Objective: To investigate the factors affecting the chronicity of childhood primary immune thrombo-cytopenia (ITP) and compare the efficiency of different first-line treatment regimens.
Methods: Children with ITP hospitalized in our hospital from September 2013 to October 2018 were retrospectively analyzed.
Results: Three hundred and one children (150 males and 151 females) were included in this study, with a median age of 8 (0.17-17) years old, and 110 (36.5%), 92 (30.6%), and 99 (32.9%) cases were grouped into newly diagnosed, persistent, and chronic ITP, respectively. The median of follow-up was 41.92 (1.07-74.03) months. At the end of the follow-up (October 2019), among the 202 newly diagnosed/persistent ITP children, 79 cases (59 newly diagnosed and 20 persistent ITP) achieved remission within 1 year after initial diagnosis, with a remission rate of 39.3%; 122 cases (50 newly diagnosed and 72 persistent ITP) developed chronic disease, with a chronicity rate of 60.7%; one case underwent splenectomy. In 99 cases with chronic ITP, 5 cases underwent splenectomy. Multivariable logistic regression analysis showed that, the insidious onset of symptoms (OR=3.754, 95%CI: 1.882-7.488, P=0.000) increased the risk of chronicity, while the positive antibody to anti-platelet membrane glycoprotein (OR=0.446, 95%CI: 0.224-0.888, P=0.021) might reduce the risk of chronicity. And no difference was found by the analysis of subtype of anti-platelet membrane glycoprotein (P=0.305). The efficacy of the first-line treatment of intravenous immunoglobulin (IVIG) alone or combined with steroid was better than that of steroid alone (P=0.028, 0.028), however, the efficiency was not significantly different between IVIG alone and combined with steroid (P=0.086).
Conclusion: Insidious onset of symptoms in pediatric ITP increases the risk of chronicity, while the positive titer of anti-platelet membrane glycoprotein may reduce the risk. In the first-line treatment for the newly diagnosed/persistent children. The efficacy of IVIG alone or combined with steroid is better than that of steroid alone.
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http://dx.doi.org/10.19746/j.cnki.issn.1009-2137.2021.02.042 | DOI Listing |
J Transl Med
January 2025
Comprehensive Cancer Center, Department of Oncology, Nanjing Drum Tower Hospital Clinical College of Nanjing Medical University, 321 Zhongshan Road, Nanjing, 210008, China.
Objectives: GPC3 has been recognized as a promising target for immunotherapy in hepatocellular carcinoma (HCC). However, the GPC3-targeted immunotherapies have shown limited therapeutic efficacy. The use of anti-PD-1/PD-L1 monoclonal antibodies in HCC treatment is considerably constrained.
View Article and Find Full Text PDFMol Syst Biol
January 2025
Chair of Proteomics and Bioanalytics, Technical University of Munich, Freising, Germany.
The DNA-damaging agent Gemcitabine (GEM) is a first-line treatment for pancreatic cancer, but chemoresistance is frequently observed. Several clinical trials investigate the efficacy of GEM in combination with targeted drugs, including kinase inhibitors, but the experimental evidence for such rationale is often unclear. Here, we phenotypically screened 13 human pancreatic adenocarcinoma (PDAC) cell lines against GEM in combination with 146 clinical inhibitors and observed strong synergy for the ATR kinase inhibitor Elimusertib in most cell lines.
View Article and Find Full Text PDFClin Lymphoma Myeloma Leuk
December 2024
Incyte Corporation, Wilmington, DE.
Myelofibrosis (MF) is a rare myeloproliferative neoplasm characterized by progressive bone marrow fibrosis and splenomegaly. Ruxolitinib is the standard-of-care first-line treatment option for MF. This review summarizes real-world effectiveness and safety of ruxolitinib in more than 4500 patients with MF from real-world settings, including expanded-access and phase 4 trials, as well as registry, postmarketing, and retrospective studies in the 10 years since regulatory approval.
View Article and Find Full Text PDFBMJ Case Rep
January 2025
Medical Oncology, Alicante Institute for Health and Biomedical Research, Alicante, Comunidad Valenciana, Spain.
The increasing use of immune checkpoint inhibitors in clinical practice is associated with a broad spectrum of immune-related adverse events, such as cardiac, rheumatological and neurological toxicities. Myocarditis is a life-threatening complication, and the concurrent development of myocarditis, myositis and/or myasthenia leads to difficulties in diagnosis, management and treatment. We describe a case presenting with this triple M overlap syndrome following pembrolizumab treatment.
View Article and Find Full Text PDFWorld Neurosurg
January 2025
Department of Neurosurgery, Hospital Nacional Dos de Mayo, Lima, Peru.
Introduction: Microsurgery for paraclinoid aneurysms remains the first line of treatment in resource-constrained settings. The authors describe their institutional experience and evaluate functional outcomes after microsurgical treatment of paraclinoid aneurysms.
Methods: A retrospective review of clinical records was conducted.
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