Journal
CLINICAL RHEUMATOLOGY
Volume 41, Issue 3, Pages 869-876Publisher
SPRINGER LONDON LTD
DOI: 10.1007/s10067-021-05931-0
Keywords
Autoimmune disease; Autologous transplantation; Outpatient care; Patient-reported outcomes; Refractory; Rheumatic disease
Categories
Ask authors/readers for more resources
Autologous hematopoietic stem cell transplantation (HSCT) without anti-thymocyte globulin (ATG) can be a viable treatment alternative for patients with severe, refractory autoimmune rheumatic diseases (ARDs). This study demonstrated that some patients achieved remission and improved functionality post-HSCT, highlighting the potential benefits of HSCT for selected patients with ARDs. Further research is needed to identify the optimal candidate and conditioning regimen for HSCT when ATG is not available.
Autologous hematopoietic stem cell transplantation (HSCT) has been recognized as treatment alternative for patients with severe, refractory autoimmune rheumatic diseases (ARDs). Usually, anti-thymocyte globulin (ATG)-containing conditioning regimens are employed; however, ATG is unavailable in some developing nations. We report our 15-year clinical experience autografting patients with ARDs with an ATG-free conditioning regimen and a brief assessment of patient-reported outcomes post-HSCT. All patients had active disease and were resistant to multiple lines of treatment. Event-free survival (EFS) was assessed using the Kaplan-Meier method. Eight patients underwent autologous HSCT. Diagnoses included juvenile idiopathic arthritis (n = 3), systemic lupus erythematosus (n = 2), systemic sclerosis (n = 2), and rheumatoid arthritis (n = 1). Median time from diagnosis to HSCT was 3 years (0.75-19). Hematological recovery was documented in all recipients, and 4 patients (50%) completed the procedure in a completely ambulatory setting. Five (62.5%) patients achieved complete response and 3 (37.5%) partial response. The median EFS was 7 months (95% CI, 4.97-9.02), and the 1-year EFS rate was 21.9% (95% CI, 18.25-25.76). Transplant-related mortality was 0%, and 1 recipient died 8 years post-HSCT due to chronic kidney disease. Six (75%) patients presented steroid dosage reduction post-HSCT, and 2 (25%) perceived improvement in functionality despite having relapsed. HSCT is a viable treatment alternative for selected patients with severe therapy-resistant ARDs, as an improvement in disease management and quality of life was documented. The need remains to elucidate the characteristics of the optimal HSCT candidate, as well as the adequate conditioning regimen when ATG is not available.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available