Improvements in the function, quality of life, and longevity of patients with Duchenne muscular dystrophy (DMD) have been achieved through a multidisciplinary approach to management across a range of health-care specialties. In part 3 of this update of the DMD care considerations, we focus on primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Many primary care and emergency medicine clinicians are inexperienced at managing the complications of DMD. We provide a guide to the acute and chronic medical conditions that these first-line providers are likely to encounter. With prolonged survival, individuals with DMD face a unique set of challenges related to psychosocial issues and transitions of care. We discuss assessments and interventions that are designed to improve mental health and independence, functionality, and quality of life in critical domains of living, including health care, education, employment, interpersonal relationships, and intimacy.

ABSTRACT. Objective. To compare the accuracy of biomarkers for identifying acute chest syndrome (ACS) in patients with sickle cell disease presenting to a pediatric emergency department (ED).Methods. We conducted a 13-month-long (2002-2003) cohort study with nested case-control in patients with sickle cell disease presenting to the pediatric ED with vaso-occlusive crises or fever in which we compared levels of secretory phospholipase A2 (sPLA2), endothelin-1, interleukin-6 (IL-6), and peripheral white blood cell count (WBC) in cases that were complicated by ACS and in control subjects with uncomplicated illnesses. For diagnosis, a test was considered to be accurate when the area under its receiver operator characteristic curve (AUC) was >0.70. Laboratory tests with AUC values >0.70 were entered into a binary recursive partitioning model for diagnosis.Results. For the period of study, samples from 72 visits were obtained from 51 patients who presented with vaso-occlusive crises (range: 1-4 visits per patient; 15 were enrolled more than once A cute chest syndrome (ACS), a combination of respiratory symptoms and radiographic evidence of new pulmonary infiltrates, is the second leading cause of hospitalization in patients with sickle cell disease (SCD). 1 The success of simple blood transfusions in the treatment of overt ACS is well established and raises the possibility of using such therapy earlier in the course of illness. 2-4 Symptoms of impending ACS, however, may be subtle, limiting the utility of the clinical examination. In fact, a recent study reported that half of ACS cases were diagnosed after patients had been admitted to the hospital with other complaints (most commonly, vaso-occlusive crises [VOC]). 2 A strategy of routine chest radiographs in patients who present with VOC may identify early cases of ACS but is undesirable because of the hazards associated with repeated exposure to radiation. Fortunately, other possibilities exist. A promising test is the level of secretory phospholipase A2 (sPLA2). In one small study, elevation of this enzyme predicted impending ACS in children with VOC. 5 No attempts have been made, however, to validate these findings at other sites. Another test is the endothelin-1 level in plasma, which has been shown to rise in VOC 6 but has not been evaluated for predicting ACS. Finally, peripheral blood leukocytes and interleukin (IL) levels increase in many inflammatory conditions and so may have value for detecting ACS. Among ILs, an attractive target for study is IL-6, a pleiotropic proinflammatory protein with many biologic activities that is reported to correlate positively with the number and adhesiveness of neutrophils in SCD. 7,8 For now, it is not clear how these biomarkers change in response to ACS. In this study, we addressed this question. Specifically, we validated the accuracy of sPLA2 in a new setting and compared its performance with that of other tests. For ACS detection, we also defined optimal test cutoffs and evaluated a strategy that combines these tests for...

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Background:Prescription fill rates for children being discharged from the emergency department (ED) after asthma exacerbations are low, placing the child at risk for additional ED visits or admissions for asthma. This article describes the implementation of an ED asthma prescription delivery service designed to improve pharmacy prescription capture and decrease ED revisit rates.Methods:A core group developed a service to provide asthma prescriptions and education to patients in their ED room before discharge. The project assessed the percent of ED asthma patients who filled ED asthma prescriptions at the hospital outpatient pharmacy, 7-, 14-, and 30-day ED revisit rates, and patient satisfaction.Intervention:Patients/families who chose to participate in the service received asthma prescriptions and education at the ED bedside. Within 1–3 days, ED outreach nurses obtained patient satisfaction survey responses via telephone.Results:There was a statistically significant increase in the number of patients who filled ED asthma prescriptions at the hospital outpatient pharmacy (22.2% versus 33.8%; P < 0.0001). The decrease in 7-, 14-, or 30-day ED revisit rates for patients who received the medication delivery service compared with standard of care was not statistically significant. Patients were satisfied to very satisfied with the service.Conclusion:Postimplementation of a medication delivery program within the ED, there was an increase in the percentage of patients who filled ED asthma medication prescriptions at the hospital outpatient pharmacy. There was no difference in ED revisit rates for patients who enrolled in the prescription delivery service versus standard of care.