Musiime Victor, Bwakura-Dangarembizi Mutsa, Szubert Alexander J, Mumbiro Vivian, Mujuru Hilda A, Kityo Cissy M, Lugemwa Abbas, Doerholt Katja, Chabala Chishala, Makumbi Shafic, Mulenga Veronica, McIlleron Helen, Burger David, Natukunda Eva, Shakeshaft Clare, Linda Kyomuhendo Jovia, Nathoo Kusum, Monkiewicz Lara, Yawe Ibrahim, Kapasa Monica, Nyathi Mary, Lungu Joyce, Nduna Bwendo, Ndebele Wedu, South Annabelle, Mwamabazi Mwate, Musoro Godfrey, Griffiths Anna, Zyambo Khozya, Nazzinda Rashidah, Zimba Kevin, Zhang Yingying, Walker Simon, Turkova Anna, Walker A Sarah, Bamford Alasdair, Gibb Diana M
Makerere University, College of Health Sciences, School of Medicine, Department of Paediatrics and Child Health, Kampala, Uganda.
Joint Clinical Research Centre, Kampala, Uganda.
N Engl J Med. 2025 May 15;392(19):1917-1932. doi: 10.1056/NEJMoa2404597.
Children living with human immunodeficiency virus (HIV) have limited options for second-line antiretroviral therapy (ART).
In this open-label trial with a 2-by-4 factorial design, we randomly assigned children with HIV who had first-line treatment failure to receive second-line therapy with tenofovir alafenamide fumarate (TAF)-emtricitabine or standard care (abacavir or zidovudine, plus lamivudine) as the backbone and dolutegravir or ritonavir-boosted darunavir, atazanavir, or lopinavir as the anchor drug. The primary outcome was a viral load of less than 400 copies per milliliter at 96 weeks. We hypothesized that TAF-emtricitabine would be noninferior to standard care, that dolutegravir and ritonavir-boosted darunavir would each be superior to ritonavir-boosted lopinavir and atazanavir analyzed in combination, and that ritonavir-boosted atazanavir would be noninferior to ritonavir-boosted lopinavir. Safety was also assessed.
A total of 919 children underwent randomization; 458 were assigned to receive TAF-emtricitabine, and 461 to receive standard care. Assigned anchor drugs were dolutegravir (229 participants), ritonavir-boosted darunavir (232), ritonavir-boosted atazanavir (231), and ritonavir-boosted lopinavir (227). The median age of participants was 10 years, and 497 (54.1%) were male. The median viral load at baseline was 17,573 copies per milliliter. At week 96, TAF-emtricitabine was superior to standard care: the adjusted difference in the percentage of participants with a viral load of less than 400 copies per milliliter was 6.3 percentage points (95% confidence interval [CI], 2.0 to 10.6; P = 0.004). Dolutegravir was superior to ritonavir-boosted lopinavir and atazanavir analyzed in combination (adjusted difference, 9.7 percentage points; 95% CI, 4.8 to 14.5; P<0.001), but ritonavir-boosted darunavir was not (adjusted difference, 5.6 percentage points; 95% CI, 0.3 to 11.0; P = 0.04 [prespecified threshold, P = 0.03]). Ritonavir-boosted atazanavir was noninferior to ritonavir-boosted lopinavir. One child died, and 29 (3.2%) had serious adverse events, with no significant between-group differences.
Second-line ART regimens including TAF-emtricitabine and dolutegravir were effective for children, with no evidence of safety concerns. Ritonavir-boosted darunavir was also effective. (Funded by the European and Developing Countries Clinical Trials Partnership and others; CHAPAS-4 ISRCTN Registry number, ISRCTN22964075.).
感染人类免疫缺陷病毒(HIV)的儿童接受二线抗逆转录病毒疗法(ART)的选择有限。
在这项采用2×4析因设计的开放标签试验中,我们将一线治疗失败的HIV感染儿童随机分配接受以富马酸替诺福韦艾拉酚胺(TAF)-恩曲他滨或标准治疗(阿巴卡韦或齐多夫定加拉米夫定)为基础,多替拉韦或利托那韦增强的达芦那韦、阿扎那韦或洛匹那韦为锚定药物的二线治疗。主要结局是96周时病毒载量低于每毫升400拷贝。我们假设TAF-恩曲他滨不劣于标准治疗,多替拉韦和利托那韦增强的达芦那韦各自优于联合分析的利托那韦增强的洛匹那韦和阿扎那韦,且利托那韦增强的阿扎那韦不劣于利托那韦增强的洛匹那韦。同时也评估了安全性。
共有919名儿童接受随机分组;458名被分配接受TAF-恩曲他滨,461名接受标准治疗。分配的锚定药物为多替拉韦(229名参与者)、利托那韦增强的达芦那韦(232名)、利托那韦增强的阿扎那韦(231名)和利托那韦增强的洛匹那韦(227名)。参与者的中位年龄为10岁,497名(54.1%)为男性。基线时的中位病毒载量为每毫升17573拷贝。在第96周时,TAF-恩曲他滨优于标准治疗:病毒载量低于每毫升400拷贝的参与者百分比的调整差异为6.3个百分点(95%置信区间[CI],2.0至10.6;P = 0.004)。多替拉韦优于联合分析的利托那韦增强的洛匹那韦和阿扎那韦(调整差异,9.7个百分点;95%CI,4.8至14.5;P<0.001),但利托那韦增强的达芦那韦并非如此(调整差异,5.6个百分点;95%CI,0.3至11.0;P = 0.04[预设阈值,P = 0.03])。利托那韦增强的阿扎那韦不劣于利托那韦增强的洛匹那韦。1名儿童死亡,29名(3.2%)发生严重不良事件,组间无显著差异。
包括TAF-恩曲他滨和多替拉韦的二线ART方案对儿童有效,且无安全问题的证据。利托那韦增强的达芦那韦也有效。(由欧洲和发展中国家临床试验合作组织及其他机构资助;CHAPAS-4国际标准随机对照试验编号,ISRCTN22964075。)