February 2021, Vol 12, No 1

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).

Acute graft-versus-host disease (GVHD) is a major complication after allogeneic hematopoietic stem-cell transplant (HSCT). The rate of GVHD is between 34% and 51% within 100 days of undergoing transplant.

A new step-up dosing schedule with glofitamab, an investigational T-cell engaging bispecific antibody, has demonstrated strong clinical activity, with high complete response rates in patients with hard-to-treat relapsed or refractory non-Hodgkin lymphoma (NHL), according to data presented at the ASH 2020 annual meeting.

Momelotinib, a selective and orally bioavailable inhibitor of Janus kinase (JAK) 1, JAK2, and ACVR1, improved overall survival and sustained efficacy outcomes in patients with intermediate- or high-risk myelofibrosis, according to updated findings from the phase 3 SIMPLIFY-1 and SIMPLIFY-2 clinical trials presented at ASH 2020. Momelotinib was of benefit to patients who previously received treatment with ruxolitinib (Jakafi) and those who had not received a JAK inhibitor.

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.

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 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.

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 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.

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.