Definitions and outcome measures for bullous pemphigoid: Recommendations by an international panel of experts

Abstract

Our scientific knowledge of bullous pemphigoid (BP) has dramatically progressed in recent years. However, despite the availability of various therapeutic options for the treatment of inflammatory diseases, only a few multicenter controlled trials have helped to define effective therapies in BP. A major obstacle in sharing multicenter-based evidences for therapeutic efforts is the lack of generally accepted definitions for the clinical evaluation of patients with BP. Common terms and end points of BP are needed so that experts in the field can accurately measure and assess disease extent, activity, severity, and therapeutic response, and thus facilitate and advance clinical trials. These recommendations from the International Pemphigoid Committee represent 2 years of collaborative efforts to attain mutually acceptable common definitions for BP and proposes a disease extent score, the BP Disease Area Index. These items should assist in the development of consistent reporting of outcomes in future BP reports and studies.

Assessment of Bullous Pemphigoid Disease Area Index during Treatment: A Prospective Study of 30 Patients

“…BPDAI was calculated according to the International Pemphigoid Committee recommendations [15]. At each visit, BPDAI and the BPDAI pruritus component were recorded separately.…”

“…Two scoring systems for pemphigus were first introduced and validated: the Autoimmune Bullous Skin Disorder Intensity Score in 2007 [11] and the Pemphigus Disease Area Index, which was developed by an International Pemphigus Committee in 2008 [12,13,14]. In 2011, an international panel of experts proposed a disease extent score, the Bullous Pemphigoid Disease Area Index (BPDAI) to assist in the development of consistent reporting of outcome in future BP reports and studies [15]. Since then, only one study used the BPDAI in practice, aiming to correlate this score with the titres of circulating autoantibodies in a series of BP patients (anti-BP180, anti-BP230) [16].…”

A randomised controlled trial to compare the safety, effectiveness and cost-effectiveness of doxycycline (200 mg/day) with that of oral prednisolone (0.5 mg/kg/day) for initial treatment of bullous pemphigoid: the Bullous Pemphigoid Steroids and Tetracyclines (BLISTER) trial

“…Consideration should be given to adopting clearer definitions of remission and early, intermediate and late observation points as recommended in 2012 by an international group. 57 There is also scope for establishing a core outcome set for future clinical trials of pemphigoid which includes outcomes that are important to patients so that the results of new trials can be compared in meta-analysis.…”

Intravenous immunoglobulin as clinical immune-modulating therapy

“…A recent review of 41 published case reports showed that a cycle of immunoglobulin of 2 g/kg was clinically effective in about 80% of the patients and led to the withdrawal of other immunosuppressive treatment. 21 In view of the limitations owing to diverse definitions for outcome measures, 22 it is difficult to compare the efficacy of therapeutic alternatives and to indicate a schedule of treatment.…”