As with many rare diseases, the treatment of scleroderma and its associated conditions can be complicated. Three scleroderma experts will outline approaches to scleroderma skin involvement, interstitial lung disease, and pulmonary hypertension during the Wednesday Clinical Practice session Scleroderma Treatment 2019. The session will take place from 9:00 – 10:30 am in the Sidney Marcus Auditorium, Building A in the Georgia World Congress Center.
Laura Hummers, MD, ScM, Associate Professor of Medicine at Johns Hopkins University Division of Rheumatology, will open the session with a discussion of treatment options for scleroderma skin involvement.
“Although most patients with scleroderma have some degree of tight skin, there is a subset of patients with diffuse cutaneous scleroderma who have very dramatic involvement of the skin, with tightness and hardening in a lot of areas,” Dr. Hummers said. “That process can be painful, sometimes itchy, and can lead to dramatic loss of function and motion of the joints.”
Dr. Hummers will share with attendees the diagnosis of diffuse cutaneous scleroderma, and how to distinguish patients who do and do not need treatment.
“About two-thirds of patients with scleroderma have limited skin involvement that is not amenable to the therapies like the diffuse group would be,” Dr. Hummers said.
Her presentation will also include information on how to address skin involvement in the context of other conditions, such as lung involvement. She will also walk through some of the standard therapies for the condition and the data supporting their use.
In addition to discussing current therapies, Dr. Hummers will provide an overview of some of the recent clinical trials and what can be learned about those potential medications and the impact they do or do not have on skin disease. Dr. Hummers will wrap up her presentation discussing refractory patients, and treatment approaches to use after failure of first-line therapies.
Interstitial Lung Disease
The session’s second speaker, Lesley Ann Saketkoo, MD, MPH, Director of the New Orleans Scleroderma and Sarcoidosis Patient Care and Research Center between LSU and Tulane Schools of Medicine, will provide an overview of treatment options for systemic sclerosis interstitial lung disease (SSc-ILD).
Dr. Saketkoo plans to open her presentation by discussing the development and history of treatments used for SSc-ILD, as well as the potential pathogenetic mechanisms being targeted in the pharmaceutical treatment of the condition. Her presentation will also include an overview of non-pharmaceutical treatments such as exercise and sleep.
“I will also examine common comorbidities, especially ones that can be confused with the disease itself,” Dr. Saketkoo said. “These comorbidities, such as reflux or postnasal drip, need different types of treatments and management.”
Part of the presentation will also provide updates on recent clinical trials in SSc-ILD.
For example, earlier this year the FDA approved the first treatment for patients with SSc-ILD. Nintedanib (Ofev) capsules were approved based on results of a study of 576 patients with SSc-ILD aged 20 to 79. After treatment for at least 52 weeks, those patients in the trial assigned to nintedanib had less lung function decline as measured by forced vital capacity than those patients assigned to placebo.
Nintedanib is not the only potential new treatment for SSc-ILD in the pipeline. Dr. Saketkoo will also discuss the results of some new study experiences with rituximab and mycophenolate.
Finally, Dr. Saketkoo also plans to share ways that she approaches patient counseling, and highlight that the groundwork for any successful treatment is shared decision making with the patient.
Pulmonary Hypertension
Lorinda Chung, MD, MS, Professor of Medicine and Dermatology at Stanford University School of Medicine, will wrap up the session with a discussion of treatment options for SSc-associated pulmonary hypertension. Dr. Chung hopes that attendees will leave with a better understanding of the most up-to-date treatment options for scleroderma patients with pulmonary arterial hypertension.
“Pulmonary arterial hypertension is one of the major causes of death in patients with scleroderma,” Dr. Chung said. “Patients with scleroderma can get different forms of pulmonary hypertension, but pulmonary arterial hypertension is the most common type, and the only form we have treatments available for.”
Because there are so many treatments available, experts have developed screening algorithms to help identify patients with scleroderma at high risk for pulmonary arterial hypertension who should undergo the definitive diagnostic test called a right heart catheterization.
Dr. Chung also will provide an update on these treatments and some of the changes that have occurred in the past few years that have altered the way patients with pulmonary arterial hypertension are approached.
For example, a pivotal study showed that upfront combination therapy in patients with all forms of pulmonary arterial hypertension, but particularly scleroderma-associated pulmonary arterial hypertension, have better outcomes if they are treated upfront with a PDE-5 inhibitor such as tadalafil, and an endothelin receptor antagonist.