Designing ATHN 7: Safety, effectiveness and practice of treatment of people with haemophilia in the United States through a natural history cohort study
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.
Iorio A, Stonebraker JS, Chambost H, et al.; Data and Demographics Committee of the World Federation of Hemophilia. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med 2019; 171(8): 540–546. doi: 10.7326/M19-1208.IorioAStonebrakerJSChambostHData and Demographics Committee of the World Federation of Hemophilia. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries2019171854054610.7326/M19-120831499529Open DOISearch in Google Scholar
Marchesini E, Morfini M, Valentino L. Recent advances in the treatment of haemophilia A: a review. Biologics 2021; 15: 221–235. doi: 10.2147/BTT.S252580.MarchesiniEMorfiniMValentinoLRecent advances in the treatment of haemophilia A: a review20211522123510.2147/BTT.S252580821453934163136Open DOISearch in Google Scholar
Weyand AC, James PD. Sexism in the management of bleeding disorders. Res Pract Thromb Haemost 2020; 5(1): 51–54. doi: 10.1002/rth2.12468.WeyandACJamesPDSexism in the management of bleeding disorders202051515410.1002/rth2.12468784507033537529Open DOISearch in Google Scholar
van Galen KPM, d'Oiron R, James P, et al. A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH. J Thromb Haemost 2021; 19(8): 1883–1887. doi: 10.1111/jth.15397.van GalenKPMd'OironRJamesPA new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH20211981883188710.1111/jth.15397836171334327828Open DOISearch in Google Scholar
Berntorp E, Fischer K, Hart DP, et al. Haemophilia. Nat Rev Dis Primers 2021; 7(1): 45. doi: 10.1038/s41572-021-00278-x.BerntorpEFischerKHartDPHaemophilia2021714510.1038/s41572-021-00278-x34168126Open DOISearch in Google Scholar
Chen S-L. Economic costs of haemophilia and the impact of prophylactic treatment on patient management. Am J Manag Care 2016; 22(5 Suppl): s126–33.ChenS-LEconomic costs of haemophilia and the impact of prophylactic treatment on patient management2016225 Suppls12633Search in Google Scholar
Gualtierotti R, Tafuri F, Arcudi S, et al. Current and emerging approaches for pain management in hemophilic arthropathy. Pain Ther 2022; 11(1): 1–15. doi: 10.1007/s40122-021-00345-x.GualtierottiRTafuriFArcudiSCurrent and emerging approaches for pain management in hemophilic arthropathy202211111510.1007/s40122-021-00345-x886124335020184Open DOISearch in Google Scholar
Barry V, Buckner TW, Lynch ME, et al. An evaluation of PROMIS health domains in adults with haemophilia: A cross-sectional study. Haemophilia 2021; 27(3): 375–382. doi: 10.1111/hae.14321.BarryVBucknerTWLynchMEAn evaluation of PROMIS health domains in adults with haemophilia: A cross-sectional study202127337538210.1111/hae.1432133866654Open DOISearch in Google Scholar
DeKoven M, Karkare S, Kelley LA, et al. Understanding the experience of caring for children with haemophilia: cross-sectional study of caregivers in the United States. Haemophilia 2014; 20(4): 541–9. doi: 10.1111/hae.12379.DeKovenMKarkareSKelleyLAUnderstanding the experience of caring for children with haemophilia: cross-sectional study of caregivers in the United States2014204541910.1111/hae.1237924472032Open DOISearch in Google Scholar
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.OldenburgJMahlanguJNKimBEmicizumab prophylaxis in hemophilia A with inhibitors2017377980981810.1056/NEJMoa170306828691557Open DOISearch in Google Scholar
Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med 2018; 379(9): 811–822. doi: 10.1056/NEJMoa1803550.MahlanguJOldenburgJPaz-PrielIEmicizumab prophylaxis in patients who have hemophilia A without inhibitors2018379981182210.1056/NEJMoa180355030157389Open DOISearch in Google Scholar
Ellsworth P, Ma A. Factor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates? Hematology Am Soc Hematol Educ Program 2021; 2021(1): 219–225. doi: 10.1182/hematology.2021000253.EllsworthPMaAFactor-mimetic and rebalancing therapies in hemophilia A and B: the end of factor concentrates?20212021121922510.1182/hematology.2021000253879112334889356Open DOISearch in Google Scholar
McGill JR, Simhadri VL, Sauna ZE. HLA Variants and inhibitor development in hemophilia A: a retrospective case-controlled study using the ATHN dataset. Front Med (Lausanne) 2021; 8: 663396. doi: 10.3389/fmed.2021.663396.McGillJRSimhadriVLSaunaZEHLA Variants and inhibitor development in hemophilia A: a retrospective case-controlled study using the ATHN dataset2021866339610.3389/fmed.2021.663396813940534026790Open DOISearch in Google Scholar
Male C, Andersson NG, Rafowicz A, et al. Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica 2021; 106(1): 123–129. doi: 10.3324/haematol.2019.239160.MaleCAnderssonNGRafowiczAInhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study2021106112312910.3324/haematol.2019.239160777624631919092Open DOISearch in Google Scholar
Garagiola I, Palla R, Peyvandi F. Risk factors for inhibitor development in severe hemophilia a. Thromb Res 2018; 168: 20–27. doi: 10.1016/j.thromres.2018.05.027.GaragiolaIPallaRPeyvandiFRisk factors for inhibitor development in severe hemophilia a2018168202710.1016/j.thromres.2018.05.02729879570Open DOISearch in Google Scholar
Valentino LA, Kempton CL, Kruse-Jarres R, et al.; International Immune Tolerance Induction Study Investigators. US Guidelines for immune tolerance induction in patients with haemophilia A and inhibitors. Haemophilia 2015; 21(5): 559–67. doi: 10.1111/hae.12730.ValentinoLAKemptonCLKruse-JarresRInternational Immune Tolerance Induction Study Investigators. US Guidelines for immune tolerance induction in patients with haemophilia A and inhibitors20152155596710.1111/hae.1273026032231Open DOISearch in Google Scholar
Davis JA, Klamroth R, Benchikh El Fegoun S, Habis R, de Moerloose P. A survey among patients with haemophilia and inhibitors or their caregivers, seeking treatment in nonhaemophilia treatment centres. Haemophilia 2020; 26(4): e205–e208. doi: 10.1111/hae.13983.DavisJAKlamrothRBenchikh El FegounSHabisRde MoerloosePA survey among patients with haemophilia and inhibitors or their caregivers, seeking treatment in nonhaemophilia treatment centres2020264e205e20810.1111/hae.1398332267042Open DOISearch in Google Scholar
Valentino LA, Baker JR, Butler R, et al. Integrated hemophilia patient care via a national network of care centers in the United States: a model for rare coagulation disorders. J Blood Med 2021; 12: 897–911. doi: 10.2147/JBM.S325031.ValentinoLABakerJRButlerRIntegrated hemophilia patient care via a national network of care centers in the United States: a model for rare coagulation disorders20211289791110.2147/JBM.S325031854426534707421Open DOISearch in Google Scholar
World Medical Association. WMA Declaration of Helsinki – Ethical principles for medical research involving human subjects. Available at: https://www.wma.net/policies-post/wma-declaration-of-helsinki-ethical-principles-formedical-research-involving-human-subjects/ (accessed 21 November 2022).World Medical AssociationAvailable at: https://www.wma.net/policies-post/wma-declaration-of-helsinki-ethical-principles-formedical-research-involving-human-subjects/ (accessed 21 November 2022).Search in Google Scholar
Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost 2014; 12(11): 1935–9. doi: 10.1111/jth.12672.BlanchetteVSKeyNSLjungLRManco-JohnsonMJvan den BergHMSrivastavaASubcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH201412111935910.1111/jth.1267225059285Open DOISearch in Google Scholar
Makris M, Calizzani G, Fischer K, et al. EUHASS: The European Haemophilia Safety Surveillance system. Thromb Res 2011; 127 Suppl 2: S22–5. doi: 10.1016/S0049-3848(10)70150-X.MakrisMCalizzaniGFischerKEUHASS: The European Haemophilia Safety Surveillance system2011127Suppl 2S22510.1016/S0049-3848(10)70150-X21193110Open DOISearch in Google Scholar
Boylan B, Miller CH. Effects of pre-analytical heat treatment in factor VIII (FVIII) inhibitor assays on FVIII antibody levels. Haemophilia 2018; 24(3): 487–491. doi: 10.1111/hae.13435.BoylanBMillerCHEffects of pre-analytical heat treatment in factor VIII (FVIII) inhibitor assays on FVIII antibody levels201824348749110.1111/hae.13435607256529461004Open DOISearch in Google Scholar