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

STAT1-GOF disease is characterized by infections and autoimmunity. In this article, overall survival after allogeneic HSCT in an international cohort of 36 patients is 72.2%, markedly improved from an earlier report. Pre-treatment with JAK inhibitors is associated with better event-free survival.

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Autori principali:	Emilie Pauline Buddingh, Mary Slatter, Juan Carlos Aldave Becerra, Laura Alonso Garcia, Erik von Asmuth, Safa Baris, Oscar de la Calle-Martín, Alice Y. Chan, Su-Wan Bianca Chan, Shanmuganathan Chandrakasan, Deepakbabu Chellapandian, Jasmeen Dara, Susan Farmand, Anders Fasth, Lisa Forbes Satter, Renata Formankova, Eyal Grunebaum, Steven J. Keogh, Ayça Kiykim, Jörn-Sven Kühl, Alexandra Laberko, Timothy Ronan Leahy, Caroline Lindemans, Caridad Martinez, Laura Martínez-Martínez, William Glenn Mitchell, Emma Morris, Joseph H. Oved, Maria Polacik, Jacques G. Rivière, Chaim M. Roifman, Sara Sebnem Kilic, Petr Sedlacek, Ami J. Shah, Linda Vong, Arjan C. Lankester, Michael H. Albert, Bénédicte Neven, Troy Torgerson, Jennifer Leiding, Catharina Schuetz, Inborn Errors Working Party (IEWP), European Society for Blood and Marrow Transplantation (EBMT), and Primary Immunodeficiency Treatment Consortium (PIDTC)
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Rockefeller University Press 2025-07-01
Serie:	Journal of Human Immunity
Accesso online:	https://rupress.org/jhi/article-pdf/doi/10.70962/jhi.20250027/1948050/jhi_20250027.pdf
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