Ruxolitinib as a Salvage Therapy for Hemophagocytic Lymphohistiocytosis

Study Overview
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Brief Summary
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This study aimed to investigate the efficacy and safety of different doses of ruxolitinib as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis(HLH).

Official Title
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Clinical Study of Different Doses of Ruxolitinib as a Salvage Therapy for Refractory/Relapsed Hemophagocytic Lymphohistiocytosis

Conditions
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Hemophagocytic Lymphohistiocytosis

Intervention / Treatment
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* Drug: low dose ruxolitinib
* Drug: high dose ruxolitinib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: meet hemophagocytic lymphohistiocytosis (HLH)-04 diagnostic criteria; treated with HLH-94 no less than 2 weeks before enrollment and did not achieve at least PR; or relapsed patients after remission； Life expectancy exceeds 1 month; Age≥1 year old and ≤75 years old, gender is not limited； Before the start of the study, total bilirubin ≤ 10 times the upper limit of normal; serum creatinine ≤ 1.5 times normal； Serum human immunodeficiency virus(HIV) antigen or antibody negative； Hepatitis C virus (HCV) antibody is negative, or HCV antibody is positive, but HCV RNA is negative； Both hepatitis B virus (HBV) surface antigen and HBV core antibody were negative. If any of the above is positive, peripheral blood hepatitis B virus DNA titer should be detected, and less than 1×103 copies/ml can enter the group； Informed consent. Exclusion Criteria: Pregnancy or lactating Women； Allergic to ruxolitinib； Active bleeding of the internal organs； uncontrollable infection； Serious mental illness; Non-melanoma skin cancer history; Patients unable to comply during the trial and/or follow-up phase; Participate in other clinical research at the same time.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 75 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
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Low dose<br> | Drug: low dose ruxolitinib<br>* The dose for adult patients(Age>=14 years) is generally 10 mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 5mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 2.5mg twice daily.<br>|
| Experimental: High dose<br> | Drug: high dose ruxolitinib<br>* The dose for adult patients(Age>=14 years) is generally 20mg twice daily. For children(Age<14 years ,weight >=25kg),the dose was generally 10mg twice daily. For children(Age<14 years, weight <25kg), the dose was generally 5mg twice daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response rate | complete response (CR) and partial response (PR) rates | 1 years | 
| Progression Free Survival | from date of inclusion to date of progression, relapse, or death from any cause Overall Survival | 1 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival | from the date of inclusion to date of death, irrespective of cause Adverse Events | 1 years | 
| Adverse events | Adverse events including myelosuppression, infection, bleeding and so on. | 1 year |