Low intensity lympho-ablative regimen followed by autologous hematopoietic stem cell transplantation in severe forms of multiple sclerosis: A MRI-based clinical study

Daniela Curro, Luisa Vuolo, Francesca Gualandi, Andrea Bacigalupo, Luca Roccatagliata, Elisabetta Capello, Antonio Uccelli, Riccardo Saccardi, Maria Pia Sormani, Gianluigi Mancardi

Research output: Contribution to journalArticlepeer-review

Abstract

Background: Autologous hematopoietic stem cell transplantation (AHSCT) has been successfully used to treat aggressive forms of multiple sclerosis (MS) that are unresponsive to approved therapies. In the last years, in view of the risk of mortality related to the procedure, the utilization of low-intensity conditioning regimens has been considered. Objective: To report magnetic resonance imaging (MRI) and clinical data in a small cohort of patients treated with a low-intensity lympho-ablative regimen, followed by AHSCT. Methods: Seven patients affected by relapsing-remitting MS (RRMS) underwent AHSCT, with cyclo-phosphamide 120 mg/kg in 2 days as the conditioning regimen; and were then followed with serial MRI evaluations until 36 months, with clinical evaluations until 60 months. Results: The mean number of gadolinium (Gd)-enhancing lesions significantly decreased after treatment, but a complete suppression of inflammatory activity was not obtained. No deaths occurred, but every patient developed adverse events, although not severe. After 5 years of follow-up, two patients remained stable, one patient markedly improved and four patients had a mild progression of the disease. Only one patient experienced a relapse after treatment. Conclusion: A low-intensity conditioning regimen with AHSCT has a profound effect on MRI inflammation and relapses, but is not able to completely abrogate MRI activity and disease progression of aggressive RRMS.

Original languageEnglish
Pages (from-to)1423-1430
Number of pages8
JournalMultiple Sclerosis Journal
Volume21
Issue number11
DOIs
Publication statusPublished - 2015

Keywords

  • Aggressive multiple sclerosis
  • Autologous hematopoietic stem cell transplantation
  • Conditioning regimen
  • Lympho-ablation
  • Multiple sclerosis
  • Relapsing-remitting multiple sclerosis
  • Transplantation
  • Treatment methods

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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