Relationship Between Single-Nucleotide Polymorphisms of Tumor Necrosis Factor Alpha, Interleukin-10, Factor II and Factor V with Risk of Inhibitor Development in Patients with Severe Hemophilia A
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.
AbstractInherited bleeding disorders are not uncommon in pediatric practice: most of them being chronic, require lifelong replacement therapy. To frame a management policy, it is essential to assess the load and pattern of bleeding disorders in the local population. However, there is paucity of data reporting the clinical spectrum of coagulation and platelet function disorders in Indian children. Hence to find out the exact burden and clinico-investigational profile of these patients we conducted this study. In this retrospective case review, detailed clinical information was extracted from case records in 426 children wit...
ConclusionsThis analysis evaluated the pharmacokinetics of BAY 94-9027 and its sources of IIV. Using the model, determination of individual patient pharmacokinetics was possible with few FVIII samples, and a sparse sampling design to support pharmacokinetic-guided dosing was identified.