Jakafi (ruxolitinib)

P3, N=438, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China

P=N/A, N=92, Recruiting, Institute of Hematology & Blood Diseases Hospital, China | Not yet recruiting --> Recruiting | N=30 --> 92

Momelotinib demonstrated meaningful clinical benefit and acceptable safety in cytopenic patients pretreated with ruxolitinib, which supports its role after ruxolitinib failure.

P1, N=66, Active, not recruiting, Stanford University | Recruiting --> Active, not recruiting | Trial completion date: Nov 2027 --> May 2028 | Trial primary completion date: Nov 2027 --> May 2028

Patients with active disease received immunosuppressive therapy, chemotherapy-based regimens and/or ruxolitinib...Based on these findings, we developed a risk model stratifying patients into three groups with distinct 3-year OS rates: 69.9% (95% CI, 44.6-85.3) in patients with neither factor, 24.8% (95% CI, 7.0-48.2) in those with one factor and 0% in those with both factors (p < 0.001). ALT and ferritin are practical biomarkers for risk stratification and treatment.

P2/3, N=1000, Not yet recruiting, Bambino Gesù Hospital and Research Institute

Integrated PK/PD model simulations suggested that a flat starting dose of navitoclax 200 mg for baseline platelets > 150 × 109/L, and 100 mg for ≤ 150 × 109/L with ruxolitinib minimized thrombocytopenia risk while maintaining efficacy. Dose reductions of 25 mg for the 100 mg start and 50 mg (plus 25 mg if needed) for the 200 mg start optimized the benefit-risk balance.

P1/2, N=216, Active, not recruiting, Bristol-Myers Squibb | Trial completion date: May 2026 --> Aug 2028 | Trial primary completion date: May 2026 --> Aug 2028

P2, N=42, Recruiting, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

Although etoposide with dexamethasone remains the most frequently used first-line regimen, various new therapies are now being employed in the management of HLH. The interferon gamma inhibitor emapalumab, the Janus kinase signal transducer and activator of transcription pathway inhibitor ruxolitinib, and the interleukin 6 (IL-6) inhibitor tocilizumab have been trialed in HLH management, with additional treatment options being inhibition of IL-18 and tumor necrosis factor alpha. Here, we summarize current management options for HLH; we also propose a new grading system for HLH based on Common Terminology Criteria for Adverse Events version 5.0 as well as on known prognostic factors (eg, abnormal bilirubin and transaminase levels, elevated creatinine level, respiratory failure, neutropenia, hypertriglyceridemia, hypoalbuminemia, and coagulopathy), which could standardize the diagnosis and guide prompt and appropriate management.

P3, N=180, Not yet recruiting, Mesoblast, Ltd.

P1, N=96, Not yet recruiting, Philogen S.p.A.