Lurbinectedin, a novel antineoplastic agent, was granted the orphan drug designation by the United States Food and Drug Administration (US FDA) and approved for use in relapsed/refractory small cell lung cancer in June 2020. The approval was granted after its efficacy was demonstrated in a multicenter open-label, multi-cohort study enrolling 105 participants. Since then, real-world studies have examined the efficacy and safety profiles of lurbinectedin in clinical practice. By examining these outcomes, this review aims to provide clinicians with the tools necessary to make informed clinical decisions.
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| http://dx.doi.org/10.1080/1120009X.2024.2302736 | DOI Listing |
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Olutasidenib in combination with azacitidine induces durable complete remissions in patients with relapsed or refractory mIDH1 acute myeloid leukemia: a multicohort open-label phase 1/2 trial.
J Hematol Oncol
January 2025
Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL, USA.
Background: Olutasidenib is a potent, selective, oral, small molecule inhibitor of mutant IDH1 (mIDH1) which induced durable remissions in high-risk, relapsed/refractory (R/R) mIDH1 AML patients in a phase 1/2 trial. We present a pooled analysis from multiple cohorts of the phase 1/2 trial of patients with R/R AML who received combination olutasidenib and azacitidine therapy.
Methods: Adult patients with mIDH1 AML received 150 mg olutasidenib twice daily plus standard-of-care azacitidine (OLU + AZA) and were evaluated for response and safety.
Relapsed childhood T-cell acute lymphoblastic leukemia and lymphoblastic lymphoma.
Haematologica
January 2025
Division of Oncology, the Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pediatrics, the University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.
While outcomes for pediatric acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) have improved dramatically in recent decades, relapsed and refractory disease remain a significant therapeutic challenge. This is particularly true for patients with T-cell ALL and LBL, where survival for patients with relapsed/refractory disease remains dismal. Recent efforts to comprehensively profile the genomics of T-ALL/LBL to improve understanding of disease biology have enhanced our ability to identify high-risk patients at diagnosis who are more likely to relapse and have also identified novel targets for precision medicines.
View Article and Find Full Text PDFEpigenetic Role of Long Non-coding RNAs in Multiple Myeloma.
Curr Oncol Rep
January 2025
Department of Molecular Oncology, Cancer Institute (WIA), Chennai, TN, India.
Purpose Of The Review: This review aims to explore the pivotal role of long non-coding RNAs (lncRNAs) as epigenetic regulators in the pathogenesis of multiple myeloma (MM). Additionally, we have portrayed the dual role of lncRNAs in the epigenetic landscape of MM pathobiology.
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CXCR4-directed endoradiotherapy with [Lu]Pentixather added to total body irradiation for myeloablative conditioning in patients with relapsed/refractory acute myeloid leukemia.
Theranostics
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Department of Internal Medicine III, School of Medicine and Health, Technical University of Munich, Munich, Germany.
Article Synopsis
- Despite improvements in targeted therapy for Acute Myeloid Leukemia (AML), the prognosis remains poor, particularly for patients with relapsed or refractory disease.
- Allogeneic hematopoietic stem cell transplantation (alloSCT) is the main curative option for high-risk patients, but the best conditioning approach is still uncertain for those who are chemotherapy-refractory.
- A study on seven AML patients who received CXCR4-directed endoradiotherapy (ERT) combined with total body irradiation and chemotherapy prior to alloSCT showed promising outcomes, with 6 out of 7 patients achieving response and successful engraftment, offering insights into a potentially effective treatment strategy for advanced cases.
[The mechanisms and salvage treatment strategies underlying positive relapse following CD19 CAR-T cell therapy in B-acute lymphoblastic leukemia].
Zhonghua Xue Ye Xue Za Zhi
October 2024
Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Hubei Clinical Medical Center of Cell Therapy for Neoplastic Disease, Wuhan 430022, China.
Approximately 50% of patients suffering from relapsed/refractory B-acute lymphoblastic leukemia (R/R B-ALL), experience relapse within one year, with around 60% of these relapses being antigen-positive, despite the transformative impact of chimeric antigen receptor (CAR) T cell therapy. The mechanisms underlying relapse are primarily associated with tumor heterogeneity, CAR-T cell dysfunction, subopimal in vivo expansion and persistence, and an inhibitory immune microenvironment. This review aims to investigate salvage strategies designed to enhance outcomes for patients undergoing relapse or disease progression following the CAR-T cell therapy.
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