Abstract P-389: A REVIEW OF THE ETHICAL CONSIDERATIONS RELATING TO LONG TERM VENTILATION IN CHILDREN WITH SPINAL MUSCULAR ATROPHY TYPE -1. HAS NUSINERSEN CHANGED THE LANDSCAPE?

Abstract

Aims & Objectives: Increasing numbers of children and young adults are recipients of long-term ventilation at home. The decision to opt for long-term ventilation in children with Spinal Muscular Atrophy Type-1 (SMA-1) is one that has historically polarised opinion amongst clinicians. Whilst in other neuromuscular conditions outcomes and positive impact on quality of life are evident, outcome studies and quality of life surveys that focus on SMA-1 are scarce, with variable results and consist of small numbers. Methods In December 2016 the antisense oligonucleotide Nusinersen became the first approved drug to be used to treat SMA-1. In clinical trials, the drug halted the disease progression and was found to significantly improve motor function in around 60% of infants with the condition. The drug, which comes at a significant cost, has allowed for some cautious optimism amongst the medical community and has, in the author’s opinion, significantly changed the ethical landscape relating to long term ventilation decisions surrounding SMA-1. Results Balancing the four ethical principles of beneficence, non-maleficence, autonomy and distributive justice in this setting has always been a bioethical conundrum. Clinical management of these children varies internationally and is influenced by local societal norms, legal frameworks and whether healthcare is publicly or privately funded. Conclusions The article examines what ethical challenges paediatricians and families face in long-term ventilation decisions in children with the condition as well as how Nusinersen is bound to influence the ethical decision making in favour of LTV provision for these children.