Study Investigates Patient-Reported Outcomes in Scleroderma Lung Disease

Patients with systemic sclerosis‐related interstitial lung disease (SSc‐ILD) experience improvements in their health-related quality of life and report positive outcomes following treatment with mycophenolate mofetil (MMF) or cyclophosphamide (CYC), according to results of the Scleroderma Lung Study (SLS) II.

This study was a randomized controlled trial to evaluate the safety and efficacy of MMF and CYC, conducted over a 2-year period. For the SLS II, the researchers randomly assigned 142 patients into 2 groups: 72 participants received oral CYC for 1 year followed by 1 year of placebo while 69 received MMF.

The primary end point for the SLS II was the percentage improvement in forced vital capacity (FVC), measured quarterly over the study period. Both groups experienced significant improvement in FCV during the study.

The researchers also examined whether and how patient-reported outcomes (PROs) changed in response to the therapeutic agents and whether changes in the FVC%‐predicted in SLS II correlated to changes in PROs. They also looked for correlations between PROs and SSc‐ILD disease severity.

Participants reported improvements in several outcomes, regardless of whether they had received CYC or MMF. The researchers found significant improvement in both treatment cohorts in the scores on the Transitional Dyspnea Index (TDI) and St. George’s Respiratory Questionnaire (SGRQ) during the study period.

Results showed that 29% of participants receiving CYC therapy and 24% of those receiving MMF therapy met or exceeded the minimally clinical important difference estimates for TDI. Moreover, 28% of participants receiving CYC therapy and 25% of those receiving MMF therapy met or exceeded the minimally clinical important difference estimates for SGRQ.

In addition, baseline FVC%‐predicted was not associated with baseline TDI or SGRQ scores, but “improvements in FVC%‐predicted were weakly associated with improvements in [TDI] and SGRQ scores,” the researchers noted.

“Treatment with CYC and MMF improved overall health‐related quality of life in patients with SSc‐ILD,” the researchers concluded. “The relationship between PRO measures and the FVC was relatively weak, suggesting that PROs provide complementary information about treatment efficacy not captured by changes in the FVC alone in this patient population.”


—Rebecca Mashaw



Volkmann ER, Tashkin DP, LeClair H, et al. Treatment with mycophenolate and cyclophosphamide leads to clinically meaningful improvements in patient‐reported outcomes in scleroderma lung disease: results of Scleroderma Lung Study II. ACR Open Rheumatol. Published online May 20, 2020.