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In adult patients with relapsed/refractory Langerhans cell histiocytosis (LCH), a treatment regimen of XTD regimen (Selinexor, Thalidomide and Dexamethasone) is planned to be used.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adult patients with relapsed or refractory LCH | Experimental | (1) Relapsed or refractory LCH; (2) Age ≥ 18 years; (3) Multi-focal single system or multi-system involvement; (4) ECOG PS 0-2 score |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Selinexor | Drug | The combined treatment period includes 12 cycles: receiving Selinexor (60mg, D1, 8, 15, 22), Thalidomide (100mg, D1-28), and Dexamethasone (40mg, D1, 8, 15, 22) as oral treatment, with each cycle lasting 28 days, for a total of 12 cycles of combined treatment, or until disease progression, death, or occurrence of intolerable toxicity. Alternatively, until disease progression, death, or occurrence of intolerable toxicity. |
| Measure | Description | Time Frame |
|---|---|---|
| PFS | PFS defined as the time from XTD initiation to first documented disease progression, relapse after XTD, death from any cause, or last follow-up. | From enrollment to the end of treatment at 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| ORR | The overall response rate (ORR) was defined as the cumulative proportion of patients attaining either a complete response (CR) or partial response (PR) . | From enrollment to the end of treatment at 8 weeks |
| OS |
| Measure | Description | Time Frame |
|---|---|---|
| Correlation between the positivity of NGS in MAPK pathway and therapeutic efficacy/PFS | Correlation between the positivity of NGS in MAPK pathway and therapeutic efficacy/PFS | From enrollment to the end of treatment at 8 weeks |
| Fact-G |
Inclusion Criteria:
Exclusion Criteria:
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| 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, beijing, | Beijing | China |
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A multicenter, prospective, interventional study, with a planned number of subjects: approximately 40 cases.
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|
| Thalidomide (100mg) | Drug | The combined treatment period includes 12 cycles: receiving Selinexor (60mg, D1, 8, 15, 22), Thalidomide (100mg, D1-28), and Dexamethasone (40mg, D1, 8, 15, 22) as oral treatment, with each cycle lasting 28 days, for a total of 12 cycles of combined treatment, or until disease progression, death, or occurrence of intolerable toxicity. Alternatively, until disease progression, death, or occurrence of intolerable toxicity. |
|
| Dexamethasone | Drug | The combined treatment period includes 12 cycles: receiving Selinexor (60mg, D1, 8, 15, 22), Thalidomide (100mg, D1-28), and Dexamethasone (40mg, D1, 8, 15, 22) as oral treatment, with each cycle lasting 28 days, for a total of 12 cycles of combined treatment, or until disease progression, death, or occurrence of intolerable toxicity. Alternatively, until disease progression, death, or occurrence of intolerable toxicity. |
|
OS was measured from XTD initiation to death or last follow-up
| From enrollment to the end of treatment at 8 weeks |
| 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 at 8 weeks |
The score of Functional Assessment of Cancer Therapy - General
| From enrollment to the end of treatment at 8 weeks |
| ID | Term |
|---|---|
| D006646 | Histiocytosis, Langerhans-Cell |
| ID | Term |
|---|---|
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D015614 | Histiocytosis |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C585161 | selinexor |
| D013792 | Thalidomide |
| D003907 | Dexamethasone |
| ID | Term |
|---|---|
| D010797 | Phthalimides |
| D010795 | Phthalic Acids |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D010881 | Piperidones |
| D010880 | Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D054833 | Isoindoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
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