Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.
Clearly diagnose and treat adult patients with multisystem or single-system multifocal LCH;
Aged between 18 and 70 years;
Multisystem involvement or single-system multifocal disease;
No prior systemic treatment (patients who have only received local radiotherapy or surgery may be enrolled);
Patients with LCH involving the central nervous system;
Single-system single-lesion LCH;
Subjects who have undergone major surgery within 4 weeks prior to the first dose in the study;
Subjects who have received radiotherapy within 4 weeks prior to the first dose in the study; subjects with a history of myocardial infarction within the past year;
Single group
Cytarabine (Ara-C)
DrugThe treatment regimen consists of Ara-C administered at a dose of 100 mg/m² from days 1 to 5, combined with thalidomide at a dose of 100 mg from days 1 to 35, repeated every 35 days for a total of 12 cycles. Following this, thalidomide will be given as monotherapy at a dose of 100 mg from days 1 to 28, administered every 28 days for an additional 12 cycles of maintenance treatment. In total, participants will undergo 24 cycles of treatment.
Thalidomide (100mg)
DrugThe treatment regimen consists of AraC administered at a dose of 100 mg/m² from days 1 to 5, combined with thalidomide at a dose of 100 mg from days 1 to 35, repeated every 35 days for a total of 12 cycles. Following this, thalidomide will be given as monotherapy at a dose of 100 mg from days 1 to 28, administered every 28 days for an additional 12 cycles of maintenance treatment. In total, participants will undergo 24 cycles of treatment.
Event-Free Survival (EFS)
The primary endpoint was event-free survival (EFS), with events defined as poor response (stable disease or progressive disease) to cytarabine and thalidomide therapy, or death from any cause. EFS was measured from initiation of cytarabine and thalidomide therapy until the date of the first documented event, with censoring of patients without events at the last follow-up.
Overall survival (OS)
OS was defined as the time from cytarabine and thalidomide initiation to death or last follow-up.
Overall response rate (ORR)
The overall response rate (ORR) was defined as the cumulative proportion of patients achieving either a complete response (CR) or partial response (PR) following therapy.
Adverse events
Toxicities were recorded and graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
FACT-G (Functional Assessment of Cancer Therapy-General)
Score of FACT-G (Functional Assessment of Cancer Therapy-General)
Correlation between next-generation sequence (NGS) / circulating free DNA (cfDNA) and efficacy/EFS
Correlation between the positivity of next-generation sequence (NGS) / circulating free DNA (cfDNA) and efficacy/EFS
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