RESUMO
Background: Real-world data assessing treatment outcomes in patients with hemophilia A in routine clinical practice are limited. Objective: To evaluate the effectiveness and safety of octocog alfa in patients with moderate/severe hemophilia A receiving treatment in clinical practice. Design: The international Antihemophilic Factor Hemophilia A Outcome Database study is an observational, noninterventional, prospective, multicenter study. Methods: This planned interim data read-out was conducted following 7 years of observation of patients receiving octocog alfa (cut-off, 30 June 2020). The primary endpoint was joint health status, assessed by the Gilbert Score. Secondary endpoints included annualized bleeding rates (ABRs), Hemophilia Joint Health Score (HJHS), health-related quality of life, consumption, and safety. This post hoc analysis stratified data by hemophilia severity at baseline [moderate, factor VIII (FVIII) 1-5%; severe, FVIII <1%]. Results: Of the 711 patients in this analysis, 582 (82%) were receiving prophylaxis with octocog alfa at enrollment, and 498 (70%) had severe disease. Median Gilbert Scores were higher with on-demand therapy versus prophylaxis and scores were comparable in moderate and severe disease. In patients receiving prophylaxis, there was an improvement in HJHS Global Gait Score over 7 years of follow-up overall and in patients with severe disease. ABRs and annualized joint bleeding rates were low across all 7 years. An ABR of zero was reported in 34-56% of prophylaxis patients versus 20-40% in the on-demand group. ABRs were similar in severe and moderate disease. In total, 13/702 (1.9%) patients experienced 18 treatment-related adverse events. Conclusion: These data demonstrate the long-term effectiveness and safety of octocog alfa in patients with moderate and severe hemophilia A, especially in those receiving prophylaxis. The high number of patients receiving on-demand treatment experiencing zero bleeds could be due to selection bias within the study, with patients with less severe disease more likely to be receiving on-demand treatment. Trial registration: ClinicalTrials.gov: NCT02078427.
RESUMO
Se presenta un caso de encefalitis por Staphylococcus aureus meticilino sensible. Se trata de una paciente de sexo femenino, de 20 años de edad, quien fue atendida en la Fundación Centro Colombiano de Epilepsia y Enfermedades Neurológicas, de Cartagena, por presentar cefalea, progresivo deterioro del estado cognitivo y focalidad neurológica súbita con limitación a la emisión del lenguaje. Hallazgos en cultivo de LCR revelan crecimiento de Staphylococcus aureus meticilino sensible. La evolución de esta paciente fue satisfactoria. Se hace una revisión del caso, se realiza análisis de infrecuencia de encefalitis por Staphylococcusa ureus meticilino sensible sin foco de infección primario establecido.
A case of encephalitis Staphylococcus aureusmethicillin sensitive is presented. This is a female patient of 20 years old, who was treated at ColombianFoundation Center for Epilepsy and Neurological Diseases, in Cartagena. Her symptoms were headaches, progressive deterioration of cognitive status, and sudden neurological deficit with limitation on the issuance of language. CSF culture findings reveal growth of S. aureusmethicillin sensitive. The evolution of this patient was satisfactory. A review of the case is made, analysis infrequency of encephalitis is carried by Staphylococcus aureus methicillin sensitive in primary, focus of infection established.
Assuntos
Staphylococcus aureus , Encefalite , MeticilinaRESUMO
OBJECTIVE: To describe the relationship between sleep architecture and behavioral measures in unmedicated children and adolescents with Tourette syndrome (TS), attention-deficit hyperactivity disorder (ADHD), TS and comorbid ADHD (TS + ADHD), and healthy controls. The study also set out to examine differences in sleep architecture with each diagnosis. METHOD: A cross-sectional, 2-night consecutive polysomnographic sleep study was conducted in 90 children. All participants were matched for age, gender, and level of intelligence. RESULTS: Scores on the Child Behavior Checklist delinquency measure were modestly but significantly correlated with the number of movements during REM sleep (r = .36, p = .003). Significant correlations were also noted among the number of total arousals and arousals from slow wave sleep (SWS), and scores on the measures of conduct disorder, hyperactivity/immaturity, and restless/disorganized behaviors. There were a few significant differences in sleep architecture among the diagnostic groups. The ADHD-only group exhibited a significantly higher number of total arousals (p < .01) and arousals from SWS (p < .01) compared with the other three study groups. DISCUSSION: Our findings indicate that children with TS and/or ADHD and who have more arousals from sleep are significantly more likely to have issues with conduct disorder, hyperactivity/immaturity, and restless/disorganized behavior. It was also noted that having ADHD, alone or comorbid with TS, is associated with a significantly greater number of movements during both non-REM and REM sleep. This study underscores the compelling need for the diagnosis and treatment of any sleep disorders in children with TS and/or ADHD so as to facilitate better management of problem behaviors.
