This article is more than 5 years old. Information may no longer be current.

Bevacizumab may be an effective treatment option in hereditary hemorrhagic telangiectasia

After 3 months of treatment with bevacizumab, 80% of study participants with hereditary hemorrhagic telangiectasia had decreased cardiac output — of which 12% experienced normalization of cardiac output, according to data published in the Journal of the American Medical Association.

In a preliminary, single-center trial, researchers pooled data on 25 patients age 18 to 70 years with confirmed hereditary hemorrhagic telangiectasia affecting the liver, and high cardiac index associated with the illness. Participants received six injections of 5 mg/kg bevacizumab (Avastin, Genentech) every 14 days for 2.5 months duration, with 6 months of follow-up after treatment initiation.

Among 23 patients with available data after six months, 20 indicated a response to treatment, of which five participants experienced cardiac index normalization and 15 experienced a partial response. Median cardiac index decreased from 5.05 L/min/m² to 4.1 L/min/m² after 6 months.

Further, the mean duration of nose bleeds also decreased from 221 minutes per month to 134 minutes after 3 months and 43 minutes after 6 months (P=.008).

“This preliminary study suggests that bevacizumab may be a therapeutic option in the treatment of [hereditary hemorrhagic telangiectasia],” the researchers wrote. “We do not know if this treatment could be definitive or a bridging therapy while patients are waiting for a liver transplant. Longer follow-up studies are necessary to determine the duration of … efficacy and whether maintenance therapy is required.”

Disclosure: Researcher Gilles Paintaud reported having served as a consultant for Laboratoires Français du Fractionnemen et des Biotechnologies, Roche Pharma, Wyeth, Janssen and Pierre-Fabre Laboratories. The study was financed by Hospices Civils de Lyon with a grant supported by the National Research Program and by Roche Laboratories.