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December 8, 2025BloodOpen Access

HLH-JAK, the first OPEN-label, phase II study evaluating ANTI-JAK1 itacitinib, for NON severe HLH in adults.

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Authors

MMMarc Michel

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Overview

Phase II trial shows 71% response rate in non-severe HLH adults treated with itacitinib, suggesting effective use of anti-JAK1 agents.

Key Points

  • To evaluate the efficacy and safety of itacitinib, an anti-JAK1 inhibitor, in adults with non-severe hemophagocytic lymphohistiocytosis (HLH).
  • Open-label, phase II study conducted across 23 French medical centers
  • Included adult patients meeting HLH diagnostic criteria and without severe organ failure
  • Primary endpoint: response rate at day 15, defined as complete or partial response based on clinical parameters.
  • 35 patients were enrolled, with a response rate of 71% at day 15, exceeding the efficacy threshold
  • Of the responders, 15 achieved complete response and 10 partial response
  • The treatment was well tolerated, with only mild adverse effects and no grade ≥3 events reported.

Cite This Study

Marc Michel (2025) studied this question.

synapsesocial.com/papers/69362f604fa91c937236dd37https://doi.org/10.1182/blood-2025-190
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1OA12 JAK inhibitors, an emerging therapeutic target in haemophagocytic lymphohistiocytosis: results from a service evaluation of the UK HLH MDT2026
  2. 2Dual anti-HLH and antitumor efficacy of golidocitinib in relapsed/refractory PTCL-associated hemophagocytic lymphohistiocytosis: A retrospective safety and activity analysis2025
  3. 3HLHRUXO: A prospective trial of a ruxolitinib-containing regimen for children with hemophagocytic lymphohistiocytosis2026
  4. 4Ruxolitinib in malignancy-associated hemophagocytic lymphohistiocytosis (M-HLH): A systematic literature review2025
  5. 5Tofacitinib for elderly onset hemophagocytic lymphohistiocytosis with gene mutations: a case report2024 · 2 citations