Management of symptoms and prevention of life-threatening hemorrhage in immune thrombocytopenia (ITP) must be balanced against adverse effects of therapies. Because current treatment guidelines based on platelet count are confounded by variable bleeding phenotypes, there is a need to identify new objective markers of disease severity for treatment stratification. In this cross-sectional prospective study of 49 patients with ITP and nadir platelet counts <30 × 109/L and 18 aged-matched healthy controls, we used susceptibility-weighted magnetic resonance imaging to detect cerebral microbleeds (CMBs) as a marker of occult hemorrhage. CMBs were detected using a semiautomated method and correlated with clinical metadata using multivariate regression analysis. No CMBs were detected in health controls. In contrast, lobar CMBs were identified in 43% (21 of 49) of patients with ITP; prevalence increased with decreasing nadir platelet count (0/4, ≥15 × 109/L; 2/9, 10-14 × 109/L; 4/11, 5-9 × 109/L; 15/25 <5 × 109/L) and was associated with longer disease duration (P = 7 × 10-6), lower nadir platelet count (P = .005), lower platelet count at time of neuroimaging (P = .029), and higher organ bleeding scores (P = .028). Mucosal and skin bleeding scores, number of previous treatments, age, and sex were not associated with CMBs. Occult cerebral microhemorrhage is common in patients with moderate to severe ITP. Strong associations with ITP duration may reflect CMB accrual over time or more refractory disease. Further longitudinal studies in children and adults will allow greater understanding of the natural history and clinical and prognostic significance of CMBs.
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http://dx.doi.org/10.1182/blood.2020004858 | DOI Listing |
Mol Imaging
January 2025
Nuclear Medicine Department, Montpellier University Hospital, University of Montpellier, Montpellier, France.
Immunologic thrombocytopenic purpura (ITP) is a condition that affects four to 18 per 100 000 children every year. In most cases, spontaneous remission occurs, but splenectomy may be proposed. Exploring the site of platelet sequestration can help to better predict potential poor responders to splenectomy, but In-radiolabeled platelet scintigraphy (IPS) can be difficult to perform in children with very few platelets.
View Article and Find Full Text PDFJ Immunother Precis Oncol
February 2025
Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Introduction: Uveal melanoma remains a disease with aggressive behavior and poor prognosis despite advances in clinical management. Because monotherapy with immune checkpoint inhibitors has led to limited improvement in response rates, combination with other agents that act on the biological basis of oncogenesis has been proposed as a possible therapeutic strategy.
Methods: We designed a phase 1b trial to test the safety and tolerability of selinexor in combination with immune checkpoint inhibitors in patients with advanced uveal melanoma.
Rev Med Liege
January 2025
Service d'Hématologie clinique, CHU 20 Août, Casablanca, Maroc.
We conducted a retrospective study of 83 cases of immune thrombocytopenia (IT) in patients under 20 years of age. The aim was to provide an overview of IT in our young patients. The median age was 10 years, with a predominance of females (71 %).
View Article and Find Full Text PDFJ Immunother Precis Oncol
February 2025
Section of Benign Hematology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Thrombotic thrombocytopenic purpura (TTP) is characterized by thrombotic microangiopathy resulting from decreased activation of the von Willebrand factor-cleaving protease (ADAMTS13). TTP can cause organ damage and is often fatal if the appropriate treatment is not started immediately. Although primary immune TTP is the most common form of TTP, secondary immune etiologies, including complications from immune checkpoint inhibitors (ICIs), have also been reported.
View Article and Find Full Text PDFBr J Clin Pharmacol
January 2025
Department of Pediatrics, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, China.
Aims: Research on hydroxychloroquine (HCQ) for children with chronic immune thrombocytopenia (ITP) is limited. The association between antinuclear antibody (ANA) positivity and its efficacy remains unclear.
Methods: This retrospective cohort study compared the clinical characteristics of children with chronic ITP who received HCQ with those who did not, as well as patients who responded to HCQ at 3 months with those who did not.
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