Guidance on JAK inhibitor treatment for inborn errors of JAK-STAT signaling (2026). An international consensus statement on behalf of the ESID/EBMT-IEWP and ERN-RITA - PubMed
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- #JAK inhibitors
- #Consensus guidelines
- #Inborn errors of immunity
- Consensus provides structured guidance for JAK inhibitor (JAKi) treatment in JAK-STAT gain-of-function inborn errors of immunity (IEI), addressing limited evidence and off-label use.
- Recommendations cover five areas: JAKi indications/contraindications, pre-treatment assessment, pharmacology/dosing, monitoring, and use with hematopoietic stem cell transplantation.
- Development involved systematic review, expert consensus via Delphi method, retrospective cohort data, and patient input, resulting in 51 evidence-based statements.
- Aims to support clinicians across all age groups, serving as the current international standard of care pending further prospective trials.