Immune thrombocytopenia (ITP), resulting from antibody-mediated platelet destruction combined with impaired platelet production, is a rare cause of thrombocytopenia in both children and adults. The decision to treat newly diagnosed patients is based on several factors, including the desire to increase platelet count to prevent bleeding, induce remission, and improve health-related quality of life (HRQoL). At present, standard first-line therapy is corticosteroids. While this treatment does increase the platelet count in many patients, a high percentage still relapse after discontinuation of therapy. For this reason, alteration or intensification of first-line therapy that results in superior long-term remission rates is desirable. The objective of this review is to outline different upfront strategies for newly diagnosed patients with ITP in an effort to potentially enhance remission rates and prevent relapse, taking into account an assessment of the risks and benefits of each approach. We primarily focus on adults with ITP, highlighting pediatric data and practice when applicable.
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http://dx.doi.org/10.1016/j.blre.2021.100822 | DOI Listing |
J Clin Med
January 2025
College of Pharmacy, King Saud Bin Abdulaziz University for Health Sciences, Riyadh 11461, Saudi Arabia.
Owing to the growing use of immune checkpoint inhibitors (ICIs) in the treatment of cancer, a wide spectrum of toxicity has arisen among cancer patients. Yet, limited ICI toxicity-related research is currently conducted in our region. This is a retrospective observational study conducted on adult cancer patients who received at least one cycle of ICI single therapy.
View Article and Find Full Text PDFChildren (Basel)
January 2025
Division of Critical Care Medicine, Nicklaus Children's Hospital, 3100 SW 62nd Avenue, Miami, FL 33155, USA.
Thrombocytopenia frequently occurs in patients before, during, and after admission to Pediatric Intensive Care Units (PICUs). In critically ill children, it is often due to multifactorial causes and can be a sign of significant organ dysfunction. This review summarizes the potential causes/mechanisms of thrombocytopenia in acutely ill children, their identification, and treatments, with special attention paid to septic patients.
View Article and Find Full Text PDFAnn Hematol
January 2025
Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology- Oncology, Key Laboratory of Major Diseases in Children, Beijing Children's Hospital, National Key Discipline of Pediatrics (Capital Medical University, Ministry of Education, Capital Medical University, National Center for Children's Health, Beijing, 100045, China.
Mycoplasma pneumoniae (M. pneumoniae), as one of the susceptible pathogens during childhood, may lead to severe mycoplasmal pneumonia and affect platelet fluctuations. We prospectively collected data on persistent/chronic ITP children who were infected with M.
View Article and Find Full Text PDFCancer
February 2025
Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Background: Talabostat, an oral small molecule inhibitor of dipeptidyl peptidases (DPP4 and DPP8/9), has shown synergistic activity with immune checkpoint inhibitors in preclinical studies. This open label, phase 2 basket trial assessed the antitumor activity of combining talabostat and pembrolizumab (anti-programmed death-1 antibody) in advanced solid tumor patients.
Methods: The primary objective was assessment of dose-limiting toxicity (DLT) rates in the first six patients (lead-in stage) and response rate (efficacy stage; included cohort A [checkpoint inhibitor (ICI) naive] and cohort B [ICI pretreated]) for the study treatment using the Response Evaluation Criteria in Solid Tumors (RECIST) v1.
Eur J Pediatr
January 2025
Pediatric Hematology and Oncology, Liv Hospital, Gaziantep, Turkey.
Unlabelled: Spondyloenchondrodysplasia (SPENCD) is a rare genetic disorder characterized with skeletal dysplasia, immune dysregulation, and neurological impairment. Patients diagnosed with SPENCD at a single pediatric hematology center were included in the study. The patients' clinical characteristics, symptoms at presentation, imaging and laboratory results, and genetic analysis results were collected retrospectively from their files.
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