The treatment for primary immune thrombocytopenia with romiplostim in adult and paediatric patients: use experience at a Spanish university hospital.

WHAT IS KNOWN AND OBJECTIVE: Primary immune thrombocytopenia (ITP) is characterized by accelerated platelet destruction, as well as suboptimal platelet production. Thrombopoietin (TPO) receptor agonists bind to and activate human TPO receptor, and have been shown to increase platelet counts. In this study, we assessed the effectiveness and safety of long-term administration of TPO agonist romiplostim in adult and paediatric patients. METHODS: This is a retrospective observational study that included every ITP patient (adults and children) who received romiplostim since its inclusion in our institutional formulary. Data on patients' demographics, romiplostim doses, platelet counts, use of rescue medication and concurrent therapies were collected. Outcomes for effectiveness evaluation were proportion of patients who achieved a platelet response (platelet count >50 × 10(9) per litre and double the platelet count at baseline on any scheduled visit, excluding counts obtained within 8 weeks after receipt of rescue medications), proportion of patients who achieved a durable response (platelet responses during 6 or more weeks of the last 8 weeks of treatment), proportion of patients needing rescue medication, proportion of patients able to stop or reduce concurrent treatment and mean number of weekly platelet responses. Safety was assessed on the basis of the incidence of adverse events documented on the patients' medical records. RESULTS AND DISCUSSION: This study enrolled ten adults and four paediatric patients. None of the paediatric patients and one adult patient had been splenectomized (contraindicated in the other adults). In the adult population, eight achieved a response at least once during treatment, and 1 achieved a durable response. Four patients needed rescue medication (mostly intravenous immunoglobulins). Three patients were able to stop concurrent ITP therapies, and the mean number of weekly platelet responses was 6. All four paediatric patients achieved a response at least once during treatment, and three achieved durable responses. Three patients needed rescue medication. The only patient who was receiving concurrent ITP medication was able to stop it, and the mean number of weekly platelet responses was 25. No serious adverse events were registered during treatment in either population. WHAT IS NEW AND CONCLUSION: The effectiveness of romiplostim was variable with few adult patients achieving a durable response. Our paediatric patients responded better with most achieving a durable response. The treatment was safe for both groups of patients. Studies should be conducted to identify patients more likely to benefit from this treatment.

Cognitive functioning in children with multiple sclerosis.

OBJECTIVE: To examine the cognitive functioning of children with multiple sclerosis (MS). METHODS: Six children with a diagnosis of clinically definite MS were evaluated using a neuropsychological test battery. RESULTS: The majority of the children showed deficits in at least two of the administered subtests, with IQ scores within the deficient classification. CONCLUSIONS: Verbal and non-verbal skills were equally impaired, and patients who were older at the moment of the onset of the disease had a better cognitive performance. Cognitive deficits should be regarded as a common occurrence in the course of MS in children.

Facturación interna de los productos finales elaborados por el Servicio de Farmacia Hospitalaria / Internal invoicing of end products manufactured by a hospital Pharmacy Department

Objetivo: Calcular los costes reales de los productos finales elaborados por el Servicio de Farmacia. Material y métodos: Se valora el nivel de calidad del Servicio de Farmacia, su producción global en Unidades Relativas de Valor (URVs) y su grado de complejidad. Se calcula el coste real de la URV y de los productos finales elaborados. Resultados: El Servicio de Farmacia se clasifica en el nivel II de calidad. El número total de URVs producidas es de 2.334.355,86, con un grado de complejidad de 2,20, siendo el coste de la URV de 0,87 euros. La imputación del coste del Servicio de Farmacia por URV presenta grandes discrepancias frente al sistema tradicional de reparto, por consumo de medicamentos. Conclusiones: La incorporación de un Catálogo de Productos en los Servicios de Farmacia permite conocer la actividad realizada de forma global y por servicio, su complejidad media, los puntos fuertes y de mejora para incluirlos en la planificación del Servicio de Farmacia. Además, permite una facturación más real de los productos a los servicios peticionarios (AU)

[Internal invoicing of end products manufactured by a hospital Pharmacy Department] / Facturación interna de los productos finales elaborados por el Servicio de Farmacia Hospitalaria.

OBJECTIVE: To calculate actual costs of end products manufactured by a Pharmacy Department. MATERIAL AND METHODS: Quality standards, overall production in relative value units (RVUs), and complexity degree of a pharmacy department are assessed. Actual cost of RVUs and manufactured end products is calculated. RESULTS: The Pharmacy Department is classified as quality level II. The total number of produced RVUs is 2,334,355.86, with a complexity degree of 2.20 and a cost per RVU of 0.87 euros. Pharmacy Department cost imputation per RVU has relevant discrepancies when compared to traditional shared cost systems regarding drug consumption. CONCLUSIONS: The inclusion of a product catalogue within Pharmacy Departments allows overall and department-specific activities to be acknowledged, as well as mean complexity, strengths and issues in need of improvement, in order to include them within pharmacy department planning. Furthermore, it allows for more realistic product charging to requesting departments.

Clinical evaluation of norbolethone, a new anabolic steroid

Norbolethone may be considered an excellent metabolic activator due to the following reasons: 1. Substantial increase in lean body weight in a big majority of the subjects. 2. Stimulation of appetite in those suffering from chronic or refractory anorexia not affected by vitamins and other anabolics previously taken3. Improvement of the feeling of well-being in those patients even suffering from malignancies and intractable pain4. Increase of alertness and strength which would have taken a long time in convalescent subjects, post-infectious and post-surgical5. Persistence of excellent results even after administration of the drug had been terminated6. Reversal of negative nitrogen balance7. Insignificant effect on blood and urine values. SGOT values increased but were restored to normal8. Increase in serum protein and nitrogen balance positive of protein build up9. Side reactions that occur later with the drug are not severe enough to require suspension of therapy and disappear gradually even only on reduction of the dose. The 11 dropouts earlier in the study were not caused by intolerance to the drug but due to the fact that these subjects thought they had received enough of the drug when their conditions improved. (Conclusion)

Enhancement of incisional wound healing and neovascularization in normal rats by thrombin and synthetic thrombin receptor-activating peptides.

To better define thrombin-receptor interactions, we synthesized human thrombin peptides and identified binding-domain peptides that bind thrombin receptors and activate mitogenic signals (Glenn, K.C., G.H. Frost, J.S. Bergmann, and D.H. Carney. 1988. Pept. Res. 1:65-73). Treatment of full dermal dorsal incisions with a single topical application of thrombin receptor-activating peptide (TRAP-508) or human alpha-thrombin in saline enhances 7-d incisional breaking strength in normal rats up to 82% or 55% over saline-treated controls, respectively. Control wounds require approximately 11.5 d to achieve breaking strength equivalent to TRAP-treated wounds at day 7. Thus, a single application of TRAP accelerates healing, shifting the time course forward by up to 4.5 d. Histological comparisons at day 7 show more type I collagen, less evidence of prolonged inflammation, and an increase in number and maturity of capillaries in TRAP- and thrombin-treated incisions. Angiograms also show 50-65% more functional vascularization going across thrombin- and TRAP-treated surgical incisions. Thus, alpha-thrombin and thrombin peptides, such as those released following injury, appear to initiate or enhance signals required for neovascularization and wound healing. The ability to accelerate normal wound healing events with synthetic peptides representing receptor binding domains of human thrombin may offer new options for management of wound healing in man.

A synthetic peptide representing the thrombin receptor-binding domain enhances wound closure in vivo.

Our studies of alpha-thrombin as a growth factor have led to the development of a synthetic peptide (p508) that in vitro competes with thrombin for binding to high affinity receptors, and enhances mitogenic activity. To determine if this peptide could be used to accelerate wound closure in vivo, full thickness 6 mm dermal biopsy wounds on the dorsal skin of anesthetized rats were treated with p508 peptide, thrombin or PBS as control. At day 7, the p508 treated wound areas were 20% to 50% smaller than either thrombin or PBS treated wound sites. This suggests that p508 enhances aspects of wound healing, and avoids the normal in vivo regulatory mechanisms of intact thrombin.