文献综述

贝达喹啉和德拉马尼在治疗儿童耐药结核病中的研究进展

  • 王希羽 综述 ,
  • 许红梅 审校
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  • 重庆医科大学附属儿童医院感染科 国家儿童健康与疾病临床医学研究中心 儿童发育疾病研究教育部重点实验室 儿科学重庆市重点实验室(重庆 400010)

收稿日期: 2022-08-08

  网络出版日期: 2023-05-10

Advances in the study of bedaquiline and delamanid in the treatment of drug-resistant tuberculosis in children

  • Xiyu Reviewer: WANG ,
  • Hongmei Reviser: XU
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  • Department of Infection Children's Hospital of Chongqing Medical University, National Clinical Research Center for Child Health and Disorders, Ministry of Education Key Laboratory of Child Development and Disorders, Chongqing Key Laboratory of Pediatrics, Chongqing 400010, China

Received date: 2022-08-08

  Online published: 2023-05-10

摘要

结核病是全球重大公共卫生问题之一,耐药结核病由于治疗时间长、方案复杂、不良反应多、疗效差,成为实现终结结核病目标的主要障碍之一。近年来,贝达喹啉和德拉马尼逐渐成为有应用前途的新药,目前国内缺乏关于这两种药物在儿童耐药结核病中的研究应用。文章对贝达喹啉和德拉马尼在耐药结核病中的有效性、安全性及儿童中的应用进展进行综述,从而为儿童耐药结核病的治疗构建新的有效方案提供参考及帮助。

本文引用格式

王希羽 综述 , 许红梅 审校 . 贝达喹啉和德拉马尼在治疗儿童耐药结核病中的研究进展[J]. 临床儿科杂志, 2023 , 41(5) : 387 -393 . DOI: 10.12372/jcp.2023.22e1064

Abstract

Tuberculosis (TB) is one of the major global public health concerns, and drug-resistant TB is one of the major obstacles to achieve the goal of ending TB due to the long treatment period, complicated regimens, multiple adverse effects and poor efficacy. In recent years, bedaquiline and delamanid have gradually become promising new drugs, but there is lack of researches on the application of these drugs in children with drug-resistant tuberculosis in China. This article reviews the efficacy and safety of bedaquiline and delamanid in drug-resistant tuberculosis and the progress of their use in children, in order to help construct new and effective regimens for the treatment of drug-resistant tuberculosis in children.

参考文献

[1] World Health Organization. Global tuberculosis raport 2021 [EB/OL]. [2022-08-08]. https://www.who.int/teams/global-tuberculosis-programme/tb-reports/global-tuberculosis-report-2021.
[2] Dodd PJ, Sismanidis C, Seddon JA. Global burden of drug-resistant tuberculosis in children: a mathematical modelling study[J]. Lancet Infect Dis, 2016, 16(10): 1193-1201.
[3] Jenkins HE, Tolman AW, Yuen CM, et al. Incidence of multidrug-resistant tuberculosis disease in children: systematic review and global estimates[J]. Lancet, 2014, 383(9928): 1572-1579.
[4] Jenkins HE, Yuen CM. The burden of multidrug-resistant tuberculosis in children[J]. Int J Tuberc Lung Dis, 2018, 22(5): 3-6.
[5] Tadolini M, Garcia-Prats AJ, D'ambrosio L, et al. Compassionate use of new drugs in children and adolescents with multidrug-resistant and extensively drug-resistant tuberculosis: early experiences and challenges[J]. Eur Respir J, 2016, 48(3): 938-943.
[6] Diacon AH, Pym A, Grobusch MP, et al. Multidrug-resistant tuberculosis and culture conversion with bedaquiline[J]. N Engl J Med, 2014, 371(8): 723-732.
[7] Field SK. Bedaquiline for the treatment of multidrug-resistant tuberculosis: great promise or disappointment?[J]. Ther Adv Chronic Dis, 2015, 6(4): 170-184.
[8] Dhillon J, Andries K, Phillips PP, et al. Bactericidal activity of the diarylquinoline TMC207 against Mycobacterium tuberculosis outside and within cells[J]. Tuberculosis (Edinb), 2010, 90(5): 301-305.
[9] 刘盛盛, 王莲芝, 唐神结. 耐多药和利福平耐药结核病化学治疗研究进展[J]. 中华结核和呼吸杂志, 2020, 43(4): 371-375.
[10] Pym AS, Diacon AH, Tang SJ, et al. Bedaquiline in the treatment of multidrug- and extensively drug-resistant tuberculosis[J]. Eur Respir J, 2016, 47(2): 564-574.
[11] Borisov SE, Dheda K, Enwerem M, et al. Effectiveness and safety of bedaquiline-containing regimens in the treatment of MDR- and XDR-TB: a multicentre study[J]. Eur Respir J, 2017, 49(5):1700387.
[12] Koirala S, Borisov S, Danila E, et al. Outcome of treatment of MDR-TB or drug-resistant patients treated with bedaquiline and delamanid: results from a large global cohort[J]. Pulmonology, 2021, 27(5): 403-412.
[13] Cox E, Laessig K. FDA approval of bedaquiline--the benefit-risk balance for drug-resistant tuberculosis[J]. N Engl J Med, 2014, 371(8): 689-691.
[14] Schnippel K, Ndjeka N, Maartens G, et al. Effect of bedaquiline on mortality in South African patients with drug-resistant tuberculosis: a retrospective cohort study[J]. Lancet Respir Med, 2018, 6(9): 699-706.
[15] Achar J, Hewison C, Cavalheiro AP, et al. Off-label use of bedaquiline in children and adolescents with multidrug-resistant tuberculosis[J]. Emerg Infect Dis, 2017, 23(10): 1711-1713.
[16] Harausz EP, Garcia-Prats AJ, Seddon JA, et al. New and repurposed drugs for pediatric multidrug-resistant tuberculosis. Practice-based recommendations[J]. Am J Respir Crit Care Med, 2017, 195(10): 1300-1310.
[17] Gubkina MF, Khokhlova JY, Yukhimenko NV, et al. Prolonged use of bedaquiline in the treatment for MDR-TB in a child[J]. IDCases, 2021, 26: e01311.
[18] Hughes JA, Solans BP, Draper HR, et al. Pharmacokinetics and safety of bedaquiline in HIV-positive and negative older children and adolescents with rifampicin-resistant tuberculosis[J]. Clin Infect Dis, 2022, 75(10):1772-1780.
[19] Solodovnikova V, Kumar AMV, Hurevich H, et al. Effectiveness and safety of delamanid- or bedaquiline-containing regimens among children and adolescents with multidrug resistant or extensively drug resistant tuberculosis: a nationwide study from Belarus, 2015-19[J]. Monaldi Arch Chest Dis, 2021, 91(1). doi: 10.4081/monaldi.2021.1646.
[20] Das M, Mamnoon F, Mansoor H, et al. New TB drugs for the treatment of children and adolescents with rifampicin-resistant TB in Mumbai, India[J]. Int J Tuberc Lung Dis, 2020, 24(12): 1265-1271.
[21] Wu HY, Tian Y, Wang XD, et al. Twenty-four-week interim outcomes of bedaquiline-containing regimens in treatment of adolescents with rifampicin-resistant tuberculosis: a retrospective cohort study in China[J]. J Paediatr Child Health, 2022, 58(1): 116-121.
[22] Moodliar R, Aksenova V, Frias MVG, et al. Bedaquiline for multidrug-resistant TB in paediatric patients[J]. Int J Tuberc Lung Dis, 2021, 25(9): 716-724.
[23] Evaluating the pharmacokinetics, safety, and tolerability of bedaquiline in HIV-infected and HIV-uninfected infants, children, and adolescents with multidrug-resistant tuberculosis[EB/OL]. [2022-08-08]. https://clinicaltrials.gov/ct2/show/NCT02906007.
[24] Pontali E, Sotgiu G, Tiberi S, et al. Cardiac safety of bedaquiline: a systematic and critical analysis of the evidence[J]. Eur Respir J, 2017, 50(5):1701462.
[25] Brust JCM, Gandhi NR, Wasserman S, et al. Effectiveness and Cardiac safety of bedaquiline-based therapy for drug-resistant tuberculosis: a prospective cohort study[J]. Clin Infect Dis, 2021, 73(11): 2083-2092.
[26] Marais BJ. Newer drugs for tuberculosis prevention and treatment in children[J]. Indian J Pediatr, 2019, 86(8): 725-731.
[27] 抗结核新药贝达喹啉临床应用专家共识(2020年更新版)[J]. 中华结核和呼吸杂志, 2021, 44(2): 81-87.
[28] World Health Organization. The use of bedaquiline in the treatment of multidrug-resistant tuberculosis: interim guidance[M/OL]. [2022-08-08]. Geneva: World Health Organization, 2013. https://apps.who.int/iris/bitstream/handle/10665/84879/9789241505482_eng.pdf.
[29] Provisional CDC guidelines for the use and safety monitoring of bedaquiline fumarate (Sirturo) for the treatment of multidrug-resistant tuberculosis[J]. MMWR Recomm Rep, 2013, 62(Rr-09): 1-12.
[30] Rapid communication: key changes to treatment of multidrug- and rifampicin-resistant tuberculosis (MDR/RR-TB)[EB/OL]. [2022-08-08]. https://www.who.int/publications/i/item/WHO-CDS-TB-2018.18.
[31] World Health Organization. WHO consolidated guidelines on drug-resistant tuberculosis treatment 2019[M/OL]. [2022-08-08]. Geneva, World Health Organization, 2019. https://www.who.int/publications/i/item/9789241550529.
[32] Liu Y, Matsumoto M, Ishida H, et al. Delamanid: from discovery to its use for pulmonary multidrug-resistant tuberculosis (MDR-TB)[J]. Tuberculosis (Edinb), 2018, 111: 20-30.
[33] Gler MT, Skripconoka V, Sanchez-Garavito E, et al. Delamanid for multidrug-resistant pulmonary tuberculosis[J]. N Engl J Med, 2012, 366(23): 2151-2160.
[34] Skripconoka V, Danilovits M, Pehme L, et al. Delamanid improves outcomes and reduces mortality in multidrug-resistant tuberculosis[J]. Eur Respir J, 2013, 41(6): 1393-400.
[35] Blair HA, Scott LJ. Delamanid: a review of its use in patients with multidrug-resistant tuberculosis[J]. Drugs, 2015, 75(1): 91-100.
[36] Gupta R, Geiter LJ, Wells CD, et al. Delamanid for extensively drug-resistant tuberculosis[J]. N Engl J Med, 2015, 373(3): 291-292.
[37] Von Groote-Bidlingmaier F, Patientia R, Sanchez E, et al. Efficacy and safety of delamanid in combination with an optimised background regimen for treatment of multidrug-resistant tuberculosis: a multicentre, randomised, double-blind, placebo-controlled, parallel group phase 3 trial[J]. Lancet Respir Med, 2019, 7(3): 249-259.
[38] Esposito S, D'ambrosio L, Tadolini M, et al. ERS/WHO Tuberculosis Consilium assistance with extensively drug-resistant tuberculosis management in a child: case study of compassionate delamanid use[J]. Eur Respir J, 2014, 44(3): 811-815.
[39] Esposito S, Bosis S, Tadolini M, et al. Efficacy, safety, and tolerability of a 24-month treatment regimen including delamanid in a child with extensively drug-resistant tuberculosis: a case report and review of the literature[J]. Medicine (Baltimore), 2016, 95(46): e5347.
[40] Ghosh S, Breitscheidel L, Lazarevic N, et al. Com-passionate use of delamanid in adults and children for drug-resistant tuberculosis: 5-year update[J]. Eur Respir J, 2021, 57(5): 2002483.
[41] European Medicines Agency, Amsterdam, The Netherlands. Deltyba European public assessment report (EPAR)[EB/OL]. [2022-08-08]. https://www.ema.europa.eu/en/documents/variation-report/deltyba-h-c-2552-ii-0040-epar-assessment-report-variation_en.pdf.
[42] European Medicines Agency, Amsterdam, The Netherlands. Deltyba European public assessment report (EPAR)[EB/OL]. [2022-08-08]. https://www.ema.europa.eu/en/documents/variation-report/deltyba-h-c-002552-x-0046-g-epar-assessment-report-variation_en.pdf.
[43] Garcia-Prats AJ, Frias M, Van Der Laan L, et al. Delamanid added to an optimized background regimen in children with multidrug-resistant tuberculosis: results of a phase I/II clinical trial[J]. Antimicrob Agents Chemother, 2022, 66(5): e0214421.
[44] Pharmacokinetic and safety trial to determine the appropriate dose for pediatric patients with multidrug resistant tuberculosis[EB/OL]. [2022-08-08]. https://clinicaltrials.gov/ct2/show/NCT01856634.
[45] A 6-month safetyefficacy, and pharmacokinetic (PK) trial of delamanid in pediatric participants with multidrug resistant tuberculosis (MDR-TB)[EB/OL]. [2022-08-08]. https://clinicaltrials.gov/ct2/show/NCT01859923.
[46] Evaluating the Pharmacokinetics, Safety, and Tolerability of Delamanid in Combination With Optimized Multidrug Background Regimen (OBR) for Multidrug-Resistant Tuberculosis (MDR-TB) in HIV-Infected and HIV-Uninfected Children With MDR-TB[EB/OL]. [2022-08-08]. https://clinicaltrials.gov/ct2/show/NCT03141060.
[47] Ryan N, Lo J. Delamanid: first global approval[J]. Drugs, 2014, 74(9): 1041-1045.
[48] Lewis JM, Sloan DJ. The role of delamanid in the treatment of drug-resistant tuberculosis[J]. Ther Clin Risk Manag, 2015, 11: 779-791.
[49] Sasaki T, Svensson EM, Wang X, et al. Population Pharmacokinetic and concentration-QTc analysis of delamanid in pediatric participants with multidrug-resistant tuberculosis[J]. Antimicrob Agents Chemother, 2022, 66(2): e0160821.
[50] World Health Organization. The use of delamanid in the treatment of multidrug-resistant tuberculosis in children and adolescents: interim policy guidance[M/OL]. [2022-08-08]. Geneva: World Health Organization, 2016. https://www.who.int/publications/i/item/9789241549899.
[51] World Health Organization. WHO consolidated guidelines on drug-resistant tuberculosis treatment[M/OL]. [2022-08-08]. Geneva: World Health Organization, 2016. https://www.who.int/publications/i/item/9789241550529
[52] World Health Organization. WHO consolidated guidelines on tuberculosis: Module 5: Management of tuberculosis in children and adolescents[M/OL]. [2022-08-08]. Geneva: World Health Organization, 2016. https://www.who.int/publications/i/item/9789240046764.
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