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Updated: Aug 4, 2026
Preparation and Characterization of Lipophilic Doxorubicin Pro-drug Micelles
Published on: August 2, 2016
Phase II trial of liposomal doxorubicin (Doxil) in advanced soft tissue sarcomas
T Chidiac1, G T Budd, R Pelley
1The Taussig Cancer Center, Cleveland Clinic Foundation, OH 44195, USA.
Purpose:
To assess the objective response rate, toxicity experienced, progression-free survival, and overall survival of patients with previously untreated advanced soft tissue sarcomas treated with a liposomal doxorubicin formulation (Doxil).
Methods:
Patients with metastatic or recurrent soft tissue sarcoma who had received no prior chemotherapy for advanced disease were treated with liposomal doxorubicin (Doxil) according to a two stage accrual design. Doxil was administered at 50 mg/m2 every 4 weeks. A total of 15 patients were treated and are evaluable for response and toxicity.
Results:
The male/female ratio was 7/8, the median age was 60 years (34-75) and the ECOG performance status was 0-1 in >90% of patients. Leiomyosarcoma (7/15) and malignant fibrous histiocytoma (2/15) were the most common histologic diagnoses. No objective responses were observed in the 15 evaluable patients. No lethal toxicity occurred. Grade 3-4 leukopenia or neutropenia were reported in 3/15 (20%) patients. Grade 3 mucositis or hand-foot syndrome occurred in 2/15 (13%) and 1/15 (7%) patients respectively and seemed more severe in older patients. The median time to progression was 1.9 months (range 0.9-6.2). Twelve patients have now died. The Kaplan-Meier estimate of median overall survival is 12.3 months. As called for in the study design, accrual was terminated because no responses were obtained in the first 15 patients.
Conclusion:
Though well-tolerated, Doxil given according to this dose and schedule to patients with advanced soft tissue sarcoma had no significant therapeutic activity. A correlation between older age and skin/mucosal toxicity of Doxil is suggested in this study but needs confirmation. Future investigations of Doxil in soft tissue sarcomas should use a different schedule and dose.
Insights
Liposomal doxorubicin (Doxil) showed no significant therapeutic activity in advanced soft tissue sarcomas. This well-tolerated treatment did not improve progression-free survival, prompting recommendations for different dosing in future studies.
Area of Science:
- Oncology
- Pharmacology
Background:
- Soft tissue sarcomas are rare cancers.
- Liposomal doxorubicin (Doxil) is an established chemotherapy agent.
- Novel formulations and schedules are explored for improved efficacy.
Purpose of the Study:
- To evaluate the efficacy and safety of liposomal doxorubicin (Doxil) in previously untreated advanced soft tissue sarcomas.
- To determine objective response rate, progression-free survival, and overall survival.
- To assess toxicity profiles of Doxil in this patient population.
Main Methods:
- A two-stage accrual design was used for 15 evaluable patients with advanced soft tissue sarcoma.
- Liposomal doxorubicin (Doxil) was administered at 50 mg/m2 every 4 weeks.
- Response, survival, and toxicity were assessed.
Main Results:
- No objective responses were observed in the 15 evaluable patients.
- Median progression-free survival was 1.9 months; median overall survival was 12.3 months.
- Grade 3-4 leukopenia/neutropenia occurred in 20%, and mucositis/hand-foot syndrome in 13%/7% respectively, with increased toxicity in older patients.
Conclusions:
- Liposomal doxorubicin (Doxil) at this dose and schedule demonstrated no significant therapeutic activity in advanced soft tissue sarcomas.
- The treatment was generally well-tolerated, but older patients may experience more severe skin and mucosal toxicity.
- Future research should investigate alternative dosing schedules for Doxil in soft tissue sarcomas.
