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CASE REPORTS
JOURNAL ARTICLE
Autologous stem cell transplantation in a patient with severe systemic sclerosis.
Acta Reumatológica Portuguesa 2014 July
UNLABELLED: Systemic Sclerosis (SSc) is a chronic disease of the connective tissue, whose pathogenesis involves abnormalities of the immunological system. It has a variable course and there is a subgroup of patients with rapidly progressive disease or unresponsive to conventional treatment. These patients can benefit from intensive immunosuppression and autologous hematopoietic stem cell transplant.
CLINICAL CASE: 19-year-old (y.o.) woman diagnosed with SSc at the age of 13 y.o. with cutaneous, vascular and articular involvement with initial response to methotrexate. Three years later the disease progressed with severe digestive involvement (dysphagia, delayed gastric emptying and weight loss) needing gastrostomy for nutritional support. She was treated with cyclophosphamide without improvement In May 2012 she had an autologous transplant with myeloablative regimen (BEAM): carmustine 300 mg/m2x1 day; etoposido 120 mg/kgx4 days; cytarabine 120 mg/kg 12/12:hx4 days; melphalan 140 mg/m2x1 day. A year and a half after transplantation she is asymptomatic, without any signs or symptoms of the disease, feeds by mouth and the gastric emptying study is normal. Currently she is free of medication.
CLINICAL CASE: 19-year-old (y.o.) woman diagnosed with SSc at the age of 13 y.o. with cutaneous, vascular and articular involvement with initial response to methotrexate. Three years later the disease progressed with severe digestive involvement (dysphagia, delayed gastric emptying and weight loss) needing gastrostomy for nutritional support. She was treated with cyclophosphamide without improvement In May 2012 she had an autologous transplant with myeloablative regimen (BEAM): carmustine 300 mg/m2x1 day; etoposido 120 mg/kgx4 days; cytarabine 120 mg/kg 12/12:hx4 days; melphalan 140 mg/m2x1 day. A year and a half after transplantation she is asymptomatic, without any signs or symptoms of the disease, feeds by mouth and the gastric emptying study is normal. Currently she is free of medication.
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