Orlando skyline at night, Photo by Bill Dickinson.

The World Federation of Hemophilia (WFH) 2016 World Congress took place July 24-28 in Orlando, Florida.

Drug can prevent bleeding in hemophilia A and B

Structure of RNA

Results from an ongoing phase 1 study suggest fitusiran, a small interfering RNA therapeutic targeting antithrombin (AT), can restore hemostasis and prevent bleeding in patients with hemophilia A or B, with or without inhibitors. In patients with inhibitors, fitusiran exhibited preliminary evidence of reduced bleeding. In patients without inhibitors, fitusiran reduced the... [Read Article]

Gene therapy reduces need for FIX prophylaxis

DNA helices
Image courtesy of NIGMS

The gene therapy AMT-060 can reduce the need for factor IX (FIX) prophylaxis in patients with severe hemophilia B, results of a phase 1/2 study suggest. All of the patients treated in the low-dose cohort of this study have had sustained improvements in their disease phenotype and continue... [Read Article]

Gene therapy shows promise for severe hemophilia A

DNA helix
Image by Spencer Phillips

An investigational gene therapy can safely reduce bleeding in patients with severe hemophilia A, a phase 1/2 study suggests. The therapy is BMN 270, a recombinant adeno-associated virus (AAV) vector coding for human coagulation factor VIII (FVIII). Six of the 7 patients treated with the highest dose of... [Read Article]

mAb could change treatment of hemophilia A, doc says

Monoclonal antibodies
Photo by Linda Bartlett

Emicizumab, a bispecific monoclonal antibody (mAb), could potentially change the treatment paradigm of hemophilia A, according to a speaker at the World Federation of Hemophilia 2016 World Congress. Results of a phase 1/2 study suggest that emicizumab can be safe and effective as once-weekly prophylaxis in patients with... [Read Article]

How hemophilia B impacts education, work

Woman at work

Even mild or moderate hemophilia B can have a negative impact on the education and careers of patients, according to the B-HERO-S study. Ninety-four percent of the patients studied said their disease had a negative impact on their education, and 95% said hemophilia had a negative impact on their work. More than... [Read Article]

Patients can safely receive less FVIII, study suggests

Antihemophilic factor

It may be possible for hemophilia patients to receive less factor VIII (FVIII) without increasing their risk of bleeding, according to a study presented at the World Federation of Hemophilia 2016 World Congress. The study showed that hemophilia A patients who received prophylactic FVIII from the home infusion provider Option Care received 6... [Read Article]

Therapy seems safe, effective in kids with hemophilia

Vial of Adynovate
Photo courtesy of Baxalta

Results of a phase 3 study suggest the full-length recombinant factor VIII therapy Adynovate (BAX 855) can be safe and effective as twice-weekly prophylaxis and to control bleeding in children with hemophilia A. None of the patients in this study developed inhibitory antibodies, and there were... [Read Article]