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.