STUDY: Long-term results from Italian study, autologous HSCT for MS

[ActiveMSers]

Just released Italian study. This is the treatment I received--BEAM with ATG. The abstract doesn't differentiate between long-term success in RRMS and SPMS, but I would imagine most of the post 7 year results are SPMS since this type of therapy was not used as much for RRMS until the mid 2000s.

Sustained gains are quite limited in SPMSers (3%) whereas about a third of RRMSers had sustained gains. This is a reminder that this treatment won't significantly reverse disease damage in most cases. The more impressive stat is that 2/3rds of patients (both RRMS and SPMS) had stability or gains after 5 years. This rate is likely a good deal higher for RRMS patients only. For folks with aggressive MS, that's eye-opening potency and potentially life saving. - Dave

Autologous haematopoietic stem cell transplantation with an intermediate intensity conditioning regimen in multiple sclerosis: the Italian multi-centre experience

Background: Over recent years numerous patients with severe forms of multiple sclerosis (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 protocols and on the disease phase.

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 patients were collected after a median follow-up period of 48.3 (range = 0.8–126) months.

Results: Two patients (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 Expanded Disability Status Scale improvement > 1 point after AHSCT as compared with one out of 36 (3%) patients with a secondary progressive 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.

[ActiveMSers]

Dr. Burt of Northwestern found some issues with this study that are not apparent when reading the abstract. He feels patient selection is critical and that the Italians may have had more SPMS patients than actually reported, skewing the success of the treatment with RRMSers. - Dave

Mancardi et al.’s multi-center Italian study reported some patients recorded as having relapsing–remitting disease who had a disease duration of up to 18 years, were up to 52 years old, and or had an EDSS as high as 7.5. This emphasizes the importance of auditing patients enrolled at each site to ensure appropriate selection and universal understanding of the difference between inflammatory versus progressive MS. Patient selection is essential for good outcome following HSCT for MS since ‘no inflammation, no response’.