The comparison of tandem autologous SCT with intensified chemotherapy was the only one where patients with non-metastatic breast cancer not only survived longer without recurrence of breast cancer ("event-free survival"), but also lived longer ("overall survival"). However, these indications are only found in one trial and only apply to a specific therapy regimen (WSG AM-01Trial), so that they cannot be generalized.

In general, IQWiG is concerned that some of the available trials are quite old. Nowadays, other chemotherapy regimens are normally used, particularly for patients with advanced non-metastatic breast cancer.

Further testing must only take place within clinical trials

High-dose chemotherapy in combination with autologous SCT appeared in the 1980s as a promising treatment and was introduced into medical care without adequate clinical trialling (in IQWiG's view an ethically questionable act). When the results of the first reproducible RCTs became available at the end of the 1990s, disillusionment set in and the number of transplants in breast cancer fell dramatically: in 2002 only 316 patients in Europe received transplants whereas in 1997 this figure was 2626.

For patients with metastatic breast cancer in particular, for whom there is still no curative treatment, alternatives must be tested. This also applies to therapies combined with autologous SCT. In view of the risks associated with autologous SCT, the authors of the final report believe that this testing should only take place within controlled trials. In order to better assess the benefits and harms, it would be particularly useful to investigate in further clinical trials whether treatment, optionally with autologous SCT, can extend the life of patients with metastatic breast cancer. This would be a trial looking at the primary outcome of overall survival.

Commenting procedure

IQWiG published the preliminary results in the form of the preliminary report in the middle of June 2008 and interested parties were invited to submit comments. When the comments stage ended, the preliminary report was revised and sent as a final report to the contracting agency, the Federal Joint Committee, at the end of April 2009. As the only written comment submitted did not raise any questions that had to be discussed, no oral debate took place. Appraisal of this written comment was incorporated into the discussion part of the final report. The written comment itself was recorded separately and published simultaneously with the final report. The report was produced in collaboration with external experts.

Source: Institute for Quality and Efficiency in Health Care