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allogeneic hematopoietic stem cell transplantation

Lukas Mayerhoff, Moritz Lehne, Lennart Hickstein, Tayyab Salimullah, Sigurd Prieur, Simu K Thomas, Jie Zhang
AIM: Quantify hematopoietic stem cell transplantation (HSCT) costs in German patients with acute lymphoblastic leukemia (ALL), diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL). METHODS: The primary outcome was direct and indirect costs in patients with ALL/DLBCL/FL who received HSCT between 2010 and 2014. Costs were evaluated two to four quarters before to eight quarters after HSCT. RESULTS: Among 258 patients with HSCT, direct costs were €290,125/patient (pediatric ALL), €246,266/patient (adult ALL), €230,399/patient (DLBCL/FL allogeneic) and €107,457/patient (DLBCL/FL autologous)...
December 5, 2018: Journal of Comparative Effectiveness Research
Kerstin Rauwolf, Heidi Herbrüggen, Stefan Zöllner, Heike Thorer, Olga Makarova, Thomas Kaiser, Aleksandra Pettke, Claudia Rossig, Birgit Burkhardt, Andreas H Groll
Chronic hepatitis C virus (HCV) infection carries increased risks for morbidity and mortality in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) but has become curable through the advent of directly acting antiviral compounds. Current guidelines of the American Society for Blood and Marrow Transplantation (ASBMT) recommend that HCV-infected HSCT candidates preferably start and complete therapy prior to transplant. However, this is often not feasible due to time constraints or treatment limiting comorbidities, conditions and treatments...
December 5, 2018: Journal of Viral Hepatitis
Jing Liu, Xiao-Su Zhao, Yan-Rong Liu, Lan-Ping Xu, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, Qiao-Zhen Fan, Xiao-Jun Huang, Ying-Jun Chang
Background: Several studies have shown that detection of minimal residual disease (MRD) in acute myeloid leukemia (AML) is an independent prognostic factor. This study aimed to evaluate the significance of dynamic MRD pretransplantation on outcome of AML patients receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT). Methods: We retrospectively analyzed 145 consecutive AML patients undergoing allo-HSCT in complete remission status between June 2013 and June 2016...
December 5, 2018: Chinese Medical Journal
Adam W Bartlett, Megan P Cann, Daniel K Yeoh, Anne Bernard, Anne L Ryan, Christopher C Blyth, Rishi S Kotecha, Brendan J McMullan, Andrew S Moore, Gabrielle M Haeusler, Julia E Clark
BACKGROUND: A thorough understanding of local and contemporary invasive fungal infection (IFI) epidemiology in immunocompromised children is required to provide a rationale for targeted prevention and treatment strategies. METHODS: Retrospective data over 10 years from four tertiary pediatric oncology and hematopoietic stem cell transplant (HSCT) units across Australia were analyzed to report demographic, clinical, and mycological characteristics of IFI episodes, and crude IFI prevalence in select oncology/HSCT groups...
December 4, 2018: Pediatric Blood & Cancer
Chan-Young Ock, Heewon Seo, Dae-Yoon Kim, Byung Joo Min, Yoomi Park, Hyun Sub Cheong, Hyung-Lae Kim, Eun-Young Song, Inho Kim, Sung-Soo Yoon, Ju Han Kim, Youngil Koh
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been the only treatment option for acute myeloid leukemia (AML) refractory to induction chemotherapy, with only 10-20% of patients achieving long-term survival. Certain donor genotypes may confer leukemia-clearing effects after allo-HSCT. We performed whole-exome sequencing of five pairs of the germ lines in AML patients who achieved long-term remission after allo-HSCT and in their donors, and found two significant variants: EGFR c.2982C > T and CDH11 c...
December 3, 2018: Leukemia & Lymphoma
Juliane Grimm, Marius Bill, Madlen Jentzsch, Stefanie Beinicke, Janine Häntschel, Karoline Goldmann, Julia Schulz, Michael Cross, Georg-Nikolaus Franke, Gerhard Behre, Vladan Vucinic, Wolfram Pönisch, Thoralf Lange, Dietger Niederwieser, Sebastian Schwind
Age-related somatic mutations linked to clonal hematopoiesis have been found in apparently healthy individuals and increase the risk of developing hematologic malignancies. In acute myeloid leukemia (AML) the clinical relevance of clonal hematopoiesis remains controversial and data on patients with detectable clonal hematopoiesis, consolidated with hematopoietic stem cell transplantation are limited. We analyzed samples from 113 AML patients in complete remission prior to hematopoietic stem cell transplantation for the presence of clonal hematopoiesis-associated mutations...
November 30, 2018: Bone Marrow Transplantation
Andrea Bacigalupo
Allogeneic hematopoietic stem-cell transplantation remains the only curative treatment for patients with acquired severe aplastic anemia (SAA). When a matched sibling is not available, one can search for a matched unrelated donor or a cord blood unit (CB) in the international registries or, more recently, for an HLA haploidentical (HAPLO) family member. International guidelines call for a course of antithymocyte globulin (ATG) and cyclosporine before a patient with SAA receives a transplant from a donor other than an HLA identical sibling, but whether this is necessary for patients age <20 years is less clear...
November 30, 2018: Hematology—the Education Program of the American Society of Hematology
Charlotte M Niemeyer
Juvenile myelomonocytic leukemia (JMML) is a unique clonal hematopoietic disorder of early childhood characterized by hyperactivation of the RAS signal transduction pathway. Approximately 90% of patients harbor molecular alteration in 1 of 5 genes ( PTPN11, NRAS, KRAS, NF1, CBL ), which define genetically and clinically distinct JMML subtypes. Three subtypes, PTPN11- , NRAS- , and KRAS -mutated JMML, are characterized by heterozygous somatic gain-of-function mutations in non syndromic children, while two subtypes, JMML in neurofibromatosis type 1 and in JMML in children with CBL syndrome, are characterized by germ line RAS disease and acquired biallelic inactivation of the respective tumor suppressor genes in hematopoietic cells...
November 30, 2018: Hematology—the Education Program of the American Society of Hematology
Betty Ky Hamilton
Allogeneic hematopoietic cell transplantation is a potentially curative therapy for many malignant and nonmalignant hematologic diseases. Graft-versus-host disease (GVHD) is a common complication after transplantation and remains a major cause of morbidity and mortality, limiting the success of a potentially curative transplant. This paper reviews the current and emerging strategies in GVHD prevention and treatment. New insights are leading the way to the development of novel targeted approaches to minimize the risk of disease relapse and infection...
November 30, 2018: Hematology—the Education Program of the American Society of Hematology
Julius C Fischer, Chia-Ching Lin, Simon Heidegger, Alexander Wintges, Martin Schlapschy, Matthias Beudert, Stephanie E Combs, Florian Bassermann, Arne Skerra, Tobias Haas, Hendrik Poeck
PURPOSE: Type I interferon (IFN-I) and IL-22 modulate regeneration of the thymus and intestinal epithelial cells (IECs) after cytotoxic stress like irradiation. Radiation-induced damage to thymic tissues and IECs is a crucial aspect during the pathogenesis of inadequate immune reconstitution and acute graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) with myeloablative total body irradiation (TBI), respectively. IL-22 and IFN-I reduce severity of acute GVHD after allo-HSCT with myeloablative TBI...
November 29, 2018: International Journal of Radiation Oncology, Biology, Physics
Annefleur C Langedijk, Mariëlle van Aalst, Bob Meek, Ester M M van Leeuwen, Sacha Zeerleder, Ellen Meijer, Mette D Hazenberg, Martin P Grobusch, Abraham Goorhuis
Infection with Streptococcus pneumoniae is a life-threatening, but vaccine preventable complication in patients with allogeneic hematopoietic stem cell transplantation (allo-HSCT). The international consensus on post allo-HSCT immunization schedules, starting 3-6 months after HSCT, focuses on short-term immunogenicity while long-term immunogenicity is not well characterized. The current Dutch immunization schedule, which starts at 12 months post allo-HSCT, was developed as a result of concerns on the coverage of long-term immunogenicity in international guidelines...
November 27, 2018: Vaccine
Dong Wang, Feng Zhu, Kai-Lin Xu
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been the only method for curing many hematopoietic benign or malignant diseases, from which a number of patients obtain long-term survival. However, graft versus host disease (GVHD) deeply hinders the development and clinical use of HSCT. Up to now, immune response triggered by donor's T cell has been considered to be the pathogenesis of GVHD. On the other side, donor's T cell has indispensable effect in competing with the tumor cells and reducing the disease recurrence rate...
December 2018: Zhongguo Shi Yan Xue Ye Xue za Zhi
Mahmoud Elsawy, Barry E Storer, Filippo Milano, Brenda M Sandmaier, Colleen Delaney, Rachel B Salit, Ahmed H Rashad, Ann E Woolfrey, Frederick R Appelbaum, Rainer Storb, Mohamed L Sorror
The Hematopoietic Cell Transplantation-specific Comorbidity Index (HCT-CI) was developed and validated to weigh the burden of pretransplant comorbidities and estimate their impact on post-transplant risks of non-relapse mortality (NRM). Recently, the HCT-CI was augmented by the addition of both age and values of three markers namely, ferritin, albumin, and platelet count. The HCT-CI research so far has been almost exclusively limited to recipients of allogeneic HCT from human leucocyte antigen (HLA)-matched grafts...
November 27, 2018: Biology of Blood and Marrow Transplantation
Betul Oran, Naval Daver
No abstract text is available yet for this article.
November 27, 2018: Biology of Blood and Marrow Transplantation
Masaharu Akiyama, Masayoshi Yamaoka, Wataru Ohyama, Kentaro Yokoi, Shuichi Ashizuka, Daisuke Aizawa, Masahiro Ikegami, Hideaki Suzuki, Koji Ozaki, Hiroyuki Ida, Yuki Yuza
We report on a 16-year-old Japanese boy in whom an esophageal squamous cell carcinoma (ESCC) developed 12 years after allogeneic hematopoietic stem cell transplantation was performed for aplastic anemia. A high frequency of microsatellite instability was detected in samples of ESCC. Moreover, the detection of pathogenic variants, including single nucleotide substitution of TP53 (c.346C>T) and BRCA2 (c.6952C>T) and splicing of KDM6A (c.1194+2T>G), suggest that the development of ESCC in the patient was triggered by impairment of checkpoint and repair for DNA damage and epigenetic modification through accumulation of gene mutations induced by chronic graft-versus-host disease and prolonged administration of tacrolimus...
November 28, 2018: Journal of Pediatric Hematology/oncology
S R Safayi, F Shahi, M Ghalamkari, M Mirzania, M Khatuni, F Hirmandi Niasar
Background: Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially cure for acute myeloid leukemia (AML). Patients who undergone HSCT are at increased risk of infection due to impaired immunity. Objective: To evaluate the rate of bacterial, viral and fungal infection and its relationship with 2-year overall survival of AML patients who had undergone HSCT. Methods: This was a retrospective cross-sectional study of 49 patients who underwent allogenic bone marrow transplantation (BMT) from full-matched donors at BMT Center, Imam Khomeini Hospital Complex, Tehran, Iran, from 2006 to 2013...
2018: International Journal of Organ Transplantation Medicine
Z M Zhang, Y R Lai, Q C Li, L Luo, R R Liu, L L Shi, L J Liu
Objective: To explore the diagnosis, treatment and prognosis of autoimmune hemolytic anemia (AIHA) after allo-HSCT in patients with thalassemia major (TM). Methods: A retrospective analysis of AIHA status after allo-HSCT in 291 TM patients from July 2007 to December 2017 was conducted. Results: Five of the 291 TM patients (1.72%) were diagnosed with post-transplant AIHA. The median time of AIHA was 7 (5-12) months after HSCT. All post-transplant AIHA patients were positive in direct and indirect Coombs test, the main clinical manifestations were dizziness, fatigue, pale complexion, skin and sclera yellow, and soy sauce urine...
November 14, 2018: Zhonghua Xue Ye Xue za Zhi, Zhonghua Xueyexue Zazhi
Wen-Jing Yu, Xiao-Dong Mo, Xiao-Hui Zhang, Lan-Ping Xu, Yu Wang, Chen-Hua Yan, Huan Chen, Yu-Hong Chen, Wei Han, Feng-Rong Wang, Jing-Zhi Wang, Kai-Yan Liu, Xiao-Jun Huang
The aim of this study was to investigate the occurrence and severity of chemotherapy plus donor lymphocyte infusion (Chemo-DLI) associated chronic graft-versus-host disease (cGVHD) in a consecutive cohort of patients with acute leukemia who experienced relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT; n = 104). The 5-year cumulative incidence of complete remission (CR) after Chemo-DLI was 81.0% (95% CI, 73.3-88.7%) and 84.6% (95% CI, 74.5-94.7%) in the moderate and severe cGVHD groups, respectively, which was significantly higher than that of the mild cGVHD 40...
November 25, 2018: Biology of Blood and Marrow Transplantation
Joshua N Kellner, Eveline M Delemarre, Eric Yvon, Stefan Nierkens, Jaap J Boelens, Ian McNiece, Amanda Olson, Yago Nieto, Stefan Ciurea, Uday Popat, Sairah Ahmed, Richard Champlin, Jennifer Ramos, Mitsutaka Nishimoto, Hongbing Ma, Zeng Ke, Peter Thall, Joseph D Khoury, Robert Negrin, Borje Andersson, Simrit Parmar
Incubation of umbilical cord blood (UCB) derived regulatory T-cells (Tregs) with fucosyltransferase enzyme improves their ability to home to the target tissue to prevent graft vs. host disease (GVHD). We report results of 5 patients (Double UCB Transplant, n=2; Peripheral Blood Matched Unrelated Donor Transplant, n=3) who received UCB-Tregs (Dose level = 1×106/kg), infused one day prior to the donor graft. All patients received their designated UCB-Treg dose without any infusion reaction. The ratio of conventional T-cells in donor graft was at least 10 times higher than infused UCB-Tregs (ratio range, 12-356)...
November 2, 2018: Oncotarget
Josef Finsterer
Metabolic myopathies are a heterogeneous group of disorders characterized by inherited defects of enzymatic pathways involved in muscle fiber energetics. Diagnosing metabolic myopathies requires a thoroughly taken individual and family history, a meticulous neurologic exam, exercise tests, blood and urine tests, needle-electromyography, nerve-conduction studies, muscle biopsy, targeted genetic tests, or next-generation sequencing. There is limited evidence from the literature to guide treatment of metabolic myopathies...
December 2018: Expert Review of Neurotherapeutics
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