In this issue Okano et al1 report on the outcome of 23 patients with activated phosphoinositide 3-kinase δ (PI3Kδ) syndrome (APDS), 9 of whom received hematopoietic stem cell transplantation (HSCT) for a severe clinical course. This experience adds to recent reports of the clinical spectrum of the disease in large coh ort studies,2-4 including outcomes of HSCT in 11 patients with APDS.5 Altogether, these studies raise the important questions of which patients should be considered for HSCT and when and what would be the optimal preparative regimens to facilitate durable engraftment and reduce the risk of transplan t-related morbidity and mortality. (Source: Journal of Allergy and Clinical Immunology)
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Τετάρτη 10 Οκτωβρίου 2018
Hematopoietic stem cell transplantation for activated phosphoinositide 3-kinase δ syndrome: Who, when, and how?
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