Efanesoctocog alfa in hemophilia A: a profile of its use
AbstractEfanesoctocog alfa [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl; ALTUVIIIO®], indicated for routine prophylaxis, on-demand treatment of bleeding episodes, and perioperative management of bleeding, is a useful addition to the therapies available for the management of hemophilia A in adults and children. Its half-life is three- to four-fold longer than existing (standard and extended half-life) factor VIII replacement products, which enables once-weekly administration. In a phase 3 trial, pretreated adolescents and adults (aged ≥ 12 years) receiving efanesoctocog alfa prophylaxis had a l...
Source: Drugs and Therapy Perspectives - February 24, 2024 Category: Drugs & Pharmacology Source Type: research
Comparison of thrombotic adverse events in patients treated with factor VIII products and emicizumab using the 2018-2022 FAERS data
CONCLUSION: Thrombotic AEs in the FAERS data were about three times more frequently reported for emicizumab than for FVIII products. More research and efforts in the future are warranted for monitoring, elucidating, and preventing the potential risk of thrombotic AEs of hemophilia therapy, including emicizumab.PMID:38395359 | DOI:10.1016/j.jtha.2024.02.009 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - February 23, 2024 Category: Hematology Authors: Hyunjeong Cho Ki Young Yoo Ju-Young Shin Eun-Kyoung Lee BongKyoo Choi Source Type: research
Innovative Therapies for Acquired Hemophilia A
Semin Thromb Hemost DOI: 10.1055/s-0044-1779737Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder which can be life-threatening. AHA is due to autoantibodies against coagulation factor VIII. Disease onset may be idiopathic (approximately half of the cases) or triggered by autoimmune disorders, cancers, drugs, infections, or pregnancy. Besides treating the underlying disorder, specific AHA treatments include management of bleeding and inhibitor eradication. Various first-line and second-line hemostatic and immunosuppressive agents are currently available for the management of AHA. Recently, the hemostatic dr...
Source: Seminars in Thrombosis and Hemostasis - February 23, 2024 Category: Hematology Authors: Franchini, Massimo Focosi, Daniele Tags: Review Article Source Type: research
ADH-Enhancer: an attention-based deep hybrid framework for enhancer identification and strength prediction
Brief Bioinform. 2024 Jan 22;25(2):bbae030. doi: 10.1093/bib/bbae030.ABSTRACTEnhancers play an important role in the process of gene expression regulation. In DNA sequence abundance or absence of enhancers and irregularities in the strength of enhancers affects gene expression process that leads to the initiation and propagation of diverse types of genetic diseases such as hemophilia, bladder cancer, diabetes and congenital disorders. Enhancer identification and strength prediction through experimental approaches is expensive, time-consuming and error-prone. To accelerate and expedite the research related to enhancers iden...
Source: Briefings in Bioinformatics - February 22, 2024 Category: Bioinformatics Authors: Faiza Mehmood Shazia Arshad Muhammad Shoaib Source Type: research
ADH-Enhancer: an attention-based deep hybrid framework for enhancer identification and strength prediction
Brief Bioinform. 2024 Jan 22;25(2):bbae030. doi: 10.1093/bib/bbae030.ABSTRACTEnhancers play an important role in the process of gene expression regulation. In DNA sequence abundance or absence of enhancers and irregularities in the strength of enhancers affects gene expression process that leads to the initiation and propagation of diverse types of genetic diseases such as hemophilia, bladder cancer, diabetes and congenital disorders. Enhancer identification and strength prediction through experimental approaches is expensive, time-consuming and error-prone. To accelerate and expedite the research related to enhancers iden...
Source: Briefings in Bioinformatics - February 22, 2024 Category: Bioinformatics Authors: Faiza Mehmood Shazia Arshad Muhammad Shoaib Source Type: research
The spectrum of neutralizing and non-neutralizing anti-FVIII antibodies in a nationwide cohort of 788 persons with hemophilia A
ConclusionIn this cross-sectional study, we identified non-neutralizing antibodies in a relatively large proportion of persons with hemophilia A. In contrast, in our population, consisting of persons highly exposed to FVIII concentrates, (very low-titer) inhibitors were detected only in a small proportion of persons, reflecting a well-tolerized population. Hence, our findings suggest that only a small subpopulation of non-neutralizing FVIII-specific antibodies is associated with clinically relevant inhibitors. (Source: Frontiers in Immunology)
Source: Frontiers in Immunology - February 22, 2024 Category: Allergy & Immunology Source Type: research
Real-world long-term safety and effectiveness of turoctocog alfa in the treatment of haemophilia A in Japan: results from a multicentre, non-interventional, post-marketing study
CONCLUSION: Long-term use of turoctocog alfa therapy in clinical practice posed no newly identified safety issues and was effective for prophylaxis and treatment of bleeds in patients with haemophilia A in Japan.PMID:38376107 | DOI:10.1080/16078454.2024.2316540 (Source: Hematology)
Source: Hematology - February 20, 2024 Category: Hematology Authors: Azusa Nagao Ayumi Deguchi Keiji Nogami Source Type: research
Recent and anticipated novel drug approvals for 2024
CONCLUSION: More oncology agents, including gene therapies, oral agents, and monoclonal antibodies, are in the pipeline this year. Additional diseases targeted by new novel drugs, including cellular and gene therapies, are hemophilia, nonalcoholic steatohepatitis, Alzheimer's disease, and rare diseases such as galactosemia and epidermolysis bullosa.PMID:38373160 | DOI:10.1093/ajhp/zxae046 (Source: American Journal of Health-System Pharmacy : AJHP)
Source: American Journal of Health-System Pharmacy : AJHP - February 19, 2024 Category: Drugs & Pharmacology Authors: Matthew H Rim Brittany L Karas Farah Barada Andrew M Levitsky Source Type: research
A Generative and Causal Pharmacokinetic Model for Factor VIII in Hemophilia A: A Machine Learning Framework for Continuous Model Refinement
In conclusion, the proposed approach introduces interesting new possibilities for model development. In the context of rare disease, the introduction of generative models facilitates sharing of synthetic data, enabling the iterative improvement of population PK models.PMID:38372445 | DOI:10.1002/cpt.3203 (Source: Clinical Pharmacology and Therapeutics)
Source: Clinical Pharmacology and Therapeutics - February 19, 2024 Category: Drugs & Pharmacology Authors: Alexander Janssen Louk Smalbil Frank C Bennis Marjon H Cnossen Ron A A Math ôt OPTI-CLOT study group and SYMPHONY consortium Source Type: research
