To assess HRQL and health state values in a sample of patients with different levels of severity of ALS
2 outpatient clinics
N = 77
Age 58.1 ± 12.1 (range 27–79), 36% female, time since diagnosis 25.3 months ±22.6 (range 1–112), ALSFRS-R score not presented
Severity classified according to the ALS/HSS: level 1 mild (n = 15, 20%), level 2 moderate (n = 21, 27%), level 3 severe (n = 22, 29%), level 4 terminal (n = 19, 25%)
EQ-5D-3L, EQVAS
EQ-5D score not presented for total sample, EQVAS 0.55 ± 0.22 (median 0.5)
The percentage of total sample who endorsed the worst response options of the EQ-5D increased with ALS severity across all dimensions; mean EQVAS scores decreased with increasing ALS severity from 0.74 to 0.37
Not available
54–80% of individuals with ALS reported moderate/severe problems in 4/5 EQ-5D dimensions (exception of 27% of people for anxiety/depression)
Not assessed: primary objective was not to assess psychometric properties of generic preference-based measure
To determine the concurrent validity of three self-administered HRQL questionnaires in patients with ALS
Outpatient clinic
N = 19
Age 60.5 (range 36–76), 42% female, time since diagnosis 20.5 months (range 2–62), ALSFRS-R score not presented, FVC 64% (range 17%–91%)
ALS severity not presented, diagnosis classified according to the El Escorial criteria: definite (n = 9, 47%), probable (n = 4, 21%), possible (n = 5, 26%), suspected (n = 1, 5%)
QWB-SA
QWB-SA 0.43 (range 0-1)
Not available
QWBa: +SIP/ALS (r = 0.55), +SF-36 (r = 0.21)
Not available
Methodological qualityh: adequate Ratingi: sufficient (inconsistent based on majority) grading of quality of evidencej: very low
HRQL: health-related quality of life, EQ-5D: EuroQol Five Dimension, EQVAS: EuroQol Visual Analogue Scale, ALSFRS-R: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised, BDI: Beck Depression Inventory, ALSSS: Amyotrophic Lateral Sclerosis Severity Scale, MG: myasthenia gravis, FSHD: facioscapulohumeral muscular dystrophy, MND: motor neuron disease; ALSAQ-40 :ALS Assessment Questionnaire 40 subscales; ALSPM: physical mobility, ALSADL/I: activities of daily living/independence; ALSED: eating and drinking; ALSCOM: communication; ALSER: emotional reactions, ALS/HSS: ALS Health State Scale; FVC: forced vital capacity, QWB-SA: Quality of Well-Being Self-Administered Scale, SF-36: Short Form 36, SIP/ALS-19: Sickness Impact Profile ALS-19. Range of health utility scores from −0.306–0.885, with higher scores representing better health [36]. aSpearman’s rank correlation coefficient. bMeasures social support. cProposed clinical stages developed by Jones et al. estimated using ALSFRS-R scores and modified King’s ALS staging system to indicate ALS severity. dHigh severity: patients needed caregiver’s assistance. eLow severity: patients did not need caregiver’s assistance. fPearson’s product-moment correlation coefficient. gCorrelation is significant at the 0.01 level (two-tailed). hDetermined using COSMIN’s risk of bias checklist27. iResults rated against COSMIN’s criteria for good measurement properties26: 50% of correlations (QWB: +SIP/ALS (r = 0.55), +SF-36 (r = 0.21)) in accordance with hypotheses, results rated as sufficient with an inconsistent rating from the majority of results. jDetermined using the GRADE approach. More details are described in detail in the COSMIN manual26.