What is the role of hematopoietic stem cell transplantation (HSCT) in the treatment of autoimmune lymphoproliferative syndrome (ALPS)?

Updated: Jul 29, 2019
  • Author: Akaluck Thatayatikom, MD, RhMSUS; Chief Editor: Harumi Jyonouchi, MD  more...
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Answer

For reasons that remain unclear, the cytopenias in most patients with ALPS improve with age. Nevertheless, hematopoietic stem cell transplantation (HSCT) has been successful in ALPS patients and can be considered a treatment option for those with severe, recalcitrant disease and a matched donor. Sibling donors should be screened for and should be free of a mutation in the apoptosis pathway.

The mortality of a matched unrelated donor-derived allogeneic bone marrow transplant is too high to warrant the procedure in most ALPS patients, many of whom have a near-normal life expectancy. Most patients with cytopenias secondary to ALPS can be managed with immunosuppressive agents. Few patients with recalcitrant autoimmune cytopenias are sick enough as a result of their cytopenias to justify the risks and mortality associated with allogeneic bone marrow transplantation. [45]


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