Efficacy and Safety of Interferon-Gamma Monoclonal Antibody Combined With Dexamethasone in the Treatment of Refractory Adult Secondary Hemophagocytic Lymphohistiocytosis

Fudan University22 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Adult Refractory Secondary Hemophagocytic Lymphohistiocytosis Secondary Hemophagocytic Lymphohistiocytosis HLH

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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.

Time frame: Up to 8 weeks after initiation of treatment

Secondary Outcomes

Progression-Free Survival (PFS)

Time from treatment start to disease progression or death from any cause, whichever occurs first.

Time frame: Up to 12 months after treatment initiation

Overall Survival (OS)

Time from treatment start to death from any cause.

Time frame: 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.

Time frame: Up to 28 days after the last dose of study treatment