Archives De Pediatrie最新文献_第2页

Implementation in the Ile-de-France region of French clinical guidelines for the management of newborns at risk of early-onset neonatal infection. 在法兰西岛大区实施法国早发性新生儿感染风险管理临床指南。

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-15 DOI: 10.1016/j.arcped.2025.11.002

Mathilde Letouzey, Elisabeth Outtier, Tiphaine Biaggi, Anne Rousseau, Pascal Boileau, Laurence Foix-L'Hélias

Background: Accurately targeting newborns suspected of early-onset bacterial neonatal infection (EONI) and optimizing the administration of antibiotics is challenging. French clinical guidelines (HAS/SFN) on the management of newborns over 34 weeks at risk of EONI were deployed in September 2017.

Objective: Our objective was to assess their implementation 3 years later in the Paris area, representing approximately 180,000 births.

Methods and settings: We conducted an observational study in all maternity units of the Ile-de-France region from July 2020 to January 2021. We sent a self-administered questionnaire on the application of the new guidelines by email addressed to a pediatrician in each maternity unit.

Results: Out of the 80 maternity units in the Ile-de-France region, 78 participated. Among the participating units, 40 (51 %) reported applying the new clinical guidelines fully, 28 (36 %) partially, while 10 (13 %) reported not applying them. Among participants, 45 (66 %) maternity units found the implementation of the clinical guidelines feasible, and nearly 80 % of them observed that applying them resulted in fewer additional tests, such as blood samples or peripheral bacteriological tests. Only 35 % of maternity units no longer collect gastric fluid sample, even though this practice is no longer recommended. Among the difficulties identified by the 10 centers that reported not implementing the guidelines were a lack of equipment, insufficient numbers of competent personnel, and insufficient time to train relevant professionals.

Conclusion: Three years after the publication of the French clinical guidelines (HAS/SFN) on the management of newborns over 34 weeks at risk of EONI, this study showed that over 85 % of responding maternity units in the Ile-de-France region reported have implemented the guidelines either fully or partially.

背景：准确定位疑似早发型新生儿细菌性新生儿感染（EONI）并优化抗生素给药具有挑战性。法国临床指南（HAS/SFN）关于34周以上EONI风险新生儿的管理于2017年9月发布。目标：我们的目标是评估3年后在巴黎地区的实施情况，该地区约有18万名新生儿。方法和环境：我们于2020年7月至2021年1月在法兰西岛地区的所有产科单位进行了一项观察性研究。我们通过电子邮件向每个产科病房的儿科医生发送了一份关于新指南应用情况的自我管理问卷。结果：在法兰西岛地区的80个产科单位中，有78个参加了该计划。在参与的单位中，40个（51%）报告完全应用新的临床指南，28个（36%）部分应用，而10个（13%）报告未应用。在参与者中，有45家（66%）产科单位认为实施临床指南是可行的，其中近80%的单位观察到，应用这些指南可以减少额外的检查，如血液样本或外周细菌学检查。只有35%的产科单位不再收集胃液样本，尽管这种做法不再被推荐。据报告，未执行指导方针的10个中心指出的困难包括缺乏设备、合格人员数量不足以及培训相关专业人员的时间不足。结论：在法国临床指南（HAS/SFN）关于34周以上EONI风险新生儿管理的出版三年后，这项研究表明，在法兰西岛地区，85%以上的响应产科单位完全或部分地实施了指南。

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引用次数: 0

Respiratory care in Duchenne muscular dystrophy 杜氏肌营养不良症的呼吸护理

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00250-7

Hélène Prigent

Respiratory complications are amajor cause of morbidity and mortality in Duchenne muscular dystrophy (DMD). Progressive weakness of inspiratory and expiratory muscles leads to ineffective cough, mucus retention, sleep-disordered breathing, nocturnal hypoventilation, and ultimately chronic respiratory failure. Before the advent of mechanical ventilation, respiratory failure was the leading cause of early death. Advances in multidisciplinary care, including noninvasive ventilation (NIV), airway clearance techniques, corticosteroid therapy, scoliosis management, and cardioprotective treatments, have markedly improved survival and quality of life. Respiratory decline typically follows loss of ambulation, reflecting the progressive loss of muscle strength. Hypoventilation first appears during REM sleep and evolves into daytime hypercapnia. Regular monitoring—annually before and biannually after loss of ambulation—is essential. Key respiratory monitoring parameters include decreased vital capacity, maximal inspiratory and expiratory pressures, and peak cough flow. Sleep studies are critical for early detection of nocturnal hypoventilation and sleep-disordered breathing. NIV remains the cornerstone of therapy, improving gas exchange, sleep quality, and life expectancy. As weakness increases, patients may become ventilator dependent; invasive ventilation is reserved for NIV failure. Adjunctive strategies such as lung volume recruitment and mechanical insufflation–exsufflation enhance cough efficacy and secretion clearance. Long-term corticosteroid therapy delays respiratory decline, while scoliosis correction and infection prevention further optimize outcomes. Nutritional management also supports respiratory health. Overall, early detection, individualized ventilatory management, and comprehensive multidisciplinary care are critical to improving survival and quality of life in patients with DMD.

呼吸系统并发症是杜氏肌营养不良症（DMD）发病和死亡的主要原因。吸气和呼气肌的进行性无力导致无效咳嗽、粘液潴留、睡眠呼吸障碍、夜间换气不足，最终导致慢性呼吸衰竭。在机械通气出现之前，呼吸衰竭是早期死亡的主要原因。多学科治疗的进步，包括无创通气（NIV）、气道清除技术、皮质类固醇治疗、脊柱侧凸管理和心脏保护治疗，显著提高了生存率和生活质量。呼吸衰退通常伴随着行走能力的丧失，反映了肌肉力量的逐渐丧失。低通气首先出现在快速眼动睡眠期间，并演变为白天高碳酸血症。定期监测——丧失行动能力之前每年一次，丧失行动能力之后每两年一次——是必要的。关键的呼吸监测参数包括肺活量下降、最大吸气和呼气压力以及咳嗽流量峰值。睡眠研究对于早期发现夜间低通气和睡眠呼吸障碍至关重要。NIV仍然是治疗的基石，改善气体交换，睡眠质量和预期寿命。随着虚弱程度的增加，患者可能会依赖呼吸机；有创通气用于无创通气失败。辅助策略，如肺容量增加和机械吸气-呼气，可提高咳嗽疗效和分泌物清除。长期皮质类固醇治疗可延缓呼吸衰退，而脊柱侧凸矫正和感染预防可进一步优化预后。营养管理也有助于呼吸系统健康。总之，早期发现、个体化通气管理和综合多学科护理对改善DMD患者的生存和生活质量至关重要。

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引用次数: 0

Corticosteroid treatment in Duchenne muscular dystrophy 皮质类固醇治疗杜氏肌营养不良症

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00253-2

Stéphanie Fontaine-Carbonnel

Duchenne muscular dystrophy (DMD) is a severe degenerative disease that remains incurable. Its prognosis has been transformed by multidisciplinary care, which has significantly improved the life expectancy of patients. Corticosteroids have been used for over 20 years and have modified the care of young boys with DMD: They delay the loss of ambulation, support the gradual introduction of technical mobility aids, and reduce the frequency of scoliosis surgery. Corticoids are started and maintained at the theoretical dose of 0.75 mg/kg/day of prednisone/prednisolone or 0.9 mg/kg/day of deflazacort, adjusting the dose to weight if the benefit–risk ratio favors treatment. If introduced early enough, when muscle degeneration is not too advanced, they prolong the ability to climb stairs, rise from the floor, walk, and bring the hands to the mouth. They have been suggested to contribute to delaying the onset of respiratory failure and cardiomyopathy. The well-known side effects of corticosteroids require specific management (to prevent obesity and osteoporosis) and/or adjustment of their dosage. The only reasons to discontinue treatment when the patient and family are adhering to it are the uncontrolled side effects. Thus, in many countries including France, as of late 2025, the only routinely prescribed symptomatic treatment for DMD remains conventional corticosteroids, which will be discussed in this article, followed by a description of vamorolone, a dissociative steroidal compound, and givinostat, a histone deacetylase inhibitor.

杜氏肌营养不良症（DMD）是一种严重的退行性疾病，目前仍无法治愈。多学科治疗改变了该病的预后，显著提高了患者的预期寿命。皮质类固醇已经使用了20多年，并改善了患有DMD的年轻男孩的护理：它们延迟了行动能力的丧失，支持逐渐引入技术活动辅助设备，并减少了脊柱侧凸手术的频率。皮质激素开始并维持在理论剂量0.75 mg/kg/天的强的松/泼尼松或0.9 mg/kg/天的地氮柯，如果获益-风险比有利于治疗，调整剂量与体重。如果尽早使用，在肌肉退化不太严重的时候，它们可以延长爬楼梯、从地板上站起来、走路和用手到嘴的能力。它们被认为有助于延缓呼吸衰竭和心肌病的发作。众所周知，皮质类固醇的副作用需要特殊的管理（防止肥胖和骨质疏松）和/或调整其剂量。当病人和家人坚持治疗时，停止治疗的唯一原因是无法控制的副作用。因此，在包括法国在内的许多国家，截至2025年底，DMD唯一的常规对症治疗仍然是常规的皮质类固醇，这将在本文中讨论，然后是对氨莫洛酮（一种解离性类固醇化合物）和吉维司他（一种组蛋白去乙酰化酶抑制剂）的描述。

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引用次数: 0

Duchenne muscular dystrophy in 2025 2025年的杜氏肌萎缩症

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00245-3

Isabelle Desguerre

引用次数: 0

Neuropsychological management in Duchenne muscular dystrophy: A critical overview and future directions 杜氏肌营养不良症的神经心理学治疗：关键综述和未来方向

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00252-0

Nathalie Angeard

Pediatric neuromuscular disorders constitute a highly heterogeneous group of over 200 distinct conditions, varying in etiology, affected structures (e.g., peripheral motor neurons, neuromuscular junction, or muscle tissue), central nervous system involvement, age of onset, severity, and progression. Among these, Duchenne muscular dystrophy (DMD) is the most common and extensively studied genetic neuromuscular disorder in childhood. Increasing evidence suggests that DMD is not solely a motor condition but also involves a distinct neuropsychological phenotype, frequently characterized by cognitive impairments and comorbid neurodevelopmental disorders. This article provides a comprehensive review of the neuropsychological profile associated with DMD, with particular emphasis on language, memory, executive functions, and social cognition. The developmental trajectory of these domains is discussed in light of recent findings, including genotype–phenotype correlations. Furthermore, we offer recommendations for systematic neuropsychological assessment and screening for early non-motor symptoms, and propose emerging intervention strategies to support cognitive development in individuals with DMD.

小儿神经肌肉疾病是一个高度异质性的群体，有200多种不同的病症，在病因、受影响的结构（如外周运动神经元、神经肌肉连接或肌肉组织）、中枢神经系统受累、发病年龄、严重程度和进展等方面各不相同。其中，杜氏肌营养不良症（DMD）是儿童时期最常见和研究最广泛的遗传性神经肌肉疾病。越来越多的证据表明，DMD不仅是一种运动疾病，而且还涉及一种独特的神经心理学表型，通常以认知障碍和共病神经发育障碍为特征。本文提供了与DMD相关的神经心理学概况的全面回顾，特别强调语言，记忆，执行功能和社会认知。根据最近的发现，包括基因型-表型相关性，讨论了这些结构域的发育轨迹。此外，我们还建议对早期非运动症状进行系统的神经心理学评估和筛查，并提出新的干预策略，以支持DMD患者的认知发展。

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引用次数: 0

Gene therapy in Duchenne muscular dystrophy 杜氏肌营养不良症的基因治疗

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00254-4

V. Laugel

Duchenne muscular dystrophy (DMD) is a severe X-linked myopathy caused by mutations in the DMD gene, resulting in the absence of functional dystrophin. Gene therapy seems to represent a rational therapeutic strategy, aiming to restore dystrophin expression through delivery of engineered microdystrophin constructs using adeno-associated virus (AAV) vectors. Preclinical studies in murine and canine models demonstrated robust dystrophin restoration, histological improvement, and functional benefit, supporting the transition to clinical trials. Over the past decade, five AAV-microdystrophin programs have entered large-scale human testing. In 2023, delandistrogene moxeparvovec (Sarepta/Roche) received accelerated approval from the U.S. Food and Drug Administration for ambulatory pediatric patients, marking the first regulatory authorization of a gene therapy for DMD. By contrast, fordadistrogene movaparvovec (Pfizer) showed encouraging biomarker results but was associated with immune-mediated serious adverse events, including thrombotic microangiopathy cases and patient deaths due to acute liver failure, ultimately leading to program discontinuation. Other investigational candidates—GNT0004 (Généthon), SGT-003 (Solid Biosciences), and RGX-202 (Regenxbio)—incorporate distinct promoter designs and microdystrophin cassettes and are currently in early- to mid-phase evaluation. Key issues include immunogenicity against AAV capsids and transgene products, durability of expression, and the need for re-dosing or combinatorial strategies. Gene therapy management also raises difficult economic and logistical challenges for healthcare systems. Balancing rapid patient access to potentially disease-modifying therapies with rigorous scientific and regulatory standards is essential to ensure safe and durable benefit for individuals with DMD.

杜氏肌营养不良症（DMD）是一种严重的x连锁肌病，由DMD基因突变引起，导致功能性肌营养不良蛋白缺失。基因治疗似乎是一种合理的治疗策略，旨在通过使用腺相关病毒（AAV）载体传递工程化微肌营养不良蛋白构建物来恢复肌营养不良蛋白的表达。在小鼠和犬模型中进行的临床前研究表明，抗肌营养不良蛋白的恢复、组织学改善和功能改善，支持向临床试验的过渡。在过去的十年里，五个aav -微营养不良蛋白项目已经进入了大规模的人体测试。2023年，delandistrogene moxeparvovec （Sarepta/罗氏）获得美国食品和药物管理局（fda）加速批准，用于门诊儿科患者，标志着首个DMD基因治疗的监管批准。相比之下，福达反相基因莫帕韦克（辉瑞）显示出令人鼓舞的生物标志物结果，但与免疫介导的严重不良事件相关，包括血栓性微血管病变病例和急性肝功能衰竭导致的患者死亡，最终导致项目终止。其他正在研究的候选药物——gnt0004 （g生物科学公司）、SGT-003 （Solid Biosciences公司）和RGX-202 （Regenxbio公司）——采用不同的启动子设计和微肌营养不良蛋白盒，目前处于早期到中期评估阶段。关键问题包括针对AAV衣壳和转基因产物的免疫原性，表达的持久性以及重新给药或组合策略的需要。基因治疗管理也给卫生保健系统带来了困难的经济和后勤挑战。平衡患者快速获得潜在的疾病改善疗法与严格的科学和监管标准对于确保DMD患者安全和持久的益处至关重要。

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引用次数: 0

Orthopedic management in Duchenne muscular dystrophy 杜氏肌营养不良症的骨科治疗

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00251-9

Mathilde Gaumé , Vincent Cunin , Carole Vuillerot

This article provides a comprehensive overview of the orthopedic management strategies in Duchenne muscular dystrophy (DMD). DMD manifests with severe muscle wasting, respiratory insufficiency, and cardiomyopathy, with initial symptoms emerging in early childhood. By the age of 10–12 years, most patients require wheelchairs due to muscle degeneration, which often leads to spinal deformities and joint contractures. The absence of dystrophin causes the breakdown of the dystrophin glycoprotein complex, causingmuscle degeneration, necrosis, and fibrosis. Current treatments focus on symptom management, including physical therapy, corticosteroids, orthopedic surgery, ventilatory and nutritional support, and cardiac care. Long-term glucocorticoid therapy can extend mobility and life expectancy, delay the need for ventilation, and reduce the rates of scoliosis surgery. Orthopedic care focuses on prevention to preserve motor function and bone health and involves an interdisciplinary team, early use of orthotic devices, and promotion of proper posture. Common deformities include varus equinus, hip flexor retractions, scoliosis, and pelvic obliquity. Scoliosis management involves posterior fusion surgery extended to the pelvis or limited to the L5 vertebra. Preoperative evaluation should include assessing the risk of hip flexion contracture decompensation following arthrodesis. There now seems to be increasing consensus favoring lower extremity surgery only in exceptional cases. This shift is due to the limited success of surgery, with frequent recurrence of muscle retractions and minimal expected benefits. Fracture management involves bisphosphonate therapy post-fracture, with vertebral compression and lower limb fracture being the most prevalent.

本文综述了杜氏肌营养不良症（DMD）的骨科治疗策略。DMD表现为严重的肌肉萎缩、呼吸功能不全和心肌病，最初症状出现在儿童早期。到10-12岁时，大多数患者由于肌肉退行性变而需要轮椅，这往往导致脊柱畸形和关节挛缩。肌营养不良蛋白的缺乏导致肌营养不良蛋白糖蛋白复合物的分解，引起肌肉变性、坏死和纤维化。目前的治疗侧重于症状管理，包括物理治疗、皮质类固醇、骨科手术、通气和营养支持以及心脏护理。长期糖皮质激素治疗可以延长活动能力和预期寿命，延迟通气的需要，并减少脊柱侧凸手术的发生率。骨科护理的重点是预防，以保持运动功能和骨骼健康，包括一个跨学科的团队，早期使用矫形装置，并促进正确的姿势。常见的畸形包括马蹄内翻、髋屈肌后缩、脊柱侧凸和骨盆倾斜。脊柱侧凸的治疗包括后路融合手术，可延伸至骨盆或局限于L5椎体。术前评估应包括评估关节融合术后髋关节屈曲挛缩失代偿的风险。现在似乎有越来越多的共识赞成下肢手术只在特殊情况下。这一转变是由于手术成功率有限，肌肉收缩频繁复发，预期收益甚微。骨折治疗包括骨折后双膦酸盐治疗，椎体压迫和下肢骨折是最常见的。

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引用次数: 0

Genetics and pathophysiology of Duchenne muscular dystrophy 杜氏肌营养不良症的遗传与病理生理

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00246-5

Helge Amthor , Aurélie Avril , France Leturcq

Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder caused by mutations in the DMD gene located on the Xp21.2–Xp21.1 region of the X chromosome. This gene encodes dystrophin, a cytoskeletal protein that is critical for the stability of muscle fibers. With an incidence of approximately 1 in 5,000 males, DMD is the most common form of inherited muscular dystrophy in childhood. The DMD gene produces multiple tissue-specific dystrophin isoforms. Absence of the full-length Dp427 isoform leads to DMD, while residual expression results in milder phenotypes, such as Becker muscular dystrophy (BMD). Mostmutations are intragenic deletions, and the reading-frame rule predicts disease severity: Out-of-frame mutations abolish dystrophin synthesis, whereas in-frame deletions produce partially functional proteins. Dystrophin anchors the cytoskeleton to the dystrophin-associated protein complex (DAPC), linking contractile elements to the extracellular matrix. Loss of dystrophin disrupts sarcolemmal stability, leading to calcium influx, mitochondrial dysfunction, oxidative stress, and myofiber necrosis. This is followed by inflammation, fibrosis, and fatty infiltration. The mislocalization of neuronal nitric oxide synthase (nNOS) and reduced vascular endothelial growth factor (VEGF) impair vasoregulation and exacerbate ischemic injury. Brain involvement results from the loss of the Dp427, Dp140, and Dp71 dystrophin isoforms, which regulate synaptic and glial membrane architecture. The deficiency of these proteins contributes to cognitive impairment through disrupted GABAergic signaling, altered neurovascular function, and an imbalance in neuronal excitatory–inhibitory transmission.

杜氏肌营养不良症（DMD）是一种严重的X连锁隐性疾病，由位于X染色体Xp21.2-Xp21.1区域的DMD基因突变引起。该基因编码肌营养不良蛋白，这是一种细胞骨架蛋白，对肌肉纤维的稳定性至关重要。DMD是儿童时期最常见的遗传性肌肉萎缩症，发病率约为1 / 5000。DMD基因产生多种组织特异性肌营养不良蛋白亚型。缺少全长Dp427亚型会导致DMD，而剩余的表达会导致较轻的表型，如贝克肌营养不良症（BMD）。大多数突变是基因内缺失，而阅读框规则预测疾病的严重程度：框外突变消除营养不良蛋白合成，而框内缺失产生部分功能蛋白。肌营养不良蛋白将细胞骨架锚定在肌营养不良蛋白相关蛋白复合物（DAPC）上，将收缩元件与细胞外基质连接起来。肌营养不良蛋白的丧失破坏肌层稳定性，导致钙内流、线粒体功能障碍、氧化应激和肌纤维坏死。随后是炎症、纤维化和脂肪浸润。神经元一氧化氮合酶（nNOS）的错定位和血管内皮生长因子（VEGF）的减少损害血管调节，加剧缺血性损伤。Dp427、Dp140和Dp71抗营养不良蛋白亚型的缺失导致脑受损伤，而Dp427、Dp140和Dp71抗营养不良蛋白亚型调节突触和胶质膜结构。这些蛋白的缺乏通过破坏gaba能信号、改变神经血管功能和神经元兴奋抑制传递的不平衡而导致认知障碍。

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引用次数: 0

Multidisciplinary management of Duchenne muscular dystrophy from childhood to adulthood 儿童期至成年期杜氏肌营养不良症的多学科治疗

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00248-9

Juliette Ropars , Emmanuelle Salort-Campana

Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder characterized by muscle degeneration and weakness, leading to significant physical disability and multisystem complications. The management of DMD requires a coordinated, multidisciplinary approach to address the evolving needs of patients from childhood through adulthood, with a focus on patient quality of life. Early diagnosis and proactive interventions are critical to optimizing outcomes and may include physical therapy, respiratory support, cardiac monitoring, and orthopedic care. In children, the focus is on preserving mobility, managing scoliosis, preventing cardiac and respiratory complications, and ensuring access to education and psychosocial support. As patients transition to adulthood, the challenges shift to maintaining independence, managing advanced respiratory and cardiac complications, and addressing vocational and social integration. Transition programs must be carefully planned to bridge pediatric and adult care systems, ensuring continuity and minimizing gaps in care. Adult care involves specialized teams to manage complex medical needs, including advanced respiratory support, cardiomyopathy, and palliative care. Anticipatory directives play a crucial role in aligningmedical interventions with patient and family preferences, particularly in advanced stages of the disease. This article outlines the principles of multidisciplinary care for DMD, emphasizing the importance of tailored interventions at each life stage, the challenges of transition, and the ethical considerations of anticipatory planning. By integrating medical, psychosocial, and rehabilitative strategies, healthcare providers can improve the quality of life and longevity of individuals with DMD.

杜氏肌营养不良症（DMD）是一种严重的进行性神经肌肉疾病，以肌肉变性和无力为特征，导致严重的身体残疾和多系统并发症。DMD的管理需要一个协调的、多学科的方法来解决患者从童年到成年的不断变化的需求，重点是患者的生活质量。早期诊断和积极干预对优化结果至关重要，可能包括物理治疗、呼吸支持、心脏监测和骨科护理。在儿童中，重点是保持活动能力，管理脊柱侧凸，预防心脏和呼吸系统并发症，并确保获得教育和社会心理支持。随着患者进入成年期，挑战转变为维持独立性，管理晚期呼吸和心脏并发症，以及解决职业和社会融合问题。过渡方案必须仔细规划，以衔接儿科和成人护理系统，确保连续性并尽量减少护理差距。成人护理涉及专业团队来管理复杂的医疗需求，包括高级呼吸支持、心肌病和姑息治疗。预见性指示在将医疗干预与患者和家庭的偏好结合起来方面发挥着至关重要的作用，特别是在疾病的晚期。本文概述了DMD的多学科护理原则，强调了在每个生命阶段量身定制的干预措施的重要性，过渡的挑战，以及预期计划的伦理考虑。通过整合医疗、社会心理和康复策略，医疗保健提供者可以改善DMD患者的生活质量和寿命。

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引用次数: 0

Title Page 标题页

IF 1.3 4区医学 Q3 PEDIATRICS

Archives De Pediatrie

Pub Date : 2025-12-01 DOI: 10.1016/S0929-693X(25)00242-8

引用次数: 0