Cost-effectiveness analysis of low-dose prophylaxis versus on-demand treatment for moderate-to-severe hemophilia A in India

References

• Mandal P, Phukan A, Bhowmik A, et al. Effect of tertiary prophylaxis with low-dose factor VIII in quality of life in adult patients with severe hemophilia A. J Appl Hematol. 2019;10:88. doi:10.4103/joah.joah_37_19
   Google Scholar
• Kar A, Phadnis S, Dharmarajan S, et al. Epidemiology & social costs of haemophilia in India. Indian J Med Res. 2014;140(1):19–31.
   PubMedGoogle Scholar
• Report on the Annual Global Survey 2020 is published by the World Federation of Hemophilia. (2020). https://www1.wfh.org/publications/files/pdf-2045.pdf.
   Google Scholar
• Sachdeva A, Gunasekaran V, Ramya HN, et al. Consensus statement of the Indian academy of pediatrics in diagnosis and management of hemophilia. Indian Pediatr. 2018;55(7):582–590. doi:10.1007/s13312-018-1302-8
   PubMed Web of Science ®Google Scholar
• Goudarzi R, Seraji S, Amiresmaili M, et al. Safety and efficacy of secondary prophylactic in patients with severe hemophilia type A and B: a systematic review and meta-analysis study. Health Technol Assess Action. 2020;3(1):1–7.
   Google Scholar
• Singh A, Mehta S, Goyal LK, et al. Low dose prophylaxis vis-a-vis on-demand treatment strategies for hemophilia: a cost effective and disability attenuating approach. J Assoc Physicians India. 2019;67(11):52–55.
   PubMedGoogle Scholar
• Tang L, Xu W, Li CG, et al. Describing the quality of life of boys with haemophilia in China: results of a multicentre study using the CHO-KLAT. Haemophilia. 2018;24(1):113–119. doi:10.1111/hae.13349
   PubMed Web of Science ®Google Scholar
• Wu R, Sun J, Xiao J, et al. A prospective study of health-related quality of life of boys with severe haemophilia A in China: comparing on-demand to prophylaxis treatment. Haemophilia. 2017;23(3):430–436. doi:10.1111/hae.13198
   PubMed Web of Science ®Google Scholar
• Poon MC, Lee A. Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? Thromb J. 2016;14(Suppl. 1):32. doi:10.1186/s12959-016-0096-y
   PubMed Web of Science ®Google Scholar
• Sidharthan N, Sudevan R. Low dose prophylaxis in hemophilia care. Indian J Hematol Blood Transfus. 2020;36(1):16–25. doi:10.1007/s12288-019-01147-0
   PubMed Web of Science ®Google Scholar
• Verma S, Tripathi A, Sharma G, et al. Low dose long-acting factor VIII prophylaxis in pediatric and young adult patients with hemophilia A: short-term single-center experience from a developing country. J Hematol Allied Sci. 2021;1:75–80. doi:10.25259/JHAS_13_2021
   Google Scholar
• Verma SP, Dutta TK, Mahadevan S, et al. A randomized study of very low-dose factor VIII prophylaxis in severe haemophilia - A success story from a resource limited country. Haemophilia. 2016;22(3):342–348. doi:10.1111/hae.12838
   PubMed Web of Science ®Google Scholar
• Zhao Y, Xiao J, Yang R, et al. Efficacy of standard prophylaxis versus on-demand treatment with Bayer's sucrose-formulated recombinant FVIII (rFVIII-FS) in Chinese children with severe hemophilia A. Pediatr Hematol Oncol. 2017;34(3):146–148. doi:10.1080/08880018.2017.1313921
   Web of Science ®Google Scholar
• Tang L, Wu R, Sun J, et al. Short-term low-dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi-centre pilot study in China. Haemophilia. 2013;19(1):27–34. doi:10.1111/j.1365-2516.2012.02926.x
   PubMed Web of Science ®Google Scholar
• Zahedi Z, Karimi M, Keshavarz K, et al. A cost-effectiveness analysis of the prophylaxis versus on-demand regimens in severe hemophilia A patients under 12 years old in southern Iran. Hematology. 2021;26(1):240–248. doi:10.1080/16078454.2021.1885123
   PubMed Web of Science ®Google Scholar
• Sudevan R, Beenakumari AA, Ganapathy R, et al. Intermediate dose prophylaxis in adults with haemophilia: a clinical audit from a resource limited setting. Indian J Hematol Blood Transfus. 2020;36(2):374–376. doi:10.1007/s12288-019-01189-4
   PubMed Web of Science ®Google Scholar
• Gulshan S, Mandal PK, Phukan A, et al. Is Low dose a New dose to initiate hemophilia A prophylaxis? - A systematic study in eastern India. Indian J Pediatr. 2020;87(5):345–352. doi:10.1007/s12098-019-03179-w
   PubMed Web of Science ®Google Scholar
• Kavakli K, Yang R, Rusen L, et al. Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II). J Thromb Haemost. 2015;13(3):360–369. doi:10.1111/jth.12828
   PubMed Web of Science ®Google Scholar
• Manco-Johnson MJ, Lundin B, Funk S, et al. Effect of late prophylaxis in hemophilia on joint status: a randomized trial. J Thromb Haemost. 2017;15(11):2115–2124. doi:10.1111/jth.13811
   PubMed Web of Science ®Google Scholar
• Debanjana BPK M, Manik M, Tapan Kumar S. Study on secondary versus tertiary prophylaxis in hemophilia children under 12 years of Age in a tertiary care hospital in eastern India. J Indian Med Assoc. 2020;118(12):49–53.
   Google Scholar
• Chozie NA, Primacakti F, Gatot D, et al. Comparison of the efficacy and safety of 12-month low-dose factor VIII tertiary prophylaxis vs on-demand treatment in severe haemophilia A children. Haemophilia. 2019;25(4):633–639. doi:10.1111/hae.13770
   PubMed Web of Science ®Google Scholar
• Ghosh K, Ghosh K. Management of haemophilia in developing countries: challenges and options. Indian J Hematol Blood Transfus. 2016;32(3):347–355. doi:10.1007/s12288-015-0562-x
   PubMed Web of Science ®Google Scholar
• Representation of State-wise AHF Support, Chapters and HTCs as on 1st March 2020. Hemophilia Federation (India); [Available from: https://hemophilia.in/index.php/ahf-status.
   Google Scholar
• Bansal D, Totadri S. Comprehensive hemophilia management in India – miles to Go before We sleep. Indian Pediatr. 2018;55:559–560. doi:10.1007/s13312-018-1433-y
   PubMed Web of Science ®Google Scholar
• Hemophilia Federation (India). [Available from: https://www.hemophilia.in/index.php/hemophilia-treatment-centers.
   Google Scholar
• Singh P, Mukherjee K. Cost-Benefit analysis and assessment of quality of care in patients with hemophilia undergoing treatment at national rural health mission in Maharashtra, India. Value Health Reg Issues. 2017;12:101–106. doi:10.1016/j.vhri.2016.11.003
   PubMedGoogle Scholar
• Ghosh K. Evolution of hemophilia care in India. Indian J Hematol Blood Transfus. 2019;35(4):716–721. doi:10.1007/s12288-018-1059-1
   PubMed Web of Science ®Google Scholar
• Blanchette VS. Prophylaxis in the haemophilia population. Haemophilia. 2010;16:181–188. doi:10.1111/j.1365-2516.2010.02318.x
   PubMed Web of Science ®Google Scholar
• Zhou ZY, Raimundo K, Patel AM, et al. Model of short- and long-term outcomes of Emicizumab prophylaxis treatment for persons with hemophilia A. J Manag Care Spec Pharm. 2020;26(9):1109–1120.
   PubMed Web of Science ®Google Scholar
• National Institute for Health and Care Excellence. (2004). Guide to the methods of technology appraisal. London: National Institute for Health and Care Excellence.
   Google Scholar
• Bank TW. India [Available from: https://data.worldbank.org/country/india.].
   Google Scholar
• Jaramillo HE C, Viscaya M M, Mejia AE. Cost-utility analysis of primary prophylaxis, compared with on-demand treatment, for patients with severe hemophilia type a in Colombia. Int J Technol Assess Health Care. 2016;32(5):337–347. doi:10.1017/S0266462316000544
   PubMed Web of Science ®Google Scholar
• A Guide to Minimum Wage in India in 2021. (2021). Available from: https://www.india-briefing.com/news/guide-minimum-wage-india-2021-19406.html/.
   Google Scholar
• National Cost database. National Health System Cost Database for India; [Available from: https://www.healtheconomics.pgisph.in/costing_web/.
   Google Scholar
• PMJAY. [Available from: https://pmjay.gov.in/sites/default/files/2020-01/HBP_2.0-For_Website_V2.pdf.
   Google Scholar
• Howren MB. Quality-Adjusted life years (QALYs). In: Gellman MD, Turner JR, editor. Encyclopedia of behavioral medicine. New York, NY: Springer New York; 2013. p. 1605–1606.
   Google Scholar
• Wichmann AB, Adang EM, Stalmeier PF, et al. The use of quality-adjusted life years in cost-effectiveness analyses in palliative care: mapping the debate through an integrative review. Palliat Med. 2017;31(4):306–322. doi:10.1177/0269216316689652
   PubMed Web of Science ®Google Scholar
• Mancuso ME, Graca L, Auerswald G, et al. Haemophilia care in children-benefits of early prophylaxis for inhibitor prevention. Haemophilia. 2009;15(Suppl. 1):8–14. doi:10.1111/j.1365-2516.2008.01947.x
   PubMedGoogle Scholar
• Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT study). J Thromb Haemost. 2011;9(4):700–710. doi:10.1111/j.1538-7836.2011.04214.x
   PubMed Web of Science ®Google Scholar
• Mancuso ME, Male C, Kenet G, et al. Prophylaxis in children with haemophilia in an evolving treatment landscape. Haemophilia. 2021;27(6):889–896. doi:10.1111/hae.14412
   PubMed Web of Science ®Google Scholar
• Saxena K. Barriers and perceived limitations to early treatment of hemophilia. J Blood Med. 2013;4:49–56. doi:10.2147/JBM.S43734
   PubMedGoogle Scholar
• Li P, Chen Z, Cheng X, et al. PK-tailored tertiary prophylaxis in patients with severe hemophilia A at Beijing children's hospital. Pediatr Investig. 2019;3(1):45–49. doi:10.1002/ped4.12122
   PubMedGoogle Scholar
• Gringeri A, Lambert T, Street A, et al. Tertiary prophylaxis in adults: is there a rationale? Haemophilia. 2012;18(5):722–728. doi:10.1111/j.1365-2516.2012.02843.x
   PubMed Web of Science ®Google Scholar
• Sidharthan N, Sudevan R, Narayana Pillai V, et al. Low-dose prophylaxis for children with haemophilia in a resource-limited setting in south India-A clinical audit report. Haemophilia. 2017;23(4):e382–e3e4. doi:10.1111/hae.13272
   PubMed Web of Science ®Google Scholar