International Journal of Hematology最新文献

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Dynamics of clonal hematopoiesis and risk of hematologic malignancy. 克隆造血的动态变化与血液恶性肿瘤的风险。
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2024-08-08 DOI: 10.1007/s12185-024-03829-6
Christopher Maximilian Arends, Siddhartha Jaiswal
{"title":"Dynamics of clonal hematopoiesis and risk of hematologic malignancy.","authors":"Christopher Maximilian Arends, Siddhartha Jaiswal","doi":"10.1007/s12185-024-03829-6","DOIUrl":"10.1007/s12185-024-03829-6","url":null,"abstract":"<p><p>The age-related expansion of hematopoietic stem cell clones carrying somatic mutations is known as clonal hematopoiesis and is linked to hematologic malignancies, cardiovascular diseases, and increased mortality. As the risk for adverse outcomes increases substantially with clone size, a precise understanding of the mechanisms that promote clonal expansion is crucial to identify potential therapeutic targets. Clonal expansion and progression to myeloid malignancies are driven by a complex interplay of cell-intrinsic and extrinsic factors that remain incompletely understood. Here, we review how recently proposed methods to estimate clonal expansion rates have been implemented to study the natural history of clonal hematopoiesis and identify factors that promote clonal expansion. We discuss how these factors relate to progression to myeloid malignancies and recapitulate recent risk prediction models. While we are still in the early stages of understanding clonal expansion, analysis of large-scale biobank data in combination with experimental models will help to discover causal factors promoting or suppressing clone growth, define mechanisms, and identify potential targets for clinical intervention in the future.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"318-326"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141901703","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}
引用次数: 0
Comparative inhibitory effects of bepotastine and diphenhydramine on rituximab-induced infusion reactions. bepotastine和苯海拉明对利妥昔单抗诱导的输注反应的比较抑制作用。
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2025-04-29 DOI: 10.1007/s12185-025-03990-6
Tomoki Hori, Kazuhiro Yamamoto, Tomoaki Nakagawa, Rinako Nakagawa, Masami Okayama, Tamika Sudou, Moe Hamasaki, Mai Yasuda, Shinya Kobayashi, Fumihiko Nakamura, Hideo Yagi, Yumi Kitahiro, Shigeki Ikushima, Ikuko Yano
{"title":"Comparative inhibitory effects of bepotastine and diphenhydramine on rituximab-induced infusion reactions.","authors":"Tomoki Hori, Kazuhiro Yamamoto, Tomoaki Nakagawa, Rinako Nakagawa, Masami Okayama, Tamika Sudou, Moe Hamasaki, Mai Yasuda, Shinya Kobayashi, Fumihiko Nakamura, Hideo Yagi, Yumi Kitahiro, Shigeki Ikushima, Ikuko Yano","doi":"10.1007/s12185-025-03990-6","DOIUrl":"10.1007/s12185-025-03990-6","url":null,"abstract":"<p><strong>Purpose: </strong>Infusion-related reaction (IRR) is a common adverse event induced by rituximab. Although first-generation histamine 1 receptor antagonists (H1RAs) are commonly used to prevent IRR, evidence on IRR suppression by the second-generation H1RA bepotastine is scarce. In this study, we assessed the inhibitory effects of bepotastine on rituximab-induced IRR and compared them with those of the first-generation H1RA diphenhydramine.</p><p><strong>Methods: </strong>We retrospectively evaluated IRR incidence in patients with B-cell non-Hodgkin lymphoma who received their first dose of rituximab.</p><p><strong>Results: </strong>The incidence of any grade IRR was 9.8% in the bepotastine group (n = 92), which was significantly lower than the 30.2% rate in the diphenhydramine group (n = 96; p < 0.001). The incidence of grade 2 or higher IRR was similar between the two groups (6.5% vs. 12.5%; p = 0.16). Multivariable logistic regression analysis revealed that the risk of any grade IRR incidence was higher in patients with B symptoms and bulky disease. Premedication with bepotastine was an independent factor in reducing the risk of any grade IRR incidence (odds ratio = 0.19, 95% confidence interval: 0.08-0.47).</p><p><strong>Conclusion: </strong>Bepotastine may be more effective than diphenhydramine in reducing the incidence of rituximab-induced IRR, particularly low-grade reactions.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"413-420"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12380942/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143982692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","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 in cancer predisposition syndromes. 癌症易感综合征的克隆造血。
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2024-12-06 DOI: 10.1007/s12185-024-03878-x
Kenichi Yoshida
{"title":"Clonal hematopoiesis in cancer predisposition syndromes.","authors":"Kenichi Yoshida","doi":"10.1007/s12185-024-03878-x","DOIUrl":"10.1007/s12185-024-03878-x","url":null,"abstract":"<p><p>After recent advances in sequencing technologies led to the discovery of novel genes associated with predisposition to hematological malignancies, studies have now shown that myeloid neoplasms associated with germline variants are more common than previously estimated. Based on these findings, myeloid neoplasms with germline predisposition have emerged as a unique category in the recent World Health Organization classification of Haematolymphoid Tumors. Clonal hematopoiesis is common in healthy individuals, particularly in older people. In patients with germline predisposition to hematological malignancies, clonal hematopoiesis is frequently observed at younger ages and is often associated with unique disease-specific driver mutations, some of which are hypothesized to compensate for the inherited defect. This review summarizes recent findings on clonal hematopoiesis in cancer predisposition syndromes.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"351-357"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791898","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}
引用次数: 0
High-dose thiotepa may induce inappropriate secretion of antidiuretic hormone syndrome (SIADH) during conditioning treatment for allogeneic stem cell transplantation. 大剂量硫替帕可能诱导异基因干细胞移植调理治疗过程中抗利尿激素综合征(SIADH)的不适当分泌。
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2025-07-14 DOI: 10.1007/s12185-025-04032-x
Maria Assunta Limongiello, Elisabetta Metafuni, Sabrina Giammarco, Eugenio Galli, Federica Sorà, Francesco Autore, Silvia Baroni, Patrizia Chiusolo, Simona Sica
{"title":"High-dose thiotepa may induce inappropriate secretion of antidiuretic hormone syndrome (SIADH) during conditioning treatment for allogeneic stem cell transplantation.","authors":"Maria Assunta Limongiello, Elisabetta Metafuni, Sabrina Giammarco, Eugenio Galli, Federica Sorà, Francesco Autore, Silvia Baroni, Patrizia Chiusolo, Simona Sica","doi":"10.1007/s12185-025-04032-x","DOIUrl":"10.1007/s12185-025-04032-x","url":null,"abstract":"<p><p>We conducted a retrospective, single-center, observational cohort study in patients with inappropriate secretion of antidiuretic hormone syndrome (SIADH) induced by high-dose thiotepa during conditioning for allogeneic stem cell transplantation (allo-HSCT). Between May 2018 and December 2023, 431 patients underwent allo-HSCT. All patients who developed SIADH had received TBF conditioning, and the incidence of SIADH among TBF-treated patients was 7.3%. Despite its limitations as a retrospective study, this real-life analysis shows that hyponatremia with corresponding serum hypo-osmolality and persistent urinary sodium excretion is compatible with a diagnosis of SIADH in the presence of normovolemia. Fluid balance and electrolyte levels should be closely monitored during conditioning to ensure prompt detection of SIADH promptly and enable immediate treatment.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"368-371"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144636978","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}
引用次数: 0
Successful treatment of myeloid blast phase chronic myelogenous leukemia with the JAK2 V617 F mutation by combination therapy with asciminib and ropeginterferon alfa-2b in an elderly patient. 阿西米尼联合干扰素α -2b成功治疗1例老年患者JAK2 V617 F突变的髓系母细胞期慢性髓系白血病
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2025-04-29 DOI: 10.1007/s12185-025-03994-2
Satoko Oka, Yuina Akagi, Takaya Mituyoshi, Kazuo Ono
{"title":"Successful treatment of myeloid blast phase chronic myelogenous leukemia with the JAK2 V617 F mutation by combination therapy with asciminib and ropeginterferon alfa-2b in an elderly patient.","authors":"Satoko Oka, Yuina Akagi, Takaya Mituyoshi, Kazuo Ono","doi":"10.1007/s12185-025-03994-2","DOIUrl":"10.1007/s12185-025-03994-2","url":null,"abstract":"<p><p>The co-occurrence of JAK2 V617F mutations and the BCR::ABL1 translocation in the same patient is rare, and the current standard treatment for aggressive myeloid blast phase chronic myeloid leukemia (CML-myeloid BP) with JAK2 V617F mutations remains inadequate, particularly in transplant-ineligible patients. Asciminib, a first-in-class allosteric inhibitor of BCR::ABL1 kinase that specifically targets the ABL1 myristoyl pocket, has emerged as a novel alternative to standard tyrosine kinase inhibitor (TKI) therapy. Ropeginterferon alfa-2b (ropegIFNα2b) is a novel site-selective, monopegylated recombinant human IFN with long-term safety and efficacy in patients with polycythemia vera (PV). Here, we report a case of successful combination therapy with asciminib and ropegIFNα2b in a patient with CML-myeloid BP who had a long history of PV with JAK2 V617F refractory to induction chemotherapy with several TKIs. The combination of asciminib and ropegIFNα2b is a promising new treatment option for these patients.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"444-448"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12380864/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
COVID-19-induced thrombotic microangiopathy in patients with multiple myeloma receiving carfilzomib treatment. 接受卡非佐米治疗的多发性骨髓瘤患者covid -19诱导的血栓性微血管病变
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2025-05-05 DOI: 10.1007/s12185-025-03996-0
Kaito Takikawa, Mana Sota, Shiori Nakashima, Yui Imai, Hirofumi Nakano, Tomoyuki Uchida, Morihiro Inoue, Masao Hagihara, Akiko Torii
{"title":"COVID-19-induced thrombotic microangiopathy in patients with multiple myeloma receiving carfilzomib treatment.","authors":"Kaito Takikawa, Mana Sota, Shiori Nakashima, Yui Imai, Hirofumi Nakano, Tomoyuki Uchida, Morihiro Inoue, Masao Hagihara, Akiko Torii","doi":"10.1007/s12185-025-03996-0","DOIUrl":"10.1007/s12185-025-03996-0","url":null,"abstract":"<p><p>A 62-year-old woman experienced severe renal insufficiency and significant thrombocytopenia during the 7th cycle of carfilzomib and dexamethasone therapy for relapsed multiple myeloma. She was diagnosed with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection from a routine check upon admission. She was subsequently diagnosed with thrombotic microangiopathy (TMA) as well, and platelet fragments were detected in her blood. Treatment with plasma exchange, hemodialysis, and antiviral medications was successful. Although coronavirus disease 2019 (COVID-19) was identified as the primary trigger of TMA, carfilzomib was probably a predisposing factor that contributed to endothelial damage. Even though severe cases of COVID-19 have been less frequent since the Omicron variant became dominant, patients with MM, especially those under treatment with carfilzomib, should be given repeated booster vaccinations. Antiviral medication should also be considered in patients infected with COVID-19, even if symptoms are mild.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"449-453"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144005338","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}
引用次数: 0
Ropeginterferon-α2b discontinuation after long-term exposure: four cases from a single institution. 长期暴露后停用ropeg干扰素-α2b:同一机构4例
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2025-05-21 DOI: 10.1007/s12185-025-04008-x
Shuichi Shirane, Tadaaki Inano, Yasutaka Fukuda, Tomonori Ochiai, Naoko Midorigawa, Soji Morishita, Miki Ando, Norio Komatsu
{"title":"Ropeginterferon-α2b discontinuation after long-term exposure: four cases from a single institution.","authors":"Shuichi Shirane, Tadaaki Inano, Yasutaka Fukuda, Tomonori Ochiai, Naoko Midorigawa, Soji Morishita, Miki Ando, Norio Komatsu","doi":"10.1007/s12185-025-04008-x","DOIUrl":"10.1007/s12185-025-04008-x","url":null,"abstract":"<p><p>Polycythemia vera (PV) is a Philadelphia chromosome-negative myeloproliferative neoplasm driven by JAK2 mutations, leading to the overproduction of blood cells. Ropeginterferon-α-2b (RopegIFN) has emerged as a promising therapy, capable of lowering the JAK2V617F allele burden and maintaining a complete hematologic response (CHR). Here, we report the cases of four patients with PV who discontinued RopegIFN after achieving CHR. One patient had sustained long-term remission post-discontinuation, with the JAK2V617F allele burden reduced below 10%, and met the criteria for an \"operational cure.\" However, the other three patients experienced rising blood counts, resulting in loss of CHR. These findings underscore the importance of a robust molecular response in predicting sustainable remission. Continuous therapy remains the standard approach due to the lack of predictive models to identify patients who can safely discontinue RopegIFN. In the future, it will be necessary to verify whether patients who have achieved an \"operational cure\" can remain treatment free.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"458-461"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119316","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}
引用次数: 0
Discovering the hidden link: hematological disorders caused by copper deficiency. 发现隐藏的环节:缺铜引起的血液病。
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2025-07-14 DOI: 10.1007/s12185-025-04036-7
Akiyoshi Takami, Kaori Uchino
{"title":"Discovering the hidden link: hematological disorders caused by copper deficiency.","authors":"Akiyoshi Takami, Kaori Uchino","doi":"10.1007/s12185-025-04036-7","DOIUrl":"10.1007/s12185-025-04036-7","url":null,"abstract":"<p><p>Copper deficiency can lead to anemia, leukopenia, or thrombocytopenia, often mimicking vitamin B<sub>12</sub> deficiency or myelodysplastic syndrome. We review the crucial role of copper in iron metabolism and hematopoiesis, highlighting how high-dose zinc supplementation, gastrointestinal surgeries, and long-term nutritional support can induce deficiency. The pathophysiology involves compromised ferroxidase activity, which hampers iron utilization, and reduced neutrophil survival due to impairment of cytochrome c oxidase and superoxide dismutase. Timely recognition is vital, as most cases respond to copper repletion and zinc reduction. Institutional data underscore the need for routine copper monitoring, and management strategies aim to optimize patient care by maintaining adequate copper levels.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"358-367"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144626255","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}
引用次数: 0
In vitro evaluation of global coagulation potential of emicizumab and warfarin using rotational thromboelastometry. 使用旋转血栓弹性测量法体外评价艾美珠单抗和华法林的整体凝血电位。
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2025-04-28 DOI: 10.1007/s12185-025-03986-2
Takahiro Kajimoto, Yuto Nakajima, Nobuyuki Tsujii, Keiji Nogami
{"title":"In vitro evaluation of global coagulation potential of emicizumab and warfarin using rotational thromboelastometry.","authors":"Takahiro Kajimoto, Yuto Nakajima, Nobuyuki Tsujii, Keiji Nogami","doi":"10.1007/s12185-025-03986-2","DOIUrl":"10.1007/s12185-025-03986-2","url":null,"abstract":"<p><p>Warfarin inhibits the generation of vitamin K-dependent proteins and emicizumab can prevent bleeding episodes in people with hemophilia A (PwHA), but their combined hemostatic potential remains unclear. We analyzed the coagulation potential of emicizumab combined with warfarin in a simulated model of PwHA. Nineteen samples were collected from ten patients taking warfarin, and the prothrombin time-international normalized ratio (PT-INR) was used to define near-normal (INR 1.2-1.48; n = 4), subtherapeutic (INR 1.56-1.9; n = 7) and therapeutic (INR > 2.0; n = 8) groups. Factor (F)VIII activity (FVIII:C) was neutralized using an anti-FVIII inhibitor antibody (termed FVIII-depleted) before the addition of emicizumab (50 µg/mL). Coagulation potential was measured using Ca2 + -triggered rotational thromboelastometry, and was compared with that in emicizumab-treated PwHA. The average PT-INR in the near-normal, subtherapeutic, and therapeutic groups was 1.3 ± 0.1, 1.7 ± 0.1, and 2.4 ± 0.3, respectively. The hemostatic potential in FVIII-depleted samples mixed with emicizumab in the near-normal group was comparable to that in emicizumab-treated PwHA. The coagulation potential in FVIII-depleted samples after addition of emicizumab in the subtherapeutic and therapeutic groups were lower than that in emicizumab-treated PwHA. PT-INR monitoring could be informative in emicizumab-treated PwHA due to the influence of vitamin K-dependent proteins.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"381-391"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144014398","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}
引用次数: 0
VEXAS syndrome. VEXAS 综合征
IF 1.8 4区 医学
International Journal of Hematology Pub Date : 2025-09-01 Epub Date: 2024-05-31 DOI: 10.1007/s12185-024-03799-9
Hideaki Nakajima, Hiroyoshi Kunimoto
{"title":"VEXAS syndrome.","authors":"Hideaki Nakajima, Hiroyoshi Kunimoto","doi":"10.1007/s12185-024-03799-9","DOIUrl":"10.1007/s12185-024-03799-9","url":null,"abstract":"<p><p>VEXAS syndrome is a recently identified, adult-onset autoinflammatory disease caused by somatic mutations in UBA1. UBA1 is an X-linked gene encoding E1 ubiquitin activating enzyme and its mutation in hematopoietic stem and progenitor cells leads to their clonal expansion and myeloid-skewed differentiation. UBA1 mutations in VEXAS are clustered at the second methionine (p.Met41), eliminating UBA1b isoform translated from p.Met41. Loss of UBA1b impairs ubiquitination and activates innate immune pathways, leading to systemic autoinflammation manifested as recurrent fever, chondritis, pulmonary involvement, vasculitis, or neutrophilic dermatitis. VEXAS syndrome is frequently associated with hematological disorders such as myelodysplastic syndrome (MDS), plasma cell dyscrasia and venous thromboembolism. Macrocytic anemia/macrocytosis and vacuoles in myeloid/erythroid precursors are prominent features of VEXAS syndrome, and their presence in patients with autoinflammatory symptoms prompts physicians to screen for UBA1 variant. Treatment of VEXAS syndrome is challenging and no consistently effective therapies have been established. Anti-inflammation therapies including glucocorticoids and anti-interleukin-6 have shown limited efficacy, while azacytidine and JAK inhibitors such as ruxolitinib were found to induce favorable, mid-term responses. Hematopoietic stem cell transplantation is the only curative option for VEXAS and should be considered for younger, fit patients with poor prognostic factors or recalcitrant symptoms.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"341-350"},"PeriodicalIF":1.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12380878/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141179335","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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