Hematology-Oncology
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Hetrombopag vs. Thrombopoietin for Platelet Engraftment After Allogeneic Stem Cell Transplantation: New Evidence from a Multicenter Randomized Controlled Trial
Hetrombopag demonstrates comparable efficacy and safety to thrombopoietin for platelet engraftment after allogeneic hematopoietic stem cell transplantation, offering a promising alternative in hematologic care.
Intermediate-Dose Cytarabine Matches High-Dose Efficacy with Fewer Side Effects in Postinduction AML Therapy
A major RCT shows intermediate-dose cytarabine is noninferior to high-dose as postinduction therapy in AML, with similar survival and lower toxicity.
Bexmarilimab Plus Azacitidine in High-Risk Myelodysplastic Syndrome and Relapsed/Refractory Acute Myeloid Leukaemia: Phase 1 Dose-Escalation Results
The combination of bexmarilimab and azacitidine shows manageable safety and promising efficacy in high-risk myelodysplastic syndrome and relapsed/refractory acute myeloid leukaemia, supporting further investigation.
Avatrombopag in Pediatric Immune Thrombocytopenia: Evidence from the AVA-PED-301 Phase 3b Trial
The AVA-PED-301 phase 3b trial demonstrates avatrombopag’s efficacy and safety as an oral thrombopoietin receptor agonist for children and adolescents with persistent or chronic immune thrombocytopenia.
Mitapivat Offers New Hope for Adults with Non-Transfusion-Dependent α- and β-Thalassaemia: Results from the Phase 3 ENERGIZE Trial
Mitapivat significantly improves haemoglobin levels in adults with non-transfusion-dependent α- or β-thalassaemia, offering a promising oral therapeutic option with manageable safety profile.
Subcutaneous Blinatumomab in Relapsed/Refractory B-ALL: Clinical Promise and the Future of Delivery Modalities
Subcutaneous blinatumomab demonstrates promising efficacy and manageable safety in adults with relapsed/refractory B-cell acute lymphoblastic leukemia, with potential to shift future administration paradigms.
Haematopoietic Gene Therapy Without Conditioning for Fanconi Anaemia-A: Results and Clinical Implications from the FANCOLEN-1 Trials
Gene therapy using autologous, gene-corrected stem cells without conditioning offers sustained engraftment and reverses bone marrow failure in Fanconi anaemia-A, with a favorable safety profile.
Belantamab Mafodotin Plus Bortezomib and Dexamethasone in Relapsed or Refractory Multiple Myeloma: Updated Survival Outcomes from the DREAMM-7 Trial and Real-World Insights
The DREAMM-7 trial demonstrates that belantamab mafodotin with bortezomib and dexamethasone significantly improves survival in relapsed/refractory multiple myeloma, suggesting a new standard of care.
Fertility Outcomes in Advanced-Stage Classic Hodgkin Lymphoma: BrECADD Versus eBEACOPP in the HD21 Trial
The HD21 trial shows BrECADD offers superior gonadal function recovery and higher parenthood rates, especially in men, compared with eBEACOPP for advanced-stage classic Hodgkin lymphoma, supporting BrECADD as the preferred first-line therap
Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results
A single AAV gene therapy infusion for severe hemophilia B led to sustained factor IX expression, substantial reduction in bleeding and factor use, and no late safety concerns over 13 years.
Pirtobrutinib Demonstrates Superior Efficacy and Tolerability in cBTKi-Pretreated CLL/SLL: Insights from the BRUIN CLL-321 Phase III Trial
Pirtobrutinib significantly improves progression-free survival and tolerability over standard regimens in covalent BTK inhibitor-pretreated CLL/SLL, offering a promising new option for this challenging population.
Long-Term Outcomes of Cilta-Cel CAR-T Therapy in Relapsed/Refractory Multiple Myeloma: Five-Year Remission Achieved in One-Third of Patients
A recent J Clin Oncol study shows that nearly 33% of RRMM patients treated with cilta-cel achieved ≥5 years of progression-free survival, highlighting durable responses and promising biomarkers.
Advances in Graft-versus-Host Disease Prophylaxis: Cyclophosphamide and Cyclosporin in Allogeneic Stem Cell Transplantation
Post-transplant cyclophosphamide plus cyclosporin significantly improves GVHD-free, relapse-free survival compared to standard prophylaxis in matched related donor stem cell transplantation.
Mitochondrial Donation and Preimplantation Genetic Testing: Innovations in Reducing mtDNA Disease Transmission
Mitochondrial donation by pronuclear transfer and preimplantation genetic testing effectively reduce transmission of pathogenic mtDNA variants, offering hope for families at risk of mitochondrial diseases.
MRD-Guided Consolidation Therapy in Newly Diagnosed Multiple Myeloma: Insights from the MIDAS Phase 3 Trial
MRD-guided consolidation strategies in transplant-eligible multiple myeloma patients did not significantly improve deep remission rates over conventional approaches, questioning the clinical utility of MRD-adapted intensification.
Ivosidenib Plus Azacitidine Significantly Improves Survival in IDH1-Mutated AML: Long-Term Results from the AGILE Study
The AGILE trial shows ivosidenib plus azacitidine more than triples median overall survival in newly diagnosed IDH1-mutated AML patients ineligible for intensive chemotherapy, with improved hematologic recovery and safety.
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