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https://www.readbyqxmd.com/read/28346229/clinical-efficacy-of-gene-modified-stem-cells-in-adenosine-deaminase-deficient-immunodeficiency
#1
Kit L Shaw, Elizabeth Garabedian, Suparna Mishra, Provaboti Barman, Alejandra Davila, Denise Carbonaro, Sally Shupien, Christopher Silvin, Sabine Geiger, Barbara Nowicki, E Monika Smogorzewska, Berkley Brown, Xiaoyan Wang, Satiro de Oliveira, Yeong Choi, Alan Ikeda, Dayna Terrazas, Pei-Yu Fu, Allen Yu, Beatriz Campo Fernandez, Aaron R Cooper, Barbara Engel, Greg Podsakoff, Arumugam Balamurugan, Stacie Anderson, Linda Muul, G Jayashree Jagadeesh, Neena Kapoor, John Tse, Theodore B Moore, Ken Purdy, Radha Rishi, Kathey Mohan, Suzanne Skoda-Smith, David Buchbinder, Roshini S Abraham, Andrew Scharenberg, Otto O Yang, Kenneth Cornetta, David Gjertson, Michael Hershfield, Rob Sokolic, Fabio Candotti, Donald B Kohn
BACKGROUND: Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that has shown clinical benefit for adenosine deaminase-deficient (ADA-deficient) SCID when combined with reduced intensity conditioning (RIC) and ERT cessation. Clinical safety and therapeutic efficacy were evaluated in a phase II study. METHODS: Ten subjects with confirmed ADA-deficient SCID and no available matched sibling or family donor were enrolled between 2009 and 2012 and received transplantation with autologous hematopoietic CD34+ cells that were modified with the human ADA cDNA (MND-ADA) γ-retroviral vector after conditioning with busulfan (90 mg/m2) and ERT cessation...
March 27, 2017: Journal of Clinical Investigation
https://www.readbyqxmd.com/read/28345415/chondrogenic-gene-expression-differences-between-chondrocytes-from-osteoarthritic-and-non-oa-trauma-joints-in-a-3d-collagen-type-i-hydrogel
#2
Vivek Jeyakumar, Florian Halbwirth, Eugenia Niculescu-Morzsa, Christoph Bauer, Hannes Zwickl, Daniela Kern, Stefan Nehrer
Objective The purpose of the current study was to compare the donor age variation of chondrocytes from non-OA (osteoarthritic) trauma joints in patients of young to middle age (20.5 ± 3.7, 31.8 ± 1.9, 41.9 ± 4.1 years) embedded in matrix-associated autologous chondrocyte transplantation (MACT) grafts (CaReS). The chondrocyte-specific gene expression of CaReS grafts were then compared to chondrocytes from OA joints (in patients aged 63.8 ± 10 years) embedded in a collagen type I hydrogel. Design OA chondrocytes and articular chondrocyte-laden grafts were cultured over 14 days in chondrogenic growth medium...
April 2017: Cartilage
https://www.readbyqxmd.com/read/28345003/expansion-of-human-tregs-from-cryopreserved-umbilical-cord-blood-for-gmp-compliant-autologous-adoptive-cell-transfer-therapy
#3
Howard R Seay, Amy L Putnam, Judit Cserny, Amanda L Posgai, Emma H Rosenau, John R Wingard, Kate F Girard, Morey Kraus, Angela P Lares, Heather L Brown, Katherine S Brown, Kristi T Balavage, Leeana D Peters, Ashley N Bushdorf, Mark A Atkinson, Jeffrey A Bluestone, Michael J Haller, Todd M Brusko
Umbilical cord blood is a traditional and convenient source of cells for hematopoietic stem cell transplantation. Thymic regulatory T cells (Tregs) are also present in cord blood, and there is growing interest in the use of autologous Tregs to provide a low-risk, fully human leukocyte antigen (HLA)-matched cell product for treating autoimmune diseases, such as type 1 diabetes. Here, we describe a good manufacturing practice (GMP)-compatible Treg expansion protocol using fluorescence-activated cell sorting, resulting in a mean 2,092-fold expansion of Tregs over a 16-day culture for a median yield of 1...
March 17, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28344788/lymphoma-development-and-survival-in-refractory-coeliac-disease-type-ii-histological-response-as-prognostic-factor
#4
P Nijeboer, Rlj van Wanrooij, T van Gils, N J Wierdsma, G J Tack, B I Witte, H J Bontkes, O Visser, Cjj Mulder, G Bouma
BACKGROUND: Refractory coeliac disease type II (RCDII) frequently transforms into an enteropathy-associated T-cell lymphoma (EATL) and therefore requires intensive treatment. Current evaluated treatment strategies for RCDII include cladribine (2-CdA) and autologous stem cell transplantation (auSCT). OBJECTIVE: The purpose of this study was to evaluate long-term survival and define clear prognostic criteria for EATL development comparing two treatment strategies...
March 2017: United European Gastroenterology Journal
https://www.readbyqxmd.com/read/28341874/the-multi-faceted-potential-of-cd38-antibody-targeting-in-multiple-myeloma
#5
Rory M Shallis, Christopher M Terry, Seah H Lim
CD38, an adenine dinucleotide phosphate (ADP) ribose cyclase and a cyclic ADP ribose hydrolase, is widely expressed on the surface of multiple myeloma (MM) cells. It is known to play a pivotal role in the downstream pathways that mediate MM cell growth, signal transduction, and adhesion. The clinical use of CD38 monoclonal antibodies (MoAbs), such as daratumumab, either as monotherapy or in combination with other anti-MM agents, has produced impressive results in patients who have failed standard MM therapy...
March 24, 2017: Cancer Immunology, Immunotherapy: CII
https://www.readbyqxmd.com/read/28340882/autologous-and-allogeneic-hematopoietic-cell-transplantation-in-peripheral-t-nk-cell-lymphomas-a-histology-specific-review
#6
REVIEW
Tejaswini M Dhawale, Andrei R Shustov
Peripheral T-cell lymphoma and natural killer/T-cell lymphomas (PT/NKCL) make up a diverse subgroup of non-Hodgkin's lymphomas characterized by an aggressive clinical course. The use of hematopoietic stem cell transplantation (HSCT) in the treatment of PT/NKCL remains controversial because of the absence of randomized controlled trials. The best available data suggest that certain subtypes of PT/NKCL may benefit more from the application of HSCT than other subtypes and that this benefit results from their unique clinical characteristics and underlying biology...
April 2017: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/28340875/angioimmunoblastic-t-cell-lymphoma
#7
REVIEW
Alessandro Broccoli, Pier Luigi Zinzani
Angioimmunoblastic T-cell lymphoma is a follicular T-helper-derived neoplasm displaying a peculiar morphologic appearance and biological complexity. New mutations have been described that contribute to elucidating the underlying pathogenetic events. The disease behaves aggressively and typically affects elderly patients. The outcomes reported with anthracycline-containing regimens are poor; therefore autologous transplantation in first remission should be offered whenever possible. Newer approaches are urgently needed for relapsed and refractory patients...
April 2017: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/28340873/peripheral-t-cell-lymphoma-not-otherwise-specified-a-review-of-current-disease-understanding-and-therapeutic-approaches
#8
REVIEW
Musa Al-Zahrani, Kerry J Savage
Peripheral T-cell lymphoma, not otherwise specified (PTCL-NOS), corresponds with a heterogeneous group of mature T-cell lymphomas. Recent gene expression profiling studies have identified at least two molecular subgroups (GATA3 and TBX2). Standard treatment and outcomes remain poor. High-dose chemotherapy with autologous stem cell transplantation is incorporated into primary therapy for young fit patients but remains ineffective for most and has not been tested in a randomized study. Several novel agents have been approved for use in relapsed/refractory PTCLs, and although response rates are modest for most, durable remissions have been reported...
April 2017: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/28340834/hepatic-artery-reconstruction-with-autologous-inferior-mesenteric-artery-graft-in-living-donor-liver-transplant-recipients
#9
M Özbilgin, T Ünek, T Egeli, C Ağalar, S Ozkardesler, F Obuz, H Ellidokuz, S Karademir, I Astarcıoğlu
INTRODUCTION: In living donor liver transplantation (LDLT), hepatic arterial continuity is crucial to avoid biliary leakage, biliary stricture, cholangitis, and graft and patient loss. Sometimes there exist factors making anastomosis difficult or even impossible. In these cases, a vascular graft may be needed to bridge the two arteries for revascularization. METHOD: Medical records of 297 patients who underwent LDLT between June 2000 and July 2016 at the Hepatopancreatobiliary Surgery and Liver Transplantation Unit of Dokuz Eylul University Hospital were reviewed retrospectively...
April 2017: Transplantation Proceedings
https://www.readbyqxmd.com/read/28339301/cultivation-of-human-oral-mucosal-explants-on-contact-lenses
#10
Barbara Zsebik, László Ujlaky-Nagy, Gergely Losonczy, György Vereb, Lili Takács
Purpose/Aim: Autologous cultivated oral mucosal (OM) epithelial transplantation has been successfully used as corneal epithelial replacement in bilateral limbal stem cell deficiency. Recently, lotrafilcon A contact lens (CL) surface was described as a suitable carrier for cultured stem cells in corneal epithelial transplantation. Our aim was to establish explant cultures from human OM on CL carriers that are free of animal-derived materials and feeder cells. MATERIALS AND METHODS: Human cadaveric 2 mm OM explants were sutured onto CL surfaces and cultivated with fetal calf serum (FCS) or human serum (HS) supplemented culture medium without feeder cells...
March 24, 2017: Current Eye Research
https://www.readbyqxmd.com/read/28338672/posttransplant-maintenance-therapy-in-multiple-myeloma-the-changing-landscape
#11
REVIEW
S Sengsayadeth, F Malard, B N Savani, L Garderet, M Mohty
Transplant-eligible patients with multiple myeloma (MM) now have extended survival after diagnosis owing to effective modern treatment strategies that include new agents in induction therapy, autologous stem cell transplant (ASCT), consolidation therapy and posttransplant maintenance therapy. Standard of care for newly diagnosed, fit patients includes ASCT and, often nowadays, posttransplant maintenance. Several large studies have shown the efficacy of maintenance with thalidomide, lenalidomide and bortezomib in the treatment scheme of MM with regards to prolonging progression-free survival and, to a lesser degree, overall survival...
March 24, 2017: Blood Cancer Journal
https://www.readbyqxmd.com/read/28337445/rapid-chondrocyte-isolation-for-tissue-engineering-applications-the-effect-of-enzyme-concentration-and-temporal-exposure-on-the-matrix-forming-capacity-of-nasal-derived-chondrocytes
#12
Srujana Vedicherla, Conor Timothy Buckley
Laboratory based processing and expansion to yield adequate cell numbers had been the standard in Autologous Disc Chondrocyte Transplantation (ADCT), Allogeneic Juvenile Chondrocyte Implantation (NuQu®), and Matrix-Induced Autologous Chondrocyte Implantation (MACI). Optimizing cell isolation is a key challenge in terms of obtaining adequate cell numbers while maintaining a vibrant cell population capable of subsequent proliferation and matrix elaboration. However, typical cell yields from a cartilage digest are highly variable between donors and based on user competency...
2017: BioMed Research International
https://www.readbyqxmd.com/read/28336110/patient-generated-subjective-global-assessment-as-a-prognosis-tool-in-patients-with-multiple-myeloma
#13
Hae Su Kim, Ji Yun Lee, Sung Hee Lim, Jaewon Cho, Seok Jin Kim, Jun Ho Jang, Won Seog Kim, Chul Won Jung, Kihyun Kim
OBJECTIVE: Disease-related weight loss is relatively common in patients with newly diagnosed multiple myeloma (MM), but limited data exist regarding the effects of nutritional status on survival. The aim of this study was to assess the relationship between malnutrition (as measured by Patient-Generated Subjective Global Assessment [PG-SGA]) and clinical characteristics of patients with MM, and to investigate the association between the PG-SGA score before chemotherapy and overall survival in MM patients...
April 2017: Nutrition
https://www.readbyqxmd.com/read/28334700/single-agent-pixantrone-as-a-bridge-to-autologous-stem-cell-transplantation-in-a-patient-with-refractory-diffuse-large-b-cell-lymphoma
#14
Lorena Appio, Carlo Landoni, Maria La Targia, Vanda Bertolli, Martina Chiarucci, Giovanni Crovetti, Elisabetta Vassenna, Giovanni Serio, Marco Bregni
Aggressive non-Hodgkin lymphoma is associated with poor long-term survival after relapse or resistance to chemotherapy. We report a case of aggressive non-Hodgkin lymphoma refractory to first-line R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone) and second-line R-DHAP (rituximab, dexamethasone, cytarabine, and cisplatin) chemotherapy treatments. The patient achieved remission with single-agent pixantrone, and received a consolidation with high-dose BEAM (BCNU, etoposide, cytarabine, and melphalan) chemotherapy and autologous stem cell transplantation...
March 24, 2017: Chemotherapy
https://www.readbyqxmd.com/read/28333539/age-is-not-a-contraindication-for-cartilage-surgery-a-critical-analysis-of-standardized-outcomes-at-long-term-follow-up
#15
Giuseppe Filardo, Luca Andriolo, Andrea Sessa, Francesca Vannini, Alberto Ferruzzi, Maurilio Marcacci, Elizaveta Kon
BACKGROUND: Age is commonly accepted as a negative prognostic factor in cartilage surgery, and cutoff values of 30 to 40 years have been previously suggested as treatment indication. However, the lower outcome scores documented in older patients do not take in consideration the decreasing requirements of an aging joint. PURPOSE: To analyze the real effect of age in terms of recovery with respect to the functional level expected for different age categories of patients treated for cartilage lesions...
March 1, 2017: American Journal of Sports Medicine
https://www.readbyqxmd.com/read/28332991/accelerated-total-lymphoid-irradiation-containing-salvage-regimen-for-patients-with-refractory-and-relapsed-hodgkin-lymphoma-20%C3%A2-years-of-experience
#16
Andreas Rimner, Shona Lovie, Meier Hsu, Monica Chelius, Zhigang Zhang, Karen Chau, Alison J Moskowitz, Matthew Matasar, Craig H Moskowitz, Joachim Yahalom
PURPOSE: We report the long-term results of integrated accelerated involved field radiation therapy (IFRT) followed by total lymphoid irradiation (TLI) as part of the high-dose salvage regimen followed by autologous bone marrow transplantation or autologous stem cell transplantation in patients with relapsed or refractory Hodgkin lymphoma (HL). METHODS AND MATERIALS: From November 1985 to July 2008, 186 previously unirradiated patients with relapsed or refractory HL underwent salvage therapy on 4 consecutive institutional review board-approved protocols...
April 1, 2017: International Journal of Radiation Oncology, Biology, Physics
https://www.readbyqxmd.com/read/28332063/decreased-interaction-between-zo-1-and-occludin-is-involved-in-alteration-of-tight-junctions-in-transplanted-epiphora-submandibular-glands
#17
Chong Ding, Xin Cong, Xue-Ming Zhang, Sheng-Lin Li, Li-Ling Wu, Guang-Yan Yu
Tight junctions (TJs) in salivary epithelium play an important role in regulating saliva secretion. Autologous transplantation of submandibular glands (SMGs) is an effective method to treat severe dry eye syndrome. However, epiphora occurs in some patients 6 months after transplantation. We previously found that the acinar TJs are enlarged in rabbit SMGs after long-term transplantation, but the exact TJ components involved in the epiphora are still unknown. Here, we found that the mRNA and protein expression of ZO-1 and occludin were increased in the transplanted SMGs obtained from epiphora patients, while other TJs were unchanged...
March 22, 2017: Journal of Molecular Histology
https://www.readbyqxmd.com/read/28331981/vaccination-in-oncology-practice-and-predictors
#18
Ali Alkan, Ebru Karcı, Arzu Yaşar, Gülseren Tuncay, Elif Berna Köksoy, Muslih Ürün, Filiz Çay Şenler, Ahmet Demirkazık, Güngör Utkan, Hakan Akbulut, Yüksel Ürün
PURPOSE: Despite, vaccination is a highly effective and widely recommended for prevention of certain infections, vaccination coverage is very low. The purposes of this study were to evaluate the attitudes of medical oncologists towards vaccination and to identify predictors of intention to recommend vaccination in patients with cancer. METHODS: A structured questionnaire is formed to evaluate the daily practice of vaccination. Turkish medical oncologists were invited to this study via email, SMS, or phone call...
March 22, 2017: Supportive Care in Cancer: Official Journal of the Multinational Association of Supportive Care in Cancer
https://www.readbyqxmd.com/read/28331132/orbital-malt-lymphoma-after-autologous-stem-cell-transplantation-for-follicular-lymphoma-as-relapse-of-diffuse-large-b-cell-lymphoma
#19
Toshihiko Matsuo, Takehiro Tanaka, Nobuharu Fujii
We report a patient who developed orbital MALT lymphoma after autologous peripheral blood stem cell transplantation for follicular lymphoma as relapse of diffuse large B-cell lymphoma. A 54-year-old woman with systemic lymphadenopathy was diagnosed with diffuse large B-cell lymphoma by left supraclavicular lymph node biopsy, and underwent 6 courses of R-CHOP chemotherapy with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisolone, leading to complete response. Five years later in the follow-up, an abdominal mass with abnormal uptake was found by whole-body 2-[(18)F]fluoro-2-deoxy-D-glucose positron emission tomography, and computed tomography-guided biopsy demonstrated follicular lymphoma...
2017: Journal of Clinical and Experimental Hematopathology: JCEH
https://www.readbyqxmd.com/read/28330512/evaluating-risk-factors-for-clostridium-difficile-infection-in-stem-cell-transplant-recipients-a-national-study
#20
Nishi N Shah, William McClellan, Christopher R Flowers, Sagar Lonial, Hannah Khoury, Edmund K Waller, Amelia Langston, Ajay K Nooka
OBJECTIVE Large-scale studies evaluating risk factors for Clostridium difficile infection (CDI), a leading cause of infectious diarrhea among patients undergoing stem cell transplantation (SCT), are lacking. We have evaluated risk factors for CDI among both autologous SCT (auto-SCT), and allogeneic SCT (allo-SCT) recipients using the National Inpatient Sample (NIS) database provided by the Healthcare Cost and Utilization Project (HCUP). METHODS We used patient data obtained from the NIS database for all adult patients admitted for auto- and allo-SCTs from January 2001 to December 2010...
March 23, 2017: Infection Control and Hospital Epidemiology
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