New Canadian study to improve eradication of inhibitors in Hemophilia A patients

Octapharma announced today the launch of the Canadian PREVAIL (Immune Tolerance Induction in Hemophilia A Patients using wilate®) study for patients with Hemophilia A.

The PREVAIL study will enroll hemophilia A patients who have recently developed inhibitors to a FVIII concentrate or who have had long-standing inhibitors to FVIII. Immune tolerance induction (ITI) is the only method of eradicating the inhibitor, and even patients with long standing inhibitors may be able to reduce reliance on bypassing agents for their treatment. The aim of PREVAIL is to better understand patient and therapy related variables on the course and outcome of ITI in a Canadian setting.

According to the coordinating investigators, Dr. Paul Moorehead from the Janeway Children’s Health and Rehabilitation Centre in St. John’s and Dr. Mark Belletrutti from Stollery Children’s Hospital in Edmonton, inhibitor development is the most serious complication of hemophilia A treatment today, occurring in up to 30-40 percent of newly treated patients with severe hemophilia A. “The presence of a FVIII inhibitor in the body will rapidly inactivate the infused factor VIII concentrate, thus making bleed control and prevention of joint damage a clinical challenge in these patients. ITI can result in inhibitor resolution, but can take several years to succeed“, said Dr. Moorehead.

“Although several treatment regimens are used routinely, optimal regimen for ITI is yet to be defined. Persons living with FVIII inhibitors have high morbidity, mortality and associated increases in cost of care and this study is an important step to improve treatment and patient outcomes in Canada”, said Dr. Belletrutti.

The PREVAIL study is the first Canadian, 10-year study of hemophilia A inhibitor patients treated with Wilate for either first occurrence of inhibitors, inhibitors refractory to previous ITI attempts, or relapsed inhibitors to FVIII. Moderate and severe hemophilia A persons of all ages living with inhibitors are eligible and patient enrolment for the study is now open. The comprehensive data collection includes ITI outcome data, bleeding rates during ITI, joint health during ITI, adherence to treatment and relapse rates. Optional sub-studies evaluate quality of life (QoL) during ITI and shed light on inhibitor biology for better inhibitor management. The PREVAIL study will complement currently available data and provide important, prospective information to support the management of patients with hemophilia A who develop inhibitors.

”Given the huge impact inhibitors can have on a person’s life and ability to manage their hemophilia, the Canadian Hemophilia Society (CHS) is supportive of studies on inhibitors and pleased to see the start of this study to collect long term ITI data in Canada to help improve approaches to treatment," said Paul Wilton, CHS President.

For additional information about the study, please contact medinfo@octapharma.ca

About Haemophilia A

Haemophilia A is an X-linked hereditary disorder caused by FVIII deficiency which, if left untreated, leads to haemorrhages in muscles and joints and consequently to arthropathy and severe morbidity. FVIII replacement prophylactic treatment reduces the number of bleeding episodes and the risk of permanent joint damage. This disorder affects one in every 5,000 to 10,000 men worldwide. Globally, 75% of haemophilia cases are left undiagnosed or untreated. The development of neutralising FVIII antibodies (FVIII inhibitors) against infused FVIII represents the most serious treatment complication. The cumulative risk of FVIII inhibitor development is reported to be currently up to 39%.

About Octapharma

The vision of Octapharma is “Our passion drives us to provide new health solutions advancing human life”. Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. As a family-owned company, Octapharma believes in investing to make a difference in people’s lives and has been doing so since 1983; because it’s in our blood. Our company values are Ownership, Integrity, Leadership, Sustainability and Entrepreneurship.

In 2017, the Group achieved €1.72 billion in revenue, an operating income of €349 million and invested €287 million to ensure future prosperity. Octapharma employs around 7,700 people worldwide to support the treatment of patients in 113 countries with products across three therapeutic areas:

• Haematology (coagulation disorders)

• Immunotherapy (immune disorders)

• Critical care

 
Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden. 

For more information visit www.octapharma.com

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