Use Of A Response-Adapted Ruxolitinib-Containing Regimen For The Treatment Of Hemophagocytic Lymphohistiocytosis

Summary

This study is a multi-site Phase Ib/II, 2-arm non-randomized clinical trial to determine the efficacy and tolerability of a response-adapted regimen combining ruxolitinib, dexamethasone, and etoposide as Frontline therapy for patients with newly diagnosed hemophagocytic lymphohistiocytosis (HLH) or as Salvage therapy for patients with relapsed/refractory HLH. Primary Objective * To determine the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with newly diagnosed HLH. Secondary Objectives * To describe the efficacy and tolerability of a response-adapted ruxolitinib-containing regimen for patients with relapsed/refractory HLH. * To describe the overall response and outcome for patients with newly diagnosed or relapsed/refractory HLH who are treated with this response-adapted ruxolitinib-containing regimen. Exploratory Objectives * To estimate the pharmacokinetic (PK) parameters of ruxolitinib, assess covariates of ruxolitinib pharmacokinetics, and test whether the drug's effectiveness is correlated with systemic drug exposure. * To query specific immunologic biomarkers and determine whether the levels of these biomarkers correlate with disease response and outcome.

Timeline

Start

2021-07-13

Primary completion

2025-11-07

Completion

2026-08

Publications

• Meyer LK, Ryan K, Panetta JC, Crews KR, Albeituni S, Degar BA, Hermiston ML, Talano JM, Torno LL, Zambidis ET, Trone DJ, Sabin ND, Maron G, Flerlage T, Thomas PG, Cheng C, Campbell PK, Nichols KE, Hines MR. HLHRUXO: A prospective trial of a ruxolitinib-containing regimen for children with hemophagocytic lymphohistiocytosis. Blood Adv. 2026 Jun 4:bloodadvances.2026020265. doi: 10.1182/bloodadvances.2026020265. Online ahead of print.

Drugs

Indications