Multiple Sclerosis
Multiple sclerosis (MS) is a chronic, autoimmune, degenerative disease affecting nearly 1 million adults in the United States. [1] Most patients present with symptoms between 20 and 40 years of age but have varying rates of disability impairment. [2]
There is currently no cure for MS. However, research has shown that autologous hematopoietic cell transplantation (HCT) is an efficient and safe treatment for active relapsing forms of MS to help prevent relapse, lesion-activity, and worsening disability while limiting unexpected adverse events. [2] In addition, patients with aggressive forms of MS may particularly benefit from transplant accompanied by high-dose immunotherapy. [3,4] Together, these treatments suppress the autoimmune inflammatory response and promote immune recovery.
The American Society for Transplantation and Cellular Therapy (ASTCT) has recommended autologous transplant be considered for “standard of care, clinical evidence available” treatment of refractory-relapsing MS. [2]
Advances
Recent research shows promising treatment options and provides recommendations for clinical decision-making for patients with aggressive forms of MS, including:
Outcomes
Data in this section was published by Muraro et al. using data from CIBMTR. [3]
Figure 1. Multiple Sclerosis Progression-Free Survival after Autologous HCT, 1995 to 2006
Figure 2. Overall Survival after Autologous HCT, 1995 to 2006
Referral Timing Guidelines
These guidelines highlight disease categories that include patients at risk for disease progression and who should be referred for a consultation for autologous or allogeneic transplantation.
Transplant Consultation Guidelines: Multiple Sclerosis
- After MS relapse, with ≥ 2 relapse episodes in past 3 years, while on disease modifying therapy
- Refer patient prior to progression of severe disability: patient must be able to walk 100 meters (with unilateral assistance: cane, crutch or brace)
View complete Referral Timing Guidelines
References
- National Multiple Sclerosis Society. Landmark Study Estimates Nearly 1 Million in the U.S. Have Multiple Sclerosis. February 15, 2019. Accessed June 9, 2022. https://www.nationalmssociety.org/About-the-Society/News/Landmark-Study-Estimates-Nearly%C2%A01-Million-in-the-U
- Cohen JA, Baldassari LE, Atkins HL, et al. Autologous Hematopoietic Cell Transplantation for Treatment-Refractory Relapsing Multiple Sclerosis: Position Statement from the American Society for Blood and Marrow Transplantation. Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation. 2019;25(5):845-854. doi:10.1016/j.bbmt.2019.02.014.
- Muraro PA, Pasquini M, Atkins HL, et al. Long-term Outcomes After Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis. JAMA neurology. 2017;74(4):459-469. doi:10.1001/jamaneurol.2016.5867.
- Nash RA, Hutton GJ, Racke MK, et al. High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS. Neurology. 2017;88(9):842-852. doi:10.1212/WNL.0000000000003660.
- Sharrack B, Saccardi R, Alexander T, et al. Autologous hematopoietic 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). Bone marrow transplantation. 2020;55(2):283-306. doi:10.1038/s41409-019-0684-0.
- Miller AE, Chitnis T, Cohen BA, Costello K, Sicotte NL, Stacom R. Autologous Hematopoietic Stem Cell Transplant in Multiple Sclerosis: Recommendations of the National Multiple Sclerosis Society. JAMA neurology. 2021;78(2):241-246. doi:10.1001/jamaneurol.2020.4025.