A trial of alemtuzumab adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency
- Author(s): Dvorak, CC
- Horn, BN
- Puck, JM
- Adams, S
- Veys, P
- Czechowicz, A
- Cowan, MJ
- et al.
Published Web Locationhttp://onlinelibrary.wiley.com/doi/10.1111/petr.12310/full
For infants with SCID the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with alemtuzumab monotherapy to overcome NK-cell mediated immunologic barriers to engraftment. We enrolled four patients who received CD34-selected haploidentical cells, two of whom failed to engraft donor T cells. The two patients who engrafted had delayed T-cell reconstitution, despite rapid clearance of circulating alemtuzumab. Although well-tolerated, alemtuzumab failed to overcome immunologic barriers to donor engraftment. Furthermore, alemtuzumab may slow T-cell development in patients with SCID in the setting of a T-cell depleted graft. © 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
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