DEP-Ru Regimen as a Salvage Therapy for HLH
Study Details
Study Description
Brief Summary
This study aimed to investigate the efficacy and safety of ruxolitinib together with liposomal doxorubicin, etoposide and methylprednisolone (DEP-Ru) as a salvage therapy for refractory/relapsed hemophagocytic lymphohistiocytosis.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: DEP-Ru doxorubicin (doxorubicin hydrochloride liposome injection) 25 mg/m2 day 1; etoposide 100 mg/m2 was administered day1; methylprednisolone 2 mg/kg days 1 to 5,then gradually reduce; ruxolitinib 0.3mg/kg/d。This regimen was repeated after 2 weeks. |
Drug: DEP-Ru
Drug: ruxolitinib
Drug: doxorubicin
Drug: etoposide
Drug: methylprednisolone
|
Outcome Measures
Primary Outcome Measures
- Evaluation of treatment response [Change from before and 2,4,6 and 8 weeks after initiating DEP-Ru salvage 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%.
Secondary Outcome Measures
- Survival [from the time patients received DEP-Ru salvage therapy up to 24 months or June 2021]
survival
- Adverse events that are related to treatment [through study completion, an average of 2 years]
Adverse events including myelosuppression, infection, bleeding and so on.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
meet HLH-04 diagnostic criteria;
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treated with HLH-94 no less than 2 weeks before enrollment and did not achieve at least PR; or relapsed patients after remission；
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Life expectancy exceeds 1 month;
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Age≥1 year old and ≤70 years old, gender is not limited；
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Before the start of the study, total bilirubin ≤ 10 times the upper limit of normal; serum creatinine ≤ 1.5 times normal；
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Serum HIV antigen or antibody negative；
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HCV antibody is negative, or HCV antibody is positive, but HCV RNA is negative；
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Both 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；
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Echocardiographic examination of LVEF ≥ 50%;
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Informed consent.
Exclusion Criteria:
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Heart function above grade II (NYHA)；
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Accumulated dose of doxorubicin above 300mg/m2 or epirubicin above 450mg/m2；
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Pregnancy or lactating Women；
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Allergic to Pegaspargase, doxorubicin or etoposide；
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Active bleeding of the internal organs；
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uncontrollable infection；
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Serious mental illness;
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Non-melanoma skin cancer history;
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Patients unable to comply during the trial and/or follow-up phase;
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Participate in other clinical research at the same time.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Beijing Friendship Hospital, Capital Medical University | Beijing | Beijing | China | 100050 |
Sponsors and Collaborators
- Beijing Friendship Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
- DEP-Ru HLH