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Venetoclax/decitabine for a pediatric patient with chronic myelomonocytic leukemia
Author(s) -
Molina John C.,
Asare Julie M.,
Tuschong Laura,
West Robert R.,
Calvo Katherine R.,
Persky Rebecca,
Boyce Alison M.,
Hammoud Dima A.,
Holland Steven M.,
Hickstein Dennis,
Shah Nirali N.
Publication year - 2021
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28865
Subject(s) - decitabine , medicine , chronic myelomonocytic leukemia , venetoclax , myelodysplastic syndromes , pediatrics , oncology , leukemia , bone marrow , biochemistry , gene expression , chemistry , chronic lymphocytic leukemia , dna methylation , gene
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.