Long-Term Nirogacestat for Progressive Desmoid Tumors: Durable Responses, Continued Tumor Shrinkage, and a Manageable Safety Profile
Posted inHematology-Oncology news Oncology

Long-Term Nirogacestat for Progressive Desmoid Tumors: Durable Responses, Continued Tumor Shrinkage, and a Manageable Safety Profile

Extended follow-up from the phase III DeFi trial shows that continuous nirogacestat yields further tumor shrinkage, durable objective responses (ORR 45.7% at up to 4 years), sustained patient-reported benefits, and a consistent, manageable safety profile over a median 33.6 months of exposure.
Engasertib Shows Promise for Recurrent Epistaxis in HHT: Proof‑of‑Concept Trial Demonstrates Reduced Bleeding with Acceptable Short‑Term Safety
Posted inHematology-Oncology news Otorhinolaryngology

Engasertib Shows Promise for Recurrent Epistaxis in HHT: Proof‑of‑Concept Trial Demonstrates Reduced Bleeding with Acceptable Short‑Term Safety

In a randomized, double‑blind, placebo‑controlled proof‑of‑concept trial, oral engasertib reduced epistaxis frequency and duration in hereditary hemorrhagic telangiectasia (HHT) with a safety profile similar to placebo except for reversible rash and occasional hyperglycemia.
Ropeginterferon alfa-2b Superior to Anagrelide as Second-line Therapy in Hydroxyurea‑Intolerant or ‑Refractory Essential Thrombocythaemia: SURPASS‑ET Phase 3 Results
Posted inHematology-Oncology news

Ropeginterferon alfa-2b Superior to Anagrelide as Second-line Therapy in Hydroxyurea‑Intolerant or ‑Refractory Essential Thrombocythaemia: SURPASS‑ET Phase 3 Results

SURPASS‑ET, a phase 3 randomized trial, showed ropeginterferon alfa‑2b achieved durable ELN responses in 43% of hydroxyurea‑intolerant/refractory ET patients with leukocytosis versus 6% with anagrelide, with a favorable safety profile and fewer serious adverse events.
Fixed‑Duration Venetoclax After RBAC Improves 2‑Year PFS in High‑Risk Older Mantle Cell Lymphoma: Final Results of the FIL_V‑RBAC Phase 2 Study
Posted inHematology-Oncology news

Fixed‑Duration Venetoclax After RBAC Improves 2‑Year PFS in High‑Risk Older Mantle Cell Lymphoma: Final Results of the FIL_V‑RBAC Phase 2 Study

In a multicentre, risk‑adapted phase 2 trial, adding fixed‑duration venetoclax after rituximab‑bendamustine‑cytarabine (RBAC) produced a 2‑year progression‑free survival of 60% in older patients with high‑risk mantle cell lymphoma (MCL). Toxicities were manageable, neutropenia was most common, and one treatment‑related death occurred.
Nationwide China Study Reveals Age-Specific Burden and Survival Gaps in Acute Leukaemia: Good Outcomes for Children and APL; Poor Prognosis in Older Adults
Posted inHematology-Oncology news Oncology

Nationwide China Study Reveals Age-Specific Burden and Survival Gaps in Acute Leukaemia: Good Outcomes for Children and APL; Poor Prognosis in Older Adults

A large linkage of Chinese national registries (628.4 million population) estimates 43,275 acute leukaemia cases in 2019, describes age-specific incidence peaks in early childhood and older age, reports major survival gains for children and APL, and highlights dismal outcomes for patients ≥60 years.
IL‑10–Armored anti‑CD19 CAR T Cells for R/R B‑ALL: Promising Phase 1 Safety Signals and a New Strategy to Tame Inflammatory Toxicity
Posted inHematology-Oncology news

IL‑10–Armored anti‑CD19 CAR T Cells for R/R B‑ALL: Promising Phase 1 Safety Signals and a New Strategy to Tame Inflammatory Toxicity

A phase 1, single‑arm study evaluated IL‑10–expressing anti‑CD19 CAR T cells in relapsed/refractory B‑ALL, reporting feasibility and encouraging safety signals suggesting attenuated inflammatory toxicity while preserving anti‑leukemic activity. Larger controlled studies are needed to confirm benefit and infectious risk.
Metformin Triggers Ferroptosis in AML Through Lipid Remodeling: A Repurposing Opportunity for Metabolic Subtypes
Posted inHematology-Oncology news

Metformin Triggers Ferroptosis in AML Through Lipid Remodeling: A Repurposing Opportunity for Metabolic Subtypes

A new ex vivo study shows metformin induces ROS-driven ferroptosis in acute myeloid leukemia (AML), particularly in samples with altered lipid metabolism (IDH2, FLT3 mutations). Lipidomic remodeling, CD36-mediated fatty acid uptake, and DGAT1 activity determine sensitivity—suggesting biomarker-guided repurposing strategies and combination approaches.