Therapeutic Advances in Hematology最新文献

{"title":"Future needs for continuing innovation in hemophilia: improving outcomes for individuals of all severities, including women and those in resource-constrained regions.","authors":"Jan Blatný, Jan Astermark, Cristina Catarino, Gerry Dolan, Karin Fijnvandraat, Cédric Hermans, Katharina Holstein, Víctor Jiménez-Yuste, Robert Klamroth, Michelle Lavin, Peter J Lenting, Sébastien Lobet, Maria Elisa Mancuso, Jayashree Motwani, James S O'Donnell, Christoph Königs","doi":"10.1177/20406207241285143","DOIUrl":"https://doi.org/10.1177/20406207241285143","url":null,"abstract":"<p><p>Over recent decades, management of people with hemophilia (PwH) has been greatly improved by scientific advances that have resulted in a rich and varied therapeutic landscape. Nevertheless, treatment limitations continue to drive innovation, and emerging options have the potential to realize further improvement. We advocate four general principles to optimize benefits from innovation: individualizing the treatment approach, targeting 'normal,' making the most of available resources, and considering treatment affordability. Ultimately, all PwH-men and women, of all ages and severities, and worldwide-should have access to treatment that fully prevents bleeding, while allowing personal, social, family, and professional lives of choice. Clearly, we are not there yet, but developing goals/milestones based on the principles we describe may help to achieve this.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241285143"},"PeriodicalIF":3.4,"publicationDate":"2024-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11459663/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142393604","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The efficacy and safety of third-party umbilical blood/umbilical cord mesenchymal stem cell assisted related haploid hematopoietic stem cell transplantation in pediatric patients with acute leukemia: an observational study.","authors":"Chang Liu, Minyuan Liu, Xin Liu, Bohan Li, Li Gao, Shuiyan Wu, Qi Ji, Zhiqi Zhang, Senlin Zhang, Peifang Xiao, Jun Lu, Jie Li, Shaoyan Hu","doi":"10.1177/20406207241277549","DOIUrl":"10.1177/20406207241277549","url":null,"abstract":"<p><strong>Background: </strong>There is limited data on third-party umbilical cord blood (UCB) or mesenchymal stem cell (MSC) transplantation-assisted haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in pediatric patients.</p><p><strong>Objective: </strong>To evaluate the efficacy and safety of UCB and MSC transplantation-assisted haplo-HSCT in pediatric patients with acute leukemia (AL).</p><p><strong>Design: </strong>Observational study.</p><p><strong>Methods: </strong>Clinical data of 152 children with AL undergoing haplo-HSCT at the Children's Hospital of Soochow University between January 2020 and June 2022 were collected. The patients were divided into the haplo-HSCT + UCB group (<i>n</i> = 76), haplo-HSCT + MSC group (<i>n</i> = 31), and haplo-HSCT group (<i>n</i> = 45). Hematopoietic reconstruction time, complications within 30 days after transplantation, and survival and recurrence at 3 years after transplantation were compared among the groups.</p><p><strong>Results: </strong>Multivariate analysis revealed that haplo-HSCT with MSC and human leukocyte antigen (HLA) matching ⩾6/10 were independent factors reducing engraftment syndrome (ES) incidence. There were no significant differences among the groups in the hematopoietic reconstruction time or incidence of complications within 30 days after transplantation (<i>p</i> > 0.05). Overall survival, relapse-free survival, cumulative incidence of relapse, cumulative incidence of hematological relapse, and 3-year transplant-related mortality were not significantly different (<i>p</i> > 0.05). The incidence of adverse reactions in the haplo-HSCT + UCB group was 97.3% within 4 h after UCB infusion, with a particularly high occurrence rate of 94.7% for hypertension. No transfusion-related adverse reactions occurred after the transfusion of umbilical cord MSC in the haplo-HSCT + MSC group.</p><p><strong>Conclusion: </strong>MSC-assisted haplo-HSCT can reduce ES incidence after transplantation in pediatric patients with AL. UCB infusion is associated with a high incidence of reversible hypertension. However, no adverse reactions were observed in umbilical cord MSC transfusion.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241277549"},"PeriodicalIF":3.4,"publicationDate":"2024-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11452895/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142381614","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic impact of <i>IL7R</i> mutations on acute myeloid leukemia.","authors":"Qiqi Tao, Qiaoyuan Wu, Yutong Xue, Changkun Chen, Ya Zhou, Ruoyang Shao, Haiyan Zhang, Hui Liu, Xiangzong Zeng, Lingling Zhou, Qifa Liu, Hua Jin","doi":"10.1177/20406207241279533","DOIUrl":"https://doi.org/10.1177/20406207241279533","url":null,"abstract":"<p><strong>Background: </strong>Interleukin-7 receptor (<i>IL7R</i>) mutation has been demonstrated to be an adverse prognostic factor in acute lymphoblastic leukemia (ALL) patients. However, the effects of the <i>IL7R</i> mutation on acute myeloid leukemia (AML) have rarely been reported. Here, we investigated <i>IL7R</i> mutations and their effects on AML patients.</p><p><strong>Methods: </strong>A total of 346 newly diagnosed AML patients from January 2017 to July 2020 at Nanfang Hospital were analyzed in this study. A genomic panel of 167 gene targets was detected by next-generation sequencing.</p><p><strong>Results: </strong>Among 346 patients, 33 (9.5%) AML patients carried <i>IL7R</i> mutations. With a median follow-up of 50.7 months (95% confidence interval (CI) 17.3-62.2), the 5-year overall survival (OS) rates were 51.5% (95% CI 37.0%-71.0%) and 72.2% (95% CI 67.4%-77.3%; <i>p</i> = 0.008), the 5-year event-free survival (EFS) rates were 36.1% (95% CI 23.2%-57.1%) and 58.1% (95% CI 52.9%-63.8%; <i>p</i> = 0.005), the 5-year non-relapse mortality (NRM) were 21.4% (95% CI 8.5%-38.2%) and 6.2% (95% CI 3.7%-9.5%; <i>p</i> = 0.004) in the IL7R mutant (<i>IL7R</i> ^<i>MUT</i> ) group and non-IL7R mutant (<i>IL7R</i> ^<i>WT</i> ) group, respectively. There is no significant difference in the disease-free survival (75.1% vs 73.5%, <i>p</i> = 0.885) and cumulative incidence of relapse (25.7% vs 25.2%, <i>p</i> = 0.933) between <i>IL7R</i> ^<i>MUT</i> and <i>IL7R</i> ^<i>WT</i> group. Furthermore, patients who underwent hematopoietic stem cell transplantation (HSCT) still had more adverse outcomes in the <i>IL7R</i> ^<i>MUT</i> group than in the <i>IL7R</i> ^<i>WT</i> group (5-year OS: 61.9% vs 85.3%, <i>p</i> = 0.003). In the <i>TET2</i> (<i>p</i> = 0.013) and DNA methyltransferase 3A (<i>DNMT3A; p</i> = 0.046) mutation subgroups, the presence of <i>IL7R</i> mutations was associated with worse OS than in AML patients without <i>IL7R</i> mutations.</p><p><strong>Conclusion: </strong>Our study demonstrated that the <i>IL7R</i> mutation is associated with an inferior prognosis for AML patients. Patients with <i>IL7R</i> mutations have higher NRM, shorter OS, and EFS than patients without <i>IL7R</i> mutations, even patients who have undergone HSCT. Future larger and multicentric prospective studies will be explored.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241279533"},"PeriodicalIF":3.4,"publicationDate":"2024-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11439168/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354313","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Successful rechallenge with Erwinia chrysanthemi asparaginase after pegaspargase-induced hypertriglyceridemia: a case report.","authors":"Gaia Ciolli, Andrea Pasquini, Francesco Mannelli, Barbara Scappini, Giacomo Gianfaldoni, Elisa Quinti, Laura Fasano, Jessica Caroprese, Francesca Crupi, Alessandro Maria Vannucchi, Matteo Piccini","doi":"10.1177/20406207241270846","DOIUrl":"https://doi.org/10.1177/20406207241270846","url":null,"abstract":"<p><p>Polyethylene-glycolated <i>Escherichia coli</i>-derived l-asparaginase (pegaspargase, pASP) is an essential component of paediatric-inspired regimens for the treatment of acute lymphoblastic leukaemia/lymphoma; nonetheless, is characterised by severe and potentially life-threatening toxicities, such as hypertriglyceridemia. Grades 3-4 events have been reported in ~1%-18% of paediatric patients and in sparse reports in adults. There is limited evidence on the safety of asparaginase rechallenge in patients experiencing severe pASP-related hypertriglyceridemia. Herein we present the case of a young adult patient diagnosed with T-LBL who experienced an asymptomatic severe pASP-related hypertriglyceridemia and was safely re-exposed to ASP using Erwinia chrysanthemi asparaginase (crisantapase), with only mild transient hypertriglyceridemia recurrence.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241270846"},"PeriodicalIF":3.4,"publicationDate":"2024-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11425753/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354314","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adding venetoclax or hypomethylating agents to induction chemotherapy as first-line treatment for adults with acute myeloid leukemia: a retrospective case-cohort study","authors":"Fangfei Xu, Kuangguo Zhou, Duanhao Gong, Wei Huang","doi":"10.1177/20406207241275850","DOIUrl":"https://doi.org/10.1177/20406207241275850","url":null,"abstract":"Background:The response rate of traditional first-line induction chemotherapy (IC) for newly diagnosed acute myeloid leukemia needs to be improved, but it is not clear whether adding venetoclax or hypomethylating agents (HMAs) to IC will improve the response rate.Objective:To determine whether venetoclax or HMAs could increase the response rate of IC in patients with newly diagnosed acute myeloid leukemia (AML).Design:A retrospective, propensity score matching analysis.Methods:Newly diagnosed AML patients at Tongji Hospital between 2021 and 2023 were included in this study. By matching cases and controls based on age, gender, baseline bone marrow blast cell proportion, type of AML, and the National Comprehensive Cancer Network (NCCN) risk stratification group, we compared the response rate (CR, CR/CRi, ORR, and MRD negative) and hematological adverse events in newly diagnosed AML treated with IC plus venetoclax or HMAs versus IC alone after one cycle of IC.Results:The addition of venetoclax could improve CR/CRi of IC (83.8% for IC plus venetoclax vs 66.1% for IC alone, p = 0.029). The addition of venetoclax to IA regimen did not improve CR/CRi of IA regimen (76.9% for IA plus venetoclax vs 76.2% for IA alone, p = 0.986). The addition of HMAs could not only improves CR/CRi of IC (85.3%% for IC plus HMAs vs 65.4% for IC alone, p = 0.002) but also improves CR/CRi of IA regimen (91.3% for IA plus HMAs vs 70.0% for IA alone, p = 0.034). The addition of HMAs could improve CR/CRi of patients with adverse mutations (FLT3, IDH1/2, K/NRAS) after IC. The addition of venetoclax and HMAs both extended the duration of agranulocytosis and thrombocytopenia.Conclusion:Adding HMAs might improve CR/CRi of IC including IA. Adding venetoclax might not improve CR/CRi of IA. A well-designed prospective randomized controlled study is now warranted.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"1 1","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Is less more? Intravenous immunoglobulin for pediatric immune thrombocytopenia","authors":"Eyal Elron, Joanne Yacobovich, Orly Efros, Osama Tanous, Sarina Levy-Mendelovich, Esti Shamba, Orna Steinberg-Shemer, Tracie Goldberg, Shai Izraeli, Oded Gilad","doi":"10.1177/20406207241279202","DOIUrl":"https://doi.org/10.1177/20406207241279202","url":null,"abstract":"Objectives:Treatment of pediatric immune thrombocytopenia (ITP) is guided by the risk of bleeding. Intravenous immunoglobulin (IVIg) is one of the first-line therapy options for new-onset pediatric ITP. However, the exact optimal dose of IVIg has not been determined.Methods:This retrospective cohort study included all hospitalized children with newly diagnosed ITP receiving IVIg as first-line therapy during 2010–2020. We compared the safety and efficacy of two common IVIg dose regimens, 1 and 2 g/kg. Outcomes were short and long-term treatment responses and adverse events to the different doses.Results:A total of 168 children were included in our cohort. Eighty-two children were treated with 1 g/kg of IVIg and 86 with 2 g/kg. There was no difference in sustained response (platelet count &gt; 20 × 10<jats:sup>9</jats:sup>, &gt; 14 days) between the groups (74.3% vs 76.7%, respectively, p = 0.72) and maximal platelet counts following treatment ( p = 0.44). No difference was found regarding the percentage of chronic ITP between the two groups (24.4% in the 1 g/kg group as compared to 17.4% in the 2 g/kg group; p = 0.34). Logistic regression analysis demonstrated there was no effect of the IVIg dose on treatment failure and development of chronic ITP. As anticipated, 47.7% of adverse events were in the 2 g/kg group and 32.9% in the 1 g/kg group, with borderline statistical significance ( p = 0.06).Conclusion:The initial treatment of newly diagnosed pediatric ITP using a 1 g/kg IVIg regimen may give comparable results to the double dose of 2 g/kg in attaining a prolonged safe hemostatic threshold, without impacting the incidence of chronic disease.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"215 1","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251402","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Systematic literature review of the indirect costs and humanistic burden of β-thalassemia","authors":"Yesim Aydinok, Sneha Purushotham, Aylin Yucel, Mrudula Glassberg, Sohan Deshpande, Barbara Potrata, Myrto Trapali, Farrukh Shah","doi":"10.1177/20406207241270872","DOIUrl":"https://doi.org/10.1177/20406207241270872","url":null,"abstract":"Background:β-Thalassemia is an inherited blood disorder requiring lifetime management of anemia and its complications.Objective:This study aimed to determine the indirect costs and humanistic burden of β-thalassemia.Design:A systematic literature review was conducted.Data sources and methods:Searches were conducted in Embase, MEDLINE, MEDLINE In-Process, and EconLit (November 1, 2010, to November 25, 2020). Studies reporting indirect costs and health-related quality of life (HRQoL) for patients with β-thalassemia were eligible.Results:Seventy-five publications were included. Mean annual days lost due to transfusion-related absenteeism ranged from 15.6 to 35 days. Patients spent a mean of 592 min (standard deviation (SD): 349) daily on disease management on transfusion days and 91 min (SD: 221) daily on non-transfusion days. Patients with non-transfusion-dependent β-thalassemia (NTDT) showed worse HRQoL versus those with transfusion-dependent β-thalassemia (TDT) on the 36-item Short Form Health Survey (75.8 vs 66.5; p = 0.021). Caregivers of patients with TDT had more severe stress compared with patients (20.17 vs 18.95; p = 0.006), as measured by the standardized Cohen Perceived Stress Questionnaire.Conclusion:TDT is associated with substantial indirect costs and caregiver burden, and NTDT is associated with worse HRQoL. There is an unmet need for novel treatments in both TDT and NTDT that minimize patient and caregiver burden.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"179 1","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251403","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Phase I/II study of the clinical activity and safety of GSK3326595 in patients with myeloid neoplasms","authors":"Justin Watts, Mark D. Minden, Kimo Bachiashvili, Andrew M. Brunner, Sameem Abedin, Timothy Crossman, Magdalena Zajac, Veronica Moroz, Jacqueline L. Egger, Aarti Tarkar, Brandon E. Kremer, Olena Barbash, Gautam Borthakur","doi":"10.1177/20406207241275376","DOIUrl":"https://doi.org/10.1177/20406207241275376","url":null,"abstract":"Background:GSK3326595 is a potent, selective, reversible protein arginine methyltransferase 5 (PRMT5) inhibitor under investigation for treatment of myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML). In preclinical models of AML, PRMT5 inhibition decreased proliferation and increased cell death, supporting additional clinical research in myeloid neoplasms.Objectives:To determine the clinical activity, safety, tolerability, dosing, additional measures of clinical activity, pharmacokinetics, and pharmacodynamics of GSK3326595.Design:In part 1 of this open-label, multicenter, multipart, phase I/II study, adults with relapsed/refractory myeloid neoplasms (e.g., MDS, CMML, and AML) received monotherapy with 400 or 300 mg oral GSK3326595 once daily. Study termination occurred prior to part 2 enrollment.Methods:Clinical activity was determined by the clinical benefit rate (CBR; proportion of patients achieving complete remission (CR), complete marrow remission (mCR), partial remission, stable disease (SD) &gt;8 weeks, or hematologic improvement). Adverse events (AEs) were assessed by incidence and severity. Exploratory examination of spliceosome mutations was performed to determine the relationship between genomic profiles and clinical response to GSK3326595.Results:Thirty patients with a median age of 73.5 years (range, 47–90) were enrolled; 13 (43%) and 17 (57%) received 400 and 300 mg of GSK3326595, respectively. Five (17%) patients met CBR criteria: 4 (13%) with SD &gt;8 weeks and 1 (3%) achieving mCR. Of five patients with clinical benefit: three had SRSF2 mutation, one U2AF1, and one was splicing factor wild-type. Frequent GSK3326595-related AEs were decreased platelet count (27%), dysgeusia (23%), fatigue (20%), and nausea (20%). GSK3326595 had rapid absorption, with a T<jats:sub>max</jats:sub> of approximately 2 h and a terminal half-life of 4–6 h.Conclusion:GSK3326595 monotherapy had limited clinical activity in heavily pretreated patients despite robust target engagement. The safety profile was broadly consistent with other published PRMT5 inhibitor studies.Trial registration:ClinicalTrials.gov: NCT03614728.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 1","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251404","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Targeting B-cell maturation antigen for treatment and monitoring of relapsed/refractory multiple myeloma patients: a comprehensive review","authors":"David Yashar, Bernard Regidor, Marissa-Skye Goldwater, Sean Bujarski, Ashley Del Dosso, James R. Berenson","doi":"10.1177/20406207241275797","DOIUrl":"https://doi.org/10.1177/20406207241275797","url":null,"abstract":"Despite major therapeutic advancements in recent years, multiple myeloma (MM) remains an incurable disease with nearly all patients experiencing relapsed and refractory disease over the course of treatment. Extending the duration and durability of clinical responses will necessitate the development of therapeutics with novel targets that are capable of robustly and specifically eliminating myeloma cells. B-cell maturation antigen (BCMA) is a membrane-bound protein expressed predominantly on malignant plasma cells and has recently been the target of several novel therapeutics to treat MM patients. This review will focus on recently approved and currently in development agents that target this protein, including bispecific antibodies, antibody-drug conjugates, and chimeric antigen receptor T-cell therapies. In addition, this protein also serves as a novel serum biomarker to predict outcomes and monitor disease status for MM patients; the studies demonstrating this use of BCMA will be discussed in detail.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"65 1","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251405","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Vedolizumab for second-line treatment of steroid-refractory gastrointestinal late acute graft-versus-host disease.","authors":"Yingling Zu, Ruirui Gui, Zhen Li, Juan Wang, Pei Li, Ying Liu, Xiaofeng Dong, Jian Zhou","doi":"10.1177/20406207241276982","DOIUrl":"10.1177/20406207241276982","url":null,"abstract":"<p><strong>Background: </strong>Late acute graft-versus-host disease (aGVHD) is a complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT) with little data regarding treatment and outcomes. There is no standard treatment for gastrointestinal (GI) late aGVHD, especially for steroid-refractory (SR) GI late aGVHD. Vedolizumab, a monoclonal antibody inhibiting the migration of both naive and activated lymphocytes into the GI endothelium, has been verified to be effective for SR GI aGVHD.</p><p><strong>Methods: </strong>We retrospectively analyzed the clinical efficacy and safety of vedolizumab as the second line for SR GI late aGVHD in seven patients after allo-HSCT.</p><p><strong>Results: </strong>Four patients received two doses of vedolizumab infusion, while three patients received only one dose of vedolizumab infusion. The complete response and partial response rates were 57.1% (4/7) and 42.9% (3/7), respectively. No patient progressed to chronic GVHD during the period of follow-up. There was no severe adverse event related to vedolizumab.</p><p><strong>Conclusion: </strong>Our data suggest that vedolizumab is expected to ameliorate SR GI late aGVHD. Further data on the treatment timing, efficacy, and safety of vedolizumab are warranted in prospective clinical trials.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241276982"},"PeriodicalIF":3.4,"publicationDate":"2024-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11380122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142154997","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}