Adult Refractory Secondary Hemophagocytic Lymphohistiocytosis, HLH, Secondary Hemophagocytic Lymphohistiocytosis
Conditions
Brief summary
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.
Interventions
None listed
Sponsors
Fudan University
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age ≥ 18 years * Confirmed diagnosis of secondary hemophagocytic lymphohistiocytosis (HLH) according to HLH-2004 criteria * Refractory HLH defined as inadequate response or progression after at least 2 weeks of standard first-line or second-line therapy * Able to provide written informed consent
Exclusion criteria
* Active infection with mycobacteria, histoplasma, CMV, herpes zoster, or HIV * Left ventricular ejection fraction \< 50% * Concurrent use of other anti-neoplastic or investigational agents * Other uncontrolled infections judged by the investigator * Psychiatric disorders or poor protocol compliance
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Response Rate (ORR) | Up to 8 weeks after initiation of treatment | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Progression-Free Survival (PFS) | Up to 12 months after treatment initiation | Time from treatment start to disease progression or death from any cause, whichever occurs first. |
| Overall Survival (OS) | Up to 12 months after treatment initiation | Time from treatment start to death from any cause. |
| Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) | Up to 28 days after the last dose of study treatment | Number and severity of treatment-emergent adverse events and serious adverse events, graded according to NCI-CTCAE v5.0. |
Countries
China
Outcome results
None listed