HLH
Conditions
Brief summary
This study is a prospective single-arm clinical study, focusing on Hemophagocytic lymphohistocytosis，to evaluate the clinical efficacy and safety of zanubrutinib.
Interventions
DRUGZanubrutinib
The dosage of zanubrutinib is 160mg twice a day (80mg twice a day for patients over 75 years old) for 2 months. The remission rate is evaluated after treatment. If it is above PR, continue the dose maintenance treatment.
Sponsors
Beijing Friendship Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
14 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Unlimited gender, age ≥ 14 years old; 2. HLH was diagnosed according to HLH-04 diagnostic criteria; 3. Before the study, there was no severe liver function injury; Serum creatinine ≤ 1.5 times the upper limit of normal value; 4. Sign informed consent
Exclusion criteria
1. Allergic to zebutinib; 2. Currently active important cardiovascular diseases with clinical significance; The functions of important organs such as heart and lung unrelated to HLH were seriously abnormal; 3. Known human immunodeficiency virus (HIV) or active hepatitis B (HBV) or hepatitis C (HCV) infection; 4. Pregnant or lactating women and patients of childbearing age who refuse to take appropriate contraceptive measures during this trial; 5. Serious mental illness; 6. Active massive hemorrhage of internal organs; 7. Uncontrollable infection; 8. At the same time, participate in other clinical researchers
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| evaluation of treatment response | Change from before and 2,4,6 and 8 weeks after initiating therapy | A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, and triglyceride; hemoglobin; neutrophil counts; platelet counts; and alanine aminotransferase (ALT). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was\>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; for patients with an initial neutrophil count of\<0.5 ×109/L, a response was defined as an increase by at least 100% to\>0.5×109/L; for patients with a neutrophil count of 0.5 to 2.0× 109/L, an increase by at least 100% to \>2.0 × 109/L was considered a response; and for patients with ALT \>400 U/L,response was defined as an ALT decrease of at least 50%. |
| Progression Free Survival | 6 months | from date of inclusion to date of progression, relapse, or death from any cause |
| Adverse events | 6 months | Adverse events including myelosuppression, infection, hemorrhage |
Countries
China
Outcome results
None listed