Year: 2007
Type: article
Study design: Observational Study
Abstract: The objective of high dose chemotherapy followed by hematopoietic stem cell rescue is to halt autoimmunity in patients with severe AI. Few patients have received this treatment to date and this report summarizes the activity of HCT for AI from centers in North and South America. One-hundred and ninety-eight patients who underwent HCT primarily for the treatment of AI, were reported to the CIBMTR from 1996-2005 from 43 centers. Most received an autologous HCT (autoHCT) (171 of 198). The most common indications for autoHCT were multiple sclerosis (MS) (45%), scleroderma (SSC) (35%) and systemic lupus erythematous (SLE) (8%). Twenty-seven patients received allogeneic HCT (alloHCT) for SSC (n=7), amyotrophic lateral sclerosis (n=6), autoimmune cytopenias (n=6), SLE (n=2), autoimmune enteropathy (n=1) and unclassified AI (n=3). The median age at HCT did not differ by transplant type (41 vs. 36 years for autoHCT and alloHCT, respectively) and most patients (66%) had a good performance score at HCT. The median time from diagnosis to transplant was 58 and 34 months for autoHCT and alloHCT, respectively. Irradiation-containing conditioning regimens were used in 109 cases (61%) and cyclophosphamide either alone or in combination with other agents without irradiation in 45 cases (25%). Most autoHCT recipients received total body irradiation, cyclophosphamide and anti-thymocyte globulin (64%). Peripheral stem cell cells were the predominant graft type; and 56% of these grafts were manipulated ex vivo (CD34+ cell selection or T-cell depletion). The probabilities of 100-day mortality were 10% (95% confidence interval [CI], 6-15%) and 17% (95% CI, 5-35%) after autoHCT and alloHCT, respectively. Corresponding one-year probabilities of overall survival were 89% (95% CI, 83-93%) and 75% (95% CI, 54-91%). One-year probability of overall survival post-autoHCT were 96%, 82% and 73% for MS, SSC and SLE respectively. In summary, these collective data from CIBMTR, MS and SSC are the most common autoimmune disease indications for HCT.
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2045575444