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An Expert’s Guide to ASH 2022: Preview of the Top Abstracts

Catherine M. Broome, MD

Associate Professor of Medicine
Lombardi Cancer Center
MedStar Georgetown University
Washington, DC

Catherine M. Broome, MD: consultant/advisor/speaker: Alexion, Incyte, Sanofi; researcher: Argenx.

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John M. Burke, MD

Associate Chair
US Oncology Hematology Research Program
Rocky Mountain Cancer Centers
Aurora, Colorado

John M. Burke, MD: consultant/advisor/speaker: AbbVie, Adaptive Biotechnologies, AstraZeneca, BeiGene, Bristol-Myers Squibb, Epizyme, Kura, Kymera, MorphoSys, Novartis, Nurix, Roche/Genentech, Seattle Genetics, TG Therapeutics, Verastem, X4 Pharmaceuticals; researcher: MorphoSys; speakers bureau: BeiGene, Seattle Genetics.

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Corey Cutler, MD, MPH, FRCPC

Associate Professor of Medicine
Harvard Medical School
Medical Director, Stem Cell Transplantation
Dana-Farber Cancer Institute
Boston, Massachusetts

Corey Cutler, MD, MPH, FRCPC: consultant/advisor/speaker: CSL Behring, CTI Biopharma, Equillium, Incyte, Janssen, Jazz, Mallinckrodt, Sanofi; other: Cimeio.

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David Dingli, MD, PhD

Professor of Medicine
Division of Hematology
Mayo Clinic College of Science and Medicine
Rochester, Minnesota

David Dingli, MD, PhD: consultant: Alexion, Apellis, Janssen, Novartis, Sanofi, Takeda; independent contractor: Sorrento Therapeutics.

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Amir T. Fathi, MD

Director, Leukemia Program
Massachusetts General Hospital
Associate Professor of Medicine
Harvard Medical School
Boston, Massachusetts

Amir T. Fathi, MD: consultant/advisor/speaker/: AbbVie, Agios, Amgen, Astellas, Bristol-Myers Squibb, Celgene, Daiichi Sankyo, Enclear, Foghorn, Forma, Genentech, Immunogen, Ipsen, Kite, Mablytics, MorphoSys, Novartis, Orum, Pfizer, PureTech, Servier, Takeda.

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Shaji K. Kumar, MD

Mark and Judy Mullins Professor of Hematological Malignancies
Myeloma Amyloidosis Dysproteinemia Group
Consultant, Division of Hematology
Mayo Clinic
Rochester, Minnesota

Shaji K. Kumar, MD: consultant/advisor (paid to institution): AbbVie, Amgen, Antengene, ArcellX, AstraZeneca, Beigene, Bluebird Bio, Bristol-Myers Squibb, Janssen, K36, Loxo Oncology, Oncopeptides, Roche/Genentech, Sanofi, Secura Biotherapeutics, Takeda, Trillium; researcher (paid to institution): AbbVie, Allogene, Amgen, AstraZeneca, Bristol-Myers Squibb, Carsgen, GlaxoSmithKline, Janssen, Novartis, Regeneron, Roche/Genentech, Takeda.

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Sagar Lonial, MD

Professor and Chair
Department of Hematology and Medical Oncology
Chief Medical Officer
Winship Cancer Institute of Emory University
Atlanta, Georgia

Sagar Lonial, MD: consultant/advisor/speaker: AbbVie, Bluebird, Bristol-Myers Squibb, Celgene, GlaxoSmithKline, Janssen, Pfizer, Takeda; other financial or material support: TG Therapeutics

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Peter Martin, MD

Associate Professor of Medicine
Department of Medicine, Division of Hematology-Oncology
Weill Cornell Medical College
New York, New York

Peter Martin, MD: consultant/advisor/speaker: ADC Therapeutics, AstraZeneca, BeiGene, Bristol-Myers Squibb, Daiichi Sankyo, Epizyme, Genentech, Gilead Sciences, Janssen, Takeda.

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Marshall Mazepa, MD

Assistant Professor of Medicine
Division of Hematology, Oncology and Transplantation
University of Minnesota
Minneapolis, Minnesota

Marshall Mazepa, MD: non-CME/CE services: Sanofi.

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Srdan Verstovsek, MD, PhD

Division of Cancer Medicine
Department of Leukemia
The University of Texas MD Anderson Cancer Center
Houston, Texas

Srdan Verstovsek, MD, PhD: researcher: Bristol-Myers Squibb, CTI, Incyte, PharmaEssentia, Protagonist, Roche, Sierra.

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Eunice S. Wang, MD

Professor of Oncology
Chief, Leukemia Service
Department of Medicine
Roswell Park Comprehensive Cancer Center
Buffalo, New York

Eunice S. Wang, MD: consultant/advisor/speaker: AbbVie, Amgen, Astellas, Bristol-Myers Squibb, Gilead Sciences, GlaxoSmithKline, Janssen, Jazz, Kite, Novartis, NuProbe, Pfizer, PharmaEssentia, Stemline.

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Allison Wheeler, MD, MSCI

Associate Professor
Department of Pathology, Microbiology, and Immunology
Department of Pediatrics
Vanderbilt University Medical Center
Nashville, Tennessee

Allison Wheeler, MD, MSCI: consultant/advisor/speaker: Bayer, Bioverativ, CSL Behring, HEMA Biologics, Novo Nordisk, Octapharma, Pfizer, Sanofi, Takeda.

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Amer Zeidan, MBBS, MPH

Associate Professor, Internal Medicine
Leader, Leukemia and Myeloid Disease Aligned Research Team (DART)
Director, Hematology Early Therapeutics Research
Yale Cancer Center and Smilow Cancer Hospital
Yale University School of Medicine
New Haven, Connecticut

Amer Zeidan, MBBS, MPH: researcher: AbbVie, ADC Therapeutics, Amgen, Aprea, Astex, Boehringer Ingelheim, Cardiff Oncology, Celgene/Bristol-Myers Squibb, Incyte, Medimmune/AstraZeneca, Novartis, Otsuka, Pfizer, Takeda, Trovagene; consultant: Acceleron, Agios, Amgen, Aprea, Astellas, Astex, BeyondSpring, Boehringer Ingelheim, Cardiff Oncology, Cardinal Health, Celgene/Bristol-Myers Squibb, Daiichi Sankyo, Epizyme, Gilead Sciences, Incyte, Ionis, Janssen, Jazz, Kura, Novartis, Otsuka, Pfizer, Seattle Genetics, Syndax, Taiho, Takeda, Trovagene, Tyme.

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Released: December 7, 2022

During the 2022 American Society of Hematology (ASH) Annual Meeting and Exposition, important results from many clinical trials will be reported. Below, experts have highlighted their most anticipated abstracts, which we will cover online as a part of CCO’s Independent Conference Coverage of ASH 2022. Remember to check the CCO website often as the meeting unfolds for downloadable slidesets summarizing the data from these studies and more, as well as after the meeting for CME-certified online activities featuring expert analyses and perspectives on the clinical implications of the new data.

Top Picks: Leukemia
At ASH 2022, several studies in leukemia are being presented as part of the general session or as late-breaking abstracts. Many other interesting studies also are being reported. Amir T. Fathi, MD, and Eunice S. Wang, MD, identified the following as the top abstracts to watch.

  • Magrolimab with venetoclax and azacitidine in older/unfit patients with newly diagnosed or relapsed/refractory (R/R) acute myeloid leukemia (AML) (Abstract 61). Magrolimab in combination with a hypomethylating agent (HMA) has been associated with high rates of marrow response and remission in newly diagnosed, older patients with myelodysplastic syndromes (MDS) and AML, with particular promise in patients with TP53 mutation. This phase I/II study of a triplet containing magrolimab, azacitidine, and venetoclax appears to yield an even higher rate of marrow response and remission, with a promising overall survival (OS) rate in a small cohort of patients with TP53 mutation.
  • KOMET-001: phase I/II study of ziftomenib in patients with R/R AML (Abstract 64). The emergence of menin inhibitors in clinical trials is quite exciting. These drugs appear to have marked activity in R/R patients with NPM1 or KMT2A alterations. Differentiation syndrome can be seen as an adverse event (AE) and needs to be closely monitored and managed.
  • Long-term follow-up of VIALE-A, a phase III trial of venetoclax/azacitidine in patients with untreated AML who are ineligible for intensive chemotherapy (Abstract 219). The VIALE-A long-term follow-up demonstrates sustained OS benefit with venetoclax/azacitidine in patients with AML ineligible for intensive chemotherapy compared with placebo plus azacitidine in all subgroups. Of note, the median OS for patients with measurable residual disease (MRD) <10-3 who had achieved either complete remission (CR) or CR with incomplete hematologic recovery was 34.2 months, and the median OS for patients with IDH1/2 mutations receiving venetoclax/azacitidine was 19.9 months. The VIALE-A follow-up analysis confirms the long-term survival benefit for patients receiving venetoclax/azacitidine, with no new safety findings.
  • GMALL-Initial1: inotuzumab ozogamicin induction followed by chemotherapy in de novo B-lymphoblastic leukemia (Abstract 212). The treatment of B-cell acute lymphoblastic leukemia (ALL) in older patients is challenging. Traditional intensive induction does not lead to long-term remissions and cures in the majority of patients. Incorporating the CD22 antibody–drug conjugate inotuzumab ozogamicin into frontline therapy is a promising strategy, and these emerging data suggest high levels of efficacy and impressive survival in patients older than 55 years of age.
  • ECOG-ACRIN E1910: randomized phase III trial of blinatumomab consolidation therapy in newly diagnosed adult B-lineage ALL in MRD-negative remission (Abstract LBA-1). Blinatumomab, when added to consolidation chemotherapy, resulted in a significant OS improvement in patients with newly diagnosed B-lineage ALL who were MRD negative after intensification chemotherapy with no new safety signals noted. These data suggest that the addition of blinatumomab to consolidation chemotherapy represents a new standard of care for patients with BCR::ABL1-negative B-lineage ALL who are 30-70 years of age.
  • Low-dose dasatinib as frontline therapy in newly diagnosed chronic myeloid leukemia (CML) (Abstract 619). After a median follow-up of 5 years, dasatinib 50 mg daily continues to be effective and safe in 83 patients with newly diagnosed chronic-phase CML. The 5-year event-free survival and OS rates were 97% and 98%, respectively. These data support low-dose dasatinib therapy as a reasonable approach in newly diagnosed CML.

Additional studies to watch in leukemias:

  • AUGMENT 101: updated data from the phase I study of revumenib in KMT2A-rearranged or NPM1 mutant AML (Abstract 63).
  • Updated results from a phase I/II study of venetoclax/azacitidine in combination with gilteritinib in patients with newly diagnosed or R/R FLT3-mutated AML who are ineligible for intensive chemotherapy (Abstract 831).
  • V-FAST Master: subgroup analysis by FLT3 mutation type in adult patients with newly diagnosed AML who received CPX-351 plus midostaurin (Abstract 1436).
  • Retrospective safety and efficacy analysis of reduced venetoclax exposure by limiting venetoclax administration to concurrent azacitidine administration days in adult patients with newly diagnosed AML (Abstract 222).
  • 2-cohort comparison within the Graall-2014/B study of blinatumomab during consolidation in adult patients with high-risk Philadelphia chromosome–negative precursor B-cell ALL (Abstract 211).
  • ASC4MORE: randomized, multicenter, open-label phase II study of asciminib in combination with imatinib in patients with chronic phase CML (Abstract 80).

Top Picks: Lymphoma/Chronic Lymphocytic Leukemia
John M. Burke, MD, and Peter Martin, MD, have identified several key studies in lymphoma and chronic lymphocytic leukemia (CLL) that you should check out at ASH 2022, including plenary and late-breaking abstracts.

  • MATRix/IELSG43: a phase III trial evaluating nonmyeloablative chemoimmunotherapy (CIT) vs high-dose chemotherapy (HDC) followed by autologous stem cell transplant (ASCT) in primary central nervous system (CNS) lymphoma (Abstract LBA-3). The results confirm a survival benefit with HDC-ASCT compared with CIT without ASCT, indicating that patients with primary CNS lymphoma deemed fit enough should undergo consolidation ASCT after induction.
  • TRIANGLE: a 3-arm phase III study in patients 65 years of age or younger with previously untreated advanced-stage mantle cell lymphoma (MCL) evaluating the addition of ibrutinib to standard-of-care CIT followed by ASCT and maintenance rituximab vs standard of care vs ibrutinib-containing treatment without ASCT (Abstract 1). The results show strong efficacy with the addition of ibrutinib during induction and as maintenance with or without ASCT compared with standard of care. Could this be the end of ASCT in MCL?
  • Pooled analysis evaluating MRD negativity as a surrogate endpoint of response to induction therapy in newly diagnosed, diffuse large B-cell lymphoma (Abstract 322). The detection of circulating tumor DNA by a new technology called PhasED-seq was better at predicting patient outcomes than standard end-of-treatment PET/CT, with the failure to achieve MRD negativity being highly predictive of relapse.
  • ALPINE: a phase III study evaluating zanubrutinib vs ibrutinib in R/R CLL/small lymphocytic lymphoma (SLL) (Abstract LBA-6). This final analysis showing improved progression-free survival (PFS) and safety with zanubrutinib compared with ibrutinib in patients with R/R CLL/SLL is the first demonstration of superiority of one Bruton tyrosine kinase inhibitor over another.

The following are additional studies to see in lymphoma and CLL:

  • A phase II trial evaluating acalabrutinib in combination with lenalidomide and rituximab with response-adapted modification of treatment intensity by real-time MRD monitoring in newly diagnosed MCL (Abstract 73). 
  • EPCORE NHL-2: a phase I/II trial evaluating SC epcoritamab in combination with rituximab and lenalidomide in patients with previously untreated (initial results: Abstract 611) and R/R (updated results: Abstract 609) follicular lymphoma.
  • SYMPHONY-1: preliminary findings from a phase 1b safety run-in trial of tazemetostat plus rituximab and lenalidomide in R/R follicular lymphoma (Abstract 954). 
  • SGN35-027 Part B: updated results from a phase II trial evaluating the combination of brentuximab vedotin, nivolumab, doxorubicin, and dacarbazine in patients with previously untreated, bulky stage I/II or stage III/IV classical Hodgkin lymphoma (Abstract 314).  
  • Updated results of a multicohort pilot study evaluating short-course brentuximab vedotin with doxorubicin, vinblastine, and dacarbazine chemotherapy ± consolidative radiation therapy in newly diagnosed, early-stage, unfavorable-risk Hodgkin lymphoma, including a report of the potential utility of baseline metabolic tumor volume and PET2 to identify patients at higher risk for treatment failure (Abstract 730).
  • CAPTIVATE: a 5-year follow-up of a patient cohort with confirmed, undetectable MRD who received continued ibrutinib vs placebo (fixed-duration treatment) in this phase II study evaluating ibrutinib plus venetoclax in previously untreated CLL/SLL (Abstract 92).
  • A phase II trial evaluating triplet therapy with acalabrutinib, venetoclax, and obinutuzumab in previously untreated CLL, including updated results for a cohort of patients with any genetic risk profile and initial results for a new cohort of patients with high-risk, TP53-mutated disease (Abstract 344).

Top Picks: Multiple Myeloma
In multiple myeloma (MM), Shaji K. Kumar, MD, and Sagar Lonial, MD, identified several interesting and potentially practice-changing studies to watch.

  • UK MCRI Myeloma XI: the results evaluating maintenance lenalidomide or observation following ASCT from the transplant-eligible arm of a multifactorial, adaptive-design phase III trial in patients with newly diagnosed MM (Abstract 570). The duration of lenalidomide maintenance therapy continues to be an important question based on the DETERMINATION study, which supports longer therapy in combination with ASCT. This analysis from the UK-based Myeloma XI trial seeks to better define optimal duration. 
  • DSMM XIII: The median 5.6-year follow-up results of a multicenter phase III trial comparing the safety and efficacy of continuous lenalidomide dexamethasone (Rd) vs Rd induction followed by intensity-reduced melphalan, ASCT, and lenalidomide maintenance in 348 patients aged 60-75 with newly diagnosed MM (Abstract 116). This long-term analysis suggested that PFS and OS were not superior with ASCT and lenalidomide maintenance vs continuous Rd.
  • IFN2017-03: a phase III trial of daratumumab lenalidomide (DR) vs lenalidomide dexamethasone (Rd) in frail patients with newly diagnosed MM older than 65 years of age (Abstract 569). The results at 12 months show an improved overall response rate (ORR) in the DR arm, with similar discontinuation for AEs in both arms. DR with limited use of dexamethasone is relevant for many older patients, for whom the ongoing use of dexamethasone is a significant challenge in care and AE management.
  • KarMMa-2: the results from cohort 2a of a multicenter phase II trial evaluating the efficacy and safety of idecabtagene vicleucel (ide-cel) CAR T-cell therapy in 37 patients with MM who experienced early relapse after frontline induction, ASCT, and lenalidomide-containing maintenance therapy (Abstract 361). The results demonstrate that even in this cohort of patients with functional high-risk disease, there is the potential benefit of the use of CAR T-cell therapy in earlier lines of treatment.
  • Real-world safety and survival analysis of ide-cel CAR T-cell therapy in 50 patients with heavily pretreated R/R MM who previously received B-cell maturation antigen (BCMA)–targeted therapy (Abstract 766). The results demonstrated lower response rates compared with those who had not received previous BCMA-targeted therapy, particularly those with previous BCMA-targeted treatment <6 months before ide-cel infusion. The timing of ide-cel CAR T-cell therapy administration following BCMA-targeted therapy warrants further inquiry. Additional studies, including a retrospective analysis of patients who previously received ide-cel or ciltacabtagene autoleucel (Abstract 250), are exploring outcomes with subsequent salvage regimens after BCMA-targeted therapy.

Additional studies to watch in MM:

  • ASCENT: a multicenter phase II trial of carfilzomib, lenalidomide, daratumumab, and dexamethasone in high-risk smoldering multiple myeloma (SMM) (Abstract 757).
  • GEM-CESAR: a post hoc update of a multicenter phase II trial evaluating MRD negativity 2 years after maintenance in high-risk SMM following an intensive treatment regimen and SCT (Abstract 118).
  • OPTIMUM/MUKnine: a phase II trial evaluating intensified post-SCT consolidation with daratumumab plus bortezomib, lenalidomide, and dexamethasone following induction with daratumumab, bortezomib, lenalidomide, cyclophosphamide, and dexamethasone in ultra high–risk newly diagnosed MM or primary plasma cell leukemia (Abstract 758). 
  • MonumenTAL-1: updated results for patients with R/R MM who were treated at recommended phase II doses from the phase I/II trial of talquetamab, a bispecific antibody targeting CD3 and GPRC5D receptors (Abstract 157).
  • CC-92480-MM-001: dose-expansion cohort results from a phase I/II trial of mezigdomide, a cereblon E3 ubiquitin ligase complex modulator, in combination with dexamethasone for heavily pretreated R/R MM (Abstract 568).
Top Picks: Myelodysplastic Syndromes/Myeloproliferative Neoplasms

At ASH 2022, several studies in MDS/myeloproliferative neoplasms (MPNs) will be presented.

In MPNs, Srdan Verstovsek, MD, PhD, has identified several key studies of interest.

  • MOMENTUM: updated results to be presented from this phase III trial of momelotinib vs danazol for patients with symptomatic, anemic myelofibrosis (MF) who were previously treated with a JAK inhibitor (Abstract 627). Previous results showed that momelotinib (a JAK1/2 and ACVR1 inhibitor) is associated with improved symptom, anemia, and spleen size control vs danazol. It will be important to understand the full scale of possible benefits and AEs with momelotinib, which may be approved in the upcoming year.
  • An analysis of changes in JAK2V617F molecular responses to ruxolitinib and the impact on clinical outcomes in patients with polycythemia vera (PV) or essential thrombocythemia (ET) (Abstract 741). Per the abstract, some patients achieved JAK2V617F molecular responses, and these responses were associated with improved outcomes. It has been hoped that molecular responses could act as a reliable prognostic indicator in ET and PV. Although studies of interferon in these MPNs demonstrated a possible connection between treatment-induced decrease of JAK2 allele burden and clinical outcomes, this has not been shown convincingly to date with other agents. The results of this study could lead us to revisit common goals of therapy for ET and PV and whether to incorporate the evaluation of molecular signatures with different therapies for these patients.
  • P1101MF: a phase II study of ropeginterferon alfa-2b for patients with Dynamic International Prognostic Scoring System low/intermediate-1–risk, prefibrotic primary MF (Abstract 629). Data show that ropeginterferon alfa-2b appears safe and effective in this population. The focus of clinical trials of therapeutics for MF has traditionally been on patients with higher-risk disease, but controlling thrombotic risk in patients with prefibrotic/low-risk disease could potentially prevent the progression of MF to the fibrotic/advanced stage and may benefit many patients. This study could open the door to examining novel therapeutic strategies for this often-forgotten patient group.

Additional studies to watch

  • The final results from a phase II study of parsaclisib (a PI3K delta inhibitor) as an add-on to ruxolitinib therapy for patients with MF and suboptimal responses to ruxolitinib (Abstract 236).
  • REFINE Cohort 3: data from a cohort of patients with JAK inhibitor–naive, intermediate-risk or high-risk MF in a phase II study of navitoclax (a BCL-XL/BCL-2 inhibitor) plus ruxolitinib (Abstract 237).
  • A phase I study of selinexor plus ruxolitinib for patients with treatment-naive MF (Abstract 1734).

In MDS, Amer Zeidan, MBBS, MPH, has identified the following abstracts to watch.

  • A study validating the 2022 WHO and International Consensus systems for genetic and morphologic MDS classification (Abstract 463).
  • A multicenter phase II trial evaluating the efficacy and safety of ruxolitinib for the treatment of 29 patients with symptomatic chronic myelomonocytic leukemia (Abstract 457). The abstract reports clinical benefit to ruxolitinib treatment.in 61 patients with low-risk MDS0del(5q) with anemia who were not transfusion dependent (Abstract 460). The abstract reports that low-dose lenalidomide lengthens the time to transfusion dependence.
  • STIMULUS-MDS1: a randomized, double-blind, placebo-controlled phase II trial evaluating the efficacy and safety of sabatolimab plus HMAs vs placebo plus HMAs in 127 patients with intermediate-risk, high-risk, or very high–risk MDS (Abstract 853). The abstract reports improvements in complete remission and PFS in the sabatolimab arm that were not statistically significant.

Top Picks: Nonmalignant Hematologic Disorders
At ASH 2022, several studies on nonmalignant hematologic disorders are being presented as part of the general session or as late-breaking abstracts. Corey Cutler, MD, MPH, FRCPC; David Dingli, MD, PhD; Marshall Mazepa, MD; Catherine M. Broome, MD; and Allison Wheeler, MD, MSCI, have identified the following as the top abstracts to watch.

  • ADVANCE IV: a multicenter, double-blind phase III clinical trial evaluating the efficacy and safety of IV efgartigimod vs placebo in adults with primary, persistent, or chronic immune thrombocytopenia (ITP; Abstract 3). Chronic ITP remains a challenging disorder to treat, with many patients who either fail to respond or lose their response to currently available therapy. Ergartigimod, a human IgG1 Fc-fragment, competitively binds to FcRn to prevent recycling of IgG and hence reduce pathogenic IgG autoantibodies. The results of this highly anticipated trial will be presented at the upcoming ASH meeting. The results will show that efgartigimod led to early and sustainable platelet recovery, including more weeks with platelet levels above ≥50×109/L, thus achieving the primary and secondary endpoints of the study. Efgartigimod could potentially become the first in a new class of therapies for ITP FcRn inhibitors, bringing an important new therapeutic option for patients with this challenging disease.
  • REACH4: the primary findings from a phase I/II study evaluating ruxolitinib in pediatric patients with treatment-naive or steroid-refractory acute graft-vs-host disease (GVHD; Abstract 572). The data confirm high ORRs and safety, as seen in the adult patient population.
  • BMT CTN 1703: a phase III study of post transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil vs tacrolimus/mycophenolate mofetil as treatment for GVHD in patients aged 18 years or older with hematologic malignancies and undergoing reduced-intensity conditioning allogeneic hematopoietic cell transplant (Abstract LBA-4). Based on the abstract for the study, the trial met its primary endpoint of achieving adjusted 1-year GVHD/relapse or PFS in this patient population.
  • Apply-PNH: a randomized, active comparator–controlled phase III study of monotherapy with iptacopan, a proximal complement inhibitor of factor B, vs IV terminal complement inhibition with eculizumab or ravulizumab in 97 adult patients with paroxysmal nocturnal hemoglobinuria and mean hemoglobin <10 g/dL who previously were receiving standard-of-care therapy with eculizumab or ravulizumab (Abstract LBA-2). The abstract for that study reports superior efficacy and a favorable safety profile in favor of iptacopan vs standard eculizumab or ravulizumab therapy.
  • CADENZA phase III study: the long-term effects analyses of biweekly doses of sutimlimab at 6.5 g (if <75 kg) or 7.5 g (if ≥75 kg) in patients with cold agglutinin disease (CAD) for up to 1 year (Abstract 31). The abstract reports sustained improvements in patient-reported outcomes and quality of life beyond 26 weeks in patients with CAD and 1 year or more after the initiation of sutimlimab.

Additional studies to watch in nonmalignant hematologic disorders:

  • Results from the phase III XTEND-1 study of weekly efanesoctocog alfa prophylaxis (arm A) on pain in adults and adolescents 12 years of age or older with severe hemophilia A previously receiving standard factor VIII prophylaxis (Abstract 2474).
  • Health-related quality of life results from the phase III ATLAS-PPX study comparing fitusiran prophylaxis vs prior prophylaxis with clothing factor concentrates or bypass agents in people with hemophilia A or B irrespective of inhibitor status (Abstract 3559).
  • An open-label, proof-of-concept phase II study evaluating the efficacy and safety of vemircopan, a complement factor D inhibitor, as monotherapy in patients with paroxysmal nocturnal hemoglobinuria (Abstract 294).
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