February 2021, Vol 12, No 1

The use of a screening method followed by a financial toxicity intervention that involves navigators, pharmacists, and financial counselors demonstrated significantly improved quality of life for patients with hematologic malignancies, said lead investigator Thomas Greg Knight, MD, Department of Hematologic Oncology and Blood Disorders, Levine Cancer Institute, Atrium Health, Charlotte, NC, at ASH 2020.

The investigational drug pevonedistat in combination with azacitidine injection (Vidaza) leads to longer event-free survival (EFS) and a higher complete response rate than azacitidine alone in patients with high-risk myelodysplastic syndromes (MDS), according to results from a phase 2, open-label, international clinical trial. The findings were presented by Mikkael A. Sekeres, MD, MS, Director, Leukemia Program, Cleveland Clinic, OH, at ASH 2020.

Results of a phase 1 clinical trial showed that approximately 66% of patients with chronic-phase chronic myeloid leukemia (CML) had a major cytogenetic response (MCyR) to the novel oral BCR-ABL1 tyrosine kinase inhibitor (TKI) vodobatinib, regardless of whether they previously received ponatinib therapy. Jorge E. Cortes, MD, Director, Georgia Cancer Center, Augusta University, presented the results at ASH 2020.

Magrolimab, a first-in-class investigational antibody targeting CD47, showed good efficacy when combined with azacitidine injection (Vidaza) regardless of TP53 mutation in patients with treatment-naïve acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy, according to data presented at ASH 2020. The results also showed that this combination did not lead to significant immune-related side effects.

The selective, oral retinoic acid receptor alpha (RARA) agonist, SY-1425, combined with azacitidine showed encouraging activity in patients with relapsed or refractory acute myeloid leukemia (AML) that is overexpressing the RARA gene in a phase 2 clinical trial presented at ASH 2020. The RARA gene is a novel target in patients with AML.

Maintenance therapy with azacitidine (Onureg) tablets improved overall survival (OS) in patients with acute myeloid leukemia (AML) who were in remission after intensive chemotherapy in the phase 3 QUAZAR AML-001 study. Survival was extended regardless of the patients’ measurable residual disease (MRD) status at study entry, reported Gail J. Roboz, MD, Director, Clinical and Translational Leukemia Program, Weill Medical College of Cornell University, New York, NY, at ASH 2020.

The randomized phase 2 CAPTIVATE clinical trial showed that first-line therapy with ibrutinib (Imbruvica) and venetoclax (Venclexta) for a fixed duration (ie, 12 cycles) led to a 30-month progression-free survival (PFS) in more than 95% of patients with chronic lymphocytic leukemia (CLL) and undetectable minimal residual disease (MRD). The results of the MRD cohort of the study were presented at ASH 2020.

The combination of 2 novel agents—umbralisib and ublituximab (U2)—represents a promising new treatment option for patients with chronic lymphocytic leukemia (CLL). In the phase 3 multicenter clinical trial UNITY-CLL, the median progression-free survival (PFS) was significantly longer with U2 than with standard-of-care chemoimmunotherapy, reported John G. Gribben, MD, DSc, FRCP, Centre Lead, Centre for Haemato-Oncology, Barts Cancer Institute, Queen Mary University of London, England, at ASH 2020.

The recent FDA approval of the first FGFR inhibitor, pemigatinib (Pemazyre), and the positive results from the phase 3 study of the first IDH1 inhibitor, ivosidenib (Tibsovo), represent major breakthroughs in the treatment of patients with cholangiocarcinoma (CCA), a rare cancer associated with poor outcomes. However, the duration of response with these agents is still relatively short.

Targeted therapy has improved survival for patients with cancer across a broad spectrum of disease sites, but until recently, progress has been slow in the treatment of patients with cholangiocarcinoma (CCA).