Gary J. Schiller
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View article: <i>IDH2</i> mutation is associated with favorable outcome among older adults with newly diagnosed acute myeloid leukemia treated with hypomethylating agent-based therapy
<i>IDH2</i> mutation is associated with favorable outcome among older adults with newly diagnosed acute myeloid leukemia treated with hypomethylating agent-based therapy Open
Mutations of isocitrate dehydrogenase (IDH) are recurrent in newly diagnosed (ND) acute myeloid leukemia (AML) and the prevalence increases with age. The prognostic impact of IDH mutations in AML remains controversial. IDH inhibitors gener…
View article: A phase 1/1b open-label, dose escalation, first-in-human study to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-leukemic activity of the orally available clk inhibitor, BH-30236, in adults with R/R AML or HR-MDS
A phase 1/1b open-label, dose escalation, first-in-human study to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-leukemic activity of the orally available clk inhibitor, BH-30236, in adults with R/R AML or HR-MDS Open
Background: Dysregulated alternative splicing (AS) is hallmark of cancer which plays an important role in regulating proliferation, apoptosis, immune surveillance, and therapeutic resistance. Components of the spliceosome machinery are fre…
View article: Ziftomenib in combination with venetoclax and azacitidine in relapsed/refractory NPM1-m or KMT2A-r acute myeloid leukemia: Updated phase 1a/b safety and clinical activity results from KOMET-007
Ziftomenib in combination with venetoclax and azacitidine in relapsed/refractory NPM1-m or KMT2A-r acute myeloid leukemia: Updated phase 1a/b safety and clinical activity results from KOMET-007 Open
Introduction: Leukemogenesis is driven by nucleophosmin 1 mutations(NPM1-m) or lysine methyltransferase 2A rearrangements(KMT2A-r) in ~35–40% of acute myeloid leukemia (AML) cases. Nearly half of AML patients will develop relapsed/refracto…
View article: RAS mutations negate the favorable impact of NPM1 in older patients with newly diagnosed Acute Myeloid Leukemia treated with ven/HMA
RAS mutations negate the favorable impact of NPM1 in older patients with newly diagnosed Acute Myeloid Leukemia treated with ven/HMA Open
Background: Activating RAS mutations play an important role in the pathogenesis of acute myeloid leukemia (AML) and occur in approximately 15-20% of patients with newly diagnosed (ND) AML. Although RAS mutations do not independently impact…
Promising results from an ongoing Phase I multicenter study of senti-202, a first-in-class, CD33 and/or FLT3 & not endomucin (EMCN), selective off-the-shelf logic gated CAR NK cell therapy in adults with Relapsed/Refractory Acute Myeloid Leukemia (R/R AML) Open
Despite recent improvements in treatment, R/R AML is associated with poor outcomes. Novel well-tolerated therapies that can lead to measurable residual disease negative (MRD-) and durable complete remissions (CR) are urgently needed. SENTI…
View article: Associations between chronic kidney disease, proteinuria, and thrombotic risk in SCD
Associations between chronic kidney disease, proteinuria, and thrombotic risk in SCD Open
Background: Sickle cell disease (SCD) is characterized by early onset of frailty and age-related complications, including heart disease, hypertension, venous thromboembolism (VTE), and chronic kidney disease (CKD). While recent studies hav…
A phase 2, open-label study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of DISC-3405 in participants with polycythemia vera (PV) Open
Background and Significance: Polycythemia vera (PV) is a myeloproliferative neoplasm associated with pathologic erythrocytosis, most often driven by somatic mutations in the JAK2 gene. A key treatment goal is maintaining hematocrit (HCT) b…
View article: Efficacy and safety of loncastuximab tesirine and rituximab (Lonca-R) followed by dose-adjusted rituximab with etoposide, doxorubicin, cyclophosphamide, vincristine and prednisone (DA-R-EPOCH) in previously untreated high-risk diffuse large B-cell lymphoma (DLBCL): Preliminary results from a University of California Malignancy Consortium (UCHMC) Phase II trial, UCDCC#303.
Efficacy and safety of loncastuximab tesirine and rituximab (Lonca-R) followed by dose-adjusted rituximab with etoposide, doxorubicin, cyclophosphamide, vincristine and prednisone (DA-R-EPOCH) in previously untreated high-risk diffuse large B-cell lymphoma (DLBCL): Preliminary results from a University of California Malignancy Consortium (UCHMC) Phase II trial, UCDCC#303. Open
Background: Newly diagnosed DLBCL is a potentially curable malignancy. However, there are high-risk subsets that respond poorly to and have worse outcomes using standard first-line immunochemotherapy with rituximab, cyclophosphamide, doxor…
Optimizing capillary leak syndrome prevention and management in patients receiving tagraxofusp for blastic plasmacytoid dendritic cell neoplasm Open
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive, orphan hematologic malignancy characterized by cells expressing CD123 and other markers. The only drug approved for BPDCN is tagraxofusp, a first-in-class CD123-targete…
Emerging Therapies for Sickle Cell Disease: From Symptom Management to Curative Gene Therapy Open
Sickle cell disease (SCD) is a hereditary hemoglobinopathy caused by a point mutation in the β-globin gene, leading to the production of hemoglobin S and resulting in chronic hemolytic anemia, vaso-occlusion, and progressive organ damage. …
View article: Lower dose and weekly schedules of selinexor in multiple myeloma - updated evidence on safety and efficacy
Lower dose and weekly schedules of selinexor in multiple myeloma - updated evidence on safety and efficacy Open
Background Selinexor, a first-in-class, oral exportin-1 inhibitor, showed activity in penta-refractory multiple myeloma (MM) in early trial exploration; however, the side-effect profile of twice-weekly dosing led to hesitant incorporation …
View article: Final safety and efficacy results from a phase 1/2 study of tagraxofusp, a CD123-targeted therapy, for myelofibrosis
Final safety and efficacy results from a phase 1/2 study of tagraxofusp, a CD123-targeted therapy, for myelofibrosis Open
Patients with myelofibrosis (MF) who are resistant to or relapse after Janus kinase inhibitor (JAKi) therapy have limited treatment options and typically poor prognoses. CD123 is overexpressed in various myeloid malignancies, including MF.…
View article: Supplementary Data1 from Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in <i>FLT3</i>-ITD Mutant/<i>TP53</i> Wild-type Acute Myeloid Leukemias
Supplementary Data1 from Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in <i>FLT3</i>-ITD Mutant/<i>TP53</i> Wild-type Acute Myeloid Leukemias Open
Supplementary Data
View article: Data from Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in <i>FLT3</i>-ITD Mutant/<i>TP53</i> Wild-type Acute Myeloid Leukemias
Data from Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in <i>FLT3</i>-ITD Mutant/<i>TP53</i> Wild-type Acute Myeloid Leukemias Open
Purpose:Acute myeloid leukemia (AML) is characterized by frequent mutations in FMS-like tyrosine kinase 3 (FLT3), overexpression of murine double minute 2 (MDM2), and TP53 wild-type (WT). Monotherapies targeting FLT3 frequently result in t…
View article: Human cancer-targeted immunity via transgenic hematopoietic stem cell progeny
Human cancer-targeted immunity via transgenic hematopoietic stem cell progeny Open
Adoptive transfer of genetically engineered T cells expressing a tumor-antigen-specific transgenic T cell receptor (TCR) can result in clinical responses in a variety of malignancies. However, these responses are frequently short-lived, an…
View article: Risk Factors for Solid Organ Graft Failure and Death in Hematopoietic Cell Transplant Recipients Undergoing Solid Organ Transplantation: A Retrospective Center for International Blood and Marrow Transplant Research and Organ Procurement and Transplantation Network Study
Risk Factors for Solid Organ Graft Failure and Death in Hematopoietic Cell Transplant Recipients Undergoing Solid Organ Transplantation: A Retrospective Center for International Blood and Marrow Transplant Research and Organ Procurement and Transplantation Network Study Open
Background. There is a growing population of hematopoietic cell transplantation (HCT) survivors who later require a solid organ transplant (SOT). However, there are limited data on survival, risk factors (RFs) for SOT graft loss, and death…
View article: Risk Factors for Solid Organ Graft Failure and Death in Solid Organ Transplant Recipients Undergoing Hematopoietic Cell Transplantation: A Retrospective Center for International Blood and Marrow Transplant Research (CIBMTR) and Organ Procurement and Transplantation Network (OPTN) Study
Risk Factors for Solid Organ Graft Failure and Death in Solid Organ Transplant Recipients Undergoing Hematopoietic Cell Transplantation: A Retrospective Center for International Blood and Marrow Transplant Research (CIBMTR) and Organ Procurement and Transplantation Network (OPTN) Study Open
Background. There is a growing population of solid organ transplant (SOT) survivors who subsequently require a hematopoietic cell transplant (HCT), although there are limited data on survival, risk factors for SOT graft loss, and death in …
View article: Azacitidine, Venetoclax, and Revumenib for Newly Diagnosed <i>NPM1</i> -Mutated or <i>KMT2A</i> -Rearranged AML
Azacitidine, Venetoclax, and Revumenib for Newly Diagnosed <i>NPM1</i> -Mutated or <i>KMT2A</i> -Rearranged AML Open
PURPOSE Azacitidine and venetoclax is a standard frontline treatment regimen for newly diagnosed older adults with AML; however, long-term outcomes remain poor. Revumenib is an oral menin inhibitor with clinical activity in AML patients wi…
View article: A phase 1 study of IO-202, an anti-LILRB4 antibody, in chronic myelomonocytic leukemia and acute myeloid leukemia
A phase 1 study of IO-202, an anti-LILRB4 antibody, in chronic myelomonocytic leukemia and acute myeloid leukemia Open
IO-202 is a humanized immunoglobulin G1 monoclonal antibody with high affinity and specificity for leukocyte immunoglobulin-like receptor B4 (LILRB4; ILT3), which is predominantly expressed in monocytes and monocytic blasts. IO-202 induces…
View article: Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in <i>FLT3</i> -ITD Mutant/ <i>TP53</i> Wild-type Acute Myeloid Leukemias
Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition with Quizartinib and Milademetan in <i>FLT3</i> -ITD Mutant/ <i>TP53</i> Wild-type Acute Myeloid Leukemias Open
Purpose: Acute myeloid leukemia (AML) is characterized by frequent mutations in FMS-like tyrosine kinase 3 (FLT3), overexpression of murine double minute 2 (MDM2), and TP53 wild-type (WT). Monotherapies targeting FLT3 frequently result in …
View article: Three-year analysis of adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia treated with brexucabtagene autoleucel in ZUMA-3
Three-year analysis of adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia treated with brexucabtagene autoleucel in ZUMA-3 Open
Brexucabtagene autoleucel (brexu-cel) is an autologous anti-CD19 CAR T-cell therapy approved in the US to treat adults aged ≥18 years (≥26 years in the EU) with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL). Brexu-c…
View article: Olutasidenib demonstrates significant clinical activity in mutated <i>IDH1</i> acute myeloid leukaemia arising from a prior myeloproliferative neoplasm
Olutasidenib demonstrates significant clinical activity in mutated <i>IDH1</i> acute myeloid leukaemia arising from a prior myeloproliferative neoplasm Open
Summary Acute myeloid leukaemia (AML) arising from a myeloproliferative neoplasm (MPN) is more aggressive and less responsive to therapies compared to de novo AML. Olutasidenib, an oral small‐molecule inhibitor of mutated IDH1 (mIDH1), sho…
View article: IO-202, a Novel Anti-LILRB4 Antibody, with Azacitidine for Hypomethylating Agent-Naive Chronic Myelomonocytic Leukemia: Phase 1b Expansion Cohort Results
IO-202, a Novel Anti-LILRB4 Antibody, with Azacitidine for Hypomethylating Agent-Naive Chronic Myelomonocytic Leukemia: Phase 1b Expansion Cohort Results Open
Introduction Chronic myelomonocytic leukemia (CMML) is a rare and lethal hematological malignancy. Hypomethylating agents (HMAs), including azacitidine (AZA), are the only approved treatment for CMML, yet they offer complete remission (CR)…
View article: A Phase 1/1b Open-Label, Dose Escalation, First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Anti-Leukemic Activity of the Orally Available CDC-like Kinase Inhibitor, BH-30236, in Adults with R/R AML or HR-MDS
A Phase 1/1b Open-Label, Dose Escalation, First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Anti-Leukemic Activity of the Orally Available CDC-like Kinase Inhibitor, BH-30236, in Adults with R/R AML or HR-MDS Open
Background: Alternative splicing (AS) is a primary mechanism for mRNA transcript diversification and protein expression regulation. AS is a new hallmark of cancers including acute myelogenous leukemia (AML) and myelodysplastic syndrome (MD…
View article: R289, a Dual Irak 1/4 Inhibitor, in Patients with Relapsed/Refractory (R/R) Lower-Risk Myelodysplastic Syndrome (LR-MDS): Initial Results from a Phase 1b Study
R289, a Dual Irak 1/4 Inhibitor, in Patients with Relapsed/Refractory (R/R) Lower-Risk Myelodysplastic Syndrome (LR-MDS): Initial Results from a Phase 1b Study Open
Introduction: Interleukin receptor-associated kinases (IRAK) 1 and 4 are critical for downstream signaling of IL-1R family and most toll-like receptors (TLR), promoting proinflammatory cytokine production, bone marrow inflammation and cell…
View article: Combination of Olutasidenib and Azacitidine Induces Durable Complete Remissions in m<i>IDH1</i> Acute Myeloid Leukemia: A Multicohort Open-Label Phase 1/2 Trial
Combination of Olutasidenib and Azacitidine Induces Durable Complete Remissions in m<i>IDH1</i> Acute Myeloid Leukemia: A Multicohort Open-Label Phase 1/2 Trial Open
Introduction: Treatment of relapsed/refractory (R/R) acute myeloid leukemia (AML) remains a significant challenge as standard chemotherapies have limited efficacy and may be contraindicated in elderly or frail patients. Relapse and resista…