Successful autologous stem cell transplantation (ASCT) in a patient with juvenile dermatomyositis

We report a 16 year old patient, who was diagnosed with JDM four years ago with severe muscle weakness and skin involvement. Despite therapy including methotrexate, steroids, immunoglobulins, cyclosporine A and rituximab a sustained remission could not be achieved. The patient developed progressive contractures due to persistent inflammatory reactions. Due to treatment-refractory disease immunoablation followed by an autologous stem cell transplantation (ASCT) was performed. The stem cells were mobilized by application of 2 g/m2 cyclophosphamide. After immunoablation using ATG (thymoglobuline®) 10 mg/kg, cyclophosphamide 120 mg/kg and fludarabine 150 mg/m2, CD3/CD19-depleted CD34+stem cells (7.5 × 106/kg) including 2,9 × 104/kg Tcells were transferred. The haematological reconstitution with leucocytes >1000/ml and granulocytes >500/μl was achieved on day +7 and day +8, respectively. Substitution of erythrocytes or platelets was not necessary. No severe infections or organ toxicity (WHO) were observed.