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A. Parnes, J. Mahlangu, S. Pipe, I. Paz‐Priel, M. Lehle, P. Trask, V. Jiménez‐Yuste (2021)
Patient preference for emicizumab versus prior factor therapy in people with haemophilia A: Results from the HAVEN 3 and HAVEN 4 studiesHaemophilia, 27
A. Shapiro, Ian Mitchell, Sonia Nasr (2018)
The future of bypassing agents for hemophilia with inhibitors in the era of novel agentsJournal of Thrombosis and Haemostasis, 16
S. Royal, W. Schramm, E. Berntorp, P. Giangrande, A. Gringeri, C. Ludlam, B. Kroner, T. Szucs (2002)
Quality‐of‐life differences between prophylactic and on‐demand factor replacement therapy in European haemophilia patientsHaemophilia, 8
V. Jiménez‐Yuste, R. Klamroth, G. Castaman, J. Hovinga, C. Shanmukhaiah, S. Rangarajan, J. Chavez, R. Martinez, G. Kenet, S. Robson, C. Schmitt, O. Meier, M. Ozelo (2021)
Second interim analysis results from the STASEY trial: A single-arm, multicentre, open-label, phase III clinical trial to evaluate the safety and tolerability of emicizumab prophylaxis in persons with haemophilia A (PwHA) with FVIII inhibitorsHämostaseologie, 41
C. Schmitt, J. Adamkewicz, Jin Xu, Claire Petry, O. Catalani, G. Young, C. Négrier, M. Callaghan, G. Levy (2020)
Pharmacokinetics and Pharmacodynamics of Emicizumab in Persons with Hemophilia A with Factor VIII Inhibitors: HAVEN 1 StudyThrombosis and Haemostasis, 121
P. Cortesi, G. Castaman, G. Trifirò, S. Creazzola, G. Improta, G. Mazzaglia, A. Molinari, L. Mantovani (2019)
Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with InhibitorsThrombosis and Haemostasis, 120
J. Oldenburg, J. Mahlangu, W. Buján, P. Trask, M. Callaghan, G. Young, E. Asikanius, F. Peyvandi, E. Santagostino, R. Kruse-Jarres, C. Négrier, C. Kessler, Jin Xu, J. Windyga, M. Shima, S. Mackensen (2018)
The effect of emicizumab prophylaxis on health‐related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 StudyHaemophilia, 25
P. Lenting (2020)
Laboratory monitoring of hemophilia A treatments: new challenges.Blood advances, 4 9
R. Nuss, H. Hedegaard, B. Riske, R. Hoffman, S. Michael, M. Manco‐Johnson (1998)
Medical care for haemophiliaHaemophilia, 4
G. Batsuli, K. Zimowski, Kelly Tickle, S. Meeks, R. Sidonio (2019)
Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxisHaemophilia, 25
M. Witkop, Anne Neff, T. Buckner, Michael Wang, Katharine Batt, C. Kessler, Doris Quon, Lisa Boggio, Michael Recht, K. Baumann, R. Gut, David Cooper, Christine Kempton (2017)
Self‐reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P‐FiQ) studyHaemophilia, 23
A. Srivastava, E. Santagostino, A. Dougall, S. Kitchen, M. Sutherland, S. Pipe, M. Carcao, J. Mahlangu, M. Ragni, J. Windyga, A. Llinás, N. Goddard, Richa Mohan, P. Poonnoose, B. Feldman, Sandra Lewis, H. Berg, Glenn Pierce (2020)
WFH Guidelines for the Management of Hemophilia, 3rd editionHaemophilia, 26
T. Lambert, G. Benson, G. Dolan, C. Hermans, V. Jiménez‐Yuste, R. Ljung, M. Morfini, S. Zupancic-Salek, E. Santagostino (2018)
Practical aspects of extended half-life products for the treatment of haemophiliaTherapeutic Advances in Hematology, 9
G. Batsuli, A. Greene, S. Meeks, R. Sidonio (2021)
Emicizumab in tolerized patients with hemophilia A with inhibitors: A single‐institution pediatric cohort assessing inhibitor statusResearch and Practice in Thrombosis and Haemostasis, 5
Isabella McCary, C. Guelcher, J. Kuhn, R. Butler, G. Massey, M. Guerrera, Lance Ballester, L. Raffini (2020)
Real‐world use of emicizumab in patients with haemophilia A: Bleeding outcomes and surgical proceduresHaemophilia, 26
M. Carcao, C. Escuriola-Ettingshausen, E. Santagostino, J. Oldenburg, R. Liesner, B. Nolan, A. Bátorová, S. Haya, G. Young (2019)
The changing face of immune tolerance induction in haemophilia A with the advent of emicizumabHaemophilia, 25
K. Knobe, E. Berntorp (2011)
Haemophilia and joint disease: pathophysiology, evaluation, and managementJournal of Comorbidity, 1
Patrick Ebbert, F. Xavier, Craig Seaman, M. Ragni (2019)
Emicizumab prophylaxis in patients with haemophilia A with and without inhibitorsHaemophilia, 26
S. Lacroix-Desmazes, J. Voorberg, D. Lillicrap, D. Scott, K. Pratt (2020)
Tolerating Factor VIII: Recent ProgressFrontiers in Immunology, 10
J. Oldenburg, M. Shima, R. Kruse-Jarres, E. Santagostino, J. Mahlangu, M. Lehle, Nives Bienz, S. Chebon, E. Asikanius, P. Trask, M. Mancuso, V. Jiménez‐Yuste, S. Mackensen, G. Levy (2020)
Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional studyPediatric Blood & Cancer, 67
G. Young, R. Liesner, Tiffany Chang, R. Sidonio, J. Oldenburg, V. Jiménez‐Yuste, J. Mahlangu, R. Kruse-Jarres, Michael Wang, Marianne Uguen, M. Doral, Lilyan Wright, C. Schmitt, G. Levy, M. Shima, M. Mancuso (2019)
A multicenter, open-label, phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors.Blood
J. Wight, S. Paisley (2003)
The epidemiology of inhibitors in haemophilia A: a systematic reviewHaemophilia, 9
A. Barg, T. Livnat, I. Budnik, Einat Avishai, T. Brutman-Barazani, I. Tamarin, D. Bashari, M. Misgav, G. Kenet (2020)
Emicizumab treatment and monitoring in a paediatric cohort: real‐world dataBritish Journal of Haematology, 191
T. Kitazawa, T. Igawa, Zenjiro Sampei, A. Muto, T. Kojima, T. Soeda, K. Yoshihashi, Yukiko Okuyama-Nishida, Hiroyuki Saito, H. Tsunoda, Tsukasa Suzuki, H. Adachi, Taro Miyazaki, Shinya Ishii, Mika Kamata-Sakurai, T. Iida, Aya Harada, Keiko Esaki, Miho Funaki, Chifumi Moriyama, Eriko Tanaka, Y. Kikuchi, Tetsuya Wakabayashi, M. Wada, M. Goto, T. Toyoda, Atsunori Ueyama, Sachiyo Suzuki, K. Haraya, Tatsuhiko Tachibana, Y. Kawabe, M. Shima, A. Yoshioka, K. Hattori (2012)
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A modelNature Medicine, 18
K. Kelley, Belinda Clark, V. Brown, J. Sitzia (2003)
Good practice in the conduct and reporting of survey research.International journal for quality in health care : journal of the International Society for Quality in Health Care, 15 3
M. Manco‐Johnson, B. Lundin, S. Funk, C. Peterfy, D. Raunig, M. Werk, C. Kempton, M. Reding, S. Goranov, L. Gercheva, L. Rusen, V. Uscăţescu, M. Pierdominici, S. Engelen, J. Pocoski, D. Walker, W. Hong (2017)
Effect of late prophylaxis in hemophilia on joint status: a randomized trialJournal of Thrombosis and Haemostasis, 15
M. Mancuso, J. Mahlangu, R. Sidonio, P. Trask, Marianne Uguen, Tiffany Chang, M. Shima, G. Young, J. Oldenburg, S. Mackensen (2020)
Health‐related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors—Results from the HAVEN 2 studyHaemophilia, 26
Pei-Chin Lin, Yu‐Mei Liao, S. Tsai, Tai‐Tsung Chang (2011)
Immune tolerance induction therapy for patients with hemophilia A and FVIII inhibitors particularly using low‐dose regimensPediatric Blood & Cancer, 57
M. Skinner, C. Négrier, I. Paz‐Priel, S. Chebon, V. Jiménez‐Yuste, M. Callaghan, M. Lehle, M. Niggli, J. Mahlangu, A. Shapiro, M. Shima, A. Campinha-Bacote, G. Levy, J. Oldenburg, S. Mackensen, S. Pipe (2021)
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 studiesHaemophilia, 27
Zheng-Yi Zhou, K. Raimundo, Anisha Patel, Simeng Han, Yusi Ji, H. Fang, J. Zhong, K. Betts, A. Mahajerin (2020)
Model of Short- and Long-Term Outcomes of Emicizumab Prophylaxis Treatment for Persons with Hemophilia AJournal of Managed Care & Specialty Pharmacy, 26
M. Callaghan, C. Négrier, I. Paz‐Priel, Tiffany Chang, S. Chebon, M. Lehle, J. Mahlangu, G. Young, R. Kruse-Jarres, M. Mancuso, M. Niggli, Monet Howard, Nives Bienz, M. Shima, V. Jiménez‐Yuste, C. Schmitt, E. Asikanius, G. Levy, S. Pipe, J. Oldenburg (2020)
Long-term outcomes with emicizumab prophylaxis for hemophilia A with/without FVIII inhibitors from the HAVEN 1-4 studies.Blood
M. García-Dasí, J. Aznar, V. Jiménez‐Yuste, C. Altisent, S. Bonanad, E. Mingot, F. Lucía, F. Giménez, M. López, P. Marco, R. Pérez, M. Fernández, M. Paloma, B. Galmés, S. Herrero, J. García-Talavera (2015)
Adherence to prophylaxis and quality of life in children and adolescents with severe haemophilia AHaemophilia, 21
Anisha Patel, S. Corman, S. Chaplin, K. Raimundo, R. Sidonio (2019)
Economic impact model of delayed inhibitor development in patients with hemophilia a receiving emicizumab for the prevention of bleeding eventsJournal of Medical Economics, 22
AbstractBackgroundEmicizumab is a bispecific monoclonal antibody approved in the United States (US) for the treatment of people with haemophilia A (PwHA) with or without factor VIII (FVIII) inhibitors. Changes to haematologists’ practices since the approval of emicizumab are of interest to the haemophilia A community.AimTo identify the clinical characteristics of PwHA receiving emicizumab (PwHArE) in the real-world setting and gain insight into the disease management practices of haematologists treating PwHArE.MethodsIn total, 50 haematologists across the US completed a one-time, 30-minute, online, qualitative survey consisting of 55 questions (including 11 screening questions) in May 2019. Haematologists were required to be board-certified in haematology, practising in the US, ≥2 years post-residency experience, and currently treating ≥3 PwHA with emicizumab.ResultsHaematologists reported their PwHArE were mostly adults (aged ≥18 years; 66%) with severe phenotypes (66%), with and without FVIII inhibitors. Haematologists perceived that PwHArE had similar or better treatment adherence (40% and 50%, respectively) compared with PwHA on other treatments, sought the same or lower levels of routine care (72% and 14%, respectively), and were similarly or more physically active (52% and 32%, respectively). Additionally, most haematologists currently using immune tolerance induction (ITI) in PwHArE reported using lower doses of FVIII (73%) and shorter durations (45%) for ITI.ConclusionsAvailability of emicizumab has resulted in changes in the care of PwHArE, including bleed management, FVIII monitoring, activity guidance, surgery, and use of ITI. Understanding patterns of disease management can inform clinical care.
The Journal of Haemophilia Practice – de Gruyter
Published: Jan 1, 2021
Keywords: emicizumab; haemophilia A; physicians; surveys and questionnaires
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