- Nicolas Waespe1,
- Machiel Van Den Akker2,
- Robert J. Klaassen3,
- Lani Lieberman4,
- Meredith S. Irwin1,
- Salah S. Ali1,
- Mohamed Abdelhaleem5,
- Bozana Zlateska6,
- Mira Liebman1,
- Michaela Cada1,
- Tal Schechter1 and
- Yigal Dror1,*
- 1 Division of Hematology/ Oncology, The Hospital for Sick Children, Toronto, Canada;
- 2 Pediatric Hematology/Oncology, UZ Brussel, Jette, Belgium;
- 3 Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, Canada;
- 4 Department of Laboratory Medicine, University Health Network, Toronto, Canada;
- 5 Division of Hematopathology, The Hospital for Sick Children, Toronto, Canada;
- 6 Genetics and Genome Biology Program, The Hospital for Sick Children, Toronto, Canada
- ↵* Corresponding author; email:
Advanced myelodysplastic syndrome harbors a high risk of progression to acute myeloid leukemia and poor prognosis. In children, there is no established treatment to prevent or delay progression to leukemia prior to hematopoietic stem cell transplantation. Azacitidine is a hypomethylating agent, which was shown to slow progression to leukemia in adults with myelodysplastic syndrome. There is little data on the efficacy of azacitidine in children. We reviewed 22 pediatric patients with advanced myelodysplastic syndrome from a single center, diagnosed between January 2000 and December 2015. Of those, eight patients received off-label azacitidine before hematopoietic stem cell transplantation. A total of 31 cycles were administered and modification or delay occurred in four of them due to cytopenias, infection, nausea/vomiting, and transient renal impairment. Bone marrow blast percentages in azacitidine- treated patients decreased significantly from a median of 15% (range 9-31%) at start of treatment to 5.5% (0-12%, p=0.02) before hematopoietic stem cell transplantation. Following azacitidine treatment, four patients (50%) achieved marrow remission, and none progressed. In contrast, three untreated patients (21.4%) had progressive disease characterized by >50% increase in blast counts or progression to leukemia. Azacitidine-treated patients had significantly increased 4- year event-free survival (p=0.04); predicted 4 year-overall survival was 100% versus 69.3% in untreated patients (p=0.1). In summary, azacitidine treatment prior to hematopoietic stem cell transplantation was well tolerated in pediatric patients with advanced myelodysplastic syndrome, led to partial or complete bone marrow response in seven of eight patients (87.5%), and correlated with superior event-free survival in this cohort.
- Received March 16, 2016.
- Accepted August 18, 2016.
- Copyright © 2016, Ferrata Storti Foundation