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Biology of Blood and Marrow Transplantation

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https://www.readbyqxmd.com/read/28647558/durable-chimerism-and-long-term-survival-following-unrelated-umbilical-cord-blood-transplantation-for-pediatric-hemophagocytic-lymphohistiocytosis-a-single-center-experience
#1
Sachit A Patel, Heather A Allewelt, Jesse D Troy, Paul L Martin, Timothy A Driscoll, Vinod K Prasad, Joanne Kurtzberg, Kristin M Page, Suhag H Parikh
Hemophagocytic Lymphohistiocytosis (HLH) is a life-threatening disorder of immune dysregulation characterized by fever, hepatosplenomegaly, cytopenias, CNS disease, increased inflammatory markers and hemophagocytosis. Currently, allogeneic hematopoietic stem cell transplantation is the only curative approach for patients with HLH with reported survival ranging from 50-70% with myeloablative conditioning (MAC) regimens. However, donor availability and transplant related mortality associated with conventional MAC are major barriers to success...
June 21, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28642072/recombinant-human-thrombopoietin-promotes-platelet-engraftment-and-improves-prognosis-of-patients-with-myelodysplastic-syndromes-and-aplastic-anemia-after-allogeneic-hematopoietic-stem-cell-transplantation
#2
Hong Wang, Man Huang, Ying Zhao, Jia-Qian Qi, Chun Chen, Ya-Qiong Tang, Hui-Ying Qiu, Cheng-Cheng Fu, Xiao-Wen Tang, De-Pei Wu, Chang-Geng Ruan, Yue Han
Poor platelet graft function (PPGF) is a significant complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, no optimal treatment has been recommended. This study investigated aspects of platelet recovery after allo-HSCT, including prognostic value and the effect of recombinant human thrombopoietin (rhTPO). We retrospectively analyzed 275 patients who received allo-HSCT in our center. Of them, 135 (49.1%) patients had good platelet graft function (GPGF) and 140 (50.9%) had PPGF...
June 19, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28624476/measuring-immune-response-to-commonly-used-vaccinations-in-adult-recipients-of-allogeneic-hematopoietic-cell-transplantation
#3
REVIEW
Lucy Boyce Kennedy, Zhuoyan Li, Bipin N Savani, Per Ljungman
Recipients of hematopoietic cell transplantation are at risk for potentially preventable infectious complications due to defects in humoral and cell-mediated immunity. Studies of vaccine immunogenicity in HCT recipients have shown that antibody response rates depend on age, the type of vaccine, and presence or absence of GVHD. However, there are few large-scale studies assessing the immune response to vaccination in HCT recipients. Additionally, HCT recipients have much higher rates of potentially preventable infections compared to the general population even after vaccination...
June 14, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28627425/a-multicenter-retrospective-analysis-stressing-importance-of-long-term-follow-up-after-hematopoietic-cell-transplantation-for-%C3%AE-thalassemia
#4
S Chaudhury, M Ayas, C Rosen, M Ma, M Viqaruddin, S Parikh, S Kharbanda, K Y Chiang, A Haight, M Bhatia, Gmt Guilcher, A Thompson, S Shenoy
Allogeneic hematopoietic cell transplantation (HCT) is curative in patients with β-thalassemia major. However, the majority of reports on HCT outcomes lack long-term follow-up data with the exception of single center reports. An international multicenter retrospective data collection and analysis was conducted in 176 β-Thalassemia patients who were 1 year or beyond after first HCT to evaluate follow up methods and outcomes at 7 centers- Median age at HCT was 5.5 years (range, 0.6- 18.5) and median follow-up was 7 years (range, 1-20)...
June 13, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28619667/taking-the-good-with-the-bad-survival-and-late-toxicity-among-young-hematopoietic-cell-transplantation-survivors
#5
Lucie M Turcotte
No abstract text is available yet for this article.
June 12, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28606646/current-use-and-trends-in-hematopoietic-cell-transplantation-in-the-united-states
#6
EDITORIAL
Anita D'Souza, Stephanie Lee, Xiaochun Zhu, Marcelo Pasquini
Hematopoietic cell transplantation (HCT) is an established curative treatment for a number of malignant and non-malignant diseases involving the hematopoietic system and some solid tumors. In this report, we provide information about the number of HCT procedures performed in the United States (US) in 2015 and analyze trends and outcomes of HCT as reported to the Center for International Blood and Marrow Transplant Research(®) (CIBMTR(®)). We show that the numbers of HCT performed annually continue to increase, the indications for HCT, preferred donor sources and GVHD prophylaxis continue to evolve...
June 9, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28603071/evaluation-of-performance-status-and-hematopoietic-cell-transplantation-specific-co-morbidity-index-on-unplanned-admission-rates-in-patients-with-multiple-myeloma-undergoing-outpatient-autologous-stem-cell-transplantation
#7
C Obiozor, D P Subramaniam, C Divine, L Shune, A K Singh, T L Lin, S Abhyankar, G J Chen, J McGuirk, S Ganguly
Although, outpatient autologous stem cell transplant (ASCT) is safe and feasible in most instances, some patients undergoing planned outpatient transplantation for multiple myeloma (MM) will need inpatient admission for transplant related complications. We aim to evaluate the difference, if any, between outpatient and inpatient ASCT cohorts of MM patients in terms of admission rate, transplant outcome, and overall survival. We also plan to assess if hematopoietic cell transplantation-comorbidity index (HCT-CI) and Karnofsky Performance Status (KPS) could predict unplanned admissions after adjusting for confounding factors...
June 8, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28603070/clinical-utility-of-quantitative-pcr-for-chimerism-and-engraftment-monitoring-after-allogeneic-stem-cell-transplantation-for-hematologic-malignancies
#8
Müberra Ahci, Karin Stempelmann, Ulrike Buttkereit, Pietro Crivello, Mirko Trilling, Andreas Heinold, Nina Kristin Steckel, Michael Koldehoff, Peter A Horn, Dietrich W Beelen, Katharina Fleischhauer
Although quantitative PCR (qPCR) has been explored for chimerism monitoring after allogeneic stem cell transplantation (SCT), evidence regarding its clinical utility compared to standard short tandem repeat (STR) is still limited. We retrospectively studied commercial qPCR and STR chimerism with respective positivity thresholds of 0.1% and 1%, in 359 peripheral blood (PB) and 95 bone marrow (BM) samples from 30 adult patients after first HLA-matched SCT for myeloid malignancies or acute lymphatic leukemia. Concordance between the two methods was 79...
June 8, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28603069/the-case-for-high-resolution-extended-6-loci-hla-typing-for-identifying-related-donors-in-the-indian-subcontinent
#9
Rajat Kumar Agarwal, Ankita Kumari, Amit Sedai, Lalith Parmar, Rakesh Dhanya, Lawrence Faulkner
Three-loci low- or intermediate-resolution HLA typing is generally considered adequate in the related blood and marrow transplantation (BMT) context. However, a single high-resolution (HR) mismatch may have a similar adverse impact on BMT outcome as a low resolution (LR) one. We sought to determine the frequency of mismatches which may go undetected when standard typing (LR or HR/3-loci) is employed as against HR/6-loci typing for related donors compatibility testing and assess its impact on relevant BMT outcomes...
June 8, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28603068/genetic-effects-on-hematopoietic-stem-cell-transplant-prognosis-and-outcomes-more-than-just-histocompatibility
#10
EDITORIAL
Jai N Patel, Lawrence J Druhan
No abstract text is available yet for this article.
June 8, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28602958/allogeneic-hematopoietic-cell-transplantation-using-treosulfan-based-conditioning-for-treatment-of-marrow-failure-disorders
#11
Lauri M Burroughs, Akiko Shimamura, Julie-An Talano, Jennifer Domm, Kelsey Baker, Colleen Delaney, Haydar Frangoul, David Margolis, K Scott Baker, Amy Geddis, Brenda M Sandmaier, H Joachim Deeg, Rainer Storb, Ann E Woolfrey
Hematopoietic cell transplantation (HCT) is effective in the treatment of inherited marrow failure disorders and other non-malignant diseases. Conventional myeloablative conditioning regimens have been associated with high transplant related mortality particularly in patients with co-morbid conditions. Here we report on 14 patients with marrow failure disorders (Shwachman-Diamond syndrome n=3, Diamond Blackfan anemia n=4, GATA2 deficiency n=2, paroxysmal nocturnal hemoglobinuria n=4, and an undefined marrow failure disorder n=1) who underwent HCT on a prospective phase II multi-center clinical trial...
June 7, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28600031/hematopoietic-cell-transplantation-in-myelodysplastic-syndromes-after-treatment-with-hypomethylating-agents
#12
Moreno Festuccia, Kelsey Baker, Theodore A Gooley, Brenda M Sandmaier, H Joachim Deeg, Bart L Scott
The prognosis of patients with myelodysplastic syndromes (MDS) following failure of hypomethylating agent (HMA) therapy is poor. Allogeneic hematopoietic cell transplantation (HCT) can be effective in curing patients who have failed therapy with HMA. However, published results have not addressed the outcomes with HCT in this setting. We identified 125 MDS patients who had been treated with HMA and underwent subsequent HCT. Among these, 68 were considered HMA failures, and 57 responders. Failure was defined as progression to higher grade MDS or acute myeloid leukemia (AML), lack of hematological improvement after at least 4 HMA cycles, or loss of response after initial improvement...
June 6, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28602892/cell-based-therapy-using-umbilical-cord-blood-for-novel-indications-in-regenerative-therapy-and-immune-modulation-an-updated-systematic-scoping-review-of-the-literature
#13
REVIEW
Mina Rizk, Joseph Aziz, Risa Shorr, David S Allan
BACKGROUND: Cell-based therapy using umbilical cord blood is used increasingly for novel applications. To balance heightened public expectations and ensure appropriateness of emerging cell-based treatment choices, regular evidence-based assessment of novel cord blood-derived therapies is needed. METHODS AND RESULTS: We performed a systematic search of the literature and identified 57 studies (814 patients) for analysis. 16 studies (353 patients) included control groups for comparison...
June 5, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28602891/manufacture-of-autologous-cd34-selected-grafts-in-the-niaid-sponsored-halt-ms-and-scot-multicenter-clinical-trials-for-autoimmune-diseases
#14
Carolyn A Keever-Taylor, Shelly Heimfeld, Kaitlyn C Steinmiller, Richard A Nash, Keith M Sullivan, Christine W Czarniecki, Tomeka C Granderson, Julia S Goldstein, Linda M Griffith
To ensure comparable grafts for autologous HCT in NIAID-sponsored IND protocols for multiple sclerosis (HALT-MS) and systemic sclerosis (SCOT), a DMF approach to control manufacture was implemented, including a common Master Production Batch Record, and site-specific SOPs with "Critical Elements". We assessed comparability of flow cytometry and controlled rate cryopreservation among sites, and stability of cryopreserved grafts, using hematopoietic progenitor cells (HPC) from healthy donors. Auto-CD34+HPC graft specifications included ≥70% viable CD34+ cells before cryopreservation...
June 5, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28602890/posterior-reversible-encephalopathy-syndrome-after-hematopoietic-cell-transplantation-in-children-with-hemoglobinopathies
#15
Javid Gaziev, Simone Marziali, Katia Paciaroni, Antonella Isgrò, Francesca Di Giuliano, Giorgia Rossi, Marco Marziali, Gioia De Angelis, Cecilia Alfieri, Michela Ribersani, Marco Andreani, Maria Giuseppina Palmieri, Fabio Placidi, Andrea Romigi, Francesca Izzi, Roberto Floris, Nicola Biagio Mercuri
Posterior reversible encephalopathy syndrome (PRES) is a serious adverse event associated with calcineurin inhibitors (CI) used for graft-versus-host disease (GVHD) prophylaxis. We compared the incidence of PRES in children with thalassemia (n=222, 1.4-17.8 years old) versus sickle cell disease (SCD; n=59, 2-17 years old) who underwent hematopoietic cell transplantation (HCT) from HLA-matched siblings or alternative donors and analyzed the risk factors for PRES. Overall, 31 children developed CI-related PRES (11%), including 30 patients with seizures and 1 patient without seizures...
June 5, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28578010/unrelated-umbilical-cord-blood-transplantation-for-sickle-cell-disease-following-reduced-intensity-conditioning-results-of-a-phase-i-trial
#16
Allistair Abraham, Andrew Cluster, David Jacobsohn, David Delgado, Monica L Hulbert, Divyesh Kukadiya, Lisa Murray, Shalini Shenoy
Hematopoietic stem cell transplantation from HLA-matched sibling donors results in disease free survival of >90% in patients with sickle cell disease (SCD) but only about 18% have suitable donors. Unrelated cord blood transplantation (UCBT) is one way to expand donor options for patients with severe SCD but historically has been associated with high graft rejection rates (50-62%). We hypothesized that the addition of thiotepa to a previously tested reduced intensity conditioning (RIC) regimen would support engraftment after UCBT in SCD patients...
May 31, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28578009/lessons-learned-from-early-experiences-with-vedolizumab-for-steroid-refractory-acute-graft-versus-host-disease-with-gastrointestinal-involvement
#17
LETTER
Adomas Bukauskas, Laimonas Griskevicius, Valdas Peceliunas
No abstract text is available yet for this article.
May 31, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28554856/beneficial-role-of-low-dose-atg-in-unrelated-stem-cell-transplantation-for-adult-patients-with-acquired-severe-aplastic-anemia-reduction-of-graft-versus-host-disease-gvhd-and-improvement-of-gvhd-free-failure-free-survival-rate
#18
Sung-Soo Park, Dae Hun Kwak, Young-Woo Jeon, Jae-Ho Yoon, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Seok Lee, Chang-Ki Min, Seok-Goo Cho, Dong-Wook Kim, Woo-Sung Min, Jong Wook Lee
Stem cell transplantation from an unrelated donor (URD SCT) is often considered in patients with severe aplastic anemia (SAA) whom immunosuppressive therapy failed and matched sibling donor is not available. To reduce the incidence of graft-versus-host disease (GVHD) in URD SCT, introduction of antithymocyte globulin (ATG) to the conditioning regimen has been proposed. Although ATG was shown to play a role in reducing GVHD in a cohort with diverse hematologic diseases, its role in SAA remains uncertain. The aim of this study was to determine the efficacy and toxicity of ATG in URD SCT for adult patients with SAA...
May 26, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28552421/reduced-intensity-conditioning-with-busulfan-fludarabine-and-antithymocyte-globulin-for-hematopoietic-cell-transplantation-from-unrelated-or-haploidentical-family-donors-in-patients-with-acute-myeloid-leukemia-in-remission
#19
Kyoo-Hyung Lee, Je-Hwan Lee, Jung-Hee Lee, Dae-Young Kim, Han-Seung Park, Eun-Ji Choi, Sun-Hye Ko, Miee Seol, Young-Shin Lee, Young-A Kang, Mijin Jeon, Seunghyun Baek, You-Lee Kang, Sung-Han Kim, Sung-Cheol Yun, Hawk Kim, Jae-Cheol Jo, Yunsuk Choi, Young-Don Joo, Sung-Nam Lim
To investigate the role of antithymocyte globulin (ATG)-containing reduced-intensity conditioning (RIC) in hematopoietic cell transplantation (HCT) from unrelated (UD) or haploidentical family donors (HFD), we conducted a phase 2 trial of 237 patients (age range, 16 to 69 years) with acute myeloid leukemia (AML) in remission. Patients undergoing UD-HCT (n = 93) or HFD-HCT (n = 59) received RIC comprising busulfan, fludarabine, and ATG, 9 mg/kg, whereas those undergoing HCT from matched sibling donors (MSD, n = 85) received myeloablative busulfan and cyclophosphamide conditioning or aforementioned RIC with ATG, 4...
May 25, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28549771/calcineurin-and-mtor-inhibitor-free-post-transplantation-cyclophosphamide-and-bortezomib-combination-for-graft-versus-host-disease-prevention-after-peripheral-blood-allogeneic-hematopoietic-stem-cell-transplantation-a-phase-i-ii-study
#20
A Samer Al-Homsi, Kelli Cole, Marlee Muilenburg, Austin Goodyke, Muneer Abidi, Ulrich Duffner, Stephanie Williams, Jessica Parker, Aly Abdel-Mageed
Graft-versus-host disease (GVHD) hampers the utility of allogeneic hematopoietic stem cell transplantation (AHSCT). The purpose of this study was to determine the feasibility, safety, and efficacy of a novel combination of post-transplantation cyclophosphamide (PTC) and bortezomib for the prevention of GVHD. Patients undergoing peripheral blood AHSCT for hematological malignancies after reduced-intensity conditioning with grafts from HLA-matched related or unrelated donors were enrolled in a phase I/II clinical trial...
May 23, 2017: Biology of Blood and Marrow Transplantation
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