Acthar Gel May Improve Sarcoidosis Symptoms, Real-world Study Shows

Acthar Gel (repository corticotropin injection, or RCI) can improve sarcoidosis symptoms, a real-world study shows.

Titled “Repository corticotropin injection in patients with advanced symptomatic sarcoidosis: retrospective analysis of medical records,” the study was published in the journal Therapeutic Advances in Respiratory Disease.

Acthar Gel, marketed by Mallinckrodt Pharmaceuticals, is a therapy delivered via injection under the skin or into the muscles; it acts primarily via release of adrenocorticotropic hormone, which is thought to limit inflammation through a number of different molecular mechanisms.

Although Acthar Gel has been approved for the treatment of sarcoidosis, limited data is available about its use in real-world settings (that is, outside of carefully controlled settings of clinical trials).

In the study, researchers sought real-world data by reviewing medical records of adults with sarcoidosis who were treated with Acthar Gel in the three years preceding the study.

The goal was “to describe patient demographic and clinical characteristics, utilization patterns of RCI [Acthar Gel] and concomitant therapies, and physicians’ assessments of RCI effects on patients’ health status.”

In total, medical data from 302 sarcoidosis patients were analyzed. The group had a mean age of 51 years, and 52% were female.

All patients analyzed had evidence of pulmonary sarcoidosis. Apart from the lungs, 42% had involvement of at least one additional organ — most commonly the skin, joints, heart, and eyes.

Most (64%) had stage 3 or 4 sarcoidosis, as evidenced by biopsies and chest imaging, and 30% of them had been hospitalized for sarcoidosis-related reasons in the year preceding the study.

The majority of the patient cohort (80%) were using Acthar Gel for the first time. When the data was collected, less than half (142 patients, 47%) had completed a course of Acthar Gel treatment; the remainder were continuing treatment.

Overall, treatment with Acthar Gel lasted for an average of 32.5 weeks.

Interestingly, dosing for Acthar Gel varied considerably from person-to-person, “because patients had individualized dosing and dose adjustments,” the researchers wrote.

In total, 49 different starting dosing regimens were identified; the most common starting dosages (40%) were between 41 and 80 units/week. Most patients (79%) had no change in dosage over the course of their treatment.

The researchers noted that the use of other medications, such as corticosteroids, was more prevalent in the three months before Acthar Gel treatment, than it was during the treatment or in the three months after.

“The percentage of patients who used corticosteroids decreased from 61.3% during the 3 months before initiation of RCI to 12.9% 3 months after RCI therapy,” they wrote. “These patterns suggest that reported concomitant medication use decreased during and 3 months after RCI treatment.”

Based on physicians’ assessments, nearly all of those analyzed (95%) experienced at least some kind of improvement after treatment with Acthar Gel, with 54% experiencing improvement in at least two different symptoms.

“The most commonly reported types of sarcoidosis symptom improvements were: overall symptoms (73%), lung function (38%), inflammation (33%), reduction or discontinuation of corticosteroid use (32%), and improved patient quality of life (32%),” the researchers wrote.

Overall, “the findings suggest that RCI is a viable treatment option for patients with advanced symptomatic sarcoidosis,” the researchers said, adding that the findings “provide insights on patient characteristics and practice patterns to help clinicians determine appropriate use of RCI.”

Of note, three of the researchers are current or former employees of Mallinckrodt, which also funded the study.

According to the team, further studies are warranted. A clinical trial (NCT03320070) testing Acthar Gel in pulmonary sarcoidosis patients is currently ongoing, and is sponsored by Mallinckrodt.