Background: Congenital nephrotic syndrome of the Finnish type (CNF) is a rare, severe glomerular disease caused by mutations in the NPHS1 gene, which codes for nephrin. It is characterised by massive proteinuria and severe edoema. Progression to end-stage kidney failure occurs during early childhood and the only curative treatment is kidney transplantation. Nowadays, patients need aggressive medical treatment, which includes daily albumin infusions (for months) until they get clinical stability to receive transplant.Objective: In our paediatric hospital, we implemented a multidisciplinary program for the home infusion of albumin with outpatient follow-up. The aim of the study was to assess the safety and efficacy of this program for the first four years of its implementation.Material and Methods: Retrospective observational study of CNF paediatric patients treated with home albumin infusion therapy from March 2014 to July 2018 at a tertiary care paediatric hospital. Information on albumin administration was obtained from the electronic prescription assistance program and details on clinical and care-related variables from the hospital's electronic information systems.Results: Four patients with CNF received albumin infusions for 18, 21, 22 months, and 3 years. The treatment was safe, and the complication rates were to be expected considering the severity of disease. Patients required a median of two hospital admissions a year (19 in total); 47% due to catheter-related complications, but there were just three catheter infections.Conclusions: In our experience, home albumin infusion therapy is safe and effective and helps to improve children health and quality of life.

BackgroundOrphan drugs (ODs) are designed to treat rare diseases (RD), which are those affecting a small number of people (prevalence <1/2000 inhabitants).PurposeTo assess the economic impact of drugs used to treat RD in a hospital outpatient paediatric pharmacy (HOPP) and a paediatric day hospital (PDH).Material and methodsObservational, retrospective, transversal study conducted at a third-level maternal and child University Hospital during 2016. All paediatric patients(<18 years’ old) were included, as well as adults with cystic fibrosis. Chemotherapy treatments administered in the Oncology Day Hospital were excluded.Pharmaceutical drug, active ingredient, number of packages, real unit cost, consumption data, medical service and treating unit were collected from pharmacy-dispensing software(Silicon®)The Orphanet® database was used to classify the drugs in ODs (recognised as orphan by the European Union or abroad) or drugs without orphan designation.ResultsFour hundred and ten drugs were identified to treat RD and 51 (12.4%) were ODs: 42 were dispensed in the HOPP and nine were administered in the PDH.There were 2442 patients who received at least one drug to treat RD: 2044 from the HOPP and 562 from the PDH (164 patients received treatment in both settings) Of all patients, 441 (18.1%) received at least one OD: 420 (20.5%) in the HOPP and 23 (4.1%) in the PDH (two patients in both).Drugs used to treat RDs accounted for €7.7 million: €3.8 million in the HOPP and €3.9 million in the PDH.OD cost represented 36.3% of the total pharmaceutical expenditure in drugs at the HOPP and 71% at the PDH.Bosentan, adalimumab, ivacaftor, ataluren and sildenafil were the five drugs with the greatest economic impact in the HOP budget and eculizumab, idursulfase, elosulfase, galsulfase and velaglucerase in the PDH budget.ConclusionPharmacological treatment with ODs has a great impact on direct medical costs, involving more than 50% of total pharmaceutical expenditure. Although it is more common in the outpatient pharmacy than in the day hospital (20.5% and 4.1% of the assisted patients, respectively), the OD cost reaches 71% of the expenditure on drugs in the PDH setting.The HOPP and the PDH need to develop strategies focusing on ODs, but also on treatments in special situations and extemporaneous drug formulations used to treat patients affected by RD.References and/or AcknowledgementsThanks to all authors for their involvementNo conflict of interest

BackgroundA pharmacist in the intensive care unit (ICU) as a component of multi-professional staff may improve the care provided to patients, particularly by monitoring the drugs administered, reducing preventable adverse drug events (ADE) and identifying drug interactions and errors.PurposeEvaluate the interventions of a critical care pharmacist (CCP) as a component of team-based care in a Spanish neurotrauma ICU (NTICU).Material and methodsProspective observational study with patients admitted in a NTICU for 5 weeks (including only working days). CCP collaborates with a multidisciplinary team selecting the medication therapy, dosage, duration and monitoring, based on physician diagnosis and team’s goals for the patient. CCP is also responsible for clinical services and electronic verification of medication orders.ResultsOut of 54, only 42 patients were monitored, with a mean age of 57 years (31–85), of which 31 were males (74%). Eleven patients were admitted for polytrauma (26%), eight for severe traumatic brain injury (19%), six for acute spinal cord injury (14%), three for cerebrovascular accident (7%), two for necrotising fasciitis (5%) and 12 (28%) for other causes. The median days of admission were 14. There were only five deaths during the study period.A total of 116 interventions were done, almost three interventions per patient and five per day of dedication of the CCP.The majority of interventions were related to artificial nutrition monitoring (28) and about the management of antimicrobial optimisation (27): nine discontinuations of antibiotic prophylaxis, six antibiotic dose adjustments, four recommendations to de-escalate the antibiotic and three antibiotic changes because they did not cover the pathogen. Twenty-two interventions were related to drug administration, 11 with conciliation, eight with intravenous-to-enteral conversion, five of thromboembolism prophylaxis, four drug-related questions, three discontinuations by duplications, two stopped because of ADE and one interaction.According to an internal hospital protocol, 26% of interventions were considered of high clinical impact.ConclusionAs most of the interventions were related to artificial nutrition adjustments, antimicrobial optimisation management and drug administration, a checklist was designed, containing such points where the pharmacist is mostly involved, to monitor critical patients in a standardised way and to simplify the detection of discrepancies.References and/or AcknowledgementsThanks to all authors for their involvement.No conflict of interest

BackgroundSerious burns produce plasma extravasation which develops an important loss of immunoglobulins (Ig). In patients with a burned surface area (BSA) >15% IgG plasmatic levels decrease until 40 hours’ post-burn.PurposeTo evaluate the use of non-specific Ig in burned paediatric patients based on the current protocol of the hospital.Material and methodsRetrospective observational study, which includes all paediatric patients with ≥15% BSA hospitalised between August 2012 and July 2017.Biodemographic data were registered as: (sex, age, weight), burn data (BSA) and Ig administration data (plasmatic levels, dose and number of administrations).The existing protocol about the use of Ig in burned paediatric patients (BSA ≥15%) was analysed. It recommends the determination of IgG 24 to 48 hours’ post-burn and the infusion of non-specific Ig (400 mg/kg) if patients have below-normal levels.ResultsThirty-one patients (12 females) with a median age of 2 years (8 m – 15 y) and a weight of 13 kg (7.5–67 kg) were enrolled in the study. The median BSA was 20% (15%–55%).Eighteen patients (58%) accomplished all the recommendations specified in the protocol.Determination of IgG levels was made in 26 patients (83.9%). Eighteen (69.2%) had below-normal levels and a median BSA of 23.5% (15–55). In the subgroup of patients with BSA ≥20% (20–55) the determination was done in the 94% (15/16) and 14 (93%) who had below-normal levels.Non-specific Ig was administered in 61% (19) of the patients at a dose of 400 mg/kg. No IgG determination was repeated after the first infusion in six patients (31.6%). Seven patients with a median BSA of 46% (16–55) needed more than one Ig infusion.ConclusionAll patients received the dose of Ig indicated in the protocol (400 mg/kg).In patients with BSA>20%, determination of plasmatic levels is essential because it was detected that more than 90% of the patients had below–normal levels of IgG.No IgG determination after the first infusion was repeated in more than 30% of the patients, therefore a proposal to improve the protocol is the need to repeat IgG determination in all the patients who have received an infusion to corroborate the achievement of normal IgG levels.References and/or AcknowledgementsThanks to all authorsNo conflict of interest

BackgroundIntravenous immunoglobulin (IVIG) is being increasingly used to treat neuroimmunological diseases. Randomised clinical trials (RCT) have proved its efficacy in certain indications, but evidence is scarce in others.PurposeEvaluate the prevalence, level of evidence and degree of recommendation of IVIG in different neuroimmunological indications.Material and methodsAmbispective observational study involving three tertiary hospitals including patients diagnosed with neuroimmunological diseases chronically receiving IVIG.Sex, age and main diagnosis were recorded for each patient. Demographic and clinical data were collected from electronic medical record and pharmacy dispensing software.The adequacy analysis (degree of evidence and recommendation) was contrasted against the British National Health System Clinical Guide for the use of IVIG. For indications with insufficient evidence, further research was performed.ResultsOne hundred and seventeen patients were included (51 females) with a median age of 53 (18–85).Most were diagnosed with chronic inflammatory demyelinating polyradiculoneuropathy, CIDP (45); followed by myasthenia gravis, MG (25); multifocal motor neuropathy, MMN (12); lower motorneuron, LMN (4); Guillain–Barre syndrome, SGB (5), stiff person syndrome (3), CIDP-like neuropathy (3), Eaton–Lambert syndrome, LEMS (3); Bickerstaff encephalitis, EB (3); inclusion body myositis, IBM (2); amyotrophic lateral sclerosis, ALS (2); anti-GAD +paraneoplastic syndrome (2); polymyositis (2); autoimmune epilepsy (1); transverse myelitis (1); Kabuki syndrome (1); Kinsbourne syndrome (1); Sjögren syndrome (1); and idiopathic lumbosacral plexopathy, PLSI (1).There is a high level of evidence (A, Ia) and degree of recommendation for the use of IVIG in CIDP, SGB, CIDP-like-neuropathy and MMN (55.5%). In stiff person syndrome and LEMS, however, the degree of recommendation is lower due to the absence of meta-analysis (A, Ib) (5.12%).For LMN, EB, anti-GAD +paraneoplastic syndrome, myelitis, polymyositis, epilepsy, Kabuki syndrome, Kinsbourne Syndrome and Sjögren syndrome there are not high-quality RCT, so the degree of recommendation and evidence are low (C, III) (13.6%).There are not recommendations for using IVIG in PLSI and ALS (D, IV) (2.5%).ConclusionIn neuroimmunological diseases, IVIG are used for indications with a high level of evidence (I-II) and degree of recommendation (A-B). However, 16% of indications with low evidence (III-IV) and recommendation (C-D) were recorded. Pharmacy services must guarantee the correct use of IGIV.References and/or AcknowledgementsThanks to all authors for their contributionsNo conflict of interest