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Experimental and clinical observations have indicated that high-dose immunosuppression followed by autologous stem cell transplantation (ASCT) or allogeneic transplntation can induce remissions in severe, refractory, autoimmune diseases including multiple sclerosis (MS). Control of the disease is unsatisfactory in most of the patients, especially those with rapidly evolving relapsing–remitting course, and those with chronic progressive disease. A recent review writes: “it is not a treatment for the general population of MS patients but only for selected cases that do not respond to standard therapies and worsen rapidly, i.e. in situations where benefits are expected to counterbalance morbidity and mortality risks

More recent evidence further supports that patients need to be carefully selected. An observational report from Joachim Burman, M.D., a neurologist at Uppsala University Hospital, and colleagues from the major hospitals in Sweden just published in the Journal of Neurology, Neurosurgery and Psychiatry evaluated safety and adverse effects among 48 patients who received transplants from May 2004 through April 2013 (Burman et al., 2014).
Outcomes at the 5-year mark were encouraging and consistent with those of other studies:
87% relapse-free survival.
85% survival without new lesions on MRI.
77% progression-free on the EDSS.
68% disease-free survival (no relapses, no new MRI lesions, no EDSS progression).
None of the patients died.

The consensus on HSCT in autoimmune diseases provides an indication of HSCT in patients with progressive MS unresponsive to conventional therapy and EDSS between 3.0 and 6.0. The forms of the disease that might benefit from transplantation are: relapsing-remitting, primary or secondary progressive, and the “malignant” form, provided there is evidence of inflammatory activity at the time of transplant indication.

Considering the lack of options for this patient, an autologous stem cell transplants should be considered medially necessary.

References:

Source Reference: Burt RK, et al “Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis” JAMA 2019

Atkins H “Stem cell transplantation to treat multiple sclerosis” JAMA 2019

Athanasios Fassas, Gian Luigi Mancardi, Autologous hemopoietic stem cell transplantation for multiple sclerosis: Is it worthwile? Autoimmunity, Volume 41, Issue 8 December 2008 , pages 601 – 610

Gianluigi Mancardi MD, Riccardo Saccardi Autologous haematopoietic stem-cell transplantation in multiple sclerosis The Lancet Neurology, Volume 7, Issue 7, Pages 626 -636, July 2008

Pasquini MC, Griffith LM, Arnold DL, Atkins HL, Bowen JD, Chen JT, Freedman MS, Kraft GH, Mancardi GL, Martin R, Muraro PA, Nash RA, Racke MK, Storek J, Saccardi R. Hematopoietic stem cell transplantation for multiple sclerosis: collaboration of the CIBMTR and EBMT to facilitate international clinical studies. Biol Blood Marrow Transplant. 2010 Aug;16(8):1076-83.

Burman J et al, Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience. J Neurol Neurosurg Psychiatry. 2014 Oct;85(10):1116-21. Stangel M, Multiple sclerosis: Hematopoietic stem cell transplantation: hope and hype. Nat Rev Neurol. 2009 Jun;5(6):300-2.

Maria Carolina de Oliveira Rodrigues, Guidelines of the Brazilian society of bone Marrow transplantation on hematopoietic stem cell transplantation as a treatment for the autoimmune diseases systemic sclerosis and multiple sclerosis. Rev Bras Hematol Hemoter. 2013; 35(2): 134–143.