Clinical Study on the Treatment of Pediatric Hemophagocytic Lymphohistiocytosis Based on Cytokine Guided Risk Stratification:A Multicenter Randomized Controlled Study

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1 day
Maximum Age: 18
Healthy Volunteers: f
View:

• 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.

Locations
Other Locations
China
The Children's Hospital of Zhejiang University School of Medicine
RECRUITING
Hangzhou
Contact Information
Primary
Xiaojun Xu, MD
xuxiaojun@zju.edu.cn
+8657188873617
Backup
Zebin Luo, MD
645747676@qq.com
+8618758196529
Time Frame
Start Date: 2022-09-01
Estimated Completion Date: 2025-12-31
Participants
Target number of participants: 400
Treatments
Experimental: Non-severe DXM group
Dexamethasone (DXM): week1-2: 10 mg/m2.d, week 3-4: 5 mg/m2.d, week5-6: 2.5 mg/m2.d, week7: 1.25 mg/m2.d, week8: tapering.
Experimental: Non-severe Ruxo group
Ruxolitinib(Ruxo): body weight (BW)\<10kg: 2.5mg Bid; 10-20kg: 5mg Bid; \>20kg: 10mg Bid; Orally for 4 weeks.
Experimental: Severe HLH-94 group
DXM: week1-2: 10 mg/m2.d, week 3-4: 5 mg/m2.d, week5-6: 2.5 mg/m2.d, week7: 1.25 mg/m2.d, week8: tapering.~Etoposide (VP16): 100-150mg/m2 twice in the first two weeks, and once every week to week 8.
Experimental: Severe HLH-94 plus ruxolitinib group
DXM: week1-2: 10 mg/m2.d, week 3-4: 5 mg/m2.d, week5-6: 2.5 mg/m2.d, week7: 1.25 mg/m2.d, week8: tapering.~Etoposide (VP16): 100-150mg/m2 twice in the first two weeks, and once every week to week 8.~Ruxolitinib(Ruxo): body weight (BW)\<10kg: 2.5mg Bid; 10-20kg: 5mg Bid; \>20kg: 10mg Bid; Orally for 4 weeks.
Sponsors
Collaborators: Hunan Provincial People's Hospital, Wuhan Children's Hospital, The Second Hospital of Anhui Medical University, Children's Hospital of Nanjing Medical University, The Third Xiangya Hospital of Central South University, Children's Hospital of Fudan University, The Affiliated Hospital of Qingdao University, First Affiliated Hospital, Sun Yat-Sen University, Shanghai Children's Hospital, Shenzhen Children's Hospital, Second Affiliated Hospital of Wenzhou Medical University, West China Second University Hospital, Qilu Hospital of Shandong University, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Union hospital of Fujian Medical University, Tongji Hospital, Shanghai Children's Medical Center, Institute of Hematology & Blood Diseases Hospital, China, Zunyi Medical College, Children's Hospital of Soochow University
Leads: The Children's Hospital of Zhejiang University School of Medicine

This content was sourced from clinicaltrials.gov