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P.249 Efficacy of autologous hematopoietic stem cell transplantation in diffuse cutaneous systemic sclerosis: a case series linking outcomes to patient age and conditioning regimen

jsrd · 2026-06-05 · canonical JSON source

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Introduction Diffuse cutaneous systemic sclerosis (dcSSc) is a severe autoimmune disease characterized by progressive skin fibrosis and internal organ involvement. Autologous hematopoietic stem cell transplantation (AHSCT) has emerged as a potential therapeutic option for patients with refractory dcSSc following failure of conventional immunosuppressive treatments.Material and Methods This case series reports outcomes of three Thai patients with progressive dcSSc (aged 19, 52, and 35 years) who underwent AHSCT after failing standard therapies including methotrexate, mycophenolate mofetil, cyclophosphamide, and rituximab. The conditioning regimen consisted of fludarabine (30 mg/m 2), cyclophosphamide (40-60 mg/m2), and anti-thymocyte globulin (2.5 mg/kg). Clinical outcomes were assessed using modified Rodnan skin score (mRSS) and monitoring for transplant-related complications.Results All patients achieved neutrophil engraftment within 7-9 days post-transplant. Two patients (aged 19 and 35) demonstrated sustained clinical improvement with dramatic mRSS reductions from 29 to 4 and 20 to 4, respectively. The older patient (aged 52) showed initial improvement (mRSS 32 to 15) but experienced early relapse with progression of myocarditis within two months. Complications included cytomegalovirus reactivation in all cases, Epstein-Barr virus reactivation, drug-induced liver injury, and one case of disseminated Mycobacterium malmoense infection, all successfully managed.Conclusions AHSCT can achieve significant clinical improvement in selected patients with refractory dcSSc, particularly younger individuals without severe cardiac involvement. The reduced cyclophosphamide dose (40 mg/m 2) may have contributed to early relapse in the older patient, highlighting the importance of individualized conditioning intensity and careful patient selection for optimal outcomes.Abstract P.249 Table 1