Kristin Highland, MD: Practices and Metrics for Improving Scleroderma Care


The rare disease patient population is smaller and in need of immediate treatment. What's next after the SENSCIS trial results?

A new study presented at the American Thoracic Society (ATS) 2019 International Meeting in Dallas, TX, this week showed that nintedanib treatment resulted in improved forced vital capacity (FVC) decline among patients with systemic sclerosis-associated interstitial lung disease (ILD).

Results from the one-year year Safety and Efficacy of Nintedanib in Systemic Sclerosis (SENSCIS) trial indicated that nintedanib has a similar benefit for patients with the rare, heterogenous associated form of ILD as it does for patients with idiopathic pulmonary fibrosis. As study author Kristin B. Highland, MD, director of the Rheumatic Lung Disease Program at the Cleveland Clinic, explained to MD Magazine®, forced vital capacity is the best metric of care for these patients; its associated with lung function, and therefore survival.

MD Mag: With what do patients with scleroderma generally present, and why is FVC a valuable metric of care?

Highland: Scleroderma patients are either going to present to the rheumatologist first, where they have complaints such as skin thickening , Raynaud’s phenomenon, digital ulcers, muscle involvement, or joint discomfort. And it's up to the rheumatologist to recognize that the patient may in fact have interstitial lung disease. Sometimes they may not even know that they have interstitial lung disease because they're living a very sedentary lifestyle.

So it's very important that the rheumatologist have seen these patients are screening for interstitial lung disease with HR-CT pulmonary function testing, because interstitial lung disease is the leading cause of mortality in scleroderma. On the contrary, a lot of patients are presenting to the pulmonologist with interstitial lung disease, and it's important for the pulmonologist to recognize that the patient may in fact have scleroderma.

Sometimes it's obvious when they have extensive skin involvement, but sometimes the extra pulmonary manifestations can be more subtle. And so, treatment strategies for scleroderma interstitial lung disease are different than for idiopathic pulmonary fibrosis.

So forced vital capacity is really our best surrogate for overall lung function and it does track with what we see on chest CT; it does track with survival. This is an orphan disease—it would be a little difficult to power design the study to be a mortality study. So first vital capacity, at this point in time, is probably our best surrogate in in all comers with interstitial lung disease.

How will follow-up be managed in this smaller patient population in need of immediate care?

Well I think that's a valid point. I think we will have some data to inform the stability of the treatment effect that we saw in the SENSCIS study. SENSCIS-ON is a continuation study for this trial, and the vast majority of patients did roll over to the continuation study. So we'll be able to follow these patients for a number of years following an open-label study—granted limitations.

But we’ll be able to follow these patients for a number of years following completion of the SENSCIS study to see how they behave long-term. We'll have that data. I anticipate that there'll be real-world registries of scleroderma patients that get treated.

There are a number of registries that exist right now the EUSTAR registry is a very, very large European registry of patients with scleroderma—not just scleroderma interstitial lung disease—that are tracking all aspects of disease. And I think that registry will help make these data more robust, and will make it applicable to the “real-world setting."

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