Hemophagocytic lymphohistiocytosis
Conditions
Interventions
experimental group:Patients received oral ruxolitinib dosed on 2.5mg, 5 mg or 10 mg twice daily depending on the body weight (=10kg,=20kg or>20kg, respectively)
Sponsors
Beijing Children's Hospital Affiliated to Capital Medical University, National Center for Children's Health
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: At the same time, the following three conditions are met: 1. Patients with hlh-2004 were diagnosed; 2. Both male and female, patients under the age of 18 years; 3. Patients willing to sign informed consent.
Exclusion criteria
Exclusion criteria: 1. Patients with HLH related to tumor and parasite infection were identified; 2. Patients who are allergic to RUX or have more severe allergic constitution; 3. Patients with severe renal dysfunction (glomerular filtration rate 20); 5. Patients with moderate or severe cardiac dysfunction (NYHA grade greater than II grade); 6. Patients who are unable to comply during the trial and / or follow-up phase; 7. Patients who also participated in other clinical studies.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| the overall response (OR) rate, including proportion of patients achieving a complete response (CR), proportion of a partial response (PR) and HLH improvement rate.; | — |
Secondary
| Measure | Time frame |
|---|---|
| event-free survival time (EFS, defined as the time from initial ruxolitinib treatment to the first occurrence of disease progression, relapse or death; for non-responders, EFS is defined as date of enrollment plus 1 day);Overall Survival Rate;the duration of response;dynamic changes of key biomarkers during treatment;the association between treatment response and risk stratification;safety; | — |
Countries
China
Contacts
Public ContactRui Zhang
Beijing Children's Hospital Affiliated to Capital Medical University
Outcome results
None listed