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The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study
CONCLUSION: These data demonstrate the long-term effectiveness and safety of octocog alfa in patients with moderate and severe hemophilia A, especially in those receiving prophylaxis. The high number of patients receiving on-demand treatment experiencing zero bleeds could be due to selection bias within the study, with patients with less severe disease more likely to be receiving on-demand treatment.TRIAL REGISTRATION: ClinicalTrials.gov: NCT02078427.PMID:38371314 | PMC:PMC10874143 | DOI:10.1177/20406207231218624 (Source: Adv Data)
Source: Adv Data - February 19, 2024 Category: Epidemiology Authors: Margareth C Ozelo Cedric Hermans Manuel Carcao Beno ît Guillet Joan Gu Randy Guerra Leilei Tang Kate Khair 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

GSE242926 The Role of microRNAs in Defining LSECs Cellular Identity and in Regulating F8 Gene Expression
Contributors : Muhammad A Jamil ; Osman El-MaarriSeries Type : Non-coding RNA profiling by high throughput sequencingOrganism : Homo sapiensCoagulation Factor VIII (FVIII) plays a pivotal role within the coagulation cascade, and deficiencies in its levels, as seen in Hemophilia A, can lead to significant health implications. Liver sinusoidal endothelial cells (LSECs) are the main producers and contributors of FVIII in blood, a fact we have previously elucidated through mRNA expression profiling when comparing these cells to other endothelial cell types. Our current investigation delves into small microRNAs, analyzing thei...
Source: GEO: Gene Expression Omnibus - February 19, 2024 Category: Genetics & Stem Cells Tags: Non-coding RNA profiling by high throughput sequencing Homo sapiens Source Type: research

The role of microRNAs in defining LSECs cellular identity and in regulating F8 gene expression
Discussion: Our investigation identified a panel of 10 microRNAs (miR-429, miR-200b-3p, miR-200a-3p, miR-216b-5p, miR-1185-5p, miR-19b-3p, miR-192-5p, miR-122-5p, miR-30c-2-3p, and miR-30a-5p) that distinctly define LSEC identity. Furthermore, our scrutiny extended to microRNAs implicated in F8 regulation, revealing a subset (miR-122-5p, miR-214-3p, miR-204-3p, and miR-2682-5p) whose expression intricately correlates with F8 expression within LSECs. This microRNA cohort emerges as a crucial modulator of F8, both directly through suppression and indirect effects on established F8-related transcription factors. The above mic...
Source: Frontiers in Genetics - February 19, 2024 Category: Genetics & Stem Cells Source Type: research

Development of a Coagulation Disorders Unit
Semin Thromb Hemost DOI: 10.1055/s-0044-1779633Our Coagulation Disorders Unit in Helsinki, Finland, provides 24/7 services for local and national hospitals and colleagues upon requests regarding bleeding and thrombosis diagnostics and management, including follow-up. The unit has a tight connection between the clinic and laboratory, and its maintenance and sharing knowledge and observations have been priorities, already for over 20 years and will continue to be of major importance. The consultation service is provided by phone during daytime and on-call hours, and in written form sent electronically to the consulting stake...
Source: Seminars in Thrombosis and Hemostasis - February 19, 2024 Category: Hematology Authors: Szanto, Timea Helin, Tuukka Joutsi-Korhonen, Lotta Lehtinen, Anna-Elina El Beayni, Nancy Lep äntalo, Aino Lassila, Riitta Tags: Review Article Source Type: research

Pharmacokinetic –Pharmacodynamic Modelling in Hemophilia A: Relating Thrombin and Plasmin Generation to Factor VIII Activity After Administration of a VWF/FVIII Concentrate
ConclusionOur predictive performance displayed adequate thrombin pharmacodynamic predictions of the original model, but a new pharmacokinetic model was required. The pharmacodynamic model is not factor specific and applicable to multiple factor concentrates. A prospective study is needed to validate the impact of the FVIII dosing pharmacodynamic model on bleeding reduction in patients. (Source: European Journal of Drug Metabolism and Pharmacokinetics)
Source: European Journal of Drug Metabolism and Pharmacokinetics - February 17, 2024 Category: Drugs & Pharmacology Source Type: research

Decoding the complexity of on-target integration: characterizing DNA insertions at the CRISPR-Cas9 targeted locus using nanopore sequencing
CRISPR-Cas9 technology has advanced in vivo gene therapy for disorders like hemophilia A, notably through the successful targeted incorporation of the F8 gene into the Alb locus in hepatocytes, effectively cur... (Source: BMC Genomics)
Source: BMC Genomics - February 17, 2024 Category: Genetics & Stem Cells Authors: Juan-Juan Zhao, Xin-Yu Sun, Sai-Ning Tian, Zong-Ze Zhao, Meng-Di Yin, Mei Zhao, Feng Zhang, Si-Ang Li, Zhi-Xue Yang, Wei Wen, Tao Cheng, An Gong, Jian-Ping Zhang and Xiao-Bing Zhang Tags: Research Source Type: research

Efanesoctocog alfa: the renaissance of Factor VIII replacement therapy
Haematologica. 2024 Feb 15. doi: 10.3324/haematol.2023.284498. Online ahead of print.ABSTRACTEfanesoctocog alfa (ALTUVIIIOTM, Sanofi-SOBI) is a B domain-deleted single-chain Factor VIII (FVIII) connected to D'D3 domain of von Willebrand Factor (VWF). Its ingenious design allows efanesoctocog alfa to operate independently of endogenous VWF and results in an outstanding 3-4 times longer half-life compared to standard and extended half-life (EHL) FVIII products. The prolonged half-life ensures sustained high levels of factor activity, maintaining normal to near-normal ranges for the majority of the week, facilitating the conv...
Source: Haematologica - February 15, 2024 Category: Hematology Authors: Yesim Dargaud Alexandre Leuci Alejandra Reyes Ruiz Sebastien Lacroix-Desmazes Source Type: research