期刊
BONE MARROW TRANSPLANTATION
卷 40, 期 7, 页码 633-642出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.bmt.1705778
关键词
hematopoietic cell transplantation; nonmyeloablative conditioning; primary immunodeficiency disorders; severe combined immunodeficiency; bone marrow transplantation
资金
- NCI NIH HHS [CA15704, CA076930, P01 CA078902, CA78902] Funding Source: Medline
- NHLBI NIH HHS [HL085288, HL36444] Funding Source: Medline
- NICHD NIH HHS [HD043376, HD17427] Funding Source: Medline
This study was designed to determine the safety of a nonmyeloablative regimen in patients with primary immunodeficiency disorders (PID) who had infections, organ dysfunction or other risk factors that precluded conventional hematopoietic cell (HC) transplant. Fourteen patients received HLA-matched related (n=6) or unrelated (n=8) HC grafts from marrow (n=8), peripheral blood mononuclear cells (n=5) or umbilical cord blood (n=1), either without conditioning (n=1), or after 200 cGy total body irradiation alone (n=3) or with 90 mg/m(2) fludarabine (n=10). All patients were given postgrafting immunosuppression with mycophenolate mofetil and cyclosporine. Mixed (n=5) or full (n=8) donor chimerism was established in 13 patients, and one patient rejected the graft. Eight patients developed acute grade III (n=1) and/or extensive chronic GVHD (n=8). With a median follow-up of 4.9 ( range, 0.7-8.1) years, the 3-year overall survival, event-free survival and transplant-related mortality were 62, 62 and 23%, respectively. Correction of immune dysfunction was documented in 8 of 10 patients with stable donor engraftment. These preliminary results indicated that this approach was associated with stable donor engraftment and a low incidence of early mortality and, thus, can be considered for certain high-risk patients with PID. However, there was a risk of GVHD, which is an undesirable outcome for this group of patients.
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