Routine clinical care data for population pharmacokinetic modeling: the case for Fanhdi/Alphanate in hemophilia A patients
This study shows the feasibility of using real-world data for the development of a population PK model. Evaluation and comparison of the model for Bayesian forecasting resulted in similar results as a model developed using rich sampling data.
This study has been followed by unparalleled advances in gene therapy for hemophilia A and B, leading to clotting factor activity approaching normal or near-normal levels associated with a "zero bleed rates" in previously severely affected patients following a single administration of AAV vectors. Thus, AAV gene therapies are likely to alter the treatment paradigm for hemophilia A and B. This review explores recent progress and the remaining limitations that need to be overcome for wider availability of this novel treatment of inherited bleeding disorders. PMID: 31808868 [PubMed - in process]
Authors: Menegatti M, Biguzzi E, Peyvandi F Abstract Autoantibodies toward clotting factors may develop in people suffering from autoimmune or neoplastic diseases, after drug intake or even in subjects without apparent conditions. They are more commonly directed against factor VIII (FVIII) or von Willebrand factor leading to acquired hemophilia A or acquired von Willebrand syndrome, respectively. Rarely, autoantibodies develop against other clotting factors, such as fibrinogen, FII, FV, FVII, FX, FXI, and FXIII. The clinical picture of an acquired bleeding disorder includes a wide spectrum of clinical manifestation...
Condition: Adult Patients (>= 18 Years) Diagnosed With Acquired Hemophilia A (AHA) Based on a Reduced FVIII Activity and a Positive Nijmegen Bethesda Inhibitor Titer Intervention: Drug: Emicizumab Injection Sponsors: GWT-TUD GmbH; Hoffmann-La Roche; Hannover Medical School Not yet recruiting
Authors: Singh A, Mehta S, Goyal LK, Mehta S, Sharma BS Abstract Aim: To assess effect of low dose prophylaxis in hemophilics in terms of bleeding, joint function, QoL and cost-effectiveness. Methods: Analytic study done during one year among 70 adult hemophilics. In observation period (12 weeks), on-demand factor and during prophylaxis (12 weeks), low dose factor was given (Factor VIII 10 IU/KgBW biweekly for haemophilia A and Factor IX 20 IU/KgBW weekly for haemophilia B). Clinical joint assessment was done by Gilbert score and improvement by WFH definitions. Results: Bleed reduced by 68.99% in moderate h...
Conclusions: The results of this PMS analysis in Japan support its known safety profile and identified no new safety risks for people living with HIV/AIDS in Japan currently on, or beginning treatment with, fosamprenavir. PMID: 31794279 [PubMed - as supplied by publisher]
CONCLUSION: Our results suggest a potential benefit of TXA when used in combination with FVIII in prophylactic setting. PMID: 31782901 [PubMed - as supplied by publisher]
CONCLUSION: These data support a role for microRNAs in fine-tuning F8 gene regulation. Based on our findings, our current model suggests that in HA cases where the F8 gene is normal and is predicted to express normal levels of FVIII, F8 mRNA 3' UTR targeting miRNAs may be responsible for a FVIII-deficiency phenotype clinically manifesting as HA. PMID: 31785023 [PubMed - as supplied by publisher]
Condition: Hemophilia A Without Inhibitor Interventions: Device: PA measurement with Fitbit Charge 3; Device: PA measurement with Fitbit and ActiGraph GT3X Sponsors: Oslo University Hospital; Norwegian School of Sport Sciences Enrolling by invitation
ConclusionThe results demonstrated that turoctocog alfa can be subjected to variable storage conditions, including cycling between 5 °C and ≤ 40 °C, and subsequent storage for 3 months up to 40 °C, without loss of stability. This suggests that turoctocog alfa may offer greater product storage flexibility for patients in everyday practice, with a potential reduction in wastage.
Human Gene Therapy, Ahead of Print.