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The Frontline and Relapsed Rhabdomyosarcoma (FaR-RMS) clinical trial is an overarching, multinational study for children and adults with rhabdomyosarcoma (RMS). The trial, developed by the European Soft Tissue Sarcoma Study Group (EpSSG), incorporates multiple different research questions within a multistage design with a focus on (i) novel regimens for poor prognostic subgroups, (ii) optimal duration of maintenance chemotherapy, and (iii) optimal use of radiotherapy for local control and widespread metastatic disease. Additional sub-studies focusing on biological risk stratification, use of imaging modalities, including [F]FDG PET-CT and diffusion-weighted MRI imaging (DWI) as prognostic markers, and impact of therapy on quality of life are described. This paper forms part of a Special Issue on rhabdomyosarcoma and outlines the study background, rationale for randomisations and sub-studies, design, and plans for utilisation and dissemination of results.
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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10931395 | PMC |
http://dx.doi.org/10.3390/cancers16050998 | DOI Listing |
Clin Lymphoma Myeloma Leuk
August 2025
Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, Ann Arbor, MI. Electronic address:
Blinatumomab is a bispecific T-cell engager that has recently transformed front-line treatment for many patients with Philadelphia chromosome (Ph)-negative B-cell acute lymphoblastic leukemia (B-ALL). It was originally studied in relapsed/refractory disease, then moved to targeting measurable residual disease (MRD), and has since been shown to improve outcomes for almost every age group when added to consolidation chemotherapy. The evidence supporting blinatumomab is most robust in adult and standard-risk pediatric age groups, but its benefit in adolescents and young adults and high-risk pediatric patients is not yet understood.
View Article and Find Full Text PDFExpert Opin Pharmacother
September 2025
Department of Haematology, Sir Charles Gairdner Hospital, Nedlands, WA, Australia.
Introduction: There have been recent major advances in the management and treatment of mantle cell lymphoma (MCL). This uncommon subtype of mature B-cell lymphoma has a heterogeneous clinical course, including a spectrum of indolent and aggressive disease. While historically regarded as an incurable disease with a poor long-term prognosis, recent developments have improved outcomes.
View Article and Find Full Text PDFAm J Hematol
September 2025
Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA.
Anaplastic lymphoma kinase-positive large B-cell lymphoma (ALK+ LBCL) is a rare, aggressive subtype of diffuse large B-cell lymphoma with poor outcomes using standard chemotherapy. In this multi-institutional retrospective study, we analyzed 39 cases of ALK+ LBCL identified at six US academic centers from 2002 to 2024, with treatment including conventional cytotoxic regimens in frontline and biologically informed and nonchemotherapy-based strategies in the relapsed setting. Ninety-two percent of patients received frontline anthracycline-based chemotherapy; 43% received intensified regimens, and 15% underwent upfront autologous stem cell transplantation (ASCT).
View Article and Find Full Text PDFAm J Hematol
September 2025
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Leuk Lymphoma
September 2025
Division of Hematology and Oncology, La Jolla, CA, USA.
Hairy cell leukemia (HCL) is a rare, indolent lymphoproliferative disorder that is highly responsive to purine analog therapy. While agents such as cladribine and pentostatin remain standard front-line therapies, their long-term toxicities-particularly myelosuppression and immune dysfunction-have led to growing interest in chemotherapy-free agents. Recent advances in molecular characterization of HCL, including the near-universal presence of BRAF V600E mutations, have led to an era of targeted therapeutics for the treatment of this disease.
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