Bone marrow transplantation is a justifiable treatment for active relapsing multiple sclerosis: for
Author(s):
G. Mancardi
G. Mancardi
Affiliations:
ECTRIMS Online Library. Mancardi G. Sep 15, 2016; 147002; 149
Gian Luigi Mancardi
Gian Luigi Mancardi
Contributions
Abstract

Abstract: 149

Type: Oral

Intense immunosuppression followed by autologous haematopoietic stem cell transplantation (AHSCT) is a possible therapeutic strategy utilized in the last 20 years to treat severe autoimmune disorders unresponsive to traditional therapies. The target of this treatment is the eradication of self reactive abnormal immune system by intense immunosuppression, followed by the infusion of autologous haematopoietic stem cells aimed to restore the haemato-lymphopoietic system which could become more tolerant for a prolonged period of time. In the first studies mainly progressive MS cases with advanced disability were enrolled, the clinical results were not entirely satisfactory and the mortality risk was unacceptably high. The subsequent experience indicated that the patient that can take the largest advantage from the procedure is a MS patient in an earlier phase of the disease, with a high clinical and MR activity, in spite of the approved therapy. More recently, numerous studies demonstrated the profound efficacy of AHSCT, showing that medium intensity conditioning regimens such as BEAM ((carmustine, cytarabine, etoposide and melphalan) plus polyclonal anti-thymocyte gobulin (ATG) are significantly superior to Mitoxantrone in reducing the MR activity (Mancardi et al 2015); and that 5 years after AHSCT, the proportion of aggressive MS patients with sustained clinical remission and without MRI activity is by far higher (Burman et al 2014; Nash et al 2015) than the number of cases obtained with all the present available therapies. Moreover, if a high intensity conditioning regimen with busulfan and both CD34 selection and ATG is utilized, the absence of relapses and MR activity can be observed for an extended period of time (Atkins et al 2016). The mortality risk however still remains, even if it decreased in the last years around 1-2% of treated cases. AHSCT is a therapy reserved to a population of aggressive MS cases refractory to approved therapies and now should move toward a comparative trial that could definitely asses its efficacy.

Disclosure: Dr Mancardi has received honoraria for lecturing, travel expenses for attending meetings and financial support for research from Bayer Schering, Biogen Idec, Genzyme, Merck Serono, Novartis, Sanofi Aventis, Teva Pharmaceuticals

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