Ranchin Bruno, Schmitt Claus Peter, Warady Bradley A, Hataya Hiroshi, Jones Joanne, Lalji Rowena, Licht Christoph, Mosca Melodie, Stronach Lynsey, Vidal Enrico, Walle Johan Vande, Shroff Rukshana
Pediatric Nephrology Unit, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Université de Lyon, Lyon, France.
Centre de référence des maladies rénales rares, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 boulevard Pinel, 69677, Bron Cedex, France.
Pediatr Nephrol. 2024 Sep;39(9):2579-2591. doi: 10.1007/s00467-023-06233-0. Epub 2023 Dec 23.
Children requiring long-term kidney replacement therapy are a "rare disease" cohort. While the basic technical requirements for hemodialysis (HD) are similar in children and adults, key aspects of the child's cardiovascular anatomy and hemodynamic specifications must be considered. In this article, we describe the technical requirements for long-term HD therapy for children and the devices that are currently available around the world. We highlight the characteristics and major technical shortcomings of permanent central venous catheters, dialyzers, dialysis machines, and software available to clinicians who care for children. We show that currently available HD machines are not equipped with appropriately small circuits and sensitive control mechanisms to perform safe and effective HD in the youngest patients. Manufacturers limit their liability, and health regulatory agencies permit the use of devices, only in children according to the manufacturers' pre-specified weight limitations. Although registries show that 6-23% of children starting long-term HD weigh less than 15 kg, currently, there is only one long-term HD device that is cleared for use in children weighing 10 to 15 kg and none is available and labelled for use in children weighing less than 10 kg anywhere in the world. Thus, many children are being treated "off-label" and are subject to interventions delivered by medical devices that lack pediatric safety and efficacy data. Moreover, recent improvements in dialysis technology offered to adult patients are denied to most children. We, in turn, advocate for concerted action by pediatric nephrologists, industry, and health regulatory agencies to increase the development of dedicated HD machines and equipment for children.
需要长期肾脏替代治疗的儿童是一个“罕见病”群体。虽然儿童和成人血液透析(HD)的基本技术要求相似,但必须考虑儿童心血管解剖结构和血流动力学指标的关键方面。在本文中,我们描述了儿童长期HD治疗的技术要求以及目前世界各地可用的设备。我们强调了永久性中心静脉导管、透析器、透析机和可供照顾儿童的临床医生使用的软件的特点和主要技术缺陷。我们表明,目前可用的HD机器没有配备适当小的回路和灵敏的控制机制,无法在最年幼的患者中进行安全有效的HD。制造商限制其责任,卫生监管机构仅允许根据制造商预先指定的体重限制在儿童中使用设备。尽管登记数据显示,开始长期HD治疗的儿童中有6%-23%体重不足15千克,但目前只有一种长期HD设备被批准用于体重10至15千克的儿童,世界上任何地方都没有可供体重不足10千克的儿童使用且有标签的设备。因此,许多儿童正在接受“超说明书”治疗,并且要接受缺乏儿科安全性和有效性数据的医疗设备的干预。此外,提供给成年患者的透析技术的最新改进大多数儿童无法受益。相应地,我们倡导儿科肾病学家、行业和卫生监管机构采取协调行动,以增加针对儿童的专用HD机器和设备的开发。