Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma.

ABSTRACT:

Background

Despite current therapies, diffuse cutaneous systemic sclerosis (scleroderma) often has a devastating outcome. We compared myeloablative CD34+ selected autologous hematopoietic stem-cell transplantation with immunosuppression by means of 12 monthly infusions of cyclophosphamide in patients with scleroderma.

Methods

We randomly assigned adults (18 to 69 years of age) with severe scleroderma to undergo myeloablative autologous stem-cell transplantation (36 participants) or to receive cyclophosphamide (39 participants). The primary end point was a global rank composite score comparing participants with each other on the basis of a hierarchy of disease features assessed at 54 months: death, event-free survival (survival without respiratory, renal, or cardiac failure),

SUBMITTER: Sullivan KM 

PROVIDER: S-EPMC5846574 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature