Life after Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis

Abstract

Stem cell transplantation has been investigated as treatment for severe and progressive systemic sclerosis (SSc) for the past 25 years. To date, more than 1000 SSc patients have been transplanted worldwide. Overall and event-free survival have increased over the years, reflecting stricter patient selection criteria and better clinical management strategies. This review addresses long-term outcomes of transplanted SSc patients, considering phase I/II and randomized clinical trials, as well as observational studies and those assessing specific aspects of the disease. Clinical outcomes are discussed comparatively between studies, highlighting advances, drawbacks and controversies in the field. Areas for future development are also discussed.

Keywords: long-term outcomes; progression-free survival; stem cell transplantation; systemic sclerosis.

Figure 1

Schematic representation of the transplant procedure. Autologous stem cells are mobilized from the bone marrow to the peripheral blood, from where they are harvested by leukapheresis and cryopreserved (1). The graft can be selected before cryopreservation or remain unmanipulated. The patient then undergoes an immunoablative conditioning regimen (2), followed by intravenous administration of the autologous cells, which are thawed immediately before infusion (3). After a period of aplasia, there is reconstitution of the immune system (4), and the patient is discharged from the hospital. Long-term outcomes are evaluated over time.