When you first attend clinic, clinicians will assess how you are doing now so that they can have ‘standard baseline’ information. These include recording:

• nutritional status, including swallowing problems
  (see: The International Standards of Care for SMA / Nutrition, Growth & Bone Health)
• scoliosis
  (see: The International Standards of Care for SMA / Orthopaedic Management)
• fractures
  (see: The International Standards of Care for SMA / scroll down to ‘Impact on the Bones’ and ‘Bone Health for All’)
• ventilation / respiratory events; respiratory function tests
  (see: The International Standards of Care for SMA / Breathing (Respiratory & Pulmonary) Care).
• motor function using SMA validated scales appropriate for the level of function of the patient (see below).

Our thanks to the Sheffield Team for talking this through:

Most often measures are in the form of a scale which captures some ‘functional measure’ of your abilities. Which ones are used depend to some extent on age but also on what will provide useful information. Many focus on ‘motor performance’ and it’s recognised that they only capture what this is like on the actual day of assessment – people can have ‘off’ days or really great days.

Scales used include:

• The Revised Hammersmith Scale (RHS)
• Revised Upper limb Module (RULM)
• CHOP-Infant Test of Neuromuscular Disorders (CHOP-INTEND)
• Hammersmith Infant Neurological Examination – motor milestones (HINE)
• WHO Motor Milestones (WHO MM)
• The Adult version of the CHOP-INTEND, ATEND – which is in development

Some scales record patient reported outcome measures, such as:

• The Egan Klassifikation 2 scale (EK2)

Some give information on fatigue such as:

• The 6MWT (6-minute walk test) – but aren’t useful for many who have SMA

At the beginning of treatment, your physio will discuss with you, which scales are most suitable for you. They will be looking for scales that can show your current motor functional ability as well as ones that will show where change might occur for you.

Ideally you will remain on one key scale for the length of the Managed Access Agreement. However, it is recognised that it is essential to capture any changes happening and it may be that a scale you are currently using is no longer helpful. For example, a child may become too strong to be measured on the CHOP -INTEND so has to transfer to the RHS; a young child may no longer be able to do the CHOP-INTEND due to their age, so starts to be assessed on the ATEND.

Additionally, though, as not all scales measure the same thing, you may be measured on more than one scale so that information that is important for you is captured. So, for example, an adult who is a non-sitter may be assessed on three different scales – the ATEND (motor ability measure), the EK2 (patient reported outcomes) and the RULM (Upper limb strength / ability) – as they all capture different aspects of what’s going on for you. Your physio will discuss with you if you/your child needs to change or use more than one scale.

For each scale, the MAA defines how many points someone needs to drop for it to be considered that they have deteriorated. You can find more detail of this in:

Page 3 Table 1 of the Nusinersen Managed Access Agreement Variation 2

NHS England has also clarified that the equivalent measure of deterioration in the RULM scale is a deterioration of more than or equal to 2 points (>=2 points).

The MAA also recognises that people can have ‘off days’ so states that there need to be two consecutive measures taken that both show deterioration.

NICE states:

"If the patient has a worsening in any motor scale score the patient’s next assessments must take place within the next six months. Any patient not complying with the assessment schedule (without good reason) may be deemed as not complying with the terms of the managed access agreement and access to treatment may cease."

However, if you’re maintaining stability or improving on another scale that has been agreed as an important measurement for you, AND in the opinion of the treating clinician you’re continuing to receive clinical benefit from the treatment you are receiving, then the MAA says:

"Continuation of treatment may be considered’ and that ‘These cases should be discussed with the NHS England Clinical Panel."

We suggest you don’t worry about this until or if you get to this point, at which time we would expect you to be having quality discussions with your team about treatment options. In the unlikely event that you don’t feel listened to you might want to get in touch with one of the Patient Groups to talk through possible options open to you.