Reduced Intensity Vs Myeloablative Conditioning Regimen for Pediatric Therapy-Related Myelodysplastic Syndrome/Acute Myeloid Leukemia

• Published in: Biology of blood and marrow transplantation 2019-03, Vol.25 (3), p.S14-S15
• Main Authors: Sharma, Akshay, Brooke, Russell J., Bhatt, Neel S., Li, Ying, Allewelt, Heather B., Bies, Joshua J., Brondon, Jennifer E., Gloude, Nicholas J., Goeckerman, Erica S., Harris, Katherine M., Ho, Richard H., Hudspeth, Michelle, Huo, Jeffrey S., Jacobsohn, David A., Kasow, Kimberly A., Katsanis, Emmanuel, Kaviany, Saara, Ktena, Yiouli P., Lauhan, Colette, Lopez-Hernandez, Gerardo, Myers, Kasiani C., Naik, Swati, Olaya-Vargas, Alberto, Onishi, Toshihiro, Ramos, Kristie N., Rangarajan, Hemalatha G., Roehrs, Philip A., Sampson, Megan E., Shook, David R., Skiles, Jodi L., Somers, Katherine M., Spiegel, Courtney, Symons, Heather J., Uber, Allison, Triplett, Brandon M.
• Format: Article
• Language: English
• ISSN: 1083-8791; 1523-6536
• DOI: 10.1016/j.bbmt.2018.12.081

Therapy-related myelodysplastic syndrome and acute myeloid leukemia (t-MDS/AML) are rare but well-recognized complications of cancer therapy. Allogeneic hematopoietic cell transplantation (HCT) with myeloablative conditioning (MAC) remains the mainstay of treatment but is associated with a high incidence of non-relapse mortality (NRM). Reduced intensity conditioning (RIC) may reduce NRM in these heavily pre-treated patients. The primary objective of this study was to compare outcomes following RIC and MAC regimens in patients receiving an allogeneic HCT. Retrospective chart review of patients =90) was the only factor that was found to be statistically associated with improved EFS (HR 0.49, p=0.01). NRM was noted in 61% of patients after MAC compared to 28% after RIC, however disease relapse was a major cause of death after RIC (Fig C & D). The majority of transplants were performed after 2005 (81.6%) and outcomes seem to be slightly better in contemporary era due to improved supportive care and decreased NRM. This is one of the largest reported cohorts of pediatric patients receiving allogeneic transplantation for t-MDS/AML. Outcomes are poor relative to de novo MDS/AML due to high rate of NRM with MAC and high rate of disease related death after RIC. Novel strategies that lead to a reduction in transplant-related mortality in these heavily pre-treated patients while providing sufficient disease control are needed to improve survival in t-MDS/AML patients receiving HCT.