For patients affected by systemic scleroderma (also known as systemic sclerosis), a chronic autoimmune disease marked by hardening of the skin and internal organ involvement, the need for treatments is well understood.

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“Scleroderma has the highest mortality rates of any rheumatic disease,” says Dinesh Khanna, M.D., M.Sc., director of the Michigan Medicine Scleroderma Program. “This disease may be rare, but it can have a profound effect on young people and can be quite disabling for patients at any age.”

Apart from its effects on the skin, systemic scleroderma can also harden the internal organs. The condition ranges in severity and can cause pain, stiffness and fatigue.

Khanna’s program is one of few in the nation dedicated to helping scleroderma patients manage their condition and one of the top three in the nation based on research funding and patient volume.

Although there are currently no approved treatments for the estimated 125,000 people in the United States with scleroderma, Khanna and his team have been spearheading new clinical trials that could help shape the future of care.

He spoke more about efforts and progress.

A new outcome measure for scleroderma

When performing scleroderma treatment trials in the past, Khanna and other experts relied on only one outcome measure: the modified Rodnan Skin Score.

“The score measures skin thickness,” Khanna says. “We pinch the skin at 17 different spots and measure skin thickening in patients. It’s a primary outcome measure in systemic sclerosis clinical trials.”

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But, he notes, the skin score had shortcomings. Skin is one aspect of the disease, but scleroderma also affects other organs.

“The scleroderma research field needed a combined measure that captures the overall disease and addresses what is important to a patient,” Khanna says.

Which is why he led the development of the American College of Rheumatology Provisional Composite Response Index for Clinical Trials in Early Diffuse Cutaneous Systemic Sclerosis, or ACR CRISS, in 2016.

The two-part measure first examines significant heart, lung and kidney involvement and then changes in baseline in five variables — the modified Rodnan Skin Score (skin thickness); forced vital capacity (a spirometry measurement for lung function preservation); patient global assessment (to measure overall well-being); the Health Assessment Questionnaire Disability Index (a measure of functional disability); and physician global assessment (to assess overall scleroderma).

After examining those variables collectively, the new composite measure calculates the probability of patient improvement after one year of treatment.

The new measure was recently endorsed by the American College of Rheumatology Board of Directors at its 2018 annual meeting to be used in future systemic sclerosis clinical trials.