Health Problem: Multiple sclerosis (MS) is a potentially disabling autoimmune disease that impacts the central nervous system. MS is estimated to affect 400,000 to 1,000,000 people in the United States and 2,300,000 people worldwide.

Technology Description: During hematopoietic stem cell transplantation (HSCT), a patient undergoes a partially or fully myeloablative conditioning regimen, followed by infusion of autologous or allogenic hematopoietic stem cells. The goal of HSCT for MS is to eradicate the dysfunctional immune cells mediating the disease and enable the formation of a more functional system using hematopoietic stem cells.

Controversy: HSCT involves the administration of high levels of chemotherapeutic agents and other drugs with notable toxicity. The benefits of HSCT must therefore be weighed against the risks of adverse events. A variety of conditioning regimens with a range of intensities are used in concert with HSCT; optimal protocols and accompanying patient selection criteria have not been established. With regard to MS disease type, although there are several treatment options for relapsing progressive MS, few viable treatments are available for primary progressive MS. It is unclear whether HSCT is an appropriate treatment for progressive versus relapsing forms of MS or whether other factors such as age and disease duration impact treatment success.

Key Questions:

  • Is HSCT effective in treating patients with MS?
  • How does HSCT compare with alternative therapies for MS?
  • Is HSCT safe for treatment of MS?
  • Have definitive patient selection criteria been identified for HSCT in patients with MS?

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