American Journal of Hematology最新文献

{"title":"Long-Term Outcome of Fanconi Anemia Patients From the Italian Registry on Behalf of the Marrow Failure Study Group of the AIEOP (Italian Association for Pediatric Haematology-Oncology)","authors":"Erica Ricci, Francesca Bagnasco, Filomena Pierri, Antonio Risitano, Piero Farruggia, Maura Faraci, Camilla Frieri, Paola Corti, Ugo Ramenghi, Walter Barberi, Giuseppe Menna, Giovanna Giagnuolo, Marta Pillon, Maurizio Miano, Paolo Di Bartolomeo, Marco Zecca, Elena Mastrodicasa, Rosamaria Mura, Beatrice Pinazzi, Laura Luti, Maria Licciardello, Angelica Barone, Marinella Veltroni, Giovanni Palazzi, Federico Verzegnassi, Francesca Patriarca, Daniela Onofrillo, Simone Cesaro, Roberta Ghilardi, Irene D'Alba, Rosa Angarano, Luca Arcuri, Andrea Beccaria, Ramona Tallone, Adriana Zatterale, Carlo Dufour","doi":"10.1002/ajh.27724","DOIUrl":"https://doi.org/10.1002/ajh.27724","url":null,"abstract":"We analyzed 193 Fanconi anemia patients from the Italian Registry, focusing on hematological outcome, cancer risk, and mortality, both in transplanted (<i>n</i> = 130, 67.4% of the cohort) and non-transplanted (<i>n</i> = 63, 36.6% of the cohort) patients. After a median follow-up of 7 years, almost all patients developed cytopenia that was more frequent in patients receiving hematopoietic stem cell transplantation (HSCT). The cumulative overall survival from birth was 91.0% at age 10 years, 71.6% at age 20, and 47.4% at age 30 years; the median survival age was 29.1 years. When stratifying patients by indication for transplantation (moderate vs. severe cytopenia vs. persistent poor prognosis cytogenetic alterations/acute myeloid leukemia), we found a 5-year cumulative mortality higher, though not significantly (<i>p</i> = 0.281) in the last group. Cancers were the second most common cause of death in the whole cohort after infections. Head and neck squamous cell carcinoma was the most frequent cancer, followed by hematologic neoplasms. The cumulative incidence of solid/hematological malignancy remarkably increased after 20 years of age and was 51.7% at age 40 years. The risk of malignancies was greater in subjects who received HSCT (sub-distribution azard ratio 2.9, 95% CI: 1.1–7.5, <i>p</i> = 0.024). We also identified a small group of patients with stable or even improved cytopenia over time without transplant, thus confirming that bone marrow failure is not automatic in all patients and heightening the importance of tight monitoring to surveil on the worsening of hematopoietic function and cancer occurrence. Overall, this study provides important findings that may help to make robust clinical decisions.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"58 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144228897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The 2024 Three‐Strata Baseline Anemia Definition of the Revised IWG‐ELN Criteria Dissects Survival in Ruxolitinib‐Treated Myelofibrosis Patients","authors":"Margherita Maffioli, Barbara Mora, Alessandra Iurlo, Elena Maria Elli, Maria Chiara Finazzi, Mirko Farina, Elisa Rumi, Marianna Caramella, Maria Cristina Carraro, Mariella D'Adda, Alfredo Molteni, Elda Mimiola, Francesca Lunghi, Alessandro Vismara, Marta Ubezio, Maria Chiara Di Chio, Michela Anghilieri, Daniele Cattaneo, Matteo Giovanni Della Porta, Lorenza Bertù, Marta Coscia, Francesco Passamonti","doi":"10.1002/ajh.27734","DOIUrl":"https://doi.org/10.1002/ajh.27734","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"70 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144236978","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcome of Chronic Myeloid Leukemia Patients Not in Deep Molecular Response: Results From the GIMEMA LabNet CML Network Database","authors":"Fabio Stagno, Rosalba Cucci, Giovanni Marsili, Fausto Castagnetti, Sara Galimberti, Barbara Izzo, Federica Sorà, Simona Soverini, Monica Messina, Alfonso Piciocchi, Massimiliano Bonifacio, Daniela Cilloni, Alessandra Iurlo, Giovanni Martinelli, Gianantonio Rosti, Paola Fazi, Marco Vignetti, Massimo Breccia, Alessandro Allegra, Fabrizio Pane","doi":"10.1002/ajh.27733","DOIUrl":"https://doi.org/10.1002/ajh.27733","url":null,"abstract":"&lt;p&gt;Chronic myeloid leukemia (CML) is currently managed as a chronic disease requiring long-term treatment and a close molecular monitoring in many patients [&lt;span&gt;1&lt;/span&gt;]. Evidence suggests that in a substantial number of patients who achieved a stable sustained deep molecular response (DMR) the treatment with tyrosine kinase inhibitors (TKIs) can be safely discontinued [&lt;span&gt;2, 3&lt;/span&gt;]. Hence, treatment-free remission (TFR) is a strategy increasingly considered as a feasible treatment goal in about 20%–40% of CML patients [&lt;span&gt;4&lt;/span&gt;]. Nevertheless, a proportion of patients with CML in chronic-phase (CP) treated with TKIs still remain in stable major molecular remission (MR3) or less (MR2 only) without achieving a DMR, therefore requiring long-term TKIs therapy [&lt;span&gt;5&lt;/span&gt;] as well as a long-term molecular monitoring [&lt;span&gt;6&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;The Italian Group for Adult Hematological Malignancies (GIMEMA) activated in 2008 a project named GIMEMA LabNet CML network with the aim of reassuring a nationwide fast and harmonized molecular diagnostic and monitoring process to all CML patients. The network is now made up of 51 standardized laboratories for clinical and research purposes linked with 144 hematological centers. The connection between the hematology centers and laboratories is managed by a web-based general data protection regulation (GDPR) compliant platform. LabNet digital infrastructure manages the traffic of molecular diagnostic tests between clinical centers and laboratories with the aim of providing a standardized evaluation of minimal residual disease. Therefore, if not all, most of the CML patients living in Italy are monitored for their disease at the same level of accuracy and harmonization.&lt;/p&gt;\u0000&lt;p&gt;The aim of our analysis was to describe, in the pure real-life scenario of the GIMEMA LabNet CML network, the long-term outcome of those CML patients in stable MR3/MR2. The LabNet CML database represented the data source of our analysis. To participate in the network, laboratories fulfilled quality controls and regularly participated in quality control rounds.&lt;/p&gt;\u0000&lt;p&gt;At the time of the current analysis, the LabNet database contained data of 9699 patients affected by CML with evaluable samples for &lt;i&gt;BCR::ABL1&lt;/i&gt; transcripts. All patients gave written informed consent. We selected the patient cohort by analyzing all those with CP-CML treated frontline with Imatinib (IM), Dasatinib (DAS), or Nilotinib (NIL) at conventional doses who achieved, within 6 months, an MR3 or less (MR2) and showed evaluable samples for molecular response at least 24 months from the first MR3 or MR2 achievement. We collected only &lt;i&gt;BCR::ABL1&lt;/i&gt; kinetic and molecular data according to clinical practice. The LabNet CML Network adopted Subjective Objective Assessment and Plan (SOAP) notes according to the European LeukemiaNet guidelines [&lt;span&gt;6&lt;/span&gt;]. The &lt;i&gt;BCR::ABL1&lt;/i&gt; transcript levels were measured in each laboratory from peripheral blood samp","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"39 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144219416","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Characteristics and Survival Outcomes of Solitary Plasmacytomas: A 30‐Year Experience of the Greek Myeloma Study Group on 175 Patients","authors":"Eirini Katodritou, Efstathios Kastritis, Dimitra Dalampira, Nikolaos Kanellias, Vasiliki Labropoulou, Gerasimos Kyriakidis, Vasiliki Douka, Sosana Delimpasi, Dionysios Stoumbos, Emmanouil Spanoudakis, Sotirios Papageorgiou, Despina Fotiou, Annita‐Ioanna Gkioka, Theodora Triantafyllou, Ioannis Ntanasis‐Stathopoulos, Theodosia Papadopoulou, Kyriaki Tsirou, Aggeliki Sevastoudi, Aikaterini Daiou, Foteini Theodorakakou, Nikolaos Giannakoulas, Vasiliki Pappa, Avgi Lalayianni, Anastasia Pouli, Marie‐Christine Kyrtsonis, Maria Kotsopoulou, Evgenia Verrou, Maria Gavriatopoulou, Evangelos Terpos, Meletios‐Athanasios Dimopoulos","doi":"10.1002/ajh.27725","DOIUrl":"https://doi.org/10.1002/ajh.27725","url":null,"abstract":"In this multi‐institutional retrospective study, we analyzed the characteristics and outcomes of 118 patients with solitary bone plasmacytoma (SBP) and 57 with extramedullary plasmacytoma (SEP) diagnosed over 30 years. We also evaluated the impact of systemic therapy (ST), which is not routinely recommended, compared to standard radiation therapy (RT). The median age was 62 years (range: 17–85). Treatment included RT (<jats:italic>n</jats:italic> = 94), RT with ST (<jats:italic>n</jats:italic> = 47), ST alone (<jats:italic>n</jats:italic> = 22), and surgical excision alone (<jats:italic>n</jats:italic> = 12). Overall and complete response (CR) rates were 93% and 53%, respectively; 70 patients relapsed, 56 progressing to multiple myeloma (MM). The median follow‐up was 10 years (95% CI: 7.1–13). Median estimated overall survival (OS) was 18.5 years with a 5‐ and 10‐year OS rate of 85% and 70%, respectively, similar across groups (<jats:italic>p</jats:italic> &gt; 0.05). Median progression‐free survival (PFS) was 75 months (95% CI: 53–97), with a 5‐ and 10‐year PFS rates of 57% and 44%. The 5‐ and 10‐year MM‐free survival (MMFS) was 66% and 53%, respectively. We identified age ≤ 60 years, achieving CR, and an abnormal serum FLC ratio at diagnosis as the strongest prognosticators for OS (HR: 0.25), PFS (HR: 0.54), and MMFS (HR: 5 + 0.4), respectively (<jats:italic>p</jats:italic> &lt; 0.05); ST alone or combined with RT did not significantly improve survival outcomes or MMFS (<jats:italic>p</jats:italic> &gt; 0.05). In conclusion, ST increased toxicity without offering outcome benefits, reaffirming RT as the cornerstone of SP management. Despite therapeutic advancements in MM, the persistent challenge of progression to MM underscores the importance of identifying high‐risk SP patients, enabling early intervention with more aggressive treatments.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"8 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144210964","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Impact of Graft Cryopreservation on Allogeneic Stem Cell Transplantation: An Italian, Registry‐Based Study on Behalf of the “Gruppo Italiano Per Il Trapianto di Midollo Osseo, Cellule Staminali Emopoietiche e Terapia Cellulare” (GITMO)","authors":"Irene Defrancesco, Virginia Valeria Ferretti, Patrizia Chiusolo, Domenico Russo, Chiara Nozzoli, Attilio Olivieri, Massimiliano Gambella, Irene Maria Cavattoni, Stefania Bramanti, Stella Santarone, Renato Fanin, Roberto Cairoli, Simona Piemontese, Matteo Parma, Francesco Onida, Alessandro Busca, Luca Castagna, Angela Cuoghi, Domenico Pastore, Nicola Mordini, Fabio Benedetti, Cristina Skert, Carlo Borghero, Anna Paola Iori, Franca Fagioli, Vincenzo Pavone, Carmine Selleri, Simone Cesaro, Maurizio Musso, Marco Ladetto, Daniele Vallisa, Paola Carluccio, Alessandra Picardi, Monica Tozzi, Alessandra Biffi, Giuseppe Milone, Maura Faraci, Arcangelo Prete, Lucia Prezioso, Antonio Maria Risitano, Francesco Paolo Tambaro, Veronica Tintori, Piero Galieni, Fabrizio Pane, Caterina Zerbi, Antonio Bianchessi, Giulia Losi, Francesco Romano, Alessia Taurino, Elena Oldani, Nicola Polverelli, Francesca Bonifazi, Massimo Martino","doi":"10.1002/ajh.27731","DOIUrl":"https://doi.org/10.1002/ajh.27731","url":null,"abstract":"The coronavirus disease 2019 (COVID‐19) pandemic created major challenges for allogeneic hematopoietic stem cell transplantation (allo‐HSCT). Scientific societies and authorities recommended cryopreserving grafts before starting conditioning regimens, despite limited data on the clinical impact. The Italian Group for Bone Marrow Transplantation (GITMO) conducted a registry‐based study involving 3492 patients who underwent allo‐HSCT between March 2018 and September 2021. The cryopreserved cohort (<jats:italic>n</jats:italic> = 976) included patients who received cryopreserved grafts during the pandemic and was compared to the historical cohort (<jats:italic>n</jats:italic> = 2516). Graft cryopreservation was associated with a lower day 30 incidence of neutrophil and platelet engraftment (adjusted sHR = 0.8 and 0.7, <jats:italic>p</jats:italic> = 0.031 and <jats:italic>p</jats:italic> &lt; 0.001, respectively) and delayed hematopoietic recovery. However, primary graft failure rates at day +30 were similar in the cryo and historical cohort (4% vs. 5%, respectively; <jats:italic>p</jats:italic> = 0.337), also after adjustment (RR = 1.19, <jats:italic>p</jats:italic> = 0.518). Day 100 incidence of grade II‐IV acute GVHD was comparable between the two groups (adjusted sHR = 1.2, <jats:italic>p</jats:italic> = 0.194). Regarding chronic GVHD incidence, we found that it was higher in patients aged &lt; 18 years in the cryo group (adjusted sHR = 3.9, <jats:italic>p</jats:italic> = 0.002), but lower in those aged 18–55 years (adjusted sHR = 0.7, <jats:italic>p</jats:italic> = 0.008). Cumulative incidence of relapse did not differ between historical and cryo cohort (adjusted sHR 1.0. <jats:italic>p</jats:italic> = 0.943), as well as non‐relapse mortality (adjusted sHR 1.1, <jats:italic>p</jats:italic> = 0.196) and relapse‐free survival (adjusted sHR = 1.1, <jats:italic>p</jats:italic> = 0.197). However, a shorter overall survival was observed in the cryopreserved group (adjusted HR = 1.2, <jats:italic>p</jats:italic> = 0.038). Transplant centers should carefully balance the benefits and drawbacks of cryopreservation in allo‐HSCT.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"1 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144201637","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Challenging the Adverse Label: Diverse Outcomes of ELN 2022 Adverse Cytogenetic Subgroups in Acute Myeloid Leukemia Patients Allografted in First Remission: From EBMT ALWP","authors":"Ali Bazarbachi, Jacques-Emmanuel Galimard, Iman Abou Dalle, Gérard Socié, Jurjen Versluis, Depei Wu, Matthias Eder, Hélène Labussière-Wallet, Ibrahim Yakoub-Agha, Johan Maertens, Edouard Forcade, Tobias Gedde-Dahl, Goda Choi, Cristina Castilla-Llorente, Frederic Baron, Eolia Brissot, Jordi Esteve, Arnon Nagler, Mohamad Mohty, Fabio Ciceri","doi":"10.1002/ajh.27726","DOIUrl":"https://doi.org/10.1002/ajh.27726","url":null,"abstract":"According to the European LeukemiaNet (ELN) 2022 classification, acute myeloid leukemia (AML) patients with intermediate or adverse risk are offered allogeneic hematopoietic stem cell transplantation (allo-HSCT) in first remission. In this EBMT study, we included 1735 adult AML patients with ELN-2022 adverse-risk cytogenetics allografted between 2010 and 2022 in first remission (67% de novo AML, median age 56 years). Eleven non-overlapping adverse-risk cytogenetics groups were defined. The five most frequent were: Group 1 [<i>n</i> = 394; monosomy 17 or abn(17p); 2-year leukemia-free survival (LFS) 22%, and overall survival (OS) 25%]; Group 2 [<i>n</i> = 313; complex karyotype (CK) involving monosomy 5, monosomy 7, or del(5q) without monosomy 17 or abn(17p); LFS 27%, OS 37%]; Group 3 [<i>n</i> = 201; monosomy 5, monosomy 7, or del(5q) without CK and without monosomy 17 or abn(17p); LFS: 55%, OS: 63%]; Group 4 [<i>n</i> = 256; CK without monosomal karyotype (MK) or adverse additional cytogenetic abnormalities (ACA); LFS 50%, OS 61%]; Group 5 [<i>n</i> = 213; t(v, 11q23) without adverse ACA; LFS 50%, OS 59%]. In multivariable analysis, compared to CK without adverse ACA, LFS was negatively affected by monosomy 17 or 17p abnormalities, monosomy 5, 7, or del(5q) in the presence of CK, and t(8;16). In conclusion, this study revealed a very poor outcome of allografted AML patients with monosomy 17 or 17p abnormalities or CK involving monosomy 5, monosomy 7, and del5q. Outcomes were relatively favorable for most other ELN 2022 adverse categories, including CK with or without MK other than 5, 7, and 17, indicating that allo-HSCT can overcome their poor outcome.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"146 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144176952","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identification of a CAR-Derived Clone by NGS-Based MRD After Fully Human BCMA CAR T-Cell Therapy in Multiple Myeloma","authors":"Wenqiang Yan, Jiao Chang, Chenxing Du, Yuntong Liu, Rui Lv, Hesong Zou, Tengteng Yu, Shuaishuai Zhang, Tingyu Wang, Weiwei Sui, Shuhui Deng, Yan Xu, Wenyang Huang, Shuhua Yi, Dehui Zou, Jianxiang Wang, Lugui Qiu, Yujiao Jia, Gang An","doi":"10.1002/ajh.27732","DOIUrl":"https://doi.org/10.1002/ajh.27732","url":null,"abstract":"&lt;p&gt;B cell maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cell therapy has significantly improved survival outcomes in patients with relapsed or refractory multiple myeloma (RRMM), achieving unprecedented depth of response compared to conventional salvage regimens [&lt;span&gt;1-3&lt;/span&gt;]. Minimal residual disease (MRD) negativity has emerged as a strong predictor of favorable prognosis in this setting [&lt;span&gt;4&lt;/span&gt;] and is increasingly recognized as a surrogate endpoint for accelerated drug approvals of novel agents.&lt;/p&gt;\u0000&lt;p&gt;Next-generation flow cytometry (NGF) and next-generation sequencing (NGS)-based MRD assessments are the most widely adopted approaches, offering sensitivities of approximately 10&lt;sup&gt;−5&lt;/sup&gt; and 10&lt;sup&gt;−6&lt;/sup&gt;, respectively. In addition to their high sensitivity, NGS-MRD enables longitudinal tracking of clonal dynamics and the identification of potential subclonal evolution. However, the interpretation of NGS-MRD in the context of genetically engineered T cell products remains limited and poorly characterized.&lt;/p&gt;\u0000&lt;p&gt;In our center, most patients attained NGS-MRD negativity following BCMA CAR-T infusion in real-world clinical settings. Unexpectedly, in a subset of these patients, we identified a persistent novel clone sequence that was not related to the original tumor clone post-infusion. This novel clone gradually declined over time but remained detectable in serial MRD assessments. Whether this uncommon finding reflects clonal evolution, tumor relapse, secondary neoplasm, or a previously unrecognized signal derived from the CAR-T product itself remains to be determined. To investigate this phenomenon, we conducted a retrospective study utilizing a multi-modal approach to characterize the origin and clinical relevance of this novel clone.&lt;/p&gt;\u0000&lt;p&gt;We retrospectively analyzed 55 myeloma patients who received BCMA CAR-T cell therapy at our center and had at least one NGS-MRD assessment post-infusion, as part of real-world observational studies or investigator-initiated trials (IIT). Written informed consent was obtained from all patients, and the study was approved by the Ethics Committee of the Blood Diseases Hospital.&lt;/p&gt;\u0000&lt;p&gt;Fluorescence in situ hybridization (FISH) and NGF-based MRD were performed as previously described [&lt;span&gt;5&lt;/span&gt;]. NGS-MRD analysis was conducted using the Neo-MRD assay (Neoimmune, Shenzhen, China), targeting V(D)J rearrangements in IGH, IGK, and IGL genes. A two-step PCR was applied: a 28-cycle multiplex PCR to amplify V and J segments, followed by a 12-cycle universal PCR to add Illumina adaptors. Libraries were sequenced on the NovaSeq 6000 platform (paired-end, 150 bp). CDR3 sequences with fewer than three reads per million were excluded. Dominant clones were defined based on &gt; 3% clonal frequency and &gt; 0.2% nucleated cell content and were tracked across timepoints.&lt;/p&gt;\u0000&lt;p&gt;Lentiviral vector copy number (VCN) was quantified using digital droplet PCR (ddPCR) in 27 bone marrow (BM) DNA ","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"149 2 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144176663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Benefit of Early Daratumumab Use in Multiple Myeloma is Undefined.","authors":"Ruben Van Dijck, Zoran Erjavec, John-John B Schnog","doi":"10.1002/ajh.27728","DOIUrl":"https://doi.org/10.1002/ajh.27728","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144172198","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CPX-351 (Vyxeos) in Acute Myeloid Leukemia: Time to Move on?","authors":"Naseema Gangat, Ayalew Tefferi","doi":"10.1002/ajh.27730","DOIUrl":"https://doi.org/10.1002/ajh.27730","url":null,"abstract":"CPX-351 has similar efficacy but more toxicity compared to venetoclax+hypomethylating agent therapy.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"134 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144165663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mean Corpuscular Hemoglobin Modulates HbF/F-Cell and Clinical Response to Gene Therapy and Hydroxyurea in Sickle Cell Disease.","authors":"Martin H Steinberg, Abdullah Kutlar, Paola Sebastiani","doi":"10.1002/ajh.27729","DOIUrl":"https://doi.org/10.1002/ajh.27729","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144155523","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}