期刊
ARTHRITIS RESEARCH & THERAPY
卷 21, 期 -, 页码 -出版社
BMC
DOI: 10.1186/s13075-019-1823-0
关键词
Systemic sclerosis; Scleroderma; Haematopoietic stem cell transplantation; CD34
类别
资金
- Ministry of Health, Labor, and Welfare [H24-Jitsuyoka-Kokusai-004]
- Practical Research Project for Rare/Intractable Diseases from Japan Agency for Medical Research and development, AMED
- Japan Society for the Promotion of Science [JSPS KAKENHI 16K19603, 15K09527]
- Grants-in-Aid for Scientific Research [15K09527] Funding Source: KAKEN
BackgroundThe effectiveness of autologous haematopoietic stem cell transplantation (auto-HSCT) in treating severe systemic sclerosis (SSc) is established; however, the necessity of purified CD34+ cell grafts and the appropriate conditioning regimen remain unclear. This study aimed to compare the efficacy and safety of CD34-selected auto-HSCT with unmanipulated auto-HSCT to treat severe SSc.MethodsThis study was a post hoc analysis of a phase I/II clinical trial conducted in Japan. Nineteen patients with severe SSc were enrolled. Peripheral blood stem cells (PBSCs) were mobilised with cyclophosphamide (4g/m(2)) and filgrastim (10g/kg/day). Following PBSC collection by apheresis, CD34+ cells were immunologically selected in 11 patients. All patients were treated with high-dose cyclophosphamide (200mg/kg) monotherapy as a conditioning regimen and received CD34-selected (n=11) or unmanipulated auto-HSCT (n=8). Changes in skin sclerosis and pulmonary function were assessed over an 8-year follow-up period. Differences in the changes, toxicity, progression-free survival (PFS) and overall survival were compared between patients who had received CD34-selected auto-HSCT and those who had received unmanipulated auto-HSCT.ResultsSkin sclerosis progressively improved after transplantation over an 8-year follow-up period in both groups, and the improvement was significantly greater in the CD34-selected group than in the unmanipulated group. Forced vital capacity in the CD34-selected group continuously increased over 8years, whereas in the unmanipulated group it returned to baseline 3years after transplantation. Toxicity and viral infections, such as cytomegalovirus infection and herpes zoster, were more frequently found in the CD34-selected group than in the unmanipulated group. The frequency of severe adverse events, such as bacterial infections or organ toxicity, was similar between the two groups. No treatment-related deaths occurred in either treatment group. PFS of the CD34-selected group was greater than that of the unmanipulated group, and the 5-year PFS rates of the CD34-selected and unmanipulated group were 81.8% and 50% respectively.ConclusionsCD34-selected auto-HSCT may produce favourable effects on improvement of skin sclerosis and pulmonary function compared with unmanipulated auto-HSCT. Use of CD34-selected auto-HSCT with high-dose cyclophosphamide monotherapy as a conditioning regimen may offer an excellent benefit-to-risk balance.
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