Venetoclax/decitabine for a pediatric patient with chronic myelomonocytic leukemia

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.

Automated summary

Chronic myelomonocytic leukemia (CMML) is a common myelodysplastic syndrome/myeloproliferative disorder in the elderly, but rare in adolescents. This case report describes an adolescent with monosomy 7 CMML presenting with central diabetes insipidus (DI), treated with venetoclax and decitabine as a bridge to hematopoietic stem cell transplantation (HSCT).