Department of Pediatrics, Division of Respiratory Medicine, Rady Children's Hospital San Diego, University of California San Diego, San Diego, California.
Department of Orthopedics, Division of Rehabilitation Medicine, Rady Children's Hospital San Diego, University of California San Diego, San Diego, California.
J Clin Sleep Med. 2021 Oct 1;17(10):1973-1980. doi: 10.5664/jcsm.9400.
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that leads to chronic respiratory insufficiency and failure. Use of home noninvasive ventilation (NIV) has been linked to improved outcomes including reduced mortality. Despite the importance of NIV, factors promoting optimal NIV usage and determinants of nonadherence have not been rigorously examined. Moreover, given that respiratory issues in DMD span between childhood and adulthood, examination across a broad age group is needed. The objectives of this study were to (1) evaluate NIV usage across a broad spectrum of patients with DMD, including both children and adults, and (2) identify biological and socioeconomic determinants of NIV usage and NIV nonadherence.
We performed a retrospective review of all patients with DMD from February 2016 to February 2020 who underwent evaluation at associated pediatric and adult neuromuscular disease clinics. NIV use was determined objectively from device downloads. A priori, we defined nonadherence as < 4 hours use per night, quantified as the percentage of nights below this threshold across a 30-day period within 6 months of a clinic visit. We also assessed the average hours of NIV usage over this time period. Predictors examined included demographics, social determinants, and pulmonary function.
33 patients with DMD were identified, 29 (87%) of whom were using NIV (13 age < 21 years). Mean age was 22.9 ± 6.6 years (range 13-39 years), body mass index was 23.4 ± 10.4 kg/m, and seated forced vital capacity was 23% ± 18% predicted. Mean nightly NIV usage was 7.4 ± 3.8 hours and mean percentage of nonadherent nights was 13% ± 30%. In univariable analysis, age did not predict use. Those with lower forced vital capacity had higher NIV usage hours ( = .01) and a trend toward less nonadherence ( = .06). Higher estimated household income demonstrated a trend toward increased usage hours and less nonadherence (both = .08). Multivariable analysis found increased usage hours were predicted best by higher income, higher inspiratory positive airway pressure, and higher bicarbonate. Nonadherence was higher in those with lower income or higher forced vital capacity.
In this cohort of adult and pediatric patients with DMD, most individuals were using NIV. While usage hours were higher with lower lung function, substantial variability remains unexplained by examined factors. Nonadherence was observed in some individuals, including those with advanced disease. Further investigations should focus on evaluating patient-oriented outcomes to define optimal NIV usage across the spectrum of disease and determine strategies to counteract issues with nonadherence.
Hurvitz MS, Bhattacharjee R, Lesser DJ, Skalsky AJ, Orr JE. Determinants of usage and nonadherence to noninvasive ventilation in children and adults with Duchenne muscular dystrophy. . 2021;17(10):1973-1980.
杜氏肌营养不良症（DMD）是一种神经肌肉疾病，导致慢性呼吸功能不全和衰竭。家庭使用无创通气（NIV）已被证明可改善预后，包括降低死亡率。尽管 NIV 很重要，但促进最佳 NIV 使用的因素和不依从的决定因素尚未得到严格检查。此外，鉴于 DMD 的呼吸问题跨越儿童期和成年期，需要在广泛的年龄组中进行检查。本研究的目的是：（1）评估广泛的 DMD 患者的 NIV 使用情况，包括儿童和成人；（2）确定 NIV 使用和 NIV 不依从的生物学和社会经济决定因素。
我们对 2016 年 2 月至 2020 年 2 月在相关儿科和成人神经肌肉疾病诊所接受评估的所有 DMD 患者进行了回顾性分析。通过设备下载客观地确定 NIV 使用情况。我们预先定义不依从性为每晚使用时间<4 小时，通过在就诊前 6 个月内的 30 天内每晚低于该阈值的百分比来量化。我们还评估了这段时间内的平均 NIV 使用时间。检查的预测因素包括人口统计学、社会决定因素和肺功能。
共确定了 33 名 DMD 患者，其中 29 名（87%）正在使用 NIV（13 名年龄<21 岁）。平均年龄为 22.9 ± 6.6 岁（范围 13-39 岁），体重指数为 23.4 ± 10.4 kg/m，坐位用力肺活量为预计值的 23%±18%。平均每晚 NIV 使用时间为 7.4 ± 3.8 小时，平均不依从性夜间百分比为 13%±30%。在单变量分析中，年龄并不能预测使用情况。用力肺活量较低者 NIV 使用时间较长（ =.01），不依从性趋势较低（ =.06）。较高的估计家庭收入显示出与较高的使用时间和较低的不依从性相关的趋势（两者均 =.08）。多变量分析发现，较高的收入、较高的吸气正压和较高的碳酸氢盐水平可以更好地预测使用时间的增加。收入较低或用力肺活量较低的患者不依从性较高。
在本队列中，大多数患有 DMD 的成年和儿科患者都在使用 NIV。虽然随着肺功能下降，使用时间增加，但通过检查的因素，仍存在大量无法解释的差异。一些患者（包括患有晚期疾病的患者）存在不依从现象。进一步的研究应集中在评估以患者为导向的结果上，以确定疾病谱中最佳的 NIV 使用情况，并确定克服不依从问题的策略。
Hurvitz MS, Bhattacharjee R, Lesser DJ, Skalsky AJ, Orr JE. Determinants of usage and nonadherence to noninvasive ventilation in children and adults with Duchenne muscular dystrophy.. 2021;17(10):1973-1980.
