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A phase II study of thalidomide in patients with brain metastases from malignant melanoma
Lene W Vestermark1, Susanne Larsen, Birgit Lindeløv
1Department of Oncology, Odense University Hospital, Odense C, Denmark. lene.vestermark@ouh.regionsyddanmark.dk
Introduction:
Brain metastases develop in nearly half of the patients with advanced melanoma and in 15 to 20% of these patients CNS is the first site of relapse. Overall median survival is short, ranging from 2 to 4 months. Thalidomide has antiangiogenic and immunomodulatory effects. Results obtained in prior trials indicate that Thalidomide acts as a cytostatic agent in metastatic melanoma. We evaluated single agent antitumour activity and toxicity of Thalidomide in a phase II setting in patients with brain metastases associated with metastatic melanoma.
Material And Methods:
Patients with measurable metastatic melanoma in progression and with PS < or = 2 were enrolled in the study. Thalidomide was given orally. Dose was escalated over 4 weeks from 100 mg/day to 400 mg/day. Primary objective of the study was to determine response rate, according to RECIST. Secondary objectives were to estimate time to progression, overall survival and to evaluate tolerability of the regimen.
Results:
Twenty five men and 11 women were enrolled in the study, median age 48 years. Among 36 eligible patients 35 were evaluable for response. None of the patients obtained a response in brain metastases. Three patients obtained a partial response in extracranial lesions. Toxicity was acceptable and manageably. Median time to progression and overall survival time was 1.7 and 3.1 months, respectively.
Conclusion:
There were no objective responses in the brain but single agent Thalidomide has some activity in melanoma patients with brain metastases. It has encouraged us to investigate Thalidomide in combination with Temozolomide, a very lipophilic agent, in this group of patients.
Insights
Single-agent Thalidomide showed no objective response in melanoma patients with brain metastases. However, it demonstrated manageable toxicity and some activity in extracranial lesions, prompting further combination studies.
Area of Science:
- Oncology
- Melanoma Research
- Neuro-oncology
Background:
- Advanced melanoma frequently metastasizes to the brain, with limited survival outcomes.
- Thalidomide exhibits antiangiogenic and immunomodulatory properties, with prior evidence of cytostatic effects in melanoma.
Purpose of the Study:
- To evaluate the single-agent antitumour activity and toxicity of Thalidomide in patients with melanoma brain metastases.
- To determine the response rate, time to progression, and overall survival.
Main Methods:
- Phase II study of orally administered Thalidomide with dose escalation from 100 mg/day to 400 mg/day over 4 weeks.
- Patients with measurable metastatic melanoma in progression and performance status <= 2 were enrolled.
- Response was assessed by RECIST criteria; tolerability and survival were secondary objectives.
Main Results:
- No objective responses were observed in brain metastases among 35 evaluable patients.
- Three patients achieved partial response in extracranial lesions.
- Median time to progression was 1.7 months, and median overall survival was 3.1 months, with acceptable toxicity.
Conclusions:
- Single-agent Thalidomide did not yield objective responses in melanoma brain metastases.
- Thalidomide demonstrated some activity in extracranial melanoma lesions and was well-tolerated.
- These findings support further investigation of Thalidomide in combination with other agents, such as Temozolomide.
