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 hemato...

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Bibliographic Details
Published inPediatric blood & cancer Vol. 68; no. 3; pp. e28865 - n/a
Main Authors 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.
Format Journal Article
LanguageEnglish
Published United States Wiley Subscription Services, Inc 01.03.2021
Subjects
5-aza-2'-deoxycytidine
Chronic myelomonocytic leukemia
Diabetes insipidus
Hematology
Hematopoietic stem cells
Leukemia
Monosomy
Myelodysplastic syndrome
Myelodysplastic syndromes
Myelomonocytic leukemia
Myeloproliferative diseases
Oncology
Pediatrics
Stem cell transplantation
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Summary: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.
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AUTHOR CONTRIBUTIONS
John C. Molina, Julie M. Asare, and Nirali N. Shah wrote the first draft of this manuscript. John C. Molina, Julie M. Asare, Rebecca Persky, Alison M. Boyce, Steven M. Holland, Dennis Hickstein, and Nirali N. Shah all provided patient care. Katerine R. Calvo provided pathology expertise, and Dima A. Hammoud provided radiology expertise. Robert R. West and Laura Tuschong genotyped and tracked mutations.
ISSN:1545-5009
1545-5017
DOI:10.1002/pbc.28865