What is the role of hematopoietic stem cell transplantation (HSCT) in the treatment of X-linked lymphoproliferative (XLP) syndrome?

Updated: Jan 27, 2019
  • Author: Karen Seiter, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Hematopoietic stem cell transplantation (HSCT), which includes the transfer of bone marow, mobilized CD34+ cells from peripheral blood, or umbilical cord–derived CD34+ cells, is currently the only definitive treatment for XLP syndrome. However, success is dependent on the availability of an appropriate donor who is human leukocyte antigen matched. A number of factors must be considered prior to HSCT, including the disease status, previous treatments, and the type of pre-conditioning regimen. An EBV-positive donor is preferred in patients with EBV-driven disease.

Several studies have evaluated the clinical outcomes of patients undergoing HSCT using either myeloablative-conditioning regimens or reduced-intensity-conditioning (RIC) regimens. [5, 21, 22] These studies revealed similar overall post-transplantation survival rates with RIC and myeloablative protocols in XLP1, with both averaging about 80%. [5, 22]  The outcomes of allogeneic HSCT for XLP2 are less certain at this time. Early evidence suggests that reduced-intensity conditioning regimens should be considered due to very poor early experience with myeloablative preparative regimens. [22]


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