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Article

Improved outcome of HSCT in STAT1 gain-of-function disease following JAK inhibition bridging

27 Jun 2025

Abstract excerpt

) are associated with infections, including chronic mucocutaneous candidiasis and autoimmunity. Morbidity is high, and disease manifestations can be life-threatening. Curative allogeneic hematopoietic stem cell transplantation (HSCT) historically has had poor outcomes. We identified 36 patients with STAT1 GOF disease, receiving 40 HSCT procedures in 2010-2023, in a combined effort of the EBMT-IEWP and the PIDTC....

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PMID: 41142635
DOI: 10.70962/jhi.20250027

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