American Journal of Hematology最新文献

{"title":"Respiratory Viruses in Patients With Hematological Malignancy in Boreal Autumn/Winter 2023–2024: EPICOVIDEHA-EPIFLUEHA Report","authors":"Jon Salmanton-García, Francesco Marchesi, Milan Navrátil, Klára Piukovics, Maria Ilaria del Principe, Marianna Criscuolo, Yavuz M. Bilgin, Nicola S. Fracchiolla, Antonio Vena, Alessandra Romano, Iker Falces-Romero, Nicola Sgherza, Inmaculada Heras-Fernando, Monika M. Biernat, Verena Petzer, Pavel Žák, Barbora Weinbergerová, Michail Samarkos, Nurettin Erben, Jens van Praet, Alberto López-García, Jorge Labrador, Tobias Lahmer, Ľuboš Drgoňa, Maria Merelli, Annarosa Cuccaro, Sonia Martín-Pérez, Julio Dávila-Valls, Francesca Farina, Chiara Cattaneo, László Imre Pinczés, Ferenc Magyari, Ildefonso Espigado, Caterina Buquicchio, Donald C. Vinh, Igor Stoma, Martin Čerňan, Lucia Prezioso, Mario Virgilio Papa, Gaëtan Plantefeve, Reham Abdelaziz Khedr, Josip Batinić, Gabriele Magliano, Simge Erdem, Sofya Khostelidi, Natasha Čolović, Davide Nappi, Patricia García-Ramírez, Jakub Góra, Marta Callejas-Charavia, Jędrzej Tłusty, Martijn Bakker, Elwira Wojtyniak, Darko Antić, Agnieszka Magdziak, Michelina Dargenio, Larisa Idrizović, Nikola Pantić, Zlate Stojanoski, Noha Eisa, Vladimir Otašević, Monia Marchetti, Erica Mackenzie, Carolina Garcia-Vidal, Avinash Aujayeb, Ahlam Almasari, Carolina Miranda-Castillo, Eleni Gavriilaki, Nicola Coppola, Alessandro Busca, Tatjana Adžić-Vukičević, Martin Schönlein, Ditte Stampe Hersby, Stefanie K. Gräfe, Andreas Glenthøj, Tommaso Francesco Aiello, Milche Cvetanoski, Mirjana Mitrović, Claudio Cerchione, Romane Prin, Gina Varricchio, Elena Arellano, Raúl Córdoba, Jiří Mayer, Benjamín Víšek, Dominik Wolf, Amalia N. Anastasopoulou, Mario Delia, Pellegrino Musto, Dario Leotta, Martina Bavastro, Alessandro Limongelli, Mariarita Sciumè, Lukas van den Ven, Luana Fianchi, Sara Caterina Brunetti, Joanna Drozd-Sokołowska, Anna Dąbrowska-Iwanicka, Oliver A. Cornely, Livio Pagano","doi":"10.1002/ajh.27565","DOIUrl":"https://doi.org/10.1002/ajh.27565","url":null,"abstract":"Community-acquired respiratory viral infections (CARV) significantly impact patients with hematological malignancies (HM), leading to high morbidity and mortality. However, large-scale, real-world data on CARV in these patients is limited. This study analyzed data from the EPICOVIDEHA-EPIFLUEHA registry, focusing on patients with HM diagnosed with CARV during the 2023–2024 autumn–winter season. The study assessed epidemiology, clinical characteristics, risk factors, and outcomes. The study examined 1312 patients with HM diagnosed with CARV during the 2023–2024 autumn–winter season. Of these, 59.5% required hospitalization, with 13.5% needing ICU admission. The overall mortality rate was 10.6%, varying by virus: parainfluenza (21.3%), influenza (8.8%), metapneumovirus (7.1%), RSV (5.9%), or SARS-CoV-2 (5.0%). Poor outcomes were significantly associated with smoking history, severe lymphopenia, secondary bacterial infections, and ICU admission. This study highlights the severe risk CARV poses to patients with HM, especially those undergoing active treatment. The high rates of hospitalization and mortality stress the need for better prevention, early diagnosis, and targeted therapies. Given the severe outcomes with certain viruses like parainfluenza, tailored strategies are crucial to improving patient outcomes in future CARV seasons.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"39 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142874802","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":"Heavy Metal Toxicity Secondary to Ayurvedic Medications","authors":"Bingwen Eugene Fan, Natasha Fay Anthony, Kian Guan Eric Lim, Yin Wen Lai, Ponnudurai Kuperan, Hemalatha Shanmugam","doi":"10.1002/ajh.27571","DOIUrl":"https://doi.org/10.1002/ajh.27571","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"5 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142879688","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":"Efficacy and Long-Term Outcomes of Eltrombopag Treatment Within 6 Months of Diagnosis in Patients With Steroid Unresponsive or Dependent Immune Thrombocytopenia","authors":"Huyen Tran, Caroline Dix, Robert Bird, Sanjeev Chunilal, Tim Brighton, John Reynolds, Simon He, Christine Mazis, Andrew Grigg","doi":"10.1002/ajh.27568","DOIUrl":"https://doi.org/10.1002/ajh.27568","url":null,"abstract":"<p>Immune thrombocytopenia (ITP) is an autoimmune disorder associated with the production of autoantibodies directed against platelet glycoprotein complexes, resulting in both accelerated destruction of platelets and a relative reduction in platelet production. In adults, the majority of the acute cases respond to initial steroid therapy, but 50% relapse by 6 months and an additional 25% relapse beyond 1 year [<span>1</span>]. The non-responders and early relapsers can be difficult to manage and require additional therapies. These include intravenous immunoglobulin (IVIg), splenectomy, and rituximab, all of which can be problematic with variable and non-sustained responses, and specific thrombotic and infective risks with splenectomy [<span>2</span>].</p>\u0000<p>Thrombopoietin receptor agonists (TPO-RA) exert their effects in ITP by increasing platelet production. Clinical trials have shown that TPO-RA are efficacious and well tolerated in chronic ITP patients, with some patients achieving a lasting response after cessation [<span>3</span>]. At the time, the study described in this manuscript was designed (2014), little was known about the short- and long-term response to TPO-RA in patients with newly diagnosed ITP. Hence, this study was initiated to evaluate the early- and long-term efficacy and safety of eltrombopag in adult non-splenectomized patients with steroid-unresponsive or dependent ITP within 6 months of diagnosis.</p>\u0000<p>Patients at six Australian centers were enrolled into this multicenter, single arm, prospective open-label study between February 2014 and February 2016. Patients were 18 years or older and with ITP diagnosed within the prior 6 months which was steroid-unresponsive or -dependent and, if used, without a sustained response to IVIg. Full inclusion and exclusion criteria can be found in the Supporting Information. The study was conducted following approval from the ethics committees of participating institutions and patients provided written informed consent. The study was registered as a clinical trial [ACTRN12613000721707].</p>\u0000<p>Patients with platelets < 10 × 10<sup>9</sup>/L commenced 75 mg eltrombopag daily; those with a platelet count ≥ 10 × 10<sup>9</sup>/L commenced 50 mg daily. The dose was progressively increased by 25 mg increment every 2 weeks to maximum of 150 mg daily (100 mg daily if East Asian) if the platelet count remained ≤ 30 × 10<sup>9</sup>/L or there was clinically significant bleeding. The dose in responding patients was maintained until the week 12 response assessment, unless the platelet count was ≥ 200 × 10<sup>9</sup>/L. Subsequently, the dose was adjusted to the minimum required to maintain a platelet count > 30 × 10<sup>9</sup>/L but ideally > 50 × 10<sup>9</sup>/L, preferably in the absence of concomitant steroids. After 6 months and beyond, patients who achieved at least a stable partial response (PR; platelets > 50 × 10<sup>9</sup>/L) had eltrombopag tapered by 25 mg each 3 weeks,","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"63 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142874800","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":"Outcomes of CD19 CAR T in Transformed Indolent Lymphoma Compared to De Novo Aggressive Large B-Cell Lymphoma","authors":"Swetha Kambhampati Thiruvengadam, Reid Merryman, Yan Wang, Charles Gaulin, Evandro Bezerra, Timothy Voorhees, Madhav R. Seshadri, Ayo Falade, Alma Habib, Amy A. Ayers, Megumi Bailey, Annette Brown, Neil Bailey, Krish Patel, Charalambos B. Andreadis, Adam S. Kittai, Caron Jacobson, Joycelynne Palmer, Stephen J. Forman, Loretta Nastoupil, Lihua E. Budde","doi":"10.1002/ajh.27548","DOIUrl":"https://doi.org/10.1002/ajh.27548","url":null,"abstract":"Chimeric antigen receptor (CAR) T-cell therapy has revolutionized treatment of aggressive large B-cell lymphoma (aLBCL). Patients with transformed indolent non-Hodgkin lymphoma (tiNHL) were included in key CAR trials, but outcomes of CAR for this distinct, historically high-risk group are poorly understood. We conducted a multicenter retrospective study of 1182 patients with aLBCL receiving standard-of-care CAR T between 2017 and 2022, including 338 (29%) with tiNHL. Rates of grade ≥ 3 cytokine release syndrome (CRS) were similar between tiNHL and de novo cohorts (7% vs. 8%, <i>p</i> = 0.6), while grade ≥ 3 immune effector cell-associated neurotoxicity syndrome was lower in tiNHL (21% vs. 27%, <i>p</i> = 0.02). Overall response rate was similar in both cohorts (83% vs. 81%, <i>p</i> = 0.3), while complete response rate was higher in tiNHL (67% vs. 59%, <i>p</i> = 0.017). With a median follow-up of 22.3 months, the progression/relapse-free (PFS) and overall survival (OS) were similar between the tiNHL and de novo cohorts (24-month PFS 41% [95% CI: 35%–46%] vs. 38% [95% CI: 35%–42%]; 24-month OS 58% [95% CI: 52%–63%] vs. 52% [95% CI: 48%–56%], respectively). After adjusting for key risk factors, there was a trend toward a lower hazard of disease progression, relapse or death post-CAR for tiNHL patients compared to de novo aLBCL patients (HR: 0.84 [95% CI: 0.69–1.0], <i>p</i> = 0.07). Elevated LDH, advanced stage, prior bendamustine within 12 months of CAR, receipt of bridging therapy, CNS involvement, and ≥ 3 prior lines of therapy were each associated with inferior PFS. In conclusion, CAR T therapy is highly effective with an acceptable toxicity profile in patients with tiNHL.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"20 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142874797","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":"Rituximab Plus Bortezomib for Relapsed and Refractory Warm Autoimmune Hemolytic Anemia: A Prospective Phase 2 Trial","authors":"Xijuan Lin, Chen Yang, Junling Zhuang, Jian Li, Daobin Zhou, Miao Chen, Bing Han","doi":"10.1002/ajh.27540","DOIUrl":"https://doi.org/10.1002/ajh.27540","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"54 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142869932","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":"Treatment Effectiveness of Venetoclax-Based Therapy After Bruton Tyrosine Kinase Inhibitors in Chronic Lymphocytic Leukemia: An International Real-World Study","authors":"Nilanjan Ghosh, Toby A. Eyre, Jennifer R. Brown, Nicole Lamanna, Beenish S. Manzoor, Catherine C. Coombs, Hande H. Tuncer, Chaitra Ujjani, Lori A. Leslie, Lindsey E. Roeker, Matthew S. Davids, Joanna M. Rhodes, Alan P. Skarbnik, Wendy Sinai, Isabelle Fleury, Brian T. Hill, Nicolas Martinez-Calle, Paul M. Barr, Dureshahwar Jawaid, Nnadozie Emechebe, Laurie Pearson, Frederick Lansigan, Yun Choi, Christopher E. Jensen, Bita Fakhri, Deborah M. Stephens, Steven E. Marx, Stephen J. Schuster, Michael Coyle, Irina Pivneva, Talissa Watson, Annie Guerin, Mazyar Shadman","doi":"10.1002/ajh.27563","DOIUrl":"https://doi.org/10.1002/ajh.27563","url":null,"abstract":"&lt;p&gt;The treatment landscape of chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) has evolved drastically with the introduction of targeted agents, including covalent Bruton tyrosine kinase inhibitors (cBTKis) and B-cell lymphoma 2 inhibitors (BCL2is) [&lt;span&gt;1&lt;/span&gt;]. However, the development of cBTKi resistance (leading to disease progression) and intolerance due to adverse events limits durable cBTKi efficacy [&lt;span&gt;1&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;Venetoclax-based therapy, including fixed-treatment-duration combinations, has demonstrated improved and sustained efficacy and manageable toxicity versus chemotherapy/chemoimmunotherapy (CT/CIT) for patients with previously untreated or relapsed/refractory CLL in phase 3 clinical trials [&lt;span&gt;1&lt;/span&gt;]. Because cBTKis were not yet or were just recently approved during patient recruitment for the initial venetoclax landmark clinical trials, there were few patients with previous exposure to cBTKis and, thus, limited evidence supporting venetoclax efficacy in these settings [&lt;span&gt;2&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;Although clinical trials like M14-032 have established the clinical benefit of venetoclax after cBTKis, real-world studies report on small subgroups of patients with CLL or patients who received venetoclax primarily in later lines of therapy, following CT/CIT [&lt;span&gt;2-5&lt;/span&gt;]. Additionally, there is limited literature evaluating real-world venetoclax outcomes among patients stratified by reasons for cBTKi discontinuation [&lt;span&gt;3&lt;/span&gt;], venetoclax-based regimen (particularly, VR combination) [&lt;span&gt;4, 5&lt;/span&gt;], or prior CT/CIT exposure. This study assessed real-world clinical outcomes of patients with CLL/SLL, who received venetoclax-based therapy after cBTKi therapy, overall and stratified by line of therapy, prior CT/CIT exposure, discontinuation of cBTKi therapy due to intolerance or disease progression, and for treatment with venetoclax + rituximab (VR).&lt;/p&gt;\u0000&lt;p&gt;Data for the current analysis (06/01/2018–12/27/2023) were collected as part of the CLL Collaborative Study of Real-World Evidence (CORE; Supporting Information). Adult patients diagnosed with CLL/SLL were included if they initiated a first-line (1 L) or new line of therapy in a relapsed/refractory setting after 02/12/2014 (US approval date of ibrutinib for CLL/SLL) and if they received a venetoclax-based therapy (i.e., monotherapy or combination therapy with rituximab or obinutuzumab) following discontinuation of a cBTKi-based regimen.&lt;/p&gt;\u0000&lt;p&gt;Clinical outcomes assessed following initiation of venetoclax-based therapy included overall response rate (ORR), time to next treatment or death (TTNT-D), and progression-free survival (PFS). Outcomes were assessed for the overall population and stratified by line of therapy (1 L cBTKi➔second-line [2 L] venetoclax; 2 L cBTKi➔third-line [3 L] venetoclax), CT/CIT exposure before cBTKi treatment for 2 L cBTKi➔3 L venetoclax, primary reason for cBTKi discontinuation (either intolerance [D&lt;sub&gt;I&lt;/sub&gt; gr","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"23 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142849556","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":"Risk Factors and Prognostic Markers for Posterior Reversible Encephalopathy Syndrome in Patients With Thalassemia Major After Allogeneic Hematopoietic Stem Cell Transplantation","authors":"Meiru Bu, Kemei Deng, Meiqing Wu, Lingling Shi, Yuhong Qin, Muliang Jiang, Bihong T. Chen","doi":"10.1002/ajh.27566","DOIUrl":"https://doi.org/10.1002/ajh.27566","url":null,"abstract":"&lt;p&gt;Posterior reversible encephalopathy syndrome (PRES) has been known as a neurological complication in patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). PRES is an acute or subacute syndrome with various neurological symptoms such as headache, encephalopathy, visual disturbance and seizures [&lt;span&gt;1&lt;/span&gt;]. Neuroimaging may show subcortical white matter vasogenic edema typically in bilateral parieto-occipital lobes [&lt;span&gt;1&lt;/span&gt;]. The etiology of PRES is not clear, which has been speculated as cerebrovascular dysregulation associated with exposure to toxic agents such as chemotherapeutic drugs and immunosuppressive agents [&lt;span&gt;1&lt;/span&gt;]. Several studies have indicated that severe hypertension, Pesaro class III, and acute graft-versus-host disease (aGVHD) are potential risk factors for PRES in patients with transfusion-dependent thalassemia (TDT) after allo-HSCT [&lt;span&gt;2, 3&lt;/span&gt;]. Severity of brain magnetic resonance imaging (MRI) findings and the length of in-hospital stay were independent risk factors related to adverse outcomes from PRES in patients with TDT after allo-HSCT [&lt;span&gt;4&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;We enrolled consecutive patients with TDT after allo-HSCT from October 2010 and May 2023 and assessed their clinical factors and brain MRI findings. Univariate and multivariate binary logistic regression were used to identify the risk factors for the occurrence and the prognosis of PRES. Receiver operating characteristic (ROC) curve analysis was performed to obtain the area under the curve (AUC) values for assessing the prediction efficacy. The study methods are detailed in the Supporting Information.&lt;/p&gt;\u0000&lt;p&gt;A total of 770 patients diagnosed with TDT underwent allo-HSCT during the study interval (Table S1). All patients underwent one allo-HSCT, with the mean age at 7.36 years. Among them, 64 patients had PRES with an incidence of 8.3%. The median time of onset for PRES from the start of allo-HSCT was 77 days (ranging from 12 to 441 days). Ten patients who developed PRES had poor prognosis, with a score of 3–6 on the modified Rankin Scale (mRS) clinical outcome scale (Supporting Information: Methods). Three of the 64 patients with PRES died, resulting in a mortality rate of 4.69%. Among the cohort of 64 cases with PRES, 45 cases showed mild brain MRI findings, 16 cases showed moderate MRI findings, and 3 cases exhibited severe MRI abnormalities. We found 6 patients showing hyperintensity on diffusion-weighted images (DWI &lt;i&gt;b&lt;/i&gt; values = 1000s/mm&lt;sup&gt;2&lt;/sup&gt;) and hypointensity in apparent diffusion coefficient (ADC). A summary of MRI findings of PRES is presented in Figure 1. The median duration for complete resolution of lesions during follow-up was 14 days (range 6–99 days). The median interval for lesion improvement was 21 days (range 3–66 days), with the earliest documented improvement observed at 3 days post-diagnosis.&lt;/p&gt;\u0000&lt;figure&gt;&lt;picture&gt;\u0000&lt;source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/2118989b-c890-","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"23 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142849237","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 Use of Eltrombopag and Avatrombopag in Pediatric ITP in China: A Real-World Multicenter Retrospective Cohort Study","authors":"Jingjing Liu, Zhifa Wang, Nan Wang, Jingyao Ma, Yu Hu, Jie Ma, Lijuan Wang, Yan Liu, Juntao Ouyang, Zhenping Chen, Xiaoling Cheng, Runhui Wu","doi":"10.1002/ajh.27554","DOIUrl":"https://doi.org/10.1002/ajh.27554","url":null,"abstract":"&lt;p&gt;Immune thrombocytopenia (ITP) is an acquired bleeding disorder characterized by a reduced platelet count of less than 100 × 10&lt;sup&gt;9&lt;/sup&gt;/L, with an estimated incidence of 2–5/100 000 children per year [&lt;span&gt;1&lt;/span&gt;]. Since 2014, the China National Medical Products Administration (NMPA) has approved five TPO-RAs for treating ITP in China. Among these, eltrombopag (ELT) and avatrombopag (AVA) are the two most evidence-based and available oral medications for pediatric ITP; both demonstrated promising efficacy and tolerability in clinical trials [&lt;span&gt;2, 3&lt;/span&gt;]. Because no head-to-head studies have been conducted, making direct comparisons between the two drugs is difficult. To address this gap, we conducted a multicenter, retrospective, observational cohort study to evaluate the efficacy and safety of ELT versus AVA in pediatric patients with ITP in China, to offer personalized treatment guidance based on factors such as the severity of bleeding, medication adherence, and family economic conditions.&lt;/p&gt;\u0000&lt;p&gt;Patients younger than 18 with a confirmed diagnosis of ITP who received treatment with ELT or AVA in 3 Children's Specialty Hospitals from April 2017 to December 2023, with medication duration for at least 2 months and with a total observation period of more than 6 months. For this retrospective study, ethics approval was obtained from the institutional review boards of Beijing Children's Hospital, Capital Medical University (Ethics approval NO. [2024]-Y-098-D).&lt;/p&gt;\u0000&lt;p&gt;The initial dosage of ELT was determined by age and body weight. For children aged 1–5 years, the starting dose was 1.5 mg/kg/day. In those aged 6–17 years, the dose was 37.5 mg/day for individuals weighing less than 27 kg and 50 mg/day for those weighing 27 kg or more. Similarly, the starting dose of AVA was 10 mg/day for children aged 1–6 years and 20 mg/day for those aged 6–18 years.&lt;/p&gt;\u0000&lt;p&gt;The current study defines several key definitions and outcomes [&lt;span&gt;4&lt;/span&gt;]:(1) Complete Response (CR): a platelet count ≥ 100 × 10&lt;sup&gt;9&lt;/sup&gt;/L and no bleeding within 2 months. (2) Response (R): a platelet count of 30–100 × 10&lt;sup&gt;9&lt;/sup&gt;/L, with at least a twofold increase from baseline and no bleeding within 2 months. (3) No Response (NR): a platelet count &lt; 30 × 10&lt;sup&gt;9&lt;/sup&gt;/L, less than a twofold increase from baseline, or bleeding after dose titration of ELT or AVA within 2 months. (4) Overall Response (OR): the combined number of patients achieving CR and R. (5) Durable Response (DR): a platelet count &gt; 30 × 10&lt;sup&gt;9&lt;/sup&gt;/L with at least a twofold increase from baseline at 6 months. (6) Remission: a platelet count ≥ 30 × 10&lt;sup&gt;9&lt;/sup&gt;/L at 12 months. (7) SRoT: a platelet count ≥ 30 × 10&lt;sup&gt;9&lt;/sup&gt;/L for at least 6 months off ELT or AVA treatment. (8) Time to typer: defined as the period from the initiation of ELT/AVA therapy to the commencement of dosage tapering within the first 12 months. (9) Rescue therapy, including IVIG, platelet transfusion, and high-do","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"48 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142841749","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":"Blastic Plasmocytoid Dendritic Cell Neoplasm With Pseudo-Lymphoid Morphology Mimicking Lymphoid Malignancy","authors":"Romain Ravel-Chapuis, Gérard Buchonnet, Catherine Boutet, Francine Garnache Ottou, Anne Roggy, Florian Renosi, Olivier Matray, Victor Bobée","doi":"10.1002/ajh.27567","DOIUrl":"https://doi.org/10.1002/ajh.27567","url":null,"abstract":"&lt;p&gt;A 60-year-old male presented with spontaneous hematomas and cervical adenopathy. Laboratory workup revealed hemoglobin 12.3 g/dL, platelet 56 × 10&lt;sup&gt;9&lt;/sup&gt;/L, and leukocyte 14.7 × 10&lt;sup&gt;9&lt;/sup&gt;/L. The automated cell counter (Sysmex XN) reported 9% neutrophils (1.32 × 10&lt;sup&gt;9&lt;/sup&gt;/L) and 86% lymphocytes (12.6 × 10&lt;sup&gt;9&lt;/sup&gt;/L), suggestive of a lymphoproliferative disorder. Blood smear showed atypical lymphoid cells with normal size, mature chromatin, and scant cytoplasm Figure 1 (Panel A), while numerous lysed cells were also observed (Panel B). About 10% of these cells displayed cytoplasmic vacuoles, occasionally coalescing, and 10% appeared larger with grayish cytoplasm (Panel C and D). Due to the apparent lymphocytosis, flow cytometry immunophenotyping was performed (Navios EX, Beckman-Coulter). Surprisingly, 62% of the leukocytes consisted of a CD45+low blastic population. These cells were negative for common lymphoid (CD19/CD20/CD22/CD24/cCD79a/CD3/CD5/CD8/CD10) and myeloid antigens (CD34/CD38/CD13/CD14/CD42b/CD61/CD64/CD65/CD117/cMPO), but were positive for CD4(weak)/CD56/CD123(strong)/HLA-DR(strong)/CD33 (Panel E and F). A cutaneous lesion was found on the patient's back, and bone marrow aspiration confirmed 82% of the same population with additional phenotypic markers indicative of pDC lineage (CD303+weak/CD304+/cTCL1+/BadLamp+), supporting the diagnosis of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The neurological examination was normal, and cerebrospinal involvement was inconclusive due to hemorrhagic cerebrospinal fluid samples. Furthermore, the mutational profile performed on bone marrow aspirate was consistent with BPDCN, revealing mutations in &lt;i&gt;TET2, ASXL1&lt;/i&gt;, and &lt;i&gt;NF1&lt;/i&gt;, as well as a subclonal &lt;i&gt;NRAS&lt;/i&gt; mutation and deletions in the &lt;i&gt;IKZF1&lt;/i&gt; and &lt;i&gt;ETV6&lt;/i&gt; loci.&lt;/p&gt;\u0000&lt;p&gt;BPDCN is a rare hematologic malignancy, with diverse morphological presentations. While the classic blastic morphology, often exhibiting “pearl necklace” cytoplasmic appearances and “hand-mirror-like” projections, is the most common presentation, this case displayed an unusual morphology dominated by pseudo-lymphoid cells. Although a subset of pseudo-lymphoid cells is commonly observed in BPDCN, this case was remarkable in that nearly all cells exhibited this distinctive morphology. Such atypical presentation can be deceptive, initially mimicking lymphoid malignancies. Here, the immunophenotypic profile was crucial in establishing the accurate diagnosis.&lt;/p&gt;\u0000&lt;figure&gt;&lt;picture&gt;\u0000&lt;source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/066ddd2e-9d43-407c-8ef9-7a0c7829025e/ajh27567-fig-0001-m.jpg\"/&gt;&lt;img alt=\"Details are in the caption following the image\" data-lg-src=\"/cms/asset/066ddd2e-9d43-407c-8ef9-7a0c7829025e/ajh27567-fig-0001-m.jpg\" loading=\"lazy\" src=\"/cms/asset/606f65ba-04c1-4c37-bab4-d263c99fa856/ajh27567-fig-0001-m.png\" title=\"Details are in the caption following the image\"/&gt;&lt;/picture&gt;&lt;figcaption&gt;\u0000&lt;div&gt;&lt;strong&gt;FIGURE 1&lt;span sty","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"1 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142841774","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":"Pseudothrombocytopenia due to Phagocytosis of Platelets by Polymorphonuclear Leukocytes","authors":"Iliana Stamatiou, Zoe Bezirgiannidou, Evangelia Charitaki, Ioannis Kotsianidis, Konstantinos Liapis","doi":"10.1002/ajh.27562","DOIUrl":"https://doi.org/10.1002/ajh.27562","url":null,"abstract":"&lt;p&gt;A 75-year-old woman presented to the emergency department with progressive abdominal pain, fever, and diarrhea after taking levofloxacin for a respiratory tract infection. On evaluation, she was in clinical shock, with blood pressure 88/58 mmHg and heart rate 122 beats per minute. A complete blood count provided by the Sysmex XN-1000 analyzer showed leukocytosis (13.8 × 10&lt;sup&gt;9&lt;/sup&gt;/L, 95% neutrophils) and thrombocytopenia (22 × 10&lt;sup&gt;9&lt;/sup&gt;/L). She had acidosis, renal impairment, coagulopathy, and elevated C-reactive protein level. Because of the thrombocytopenia, an examination of a peripheral-blood smear was performed in the hematology laboratory, which showed vacuolated neutrophils that contained phagocytized platelets. Of 200 neutrophils examined, 161 (80%) contained between one and six platelets (Figure 1). These findings indicated spurious thrombocytopenia. The emergency department staff were notified by the laboratory that the patient's platelet count was normal. Subsequently, she underwent internal jugular-vein catheterization for fluid resuscitation without oozing or hematoma. Pseudomembranous colitis was diagnosed on the basis of a positive &lt;i&gt;Clostridioides difficile&lt;/i&gt; stool test. She was treated with metronidazole and vancomycin, but her course was complicated by renal failure necessitating hemodialysis. Eventually, she made a full recovery. During hospitalization, multiple routinely prepared films from EDTA-anticoagulated blood consistently demonstrated platelet phagocytosis but with the resolution of the colitis, the phenomenon became progressively less pronounced. The automated platelet count became normal within 30 days.&lt;/p&gt;\u0000&lt;figure&gt;&lt;picture&gt;\u0000&lt;source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/43ba7b26-cdb9-4a52-851b-76e271182229/ajh27562-fig-0001-m.jpg\"/&gt;&lt;img alt=\"Details are in the caption following the image\" data-lg-src=\"/cms/asset/43ba7b26-cdb9-4a52-851b-76e271182229/ajh27562-fig-0001-m.jpg\" loading=\"lazy\" src=\"/cms/asset/533e4fe2-c86d-4138-a00c-28ff5b938961/ajh27562-fig-0001-m.png\" title=\"Details are in the caption following the image\"/&gt;&lt;/picture&gt;&lt;figcaption&gt;\u0000&lt;div&gt;&lt;strong&gt;FIGURE 1&lt;span style=\"font-weight:normal\"&gt;&lt;/span&gt;&lt;/strong&gt;&lt;div&gt;Open in figure viewer&lt;i aria-hidden=\"true\"&gt;&lt;/i&gt;&lt;span&gt;PowerPoint&lt;/span&gt;&lt;/div&gt;\u0000&lt;/div&gt;\u0000&lt;div&gt;Four fields of the peripheral-blood smear, showing ingestion of platelets by neutrophilic granulocytes (May-Grünwald-Giemsa stain, ×1000).&lt;/div&gt;\u0000&lt;/figcaption&gt;\u0000&lt;/figure&gt;\u0000&lt;p&gt;A peripheral-blood smear should always be examined in new cases of thrombocytopenia or whenever the platelet count is unexpectedly low, in order to confirm the thrombocytopenia. The blood smear may be requested by physicians or initiated by laboratory staff [&lt;span&gt;1&lt;/span&gt;]. As seen here, a laboratory-initiated blood smear, is particularly valuable because it may permit earlier recognition of pseudothrombocytopenia. The incidence of pseudothrombocytopenia is 1.9% among hospitalized patients and 0.15% in the outpatient setting [","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"26 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142825503","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}