Annals of Hematology最新文献

{"title":"Tandem autologous hematopoietic stem cell transplantation in multiple myeloma: A historical perspective and current challenges.","authors":"Xueting Wang, Yushan Cui, Yaomei Wang, Baijun Fang","doi":"10.1007/s00277-025-06563-z","DOIUrl":"https://doi.org/10.1007/s00277-025-06563-z","url":null,"abstract":"<p><p>Multiple myeloma (MM) is a heterogeneous and relapse-prone hematologic malignancy that remains incurable. For newly diagnosed patients aged 70 years or younger, who are eligible for transplantation, autologous hematopoietic stem cell transplantation (auto-HSCT) is the preferred first-line treatment. In patients with high-risk multiple myeloma (HRMM), some studies have demonstrated that tandem auto-HSCT provides notable benefits over single auto-HSCT, particularly in extending progression-free survival (PFS) and overall survival (OS). Although allogeneic hematopoietic stem cell transplantation (allo-HSCT) currently offers the only potential for long-term cure in MM, its application is limited by high transplant-related mortality (TRM) and the risk of graft-versus-host disease (GVHD). In recent years, the emergence of novel therapies, including proteasome inhibitors, immunomodulatory drugs, monoclonal antibodies, and chimeric antigen receptor T-cell (CAR-T) therapy, has posed new challenges to the role of tandem auto-HSCT in MM treatment. This review aims to critically examine the efficacy differences between tandem and single auto-HSCT, and sequential allo-HSCT following auto-HSCT. Furthermore, it will rigorously evaluate the role and challenges of tandem auto-HSCT within the evolving therapeutic landscape.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145063449","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":"Access to hydroxyurea in Sub-Saharan Africa remains a neglected response to sickle cell disease: reply to use of hydroxyurea in French-speaking Sub-Saharan Africa article.","authors":"Paulo João Ferreira de Almeida","doi":"10.1007/s00277-025-06513-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06513-9","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145063389","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":"Impact of best response to ibrutinib plus Bendamustine and rituximab on PFS in MCL: a secondary analysis of SHINE.","authors":"Yuko Mishima, Daigo Hashimoto, Michiko Ichii, Noriko Fukuhara, Toshiki Uchida, Koji Kato, Ai Omi, Yosuke Koroki, Kaname Shiga, Dai Maruyama","doi":"10.1007/s00277-025-06569-7","DOIUrl":"https://doi.org/10.1007/s00277-025-06569-7","url":null,"abstract":"<p><p>Ibrutinib is a first-in-class oral inhibitor of Bruton's tyrosine kinase, which was investigated for the first-line treatment of mantle cell lymphoma (MCL) in the randomized, double-blind, phase 3 SHINE study. In SHINE, ibrutinib plus bendamustine and rituximab (BR) demonstrated superior progression-free survival (PFS) versus placebo plus BR in patients aged ≥ 65 years with previously untreated stage II-IV MCL. In this secondary efficacy analysis of SHINE, we assessed the correlation between best response and PFS (n = 523; 70% male; median age 71.0 years). After a median follow-up of 94.5 months, patients achieving complete response (CR) had longer median PFS in the ibrutinib (97.8 months) and placebo (87.9 months) arms than those with partial response (PR; 27.6 and 16.7 months, respectively) or progressive/stable disease (2.9 and 3.4 months, respectively). In the multivariate logistic regression analysis, patients receiving ibrutinib plus BR were more likely to achieve CR than those receiving placebo plus BR (odds ratio 1.48; 95% confidence interval 1.00-2.22). In conclusion, prolonged long-term PFS was more likely in patients with MCL who achieved CR following treatment with either ibrutinib plus BR or placebo plus BR, while CR was more likely in patients who had received ibrutinib plus BR. TRIAL REGISTRATION: EU Clinical Trials Register (EudraCT) identifier 2012-004056-11 (registered 15 January 2013) WHO Universal Trial Number U1111-1137-0389.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145051808","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":"An unconquered challenge in MDS: review of pathophysiology, clinical manifestations, and management options of MDS with thrombocytopenia.","authors":"Xiaoyi Chen, Mihir Shukla, Jun H Choi","doi":"10.1007/s00277-025-06374-2","DOIUrl":"https://doi.org/10.1007/s00277-025-06374-2","url":null,"abstract":"<p><p>Myelodysplastic syndromes (MDS) is a heterogeneous group of myeloid clonal disorder resulting in bone marrow failure with a tendency to acute myeloid leukemia transformation. MDS is characterized by a variable degree of clonal cytopenia. Compared to anemia, thrombocytopenia is less common but presents more significant challenges due to high risk of acute complications and dearth effective treatment options. Platelet transfusions are effective in increasing platelet counts but provide limited and transient benefits, along with associated risks of transfusions. Anti-fibrinolytic drugs have been attempted including in clinical trial settings but its efficacy remains unproven. Successful development of thrombopoietin agonists appeared promising especially in other conditions associated with thrombocytopenia but its utility in MDS has been controversial. Two of the novel thrombopoietin receptor agonists (TPO-RA), romiplostim and eltrombopag have established clinical activity in immune thrombocytopenic purpura (ITP) and have been explored for the treatment of thrombocytopenia in MDS. Due to early research data showing TPO-RA leading to a small increase in blast counts and possibly promoting leukemic transformation, subsequent clinical trials sought to establish its safety and efficacy in MDS. Despite considerable amount of evidence demonstrating favorable safety profiles in lower risk MDS, many hematologists are often hesitant to use TPO-RA to treat thrombocytopenia in MDS due to theoretical concern of stimulating blasts. In higher risk MDS the safety is not proven and certainly requires more investigation. In this review, we aim to highlight pathophysiology of thrombocytopenia in MDS and provide comprehensive management strategies supported by past and current clinical research data.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145038941","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":"Multicenter, real-world observational study of AVA therapy following ELT/HET switching in Chinese children with persistent/chronic primary ITP.","authors":"Jingjing Liu, Zhifa Wang, Nan Wang, Jingyao Ma, Lijuan Wang, Yan Liu, Jinxi Meng, Shuyue Dong, Yu Hu, Juntao Ouyang, Zhenping Chen, Xiaoling Cheng, Runhui Wu","doi":"10.1007/s00277-025-06567-9","DOIUrl":"10.1007/s00277-025-06567-9","url":null,"abstract":"<p><p>Avatrombopag (AVA), a second-generation thrombopoietin receptor agonist (TPO-RA), has demonstrated efficacy in pediatric persistent/chronic immune thrombocytopenia (ITP). However, critical evidence gaps persist regarding treatment-switching strategies between TPO-RAs, particularly when transitioning from eltrombopag (ELT) or hetrombopag (HET) to AVA. This multicenter cohort study evaluated 55 pediatric ITP patients unresponsive to or relapsing after ELT (n = 46) or HET (n = 9) who underwent AVA switch therapy. Outcomes included platelet response (≥ 30 × 10⁹/L without rescue therapy), bleeding events, concomitant medication reduction, and safety. Sustained response rates reached 48.4% (ELT-to-AVA) and 33.3% (HET-to-AVA), with median response durations of 10 and 7 days respectively. Platelet elevation during AVA treatment was resolved with dosage changes or discontinuation. AVA significantly reduced bleeding, ITP medications, and rescue therapy, with side effects such as gastrointestinal symptoms, headaches, and fatigue (grades 1-2). AVA demonstrates potential as a safe and effective bridging therapy for TPO-RA refractory pediatric ITP, offering hematological stabilization while reducing treatment burden. These findings address current evidence deficiencies in TPO-RA switching protocols.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032549","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":"An Italian real-world multicenter study of patients with refractory/relapsed functional high-risk multiple myeloma patients treated with second-line therapies.","authors":"Danilo De Novellis, Salvatore Palmieri, Stefano Rocco, Daniele Derudas, Roberta Della Pepa, Daniela Roccotelli, Daniela Esposito, Chiara Masucci, Emilia Gigliotta, Maria Lucia Barone, Emanuela Morelli, Antonio Lazzaro, Rosario Bianco, Fabrizio Accardi, Luana Marano, Matteo Bonanni, Anna Maria Della Corte, Bianca Serio, Eleonora Urciuoli, Michela Rizzo, Raffaele Fontana, Manuela Arcamone, Rossella Iula, Anna Dandolo, Aldo Leone, Maria Gabriella Rascato, Maria Di Perna, Antonietta Pia Falcone, Lucia Morello, Gianpaolo Marcacci, Nunziata Giuseppe Rodolfo, Ferdinando Frigeri, Catello Califano, Angelo Michele Carella, Antonio Maria Risitano, Mario Annunziata, Fabrizio Pane, Valentina Giudice, Cirino Botta, Carmine Selleri","doi":"10.1007/s00277-025-06572-y","DOIUrl":"https://doi.org/10.1007/s00277-025-06572-y","url":null,"abstract":"<p><p>Functional high risk multiple myeloma (FHRMM) remains a challenging entity with poor outcomes and limited survival, and there is no international consensus on optimal second-line therapeutic strategies in relapsed/refractory patients. In this multicenter real-world retrospective study, we investigated clinical characteristics and outcomes of a total of 62 FHRMM patients previously treated with a first-line daratumumab-based quadruplet regimen or who relapsed within 12 months after frontline autologous stem cell transplantation (ASCT). In our cohort, the overall response rate was 61%, with 42% of patients achieving a very good partial response (VGPR) or better. Similarly, median progression-free survival (PFS) was not reached with an estimated 12-month PFS rate of 54%, as well as median overall survival (OS) with a 12-month OS rate of 72%. Factors associated with worse PFS included extramedullary disease, prior lenalidomide maintenance, lack of ASCT consolidation, an ECOG score ≥ 2, advanced disease stage, and salvage therapy without carfilzomib-lenalidomide-dexamethasone. In conclusion, second-line management of FHRMM following daratumumab-bortezomib-thalidomide-dexamethasone induction is highly challenging and variable across centers, due to the lack of standardized international guidelines. Carfilzomib-based regimens demonstrated some clinical benefits, especially in lenalidomide-naïve patients; however, outcomes remained suboptimal in FHRMM population who may benefit from novel therapies administered as earlier treatment lines. Larger prospective trials are needed to optimize FHRMM clinical management and improve patient outcomes.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028895","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":"An international prognostic index to predict the early chemoimmunotherapy failure of diffuse large B-cell lymphoma.","authors":"Qifan Xu, Yang Li, Beibei Xin, Chenyang Wang, Xinya Tao, Shihai Li, Hui Xiong, Xiaohua Zhou, Li Wang, Weili Zhao","doi":"10.1007/s00277-025-06525-5","DOIUrl":"https://doi.org/10.1007/s00277-025-06525-5","url":null,"abstract":"<p><strong>Background: </strong> Approximately 30-40% of diffuse large B-cell lymphoma (DLBCL) patients will develop relapse/refractory disease, who may benefit from novel therapies, such as CAR-T cell therapy. Thus, accurate identification of individuals at high risk of early chemoimmunotherapy failure (ECF) is crucial. Methods. Two prognostic models were developed to predict the ECF of DLBCL using clinical variables, namely the ECF-IPI-basic model (n = 1200) and the ECF-IPI-advance model (n = 699), respectively. 8 variables included age, gender, Ann Arbor stage, Hans classification, MYC and BCL2 double expression (DE), number of extranodal involvement sites, lactate dehydrogenase (LDH) and Eastern Cooperative Oncology Group performance status (ECOG PS) were considered to construct the basic model. The advanced model incorporated four additional biomarkers, interleukin-8 (IL-8), interleukin-2 receptor (IL-2R), β2-microglobulin (β2-MG), and D-dimer, totaling 12 predictive variables. Results. The ECF-IPI-basic model includes 5 variables, which was constructed with the formula of Age + Ann Arbor stage + DE (MYC and BCL2 double expression) + ECOG + LDH (lactate dehydrogenase). The ECF-IPI-advance model includes 7 variables, specifically, it was constructed with the formula of Age × Sex + Ann Arbor stage + DE + ECOG + LDH + IL-2R. Compared with the IPI score, greater discriminatory capacity was observed in both of the ECF-IPI-basic model (AUC, 0.768 vs. 0.701, p < 0.001) and the ECF-IPI-advance model (AUC, 0.824 vs. 0.724, p < 0.001) in identifying ECF. Conclusions. Overall, this study provides two potent ECF-IPI models that can effectively distinguish the patients with ECF from DLBCL, contributing to improve the prognosis of DLBCL.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028889","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":"Expanding the frontier of CAR therapy: comparative insights into CAR-T, CAR-NK, CAR-M, and CAR-DC approaches.","authors":"Yiling Zhang, Rong Hu, Xiaoling Xie, Yuhua Li","doi":"10.1007/s00277-025-06538-0","DOIUrl":"https://doi.org/10.1007/s00277-025-06538-0","url":null,"abstract":"<p><p>Chimeric antigen receptor (CAR) therapies have demonstrated remarkable clinical efficacy in hematological malignancies, validating their therapeutic potential. However, challenges such as therapeutic resistance and limited accessibility hinder their broader application. To overcome these limitations, alternative CAR-based cell therapies, including CAR-Natural Killer (CAR-NK), CAR-macrophage (CAR-M), and CAR-dendritic cell (CAR-DC) therapies, have been proposed. Compared with CAR-T, CAR-NK cells have a higher safety profile in terms of cytokine release syndrome (CRS) and neurotoxicity, while being naturally cytotoxic, making them a promising option. Despite these advantages, CAR-NK therapy is limited by issues such as insufficient tissue infiltration and low transduction efficiency. CAR-M cells, with their potent infiltration capabilities and ability to function as antigen-presenting cells, also hold promise but face challenges related to suboptimal viral transduction efficiency. CAR-DCs are emerging as a highly promising approach and are currently undergoing active investigation. This review summarizes the profiles, current clinical trials, and comparative advantages and limitations of CAR-T, CAR-NK, CAR-M, and CAR-DC therapies. Finally, we discuss the key challenges to be addressed and the future prospects of these evolving CAR-based cell therapies.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028834","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":"Combined heterozygosity for the highly unstable variant hemoglobin Taybe, and α-thalassemia as a rare cause of hemolytic anemia.","authors":"Saskia N Nagel, Joaquin Brintrup, Yazan Ghannam, Andreas Stallmach, Andreas Hochhaus, Cristina Ripoll, Holger Cario, Karin G Schrenk","doi":"10.1007/s00277-025-06578-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06578-6","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022774","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":"Age interaction in associations between geriatric nutritional risk index and prognosis in diffuse large B-cell lymphoma.","authors":"Kei Fujita, Hikaru Tsukasaki, Shin Lee, Tetsuji Morishita, Eiju Negoro, Kana Oiwa, Takeshi Hara, Hisashi Tsurumi, Takanori Ueda, Takahiro Yamauchi","doi":"10.1007/s00277-025-06477-w","DOIUrl":"https://doi.org/10.1007/s00277-025-06477-w","url":null,"abstract":"<p><p>To evaluate whether age modifies the association between the geriatric nutritional risk index (GNRI) and overall survival (OS) in patients aged ≥ 18 years with newly diagnosed diffuse large B-cell lymphoma (DLBCL), we conducted a multi-centre retrospective study of 552 patients. Multivariable Cox regression with restricted cubic spline (RCS) modelling showed that GNRI was significantly associated with OS, but the relationship was non-linear (P for non-linearity = 0.0158). A three-dimensional Cox-RCS model revealed that older age was associated with a higher mortality risk. The association between lower GNRI and worse OS was consistent across all age groups, including younger patients. Among patients with low GNRI (< 92), the most common pattern of malnutrition involved normal albumin (Alb) and body mass index (BMI). This pattern accounted for approximately 40% of low-GNRI patients, regardless of age group (based on thresholds of 65, 70, or 75 years). Survival curves demonstrated that a low GNRI (< 92) predicted poor prognosis, regardless of whether Alb or BMI was the main contributing factor. There was no evidence of interaction between age and GNRI on prognosis. Malnutrition, as measured by GNRI, adversely affects survival in both younger and older DLBCL patients.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022788","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}