Fundação de Medicina Tropical do Amazonas and Universidade Estadual do Amazonas, Manaus, AM, Brasil.
Am J Trop Med Hyg. 2011 Feb;84(2):255-60. doi: 10.4269/ajtmh.2011.10-0155.
Miltefosine has been used in the treatment of several new world cutaneous leishmaniasis (CL) species with variable efficacy. Our study is the first evidence on its clinical efficacy in Leishmania (Viannia) guyanensis. In this phase II/III randomized clinical trial, 90 CL patients were randomly allocated (2:1) to oral miltefosine (2.5 mg/kg/day/28 days) (N = 60) or parenteral antimony (15-20 mg/Sb/kg/day/20 days) (N = 30) according to age groups: 2-12 y/o and 13-65 y/o. Patients were human immunodeficiency virus (HIV) noninfected parasitological proven CL without previous treatment. Definitive cure was accessed at 6 months follow-up visit. No severe adverse events occurred. Vomiting was the most frequent adverse event (48.3%) followed by nausea (8.6%) and diarrhea (6.7%). Cure rates were 71.4% (95% confidence interval [CI] = 57.8-82.7) and 53.6% (95% CI = 33.9-72.5) (P = 0.05) for miltefosine and antimonial, respectively. There were no differences in cure rates between age groups within the same treatment arms. Miltefosine was safe and relatively well tolerated and cure rate was higher than antimony.
米替福新已用于多种新世界皮肤利什曼病（CL）的治疗，疗效不一。本研究是其在利什曼（Viannia）圭亚那亚种中的临床疗效的首次证据。在这项 II/III 期随机临床试验中，90 例 CL 患者根据年龄组（2-12 岁和 13-65 岁）按 2:1 的比例随机分为口服米替福新（2.5 mg/kg/天/28 天）（N=60）或静脉注射锑（15-20 mg/Sb/kg/天/20 天）（N=30）。所有患者均为人免疫缺陷病毒（HIV）阴性、寄生虫学证实未经治疗的 CL。在 6 个月随访时评估治愈情况。未发生严重不良事件。最常见的不良事件为呕吐（48.3%），其次为恶心（8.6%）和腹泻（6.7%）。米替福新组的治愈率为 71.4%（95%置信区间 [CI] = 57.8-82.7），而锑组的治愈率为 53.6%（95% CI = 33.9-72.5）（P=0.05）。在相同治疗组中，不同年龄组的治愈率无差异。米替福新安全且相对耐受良好，治愈率高于锑。
