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Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Survivorship and Adverse Late Sequelae

While the issues of long-term complications of cancer and its treatment cross many disease categories, there are several important issues that relate to the treatment of myeloid malignancies that are worth stressing. (Refer to the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.)

The Children's Cancer Survivor Study examined 272 survivors of childhood acute myeloid leukemia (AML) who did not undergo a hematopoietic stem cell transplant (HSCT).[1] This study identified second malignancies (cumulative incidence, 1.7%) and cardiac toxic effects (cumulative incidence, 4.7%) as significant long-term risks. Cardiomyopathy has been reported in 4.3% of survivors of AML based on Berlin-Frankfurt-Münster studies. Of these, 2.5% showed clinical symptoms.[2] A retrospective study of cardiac function of children treated with United Kingdom Medical Research Council-based regimens at a median of 13 months following treatment reported a mean detrimental change in left ventricular stroke volume of 8.4% compared with baseline values.[3] For pediatric patients, the risk of developing early toxicity was 13.7% and the risk of developing late cardiac toxic effects (defined as 1 year after completing first-line therapy) was 17.4%. Early cardiac toxic effects was a significant predictor of late cardiac toxic effects and the development of clinical cardiomyopathy requiring long-term therapy.[4] Retrospective analysis of a single study suggests cardiac risk may be increased in children with Down syndrome,[5] but prospective studies are required to confirm this finding.

For children undergoing chemotherapy only for treatment of AML, renal, gastrointestinal, and hepatic late adverse effects have been reported to be rare.[6] A Nordic Society for Pediatric Hematology and Oncology retrospective trial of children treated for AML with chemotherapy only at a median follow-up of 11 years, based on a self-reported use of health care services, demonstrated similar health care usage and marital status as their siblings.[7] A COG study using a health-related, quality-of-life comparison reported an overall 21% of 5-year survivors having a severe or life-threatening chronic health condition; when compared by type of treatment, this percentage was 16% for the chemotherapy-only treated group, 21% for the autologous HSCT treated group, and 33% for those who received an allogeneic HSCT.[8]

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