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Hematopoietic cell transplant

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https://www.readbyqxmd.com/read/29242305/worldwide-study-of-hematopoietic-allogeneic-stem-cell-transplantation-in-pyruvate-kinase-deficiency
#1
Stephanie van Straaten, Marc Bierings, Paola Bianchi, Kensuke Akiyoshi, Hitoshi Kanno, Isabel Badell Serra, Jing Chen, Xiaohang Huang, Eduard van Beers, Supachai Ekwattanakit, Tayfun Güngör, Wijnanda Adriana Kors, Frans Smiers, Reinier Raymakers, Lucrecia Yanez, Julian Sevilla, Wouter van Solinge, Jose Carlos Segovia, Richard Van Wijk
No abstract text is available yet for this article.
December 14, 2017: Haematologica
https://www.readbyqxmd.com/read/29242302/a-distinct-subtype-of-epstein-barr-virus-positive-t-nk-cell-lymphoproliferative-disorder-adult-patients-with-chronic-active-epstein-barr-virus-infection-like-features
#2
Keisuke Kawamoto, Hiroaki Miyoshi, Takaharu Suzuki, Yasuji Kozai, Koji Kato, Masaharu Miyahara, Toshiaki Yujiri, Naoki Oishi, Ilseung Choi, Katsumichi Fujimaki, Tsuyoshi Muta, Masaaki Kume, Sayaka Moriguchi, Shinobu Tamura, Takeharu Kato, Hiroyuki Tagawa, Junya Makiyama, Yuji Kanisawa, Yuya Sasaki, Daisuke Kurita, Kyohei Yamada, Joji Shimono, Hirohito Sone, Jun Takizawa, Masao Seto, Hiroshi Kimura, Koichi Ohshima
The characteristics of adult patients with chronic active Epstein-Barr virus infection (adult-onset CAEBV) are poorly recognized, hindering early diagnosis and an improved prognosis. Adult-onset CAEBV (n = 54) diagnosed between 2005 and 2015 were conducted. Adult-onset was defined as an estimated age of onset ≥15 years. To characterize the clinical features of adult-onset CAEBV, we compared them to those of pediatric-onset (estimated age of onset <15 years) patients (n = 75). We compared the prognosis of adult-onset CAEBV with that for patients with nasal-type (n = 37) and non-nasal-type (n = 45) extranodal NK/T-cell lymphoma (ENKTL)...
December 14, 2017: Haematologica
https://www.readbyqxmd.com/read/29242294/in-vivo-il-12-il-23p40-neutralization-blocks-th1-th17-response-after-allogeneic-hematopoietic-cell-transplantation
#3
Joseph Pidala, Francisca Beato, Jongphil Kim, Brian Betts, Heather Jim, Elizabeth Sagatys, John E Levine, James Lm Ferrara, Umut Ozbek, Ernesto Ayala, Marco Davila, Hugo F Fernandez, Teresa Field, Mohamed A Kharfan-Dabaja, Divis Khaira, Farhad Khimani, Frederick L Locke, Asmita Mishra, Michael Nieder, Taiga Nishihori, Lia Perez, Marcie Riches, Claudio Anasetti
T helper 1 and T helper 17 lymphocytes mediate acute graft vs. host disease. Interleukin 12 is critical for T helper 1 differentiation and interleukin 23 for T helper 17 maintenance. Interleukin 12 and 23 are heterodimeric cytokines that share the p40 subunit (IL-12/IL-23p40). In a randomized, blinded, placebo-controlled trial, we examined the biologic impact and clinical outcomes following IL-12/IL-23p40 neutralization using ustekinumab. 30 patients received peripheral blood mobilized hematopoietic cell transplantation from HLA-matched sibling or unrelated donors, received sirolimus plus tacrolimus as graft vs...
December 14, 2017: Haematologica
https://www.readbyqxmd.com/read/29242111/autologous-transplantation-in-follicular-lymphoma-with-early-therapy-failure-a-nlcs-and-cibmtr-analysis
#4
Carla Casulo, Jonathan W Friedberg, Kwang Woo Ahn, Christopher Flowers, Alyssa DiGilio, Sonali M Smith, Sairah Ahmed, David Inwards, Mahmoud Aljurf, Andy I Chen, Hannah Choe, Jonathon Cohen, Edward Copelan, Umar Farooq, Timothy S Fenske, Cesar Freytes, Sameh Gaballa, Siddhartha Ganguly, Yogesh Jethava, Rammurti T Kamble, Vaishalee P Kenkre, Hillard Lazarus, Aleksandr Lazaryan, Richard F Olsson, Andrew R Rezvani, David Rizzieri, Sachiko Seo, Gunjan L Shah, Nina Shah, Melham Solh, Anna Sureda, Basem William, Aaron Cumpston, Andrew D Zelenetz, Brian K Link, Mehdi Hamadani
Patients with follicular lymphoma (FL) experiencing early therapy failure (ETF) within two years of frontline chemoimmunotherapy have poor overall survival (OS). We analyzed data from the Center for International Blood and Marrow Transplant Research (CIBMTR) and the National LymphoCare Study (NLCS) to determine whether autologous hematopoietic cell transplant (autoHCT) can improve outcomes in this high-risk FL subgroup.ETF was defined as failure to achieve at least partial response after frontline chemoimmunotherapy or lymphoma progression within two years of frontline chemoimmunotherapy...
December 11, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29241731/efficacy-of-lentiviral-mediated-gene-therapy-in-an-omenn-syndrome-rag2-mouse-model-is-not-hindered-by-inflammation-and-immune-dysregulation
#5
Valentina Capo, Maria Carmina Castiello, Elena Fontana, Sara Penna, Marita Bosticardo, Elena Draghici, Luigi P Poliani, Lucia Sergi Sergi, Rosita Rigoni, Barbara Cassani, Monica Zanussi, Paola Carrera, Paolo Uva, Kerry Dobbs, Nicolò Sacchetti, Luigi D Notarangelo, Niek P van Til, Gerard Wagemaker, Anna Villa
BACKGROUND: Omenn syndrome (OS) is a rare severe combined immunodeficiency associated with autoimmunity, caused by defects of the lymphoid-specific V(D)J recombination. Most patients carry hypomorphic mutations in recombination activating genes (RAG) 1 or 2. Hematopoietic stem cell (HSC) transplantation is the standard treatment, however gene therapy (GT) may represent a valid alternative, especially for patients lacking a matched donor. OBJECTIVE: To determine the efficacy of lentiviral vector (LV) mediated GT in the murine model of OS (Rag2R229Q/R229Q) in correcting immunodeficiency and autoimmunity...
December 11, 2017: Journal of Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/29241729/long-term-follow-up-of-ipex-syndrome-patients-after-different-therapeutic-strategies-an-international-multicenter-retrospective-study
#6
Federica Barzaghi, Laura Cristina Amaya Hernandez, Benedicte Neven, Silvia Ricci, Zeynep Yesim Kucuk, Jack Bleesing, Zohreh Nademi, Mary Anne Slatter, Erlinda Rose Ulloa, Anna Shcherbina, Anna Roppelt, Austen Worth, Juliana Silva, Alessandro Aiuti, Luis Murguia-Favela, Carsten Speckmann, Magda Carneiro-Sampaio, Juliana Folloni Fernandes, Safa Baris, Ahmet Ozen, Elif Karakoc-Aydiner, Ayca Kiykim, Ansgar Schulz, Sandra Steinmann, Lucia Dora Notarangelo, Eleonora Gambineri, Paolo Lionetti, William Thomas Shearer, Lisa Forbes, Caridad Martinez, Despina Moshous, Stephane Blanche, Alain Fisher, Frank M Ruemmele, Come Tissandier, M Ouachee-Chardin, Frédéric Rieux-Laucat, Marina Cavazzana, Waseem Qasim, Barbarella Lucarelli, Michael H Albert, Ichiro Kobayashi, Laura Alonso, Cristina Diaz De Heredia, Hirokazu Kanegane, Anita Lawitschka, Jong Jin Seo, Marta Gonzalez-Vicent, Miguel Angel Diaz, Rakesh Kumar Goyal, Martin G Sauer, Akif Yesilipek, Minsoo Kim, Yesim Yilmaz-Demirdag, Monica Bhatia, Julie Khlevner, Erick J Richmond Padilla, Silvana Martino, Davide Montin, Olaf Neth, Agueda Molinos-Quintana, Justo Valverde-Fernandez, Arnon Broides, Vered Pinsk, Antje Ballauf, Filomeen Haerynck, Victoria Bordon, Catharina Dhooge, Maria Laura Garcia-Lloret, Robbert G Bredius, Krzysztof Kałwak, Elie Haddad, Markus Gerhard Seidel, Gregor Duckers, Sung-Yun Pai, Christopher C Dvorak, Stephan Ehl, Franco Locatelli, Frederick Goldman, Andrew Richard Gennery, Mort J Cowan, Maria Grazia Roncarolo, Rosa Bacchetta
BACKGROUND: Immunedysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined. OBJECTIVE: To evaluate disease onset, progression and long-term outcome of the two main treatments in long-term IPEX survivors...
December 11, 2017: Journal of Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/29240253/outcome-following-second-allogeneic-hematopoietic-cell-transplantation-a-single-center-experience
#7
Hassan A Aljasem, Hans A Messner, Jeffrey H Lipton, Dennis Dong Hwan Kim, Auro Viswabandya, Santhosh Thyagu, Uday Deotare, Fotios V Michelis
OBJECTIVE: Second allogeneic hematopoietic cell transplantation (HCT) may be indicated following relapse or graft failure following first HCT. Our retrospective single-center study sought to investigate parameters that influence post-second allogeneic HCT survival METHOD: We investigated 92 patients that underwent second allogeneic HCT between 1980 and 2016 for relapse or graft failure following first HCT. Median age at second HCT was 41 years (range 16-68), performed for relapse in 59 patients (64%) and for graft failure in 33 patients (36%) RESULTS: On univariate analysis, 3-year OS of the entire cohort was 35% (95%CI=25-45)...
December 14, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/29239916/early-experience-with-clinimacs-prodigy-ccs-ifn-gamma-system-in-selection-of-virus-specific-t-cells-from-third-party-donors-for-pediatric-patients-with-severe-viral-infections-after-hematopoietic-stem-cell-transplantation
#8
Krisztián Kállay, Csaba Kassa, Marienn Réti, Éva Karászi, János Sinkó, Vera Goda, Anita Stréhn, Katalin Csordás, Orsolya Horváth, Attila Szederjesi, Szabolcs Tasnády, Apor Hardi, Gergely Kriván
Viral reactivation is a frequent complication of allogeneic hematopoietic stem cell transplantation especially in children. For refractory cases, rapid virus-specific T-cell therapy would be ideally implemented within a few days. Over the course of a year in our pediatric cohort of 43 allogeneic transplantation, 9 patients fulfilled criteria for virus-specific T-cell therapy. Viral infections were due to cytomegalovirus (CMV) in 3, Epstein-Barr virus (EBV) in 2, and adenovirus (AdV) in 1 case, whereas >1 virus was detected in 3 cases...
December 12, 2017: Journal of Immunotherapy
https://www.readbyqxmd.com/read/29239076/allogeneic-hematopoietic-stem-cell-transplantation-is-associated-with-cure-and-durable-remission-of-late-onset-primary-isolated-central-nervous-system-hemophagocytic-lymphohistiocytosis
#9
Sajad Khazal, Veronika Polishchuk, Gary Soffer, Samantha Prinzing, Jonathan Gill, Kris M Mahadeo
Primary isolated CNS presentation of HLH is exceedingly rare and typically associated with significant morbidity and mortality. We describe an adolescent patient with late-onset, primary isolated CNS HLH and a compound heterozygous PRF1 mutation (c50delT (p.L17 fs); c.1229G>C (p.R410P)), not previously reported with this phenotype. He was successfully treated with allogeneic HSCT following a reduced-intensity conditioning regimen, despite a high pre-HSCT comorbidity index. Two years after transplant, he is alive and in disease remission...
December 13, 2017: Pediatric Transplantation
https://www.readbyqxmd.com/read/29238995/test-dose-pharmacokinetics-in-pediatric-patients-receiving-once-daily-iv-busulfan-conditioning-for-hematopoietic-stem-cell-transplant-a-reliable-approach
#10
Kristina M Brooks, Paul Jarosinski, Thomas Hughes, Elizabeth Kang, Nirali N Shah, John B Le Gall, Dennis D Hickstein, Suk See De Ravin, Jomy M George, Parag Kumar
Intravenous (IV) busulfan test dose pharmacokinetics (PK) has been shown to accurately predict once-daily dose requirements and improve outcomes in adult transplant patients, but there are limited data to support this approach in children. Test doses of busulfan ∼0.8 mg/kg were infused over 2 to 3 hours, followed by serial sampling to 4-6 hours postinfusion in pediatric hematopoietic stem cell transplant recipients (n = 5). Once-daily busulfan doses were calculated based on a myelosuppressive area under the concentration-time curve (AUC) target of ∼3700 to 4000 μmol·min/L and assumed dose-proportionality to the test dose...
December 14, 2017: Journal of Clinical Pharmacology
https://www.readbyqxmd.com/read/29238988/plerixafor-and-granulocyte-colony-stimulating-factor-for-mobilization-of-hematopoietic-stem-cells-for-autologous-transplantation-in-chinese-patients-with-non-hodgkin-s-lymphoma-a-randomized-phase-3-study
#11
Jun Zhu, Huiqiang Huang, Huan Chen, Xi Zhang, Zengjun Li, Depei Wu, Daobin Zhou, Yongping Song, Yu Hu, Yingmin Liang, Hanyun Ren, He Huang, Nainong Li, Hu Chen, Jiong Hu, Jianyong Li, Robin Meng, Junlong Wu, Dong Yu, Xiaojun Huang
BACKGROUND: This Phase 3 randomized, double-blind study evaluated the efficacy and safety of plerixafor plus granulocyte-colony-stimulating factor for the mobilization of hematopoietic stem cells in Chinese patients with non-Hodgkin's lymphoma. STUDY DESIGN AND METHODS: Adults (ages 18-75 years) with non-Hodgkin's lymphoma in first or second complete or partial remission, without previous hematopoietic stem cell mobilization or autologous transplant, were included...
December 13, 2017: Transfusion
https://www.readbyqxmd.com/read/29237963/analysis-of-factors-affecting-initial-cyclosporine-level-and-its-impact-on-post-transplant-outcomes-in-acute-leukemia
#12
Alok Gupta, Sachin Punatar, Jayant Gawande, Libin Mathew, Sadhana Kannan, Navin Khattry
BACKGROUND: Trough cyclosporine (CsA) blood level can influence incidence of graft-versus-host disease (GVHD) and relapse in patients with acute leukemia undergoing allogeneic hematopoietic stem cell transplant (HSCT). We sought to determine factors affecting initial trough CsA level (CsA-1) and its impact on transplant outcome in acute leukemia. MATERIALS AND METHODS: Seventy-seven patients underwent HSCT for acute leukemia between January 2008 and March 2013 and were included...
October 2017: Journal of Cancer Research and Therapeutics
https://www.readbyqxmd.com/read/29235254/mesenchymal-stem-cells-a-double-edged-sword-in-radiation-induced-lung-injury
#13
Yi Yao, Zhongliang Zheng, Qibin Song
Radiation therapy is an important treatment modality for multiple thoracic malignancies. However, radiation-induced lung injury (RILI), which is the term generally used to describe damage to the lungs caused by exposure to ionizing radiation, remains a critical issue affecting both tumor control and patient quality of life. Despite tremendous effort, there is no current consensus regarding the optimal treatment approach for RILI. Because of a number of functional advantages, including self-proliferation, multi-differentiation, injury foci chemotaxis, anti-inflammation, and immunomodulation, mesenchymal stem cells (MSCs) have been a focus of research for many years...
December 13, 2017: Thoracic Cancer
https://www.readbyqxmd.com/read/29235226/reduced-therapeutic-effect-of-antiviral-drugs-in-patients-with-hepatitis-b-virus-reactivation-after-hematopoietic-stem-cell-transplantation
#14
Masamichi Kimura, Koji Nishikawa, Hisashi Sakamaki, Masashi Mizokami, Kiminori Kimura
BACKGROUND: Patients with resolved Hepatitis B virus (HBV) infection following hematopoietic stem cell transplantation (HSCT) are potentially at high risk of HBV reactivation. Although anti-viral drug therapy is recommended when HBV DNA reappears in the serum, drug efficacy after HBV reactivation remains unclear. METHODS: Host immune response against HBV was investigated via immunological analyses at 12 months after entecavir (ETV) treatment in six HSCT-treated and five non-HSCT-treated patients with HBV reactivation, and 18 patients with chronic hepatitis B (CHB)...
December 13, 2017: Hepatology Research: the Official Journal of the Japan Society of Hepatology
https://www.readbyqxmd.com/read/29235178/the-gp130-cytokine-il-11-regulates-engraftment-of-vav1-hematopoietic-stem-and-progenitor-cells-in-lethally-irradiated-recipients
#15
Serena De Vita, Yanhua Li, Chad E Harris, Meaghan K McGuinness, Clement Ma, David A Williams
During bone marrow transplantation (BMT), hematopoietic stem and progenitor cells (HSPCs) respond to signals from the hematopoietic microenvironment (HM) by coordinately activating molecular pathways through Rho GTPases, including Rac. We have previously shown that deletion of Vav1, a hematopoietic-specific activator of Rac, compromises engraftment of transplanted adult HSPCs without affecting steady-state hematopoiesis in adult animals. Here, we show that Vav1-/- fetal HSPCs can appropriately seed hematopoietic tissues during ontogeny but cannot engraft into lethally irradiated recipients...
December 13, 2017: Stem Cells
https://www.readbyqxmd.com/read/29234595/ibrutinib-as-a-bridge-to-transplant-in-high-risk-chronic-lymphocytic-leukemia-a-case-report-and-review-of-the-literature
#16
Arcari Annalisa, Bassi Simona, Pochintesta Lara, Trabacchi Elena, Moroni Carlo Filippo, Rossi Angela, Zanlari Luca, Vallisa Daniele
The treatment landscape of chronic lymphocytic leukemia (CLL) has been challenged by the advent of novel classes of drugs, such as B-cell receptor (BCR)-inhibitors and BCL-2 antagonists. In selected high-risk patients, the choice to start allogeneic hematopoietic stem cell transplantation (alloHCT) or continue these agents is a matter of debate. Furthermore, published data about the impact on the feasibility of alloHCT and the optimal timing of administration are limited. Here we present a case of relapsed TP53 mutated CLL treated with ibrutinib as a bridge to alloHCT, discussing risks and benefits of different treatment options in a "real life" situation...
2017: Leukemia Research Reports
https://www.readbyqxmd.com/read/29233822/hematopoietic-stem-cell-gene-therapy-for-ifn%C3%AE-r1-deficiency-protects-mice-from-mycobacterial-infections
#17
Miriam Hetzel, Adele Mucci, Patrick Blank, Ariane Hai Ha Nguyen, Jan Schiller, Olga Halle, Mark-Philipp Kühnel, Sandra Billig, Robert Meineke, Daniel Brand, Vanessa Herder, Wolfgang Baumgärtner, Franz-Christoph Bange, Ralph Goethe, Danny Jonigk, Reinhold Förster, Bernhard Gentner, Jean-Laurent Casanova, Jacinta Bustamante, Axel Schambach, Ulrich Kalinke, Nico Lachmann
Mendelian Susceptibility to Mycobacterial Disease (MSMD) is a rare primary immunodeficiency, characterized by severe infections caused by weakly virulent mycobacteria. Bi-allelic null mutations in genes encoding interferon gamma (IFNγ) receptor 1 or -2 (IFNGR1, IFNGR2) result in a life-threatening disease phenotype in early childhood. Recombinant IFNγ therapy is inefficient and hematopoietic stem cell transplantation (HSCT) has a poor prognosis. Thus, we developed a HSC gene therapy approach using lentiviral vectors expressing Ifnγr1 either constitutively or myeloid-specifically...
December 12, 2017: Blood
https://www.readbyqxmd.com/read/29232940/no-benefit-of-hypomethylating-agents-compared-to-supportive-care-for-higher-risk-myelodysplastic-syndrome
#18
Sang Kyun Sohn, Joon Ho Moon, In Hee Lee, Jae Sook Ahn, Hyeoung Joon Kim, Joo Seop Chung, Ho Jin Shin, Sung Woo Park, Won Sik Lee, Sang Min Lee, Hawk Kim, Ho Sup Lee, Yang Soo Kim, Yoon Young Cho, Sung Hwa Bae, Ji Hyun Lee, Sung Hyun Kim, Ik Chan Song, Ji Hyun Kwon, Yoo Jin Lee
Background/Aims: This study evaluated the role of hypomethylating agents (HMA) compared to best supportive care (BSC) for patients with high or very-high (H/VH) risk myelodysplastic syndrome (MDS) according to the Revised International Prognostic Scoring System. Methods: A total of 279 H/VH risk MDS patients registered in the Korean MDS Working Party database were retrospectively analyzed. Results: HMA therapy was administered to 205 patients (73...
December 15, 2017: Korean Journal of Internal Medicine
https://www.readbyqxmd.com/read/29232542/allogeneic-transplantation-for-follicular-lymphoma-does-one-size-fit-all
#19
Mehdi Hamadani, Mary M Horowitz
Follicular lymphoma (FL) exhibits striking biologic and clinical heterogeneity. Patients with newly diagnosed asymptomatic or low-bulk disease may be observed or managed with immunotherapies alone. Chemoimmunotherapy is considered a standard treatment for patients with advanced, symptomatic disease. In patients with FL who achieve at least a partial remission after first-line chemoimmunotherapy, autologous (auto-) hematopoietic cell transplantation (HCT) consolidation is not recommended; however, most patients with FL experience disease relapse after frontline therapies, with the experience of therapy failure within 2 years of first-line treatments predicting poor survival...
December 2017: Journal of Oncology Practice
https://www.readbyqxmd.com/read/29232422/atypical-disseminated-herpes-zoster-management-guidelines-in-immunocompromised-patients
#20
Daniel J Lewis, Megan J Schlichte, Harry Dao
Reactivation of the varicella-zoster virus (VZV) causes dermatomal herpes zoster (HZ) and more rarely severe disseminated HZ including diffuse rash, encephalitis, hepatitis, and pneumonitis. An atypical form of VZV infection, disseminated HZ has been described primarily in immunocompromised hosts. We report 2 cases of atypical disseminated HZ in immunocompromised patients presenting with diffuse, nondermatomal, vesicular eruptions. We also provide a review of the literature and summarize the current guidelines for the treatment and prophylaxis of HZ in patients with human immunodeficiency virus (HIV) infection, solid organ transplantation (SOT), and hematopoietic stem cell transplantation (HSCT)...
November 2017: Cutis; Cutaneous Medicine for the Practitioner
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