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Benefits of aHSCT over alemtuzumab in patients with multiple sclerosis besides disability and relapses: Sustained improvement in cognition and quality of life.
Braun, Bente; Fischbach, Felix; Richter, Johanna; Pfeffer, Lena Kristina; Fay, Heike; Reinhardt, Stefanie; Friese, Manuel A; Stellmann, Jan-P; Kröger, Nicolaus M; Heesen, Christoph; Häußler, Vivien.
Afiliación
  • Braun B; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Fischbach F; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Richter J; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Pfeffer LK; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Fay H; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Reinhardt S; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Friese MA; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Stellmann JP; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany; APHM, Hopital de la Timone, CEMEREM, Marseille, Fra
  • Kröger NM; Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Heesen C; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany.
  • Häußler V; Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg 20251, Germany; Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg 20246, Germany. Electronic address: v.haeussler@uke.de.
Mult Scler Relat Disord ; 82: 105414, 2024 Feb.
Article en En | MEDLINE | ID: mdl-38176284
ABSTRACT

BACKGROUND:

Autologous hematopoietic stem cell transplantation (aHSCT) exhibits promising results for multiple sclerosis (MS) in the short term. We investigated the long-term outcome differences in disease progression and cognitive impairment after aHSCT and alemtuzumab treatment.

METHODS:

20 patients receiving aHSCT and 21 patients treated with alemtuzumab between 2007 and 2020 were included in this monocentric observational cohort study. The primary objective was to compare the outcome of both groups with regards to achieving No Evidence of Disease Activity (NEDA-3), defined by the absence of relapses, EDSS progression, and MRI activity. Secondary endpoints in the study included the assessment of neurocognitive functioning, quality of life (QoL), Multiple Sclerosis Functional Composite (MSFC), and EDSS improvement.

RESULTS:

Baseline characteristics between both groups were comparable, except for a longer disease duration in the alemtuzumab group of 11.3 years compared to 5.4 years in aHSCT-treated patients (p = 0.002) and a longer mean follow-up time in the aHSCT cohort of 9.0 (range 2.8-15.7) years compared to 5.9 years (range 0.9-9.2) in alemtuzumab patients. NEDA-3 was more frequently observed in the aHSCT group with 75.0 % and 55.0 % at five and 10 years, respectively, than in the alemtuzumab group with only 40.0 % at five years (p = 0.012). Relapse free survival was higher in the aHSCT group (p < 0.001). None of the aHSCT-treated patients showed new T2-lesions six months after therapy initiation until the end of the observational period in contrast to 35.0 % of the alemtuzumab-treated patients showing new T2-lesions (95 %CI 14.2-98.9, p = 0.002). aHSCT-treated patients showed significantly improved cognitive performance in five out of 12 cognitive tests whereas alemtuzumab treated patients deteriorated in four out of 12 tests. Quality of life remained on a constant level for up to 10 years in patients receiving aHSCT with improved scores for the subscale fatigue (p = 0.013).

CONCLUSION:

aHSCT seems to be superior to alemtuzumab in maintaining long-term NEDA-3 status, improving cognition and stabilizing quality of life for up to 10 years.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Esclerosis Múltiple Recurrente-Remitente / Esclerosis Múltiple Tipo de estudio: Observational_studies Aspecto: Patient_preference Límite: Humans Idioma: En Revista: Mult Scler Relat Disord Año: 2024 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Países Bajos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Esclerosis Múltiple Recurrente-Remitente / Esclerosis Múltiple Tipo de estudio: Observational_studies Aspecto: Patient_preference Límite: Humans Idioma: En Revista: Mult Scler Relat Disord Año: 2024 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Países Bajos