Cytokine Guided Risk Stratification and Treatment in Pediatric Hemophagocytic Lymphohistiocytosis

• Status: Not yet recruiting
• Trial end date: 2025-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The Children's Hospital of Zhejiang University School of Medicine
• Collaborators: Children's Hospital of Fudan University; Children's Hospital of Nanjing Medical University; Children's Hospital Of Soochow University; First Affiliated Hospital, Sun Yat-Sen University; Hunan Provincial People's Hospital; Institute of Hematology & Blood Diseases Hospital; Qilu Hospital of Shandong University; Second Affiliated Hospital of Wenzhou Medical University; Shanghai Children's Hospital; Shanghai Children's Medical Center; Shenzhen Children's Hospital; The Affiliated Hospital of Qingdao University; The Second Hospital of Anhui Medical University; The Third Xiangya Hospital of Central South University; Tongji Hospital; Union hospital of Fujian Medical University; West China Second University Hospital; Wuhan Children's Hospital; Xinhua Hospital, Shanghai Jiao Tong University School of Medicine; Zunyi Medical College
• Treatments: Dexamethasone; Etoposide

Criteria

Inclusion Criteria:

• Age from one day to 18 years old；

• Newly diagnosed HLH, fulfilling the HLH criteria;

• To observed the early diagnosis role of cytokines, patients who is suspected to be HLH and fulfill 3 out of 8 criteria can be pre-enrolled.

Exclusion Criteria:

• treated with steroids or etoposide within 72 hours before diagnosis.