Table 1 Summary of HRQoL and work productivity measures completed by patients and caregivers in the survey

Adult patients

 EQ-5D-5L

Generic (HRQoL)

Previously used in NF1 [12]

0–1; higher score indicates better HRQoL

 PROMIS® GH

Generic (HRQoL)

Previously used in NF1 PN [11]

0–100; higher score indicates better HRQoL

 INF1-QOL

NF1-specific (HRQoL)

Validated for assessment of HRQoL in patients with NF1, with significant bivariate correlations reported with EQ-5D and physician rated clinical severity scores [12]

0–42; higher score indicates worse HRQoL

Caregivers of paediatric patients

 EQ-5D-5L

Generic (HRQoL)

Previously used to measure caregiver HRQoL [17, 18]

0–1; higher score indicates better HRQoL

 CarerQoL

Caregiver-specific (care-related Qol)

Used in informal care research and economic evaluation; validated for assessment of care-related QoL in informal caregivers, with significant multivariate correlations identified with other measures such as EQ-5D [19,20,21,22]

0–100; higher score indicates better care-related QoL

 WPAI:CG

Caregiver-specific (work productivity)

Validated for assessment of productivity loss in caregivers through evidence of strong, significant correlations with existing measures [23, 24]

0%–100%; higher score indicates greater impairment to work productivity