19 December 2017

Novel gene therapy for haemophilia A

Professor John Pasi, of Barts and the London School of Medicine and Dentistry, UK, and colleagues reported on a novel gene therapy for adults with severe haemophilia A at the conference in Atlanta, Georgia, USA.

The team showed that a single infusion of the gene therapy, using a viral vector with a functional copy of the gene responsible for producing the essential blood clotting protein Factor VIII, led to improved levels of the protein. Out of the 13 patients treated, 11 achieved normal or near-normal Factor VIII levels over the following 19 months.

In addition, ten of the patients had no bleeding episodes requiring Factor VIII treatment from four weeks after infusion through the last follow-up visit.

Professor Pasi said: "The clinical data to date for this investigational gene therapy exceeded our expectations, in terms of increasing Factor VIII levels and reducing the annualised bleed rate.

“We are researching whether it may be possible for haemophilia A patients to reduce or eliminate Factor VIII treatment over an extended timeline."


Rivaroxaban for venous thromboembolism in cancer

A study found good results for the direct oral anti-coagulant rivaroxaban in treating venous thromboembolism in cancer patients. Dr Annie Young of the University of Warwick, UK, and her team recruited 406 patients who were randomly given either rivaroxaban or the low-molecular-weight heparin.

Venous thromboembolism rates were lower in those given rivaroxaban after six months of treatment - 4% versus 11% on heparin. However, the rivaroxaban group had more major bleeding events.

Dr Young said: "Clinicians are already adopting Direct Oral Anti-Coagulants into practice for these patients, and now they have data from this study to indicate that they are potentially safe in cancer patients."


Source: Studies presented at the 59th American Society of Hematology Annual Meeting in Atlanta, USA.

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