HemFitBit, an Observational Study of Physical Activity in People With Moderate and Severe Haemophilia A
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
CONCLUSIONS: This regimen may be considered to induce FVIII tolerance in HA patients with refractory inhibitors. Further characterization of the FVIII-specific immune response is necessary to clarify the mechanism of immune tolerance. PMID: 31985872 [PubMed - as supplied by publisher]
CONCLUSION: Emicizumab improved ex-vivo coagulation potential in plasma from AHA patients. PMID: 31984625 [PubMed - as supplied by publisher]
Conclusions: Valoctocogene roxaparvovec was found to be cost-saving-on average by about $6.8 million per patient-and more effective than prophylactic therapy for treatment of hemophilia A. The comparative benefit of gene therapy was observed across a broad range of simulated patients that were representative of the real-world severe hemophilia A population. PMID: 31971453 [PubMed - as supplied by publisher]
Publication date: 25–31 January 2020Source: The Lancet, Volume 395, Issue 10220Author(s): Geoffrey C Colin, Patrick Mailleux, Anne-Catherine Bafort, Christophe Van Bellinghen, François Buche
Condition: Hemophilia Intervention: Other: Experimental group Sponsor: Real Fundación Victoria Eugenia Not yet recruiting
Abstract Hemophilia B is an X-linked recessive bleeding disorder caused by abnormalities in the coagulation factor IX gene. Without prophylactic treatment, patients experience frequent spontaneous bleeding episodes. Well-characterized animal models are valuable for determining the pathobiology of the disease and testing novel therapeutic innovations. Here, we generated a porcine model of hemophilia B using a combination of CRISPR/Cas9 and somatic cell nuclear transfer. Moreover, we tested the possibility of hemophilia B therapy by gene insertion. Frequent spontaneous joint bleeding episodes that occurred in hemoph...
Notice NOT-HL-19-747 from the NIH Guide for Grants and Contracts
Authors: Rodriguez-Merchan EC Abstract Introduction: Hemophilia is a very expensive disease. Ameliorations in the management of hemophilia causes increased patient survival, more complex disease management, and augmented treatment costs. A literature search related to the cost of hemophilia was analyzed.Covered areas: Clotting factor concentrates, which are the keystone of management, are costly. In fact, the yearly expenditure in the USA is 250,000 USD per adult patient. Indirect expenses also play a part to the economic load, and include lost productive capacity, uncompensated caretaker tariffs, and hemophilia-re...