ALPS and stem cell transplantation – pro
For reasons that remain unclear, the cytopenias in most patients with ALPS improve with age. Nevertheless, stem cell transplantation has been successful in ALPS patients and can be considered a treatment option for those with severe, recalcitrant disease and a matched donor. In this case,t here is a donor but the disease does not appear to be very severe.Becasue f teh ararity fo this disease, only case reports are available for transplantation.
A recente review says that 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. The stated reasons are the child missing school adn requiring home isntructions and petechiae etc fomr thrombocytopenia, which do nto appear tob esevere enoguh to justify sibling transplantation.
http://emedicine.medscape.com/article/1663330-treatment#d9, Accessed 10/20/16
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