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The goal of this clinical trial is to learn about efficacy and safety of GTP regimen in refractory/recurrent hemophagocytic lymphohistiocytosis. The main questions it aims to answer are:
Refractory/recurrent hemophagocytic lymphohistiocytosis will be treated with emapalumab combined with teniposide and methylprednisolone. Observed the overall response rate and side effects.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GTP regimen treated patients | Experimental | R/R HLH patients treated with GTP regimen |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GTP regimen | Drug | Refractory/recurrent hemophagocytic lymphohistiocytosis patients will be treated with GTP regimen (Emapalumab+Teniposide+Methylprednisolone) |
|
| Measure | Description | Time Frame |
|---|---|---|
| ORR | Overall remission rate | 1 year |
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Inclusion Criteria:
Male and female patients ≥1 month of age with HLH
Definitive diagnosis of HLH: meets the molecular diagnosis of pHLH or has a family history of HLH, or meets at least 5 of the 8 diagnostic criteria of HLH-2004.
The investigators assessed the presence of refractory/recurrent HLH disease. Patients must meet one of the following criteria assessed by the investigator:
According to the researchers, the expected survival was more than 2 weeks.
The informed consent is signed by the patient or his legal guardian (patients under 18 years), or by the patient's legally authorized representative.
Women who are fertile are willing to use a highly effective contraceptive method from the start of the study until 6 months after the end of the last course of treatment.
Exclusion Criteria:
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| ID | Term |
|---|---|
| D012008 | Recurrence |
| D051359 | Lymphohistiocytosis, Hemophagocytic |
| ID | Term |
|---|---|
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D015616 | Histiocytosis, Non-Langerhans-Cell |
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| D015614 | Histiocytosis |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |