Blood advances最新文献

{"title":"Peripheral T-cell lymphoma-NOS in children and adolescents: a review from the Children's Oncology Group NHL Committee.","authors":"Kaitlin J Devine, Lindsay Schwartz, Nader Kim El-Mallawany","doi":"10.1182/bloodadvances.2024013689","DOIUrl":"10.1182/bloodadvances.2024013689","url":null,"abstract":"<p><strong>Abstract: </strong>Peripheral T-cell lymphoma not otherwise specified (PTCL-NOS) is a rare mature T-cell non-Hodgkin lymphoma (NHL) seen in both children and adults. Although it is the most common nonanaplastic mature T-cell lymphoma of childhood, it is quite rare and, therefore, the standard of care remains largely undefined. It is a disease characterized by clinical and pathological heterogeneity and is generally associated with an aggressive clinical course and poor prognosis in adults. Retrospective reports on treatment outcomes for pediatric PTCL-NOS are limited by small cohorts, variable clinical presentations, and heterogeneous treatment regimens. Although published survival rates in children appear encouraging compared with those from prospective studies in adults, the prognosis is guarded, and relatively low curative outcomes are in stark contrast to more common pediatric NHL. Although recent landmark gene profiling studies have shed light on the molecular landscape of the disease in adults, identifying molecular subgroups with prognostic significance, the biology of PTCL-NOS remains unclear in children. Here, we review the clinical presentation and diagnosis, historical treatment approaches, current knowledge of the disease biology, and the role of hematopoietic stem cell transplant (HSCT) in PTCL-NOS in children to pursue a better understanding of this heterogeneous condition and empower physicians to use this information to best support our pediatric population. Studies focusing on pediatric PTCL-NOS are required to unravel the disease biology in children, improve risk stratification, and better define upfront treatment through the role of targeted agents and HSCT, as we look to future directions of the care of children with PTCL-NOS.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1420-1431"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11960635/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143000273","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":"Differential effects of C5 inhibition, C3 inhibition, and alternative pathway inhibition on bacterial killing in vitro.","authors":"Mendy Ter Avest, Zenno de Boer, Saskia M C Langemeijer, Nicole C A J van de Kar, Marien I de Jonge, Rob Ter Heine, Jeroen D Langereis","doi":"10.1182/bloodadvances.2024013587","DOIUrl":"10.1182/bloodadvances.2024013587","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1280-1285"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11950765/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143000067","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":"Pediatric relapsed/refractory ALK-positive anaplastic large cell lymphoma treatment and outcomes in the targeted-drug era.","authors":"Lianna J Marks, Victor Ritter, Jennifer E Agrusa, Kala Y Kamdar, Julie Rivers, Rebecca Gardner, Matthew J Ehrhardt, Kaitlin J Devine, Charles A Phillips, Anne Reilly, Keith August, Joanna Weinstein, Prakash Satwani, Christopher J Forlenza, Christine Moore Smith, Chelsee Greer, Zeinab Afify, Carol H Lin, Jennifer A Belsky, Hilda Ding, David Hoogstra, Keri Toner, Michael P Link, Liora M Schultz, Eric J Lowe, Catherine Aftandilian","doi":"10.1182/bloodadvances.2024014745","DOIUrl":"10.1182/bloodadvances.2024014745","url":null,"abstract":"<p><strong>Abstract: </strong>Treatment options for patients with relapsed or refractory (R/R) anaplastic large cell lymphoma (ALCL) have increased in the era of targeted therapies such as brentuximab vedotin (BV) and anaplastic lymphoma kinase (ALK) inhibitors. However, there is no standard treatment and published data evaluating their use are limited. The goal of this retrospective study was to describe current real-world treatment and outcomes of pediatric, adolescent, and young adult patients with R/R ALK-positive ALCL. We conducted a retrospective, multi-institutional study identifying 81 patients with R/R ALK-positive ALCL aged ≤21 years at initial diagnosis treated between 2011 and 2022 across 18 institutions. Median time from diagnosis to relapse was 8.9 months (range, 2.6-131.9). Initial reinduction regimens included ALK-inhibitor monotherapy (n = 37, 46%), BV monotherapy (n = 19, 23%), chemotherapy without targeted therapy (n = 12, 15%), chemotherapy with targeted therapy (n = 9, 11%), or vinblastine monotherapy (n = 4, 5%), with 83% of patients achieving a complete response to initial reinduction regimen. Fifty-eight patients received a hematopoietic stem cell transplant (HSCT), 11 autologous and 48 allogeneic, with 1 receiving both. Duration of treatment for patients receiving BV or the ALK-inhibitor crizotinib (CZ) varied widely (BV, 1-11 years; CZ, 2-10 years). Five-year event-free survival was 63% (95% confidence interval [CI], 53-75) and 5-year overall survival was 91% (95% CI, 84-98). This is, to our knowledge, the largest collection of patients with R/R ALK-positive ALCL treated in the era of targeted therapy. Patients achieved excellent responses to ALK-inhibitor or BV monotherapy, but questions remain about duration of therapy and role of HSCT.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1356-1365"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954107/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022261","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":"Anxiety and depression in patients with histiocytic neoplasms and their associated clinical features.","authors":"Anne S Reiner, Yesne Alici, Denise D Correa, Dana Bossert, Allison M Sigler, Deanna Fournier, Kathleen Brewer, Gaurav Goyal, Thomas M Atkinson, Priya Marathe, Jun J Mao, Katherine S Panageas, Eli L Diamond","doi":"10.1182/bloodadvances.2024014850","DOIUrl":"10.1182/bloodadvances.2024014850","url":null,"abstract":"<p><strong>Abstract: </strong>Anxiety and depression are common in many cancers but, to our knowledge, have not been systematically studied in patients with histiocytic neoplasms (HNs). We sought to estimate rates of anxiety and depression and identify clinical features and patient-reported outcomes (PROs) associated with anxiety and depression in patients with HNs. A registry-based cohort of patients with HNs completing PROs including the hospital anxiety and depression scale (HADS) from 2018 to 2023 was identified. Moderate or severe anxiety or depression were respectively defined as a score of ≥11 on the HADS anxiety or depression subscales. Associations of variables, including other validated PROs, with moderate or severe anxiety or depression were modeled with logistic regression to estimate odds ratios and 95% confidence intervals. In 215 patients, ∼1 in 3 met the criteria for anxiety or depression, and 1 in 7 met the criteria for moderate or severe anxiety or depression. These estimates remained stable over a 12-month trajectory. Rates of depression, but not anxiety, significantly differed across HN types, with patients with Erdheim-Chester disease experiencing the highest rate. In addition, neurologic involvement, unemployment, and longer undiagnosed illness interval were significantly associated with increased risk of depression. Financial burden, financial worry, and severe disease-related symptoms were correlated with increased risk of both anxiety and depression. Conversely, increased general and cognitive health-related quality of life (HRQoL) were correlated with decreased risk of both anxiety and depression. In patients with HN, anxiety and depression are prevalent, stable over time, and correlated with financial burden, symptom severity, and HRQoL. This trial was registered at www.clinicaltrials.gov as #NCT03329274.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1376-1386"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11957516/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142766395","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":"Elderberry wine and promyelocytic leukemia.","authors":"Steven D Gore","doi":"10.1182/bloodadvances.2025015905","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025015905","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":"9 6","pages":"1474-1475"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143699711","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":"Subclonal TP53 and KRAS variants combined with poor treatment response identify ultrahigh-risk pediatric patients with T-ALL.","authors":"Tamara Kempter, Paulina Richter-Pechańska, Katarzyna Michel, Tobias Rausch, Büşra Erarslan-Uysal, Cornelia Eckert, Martin Zimmermann, Martin Stanulla, Martin Schrappe, Gunnar Cario, Stefan Köhrer, Andishe Attarbaschi, Jan O Korbel, Joachim B Kunz, Andreas E Kulozik","doi":"10.1182/bloodadvances.2024014209","DOIUrl":"10.1182/bloodadvances.2024014209","url":null,"abstract":"<p><strong>Abstract: </strong>Variations in the TP53 and KRAS genes indicate a particularly adverse prognosis in relapsed pediatric T-cell acute lymphoblastic leukemia (T-ALL). We hypothesized that these variations might be subclonally present at disease onset and contribute to relapse risk. To test this, we examined 2 cohorts of children diagnosed with T-ALL: cohort 1 with 81 patients who relapsed and 79 who matched nonrelapsing controls, and cohort 2 with 226 consecutive patients, 30 of whom relapsed. In cohort 1, targeted sequencing revealed TP53 clonal and subclonal variants in 6 of 81 relapsing patients but none in the nonrelapsing group (P = .014). KRAS alterations were found in 9 of 81 relapsing patients compared with 2 of 79 nonrelapsing patients (P = .032). Survival analysis showed that none of the relapsed patients with TP53 and/or KRAS alterations survived, whereas 19 of 67 relapsed patients without such variants did, with a minimum follow-up time of 3 years (P = .023). In cohort 2, none of the relapsing patients but 10 of 196 nonrelapsing patients carried TP53 or KRAS variants, indicating that mutation status alone does not predict poor prognosis. All 10 nonrelapsing patients with mutations had a favorable early treatment response. Among the total cohort of 386 patients, 188 showed poor treatment response, of whom 69 relapsed. Of these poor responders, 9 harbored TP53 or KRAS variants. In conclusion, subclonal TP53 and KRAS alterations identified at the time of initial diagnosis, along with a poor treatment response, characterize a subset of children with T-ALL who face a dismal prognosis and who may benefit from alternative treatment approaches.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1267-1279"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11950767/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142982813","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":"American Society of Hematology living guidelines on use of anticoagulation for thromboprophylaxis for patients with COVID-19: executive summary.","authors":"Deborah M Siegal, Eric K Tseng, Holger J Schünemann, Pantep Angchaisuksiri, Adam Cuker, Kathryn Dane, Maria T DeSancho, David Diuguid, Daniel O Griffin, Frederikus A Klok, Alfred Ian Lee, Ignacio Neumann, Ashok Pai, Marc Righini, Kristen M Sanfilippo, Deirdra R Terrell, Elie A Akl, Reyad Al Jabiri, Yazan Al Jabiri, Angela M Barbara, Antonio Bognanni, Imad Bou Akl, Mary Boulos, Romina Brignardello-Petersen, Matthew Chan, Rana Charide, Luis E Colunga-Lozano, Karin Dearness, Andrea J Darzi, Heba Hussein, Samer G Karam, Philipp Kolb, Razan Mansour, Gian Paolo Morgano, Rami Z Morsi, Giovanna Muti-Schünemann, Menatalla K Nadim, Atefeh Noori, Binu A Philip, Thomas Piggott, Yuan Qiu, Yetiani Roldan Benitez, Finn Schünemann, Adrienne Stevens, Karla Solo, Wojtek Wiercioch, Reem A Mustafa, Robby Nieuwlaat","doi":"10.1182/bloodadvances.2024014219","DOIUrl":"10.1182/bloodadvances.2024014219","url":null,"abstract":"<p><strong>Background: </strong>COVID-19-related critical and acute illness is associated with an increased risk of venous thromboembolism (VTE). These evidence-based recommendations of the American Society of Hematology (ASH) are intended to support patients, clinicians, and other health care professionals in decisions about using anticoagulation for thromboprophylaxis for patients with COVID-19-related critical illness; patients with COVID-19-related acute illness; and those being discharged from the hospital, who do not have suspected or confirmed VTE.</p><p><strong>Methods: </strong>ASH formed a multidisciplinary panel, including patient representatives. The Michael G. DeGroote Cochrane Canada and MacGRADE Centres at McMaster University supported guideline development, including performing systematic reviews (up to June 2023). The panel prioritized clinical questions and outcomes according to their importance for clinicians and patients. The panel used the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach to assess certainty in the evidence and make recommendations.</p><p><strong>Results: </strong>This is an executive summary of 3 updated recommendations that have been published, which concludes the living phase of the guidelines. For patients with COVID-19-related critical illness, the panel issued conditional recommendations suggesting (a) prophylactic-intensity over therapeutic-intensity anticoagulation and (b) prophylactic-intensity over intermediate-intensity anticoagulation. For patients with COVID-19-related acute illness, conditional recommendations were suggested (a) prophylactic-intensity over intermediate-intensity anticoagulation, and (b) therapeutic-intensity over prophylactic-intensity anticoagulation. The panel issued a conditional recommendation suggesting against the use of postdischarge anticoagulant thromboprophylaxis.</p><p><strong>Conclusions: </strong>These conditional recommendations were made based on low or very low certainty in the evidence, underscoring the need for additional, high-quality, randomized controlled trials for patients with COVID-19.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1247-1260"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11950770/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142495372","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":"Imetelstat: drugging telomerase in transfusion-dependent low- and intermediate-1-risk MDS.","authors":"Joseph Cannova, Rafael Madero-Marroquin, Olatoyosi Odenike","doi":"10.1182/bloodadvances.2024013929","DOIUrl":"10.1182/bloodadvances.2024013929","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1390-1391"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11957495/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142944974","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 A-HIPI prediction model in advanced-stage Hodgkin lymphoma: identification of risk groups and creation of an online tool.","authors":"Matthew J Maurer, Susan K Parsons, Jenica N Upshaw, Angie Mae Rodday, Raphael Mwangi, Sára Rossetti, Jonathan W Friedberg, Andrea Gallamini, Massimo Federico, Eliza Hawkes, David Hodgson, Peter Johnson, Brian K Link, Eric Mou, Kerry J Savage, Pier Luigi Zinzani, Andrew M Evens","doi":"10.1182/bloodadvances.2024014689","DOIUrl":"10.1182/bloodadvances.2024014689","url":null,"abstract":"","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1366-1369"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954104/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142944979","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":"Transcriptomic atlas reveals organ-specific disease tolerance in sickle cell mice.","authors":"Anne Grunenwald, Julie Peliconi, Julien Lavergne, Margot Revel, Elodie Voilin, Tania Robe-Rybkine, Gilles Crambert, Jordan D Dimitrov, Olivier Blanc-Brude, Lubka T Roumenina","doi":"10.1182/bloodadvances.2024013435","DOIUrl":"10.1182/bloodadvances.2024013435","url":null,"abstract":"<p><strong>Abstract: </strong>Sickle cell disease (SCD) is the most common genetic disease in the world and a societal challenge. SCD is characterized by multiorgan injury related to intravascular hemolysis. To understand tissue-specific responses to intravascular hemolysis and exposure to heme, we present a transcriptomic atlas of the primary target organs of hemoglobin S (HbSS) vs hemoglobin 1 (HbAA) transgenic SCD mice. We explored the transcriptomes of the liver, kidney, heart, lung, and bone marrow from HbAA and HbSS Townes littermates at resting state and their changes after the injection of heme, assessed by RNA sequencing. Inflammation and myeloid cell signatures were omnipresent in resting HbSS organs, with the liver being the most affected. The injection of heme triggered a robust inflammatory response in HbAA mice. Signatures of exposure to heme in HbAA mice were downstream of toll like receptor 4, sensor of lipopolysaccharides but also of heme, interleukin-1β (IL-1β), IL-6, and interferon gamma, similarly to HbSS mice at rest. Nevertheless, HbSS mice were strikingly unresponsive to the heme administration, irrespective of the organ. This tolerance was driven by upregulation of the heme-detoxifying enzyme heme oxygenase-1 and was abrogated by its specific inhibition. Therefore, HbSS mice develop robust protective mechanisms, which may explain how they and patients with SCD survive bouts of severe hemolysis.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1405-1419"},"PeriodicalIF":7.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11960547/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143022263","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}