Zaoutis Theoklis E, Jafri Hasan S, Huang Li-Min, Locatelli Franco, Barzilai Asher, Ebell Wolfram, Steinbach William J, Bradley John, Lieberman Jay M, Hsiao Chih-Cheng, Seibel Nita, Laws Hans-Juergen, Gamba Melinda, Petrecz Maria, Taylor Arlene F, Strohmaier Kim M, Chow Joseph W, Kartsonis Nicholas A, Ngai Angela L
Children's Hospital of Philadelphia, Division of Infectious Diseases, 34th Street and Civic Center Boulevard, CHOP North, Suite 1527, Philadelphia, PA 19104, USA.
Pediatrics. 2009 Mar;123(3):877-84. doi: 10.1542/peds.2008-1158.
We evaluated the safety, tolerability, and efficacy of caspofungin in pediatric patients with invasive aspergillosis, invasive candidiasis, or esophageal candidiasis.
This was a multicenter, prospective, open-label study in children 3 months to 17 years of age with proven or probable invasive aspergillosis, proven invasive candidiasis, or proven esophageal candidiasis. All of the patients received caspofungin 70 mg/m(2) on day 1, followed by 50 mg/m(2) per day (maximum: 70 mg/day), as primary or salvage monotherapy. Favorable response was defined as complete resolution of clinical findings and microbiologic (or radiographic/endoscopic) eradication (complete response) or significant improvement in these parameters (partial response). Efficacy was assessed at the end of caspofungin therapy in patients with a confirmed diagnosis who received >/=1 dose of caspofungin. The primary safety evaluation was the proportion of patients with clinical or laboratory drug-related adverse events.
Of the 49 patients enrolled, 3 were <2 years of age, 30 were 2 to 11 years of age, and 16 were 12 to 17 years of age. Forty-eight patients had confirmed disease: invasive aspergillosis (10), invasive candidiasis (37), and esophageal candidiasis (1). Eight of 10 patients with invasive aspergillosis had pulmonary involvement; 34 of 37 patients with invasive candidiasis had candidemia. Caspofungin was given for 2 to 87 days. Success at end of therapy was achieved in 5 of 10 patients with invasive aspergillosis, 30 of 37 with invasive candidiasis, and 1 of 1 with esophageal candidiasis. One patient (invasive candidiasis) relapsed during the 28-day follow-up period. Drug-related clinical or laboratory adverse events occurred in 27% and 35% of patients, respectively. There were no serious drug-related adverse events or discontinuations of caspofungin because of toxicity.
Caspofungin was generally well tolerated in pediatric patients aged 6 months through 17 years. Efficacy outcomes in patients with invasive aspergillosis or invasive candidiasis were consistent with previous adult studies in these indications.
我们评估了卡泊芬净在患有侵袭性曲霉病、侵袭性念珠菌病或食管念珠菌病的儿科患者中的安全性、耐受性和疗效。
这是一项针对3个月至17岁已确诊或疑似患有侵袭性曲霉病、确诊侵袭性念珠菌病或确诊食管念珠菌病儿童的多中心、前瞻性、开放标签研究。所有患者在第1天接受70mg/m²的卡泊芬净治疗，随后每天接受50mg/m²（最大剂量：70mg/天），作为一线或挽救性单药治疗。良好反应定义为临床症状完全缓解且微生物学（或影像学/内镜检查）清除（完全缓解）或这些参数有显著改善（部分缓解）。在确诊且接受≥1剂卡泊芬净治疗的患者中，在卡泊芬净治疗结束时评估疗效。主要安全性评估是发生临床或实验室药物相关不良事件的患者比例。
在纳入的49例患者中，3例年龄小于2岁，30例年龄在2至11岁之间，16例年龄在12至17岁之间。48例患者确诊患病：侵袭性曲霉病（10例）、侵袭性念珠菌病（37例）和食管念珠菌病（1例）。10例侵袭性曲霉病患者中有8例有肺部受累；37例侵袭性念珠菌病患者中有34例有念珠菌血症。卡泊芬净给药2至87天。10例侵袭性曲霉病患者中有5例在治疗结束时取得成功，37例侵袭性念珠菌病患者中有30例，1例食管念珠菌病患者中有1例。1例患者（侵袭性念珠菌病）在28天随访期内复发。分别有27%和35%的患者发生药物相关的临床或实验室不良事件。没有严重的药物相关不良事件，也没有因毒性而停用卡泊芬净的情况。
卡泊芬净在6个月至17岁的儿科患者中总体耐受性良好。侵袭性曲霉病或侵袭性念珠菌病患者的疗效结果与先前针对这些适应症的成人研究一致。
