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Aplidin in patients with advanced dedifferentiated liposarcomas: a French Sarcoma Group Single-Arm Phase II study
M Toulmonde1, A Le Cesne2, S Piperno-Neumann3
1Department of Medical Oncology, Institut Bergonié, Bordeaux.
Background:
Preclinical data have suggested a therapeutic role of JUN pathway activation in dedifferentiated liposarcoma (DDLPS) tumorigenesis. Aplidin is a drug inducing apoptosis through a strong, sustained activation of c-Jun NH2-terminal kinase.
Methods:
This phase II trial included patients with progressive advanced DDLPS. They received Aplidin 5 mg/m(2) days 1-15, 28-day cycle until disease progression or unacceptable toxicity. The primary end point was the 3-month nonprogression rate (PFS3) defined as the proportion of patients with nonprogressive disease at 3 months. A PFS3 of 40% considered as a reasonable objective to claim drug efficacy.
Results:
Between August 2012 and May 2013, 24 patients were included. Sixteen had received prior chemotherapy. Twenty-two were assessable for efficacy. The PFS3 was 9.1% [95% confidence interval (CI) 1.1-29.2]. Median progression-free and overall survivals were 1.6 months (95% CI 1.4-2.6) and 9.2 months (95% CI 6.6-). The most frequent adverse events of any grade were nausea, fatigue, anorexia, vomiting and diarrhea.
Conclusion:
Aplidin did not meet the primary end point of this trial and do not deserve further investigation in DDLPS.
Clinicaltrialsgov Identifier:
NCT01876043.
Insights
Aplidin did not show efficacy in treating dedifferentiated liposarcoma (DDLPS). The phase II trial found the drug did not meet its primary endpoint, suggesting no further investigation is warranted for DDLPS treatment.
Area of Science:
- Oncology
- Pharmacology
Background:
- Preclinical studies indicated JUN pathway activation in dedifferentiated liposarcoma (DDLPS) tumorigenesis.
- Aplidin, a drug, induces apoptosis via sustained c-Jun NH2-terminal kinase activation.
Purpose of the Study:
- To evaluate the efficacy of Aplidin in patients with advanced DDLPS.
- To determine if Aplidin could achieve a 3-month nonprogression rate (PFS3) of 40%.
Main Methods:
- A phase II trial administered Aplidin (5 mg/m(2) on days 1-15 of a 28-day cycle) to patients with progressive advanced DDLPS.
- The primary endpoint was the PFS3, with disease progression or unacceptable toxicity as reasons for discontinuation.
Main Results:
- Twenty-two patients were assessable for efficacy; the PFS3 was 9.1% (95% CI 1.1-29.2), falling short of the 40% target.
- Median progression-free survival was 1.6 months, and median overall survival was 9.2 months.
- Common adverse events included nausea, fatigue, anorexia, vomiting, and diarrhea.
Conclusions:
- Aplidin failed to meet the primary endpoint in this DDLPS trial.
- The drug does not warrant further investigation for DDLPS treatment.
