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Autologous hematopoietic stem cell transplantation in neuromyelitis optica: A registry study of the EBMT Autoimmune Diseases Working Party
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2015 (English)In: Multiple Sclerosis, ISSN 1352-4585, E-ISSN 1477-0970, Vol. 21, no 2, 189-197 p.Article in journal (Refereed) Published
Abstract [en]

BACKGROUND:

Neuromyelitis optica (NMO) is an inflammatory autoimmune disorder of the central nervous system, hallmarked by pathogenic anti-aquaporin 4 antibodies. NMO prognosis is worse compared with multiple sclerosis.

OBJECTIVE:

The European Group for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) conducted a retrospective survey to analyze disease outcome following autologous stem cell transplantation (ASCT).

METHODS:

This retrospective multicenter study assessed the efficacy and safety of ASCT in 16 patients suffering from refractory NMO reported to the EBMT registry between 2001 and 2011.

RESULTS:

Fifteen patients were successfully mobilized with cyclophosphamide (Cy) and G-CSF, one with G-CSF alone. All patients received an unmanipulated autologous peripheral blood stem cell graft, after conditioning with BEAM plus anti-thymocyte globulin (ATG, n = 9 patients), thiotepa-Cy (n = 3) or Cy (200 mg/kg) plus ATG (n = 4). After a median follow-up of 47 months, three of 16 cases were progression and treatment free, while in the remaining 13 patients further treatments were administered for disability progression or relapse after ASCT. Altogether, relapse-free survival at three and five years was 31% and 10%, respectively, while progression-free survival remained 48% at three and five years.

CONCLUSIONS:

In these NMO patients, highly resistant to conventional treatment, ASCT allows for temporary control of the disease, despite a tendency to progress or relapse in the long term.

Place, publisher, year, edition, pages
2015. Vol. 21, no 2, 189-197 p.
National Category
Neurosciences Neurology
Identifiers
URN: urn:nbn:se:uu:diva-233778DOI: 10.1177/1352458514541978ISI: 000349764700012PubMedID: 25078274OAI: oai:DiVA.org:uu-233778DiVA: diva2:754264
Available from: 2014-10-10 Created: 2014-10-10 Last updated: 2017-12-05Bibliographically approved

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Burman, JoachimCarlson, Kristina

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