This study aimed to investigate the efficacy and safety of venetoclax combined with dexamethasone and etoposide as a salvage therapy for hemophagocytic lymphohistiocytosis.
Hemophagocytic lymphohistiocytosis (HLH) is a rare and aggressive disease with high mortality and poor prognosis. The HLH-94 and HLH-04 regimens are most commonly used, but approximately 30% of the patients remain unresponsive. Currently, there is no unified evidence-based salvage treatment. There is no unified salvage regimen. This study plans to enroll 20 patients with refractory and recurrent hemophagocytic lymphohistiocytosis. Subjects will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.The safety was evaluated once a week, and the efficacy was evaluated once every 2 weeks. Overall response rate will be evaluated at 8 weeks. Patients were followed up every 3 months thereafter.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Venetoclax 100mg qd d1，200mg qd d2，400mg qd d3-56
Dexamethasone 10mg/m2 qd d1-14, 5mg/m2 qd d15-28, 2.5 mg/m2 qd d29-42, and 1.25mg/m2 qd43-56
Etoposide 75mg/m2 qw d1-56
Beijing Friendship Hospital, Capital Medical University
Beijing, Beijing Municipality, China
RECRUITINGOverall response rate
The percentage of cases with complete response (CR) and partial response (PR) after treatment in the total evaluable cases
Time frame: 1 year
Incidence and severity of adverse effects
The percentage of cases with adverse effects and its severity.
Time frame: 1 years
Overall survival
OS is defined as the time from the initiaion of treatment to death from any cause or July 2025.
Time frame: 1 years
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