316
Views
0
CrossRef citations to date
0
Altmetric
Meta-analysis

Efficacy/effectiveness and safety of emicizumab prophylaxis of people with hemophilia A: a systematic review and meta-analysis

, ORCID Icon, , , , & show all
Pages 1087-1097 | Received 26 Aug 2023, Accepted 27 Nov 2023, Published online: 13 Dec 2023

References

  • Seaman CD, Xavier F, Ragni MV. Hemophilia A (factor VIII deficiency). Hematol Oncol Clin North Am. 2021;35(6):1117–1129. doi: 10.1016/j.hoc.2021.07.006
  • Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the management of hemophilia, 3rd edition. Haemophilia Internet. 2020 [[cited 2022 Aug 1]];26:1–158. doi: 10.1111/hae.14046
  • Mancuso ME, Bidlingmaier C, Mahlangu JN, et al. The predictive value of factor VIII/factor IX levels to define the severity of hemophilia: communication from the SSC of ISTH. J Thromb Haemost Internet. 2018 [[cited 2023 May 13]];16(10):2106–2110. doi: 10.1111/jth.14257
  • Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost Internet. 2014 [[cited 2022 Aug 12]];12(11):1935–1939. doi: 10.1111/jth.12672
  • Olasupo OO, Lowe MS, Krishan A, et al. Clotting factor concentrates for preventing bleeding and bleeding‐related complications in previously treated individuals with haemophilia a or B. Cochrane Database Of Systematic Reviews [Internet]. 2021;2021(8). doi: 10.1002/14651858.CD014201
  • Chai-Adisaksopha C, Hillis C, Thabane L, et al. A systematic review of definitions and reporting of bleeding outcome measures in haemophilia. Haemophilia Internet. 2015 [[cited 2022 Aug 7]];21(6):731–735. doi: 10.1111/hae.12750
  • Kempton CL, Meeks SL. Toward optimal therapy for inhibitors in hemophilia. Blood Internet. 2014 [[cited 2022 Nov 16]];124(23):3365–3372. doi: 10.1182/blood-2014-05-577643
  • Uchida N, Sambe T, Yoneyama K, et al. A first-in-human phase 1 study of ACE910, a novel factor VIII–mimetic bispecific antibody, in healthy subjects. Blood Internet. 2016 [[cited 2022 Aug 9]];127(13):1633–1641. doi: 10.1182/blood-2015-06-650226
  • Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia a with inhibitors. N Engl J Med. 2017;377(9):809–818. doi: 10.1056/NEJMoa1703068
  • Young G, Liesner R, Chang T, et al. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia a with inhibitors. Blood Internet. 2019;134(24):2127–2138. doi: 10.1182/blood.2019001869
  • Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia a without inhibitors. N Engl J Med Internet. 2018;379(9):811–822. doi: 10.1056/NEJMoa1803550
  • Pipe SW, Shima M, Lehle M, et al. Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia a (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study. Lancet Haematol Internet. 2019;6(6):e295–e305. doi: 10.1016/S2352-3026(19)30054-7
  • Yang R, Wang S, Wang X, et al. Prophylactic emicizumab for hemophilia a in the Asia‐Pacific region: a randomized study (HAVEN 5). Res Pract Thromb Haemost Internet. 2022;6:e12670. doi: 10.1002/rth2.12670
  • Négrier C, Mahlangu J, Lehle M, et al. Emicizumab in people with moderate or mild haemophilia a (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study. Lancet Haematol Internet. 2023;10:e168–e177. doi: 10.1016/S2352-3026(22)00377-5
  • Misgav M, Brutman‐Barazani T, Budnik I, et al. Emicizumab prophylaxis in haemophilia patients older than 50 years with cardiovascular risk factors: Real‐world data. Haemophilia Internet. 2021;27(2):253–260. doi: 10.1111/hae.14261
  • Zharkov PА, Voronin KA, Andreeva TA, et al. Bleeding rate reduction in children with hemophilia a and inhibitors treated with emicizumab in the real-world clinical setting. Pediatr Hematol Immunopathol Internet. 2022;21(1):66–71. doi: 10.24287/1726-1708-2022-21-1-66-71
  • Glonnegger H, Andresen F, Kapp F, et al. Emicizumab in children: bleeding episodes and outcome before and after transition to emicizumab. BMC Pediatr Internet. 2022;22(1):487. doi: 10.1186/s12887-022-03546-1
  • Batt K, Schultz BG, Caicedo J, et al. A real-world study comparing pre-post billed annualized bleed rates and total cost of care among non-inhibitor patients with hemophilia a switching from FVIII prophylaxis to emicizumab. Current Medical Research And Opinion Internet. 2022;38(10):1685–1693. doi: 10.1080/03007995.2022.2105072
  • Liu G, Huang K, Li G, et al. Real-world experience of emicizumab prophylaxis in young children with hemophilia A: retrospective data from China. Front Pediatr Internet. 2022;10:1–7. doi: 10.3389/fped.2022.992267
  • Page MJ, Moher D, Bossuyt PM, et al. PRISMA 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews. BMJ Internet. 2021:n160. doi: 10.1136/bmj.n160
  • Higging J, Thomas J, Chandler J, et al. Cochrane handbook for systematic reviews of interventions version 6.3 (updated February 2022) [Internet]. Cochrane; 2022. www.training.cochrane.org/handbook
  • Landis JR, Koch GG. The measurement of observer agreement for categorical data published by. International Biometric Society Stable https://www.jstor.org/stable/2529310.1977;33:159–174.
  • Rohatgi A WebPlotdigitizer [Internet]. Pacifica (CA USA); 2022. https://automeris.io/WebPlotDigitizer
  • van Balen EC, O’Mahony B, Cnossen MH, et al. Patient‐relevant health outcomes for hemophilia care: development of an international standard outcomes set. Res Pract Thromb Haemost Internet. 2021;5(4):e12488. https://linkinghub.elsevier.com/retrieve/pii/S2475037922013681
  • Chen Y, Cheng SJ, Thornhill T, et al. Health care costs and resource use of managing hemophilia A: a targeted literature review. J Manag Care Spec Pharm Internet. 2023;29(6):647–658. doi: 10.18553/jmcp.2023.29.6.647
  • Sterne JA, Hernán MA, Reeves BC, et al. ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions. BMJ Internet. 2016;355:i4919. https://www.bmj.com/lookup/doi/10.1136/bmj.i4919
  • Guyatt G, Oxman AD, Akl EA, et al. GRADE guidelines: 1. Introduction — GRADE evidence profiles and summary of findings tables. J Clin Epidemiol Internet. 2011 [[cited 2023 Apr 30]];64(4):383–394.doi: 10.1016/j.jclinepi.2010.04.026
  • Shima M, Hanabusa H, Taki M, et al. Factor VIII–mimetic function of humanized bispecific antibody in hemophilia a. N Engl J Med Internet. 2016;374(21):2044–2053. doi: 10.1056/NEJMoa1511769
  • Shima M, Nogami K, Nagami S, et al. A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia a without inhibitors. Haemophilia. 2019;25(6):979–987. doi: 10.1111/hae.13848
  • Oldenburg J, Mahlangu JN, Bujan W, et al. The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia a with inhibitors: HAVEN 1 study. Haemophilia Internet. 2019;25(1):33–44. doi: 10.1111/hae.13618
  • Skinner MW, Négrier C, Paz‐Priel I, et al. The effect of emicizumab prophylaxis on long‐term, self‐reported physical health in persons with haemophilia a without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies. Haemophilia Internet. 2021;27(5):854–865. doi: 10.1111/hae.14363
  • Mannucci PM, Kessler CM, Germini F, et al. Bleeding events in people with congenital haemophilia a without factor VIII inhibitors receiving prophylactic factor VIII treatment: a systematic literature review. Haemophilia Internet. 2023 [[cited 2023 Jul 10]];29(4):954–962. doi: 10.1111/hae.14803
  • Chai-Adisaksopha C, Nevitt SJ, Simpson ML, et al. Bypassing agent prophylaxis in people with hemophilia a or B with inhibitors. Cochrane Database Syst Rev Internet. 2017 [cited 2023 Jul 10];2020(3): doi: 10.1002/14651858.CD011441.pub2
  • López-Fernández MF, Roca CA, Álvarez-Román MT, et al. Spanish consensus guidelines on prophylaxis with bypassing agents in patients with haemophilia and inhibitors. Thromb Haemost Internet. 2016;115(5):872–895. doi: 10.1160/TH15-07-0568
  • Sampei Z, Igawa T, Soeda T, et al. Identification and multidimensional optimization of an asymmetric bispecific IgG antibody mimicking the function of factor VIII cofactor activity) identification and multidimensional optimization of an asymmetric bispecific IgG antibody mimicking the func. PLoS One Internet. 2013 [[cited 2022 Oct 4]];8(2):57479.doi: 10.1371/journal.pone.0057479
  • Euser AM, Zoccali C, Jager KJ, et al. Cohort studies: prospective versus retrospective. Nephron Clin Pract Internet. 2009 [[cited 2023 Jul 10]];113(3):c214–c217.doi: 10.1159/000235241
  • Fischer K, Poonnoose P, Dunn AL, et al. Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective. Haemophilia Internet. 2017;23(1):11–24. doi: 10.1111/hae.13088
  • Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia a. Haemophilia Internet. 2014 [[cited 2023 Jul 10]];20(5):607–615. doi: 10.1111/hae.12438
  • Hartmann R, Feenstra T, Valentino L, et al. In vitro studies show synergistic effects of a procoagulant bispecific antibody and bypassing agents. J Thromb Haemost. 2018;16(8):1580–1591. doi: 10.1111/jth.14203
  • Aledort LM. Deaths associated with emicizumab in patients with hemophilia a. N Engl J Med Internet. 2019 [[cited 2023 May 4]];381(19):1878–1879. doi: 10.1056/NEJMc1909742
  • Makris M, Iorio A, Lenting PJ. Emicizumab and thrombosis: the story so far. J Thromb Haemost. 2019;17(8):1269–1272. doi: 10.1111/jth.14556
  • Linari S, Castaman G. Concomitant use of rFviia and emicizumab in people with hemophilia a with inhibitors: Current perspectives and emerging clinical evidence. Ther Clin Risk Manag Internet. 2020;16:461–469. https://www.dovepress.com/concomitant-use-of-rfviia-and-emicizumab-in-people-with-hemophilia-a-w-peer-reviewed-article-TCRM
  • Kizilocak H, Marquez-Casas E, Malvar J, et al. Safety of FEIBA and emicizumab (SAFE): dose escalation study evaluating the safety of in vivo administration of activated prothrombin complex concentrate in haemophilia a patients on emicizumab. Haemophilia Internet. 2023;29(1):100–105. doi: 10.1111/hae.14684
  • Gundabolu K, Goldsweig A, Bhatt VR, et al. ST‐Segment elevation myocardial infarction (STEMI) and pulmonary embolism in a hemophilia a patient receiving emicizumab and recombinant activated factor VII. Haemophilia Internet. 2020;26(1). doi: 10.1111/hae.13871
  • Maria A, Alessandro C, Declan N, et al. Hemorrhagic and thrombotic adverse events associated with emicizumab and extended half-life factor VIII replacement drugs: EudraVigilance data of 2021. J Thromb Haemost. 2023;21(3):546–552. doi: 10.1016/j.jtha.2023.01.010
  • Schmitt C, Emrich T, Chebon S, et al. Low immunogenicity of emicizumab in persons with haemophilia a. Haemophilia Internet. 2021;27(6):984–992. doi: 10.1111/hae.14398
  • Shea BJ, Reeves BC, Wells G, et al. AMSTAR 2: a critical appraisal tool for systematic reviews that include randomised or non-randomised studies of healthcare interventions, or both. BMJ. 2017;358:1–9. doi: 10.1136/bmj.j4008
  • Gogia P, Tarantino M, Schramm W, et al. New directions to develop therapies for people with hemophilia. Expert Rev Hematol Internet. 2023;16(6):417–433. doi: 10.1080/17474086.2023.2184341
  • Blair HA. Emicizumab : A Review in Haemophilia A. Drugs Internet. 2019;79(15):1697–1707. doi: 10.1007/s40265-019-01200-2
  • Teranishi Y, Soeda T, Koga H, et al. New factor VIII function-mimetic bispecific antibodies engineered from emicizumab for further improving the treatment of hemophilia a. Res Pract Thromb Haemost Internet. 2020;4:579 [[cited 2023 Jun 6]]. Available from: https://abstracts.isth.org/abstract/new-factor-viii-function-mimetic-bispecific-antibodies-engineered-from-emicizumab-for-further-improving-the-treatment-of-hemophilia-a/
  • Østergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia a mice. 2021 [cited 2023 Jun 5]. Available from: http://ashpublications.org/blood/article-pdf/138/14/1258/1827162/bloodbld2020010331.pdf

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.