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Associate Chair
US Oncology Hematology Research Program
Rocky Mountain Cancer Centers
Aurora, Colorado
John M. Burke, MD, has disclosed that he has received consulting fees from AbbVie, Adaptive Biotechnologies, AstraZeneca, Epizyme, Kura, MorphoSys, Roche/Genentech, and Verastem and fees for non-CME/CE services from Seattle Genetics.
Associate Professor
Oncology and Medicine
The Johns Hopkins University School of Medicine
Baltimore, Maryland
Amy E. DeZern, MD, MHS, has disclosed that she has received consulting fees from Bristol-Myers Squibb, Geron, Gilead Sciences, Novartis, Taiho, and Takeda.
Mark and Judy Mullins Professor of Hematological Malignancies
Chair, Myeloma Amyloidosis Dysproteinemia Group
Consultant, Division of Hematology
Mayo Clinic
Rochester, Minnesota
Shaji K. Kumar, MD, has disclosed that he has received funds for research support from AbbVie, Bristol-Myers Squibb, Celgene, Genentech, Janssen, MedImmune, Oncopeptides, Takeda, and TeneoBio and consulting fees from AbbVie, Bristol-Myers Squibb, Celgene, Genentech, Janssen, Oncopeptides, and Takeda
Professor and Chair
Department of Hematology and Medical Oncology
Chief Medical Officer
Winship Cancer Institute of Emory University
Atlanta, Georgia
Sagar Lonial, MD, has disclosed that he has received funds for research support from Bristol-Myers Squibb, Celgene, Janssen, and Takeda; consulting fees from AbbVie, Bluebird, Bristol-Myers Squibb, Celgene, GlaxoSmithKline, Janssen, and Takeda; and fees for non-CME/CE services from TG Therapeutics.
Associate Professor of Medicine
Department of Medicine, Division of Hematology-Oncology
Weill Cornell Medical College
New York, New York
Peter Martin, MD, has disclosed that he has received consulting fees from BeiGene, BMS/Celgene, Gilead/Kite, Incyte, Janssen, Karyopharm, Merck, MorphoSys, Regeneron, Takeda.
Associate Professor of Medicine
Division of Oncology
Department of Medicine
Washington University School of Medicine in St Louis
St Louis, Missouri
Mark A. Schroeder, MD, has disclosed that he has received consulting fees from AbbVie, Merck, and Takeda; funds for research support from Amgen, Celgene, Cellect, Fortis, Genentech, Incyte, Janssen, PBD Inc., Sanofi Genzyme, and Seattle Genetics and fees for non-CME/CE services from Astellas, Dova, Flatiron, GlaxoSmithKline, Incyte, Janssen, Novo Nordisk, Partners Therapeutics, Pfizer, and Sanofi Genzyme.
Professor
Department of Pediatrics
Weill Cornell Medicine
New York, New York
Sujit Sheth, MD, has disclosed that he has received funds for research support from Bristol-Myers Squibb/Celgene, DisperSol, and La Jolla and consulting fees from Agios, Bristol-Myers Squibb/Celgene, and CRISPR/Vertex.
Professor of Oncology
Division of Hematologic Malignancies
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland
B. Douglas Smith, MD, has disclosed that he has received consulting fees from Bristol-Myers Squibb/Celgene, Cellularity, Jubilant, Novartis, and Pfizer.
Professor
Division of Cancer Medicine
Department of Leukemia
The University of Texas MD Anderson Cancer Center
Houston, Texas
Srdan Verstovsek, MD, PhD, has disclosed that he has received funds for research support from AstraZeneca, Blueprint, Celgene, CIT BioPharma, Genentech, Gilead Sciences, Incyte, Italfarmaco, Novartis, NS Pharma, Pharma Essentia, Promedior, Protagonist Therapeutics, Roche, and Sierra Oncology and consulting fees from Bristol-Myers Squibb, Celgene, Constellation, Incyte, Novartis, and Sierra Oncology.
Professor of Oncology
Chief, Leukemia Service
Department of Medicine
Roswell Park Comprehensive Cancer Center
Buffalo, New York
Eunice S. Wang, MD, has disclosed that she has received consulting fees from AbbVie/Genentech, Astellas/Jazz, Bristol-Myers Squibb/Celgene/Kite, Gilead Sciences, GlaxoSmithKline, Kura Oncology, Mana, Novartis, Pfizer, Rafael, Stemline, and Takeda and fees for non-CME/CE services from Dava Oncology, Kura Oncology, Pfizer, and Stemline.
During the 2021 American Society of Hematology annual meeting (ASH 2021), important results from several key clinical trials in malignant and nonmalignant hematologic diseases will be reported. Below, experts have highlighted their most anticipated abstracts, which will be covered online as a part of CCO’s Independent Conference Coverage of ASH 2021. As ASH 2021 unfolds, remember to check the CCO website often for downloadable slidesets summarizing the data from these studies and more, and then again after the meeting for CME-certified online activities featuring expert analyses and perspectives on the clinical implications of the new data.
Top Picks: Acute and Chronic Leukemias
Eunice S. Wang, MD, and B. Douglas Smith, MD, have identified key studies for patients with leukemias at ASH 2021. In acute myeloid leukemia (AML), numerous studies are reporting results of venetoclax in combinations for patients with newly diagnosed or relapsed/refractory (R/R) AML, including interim results from a phase II trial of venetoclax plus decitabine in younger adult patients (<60 years of age) with newly diagnosed, European LeukemiaNet adverse-risk AML. Grenet and colleagues will report on the outcomes comparison from a multicenter retrospective study of CPX-351 vs hypomethylating agent plus venetoclax as frontline therapy in patients with AML. The abstract for that study indicates a favorable survival benefit favoring CPX-351 in the overall cohort and in clinically relevant subgroups. Yilmaz and colleagues will report on data from a study of quizartinib with decitabine and venetoclax in patients with FLT3-ITD–mutated AML showing activity in heavily pretreated and FLT3 inhibitor–exposed disease. Also, in FLT3-ITD–mutated AML, final response and survival endpoints will be presented from a multicenter phase Ib trial of venetoclax plus gilteritinib in R/R AML showing high rates of complete response plus complete responses with incomplete platelet recovery and FLT3 mutation clearance in most patients with prolonged survival. We will also see data from the phase III study of gilteritinib plus azacitidine vs azacitidine monotherapy for patients with AML and the FLT3 mutation ineligible for intensive induction therapy. Montesinos and colleagues will report data from the phase III AGILE trial, examining the safety and efficacy of the IDH1 inhibitor ivosidenib combined with azacitidine vs azacitidine plus placebo as first-line therapy in patients with AML and the IDH1 mutation.
In acute lymphoblastic leukemia (ALL), researchers will provide first results from the measurable residual disease–stratified, risk-adapted GMALL Trial 08/2013 in adults with newly diagnosed ALL/lymphoblastic lymphoma treated with multiple therapies.
Additional studies to watch in acute and chronic leukemias:
Top Picks: Lymphomas
John M. Burke, MD, and Peter Martin, MD, have selected numerous key studies in lymphomas to be presented at ASH 2021 with the potential to change the standard of care (SoC) for these diseases. For decades, R-CHOP has been the standard chemoimmunotherapy treatment for patients with advanced diffuse large B-cell lymphoma (DLBCL), and many attempts to improve upon R-CHOP have failed. The phase III POLARIX study is comparing polatuzumab vedotin with R-CHP (pola-R-CHP) vs R-CHOP in previously untreated DLBCL. Data from the abstract to be presented at ASH suggest an improvement in progression-free survival favoring pola-R-CHP with only modest changes in the toxicity profile, but no overall survival benefit was observed. According to the experts, the pola-R-CHP regimen will become a new standard option for patients with advanced DLBCL. Locke and colleagues will report the primary results from the phase III ZUMA-7 trial of axicabtagene ciloleucel CAR T-cell therapy vs SoC salvage chemotherapy and autologous stem cell transplant (ASCT) in patients with DLBCL who had relapsed within 12 months of initial chemoimmunotherapy and who were, therefore, at elevated risk of an unfavorable outcome from the latter approach. Data to be presented at ASH suggest improvement in the primary endpoint of event-free survival. The pivotal phase III TRANSFORM study, which has a similar design to that of ZUMA-7, is comparing the efficacy and safety of lisocabtagene maraleucel CAR T-cell therapy to SoC of chemoimmunotherapy followed by ASCT in patients with DLBCL who had relapsed within 12 months of initial chemoimmunotherapy. The abstract for that study also suggests improvement in event-free survival, progression-free survival, and complete response rate. Based on the encouraging results for these 2 large phase III studies of CAR T-cell therapy, experts are of the opinion that axicabtagene ciloleucel or lisocabtagene maraleucel therapy may become a new standard approach for patients whose DLBCL relapses within 1 year of their initial chemoimmunotherapy. Bishop and colleagues will report data from the phase III BELINDA trial of tisagenlecleucel CAR T-cell therapy vs SoC as second-line treatment in 322 patients with primary refractory or aggressive B-cell non-Hodgkin lymphoma who experienced progression within 12 months of first-line therapy.
In the setting of heavily pretreated follicular lymphoma (FL), Budde and colleagues will report promising results from a pivotal phase I/II study of mosunetuzumab, an anti-CD20 and anti-CD3 bispecific antibody used to redirect T-cells to eliminate malignant B-cells, in 90 patients with relapsed FL and a media of 3 previous therapies (range: 2-10). The abstract to be presented at ASH shows deep and durable remission rates with mosunetuzumab monotherapy, including in patients who had experienced disease progression within 24 months from start of initial therapy. Thieblemont and colleagues will present efficacy data from a phase II trial of tisagenlecleucel in 97 patients with R/R FL who had received at least 2 previous therapies or with disease relapse after ASCT. The abstract for that study reports a high (86%) and durable response rate in the 94 patients evaluable for the primary efficacy analysis. Finally, Lynch and colleagues will report on the efficacy and safety data from the phase II CITADEL-203 study of parsaclisib in 126 patients with R/R FL and at least 2 previous therapies.
Additional studies to watch in lymphomas:
Top Picks: Multiple Myeloma
Shaji Kumar, MD, and Sagar Lonial, MD, have selected several exciting studies in multiple myeloma (MM) at ASH 2021. These include an update from the phase II GRIFFIN trial in newly diagnosed MM, which assessed first-line daratumumab plus VRd vs VRd alone followed by ASCT and maintenance therapy with either daratumumab plus lenalidomide or lenalidomide alone. These data will reinforce the initial conclusions and provide longer-term insight into the use of daratumumab/lenalidomide maintenance. We will also see results from the phase III GMMG-HD7 study of isatuximab plus VRd vs VRd alone in ASCT-eligible patients with newly diagnosed MM, with the primary endpoint of measurable residual disease negativity after induction therapy.
Also to be presented at the meeting are results from the Spanish GEM2014MAIN trial evaluating ixazomib plus Rd vs Rd maintenance in newly diagnosed MM. In addition, Kaufman and colleagues will report on the safety and preliminary efficacy from the expansion cohort of the phase I/II study of venetoclax plus daratumumab and dexamethasone vs daratumumab plus bortezomib and dexamethasone in patients with t(11;14) translocation R/R MM.
As in recent years, multiple studies will present data for anti-BCMA–targeted therapies including a phase I/II trial showing deep and durable responses with the bispecific antibody REGN5458. With the influx of potential anti-BCMA therapies available for patients with R/R MM, additional novel targets are also being explored. Data will be presented from the phase I MonumenTAL-1 trial of single-agent talquetamab, a bispecific antibody that binds to GPRC5D in R/R MM. Data from a phase I trial will be presented and will show a first glimpse of a novel CAR T-cell therapy targeting GPRC5D, MCARH109, for R/R MM. We will also see updated results from the phase I trial of cevostamab, a FcRH5xCD3 bispecific antibody, which has continued to show clinically meaningful activity for patients with R/R MM. Also to be presented at the meeting are results from the dose-expansion portion of the phase I/II study of iberdomide, a potent novel cereblon E3 ligase modulator, in combination with dexamethasone in patients with heavily pretreated, triple class–exposed R/R MM.
Additional studies to watch in MM:
Top Picks: Myelodysplastic Syndromes/Myeloproliferative Neoplasms
Amy E. DeZern, MD, MHS, and Srdan Verstovsek, MD, PhD, have identified several highly anticipated studies in myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPNs) to be presented at ASH 2021. For MDS, Bernard and colleagues will present data on the molecular International Prognosis Scoring System that has been validated in 3675 patients with MDS. Of importance, this system incorporates molecular data formally for prognostication, which other MDS prognostic scoring systems have not. In addition, data will be presented from a phase Ib study of sabatolimab (MBG453), a novel immuno-myeloid therapy targeting TIM-3, plus hypomethylating agents in patients with very high–risk/high-risk MDS (N = 53) and newly diagnosed AML (N = 48). The abstract indicates that the combination of sabatolimab and hypomethylating agents is safe and effective with durable clinical responses seen in patients with very high–risk/high-risk MDS and newly diagnosed AML. We will also see data with CPX-351 (daunorubicin and cytarabine liposome for injection) in a phase I pilot study in transplant eligible, higher-risk patients with MDS (N = 19) and a phase II study in 31 patients with higher-risk MDS who were previously untreated with hypomethylating agents or chemotherapy and who were 70 years of age or younger. Finally, Sekeres and colleagues will report on the phase III PANTHER study of pevonedistat plus azacitidine vs azacitidine alone as first-line treatment in patients with higher-risk MDS/chronic myelomonocytic leukemia or AML with 20% to 30% marrow blasts.
In myelofibrosis, updated clinical and translational data will be presented from the phase II MANIFEST trial investigating pelabresib (CPI-0610) monotherapy in patients with advanced myelofibrosis who are intolerant/refractory to or ineligible for ruxolitinib and have a poor prognosis. In polycythemia vera (PV), Hoffman and colleagues will present data from a phase II trial of rusfertide (PTG-300) in 63 patients with PV who had at least 3 phlebotomies with or without concurrent cytoreductive therapy to maintain hematocrit <45% within the 24 week of treatment. Of importance, the primary goal of therapy for PV is to maintain hematocrit <45%, which is proven to decrease thrombotic risk and associated mortality in patients with PV. Data from this abstract indicate that, following initiation of rusfertide, therapeutic phlebotomy was essentially eliminated with hematocrit maintained consistently <45% with a reduction in red blood cell counts and an increase in mean corpuscular volume and mean corpuscular hemoglobin values. In myeloid/lymphoid neoplasms, Gotlib and colleagues will report data from the multicenter phase II FIGHT-203 trial of pemigatinib in adult patients with myeloid/lymphoid neoplasms, an 8p11 chromosomal abnormality, and at least 1 previous therapy. Of note, the abstract indicates a very high rate of complete and partial responses in this aggressive form of MPN.
Additional studies to watch in MDS/MPNs:
Top Picks: Nonmalignant Hematologic Disorders
Sujit Sheth, MD, and Mark A. Schroeder, MD, have identified key studies in hemoglobinopathies and graft-vs-host disease (GVHD) with the potential to change clinical practice. In hemophilia, highly anticipated results will be presented from a plenary session abstract from the multicenter phase III ATLAS-INH study of fitusiran, an investigational siRNA therapeutic targeting antithrombin, as prophylaxis in patients with hemophilia A or B (N = 57). Data from the abstract suggest that this study provides evidence to support an alternative, noncoagulation factor–based prophylactic treatment for patients with both FVIII and FIX inhibitors. There is currently one similar nonfactor–based treatment for FVIII deficiency—emicizumab—but no such approach for patients with FIX inhibitor patients. Moreover, the abstract reports good efficacy and a tolerable safety profile, without increased risk of thrombosis. In a related abstract, Srivastava and colleagues will present efficacy and safety data from the phase III ATLAS-A/B study of fitusiran prophylaxis vs on-demand treatment with factor concentrates in patients with hemophilia A or B without inhibitors. The study met its key primary endpoint of reducing annualized bleeding rate in the efficacy period (Day 29 after fitusiran first-dose up to Day 246), and secondary endpoints of reducing annualized spontaneous bleeding, annualized joint bleeding rate, and improved health-related quality of life in the treatment period. Experts comment that having additional options for the treatment of patients with hemophilia using FVIII and FIX inhibitors is a major step forward in advancing care.
In sickle cell disease (SCD), we will see data from the phase I study of etavopivat, an allosteric activator of pyruvate kinase-R, reporting a reduction in markers of inflammation, hypercoagulability, and tissue hypoxia, and for the phase I study of mitapivat (AG-348) in adult patients with SCD reporting evidence of increased ATP, increased hemoglobin levels, and decreased markers of hemolysis, suggesting that the red cells in these individuals are turning over less rapidly. Both of these studies provide evidence to support the proposed mechanism of increasing pharmacokinetic activity in red cells in patients with SCD. The need for additional treatment options for patients with SCD, used alone or in combination for potential additive or synergistic effects, will advance care and potentially increase life expectancy for these individuals.
Several new promising therapies for chronic and acute GVHD are advancing clinical development and are being reported at the ASH annual meeting. In chronic GVHD, Lee and colleagues will report on the safety and tolerability from a phase II of axatilimab, a CSF-1R humanized antibody, in 40 patients aged 6 years or older with active disease despite at least 2 prior lines of systemic therapy. The abstract for that study indicates an encouraging response rate of 66% in 25 of 38 patients evaluable for response. Koshy and colleagues report data from a phase II trial of abatacept, a recombinant fusion protein of CTLA-4 extra cellular domain linked to a modified Fc portion of human IgG1, in 39 patients with steroid-refractory chronic GVHD with multisystem involvement including skin, mouth, eyes, and gastrointestinal track. The abstract for that study also shows a promising response rate of 49% in 19 of 39 patients.
In paroxysmal nocturnal hematuria (PNH), Jang and colleagues will report on a phase II study of iptacopan (LN023), a novel, oral, selective, and potent first-in-class inhibitor of factor B, as monotherapy for patients with PNH. In a previous report, iptacopan was shown to effectively control intravascular and extravascular hemolysis, leading to rapid infusion-free improvement in hemoglobin levels in most patients treated. Data to be presented at ASH 2021 confirm those data and show that iptacopan leads to rapid and durable improvement in markers of hemolysis with a meaningful and sustainable clinical benefit based on improvement in hemoglobin. Data from this study support initiation of a phase III trial evaluating iptacopan as frontline therapy for patients with PNH.
Additional studies to watch in nonmalignant hematologic disorders:
Remember to Check the CCO Website Often During and After ASH!
These are just a few of the interesting and important abstracts selected by our expert faculty from ASH 2021. Downloadable slideset summaries of these studies and more will be available on our website as the data are released. After the meeting, comprehensive analyses by our expert faculty members will explore the clinical implications of the data in CME-certified text-based modules.
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