Background The off-label use of drugs is common in a hospital setting. However, some of these treatments have low scientific evidence.
Purpose The study aim was to describe the off-label use of drugs in the hospital and to assess the effectiveness of these treatments.
Material and methods We revised the authorised off-label applications between January 2016 and July 2018. We excluded all the off-label oncology treatments.
Clinical history, date of application, medical service, drug, indication and symptomatic improvement of disease were collected.
We considered effectiveness when the patient experienced improvement in most symptoms related to the disease (total effectiveness) or improvement in some symptoms (partial effectiveness). When the drug was not given for any reason, or the treatment was not finished for toxicity, it was considered not assessable.
Results A total of 84 applications were analysed. The evolution of these was: 32 applications in 2016, 27 in 2017 and 25 in 2018.
The medical services were: neurology (20%), nephrology (17%), nigestive (15%), ophthalmology (14%), otorhinolaryngology (6%) and other services (28%).
The most demanded drugs were rituximab (27%, n=23), botulinum toxin A (20%, n=17) and human immunoglobulin (18%, n=15).
The indications for rituximab were: membranous nephropathy (n=5), systemic lupus erythematosus (n=4), Sjögren syndrome (n=2), cryoglobulinemic vasculitis (n=2) and others (n=10).
The indications for botulinum toxin A were: achalasia (n=13), spasmodic dysphonia (n=3) and Frey syndrome (n=1).
The indications for human immunoglobulins were: myasthenic crisis (n=7), autoimmune encephalitis (n=3) and other indications (n=5).
Of all applications (n=84), 15 were not assessable: 10 because the treatment was not administrated and five because of its toxicity.
From all patients with an assessable treatment (n=69), 70% (n=48) experienced symptomatic improvement of the disease: in 48% (n=23) the treatment was totally effective and in 52% (n=25) it was partially effective.
Conclusion There is a high variability in the off-label use of drugs. It is necessary to develop protocols to unify the criteria of use of the most common treatments.
Despite low-level published evidence, the off-label treatments were effective in most patients, so they suppose a benefit for patients with few therapeutic options.
References and/or acknowledgements http://www.elsevier.es/es-revista-revista-calidad-asistencial-256-pdf-S1134282X12000760
No conflict of interest.
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