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The goal of this observational study is to learn if interferon-gamma monoclonal antibody combined with dexamethasone works to treat adults with refractory secondary hemophagocytic lymphohistiocytosis (HLH). The main questions it aims to answer are:
How well does this treatment help patients recover from refractory secondary HLH? How safe is this treatment for these patients? Participants will receive interferon-gamma monoclonal antibody and dexamethasone as part of their clinical care. Researchers will monitor participants with regular blood tests, physical exams, and safety checks for up to 8 weeks of treatment. They will collect information about how participants respond to treatment and any side effects that occur.
This study will include about 22 adult participants at Huadong Hospital Affiliated to Fudan University.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Observation group | Participants receive IFN-γ monoclonal antibody by weight-based intravenous infusion. Dexamethasone is administered orally or intravenously. Prophylaxis for infections and standard supportive care are provided as clinically needed. Treatment continues for up to 8 weeks, with regular safety and efficacy assessments. |
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| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (ORR) | The proportion of participants who achieve complete response (CR) or partial response (PR) at the end of 8-week treatment, defined by normalized or improved clinical and laboratory parameters including ferritin, triglycerides, sCD25, blood cell counts, and hemophagocytosis. | Up to 8 weeks after initiation of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Progression-Free Survival (PFS) | Time from treatment start to disease progression or death from any cause, whichever occurs first. | Up to 12 months after treatment initiation |
| Overall Survival (OS) |
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Inclusion Criteria:
Exclusion Criteria:
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This study will enroll adult participants aged 18 years and older with a confirmed diagnosis of refractory secondary hemophagocytic lymphohistiocytosis (HLH) based on the HLH-2004 diagnostic criteria. Participants must have demonstrated inadequate response or disease progression following standard first-line or second-line HLH-directed therapy. All participants must be able to provide written informed consent prior to any study-related procedures.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Huadong Hospital, Fudan University | Shanghai | Shanghai Municipality | 200040 | China |
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1 year after completion of this study
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Time from treatment start to death from any cause.
| Up to 12 months after treatment initiation |
| Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) | Number and severity of treatment-emergent adverse events and serious adverse events, graded according to NCI-CTCAE v5.0. | Up to 28 days after the last dose of study treatment |