Hematopoietic Stem Cell Transplantation in Patients with Heterozygous STAT1 Gain-of-Function Mutation

ConclusionHSCT could be an alternative and curative therapeutic option for selectedSTAT1 GOF mutant patients with progressive life-threatening disease unresponsive to conventional therapy. Morbidity and mortality-causing complications included secondary graft failure, infections, and bleeding. (Source: Journal of Clinical Immunology)

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