Annals of Hematology最新文献

{"title":"Rapid withdrawal of voxelotor can precipitate sickle cell disease related crisis.","authors":"Salam Alkindi, Ahmed Al Subhi, Anil Pathare","doi":"10.1007/s00277-025-06503-x","DOIUrl":"https://doi.org/10.1007/s00277-025-06503-x","url":null,"abstract":"<p><p>Sickle cell disease (SCD) is an inherited disorder characterized by abnormal mutation leading to formation of sickle red blood cells. The initial step is the formation of polymers and precipitation of Hemoglobin within the cell. voxelotor is a novel anti-polymerization drug approved for treatment of patients with SCD, resulting in improved hemoglobin level. Recently its clinical development was suspended, as concerns were raised regarding its safety and efficacy. We have evaluated 11 patients who were stable, on voxelotor for a mean period of 191 weeks (range, 8-369). We observed that upon its withdrawal, 8 patients (73%) showed a rapid onset of vaso-occlusive crisis, within a median of 4.7 days, precipitating hemolysis, with a significant drop of Hb, raised reticulocytes, bilirubin, and lactic dehydrogenase. Also, one patient (9.1%) developed acute chest syndrome, whereas five (45.5%) patients needed blood transfusions. Our data confirms that rapid withdrawal of voxelotor could precipitate severe SCD-related crisis.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144582912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety of low-dose radiotherapy in newly diagnosed stage I/II extranodal NK/T-cell lymphoma: a single-center retrospective analysis.","authors":"Shifeng Hao, Yukai Duan, Renjie Hua, Yuxiao Chang, Shuchang Li, Maosong Liu, Wanyue Zhao, Xiang Gao, Wenjie Gu, Qingjiang Chen, Xudong Zhang","doi":"10.1007/s00277-025-06497-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06497-6","url":null,"abstract":"<p><p>This study aimed to investigate the feasibility and safety of low-dose radiotherapy (RT) (< 50 Gy) in patients with stage I/II Extranodal NK/T-cell lymphoma (ENKTCL). Clinical and treatment data from 158 stage I/II ENKTCL patients who received combined chemoradiotherapy at the First Affiliated Hospital of Zhengzhou University from 2020 to 2022 were retrospectively analyzed to compare locoregional control (LC) and survival outcomes between high-dose (≥ 50 Gy) and low-dose (< 50 Gy) RT groups. All patients achieved an objective response (OR) after treatment, with a median follow-up of 51 months. The 5-year overall survival (OS) and progression-free survival (PFS) rates were 90.1% and 81.7% in the high-dose group (n = 130), compared to 84.0% and 68.2% in the low-dose group (n = 28), with no significant statistical differences between the two groups. Notably, no local recurrence was observed in the low-dose group (100% local control rate), and the incidence of grade ≥ 3 RT-related adverse events (AEs) was significantly lower in the low-dose group than in the high-dose group. Preliminary evidence suggests that for newly diagnosed stage I/II ENKTCL patients receiving combined chemoradiotherapy, low-dose radiotherapy may not compromise LC and survival outcomes while reducing the incidence of grade 3-4 adverse events, with 40-45 Gy appearing to be the optimal dose range within the examined parameters.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567008","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SGK1 repression by WT1 may confer a survival advantage to leukemic cells under stress conditions.","authors":"Miguel A Rubio, Sabina Cisa-Wieczorek, Ana Mozos, Helena Castellet, Elena Bussaglia, Maite Carricondo, María Isabel Hernández-Alvarez, Jorge Sierra, Josep F Nomdedéu","doi":"10.1007/s00277-025-06458-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06458-z","url":null,"abstract":"<p><p>Increased WT1 mRNA levels are pervasive in acute myeloid leukemia (AML), and this marker has been used to assess the leukemic compartment size after chemotherapy or hematopoietic cell transplants. Little is known about the effects of WT1 on the leukemic cells and their targets. This work used data obtained from gene expression arrays performed on AML samples with high and low WT1 mRNA levels to pinpoint genes that WT1 can regulate. We singled out SGK1, which showed an inverse correlation between its mRNA levels and WT1 in leukemic cell lines and AML samples. In cellular models, forced expression of WT1 reduced mRNA and protein levels of SGK1. Furthermore, WT1 repressed the SGK1 promoter activity, and accordingly, WT1 knockdown showed an increased expression of SGK1. We also detected an inverse correlation between WT1 and SGK1 during leukemic cell-line differentiation. WT1 genetic knockdown displayed decreased cell viability under nutrient deprivation. By contrast, SGK1 knockdown or pharmacologic inhibition increased resistance to apoptosis in response to serum starvation, acting on cell cycle progression. The effects of SGK1 targeting in hematologic conditions merit further investigation.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144558898","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Second allogeneic transplantation in relapsed acute leukemia - a risk worth taking?","authors":"Patrycja Zielińska, Karolina Gruenpeter, Agata Wieczorkiewicz-Kabut, Krzysztof Białas, Anna Koclęga, Izabela Noster, Izabela Szewczyk, Kinga Boral, Katarzyna Wiśniewska-Piąty, Aleksandra Butrym, Jarosław Czyż, Ewa Lutwin, Grzegorz Helbig","doi":"10.1007/s00277-025-06468-x","DOIUrl":"https://doi.org/10.1007/s00277-025-06468-x","url":null,"abstract":"<p><p>Second allogeneic stem cell transplantation (SCT2) presents a potentially curative treatment approach in patients who relapsed after first allogeneic transplant procedure. Currently there is no consensus on second allografting. The aim of our study was to analyze the outcome and prognostic factors of SCT2 in relapsed acute leukemia setting. The study comprised 60 patients at a median age of 44.7 years (37 females, 23 males) who underwent SCT2 due to relapsed acute leukemia between 2000 and 2024 in our center. Median remission duration after SCT1 was 13.6 (IQR 30.1) months and median time from relapse to SCT2 was 4 (IQR 3) months. Disease-risk index at SCT2 was as follows: low - 2 (3.3%), intermediate - 29 (48,4%), high - 27 (45%) and very high - 2 (3.3%). The same donor was used in 52 procedures (86.7%). Six patients died before engraftment due to severe infectious complications. The 2-year probability of overall survival (OS) and leukemia free survival (LFS) were 26.6% and 26.2%, respectively. Median follow-up after SCT2 was 10.7 (IQR 35) months. At the last follow-up, 22 patients (37%) were alive. The 2-year cumulative relapse incidence (RI) was 47.4% and cumulative non-relapse mortality (NRM) was 55.3%. The factors that adversely influenced survival were as follows: early relapse after SCT1 (< 6 months after transplantation) (p = 0.018) and the use of reduced intensity conditioning before SCT2 (p = 0.0272). Presented data indicate that second allograft is a feasible option in selected patients with relapsed acute leukemia. There is still room for improvement in terms of non-relapse mortality.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144551710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical significance of C-reactive protein/platelet ratio from diagnosis to allogeneic hematopoietic stem cell transplantation in acute myeloid leukemia.","authors":"Akihiko Izumi, Takayoshi Tachibana, Jun Nukui, Takuya Miyazaki, Natsuki Hirose, Takuma Ohashi, Marika Tanaka, Kengo Katsuki, Taisei Suzuki, Yuki Nakajima, Kenji Matsumoto, Shin Fujisawa, Masatsugu Tanaka, Hideaki Nakajima","doi":"10.1007/s00277-025-06470-3","DOIUrl":"https://doi.org/10.1007/s00277-025-06470-3","url":null,"abstract":"<p><p>Previous studies have shown that C-reactive protein/platelet ratio (CPR) before allogeneic hematopoietic stem cell transplantation (HSCT) is a predictor of survival in patients with hematological malignancies. This multicenter retrospective study aimed to evaluate the clinical significance of CPR from diagnosis to HSCT in patients with acute myeloid leukemia (AML) who underwent HSCT. This cohort included patients with AML who underwent their first HSCT between 2016 and 2021. CPR was evaluated at three time points: at diagnosis, after initial therapy, and pre-HSCT. The cut-off value for CPR was set at 0.05 based on previous studies. In total, 196 patients with a median age of 50 years (range: 15-72). High CPR was associated with the myeloblast ratio in the bone marrow at three points and was associated with high transfusion volume and poor performance status at HSCT. Overall survival (OS) at 2 years according to CPR at diagnosis, after initial therapy, and pre-HSCT (low vs. high) was 67.9% vs. 65.6% (P = 0.477), 72.6% vs. 54.8% (P = 0.022), and 73.1% vs. 49.7% (P < 0.001), and non-relapse mortality (NRM) was 15.8% vs. 19.0% (P = 0.557), 13.8% vs. 21.5% (P = 0.201), and 13.0% vs. 27.6% (P = 0.006), respectively. No significant differences were observed in the relapse rates. In multivariate analysis, the high CPR group pre-HSCT was associated with poor OS (HR = 1.86, 95%CI:1.13-3.07, P = 0.015) and higher NRM (HR = 2.52, 95%CI:1.26-5.04, P = 0.009). Pre-HSCT CPR was associated with post-HSCT OS and NRM. CPR is considered a marker that reflects the disease status and patient condition, suggesting the significance of CPR monitoring.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144551791","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The c.2031-2A>C MPO gene variant and the predisposition to myeloid neoplasms.","authors":"Maria Crocioni, Carlotta Nardelli, Anair Graciela Lema Fernandez, Valentina Pierini, Emanuela Falcinelli, Giuseppe Lanzarone, Caterina Matteucci, Cristina Mecucci","doi":"10.1007/s00277-025-06484-x","DOIUrl":"https://doi.org/10.1007/s00277-025-06484-x","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144537864","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"TRAF3IP3::FGFR1: a novel FGFR1 fusion identified in an aggressive case of acute myeloid leukemia.","authors":"Xue Chen, Lili Yuan, Xiaoli Ma, Fang Wang, Yang Zhang, Panxiang Cao, Ping Wu, Tong Wang, Jiaqi Chen, Xiaosu Zhou, Hongxing Liu","doi":"10.1007/s00277-025-06494-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06494-9","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144537865","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immunotherapy-associated hemostatic abnormalities: bleeding and thrombotic complications.","authors":"Yingying Li, Huiwen Jiang, Lili Luo, Heng Mei","doi":"10.1007/s00277-025-06482-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06482-z","url":null,"abstract":"<p><p>Immunotherapy, encompassing immune checkpoint inhibitors, antibody therapy, and chimeric antigen receptor (CAR)-T-cell therapy, has emerged as a groundbreaking approach by harnessing the immune system to selectively target and eliminate malignant cells. However, its widespread application has revealed a spectrum of severe, life-threatening hemostatic abnormalities, including immune thrombocytopenia, Evans syndrome, thrombotic thrombocytopenic purpura, acquired haemophilia A, thrombosis, disseminated intravascular coagulation and CAR-T-associated coagulopathy. These disorders may arise from excessive immune activation, autoantibody production, and intricate crosstalk between inflammatory pathways and coagulation cascades. Despite increasing recognition, their pathophysiological mechanisms remain incompletely understood, and standardized management strategies have yet to be established, posing significant clinical challenges. Given the safety concerns surrounding immunotherapy, timely diagnosis and appropriate intervention of these abnormalities are crucial. This review provides a comprehensive overview of the epidemiology, distinct clinical manifestations, emerging pathophysiological insights, and evolving treatment paradigms of immunotherapy-associated hemostatic abnormalities to enhance clinicians' understanding and guide therapeutic decision-making.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144537861","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sickle cell β-thalassemia diagnosed at age 40: a case report.","authors":"Christos G Nikolaidis, Despoina Gyriki, Dimitrios G Gogos, Elisavet Stavropoulou","doi":"10.1007/s00277-025-06483-y","DOIUrl":"https://doi.org/10.1007/s00277-025-06483-y","url":null,"abstract":"<p><p>Hereditary hemoglobinopathies, including sickle cell disease and thalassemias, affect thousands of newborns annually, predominantly in low-and middle-income countries. Sickle cell β-thalassemia (HbSβ-thal), a form of compound heterozygosity involving β-thalassemia, presents with a wide range of clinical severity depending on the specific mutations. However, the clinical manifestations remain poorly defined. We report the case of a 40-year-old Greek female patient presenting with symptomatic sickle cell β-thalassemia, symptoms of tissue hypoperfusion caused by markedly low hemoglobin levels and notably, bone marrow necrosis. Remarkably, her condition remained undiagnosed until her admission to the emergency department. This case underscores the importance of maintaining a high index of clinical suspicion for the late-onset diagnosis of HbSβ-thal, particularly considering its increased prevalence in certain countries. The successful treatment strategy employed in this case highlights the critical role of individualized care in managing the severe and multifaceted symptoms associated with this genetic disorder, offering valuable insights for clinicians worldwide.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144537863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials.","authors":"Jun Ho Jang, Kinuko Mitani, Yoshiaki Tomiyama, Koji Miyazaki, Koji Nagafuji, Kensuke Usuki, Nobuhiko Uoshima, Tomoaki Fujisaki, Hiroshi Kosugi, Itaru Matsumura, Ko Sasaki, Masahiro Kizaki, Masashi Sawa, Michihiro Hidaka, Naoki Kobayashi, Satoshi Ichikawa, Yuji Yonemura, Kenta Murotani, Mami Shimizu, Akira Matsuda, Keiya Ozawa, Shinji Nakao, Jong Wook Lee","doi":"10.1007/s00277-025-06337-7","DOIUrl":"10.1007/s00277-025-06337-7","url":null,"abstract":"<p><p>The thrombopoietin receptor agonist (TPO-RA) romiplostim has been shown to be efficacious in the treatment of aplastic anemia (AA) refractory to immunosuppressive therapy. However, there are few reports on predictors of TPO-RA efficacy. We analyzed patient clinical and baseline laboratory data from two clinical trials that enrolled patients with refractory AA, a phase 2 dose-finding study (NCT02094417) and a phase 2/3 study (NCT02773290), to determine predictors of romiplostim efficacy. Data from 66 patients (35 from the phase 2 dose-finding study and 31 from the phase 2/3 study), median age 47.0 (range 20-78) years, were analyzed. Of these, 51.5% (34/66) had non-severe AA, 86.4% (57/66) had received prior therapy with anti-thymocyte globulin plus cyclosporine, and 80.3% (53/66) were transfusion-dependent. At 27 weeks, no patients achieved a complete response, 57.4% (35/61) achieved a partial response, 49.0% (24/49) became red blood cell transfusion-independent, and 56.8% (21/37) became platelet transfusion-independent. Univariate logistic regression analysis identified duration from diagnosis (P = 0.040), baseline reticulocyte count (P < 0.001), and platelet count (P < 0.001) as predictors of response to romiplostim at 27 weeks. Receiver operating characteristic curve analysis identified a cutoff value of 30.77 × 10⁹/L at baseline as the threshold reticulocyte count to predict response to romiplostim in patients with refractory AA (sensitivity: 82.9%, specificity: 73.1%). Of note, the initial dose included in this analysis was lower than that used in real-world clinical practice, as data from the phase 2 dose-finding study were used. This study identified factors predicting response to romiplostim at 27 weeks.Trial registration: ClinicalTrials.gov NCT02094417 and NCT02773290.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144537862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}