Rilevazione retrospettiva multicentrica degli usi on-label e off-label dei farmaci chemioterapici utilizzati nei regimi di condizionamento nel trapianto di midollo osseo

Background. In Italy the use of anticancer drugs outside approved indications (off-label) is frequent. The objective of this retrospective study was to evaluate off-label and on-label uses in the conditioning regimens for patients receiving autologous or allogeneic stem-cell transplantation (SCT). Patients and methods. Six Italian hospitals participated in the study. We retrospectively examined all patients who received SCT in these centres over the period from June to September 2006. Results. A total of 133 patients were studied; 75.9% of the schedules used were off-label (N=101) and 24.1/% were on-label (N=32). The three most frequent therapeutic regimens were: melphalan (labelled for conditioniong regimens in patients with multiple mieloma), BEAM (off-label) and BUCY (off-label). Conclusion. Our results show that the majority of current conditioning regimens are off-label. However, this use is well-established and homogeneous across the 6 centres. Our study was completed in February 2007 and emphasized the need for a better regulation on this topic. This then happened with the AIFA Determination of 29 May 2007 that presented a revised list of on-label and off-label uses of anti-cancer drugs reimbursed by National Health System and implicitly approved the off-label uses contained in the list.