Little-Studied Blood Syndrome Gains Researchers’ Attention

New York—March 15, 2011

Motivated by the lack of treatment options for patients with antiphospholipid syndrome (APS), rheumatology researchers convened an international committee to address the problem directly. Their creation, APS ACTION (Antiphospholipid Syndrome Alliance for Clinical Trials and International Networking), is bringing together some of the foremost experts on APS—a little-known blood condition that can cause severe health consequences—to design clinical trials and registries focused on improving patient outcomes.

As a result of its first-ever meeting this past November, APS ACTION determined that distinct variations of APS—which causes frequent abnormal blood clots in arteries and veins —made patient studies into the condition very challenging. Blood clots form because the immune system mistakenly produces antibodies against phospholipid-binding plasma proteins, which puts those affected at risk for stroke and pregnancy complications.

“We concluded there are few controlled clinical trials that have included a heterogeneous group of APS patients who exhibit many different forms of the disease and antiphospholipid antibody test results. Comparison between patients is very difficult when APS looks different from one patient to another,” reported rheumatologist Doruk Erkan, M.D., who is an associate physician-scientist at the Barbara Volcker Center for Women and Rheumatic Disease and clinical co-director of the Mary Kirkland Center for Lupus Care at Hospital for Special Surgery in New York. Dr. Erkan is also the newly-elected Executive Committee Chair of APS ACTION.

Since APS ACTION formed, physicians have been vocal in their determination to advance APS research and treatment and to not be derailed by differences of opinion related to clinical practice.

“I read a post on a Facebook group for APS patients that said, ‘Finally these researchers have started to work together.’ That was sobering,” recalled Dr. Erkan. “While treatment of APS touches on contentious issues, the enthusiasm of APS ACTION participants is palpable. The urgency of the situation calls for a collaborative approach, and we’re working together to improve research, treatment and quality of life for all APS patients.”

The scarcity of comprehensive, well-designed clinical trials involving the condition and its treatment means that evidence-based recommendations to treat APS are hard to come by. In the past, this caused physicians to split into groups that emphasized different treatment regimens. But, it also inspired the creation of APS ACTION.

Emboldened by the newfound consensus of rheumatologists who often differ in their treatment recommendations for individuals with APS, a number of subcommittees have already taken up the challenge to address long-standing issues in APS research.

“The subcommittees have eagerly started to address clinical trial design, an APS patient registry, APS research and outreach to advocacy groups,” said rheumatologist Michael Lockshin, M.D., director of the Barbara Volcker Center for Women and Rheumatic Disease and also co-head of the Mary Kirkland Center for Lupus Research, both at Hospital for Special Surgery. Dr. Lockshin is also one of the executive committee members of APS ACTION.

Dr. Lockshin heads a subcommittee tasked with exploring funding options for maintaining the infrastructure of this international consortium and developing a comprehensive patient registry. “APS ACTION hit the ground running, and we want to keep up this enthusiasm by moving ahead on all APS research-related issues. Members of APS ACTION are dedicated to standardizing clinical care and research and developing evidence-based treatment recommendations, ultimately improving patient outcomes.”

About APS ACTION
Twenty-seven physicians from 19 international centers currently participate in APS ACTION. The number of centers is expected to increase when the infrastructure is finalized. For more information about APS ACTION, physicians and patients may contact Joann Vega (vegaj@hss.edu or 212-774-2795).