Omalizumab in the treatment of children with asthma: a rapid health technology assessment_Chinese Journal of Evidence-Based Medicine

Authors：

LIU Jianing ^1,2,3,4,5 , ZENG Linan ^2,3,4 , WANG Lili ⁶ , LI Hailong ^2,3,4 , HUANG Liang ^2,3,4 , CHENG Guo ^4,7,8 ,  YANG Chunsong ^2,3,4 ,  ZHANG Lingli ^1,2,3,4

1. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, P. R. China;
2. Department of Pharmacy/Evidence-Based Pharmacy Center, West China Second University Hospital, Sichuan University; Children's Medicine Key Laboratory of Sichuan Province, Chengdu 610041, P. R. China;
3. NMPA Key Laboratory for Technical Research on Drug Products in vitro and in vivo Correlation, Chengdu 610041, P. R. China;
4. Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu 610041, P. R. China;
5. West China School of Medicine, Sichuan University, Chengdu 610041, P. R. China;
6. Department of Pediatric Pulmonology and Lmmunology, West China Second University Hospital, Sichuan University, Chengdu 610041, P. R. China;
7. Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu 610041, P. R. China;
8. Laboratory of Molecular Translational Medicine, Center for Translational Medicine, Sichuan University, Chengdu 610041, P. R. China;

Corresponding?author：

YANG Chunsong, Email: yangchunsong_123@126.com; ZHANG Lingli, Email: zhanglingli@scu.edu.cn

Keywords：

Omalizumab; Pediatric asthma; Efficacy; Safety; Economics; Health technology assessment

DOI：

10.7507/1672-2531.202405095

Video：

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Objective To evaluate the efficacy, safety, and economics of omalizumab for the treatment of pediatric asthma through a rapid health technology assessment (HTA). Methods The search was conducted on INAHTA website and databases such as PubMed, Embase, Cochrane Library, SinoMed, CNKI, VIP, and WanFang Data from inception to August 2024. Literature screening, quality evaluation, and data extraction were conducted independently by two investigators. An interview was conducted to consult the medication and treatment opinions of doctors and patients to investigate its clinical application. Results A total of 28 articles, including HTA articles (4), SR/Meta analyses (18), and pharmacoeconomics articles (6) were included. Omalizumab could reduce the incidence of clinical exacerbations, decrease the number of asthma attacks/days with asthma symptoms, and improve the quality of life. The results of the safety evaluation showed that omalizumab could reduce the rate of serious adverse events, especially those related to worsening asthma. Foreign pharmacoeconomic studies showed ICER ranging from ￡30 109 to ￡78 009. A pharmacoeconomic study in China found an ICER of $211 217/QALY for omalizumab, which were both above the pre-set thresholds. Conclusion Omalizumab is a treatment for patients who suffer from moderate to severe persistent allergic asthma or moderate to severe allergic asthma. It has been found to improve disease symptoms without increasing serious adverse events. However, it is not considered cost-effective due to its high price.

Citation： LIU Jianing, ZENG Linan, WANG Lili, LI Hailong, HUANG Liang, CHENG Guo, YANG Chunsong, ZHANG Lingli. Omalizumab in the treatment of children with asthma: a rapid health technology assessment. Chinese Journal of Evidence-Based Medicine, 2025, 25(3): 303-310. doi: 10.7507/1672-2531.202405095 Copy

1.	中華醫學會兒科學分會呼吸學組, 《中華兒科雜志》編輯委員會. 兒童支氣管哮喘診斷與防治指南(2016年版). 中華兒科雜志, 2016, 54(3): 167-181.
2.	全國兒科哮喘協作組, 中國疾病預防控制中心環境與健康相關產品安全所. 第三次中國城市兒童哮喘流行病學調查. 中華兒科雜志, 2013, 51(10): 729-735.
3.	Boulet LP, Reddel HK, Bateman E, et al. The global initiative for asthma (GINA): 25 years later. Eur Respir J, 2019, 54(2): 1900598.
4.	National Asthma Education and Prevention Program. Expert panel report 3 (EPR-3): guidelines for the diagnosis and management of asthma-summary report 2007. J Allergy Clin Immunol, 2007, 120(5 Suppl): S94-S138.
5.	楊祎, 姜明燕. 兒童支氣管哮喘藥物治療研究與進展. 系統醫學, 2019, 4(2): 193-195.
6.	Global Initiative For Asthma. Global strategy for asthma management and prevention. 2024.
7.	中國醫藥教育協會慢性氣道疾病專業委員會, 中國哮喘聯盟. 重度哮喘診斷與處理中國專家共識(2024). 中華醫學雜志, 2024, 104(20): 1759-1789.
8.	中華醫學會變態反應分會呼吸過敏學組(籌), 中華醫學會呼吸病學分會哮喘學組. 中國過敏性哮喘診治指南(第一版, 2019年). 中華內科雜志, 2019, 58(9): 636-655.
9.	Arakawa H, Adachi Y, Ebisawa M, et al. Japanese guidelines for childhood asthma 2020. Allergol Int, 2020, 69(3): 314-330.
10.	Holguin F, Cardet JC, Chung KF, et al. Management of severe asthma: a European Respiratory Society/American Thoracic Society guideline. Eur Respir J, 2020, 55(1): 1900588.
11.	Asher I, Pearce N. Global burden of asthma among children. Int J Tuberc Lung Dis, 2014, 18(11): 1269-1278.
12.	Hong J, Bao Y, Chen A, et al. Chinese guidelines for childhood asthma 2016: major updates, recommendations and key regional data. J Asthma, 2018, 55(10): 1138-1146.
13.	Lanier B, Bridges T, Kulus M, et al. Omalizumab for the treatment of exacerbations in children with inadequately controlled allergic (IgE-mediated) asthma. J Allergy Clin Immunol, 2009, 124(6): 1210-1216.
14.	唐密, 張雪艷, 楊燕, 等. 三級醫療機構醫院衛生技術評估實施現況. 中國衛生資源, 2023, 26(3): 281-285.
15.	COOTHA. Omalizumab as add-on therapy to inhaled steroids for asthma. 2004.
16.	NICE. Omalizumab for treating severe persistent allergic asthma. 2013.
17.	NIHR. Omalizumab for the treatment of severe persistent allergic asthma: a systematic review and economic evaluation. 2013.
18.	周華, 車大鈿. 奧馬珠單抗治療兒童重度過敏性哮喘的快速衛生技術評估. 上海醫藥, 2020, 41(21): 13-16, 24.
19.	Lang D, Liu Z, Li D. Safety and tolerability of omalizumab in children with allergic (IgE-Mediated) asthma: a systematic review and meta-analysis. Discov Med, 2023, 35(176): 233-241.
20.	Fenu G, La Tessa A, Calogero C, et al. Severe pediatric asthma therapy: omalizumab-a systematic review and meta-analysis of efficacy and safety profile. Front Pediatr, 2023, 10: 1033511.
21.	Hanania NA, Niven R, Chanez P, et al. Long-term effectiveness and safety of omalizumab in pediatric and adult patients with moderate-to-severe inadequately controlled allergic asthma. World Allergy Organ J, 2022, 15(10): 100695.
22.	Liu L, Zhou P, Wang Z, et al. Efficacy and safety of omalizumab for the treatment of severe or poorly controlled allergic diseases in children: a systematic review and meta-analysis. Front Pediatr, 2022, 10: 851177.
23.	楊琰茗, 任丹陽, 翁稚穎, 等. 奧馬珠單抗治療中-重度過敏性哮喘兒童的文獻分析. 海峽藥學, 2022, 34(7): 113-117.
24.	Busse WW, Szefler SJ, Haselkorn T, et al. Possible protective effect of omalizumab on lung function decline in patients experiencing asthma exacerbations. J Allergy Clin Immunol Pract, 2021, 9(3): 1201-1211.
25.	王亞芹, 樊鵬利, 呂品, 等. 奧馬珠單抗治療兒童及青少年過敏性哮喘的系統評價. 中國藥物評價, 2021, 38(2): 111-114.
26.	姜紅玉, 李娟, 胡文鳳, 等. 奧馬珠單抗治療兒童中重度過敏性哮喘的有效性和安全性系統評價. 中國醫院用藥評價與分析, 2021, 21(9): 1091-1096.
27.	Fu Z, Xu Y, Cai C. Efficacy and safety of omalizumab in children with moderate-to-severe asthma: a meta-analysis. J Asthma, 2021, 58(10): 1350-1358.
28.	Henriksen DP, Bodtger U, Sidenius K, et al. Efficacy of omalizumab in children, adolescents, and adults with severe allergic asthma: a systematic review, meta-analysis, and call for new trials using current guidelines for assessment of severe asthma. Allergy Asthma Clin Immunol, 2020, 16: 49.
29.	Corren J, Kavati A, Ortiz B, et al. Patient-reported outcomes in moderate-to-severe allergic asthmatics treated with omalizumab: a systematic literature review of randomized controlled trials. Curr Med Res Opin, 2018, 34(1): 65-80.
30.	Tortajada-Girbés M, Bousquet R, Bosque M, et al. Efficacy and effectiveness of omalizumab in the treatment of childhood asthma. Expert Rev Respir Med, 2018, 12(9): 745-754.
31.	Corren J, Kavati A, Ortiz B, et al. Efficacy and safety of omalizumab in children and adolescents with moderate-to-severe asthma: a systematic literature review. Allergy Asthma Proc, 2017, 38(4): 250-263.
32.	Rodrigo GJ, Neffen H. Systematic review on the use of omalizumab for the treatment of asthmatic children and adolescents. Pediatr Allergy Immunol, 2015, 26(6): 551-556.
33.	Turner SW, Friend AJ, Okpapi A. Asthma and other recurrent wheezing disorders in children (chronic). BMJ Clin Evid, 2012, 2012.
34.	Milgrom H, Fowler-Taylor A, Vidaurre CF, et al. Safety and tolerability of omalizumab in children with allergic (IgE-mediated) asthma. Curr Med Res Opin, 2011, 27(1): 163-169.
35.	Massanari M, Milgrom H, Pollard S, et al. Adding omalizumab to the therapy of adolescents with persistent uncontrolled moderate--severe allergic asthma. Clin Pediatr (Phila), 2009, 48(8): 859-865.
36.	Chapman KR, Cartier A, Hébert J, et al. The role of omalizumab in the treatment of severe allergic asthma. Can Respir J, 2006, 13(Suppl B): 1B-9B.
37.	Nieto-Cid M, Garriga-Baraut T, Plaza-Martín AM, et al. Cost-effectiveness of omalizumab for the treatment of severe pediatric allergic asthma-results of a real-life study in Spain. Pediatr Allergy Immunol, 2023, 34(4): e13942.
38.	Rodriguez-Martinez CE, Sossa-Brice?o MP, Castro-Rodriguez JA. Cost-utility of omalizumab for the treatment of uncontrolled moderate-to-severe persistent pediatric allergic asthma in a middle-income country. Pediatr Pulmonol, 2021, 56(9): 2987-2996.
39.	Pamuk G, Le Bourgeois M, Abou Taam R, et al. The economic burden of severe asthma in children: a comprehensive study. J Asthma, 2021, 58(11): 1467-1477.
40.	Zhou H, Lu Y, Wu B, et al. Cost-effectiveness of omalizumab for the treatment of inadequately controlled severe allergic asthma in Chinese children. J Asthma, 2020, 57(1): 87-94.
41.	Yoshikawa H, Iwata M, Matsuzaki H, et al. Impact of omalizumab on medical cost of childhood asthma in Japan. Pediatr Int, 2016, 58(5): 425-428.
42.	Faria R, McKenna C, Palmer S. Optimizing the position and use of omalizumab for severe persistent allergic asthma using cost-effectiveness analysis. Value Health, 2014, 17(8): 772-782.
43.	深圳政府在線. 深圳市醫療保障局答奧馬珠單抗報銷. 2023.
44.	國家醫療保障局. 2023年國家醫保藥品目錄調整申報材料(公示版). 2023.

1. 中華醫學會兒科學分會呼吸學組, 《中華兒科雜志》編輯委員會. 兒童支氣管哮喘診斷與防治指南(2016年版). 中華兒科雜志, 2016, 54(3): 167-181.
2. 全國兒科哮喘協作組, 中國疾病預防控制中心環境與健康相關產品安全所. 第三次中國城市兒童哮喘流行病學調查. 中華兒科雜志, 2013, 51(10): 729-735.
3. Boulet LP, Reddel HK, Bateman E, et al. The global initiative for asthma (GINA): 25 years later. Eur Respir J, 2019, 54(2): 1900598.
4. National Asthma Education and Prevention Program. Expert panel report 3 (EPR-3): guidelines for the diagnosis and management of asthma-summary report 2007. J Allergy Clin Immunol, 2007, 120(5 Suppl): S94-S138.
5. 楊祎, 姜明燕. 兒童支氣管哮喘藥物治療研究與進展. 系統醫學, 2019, 4(2): 193-195.
6. Global Initiative For Asthma. Global strategy for asthma management and prevention. 2024.
7. 中國醫藥教育協會慢性氣道疾病專業委員會, 中國哮喘聯盟. 重度哮喘診斷與處理中國專家共識(2024). 中華醫學雜志, 2024, 104(20): 1759-1789.
8. 中華醫學會變態反應分會呼吸過敏學組(籌), 中華醫學會呼吸病學分會哮喘學組. 中國過敏性哮喘診治指南(第一版, 2019年). 中華內科雜志, 2019, 58(9): 636-655.
9. Arakawa H, Adachi Y, Ebisawa M, et al. Japanese guidelines for childhood asthma 2020. Allergol Int, 2020, 69(3): 314-330.
10. Holguin F, Cardet JC, Chung KF, et al. Management of severe asthma: a European Respiratory Society/American Thoracic Society guideline. Eur Respir J, 2020, 55(1): 1900588.
11. Asher I, Pearce N. Global burden of asthma among children. Int J Tuberc Lung Dis, 2014, 18(11): 1269-1278.
12. Hong J, Bao Y, Chen A, et al. Chinese guidelines for childhood asthma 2016: major updates, recommendations and key regional data. J Asthma, 2018, 55(10): 1138-1146.
13. Lanier B, Bridges T, Kulus M, et al. Omalizumab for the treatment of exacerbations in children with inadequately controlled allergic (IgE-mediated) asthma. J Allergy Clin Immunol, 2009, 124(6): 1210-1216.
14. 唐密, 張雪艷, 楊燕, 等. 三級醫療機構醫院衛生技術評估實施現況. 中國衛生資源, 2023, 26(3): 281-285.
15. COOTHA. Omalizumab as add-on therapy to inhaled steroids for asthma. 2004.
16. NICE. Omalizumab for treating severe persistent allergic asthma. 2013.
17. NIHR. Omalizumab for the treatment of severe persistent allergic asthma: a systematic review and economic evaluation. 2013.
18. 周華, 車大鈿. 奧馬珠單抗治療兒童重度過敏性哮喘的快速衛生技術評估. 上海醫藥, 2020, 41(21): 13-16, 24.
19. Lang D, Liu Z, Li D. Safety and tolerability of omalizumab in children with allergic (IgE-Mediated) asthma: a systematic review and meta-analysis. Discov Med, 2023, 35(176): 233-241.
20. Fenu G, La Tessa A, Calogero C, et al. Severe pediatric asthma therapy: omalizumab-a systematic review and meta-analysis of efficacy and safety profile. Front Pediatr, 2023, 10: 1033511.
21. Hanania NA, Niven R, Chanez P, et al. Long-term effectiveness and safety of omalizumab in pediatric and adult patients with moderate-to-severe inadequately controlled allergic asthma. World Allergy Organ J, 2022, 15(10): 100695.
22. Liu L, Zhou P, Wang Z, et al. Efficacy and safety of omalizumab for the treatment of severe or poorly controlled allergic diseases in children: a systematic review and meta-analysis. Front Pediatr, 2022, 10: 851177.
23. 楊琰茗, 任丹陽, 翁稚穎, 等. 奧馬珠單抗治療中-重度過敏性哮喘兒童的文獻分析. 海峽藥學, 2022, 34(7): 113-117.
24. Busse WW, Szefler SJ, Haselkorn T, et al. Possible protective effect of omalizumab on lung function decline in patients experiencing asthma exacerbations. J Allergy Clin Immunol Pract, 2021, 9(3): 1201-1211.
25. 王亞芹, 樊鵬利, 呂品, 等. 奧馬珠單抗治療兒童及青少年過敏性哮喘的系統評價. 中國藥物評價, 2021, 38(2): 111-114.
26. 姜紅玉, 李娟, 胡文鳳, 等. 奧馬珠單抗治療兒童中重度過敏性哮喘的有效性和安全性系統評價. 中國醫院用藥評價與分析, 2021, 21(9): 1091-1096.
27. Fu Z, Xu Y, Cai C. Efficacy and safety of omalizumab in children with moderate-to-severe asthma: a meta-analysis. J Asthma, 2021, 58(10): 1350-1358.
28. Henriksen DP, Bodtger U, Sidenius K, et al. Efficacy of omalizumab in children, adolescents, and adults with severe allergic asthma: a systematic review, meta-analysis, and call for new trials using current guidelines for assessment of severe asthma. Allergy Asthma Clin Immunol, 2020, 16: 49.
29. Corren J, Kavati A, Ortiz B, et al. Patient-reported outcomes in moderate-to-severe allergic asthmatics treated with omalizumab: a systematic literature review of randomized controlled trials. Curr Med Res Opin, 2018, 34(1): 65-80.
30. Tortajada-Girbés M, Bousquet R, Bosque M, et al. Efficacy and effectiveness of omalizumab in the treatment of childhood asthma. Expert Rev Respir Med, 2018, 12(9): 745-754.
31. Corren J, Kavati A, Ortiz B, et al. Efficacy and safety of omalizumab in children and adolescents with moderate-to-severe asthma: a systematic literature review. Allergy Asthma Proc, 2017, 38(4): 250-263.
32. Rodrigo GJ, Neffen H. Systematic review on the use of omalizumab for the treatment of asthmatic children and adolescents. Pediatr Allergy Immunol, 2015, 26(6): 551-556.
33. Turner SW, Friend AJ, Okpapi A. Asthma and other recurrent wheezing disorders in children (chronic). BMJ Clin Evid, 2012, 2012.
34. Milgrom H, Fowler-Taylor A, Vidaurre CF, et al. Safety and tolerability of omalizumab in children with allergic (IgE-mediated) asthma. Curr Med Res Opin, 2011, 27(1): 163-169.
35. Massanari M, Milgrom H, Pollard S, et al. Adding omalizumab to the therapy of adolescents with persistent uncontrolled moderate--severe allergic asthma. Clin Pediatr (Phila), 2009, 48(8): 859-865.
36. Chapman KR, Cartier A, Hébert J, et al. The role of omalizumab in the treatment of severe allergic asthma. Can Respir J, 2006, 13(Suppl B): 1B-9B.
37. Nieto-Cid M, Garriga-Baraut T, Plaza-Martín AM, et al. Cost-effectiveness of omalizumab for the treatment of severe pediatric allergic asthma-results of a real-life study in Spain. Pediatr Allergy Immunol, 2023, 34(4): e13942.
38. Rodriguez-Martinez CE, Sossa-Brice?o MP, Castro-Rodriguez JA. Cost-utility of omalizumab for the treatment of uncontrolled moderate-to-severe persistent pediatric allergic asthma in a middle-income country. Pediatr Pulmonol, 2021, 56(9): 2987-2996.
39. Pamuk G, Le Bourgeois M, Abou Taam R, et al. The economic burden of severe asthma in children: a comprehensive study. J Asthma, 2021, 58(11): 1467-1477.
40. Zhou H, Lu Y, Wu B, et al. Cost-effectiveness of omalizumab for the treatment of inadequately controlled severe allergic asthma in Chinese children. J Asthma, 2020, 57(1): 87-94.
41. Yoshikawa H, Iwata M, Matsuzaki H, et al. Impact of omalizumab on medical cost of childhood asthma in Japan. Pediatr Int, 2016, 58(5): 425-428.
42. Faria R, McKenna C, Palmer S. Optimizing the position and use of omalizumab for severe persistent allergic asthma using cost-effectiveness analysis. Value Health, 2014, 17(8): 772-782.
43. 深圳政府在線. 深圳市醫療保障局答奧馬珠單抗報銷. 2023.
44. 國家醫療保障局. 2023年國家醫保藥品目錄調整申報材料(公示版). 2023.

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