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1. Management of multiple myeloma in a patient with haemophilia with concurrent emicizumab – case report

Author

Davies Elizabeth, Carter-Brzezinski Luke, Traintafillou Margarita, Brown Rachel, and Hay Charles R M

Subjects
emicizumab, myeloma, thromboprophylaxis, thalidomide, lenalidomide, Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

With the increased life expectancies of people with severe haemophilia A, diagnoses of disorders associated with increased age are likely to become more common. Multiple myeloma is a commonly diagnosed plasma cell dyscrasia, with incidence strongly related to age. Both the disease itself and specific myeloma therapies are associated with a significant increase in rates of venous thromboembolism (VTE). Management of VTE prophylaxis can be challenging, especially in patients with a bleeding disorder. In this case report, we describe the management of a patient with concurrent diagnosis of multiple myeloma and severe haemophilia A, undergoing first line myeloma chemotherapy. The patient was successfully managed on standard of care myeloma treatment, with use of emicizumab and venous thromboprophylaxis. This case demonstrates the successful management of a haematological malignancy and a bleeding disorder using integrated and multidisciplinary patient-focused care.

Published
2021
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2. The factor VIII treatment history of non‐severe hemophilia A

Author

Abdi, Amal, Kloosterman, Fabienne R., Eckhardt, Corien L., Male, Christoph, Castaman, Giancarlo, Fischer, Kathelijn, Beckers, Erik A.M., Kruip, Marieke J.H.A., Peerlinck, Kathelijne, Mancuso, Maria Elisa, Santoro, Cristina, Hay, Charles R., Platokouki, Helen, van der Bom, Johanna G., Gouw, Samantha C., Fijnvandraat, Karin, and Hart, Dan P.

Published
2020
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3. Association of patient, treatment and disease characteristics with patient‐reported outcomes: Results of the ECHO Registry.

Author

Hay, Charles R. M., Makris, Michael, Shima, Midori, Nagao, Azusa, Jiménez‐Yuste, Víctor, Skinner, Mark, Kessler, Craig M., and von Mackensen, Sylvia

Subjects
*PATIENT reported outcome measures, *THERAPEUTICS, *PATIENT satisfaction, *JOINTS (Anatomy), *PHYSICAL mobility
Abstract

Introduction: Patient‐reported outcomes (PROs) in people living with haemophilia A (PLWHA) are often under‐reported. Investigating PROs from a single study with a diverse population of PLWHA is valuable, irrespective of FVIII product or regimen. Aim: To report available data from the Expanding Communications on Haemophilia A Outcomes (ECHO) registry investigating the associations of patient, treatment and disease characteristics with PROs and clinical outcomes in PLWHA. Methods: ECHO (NCT02396862), a prospective, multinational, observational registry, enrolled participants aged ≥16 years with moderate or severe haemophilia A using any product or treatment regimen. Data collection, including a variety of PRO questionnaires, was planned at baseline and annually for ≥2 years. Associations between PRO scores and patient, treatment and disease characteristics were determined by statistical analyses. Results: ECHO was terminated early owing to logistical constraints. Baseline data were available from 269 PLWHA from Europe, the United States and Japan. Most participants received prophylactic treatment (76.2%), with those using extended‐half‐life products (10.0%) reporting higher treatment satisfaction. Older age and body weight >30 kg/m2 (>BMI) were associated with poorer joint health. Older age was associated with poorer physical functioning and work productivity. Health‐related quality of life and pain interference also deteriorated with age and >BMI; >BMI also increased pain severity scores. Conclusion: ECHO captured a variety of disease characteristics, treatment patterns, PROs and clinical outcomes obtained in real‐world practice with ≤1 year's follow‐up. Older age, poorer joint health and >BMI adversely affected multiple aspects of participant well‐being. [ABSTRACT FROM AUTHOR]

Published
2024
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11. Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company

Author

Hay, Charles R. M., primary, Shima, Midori, additional, Makris, Michael, additional, Jiménez‐Yuste, Victor, additional, Oldenburg, Johannes, additional, Fischer, Kathelijn, additional, Iorio, Alfonso, additional, Skinner, Mark W., additional, Santagostino, Elena, additional, Mackensen, Sylvia, additional, and Kessler, Craig M., additional

Published
2020
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15. The factor VIII treatment history of non-severe hemophilia A

Author

Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Abdi, Amal, Kloosterman, Fabienne R., Eckhardt, Corien L., Male, Christoph, Castaman, Giancarlo, Fischer, Kathelijn, Beckers, Erik A.M., Kruip, Marieke J.H.A., Peerlinck, Kathelijne, Mancuso, Maria Elisa, Santoro, Cristina, Hay, Charles R., Platokouki, Helen, van der Bom, Johanna G., Gouw, Samantha C., Fijnvandraat, Karin, Hart, Dan P., INSIGHT Study Group, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Abdi, Amal, Kloosterman, Fabienne R., Eckhardt, Corien L., Male, Christoph, Castaman, Giancarlo, Fischer, Kathelijn, Beckers, Erik A.M., Kruip, Marieke J.H.A., Peerlinck, Kathelijne, Mancuso, Maria Elisa, Santoro, Cristina, Hay, Charles R., Platokouki, Helen, van der Bom, Johanna G., Gouw, Samantha C., Fijnvandraat, Karin, Hart, Dan P., and INSIGHT Study Group

Published
2020

17. Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study

Author

Oldenburg, Johannes, primary, Hay, Charles R M, additional, Jiménez-Yuste, Víctor, additional, Peyvandi, Flora, additional, Schved, Jean-François, additional, Szamosi, Johan, additional, Winding, Bent, additional, and Lethagen, Stefan, additional

Published
2019
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19. A Novel, Enriched Population Pharmacokinetic Model for Recombinant Factor VIII-Fc Fusion Protein Concentrate in Hemophilia A Patients.

Author

Bukkems, Laura H., Heijdra, Jessica M., Mathias, Mary, Collins, Peter W., Hay, Charles R. M., Tait, Robert C., Mangles, Sarah, Myers, Bethan, Evans, G., Bailiff, Benjamin, Curry, Nicola, Payne, Jeanette, Austin, Steve, Goedhart, Tine M. H. J., Leebeek, Frank W. G., Meijer, Karina, Fijnvandraat, Karin, Chowdary, Pratima, Mathôt, Ron A. A., and Cnossen, Marjon H.

Published
2020
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21. Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

Author

Pasi, K. John, primary, Rangarajan, Savita, additional, Georgiev, Pencho, additional, Mant, Tim, additional, Creagh, Michael D., additional, Lissitchkov, Toshko, additional, Bevan, David, additional, Austin, Steve, additional, Hay, Charles R., additional, Hegemann, Inga, additional, Kazmi, Rashid, additional, Chowdary, Pratima, additional, Gercheva-Kyuchukova, Liana, additional, Mamonov, Vasily, additional, Timofeeva, Margarita, additional, Soh, Chang-Heok, additional, Garg, Pushkal, additional, Vaishnaw, Akshay, additional, Akinc, Akin, additional, Sørensen, Benny, additional, and Ragni, Margaret V., additional

Published
2017
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25. Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study.

Author

Scott, Martin J., Xiang, Hua, Hart, Daniel P., Palmer, Benedict, Collins, Peter W., Stephensen, David, Sima, Camelia S., and Hay, Charles R. M.

Subjects
TREATMENT effectiveness
Abstract

Introduction: The THUNDER study provides an analysis of treatment patterns and outcomes in UK patients with severe or moderate haemophilia A (SHA/MHA) in 2015. Methods: Patients with SHA or MHA registered with the UK National Haemophilia Database (NHD) were segregated by severity, inhibitor status and age. Haemophilia joint health score (HJHS) was derived from NHD records and treatment regimen and annualized bleed/joint‐bleed rate (ABR/AJBR) from Haemtrack (HT) in HT‐compliant patients. Results: We report 1810 patients with SHA and 864 with MHA. Prophylaxis was used in 94.9% (n = 130/137) of HT‐compliant children <12 years with SHA, falling to 74.1% (n = 123/166) aged ≥40 years. Median ABR increased with age (1.0, IQR 0.0‐5.0, <12 years; 3.0 IQR, 1.0‐8.0, ≥40 years). Inhibitors were present in 159 (8.8%) SHA and 34 (3.9%) MHA. Median ABR increased from 2.0 (<12 years) to 21.0 (≥40 years) in SHA inhibitor patients using prophylaxis. Prophylaxis was used by 68.8% of HT‐compliant MHA patients (n = 106) (median FVIII baseline 0.01 IU/mL) associated with a median (IQR) ABR of 3.0 (1.0‐7.0). Median HJHS (n = 453) increased with age in SHA and MHA. Median (IQR) HJHS was higher in SHA inhibitor (17.0, 0.0‐64.5) than non‐ or past inhibitor patients (7.0, 0.0‐23.0). Conclusions: Increasing ABR with age persists despite current prophylaxis regimens. SHA and MHA had similar ABR/AJBR and HJHS, leading to a suspicion that a subgroup of MHA may be relatively undertreated. More intensive prophylaxis may improve outcomes, but this requires further study. [ABSTRACT FROM AUTHOR]

Published
2019
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26. Summary report of the First International Conference on inhibitors in haemophilia A

Author

Lacroix-Desmazes, Sebastien, Scott, David W, Goudemand, Jenny, Van Den Berg, Marijke, Makris, Michael, Van Velzen, Alice S, Santagostino, Elena, Lillicrap, David, Rosendaal, Frits R, Hilger, Anneliese, Sauna, Zuben E, Oldenburg, Johannes, Mantovani, Lorenzo, Mancuso, M Elisa, Kessler, Craig, Hay, Charles R M, Knoebl, Paul, Di Minno, Giovanni, Hoots, Keith, Bok, Amanda, Brooker, Mark, Buoso, Erica, Mannucci, Pier Mannuccio, Peyvandi, Flora, Lacroix-Desmazes, Sebastien, Scott, David W, Goudemand, Jenny, Van Den Berg, Marijke, Makris, Michael, Van Velzen, Alice S, Santagostino, Elena, Lillicrap, David, Rosendaal, Frits R, Hilger, Anneliese, Sauna, Zuben E, Oldenburg, Johannes, Mantovani, Lorenzo, Mancuso, M Elisa, Kessler, Craig, Hay, Charles R M, Knoebl, Paul, Di Minno, Giovanni, Hoots, Keith, Bok, Amanda, Brooker, Mark, Buoso, Erica, Mannucci, Pier Mannuccio, and Peyvandi, Flora

Published
2017

27. Summary report of the First International Conference on inhibitors in haemophilia A

Author

Cardiovasculaire Epi Team 1, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, Circulatory Health, Lacroix-Desmazes, Sebastien, Scott, David W, Goudemand, Jenny, Van Den Berg, Marijke, Makris, Michael, Van Velzen, Alice S, Santagostino, Elena, Lillicrap, David, Rosendaal, Frits R, Hilger, Anneliese, Sauna, Zuben E, Oldenburg, Johannes, Mantovani, Lorenzo, Mancuso, M Elisa, Kessler, Craig, Hay, Charles R M, Knoebl, Paul, Di Minno, Giovanni, Hoots, Keith, Bok, Amanda, Brooker, Mark, Buoso, Erica, Mannucci, Pier Mannuccio, Peyvandi, Flora, Cardiovasculaire Epi Team 1, JC onderzoeksprogramma Cardiovasculaire Epidemiologie, Circulatory Health, Lacroix-Desmazes, Sebastien, Scott, David W, Goudemand, Jenny, Van Den Berg, Marijke, Makris, Michael, Van Velzen, Alice S, Santagostino, Elena, Lillicrap, David, Rosendaal, Frits R, Hilger, Anneliese, Sauna, Zuben E, Oldenburg, Johannes, Mantovani, Lorenzo, Mancuso, M Elisa, Kessler, Craig, Hay, Charles R M, Knoebl, Paul, Di Minno, Giovanni, Hoots, Keith, Bok, Amanda, Brooker, Mark, Buoso, Erica, Mannucci, Pier Mannuccio, and Peyvandi, Flora

Published
2017

28. Von Willebrand Factor antigen and age explain variation in baseline FVIII: C among nonsevere hemophilia A patients with the same F8 genotype (Arg593Cys and Asn618Ser)

Author

Loomans, Janneke I., Van Velzen, Alice S., Eckhardt, Corien L., Peters, Marjolein, Astermark, Jan, Brons, Paul P., Castaman, Giancarlo, Cnossen, Marjon H., Dors, Natasja, Escuriola-Ettingshausen, Carmen, Hamulyak, Karly, Hart, Daniel P., Hay, Charles R. M., Haya, Saturnino, Van Heerde, Waander L., Hermans, Cedric, Holmstrom, Margaretha, Jimenez-Yuste, Victor, Keenan, Russell D., Klamroth, Robert, Koenigs, Christoph, Kruip, Marieke J. H. A., Laros-Van Gorkom, Britta A. P., Leebeek, Frank W. G., Liesner, Ri, Makipernaa, Anne, Male, Christoph, Mauser-Bunschoten, Evelien, Mazzucconi, Maria G., Mcrae, Simon, Meijer, Karina, Mitchell, Michael, Morfini, Massimo, Nijziel, Marten, Oldenburg, Johannes, Peerlinck, Kathelijne, Petrini, Pia, Platokouki, Helen, Rangarajan, Savita, Reitter-Pfoertner, Sylvia E., Santagostino, Elena, Schinco, Piercarla, Smiers, Frans J., Siegmund, Berthold, Tagliaferri, Annarita, Yee, Thynn T., Kamphuisen, Pieter Willem, Van der Bom, Johanna G., Fijnvandraat, Karin, Cardiovascular Centre (CVC), and Vascular Ageing Programme (VAP)

Published
2014

29. Clinical presentation of inhibitor development in non-severe hemophilia A: Half of patients have high titer inhibitors and present with bleeding complications

Author

Eckhardt, Corien L., Loomans, Janneke I., Velzen, Alice S. Van, Peters, Marjolein, Astermark, Jan, Brons, Paul P., Castaman, Giancarlo, Cnossen, Marjon H., Dors, Natasja, Escuriola-Ettingshausen, Carmen, Hamulyak, Karly, Hart, Daniel P., Hay, Charles R. M., Haya, Saturnino, Van Heerde, Waander L., Hermans, Cedric, Holmstrom, Margaretha, Jimenez-Yuste, Victor, Keenan, Russell D., Klamroth, Robert, Van Laros-Gorkom, Britta A. P., Leebeek, Frank W. G., Liesner, Ri, Makipernaa, Anne, Male, Christoph, Mauser-Bunschoten, Eveline, Mazzucconi, Maria G., Mcrae, Simon, Meijer, Karina, Mitchell, Michael, Morfini, Massimo, Nijziel, Marten, Oldenburg, Johannes, Peerlinck, Kathelijne, Petrini, Pia, Platokouki, Helen, Rangarajan, Savita, Reitter-Pfoertner, Sylvia E., Santagostino, Elena, Schinco, Piercarla, Smiers, Frans J., Siegmund, Berthold, Tagliaferri, Annarita, Yee, Thynn T., Kamphuisen, Pieter Willem, Van der Bom, Johanna G., Fijnvandraat, Karin, Cardiovascular Centre (CVC), and Vascular Ageing Programme (VAP)

Subjects
implantable cardioverter defibrillator, phenotype, screening, groups by age, neutralizing antibody, population, bleeding tendency, clinical study, blood clotting factor 8 concentrate, bleeding, exposure, hemophilia, follow up, hemophilia A, patient, human, laboratory
Abstract

Introduction and Objectives: Inhibitor development in nonsevere haemophilia A patients (FVIII:C, 2-40 IU/dL) has a heterogeneous clinical phenotype, ranging from irrelevant transient inhibitors to high titre neutralizing antibodies with severe bleeding complications. Data on inhibitor presentation are scarce and selected, favouring those with severe complications. The aim of current study was to describe the presenting symptoms of inhibitors in a large unselected cohort of nonsevere haemophilia patients. Materials and Methods: Clinical data were collected of 107 inhibitor patients derived from a source population of 2,709 nonsevere haemophilia A patients that were treated between 1980 and 2011 in 34 European and Australian centers (the INSIGHT consortium). Patients were subdivided according to age and calendar period at time of inhibitor development (10-year groups), aiming to search for age-related trends and trends over time. Results: Age at inhibitor detection varied widely between 1 and 85 years with a median age of 38 years (IQR, 15-61). Most inhibitors developed in the period of 2000-2010 (n = 64, 60%). Inhibitors developed after a median of 28 exposure days (IQR, 14-66), which was comparable between all age groups. Fifty-seven patients (56%) had high titre inhibitors (>5 BU/mL). About half of the high titre inhibitors developed in patients older than 40 years (n = 30, 53%) and 60% after the year 2000 (n = 34). In 13 (12%) inhibitor patients the inhibitor was detected during routine laboratory screening and no clinical signs of the inhibitor were present. More than half of the patients (n = 61) had an increased bleeding tendency at presentation with inhibitor. In 80 patients (80%) endogenous FVIII:C was decreased to below 5 IU/dL (including 33/44 patients with low titer inhibitors), of whom FVIII:C fell below ≤ 0.01 IU/mL in 49 patients (46%). Conclusion: Half of the patients with nonsevere haemophilia A and inhibitors developed high titre inhibitors; these were more common in the last decennium. More than half of the patients presented with bleeding complications and 46% had changed to a severe phenotype. These results stress the importance of close follow-up after exposure to therapeutic factor VIII concentrates in nonsevere haemophilia A patients.

Published
2014

30. Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

Author

Ragni, Margaret V., primary, Georgiev, Pencho, additional, Mant, Tim, additional, Creagh, Michael Desmond, additional, Lissitchkov, Toshko, additional, Bevan, David, additional, Austin, Steve, additional, Hay, Charles R, additional, Hegemann, Inga, additional, Kazmi, Rashid, additional, Chowdary, Pratima, additional, Rangarajan, Savita, additional, Soh, Chang-Heok, additional, Akinc, Akin, additional, Partisano, Angela M., additional, Sorenson, Benny, additional, and Pasi, K John, additional

Published
2016
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31. Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

Author

Pasi, K John, primary, Georgiev, Pencho, additional, Mant, Tim, additional, Lissitchkov, Toshko, additional, Creagh, Michael Desmond, additional, Bevan, David, additional, Austin, Steve, additional, Hay, Charles R, additional, Hegemann, Inga, additional, Kazmi, Rashid, additional, Chowdary, Pratima, additional, Ragni, Margaret V., additional, Soh, Chang-Heok, additional, Akinc, Akin, additional, Partisano, Angela M., additional, Sorenson, Benny, additional, and Rangarajan, Savita, additional

Published
2016
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32. A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Weekly and Monthly Dosing Results in Patients with Hemophilia A or B

Author

Pasi, K John, primary, Georgiev, Pencho, additional, Mant, Tim, additional, Creagh, Michael Desmond, additional, Lissitchkov, Toshko, additional, Bevan, David, additional, Austin, Steve, additional, Hay, Charles R, additional, Brand, Brigit, additional, Kazmi, Rashid, additional, Chowdary, Pratima, additional, Ragni, Margaret V, additional, Chen, Jihong, additional, Akinc, Akin, additional, Sorensen, Benny, additional, and Rangarajan, Savita, additional

Published
2015
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35. The use of fondaparinux in pregnancy

Author

Elsaigh, Esra, primary, Thachil, Jecko, additional, Nash, Michael J., additional, Tower, Clare, additional, Hay, Charles R. M., additional, Bullough, Sian, additional, and Byrd, Louise, additional

Published
2014
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36. Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia a

Author

UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire, UCL - (SLuc) Service d'hématologie, UCL - (SLuc) Centre de malformations vasculaires congénitales, Eckhardt, Corien L., Van Velzen, Alice S., Peters, Marjolein, Astermark, Jan, Brons, Paul P., Castaman, Giancarlo, Cnossen, Marjon H., Dors, Natasja, Escuriola-Ettingshausen, Carmen, Hamulyak, Karly, Hart, Daniel P., Hay, Charles R. M., Haya, Saturnino, Van Heerde, Waander L., Hermans, Cédric, Holmström, Margareta, Jimenez-Yuste, Victor, Keenan, Russell D., Klamroth, Robert, Laros-van Gorkom, Britta A. P., Leebeek, Frank W. G., Liesner, Ri, Mäkipernaa, Anne, Male, Christoph, Mauser-Bunschoten, Evelien, Mazzucconi, Maria G., McRae, Simon, Meijer, Karina, Mitchell, Michael, Morfini, Massimo, Nijziel, Marten, Oldenburg, Johannes, Peerlinck, Kathelijne, Petrini, Pia, Platokouki, Helena, Reitter-Pfoertner, Sylvia E., Santagostino, Elena, Schinco, Piercarla, Smiers, Frans J., Siegmund, Berthold, Tagliaferri, Annarita, Yee, Thynn T., Kamphuisen, Pieter Willem, Van Der Bom, Johanna G., Fijnvandraat, Karin, UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire, UCL - (SLuc) Service d'hématologie, UCL - (SLuc) Centre de malformations vasculaires congénitales, Eckhardt, Corien L., Van Velzen, Alice S., Peters, Marjolein, Astermark, Jan, Brons, Paul P., Castaman, Giancarlo, Cnossen, Marjon H., Dors, Natasja, Escuriola-Ettingshausen, Carmen, Hamulyak, Karly, Hart, Daniel P., Hay, Charles R. M., Haya, Saturnino, Van Heerde, Waander L., Hermans, Cédric, Holmström, Margareta, Jimenez-Yuste, Victor, Keenan, Russell D., Klamroth, Robert, Laros-van Gorkom, Britta A. P., Leebeek, Frank W. G., Liesner, Ri, Mäkipernaa, Anne, Male, Christoph, Mauser-Bunschoten, Evelien, Mazzucconi, Maria G., McRae, Simon, Meijer, Karina, Mitchell, Michael, Morfini, Massimo, Nijziel, Marten, Oldenburg, Johannes, Peerlinck, Kathelijne, Petrini, Pia, Platokouki, Helena, Reitter-Pfoertner, Sylvia E., Santagostino, Elena, Schinco, Piercarla, Smiers, Frans J., Siegmund, Berthold, Tagliaferri, Annarita, Yee, Thynn T., Kamphuisen, Pieter Willem, Van Der Bom, Johanna G., and Fijnvandraat, Karin

Abstract

Neutralizing antibodies (inhibitors) toward factor VIII form a severe complication in nonsevere hemophilia A, profoundly aggravating the bleeding pattern. Identification of high-risk patients is hampered by lack of data that take exposure days to therapeutic factor VIII concentrates into account. In the INSIGHT study, we analyzed the association between F8 mutation and inhibitor development in patients with nonsevere hemophilia A (factor VIII 2-40 IU/dL). This analysis included 1112 non-severe hemophilia A patients from 14 centers in Europe and Australia that had genotyped at least 70% of their patients. Inhibitor risk was calculated as Kaplan-Meier incidence with cumulative number of exposure days as the time variable. During 44 800 exposure days (median, 24 exposure days per patient; interquartile range [IQR], 7-90), 59 of the 1112 patients developed an inhibitor; cumulative incidence of 5.3% (95% confidence interval [CI], 4.0-6.6) after a median of 28 exposure days (IQR, 12-71). The inhibitor risk at 50 exposure days was 6.7% (95% CI, 4.5-8.9) and at 100 exposure days the risk further increased to 13.3% (95% CI, 9.6-17.0). Among a total of 214 different F8 missense mutations 19 were associated with inhibitor development. These results emphasize the importance of F8 genotyping in nonsevere hemophilia A. ( Blood . 2013; 122(11):1954-1962) © 2013 by The American Society of Hematology.

Published
2013

37. Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A

Author

Eckhardt, Corien L., primary, van Velzen, Alice S., additional, Peters, Marjolein, additional, Astermark, Jan, additional, Brons, Paul P., additional, Castaman, Giancarlo, additional, Cnossen, Marjon H., additional, Dors, Natasja, additional, Escuriola-Ettingshausen, Carmen, additional, Hamulyak, Karly, additional, Hart, Daniel P., additional, Hay, Charles R. M., additional, Haya, Saturnino, additional, van Heerde, Waander L., additional, Hermans, Cedric, additional, Holmström, Margareta, additional, Jimenez-Yuste, Victor, additional, Keenan, Russell D., additional, Klamroth, Robert, additional, Laros-van Gorkom, Britta A. P., additional, Leebeek, Frank W. G., additional, Liesner, Ri, additional, Mäkipernaa, Anne, additional, Male, Christoph, additional, Mauser-Bunschoten, Evelien, additional, Mazzucconi, Maria G., additional, McRae, Simon, additional, Meijer, Karina, additional, Mitchell, Michael, additional, Morfini, Massimo, additional, Nijziel, Marten, additional, Oldenburg, Johannes, additional, Peerlinck, Kathelijne, additional, Petrini, Pia, additional, Platokouki, Helena, additional, Reitter-Pfoertner, Sylvia E., additional, Santagostino, Elena, additional, Schinco, Piercarla, additional, Smiers, Frans J., additional, Siegmund, Berthold, additional, Tagliaferri, Annarita, additional, Yee, Thynn T., additional, Kamphuisen, Pieter Willem, additional, van der Bom, Johanna G., additional, and Fijnvandraat, Karin, additional

Published
2013
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48. Acquired hemophilia A in the United Kingdom: a 2-year national surveillance study by the United Kingdom Haemophilia Centre Doctors' Organisation

Author

Collins, Peter W., Hirsch, Sybil, Baglin, Trevor P., Dolan, Gerard, Hanley, John, Makris, Michael, Keeling, David M., Liesner, Ri, Brown, Simon A., and Hay, Charles R. M.

Abstract

Acquired hemophilia A is a severe bleeding disorder caused by an autoantibody to factor VIII. Previous reports have focused on referral center patients and it is unclear whether these findings are generally applicable. To improve understanding of the disease, a 2-year observational study was established to identify and characterize the presenting features and outcome of all patients with acquired hemophilia A in the United Kingdom. This allowed a consecutive cohort of patients, unbiased by referral or reporting practice, to be studied. A total of 172 patients with a median age of 78 years were identified, an incidence of 1.48/million/y. The cohort was significantly older than previously reported series, but bleeding manifestations and underlying diseases were similar. Bleeding was the cause of death in 9% of the cohort and remained a risk until the inhibitor had been eradicated. There was no difference in inhibitor eradication or mortality between patients treated with steroids alone and a combination of steroids and cytotoxic agents. Relapse of the inhibitor was observed in 20% of the patients who had attained first complete remission. The data provide the most complete description of acquired hemophilia A available and are applicable to patients presenting to all centers.

Published
2007
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49. Emicizumab in Hemophilia A.

Author

Kruse-Jarres, Rebecca, Hay, Charles R. M., Buckner, Tyler W., Watson, Crystal, Recht, Michael, and Aledort, Louis M.

Subjects
*HEMOPHILIA, *IMMUNOGLOBULINS, *MONOCLONAL antibodies
Published
2020
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