Massimiliano Ugo Verza
Clinical outcome measures in a prospective cohort of myasthenia gravis patients
Autori
- MASSIMILIANO UGO VERZA (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- GREGORIO SPAGNI (DEPARTMENT OF NEUROSCIENZE, UNIVERSITÀ CATTOLICA DEL SACRO CUORE, ROME, ITALY. GERMAN CENTER FOR NEURODEGENERATIVE DISEASES (DZNE) BERLIN; BERLIN, GERMANY – NEUROLOGIA)
- SILVIA FALSO (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- SARA CORNACCHINI (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- ELENA CENCINI (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- LEONARDO PALAZZO (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- ANTONIO FARINA (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- ALICE MARIOTTINI (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- ALESSANDRO BARILARO (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- LUCA MASSACESI (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
- AMELIA EVOLI (DEPARTMENT OF NEUROSCIENZE, UNIVERSITÀ CATTOLICA DEL SACRO CUORE, ROME, ITALY – NEUROLOGIA)
- VALENTINA DAMATO (DEPARTMENT OF NEUROSCIENCES, PSYCHOLOGY, DRUG RESEARCH AND CHILD HEALTH, UNIVERSITY OF FLORENCE, FLORENCE, ITALY – NEUROLOGIA)
Presentatore
MASSIMILIANO UGO VERZA
Modalità
Oral Communication
Abstract
“Introduction
Clinical assessment of myasthenia gravis (MG) relies on validated patient-reported outcome (PRO) and physician reported outcome (PhyRO) measures, coupled with post-intervention status (PIS) or the recently proposed “PASS question”. The aim of this study is to validate the PASS question and assess PIS and PASS cut-off values for PRO and PhyRO scales in a prospective cohort.
Methods
The clinical status of AChR-MG patients was assessed with MG-ADL, MG-QOL15r and QMG scores over a 1 year-period. Favourable outcomes were defined by a positive response to the PASS question and a PIS of minimal manifestation (MM)-or-better. ROC curve analysis was used to determine cut-off values of the QMG, MG-ADL and MG-QOL15r in relation to PASS and PIS.
Results
107 AChR-MG patients were included, of whom 42% females, with a median age at onset of 46 years. Patients with a favourable PASS and in MM-or-better status had a lower median QMG, MG-ADL, MG-QOL15 scores than symptomatic patients (p<0.0001). A QMG≤ 9, MG-ADL≤ 2 and MG-QOL15r≤ 6 identified patients with a favourable PASS status. Same cut-offs for the QMG and MG-ADL scores were found to identify patients in MM-or-better, while the cut-off for the MG-QOL15r was much lower (≤4). In the multivariable logistic regression both MG-ADL
(p=0.025) and MG-QOL15r (p=0.043) were independently associated with “PASS=yes”, while QMG score was not
Conclusion
PASS and PIS provide complementary information on the MG clinical status. The PASS cut-off values found for the most common MG scales support the use of the PASS question in clinical practice and clinical trials.”