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Autologous Hematopoietic Stem Cell Transplantation for the Treatment of Systemic Sclerosis

July 19, 2019

Health Problem: Systemic sclerosis (SSc) is a progressively disabling autoimmune disease of the connective tissue that causes skin thickening; the disease can thicken visceral connective tissue that results in organ impairment, organ failure, and mortality. SSc is a rare disease, estimated to affect 250 people per 1,000,000 population in the United States.

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 SSc 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 exposure to chemotherapeutic agents and intentional immunoablation, inherently exposing patients to a high risk of developing adverse events and a low, but not insignificant, risk of treatment-related mortality within 100 days after transplant. The benefits of HSCT must therefore be weighed against the risks of adverse events. Uncertainty exists regarding which patients may receive optimal benefit from this treatment due to the high degree of heterogeneity in SSc progression and symptomology. Ideally, HSCT would be utilized for patients who are nonresponsive to immunosuppressive therapy with disease-modifying antirheumatic drugs, but patients who have progressed too far or have suffered too much visceral impairment are often no longer eligible for HSCT.

Key Questions:

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