American Journal of Hematology最新文献

{"title":"Integrative Genomic and Transcriptomic Analysis Reveals Targetable Vulnerabilities in Angioimmunoblastic T‐Cell Lymphoma","authors":"Alyssa Bouska, Weiwei Zhang, Sunandini Sharma, Harald Holte, Rauf A. Shah, Waseem G. Lone, Mahfuza Afroz Soma, Ruimeng Yang, Xuxiang Liu, Syed Mehmood, Ravneet Singh Chawla, Luca Vincenzo Cappelli, Danilo Fiore, Qiang Gong, Tayla B. Heavican‐Foral, Jeffrey J. Cannatella, Catalina Amador, Aiza Arif, Lynette M. Smith, Soon Thye Lim, Choon Kiat Ong, Andrew L. Feldman, Ming‐Qing Du, Anamarija M. Perry, Laurence de Leval, Timothy C. Greiner, Kai Fu, Gunhild Trøen, Daniel Vodák, Sigve Nakken, Jan Delabie, David Weinstock, Stefano Pileri, Antonella Laginestra, KyeongJin Kim, Utpal Pajvani, Julie M. Vose, Dennis D. Weisenburger, Steven M. Horwitz, Sandeep Dave, Joseph Khoury, Giorgio Inghirami, Wing C. Chan, Javeed Iqbal","doi":"10.1002/ajh.27736","DOIUrl":"https://doi.org/10.1002/ajh.27736","url":null,"abstract":"Nodal follicular helper T‐cell (T<jats:sub>FH</jats:sub>) lymphoma of the angioimmunoblastic (AITL) subtype has a dismal prognosis. Using whole‐exome sequencing (<jats:italic>n</jats:italic> = 124), transcriptomic (<jats:italic>n</jats:italic> = 78), and methylation (<jats:italic>n</jats:italic> = 40) analysis, we identified recurrent mutations in known epigenetic drivers (<jats:italic>TET2, DNMT3A, IDH2</jats:italic><jats:sup><jats:italic>R172</jats:italic></jats:sup>) and novel ones (<jats:italic>TET3, KMT2D</jats:italic>). <jats:italic>TET2, IDH2</jats:italic><jats:sup><jats:italic>R172</jats:italic></jats:sup>, <jats:italic>DNMT3A</jats:italic> co‐mutated AITLs had poor prognosis (<jats:italic>p</jats:italic> < 0.0001). Genes regulating T‐cell receptor (TCR) signaling (<jats:italic>CD28, PLCG1, VAV1</jats:italic>, <jats:italic>FYN</jats:italic>) or activation (<jats:italic>RHOA</jats:italic><jats:sup><jats:italic>G17V</jats:italic></jats:sup>) or regulators of the PI3K‐pathway (PIK(3)C members, <jats:italic>PTEN, PHLPP1, PHLPP2</jats:italic>) were mutated. <jats:italic>CD28</jats:italic> mutation/fusion was associated with poor prognosis (<jats:italic>p</jats:italic> = 0.02). WES of purified, neoplastic T‐cell (CD3<jats:sup>+</jats:sup>PD1<jats:sup>+</jats:sup>) demonstrated high concordance with whole tumor biopsies and validated the presence of <jats:italic>TET2</jats:italic> and <jats:italic>DNMT3A</jats:italic> in tumor and non‐lymphoid cells, but other mutations (<jats:italic>CD28</jats:italic>, <jats:italic>RHOA</jats:italic><jats:sup><jats:italic>G17V</jats:italic></jats:sup>, <jats:italic>IDH2</jats:italic><jats:sup><jats:italic>R172</jats:italic></jats:sup>, <jats:italic>PLCG1</jats:italic>) in neoplastic cells. Integrated DNA‐methylation and mRNA expression analysis revealed epigenetic alterations in genes regulating TCR, cytokines, PI3K‐signaling, and apoptosis. RNA‐seq analysis identified fusion transcripts regulating TCR‐activation (8%), revealed a restricted TCR‐repertoire (α = 87%, β = 72%), and showed the presence of Epstein–Barr virus transcriptome (73%). GEP demonstrated the association of B‐cells or dendritic cells in the tumor milieu with prognosis (<jats:italic>p</jats:italic> < 0.01). RNA‐seq and WES analysis of 12 AITL‐patient‐derived‐xenografts (PDX) showed that bi‐allelic <jats:italic>TET2</jats:italic> and <jats:italic>DNMT3A</jats:italic> mutations or sub‐clonal mutations (<jats:italic>PLCG1, PHLPP2</jats:italic>) propagated in sequential passages, and gene signatures related to T<jats:sub>FH</jats:sub> and T<jats:sub>CM</jats:sub> (central‐memory) were well‐maintained through passages. Gene expression signatures associated with late PDX passages (3rd–5th) were enriched with proliferation and metabolic reprogramming‐related genes and predicted prognosis in an independent AITL series. Low <jats:italic>PHLPP2</jats:italic> mRNA expression predicted poor prognosis (<jats:italic>p</jats:italic> = 0.05) and enginee","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"31 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144278262","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":"Poor Engraftment After Posttransplant Cyclophosphamide Graft-Versus-Host DISAESE Prophylaxis in Patients With Myelofibrosis.","authors":"Haesook T Kim, Deborah J Liney, Kristen Rizza, Corey S Cutler, John Koreth, Sarah Nikiforow, Roman M Shapiro, Amar H Kelkar, Mahasweta Gooptu, Rizwan Romee, Catherine J Wu, Joseph H Antin, Jerome Ritz, Robert J Soiffer, Vincent T Ho","doi":"10.1002/ajh.27741","DOIUrl":"https://doi.org/10.1002/ajh.27741","url":null,"abstract":"<p><p>Patients with myelofibrosis (MF) often have impaired engraftment after allogeneic hematopoietic cell transplantation (alloHCT). To determine whether posttransplant cyclophosphamide (PTCY) exerts a detrimental impact on engraftment, we compared clinical outcomes of all patients receiving PTCY (N = 49) and no-PTCY (N = 89) regimens as GVHD prophylaxis after first alloHCT for MF from 2016 to 2023. Median age was 64 in both groups, and baseline characteristics were balanced except that the PTCY group received more haploidentical transplants and were more likely to have secondary MF. Among engrafted individuals, median time to neutrophil engraftment was D + 18 for PTCY and D + 15 for no-PTCY (p < 0.0001). Median time to platelet recovery was D + 33.5 and D + 26 for PTCY and no-PTCY, respectively (p = 0.0006). The graft failure rate was higher for PTCY but did not reach statistical significance (27% for PTCY and 15% for non-PTCY, p = 0.095). The need for donor lymphocyte infusion, CD34 boost, or second transplant was significantly higher for PTCY (33% vs. 13% at 1-year, respectively, p = 0.004). Most cases of graft failure in both cohorts were due to prolonged cytopenias in the absence of relapse. While T-cell chimerism at D + 30 and D + 100 was robust after PTCY, the proportion of patients with granulocyte chimerism ≥ 90% at Day + 100 was significantly lower in PTCY (76% vs. 98.2%, respectively, p = 0.0028). All other outcomes were similar except for a significantly lower chronic GVHD rate in PTCY compared with no-PTCY (p = 0.0002). GVHD prophylaxis with PTCY in patients with MF is associated with delayed engraftment, lower donor granulocyte chimerism, and increased need for additional donor cell infusions after transplant without compromising survival.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144281996","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":"Mycosis Fungoides, Sézary Syndrome, and Cutaneous B-Cell Lymphomas: 2025 Update on Diagnosis, Risk-Stratification, and Management","authors":"Alexandra C. Hristov, Trilokraj Tejasvi, Ryan A. Wilcox","doi":"10.1002/ajh.27735","DOIUrl":"https://doi.org/10.1002/ajh.27735","url":null,"abstract":"Primary cutaneous lymphomas are a rare and heterogeneous group of extranodal lymphomas that require the integration of clinical and histopathologic data for classification and treatment.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"140 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144260227","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":"Phase II Trial of Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation Conditioning With Haploidentical Donor Peripheral Blood Stem Cell Transplant","authors":"Hany Elmariah, Jongphil Kim, Rebecca Gonzalez, Elizabeth DiMaggio, Samer Sansil, Asmita Mishra, Rawan Faramand, Lia Perez, Aleksandr Lazaryan, Abu-Sayeef Mirza, Farhad Khimani, Hien Liu, Jose L. Ochoa-Bayona, Michael Nieder, Fabiana Perna, Nicholas Figura, Timothy J. Robinson, Taiga Nishihori, Claudio Anasetti, Joseph A. Pidala, Nelli Bejanyan","doi":"10.1002/ajh.27738","DOIUrl":"https://doi.org/10.1002/ajh.27738","url":null,"abstract":"&lt;p&gt;Human leukocyte antigen (HLA) haploidentical (haplo) hematopoietic cell transplantation (HCT) with post-transplant cyclophosphamide (PTCy) has expanded access to this curative therapy with rates of graft-versus-host disease (GVHD) that are similar to HLA matched donor HCT [&lt;span&gt;1&lt;/span&gt;]. The original Johns Hopkins PTCy platform uses a reduced intensity conditioning (RIC) regimen consisting of Fludarabine (Flu) 150 mg/m&lt;sup&gt;2&lt;/sup&gt;, Cy 14.5 mg/kg on Day-6 and-5, total body irradiation (TBI) 200 cGy on Day-1 (Flu/Cy/TBI) and a bone marrow graft (BMT). Though this regimen yields favorable toxicity including rates of non-relapse mortality (NRM) ~10%–15%, severe acute GVHD ~10%, and chronic GVHD ~10%, outcomes have been marred by high rates of disease relapse &gt; 50% [&lt;span&gt;1&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;While myeloablative conditioning (MAC) regimens can reduce the risk of relapse, older and frail patients cannot tolerate MAC regimens. To improve relapse after RIC haplo HCT with PTCy, investigators at the MD Anderson Cancer Center attempted a conditioning regimen consisting of Flu 160 mg/m&lt;sup&gt;2&lt;/sup&gt;, melphalan (Mel) 100–140 mg/m&lt;sup&gt;2&lt;/sup&gt;, and either Thiotepa 5 mg/kg or TBI 200 cGy [&lt;span&gt;2&lt;/span&gt;]. While they reported low 1-year relapse rates of 19%, NRM was high at 21%, resulting in 1-year disease-free survival (DFS) of 60%.&lt;/p&gt;\u0000&lt;p&gt;As haplo HCT with PTCy is increasingly used, strategies to improve outcomes are essential. We hypothesized that reducing the Mel dose to 70 mg/m&lt;sup&gt;2&lt;/sup&gt; in a Flu/Mel70/TBI regimen followed by haplo peripheral blood stem cell transplant (PBSCT) with PTCy would successfully improve DFS by reducing relapse without a significant increase in NRM compared to Flu/Cy/TBI haplo HCT in patients unfit for MAC.&lt;/p&gt;\u0000&lt;p&gt;This trial, NCT04191187, is single-institution phase II study of Flu/Mel70/TBI haplo PBSCT with PTCy/sirolimus/mycophenolate mofetil (MMF) in patients with high-risk hematologic malignancies. It was approved by the Advarra Institutional Review Board and is compliant with the Declaration of Helsinki.&lt;/p&gt;\u0000&lt;p&gt;Eligible patients were required to be ≥ 55 years or &lt; 55 years with significant comorbidities defined as an HCT comorbidity index (HCT-CI) ≥ 3, and receiving an HLA haplo donor HCT for a hematologic malignancy.&lt;/p&gt;\u0000&lt;p&gt;All patients received conditioning with Flu 30 mg/m&lt;sup&gt;2&lt;/sup&gt;/day from −6 to −2 based on actual body weight, Mel 70 mg/mg&lt;sup&gt;2&lt;/sup&gt; on day-6 based on actual body weight, and TBI 200 cGy on day-1. On day 0, patients received the PBSCT (Figure 1A). The target CD34+ cell dose was 5 × 10&lt;sup&gt;6&lt;/sup&gt; CD34+ cells/kg, with a maximum allowable dose of 7 × 10&lt;sup&gt;6&lt;/sup&gt; CD34+ cells/kg. GVHD prophylaxis consisted of PTCy/tacrolimus/MMF for the first five subjects. The protocol was subsequently modified to follow the PTCy/sirolimus/MMF platform published separately by our group [&lt;span&gt;3&lt;/span&gt;].&lt;/p&gt;\u0000&lt;figure&gt;&lt;picture&gt;\u0000&lt;source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/78a4833a-5adb-42fb-a926-7058ec195f","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"8 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144252847","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":"Common Lymphoma in an Uncommon Location","authors":"Jordan Carter,&nbsp;Jonathan M. von Reusner,&nbsp;Mark A. Sellmyer,&nbsp;Adam Bagg,&nbsp;Stefan K. Barta","doi":"10.1002/ajh.27740","DOIUrl":"10.1002/ajh.27740","url":null,"abstract":"<p>FDG avid bilateral ureteral lesions.\u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure></p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 8","pages":"1437-1439"},"PeriodicalIF":10.1,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27740","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144237808","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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,&nbsp;Francesca Bagnasco,&nbsp;Filomena Pierri,&nbsp;Antonio Risitano,&nbsp;Piero Farruggia,&nbsp;Maura Faraci,&nbsp;Camilla Frieri,&nbsp;Paola Corti,&nbsp;Ugo Ramenghi,&nbsp;Paola Quarello,&nbsp;Walter Barberi,&nbsp;Giuseppe Menna,&nbsp;Giovanna Giagnuolo,&nbsp;Marta Pillon,&nbsp;Maurizio Miano,&nbsp;Paolo Di Bartolomeo,&nbsp;Marco Zecca,&nbsp;Elena Mastrodicasa,&nbsp;Rosamaria Mura,&nbsp;Beatrice Pinazzi,&nbsp;Laura Luti,&nbsp;Maria Licciardello,&nbsp;Angelica Barone,&nbsp;Marinella Veltroni,&nbsp;Giovanni Palazzi,&nbsp;Federico Verzegnassi,&nbsp;Francesca Patriarca,&nbsp;Daniela Onofrillo,&nbsp;Simone Cesaro,&nbsp;Roberta Ghilardi,&nbsp;Irene D'Alba,&nbsp;Rosa Angarano,&nbsp;Luca Arcuri,&nbsp;Andrea Beccaria,&nbsp;Ramona Tallone,&nbsp;Adriana Zatterale,&nbsp;Carlo Dufour","doi":"10.1002/ajh.27724","DOIUrl":"10.1002/ajh.27724","url":null,"abstract":"<p>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.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 8","pages":"1387-1396"},"PeriodicalIF":10.1,"publicationDate":"2025-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27724","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":"OA","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,&nbsp;Rosalba Cucci,&nbsp;Giovanni Marsili,&nbsp;Fausto Castagnetti,&nbsp;Sara Galimberti,&nbsp;Barbara Izzo,&nbsp;Federica Sorà,&nbsp;Simona Soverini,&nbsp;Monica Messina,&nbsp;Alfonso Piciocchi,&nbsp;Massimiliano Bonifacio,&nbsp;Daniela Cilloni,&nbsp;Alessandra Iurlo,&nbsp;Giovanni Martinelli,&nbsp;Gianantonio Rosti,&nbsp;Paola Fazi,&nbsp;Marco Vignetti,&nbsp;Massimo Breccia,&nbsp;Alessandro Allegra,&nbsp;Fabrizio Pane","doi":"10.1002/ajh.27733","DOIUrl":"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;&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;&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;&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 samples","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 8","pages":"1467-1470"},"PeriodicalIF":10.1,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27733","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":"OA","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,&nbsp;Efstathios Kastritis,&nbsp;Dimitra Dalampira,&nbsp;Nikolaos Kanellias,&nbsp;Vasiliki Labropoulou,&nbsp;Gerasimos Kyriakidis,&nbsp;Vasiliki Douka,&nbsp;Sosana Delimpasi,&nbsp;Dionysios Stoumbos,&nbsp;Emmanouil Spanoudakis,&nbsp;Sotirios Papageorgiou,&nbsp;Despina Fotiou,&nbsp;Annita-Ioanna Gkioka,&nbsp;Theodora Triantafyllou,&nbsp;Ioannis Ntanasis-Stathopoulos,&nbsp;Theodosia Papadopoulou,&nbsp;Kyriaki Tsirou,&nbsp;Aggeliki Sevastoudi,&nbsp;Aikaterini Daiou,&nbsp;Foteini Theodorakakou,&nbsp;Nikolaos Giannakoulas,&nbsp;Vasiliki Pappa,&nbsp;Avgi Lalayianni,&nbsp;Anastasia Pouli,&nbsp;Marie-Christine Kyrtsonis,&nbsp;Maria Kotsopoulou,&nbsp;Evgenia Verrou,&nbsp;Maria Gavriatopoulou,&nbsp;Evangelos Terpos,&nbsp;Meletios-Athanasios Dimopoulos","doi":"10.1002/ajh.27725","DOIUrl":"10.1002/ajh.27725","url":null,"abstract":"<div>\u0000 \u0000 <p>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 (<i>n</i> = 94), RT with ST (<i>n</i> = 47), ST alone (<i>n</i> = 22), and surgical excision alone (<i>n</i> = 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 (<i>p</i> &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 (<i>p</i> &lt; 0.05); ST alone or combined with RT did not significantly improve survival outcomes or MMFS (<i>p</i> &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.</p>\u0000 </div>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 8","pages":"1334-1342"},"PeriodicalIF":10.1,"publicationDate":"2025-06-03","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,&nbsp;Virginia Valeria Ferretti,&nbsp;Patrizia Chiusolo,&nbsp;Domenico Russo,&nbsp;Chiara Nozzoli,&nbsp;Attilio Olivieri,&nbsp;Massimiliano Gambella,&nbsp;Irene Maria Cavattoni,&nbsp;Stefania Bramanti,&nbsp;Stella Santarone,&nbsp;Renato Fanin,&nbsp;Roberto Cairoli,&nbsp;Simona Piemontese,&nbsp;Matteo Parma,&nbsp;Francesco Onida,&nbsp;Alessandro Busca,&nbsp;Luca Castagna,&nbsp;Angela Cuoghi,&nbsp;Domenico Pastore,&nbsp;Nicola Mordini,&nbsp;Fabio Benedetti,&nbsp;Cristina Skert,&nbsp;Carlo Borghero,&nbsp;Anna Paola Iori,&nbsp;Franca Fagioli,&nbsp;Vincenzo Pavone,&nbsp;Carmine Selleri,&nbsp;Simone Cesaro,&nbsp;Maurizio Musso,&nbsp;Marco Ladetto,&nbsp;Daniele Vallisa,&nbsp;Paola Carluccio,&nbsp;Alessandra Picardi,&nbsp;Monica Tozzi,&nbsp;Alessandra Biffi,&nbsp;Giuseppe Milone,&nbsp;Maura Faraci,&nbsp;Arcangelo Prete,&nbsp;Lucia Prezioso,&nbsp;Antonio Maria Risitano,&nbsp;Francesco Paolo Tambaro,&nbsp;Veronica Tintori,&nbsp;Piero Galieni,&nbsp;Fabrizio Pane,&nbsp;Caterina Zerbi,&nbsp;Antonio Bianchessi,&nbsp;Giulia Losi,&nbsp;Francesco Romano,&nbsp;Alessia Taurino,&nbsp;Elena Oldani,&nbsp;Nicola Polverelli,&nbsp;Francesca Bonifazi,&nbsp;Massimo Martino","doi":"10.1002/ajh.27731","DOIUrl":"10.1002/ajh.27731","url":null,"abstract":"<p>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 (<i>n</i> = 976) included patients who received cryopreserved grafts during the pandemic and was compared to the historical cohort (<i>n</i> = 2516). Graft cryopreservation was associated with a lower day 30 incidence of neutrophil and platelet engraftment (adjusted sHR = 0.8 and 0.7, <i>p</i> = 0.031 and <i>p</i> &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; <i>p</i> = 0.337), also after adjustment (RR = 1.19, <i>p</i> = 0.518). Day 100 incidence of grade II-IV acute GVHD was comparable between the two groups (adjusted sHR = 1.2, <i>p</i> = 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, <i>p</i> = 0.002), but lower in those aged 18–55 years (adjusted sHR = 0.7, <i>p</i> = 0.008). Cumulative incidence of relapse did not differ between historical and cryo cohort (adjusted sHR 1.0. <i>p</i> = 0.943), as well as non-relapse mortality (adjusted sHR 1.1, <i>p</i> = 0.196) and relapse-free survival (adjusted sHR = 1.1, <i>p</i> = 0.197). However, a shorter overall survival was observed in the cryopreserved group (adjusted HR = 1.2, <i>p</i> = 0.038). Transplant centers should carefully balance the benefits and drawbacks of cryopreservation in allo-HSCT.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 8","pages":"1354-1364"},"PeriodicalIF":10.1,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27731","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}