Articles related to Hematology/Oncology

Perelman School of Medicine’s David Teachey, MD, reviews outcomes and implications from two recent Children’s Oncology Group trials and describes current research with daratumumab and CAR T-cell therapy.

An FDA expert panel voted 16-0, with one abstention, recommending that future FDA approvals of PI3K inhibitors be supported by randomized data. The four currently-approved PI3K inhibitors for treating hematologic malignancies received FDA approval based on single-arm data. Although these drugs have offered durable ORR and improvements in PFS, randomized data presented at the hearing showed they are associated with higher rates of serious adverse events, including fatality.

In an integrated analysis of mutations and clinical outcomes, comprising 2,200 patients with TP53-mutated myelodysplastic syndrome (MDS) with excess blasts (EB) or TP53-mutated acute myeloid leukemia (AML), the authors state that mutant TP53 AML and MDS-EB “do not differ with respect to molecular characteristics and survival” and argue these entities should be considered a single molecular disease entity. In a commentary to the above paper, TP53 and the star-crossed lovers MDS and AML, John Welch, MD, PhD of Washington University School of Medicine writes, “As a junior Hematology/Oncology fellow, I was told there were two types of physicians: splitters and mergers. That is, clinicians either seek to diagnose increasingly homogenously narrow groups of patients based on increasingly refined, shared characteristics, or they seek to find broad, overarching patterns that unite diagnostic classifications. Hematologic malignancies have been fertile ground for the diagnostic splitters of the world. On the other hand, there have been some noteworthy exceptions. Sometimes it is a technological advance that allows for the synthesis of disparate diagnoses.”

This international consortium of oncologists and researcher describe the advantages and limitations of both conventional chromosome banding and novel sequencing technologies. They state, “While one single test may not replace the totality of tests that currently exist, the active exploration and adoption of novel approaches in defined clinical settings may indeed allow for a continued and controlled evolution resulting in the successful clinical implementation of whole genome sequencing in the near future.”

This single-arm study of 40 patients with advanced neuroendocrine tumors (NETs) sought to evaluate the potential benefit of a VEGF inhibitor plus a PD-L1 inhibitor. The primary endpoint was objective radiographic response. Responses were seen in 15% to 20% of patients.

Data from a large phase III trial (Myeloma XI) were examined to determine the relationship between MRD status, PFS, and OS in post-ASCT patients randomly assigned to lenalidomide maintenance or no maintenance at 3 months after ASCT. At ASCT + 3 and ASCT + 9, MRD-negative status was associated with improved PFS and OS. Sustained MRD negativity from ASCT + 3 to ASCT + 9 or the conversion to MRD negativity by ASCT + 9 was associated with the longest PFS/OS.