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



RNA_structure_240.jpg
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_helix_Credit_NIGMS_230.jpg
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-Spencer-Phillips_230.jpg
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_Credit_Credit_Linda_Bartlett_230.jpg
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-240.jpg
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.jpg
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



adynovate-baxalta-230.png
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]