[pubmed] Patient-acceptable symptom states in myasthenia gravis

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Neurology. 2020 Aug 5:10.1212/WNL.0000000000010574. doi: 10.1212/WNL.0000000000010574. Online ahead of print.

ABSTRACT

OBJECTIVES: To estimate patient-acceptable symptom state (PASS) cut-points for myasthenia gravis (MG) health scales.

METHODS: We conducted an electronic survey that included the Myasthenia Gravis Impairment Index (MGII), EuroQol 5-Dimension (EQ5D) and a simple PASS question. PASS anchored thresholds were estimated for the MGII questionnaire through receiver operating characteristic curves. We used the MGII PASS cut-point in a validation cohort of 257 patients to estimate PASS thresholds for other clinically relevant health scales such as the quantitative myasthenia gravis score (QMGS), MG activities of daily living (MG-ADL), Myasthenia gravis composite (MGC) and myasthenia quality of life (MG-QoL15) scale.

RESULTS: One hundred twenty-four of approximately 250 invited patients answered the electronic survey (49% response rate), and 80 considered their current symptom state acceptable (PASS-positive). These had lower MGII scores than PASS-negative patients (7.76 ± 9.37 vs 25.0 ± 13.7, p < 0.0001) and better EQ5D scores (0.86 ± 0.17 vs 0.69 ± 0.18, p < 0.0001). The MGII questionnaire threshold for PASS was ≤10 points. Using this threshold in an independent dataset of 257 patients, all patients in remission or minimal manifestation status were PASS-positive. In addition, some patients in Classes I, II and IIIA also achieved PASS status. PASS thresholds for the QMGS, MG-ADL, MGC and MG-QoL15 were ≤7, 2, 3 and 8 points, respectively.

CONCLUSIONS: We have estimated thresholds for commonly-used myasthenia health scales reflecting patient-acceptable states in myasthenia patients. These thresholds indicate a global state-"being well" -rather than a change in scores -or "being better." Therefore, PASS thresholds can be used as secondary endpoints for myasthenia research.

PMID:32759200 | DOI:10.1212/WNL.0000000000010574


Source: https://pubmed.ncbi.nlm.nih.gov/3275920 ... 7&v=2.11.4