High-dose chemotherapy with autologous hematopoietic stem cell transplantation in multiple sclerosis: intermediate results of 3 years research

Relevance. Multiple sclerosis (MS) is a chronic demyelinating autoimmune disease of the central nervous system with progressive course. High-dose chemotherapy (HDCT) with autologous haematopoietic stem cell transplantation (AHSCT) is a promising course of treatment-refractory MS. The study aimed to assess the effi cacy and safety of treating MS patients with HDCT + AHSCT.Material and methods. Were studied 10 people (5 women and 5 men) with relapsing-remitting (30%), secondary progressive (60%) and primary progressive MS (10%). Patients underwent AHSCT using cyclophosphamide + rituximab chemotherapeutic regimen.Results. In 90% of cases, at least a stabilizing eff ect is shown after the procedure. Only 10% was showed signs of demyelinating activity on MRI of the spinal cord 2 years after HDCT + AHSCT. All patients had a response to therapy according to median Expanded Disability Status Scores (EDSS). We found a positive dynamics of the average EDSS score: before treatment — 5.9 points, 6 months and 1 year after treatment — 5.1 points. There was shown a positive eff ect on dynamics of neurological status, quality of life and emotional state of patients. No treatment-related mortality and uncontrolled complications were revealed.Conclusion. Timely AHSCT contributes to minimizing the level of disability and signifi cantly improves the quality of life of patients.

[1]  I. Helenowski,et al.  Real-world application of autologous hematopoietic stem cell transplantation in 507 patients with multiple sclerosis , 2021, Journal of Neurology.

[2]  M. Sormani,et al.  Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE) , 2019, Bone Marrow Transplantation.

[3]  Jeffrey A. Cohen,et al.  Autologous Hematopoietic Cell Transplantation for Treatment-Refractory Relapsing Multiple Sclerosis: Position Statement from the American Society for Blood and Marrow Transplantation. , 2019, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[4]  B. Sharrack,et al.  Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial , 2019, JAMA.

[5]  R. Zivadinov,et al.  Prospective phase II clinical trial of autologous haematopoietic stem cell transplant for treatment refractory multiple sclerosis , 2018, Journal of Neurology, Neurosurgery, and Psychiatry.

[6]  M. Sormani,et al.  Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis , 2017, Nature Reviews Neurology.

[7]  P. Manganotti,et al.  How far away from having an effective treatment option for progressive multiple sclerosis are we? , 2017, Expert opinion on pharmacotherapy.

[8]  H. Atkins,et al.  Long-term Outcomes After Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis , 2017, JAMA neurology.

[9]  D. Arnold,et al.  High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for relapsing-remitting multiple sclerosis (HALT-MS): a 3-year interim report. , 2015, JAMA neurology.