LEUKEMIAS

The FDA has granted an orphan drug designation to CA-4948, a first-in-class, small-molecule inhibitor of IRAK4, for the treatment of patients with acute myeloid leukemia and myelodysplastic syndrome.

In an interview with Targeted Oncology, Srdan Verstovsek, MD, PhD, discussed the impact of ruxolitinib on MF and other issues facing the patient population. 

GTB-3550 TriKE had demonstrated early clinical promise as a monotherapy for the treatment of high-risk myelodysplastic syndromes and refractory/relapsed acute myeloid leukemia.

A supplemental biologics license application for brexucabtagene autoleucel for the treatment of adult patients with relapsed or refractory B-cell precursors acute lymphoblastic leukemia.

Blinatumomab consolidation demonstrated significantly prolonged event-free survival in pediatric patients with high-risk first-relapse B-cell precursor acute lymphoblastic leukemia when compared with chemotherapy before allogeneic hematopoietic stem cell transplantation.

Usama Gergis, MD, MBA, reviewed that difference in acute and chronic graft-versus-host disease and the treatment available for each

Rajneesh Nath, MD, discusses his hypothesis on the benefits of targeted delivery of Iomab-B to the marrow in the older relapsed or refractory acute myeloid leukemia population.

The FDA granted Breakthrough Therapy Designation to the novel STAMP inhibitor asciminib for 2 populations of patients with Philadelphia chromosome-positive chronic myeloid leukemia.

Ten-year overall survival is low among patients with acute myeloid leukemia , but an analysis demonstrated that survival is shorter for older patients treated with chemotherapy alone, according to published findings.

In a phase 1b clinical trial presented during the 2020 American Society of Hematology Annual Meeting, investigators sought to determine the efficacy and safety of the novel TIM-3–targeting IgG4 antibody sabatolimab in combination with hypomethylating agents as treatment of patients with acute myeloid leukemia, as well as high-risk myelodysplastic syndrome.

Expanding beyond the chronic lymphocytic leukemia/ small lymphocytic lymphoma space, the BCL2 inhibitor venetoclax garnered attention in 2020 for positive reports from phase 3 clinical trials exploring its use in acute myeloid leukemia and multiple myeloma.

During a Targeted Oncology Case-Based Peer Perspective event, Jonathon B. Cohen, MD, MS, discussed therapeutic options for a 71-year-old patients with chronic lymphocytic leukemia and a genomic aberration.

The FDA has approved ponatinib for the treatment of adult patients with chronic-phase chronic myeloid leukemia with resistance or intolerance of at least 2 prior tyrosine kinase inhibitors.

Treatment with asciminib led to an almost doubling of the major molecular response rate compared with bosutinib at 24 weeks in patients with chronic-phase chronic myeloid leukemia.

High response rates and robust survival outcomes among patients with chronic-phase chronic myeloid leukemia who failed prior treatment with a second-generation tyrosine kinase inhibitor were observed in 2 clinical trials of ponatinib,.

Adult patients with acute lymphoblastic leukemia who have relapsed following hematopoietic cell transplantation have experienced considerably improved survival benefits over the past few years, which may be due to an increased use of immunotherapy.

A real-world analysis showed the positive safety and efficacy of the investigational tyrosine kinase inhibitor asciminib as treatment of patients with chronic myeloid leukemia without any alternatives in clinical practice.

Early signs of clinical activity were observed in adult patients with relapsed/refractory CD22-positive B-cell acute lymphoblastic leukemia who were treated with an investigational allogeneic off-the-shelf CD22-directed therapy.

Agents with novel mechanisms of action distinct from available ATP-competitive tyrosine kinase inhibitors have been tested in patients with previously treated, Philadelphia chromosome–positive chronic myeloid leukemia and offer promise to facilitate deeper remissions.

During a debate, Nicholas J. Short, MD, argued that intensive chemotherapy combined with a BCR-ABL tyrosine kinase inhibitor still holds the place of standard-of-care treatment for Phildelphia chromosome acute lymphoblastic leukemia, while Sabina Chiaretti, MD, PhD, made the case for targeted therapy.

There are several indications for the use of allo-SCT in adults. With the implementation of targeted therapies, these are continuously changing.

Investigation of intensive chemotherapy regimens has unfortunately resulted in a lack of improvement in outcomes for patients with acute lymphoblastic leukemia in the second of the bimodal peaks.

On the treatment of adult patients with acute lymphoblastic leukemia, long-term cure rates in elderly populations, or those older than 55 to 60 years of age, are approximately 15% to 20% and represent a clinically unmet need in this hematologic cancer.

Using a modified pediatric regimen to treat adolescents and young adults with acute lymphoblastic leukemia led to superior outcomes compared with historical adult ALL regimen results, according to a retrospective analysis that evaluated 95 AYAs aged 14 to 39 years. Findings were presented at the eighth annual Society of Hematologic Oncology virtual meeting.

In the past 5 years, 3 new treatment options have emerged to treat patients with relapsed or refractory acute lymphoblastic leukemia, providing hope for patients with this disease, but also raising clinical questions of how to choose among these agents and what is the best option for the patient at which time.

In an interview with Targeted Oncology Guillermo Garcia-Manero, MD, discussed the key findings from the QUAZAR AML-001 study and the importance of the FDA approval of CC-486 in post-remission acute myeloid leukemia.

The FDA granted approval to the oral hypomethylating agent, CC-486 (azacitidine tablets, Onureg), as a maintenance treatment for adult patients with acute myeloid leukemia who achieved a first complete remission or with incomplete blood count recovery after intensive induction chemotherapy and who are unable to complete intensive curative therapy.

The primary end point of statistically superiority in major molecular response rate at 24 weeks was met with asciminib versus bosutinib as treatment of patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase in the phase 3 ASCEMBL study.

Thomas Cluzeau, MD, PhD, discusses the rationale for evaluating azacitidine in combination with APR-246 in patients with TP53-mutant myelodysplastic syndrome and acute myeloid leukemia.

Eunice Wang, MD, discusses the excitement she has experienced in the treatment landscape of acute myeloid leukemia over the last several years due to the number of new treatment options approved by the FDA.