Posted inAllergy & Immunology Hematology-Oncology news

Precision Gene Correction via Prime Editing Restores NADPH Oxidase Activity in Patients with p47phox-Deficient Chronic Granulomatous Disease

A Phase 1/2 clinical trial demonstrates that PM359, an autologous prime-edited CD34+ cell therapy, successfully corrects the delGT mutation in NCF1, restoring essential microbicidal function in patients with p47-CGD without the off-target risks associated with traditional CRISPR-Cas9.
Precision Base-Editing and Universal CAR7 T Cells: Transforming the Treatment Landscape for Refractory T-Cell Acute Lymphoblastic Leukemia
Posted inHematology-Oncology news Oncology

Precision Base-Editing and Universal CAR7 T Cells: Transforming the Treatment Landscape for Refractory T-Cell Acute Lymphoblastic Leukemia

Recent clinical trials demonstrate the rapid evolution of CD7-targeted CAR T-cell therapies. From donor-derived models to universal base-edited cells, these innovations offer high remission rates and a viable bridge to transplant for patients with relapsed T-cell acute lymphoblastic leukemia.
Short-Course Ianalumab Combined with Eltrombopag: A Potential Shift Toward Sustained Remission in Immune Thrombocytopenia
Posted inHematology-Oncology Internal Medicine news

Short-Course Ianalumab Combined with Eltrombopag: A Potential Shift Toward Sustained Remission in Immune Thrombocytopenia

The VAYHIT2 Phase 3 trial demonstrates that adding the BAFF-R monoclonal antibody ianalumab to eltrombopag significantly extends treatment-free intervals and improves stable response rates in patients with relapsed or refractory immune thrombocytopenia (ITP) compared to eltrombopag alone.
Teclistamab plus Daratumumab Redefines Outcomes in Relapsed or Refractory Multiple Myeloma: A Paradigm Shift in Early-Line Intervention
Posted inHematology-Oncology news Oncology

Teclistamab plus Daratumumab Redefines Outcomes in Relapsed or Refractory Multiple Myeloma: A Paradigm Shift in Early-Line Intervention

The phase 3 MajesTEC-3 trial demonstrates that combining teclistamab with daratumumab significantly extends progression-free survival compared to standard therapies in patients with relapsed or refractory multiple myeloma, achieving high rates of complete response and minimal residual disease negativity.
Validated PRESEV Score Identifies Adults with Sickle Cell VOC at Low Risk for Acute Chest Syndrome — Implications for Safer Outpatient Management
Posted inHematology-Oncology news

Validated PRESEV Score Identifies Adults with Sickle Cell VOC at Low Risk for Acute Chest Syndrome — Implications for Safer Outpatient Management

An international prospective validation confirms the PRESEV score reliably identifies adult sickle cell patients hospitalized for vaso-occlusive crisis (VOC) who are at low risk for acute chest syndrome (ACS), enabling safer outpatient management strategies in selected patients.
Ziftomenib Shows Clinically Meaningful Activity in Heavily Pretreated NPM1‑Mutated AML — A Targeted Option for a Molecularly Defined Relapse Population
Posted inHematology-Oncology news

Ziftomenib Shows Clinically Meaningful Activity in Heavily Pretreated NPM1‑Mutated AML — A Targeted Option for a Molecularly Defined Relapse Population

In the phase II registration cohort of KOMET‑001, oral menin inhibitor ziftomenib achieved a CR/CRh rate of 22% (61% MRD negativity) and an ORR of 33% in relapsed/refractory NPM1‑mutated AML with manageable toxicity, supporting further study and potential use as a molecularly targeted salvage option.
RBC Transfusions in Early Sepsis Resuscitation: Common, Complex, and Potentially Harmful Above a 10 g/dL Threshold
Posted inAnesthesiology Critical Care Hematology-Oncology news

RBC Transfusions in Early Sepsis Resuscitation: Common, Complex, and Potentially Harmful Above a 10 g/dL Threshold

In a multicenter Korean sepsis cohort, early RBC transfusions were frequent and driven by illness severity. No overall 60‑day mortality difference was seen after propensity matching, but transfusion was associated with harm at hemoglobin ≥10 g/dL and a possible benefit below that threshold.
Mosunetuzumab + Polatuzumab Vedotin Outperforms R‑GemOx in Transplant‑Ineligible Relapsed/Refractory Large B‑Cell Lymphoma: Primary Results of the Phase III SUNMO Trial
Posted inHematology-Oncology news Oncology

Mosunetuzumab + Polatuzumab Vedotin Outperforms R‑GemOx in Transplant‑Ineligible Relapsed/Refractory Large B‑Cell Lymphoma: Primary Results of the Phase III SUNMO Trial

In the randomized phase III SUNMO trial, mosunetuzumab plus polatuzumab vedotin significantly improved response rates and progression‑free survival versus R‑GemOx in transplant‑ineligible relapsed/refractory large B‑cell lymphoma, with low rates of clinically significant cytokine release syndrome and improved patient‑reported outcomes.
Post‑transplant Cyclophosphamide with Sirolimus/Cyclosporine Dramatically Lowers Chronic GVHD After Unrelated Donor PBSC Transplant Without Raising Relapse Risk
Posted inHematology-Oncology news

Post‑transplant Cyclophosphamide with Sirolimus/Cyclosporine Dramatically Lowers Chronic GVHD After Unrelated Donor PBSC Transplant Without Raising Relapse Risk

In a randomized Phase II trial, replacing MMF with post‑transplant cyclophosphamide (PTCy) alongside sirolimus and cyclosporine in unrelated donor PBSC transplants markedly lowered chronic GVHD and improved 1‑year chronic GVHD‑free relapse‑free survival, without increasing relapse or early mortality, though severe infections rose.
Quizartinib Added to Standard Chemotherapy Improves Event-Free and Overall Survival in Newly Diagnosed FLT3‑ITD–Negative AML: Findings from the Phase II QUIWI Trial
Posted inHematology-Oncology news

Quizartinib Added to Standard Chemotherapy Improves Event-Free and Overall Survival in Newly Diagnosed FLT3‑ITD–Negative AML: Findings from the Phase II QUIWI Trial

In the randomized, double‑blind phase II QUIWI trial, adding quizartinib to standard induction/consolidation chemotherapy and single‑agent maintenance significantly improved event‑free survival and overall survival for adults 18–70 years with newly diagnosed FLT3‑ITD–negative AML versus placebo.
Ruxolitinib Improves Failure-Free and Overall Survival Versus Best Available Therapy in Steroid-Refractory Acute GVHD: Final 24‑Month REACH2 Analysis
Posted inHematology-Oncology news Transplantation

Ruxolitinib Improves Failure-Free and Overall Survival Versus Best Available Therapy in Steroid-Refractory Acute GVHD: Final 24‑Month REACH2 Analysis

The 24‑month final analysis of the phase III REACH2 trial shows ruxolitinib produced longer failure‑free survival, improved median overall and event‑free survival, and a longer duration of response versus best available therapy (BAT) in patients with steroid‑refractory acute GVHD.
Five-Year ZUMA-5 Follow-Up: Durable Remissions and Potential for Cure with Axicabtagene Ciloleucel in Relapsed/Refractory Follicular Lymphoma
Posted inHematology-Oncology news Oncology

Five-Year ZUMA-5 Follow-Up: Durable Remissions and Potential for Cure with Axicabtagene Ciloleucel in Relapsed/Refractory Follicular Lymphoma

Five-year outcomes from ZUMA-5 show axi-cel produces high response rates in relapsed/refractory indolent NHL, with 75% CR, median PFS ~62 months, durable remissions linked to early CAR T expansion and naïve T-cell product phenotype, and an acceptable long-term safety profile.