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Comparative Effectiveness in Multiple Sclerosis: A Methodological Comparison Publisher Pubmed



Roos I1, 96 ; Diouf I1 ; Sharmin S1 ; Horakova D2 ; Havrdova EK2 ; Patti F3, 100 ; Shaygannejad V4 ; Ozakbas S5 ; Izquierdo G8 ; Eichau S8 ; Onofrj M9 ; Lugaresi A12 ; Alroughani R15 ; Prat A16 Show All Authors
Authors
  1. Roos I1, 96
  2. Diouf I1
  3. Sharmin S1
  4. Horakova D2
  5. Havrdova EK2
  6. Patti F3, 100
  7. Shaygannejad V4
  8. Ozakbas S5
  9. Izquierdo G8
  10. Eichau S8
  11. Onofrj M9
  12. Lugaresi A12
  13. Alroughani R15
  14. Prat A16
  15. Girard M16
  16. Duquette P16
  17. Terzi M17
  18. Boz C18
  19. Grandmaison F19
  20. Sola P20
  21. Ferraro D20
  22. Grammond P21
  23. Turkoglu R24
  24. Buzzard K25, 98, 99
  25. Skibina O25, 90
  26. Yamou B26
  27. Altintas A27
  28. Gerlach O28, 87
  29. Van Pesch V29, 101
  30. Blanco Y30
  31. Maimone D31
  32. Lechnerscott J32, 95
  33. Bergamaschi R33
  34. Karabudak R34
  35. Mcguigan C35
  36. Cartechini E38
  37. Barnett M41
  38. Hughes S42
  39. Sa MJ43
  40. Solaro C44, 97
  41. Ramotello C45
  42. Hodgkinson S46
  43. Spitaleri D47
  44. Soysal A48
  45. Petersen T49
  46. Granella F50, 104
  47. De Gans K51
  48. Mccombe P52, 92
  49. Ampapa R53
  50. Van Wijmeersch B54
  51. Van Der Walt A57, 89
  52. Butzkueven H57, 89
  53. Prevost J58
  54. Sanchezmenoyo JL63
  55. Laureys G64
  56. Gouider R65
  57. Castillotrivino T66
  58. Gray O69
  59. Agueramorales E70
  60. Alasmi A71
  61. Shaw C72
  62. Deri N73
  63. Alharbi T74
  64. Fragoso Y75
  65. Csepany T76
  66. Sempere AP77
  67. Trevinofrenk I78
  68. Schepel J85
  69. Moore F86
  70. Malpas C1, 96
  71. Kalincik T1, 96

Source: Multiple Sclerosis Journal Published:2023


Abstract

Background: In the absence of evidence from randomised controlled trials, observational data can be used to emulate clinical trials and guide clinical decisions. Observational studies are, however, susceptible to confounding and bias. Among the used techniques to reduce indication bias are propensity score matching and marginal structural models. Objective: To use the comparative effectiveness of fingolimod vs natalizumab to compare the results obtained with propensity score matching and marginal structural models. Methods: Patients with clinically isolated syndrome or relapsing remitting MS who were treated with either fingolimod or natalizumab were identified in the MSBase registry. Patients were propensity score matched, and inverse probability of treatment weighted at six monthly intervals, using the following variables: age, sex, disability, MS duration, MS course, prior relapses, and prior therapies. Studied outcomes were cumulative hazard of relapse, disability accumulation, and disability improvement. Results: 4608 patients (1659 natalizumab, 2949 fingolimod) fulfilled inclusion criteria, and were propensity score matched or repeatedly reweighed with marginal structural models. Natalizumab treatment was associated with a lower probability of relapse (PS matching: HR 0.67 [95% CI 0.62-0.80]; marginal structural model: 0.71 [0.62-0.80]), and higher probability of disability improvement (PS matching: 1.21 [1.02 -1.43]; marginal structural model 1.43 1.19 -1.72]). There was no evidence of a difference in the magnitude of effect between the two methods. Conclusions: The relative effectiveness of two therapies can be efficiently compared by either marginal structural models or propensity score matching when applied in clearly defined clinical contexts and in sufficiently powered cohorts. © The Author(s), 2023.
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