Down syndrome preleukemia and leukemia

Pediatr Clin North Am. 2015 Feb;62(1):121-37. doi: 10.1016/j.pcl.2014.09.009.


Children with Down syndrome (DS) and acute leukemias acute have unique biological, cytogenetic, and intrinsic factors that affect their treatment and outcome. Myeloid leukemia of Down syndrome (ML-DS) is associated with high event-free survival (EFS) rates and frequently preceded by a preleukemia condition, the transient abnormal hematopoiesis (TAM) present at birth. For acute lymphoblastic leukemia (ALL), their EFS and overall survival are poorer than non-DS ALL, it is important to enroll them on therapeutic trials, including relapse trials; investigate new agents that could potentially improve their leukemia-free survival; and strive to maximize the supportive care these patients need.

Keywords: B-ALL; Down syndrome; GATA1; Leukemia; Myeloid leukemia of Down syndrome; Preleukemia; Transient abnormal hematopoiesis/transient myeloproliferative disorder.

MeSH terms

  • Child
  • Child, Preschool
  • Down Syndrome / blood
  • Down Syndrome / complications*
  • Down Syndrome / genetics
  • Humans
  • Leukemia / complications*
  • Leukemia / genetics
  • Leukemia / therapy
  • Mutation
  • Preleukemia / complications*
  • Preleukemia / genetics
  • Preleukemia / therapy