Kathleen Monahan , Ariel Kleman , Bicky Thapa , Aniko Szabo , Anita D'Souza , Binod Dhakal , James H. Jerkins , Marcelo C. Pasquini , Mehdi Hamadani , Parameswaran N. Hari , Saurabh Chhabra
{"title":"Propylene Glycol-Free Melphalan versus PG-Melphalan as Conditioning for Autologous Hematopoietic Cell Transplantation for Myeloma","authors":"Kathleen Monahan , Ariel Kleman , Bicky Thapa , Aniko Szabo , Anita D'Souza , Binod Dhakal , James H. Jerkins , Marcelo C. Pasquini , Mehdi Hamadani , Parameswaran N. Hari , Saurabh Chhabra","doi":"10.1016/j.bbmt.2020.08.030","DOIUrl":"10.1016/j.bbmt.2020.08.030","url":null,"abstract":"<div><p>High-dose melphalan (Mel) conditioning before autologous hematopoietic cell transplantation (autoHCT) is standard of care for patients with transplantation-eligible multiple myeloma. The traditional lyophilized Mel formulation has inadequate solubility and stability after reconstitution, leading to the use of propylene glycol (PG) as a solubilizing agent. A newer PG-free Mel preparation (Evomela) uses beta cyclodextrin captisol as a solubilizing agent and was approved by the United States Food and Drug Administration as a conditioning agent based on a single-phase IIb study showing bioequivalence. We compared the outcomes of consecutive patients with myeloma undergoing autoHCT using the 2 formulations of Mel for conditioning as our center switched from using the older formulation (PG-Mel) to the newer one (PGF-Mel). Of 294 autoHCT recipients, 162 received PG-Mel conditioning and 132 received PGF-Mel conditioning. The PGF-Mel group was older and had a lower average Karnofsky Performance Status score. PGF-Mel was associated with faster neutrophil recovery (median, 12 days versus 13 days; <em>P</em> < .001), fewer grade 3-4 infections within 30 days of autoHCT (1.5% versus 8.0%; <em>P</em> = .048), and a lower 30-day rehospitalization rate (6.8% versus 17.9%; <em>P</em> = .04), as confirmed by propensity-weighted analysis. No significant between-group differences were detected in mucositis, organ toxicity, myeloma response, or 100-day mortality.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2229-2236"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.030","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38372974","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}
Catalina Montes de Oca , Luca Castagna , Chiara De Philippis , Stefania Bramanti , Jean Marc Schiano , Thomas Pagliardini , Aude Collignon , Samia Harbi , Jacopo Mariotti , Angela Granata , Valerio Maisano , Sabine Furst , Faezeah Legrand , Christian Chabannon , Carmelo Carlo-Stella , Armando Santoro , Didier Blaise , Raynier Devillier
{"title":"Nonmyeloablative Conditioning Regimen before T Cell Replete Haploidentical Transplantation with Post-Transplant Cyclophosphamide for Advanced Hodgkin and Non-Hodgkin Lymphomas","authors":"Catalina Montes de Oca , Luca Castagna , Chiara De Philippis , Stefania Bramanti , Jean Marc Schiano , Thomas Pagliardini , Aude Collignon , Samia Harbi , Jacopo Mariotti , Angela Granata , Valerio Maisano , Sabine Furst , Faezeah Legrand , Christian Chabannon , Carmelo Carlo-Stella , Armando Santoro , Didier Blaise , Raynier Devillier","doi":"10.1016/j.bbmt.2020.08.014","DOIUrl":"10.1016/j.bbmt.2020.08.014","url":null,"abstract":"<div><p>Allogeneic hematopoietic stem cell transplantation (allo-SCT) is a valid option in patients with refractory lymphomas. HLA haploidentical stem cell transplantation (haplo-SCT) expanded the accessibility to allogeneic hematopoietic cell transplantation. The aims of study were to retrospectively assess the toxicity and efficacy of haplo-SCT using nonmyeloablative conditioning in patients with advanced lymphoma. In total, 147 patients with advanced lymphoma at 2 partner institutions were included. Patients received a uniform nonmyeloablative conditioning regimen and graft-versus-host disease (GVHD) prophylaxis. The primary endpoints were progression-free survival (PFS), overall survival (OS), GVHD, nonrelapse mortality, and GVHD, relapse-free survival (GRFS). Median follow-up was 39 months (range, 6 to 114 months). The median age was 46 years (range, 19 to 71 years). Sixty-five percent of patients were in complete remission (CR) at transplantation. Cumulative incidence of grade II to IV acute GVHD was 30% (95% confidence interval [Cl], 23% to 38%). Two-year cumulative incidence of all grades of chronic GVHD was 13% (95% CI, 8% to 20%). Two-year cumulative incidence of disease relapse was 19% (95% CI, 14% to 27%), with a higher incidence in patients not being in CR at allo-HCT (CR versus not CR: 12% versus 33%, <em>P</em> = .006). Two-year PFS, OS, and GRFS were 66% (95% CI, 59-75), 73% (95% CI, 66-81), and 56% (95% CI, 48-65), respectively. Haplo-SCT with post-transplantation cyclophosphamide may be considered a valid option for patients with aggressive lymphoma and deserves further evaluation.</p></div>","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Pages 2299-2305"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.bbmt.2020.08.014","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38296340","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}
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 , 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","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> < .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> < .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}
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 , Maximilian Christopeit , Francis Ayuk , Gaby Zeck , Christine Wolschke , 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}
{"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}
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 , 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","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}
{"title":"Masthead (Purpose and Scope)","authors":"","doi":"10.1016/S1083-8791(20)30703-5","DOIUrl":"https://doi.org/10.1016/S1083-8791(20)30703-5","url":null,"abstract":"","PeriodicalId":9165,"journal":{"name":"Biology of Blood and Marrow Transplantation","volume":"26 12","pages":"Page A1"},"PeriodicalIF":4.3,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/S1083-8791(20)30703-5","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136815785","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}
{"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 , 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","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}
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 , Mohammad Babaei , Amir Reza Radmard , Soheil Kooraki , Hamid Farajifard , Parisa Naji , Sahar Taebi , 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}