BackgroundThe introduction of PCSK9 inhibitors in the treatment of hypercholesterolaemia marks a breakthrough for patients unresponsive to traditional treatment. However, in our country, 44.9% of adults have high LDL-C levels (≥ 130 mg/dL or under treatment), so inadequate use of these innovative drugs might have a strong impact on efficiency and safety. The national atherosclerosis association published a document regarding the restricted indications for use of the PCSK9 inhibitors, and we adapted it to our centre.PurposeWe evaluated compliance with the protocol for prescription of PCSK9 inhibitors in our centre.Material and methodsIn March 2016, the pharmacy and therapeutics committee created their own protocol together with internal medicine, cardiology and endocrinology services. It turned out to be more restrictive than the national guidelines, with higher LDL-C levels required. Based on the available evidence and taking into account criteria of efficacy and safety, four indications were included: (1) HoFH: homozygous familial hypercholesterolaemia with LDL-C >120 mg/dL with the maximum tolerated dose of statin and ezetimibe; (2) HeFH: heterozygous familial hypercholesterolaemia with LDL-C >120 mg/dL with the maximum tolerated dose of statin and ezetimibe; (3) previous cardiovascular event (pCVE) with LDL-C >100 mg/dL with the maximum tolerated dose of statin and ezetimibe; and (4) any of the above with LDL-C >120 mg/dL in patients who are statin intolerant, or for whom a statin is contraindicated. We evaluated the compliance with our protocol for prescribing PCSK9 inhibitors from March to September 2016, based on computerised medical records.ResultsWe received 26 prescriptions, 20 from the internal medicine service, 3 from the endocrinology service and 3 from the cardiology service. All complied with the protocol, except for 1 that was approved by the committee for the treatment of hyperlipoproteinaemia. The following patients were treated: 18 HeFH, 6 pCVE and 1 pCVE who was statin intolerant.ConclusionIn our centre, there was high compliance with the protocol for the prescription of PCSK9 inhibitors. Due to the major economic impact of these new drugs, continuous follow-up would be required to ensure that every prescription meets the requirements of the protocol in the treatment of hypercholesterolaemia with PCSK9 inhibitors.No conflict of interest

BackgroundMacitentan is a new endothelin receptor antagonist (ERA) which was compared with placebo in the SERAPHIN trial. The results showed no superior efficacy or safety compared (indirectly) with other ERAs but better hepatic tolerance.PurposeTo assess adaptation of pulmonary arterial hypertension (PAH) treatment prescriptions under our hospital protocol and its economic impact, and to describe the clinical results of using a new ERA, macitentan.Material and methodsA use protocol for ERAs was established in September 2016 in our hospital, evaluating cost effectiveness as the main criterion for prescription. Thus bosentan was chosen as the first option for treatment as macitentan had not shown superiority with regard to either efficacy or safety. According to the use protocol, macitentan should be used in naïve patients with liver dysfunction or in those patients treated with bosentan who have developed treatment related hepatic toxicity. Several clinical parameters assessed PAH disease before and after treatment: functional class, baseline oxygen saturation (%SpO2) and NT-proBNP levels.ResultsAfter designing the use protocol for ERAs, 9 prescriptions for macitentan from the respiratory department were registered. 44% of patients (4/9) met the requirements of use of macitentan (increased transaminases before using bosentan). The treatment cost per month with a standard maintenance dose of bosentan 125 mg/12 hours was €414, while the treatment cost per month with a standard dose of macitentan 10 mg/24 hours was €2063. After 4 months of treatment with macitentan, patients with elevated transaminases had returned to normal levels but all patients maintained the same functional class and there were no clinically significant differences in%SpO2 or NT-proBNP (p>0.05).ConclusionIf compliance with the use protocol for ERAs had been 100%, 5 of the patients would have been treated with bosentan, leading to a cost saving of about €1649 for patient per month, a total amount of €74205 so far this year. Implementation of a consensual use protocol for ERAs could enhance the rational use of this drug, but further collaboration with physicians is needed to achieve better optimisation of available resources.No conflict of interest