Anti-factor VIII autoantibody eradication in acquired hemophilia A: focused strategies for resource-constrained settings

Ma AD, Carrizosa D. Acquired factor VIII inhibitors: pathophysiology and treatment. Hematol Am Soc Hematol Educ Program. 2006;2006:432-7.

Kruse-Jarres R, Kempton CL, Baudo F, Collins PW, Knoebl P, Leissinger CA, et al. Acquired hemophilia A: updated review of evidence and treatment guidance. Am J Hematol. 2017;92:695-705.

Tiede A, Collins P, Knoebl P, Teitel J, Kessler C, Shima M, et al. International recommendations on the diagnosis and treatment of acquired hemophilia A. Haematologica. 2020;105:1791-801.

Hay CR, Negrier C, Ludlam CA. The treatment of bleeding in acquired haemophilia with recombinant factor VIIa: a multicentre study. Thromb Haemost. 1997;78:1463-7.

Tiede A, Worster A. Lessons from a systematic literature review of the effectiveness of recombinant factor VIIa in acquired haemophilia. Ann Hematol. 2018;97:1889-901.

Sallah S. Treatment of acquired haemophilia with factor eight inhibitor bypassing activity. Haemophilia. 2004;10:169-73.

Baudo F, Collins P, Huth-Kühne A, Lévesque H, Marco P, Nemes L, et al. Management of bleeding in acquired hemophilia A: results from the European Acquired Haemophilia (EACH2) Registry. Blood. 2012;120:39-46.

Kruse-Jarres R, St-Louis J, Greist A, Shapiro A, Smith H, Chowdary P. et al. Efficacy and safety of OBI-1, an antihaemophilic factor VIII (recombinant), porcine sequence, in subjects with acquired haemophilia A. Haemophilia. 2015;21:162-70.

Tiede A, Kemkes-Matthes B, Knöbl P. Should emicizumab be used in patients with acquired hemophilia A? J Thromb Haemost. 2021;19:637-44.

Collins PW, Hirsch S, Baglin TP, Dolan G, Hanley J, Makris M, et al. Acquired hemophilia A in the United Kingdom: a 2-year national surveillance study by the United Kingdom Haemophilia Centre Doctors’ Organisation. Blood. 2007;109:1870-7.

Collins P, Baudo F, Knoebl P, Lévesque H, Nemes L, Pellegrini F, et al. Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2). Blood. 2012;120:47-55.

Schep SJ, van Dijk WEM, Beckers EAM, Meijer K, Coppens M, Eikenboom J, et al. Treatment of acquired hemophilia A, a balancing act: results from a 27-year Dutch cohort study. Am J Hematol. 2021;96:51-9.

Mingot-Castellano ME, Pardos-Gea J, Haya S, Bastida-Bermejo JM, Tàssies D, Marco-Rico A, et al. Management of acquired hemophilia A: results from the Spanish registry. Blood Adv. 2021;5:3821-9.

Tiede A, Klamroth R, Scharf RE, Trappe RU, Holstein K, Huth- Kühne A, et al. Prognostic factors for remission of and survival in acquired hemophilia A (AHA): results from the GTH-AH 01/2010 study. Blood. 2015;125:1091-7.

Sun B, Xue F, Feng Y, Sun J, Yu Z, Hou M, et al. Outcome of CARE: a 6-year national registry of acquired haemophilia A in China. Br J Haematol. 2019;187:653-65.

Wongrakpanich S, Uaprasert N, Akkawat B, Nanakhon T, Rojnuckarin P. Acquired factor VIII inhibitor in King Chulalongkorn Memorial Hospital. J Hematol Transfus Med. 2012;22:195-201.

Chai-Adisaksopha C, Rattarittamrong E, Norasetthada L, Tantiworawit A, Nawarawong W. Younger age at presentation of acquired haemophilia A in Asian countries: a single-centre study and systematic review. Haemophilia. 2014;20:e205-10.

Rungjirajittranon T, Suwanawiboon B, Nakkinkun Y, Leelakanok N, Kaokunakorn T, Chinthammitr Y, et al. First-line immunosuppressive therapies for acquired hemophilia A: a 25-year cohort experience and network meta-analysis. Thromb Res. 2024;241:109067.

Borg JY, Guillet B, Le Cam-Duchez V, Goudemand J, Lévesque H, SACHA Study Group. Outcome of acquired haemophilia in France: the prospective SACHA (Surveillance des Auto antiCorps au course de l’Hémophilie Acquise) registry. Haemophilia. 2013;19:564-70.

Sperr WR, Lechner K, Pabinger I. Rituximab for the treatment of acquired antibodies to factor VIII. Haematologica. 2007;92:66-71.

Lévesque H, Viallard JF, Houivet E, Bonnotte B, Voisin S, Le Cam-Duchez V, et al. Cyclophosphamide vs rituximab for eradicating inhibitors in acquired hemophilia A: a randomized trial in 108 patients. Thromb Res. 2024;237:79-87.

Wang P, Zhou R, Xue F, Zhou H, Bai J, Wang X, et al. Single-dose rituximab plus glucocorticoid versus cyclophosphamide plus glucocorticoid in patients with newly diagnosed acquired hemophilia A: a multicenter, open-label, randomized noninferiority trial. Am J Hematol. 2024;99:28-37.

Simon B, Ceglédi A, Dolgos J, Farkas P, Gaddh M, Hankó L, et al. Combined immunosuppression for acquired hemophilia A: CyDRi is a highly effective low-toxicity regimen. Blood. 2022;140:1983-92.

Dobbelstein C, Moschovakis GL, Tiede A. Reduced-intensity, risk factor-stratified immunosuppression for acquired hemophilia A: single-center observational study. Ann Hematol. 2020;99:2105-12.

Ratnasingam S, Walker PA, Tran H, Kaplan ZS, McFadyen JD, Tran H, et al. Bortezomib-based antibody depletion for refractory autoimmune hematological diseases. Blood Adv. 2016;1:31-5.

McFadyen JD, Tran H, Kaplan ZS. Factor VIII inhibitor eradication with bortezomib in acquired haemophilia A. Br J Haematol. 2017;178:986-7.

Cai H, Cao X, Wang S, Zhao Y, Li J, Zhu TN. Rituximab and bortezomib for patients with newly diagnosed acquired hemophilia: single arm, single center, prospective phase 2 study. Blood. 2022;140(Suppl 1):2715-6.

Markmann C, Bhoj VG. On the road to eliminating long-lived plasma cells- “are we there yet?” Immunol Rev. 2021;303:154-67.

Liu W, Liu J, Xue F, Yang R, Zhang L. Anti-CD38 antibody for refractory acquired hemophilia A. J Thromb Haemost. 2023;21:1515-8.

Moonla C, Polprasert C, Krittikarux S, Cheerasiri N, Akkawat B, Rojnuckarin P, Uaprasert N. Adjunctive daratumumab can induce

rapid remission in acquired haemophilia A with poor prognostic markers. Haemophilia. 2023;29:1649-52.

Allison AC, Eugui EM. Mycophenolate mofetil and its mechanisms of action. Immunopharmacology. 2000;47(2-3):85-118.

Obaji S, Rayment R, Collins PW. Mycophenolate mofetil as adjunctive therapy in acquired haemophilia A. Haemophilia. 2019;25:e59-e65.

Freedman J, Rand ML, Russell O, Davis C, Cheatley PL, Blanchette V, Garvey MB. Immunoadsorption may provide a cost-effective

approach to management of patients with inhibitors to FVIII. Transfusion. 2003;43:1508-13.

Zeitler H, Ulrich-Merzenich G, Hess L, Konsek E, Unkrig C, Walger P, et al. Treatment of acquired hemophilia by the Bonn-Malmo

Protocol: documentation of an in vivo immunomodulating concept. Blood. 2005;105:2287-93.

Callaghan MU, Negrier C, Paz-Priel I, Chang T, Chebon S, Lehle M, et al. Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood. 2021;137:2231-42.

Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU Young G, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377:809-18.

Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, et al. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019;134:2127-38.

Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, et al. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018;379:811-22.

Pipe SW, Shima M, Lehle M, Shapiro A, Chebon S, Fukutake K, 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. 2019;6:e295-e305.

Yang R, Wang S, Wang X, Sun J, Chuansumrit A, Zhou J, et al. Prophylactic emicizumab for hemophilia A in the Asia-Pacific region: a randomized study (HAVEN 5). Res Pract Thromb Haemost. 2022;6:e12670.

Négrier C, Mahlangu J, Lehle M, Chowdary P, Catalani O, Bernardi RJ, et al. Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study. Lancet Haematol. 2023;10:e168-77.

Knoebl P, Thaler J, Jilma P, Quehenberger P, Gleixner K, Sperr WR. Emicizumab for the treatment of acquired hemophilia A. Blood. 2021;137:410-9.

Shima M, Amano K, Ogawa Y, Yoneyama K, Ozaki R, Kobayashi R, et al. A prospective, multicenter, open-label phase III study of emicizumab prophylaxis in patients with acquired hemophilia A. J Thromb Haemost. 2023;21:534-45.

Tiede A, Hart C, Knöbl P, Greil R, Oldenburg J, Sachs UJ, et al. Emicizumab prophylaxis in patients with acquired haemophilia A (GTH-AHA-EMI): an open-label, single-arm, multicentre, phase 2 study. Lancet Haematol. 2023;10:e913-21.

Hart C, Klamroth R, Sachs UJ, Greil R, Knoebl P, Oldenburg J, et al. Emicizumab versus immunosuppressive therapy for the management of acquired hemophilia A. J Thromb Haemost. 2024: S1538-7836(24)00368-4. doi: 10.1016/j.jtha.2024.06.010. [Epub

ahead of print]

Moonla C, Sosothikul D, Pongtanakul B, Suwanawiboon B, Traivaree C, Natesirinilkul R, et al. Practices and challenges for hemophilia

management under resource constraints in Thailand. Orphanet. J Rare Dis. 2023;18:110.

Sosothikul D, Moonla C. Paradigm shifts in haemophilia A therapy with emicizumab prophylaxis in Asia. Ann Acad Med Singap.

;52:556-8.

Chuansumrit A, Sirachainan N, Jaovisidha S, Jiravichitchai T, Kadegasem P, Kempka K, et al. Effectiveness of monthly low dose emicizumab prophylaxis without 4-week loading doses among patients with haemophilia A with and without inhibitors: a case

series report. Haemophilia. 2023;29:382-5.

Kessakorn N, Srichumpuang C, Moonla C, Sosothikul D. Low-dose emicizumab versus low-dose factor VIII secondary prophylaxis for noninhibitor hemophilia A patients with severe bleeding phenotype. Paper presented at: The International Society on Thrombosis and Haemostasis 2024 Congress; 2024 Jun 22-26; Bangkok, Thailand.

Shima M, Nagao A, Taki M, Matsushita T, Oshida K, Amano K, et al. Long-term safety and efficacy of emicizumab for up to 5.8 years and patients’ perceptions of symptoms and daily life: a phase 1/2 study in patients with severe haemophilia A. Haemophilia. 2021;27:81-9.

Lehtinen AE, Lassila R. Do we need all that emicizumab? Haemophilia. 2022;28:e53-5.

Tang ASO, Ko CT, Saari Z, Selvaratnam V, Chew LP, Sathar J. Efficacy of reduced-dose emicizumab in hemophilia A with inhibitors: real world experience in Malaysia. Hemasphere. 2023;7(Suppl 3):e269464e.

Bansal S, Donners AAMT, Fischer K, Kshirsagar S, Rangarajan S, Phadke V, et al. Low dose emicizumab prophylaxis in haemophilia A patients: a pilot study from India. Haemophilia. 2023;29:931-4.

Patil R, Shanmukhaiah C, Gogtay NJ, Pandey P, Patil K, Jijina F, Madkaikar M. Low-dose emicizumab prophylaxis in patients with

severe hemophilia A: a retrospective study bringing new hope for our patients. J Thromb Haemost. 2024;22:1024-30.

Mannucci PM, Hermans C. Low-dose emicizumab for more equitable access to prophylaxis in resource limited countries. Haemophilia. 2024;30:575-6.

Pfrepper C, Klamroth R, Oldenburg J, Holstein K, Eichler H, Hart C, et al. Emicizumab for the treatment of acquired hemophilia A: consensus recommendations from the GTH-AHA Working Group. Hamostaseologie. 2023. doi: 10.1055/a-2197-9738. [Epub

ahead of print]