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Pegylated Liposomal Doxorubicin, An Effective, Well-tolerated Treatment For Refractory Aggressive Fibromatosis.
Published 2009 · Medicine
BACKGROUND Aggressive fibromatosis (AF) or desmoid tumour is a monoclonal proliferation which is locally invasive but does not metastasize. If local treatment fails to control the disease, systemic treatment with anti-oestrogens, non-steroidal anti-inflammatory drugs (NSAIDs) or chemotherapy can be used. Recent reports indicate that pegylated liposomal doxorubicin (PLD) is effective. METHODS Twelve patients with AF received PLD between February 2006 and May 2009. PLD was administered intravenously (iv) at 50mg/m(2) over 1h every 4 weeks. RESULTS The female/male ratio was 11:1 and median age at presentation was 29 years (range 3-53). Objective response (PR) was achieved in 4 (36%) of 11 patients. In one case ongoing shrinkage of the tumour was observed for over 12 months and partial remission was achieved at 14 months after the completion of treatment. Seven patients achieved stable disease. One patient is currently undergoing chemotherapy. Clinical benefit in terms of pain relief, improved mobility or cosmesis was observed in 11 patients. Nine patients (75%) had no evidence of progression at the end of this follow-up period and disease control has ranged from 7 to 39 months with a median of 14 months. The most severe toxicities observed were palmar-plantar erythema (4) and mucositis (3). In 6 cases (55%) toxicity resulted in dose reduction. CONCLUSION This is the largest series of patients with AF receiving PLD reported to date. PLD as a single agent therapy has acceptable toxicity and highly promising activity in unresectable AF and may provide long-term clinical benefit in some patients.