Outcomes linked to eligibility for stem cell transplantation trials in diffuse cutaneous systemic sclerosis.

OBJECTIVES: The aim of this study was to explore outcomes in a cohort of diffuse cutaneous systemic sclerosis (dcSSc) patients fulfilling eligibility criteria for stem cell transplantation (SCT) studies but receiving standard immunosuppression.

METHODS: From a large single-centre dcSSc cohort (n = 636), patients were identified using the published SCT trials' inclusion criteria. Patients meeting the trials' exclusion criteria were excluded.

RESULTS: Of the 227 eligible patients, 214 met the inclusion criteria for ASTIS, 82 for SCOT and 185 for the UPSIDE trial, and 66 were excluded based on age > 65 years, low DLco, pulmonary hypertension or creatinine clearance <40ml/min. The mean follow-up time was 12 years (SD 7). Among the eligible patients, 103 (45.4%) died. Survival was 96% at 2-, 88% at 5-, 73% at 10- and 43% at 20 years. Compared with this 'SCT-eligible' cohort, those patients who would have been excluded from SCT trials had a worse long-term survival (97% at 2-, 77% at 5-, 52% at 10- and 15% at 20 years, log rank p< 0.001). Excluded patients also had a significantly worse long-term event free survival. Hazard of death was higher in patients with higher age at onset (HR 1.05, p< 0.001), higher ESR at baseline (HR 1.01, p= 0.025) and males (HR 2.12, p= 0.008).

CONCLUSION: SCT inclusion criteria identify patients with poor outcome despite current best practice treatment. Patients meeting the inclusion criteria for SCT but who would have been excluded from the trials because of age, pulmonary hypertension, poor kidney function or DLco <40%, had worse outcomes.