A Catalyst for Antiphospholipid Syndrome Research

The Rheumatologist—March 21, 2011   

The treatment of the serious, potentially fatal autoimmune clotting disorder antiphospholipid syndrome (APS) has, in general, been empiric and based on various approaches to inhibit the clotting system. Despite many clinical studies and progress in patient outcomes, the field has nevertheless lacked a solid foundation upon which to base decision making and the development of novel approaches. The reason? The fundamental mechanisms of disease remain unknown and efforts to mount basic and clinical research programs have been limited by organizational issues, insufficient funding, and lack of quantitative standards for antibody detection.

Knowing What We Don’t Know

“This is the first effort to publish an official document about the limitations of APS research,” says CRTF co-chair, Doruk Erkan, MD, MPH, associate physician-scientist at the Barbara Volcker Center for Women and Rheumatic Diseases at Hospital for Special Surgery in New York City. For a number of reasons, this is no small achievement. “Studying the disease is very difficult because, for the autoimmune disorders of lupus or rheumatoid arthritis (RA), you are following flares; here you are following thrombotic events, which are relatively rare.” More rare still are events among patients with primary APS—those who have a sustained aPL elevation without comorbid lupus or RA. “To effectively power a trial in this setting you would need thousands of patients,” Dr. Erkan notes.

Michael Lockshin, MD, director of the Barbara Volcker Center for Women and Rheumatic Disease at Hospital for Special Surgery in New York and CRTF co-chair, speaks to the flipside of awareness, which might be called unlearning. “A big part of the challenge is going to be getting all of the world speaking the same language, meaning that, for APS treatment there’s a British approach, an Italian approach, a German approach…” and while some of these geocentric paradigms are in agreement, others conflict, and none are sufficiently based on hard clinical data. “Erkan’s paper on aspirin and primary thrombosis prevention is one of the very few that was a really solid clinical trial. The field is full of case reports of ‘here’s my experience’ type data, yet only a handful of people who have systematically looked at the results. But if we don’t do it now, then when?”

Read the full story at the-rheumatologist.org.