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Table 1 Secondary outcomes and their derivation

From: Statistical analysis plan for the motor neuron disease systematic multi-arm adaptive randomised trial (MND-SMART)

Secondary outcome

Derivation

Cognitive function and behaviour assessed by the Edinburgh Cognitive and Behavioural ALS Screen (ECAS)

Cognitive function and behaviour will be calculated by calculating the sum total score of the 16 tasks on the ECAS. The total score will be a number from 0 to 136. The higher the total score the better the cognitive function. If any of the individual tasks are missing then so will be the total score. Domain totals will also be calculated. Carer score will be separately calculated

Anxiety and depression measured by the Hospital Anxiety and Depression Scale (including anxiety and depression sub-scales as well as overall score)

The Hospital Anxiety and Depression Scale will be calculated by summing all 14 individual items. The higher the score, the worse the anxiety and depression. Anxiety and depression subscales will also be calculated. If there are any missing individual items the subscales and total score will still be calculated provided that at least 4 items (> 50%) are recorded in each sub-domain

Respiratory function measured by forced vital capacity (FVC)

A mean value of percentage predicted FVC will be calculated

Quality of life evaluation—EQ-5D-5L index and EQ-5D visual analogue scale (EQ-5D VAS)

EQ-5D VAS requires no derivation. The EQ-5D-5L index will be calculated using the cross-walk algorithm based on EQ-5D-3L. [14, 15] A secondary analysis will use the EQ-5D-5L value set for England [16, 17]

Time from randomisation to King’s stage 4A

Days from date of randomisation to date of first rating of the patient as King’s stage 4A

Time from randomisation to King’s stage 4B

Days from date of randomisation to date of first rating of the patient as King’s stage 4B