Autologous haematopoietic stem cell transplantation versus low‐dose immunosuppression in secondary–progressive multiple sclerosis

• DOI: 10.1111/ene.15280
• Publication Date: 2022-02-11T09:08:28Z
• Authors: Alice Mariottini; Giovanni Bulgarini; Benedetta Forci; Chiara Innocenti; Fabrizia Mealli; Alessandra Mattei; Chiara Ceccarelli; Anna Maria Repice; Alessandro Barilaro; Claudia Mechi; Riccardo Saccardi; Luca Massacesi
• Keywords: Immunosuppression

Abstract Background and purpose Effectiveness of autologous haematopoietic stem cell transplantation (AHSCT) in relapsing–remitting multiple sclerosis (MS) is well known, but secondary–progressive (SP)‐MS it still controversial. Therefore, AHSCT activity was evaluated SP‐MS using low‐dose immunosuppression with cyclophosphamide (Cy) as a comparative treatment. Methods In this retrospective monocentric 1:2 matched study, patients were treated intermediate‐intensity (cases) or intravenous pulses Cy (controls) at single academic centre Florence. Controls selected according to baseline characteristics adopting cardinality matching after trimming on the estimated propensity score. Kaplan–Meier Cox analyses used estimate survival free from relapses (R‐FS), disability progression (P‐FS), no evidence disease 2 (NEDA‐2). Results A total 93 included: 31 AHSCT, 62 Cy. Mean follow‐up 99 months group 91 group. R‐FS higher compared patients: Year 5, 100% versus 52%, respectively ( p < 0.0001). P‐FS did not differ between groups (at 5: 70% 81% Cy, = 0.572), nor NEDA‐2 0.379). sensitivity analysis including only “best‐matched” controls confirmed these results. Three neoplasms (2 1 AHSCT) two fatalities Cy) occurred. Conclusions This study provides Class III evidence, SP‐MS, superior effectiveness relapse activity, without differences accrual. Although suppression observed only, show advantages over disability, suggesting that becomes based more noninflammatory neurodegeneration than inflammation.