Yan Albert C, Honig Paul J, Ming Michael E, Weber Janice, Shah Kara N
Section of Dermatology, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, 3550 Market St, Second Floor, Philadelphia, PA 19104, USA.
Arch Dermatol. 2010 Jan;146(1):57-62. doi: 10.1001/archdermatol.2009.326.
Impaired skin integrity in patients with Netherton syndrome (NS) results in significant systemic absorption of topically applied medications. Some have advocated the administration of pimecrolimus, 1%, topical cream for the treatment of patients with NS. Insufficient data exist with regard to its safety, systemic absorption, and efficacy.
An exploratory study was conducted involving 3 children with NS who received twice-daily application of pimecrolimus, 1%, cream over 18 months. There were no notable abnormalities in hematologic or chemistry profiles. Blood levels of pimecrolimus ranged from 0.625 to 7.08 ng/mL, with peak levels reached during the first month in all 3 patients. Dramatic reductions were observed in the Netherton Area and Severity Assessment, Eczema Area and Severity Index, Investigator Global Evaluation of Disease, and pruritus scores compared with baseline levels.
Use of pimecrolimus, 1%, cream was well tolerated and demonstrated marked improvements in nearly all of the parameters evaluated. Patients treated with pimecrolimus responded rapidly, within the first month of treatment, and improvement persisted throughout the study period. In adult patients receiving oral pimecrolimus, blood levels as high as 54 ng/mL for 3 months have not shown clinically significant immunosuppression. Absorption of pimecrolimus, 1%, cream was detectable, but levels were much lower than expected even when applied to 50% of total body surface area. Larger studies are warranted to determine the safety and efficacy of pimecrolimus, 1%, cream in the treatment of NS.
clinicaltrials.gov Identifier: NCT00208026.
Netherton综合征（NS）患者的皮肤完整性受损，导致局部应用药物的全身吸收显著增加。一些人主张使用1%的吡美莫司乳膏治疗NS患者。关于其安全性、全身吸收和疗效的数据不足。
开展了一项探索性研究，纳入3例NS患儿，每日两次外用1%吡美莫司乳膏，为期18个月。血液学或生化指标未见明显异常。吡美莫司血药浓度范围为0.625至7.08 ng/mL，所有3例患者均在第1个月达到峰值。与基线水平相比，Netherton面积和严重程度评估、湿疹面积和严重程度指数、研究者对疾病的整体评估以及瘙痒评分均显著降低。
1%吡美莫司乳膏耐受性良好，几乎所有评估参数均有显著改善。接受吡美莫司治疗的患者在治疗的第1个月内迅速起效，且改善持续至整个研究期。接受口服吡美莫司的成年患者，血药浓度高达54 ng/mL持续3个月，未显示出临床显著的免疫抑制作用。1%吡美莫司乳膏可检测到吸收，但即使应用于50%的体表面积，血药浓度也远低于预期。有必要开展更大规模的研究以确定1%吡美莫司乳膏治疗NS的安全性和疗效。
clinicaltrials.gov标识符：NCT00208026。
