Sustained remission with azacitidine monotherapy and an aberrant precursor B-lymphoblast population in juvenile myelomonocytic leukemia.
Saman K HashmiJyotinder N PuniaAndrea N MarcoglieseAmos S GaikwadKevin E FisherAngshumoy RoyPulivarthi RaoDolores H Lopez-TerradaJo RingroseMignon L LohCharlotte M NiemeyerRachel E RauPublished in: Pediatric blood & cancer (2019)
Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. The hypomethylating agent, azacitidine, has been used as a bridging therapy to transplant. However, no patients have been treated with azacitidine without an HSCT post azacitidine. We report on an infant with JMML with somatic KRAS G12A mutation and monosomy 7 who achieved sustained remission following azacitidine monotherapy. He also developed an aberrant B-lymphoblast population which declined with similar kinetics as his JMML-associated abnormalities, suggesting that a B-lymphoblast population in JMML does not always progress to acute leukemia.
Keyphrases
- acute myeloid leukemia
- hematopoietic stem cell
- poor prognosis
- long non coding rna
- end stage renal disease
- newly diagnosed
- chronic kidney disease
- healthcare
- combination therapy
- bone marrow
- open label
- disease activity
- ulcerative colitis
- randomized controlled trial
- peritoneal dialysis
- chronic pain
- gene expression
- smoking cessation
- study protocol
- genome wide
- mesenchymal stem cells
- health insurance