Background: Over recent years numerous MS’ers with severe forms of MS refractory to conventional therapies have been treated with intense immunosuppression followed by autologous haematopoietic stem cell transplantation (AHSCT). The clinical outcome and the toxicity of AHSCT can be diverse, depending on the various types of conditioning (chemotherapy) protocols and on the disease phase.
Epub ahead of print: Mancardi et al. Autologous haematopoietic stem cell transplantation with an intermediate intensity conditioning regimen inmultiple sclerosis: the Italian multi-centre experience. Mult Scler. 2011 Nov 29.
Objectives: To report the Italian experience on all the consecutive patients with MS treated with AHSCT with an intermediate intensity conditioning regimen, named BEAM/ATG, in the period from 1996 to 2008.
Methods: Clinical and magnetic resonance imaging outcomes of 74 MS’ers were collected after a median follow-up period of 48.3 (range = 0.8-126) months.
Results: Two MS’ers (2.7%) died from transplant-related causes. After 5 years, 66% of patients remained stable or improved. Among patients with a follow-up longer than 1 year, eight out of 25 subjects with a relapsing-remitting course (31%) had a 6-12 months confirmed EDSS improvement > 1 point after AHSCT as compared with one out of 36 (3%) patients with a SP disease course (p = 0.009). Among the 18 cases with a follow-up longer than 7 years, eight (44%) remained stable or had a sustained improvement while 10 (56%), after an initial period of stabilization or improvement with median duration of 3.5 years, showed a slow disability progression.
Conclusions: This study shows that AHSCT with a BEAM/ATG conditioning regimen has a sustained effect in suppressing disease progression in aggressive MS cases unresponsive to conventional therapies. It can also cause a sustained clinical improvement, especially if treated subjects are still in the relapsing-remitting phase of the disease.
“These results confirm what we know already that in MS’ers with aggressive disease BMT works; however it comes with a very high mortality. The chance of dying from the procedure is in the order of 1 in 40. Are you prepared to take these risks?”
“I personally think other treatment options are safer than this, for example Natalizumab and Alemtuzumab.”