Article
Oncology
Marguerite Lockhart, Brigitte Tardy-Poncet, Aurelie Montmartin, Pauline Noyel, Sandrine Thouvenin, Claire Berger
Summary: For patients with severe hemophilia A receiving emicizumab treatment, it is important to adjust the dosage of bypassing agents based on thrombin-generation assay and collegial consultation to prevent bleeding risk before surgery.
PEDIATRIC BLOOD & CANCER
(2021)
Article
Hematology
Matthias M. Engelen, Johan Vandesande, Johan De Bent, Christine Van Laer, Veerle Labarque, Marc Jacquemin, Kathelijne Peerlinck, Cedric Hermans, Peter Verhamme, Thomas Vanassche
Summary: This single-center retrospective study reports the clinical and biochemical response of emicizumab in acquired hemophilia A (AHA). The results show that emicizumab can effectively control bleeding in patients with AHA and there are no safety issues.
Article
Hematology
Maissa Janbain, Nathalie Enjolras, Radu Bolbos, Marie Brevet, Jean-Claude Bordet, Yesim Dargaud
Summary: The combination of tranexamic acid (TxAc) and emicizumab showed improved hemostasis in FVIII knockout mice, reducing blood loss and joint bleeding compared to emicizumab alone. This suggests a potential benefit of using TxAc in prophylactic settings, especially for patients with breakthrough bleeds.
Article
Hematology
Christophe Schmitt, Thomas Emrich, Sammy Chebon, Elena Fernandez, Claire Petry, Koichiro Yoneyama, Anna Kiialainen, Monet Howard, Markus Niggli, Ido Paz-Priel, Tiffany Chang
Summary: Emicizumab has low immunogenicity risk in phase 3 studies, and antibodies, including neutralising antibodies, did not affect its safety profile. Routine surveillance is not necessary, but prompt evaluation by healthcare providers is recommended in cases of efficacy decline.
Article
Pharmacology & Pharmacy
Georg Gelbenegger, Ludwig Traby, Nina Rahimi, Paul Knoebl
Summary: This study presents a treatment approach for a 75-year-old male with severe Covid-19, acquired haemophilia A, and severe muscle bleeding. The treatment includes haemostatic therapy and immunosuppression. Different reagents are used to measure the activity of coagulation factor VIII, and recombinant human activated FVII and emicizumab are administered for haemostatic control. Steroids and rituximab are used for immunosuppression. The treatment is effective with no thromboembolic events.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
(2023)
Article
Pharmacology & Pharmacy
Cassandra P. Wang, Guy Young, Courtney D. Thornburg
Summary: Emicizumab is an effective alternative to prophylaxis with factor VIII replacement in individuals with hemophilia A, showing improved outcomes in reducing bleeding events. Despite a few cases of thrombotic microangiopathy and thrombotic events seen in the HAVEN 1 trial, overall, Emicizumab has an excellent safety profile with minimal side effects.
EXPERT OPINION ON DRUG SAFETY
(2021)
Review
Hematology
Amandine Hansenne, Cedric Hermans
Summary: Acquired haemophilia A is a rare and severe hemorrhagic autoimmune disease caused by autoantibodies against factor VIII. Treatment involves hemostatic control and immunosuppressive therapy, which can be challenging given the elderly population with comorbidities. Emicizumab, a bispecific antibody, has shown promise in managing AHA patients with comorbidities.
THERAPEUTIC ADVANCES IN HEMATOLOGY
(2021)
Article
Hematology
Jane A. Mason, Guy Young
Summary: This report highlights the real-world experience of using emicizumab for bleed prevention in infants with severe hemophilia A, showcasing its potential in shifting the prophylaxis paradigm in this age group.
Article
Hematology
Hande Kizilocak, Elizabeth Marquez-Casas, Jemily Malvar, Roxana Carmona, Guy Young
Summary: This study aimed to predict the approximate FVIII level in severe hemophilia A patients with inhibitors on emicizumab using global hemostasis assays. Results showed that all patients had F8EmT >10%, indicating conversion to a mild hemophilia phenotype, with a negative correlation between F8EmT and patient weight.
Article
Medicine, General & Internal
Ahmed Sobhi Hassan, Laila Metwally Sherief, Mona Hamdy, Abeer Salamah, Osama A. El-Agamy
Summary: This study assessed the effect of Emicizumab on physical activity in children with severe haemophilia A and found that Emicizumab prophylaxis improved their level of physical activity.
JOURNAL OF THE PAKISTAN MEDICAL ASSOCIATION
(2023)
Article
Hematology
Jan Astermark, Tyler W. Buckner, Laurent Frenzel, Anthony J. Hatswell, Xiaojun You, Hai Liu, Erin Goodman, Sandra Santos, Charles Hawes, David Hinds, Robert Klamroth
Summary: This study indirectly compared the bleeding rates between valoctocogene roxaparvovec and emicizumab, two treatments for haemophilia A. The results showed that valoctocogene roxaparvovec had lower bleeding rates and higher proportions of participants with no joint bleeds compared to emicizumab. Emicizumab was also more effective than FVIII prophylaxis regimens.
Article
Hematology
Eman Hassan, Lancashire Jonathan, Motwani Jayashree
Summary: The study showed that among 52 patients, 4 experienced minor adverse events with headaches, abdominal pain, nausea, and injection site reactions. 4 patients experienced major adverse events, including severe headaches, major bleeding events, development of ADAs, and recurrence of inhibitors. Emicizumab prophylaxis was discontinued in 3 patients due to adverse events, and in 1 patient due to poor compliance. No adverse events were reported in 4 previously untreated/minimally treated patients in the cohort.
Article
Hematology
Benjamin J. Samelson-Jones, Christine Guelcher, Jan Kuhn, Regina Butler, Gita Massey, Michael F. Guerrera, Leslie Raffini
Summary: This study examined the estimated costs of starting emicizumab in real-world hemophilia A patients, finding a decrease in total costs and especially in high-cost outliers. The cost of prophylaxis and total cost of bleeds also significantly decreased after starting emicizumab, for both patients with and without inhibitors.
Article
Hematology
Caroline Wall, Hua Xiang, Ben Palmer, Elizabeth Chalmers, Pratima Chowdary, Peter W. Collins, Simon Fletcher, Georgina W. Hall, Daniel P. Hart, Mary Mathias, Paul Sartain, Susan Shapiro, David Stephensen, Kate Talks, Charles R. M. Hay
Summary: The UK National Haemophilia Database (NHD) collects data from all UK persons with haemophilia A with inhibitors (PwHA-I). This study aims to determine the safety, bleeding outcomes, and early effects on joint health of emicizumab prophylaxis in a large, unselected cohort using national registry data. The results show that emicizumab prophylaxis is associated with sustained low bleeding rates and was generally well-tolerated. Rating: 9 out of 10.
Article
Hematology
Susanna Ranta, Jayashree Motwani, Jan Blatny, Martina Buehrlen, Manuel Carcao, Herve Chambost, Carmen Escuriola, Kathelijn Fischer, Mutlu Kartal-Kaess, Marloes de Kovel, Gili Kenet, Christoph Male, Beatrice Nolan, Roseline d'Oiron, Martin Olivieri, Ester Zapotocka, Nadine G. Andersson, Christoph Koenigs
Summary: Most haemophilia treatment centres prefer to initiate prophylaxis with emicizumab before 12 months of age and without concomitant FVIII. In inhibitor patients, ITI is commonly used in addition to emicizumab, but there is no common practice on how to proceed after successful ITI.
