This study aims to evaluate the efficacy and safety of toripalimab and anlotinib hydrochloride combined with standard chemotherapy in patients with refractory dermatofibrosarcoma protuberans (DFSP) resistant to imatinib therapy, and to provide evidence for the exploration of DFSP treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
All enrolled patients will receive study intervention starting on Day 1 of each 3-week cycle until disease progression, intolerable toxicity, or study withdrawal: Toripalimab 240 mg (fixed dose) administered intravenously once every 3 weeks (Q3W).
All enrolled patients will receive study intervention starting on Day 1 of each 3-week cycle until disease progression, intolerable toxicity, or study withdrawal: Anlotinib 10 mg administered orally once daily on Days 1-14 of each 3-week cycle.
All enrolled patients will receive study intervention starting on Day 1 of each 3-week cycle until disease progression, intolerable toxicity, or study withdrawal: standard chemotherapy based on anthracycline or gemcitabine once every 3 weeks (Q3W).
Objective Response Rate (ORR)
The Objective Response Rate (ORR) is defined as the percentage of patients whose best response on or before the first occurrence of disease progression is a complete response (CR) or partial response (PR). Tumor responses were assessed by investigators using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.
Time frame: From the date of first study treatment until disease progression or death from any cause, whichever occurs first, assessed up to 24months.
Duration of Response (DoR)
Duration of Response (DoR) is defined as the time from the date of first documented response (CR or PR) to date of first occurrence of disease progression as determined by the investigator, or death from any cause, whichever occurs first.
Time frame: From the date of first documented response (complete response [CR] or partial response [PR]) to the time of disease progression or death from any cause, whichever occurs first, assessed up to 24months.
Progression-Free Survival (PFS)
Progression-Free Survival (PFS) is defined as the time from the start of study treatment to the first occurrence of disease progression, or death, whichever occurs first. Tumor responses were assessed by investigators using RECIST version 1.1.
Time frame: Through study completion, an average of 2 years.
Overall Survival (OS)
Overall Survival (OS) is defined as the time from the date of the first study treatment (Day 1) to the date of death from any cause.
Time frame: Through study completion, an average of 2 years.
Disease Control Rate (DCR)
Disease Control Rate (DCR) is defined as the proportion of patients whose best response is CR, PR or SD maintained more than 8 weeks. Tumor responses were assessed by investigators using RECIST version 1.1.
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Time frame: From the date of first study treatment until disease progression or death from any cause, whichever occurs first, assessed up to 24 months.
Best Overall Response (BOR)
Best overall response (BOR) is defined as the best tumor response achieved at any time during treatment, categorized as Complete Response (CR), Partial Response (PR), Stable Disease (SD), or Progressive Disease (PD) per RECIST 1.1 criteria. Responses require confirmation at least 4 weeks later.
Time frame: Through study completion, average follow-up of 2 years.
Time to Response (TTR)
Time to response (TTR) is defined as the time from the date of first study drug administration to the date of first documented and confirmed CR or PR per RECIST 1.1.
Time frame: From first dose until first confirmed response, assessed up to 48 months.
Adverse Events (AEs)
Incidence, severity (graded per NCI CTCAE version 5.0), and causality of adverse events (AEs). Special attention will be paid to AEs of special interest associated with toripalimab, anlotinib, and anthracycline- or gemcitabine-based chemotherapy, including myelosuppression, hypertension, diarrhea, rash, liver function abnormalities, endocrine disorders, and cardiac toxicity (monitored via periodic left ventricular ejection fraction and electrocardiography).
Time frame: From first study drug administration through 40 days after the last dose; overall average follow-up duration is 2 years.