Over the last few years, several disease modifying therapies have been developed to treat spinal muscular atrophy (SMA); some have already been market-approved, which has led to new disease trajectories and phenotypes.
With these new therapies the need to instate proactive and regular daily care for the new phenotypes is crucial. Since physiotherapy seems to be difficult to translate between healthcare systems were other therapies or procedures have been more easily adapted, it is challenging to identify the most suitable approach to provide physiotherapy to patients with SMA.
In order to provide a better understanding of current practices undertaken with patients with SMA, an informal survey was conducted. The results indicated that physiotherapy is not provided on a regular basis by the NHS to children living with SMA, and that the parents of these children assume responsibility for the greater amount of physiotherapy rehabilitation themselves.
Although the need for daily care of a child with SMA is unavoidable, we believe it is important to reduce this daily burden on parents in order to shift their role away from full-time care to allow for more balanced parental responsibilities.
The aim of this project is to study the benefit of optimising the current physiotherapy practices for patients with SMA between one and six years of age treated post-symptomatically. The intention is not to change the whole system, nor to add expensive devices and rehabilitation programs that would be unaffordable and unmanageable for the care teams, but rather to show that a more personalised physiotherapy care plan combined with more frequent physiotherapy may benefit all parties. We intend to ensure a rigorous follow-up of the physiotherapy care plan, in order to provide the most suitable approach for each individual patient.
In addition, as an exploratory approach we also plan to offer access to new rehabilitation devices when the need exists, the use of which will be supervised by a trained PT. In terms of efficacy outcomes to measure the potential benefits of the adapted and personalized care plans (frequency, type of approach, increased communication between tertiary and -community PT), we will refer to the functional assessments conducted in the frame of the SMA-REACH UK study. If a patient is unable to attend their assessments, our PT team will perform the functional assessments in the frame of this project during the planned site visits.
If successful in changing the prescribed care regime, this project will help develop understanding of the importance of the frequent provision of rehabilitation by PTs to patients with SMA, and consequently reduce the care burden on the families. This project will take place in the first instance in Oxfordshire and will serve as proof of concept for lobbying changes in the health system for other counties in United Kingdom.