Oral miltefosine for Indian visceral leishmaniasis
Oral miltefosine for Indian visceral leishmaniasis
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DOI：
10.1056/nejmoa021556
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发表时间：
2002-11-28
影响因子：
158.5
通讯作者：
Berman, J
中科院分区：
文献类型：
--
作者：
Sundar, S;Jha, TK;Berman, J
背景：每年有50万例内脏利什曼病病例，主要发生在印度次大陆。几乎所有未经治疗的患者都会死亡，所有有效的药物都是胃肠外给药。米替福新是一种口服药物，已在少数患者中显示对印度内脏利什曼病具有良好的治疗指数。我们在印度进行了一项临床试验，将米替福新与最有效的标准治疗药物阿替霉素B进行比较。这项研究是一项随机、开放标签的比较，其中299名12岁或以上的患者接受口服米替福新（50或100 mg [约2.5 mg/kg体重]每日，持续28天），99名患者接受静脉内给药的阿替霉素B结果：各组在年龄、体重、既往治疗利什曼病失败的比例、脾穿刺物的寄生虫学分级和脾肿大方面匹配良好。在治疗结束时，米替福新组293例患者和阿替霉素B组98例患者的脾穿刺液。没有发现寄生虫，初步治愈率为100%。治疗结束后6个月，米替福新组299名患者中的282名（94%[95%置信区间，91 - 97]）和阿替福辛B组99名患者中的96名（97%）没有复发;这些患者被归类为治愈。呕吐和腹泻，一般持续一到两天，发生在38%和20%的患者在米替福新group.Conclusions：口服米替福新是一种有效和安全的治疗印度内脏利什曼病。米替福新可能是特别有利的，因为它可以口服给药。在寄生虫对当前药物有耐药性的地区，它也可能有帮助。
Background: There are 500,000 cases per year of visceral leishmaniasis, which occurs primarily in the Indian subcontinent. Almost all untreated patients die, and all the effective agents have been parenteral. Miltefosine is an oral agent that has been shown in small numbers of patients to have a favorable therapeutic index for Indian visceral leishmaniasis. We performed a clinical trial in India comparing miltefosine with the most effective standard treatment, amphotericin B.Methods: The study was a randomized, open-label comparison, in which 299 patients 12 years of age or older received orally administered miltefosine (50 or 100 mg [approximately 2.5 mg per kilogram of body weight] daily for 28 days) and 99 patients received intravenously administered amphotericin B (1 mg per kilogram every other day for a total of 15 injections).Results: The groups were well matched in terms of age, weight, proportion with previous failure of treatment for leishmaniasis, parasitologic grade of splenic aspirate, and splenomegaly. At the end of treatment, splenic aspirates were obtained from 293 patients in the miltefosine group and 98 patients in the amphotericin B group. No parasites were identified, for an initial cure rate of 100 percent. By six months after the completion of treatment, 282 of the 299 patients in the miltefosine group (94 percent [95 percent confidence interval, 91 to 97]) and 96 of the 99 patients in the amphotericin B group (97 percent) had not had a relapse; these patients were classified as cured. Vomiting and diarrhea, generally lasting one to two days, occurred in 38 percent and 20 percent of the patients in the miltefosine group, respectively.Conclusions: Oral miltefosine is an effective and safe treatment for Indian visceral leishmaniasis. Miltefosine may be particularly advantageous because it can be administered orally. It may also be helpful in regions where parasites are resistant to current agents.