According to a recent study, published at the 2018 AAN Annual Meeting in Los Angeles, America, autologous haematopoietic stem cell transplantation (AHSCT) is ‘safe and highly effective’ in patients with ‘aggressive’ relapsing remitting multiple sclerosis (RRMS).
The study accessed the safety and efficacy of AHSCT in 19 patients with aggressive RRMS, who had fail to respond to standard disease modifying therapies (DMTs). The patients were from across the world: seven from Sheffield (UK), seven from Uppsala (Sweden), four from Ottawa (Canada) and one from Florence (Italy). All patients received treatment between May 2004 and May 2017.
BEAM, a high-dose chemotherapy treatment that uses carmustine, etoposide, cytarabine, melphalan with antithymocyte globulin (ATG), cyclophosphamide with ATG or combination of cyclophosphamide, ATG and busulfan were used as conditioning regimens.
The median age of diagnosis was 33 years, with a median of nine months between symptom onset and AHSCT. The median Expanded Disability Status Scale (EDSS) score before treatment was 6.5.
After AHSCT treatment the EDSS score at the last follow up was 2.0 lower, showing an improvement in disability. In addition, none of these patients had any clinical relapse following treatment.
Three patients had new T2 lesions with or without gadolinium enhancement at the first six month follow up MRI, but no further new or enhancing lesions were observed in any subsequent scans. No deaths were associated with this treatment.
As a result of these findings, researchers concluded that AHSCT is ‘safe and highly effective in inducing rapid and sustain remission in this cohort and was associated with a significant improvement in their level of disability.’ They also suggested that AHSCT should be considered as a first line therapy in patients with aggressive MS.