Acta Haematologica最新文献

{"title":"Fostamatinib or Thrombopoietin for the Treatment of Chronic Immune Thrombocytopenia in Adult Patients: A Real-World Assessment of Safety, Effectiveness and Cost.","authors":"George Dranitsaris, Aaron Peevyhouse, Tiffany Wood, Yuliya Kreychman, Heather Neuhalfen, Mehdi Moezi","doi":"10.1159/000533175","DOIUrl":"10.1159/000533175","url":null,"abstract":"<p><strong>Introduction: </strong>Chronic immune thrombocytopenia purpura (ITP) in adults is a serious autoimmune disease in which platelets are prematurely destroyed, leaving the patient vulnerable to bruising and bleeding. Initial treatment starts with corticosteroids. In patients who become resistant or intolerant to corticosteroids, the thrombopoietic agents (TPOs), consisting of romiplostim (ROM), eltrombopag (ELT), and avatrombopag (AVA), or the spleen tyrosine kinase inhibitor fostamatinib (FOS), are appropriate next lines of therapy. In this study, the comparative safety, effectiveness, and cost of care between fostamatinib and the TPOs were evaluated in a real-world setting.</p><p><strong>Methods: </strong>A retrospective analysis of 17 community hematology practices across the USA was conducted to identify adult ITP patients who received one of the four agents. Data collection consisted of patient demographics, disease characteristics, as well as number and type of prior treatments. From the first day until the end of treatment, data were also collected on platelet (PLT) counts, adverse events, the use of rescue IVIG, platelet transfusions, and corticosteroids. Multivariable logistic regression analysis was used to compare PLT-related endpoints between agents.</p><p><strong>Results: </strong>A sample of 179 ITP patients who had received at least one of the four agents was identified. This resulted in a final sample of 51, 87, 127, and 44 patients who received FOS, ELT, ROM, or AVA, respectively. At month six, there were no significant differences between FOS and the TPOs in terms of the proportion of patients with the PLT count being ≥30 × 103/μL, ≥50 × 103/μL as well as the proportion of patients whose PLT levels doubled relative to baseline. The frequency of thromboembolic events (TEs) was 3.9% in FOS patients compared to 9.2%, 4.7%, and 11.4% in the ELT, ROM, and AVA groups. The mean cost per patient with FOS was $99,209 (95% CI: $59,595-$115,074), compared to $92,426 (95% CI: $68,331-$115,519), $108,482 (95% CI: $84,782-$132,182), and $131,050 (95% CI: $83,327-$179,897) for ELT, ROM, or AVA, respectively.</p><p><strong>Conclusions: </strong>In this real-world analysis, FOS was comparable to the TPOs in maintaining PLTs at clinically beneficial levels. Given these findings, the choice of therapy should be based on overall patient safety, preexisting risk factors for TEs, and cost effectiveness.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"333-343"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41123077","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Front & Back Matter","authors":"","doi":"10.1159/000531365","DOIUrl":"https://doi.org/10.1159/000531365","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47209630","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Front & Back Matter","authors":"R. Hillman, I. Ben-Bassat","doi":"10.1159/000530345","DOIUrl":"https://doi.org/10.1159/000530345","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47345119","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Challenging Management of Acute Thrombotic Microangiopathy in Pregnancy.","authors":"Bárbara Marques,&nbsp;Catarina Nora,&nbsp;Daniela Coelho,&nbsp;Patrícia Martinho,&nbsp;Teresa Fidalgo,&nbsp;Marília Gomes,&nbsp;Letícia Ribeiro,&nbsp;Catarina Geraldes,&nbsp;José Pedro Carda","doi":"10.1159/000527553","DOIUrl":"https://doi.org/10.1159/000527553","url":null,"abstract":"<p><p>Thrombotic microangiopathy is characterized by microangiopathic hemolytic anemia, thrombocytopenia, and end-organ injury. Pregnancy-associated thrombotic microangiopathy is a severe disorder with a high risk of maternal mortality and poor fetal outcomes. Preeclampsia/eclampsia and hemolysis, elevated liver enzymes, and low platelets syndrome are the most common causes, and hemolytic uremic syndrome or thrombotic thrombocytopenic purpura are rare causes. Due to overlapping clinical findings, the differential diagnosis is challenging and should be managed by a multidisciplinary team. We present a case of a 38-year-old woman at 40 weeks of second gestation, admitted with thrombotic microangiopathy being the final diagnosis not immediately clear.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 1","pages":"72-75"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9219791","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High Rates of Anti-αIIbβ3 Antibodies Produced by a Glanzmann Thrombasthenia Patient after First and Unique Red Blood Cells Administration.","authors":"Yoann Huguenin,&nbsp;Laure Levoir,&nbsp;Muriel Bouton,&nbsp;Mathieu Fiore","doi":"10.1159/000526224","DOIUrl":"https://doi.org/10.1159/000526224","url":null,"abstract":"<p><p>Immunization against the platelet αIIbβ3 glycoprotein due to blood transfusion represents one of the most severe complications in Glanzmann thrombasthenia (GT) disease. Anti-αIIbβ3 isoantibodies development may lead to ineffective platelet transfusion and can, in case of pregnancy, cross the placenta leading to fetal thrombocytopenia. We describe here the case of a girl with type I GT who developed high rates of anti-αIIbβ3 isoantibodies after first and unique blood transfusion. Surprisingly, this patient had only received red blood cell concentrates and immunization was presumably stimulated by the residual presence of platelets in concentrates. This study emphasizes the need for regular anti-αIIbβ3 antibodies screening in GT, even though patients have never been previously transfused with platelet concentrates.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 1","pages":"44-46"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10665630","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acute Myeloid Leukemia Treatment in the Elderly: A Comprehensive Review of the Present and Future.","authors":"Jun H Choi, Mihir Shukla, Maher Abdul-Hay","doi":"10.1159/000531628","DOIUrl":"10.1159/000531628","url":null,"abstract":"<p><strong>Background: </strong>Acute myeloid leukemia (AML) is a disease of the hematopoietic system that remains a therapeutic challenge despite advances in our understanding of the underlying cancer biology in the past decade. It is also an affliction of the elderly that predominantly affects patients over 60 years of age. Standard therapy involves intensive chemotherapy that is often difficult to tolerate in older populations. Fortunately, recent developments in molecular targeting have shown promising results in treating leukemia, paving the way for novel treatment strategies that are easier to tolerate.</p><p><strong>Summary: </strong>Venetoclax, a BCL-2 inhibitor, when combined with a hypomethylating agent, has proven to be a highly effective and well-tolerated drug and established itself as a new standard for treating AML in patients who are unfit for standard intensive therapy. Other targeted therapies include clinically proven and FDA-approved agents, such as IDH1/2 inhibitors, FLT3 inhibitors, and Gemtuzumab, as well as newer and more experimental drugs such as magrolimab, PI-kinase inhibitors, and T-cell engaging therapy. Some of the novel agents such as magrolimab and menin inhibitors are particularly promising, providing therapeutic options to a wider population of patients than ever before. Determining who will benefit from intense or novel low-intense therapy remains a challenge, and it requires careful assessment of individual patient's fitness and disease characteristics.</p><p><strong>Key messages: </strong>This article reviews past and current treatment strategies that harness various mechanisms of leukemia-targeting agents and introduces novel therapies on the horizon aimed at exploring therapeutic options for the elderly and unfit patient population. It also provides a strategy to select the best available therapy for elderly patients with both newly diagnosed and relapsed/refractory AML.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"431-457"},"PeriodicalIF":1.7,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10203483","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prediction of Splenectomy Outcomes in Relapsed or Refractory Immune Thrombocytopenia.","authors":"Philip Murphy","doi":"10.1159/000530678","DOIUrl":"https://doi.org/10.1159/000530678","url":null,"abstract":"A recent publication by Kwag et al suggests that first line response to intravenous immunoglobulins in patients with immune thrombocytopenia (ITP) may predict long term response to splenectomy. Predominant platelet sequestration using platelet kinetic studies may similarly predict ITP patients who respond rest to splenectomy. A study comparing response to intravenous immunoglobulins and platelet kinetic studies prior to splenectomy for ITP might identify to identify the optimal means of predicting response to splenectomy.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 4","pages":"343-344"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9974972","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Role of Follicular Regulatory T Cells/Follicular Helper T Cells in Primary Immune Thrombocytopenia.","authors":"Mingling Sun,&nbsp;Xiujuan Wang,&nbsp;Lei Wang,&nbsp;Xinyou Wang,&nbsp;Wenxia Fan,&nbsp;Ying Liu,&nbsp;Qinzhi Li,&nbsp;Ning Zhang,&nbsp;Xinhong Guo","doi":"10.1159/000529963","DOIUrl":"https://doi.org/10.1159/000529963","url":null,"abstract":"<p><strong>Introduction: </strong>Immune thrombocytopenia (ITP) is an autoimmune disease characterized by thrombocytopenia. Herein, we sought to identify potential immune-related therapeutic targets in ITP.</p><p><strong>Methods: </strong>The differentially expressed genes (DEGs) between ITP patients and controls in GSE43177 and PRJNA299534 were analyzed. The intersections of the two DEG groups were screened as common genes, and enrichment analysis was performed. Additionally, differential analysis of immune cell levels between ITP and controls was performed. Changes in the proportions of T follicular helper (Tfh) and follicular regulatory T (Tfr) cells in peripheral blood samples from ITP patients, ITP patients responding to therapy, and healthy controls were identified. The expression changes in B-cell lymphoma (Bcl)-6 and interleukin (IL)-21 were further evaluated.</p><p><strong>Results: </strong>A total of 76 common genes were identified, and enrichment analysis found that these genes were mainly associated with neutrophil-mediated immunity, the MAPK signaling pathway, and the FOXO signaling pathway. Furthermore, we found different levels of Tfh cells in patients with ITP and controls. The level of Tfh cells in the peripheral blood is significantly increased in ITP patients and declines after responding to therapy. The Tfr/Tfh ratio was reduced in ITP patients and increased after responding to therapy. IL-21 and Bcl-6 were more highly expressed in ITP patients than in controls.</p><p><strong>Conclusion: </strong>We identified abnormally expressed genes in ITP related to immune-related biological functions. We further identified the changes in Tfh and Tfr cells during ITP treatment. This provides a rationale for immunotherapy in ITP patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 4","pages":"267-276"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9983998","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic Significance of CD20 Expression in Children with Philadelphia Chromosome-Negative B-Cell Precursor Acute Lymphoblastic Leukemia.","authors":"Yu Wang,&nbsp;Yu-Juan Xue,&nbsp;Yue-Ping Jia,&nbsp;Ying-Xi Zuo,&nbsp;Ai-Dong Lu,&nbsp;Le-Ping Zhang","doi":"10.1159/000530849","DOIUrl":"10.1159/000530849","url":null,"abstract":"<p><strong>Introduction: </strong>The prognostic significance of CD20 in pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL) remains unclear. Therefore, in this study, we evaluated the prognostic value of CD20 expression in leukemia blasts in pediatric BCP-ALL at our institute.</p><p><strong>Methods: </strong>Between 2005 and 2017, 796 children with newly diagnosed Philadelphia-negative BCP-ALL were enrolled consecutively; clinical characteristics and treatment outcomes were analyzed and compared between CD20-positive and CD20-negative groups.</p><p><strong>Results: </strong>CD20 positivity was observed in 22.7% of enrolled patients. The analysis of overall and event-free survival showed that white blood cell count ≥50 × 109/L, no ETV6-RUNX1, day 33 minimal residual disease (MRD) ≥0.1%, and week 12 MRD ≥0.01% were independent risk factors. Meanwhile, in the CD20-positive group, week 12 MRD ≥0.01% was the only factor associated with long-term survival. Moreover, subgroup analysis revealed that in patients with extramedullary involvement (p = 0.047), MRD ≥0.1% on day 33 (p = 0.032), or MRD ≥0.01% at week 12 (p = 0.004), CD20 expression led to a poorer outcome compared to those without CD20 expression.</p><p><strong>Conclusions: </strong>Pediatric BCP-ALL with CD20 expression had unique clinicopathological characteristics, and MRD remained the major prognostic factor. CD20 expression had no prognostic value in pediatric BCP-ALL.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"349-357"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9754731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Geographical Distance from Quaternary Treatment Center on Clinical Trial Participation, Intensive Induction Chemotherapy, and Outcomes in Patients with Newly Diagnosed Acute Myeloid Leukemia.","authors":"Samantha A Hershenfeld,&nbsp;Eshetu G Atenafu,&nbsp;Steven Chan,&nbsp;Vikas Gupta,&nbsp;Dawn Maze,&nbsp;Andre Schuh,&nbsp;Hassan Sibai,&nbsp;Karen Yee,&nbsp;Arron Schimmer","doi":"10.1159/000531484","DOIUrl":"10.1159/000531484","url":null,"abstract":"<p><strong>Introduction: </strong>Care for patients with acute myeloid leukemia (AML) is centralized in the Ontario single-payer public healthcare system, with intensive induction chemotherapy and clinical trials only offered at specialized cancer centers with large catchment areas.</p><p><strong>Methods: </strong>We therefore conducted a retrospective single-center review of all AML patients assessed at a large specialized cancer center in Ontario, Canada.</p><p><strong>Results: </strong>Between 2012 and 2017, 1,310 patients were assessed by our center for upfront AML therapy. The median distance was 33.1 km, with 29% of patients living more than 50 km away from the center. There was no significant difference in probability of intensive induction chemotherapy or clinical trial by distance from center, both in univariate and multivariable analysis adjusting for age, sex, cytogenetics and molecular testing, and performance status. There was no significant difference in overall survival by distance from center on univariate and multivariable analysis.</p><p><strong>Conclusion: </strong>In conclusion, geographic distance from treatment center does not appear to impact choice of upfront therapy, participation in clinical trials, or clinical outcomes in this study of newly diagnosed patients with AML treated in a single-payer environment.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"366-372"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9632287","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}