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Acute myeloid leukemia (AML)-pediatric

Transplant advances and outcomes

Advances in understanding the cytogenetic and molecular abnormalities of acute myeloid leukemia (AML) have improved the ability to risk stratify both adult and pediatric patients to identify those who may benefit from hematopoietic cell transplantation (HCT).

Pediatric AML is a relatively rare disease, with an incidence of approximately 7 cases per million children younger than 15 years. [1]

Research demonstrates that allogeneic HCT in complete first remission for children and adults with high-risk AML results in outcomes comparable to those of standard-risk patients. [2] A 2017 multi-center study found that transplant survival in children with unfavorable karyotype AML has improved significantly over time due to decreased relapse risk. [3]

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).

Recommended timing for transplant consultation

High-resolution HLA typing is recommended at diagnosis for all patients.

HCT consultation should take place early after initial diagnosis for patients with AML including:

  • Age <2 years at diagnosis
  • Primary induction failure
  • Measurable (also known as minimal) residual disease after initial therapy
  • CR1 – except favorable risk AML [defined as: t(8;21)(q22;q22.1); 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

References

  1. 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
  2. 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
  3. 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
  4. NMDP and ASTCT Recommended Timing for Transplant Consultation. Download PDF
  5. National Comprehensive Cancer Network. Acute Myeloid Leukemia. (Version 2.2022). Access