Autologous hematopoietic stem cell transplantation in combination with immunoablative protocol in secondary progressive multiple sclerosis: A 10-year follow-up of the first transplanted patient
Introduction. Multiple sclerosis (MS) is an immunemediated disease of the central nervous system that affects young individuals and leads to severe disability. High dose immunoablation followed by autologous hemopoietic stem cell transplantation (AHSCT) has been considered in the last 15 ye...
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| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Ministry of Defence of the Republic of Serbia, University of Defence, Belgrade
2016-01-01
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| Series: | Vojnosanitetski Pregled |
| Subjects: | |
| Online Access: | http://www.doiserbia.nb.rs/img/doi/0042-8450/2016/0042-84501500045O.pdf |
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| Summary: | Introduction. Multiple sclerosis (MS) is an immunemediated disease of the
central nervous system that affects young individuals and leads to severe
disability. High dose immunoablation followed by autologous hemopoietic stem
cell transplantation (AHSCT) has been considered in the last 15 years as
potentialy effective therapeutic approach for agressive MS. The most recent
long-time follow-up results suggest that AHSCT is not only effective for
highly aggressive MS, but for relapsing-remitting MS as well, providing
long-term remission, or maybe even cure. We presented a 10- year follow-up of
the first MS patient being treated by immunoablation therapy and AHSCT. Case
report. A 27-year-old male experienced the first symptoms - intermitent
numbness and paresthesia of arms and legs of what was treated for two years
by psychiatrist as anxiety disorder. After he developed severe paraparesis he
was admitted to the Neurology Clinic and diagnosed with MS. Our patient
developed aggressive MS with frequent relapses, rapid disability progression
and transition to secondary progressive form 6 years after MS onset [the
Expanded Disability Status Scale (EDSS) 7.0 Ambulation Index (AI) 7]. AHSCT
was performed, cyclophosphamide was used for hemopoietic stem cell
mobilization and the BEAM protocol was used as conditionig regimen. No major
adverse events followed the AHSCT. Neurological impairment improved, EDSS
6.5, AI 6 and during a 10-year followup remained unchanged. Brain MRI
follow-up showed the absence of gadolinium enhancing lesions and a mild
progression of brain atrophy. Conclusion. The patient with rapidly evolving,
aggressive, noninflammatory MS initialy improved and remained stable, without
disability progression for 10 years, after AHSCT. This kind of treatment
should be considered in aggressive MS, or in disease modifying treatment
nonresponsive MS patients, since appropriately timed AHSCT treatment may not
only prevent disability progression but reduce the achieved level of
disability, as well. |
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| ISSN: | 0042-8450 2406-0720 |