Huntington Society of Canada, Waterloo, Ontario, Canada.
Department of Medicine, Université de Montréal, Montreal, Quebec, Canada.
BMJ Open. 2022 Jun 1;12(6):e062740. doi: 10.1136/bmjopen-2022-062740.
Disease-modifying therapies in development for Huntington's disease (HD) may require specialised administration and additional resource capacity. We sought to understand current and future capacity for HD management in Canada considering the possible introduction of an intrathecal (IT) disease-modifying treatment (DMT).
DESIGN, SETTING AND PARTICIPANTS: Using a case study, mixed methods framework, online surveys followed by semistructured interviews were conducted in late 2020 and early 2021. Neurologists from Canadian HD (n=16) and community (n=11) centres and social workers (n=16) were invited to complete online surveys assessing current HD management and potential capacity to support administration of an IT DMT.
Survey responses, anticipated demand and assumed resource requirements were modelled to reveal capacity to treat (ie, % of eligible patients) by centre. Resource bottlenecks and incremental support required (full-time equivalent, FTE) were also determined.
Neurologists from 15/16 HD centres and 5/11 community centres, plus 16/16 social workers participated. HD centres manage 94% of patients with HD currently seeking care in Canada, however, only 20% of IT DMT-eligible patients are currently seen by neurologists. One-third of centres have no access to nursing support. The average national incremental nursing, room, neurologist and social worker support required to provide IT DMT to all eligible patients is 0.73, 0.36, 0.30 and 0.21 FTE per HD centre, respectively. At peak demand, current capacity would support the treatment of 6% of IT DMT-eligible patients. If frequency of administration is halved, capacity for IT-DMT administration only increases to 11%.
In Canada, there is little to no capacity to support the administration of an IT DMT for HD. Current inequitable and inadequate resourcing will require solutions that consider regional gaps and patient needs.
针对亨廷顿病（HD），目前正在研发多种疾病修正疗法（DMT），这些疗法可能需要专门的管理和额外的资源支持。因此，我们研究了加拿大目前和未来管理 HD 的能力，以评估潜在的鞘内（IT）疾病修正治疗（DMT）的引入。
设计、地点和参与者：本研究采用案例研究，混合方法框架，于 2020 年末和 2021 年初开展了在线调查和半结构化访谈。我们邀请了加拿大 HD 中心（n=16）和社区中心（n=11）的神经科医生以及社会工作者（n=16）完成在线调查，评估当前 HD 的管理情况以及支持 IT-DMT 管理的潜在能力。
我们对调查结果、预期需求和假设的资源需求进行建模，以揭示各中心的治疗能力（即，符合条件的患者比例）。此外，还确定了资源瓶颈和增量支持需求（全职当量，FTE）。
16 个 HD 中心中的 15 个和 11 个社区中心中的 5 个以及 16 名社会工作者参与了本研究。HD 中心目前管理着在加拿大寻求治疗的 94%的 HD 患者，但仅有 20%的 IT-DMT 合格患者由神经科医生诊治。三分之一的中心无法获得护理支持。在全国范围内，为所有符合条件的患者提供 IT-DMT 所需的平均增量护理、房间、神经科医生和社会工作者支持分别为每个 HD 中心 0.73、0.36、0.30 和 0.21FTE。在需求高峰期，目前的容量只能支持 6%的 IT-DMT 合格患者的治疗。如果给药频率减半，仅能将 IT-DMT 给药的容量增加到 11%。
在加拿大，几乎没有能力支持 HD 的 IT-DMT 管理。目前资源分配不均且不足，需要考虑区域差距和患者需求来解决这些问题。
