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Disease-Specific Indications and Outcomes

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Research Spotlight
  • Structural racism variable accounts for almost all racial and ethnic disparity AML survival

    April 2022

  • Age alone should not be a barrier to HCT for adults with AML

    April 2022

  • Cyclophosphamide is Effective GVHD Prophylaxis in Matched Related and Unrelated HCT

    November 2021

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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. [1]

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. [2] 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. [3]

Outcomes

Improved Survival

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

Improved Survival Over Time, Unrelated HCT, Pediatric AML

Download slide "Improved Survival Over Time, Unrelated HCT, Pediatric AML"

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

Survival in Pediatric AML, Unrelated Marrow HCT, by Disease Status

Download slide "Survival in Pediatric AML, Unrelated Marrow HCT, by Disease Status"

Figure 3. Survival, HLA-Identical Sibling Donor HCT, by Diseases Status

Survival in Pediatric AML, HLA-Identical Sibling Donor HCT, by Diseases Status

Download slide "Survival in Pediatric AML, 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. [4]

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

Download as a slide (PPT)
View complete Referral Timing Guidelines

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/Be The Match and ASBMT Recommended Timing for Transplant Consultation.  Download (PDF)
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Slides may be downloaded and used without permission for one-time presentation or personal use.  For publication, use in print or web, or repeated presentations, please submit a Copyright Request Form.

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