乙曲替酸治疗儿童重症遗传角化性皮肤病的疗效及不良反应

An Appraisal of Acitretin Therapy on the Efficacy and Side Effects in Children with Inherited Disorders of Keratinization

目的研究乙曲替酸口服治疗儿童重度遗传角化性皮肤病的临床疗效、不良反应及对儿童生长发育的影响，评估乙曲替酸在儿童患者中应用的临床疗效及安全性。方法对22例重症角化性皮肤病患儿用乙曲替酸治疗，剂量为0．67～1．07mg/（kg·d），维持量为0～0．94mg／（kg·d）。选择身高与体质量作为儿童生长发育的检测指标，每1～2个月随访1次，共随访3～36个月。结果经2～4个月的治疗，临床痊愈率达77．3％（17／22），显效率为18．2％（4／22）。不良反应：干燥性唇炎54．5％（12／22），皮肤瘙痒36．4％（8／22），皮肤脆性增加27．3％（6／22），口干22．7％（5／22），食欲下降13．6％（3／22），听力障碍及食欲增强均为4．5％（1／22）。随访期间，儿童的身高、体质量在同年龄组正常范围。结论乙曲替酸对治疗儿童重症遗传角化性皮肤病疗效满意，尽管有较多的一般性临床不良反应发生，但对儿童生长发育未见到明显的影响，对重症患儿来说仍然是一种良好的选择。

Objective To study the acitretin as the treatment for children cases with severe inherited disorders of keratinization. Clinical data of efficacy and tolerability of acitretin therapy were available for all patients and defines guidelines for treatment. Methods 22 children with severe inherited disorders of keratinization had been treated by acitretin, the body weight and height had been adopted to investigate the growth and development of children, the treatment and maintancedose were 0.67 - 1.07 mg/（ kg · d） and 0 - 0.94 mg/ （kg ·d） respectively and the cumulative follow-up was 3 - 36 months for these 22 patients. Results The overall improvement was considerable; the clinical cured and effective rate were 77.3% and 18.2%, with only one case responding poorly. Most of those cases were showed the excellent response. Mild to moderate dry lips was most frequent （54.5 % ）, the next were pruritus （36.4%）, skin fragility （27.3%）, and dry mouth（22.7% ）. The less frequent side effects were anorexie （ 13.6% ）, and hypoacusis （4.5%）. Irreversible side effects did not occur and showed no any effected on the growth and development of those children cases during treatment in the study. Conclusion The overall improvement of acitretin therapy in treatment of severe disorders of keratinization is considerable, with only mild to moderate side effects and has no effected on the growth and development in children. Acitretin therapy represents the treatment of choice for severe inherited disorders of keratinization and it is a safe and effective treatment during the investigation.