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Hematopoietic cell transplant

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https://www.readbyqxmd.com/read/29691808/total-body-irradiation-for-hematopoietic-stem-cell-transplantation-during-early-childhood-is-associated-with-the-risk-for-diabetes-mellitus
#1
Ryuichi Nakagawa, Atsumi Hosokawa-Tsuji, Yuki Aoki, Kei Takasawa, Mitsue Maru, Keisuke Nakajima, Akito Sutani, Yuichi Miyakawa, Daisuke Tomizawa, Kenichi Kashimada, Tomohiro Morio
BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is a curative treatment for life-threatening malignancies and related diseases. Recently, the long-term prognosis of HSCT during childhood has greatly improved; however, the late adverse effects of HSCT have been found to cause substantial morbidity among long-term survivors. Although metabolic complications, such as diabetes mellitus (DM) and hyperlipidemia (HL), are the major late effects of pediatric HSCT, the clinical details are not clarified sufficiently...
April 24, 2018: Endocrine
https://www.readbyqxmd.com/read/29691471/baricitinib-induced-blockade-of-interferon-gamma-receptor-and-interleukin-6-receptor-for-the-prevention-and-treatment-of-graft-versus-host-disease
#2
Jaebok Choi, Matthew L Cooper, Karl Staser, Kidist Ashami, Kiran R Vij, Bing Wang, Lynne Marsala, Jessica Niswonger, Julie Ritchey, Bader Alahmari, Samuel Achilefu, Ikuo Tsunoda, Mark A Schroeder, John F DiPersio
The therapeutic benefits of allogeneic hematopoietic stem cell transplantation (allo-HSCT) are derived from the graft-versus-leukemia (GvL) effects of the procedure. There is a strong association between the GvL effects and graft-versus-host disease (GvHD), a major life-threatening complication of allo-HSCT. The limiting of GvHD while maintaining the GvL effect remains the goal of allo-HSCT. Therefore, identifying optimal therapeutic targets to selectively suppress GvHD while maintaining the GvL effects represents a significant unmet medical need...
April 2, 2018: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://www.readbyqxmd.com/read/29691342/human-cytomegalovirus-encodes-a-novel-flt3-receptor-ligand-necessary-for-hematopoietic-cell-differentiation-and-viral-reactivation
#3
Lindsey B Crawford, Jung Heon Kim, Donna Collins-McMillen, Byeong-Jae Lee, Igor Landais, Christine Held, Jay A Nelson, Andrew D Yurochko, Patrizia Caposio
The ability of human cytomegalovirus (HCMV) to reactivate from latent infection of hematopoietic progenitor cells (HPCs) is intimately linked to cellular differentiation. HCMV encodes UL7 that our group has shown is secreted from infected cells and induces angiogenesis. In this study, we show that UL7 is a ligand for Fms-like tyrosine kinase 3 receptor (Flt-3R), a well-known critical factor in HPC differentiation. We observed that UL7 directly binds Flt-3R and induces downstream signaling cascades, including phosphatidylinositol 3-kinase (PI3K)/Akt and mitogen-activated protein kinase (MAPK)/extracellular signal-regulated kinase (ERK) pathways...
April 24, 2018: MBio
https://www.readbyqxmd.com/read/29690908/adenosine-deaminase-deficiency-a-review
#4
REVIEW
Aisling M Flinn, Andrew R Gennery
Adenosine deaminase (ADA) deficiency leads to an accumulation of toxic purine degradation by-products, most potently affecting lymphocytes, leading to adenosine deaminase-deficient severe combined immunodeficiency. Whilst most notable affects are on lymphocytes, other manifestations include skeletal abnormalities, neurodevelopmental affects and pulmonary manifestations associated with pulmonary-alveolar proteinosis. Affected patients present in early infancy, usually with persistent infection, or with pulmonary insufficiency...
April 24, 2018: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/29689537/hiv-infection-advances-toward-a-cure
#5
Daniel C Douek
Achieving cure of HIV infection requires eliminating all replication-competent virus from the reservoir of latently infected cells or completely inhibiting infected cells from emerging from latency. Strategies include very early use of antiretroviral therapy; hematopoietic stem cell transplantation; "shock-and-kill" approaches; immune therapy with immune checkpoint inhibitors; gene therapy, including use of CC chemokine receptor 5-modified CD4+ T cells; and broadly neutralizing antibody therapy. Success is likely to require a combination of approaches...
April 2018: Topics in Antiviral Medicine
https://www.readbyqxmd.com/read/29688570/diffuse-large-b-cell-lymphoma-chemotherapy-reveals-a-combined-immunodeficiency-syndrome-in-cartilage-hair-hypoplasia
#6
Alexandre Nguyen, Nicolas Martin Silva, Hubert de Boysson, Gandhi Damaj, Achille Aouba
Cartilage hair hypoplasia (CHH) is a rare autosomal recessive ribosomopathy characterised by skeletal and integumentary system manifestations. It may also present with varied forms and intensities of haematopoietic and/or immune disorders. We report a 27-year-old female who presented a picture of combined immunodeficiency after receiving an adriamycin-based chemotherapy regimen followed by autologous stem cell transplantation. Her medical history indicated neonatal dwarfism, recurrent ear, nose and throat and respiratory infections, and hypogammaglobulinaemia, which were suggestive of a primary minor B-cell immune deficiency...
April 24, 2018: Swiss Medical Weekly
https://www.readbyqxmd.com/read/29687743/clinical-outcomes-of-treatment-with-filgrastim-versus-a-filgrastim-biosimilar-and-febrile-neutropenia-associated-costs-among-patients-with-nonmyeloid-cancer-undergoing-chemotherapy
#7
Lee S Schwartzberg, Lincy S Lal, Sanjeev Balu, Kim Campbell, Lee Brekke, Andrew DeLeon, Caitlin Elliott, Stephanie Korrer
BACKGROUND: Granulocyte colony-stimulating factors such as filgrastim are used to decrease the incidence of febrile neutropenia (FN) among patients with nonmyeloid cancers undergoing chemotherapy treatment. Although the biosimilar filgrastim-sndz has been approved in the United States since 2015, limited real-world comparisons of filgrastim-sndz versus reference filgrastim (filgrastim-ref) have been conducted. OBJECTIVE: To compare FN incidence and assess overall FN-related health care resource utilization and medical costs among U...
April 24, 2018: Journal of Managed Care & Specialty Pharmacy
https://www.readbyqxmd.com/read/29687034/preclinical-efficacy-and-safety-evaluation-of-hematopoietic-stem-cell-gene-therapy-in-a-mouse-model-of-mngie
#8
Rana Yadak, Raquel Cabrera-Pérez, Javier Torres-Torronteras, Marianna Bugiani, Joost C Haeck, Marshall W Huston, Elly Bogaerts, Steffi Goffart, Edwin H Jacobs, Merel Stok, Lorena Leonardelli, Luca Biasco, Robert M Verdijk, Monique R Bernsen, George Ruijter, Ramon Martí, Gerard Wagemaker, Niek P van Til, Irenaeus F M de Coo
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by thymidine phosphorylase (TP) deficiency resulting in systemic accumulation of thymidine (d-Thd) and deoxyuridine (d-Urd) and characterized by early-onset neurological and gastrointestinal symptoms. Long-term effective and safe treatment is not available. Allogeneic bone marrow transplantation may improve clinical manifestations but carries disease and transplant-related risks. In this study, lentiviral vector-based hematopoietic stem cell gene therapy (HSCGT) was performed in Tymp -/- Upp1 -/- mice with the human phosphoglycerate kinase (PGK) promoter driving TYMP ...
March 16, 2018: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/29685953/outcomes-of-medicare-age-eligible-nhl-patients-receiving-ric-allogeneic-transplantation-a-cibmtr-analysis
#9
Nirav N Shah, Kwang Woo Ahn, Carlos Litovich, Timothy S Fenske, Sairah Ahmed, Minoo Battiwalla, Nelli Bejanyan, Parastoo B Dahi, Javier Bolaños-Meade, Andy I Chen, Stefan O Ciurea, Veronika Bachanova, Zachariah DeFilipp, Narendranath Epperla, Nosha Farhadfar, Alex F Herrera, Bradley M Haverkos, Leona Holmberg, Nasheed M Hossain, Mohamed A Kharfan-Dabaja, Vaishalee P Kenkre, Hillard M Lazarus, Hemant S Murthy, Taiga Nishihori, Andrew R Rezvani, Anita D'Souza, Bipin N Savani, Matthew L Ulrickson, Edmund K Waller, Anna Sureda, Sonali M Smith, Mehdi Hamadani
The application of allogeneic hematopoietic cell transplantation (allo-HCT) in non-Hodgkin lymphoma (NHL) patients ≥65 years in the United States is limited by lack of Medicare coverage for this indication. Using the Center for International Blood and Marrow Transplant Research (CIBMTR) database, we report allo-HCT outcomes of NHL patients aged ≥65 years (older cohort; n = 446) compared with a cohort of younger NHL patients aged 55-64 years (n = 1183). We identified 1629 NHL patients undergoing a first reduced-intensity conditioning (RIC) or nonmyeloablative conditioning allo-HCT from 2008 to 2015 in the United States...
April 24, 2018: Blood Advances
https://www.readbyqxmd.com/read/29685874/impact-of-oral-side-effects-from-conditioning-therapy-before-hematopoietic-stem-cell-transplantation-protocol-for-a-multicenter-study
#10
Michael T Brennan, Bengt Hasséus, Allan J Hovan, Judith E Raber-Durlacher, Nicole Ma Blijlevens, Marie-Charlotte Huysmans, Karin Garming Legert, Jan-Erik Johansson, Charity G Moore, Inger von Bültzingslöwen
BACKGROUND: The oral cavity is a common site of complications related to the cytotoxic effect of high-dose chemotherapy and radiation therapy. Considering our limited understanding of the burden of illness in the oral cavity from various cytotoxic therapies, it is difficult to produce evidence-based, preventive and management protocols. A prospective multicenter study is necessary to collect data on the burden of illness from various cytotoxic regimens. OBJECTIVE: The objectives of this prospective international observational multicenter study in hematopoietic stem cell transplant (HSCT) patients are to establish the nature, incidence and temporal relationship of oral complications related to conditioning regimens (chemotherapy with or without total body irradiation), stem cell transplantation and the immunologic reactions (mainly graft-vs-host-disease) that may follow, and to determine what subjective and objective oral complications related to treatment can predict negative clinical and economic outcomes and reduced quality of life...
April 23, 2018: JMIR Research Protocols
https://www.readbyqxmd.com/read/29685843/clinical-units-to-set-up-chimeric-antigen-receptor-t-cell-therapy-car-t-cells-based-on-the-recommendations-of-the-francophone-society-of-bone-marrow-transplantation-and-cellular-therapy-sfgm-tc
#11
Ibrahim Yakoub-Agha
The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) hold its eighth practice harmonization workshops on September 2017. In a workshop dedicated to chimeric antigen receptor T-cell therapy (CAR T-cells), the society issued recommendations regarding the prerequisite for hematopoietic cellular therapy programs to set up CAR T-cell therapy. In this article we focused on the prerequisite needed, in France, for a hematopoietic transplantation unit to start a CAR T-cell program with industrial manufactured cells within investigational products or after market access authorization...
April 20, 2018: Current Research in Translational Medicine
https://www.readbyqxmd.com/read/29684567/human-herpesvirus-6b-hhv-6b-reactivation-is-a-risk-factor-for-grade-2-4-acute-graft-versus-host-disease-agvhd-after-hematopoietic-stem-cell-transplantation-hct-a-systematic-review-and-meta-analysis
#12
REVIEW
Tuan L Phan, Kristen Carlin, Per Ljungman, Ioannis Politikos, Vicki Boussiotis, Michael Boeckh, Michele L Shaffer, Danielle M Zerr
BACKGROUND/OBJECTIVE: Graft-versus-host disease (GVHD) is an important cause of morbidity and mortality following allogeneic hematopoietic cell transplantation (HCT). Many studies have suggested that HHV-6B plays a role in acute GVHD (aGVHD) following HCT. Our objective was to systematically summarize and analyze evidence regarding HHV-6B reactivation and development of aGVHD. METHODS: PubMed and EMBASE databases were searched using terms for HHV-6, HCT, and aGVHD, yielding 865 unique results...
April 20, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29684566/eltrombopag-for-the-treatment-of-of-refractory-pure-red-cell-aplasia-after-major-abo-incompatible-hematopoietic-stem-cell-transplantation
#13
Alessandro Busca, Chiara Dellacasa, Luisa Giaccone, Sara Manetta, Lucia Biale, Laura Godio, Semra Aydin, Moreno Festuccia, Lucia Brunello, Benedetto Bruno
Pure red cell aplasia (PRCS) is a well-recognized complication after allogeneic hematopoietic stem cell transplantation (HSCT). Many therapeutic options are available to treat this condition, including erythropoietin, rituximab, bortezomib, plasma exchange, immunoadsorption, donor lymphocyte infusion, mesenchymal stem cells, anti-thymocyte globulin and high-dose steroids; however treatment outcomes are often variable and can sometimes lead to disappointing results. In this brief article we report our experience with two patients with PRCA after major AB0-incompatible HSCT who were resistant to multiple therapeutic interventions and who eventually benefited from treatment with eltrombopag, a thrombopoietin mimetic approved by the Food and Drug Administration for the treatment of patients with immune thrombocytopenic purpura or severe aplastic anemia (SAA), refractory to immunosuppressive agents or not eligible to HSCT...
April 20, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29684565/thiotepa-based-intensified-reduced-intensity-conditioning-adult-double-unit-cord-blood-hematopoietic-stem-cell-transplantation-results-in-decreased-relapse-rate-and-improved-survival-compared-to-transplantation-following-standard-reduced-intensity-conditioning
#14
Prashant Sharma, Daniel A Pollyea, Clayton A Smith, Enkhtsetseg Purev, Manali Kamdar, Bradley Haverkos, Daniel Sherbenou, Rachel Rabinovitch, Andrew Hammes, Jonathan A Gutman
The "Minnesota" reduced intensity conditioning (RIC) cord-blood transplantation (CBT) regimen (standard-RIC) of fludarabine (Flu, 200mg/m2 ), cyclophosphamide (Cy, 50mg/kg), and 200cGy or 300cGy total body irradiation (TBI) is the most published RIC CBT regimen. Though well tolerated, high relapse rates remain a concern with this regimen. Intensification of conditioning may reduce relapse without increasing transplant related mortality (TRM). We performed a retrospective cohort comparison of outcomes in adult patients who underwent first double-unit CBT with standard-RIC as compared to the intensified regimen of fludarabine 150mg/m2 , cyclophosphamide 50mg/kg, thiotepa 10mg/kg and 400cGy TBI (intensified-RIC)...
April 20, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29684564/deep-npm1-sequencing-following-allogeneic-hematopoietic-cell-transplantation-improves-risk-assessment-in-adults-with-npm1-mutated-aml
#15
Yi Zhou, Megan Othus, Roland B Walter, Elihu H Estey, David Wu, Brent L Wood
Relapse is the major cause of death in patients with acute myeloid leukemia (AML) after allogeneic hematopoietic cell transplant (HCT). Measurable residual disease (MRD) detected by multiparameter flow cytometry (MFC) before and after HCT is a strong, independent risk factor for relapse. As next generation sequencing (NGS) is increasingly applied in AML MRD detection, it remains to be determined if NGS can improve prediction of post-HCT relapse. Herein, we investigated pre-HCT MRD detected by MFC and NGS in 59 adult patients with NPM1-mutated AML in morphologic remission; 45 of the 59 had post-HCT MRD determined by MFC and NGS around day 28...
April 20, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29684563/long-term-results-of-prophylactic-donor-lymphocyte-infusions-for-patients-with-multiple-myeloma-after-allogeneic-stem-cell-transplantation
#16
Maximilian Gröger, Nico Gagelmann, Christine Wolschke, Ute-Marie von Pein, Evgeny Klyuchnikov, Max Christopeit, Axel Zander, Francis Ayuk, Nicolaus Kröger
OBJECTIVE: The major reason for treatment failure after allografting in multiple myeloma (MM) is relapse. Donor lymphocyte infusions are considered a valuable post-transplant strategy mainly for relapsed patients but using them to prevent relapse in MM has been reported rarely. In the present study we examined the efficacy of prophylactic donor lymphocyte infusions after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in myeloma patients with a long-term follow-up of more than 5 years...
April 20, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29684562/bmt-following-non-myeloablative-treosulfan-conditioning-is-curative-in-a-murine-model-of-sickle-cell-disease
#17
Divya Devadasan, Chiao-Wang Sun, Erik R Westin, Li-Chen Wu, Kevin M Pawlik, Tim M Townes, Frederick D Goldman
Allogeneic hematopoietic stem cell transplantation (HSCT) can be curative for patients with sickle cell disease (SCD). However, morbidity associated with myeloablative conditioning and graft versus host disease has limited its utility. To this end, autologous HSCT for SCD using lentiviral gene-modified bone marrow (BM) or peripheral blood stem cells has been undertaken, though toxicities of fully ablative conditioning with busulfan and incomplete engraftment have been encountered. Treosulfan, a busulfan analog with a low extra-medullary toxicity profile, has been employed successfully as part of a myeloablative conditioning regimen in the allogeneic setting in SCD...
April 20, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29683948/hematopoietic-stem-cell-transplantation-from-unrelated-donors-in-2-cases-of-interleukin-10-receptor-deficiency-is-surgery-not-a-requirement
#18
Dilara F Kocacik Uygun, Vedat Uygun, Hayriye Daloğlu, Seda Öztürkmen, Gülsün Karasu, İsmail Reisli, Ersin Sayar, Hasan A Yüksekkaya, Erik-Oliver Glocker, Kaan Boztuğ, Akif Yeşilipek
Mutations in interleukin-10 and its receptors cause infantile inflammatory bowel disease (IBD), a hyperinflammatory disorder characterized by severe, treatment-refractory colitis, multiple abscesses, and enterocutaneous fistulas. Patients with infantile IBD often require several surgical interventions, including complete colectomy, and hematopoietic stem cell transplantation is currently the only known medical therapy. Traditionally, operative management has been preferred before stem cell transplantation because of the latter's increased susceptibility to procedural complications; however, surgical intervention could be delayed, and possibly reconsidered, because our 2 patients with infantile IBD demonstrated a rapid response to treatment via engraftment...
April 20, 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29681947/synergistic-integration-of-mesenchymal-stem-cells-and-hydrostatic-pressure-in-the-expansion-and-maintenance-of-human-hematopoietic-progenitor-cells
#19
Yun Gyeong Kang, Jee-Yeong Jeong, Tae-Hee Lee, Ho Sup Lee, Jung-Woog Shin
Ex vivo expansion of hematopoietic stem/progenitor cell (HSPC) has been investigated to improve the clinical outcome of HSPC transplantation. However, ex vivo expansion of HSPCs still faces a major obstacle in that HPSCs tend to differentiate when proliferating. Here, we cocultured HSPCs with mesenchymal stem cells (MSCs) and divided the HSPCs into two fractions according to whether they came into adherent to MSCs or not. Additionally, we used hydrostatic pressure (HP) to mimic the physical conditions in vivo ...
2018: Stem Cells International
https://www.readbyqxmd.com/read/29681820/use-of-eltrombopag-in-improving-poor-graft-function-after-allogeneic-hematopoietic-stem-cell-transplantation
#20
Samip Master, Ashish Dwary, Richard Mansour, Glenn M Mills, Nebu Koshy
Eltrombopag is a thrombopoietin agonist and has been used in aplastic anemia and post-transplantation thrombocytopenia. The c-MPL receptor is present on hematopoietic stem cells. There are no reports of eltrombopag utilization for improving poor graft function in the post-transplant setting. Here were report a case of a young female with post-transplant poor graft function as evident from the low absolute neutrophil count, anemia, and thrombocytopenia on day 60. Eltrombopag was started on day 72 and resulted in improvement in all 3 cell lines...
January 2018: Case Reports in Oncology
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