治疗方案。然而，在临床实践中还使用了其他各种治疗方案。
本研究旨在确定接受 TID HC 治疗的儿童与接受其他糖皮质激素治疗方案的儿童之间是否存在临床差异。此外，我们还试图确定接受替代方案治疗的儿童的生长是否存在有害影响的证据。
回顾了过去 10 年来在我们儿科内分泌门诊就诊的因 21-羟化酶缺乏而患有经典 CAH 的儿童的病历。分析的变量包括性别、最近就诊时的年龄、糖皮质激素类型、频率和剂量（mg/m2/天）、身高 z 评分、BMI z 评分、种族、最近的骨龄、生长速度 z 评分以及医生对依从性的印象（好或差）。
在 104 名患有 CAH 的儿童（51%为男性）中，50 名（48%）接受 TID HC 治疗，43 名（41%）接受泼尼松或泼尼松龙治疗，5 名（5%）接受地塞米松治疗。另外 6 名（6%）接受每日 2 次或 4 次的 HC 治疗。在 TID HC 组和替代方案组之间，在性别、身高 z 评分、BMI z 评分、种族、医生对依从性的评估、骨龄与实际年龄之比或生长速度方面均未见差异。TID HC 组的平均身高 z 评分为-0.40±1.31，而替代方案组为-0.87±1.33（p=0.075）。接受 TID HC 治疗的患者比接受替代方案治疗的患者年龄更小（p=0.027）且糖皮质激素剂量更低（p=0.001）。
我们的 CAH 患者中只有不到一半接受 TID HC 治疗。令人欣慰的是，接受常规 HC 剂量治疗和其他治疗方法的患者的生长参数和其他疾病控制指标是等效的。这些结果表明，在 CAH 儿童中，多种糖皮质激素治疗方案可能同样可行。
