Annals of the rheumatic diseases · 2026
Autologous stem cell transplantation (ASCT) for Refractory juvenile-onset systemic sclerosis (JSSc).
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Torok Kathryn S, Horvei Paulina, Li Jonathan, Rosser Franziska, Alexander Jessie L, Rose-Felker Kirsten, Sood Vibha, Olson Adam, Hogue Nicole, Vandergrift Vickie, Stanczak Heather, Miglioretti Maura, Farver Lauren, Koster Wendy, Mcguire Devin, Havrilla Haley, McIntyre Shawna, Domsic Robyn T, Lafyatis Robert A, Szabolcs Paul
Аннотация
OBJECTIVES: Juvenile-onset systemic sclerosis (JSSc) is a rare autoimmune disease characterised by vasculopathy and multiorgan fibrosis, leading to significant morbidity and early mortality. Autologous stem cell transplantation (ASCT) is an established therapy for severe adult systemic sclerosis, but data in JSSc are limited. We reported outcomes of 8 consecutive patients with treatment-refractory JSSc who received ASCT at a single centre.
METHODS: Patients with moderate-to-severe JSSc refractory to ≥ 2 disease-modifying antirheumatic drugs underwent CD34+-selected ASCT using institutional review board- and US Food and Drug Administration-approved protocols (ClinicalTrials.gov: NCT03630211). Conditioning included alemtuzumab, rituximab, antithymocyte globulin, total body irradiation, and thiotepa. Clinical outcomes were assessed at baseline and every 6 months up to 48 months post-ASCT.
RESULTS: All patients engrafted successfully (median neutrophil and platelet engraftment: 13 and 22 days, respectively). There were no treatment-related deaths or organ toxicities. Event-free survival (no death, cardiopulmonary failure, or renal crisis) was 100% at a median follow-up of 30 months. Skin scores (modified Rodnan skin score) improved by a median of 93%, and 6-minute walk distance improved by 13%. All patients discontinued immunosuppression for ≥18 months post-ASCT; 1 patient later required reinitiation for disease flare. Pulmonary function stabilised; both patients with pulmonary hypertension showed normalised pulmonary pressures. Gastrointestinal symptoms, including oesophageal motility and reflux, improved in most patients. Musculoskeletal function (Childhood Health Assessment Questionnaire and Childhood Myositis Assessment Scale), vascular symptoms, and quality of life (Scleroderma Health Assessment Questionnaire and performance scores) improved across the cohort.
CONCLUSIONS: ASCT was safe and well-tolerated in patients with refractory JSSc and resulted in durable, multisystem improvement. These findings support earlier consideration of ASCT for patients with JSSc with progressive, treatment-resistant disease.
METHODS: Patients with moderate-to-severe JSSc refractory to ≥ 2 disease-modifying antirheumatic drugs underwent CD34+-selected ASCT using institutional review board- and US Food and Drug Administration-approved protocols (ClinicalTrials.gov: NCT03630211). Conditioning included alemtuzumab, rituximab, antithymocyte globulin, total body irradiation, and thiotepa. Clinical outcomes were assessed at baseline and every 6 months up to 48 months post-ASCT.
RESULTS: All patients engrafted successfully (median neutrophil and platelet engraftment: 13 and 22 days, respectively). There were no treatment-related deaths or organ toxicities. Event-free survival (no death, cardiopulmonary failure, or renal crisis) was 100% at a median follow-up of 30 months. Skin scores (modified Rodnan skin score) improved by a median of 93%, and 6-minute walk distance improved by 13%. All patients discontinued immunosuppression for ≥18 months post-ASCT; 1 patient later required reinitiation for disease flare. Pulmonary function stabilised; both patients with pulmonary hypertension showed normalised pulmonary pressures. Gastrointestinal symptoms, including oesophageal motility and reflux, improved in most patients. Musculoskeletal function (Childhood Health Assessment Questionnaire and Childhood Myositis Assessment Scale), vascular symptoms, and quality of life (Scleroderma Health Assessment Questionnaire and performance scores) improved across the cohort.
CONCLUSIONS: ASCT was safe and well-tolerated in patients with refractory JSSc and resulted in durable, multisystem improvement. These findings support earlier consideration of ASCT for patients with JSSc with progressive, treatment-resistant disease.