Long-term efficacy of autologous hematopoietic stem cell transplantation in systemic sclerosis patients

Objective To observe the long-term efficacy and safety of autologous hematopoietic stem cell transplantation (AHSCT) for systemic sclerosis (SSc) patients. Methods Between May 2007 and June 2009,4 patients with SSc were enrolled in the study. Peripheral blood stem cells were mobilized with cyclopho-sphamide (CTX) followed by granulocyte colony stimulating factor (G-CSF). Conditioning was performed with i.v. cyclophosphamide 50 mg·kg-1·d-1 for 4 days. The results of the modified Rodnan skin score (mRSS), thoracic high-resolution computer tomography and pulmonary function were collected after transplantation. Results There was an improvement in mRSS, lung function and HRCT in the six months after AHSCT. Within six month to one year after transplantation, one patient had sustained and two patients recurred. After active treatments two patients were improved again. During the follow-up of 8.7 (4.1-9.8) years, three patients were stable and one patient died. Infection and hepatic function injury were the major complications. There was not transplant-related mortality. Conclusion AHSCT with CTX as a pre-conditioning regimen is safe and effective for SSc. The efficacy for patients with short course, rapid progress and edema is significant. However, long-term efficacy is poor, and long-term maintenance treatment is needed. Key words: Scleroderma, systemic; Peripheral blood stem cell transplantation