Key Points
Low or nondetectable MRD pre-HCT leads to similar outcomes, suggesting that MRD negativity is not an absolute prerequisite for HCT. MRD post-HCT is more important than pre-HCT, and monitoring with sensitive techniques can detect very high-risk patients early.
We report a 14 year-old female with Giant Cell Tumor of Bone, successfully treated with denosumab, who developed critical hypercalcemia after completion of therapy. Five months after her last denosumab treatment, serum calcium rose to 16.5 mg/dL (normal 8.7-10.8 mg/dL), nearly double her prior level of 8.4 mg/dL while receiving denosumab. She required emergent intervention to treat her hypercalcemia, which was attributed to rebound osteoclast activity and osteopetrotic bone. Denosumab is widely used in adults and increasingly in pediatric oncology populations and our experience demonstrates the need for close monitoring for electrolyte derangements following discontinuation.
Key Points
Children treated with blinatumomab for B-ALL with MRD had few side effects and proceeded to hematopoietic cell transplant without delay. Blinatumomab given prior to transplant reduces MRD and results in favorable leukemia-free survival, toxicity, and overall survival.
Survival outcomes for relapsed/refractory pediatric acute myeloid leukemia (R/R AML) remain dismal. Epigenetic changes can result in gene expression alterations which are thought to contribute to both leukemogenesis and chemotherapy resistance. We report results from a phase I trial with a dose expansion cohort investigating decitabine and vorinostat in combination with fludarabine, cytarabine, and G-CSF (FLAG) in pediatric patients with R/R AML [NCT02412475]. Thirty-seven patients enrolled with a median age at enrollment of 8.4 (range, 1-20) years. There
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