Ruxolitinib Combined With Dexamethasone for HLH

Study Overview
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Brief Summary
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A protocol named as HLH-DR for patients with refractory and secondary hemophagocytic lymphohistiocytosismay.

Detailed Description
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A modified protocol about ruxolitinib combined with dexamethasone which includes oral ruxolitinib (2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）. Dexamethasone (10mg/m2.d) delayes for the first 2 weeks （form week 1 to 2），(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8. Every 2 weeks, patients can be evaluated based the diagnosed index

Official Title
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Ruxolitinib Combined With Dexamethasone for Pediatric Refractory and Secondary Hemophagocytic Lymphohistiocytosis

Conditions
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Hemophagocytic Lymphohistiocytosis

Intervention / Treatment
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* Drug: Ruxolitinib
* Drug: Dexamethasone


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Secondary and refractory HLH. Exclusion Criteria: Family HLH.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 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: Randomized
Intervention Model: Sequential Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ruxolitinib and Placebo<br>Ruxolitinib 2.5 mg twice daily by oral | Drug: Ruxolitinib<br>* Ruxolitinib （2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）<br>* Other names: Jakafi;Drug: Dexamethasone<br>* Dexamethasone (10mg/m2.d) delayes for the first 2 weeks（form week 1 to 2),(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8.<br>* Other names: Hormone;|
| Placebo Comparator: Placebo and Ruxolitinib<br>Sugar pill 2.5 mg twice daily by oral | Drug: Ruxolitinib<br>* Ruxolitinib （2.5 mg twice daily for patients if the age<14 years and the weight <25kg，5 mg twice daily for patients if the age<14 years and the weight≥25kg, 10mg twice daily for patients if the age ≥14 years and<18 years）<br>* Other names: Jakafi;Drug: Dexamethasone<br>* Dexamethasone (10mg/m2.d) delayes for the first 2 weeks（form week 1 to 2),(5mg/m2.d) from week 3 to 4，(2.5mg/m2.d) from week 5 to 6），1.25mg/m2.d for week 7，and dropped off on week 8.<br>* Other names: Hormone;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Severity of disease | Serum ferritin>2000ng/ml, disease actvie; serum ferritin<2000ng/ml, disease control; | 2 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Axillary temperature | 36<Axillary temperature<37.2, disease control; 37.2<Axillary temperature<38.2, disease partly control; Axillary temperature>38.2, disease active | 2 weeks |