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Expert Point of View: William G. Blum, MD and Mikkael Sekeres, MD, MS


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Thoughts on the study by Othman et al were provided by William G. Blum, MD, Professor in the Department of Hematology and Medical Oncology at Emory University School of Medicine and Director of the Acute Leukemia Program at Winship Cancer Institute, and Mikkael Sekeres, MD, MS, Professor of Medicine and Chief of the Division of Hematology, Leukemia Section, at the Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine.

William G. Blum, MD

William G. Blum, MD

Mikkael Sekeres, MD, MS

Mikkael Sekeres, MD, MS

Speaking to The ASCO Post, Dr. Blum said he considers the findings to be “practice-changing” for the management of adults with acute myeloid leukemia (AML) and NPM1 mutation, with or without a concomitant FLT3–internal tandem duplication mutation. He noted that these investigators previously identified measurable residual disease (MRD)-positive status after two courses of intensive induction as a strong negative predictor of outcome for patients with NPM1-mutated disease.1 “Groundbreaking research presented at ASH this year has extended their findings. They now confirm that peripheral blood MRD testing by [quantitative polymerase chain reaction] for NPM1 can be used to risk-stratify patients,” he said.

Dr. Blum noted that a minority of MRD-negative patients also underwent transplant, presumably because they were perceived to be at high risk despite MRD negativity. Further analysis of this cohort may help ascertain whether patients with other high-risk factors (such as hyperleukocytosis) also benefit from early transplantation, he said.

“Among NPM1-mutated patients, however, it appears at this time that MRD status may be the best discriminator for who needs an early transplant and who does not. Patients who are MRD-positive should be offered transplant during [first complete remission], if feasible,” Dr. Blum concluded.

Additional Comments

In a press briefing, Dr. Sekeres told journalists that as a leukemia specialist, he was “particularly excited” by this abstract. “We reach a decision point, after the initial rounds of chemotherapy, as to whether or not to offer a bone marrow transplant…. We typically offer them to patients with residual disease or high-risk disease,” he said.

“This study gave very clear guidance that we can follow MRD status for NPM1-mutated AML, which has been established since 2016,1 and if residual disease is still present we should recommend transplant, based on the clear separation of the curves in this study. For the patient who is MRD-negative, based on these findings, I am now much more comfortable not offering transplant.” 

DISCLOSURE: Dr. Blum reported no conflicts of interest. Dr. Sekeres reported financial relationships with Geron, Novartis, Kurome, and BMS.

REFERENCE

1. Ivey A, Hills RK, Simpson MA, et al: Assessment of minimal residual disease in standard-risk AML. N Engl J Med 374:422-433, 2016.


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