Early hematopoietic stem cell transplant is associated with favorable outcomes in children with MDS1. Issue 4 (14th November 2012)

• Record Type: Journal Article
• Authors: Smith, Angela R.; Christiansen, Ellen C.; Wagner, John E.; Cao, Qing; MacMillan, Margaret L.; Stefanski, Heather E.; Trotz, Barbara A.; Burke, Michael J.; Verneris, Michael R.
• Is Part Of: Pediatric blood & cancer. Volume 60:Issue 4(2013:Apr.)
• Page Start: 705
• Page End: 710
• Publication Date: 2012-11-14
• Subjects: Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.24390
• Languages: English
• ISSNs: 1545-5009

Abstract

<abstract abstract-type="main" xml:lang="en"> <title>Abstract</title> <sec id="abs1-1" sec-type="section"> <title>Background</title> <p>Although hematopoietic stem cell transplantation (HSCT) is the treatment of choice for childhood myelodysplastic syndrome (MDS), there is no consensus regarding patient or disease characteristics that predict outcomes.</p> </sec> <sec id="abs1-2" sec-type="section"> <title>Procedure</title> <p>We reviewed 37 consecutive pediatric MDS patients who received myeloablative HSCT between 1990 and 2010 at a single center.</p> </sec> <sec id="abs1-3" sec-type="section"> <title>Results</title> <p>Twenty had primary MDS and 17 had secondary MDS. Diagnostic cytogenetics included monosomy 7 (n = 21), trisomy 8 (n = 7) or normal/other (n = 8). According to the modified WHO MDS classification, thirty had refractory cytopenia and seven had refractory anemia with excess blasts. IPSS scores were: low risk (n = 1), intermediate‐1 (n = 15), and intermediate‐2 (n = 21). OS and DFS at 10 years in the entire cohort was 53% and 45%. Relapse at 10 years was 26% and 1 year TRM was 25%. In multivariate analysis, factors associated with improved 3 years DFS were not receiving pre‐HSCT chemotherapy (RR = 0.30, 95% CI 0.10–0.88; <italic>P</italic> = 0.03) and a shorter interval (<140 days) from time of diagnosis to transplant (RR = 0.27, 95% CI 0.09–0.80; <italic>P</italic> = 0.02). Three years DFS in patients who did not receive pre‐HSCT chemotherapy and those who had a shorter interval to transplant (n = 16) was 80%.</p> </sec> <sec id="abs1-4" sec-type="section"> <title>Conclusion</title> <p>These results suggest that children with MDS should be referred for allogeneic HSCT soon after diagnosis and that pre‐HSCT chemotherapy does not appear to improve outcomes. Pediatr Blood Cancer 2013; 60: 705–710. © 2012 Wiley Periodicals, Inc.</p> </sec> </abstract>