Background It is known, although the estimates are very varied, that use of drugs in conditions other than those authorised or out of technical data, is particularly frequent in the onco-haematology area.
Purpose To describe the use of chemo-therapies on off-label practices in the Pharmacy Department of a tertiary hospital.
Material and methods This study included all patients treated between March 2015 and March 2017 with an off-label chemotherapeutic agent prescription. The data were collected from the clinical history of the patients and from the pharmacy programs: athos prisma®. We analysed these variables: demographic (age, sex) and treatment-related (drug involved, off-label indication, stage disease, number of previous treatment lines, treatment duration and adverse drug reactions (ADRs)).
Results A total of six types off-label drugs were requested and administered to 39 patients for eight different diseases.
Concerning haematologic indications, nine patients (23%) presented a complete response.
Patients had to discontinue treatment due to ADRs: bendamustine, doxorubicin liposomal, and fotemustine (one patient each). Treatment-related ADRs of any grade were reported in 15 (38.5%) patients. The most common were thrombocytopenia (18%) with fotemustine.
Conclusion In our assessment, off-label therapies have been effective in most patients (77%), and safe.
Evaluation of the cost of off-label therapies, in terms of medication risk and effects on the cost of healthcare, will be essential to its widespread clinical utility.
Off-label use may also become the only treatment option, especially in the case of rare tumours.
No conflict of interest
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