Publication: JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study
JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study
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Fischer, M., Olbrich, P., Hadjadj, J., Aumann, V., Bakhtiar, S., Barlogis, V., von Bismarck, P., Bloomfield, M., Booth, C., Buddingh, E. P., Cagdas, D., Castelle, M., Chan, A. Y., Chandrakasan, S., Chetty, K., Cougoul, P., Crickx, E., Dara, J., Deyà-Martínez, A., … et al. (2024). JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study. Journal of Allergy and Clinical Immunology, 153, 275-286.e18. https://doi.org/10.1016/j.jaci.2023.10.018
Abstract
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Background: Inborn errors of immunity (IEI) with dysregulated JAK/STAT signaling present with variable manifestations of immune dysregulation and infections. Hematopoietic stem cell transplantation (HSCT) is potentially curative, but initially reported outcomes were poor. JAK inhibitors (JAKi) offer a targeted treatment option that may be an alternative or bridge to HSCT. However, data on their current use, treatment efficacy and adverse events are limited.
Objective: We evaluated the current off-label JAKi treatment experience for J
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Creators (Authors)
- Fischer, Marco
- Olbrich, Peter
- Hadjadj, Jérôme
- Aumann, Volker
- Bakhtiar, Shahrzad
- Barlogis, Vincent
- von Bismarck, Philipp
- Bloomfield, Markéta
- Booth, Claire
- Buddingh, Emmeline P
- Cagdas, Deniz
- Castelle, Martin
- Chan, Alice Y
- Chandrakasan, Shanmuganathan
- Chetty, Kritika
- Cougoul, Pierre
- Crickx, Etienne
- Dara, Jasmeen
- Deyà-Martínez, Angela
- Farmand, Susan
- Formankova, Renata
- Gennery, Andrew R
- Gonzalez-Granado, Luis Ignacio
- Hagin, David
- Hanitsch, Leif Gunnar
- Hanzlikovà, Jana
- Hauck, Fabian
- Ivorra-Cortés, José
- Kisand, Kai
- Kiykim, Ayca
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Fischer, M., Olbrich, P., Hadjadj, J., Aumann, V., Bakhtiar, S., Barlogis, V., von Bismarck, P., Bloomfield, M., Booth, C., Buddingh, E. P., Cagdas, D., Castelle, M., Chan, A. Y., Chandrakasan, S., Chetty, K., Cougoul, P., Crickx, E., Dara, J., Deyà-Martínez, A., … et al. (2024). JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study. Journal of Allergy and Clinical Immunology, 153, 275-286.e18. https://doi.org/10.1016/j.jaci.2023.10.018
