Authors' objectives:

To provide a rapid review of the effectiveness and cost-effectiveness of temozolomide (TMZ) in the treatment of primary malignant brain tumours (anaplastic astrocytoma (AA) and glioblastoma multiforme (GBM)).

Authors' results and conclusions: Although the quality of the available evidence is relatively poor, gliomas do appear to show some response to TMZ. The main benefit in patients with GBM, demonstrated in one RCT and one relatively large uncontrolled study, is an increase (13%) in the estimated proportion of patients remaining progression-free at 6 months and a significant increase in median progression-free survival (PFS) of approximately 4 weeks. No significant overall survival advantage was found in comparison with procarbazine. For patients with AA, one large uncontrolled study suggests some improvement in both PFS and possibly in survival. The magnitude of any benefit in AA is difficult to quantify due to the lack of a within-study comparison of TMZ with an alternative treatment regimen. Subgroup analyses provide some suggestion of better outcomes in patients who were chemotherapy-nave, although patient numbers were small. As adjuvant chemotherapy is not commonly used in the UK, these results may be more applicable to the UK population, but require confirmation in large RCTs. TMZ appears to cause few serious adverse effects, with vomiting usually well controlled by prophylactic anti-emetic regimens. Some clinicians believe that toxicity, particularly myelosuppression, is more predictable with TMZ and this has been noted as one of the advantages of this drug over others. Empirical evidence is, however, limited. One of the major claims of benefit from TMZ is that conferred on health-related QoL. There is some evidence that QoL is improved from recurrence until the point of disease progression for patients with GBM or AA.

Authors' recommendations: It is the authors opinion that the evidence is currently too weak for firm conclusions to be drawn. However, a speculative economic model suggests some indication of benefit from TMZ, at a cost per QALY gained of around 40,000 GBP. The incidence of malignant glioma is relatively low and the overall budgetary impact for the NHS as a whole is in the order of 4 million GBP per annum. The true effectiveness of TMZ for recurrent glioma will only be determined by large RCTs comparing TMZ with best alternative care in a wider population of patients (i.e. not limited to those with favourable prognosis), with separate pre-planned analyses for those who are chemotherapy-nave.

Authors' methods: Systematic review

Project Status: Completed

URL for project: http://www.hta.ac.uk/1193

Year Published: 2001

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Health Technology Assessment programme

Contact Address: NIHR Journals Library, National Institute for Health and Care Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK

Contact Name: journals.library@nihr.ac.uk

Contact Email: journals.library@nihr.ac.uk

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