Hematology-Oncology
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Posttransplant Cyclophosphamide Appears to Neutralize the Adverse Effect of Older Unrelated Donor Age on Survival After Allogeneic HCT
A CIBMTR registry cohort (n=10,025) found donor age predicted worse overall survival with conventional calcineurin inhibitor prophylaxis but not with posttransplant cyclophosphamide (PTCy); attenuation was driven by reduced nonrelapse morta
Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes
The FANCOLEN‑1 trial shows that infusion of autologous FANCA‑corrected CD34+ cells without cytotoxic conditioning produced sustained engraftment and clinical improvement in a majority of treated patients with Fanconi anaemia‑A, with an acce
Breaking the Immortality of Cancer Cells: Israel’s Groundbreaking RNA-Targeting Drug
Israeli scientists have developed a novel small molecule drug that targets RNA structures maintaining cancer cell immortality, providing a promising new approach to treating hard-to-cure cancers with unparalleled precision.
Restrictive Red Blood Cell Transfusion Is Safe for Most Patients — Except in Neurocritical Care (and Some Bleeding Syndromes)
A Cochrane update (2025) of 69 randomized trials found restrictive RBC transfusion thresholds (typically Hb 7–8 g/dL) cut transfusion exposure ~42% without increasing 30‑day mortality overall, but liberal strategies improved long‑term neuro
Tailoring Transplant Conditioning and Maintenance Therapy in FLT3-ITD AML: Insights on NPM1 Mutations and Measurable Residual Disease
Posttransplant gilteritinib greatly benefits FLT3-ITD AML patients with NPM1 comutations, while myeloablative conditioning offers limited advantages over reduced-intensity conditioning in eradicating measurable residual disease.
Belantamab Mafodotin Plus Bortezomib and Dexamethasone Significantly Improves Overall Survival in Relapsed/Refractory Multiple Myeloma: Updated DREAMM-7 Results
The DREAMM-7 phase 3 trial shows that belantamab mafodotin combined with bortezomib and dexamethasone produces early, sustained, and statistically significant overall survival and deep, durable responses versus daratumumab-based therapy in
Understanding ‘Favism’: Diagnosis, Treatment, and Living with G6PD Deficiency
Favism, a hereditary enzyme deficiency causing red blood cell breakdown upon exposure to certain triggers like fava beans, is preventable with proper diagnosis and management. This article explores its clinical features, diagnosis, treatmen
CM313, a Novel Anti-CD38 Monoclonal Antibody, Demonstrates Promising Efficacy and Safety in Persistent or Chronic Primary Immune Thrombocytopenia: Insights from a Phase 2 Randomized Trial
The phase 2 trial shows that CM313 significantly improves platelet response rates and duration with manageable safety in adults with persistent or chronic primary immune thrombocytopenia.
Timely Opioid Administration in Pediatric Emergency Departments Reduces Hospitalizations for Acute Sickle Cell Pain
Prompt opioid delivery in pediatric EDs for sickle cell pain significantly lowers hospitalization rates, with the greatest benefit seen when the first dose is given within 60 minutes and the second dose within 30 minutes.
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
This study demonstrates the long-term effectiveness and safety of autologous gene therapy for treating ADA deficiency, showing sustained immune function and minimal adverse effects over a median follow-up of 7.5 years.
From Discovery to Breakthroughs: How Multiple Myeloma Survival Has Doubled in 20 Years
Over the past two decades, advances in therapy have doubled the median survival of multiple myeloma patients, transforming this once devastating cancer into a more manageable disease through targeted drugs and immune therapies.
Advances in Targeted and Gene Therapies: Promising Data on KRAS G12D Inhibitors, DR5 Agonists, and Ocular Gene Therapy
Recent clinical trials report encouraging tumor shrinkage with KRAS G12D inhibitor VS-7375, significant progression-free survival benefit in soft tissue sarcoma with ozekibart, and a novel gene therapy acquisition targeting wet AMD.
T-Cell Receptor Clonotypes as Prognostic Markers in Cutaneous T-Cell Lymphoma: Insights from Next-Generation Sequencing
This study identifies specific T-cell receptor clonotypes associated with aggressive subtypes and poorer survival in mycosis fungoides and Sézary syndrome, supporting TCR sequencing as a tool for refined risk stratification in cutaneous T-c
Eltrombopag in Newly Diagnosed Pediatric Immune Thrombocytopenia: A Breakthrough in Early Treatment Strategy
This randomized trial demonstrates that eltrombopag significantly improves sustained platelet response in children with newly diagnosed ITP, offering a potential early intervention option.
Ki-67 Dynamics During Neoadjuvant Chemotherapy: A Key to Better Breast Cancer Prognosis
This study highlights the prognostic significance of tumor Ki-67 changes during neoadjuvant treatment, offering potential for improved risk stratification and personalized therapy in breast cancer patients.
Restrictive Treatment Criteria Improve Prognosis Prediction in Chronic Lymphocytic Leukemia: Insights from the Brazilian Registry
A restrictive treatment initiation approach in CLL, endorsed by the Brazilian Group of CLL, identifies patients with higher risk more accurately and spares low-risk patients from unnecessary therapy, showing superior prognostic stratificati
Optimizing Treatment in Frail Elderly with Newly Diagnosed Multiple Myeloma: Insights from the IFM2017-03 Trial on Dexamethasone-Sparing Daratumumab and Lenalidomide Regimen
The IFM2017-03 phase 3 trial demonstrates that a dexamethasone-sparing regimen combining daratumumab with lenalidomide markedly improves progression-free survival in frail elderly multiple myeloma patients, without increasing serious advers
Clonal Hematopoiesis in Older Adults: Implications for Cardiovascular Risk, Bleeding, and Aspirin Use
This study reveals that clonal hematopoiesis of indeterminate potential (CHIP) in adults aged 70+ does not increase cardiovascular risk but is associated with heightened bleeding risk. Aspirin showed no additional benefits or harms for thos
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