Zanubrutinib for HLH

Study Overview
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Brief Summary
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This study is a prospective single-arm clinical study, focusing on Hemophagocytic lymphohistocytosis，to evaluate the clinical efficacy and safety of zanubrutinib.

Official Title
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Zanubrutinib Monotherapy in Patients With Hemophagocytic Lymphohistiocytosis

Conditions
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HLH

Intervention / Treatment
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* Drug: Zanubrutinib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Unlimited gender, age ≥ 14 years old; HLH was diagnosed according to HLH-04 diagnostic criteria; Before the study, there was no severe liver function injury; Serum creatinine ≤ 1.5 times the upper limit of normal value; Sign informed consent Exclusion Criteria: Allergic to zebutinib; Currently active important cardiovascular diseases with clinical significance; The functions of important organs such as heart and lung unrelated to HLH were seriously abnormal; Known human immunodeficiency virus (HIV) or active hepatitis B (HBV) or hepatitis C (HCV) infection; Pregnant or lactating women and patients of childbearing age who refuse to take appropriate contraceptive measures during this trial; Serious mental illness; Active massive hemorrhage of internal organs; Uncontrollable infection; At the same time, participate in other clinical researchers

Ages Eligible for Study
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Minimum Age: 14 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: zanubrutinib<br> | Drug: Zanubrutinib<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| evaluation of treatment response | 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%. | Change from before and 2,4,6 and 8 weeks after initiating therapy | 
| Progression Free Survival | from date of inclusion to date of progression, relapse, or death from any cause | 6 months | 
| Adverse events | Adverse events including myelosuppression, infection, hemorrhage | 6 months |