Acute Myeloid Leukemia (AML) - Pediatric
Advances in understanding the cytogenetic and molecular abnormalities of acute myelogenous leukemia (AML) have improved the ability to risk stratify both adult and pediatric patients in order to identify those who would benefit most from hematopoietic cell transplantation (HCT).
Pediatric AML is a relatively rare disease, with an incidence of approximately seven cases per million children younger than 15 years. 
Research demonstrates that allogeneic HCT in complete first remission for children and adults with high-risk AML results in outcomes comparable to outcomes of standard-risk patients.  And a 2017 multi-center study found that transplant survival in children with unfavorable karyotype AML has improved significantly over time, and was due to decrease in relapse risk. 
Figure 1 demonstrates how transplant outcomes have steadily improved over time in pediatric patients with AML undergoing unrelated donor transplantation.
Data in this section have been prepared by CIBMTR® (Center for International Blood and Marrow Transplant Research), our research program.
Figure 1. Improved Survival Over Time
Overall Survival by Disease Status and Cell Source
Review outcomes for transplants in pediatric patients with AML below. View additional AML slides showing demographic data and transplant trends.
Pediatric AML patients who receive transplants in first or second complete remission (CR) experience significantly improved survival compared to those transplanted with advanced disease (primary induction failure, active disease, or CR3 and beyond).
Figure 2. Survival, Unrelated Marrow HCT, by Disease Status
Figure 3. Survival, HLA-Identical Sibling Donor HCT, by Diseases Status
Referral Timing Guidelines
These guidelines highlight disease categories that include patients at risk for disease progression and who should be referred for a consultation for autologous or allogeneic transplantation. 
Transplant Consultation Guidelines: Pediatric AML
High-resolution HLA typing is recommended at diagnosis for all patients
HCT consultation should take place early after initial diagnosis for all patients with AML, including:
- Age <2 years at diagnosis
- Primary induction failure
- Measurable (also called minimal) residual disease after initial therapy
- CR1 – except favorable risk AML [defined as: t(8;21)(q22;q21.1); RUNX1-RUNX1T1, inv(16)(p13. 1q22) or t(16;16)(p13. 1; q22); CBFB-MYH11, mutated NPM1 without FLT3-ITD or with FLT3-ITDlow, biallelic mutated CEBPA]
- Monosomy 5 or 7
- Treatment-related leukemia
- First relapse
- CR2 and beyond, if not previously evaluated
- Creutzig U, van den Heuvel-Eibrink MM, Gibson B, et al. Diagnosis and management of acute myeloid leukemia in children and adolescents: Recommendations from an international expert panel. Blood. 2012; 120(16): 3187-3205. Access
- Burke MJ, Wagner JE, Cao Q, Ustun C, Verneris MR. Allogeneic hematopoietic cell transplantation in first remission abrogates poor outcomes associated with high-risk pediatric acute myeloid leukemia. Biol Blood Marrow Transplant. 2013; 19(7): 1021-1025. Access
- Alloin L-F, Leverger G, Dalle J-H, et al. Cytogenetics and outcome of allogeneic transplantation in first remission of acute myeloid leukemia: the French pediatric experience. Bone Marrow Transplant. 2017; 52(4): 516-521. Access
- NMDP/Be The Match and ASBMT Recommended Timing for Transplant Consultation. Download (PDF)