id	author	title	date	pages	extension	mime	words	sentences	flesch	summary	cache	txt
cord-006492-pc1vayyl	Ono, Shintaro	Hematopoietic Stem Cell Transplantation for XIAP Deficiency in Japan	2016-11-04		.txt	text/plain	3178	170	45	BACKGROUND: X-linked inhibitor of apoptosis protein (XIAP) deficiency is a rare immunodeficiency that is characterized by recurrent hemophagocytic lymphohistiocytosis (HLH) and splenomegaly and sometimes associated with refractory inflammatory bowel disease (IBD). Although hematopoietic stem cell transplantation (HSCT) is the only curative therapy, the outcomes of HSCT for XIAP deficiency remain unsatisfactory compared with those for SLAM-associated protein deficiency and familial HLH. CONCLUSION: The RIC regimen and HLH control might be important factors for successful HSCT outcomes, with improved IBD, in patients with XIAP deficiency. The transplantations were performed in different institutions, but all conditioning regimens were RIC, except that for patient 1, who was given an intermediate intensity regimen, but died of HLH and acute respiratory distress syndrome on day 27 post-HSCT, in which virus infection or reactivation might not be involved. Reduced-intensity conditioning hematopoietic cell transplantation is an effective treatment for patients with SLAMassociated protein deficiency/X-linked lymphoproliferative Disease type 1	./cache/cord-006492-pc1vayyl.txt	./txt/cord-006492-pc1vayyl.txt