CellCept, Cyclophosphamide Both Improve Patient-reported Outcomes in SSc-ILD, Phase 2 Data Show

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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patient-reported outcomes, SSc-ILD

CellCept (mycophenolate mofetil) and cyclophosphamide can both improve the health-related quality of life of people with systemic sclerosis-related interstitial lung disease, an analysis from a Phase 2 clinical trial shows.

Findings were reported in the study, “Treatment With Mycophenolate and Cyclophosphamide Leads to Clinically Meaningful Improvements in Patient‐Reported Outcomes in Scleroderma Lung Disease: Results of Scleroderma Lung Study II,” published in the journal ACR Open Rheumatology.

Scleroderma is a chronic autoimmune disorder that affects the connective tissue — the tissue that holds together and supports other tissues and organs — and is characterized by scarring, or fibrosis, of the skin and internal organs.

Interstitial lung disease is a common complication of scleroderma (known collectively as SSc-ILD), caused by excessive tissue scarring in the lungs that progressively compromises their ability to function normally.

Assessments of patient-reported outcomes (PROs) in clinical trials are important to determine how a particular medication influences patients’ experience with the disease.

“In some SSc‐ILD trials, improvements in PRO scores have paralleled improvements observed in [lung function parameters], whereas in other trials, changes in PRO scores do not parallel [these] changes. These conflicting results suggest that PROs may be measuring distinct treatment‐related effects that are important to consider in caring for patients with SSc‐ILD,” the researchers wrote in the study.

These researchers at the University of California, Los Angeles investigated how specific PROs changed in response to treatment with CellCept or cyclophosphamide in a group of 142 SSc‐ILD patients who participated in the Scleroderma Lung Study II (SLSII).

SLSII was a randomized Phase 2 trial (NCT00883129) aimed at determining if SSc‐ILD patients would benefit more from completing two years of treatment with oral CellCept or one year of treatment with oral cyclophosphamide followed by another year of placebo.

Study findings previously published in The Lancet Respiratory Medicine showed that both treatment regimens led to significant improvements in patients’ lung function, assessed by forced vital capacity (FVC; measures the total amount of air a patient is able to exhale after taking a deep breath) over the two-year study.

Data from the new analysis showed that over the course of two years, treatment with CellCept or cyclophosphamide also led to improvements in several patient-reported outcomes, including the Transitional Dyspnea Index (TDI) and the St. George’s Respiratory Questionnaire (SGRQ).

The TDI measures changes in shortness of breath — known as dyspnea — perceived by patients, while the SGRQ is a health-related quality of life measure that assesses the impact the disease has on patients’ overall health, daily life, and perceived well-being.

Additionally, analyses showed that about a third of patients in both treatment groups met or exceeded the minimum clinically important difference estimates established for TDI and SGRQ. A minimum clinically important difference is defined as the smallest improvement in a specific PRO score that is necessary for patients to perceive a meaningful improvement.

Although FVC scores were not correlated with TDI or SGRQ scores at the start of the study, a weak association was found between FVC improvements and improvements in TDI and SGRQ scores, as well as with improvements in measures of health status and functional ability, over the course of the trial.

“In summary, treatment with oral [cyclophosphamide] and [mycophenolate mofetil] led to clinically meaningful improvements in overall health status, function, and breathlessness in patients with SSc‐ILD,” the investigators wrote.

“Above all, the findings of this study demonstrate that a comprehensive evaluation combining pulmonary physiology [normal function], the radiographic extent of fibrosis, and PROs is essential to understanding the impact of treatment on progression of ILD in SSc,” they concluded.