NCT05491304 — drugset

Summary

Hemophagocytic lymphohistiocytosis (HLH) is a rapidly fatal disease caused by immune-dysregulation characterized by hypercytokinemia, with about 30%-40% of patients suffering death in children. Stratification strategy and individualized treatment is important to improve the survival. In our recent retrospective study, risk stratification based on IL-10 and IFN-γ levels well distinguished patients with different outcomes. In this multicenter prospective study, we will enroll the newly diagnosed pediatric HLH patients and divide them into low, intermediate and high-risk cytokine groups according to IFN-γ and IL-10 levels. The patients'clinical manifestation and laboratory findings will be further evaluated into severe and non-severe groups. For low/intermediate risk and non-severe patients, steroid or ruxolitinib will be used initially; while those with high risk or severe diseases, DXM+VP16±ruxolitinib will be administered. The treatment strategy could be adjusted after evaluation 48-72 hours later.

Timeline

Start: 2022-09-01
Primary completion: 2024-12-31
Completion: 2025-12-31

Publications

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Dexamethasone	Small molecule	5 mg/m2	—
Subject	Etoposide	Small molecule	100 mg/m2	—
Subject	Etoposide	Small molecule	150 mg/m2	—
Subject	Ruxolitinib	Small molecule	2.5 mg	Oral
Subject	Ruxolitinib	Small molecule	5 mg	Oral
Subject	Ruxolitinib	Small molecule	10 mg	Oral

Cytokine Guided Risk Stratification and Treatment in Pediatric Hemophagocytic Lymphohistiocytosis

Summary

Timeline

Publications

Drugs

Indications