Annals of Hematology最新文献

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Autologous stem cell transplantation from 2011 to 2022 in Japanese patients aged ≥ 65 years with relapsed or refractory diffuse large B-cell lymphoma. 2011 - 2022年日本≥65岁复发或难治性弥漫性大b细胞淋巴瘤患者自体干细胞移植研究
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-23 DOI: 10.1007/s00277-025-06519-3
Satoshi Yamasaki, Shohei Mizuno, Kota Yoshifuji, Eri Matsuki, Masashi Sawa, Takashi Akasaka, Naoyuki Uchida, Hitoji Uchiyama, Keisuke Kataoka, Nobuhiro Hiramoto, Yoshinobu Kanda, Kazuya Ishiwata, Toshio Wakayama, Takahiro Fukuda, Makoto Yoshimitsu, Makoto Onizuka, Marie Ohbiki, Yoshiko Atsuta, Ritsuro Suzuki, Shinichi Kako
{"title":"Autologous stem cell transplantation from 2011 to 2022 in Japanese patients aged ≥ 65 years with relapsed or refractory diffuse large B-cell lymphoma.","authors":"Satoshi Yamasaki, Shohei Mizuno, Kota Yoshifuji, Eri Matsuki, Masashi Sawa, Takashi Akasaka, Naoyuki Uchida, Hitoji Uchiyama, Keisuke Kataoka, Nobuhiro Hiramoto, Yoshinobu Kanda, Kazuya Ishiwata, Toshio Wakayama, Takahiro Fukuda, Makoto Yoshimitsu, Makoto Onizuka, Marie Ohbiki, Yoshiko Atsuta, Ritsuro Suzuki, Shinichi Kako","doi":"10.1007/s00277-025-06519-3","DOIUrl":"https://doi.org/10.1007/s00277-025-06519-3","url":null,"abstract":"<p><p>High-dose chemotherapy with autologous stem cell transplantation (ASCT) is an option for patients aged ≥ 65 years with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). Few data are available to select patients suitable for chimeric antigen receptor T-cell (CAR-T) therapy or bispecific antibodies. We retrospectively analyzed the risk factors for poor outcomes for 451 Japanese patients aged ≥ 65 years with R/R DLBCL who received ASCT at either second complete remission or first partial remission (n = 336 and 115, respectively) between 2011 and 2022. CAR-T became commercially available in Japan in March 2019, and the annual number of ASCTs for older patients with R/R DLBCL increased significantly until 2018. However, the number of ASCT cases plateaued in 2018. Multivariate Cox regression analysis identified ≤ 24 months from diagnosis to ASCT (hazard ratio [HR], 1.497) and performance status > 0 at ASCT (HR, 1.460) as independent predictors of overall survival (OS) and an association with late recurrence. The 3-year OS rates were 73.4% (95% confidence interval [CI], 65.8%-79.6%) in patients with > 24 months from diagnosis to ASCT and 58.6% (95% CI, 51.2%-65.2%) in those with ≤ 24 months from diagnosis to ASCT. The 3-year OS rates were 69.4% (95% CI, 62.5%-75.2%) in patients with performance status (PS) = 0 and 60.6% (95% CI, 62.5%-70.4%) in those with PS > 0. CAR-T therapy or bispecific antibodies may be used initially instead of ASCT for early relapsed and refractory patients. However, ASCT remains beneficial for older chemo-sensitive patients with late recurrence and good performance status.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144688691","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}
引用次数: 0
Biphenotypic NK-Large granular lymphocytic leukemia with aggressive clinical features: a case report and literature review. 具有侵袭性临床特征的双表型nk -大颗粒淋巴细胞白血病1例报告及文献复习。
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-23 DOI: 10.1007/s00277-025-06492-x
Beichen Liu, Tengteng Yu, Na Zhou, Shuhua Yi
{"title":"Biphenotypic NK-Large granular lymphocytic leukemia with aggressive clinical features: a case report and literature review.","authors":"Beichen Liu, Tengteng Yu, Na Zhou, Shuhua Yi","doi":"10.1007/s00277-025-06492-x","DOIUrl":"https://doi.org/10.1007/s00277-025-06492-x","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144688692","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}
引用次数: 0
Brentuximab vedotin plus CHP-like regimen for the first-line treatment of PTCL patients: a real-world single center study. Brentuximab vedotin + chp样方案用于PTCL患者的一线治疗:一项真实世界的单中心研究
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-22 DOI: 10.1007/s00277-025-06507-7
Fangshu Guan, Yi Zhang, Xing Guo, Fei Cheng, Enfan Zhang, Jingsong He, Zhen Cai, Yi Zhao
{"title":"Brentuximab vedotin plus CHP-like regimen for the first-line treatment of PTCL patients: a real-world single center study.","authors":"Fangshu Guan, Yi Zhang, Xing Guo, Fei Cheng, Enfan Zhang, Jingsong He, Zhen Cai, Yi Zhao","doi":"10.1007/s00277-025-06507-7","DOIUrl":"https://doi.org/10.1007/s00277-025-06507-7","url":null,"abstract":"<p><p>To evaluate the efficacy and safety of brentuximab vedotin (BV) combined with a CHP-like regimen (cyclophosphamide, doxorubicin, and prednisone) as first-line treatment for newly diagnosed peripheral T-cell lymphoma (PTCL). We retrospectively analyzed the clinical data of patients with PTCL who received BV in combination with a CHP-like regimen as first-line therapy between January 2021 and October 2023. Fifty patients were included in this study, with a median age of 57 years (rang, 22-86 years). The median number of BV cycles administered was six (range, 2-8 cycles). After a median follow-up of 35.2 months (95% confidence interval [CI], 30.9-39.5), the overall response rate (ORR) was 80%, with 44% of the patients achieving a complete response (CR). In patients with the ALK-positive anaplastic large cell lymphoma (ALCL) subtype, the CR rate was 70.6%. The median progression-free survival (PFS) was 39.7 months (95% CI, 14.7-NA), whereas the median overall survival (OS) was not reached (the 1-year OS rate, 90%). Multivariate analysis indicated that bone marrow infiltration at diagnosis was the only factor that was significantly associated with poor OS. Neutropenia (81.3%) was the most common adverse event, and peripheral neuropathy was observed in 16.7% of patients. This real-world study suggests that BV combined with a CHP-like regimen is an effective and safe first-line treatment for patients with newly diagnosed PTCL.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144688693","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}
引用次数: 0
Validation of the Spanish version of the EORTC QLQ-CML24 questionnaire for assessment of health-related quality of life in patients with chronic myeloid leukemia. 西班牙语版EORTC QLQ-CML24问卷对慢性髓性白血病患者健康相关生活质量评估的验证
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-22 DOI: 10.1007/s00277-025-06516-6
Elisa Lopez-Fernández, Soledad de Linares-Fernández, Maria Nieves Perez-Marfil, Alicia Rodríguez-Fernández, Magdalena Anguita-Arance, Maria Del Carmen Fernández-Valle, Maria Jose Ramírez-Sánchez, Maria Concepción Ruiz-Nuño, Werner González-Molina, Carmen Ferrer-Chaves, Carmen Avellaneda-Molina, Jose Manuel Puerta-Puerta
{"title":"Validation of the Spanish version of the EORTC QLQ-CML24 questionnaire for assessment of health-related quality of life in patients with chronic myeloid leukemia.","authors":"Elisa Lopez-Fernández, Soledad de Linares-Fernández, Maria Nieves Perez-Marfil, Alicia Rodríguez-Fernández, Magdalena Anguita-Arance, Maria Del Carmen Fernández-Valle, Maria Jose Ramírez-Sánchez, Maria Concepción Ruiz-Nuño, Werner González-Molina, Carmen Ferrer-Chaves, Carmen Avellaneda-Molina, Jose Manuel Puerta-Puerta","doi":"10.1007/s00277-025-06516-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06516-6","url":null,"abstract":"<p><strong>Objective: </strong>The EORTC QLQ-CML24 questionnaire was developed and validated to enhance research in the health-related quality of life (HRQoL) of patients with chronic myeloid leukemia (CML) as a way to more thoroughly capture the burden of disease and therapy and facilitate treatment decisions in this population. We aimed to examine the structure and psychometric properties of the Spanish version.</p><p><strong>Methods: </strong>Data were collected from 186 CML patients in 9 centers of Andalusia (Spain). The validation process integrated the analysis of feasibility, internal consistency, construct validity and convergent validity. The core EORTC QLQ-C30, Short Form-36 (SF-36), Hospital Anxiety and Depression Scale (HADS) and the Connor-Davison Resilience Scale (CD-RISC-10) were used to assess convergent validity.</p><p><strong>Results: </strong>Feasibility of the Spanish version was excellent, with all patients self-completing the questionnaire and with no missing answers. Internal consistency, assessed by Cronbach's alpha coefficients, was 0.88. The CFA goodness-of-fit indicators confirmed that the model is acceptable (Comparative Fit Index (CFI)=0.87; Tucker-Lewis Index (TLI)=0.85 and Root Mean Square Error of Approximation (RMSEA)=0.07). Strong and moderate correlations (Spearman rho values ranging from 0.4 to >0.6) were found with the scales of QLQ-C30 addressing functioning, symptoms and fatigue and with all domains of the SF-36.</p><p><strong>Conclusions: </strong>Our findings support the use of the Spanish version of the EORTC QLQ-CML24 as a valuable complementary measure to the EORTC QLQ-C30, for providing a comprehensive assessment of HRQoL in patients with CML.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144688694","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}
引用次数: 0
Factors influencing prenatal diagnosis of deletional hemoglobin H disease in thalassemia prevention and control program, Southern China. 华南地区地中海贫血防治项目中H型血红蛋白缺失病产前诊断的影响因素
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-21 DOI: 10.1007/s00277-025-06505-9
Ruirui Chen, Rui Zhang, Xiaohong He, Quanfu Zhang
{"title":"Factors influencing prenatal diagnosis of deletional hemoglobin H disease in thalassemia prevention and control program, Southern China.","authors":"Ruirui Chen, Rui Zhang, Xiaohong He, Quanfu Zhang","doi":"10.1007/s00277-025-06505-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06505-9","url":null,"abstract":"<p><p>The disease burden associated with deletional HbH disease and the health quality of these patients require attention in areas with high Thalassemia prevalence. We examined the associations between clinical and socioeconomic factors and prenatal diagnosis in couples at risk of having a fetus with deletional HbH (SEA/-α3.7 or SEA/-α4.2) disease in Southern China. Data collection was based on 521 medical records of at-risk couples identified between 2017 and 2022 at the largest prenatal diagnosis center in Shenzhen. Univariate analysis and multivariate logistic regression modelling were conducted. The fully adjusted model revealed an increasing trend in the prenatal diagnosis rate from 2019 to 2022, and socioeconomic inequalities that those with a college degree or higher being 1.92 (95% CI: 1.19-3.07) times more likely to undergo prenatal diagnosis compared to those with the lowest level of education, adjusted for three clinical indications, maternal age at expected delivery and study year. To address socioeconomic disparities in prenatal diagnosis for at-risk couples of deletional HbH disease and improve health outcomes, key priorities include: reducing financial barriers, standardizing genetic counseling with decision-support strategies, early identification of fetal thalassemia at birth if prenatal diagnosis is missed, and optimizing long-term management to enhance affected children's health quality.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144673722","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}
引用次数: 0
Distinct structural and numerical chromosome abnormalities determine the MYC status in diffuse large B-Cell lymphoma and help differentiate from Burkitt lymphoma: a cytogenetic data analysis using unsupervised and AI-driven prediction models. 不同的结构和数量染色体异常决定弥漫性大b细胞淋巴瘤的MYC状态,并有助于与伯基特淋巴瘤区分:使用无监督和人工智能驱动的预测模型的细胞遗传学数据分析。
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-19 DOI: 10.1007/s00277-025-06508-6
Rolando García, Shankar Srinivasan, Mehta Shashi, Frederick Coffman, Prasad Koduru
{"title":"Distinct structural and numerical chromosome abnormalities determine the MYC status in diffuse large B-Cell lymphoma and help differentiate from Burkitt lymphoma: a cytogenetic data analysis using unsupervised and AI-driven prediction models.","authors":"Rolando García, Shankar Srinivasan, Mehta Shashi, Frederick Coffman, Prasad Koduru","doi":"10.1007/s00277-025-06508-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06508-6","url":null,"abstract":"<p><p>The aim of this study was to identify recurrent chromosome abnormalities (RCAs) to distinguish these entities and to test their specificities in a set of predictor models. The study analyzed publicly available cytogenetic data to construct models to predict DLBCL and BL. The Fisher Exact test (2-tail) was used to assess the significance of differences in the number of aberrations between groups, as well as to determine correlations between RCAs and the two entities. A p-value less than 0.05 was considered significant. Discrimination analysis was determined by the receiver operating curve (ROC). All analyses were performed using the R package. The SAS software package was used to develop a logistic regression model. Two subsequent supervised models were constructed using a larger dataset (n = 515) to confirm initial findings. A p-value < 0.05 was considered significant. Several RCAs were associated with DLBCL, including 1p-, 1q-, -2, + 3, -4, + 5, 6p gain, 6q-, + 7, -8, 9q-, -10/-15, -10/-14, + 11, +12, 14q-, 15q-, + 16, 16q-,17p-, + 18, 19p-, and 22q-. Of these, + 7, 15q-, + 16 and + 18 were more prevalent in MYC + DLBCL vs. BL, whereas 1q gain and 13q- were consistent with BL. The specificity of supervised models ranged from 90 to 100%, whereas the accuracy of the unsupervised logistic regression model was 85%. Our findings revealed unique RCAs that may be used in combination with model classifiers to augment diagnostic accuracy and help clinicians better manage these patients.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144666884","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}
引用次数: 0
Exploring thromboembolic risk factors in polycythemia vera: from current evidence to PROSPERO study design. 真性红细胞增多症的血栓栓塞危险因素:从目前的证据到PROSPERO研究设计。
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-18 DOI: 10.1007/s00277-025-06466-z
Valerio De Stefano, Francesco Passamonti, Francesca Palandri, Francesco Ramundo, Elena Rossi, Silvia Betti, Lorenzo Fagiolo, Paola Guglielmelli, Davide Pio Abagnale, Novella Pugliese, Daniele Cattaneo, Alessandra Iurlo, Filippo Branzanti, Alessandra Dedola, Hillary Catellani, Alessia Tieghi, Marco Basso, Elisabetta Calistri, Elena Maria Elli, Elena Masselli, Erika Morsia, Giulia Benevolo, Massimo Breccia, Vincenza de Fazio, Maria Di Perna, Monia Marchetti, Marco Santoro, Agostino Tafuri, Chiara Castiglioni, Chiara Rotella, Sergio Siragusa, Alessandro Maria Vannucchi
{"title":"Exploring thromboembolic risk factors in polycythemia vera: from current evidence to PROSPERO study design.","authors":"Valerio De Stefano, Francesco Passamonti, Francesca Palandri, Francesco Ramundo, Elena Rossi, Silvia Betti, Lorenzo Fagiolo, Paola Guglielmelli, Davide Pio Abagnale, Novella Pugliese, Daniele Cattaneo, Alessandra Iurlo, Filippo Branzanti, Alessandra Dedola, Hillary Catellani, Alessia Tieghi, Marco Basso, Elisabetta Calistri, Elena Maria Elli, Elena Masselli, Erika Morsia, Giulia Benevolo, Massimo Breccia, Vincenza de Fazio, Maria Di Perna, Monia Marchetti, Marco Santoro, Agostino Tafuri, Chiara Castiglioni, Chiara Rotella, Sergio Siragusa, Alessandro Maria Vannucchi","doi":"10.1007/s00277-025-06466-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06466-z","url":null,"abstract":"<p><p>Polycythemia vera (PV) is a chronic myeloproliferative neoplasm with a substantial risk of thromboembolic events (TEs), which contribute to morbidity and mortality. Traditional thrombotic risk stratification primarily considers age and thrombosis history, yet these parameters alone do not capture the complexity of thrombotic risk. Growing evidence highlights the role of additional factors influencing the risk of TEs, underscoring the need for a more comprehensive approach to patient stratification. This paper reviews the current understanding of thromboembolic risk factors in PV and provides the rationale, methodology, and expected contributions of the PROSPERO study, a prospective, multicenter study designed to improve thrombotic risk assessment in patients with high-risk PV. By examining established (advanced age, prior TEs, cardiovascular comorbidities) and emerging thromboembolic risk factors, including specific hematologic parameters, the study aims to emphasize their impact and potential synergistic interactions on thrombotic risk. This review also evaluates the efficacy and limitations of current therapies, such as hydroxyurea (HU), interferons, and ruxolitinib, in preventing TEs, and further underscores the need for comprehensive predictive models to guide individualized management strategies. The PROSPERO study focuses on high-risk PV patients who experienced at least one prior TE and receive either HU or ruxolitinib, aiming to identify predictive factors for TEs and their individual and combined contributions to thrombotic risk by collecting longitudinal data on clinical, laboratory, and treatment-related parameters. PROSPERO aims to identify and validate new variables that can inform the development of precise, integrated prediction models. The findings are expected to enable tailored treatment approaches, ultimately reducing TE recurrence in high-risk PV populations.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144658188","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}
引用次数: 0
Treatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey. 阵发性夜间血红蛋白尿患者的治疗负担:一项深度访谈调查。
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-18 DOI: 10.1007/s00277-025-06486-9
Yasutaka Ueda, Naoshi Obara, Shikiko Ueno, Masatoshi Sakurai, Kohei Hosokawa, Tatsunori Murata, Yuta Fukuoka, Nozomi Hayama, Masami Yamashita, Yoshiaki Ogawa, Kensuke Usuki, Takayuki Ikezoe, Tatsuya Kawaguchi, Naoki Hosen, Yuzuru Kanakura, Akihiko Gotoh, Jun-Ichi Nishimura
{"title":"Treatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey.","authors":"Yasutaka Ueda, Naoshi Obara, Shikiko Ueno, Masatoshi Sakurai, Kohei Hosokawa, Tatsunori Murata, Yuta Fukuoka, Nozomi Hayama, Masami Yamashita, Yoshiaki Ogawa, Kensuke Usuki, Takayuki Ikezoe, Tatsuya Kawaguchi, Naoki Hosen, Yuzuru Kanakura, Akihiko Gotoh, Jun-Ichi Nishimura","doi":"10.1007/s00277-025-06486-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06486-9","url":null,"abstract":"<p><p>Paroxysmal nocturnal hemoglobinuria (PNH) is a lifelong, clonal hematologic disease posing life-threatening risks if untreated. The prognosis for PNH has improved with the advent of C5 inhibitors, which are now the standard of care where available. As treatment options continue to expand, healthcare providers can better address both PNH management and the impact of treatment on patients' daily lives. To investigate the burden of disease and key factors taken into consideration when patients with PNH choose their preferred treatment, we conducted an in-depth patient interview survey. Of survey participants (N = 30), 70.0% were receiving intravenous C5 inhibitors. Notably, 56.7% of all patients reported needing ≥ 1 hospital visit per month, and 46.7% required a day off from work or school for visits (33.3% among those receiving intravenous C5 inhibitors). A frequently reported burden of PNH treatment was financial concern, including the cost of treatment, hospital visits, and the negative impact on income. Additionally, burden related to waiting times and distance from the hospital and the overall time spent on outpatient PNH care were identified with similar results for patients receiving intravenous C5 inhibitors. These results suggest that the time and effort to get to treatment centers and the time required to receive treatment for PNH were critical unmet needs in PNH care. Our study indicates that persistent burdens associated with current PNH care should be taken into account alongside therapeutic effectiveness when making treatment decisions. Further analysis with a larger sample size is required to confirm these findings.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144658189","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}
引用次数: 0
Clinical features and fusion gene analysis of two Torque Teno Mini virus associated acute promyelocytic leukemia cases. 2例Torque Teno Mini病毒相关急性早幼粒细胞白血病临床特点及融合基因分析
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-18 DOI: 10.1007/s00277-025-06501-z
Deyan Liu, Jiaqi Chen, Yu Luo, Shu Yan, Hua Nan, Xue Chen, Zhongyuan Lin, Liejun Jiang, Huihe Tang, Hongyan Ma, Yumeng Niu, Jiancheng Fang, Panxiang Cao, Lili Yuan, Xiaoli Ma, Xiaosu Zhou, Fanyong Lv, Yan Dai, Hongxing Liu
{"title":"Clinical features and fusion gene analysis of two Torque Teno Mini virus associated acute promyelocytic leukemia cases.","authors":"Deyan Liu, Jiaqi Chen, Yu Luo, Shu Yan, Hua Nan, Xue Chen, Zhongyuan Lin, Liejun Jiang, Huihe Tang, Hongyan Ma, Yumeng Niu, Jiancheng Fang, Panxiang Cao, Lili Yuan, Xiaoli Ma, Xiaosu Zhou, Fanyong Lv, Yan Dai, Hongxing Liu","doi":"10.1007/s00277-025-06501-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06501-z","url":null,"abstract":"<p><p>Torque Teno Mini Virus (TTMV), a member of the Anelloviridae family, is a commensal component of the human virome. Since the initial identification of the TTMV::RARA fusion gene as a novel driver of acute promyelocytic leukemia (APL), 15 cases have been reported in retrospective studies. With advancements in diagnostic methods and increased awareness, the number of newly diagnosed cases has risen, and the clinical and molecular characteristics of TTMV::RARA-APL are becoming clearer. We systematically identified the clinical characteristics, fusion gene analysis, and treatment protocols of two pediatric APL patients harboring the TTMV::RARA fusion on this basis. While the detection of TTMV::RARA contributes to defining pathogenic fusion gene and MRD monitoring indicators in non-PML::RARA-APL cases, the precise pathogenic mechanisms of this ubiquitous symbiotic virus warrant further investigation.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144658187","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}
引用次数: 0
Consequences and impacts of PEG-IFNα2a shortage: first lessons from a MPN French center. PEG-IFNα2a缺乏的后果和影响:来自MPN法国中心的初步经验。
IF 3 3区 医学
Annals of Hematology Pub Date : 2025-07-17 DOI: 10.1007/s00277-025-06510-y
Laura Cailly, Wayne-Corentin Lambert, Jean-Richard Eveillard, Brigitte Pan-Petesch, Laura Herbreteau, Laetitia Rio, Lanig Civi, Adrian Tempescul, Florence Dalbies, Hussam Saad, Cristina Bagacean, Marie-Anne Couturier, Gaelle Guillerm, Eric Lippert, Jean-Christophe Ianotto
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引用次数: 0
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