Annals of Hematology最新文献

{"title":"Comparative efficacy and safety of BCMA-targeted CAR T cells and BiTEs in relapsed/refractory multiple myeloma: a meta-analysis of interventional and real-world studies.","authors":"Pisanupong Techaapornkun, Waranyoo Rojpalakorn, Nuthchaya Mejun, Asmita Khaniya, Arsa Thammahong, May Soe Thu, Nattiya Hirankarn, Palada Pitakkitnukun","doi":"10.1007/s00277-025-06524-6","DOIUrl":"https://doi.org/10.1007/s00277-025-06524-6","url":null,"abstract":"<p><p>Despite therapeutic advances, multiple myeloma (MM) remains incurable, especially in relapsed/refractory (R/R) cases. B-cell maturation antigen (BCMA) is a key target for novel immunotherapies, including chimeric antigen receptor T-cell (CAR-T) therapies and bispecific T-cell engagers (BiTEs), which vary in efficacy, toxicity, and accessibility. To compare the efficacy and safety of BCMA-directed CAR-T therapies and BiTEs in R/R MM through a systematic review and meta-analysis. We systematically searched PubMed, Embase, and the Cochrane Library up to October 2, 2024, for studies evaluating BCMA-directed CAR-T or BiTEs therapies in R/R MM. Twenty-six studies comprising 2,246 patients were included. A random-effects meta-analysis and meta-regression were performed to assess pooled efficacy and safety outcomes and examine the impact of CAR-T constructs and patient-level characteristics. CAR-T therapies showed a higher overall response rate (ORR) of 84% and CR/stringent CR (CR/sCR) of 55%, compared to 65% and 41%, respectively, for BiTEs. Dual-targeted CAR-T therapies (e.g., anti-BCMA + anti-CD38/CD19) had the highest efficacy (ORR 92%). CAR-T was associated with more hematologic toxicity and cytokine release syndrome, while BiTEs had fewer severe events but higher infection rates. Meta-regression confirmed CAR-T significantly outperformed BiTEs. Unlike previous analyses, this study integrates interventional and real-world data, evaluates dual-target CAR-Ts, and offers detailed product- and subgroup-level comparisons. BCMA-targeted CAR-T therapies yield deeper responses but greater toxicity. BiTEs offer safer, though less potent, alternatives, supporting more personalized decisions in BCMA-directed immunotherapy for MM.</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":"145022717","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":"Is splenectomy one of the contributory factors to pulmonary hypertension? An analysis of splenectomized hemolytic anemia and immune thrombocytopenia patients.","authors":"Zeliha Birsin, Ayşe Salihoğlu, Kardelen Ohtaroğlu Tokdil, Betül Zehra Pirdal, Seçkin Bilgiç, Burçak Kılıçkıran Avcı, Deniz Özmen, Ahmet Emre Eşkazan, Muhlis Cem Ar, Zafer Başlar, Tuğrul Elverdi","doi":"10.1007/s00277-025-06583-9","DOIUrl":"https://doi.org/10.1007/s00277-025-06583-9","url":null,"abstract":"<p><p>The development of pulmonary hypertension (PH) after splenectomy is one of the recently controversial issues. This study aims to investigate whether splenectomy itself is an independent risk factor for the development of PH or if the primary contributor to PH development is the underlying condition that necessitated splenectomy. This study was conducted prospectively. We included 21 patients with immune thrombocytopenia (ITP) and 22 with hemolytic anemia. The patients' symptoms were assessed according to a questionnaire form. Blood tests, including N-terminal pro-B type natriuretic peptide (NT-proBNP) and D-dimer levels were done and the 6-minute walk test (6MWT) was performed. PH risk was evaluated using echocardiography (ECHO) and according to the study algorithm, Q-SPECT/CT (perfusion single-photon emission computed tomography/computed tomography) and right heart catheterization (RHC) were performed on selected patients for further assessment. Only one patient in the ITP group was diagnosed as group 2 PH based on ECHO findings and 3 patients with beta thalassemia in the hemolytic anemia group were diagnosed with group 4-5 PH by RHC. There was no statistically significant difference between ECHO-assessed risk for PH in splenectomized patients with hemolytic anemia and ITP (p > 0.05). ECHO risk for PH in the hemolytic anemia patients was found to be low in patients whose hemolysis decreased and transfusion needs disappeared after splenectomy. The results of the study suggest that the development of PH after splenectomy appears to be related to the underlying condition rather than the absence of the spleen.</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":"145022765","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":"Hemoglobin levels and frailty status in generally healthy and active community-dwelling adults age 70 years and older in the three-year DO-HEALTH study.","authors":"Simon Broghammer, Michael Gagesch, Maud Wieczorek, Reto W Kressig, Markus G Manz, E John Orav, Heike A Bischoff-Ferrari","doi":"10.1007/s00277-025-06558-w","DOIUrl":"https://doi.org/10.1007/s00277-025-06558-w","url":null,"abstract":"<p><p>While frailty and anemia are prevalent conditions in aging linked to adverse outcomes, their relationship remains understudied in generally healthy older adults. We conducted a post-hoc observational study among all participants of DO-HEALTH, the largest European clinical trial designed to support healthy aging. Our analysis examined whether baseline hemoglobin levels and anemia are associated with being at least pre-frail at baseline and any yearly follow-up time point over three years. Frailty status was assessed by the Fried frailty phenotype at baseline and at each annual follow-up visit. Participants were considered at least pre-frail by the presence of any frailty domain (fatigue, unintentional weight loss, slowness, weakness, low activity). For both hemoglobin levels and anemia as predictors, we used regression models based on generalized estimating equations controlling for potential confounders. In total, 2,123 of 2,157 participants had available frailty status and hemoglobin levels at baseline and were included in the analysis (mean age 74.9 years; 61.7% women). At baseline, 46.4% (n = 986) were at least pre-frail, while the mean hemoglobin level was 139.7 g/L (SD 12.5), and 6.7% (n = 141) had anemia. In our multivariate prospective analysis, each 10-g/L decrease in baseline hemoglobin levels was associated with 8% higher odds of pre-frailty (adjusted Odds Ratio [aOR] 1.08, 95%CI 1.01-1.16, p 0.02), and baseline anemia was associated with 39% higher odds of pre-frailty over three years (aOR = 1.39, 95%CI 1.02-1.88, p 0.03). In conclusion, our results provide novel insights into the association between hemoglobin levels, anemia, and pre-frailty in generally healthy and active older adults.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145013685","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":"Comparison of prognostication by IPSS-M, IPSS-R and AIPSS-MDS in the context of limited availability of molecular data in daily clinical practice.","authors":"Felicitas Schulz, Carolin Kellersmann, Beate Betz, Barbara Hildebrandt, Annika Kasprzak, Corinna Strupp, Felicitas Thol, Michael Heuser, Christina Ganster, Fabian Beier, Katja Sockel, Wolf-Karsten Hofmann, Andrea Kuendgen, Paul Jaeger, Michael Pfeilstoecker, Michael Lauseker, Sascha Dietrich, Nobert Gattermann, Kathrin Nachtkamp, Detlef Haase, Ulrich Germing","doi":"10.1007/s00277-025-06570-0","DOIUrl":"https://doi.org/10.1007/s00277-025-06570-0","url":null,"abstract":"<p><p>The IPSS-M was developed to revolutionize the prediction of MDS patients' survival by incorporating molecular data. To compensate for lack of access to molecular analyses, the AIPSS-MDS, a supervised machine learning algorithm exclusively based on clinical and cytogenetic data, was developed by the Spanish MDS Group. We used data of the Düsseldorf MDS Registry and included 207 of more than 8500 registry patients whose IPSS-M-requested complete molecular data were known to compare and validate prognostication regarding OS and LFS of the IPSS-M, IPSS-R and AIPSS-MDS. All three tools reliably prognosticated median OS of patients even in a comparatively small patient cohort. The IPSS-M provided the most accurate prediction of median OS while the frequent lack of molecular data persists as an obstacle in daily clinical practice. Due to these circumstances, the IPSS-R remains the prognostication tool with the widest applicability. Based on our data, prognostication using the AIPSS-MDS is also feasible but less precise.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145005881","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":"Unicentric Castleman disease treated with rituximab before surgery: clinicopathologic findings.","authors":"Marco Paulli, Giuseppe Neri, Francesca Antoci, Edoardo D'Este, Marco Minetto, Federico Carpi, Martina La Fauci, Marcello Gambacorta, Marco Lucioni, Luca Arcaini","doi":"10.1007/s00277-025-06527-3","DOIUrl":"https://doi.org/10.1007/s00277-025-06527-3","url":null,"abstract":"<p><p>Castleman disease (CD) is a rare lymphoproliferative disorder with unique clinicopathological features, including two distinct clinical subtypes categorized as unicentric (UCD) and multicentric (MCD). UCD usually involves a single lymph node site presenting with no or minimal local symptoms. Histologically, most UCD cases exhibit regressive hyaline vascular germinal centers, characterized by penetrating vessels, dendritic hyperplasia/dysplasia, and increased interfollicular vascularity. While complete surgical excision is the standard treatment for UCD, cases treated with neoadjuvant anti-CD20 therapy have been reported. Here, we describe a UCD case treated with anti-CD20, that showed only a partial response. We detail the clinical findings and the histological changes observed in the surgical specimen following therapy, and discuss the potential influence of the local microenvironment on the therapeutic efficacy of anti-CD20.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145005862","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":"Bone marrow mastocytosis associated with primary cutaneous follicle center lymphoma: an unusual case report.","authors":"Max Vincent John, Ingrid Simonitsch-Klupp, Johannes Griss, Cora Waldstein, Harald Herrmann, Alexander Gaiger, Karoline V Gleixner, Wolfgang R Sperr, Peter Valent","doi":"10.1007/s00277-025-06588-4","DOIUrl":"https://doi.org/10.1007/s00277-025-06588-4","url":null,"abstract":"<p><p>In a subset of patients with systemic mastocytosis (SM), an associated hematologic neoplasm (AHN) is identified. Most AHN are myeloid neoplasms, whereas lymphoid neoplasms are uncommon. We report on a 70-year-old female patient with bone marrow mastocytosis (BMM) associated with primary cutaneous follicle center lymphoma (PCFCL). Initially, the patient had developed BMM at the age of 61 years. Since then, she suffered from recurrent anaphylaxis and vitiligo. A BM investigation revealed several compact aggregates of MC exhibiting CD25 and the KIT mutation D816V. The basal serum tryptase level ranged between 26.0 and 30.9 ng/ml. Nine years after the initial diagnosis of BMM, the patient developed cutaneous nodular reddish lesions on her scalp. Histologic examination excluded mastocytosis and revealed PCFCL. Radiation therapy resulted in regression of cutaneous lesions. This unusual case demonstrates that patients with BMM may develop a lymphoid neoplasm in extramedullary sites, including skin. However, both neoplasms were separable from each other by site and pathology, which would argue for two separate etiologies and against a classical AHN.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145005866","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":"A cohort of allogeneic hematopoietic stem cell transplantation for pediatric rare diseases (allo-HSCT-PRD): design and preliminary results.","authors":"Shiwen Hu, Ping Wang, Zifeng Li, Wenjin Jiang, Lian Liu, Wenjuan Ma, Ying Huang, Xiaochuan Wang, Xiaonan Du, Xiaowen Qian, Hongsheng Wang, Yi Wang, Yunhui Zhang, Xiaowen Zhai","doi":"10.1007/s00277-025-06600-x","DOIUrl":"https://doi.org/10.1007/s00277-025-06600-x","url":null,"abstract":"<p><p>Rare diseases in children have attracted widespread attention worldwide due to their rarity and difficulty in diagnosis and treatment. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is emerging as a promising and curable therapy for multiple rare diseases. However, rare disease research in China is relatively backward, prompting us to construct the first cohort of allo-HSCT for pediatric rare diseases (allo-HSCT-PRD) involving those who underwent allo-HSCT at the Children's Hospital of Fudan University from 1 January 2014 to 31 October 2024. We concurrently developed a standard data collection, management, and follow-up process, and a dedicated multidisciplinary team to support the work. The allo-HSCT-PRD consisted of 480 participants who were categorized into inborn errors of immunity (IEI) and inborn errors of metabolism (IEM), with 428 and 52 cases, respectively. Among all subjects, the engraftment rate was 84.6%, and the 5-year overall survival (OS) rate was 78.5% (95%CI 74.9% - 82.4%). In particular, patients in the IEM group had greater age and higher implantation and 5-year OS rate. Our next step is to extend our work experience to build allo-HSCT-PRD into a multi-center cohort to boost the development of disease models, exploration of pathogenesis, and clinical trials for new medications.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145005874","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":"Advancement of clinical practice in delivering CAR T-cell therapy: impact on healthcare resource utilization and comparison with autologous stem cell transplantation in patients with relapsed/refractory large B-cell lymphomas.","authors":"Martin Fehr, Matthias Naegele, Michael Greiling","doi":"10.1007/s00277-025-06564-y","DOIUrl":"https://doi.org/10.1007/s00277-025-06564-y","url":null,"abstract":"<p><p>In patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL) who are either refractory to first-line therapy or relapse within 12 months, chimeric antigen receptor (CAR) T-cell therapy is more effective than salvage chemotherapy followed by high-dose chemotherapy and autologous stem cell transplantation (ASCT) as second-line therapy. Adoption of CAR T-cell therapy into routine clinical practice involves a period of adaptation and refinement of clinical processes. We aimed to document the evolution of clinical processes for CAR T-cell therapy during 2022 and 2023, and compare healthcare resource utilization (HCRU) associated with CAR T-cell and ASCT processes in routine clinical practice. ClipMed^PPM software-based process modeling was used to assess HCRU for patients with R/R LBCL receiving CAR T-cell or ASCT therapy, mapping 991 and 1174 processes associated with CAR T-cell therapy in 2023 and 2022, respectively, and 1874 processes associated with ASCT over both years. Improvements in lymphodepletion therapy administration and assessment and management of CAR T-cell therapy-specific adverse events led to a 5-day (30%) reduction in hospitalization and a 15% decrease in total personnel time in the CAR T-cell therapy process from 2022 to 2023. HCRU for CAR T-cell therapy was almost half that of ASCT, with 77% less personnel time for therapy administration. Hospitalization for CAR T-cell therapy was 70%-75% shorter than for ASCT therapy (11-13 vs. 44 days). These patient-centered process efficiencies provide patients with reduced hospitalization time. Understanding this evolution is vital for addressing complexities of advanced treatments, enhancing patient care quality, and optimizing resource allocation.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145005826","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 Dara-VTD induction therapy on stem cell mobilization outcomes in newly diagnosed multiple myeloma patients undergoing autologous stem cell transplantation: a multicenter study.","authors":"Roberta Della Pepa, Salvatore Palmieri, Stefano Rocco, Novella Pugliese, Aldo Leone, Simona Avilia, Marialucia Barone, Rosa Rosamilio, Fabio Trastulli, Danilo De Novellis, Raffaele Fontana, Bianca Serio, Denise Morini, Lorenzo Esposito, Laura De Fazio, Roberta Spisso, Carmine Selleri, Catello Califano, Alessandra Picardi, Mario Annunziata, Fabrizio Pane","doi":"10.1007/s00277-025-06581-x","DOIUrl":"https://doi.org/10.1007/s00277-025-06581-x","url":null,"abstract":"<p><p>Daratumumab combined with bortezomib, thalidomide, and dexamethasone (Dara-VTD) is a highly effective induction therapy for newly diagnosed multiple myeloma (NDMM) patients eligible for autologous stem cell transplantation (ASCT). However, its impact on stem cell mobilization requires a critical evaluation. This study examines the effects of Dara-VTD on stem cell mobilization and collection outcomes. A multicenter retrospective study included 81 consecutive NDMM patients treated with Dara-VTD (from November 2021 to June 2023). Data on stem cell mobilization and collection were compared with 93 historical VTD patients. Mobilization regimens included cyclophosphamide (CTX), vinorelbine + CTX, and chemotherapy-free approaches, with plerixafor used as rescue therapy. Mobilization success was evaluated by CD34 + cell yield, additional agent use, and leukapheresis sessions required. The median CD34 + yield in the Dara-VTD group was 5.1 million cells/kg, with 96.3% of patients achieving > 2 × 10^6 cells/kg of body weight. Plerixafor use was significantly higher in the Dara-VTD group (56.2%) compared to VTD (4.3%), and CTX-based regimens showed superior mobilization efficacy (p = 0.01). Engraftment was faster in the Dara-VTD group, with median neutrophil and platelet recovery at 11 and 13 days, compared to 12 and 17 days in the VTD group (p < 0.05). Dara-VTD maintains the feasibility of ASCT, with comparable stem cell mobilization and collection outcomes to VTD. Mobilization success is influenced by individualized strategies, with CTX and plerixafor playing key roles in optimizing stem cell yield. Despite the challenges posed by daratumumab, stem cell mobilization remains effective, and Dara-VTD does not compromise the transplant process.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144999535","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":"Correction to: 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, Juan Antonio Vera-Goñi, 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/s00277-025-06603-8","DOIUrl":"https://doi.org/10.1007/s00277-025-06603-8","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144999541","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}