A 34-Year-Old Woman with 2-Year History of Therapy-Resistant, Rapidly Progressive SSc Successfully Treated by Autologous Hematopoietic Stem Cell Transplantation

Abstract

A subgroup of diffuse cutaneous systemic sclerosis (dSSc) patients has a very poor prognosis, making the need for an effective disease modifying therapy especially apparent. Some immunosuppressive agents such as cyclophosphamide have demonstrated some positive impact on disease expression, but the dose is limited by bone marrow and other toxicity. For this reason, it was proposed 15 years ago to test the oncological/hematological concept of hemato/immuno ablation followed by autologous hematopoietic stem cell transplant (HSCT) as “rescue” for severe autoimmune diseases (AD) such as SSc. Over 1,500 AD patients have so been treated in a coordinated international program, 250 of whom had SSc. Not all have responded and some patients have died as a consequence of the transplant. This case illustrates a successful outcome, despite several severe episodes of toxicity. Several randomized prospective clinical trials are running or have been recently completed to establish the place, if any, for HSCT in severe SSc.