HematologyPub Date : 2025-12-01Epub Date: 2025-01-30DOI: 10.1080/16078454.2025.2450575
Leyu Wang, Ziwei Liu, Chen Yang, Miao Chen, Bing Han
{"title":"Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety - a single-center study.","authors":"Leyu Wang, Ziwei Liu, Chen Yang, Miao Chen, Bing Han","doi":"10.1080/16078454.2025.2450575","DOIUrl":"https://doi.org/10.1080/16078454.2025.2450575","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate the short-term efficacy and safety of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.</p><p><strong>Method: </strong>Data were retrospectively collected from patients with PNH who received at least 3 months of full-dose eculizumab. Changes in clinical and laboratory indicators after 1, 3, and 6 months of eculizumab therapy and at the end of follow-up were documented. The incidence rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and adverse events were recorded.</p><p><strong>Result: </strong>A total of 48 patients, including 27 males, with a median age of 46 (12-78) years were included. Twenty-four (50%) patients had classic PNH and 24 (50%) had bone marrow failure (BMF)/PNH. Eighteen (37.5%) patients required blood transfusion. The median duration of follow-up was 6 (3-15) months. During the follow-up period, Lactate Dehydrogenase (LDH) levels were lower than those at baseline (<0.05) at all observation points. The patients showed a significant reduction in creatinine levels from baseline (<i>P</i> = 0.022 and <i>P</i> = 0.039, respectively) at 1 and 3 months. At the end of the follow-up, fifteen (83.3%) became transfusion-independent. No new thrombotic events were observed. The FACIT-Fatigue score significantly improved (<i>P</i> < 0.05). No significant differences were observed in the changes in hemoglobin or LDH levels between patients with classic PNH and those with BMF/PNH. BTH was observed in 17.4% of patients and EVH in 10.4%. Mild adverse events occurred in 22.9% of patients. No deaths or clonal evolution was observed.</p><p><strong>Conclusion: </strong>Eculizumab can effectively control the hemolytic symptoms of PNH with good tolerance for Chinese patients.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2450575"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143065405","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HematologyPub Date : 2025-12-01Epub Date: 2025-02-05DOI: 10.1080/16078454.2025.2457809
Debora S Bruno, Manoj Khanal, Xiaohong Li, Maricer P Escalon, Katherine Winfree, Lisa M Hess
{"title":"Clinical trial participation, clinical care, and patient outcomes by practice setting: a real-world database analysis of patients with Chronic Lymphocytic Leukemia or Mantle Cell Lymphoma.","authors":"Debora S Bruno, Manoj Khanal, Xiaohong Li, Maricer P Escalon, Katherine Winfree, Lisa M Hess","doi":"10.1080/16078454.2025.2457809","DOIUrl":"https://doi.org/10.1080/16078454.2025.2457809","url":null,"abstract":"<p><strong>Objective: </strong>This study was designed to compare treatment patterns, clinical trial participation, and clinical outcomes among patients with small lymphocytic lymphoma/chronic lymphocytic leukemia (CLL) or Mantle cell lymphoma (MCL) by site of care.</p><p><strong>Methods: </strong>A nationwide electronic health record (EHR)-derived de-identified database was utilized for this study. Eligible patients were diagnosed with either CLL or MCL from 2013-2022 who received systemic therapy for their disease. Overall survival (OS) was analyzed using Kaplan Meier method, censoring patients without events at last observation in the database. Cox proportional hazards regression model was used to adjust for baseline covariates.</p><p><strong>Results: </strong>A total of 6,372 patients with CLL and 3,411 with MCL met eligibility criteria for this analysis; 13.9% and 22.2%, respectively, were treated in academic settings. Academic settings were associated with higher patient volume and were more likely to treat MCL with CAR-T, enroll patients with CLL or MCL to clinical trials, and care for patients who were younger, White, and for CLL with higher rates of del(17p) mutations (all <i>p</i> < 0.01). Survival was significantly longer among patients treated in academic vs community settings (median OS not reached vs 80.5 months for CLL; 95.6 vs 68.7 months for MCL from start of first-line therapy).</p><p><strong>Discussion: </strong>Patients who received care in academic settings differed from those treated in the community; care in academic settings was associated with significantly longer OS and higher trial participation. Further research is warranted to better understand the factors that may contribute to the observed outcomes.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2457809"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255630","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HematologyPub Date : 2025-12-01Epub Date: 2025-03-18DOI: 10.1080/16078454.2025.2478650
Jian Sun, Cui Liu, Guangli Yang, Qian Li, Yang An, Yin Zhu, Pingping Zhang, Yaning Guan, Chang Peng, Zuochen Du, Pei Huang, Yan Chen
{"title":"Targeting NEDD8 in pediatric acute myeloid leukemia: an integrated bioinformatics and experimental approach.","authors":"Jian Sun, Cui Liu, Guangli Yang, Qian Li, Yang An, Yin Zhu, Pingping Zhang, Yaning Guan, Chang Peng, Zuochen Du, Pei Huang, Yan Chen","doi":"10.1080/16078454.2025.2478650","DOIUrl":"https://doi.org/10.1080/16078454.2025.2478650","url":null,"abstract":"<p><p>SUMMARYThis study systematically explored the role of NEDD8 in pediatric acute myeloid leukemia (AML) through patient sample analysis, database mining, and in vitro experiments. Our results demonstrated that NEDD8 was significantly overexpressed in newly diagnosed pediatric AML patients and was associated with poor survival outcomes. Functional enrichment analysis of the TARGET database further revealed a strong correlation between NEDD8 and cancer-related pathways. In vitro experiments showed that NEDD8 knockdown significantly inhibited the proliferation of AML cells (THP-1 and MV4-11) and induced cell cycle arrest. Collectively, these findings highlight the critical role of NEDD8 in pediatric AML pathogenesis and suggest its potential as both a prognostic biomarker and a therapeutic target.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2478650"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143657094","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Mendelian randomization reveals causal effect of Hashimoto's thyroiditis on immune thrombocytopenic purpura.","authors":"Zhen Yao, Mingzhu Xu, Zijin Wang, Shanglong Feng, Fuquan Zhang, Shengli Xue, Chengsen Cai","doi":"10.1080/16078454.2025.2484959","DOIUrl":"10.1080/16078454.2025.2484959","url":null,"abstract":"<p><strong>Introduction: </strong>Patients with immune thrombocytopenic purpura (ITP) usually express thyroid antigen-specific antibodies. The purpose of this study was to explore the causal relationship between Hashimoto's thyroiditis (HT) and ITP.</p><p><strong>Methods: </strong>A two-sample Mendelian randomization (TSMR) analysis was applied to investigate the potential causal relationship between HT and ITP in European population. Five complementary methods including inverse variance weighted (IVW), Mendelian Randomization-Egger (MR-Egger), weighted median, and weighted mode were performed in our study. Risk genes of HT and ITP were selected through Mendelian randomization (MR), and the common risk genes were further analysed by bioinformatics methods to explore the common pathogenesis of the two diseases.</p><p><strong>Results: </strong>The MR analysis revealed a potential causal relationship between HT and risk of ITP [odds ratio (OR) = 1.22; 95% confidence interval (CI) 1.01, 1.49; <i>P</i> = 0.046]. Gene eQTL data were obtained from the IEU database. HT and ITP were respectively treated as outcome variables for MR analysis, and a total of 32 common risk genes were selected, including 12 high-risk genes and 20 low-risk genes. Functional analysis including Gene Ontology (GO) and Kyoto Encyclopedia of Genes and Genome (KEGG) analysis revealed that risk genes were closely related to antigen processing and presentation, and played a crucial role in the process of various viral and bacterial infections.</p><p><strong>Conclusion: </strong>Our study demonstrated that HT may increase the risk of ITP, and revealed the role of their common risk genes in the development of the two diseases.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2484959"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143772153","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HematologyPub Date : 2025-12-01Epub Date: 2025-01-08DOI: 10.1080/16078454.2024.2445404
Minyuan Liu, Qi Ji, Senlin Zhang, Jing Qian, Bohan Li, Jie Li, Peifang Xiao, Shaoyan Hu
{"title":"Hematopoietic stem cell transplantation for purine nucleoside phosphorylase deficiency with two novel mutations: a case report and review of literature.","authors":"Minyuan Liu, Qi Ji, Senlin Zhang, Jing Qian, Bohan Li, Jie Li, Peifang Xiao, Shaoyan Hu","doi":"10.1080/16078454.2024.2445404","DOIUrl":"10.1080/16078454.2024.2445404","url":null,"abstract":"<p><strong>Purpose: </strong>We report the case of a 6-year-old boy who presented with muscular hypertonia, impaired growth, and recurrent infections, who was diagnosed with purine nucleoside phosphorylase (PNP) deficiency with two novel mutations in the <i>PNP</i> gene. He underwent a hematopoietic stem cell transplantation (HSCT) from an unrelated donor, and we observed the clinical outcome.</p><p><strong>Methods: </strong>We retrospectively analyzed the clinical manifestations and outcomes of this patient who underwent HSCT. We analyzed the results of whole exome sequencing (WES) on the patient.</p><p><strong>Results: </strong>The patient experienced repeated serious respiratory and gastrointestinal infections since birth and presented with neurological symptoms. He was found to have two novel pathogenic mutations in the <i>PNP</i> gene through WES. One hemizygous variant was c.385dup (p.Ile129Asnfs*6) in exon 4. The other was a heterozygous deletion in exon 2-6. He underwent HSCT with clinical improvement.</p><p><strong>Conclusions: </strong>We presented a patient with two novel mutations in the <i>PNP</i> gene and clinical improvement following an allo-HSCT.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2445404"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142970610","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HematologyPub Date : 2025-12-01Epub Date: 2025-02-18DOI: 10.1080/16078454.2025.2466261
Daquan Gao
{"title":"The role of non-malignant B cells in malignant hematologic diseases.","authors":"Daquan Gao","doi":"10.1080/16078454.2025.2466261","DOIUrl":"https://doi.org/10.1080/16078454.2025.2466261","url":null,"abstract":"<p><p>The tumor microenvironment (TME) represents a heterogeneous, complicated ecosystem characterized by intricate interactions between tumor cells and immune cells. During the past decade, immune cells especially T cells were found to play an important role in the progression of tumor and many related immune checkpoints drugs were created. In recent years, more and more scientists revealed the critical role of B-cells within the TME, particularly various populations of non-malignant B cells. Some studies indicated that non-malignant B cells may exert a 'double-edged sword' role in solid tumors. However, there has been comparatively less focus on the role of non-malignant B cells in hematologic malignancies. In this review, we characterized the development of B cells and summarized its functions of antitumor immunity within TME, with an emphasis on elucidating the roles and potential mechanisms of non-malignant B cells in the progression of hematologic diseases including classical Hodgkin's lymphoma, non-Hodgkin's B-cell lymphoma, non-Hodgkin's T-cell lymphoma, leukemia and multiple myeloma.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2466261"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143448952","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Impact of intermediate-dose tertiary prophylaxis on quality of life and psychological aspects of adult patients with severe/moderate hemophilia A.","authors":"Hua Gao, Jia Liu, Shiqiu Zhou, Jing Gao, Jing Tan, Rong Chen","doi":"10.1080/16078454.2024.2439061","DOIUrl":"https://doi.org/10.1080/16078454.2024.2439061","url":null,"abstract":"<p><strong>Objectives: </strong>Whether intermediate-dose tertiary prophylaxis can improve quality of life and psychological health in adults with severe/moderate hemophilia A has not been determined. This research aims to explore the impact of intermediate-dose tertiary prophylaxis with recombinant human FVIII (rhFVIII) on quality of life, anxiety and depression in such individuals transitioned from on-demand treatment.</p><p><strong>Methods: </strong>This retrospective analysis collected data from July 2019 to July 2022. Haemophilia Quality of Life Questionnaire for Adults (HAEMO-QoL-A), Patient Health Questionnaire-9 (PHQ-9), and Generalized Anxiety Disorder-7 (GAD-7) were compared before and after prophylaxis. Additionally, functional independence and joint status were analyzed at the end of the 3-year prophylaxis period, as well as their correlations with HAEMO-QoL-A, PHQ-9, and GAD-7.</p><p><strong>Results: </strong>The HAEMO-QoL-A total score decreased after prophylaxis (pre-prophylaxis: 88.90 ± 33.38 vs. post-prophylaxis: 79.59 ± 22.89) (<i>P</i> = 0.016). The mean PHQ-9 scores before and after prophylaxis were 4.44 ± 4.63 and 5.56 ± 4.30 (<i>P</i> = 0.058), respectively, while the mean GAD-7 scores were 3.15 ± 3.84 and 4.44 ± 3.99 (<i>P</i> = 0.016). Significant correlations were observed for HAEMO-QoL-A with functional independence (<i>P</i> < 0.001), mood and emotions with age (<i>P</i> = 0.032), PHQ-9 scores with knee joint rehabilitation scores (<i>P</i> = 0.047), and GAD-7 scores with treatment experience and ankle joint rehabilitation scores (<i>P</i> = 0.029, <i>P</i> = 0.039).</p><p><strong>Conclusion: </strong>Intermediate-dose tertiary prophylaxis with rhFVIII can improve quality of life but not relieve anxiety and depression in adults with severe/moderate hemophilia A. Better functional independence correlates with improved quality of life. Gait and age also influence the quality of life to some extent. We need to undertake anxiety and depression screening and provide psychological treatment when necessary.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2439061"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142970613","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HematologyPub Date : 2025-12-01Epub Date: 2025-02-09DOI: 10.1080/16078454.2025.2458359
Vickie McDonald, Sebastian Guterres, Samuel James, Maja Zakrzewski, Michał Pochopień
{"title":"The costs of treating bleeding episodes in patients with immune thrombocytopaenia in the United Kingdom.","authors":"Vickie McDonald, Sebastian Guterres, Samuel James, Maja Zakrzewski, Michał Pochopień","doi":"10.1080/16078454.2025.2458359","DOIUrl":"https://doi.org/10.1080/16078454.2025.2458359","url":null,"abstract":"<p><strong>Objectives: </strong>Immune thrombocytopaenia (ITP) is a rare autoimmune disorder characterized by low platelet count and increased risk of bleeding. This study aimed to be the first publication to characterize the economic burden of bleeding events in patients with ITP in the UK.</p><p><strong>Methods: </strong>We performed a microcosting analysis to estimate the costs associated with bleeding events in patients with ITP. Healthcare resources utilized in the management of bleeds of different severity were costed using well-established UK cost sources. The results were validated through semi-structured interviews with clinical experts.</p><p><strong>Results: </strong>The severity of bleeding events was classified into four categories, ranging from bleeding managed at home, through mild bleeding managed in the outpatient or day case setting, to serious and life-threatening bleeding events requiring inpatient admission. Total medical costs per event ranged from £2,930 for managing a mild bleeding event, through £16,711 for a serious bleeding event to £32,461 for a life-threatening event. The major cost driver for mild and serious events were intravenous immunoglobulin (IVIg) costs, amounting to £1,614 and £8,071 for the two severity categories, respectively. For life-threatening events, the costs of intensive care unit stay (£9,089) exceeded those of IVIg (£8,071).</p><p><strong>Conclusion: </strong>Real-world costs of managing bleeding in patients with ITP in the UK are substantial and greater than costs set only based on the UK NHS Tariff. Mitigating the risk of bleeding in patients with ITP is likely to yield not only clinical advantages for patients but also offer substantial cost savings to the health system.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2458359"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143382334","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HematologyPub Date : 2025-12-01Epub Date: 2025-03-27DOI: 10.1080/16078454.2025.2476254
Kristy Iglay, Molly L Aldridge, Mirella Calcinai, Eric Wolford, Aneel A Ashrani
{"title":"The global epidemiology of acquired factor X deficiency.","authors":"Kristy Iglay, Molly L Aldridge, Mirella Calcinai, Eric Wolford, Aneel A Ashrani","doi":"10.1080/16078454.2025.2476254","DOIUrl":"https://doi.org/10.1080/16078454.2025.2476254","url":null,"abstract":"<p><strong>Objectives: </strong>To summarize available data and contribute to a broader understanding of the global incidence and prevalence of acquired factor X deficiency.</p><p><strong>Methods: </strong>A comprehensive review of English-language publications from PubMed and Embase was conducted. The majority of publications on acquired factor X deficiency were associated with light-chain (AL) amyloidosis. Therefore, this review is structured to assess publications reporting on (1) acquired factor X deficiency associated with AL amyloidosis or (2) acquired factor X deficiency associated with other causes.</p><p><strong>Results: </strong>The literature includes case reports, case-series, and limited population-based reports of the epidemiology of acquired factor X deficiency. Though no definitive global incidence or prevalence estimates for AL-amyloidosis-associated acquired factor X deficiency were identified, the finding that roughly 6-14% of patients with AL amyloidosis have factor X activity levels below 45-50% of normal highlights the rarity of acquired factor X deficiency associated with AL-amyloidosis. Indeed, AL amyloidosis itself is a rare disorder with an estimated annual incidence of ∼10 cases per million population. Only case reports were available to inform the epidemiology of acquired factor X deficiency not associated with AL amyloidosis. We identified 35 cases from 29 papers published from around the globe. At least 25 of those patients experienced a bleeding event, with factor X activity levels ranging from <1% to 39%.</p><p><strong>Conclusion: </strong>More population-based data are needed to understand the epidemiology of acquired factor X deficiency; however, the limited data seem to indicate this condition is quite rare. The variation across papers in thresholds used to define deficiency highlights the need for a standardized definition to better inform drug development, resource allocation, and regulatory decision-making.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2476254"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143729958","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HematologyPub Date : 2025-12-01Epub Date: 2025-02-02DOI: 10.1080/16078454.2025.2456687
Waleed Ghanima, Francisco Javier Lucas Boronat, Valentina Carrai, Stefan Rackwitz
{"title":"Sustained response off treatment after fostamatinib in refractory immune thrombocytopenia: A series of four case reports.","authors":"Waleed Ghanima, Francisco Javier Lucas Boronat, Valentina Carrai, Stefan Rackwitz","doi":"10.1080/16078454.2025.2456687","DOIUrl":"https://doi.org/10.1080/16078454.2025.2456687","url":null,"abstract":"<p><strong>Introduction: </strong>A goal of most primary immune thrombocytopenia (ITP) treatments is reducing or discontinuing treatment while maintaining a response including an absence of bleeding events. We present four cases describing treatment with the spleen tyrosine kinase (SYK) inhibitor, fostamatinib, that showed sustained response off treatment (SROT).</p><p><strong>Case presentations: </strong>Case 1 was a 66-year-old male with chronic ITP. He was pre-treated with prednisone and rituximab before being in the FIT-2 clinical trial (placebo). He received fostamatinib in the FIT-3 open-label extension for seven weeks and maintained SROT for 2.5 years. Case 2 was a 54-year-old female patient with chronic, highly refractory ITP. SROT was achieved after 6 months of fostamatinib and was maintained for more than 16 months (in remission to date). Case 3 was a 60-year-old male with chronic ITP. He was successfully treated with cycles of corticosteroids for six years prior to fostamatinib. He was treated with fostamatinib plus prednisone for approximately two months. SROT was observed in this patient for one year. Case 4 was a 67-year-old male with persistent ITP. Before fostamatinib, he was unresponsive to high-dose dexamethasone, IVIG, eltrombopag and romiplostim. After 11 months of fostamatinib, his dose was tapered for three months and ultimately discontinued. SROT was observed for more than ten months (in remission to date).</p><p><strong>Discussion: </strong>These cases emphasize that SROT is achievable with fostamatinib in complex ITP cases unresponsive to multiple previous therapies. Additional research is needed to identify the magnitude of the underlying mechanisms, and the clinical factors associated with, and potentially predictive of, SROT.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2456687"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079696","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}