Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH

Not Applicable

Recruiting

• Conditions: Langerhans Cell Histiocytosis
• Interventions: Drug: Cytarabine (Ara-C); Drug: Thalidomide (100mg)

• Registration Number: NCT07187193
• Lead Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences

Brief Summary

Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.

Detailed Description

AraC 100 mg/m² d1-5+ thalidomide 100mg d1-35, q35 days ,for a total of 12 cycles;followed by thalidomide 100mg, qd1-28 q28d,for a total of 12 cycles of monotherapy maintenance reatment;

Study Timeline

• Study Start: 2025-04-12
• Study First Submitted: 2025-08-25
• Status Verified: 2025-09
• Study First Submitted QC: 2025-09-16
• Last Update Submitted: 2025-09-16
• Study First Posted: 2025-09-22
• Last Update Posted: 2025-09-22
• Primary Completion: 2027-04-12
• Study Completion: 2027-04-13

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• 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);
• ECOG performance status score ≤ 2;
• Judged by clinicians as suitable for treatment with this protocol;
• Patients or their families able to understand the study protocol and willing to participate in the study, providing written informed consent.

Exclusion Criteria

• 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;
• Patients with New York Heart Association (NYHA) class 3 or 4 congestive heart failure, or a history of NYHA class 3 or 4 congestive heart failure;
• Pregnant or lactating women;
• Abnormal liver and kidney function: creatinine level ≥176.8μmol/l (2mg/dl), transaminase and bilirubin levels more than 2 times the upper limit of normal (for LCH patients with liver involvement, bilirubin levels more than 10 times the upper limit of normal);
• Abnormal blood counts: absolute neutrophil count less than 1×10^9/L, platelet count less than 50×10^9/L;
• Patients or their families unable to understand the conditions and objectives of this study;
• Any other circumstances in which the investigator deems the patient unsuitable to participate in this trial.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Newly diagnosed LCH patients	Cytarabine (Ara-C)	(1) The histopathological diagnosis of LCH is confirmed; (2) Multi-system involvement or single-system with multiple lesions; (3) No prior systemic treatment (patients who have only received local radiotherapy or surgery are eligible);
Newly diagnosed LCH patients	Thalidomide (100mg)	(1) The histopathological diagnosis of LCH is confirmed; (2) Multi-system involvement or single-system with multiple lesions; (3) No prior systemic treatment (patients who have only received local radiotherapy or surgery are eligible);

Primary Outcome Measures

Name	Time	Method
Event-Free Survival (EFS)	From enrollment to the end of treatment until 3 years	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.

Secondary Outcome Measures

Name	Time	Method
Overall survival (OS)	From enrollment to the end of treatment until 3 years	OS was defined as the time from cytarabine and thalidomide initiation to death or last follow-up.
Overall response rate (ORR)	From enrollment to the end of treatment until 3 years	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	From enrollment to the end of treatment until 3 year	Toxicities were recorded and graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.

Trial Locations

Locations (1)

Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College; 🇨🇳 Beijing, China