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

Area of Science:

• Rheumatology
• Immunology
• Hematology

Background:

• Diffuse cutaneous systemic sclerosis (dcSSc) is a severe autoimmune disease.
• Stem cell transplantation (SCT) is an intensive treatment option for eligible dcSSc patients.
• Outcomes for dcSSc patients meeting SCT eligibility but receiving standard care are not well-defined.

Purpose of the Study:

• To evaluate the long-term outcomes of dcSSc patients who met SCT eligibility criteria but received standard immunosuppression.
• To compare survival rates between SCT-eligible patients and those excluded from SCT trials due to specific criteria.

Main Methods:

• Retrospective analysis of a single-center dcSSc cohort (n=636).
• Patients identified based on published SCT trial inclusion criteria.
• Comparison of outcomes between eligible patients and those excluded (e.g., age >65, low DLco, pulmonary hypertension, poor renal function).

Main Results:

• Among 227 eligible patients, 103 (45.4%) died over a mean 12-year follow-up.
• Survival rates at 10 and 20 years were 73% and 43% for eligible patients.
• Patients excluded from SCT trials had significantly worse long-term survival (52% at 10 years, 15% at 20 years).
• Higher age, ESR, and male sex were associated with increased hazard of death.

Conclusions:

• SCT eligibility criteria may identify dcSSc patients with inherently poor prognosis, even with standard treatment.
• Patients meeting SCT criteria but excluded due to comorbidities experience worse outcomes.
• Current best practice treatments may be insufficient for high-risk dcSSc patients identified by SCT criteria.