ALBERT

Description: Objective: To evaluate the effect of the implementation of a smart-phone based therapy management application over the monitoring of the hemophilia A (HA) and B (HB) patients in home-care setting. Setting: Since 2016 our hospital is using a mobile app and real time therapy management system for hemophilia patients (Haemoassist™, StatConsult) as a new tool of hemophilia patients care management complementing the existing health charts. Methods: A retrospective research conducted at one hemophilia treatment center covering four islands. HA and HB patients using the mobile app with at least 6-month follow-up period were included. Data on socio-demographics, clinical and therapy were collected before and after 6-month period of the app introduction. Sources of information were the electronic medical history (Millenium-Cerner™), pharmacy outpatient delivery program (Hospiwin™) and the above mentioned smart-phone app. The adherence was estimated calculating real factor consumption divided by the theoretical factor consumption according to prescribed prophylactic regimen for each study period. The extra doses for bleeding episodes were collected only after the intervention, due to unavailable reliable reports for mild and moderate bleeding events before the incorporation of the app. Results: A total of 28 patients with severe and moderate HA and HB on prophylactic treatment were included. The median age was 25 years (range 3-57). The median of the first period adherence was 72.7 % (range 30.77-100%) and of the second was 100% (range 51.88-100%). The median of the difference between periods was 15% (see chart 1 for more detailed information). In the post-intervention period, 12 (43 %) patients had a median of 3 bleeding events (range 1 -7) and needed extra-infusions with a median of 7 doses (range 3-17). Conclusions: The adherence to prophylactic treatment is crucial to reduce joint bleedings (Manco-Johnson MJ et al. NEJM. 2007). The use of therapy management mobile app has contributed to increase adherence in our patients allowing a precise monitoring of the administered factor and bleeding events in real time. Figure. Figure. Disclosures No relevant conflicts of interest to declare.

Description: INTRODUCTION Prophylaxis with factor VIII (FVIII) is considered the gold standard for managing hemophilia A (HA) patients without inhibitors to prevent bleedings and to preserve normal musculoskeletal function. Classically, the prophylaxis with a standard half-life FVIII is 20 to 40 IU/kg administered every other day. In practice this "one-size-fits-all" way of dosing does not work in many patients due to the interindividual variability on the concentration-time profile of FVIII, body weight, age, blood group and level of Von Willebrand factor. Pharmacokinetic (PK)-guided regimens have been proposed as an effective way to optimize prophylaxis efficacy. Different online PK tools have been developed to estimate patients' PK with only 2 blood samples. The integration of PK and clinical data can help physicians to adjust dosing. Since 2014 we have been using an online tool to perform PK estimation in patients using the FVIII product Advate® (myPKFiT®, www.mypkfit.com; Baxter Healthcare Corporation; Haemophilia 2014, 20 (Suppl.2):15). In June 2018 a new version has been launch, the version 3.0. This v3.0 includes a dose calculation update based on the ability to modify an individual dose or trough level of an individual prophylaxis regimen providing an accurate adjustment of prophylaxis dosing in comparison with version 2.0 (v2.0). This report describes the impact of the PK estimation in the infusion frequency and factor consumption, comparing both versions, in a cohort of people with HA. METHODS This is an observational case series from a single hemophilia treatment Spanish center. The inclusion criteria were: patients receiving prophylaxis with octocog alfa who accepted to participate in the PK study. Two blood samples have been collected for the estimation of FVIII PK (3-4 hours and 24-28 hours after administration of their usual dose of FVIII) in real world practice. We measured FVIII using one-stage assay. The FVIII trough levels were adjusted between 1% and 3% according to patients characteristics. Demographic data, PK parameters, infusion frequency, dosage and consumption after PK adjustment of prophylaxis using the v2.0 and v3.0 tool were analyzed. RESULTS Eleven patients were evaluated (7 severe, 3 moderate, 1 mild HA) aged 2-43 years (median 14 years). There were no differences in PK parameters between v2.0 and v3.0 as the applied algorithm for both versions is the same. PK results are shown in the Panel A of the table below. The elected trough level was between 1-3% depending of the physical activity and joint health of every individual patient. Prophylaxis regimen was modified in 6/11 patients after PK analysis with both versions of the App: 2 pediatric patients increased their FVIII dose and 3 adults increased their treatment frequency. We have only observed differences in 3 patients comparing the two versions: Patient 1 started a new infusion regimen (every 48h to 48-48-72h) with a mild increase in consumption, and patient 2 and 5 maintained the regimen every 48h with slight reductions in consumption. Results are shown in the Panel B of the table below. CONCLUSIONS Both versions of myPKFit™ are a helpful tool to adjust FVIII dosing regimens. This latest version allows to individualize even more the treatment regimen and to achieve a more efficient consumption. Prospective evaluation of the use of PK‐tailored prophylaxis in routine care and its impact on patient outcomes is needed. Table. Table. Disclosures No relevant conflicts of interest to declare.

Description: More thermolabile drugs are becoming available, and in most cases, these medications are dispensed to ambulatory patients. However, there is no regulation once medications are dispensed to patients and little is known with regard to what happens during transport and home storage. Previous studies suggest that these drugs are improperly stored. The present study was designed to determine the storage conditions of thermolabile drugs once they are dispensed to the patient in the Hospital Pharmacy Department. This is a prospective observational study to assess the temperature profile of 7 thermolabile drugs once they are dispensed to ambulatory patients at a tertiary care hospital. A data logger was added to the medication packaging. Temperature was considered inappropriate if one of the following circumstances were met: any temperature record less than or equal to 0 °C or over 25 °C; temperatures between 0–2 or 8–25 °C for a continuous period over 30 min. The time series of temperature measurements obtained from each data logger were analyzed as statistically independent variables. The data shown did not undergo any statistical treatment and must be considered directly related to thermal measurements. One hundred and fourteen patients were included and 107 patients were available for the analysis. On the whole, a mean of 50.6 days (SD 18.3) were measured and the mean temperature was 6.88 °C (SD 2.93). Three data loggers (2.8%) maintained all the measurements between 2 and 8 °C with less than 3 continuous data (