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Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.
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;
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Newly diagnosed LCH patients | Experimental | (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); |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cytarabine (Ara-C) | Drug | The 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From enrollment to the end of treatment until 3 years |
| Measure | Description | Time Frame |
|---|---|---|
| Overall survival (OS) | OS was defined as the time from cytarabine and thalidomide initiation to death or last follow-up. | From enrollment to the end of treatment until 3 years |
| Overall response rate (ORR) |
| Measure | Description | Time Frame |
|---|---|---|
| FACT-G (Functional Assessment of Cancer Therapy-General) | Score of FACT-G (Functional Assessment of Cancer Therapy-General) | From enrollment to the end of treatment until 3 years |
| Correlation between next-generation sequence (NGS) / circulating free DNA (cfDNA) and efficacy/EFS |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xinxin XX Cao, doctor | Contact | +86-18618315968 | caoxinxin@126.com | |
| Huilei HL Miao, doctor | Contact | +86-18801317695 | miaohl13@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Xinxin XX Cao, doctor | NCC, CICAMS | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| 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 | Recruiting | Beijing | China |
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| ID | Term |
|---|---|
| D006646 | Histiocytosis, Langerhans-Cell |
| ID | Term |
|---|---|
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D015614 | Histiocytosis |
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| ID | Term |
|---|---|
| D003561 | Cytarabine |
| D013792 | Thalidomide |
| ID | Term |
|---|---|
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
| D011743 | Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
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A prospective, multicenter, single-arm study.Planned enrollment of 50 patients.
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|
| Thalidomide (100mg) | Drug | The 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. |
|
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.
| From enrollment to the end of treatment until 3 years |
| Adverse events | Toxicities were recorded and graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0. | From enrollment to the end of treatment until 3 year |
Correlation between the positivity of next-generation sequence (NGS) / circulating free DNA (cfDNA) and efficacy/EFS |
| From enrollment to the end of treatment until 3 years |
|
| D008206 |
| Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006571 |
| Heterocyclic Compounds |
| D001087 | Arabinonucleosides |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D010797 | Phthalimides |
| D010795 | Phthalic Acids |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D010881 | Piperidones |
| D010880 | Piperidines |
| D054833 | Isoindoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |