Introduction
Survival is dismal for the 40-60% of children with MDS who relapse post
allogeneic HCT1,2,3. Strategies to decrease relapse
risk include use of cytoreduction prior to HCT or maintenance treatment
after HCT, data on the utility of these approaches remains
limited4-12. Rapid withdrawal of immune suppression or
use of donor lymphocyte infusion (DLI) can enhance the graft versus
leukemia effect and achieve disease control in some
cases13,14. Addition of hypomethylating agents to DLI
may provide additional benefit15 and second HCT should
be considered16-21. While several novel therapies may
alter the future landscape of MDS therapy22-28 (Table
1), the optimal approach to relapsed pediatric MDS remains unclear. We
report the management of a child who relapsed less than 70 days after
initial HCT. Our approach demonstrates that multimodal therapy may
permit prolonged survival with excellent quality of life (QOL) despite
lack of long-term cure.