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Treatment of t-AML or t-MDS
Source: Childhood Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 15, 2025 · Captured 2026-09-09.
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Context: Therapy-Related AML and Therapy-Related Myelodysplastic Neoplasms
Treatment options for t-AML or t-MDS include the following:
Hematopoietic stem cell transplant (HSCT).
The goal of treatment is to achieve an initial complete remission (CR) using AML-directed regimens and then, usually, to proceed directly to HSCT with the best available donor. However, treatment is challenging because of the following:[10]
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Increased rates of adverse cytogenetics and subsequent failure to obtain remission with chemotherapy.
Comorbidities or limitations related to chemotherapy used for the previous malignancy.
Accordingly, CR rates and overall survival (OS) rates are usually lower for patients with t-AML than for patients with de novo AML.[10-12] Also, pediatric patients with t-MDS have worse survival rates than pediatric patients with MDS not related to previous therapy.[13]
Patients with t-MDS-refractory anemia usually have not needed induction chemotherapy before transplant. The role of induction therapy before transplant is controversial in patients with refractory anemia with excess blasts-1.
Only a few reports describe the outcome of children undergoing HSCT for t-AML.
Evidence (HSCT for t-AML or t-MDS):
One study described the outcomes of 27 children with t-AML who received related- and unrelated-donor HSCT.[14]
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One study described the outcomes of 27 children with t-AML who received related- and unrelated-donor HSCT.[14]
Three-year OS rates were 18.5% (± 7.5%), and event-free survival (EFS) rates were 18.7% (± 7.5%).
One study described the outcomes of 27 children with t-AML who received related- and unrelated-donor HSCT.[14]
Poor survival was mainly the result of very high transplant-related mortality (59.6% ± 8.4%).
Another study reported a second retrospective single-center experience of 14 patients with t-AML or t-MDS who underwent transplant between 1975 and 2007.[11]
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Another study reported a second retrospective single-center experience of 14 patients with t-AML or t-MDS who underwent transplant between 1975 and 2007.[11]
The survival rate was 29%, but in this review, only 63% of patients diagnosed with t-AML or t-MDS underwent HSCT.
A multicenter study (CCG-2891) examined outcomes of 24 children with t-AML or t-MDS compared with other children enrolled on the study with de novo AML (n = 898) or MDS (n = 62). Children with t-AML or t-MDS were older and rarely had low-risk cytogenetic features.[15]
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A multicenter study (CCG-2891) examined outcomes of 24 children with t-AML or t-MDS compared with other children enrolled on the study with de novo AML (n = 898) or MDS (n = 62). Children with t-AML or t-MDS were older and rarely had low-risk cytogenetic features.[15]
The rates of achieving CR and OS at 3 years were worse in the t-AML/t-MDS group (CR rate, 50% vs. 72%; P = .016; OS rate, 26% vs. 47%; P = .007). However, if patients achieved a CR, the survival was similar (OS rate, 45% vs. 53%; P = .87).
The importance of obtaining remission to improve survival in these patients was further illustrated by another single-center report of 21 children who underwent HSCT for t-AML or t-MDS between 1994 and 2009. Of the 21 children, 12 had t-AML (11 in CR at the time of transplant), seven had refractory anemia (for whom induction was not done), and two had refractory anemia with excess blasts.[16]
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The importance of obtaining remission to improve survival in these patients was further illustrated by another single-center report of 21 children who underwent HSCT for t-AML or t-MDS between 1994 and 2009. Of the 21 children, 12 had t-AML (11 in CR at the time of transplant), seven had refractory anemia (for whom induction was not done), and two had refractory anemia with excess blasts.[16]
The survival rate of the entire cohort was 61%. Patients in remission or with refractory anemia had a disease-free survival rate of 66%.
The importance of obtaining remission to improve survival in these patients was further illustrated by another single-center report of 21 children who underwent HSCT for t-AML or t-MDS between 1994 and 2009. Of the 21 children, 12 had t-AML (11 in CR at the time of transplant), seven had refractory anemia (for whom induction was not done), and two had refractory anemia with excess blasts.[16]
For the three patients with more than 5% blasts at the time of HSCT, the survival rate was 0% (P = .015).
Because t-AML is rare in children, it is not known whether the significant decrease in transplant-related mortality after unrelated-donor HSCT noted over the past several years will translate to improved survival in this population. Patients should be carefully assessed for pre-HSCT morbidities caused by earlier therapies, and treatment approaches should be adapted to give adequate intensity while minimizing transplant-related mortality.
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Preserved source evidence · Not medical advice