Clinical experience in aggressive multiple sclerosis treatment with autologous haematopoietic stem cell transplantation
Author(s):
M. Smilowski
,
M. Smilowski
Affiliations:
L. Szczechowski
,
L. Szczechowski
Affiliations:
G. Helbig
,
G. Helbig
Affiliations:
M. Krawczyk-Kulis
,
M. Krawczyk-Kulis
Affiliations:
S. Kyrcz-Krzemien
S. Kyrcz-Krzemien
Affiliations:
ECTRIMS Online Library. Smilowski M. Sep 15, 2016; 147004; 151
Marek Smilowski
Marek Smilowski
Contributions
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Abstract

Abstract: 151

Type: Oral

Abstract Category: Therapy - disease modifying - Immunomodulation/Immunosuppression

Background: Multiple sclerosis (MS) is an inflammatory disease caused by autoimmune reactivity of T-cells towards brain myelin. Intense immunosuppression followed by autologous hematopoietic stem cell transplantation (AHSCT) has been proposed in last years as a treatment option for patients with no response for I- and II- line therapy. A majority of long-term follow-up observations after AHSCT confirmed a great potential and efficacy even for aggressive MS forms which is due to induction of reset of the immune system and elimination of aberrant auto-reactive T-limphocytes.

Objectives: The aim of this study was to evaluate the safety and efficacy of low-intensity immunosuppression followed by an AHSCT for patients with aggressive MS.

Methods: 89 patients with active relapsing-remitting MS (RRMS) and active secondary progressive MS (SPMS) were involved to the study. The qualification criteria were according to the guidelines of the European Group for Blood and Marrow Transplantation (EBMT) 2012. The group consisted of 78 patients with RRMS and 11 patients with SPMS. The stem cells were mobilized with cyclophsosphamide (Cy) and granulocyte-colony stimulating factor (G-CSF). After conditioning with high-dose chemotherapy, the stem cells were re-infused into the patient. The primary endpoint was to assess “No evidence of disease activity” (NEDA) defined as absence of relapses (Relapse free survival - RFS), absence of confirmed disability worsening (Progression Free Survival - PFS), and absence of radiological activity, detected by magnetic resonance (MRI-EFS).

Results: There were 49 female and 40 male in the follow-up group. The mean age was 38 year. The median EDSSA before AHSCT was 5,0 and it decreased to 4,25 after 3 year follow-up of the treatment. The 3 years NEDA was observed in 74% of patients. No mortality was observed. Short term side effects were limited to symptoms due to AHSCT procedures. No long term side effects have been observed so far.

Conclusions: AHSCT should be considered as an option for highly active MS treatment but only if performed according to very restrictive qualification criteria and performed in highly specialized hematological centers.

Disclosure: Lech Szczechowski - nothing to disclose

Marek Smilowski - nothing to disclose

Grzegorz Helbig - nothing to disclose

Małgorzata Krawczyk-Kulis - nothing to disclose

Slawomira Kyrcz-Krzemien - nothing to disclose

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