Geevasinga Nimeshan, Ryan Monique M
Institute for Neuromuscular Research, T.Y. Nelson Department of Neurology and Neurosurgery, The Children's Hospital at Westmead, Westmead, New South Wales, Australia.
J Paediatr Child Health. 2007 Dec;43(12):790-4. doi: 10.1111/j.1440-1754.2007.01197.x. Epub 2007 Sep 4.
Without ventilatory support, premature death from respiratory insufficiency is virtually universal in infants with spinal muscular atrophy type 1 (SMA1). With mechanical ventilation, however, long-term survival has been reported from numerous international centres. We aimed to characterize physician attitudes to the various forms of ventilatory support for children with SMA1.
We surveyed neurologists, respiratory physicians, clinical geneticists and intensivists from all major paediatric hospitals in Australia and New Zealand regarding their views on ventilatory management of SMA1.
Ninety-two of the 157 (59%) physicians surveyed replied. Respondents included 16 clinical geneticists, 19 intensive care physicians, 28 neurologists and 29 respiratory physicians. Almost half (47%) opposed invasive ventilation of children with SMA1 and respiratory failure precipitated by intercurrent illness. The majority (76%) opposed invasive ventilatory support for chronic respiratory failure in SMA1. In contrast, non-invasive ventilation was felt by 85% to be appropriate for acute respiratory deteriorations, with 49% supporting long-term non-invasive ventilatory support. Most physicians felt that decisions regarding ventilation should be made jointly by parents and doctors, and that hospital Clinical Ethics Committees should be involved in the event of discordant opinion regarding further management. A majority felt that a defined hospital policy would be valuable in guiding management of SMA1.
Respiratory support in SMA1 is an important issue with significant ethical, financial and resource management implications. Most physicians in Australian and New Zealand oppose invasive ventilatory support for chronic respiratory failure in SMA1. Non-invasive ventilation is an accepted intervention for acute respiratory decompensation and may have a role in the long-term management of SMA1. Clinical Ethics Committees and institutional policies have a place in guiding physicians and parents in the management of children with SMA1.
对于1型脊髓性肌萎缩症（SMA1）患儿，若没有通气支持，因呼吸功能不全导致的过早死亡几乎难以避免。然而，通过机械通气，众多国际中心都报道了患儿的长期存活情况。我们旨在了解医生对SMA1患儿各种通气支持形式的态度。
我们对澳大利亚和新西兰所有主要儿科医院的神经科医生、呼吸科医生、临床遗传学家和重症监护医生进行了调查，询问他们对SMA1通气管理的看法。
157名接受调查的医生中有92名（59%）回复。回复者包括16名临床遗传学家、19名重症监护医生、28名神经科医生和29名呼吸科医生。近一半（47%）的医生反对对患有SMA1且因并发疾病引发呼吸衰竭的患儿进行有创通气。大多数（76%）医生反对对SMA1慢性呼吸衰竭患儿进行有创通气支持。相比之下，85%的医生认为无创通气适用于急性呼吸恶化情况，49%的医生支持长期无创通气支持。大多数医生认为通气决策应由家长和医生共同做出，若在进一步治疗方面存在意见分歧，医院临床伦理委员会应参与其中。大多数人认为明确的医院政策对指导SMA1的治疗很有价值。
SMA1的呼吸支持是一个重要问题，具有重大的伦理、财务和资源管理意义。澳大利亚和新西兰的大多数医生反对对SMA1慢性呼吸衰竭患儿进行有创通气支持。无创通气是急性呼吸失代偿的一种可接受的干预措施，可能在SMA1的长期治疗中发挥作用。临床伦理委员会和机构政策在指导医生和家长管理SMA1患儿方面具有一定作用。
