Venetoclax/decitabine for a pediatric patient with chronic myelomonocytic leukemia

Pediatr Blood Cancer. 2021 Mar;68(3):e28865. doi: 10.1002/pbc.28865. Epub 2020 Dec 27.

Abstract

Chronic myelomonocytic leukemia (CMML) is a myelodysplastic syndrome (MDS)/myeloproliferative disorder most commonly seen in the elderly. We describe an adolescent with monosomy 7 CMML presenting as central diabetes insipidus (DI), who was treated with venetoclax and decitabine as a bridge to hematopoietic stem cell transplantation (HSCT). Central DI is a rare manifestation of monosomy 7-associated MDS including CMML, itself a rare manifestation of GATA2 deficiency, particularly in children. Venetoclax/decitabine was effective for treatment of CMML as a bridge to HSCT.

Publication types

  • Case Reports
  • Research Support, N.I.H., Intramural

MeSH terms

  • Adolescent
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Bridged Bicyclo Compounds, Heterocyclic / administration & dosage
  • Decitabine / administration & dosage
  • Humans
  • Leukemia, Myelomonocytic, Chronic / drug therapy*
  • Leukemia, Myelomonocytic, Chronic / pathology
  • Male
  • Prognosis
  • Sulfonamides / administration & dosage

Substances

  • Bridged Bicyclo Compounds, Heterocyclic
  • Sulfonamides
  • Decitabine
  • venetoclax