Obesity and loss of ambulation are associated with lower extremity oedema in Duchenne muscular dystrophy.

Patients with Duchenne muscular dystrophy have multiple risk factors for lower extremity oedema. This study sought to define the frequency and predictors of oedema. Patients aged 15 years and older were screened by patient questionnaire, and the presence of oedema was confirmed by subsequent physical exam. Twenty-four of 52 patients (46%) had oedema, 12 of whom had swelling extending above the foot and two with sores/skin breakdown. There was no significant difference in age, frequency, or duration of glucocorticoid use, non-invasive respiratory support use, forced vital capacity, cardiac medication use, or ejection fraction between patients with and without oedema (all p > 0.2). Those with oedema had a greater time since the loss of ambulation (8.4 years versus 3.5 years; p = 0.004), higher body mass index (28.3 versus 24.8; p = 0.014), and lower frequency of deflazacort use (67% versus 89%; p = 0.008). Multivariate analysis revealed a longer duration of loss of ambulation (p = 0.02) and higher body mass index (p = 0.009) as predictors of oedema. Lower extremity oedema is common in Duchenne muscular dystrophy but independent of cardiac function. Interventions focused on minimising body mass index increases over time may be a therapeutic target.

Emergency Planning as Part of Healthcare Transition Preparation for Patients with Duchenne Muscular Dystrophy.

BACKGROUND: Emergency care planning is an important component of healthcare transition, particularly for patients with medical complexity. Duchenne muscular dystrophy (DMD) is a complex, progressive pediatric-onset disease affecting multiple organ systems including impairment of cardiac and pulmonary function, high risk for fractures, fat embolism, adrenal crisis and malignant hyperthermia. Appropriate interdisciplinary emergency management is critical for survival for these patients. The purpose of this quality improvement project was to develop a process to reliably share an individualized emergency care plan (ECP) with patients and their families as part of a larger plan to develop an integrated transition program. METHODS: An interdisciplinary team of nurses and clinicians used the principles of quality improvement to develop a reliable process to assure patients with DMD received an individualized, multidisciplinary ECP at routine interdisciplinary clinic visits. Additionally, the project used surveys to assess patient and family satisfaction with the letter and whether it improved their knowledge of emergency care. RESULTS: Sixty-two patients were seen during the study timeframe. All received an ECP. Sixty-two surveys were sent and twenty-three surveys were returned. Of those that responded, the majority stated the ECP increased their knowledge of emergency care. CONCLUSION: ECPs can be developed and disseminated to patients with DMD and their caregivers. This tool can potentially promote timely and appropriate emergency care for these patients with unique and complex medical needs.

Implementation of a Portable Medical Summary for Adolescents and Young Adults With Medical Complexity in Transition to Adult Health Care.

BACKGROUND: Adolescents and young adults (AYA) with special health care needs are living into adulthood, as improvements in care increase the likelihood of survival. Fewer than half have the resources needed to transition to adult care (McPheeters et al., 2014). A portable medical summary is a concise document summarizing current medical information about a patient that can be used across healthcare systems as AYA transition to adult care. Though a consensus statement recommending the use of such a summary has existed for over a decade (American Academy of Pediatrics (AAP), American Academy of Family Physicians (AAFP), American College of Physicians-American Society of Internal Medicine (ACP-ASIM), 2002), little progress had been made in the development of a portable medical summary. METHODS: The Institute for Healthcare Improvement (IHI) Model was used to implement a process to provide a portable medical summary to AYAs with medically complex conditions (Institute for Healthcare Improvement, 2017). INTERVENTIONS: The tool was developed using the electronic health record and shared with families. Feedback from care providers and families led to modifications to improve its usefulness and feasibility. Implementation of the process was tested for four months. RESULTS: The number of AYA, ages 16-24, who were being seen for well care or chronic care management visits, and had a portable medical summary initiated, had increased from 0% to 100%. CONCLUSIONS: The use of a PMS that summarizes medical care received in the pediatric system, is one tool that may be used to bridge the gap between pediatric and adult care.