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Previously published guidelines for the diagnosis and management of primary immune thrombocytopenia (ITP) require updating largely due to the introduction of new classes of therapeutic agents, and a greater understanding of the disease pathophysiology. However, treatment-related decisions still remain principally dependent on clinical expertise or patient preference rather than high-quality clinical trial evidence. This consensus document aims to report on new data and provide consensus-based recommendations relating to diagnosis and treatment of ITP in adults, in children, and during pregnancy. The inclusion of summary tables within this document, supported by information tables in the online appendices, is intended to aid in clinical decision making.
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Complicaciones Hematológicas del Embarazo/diagnóstico , Complicaciones Hematológicas del Embarazo/tratamiento farmacológico , Púrpura Trombocitopénica Idiopática/diagnóstico , Púrpura Trombocitopénica Idiopática/tratamiento farmacológico , Adulto , Niño , Preescolar , Consenso , Femenino , Humanos , Masculino , Embarazo , Complicaciones Hematológicas del Embarazo/fisiopatología , Púrpura Trombocitopénica Idiopática/fisiopatologíaRESUMEN
OBJECTIVES: To study whether 60-Hz stimulation, compared with routine 130 Hz, improves swallowing function and freezing of gait (FOG) in patients with Parkinson disease (PD) who undergo bilateral subthalamic nucleus (STN) deep brain stimulation (DBS). METHODS: We studied 7 patients with PD who experienced FOG that persisted despite routine 130-Hz stimulation and dopaminergic medication. Each patient received 3 modified barium swallow (MBS) studies in a single day under 3 DBS conditions in the medication-on state: 130 Hz, 60 Hz, or DBS off, in a randomized double-blind manner. The laryngeal penetration and aspiration events were cautiously assessed, and a swallowing questionnaire was completed. The Unified Parkinson's Disease Rating Scale, Part III motor score, axial subscore, tremor subscore, and FOG by a questionnaire and stand-walk-sit test were also assessed. The best DBS condition (60 Hz here) producing the least FOG was maintained for 3 to 8 weeks, and patients were assessed again. Changes in measurements between the 60 Hz and 130 Hz were analyzed using paired t test, with swallowing function as primary and the remainder as secondary outcomes. Changes between other DBS conditions were further explored with Bonferroni correction. RESULTS: Compared with the routine 130 Hz, 60-Hz stimulation significantly reduced aspiration frequency by 57% on MBS study and perceived swallowing difficulty by 80% on questionnaire. It also significantly reduced FOG, and axial and parkinsonian symptoms. The benefits at 60-Hz stimulation persisted over the average 6-week assessment. CONCLUSIONS: Compared with the routine 130 Hz, the 60-Hz stimulation significantly improved swallowing function, FOG, and axial and parkinsonian symptoms in patients with PD treated with bilateral STN-DBS, which persisted over the 6-week study period. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with PD who experience FOG, STN-DBS at 60 Hz decreases aspiration events observed during MBS compared with DBS at 130 Hz.
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Estimulación Encefálica Profunda/métodos , Deglución/fisiología , Marcha/fisiología , Enfermedad de Parkinson/terapia , Núcleo Subtalámico/fisiopatología , Anciano , Sulfato de Bario , Método Doble Ciego , Femenino , Humanos , Masculino , Persona de Mediana Edad , Enfermedad de Parkinson/fisiopatología , Índice de Severidad de la Enfermedad , Resultado del TratamientoRESUMEN
OBJECTIVE: To assess the impact of immune thrombocytopenic purpura (ITP) on primary care and specialist visits and workplace productivity. RESEARCH DESIGN AND METHODS: This was a cross-sectional, descriptive study comparing ITP patients to age- and gender-matched controls. Subjects completed a one-time web-based survey, which included questions on work loss, work productivity, and physician visits. ITP patients and controls were compared on these outcomes. For ITP patients, the relationship between work-related issues and physician visits with clinical characteristics (time since diagnosis, platelet count, number of treatments received, and an ITP-specific health-related quality of life measure, the ITP-PAQ) was explored. RESULTS: A total of 1002 ITP patients and 1031 controls completed the survey. On average, ITP patients were 46 years old, diagnosed 9 years ago, and had platelet counts of 148 x 10(9)/L; 37% had undergone splenectomy. More ITP patients had primary care (20% vs. 11%) and specialist (28% vs. 11%) visits in the past month versus controls (p < or = 0.001). Higher proportions of ITP patients have ever taken sick leave (56% vs. 30%), and missed chore hours in the past week (18% vs. 13%) (p < or = 0.003). ITP patients scored significantly worse than controls on all six work productivity items. Patients diagnosed within the past year were more likely to have specialist visits and to miss chore hours versus those diagnosed less recently. Worse ITP-PAQ scores and more ITP-related treatments were related to more physician visits and worse work-related and productivity outcomes. Platelet count is not associated with these outcomes. The study is limited by the potential for biased samples due to recruitment approaches, the inherent issues of a cross-sectional study design and recall bias in questionnaire responses. CONCLUSIONS: ITP was consistently associated with more physician visits and worse work and productivity outcomes. Future research should build on these findings by calculating a comprehensive cost-of-illness of ITP including both direct and indirect costs.
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Eficiencia , Visita a Consultorio Médico/estadística & datos numéricos , Púrpura Trombocitopénica Idiopática/fisiopatología , Púrpura Trombocitopénica Idiopática/terapia , Trabajo , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Estudios de Casos y Controles , Estudios Transversales , Femenino , Humanos , Masculino , Persona de Mediana Edad , Sistemas en Línea , Consultorios Médicos , Encuestas y Cuestionarios , Lugar de Trabajo , Adulto JovenRESUMEN
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OBJECTIVE: To assess the health-related quality of life (HRQOL) of immune thrombocytopenic purpura (ITP) patients. RESEARCH DESIGN AND METHODS: This was a cross-sectional, descriptive study comparing ITP patients' HRQOL to age and gender matched controls. ITP patients from the Platelet Disorder Support Association were recruited until 1000 surveys had been completed. Controls were randomly sampled from the Harris Interactive Online Panel. ITP patients and controls completed a one-time web-based survey, including a comprehensive HRQOL assessment. ITP patients completed the SF-36, the EQ-5D, and the ITP-Patient Assessment Questionnaire (ITP-PAQ). Controls completed the SF-36 and EQ-5D only. ITP patients' SF-36 and EQ-5D scores were compared to controls in unadjusted and adjusted analyses. Associations between splenectomy status, duration of illness, and platelet count with ITP patients' HRQOL scores were also examined. RESULTS: This analysis included 1002 ITP patients and 1031 controls. ITP patients scored worse on seven of eight SF-36 domains and the Physical and Mental Summary scores (all p < 0.05) and on the EQ-5D visual analog scale (65.5 vs. 82.3; p = 0.002). ITP patients who had undergone splenectomy had similar SF-36 and EQ-5D scores to non-splenectomy patients but scored significantly worse on 5 of 10 ITP-PAQ scales: Bother, Psychological, Fear, Social Activity, and Work (all p < 0.05). ITP patients diagnosed within the past 5 years had worse Bother and Overall Quality of Life scores than less recently diagnosed patients but were similar on other ITP-PAQ scales. Lower platelet count was consistently associated with worse ITP-PAQ scores and had weaker associations with SF-36 and EQ-5D scores. CONCLUSIONS: ITP was associated with consistent and statistically significant deficits on generic HRQOL measures. The ITP-PAQ demonstrated differences based on disease severity and treatments. The self-selection bias in the two samples limits the generalizability of the results to all patients with ITP. Further research is needed in more generalizable samples.