Research & Literature

Clinical and laboratory approaches to hemophilia a.

Mansouritorghabeh H

Iranian journal of medical sciences 2015; (40(3)):194-205.

Current challenges in the management of hemophilia.

Bauer KA

The American journal of managed care 2015; (21(6 Suppl)):S112-22.

First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study.

Kreuz W, Escuriola Ettingshausen C, Vdovin V, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2016; (22(1)):87-95 doi:10.1111/hae.12774.

Inhibitor recurrence after immune tolerance induction: a multicenter retrospective cohort study.

Antun A, Monahan PE, Manco-Johnson MJ, et al.

Journal of thrombosis and haemostasis : JTH 2015; (13(11)):1980-8 doi:10.1111/jth.13143.

The factor VIII protein and its function.

Mazurkiewicz-Pisarek A, Płucienniczak G, Ciach T, Płucienniczak A

Acta biochimica Polonica 2016; (63(1)):11-16.

Releasing the brakes in coagulation Factor IXa by co-operative maturation of the substrate-binding site.

Kristensen LH, Olsen OH, Blouse GE, Brandstetter H

The Biochemical journal 2016; (473(15)):2395-411 doi:10.1042/BCJ20160336.

Aspects of prophylactic treatment of hemophilia.

Ljung R

Thrombosis journal 2016; (14(Suppl 1)):30 doi:10.1186/s12959-016-0103-3.

Pathophysiology of hemophilic arthropathy and potential targets for therapy.

Pulles AE, Mastbergen SC, Schutgens RE, et al.

Pharmacological research 2017; (115()):192-199 doi:10.1016/j.phrs.2016.11.032.

Impact of mild to severe hemophilia on engagement in recreational activities by US men, women, and children with hemophilia B: The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study.

Baumann K, Hernandez G, Witkop M, et al.

European journal of haematology 2017; (98 Suppl 86()):25-34 doi:10.1111/ejh.12852.

Acquired hemophilia A: Updated review of evidence and treatment guidance.

Kruse-Jarres R, Kempton CL, Baudo F, et al.

American journal of hematology 2017; (92(7)):695-705 doi:10.1002/ajh.24777.

Bleeding risk assessment in hemophilia A carriers from Dakar, Senegal.

Seck M, Faye BF, Sall A, et al.

Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis 2017; (28(8)):642-645 doi:10.1097/MBC.0000000000000653.

Emicizumab, a bispecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII?

Lenting PJ, Denis CV, Christophe OD

Blood 2017; (130(23)):2463-2468 doi:10.1182/blood-2017-08-801662.

Emergency Department Presentation of Iliopsoas Hematoma in a Severe Hemophiliac.

Burgess A, Douglas D, Grubish L

The American journal of emergency medicine 2018; (36(3)):529.e3-529.e4 doi:10.1016/j.ajem.2017.12.035.

Recombinant factor VIII Fc fusion protein for immune tolerance induction in patients with severe haemophilia A with inhibitors-A retrospective analysis.

Carcao M, Shapiro A, Staber JM, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2018; (24(2)):245-252 doi:10.1111/hae.13413.

[Acquired hemophilia A associated with chronic myelomonocytic leukemia successfully treated by rituximab and azacitidine].

Dartevel A, Aubanel S, Pica GM, et al.

La Revue de medecine interne 2018; (39(8)):654-657 doi:10.1016/j.revmed.2018.03.373.

Mimicking Factor VIII to Manage the Factor VIII-Deficient State.

Ragni MV

The New England journal of medicine 2018; (379(9)):880-882 doi:10.1056/NEJMe1808789.

An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B.

Machin N, Ragni MV

Journal of blood medicine 2018; (9()):135-140 doi:10.2147/JBM.S159297.

Acute Life-Threatening Hemorrhage in Neonates With Severe Hemophilia A: A Report of 3 Cases.

Moreira A, Das H

Journal of investigative medicine high impact case reports 2018; (6()):2324709618800349 doi:10.1177/2324709618800349.

The role of emicizumab, a bispecific factor IXa- and factor X-directed antibody, for the prevention of bleeding episodes in patients with hemophilia A.

Knight T, Callaghan MU

Therapeutic advances in hematology 2018; (9(10)):319-334 doi:10.1177/2040620718799997.

Evaluating the safety of emicizumab in patients with hemophilia A.

Langer AL, Etra A, Aledort L

Expert opinion on drug safety 2018; (17(12)):1233-1237 doi:10.1080/14740338.2019.1551356.

Acquired Hemophilia A: A Case Report.

Shen M, Wang S, Sessa J, et al.

Journal of pharmacy practice 2020; (33(4)):562-566 doi:10.1177/0897190019826474.

Compartment syndrome of the forearm with life-threatening bleeding after fasciotomy as the presenting sign of postpartum acquired hemophilia A: a case report.

Van Laer M, Penaloza A, Stockman W, et al.

Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis 2019; (30(3)):120-126 doi:10.1097/MBC.0000000000000799.

Timely and large dose of clotting factor IX provides better joint wound healing after hemarthrosis in hemophilia B mice.

He P, Zhang F, Zhong C, et al.

International journal of hematology 2019; (110(1)):59-68 doi:10.1007/s12185-019-02639-5.

[Mild hemophilia A diagnosed in an adult patient on the occasion of post-operative bleeding following a second knee surgery].

Nakagawa N, Fujinaga M, Chikagawa Y, et al.

[Rinsho ketsueki] The Japanese journal of clinical hematology 2019; (60(5)):398-400 doi:10.11406/rinketsu.60.398.

Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A.

van den Berg HM, Fischer K, Carcao M, et al.

Blood 2019; (134(3)):317-320 doi:10.1182/blood.2019000658.

Hemophilia A with a Rare Presentation of Hemarthrosis and Arthropathy Involving Multiple Joints in a Young Male Child.

Majid Z, Tahir F, Qadar LT, et al.

Cureus 2019; (11(4)):e4524 doi:10.7759/cureus.4524.

Hemophilia A with inhibitor: Immune tolerance induction (ITI) in the mirror of time.

Nakar C, Shapiro A

Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis 2019; (58(5)):578-589 doi:10.1016/j.transci.2019.08.008.

Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors.

Ebbert PT, Xavier F, Seaman CD, Ragni MV

Haemophilia : the official journal of the World Federation of Hemophilia 2020; (26(1)):41-46 doi:10.1111/hae.13877.

Management of intracranial hemorrhage in hemophilia A patients.

Aras M, Oral S

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery 2020; (36(9)):2041-2046 doi:10.1007/s00381-020-04528-3.

International recommendations on the diagnosis and treatment of acquired hemophilia A.

Tiede A, Collins P, Knoebl P, et al.

Haematologica 2020; (105(7)):1791-1801 doi:10.3324/haematol.2019.230771.

Cardiac surgery in patients with Hemophilia:is it safe?

Shalabi A, Kachel E, Kogan A, et al.

Journal of cardiothoracic surgery 2020; (15(1)):76 doi:10.1186/s13019-020-01123-0.

Laboratory issues in gene therapy and emicizumab.

Bowyer AE, Lowe AE, Tiefenbacher S

Haemophilia : the official journal of the World Federation of Hemophilia 2021; (27 Suppl 3()):142-147 doi:10.1111/hae.13976.

Concomitant Use of rFVIIa and Emicizumab in People with Hemophilia A with Inhibitors: Current Perspectives and Emerging Clinical Evidence.

Linari S, Castaman G

Therapeutics and clinical risk management 2020; (16()):461-469 doi:10.2147/TCRM.S205310.

ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors: Evidence from a PedNet Study.

van den Berg HM, Mancuso ME, Königs C, et al.

Thrombosis and haemostasis 2020; (120(8)):1166-1172 doi:10.1055/s-0040-1713097.

Synergistic Effect of Bypassing Agents and Sequence Identical Analogue of Emicizumab and Fibrin Clot Structure in the In Vitro Model of Hemophilia A.

Zong Y, Antovic A, Soutari NMH, et al.

TH open : companion journal to thrombosis and haemostasis 2020; (4(2)):e94-e103 doi:10.1055/s-0040-1710032.

European principles of inhibitor management in patients with haemophilia: implications of new treatment options.

Hermans C, Giangrande PLF, O'Mahony B, et al.

Orphanet journal of rare diseases 2020; (15(1)):219 doi:10.1186/s13023-020-01511-8.

Successful intraosseous factor VIII application in a haemophilic emergency.

Banchev A, Stoyanova D, Avramova B, et al.

Journal of clinical pharmacy and therapeutics 2021; (46(1)):212-214 doi:10.1111/jcpt.13252.

Primary prophylaxis was associated with lower arthropathy in Colombian men with haemophilia B: A longitudinal analysis (2015-2019).

Hernández Vargas JA, Linares A, Solano MH, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2020; (26(6)):e282-e290 doi:10.1111/hae.14136.

Mutation analysis in the F8 gene in 485 families with haemophilia A and prenatal diagnosis in China.

Feng Y, Li Q, Shi P, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2021; (27(1)):e88-e92 doi:10.1111/hae.14206.

Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A.

Long BR, Veron P, Kuranda K, et al.

Molecular therapy : the journal of the American Society of Gene Therapy 2021; (29(2)):597-610 doi:10.1016/j.ymthe.2020.12.008.

A SINE Insertion in F8 Gene Leads to Severe Form of Hemophilia A in a Family of Rhodesian Ridgebacks.

Kehl A, Haaland AH, Langbein-Detsch I, Mueller E

Genes 2021; (12(2)) doi:10.3390/genes12020134.

Key questions in the new hemophilia era: update on concomitant use of FVIII and emicizumab in hemophilia A patients with inhibitors.

Carcao M, Mancuso ME, Young G, Jiménez-Yuste V

Expert review of hematology 2021; (14(2)):143-148 doi:10.1080/17474086.2021.1875817.

Case - Acquired hemophilia A in a patient with metastatic castration-resistant prostate cancer.

Poscente M, Gan CL, Heng DYC, Alimohamed N

Canadian Urological Association journal = Journal de l'Association des urologues du Canada 2021; (15(9)):E519-E521 doi:10.5489/cuaj.7089.

Clinical phenotype of severe and moderate haemophilia: Who should receive prophylaxis and what is the target trough level?

Collins PW, Obaji SG, Roberts H, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2021; (27(2)):192-198 doi:10.1111/hae.14201.

Emicizumab in tolerized patients with hemophilia A with inhibitors: A single-institution pediatric cohort assessing inhibitor status.

Batsuli G, Greene A, Meeks SL, Sidonio RF

Research and practice in thrombosis and haemostasis 2021; (5(2)):342-348 doi:10.1002/rth2.12475.

[Effect of FVIII doses on joint structure and function in adolescents with severe hemophilia A: mid-term results of a prospective cohort study].

Meng L, Zhuang J, Sun X, et al.

Nan fang yi ke da xue xue bao = Journal of Southern Medical University 2021; (41(4)):536-542 doi:10.12122/j.issn.1673-4254.2021.04.08.

Clinicopathological features of hemophilia in a tertiary care centre of India.

Pawan PK, Mahima Y, Vijai T, Manjula L

Journal of family medicine and primary care 2021; (10(1)):295-299 doi:10.4103/jfmpc.jfmpc_1564_20.

Disruptive technology and hemophilia care: The multiple impacts of emicizumab.

Hermans C, Makris M

Research and practice in thrombosis and haemostasis 2021; (5(4)):e12508 doi:10.1002/rth2.12508.

Hemophilic arthropathy: Current knowledge and future perspectives.

Gualtierotti R, Solimeno LP, Peyvandi F

Journal of thrombosis and haemostasis : JTH 2021; (19(9)):2112-2121 doi:10.1111/jth.15444.

A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH.

van Galen KPM, d'Oiron R, James P, et al.

Journal of thrombosis and haemostasis : JTH 2021; (19(8)):1883-1887 doi:10.1111/jth.15397.

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A.

Pasi KJ, Laffan M, Rangarajan S, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2021; (27(6)):947-956 doi:10.1111/hae.14391.

Regulation and importance of factor VIII levels in hemophilia A carriers.

Cygan PH, Kouides PA

Current opinion in hematology 2021; (28(5)):315-322 doi:10.1097/MOH.0000000000000667.

Congenital hemophilia A diagnosed with postoperative hemorrhage after thyroidectomy for papillary thyroid carcinoma: a case report.

Saitou M, Okamoto M, Nagaoka R, et al.

Surgical case reports 2021; (7(1)):189 doi:10.1186/s40792-021-01272-x.

Low immunogenicity of emicizumab in persons with haemophilia A.

Schmitt C, Emrich T, Chebon S, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2021; (27(6)):984-992 doi:10.1111/hae.14398.

PERSEPT 3: A phase 3 clinical trial to evaluate the haemostatic efficacy of eptacog beta (recombinant human FVIIa) in perioperative care in subjects with haemophilia A or B with inhibitors.

Escobar M, Luck J, Averianov Y, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2021; (27(6)):911-920 doi:10.1111/hae.14418.

Low-dose immune tolerance induction therapy in children of Arab descent with severe haemophilia A, high inhibitor titres and poor prognostic factors for immune tolerance induction treatment success.

Elalfy M, Elghamry I, Hassab H, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2022; (28(1)):65-72 doi:10.1111/hae.14456.

Pediatric acquired factor VIII deficiency presenting as hemarthrosis.

Daigh LH, Chien MC, Lo CY

Pediatric blood & cancer 2022; (69(5)):e29530 doi:10.1002/pbc.29530.

Uncontrollable bleeding after tooth extraction from asymptomatic mild hemophilia patients: two case reports.

Fan G, Shen Y, Cai Y, et al.

BMC oral health 2022; (22(1)):69 doi:10.1186/s12903-022-02074-9.

Prophylactic emicizumab for hemophilia A in the Asia-Pacific region: A randomized study (HAVEN 5).

Yang R, Wang S, Wang X, et al.

Research and practice in thrombosis and haemostasis 2022; (6(2)):e12670 doi:10.1002/rth2.12670.

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.

Ozelo MC, Mahlangu J, Pasi KJ, et al.

The New England journal of medicine 2022; (386(11)):1013-1025 doi:10.1056/NEJMoa2113708.

Efanesoctocog alfa elicits functional clot formation that is indistinguishable to that of recombinant factor VIII.

Demers M, Aleman MM, Kistanova E, et al.

Journal of thrombosis and haemostasis : JTH 2022; (20(7)):1674-1683 doi:10.1111/jth.15741.

F8 gene mutation spectrum in severe hemophilia A with inhibitors: A large cohort data analysis from a single center in China.

Sun J, Li Z, Huang K, et al.

Research and practice in thrombosis and haemostasis 2022; (6(4)):e12723 doi:10.1002/rth2.12723.

Recombinant porcine factor VIII corrects thrombin generation in vitro in plasma from patients with congenital hemophilia A and inhibitors.

Négrier C, Oldenburg J, Kenet G, et al.

Research and practice in thrombosis and haemostasis 2022; (6(4)):e12731 doi:10.1002/rth2.12731.

First conditional marketing authorization approval in the European Union for hemophilia "A" gene therapy.

VandenDriessche T, Pipe SW, Pierce GF, Kaczmarek R

Molecular therapy : the journal of the American Society of Gene Therapy 2022; (30(11)):3335-3336 doi:10.1016/j.ymthe.2022.09.020.

Association between Sports Participation, Factor VIII Levels and Bleeding in Hemophilia A.

Bukkems LH, Versloot O, Cnossen MH, et al.

Thrombosis and haemostasis 2023; (123(3)):317-325 doi:10.1055/a-1983-0594.

[Gene therapy of Hemophilia: Recommendations from the German, Austrian, and Swiss Society for Thrombosis and Haemostasis Research (GTH)].

Miesbach W, Oldenburg J, Klamroth R, et al.

Hamostaseologie 2023; (43(3)):196-207 doi:10.1055/a-1957-4477.

Esophageal ulcer associated with mild hemophilia A: case report.

Nicolescu CM, Neşiu A, Uzum A, et al.

Romanian journal of morphology and embryology = Revue roumaine de morphologie et embryologie 2022; (63(3)):581-585 doi:10.47162/RJME.63.3.14.

Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.

von Drygalski A, Chowdary P, Kulkarni R, et al.

The New England journal of medicine 2023; (388(4)):310-318 doi:10.1056/NEJMoa2209226.

Long-term impact of primary prophylaxis on joint status in patients with severe hemophilia A.

Meijón Ortigueira MDM, Álvarez-Román MT, De La Corte Rodríguez H, et al.

Research and practice in thrombosis and haemostasis 2023; (7(1)):100005 doi:10.1016/j.rpth.2022.100005.

Bleeding phenotype in nonsevere hemophilia by International Society on Thrombosis and Haemostasis bleeding assessment tool, bleeding frequency, and the joint status.

Rejtő J, Kraemmer D, Grilz E, et al.

Research and practice in thrombosis and haemostasis 2023; (7(2)):100047 doi:10.1016/j.rpth.2023.100047.

Ferroptosis: a new target for iron overload-induced hemophilic arthropathy synovitis.

Han Z, Zheng L, Luo D, et al.

Annals of hematology 2023; (102(5)):1229-1237 doi:10.1007/s00277-023-05190-w.

Congenital Bleeding Disorders: Managing Central Nervous System Bleeding in an Adult Hemophiliac.

Dahra A, Mehdi Z, Gupta M, et al.

Cureus 2023; (15(3)):e36906 doi:10.7759/cureus.36906.

Antithrombin lowering in hemophilia: a closer look at fitusiran.

Young G, Lenting PJ, Croteau SE, et al.

Research and practice in thrombosis and haemostasis 2023; (7(4)):100179 doi:10.1016/j.rpth.2023.100179.

Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B.

Castaman G, Coppens M, Pipe SW

Expert review of hematology 2023; (16(12)):919-932 doi:10.1080/17474086.2023.2276206.

Large Intron Inversions in Romanian Patients with Hemophilia A-First Report.

Brinza M, Grigore A, Dragomir M, et al.

Medicina (Kaunas, Lithuania) 2023; (59(10)) doi:10.3390/medicina59101821.

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India.

Krishnamoorthy Y, Govindan D, Kannan N, et al.

Heliyon 2024; (10(5)):e27089 doi:10.1016/j.heliyon.2024.e27089.

Moderate- to vigorous-intensity physical activities for hemophilia A patients during low-dose pharmacokinetic-guided extended half-life factor VIII prophylaxis.

Srichumpuang C, Rakmanotham A, Moonla C, Sosothikul D

Orphanet journal of rare diseases 2024; (19(1)):135 doi:10.1186/s13023-024-03092-2.

The good, the bad and the ugly of pain in haemophilia: Recent evidence on the epidemiology, molecular mechanisms and knowledge gaps preventing optimal treatment.

Benemei S, Mattia C, Di Minno MND

Haemophilia : the official journal of the World Federation of Hemophilia 2024; (30(3)):589-597 doi:10.1111/hae.15002.

Liver health in hemophilia in the era of gene therapy.

Dargaud Y, Levrero M, Bailly F, et al.

Thrombosis research 2024; (240()):109064 doi:10.1016/j.thromres.2024.109064.

International Society on Thrombosis and Haemostasis clinical practice guideline for treatment of congenital hemophilia A and B based on the Grading of Recommendations Assessment, Development, and Evaluation methodology.

Rezende SM, Neumann I, Angchaisuksiri P, et al.

Journal of thrombosis and haemostasis : JTH 2024; (22(9)):2629-2652 doi:10.1016/j.jtha.2024.05.026.

Pharmacokinetic evaluation of efanesoctocog alfa: breakthrough factor VIII therapy for hemophilia A.

Yada K, Nogami K

Expert opinion on drug metabolism & toxicology 2025; (21(1)):5-14 doi:10.1080/17425255.2024.2409931.

Expert Opinion for Defining a Severe Bleeding Phenotype to Guide Prophylaxis in Patients with Nonsevere Hemophilia.

Pfrepper C, Ettingshausen CE, Klamroth R, et al.

Hamostaseologie 2026; (46(3)):206-214 doi:10.1055/a-2411-7416.

Transforming Hemophilia A Care: Insights into New Therapeutic Options.

Iurea IM, Severin E, Matei A

Life (Basel, Switzerland) 2024; (14(12)) doi:10.3390/life14121568.

Treatment of Bleeding Episodes With Efanesoctocog Alfa in Previously Treated Patients With Severe Hemophilia A in the Phase 3 XTEND-1 Study.

Weyand AC, Meunier S, Suzuki N, et al.

American journal of hematology 2025; (100(5)):813-820 doi:10.1002/ajh.27603.

Contemporary approaches to treat people with hemophilia: what's new and what's not?

Valentino LA, Santaella ME, Carlson SA, Recht M

Research and practice in thrombosis and haemostasis 2025; (9(1)):102696 doi:10.1016/j.rpth.2025.102696.

Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance.

Konkle BA, Peyvandi F, Foster GR, et al.

Journal of thrombosis and haemostasis : JTH 2025; (23(7)):2086-2094 doi:10.1016/j.jtha.2025.02.042.

Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies.

Klamroth R, von Drygalski A, Hermans C, et al.

Haemophilia : the official journal of the World Federation of Hemophilia 2025; (31(3)):391-400 doi:10.1111/hae.70017.

An Observational Study of Glycopegylated Extended Half-Life Factor Prophylaxis in Hemophilia A and B in a Tertiary Care Center in India.

Bhatt P, Patel H

Cureus 2025; (17(2)):e79815 doi:10.7759/cureus.79815.

Rebalancing Hemostasis: Fitusiran as a First-in-Class RNAi Therapy in Hemophilia A and B.

Rehman RU, Fatima R, Akilimali A

Health science reports 2026; (9(1)):e71702 doi:10.1002/hsr2.71702.

Gene Therapy of Haemophilia: Current Status and Future Directions.

Miesbach W

Hamostaseologie 2026; (46(1)):10-16 doi:10.1055/a-2751-7625.