Clinical hematology international最新文献_第3页

Central nervous system involvement in pediatric hemophagocytic lymphohistiocytosis: A single-center descriptive study of clinical features, neurodiagnostic findings, and outcomes. 小儿嗜血球淋巴组织细胞增多症涉及中枢神经系统：临床特征、神经诊断结果和结果的单中心描述性研究。

Clinical hematology international Pub Date : 2026-02-19 eCollection Date: 2026-01-01 DOI: 10.46989/001c.155719

Manan Nath, Anshul Vagrecha, Annie H Roliz, Yash D Shah, Robin Varughese, Ramya Trietel, Alan Johnson, Carolyn Fein-Levy, Sanjeev V Kothare

{"title":"Central nervous system involvement in pediatric hemophagocytic lymphohistiocytosis: A single-center descriptive study of clinical features, neurodiagnostic findings, and outcomes.","authors":"Manan Nath, Anshul Vagrecha, Annie H Roliz, Yash D Shah, Robin Varughese, Ramya Trietel, Alan Johnson, Carolyn Fein-Levy, Sanjeev V Kothare","doi":"10.46989/001c.155719","DOIUrl":"10.46989/001c.155719","url":null,"abstract":"Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening hyperimmune condition triggered by the pathological activation of T-cells, natural killer cells, and macrophages leading to a cytokine storm with widespread hyperinflammation affecting multiple organ systems, including the brain. This retrospective chart review characterizes the central nervous system (CNS) features in 22 pediatric HLH patients at a single center. CNS involvement was determined based on symptoms, exam, abnormal cerebrospinal fluid (CSF) studies, neuroimaging, and electroencephalogram (EEG) findings. Twenty-two children with HLH were analyzed, classifying them into primary HLH (pHLH) and secondary HLH (sHLH) based on genetic testing. Of 20 patients who underwent genetic testing, 6 (30%) had pathogenic pHLH mutations, while 14 were sHLH (8 with variants of unknown significance, 6 with no variants). CNS involvement was noted in 17 (77.2%) patients. Symptoms included generalized weakness, altered sensorium, seizures, and headaches. MRI abnormalities included patchy T2 FLAIR prolongation (85%) with and without contrast enhancement and subdural collection, diffuse brain atrophy (40%), microhemorrhages (15%) and diffusion restriction (15%) patients. CSF studies showed higher WBC counts in pHLH . Therapies amongst this cohort varied, with 47% receiving dexamethasone, etoposide and cyclosporine, and 29% undergoing HSCT. Mortality in the first year was 18%, with 75% of deaths involving patients with CNS involvement of HLH. Mean survival at six months was 167 days, with no further deaths in the 12-year follow-up. We conclude that frequent neurological abnormalities are noted in children with HLH highlighting the role for active surveillance of CNS involvement in this group of patients.","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 1","pages":"26-36"},"PeriodicalIF":0.0,"publicationDate":"2026-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12923291/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147273203","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Factor XIII Deficiency: A Review of Biology, Testing, and Treatment. 因子13缺乏：生物学、检测和治疗综述。

Clinical hematology international Pub Date : 2026-01-20 eCollection Date: 2026-01-01 DOI: 10.46989/001c.155180

Jeremy W Jacobs, Garrett S Booth, Victoria Costa, Cristina A Figueroa Villalba, Bipin N Savani, Brian D Adkins

{"title":"Factor XIII Deficiency: A Review of Biology, Testing, and Treatment.","authors":"Jeremy W Jacobs, Garrett S Booth, Victoria Costa, Cristina A Figueroa Villalba, Bipin N Savani, Brian D Adkins","doi":"10.46989/001c.155180","DOIUrl":"10.46989/001c.155180","url":null,"abstract":"Factor XIII (FXIII) deficiency is a rare bleeding disorder characterized by unstable hemostatic clots due to defective fibrin cross‑linking. Congenital FXIII deficiency arises from variants in the F13A1 (FXIII-A subunit) or F13B (FXIII-B subunit) genes, and classically presents with delayed umbilical stump hemorrhage, soft‑tissue and intracranial bleeding, impaired wound healing, and recurrent pregnancy loss. Acquired deficiency stems from inhibitory autoantibodies or from reduced synthesis or consumption in critical illness and surgery. Routine coagulation screening tests are normal and diagnosis relies on quantitative FXIII activity assays with or without antigenic phenotyping and, when indicated, inhibitor testing and molecular confirmation. Plasma‑derived FXIII concentrate reduces spontaneous and intracranial bleeding; recombinant FXIII‑A2 is appropriate for F13A1 defects but not patients with F13B variants. Perioperative and obstetric care target activity thresholds suited to procedural risk and individual pharmacokinetics. This review synthesizes the molecular biology, epidemiology, clinical features, diagnostic methods, and evidence‑based management of FXIII deficiency, with practical guidance for assay selection, validation, and result interpretation.","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 1","pages":"10-25"},"PeriodicalIF":0.0,"publicationDate":"2026-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12825037/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146055306","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Ciclosporin as upfront therapy in Subcutaneous Panniculitis-like T-cell Lymphoma with aggressive features and in uncomplicated disease: Two case reports and literature review. 环孢素作为侵袭性无并发症的皮下泛膜炎样t细胞淋巴瘤的前期治疗：2例报告和文献综述

Clinical hematology international Pub Date : 2026-01-05 eCollection Date: 2026-01-01 DOI: 10.46989/001c.154639

Brandon K J Tan, Kiat Hoe Ong, Yong Howe Ho, Lip Leong Chong, Christian A Gallardo

引用次数: 0

Clinical Warburg Effect in a primary cutaneous lymphoma. 原发性皮肤淋巴瘤的临床Warburg效应。

Clinical hematology international Pub Date : 2026-01-02 eCollection Date: 2026-01-01 DOI: 10.46989/001c.154811

Francisco Martins, Guilherme Fontinha, José Magalhães, José Carlos Cardoso, Joana Calvão

引用次数: 0

Spleen and Bruton's tyrosine kinase inhibitors for the management of adult immune thrombocytopenia: A systematic review and meta-analysis of randomized controlled trials. 脾脏和布鲁顿酪氨酸激酶抑制剂治疗成人免疫性血小板减少症：随机对照试验的系统回顾和荟萃分析

Clinical hematology international Pub Date : 2025-12-24 eCollection Date: 2025-01-01 DOI: 10.46989/001c.154588

Akhil D Vatvani, Timotius I Hariyanto, Alejandrina C Ramirez, Nehad Shabarek

{"title":"Spleen and Bruton's tyrosine kinase inhibitors for the management of adult immune thrombocytopenia: A systematic review and meta-analysis of randomized controlled trials.","authors":"Akhil D Vatvani, Timotius I Hariyanto, Alejandrina C Ramirez, Nehad Shabarek","doi":"10.46989/001c.154588","DOIUrl":"10.46989/001c.154588","url":null,"abstract":"Spleen tyrosine kinase (SYK) and Bruton's tyrosine kinase (BTK) inhibitors have emerged as promising targeted therapies for adult immune thrombocytopenia (ITP). However, a comprehensive synthesis of their benefits compared to placebo has not been previously conducted. This study aims to analyze the efficacy and safety of SYK and BTK inhibitors in comparison to placebo for the treatment of adult patients with ITP. A systematic search was performed across four major databases (Medline, Europe PMC, Scopus, and ClinicalTrials.gov) for randomized controlled trials (RCTs) evaluating SYK and/or BTK inhibitors versus placebo in adults with ITP. We utilized random-effects models to evaluate the risk ratio (RR) and mean difference (MD) for the occurrence of outcomes. Five RCTs across four publications were included. Both SYK and BTK inhibitors significantly improved overall platelet response (RR 3.95; 95%CI: 2.68 - 5.81, p<0.00001) and durable response rates (RR 12.50; 95%CI: 3.99 - 39.18, p<0.0001) compared to placebo. Treatment also resulted in shorter time to response and reduced need for rescue interventions. Although total AEs and rash were more common in the intervention group, these were predominantly grade 1-2 and did not lead to increased discontinuation or serious AE rates. No significant differences were observed in the specific events such as gastrointestinal symptoms, cytopenias, liver enzyme elevations, infections, or hypertension. SYK and BTK inhibitors demonstrate superior efficacy over placebo in improving platelet outcomes in adult ITP without compromising safety. These findings support their role as effective treatment options, especially for patients unresponsive to other therapies.","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 4","pages":"76-91"},"PeriodicalIF":0.0,"publicationDate":"2025-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12742321/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145851690","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Clonal hematopoiesis at the crossroads of Inflammaging and cardiovascular disease: Mechanistic insights and translational horizons. 在炎症和心血管疾病的十字路口克隆造血：机制的见解和翻译的视野。

Clinical hematology international Pub Date : 2025-12-23 eCollection Date: 2025-01-01 DOI: 10.46989/001c.146275

Soumiya Nadar, Taha K Dohadwala, Nitish Kumaresan, Shabbeer I Ahamed, Sumia Fatima

{"title":"Clonal hematopoiesis at the crossroads of Inflammaging and cardiovascular disease: Mechanistic insights and translational horizons.","authors":"Soumiya Nadar, Taha K Dohadwala, Nitish Kumaresan, Shabbeer I Ahamed, Sumia Fatima","doi":"10.46989/001c.146275","DOIUrl":"10.46989/001c.146275","url":null,"abstract":"Clonal Hematopoiesis of indeterminate potential (CHIP) has been increasingly recognised as a risk factor for cardiovascular disease (CVD). Recent epidemiological and experimental studies have linked CHIP as an independent risk factor for myocardial infarction, stroke, and coronary artery disease, with specific mutations carrying a higher CVD risk. During the aging process, somatic mutations in genes, including DNMT3A and TET2, accumulate in the hematopoietic stem cells (HSCs), conferring both epigenetic and metabolic advantages that not only drive hematopoiesis towards a pro-inflammatory myeloid lineage but also reprogram innate immune cells, promoting a persistent inflammatory state. These myeloid derivatives, via increased IL-1β and IL-6 production, establish a pro-atherogenic environment and contribute to plaque instability, leading to an increased thrombotic risk and accelerated vascular aging. Although routine screening is not recommended for asymptomatic adults, targeted detection in high-risk individuals could benefit from preventive strategies. Incorporating CHIP into risk models may enable precision prevention, but prospective trials are needed to determine whether CHIP-guided interventions improve cardiovascular outcomes.","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 4","pages":"54-63"},"PeriodicalIF":0.0,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12738160/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145851704","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Real-world outcomes in patients with relapsed and refractory multiple myeloma with prior proteasome inhibitor and lenalidomide exposure: A single-center study in Sweden. 既往有蛋白酶体抑制剂和来那度胺暴露的复发和难治性多发性骨髓瘤患者的现实世界结局：瑞典的一项单中心研究

Clinical hematology international Pub Date : 2025-12-23 eCollection Date: 2025-01-01 DOI: 10.46989/001c.146250

Vincent Luong, Muhammad Kashif, Katarina Uttervall, Anna Bohlin, Annette Öster Fernström, Ying Qu, Seina Lee, João Mendes, Sandra Van Hoorenbeeck, Eva Hellqvist Franck, Jianming He, Evren Alici, Johan Lund

{"title":"Real-world outcomes in patients with relapsed and refractory multiple myeloma with prior proteasome inhibitor and lenalidomide exposure: A single-center study in Sweden.","authors":"Vincent Luong, Muhammad Kashif, Katarina Uttervall, Anna Bohlin, Annette Öster Fernström, Ying Qu, Seina Lee, João Mendes, Sandra Van Hoorenbeeck, Eva Hellqvist Franck, Jianming He, Evren Alici, Johan Lund","doi":"10.46989/001c.146250","DOIUrl":"10.46989/001c.146250","url":null,"abstract":"The use of proteasome inhibitors (PI) and immunomodulatory drugs (IMiD) such as lenalidomide (Len) in early lines of therapy in multiple myeloma (MM) has resulted in a high proportion of patients with relapsed or refractory multiple myeloma (RRMM) being Len-refractory. Len refractoriness is associated with inferior outcomes, constituting an unmet need in clinical practice. This retrospective single-center study aimed to provide real-world characteristics and outcomes in RRMM previously exposed to PI and Len during 1-3 prior lines of therapy (LOT), stratified by Len refractoriness. RRMM patients between Jan 2017 and May 2023 were included. We studied clinical characteristics, treatments and survival outcomes. A total of 218 patients were included (n=85 Len refractory). The median progression-free survival was 13.6 months in Len-refractory versus 14.9 months in Len non-refractory patients. The median overall survival was 20.3 months in Len refractory, and not reached in Len non-refractory patients. The overall response rates to the subsequent LOT were 58.8% and 61.7% in Len refractory and non-refractory cohorts, respectively. IMiD-based and anti-CD38 monoclonal antibody-containing regimens were the most frequent subsequent LOT, 44% and 41%, respectively. This study shows suboptimal outcomes in Len refractory RRMM patients, highlighting the need to develop effective treatment options.","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 4","pages":"64-75"},"PeriodicalIF":0.0,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12739867/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145851696","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Ramadan fasting and hematological disorders: clinical considerations, risks, and management strategies. 斋月禁食和血液病：临床考虑、风险和管理策略。

Clinical hematology international Pub Date : 2025-12-11 eCollection Date: 2025-01-01 DOI: 10.46989/001c.150393

Abdulrahman Nasiri, Ahmed Alahmadi, Manal Alshammari, Ruba Alabiri, Sara Samarkandi, Huda Alfattah, Rawan Alqahtani, Tariq Alzahrani, Hadeel Samarkandi, Abeer Habash, Eysa Alsolamy, Hamad Al Ghethber, Reem Alkharras, Ghada Makhdoum, Mostafa F Mohammed Saleh, Amr Hanbali, Riad El Fakih, Hazza Alzahrani, Ali Alahmari, Mahmoud Aljurf

{"title":"Ramadan fasting and hematological disorders: clinical considerations, risks, and management strategies.","authors":"Abdulrahman Nasiri, Ahmed Alahmadi, Manal Alshammari, Ruba Alabiri, Sara Samarkandi, Huda Alfattah, Rawan Alqahtani, Tariq Alzahrani, Hadeel Samarkandi, Abeer Habash, Eysa Alsolamy, Hamad Al Ghethber, Reem Alkharras, Ghada Makhdoum, Mostafa F Mohammed Saleh, Amr Hanbali, Riad El Fakih, Hazza Alzahrani, Ali Alahmari, Mahmoud Aljurf","doi":"10.46989/001c.150393","DOIUrl":"10.46989/001c.150393","url":null,"abstract":"Ramadan fasting induces significant physiological adaptations that can affect patients with hematological disorders. While fasting is generally safe for healthy individuals, reduced hydration and nutritional intake during the 13-18 hour fasting period can pose risks for those with anemia, coagulopathies, or malignancies. High-risk patients - such as those on intensive chemotherapy, post stem-cell transplantation, or with severe anemia (hemoglobin <8 g/dL) - are advised not to fast to avoid complications (e.g. vaso-occlusive crises, thrombotic events, or worsening anemia). Patients with stable hematologic conditions, however, may fast safely with careful pre-Ramadan evaluation, medication timing adjustments, and strict hydration and nutrition plans. Given the limited clinical data, an individualized risk stratification approach is essential. Shared decision-making, balancing religious aspirations with medical safety, is encouraged. Future studies are needed to establish evidence-based guidelines, but until then, thorough risk assessment, proactive monitoring, and patient education form the cornerstone of safe Ramadan fasting for individuals with hematological disorders.","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 4","pages":"41-53"},"PeriodicalIF":0.0,"publicationDate":"2025-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12700656/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145758624","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Skills, attitude and knowledge for early-career hematologists: a pragmatic, scientific framework. 技能，态度和知识为早期职业血液学家：务实，科学的框架。

Clinical hematology international Pub Date : 2025-12-02 eCollection Date: 2025-01-01 DOI: 10.46989/001c.150336

Mohamad Mohty, Yishan Ye, Rahul Banerjee

引用次数: 0

Erdheim-Chester Disease in Adults: 11 Cases from a Canadian Rare Diseases Program. 成人埃尔德海姆-切斯特病：来自加拿大罕见病项目的11例病例。

Clinical hematology international Pub Date : 2025-10-18 eCollection Date: 2025-01-01 DOI: 10.46989/001c.145180

Stephanie Quon, Emily Leung, Mark Trinder, Liliana Wolak, Mariam Goubran, Fatimah Al-Ani, Maude Landry, Mollie Carruthers, Luke Y C Chen

{"title":"Erdheim-Chester Disease in Adults: 11 Cases from a Canadian Rare Diseases Program.","authors":"Stephanie Quon, Emily Leung, Mark Trinder, Liliana Wolak, Mariam Goubran, Fatimah Al-Ani, Maude Landry, Mollie Carruthers, Luke Y C Chen","doi":"10.46989/001c.145180","DOIUrl":"10.46989/001c.145180","url":null,"abstract":"Erdheim-Chester disease (ECD) is a rare histiocytic neoplasm with highly variable, multisystem manifestations that present significant diagnostic and therapeutic challenges. This retrospective multicenter case series included 11 adult patients diagnosed with biopsy-proven ECD across Canada between January 2015 and June 2024. The cohort comprised six females and five males with a median age of 55 years (range 41-74). PET-CT was used for disease staging and treatment monitoring in nine cases. The most commonly involved sites were bone (n=8), kidney (n=6), and lungs (n=5). BRAF V600E mutations were detected in seven patients. Treatments included vemurafenib, interferon, tocilizumab, cladribine, cobimetinib, and cytarabine. Treatment responses varied, with several patients achieving remission or stable disease, while others had progressive or end-stage disease. This study highlights the clinical heterogeneity of ECD and the value of integrating histopathology, molecular profiling, and imaging to guide management and improve outcomes.","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 4","pages":"27-36"},"PeriodicalIF":0.0,"publicationDate":"2025-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12535915/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145350527","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0