Biology of Blood and Marrow Transplantation最新文献

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Clinical-Grade Expanded Regulatory T Cells Are Enriched with Highly Suppressive Cells Producing IL-10, Granzyme B, and IL-35 临床级扩增调节性T细胞富含产生IL-10、颗粒酶B和IL-35的高抑制性细胞
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.08.034
Francesca Ulbar , Ida Villanova , Raffaella Giancola , Stefano Baldoni , Francesco Guardalupi , Bianca Fabi , Paola Olioso , Anita Capone , Rosaria Sola , Sara Ciardelli , Beatrice Del Papa , Antonello Brattelli , Ilda Ricciardi , Stefano Taricani , Giulia Sabbatinelli , Ornella Iuliani , Cecilia Passeri , Paolo Sportoletti , Stella Santarone , Antonio Pierini , Mauro Di Ianni
{"title":"Clinical-Grade Expanded Regulatory T Cells Are Enriched with Highly Suppressive Cells Producing IL-10, Granzyme B, and IL-35","authors":"Francesca Ulbar ,&nbsp;Ida Villanova ,&nbsp;Raffaella Giancola ,&nbsp;Stefano Baldoni ,&nbsp;Francesco Guardalupi ,&nbsp;Bianca Fabi ,&nbsp;Paola Olioso ,&nbsp;Anita Capone ,&nbsp;Rosaria Sola ,&nbsp;Sara Ciardelli ,&nbsp;Beatrice Del Papa ,&nbsp;Antonello Brattelli ,&nbsp;Ilda Ricciardi ,&nbsp;Stefano Taricani ,&nbsp;Giulia Sabbatinelli ,&nbsp;Ornella Iuliani ,&nbsp;Cecilia Passeri ,&nbsp;Paolo Sportoletti ,&nbsp;Stella Santarone ,&nbsp;Antonio Pierini ,&nbsp;Mauro Di Ianni","doi":"10.1016/j.bbmt.2020.08.034","DOIUrl":"10.1016/j.bbmt.2020.08.034","url":null,"abstract":"<div><p>In the setting of T cell-depleted, full-haplotype mismatched transplantation, adoptive immunotherapy with regulatory T cells (Tregs) and conventional T cells (Tcons) can prevent graft-versus-host disease (GVHD) and improve post-transplantation immunologic reconstitution and is associated with a powerful graft-versus-leukemia effect. To improve the purity and the quantity of the infused Tregs, good manufacturing practices (GMP)-compatible expansion protocols are needed. Here we expanded Tregs using an automated, clinical-grade protocol. Cells were extensively characterized in vitro, and their efficiency was tested in vivo in a mouse model. Tregs were selected by CliniMacs (CD4<sup>+</sup>CD25<sup>+</sup>, 94.5 ± 6.3%; FoxP3<sup>+</sup>, 63.7 ± 11.5%; CD127<sup>+</sup>, 20 ± 3%; suppressive activity, 60 ± 7%), and an aliquot of 100 × 10<sup>6</sup> was expanded for 14 days using the CliniMACS Prodigy System, obtaining 684 ± 279 × 10<sup>6</sup> cells (CD4<sup>+</sup>CD25<sup>+</sup>, 99.6 ± 0.2%; FoxP3<sup>+</sup>, 82 ± 8%; CD127<sup>+</sup>, 1.1 ± 0.8%; suppressive activity, 75 ± 12%). CD39 and CTLA4 expression levels increased from 22.4 ± 12% to 58.1 ± 13.3% (<em>P</em> &lt; .05) and from 20.4 ± 6.7% to 85.4 ± 9.8% (<em>P</em> &lt; .01), respectively. TIM3 levels increased from .4 ± .05% to 29 ± 16% (<em>P</em> &lt; .05). Memory Tregs were the prevalent population, whereas naive Tregs almost disappeared at the end of the culture. mRNA analysis displayed significant increases in CD39, IL-10, granzyme B, and IL-35 levels at the end of culture period (<em>P</em> &lt; .05). Conversely, IFNγ expression decreased significantly by day +14. Expanded Tregs were sorted according to TIM3, CD39, and CD62L expression levels (purity &gt;95%). When sorted populations were analyzed, TIM3<sup>+</sup> cells showed significant increases in IL-10 and granzyme B (<em>P</em> &lt; .01) .When expanded Tregs were infused in an NSG murine model, mice that received Tcons only died of GVHD, whereas mice that received both Tcons and Tregs survived without GVHD. GMP grade expanded cells that display phenotypic and functional Treg characteristics can be obtained using a fully automated system. Treg suppression is mediated by multiple overlapping mechanisms (eg, CTLA-4, CD39, IL-10, IL-35, TGF-β, granzyme B). TIM3<sup>+</sup> cells emerge as a potentially highly suppressive population.</p><p>© 2020 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2204-2210"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.034","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38406253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 8
Outcomes with Autologous or Allogeneic Stem Cell Transplantation in Patients with Plasma Cell Leukemia in the Era of Novel Agents 在新药物时代,自体或异体干细胞移植治疗浆细胞白血病的结果
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.08.035
Christopher Lemieux, Laura J. Johnston, Robert Lowsky, Lori S. Muffly, Juliana K. Craig, Parveen Shiraz, Andrew Rezvani, Matthew J. Frank, Wen-Kai Weng, Everett Meyer, Judith Shizuru, Sally Arai, Robert Negrin, David B. Miklos, Surbhi Sidana
{"title":"Outcomes with Autologous or Allogeneic Stem Cell Transplantation in Patients with Plasma Cell Leukemia in the Era of Novel Agents","authors":"Christopher Lemieux,&nbsp;Laura J. Johnston,&nbsp;Robert Lowsky,&nbsp;Lori S. Muffly,&nbsp;Juliana K. Craig,&nbsp;Parveen Shiraz,&nbsp;Andrew Rezvani,&nbsp;Matthew J. Frank,&nbsp;Wen-Kai Weng,&nbsp;Everett Meyer,&nbsp;Judith Shizuru,&nbsp;Sally Arai,&nbsp;Robert Negrin,&nbsp;David B. Miklos,&nbsp;Surbhi Sidana","doi":"10.1016/j.bbmt.2020.08.035","DOIUrl":"10.1016/j.bbmt.2020.08.035","url":null,"abstract":"<div><p>Plasma cell leukemia (PCL) is a rare and very aggressive plasma cell disorder. The optimal treatment approach, including whether to pursue an autologous (auto) or allogeneic (allo) stem cell transplantation (SCT) is not clear, given the lack of clinical trial-based evidence. This single-center retrospective study describes the outcomes of 16 patients with PCL (n = 14 with primary PCL) who underwent either autoSCT (n = 9) or alloSCT (n = 7) for PCL in the era of novel agents, between 2007 and 2019. The median age of the cohort was 58 years. High-risk cytogenetics were found in 50% of the patients. All patients received a proteasome inhibitor and/or immunomodulatory drug-based regimen before transplantation. At the time of transplantation, 10 patients (62%) obtained at least a very good partial response (VGPR). The response after autoSCT (3 months) was at least a VGPR in 6 patients (67%; complete response [CR] in 5). All patients undergoing alloSCT achieved a CR at 3 months. Maintenance therapy was provided to 5 patients (56%) after autoSCT. The median progression-free survival after transplantation was 6 months in the autoSCT group, compared with 18 months in the alloSCT group (<em>P</em> = .09), and median overall survival (OS) after transplantation in the 2 groups was 19 months and 40 months, respectively (<em>P</em> = .41). The median OS from diagnosis was 27 months and 49 months, respectively (<em>P</em> = .50). Of the 11 deaths, 10 patients (91%) died of relapsed disease. AlloSCT was not observed to offer any significant survival advantage over autoSCT in PCL, in agreement with recent reports, and relapse remains the primary cause of death in these patients.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages e328-e332"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.035","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38406255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 7
Predicting Survival after Allogeneic Hematopoietic Cell Transplantation in Myelofibrosis: Performance of the Myelofibrosis Transplant Scoring System (MTSS) and Development of a New Prognostic Model 预测骨髓纤维化患者同种异体造血细胞移植后的生存:骨髓纤维化移植评分系统(MTSS)的性能和新预后模型的发展
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.07.022
Juan-Carlos Hernández-Boluda , Arturo Pereira , Alberto Alvarez-Larran , Ana-Africa Martín , Ana Benzaquen , Lourdes Aguirre , Elvira Mora , Pedro González , Jorge Mora , Nieves Dorado , Antonia Sampol , Valentín García-Gutiérrez , Oriana López-Godino , María-Laura Fox , Juan Luis Reguera , Manuel Pérez-Encinas , María-Jesús Pascual , Blanca Xicoy , Rocío Parody , Leslie González-Pinedo , José-Luis Piñana
{"title":"Predicting Survival after Allogeneic Hematopoietic Cell Transplantation in Myelofibrosis: Performance of the Myelofibrosis Transplant Scoring System (MTSS) and Development of a New Prognostic Model","authors":"Juan-Carlos Hernández-Boluda ,&nbsp;Arturo Pereira ,&nbsp;Alberto Alvarez-Larran ,&nbsp;Ana-Africa Martín ,&nbsp;Ana Benzaquen ,&nbsp;Lourdes Aguirre ,&nbsp;Elvira Mora ,&nbsp;Pedro González ,&nbsp;Jorge Mora ,&nbsp;Nieves Dorado ,&nbsp;Antonia Sampol ,&nbsp;Valentín García-Gutiérrez ,&nbsp;Oriana López-Godino ,&nbsp;María-Laura Fox ,&nbsp;Juan Luis Reguera ,&nbsp;Manuel Pérez-Encinas ,&nbsp;María-Jesús Pascual ,&nbsp;Blanca Xicoy ,&nbsp;Rocío Parody ,&nbsp;Leslie González-Pinedo ,&nbsp;José-Luis Piñana","doi":"10.1016/j.bbmt.2020.07.022","DOIUrl":"10.1016/j.bbmt.2020.07.022","url":null,"abstract":"<div><p>Accurate prognostic tools are crucial to assess the risk/benefit ratio of allogeneic hematopoietic cell transplantation (allo-HCT) in patients with myelofibrosis (MF). We aimed to evaluate the performance of the Myelofibrosis Transplant Scoring System (MTSS) and identify risk factors for survival in a multicenter series of 197 patients with MF undergoing allo-HCT. After a median follow-up of 3.1 years, 47% of patients had died, and the estimated 5-year survival rate was 51%. Projected 5-year risk of nonrelapse mortality and relapse incidence was 30% and 20%, respectively. Factors independently associated with increased mortality were a hematopoietic cell transplantation-specific comorbidity index (HCT-CI) ≥3 and receiving a graft from an HLA-mismatched unrelated donor or cord blood, whereas post-transplant cyclophosphamide (PT-Cy) was associated with improved survival. Donor type was the only parameter included in the MTSS model with independent prognostic value for survival. According to the MTSS, 3-year survival was 62%, 66%, 37%, and 17% for low-, intermediate-, high-, and very high-risk groups, respectively. By pooling together the low- and intermediate-risk groups, as well as the high- and very high-risk groups, we pinpointed 2 categories: standard risk and high risk (25% of the series). Three-year survival was 62% in standard-risk and 25% in high-risk categories (<em>P</em> &lt; .001).</p><p>We derived a risk score based on the 3 independent risk factors for survival in our series (donor type, HCT-CI, and PT-Cy). The corresponding 5-year survival for the low-, intermediate-, and high-risk categories was 79%, 55%, and 32%, respectively (<em>P</em> &lt; .001).</p><p>In conclusion, the MTSS model failed to clearly delineate 4 prognostic groups in our series but may still be useful to identify a subset of patients with poor outcome. We provide a simple prognostic scoring system for risk/benefit considerations before transplantation in patients with MF.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2237-2244"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.022","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38205685","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 12
Individuals, Boundaries, and Graft-versus-Host Disease 个体、边界和移植物抗宿主病
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.09.001
H. Joachim Deeg
{"title":"Individuals, Boundaries, and Graft-versus-Host Disease","authors":"H. Joachim Deeg","doi":"10.1016/j.bbmt.2020.09.001","DOIUrl":"10.1016/j.bbmt.2020.09.001","url":null,"abstract":"<div><p>Hematopoietic cell transplantation generates new individuals, transplant chimeras, composed of 2 genetic partners—the patient and donor-derived cells—no longer restricted by their original genomes. Interactions of donor-derived and recipient cells occur prominently at the boundary of the recipient with a third partner, the microbiome, in particular skin and intestinal tract, leading to disruption of microbiome homeostasis. These interactions of donor and patient cells at the boundary set the stage for the development of graft-versus-host disease, an expression of the defense of individuality by recipient and donor. Establishment of tolerance and return of homeostasis at the boundary will allow for the survival of the new integrated, physiologic individual.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages e309-e312"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.09.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38477308","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
Health-Related Quality-of-Life Comparison of Adult Related and Unrelated HSC Donors: An RDSafe Study 成人相关和非相关HSC供体的健康相关生活质量比较:一项RDSafe研究
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.08.016
Galen E. Switzer , Jessica G. Bruce , Deidre M. Kiefer , Hati Kobusingye , Kaleab Z. Abebe , Rebecca Drexler , RaeAnne M. Besser , Dennis L. Confer , Mary M. Horowitz , Roberta J. King , Bronwen E. Shaw , Marcie Riches , Brandon Hayes-Lattin , Michael Linenberger , Brian Bolwell , Scott D. Rowley , Mark R. Litzow , Michael A. Pulsipher
{"title":"Health-Related Quality-of-Life Comparison of Adult Related and Unrelated HSC Donors: An RDSafe Study","authors":"Galen E. Switzer ,&nbsp;Jessica G. Bruce ,&nbsp;Deidre M. Kiefer ,&nbsp;Hati Kobusingye ,&nbsp;Kaleab Z. Abebe ,&nbsp;Rebecca Drexler ,&nbsp;RaeAnne M. Besser ,&nbsp;Dennis L. Confer ,&nbsp;Mary M. Horowitz ,&nbsp;Roberta J. King ,&nbsp;Bronwen E. Shaw ,&nbsp;Marcie Riches ,&nbsp;Brandon Hayes-Lattin ,&nbsp;Michael Linenberger ,&nbsp;Brian Bolwell ,&nbsp;Scott D. Rowley ,&nbsp;Mark R. Litzow ,&nbsp;Michael A. Pulsipher","doi":"10.1016/j.bbmt.2020.08.016","DOIUrl":"10.1016/j.bbmt.2020.08.016","url":null,"abstract":"<div><p>Multiple investigations have documented the health-related quality-of-life (HRQoL) and donation-related experiences of unrelated donors (URDs), but similar investigations of the related donor (RD) experience have been less common. The central goal of this study was to longitudinally examine and compare HRQoL of RD and URD hematopoietic stem cell (HSC) donors from predonation through 1 year postdonation. This prospective investigation included adult HSC donors ages 18 to 60 years who donated bone marrow or peripheral blood stem cells at one of 48 geographically diverse US transplant/donor centers and completed HRQoL interviews at predonation and 4 weeks and 1 year postdonation. At predonation, related donors were less ambivalent about donation (<em>t</em> = –3.30; <em>P</em> = .001), more satisfied with their decision to donate (<em>t</em> = 2.65; <em>P</em> = .009), and more likely to define themselves as donors (<em>t</em> = 2.94; <em>P</em> = .004) than were URDs. However, related donors were more concerned about the use of needles (odds ratio [OR] = 2.19; <em>P</em> = .012), about who would pay for the procedure (OR = 2.80; <em>P</em> = .011), and the possibility that they would feel responsible if the transplant failed (<em>t</em> = 2.31; <em>P</em> = .022). Shortly postdonation, related donors were more likely to report donation-related pain (<em>t</em> = 2.50; <em>P</em> = .013) and lightheadedness (OR = 3.63; <em>P</em> = .028). At 1 year postdonation, related donors were less likely to be fully recovered from donation (OR = 0.10; <em>P</em> = .010) and more likely to report a longer recovery period following donation (<em>t</em> = 2.57; <em>P</em> = .011), although this latter finding was primarily due to the percentage of related versus unrelated donors not fully recovered at 1 year postdonation (10% versus 1%). Taken together, these findings suggest that current related donor management practices may be sufficient in preparing related donors for the psychological aspects of donation but that there may be more to do in terms of calibrating the description of donation-related experiences and recovery time to the related donor group (i.e., descriptions of donation experiences based on unrelated donation may not provide best estimates of experience for this group).</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2365-2371"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.016","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38292465","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 5
Incidence and Outcome of Late Relapse after Allogeneic Stem Cell Transplantation for Myelofibrosis 同种异体干细胞移植治疗骨髓纤维化后晚期复发的发生率和结果
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.09.006
Isik Kaygusuz Atagunduz , Maximilian Christopeit , Francis Ayuk , Gaby Zeck , Christine Wolschke , Nicolaus Kröger
{"title":"Incidence and Outcome of Late Relapse after Allogeneic Stem Cell Transplantation for Myelofibrosis","authors":"Isik Kaygusuz Atagunduz ,&nbsp;Maximilian Christopeit ,&nbsp;Francis Ayuk ,&nbsp;Gaby Zeck ,&nbsp;Christine Wolschke ,&nbsp;Nicolaus Kröger","doi":"10.1016/j.bbmt.2020.09.006","DOIUrl":"10.1016/j.bbmt.2020.09.006","url":null,"abstract":"<div><p>In this cross-sectional study, we retrospectively evaluated the files of 227 patients with myelofibrosis who underwent transplantation between 1994 and 2015 for relapse later than 5 years after allogeneic stem cell transplantation (SCT). A total of 94 patients who were alive and in remission at 5 years were identified with follow-up of at least 5 years (median, 9.15 years) after SCT. Thirteen patients (14%) experienced late molecular (n = 6) or hematologic (n = 7) relapse at a median of 7.1 years while 81 patients did not experience relapse. Relapse patients received either donor lymphocyte infusion (DLI) (n = 7) and/or second transplantation (n = 4). Of those, 72.7% achieved again full donor cell chimerism and molecular remission, and after a median follow-up of 45 months, the 3-year overall survival rates for patients with or without relapse were 90.9% (95% confidence interval [CI], 77% to 100%) and 98.8% (95% CI, 96% to 100%), respectively (<em>P</em> = .13). We conclude that late relapse occurs in about 14% of the patients and the majority can be successfully salvaged with DLI and/or second allograft. All patients with molecular relapse are alive and support the long-time molecular monitoring in myelofibrosis patients after allogeneic SCT.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2279-2284"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.09.006","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38397750","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 2
Posterior Reversible Encephalopathy Syndrome after Allogeneic Stem Cell Transplantation in Pediatric Patients with Fanconi Anemia, a Prospective Study 儿童范可尼贫血患者异体干细胞移植后后部可逆性脑病综合征的前瞻性研究
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.08.021
Maryam Behfar , Mohammad Babaei , Amir Reza Radmard , Soheil Kooraki , Hamid Farajifard , Parisa Naji , Sahar Taebi , Amir Ali Hamidieh
{"title":"Posterior Reversible Encephalopathy Syndrome after Allogeneic Stem Cell Transplantation in Pediatric Patients with Fanconi Anemia, a Prospective Study","authors":"Maryam Behfar ,&nbsp;Mohammad Babaei ,&nbsp;Amir Reza Radmard ,&nbsp;Soheil Kooraki ,&nbsp;Hamid Farajifard ,&nbsp;Parisa Naji ,&nbsp;Sahar Taebi ,&nbsp;Amir Ali Hamidieh","doi":"10.1016/j.bbmt.2020.08.021","DOIUrl":"10.1016/j.bbmt.2020.08.021","url":null,"abstract":"<div><p>Posterior reversible encephalopathy syndrome (PRES) is one of the most common neurologic complications following hematopoietic stem cell transplantation (HSCT). We aimed to evaluate the incidence, clinical, and imaging features of PRES in pediatric patients with Fanconi anemia (FA) following HSCT. This prospective study included all post-HSCT patients with underlying FA disease between 2014 and 2017. Brain computed tomography scan and magnetic resonance imaging (MRI) were performed in all individuals who developed neurologic symptoms. PRES was diagnosed based on clinic-radiological evidence. Follow-up MRI was performed in all patients with PRES within two months. Forty-one patients with FA (28 males; mean age, 8.19 ± 3.25 years) were enrolled. Out of 15 patients with acute neurologic symptoms, PRES was diagnosed in 9 individuals (21.95% of the total cohort). The occurrence of PRES was significantly higher in patients who had a donor with a 1-locus mismatch (<em>P</em>= .02). Donor relation, stem cell source, and graft-versus-host disease grade did not have any significant association with the development of PRES. MRI showed asymmetric vasogenic edema in 5 patients, an overt infarct in 1 patient, and foci of microhemorrhages in 3 patients, 1 of whom developed a hemorrhagic infarct. This patient died shortly, and persistent microhemorrhages were noted in the other 2 patients. Our findings demonstrate a greater risk of developing PRES after HSCT in patients with FA compared with those with other diseases (21.95% versus 1% to 10%), and in contrast to its term, it might be irreversible and has adverse effects on HSCT outcomes. The increased vascular and endothelial fragility in FA may contribute to the higher frequency of PRES in these individuals.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages e316-e321"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.021","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38323567","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 5
Outcome of Allogeneic Hematopoietic Cell Transplantation after Venetoclax and Hypomethylating Agent Therapy for Acute Myelogenous Leukemia 异基因造血细胞移植治疗急性骨髓性白血病的效果
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.08.027
Karamjeet S. Sandhu, Sanjeet Dadwal, Dongyun Yang, Matthew Mei, Joycelynne Palmer, Amandeep Salhotra, Monzr Al Malki, Ahmed Aribi, Haris Ali, Samer Khaled, Stephen J. Forman, David Snyder, Ryotaro Nakamura, Anthony S. Stein, Guido Marcucci, Ibrahim Aldoss, Vinod Pullarkat
{"title":"Outcome of Allogeneic Hematopoietic Cell Transplantation after Venetoclax and Hypomethylating Agent Therapy for Acute Myelogenous Leukemia","authors":"Karamjeet S. Sandhu,&nbsp;Sanjeet Dadwal,&nbsp;Dongyun Yang,&nbsp;Matthew Mei,&nbsp;Joycelynne Palmer,&nbsp;Amandeep Salhotra,&nbsp;Monzr Al Malki,&nbsp;Ahmed Aribi,&nbsp;Haris Ali,&nbsp;Samer Khaled,&nbsp;Stephen J. Forman,&nbsp;David Snyder,&nbsp;Ryotaro Nakamura,&nbsp;Anthony S. Stein,&nbsp;Guido Marcucci,&nbsp;Ibrahim Aldoss,&nbsp;Vinod Pullarkat","doi":"10.1016/j.bbmt.2020.08.027","DOIUrl":"10.1016/j.bbmt.2020.08.027","url":null,"abstract":"<div><p>The combination of hypomethylating agents with the selective Bcl-2 inhibitor venetoclax (HMA-VEN) has emerged as a highly active regimen in patients with acute myelogenous leukemia (AML) in both the upfront and relapsed/refractory (r/r) settings. We report our early experience with a cohort of patients who were able to proceed to allogeneic hematopoietic cell transplantation (alloHCT) after HMA-VEN therapy. Thirty-two patients with AML (19 r/r and 13 de novo) with a median age of 62 years underwent alloHCT after HMA-VEN therapy. Twenty-two (68.8%) were in complete remission (CR)/CR with incomplete count recovery at time of HCT. With a median follow up of 14.4 months, the 1-year overall survival (OS) was 62.5%, and disease-free survival was 43.8%. The 1-year nonrelapse mortality rate was 18.8%, and the cumulative incidence of relapse was 37.5%. Among patients who underwent alloHCT in CR, the 1-year OS was 77.3%, and the cumulative incidence of nonrelapse mortality was 9.1%. The cumulative incidence of grade II-IV acute graft-versus-host disease was 43.8%. We conclude that alloHCT after HMA-VEN is therapy associated with favorable allogeneic HCT outcomes in newly diagnosed older patients with AML, as well as those with r/r AML.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages e322-e327"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.027","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38327387","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 32
Real-World Issues and Potential Solutions in Hematopoietic Cell Transplantation during the COVID-19 Pandemic: Perspectives from the Worldwide Network for Blood and Marrow Transplantation and Center for International Blood and Marrow Transplant Research Health Services and International Studies Committee COVID-19大流行期间造血细胞移植的现实问题和潜在解决方案:来自全球血液和骨髓移植网络和国际血液和骨髓移植研究中心卫生服务和国际研究委员会的观点
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.07.021
Ghada Algwaiz , Mahmoud Aljurf , Mickey Koh , Mary M. Horowitz , Per Ljungman , Daniel Weisdorf , Wael Saber , Yoshihisa Kodera , Jeff Szer , Dunia Jawdat , William A. Wood , Ruta Brazauskas , Leslie Lehmann , Marcelo C. Pasquini , Adriana Seber , Pei Hua Lu , Yoshiko Atsuta , Marcie Riches , Miguel-Angel Perales , Nina Worel , Shahrukh K. Hashmi
{"title":"Real-World Issues and Potential Solutions in Hematopoietic Cell Transplantation during the COVID-19 Pandemic: Perspectives from the Worldwide Network for Blood and Marrow Transplantation and Center for International Blood and Marrow Transplant Research Health Services and International Studies Committee","authors":"Ghada Algwaiz ,&nbsp;Mahmoud Aljurf ,&nbsp;Mickey Koh ,&nbsp;Mary M. Horowitz ,&nbsp;Per Ljungman ,&nbsp;Daniel Weisdorf ,&nbsp;Wael Saber ,&nbsp;Yoshihisa Kodera ,&nbsp;Jeff Szer ,&nbsp;Dunia Jawdat ,&nbsp;William A. Wood ,&nbsp;Ruta Brazauskas ,&nbsp;Leslie Lehmann ,&nbsp;Marcelo C. Pasquini ,&nbsp;Adriana Seber ,&nbsp;Pei Hua Lu ,&nbsp;Yoshiko Atsuta ,&nbsp;Marcie Riches ,&nbsp;Miguel-Angel Perales ,&nbsp;Nina Worel ,&nbsp;Shahrukh K. Hashmi","doi":"10.1016/j.bbmt.2020.07.021","DOIUrl":"10.1016/j.bbmt.2020.07.021","url":null,"abstract":"<div><p>The current COVID-19 pandemic, caused by SARS-CoV-2, has impacted many facets of hematopoietic cell transplantation (HCT) in both developed and developing countries. Realizing the challenges as a result of this pandemic affecting the daily practice of the HCT centers and the recognition of the variability in practice worldwide, the Worldwide Network for Blood and Marrow Transplantation (WBMT) and the Center for International Blood and Marrow Transplant Research's (CIBMTR) Health Services and International Studies Committee have jointly produced an expert opinion statement as a general guide to deal with certain aspects of HCT, including diagnostics for SARS-CoV-2 in HCT recipient, pre- and post-HCT management, donor issues, medical tourism, and facilities management. During these crucial times, which may last for months or years, the HCT community must reorganize to proceed with transplantation activity in those patients who urgently require it, albeit with extreme caution. This shared knowledge may be of value to the HCT community in the absence of high-quality evidence-based medicine.</p><p>© 2020 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2181-2189"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.021","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38205684","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 40
Impact of Donor Source on Allogeneic Hematopoietic Stem Cell Transplantation for Mature T Cell and Natural Killer Cell Neoplasms in the Kyoto Stem Cell Transplantation Group 供体来源对京都干细胞移植组成熟T细胞和自然杀伤细胞肿瘤同种异体造血干细胞移植的影响
IF 4.3
Biology of Blood and Marrow Transplantation Pub Date : 2020-12-01 DOI: 10.1016/j.bbmt.2020.07.032
Mizuki Watanabe , Junya Kanda , Yasuyuki Arai , Masakatsu Hishizawa , Momoko Nishikori , Takayuki Ishikawa , Kazunori Imada , Yasunori Ueda , Takashi Akasaka , Akihito Yonezawa , Masaharu Nohgawa , Toshiyuki Kitano , Mitsuru Itoh , Tomoharu Takeoka , Toshinori Moriguchi , Kazuhiro Yago , Nobuyoshi Arima , Naoyuki Anzai , Mitsumasa Watanabe , Tadakazu Kondo , Akifumi Takaori-Kondo
{"title":"Impact of Donor Source on Allogeneic Hematopoietic Stem Cell Transplantation for Mature T Cell and Natural Killer Cell Neoplasms in the Kyoto Stem Cell Transplantation Group","authors":"Mizuki Watanabe ,&nbsp;Junya Kanda ,&nbsp;Yasuyuki Arai ,&nbsp;Masakatsu Hishizawa ,&nbsp;Momoko Nishikori ,&nbsp;Takayuki Ishikawa ,&nbsp;Kazunori Imada ,&nbsp;Yasunori Ueda ,&nbsp;Takashi Akasaka ,&nbsp;Akihito Yonezawa ,&nbsp;Masaharu Nohgawa ,&nbsp;Toshiyuki Kitano ,&nbsp;Mitsuru Itoh ,&nbsp;Tomoharu Takeoka ,&nbsp;Toshinori Moriguchi ,&nbsp;Kazuhiro Yago ,&nbsp;Nobuyoshi Arima ,&nbsp;Naoyuki Anzai ,&nbsp;Mitsumasa Watanabe ,&nbsp;Tadakazu Kondo ,&nbsp;Akifumi Takaori-Kondo","doi":"10.1016/j.bbmt.2020.07.032","DOIUrl":"10.1016/j.bbmt.2020.07.032","url":null,"abstract":"<div><p>Although allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the key strategy to cure patients with mature T and natural killer (NK) cell lymphomas/leukemia, especially those with relapsed/refractory diseases, there is no consensus strategy for donor selection. We retrospectively analyzed the outcomes of allo-HSCT in 111 patients in 15 Japanese institutions as a multi-institutional joint research project. Thirty-nine patients received bone marrow or peripheral blood stem cell transplantation from related donors (rBMT/rPBSCT), 37 received BMT/PBSCT from unrelated donors (uBMT/uPBSCT), and 35 received cord blood transplantation (CBT). Overall survival (OS) and progression-free survival (PFS) at 4 years were 42% and 34%, respectively. The cumulative incidences of relapse and nonrelapse mortality were 43% and 25%. In multivariate analysis, CBT showed comparable OS with rBMT/rPBSCT (rBMT/rPBSCT versus CBT: hazard ratio [HR], 1.63; <em>P</em> = .264) and better OS compared with uBMT/uPBSCT (HR, 2.99; <em>P</em> = .010), with a trend toward a lower relapse rate (rBMT/rPBSCT versus CBT: HR, 2.60; <em>P</em> = .010; uBMT/uPBSCT versus CBT: HR, 2.05; <em>P</em> = .082). This superiority of CBT was more definite in on-disease patients (OS: rBMT/rPBSCT versus CBT: HR, 5.52; <em>P</em> = .021; uBMT/uPBSCT versus CBT: HR, 6.80; <em>P</em> = .007). Better disease control was also strongly associated with better OS and PFS with lower relapse rate. In conclusion, allo-HSCT is beneficial for the survival of patients with mature T and NK cell lymphomas/leukemia if performed in a timely fashion. Since CBT showed favorable survival with a lower relapse risk, it could be a preferred alternative, especially in on-disease patients.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2346-2358"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.07.032","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38224691","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 3
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