Measuring outcomes in systemic lupus erythematosus clinical trials

Abstract

The recent approval of the biologic therapy, belimumab, for treatment of systemic lupus erythematosus (SLE) by the US FDA has shifted the developmental landscape of therapeutics for this autoimmune disease. Promising therapies are currently in development for the treatment of SLE, with trials designed to emphasize clinically relevant end points. This article will discuss outcome measures that have been utilized including disease activity indices, definitions of flare, measures of damage, global assessments of disease activity and measures of health-related quality of life. Application of these outcome measures in recent trials are highlighted as illustrative examples. Contributions to the recent success of randomized controlled trials in SLE have included use of evidence-based responder indices, clear definitions of treatment failure, predefined management strategies for use of immunosuppressive agents and corticosteroids, sufficient sample sizes and efforts to identify responsive patient populations. Each completed study in SLE promises to better inform trial design and offer further opportunities for success in a field with a continuing unmet therapeutic need.

Keywords::

• disease activity
• flare
• outcome measure
• responder index
• systemic lupus erythematosus

Financial & competing interests disclosure

Vibeke Strand and Alvina D Chu have received no remuneration for preparation of this manuscript. Vibeke Strand serves as a consultant and has participated in advisory boards for: Amgen, Anthera, Astra Zeneca, BMS, Centocor, Genentech/Roche, HGS, Idera, Lilly, Medimmune, Merck Serono, NovoNordisk, Orbimed, Pfizer, Rigel, Sanofi-Aventis and UCB. She is not on any speakers bureaus and holds no stock in these companies. Vibeke Strand is a member of the clinical faculty at Stanford University, serving as Clinical Professor, Adjunct, Division of Immunology and Rheumatology. Alvina D Chu is a full-time employee at Anthera Pharmaceuticals, Inc. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Notes

BILAG: British Isles Lupus Assessment Group; L: Lupus; QOL: Quality of life; SLE: Systemic lupus erythematosus; SLEDAI: Systemic Lupus Erythematosus Disease Activity Index.