Author Of 2 Presentations
P0301 - Beyond pivotal trials inclusion criteria: real world clinical profile of multiple sclerosis patients under disease modifying treatment in Argentina. (ID 851)
- A. Barboza
- J. Rojas
- M. Gaitán
- S. Liwacki
- C. Vrech
- A. Papolla
- J. Miguez
- L. Patrucco
- J. Correale
- M. Marrodan
- M. Fiol
- L. Negrotto
- C. Ysrraelit
- E. Cristiano
- A. Carrá
- A. Chertcoff
- J. Steinberg
- A. Martinez
- C. Curbelo
- L. Cohen
- R. Alonso
- O. Garcea
- C. Pita
- B. Silva
- G. Luetic
- N. Deri
- M. Balbuena
- V. Tkachuk
- E. Carnero Contentti
- P. Lopez
- J. Pettinicchi
- A. Caride
- M. Burgos
- F. Leguizamon
- E. Knorre
- R. Piedrabuena
- P. Nofal
- G. Volman
- A. Alvez Pinheiro
- J. Hryb
- D. Tavolini
- P. Blaya
- L. Recchia
- C. Mainella
- M. Kohler
- E. Kohler
- J. Blanche
- S. Tizio
- M. Saladino
- F. Caceres
- N. Fernandez Liguori
- L. Lazaro
- E. Silva
- G. Zanga
- M. Parada Marcilla
- M. Fracaro
- F. Pagani Cassará
- G. Vazquez
- V. Sinay
- G. Sgrilli
- P. Divi
- M. Jacobo
- E. Reich
- L. Cabrera
- M. Menichini
- M. Coppola
- I. Martos
- J. Viglione
- G. Jose
- S. Bestoso
- R. Manzi
- D. Giunta
- M. Doldan
- M. Alonso Serena
Abstract
Background
Background: In multiple sclerosis (MS), randomized controlled trials (RCT) have provided relevant information about the efficacy and safety in ideal scenarios. While RCT are powerful tools for developing scientific evidence based on their high internal validity, there is always uncertainty about the generalizability, especially since the populations enrolled in such studies may differ in significant ways from those seen in clinical practice.
Objectives
Objective: to describe the frequency and clinical profile of MS patients under disease modifying treatment in Argentina that would have not fulfilled inclusion criteria in RCT.
Methods
Methods: MS patients included in the Argentinean MS and NMOSD registry (RelevarEM, NCT 03375177) were analyzed. RelevarEM is a longitudinal, strictly observational MS and NMOSD registry in Argentina. From May 2018 to March 2020, the centers and principal investigators were contacted and incorporated into the Registry. All patients with definite MS and receiving DMT at 31 December 2019 were screened, those with EDSS >6, phenotypes secondary progressive (SP) and primary progressive (PP)(with other DMT than ocrelizumab) and age <18 and >55 years old were included in the analysis.
Results
Results: A total of 1782 patients with MS receiving DMT were screened, of whom 465 (26%)would not have been included in a pivotal trial. From the 465,218 had and EDSS >6, 67 had phenotype SP and 19 PP; 292 were patients with <18 and >55 years of age (2 under 18 years old). Most prescribed DMT among patients with EDSS >6 was fingolimod (31%), among age >55 was beta interferon (35%), phenotype SP fingolimod (30%) and PP fingolimod and glatiramer acetate (each 26%).
Conclusions
Conclusion: in our registry, we found a significant number of MS patients who would have not been included in pivotal trials, receiving DMT. Real life evidence is highly relevant to assess effectiveness as well as safety of DMT in this subset of patients.
P0648 - The central vein sign as a biomarker for MS in the infratentorial brain (ID 920)
Abstract
Background
The central vein sign (CVS) is a novel imaging biomarker for the differential diagnosis of multiple sclerosis (MS). In studies at 7.0 tesla MRI, the percentage of supratentorial white matter lesions (WMLs) with CVS vary between 80% to 100% in MS patient brains. Similar results were reported at 3.0 tesla (3T) MRI in optimized sequences, such as T2*-weighted 3D echo-planar-imaging sequence and SWAN-venule. The value of the CVS for infratentorial brain remains unknown.
Objectives
The aim of this study was to determine the proportion of WMLs positive for the CVS in the brainstem and cerebellum of MS patients.
Methods
We included subjects with clinically defined MS, that showed at least one infratentorial lesion larger than 3 mm in 3D-FLAIR. Patients were scanned in a 3T MRI system (GE Medical Systems, discovery MR750) using a 32-channel coil array. MRI included 3D T2-weighted FLAIR and post-contrast SWAN-venule: [FOV = 22 cm x 16 cm; number of slices= 126; voxel resolution, 0.4 mm x 0.4 mm x 0.8 mm; TR = 47 msec; TE = 28 msec; flip angle (FA) = 8 degrees; ETL = 9; AT = 7.38 min]. The CVS, defined as a thin hypointense line or a hypointense small dot visualized in two planes, was recorded on SWAN-venule by two trained raters.
Results
Thirty MRIs were analyzed, three were excluded for motion artefacts. A total of 91 focal lesions were detected in FLAIR, 22 in the cerebellum and 69 in brainstem. Out of 91 lesions, 83 (91%) were visible in SWAN-venule and 83% were positive for the CVS.
Conclusions
The infratentorial brain is a cardinal compartment for MS diagnosis, SWAN-venule detects infratentorial WMLs and highlights the CVS in most plaques at 3.0 T MRI. The CVS could be used to discriminate MS lesions from its radiological infratentorial mimics.