Publications by authors named "R B Gerbing"
Introduction: Anthracyclines are effective in treating acute myeloid leukemia (AML) but limited by cardiotoxicity. CPX-351, a liposomal daunorubicin and cytarabine, may provide therapeutic benefit with less cardiotoxicity. Acute changes in left ventricular systolic function and cardiac biomarkers were evaluated after a cycle of CPX-351 in children with relapsed AML treated on the phase 1/2 Children's Oncology Group study, AAML1421.
View Article and Find Full Text PDFThe addition of the proteasome inhibitor bortezomib to standard chemotherapy did not improve survival in pediatric acute myeloid leukemia (AML) when all patients were analyzed as a group in the Children's Oncology Group phase 3 trial AAML1031 (NCT01371981). Proteasome inhibition influences the chromatin landscape and proteostasis, and we hypothesized that baseline proteomic analysis of histone- and chromatin-modifying enzymes (HMEs) would identify AML subgroups that benefitted from bortezomib addition. A proteomic profile of 483 patients treated with AAML1031 chemotherapy was generated using a reverse-phase protein array.
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- * Three new recurring KMT2A-rearranged groups were identified, and a significant variation in 5-year event-free survival rates was observed across 13 different groups, highlighting the impact of genetic factors on patient outcomes.
- * The research suggests incorporating five specific adverse-risk KMT2A fusions into current risk stratification models and calls for further studies to confirm the associations
Article Synopsis
- Myeloid neoplasms are a significant cause of pediatric leukaemias in infants, with 25%-50% of newborn leukemia cases showing skin-related symptoms before bone marrow involvement.
- This study examines 50 infants diagnosed with either cutaneous extramedullary disease (cEMD) or acute myeloid leukemia with cutaneous involvement (AML-cEMD), finding a common genetic mutation (KMT2A rearrangement) and notable survival rates.
- Results indicate a 66% event-free survival and a 75% overall survival rate, with some patients experiencing spontaneous remission; outcomes did not significantly differ between cEMD and AML-cEMD groups.
MLLT10 gene rearrangements with KMT2A occur in pediatric acute myeloid leukemia (AML) and confer poor prognosis, but the prognostic impact of MLLT10 in partnership with other genes is unknown. We conducted a retrospective study with 2080 children and young adults with AML registered on the Children's Oncology Group AAML0531 (NCT00372593) and AAML1031 trials (NCT01371981). Transcriptome profiling and/or karyotyping were performed to identify leukemia-associated fusions associated with prognosis.
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