Clinical Trial: Observational Study Assessing Outcomes, Treatment Patterns and Related Costs in Patients in Bullous Pemphigoid

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Multicenter Prospective Observational Study Assessing Outcomes, Adverse Events, Treatment Patterns and Related Costs in Patients Diagnosed With Bullous Pemphigoid

Brief Summary: The study is an observational, multi-center, prospective, non-interventional and open-label data collection study assessing outcomes, treatment patterns, adverse events and costs in patients diagnosed with bullous pemphigoid. The patient enrollment period will be 1 year with a follow-up (observation period) of 1 year for each patient. Four dermatology centres in France will participate. The hypothesis to be answered by the study is that superpotent topical corticosteroid therapy is properly used to treat bullous pemphigoid in real-world life as recommended by French guidelines and whether this treatment influences the medical costs by comparison with systemic therapies (e.g. methotrexate or prednisone).

Detailed Summary:

Bullous pemphigoid is the most common autoimmune blistering disease of the skin, usually affecting the elderly. It is a chronic disease characterized by spontaneous exacerbations and remissions. Bullous pemphigoid is mediated by IgG autoantibodies directed against hemidesmosomal proteins (BP180, BP230), which are involved in dermal-epidermal adhesion in the skin. The diagnosis of bullous pemphigoid is based on clinical and immunopathological findings, including skin direct immunofluorescence. The disease usually requires on average a 1-year duration of treatment although a long-term maintenance therapy may be necessary some cases. High doses oral corticosteroids have been considered as the mainstay of treatment for many years, but are deleterious, with a high rate of treatment side effects, including mortality. Topical superpotent corticosteroids have been demonstrated to be as effective but safer than high doses of oral corticosteroids, reducing side effects and mortality rate, while controlling the disease in between 95% to 100% of cases. Despite its high efficacy, topical superpotent corticosteroid therapy is often considered as poorly convenient. In addition, immunosuppressant drugs and methotrexate are poorly tolerated or frequently contra-indicated in those elderly patients with bullous pemphigoid. To date, no study has evaluated the real-world use and costs of the different treatments in this disease.

Purpose The project is an observational, multi-center, prospective, non-interventional and open-label data collection study assessing outcomes, adverse events, treatment patterns and related costs in patients diagnosed with bullous pemphigoid. The patient enrollment period will be 1 year with a follow-up (observation period) of 1 year for each patient. Four dermatology centres in France will participate.

Primary object
Sponsor: CHU de Reims

Current Primary Outcome: BPDAI (bullous pemphigoid disease area index) [ Time Frame: up to one year ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: CHU de Reims

Dates:
Date Received: June 24, 2016
Date Started: April 2013
Date Completion:
Last Updated: July 15, 2016
Last Verified: July 2016