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Real world experience with cladribine tablets for multiple sclerosis at four academic multiple sclerosis centers.
Mult Scler Relat Disord
January 2025
John L Trotter Multiple Sclerosis Center, Washington University, St. Louis, Missouri, USA.
Background: Cladribine tablets (CladT) are a multiple sclerosis (MS) disease-modifying therapy (DMT) with safety and efficacy established in the CLARITY trial and extension. A better understanding of the role of CladT in real-world populations is needed, including the clinical and radiographic trajectories of persons with MS (PwMS) treated with CladT and how CladT compares to other MS DMTs.
Methods: PwMS receiving CladT at 4 tertiary MS centers were identified and characterized.
Improvements in quality of life of people with relapsing multiple sclerosis treated with cladribine tablets during the 2-year CLARIFY-MS study: a plain language summary.
Neurodegener Dis Manag
December 2024
oDepartment of Neurology and Center for Clinical Neuroscience, First Medical Faculty, Charles University, Prague, Czech Republic.
Development of a novel tool for individual treatment trials in mucopolysaccharidosis.
J Inherit Metab Dis
January 2025
Institute of Congenital Metabolic Diseases, Paracelsus Medical University, Salzburg, Austria.
Mucopolysaccharidosis (MPS) encompasses a group of genetic lysosomal storage disorders, linked to reduced life expectancy and a significant lack of effective treatment options. Immunomodulatory drugs could have the potential to be a relevant medical approach, as the accumulation of undegraded substances initiates an innate immune response, which leads to inflammation and clinical deterioration. However, immunomodulators are not licensed for this indication.
View Article and Find Full Text PDFTreatment Intensification With Either Fludarabine, AraC, G-CSF and Idarubicin, or Cladribine Plus Daunorubicin and AraC on the Basis of Residual Disease Status in Older Patients With AML: Results From the NCRI AML18 Trial.
J Clin Oncol
November 2024
University of Birmingham College of Medicine and Health, Birmingham, United Kingdom.
Purpose: To evaluate the survival benefit of chemotherapy intensification in older patients with AML who have not achieved a measurable residual disease (MRD)-negative remission.
Methods: Five hundred twenty-three patients with AML (median age, 67 years; range, 51-79) without a flow cytometric MRD-negative remission response after a first course of daunorubicin and AraC (DA; including 165 not in remission) were randomly assigned between up to two further courses of DA or intensified chemotherapy-either fludarabine, cytarabine, granulocyte colony-stimulating factor and idarubicin (FLAG-Ida) or DA with cladribine (DAC).
Results: Overall survival (OS) was not improved in the intensification arms (DAC DA: hazard ratio [HR], 0.
The modified melphalan and busulfan-based regimen combined with maintenance therapy improved the survival of patients with refractory/relapsed AML after allogeneic transplantation: middle-term outcome of a multicenter trial.
Am J Cancer Res
October 2024
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College Tianjin 300020, China.
Article Synopsis
- - Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is currently the best curative option for patients with refractory or relapsed acute myeloid leukemia (AML), but long-term survival rates are still not ideal; researchers are working to improve conditioning regimens like the new MCBC (Melphalan, Cladribine, Busulfan, Cyclophosphamide).
- - In a study involving 56 patients treated from July 2020 to January 2022, the 2-year overall survival rate was 60.7% and relapse-free survival rate was 57.1%, with a median follow-up of about 2.3 years.
- - Key findings highlighted that patients
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