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https://www.readbyqxmd.com/read/28738402/biodistribution-of-liver-derived-mesenchymal-stem-cells-after-peripheral-injection-in-a-hemophilia-a-patient
#1
Etienne M Sokal, Catherine Anne Lombard, Véronique Roelants, Mustapha Najimi, Sharat Varma, Camillo Sargiacomo, Joachim Ravau, Giuseppe Mazza, François Jamar, Julia Versavau, Vanessa Jacobs, Marc Jacquemin, Stéphane Eeckhoudt, Catherine Lambert, Xavier Stéphenne, Françoise Smets, Cédric Hermans
BACKGROUND: With the exception of liver transplantation, there is no cure for hemophilia, which is currently managed by preemptive replacement therapy. Liver-derived stem cells are in clinical development for inborn and acquired liver diseases and could represent a curative treatment for hemophilia A. The liver is a major factor VIII (FVIII) synthesis site, and mesenchymal stem cells have been shown to control joint bleeding in animal models of hemophilia. Adult-derived human liver stem cells (ADHLSCs) have mesenchymal characteristics and have been shown able to engraft in and repopulate both animal and human livers...
August 2017: Transplantation
https://www.readbyqxmd.com/read/28737986/real-world-adherence-and-persistence-to-oral-disease-modifying-therapies-in-multiple-sclerosis-patients-over-1-year
#2
Kristen M Johnson, Huanxue Zhou, Feng Lin, John J Ko, Vivian Herrera
BACKGROUND: Disease-modifying therapies (DMTs) are indicated to reduce relapse rates and slow disease progression for relapsing-remitting multiple sclerosis (MS) patients when taken as prescribed. Nonadherence or non-persistence in the real-world setting can lead to greater risk for negative clinical outcomes. Although previous research has demonstrated greater adherence and persistence to oral DMTs compared with injectable DMTs, comparisons among oral DMTs are lacking. OBJECTIVE: To compare adherence, persistence, and time to discontinuation among MS patients newly prescribed the oral DMTs fingolimod, dimethyl fumarate, or teriflunomide...
August 2017: Journal of Managed Care & Specialty Pharmacy
https://www.readbyqxmd.com/read/28737602/treatment-of-congenital-thrombotic-thrombocytopenia-purpura-a-new-paradigm
#3
Louis M Aledort, Tammuella C Singleton, Paula J Ulsh
Congenital thrombotic thrombocytopenia purpura (cTTP) is a very rare disorder worldwide. Standard treatment of recognized cases has been to administer fresh frozen plasma as the source of ADAMTS13, to replenish the absent ADAMTS13 enzyme. An alternative source, a plasma-derived factor VIII concentrate used for hemophilia A, and found to contain this enzyme, was reported to be effective in 1 patient in the United States. We now report details on a US cohort of 8 cTTP patients who have been successfully treated for varying periods with a marketed antihemophilic factor concentrate Koate-DVI...
July 21, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28736738/preserving-and-restoring-bone-with-continuous-insulin-infusion-therapy-in-a-mouse-model-of-type-1-diabetes
#4
Jeffry S Nyman, Evangelia Kalaitzoglou, R Clay Bunn, Sasidhar Uppuganti, Kathryn M Thrailkill, John L Fowlkes
Those with type 1 diabetes (T1D) are more likely to suffer a fracture than age- and sex-matched individuals without diabetes, despite daily insulin therapy. In rodent studies examining the effect of bone- or glucose-targeting therapies on preventing the T1D-related decrease in bone strength, insulin co-therapy is often not included, despite the known importance of insulin signaling to bone mass accrual. Therefore, working toward a relevant pre-clinical model of diabetic bone disease, we assessed the effect of continuous subcutaneous insulin infusion (CSII) therapy at escalating doses on preserving bone and the effect of delayed CSII on rescuing the T1D-related bone deterioration in an established murine model of T1D...
December 2017: Bone Reports
https://www.readbyqxmd.com/read/28734965/enhanced-immunostimulatory-activity-of-in-silico-discovered-agonists-of-toll-like-receptor-2-tlr2
#5
M S Murgueitio, S Ebner, P Hörtnagl, C Rakers, R Bruckner, P Henneke, G Wolber, S Santos-Sierra
BACKGROUND: Emergent therapies in anticancer vaccination use Toll-like receptors (TLRs) agonists as dendritic cell (DC) vaccine adjuvants. DCs from the patient are isolated, stimulated with TLR agonists and tumor antigens ex vivo and then infused back into the patient. Although some TLR ligands have been tested in clinical trials, novel TLR agonists with improved immunomodulatory properties are essential to optimize treatment success. We report on the discovery of small-molecule TLR2 agonists, with favorable properties as synthetic adjuvants...
July 19, 2017: Biochimica et Biophysica Acta
https://www.readbyqxmd.com/read/28734084/thrombolysis-for-pulmonary-embolism-using-the-superficial-veins-of-the-upper-extremity
#6
Joseph Kusick, Judd Salamat, Kintur Sanghvi
This report describes a novel approach of accessing the superficial veins of the upper extremity for the treatment of pulmonary emboli (PE) with an ultrasound enhanced thrombolytic infusion catheter. In a patient suffering from saddle PE and impending right heart failure, separate basilic and cephalic venous access sites in the right arm were utilized to insert EKOS EndoWave Infusion Catheter (EKOS Corporation, Bothell, WA) insertion in each pulmonary artery (PA). This technique could be advantageous, particularly for patients at high risk for access site complications and those unable to lie supine for prolonged duration of infusion therapy...
July 22, 2017: Catheterization and Cardiovascular Interventions
https://www.readbyqxmd.com/read/28733926/perioperative-management-of-endocrine-insufficiency-after-total-pancreatectomy-for-neoplasia
#7
REVIEW
Ajay V Maker, Raashid Sheikh, Vinita Bhagia
PURPOSE: Indications for total pancreatectomy (TP) have increased, including for diffuse main duct intrapapillary mucinous neoplasms of the pancreas and malignancy; therefore, the need persists for surgeons to develop appropriate endocrine post-operative management strategies. The brittle diabetes after TP differs from type 1/2 diabetes in that patients have absolute deficiency of insulin and functional glucagon. This makes glucose management challenging, complicates recovery, and predisposes to hospital readmissions...
July 21, 2017: Langenbeck's Archives of Surgery
https://www.readbyqxmd.com/read/28732314/the-effect-of-low-dose-furosemide-in-critically-ill-patients-with-early-acute-kidney-injury-a-pilot-randomized-blinded-controlled-trial-the-spark-study
#8
Sean M Bagshaw, R T Noel Gibney, Peter Kruger, Imran Hassan, Finlay A McAlister, Rinaldo Bellomo
PURPOSE: Furosemide is commonly prescribed in acute kidney injury (AKI). Prior studies have found conflicting findings on whether furosemide modifies the course and outcome of AKI. METHODS: Pilot multi-center randomized blinded placebo-controlled trial in adult patients with AKI admitted to three intensive care units. Participants were randomly allocated to furosemide bolus and infusion or 0.9% saline placebo. Primary endpoint was worsening AKI, defined by the RIFLE criteria...
July 12, 2017: Journal of Critical Care
https://www.readbyqxmd.com/read/28731841/survey-of-nutrition-management-practices-in-centers-for-pediatric-intestinal-rehabilitation
#9
Anita M Nucci, Kipp Ellsworth, Austin Michalski, Emily Nagel, Jackie Wessel
BACKGROUND: Nutrition management of pediatric intestinal failure (IF) requires interdisciplinary coordination of parenteral nutrition (PN) and enteral nutrition (EN) support. Nutrition strategies used by specialists in pediatric intestinal rehabilitation to promote gut adaptation and manage complications have not been previously summarized. METHODS: A practice survey was distributed to members of the dietitian subgroup of the American Society for Parenteral and Enteral Nutrition Pediatric Intestinal Failure Section...
July 1, 2017: Nutrition in Clinical Practice
https://www.readbyqxmd.com/read/28729970/severe-cyclophosphamide-related-hyponatremia-in-a-patient-with-acute-glomerulonephritis
#10
Pasquale Esposito, Maria Valentina Domenech, Nicoletta Serpieri, Marta Calatroni, Ilaria Massa, Alessandro Avella, Edoardo La Porta, Luca Estienne, Elena Caramella, Teresa Rampino
Cyclophosphamide is frequently used to treat cancer, autoimmune and renal diseases, such as rapidly progressive glomerulonephritis. Its side effects are well-known, including bone marrow depression, infections, alopecia, sterility, bladder malignancy and hemorrhagic cystitis. Moreover, in some cases cyclophosphamide use has been related to the onset of hyponatremia, by development of a syndrome of inappropriate antidiuresis. Indeed, severe hyponatremia has been previously reported in patients treated with high-dose or moderate-dose of intravenous cyclophosphamide, while only few cases have been reported in patients treated with low dose...
July 6, 2017: World Journal of Nephrology
https://www.readbyqxmd.com/read/28729364/tgf-%C3%AE-transforming-growth-factor-%C3%AE-signaling-protects-the-thoracic-and-abdominal-aorta-from-angiotensin-ii-induced-pathology-by-distinct-mechanisms
#11
Stoyan N Angelov, Jie Hong Hu, Hao Wei, Nathan Airhart, Minghui Shi, David A Dichek
OBJECTIVE: The role of TGF-β (transforming growth factor-β) signaling in abdominal aortic aneurysm (AAA) formation is controversial. Others reported that systemic blockade of TGF-β by neutralizing antibodies accelerated AAA development in angiotensin II-infused mice. This result is consistent with other studies suggesting that TGF-β signaling prevents AAA. Development of a therapy for AAA that exploits the protective actions of TGF-β would be facilitated by identification of the mechanisms through which TGF-β prevents AAA...
July 20, 2017: Arteriosclerosis, Thrombosis, and Vascular Biology
https://www.readbyqxmd.com/read/28728148/the-role-of-cytotoxic-chemotherapy-in-advanced-pancreatic-neuroendocrine-tumors
#12
Sebastian Krug, Thomas M Gress, Patrick Michl, Anja Rinke
BACKGROUND: Pancreatic neuroendocrine tumors (PNETs) are rare neoplasms accounting for less than 5% of all pancreatic malignancies. These tumors are characterized by clinical and prognostical heterogeneity and are predominantly diagnosed in a metastatic stage. Cytotoxic chemotherapy, along with alkylating agents and antimetabolites as well as molecular targeted agents (everolimus, sunitinib), is used in the treatment of advanced PNETs. After the approval of lanreotide for unresectable PNETs, an additional therapeutic option has become available; however, the best sequence of therapies and patient stratification to different treatments remains challenging...
July 21, 2017: Digestion
https://www.readbyqxmd.com/read/28727609/atrial-natriuretic-peptide-a-potential-early-therapy-for-the-prevention-of-multiple-organ-dysfunction-syndrome-following-severe-trauma
#13
Rong-Wei Zhang, Li-Li Liu, Lan-Lan Zeng, Rui-Jie Li, Ye-Hua Shen, Bin Zhang, Zi-Zhuo Liu, Meng-Fei Chen, Sheng-Mao Jiang, Li-Bing Jiang, Xiao-Gang Zhao, Shou-Yin Jiang
Trauma remains a tremendous medical burden partly because of increased expenditure for the management of multiple organ dysfunction syndrome (MODS) developed during hospital stay. The intestinal barrier injury continues to be a second insult resulting in MODS which currently lacks efficient strategies for prevention. Recent studies have uncovered multi-organ protective benefits of atrial natriuretic peptide (ANP) in cardiovascular disease. However, the role of ANP in prevention of MODS following severe trauma has not been understood...
July 19, 2017: Shock
https://www.readbyqxmd.com/read/28727585/closed-continuous-irrigation-with-lidocaine-and-immediate-mobilization-for-treatment-of-pyogenic-tenosynovitis
#14
Rebecca Knackstedt, Joyce Tyler, Steven Bernard
Pyogenic flexor tenosynovitis treatment consists of either closed continuous irrigation with sterile saline or antibiotic solution, or open debridement and irrigation. These treatment approaches serve to resolve the infection, but are extremely painful and residual stiffness can be devastating to the patient. We describe herein our approach to managing pyogenic flexor tenosynovitis. To provide continuous irrigation, a butterfly catheter with needle removed is utilized with irrigation holes cut into the tubing...
July 19, 2017: Techniques in Hand & Upper Extremity Surgery
https://www.readbyqxmd.com/read/28726133/iron-protects-porcine-plasma-coagulation-kinetics-from-degradation-by-crotalus-atrox-venom
#15
Christine S Olver, Vance G Nielsen
While the administration of antivenom to treat hemotoxic snake bite injury remains the gold standard of therapy, we have demonstrated that modifying human fibrinogen with iron and carbon monoxide renders it resistant to fibrinogenolytic snake venom enzymes. In order to translate these findings into a possible biometal-based therapy complementary to antivenom administration, a preclinical model that possesses fibrinogen that closely mimics the human molecule in response to iron and carbon monoxide needed to be identified...
July 19, 2017: Biometals: An International Journal on the Role of Metal Ions in Biology, Biochemistry, and Medicine
https://www.readbyqxmd.com/read/28726113/glucocorticoid-sparing-effect-of-zoledronic-acid-in-sarcoid-hypercalcemia
#16
Mohammad Shafi Kuchay, Sunil Kumar Mishra, Beena Bansal, Khalid Jamal Farooqui, Lalitha Sekhar, Ambrish Mithal
INTRODUCTION: Glucocorticoids are regarded as first-line therapy in the management of hypercalcemia associated with sarcoidosis. However, prolonged glucocorticoid therapy leads to metabolic abnormalities, Cushingoid habitus, and impairment of bone health. This study demonstrates the efficacy and glucocorticoid-sparing effect of zoledronic acid in sarcoid hypercalcemia. METHODS: We present three patients with sarcoid hypercalcemia. They were successfully managed with oral glucocorticoids for many months...
December 2017: Archives of Osteoporosis
https://www.readbyqxmd.com/read/28725594/weekly-ascorbic-acid-infusion-in-castration-resistant-prostate-cancer-patients-a-single-arm-phase-ii-trial
#17
Torben K Nielsen, Martin Højgaard, Jon T Andersen, Niklas Rye Jørgensen, Bo Zerahn, Bent Kristensen, Trine Henriksen, Jens Lykkesfeldt, Kári J Mikines, Henrik E Poulsen
BACKGROUND: Ascorbic acid (AA) has in vivo cytotoxic properties at concentrations that can only be achieved through intravenous (IV) administration in humans. Treatment with intravenous AA is widely and increasingly used in complementary medicine despite a lack of clinical evidence for the efficacy of this treatment. METHODS: This non-comparative, single-center, phase II trial included patients with chemotherapy-naïve, metastatic castration-resistant prostate cancer (mCRPC) from an outpatient clinic to evaluate the efficacy and safety of IV AA therapy...
June 2017: Translational Andrology and Urology
https://www.readbyqxmd.com/read/28725494/apixaban-for-treatment-of-confirmed-heparin-induced-thrombocytopenia-a-case-report-and-review-of-literature
#18
Daniel E Ezekwudo, Rebecca Chacko, Bolanle Gbadamosi, Syeda Batool, Sussana Gaikazian, Theodore E Warkentin, Jo-Ann I Sheppard, Ishmael Jaiyesimi
BACKGROUND: Heparin-induced thrombocytopenia (HIT) is a life and limb-threatening condition caused by the binding of platelet-activating antibodies (IgG) to multimolecular platelet factor 4 (PF4)/heparin complexes because of heparin exposure. The by-product of this interaction is thrombin formation which substantially increases the risk of venous and/or arterial thromboembolism. Currently, only one anticoagulant, argatroban, is United States Food and Drug Administration-approved for management of HIT; however, this agent is expensive and can only be given by intravenous infusion...
2017: Experimental Hematology & Oncology
https://www.readbyqxmd.com/read/28724596/severe-destructive-nasopharyngeal-granulomatosis-with-polyangiitis-with-superimposed-skull-base-pseudomonas-aeruginosa-osteomyelitis
#19
Mitchell S von Itzstein, Jithma P Abeykoon, Daniel D Summerfield, Jennifer A Whitaker
Skull base osteomyelitis in the setting of granulomatosis with polyangiitis (GPA) is rare and entails significant diagnostic challenges. We present a case of a 65-year-old Caucasian man with a history of rheumatoid arthritis, off immunosuppression for 18 months, who presented with 2 years of chronic headaches, severe fatigue, saddle nose deformity and 20-kilogram unintentional weight loss. Maxillofacial CT revealed an extensive destructive sinonasal and erosive skull base process. Laboratory evaluation showed equivocal elevation of antiproteinase 3 antibodies with negative antineutrophil cytoplasmic antibody panel...
July 19, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28724573/a-single-dose-of-peripherally-infused-egfrviii-directed-car-t-cells-mediates-antigen-loss-and-induces-adaptive-resistance-in-patients-with-recurrent-glioblastoma
#20
Donald M O'Rourke, MacLean P Nasrallah, Arati Desai, Jan J Melenhorst, Keith Mansfield, Jennifer J D Morrissette, Maria Martinez-Lage, Steven Brem, Eileen Maloney, Angela Shen, Randi Isaacs, Suyash Mohan, Gabriela Plesa, Simon F Lacey, Jean-Marc Navenot, Zhaohui Zheng, Bruce L Levine, Hideho Okada, Carl H June, Jennifer L Brogdon, Marcela V Maus
We conducted a first-in-human study of intravenous delivery of a single dose of autologous T cells redirected to the epidermal growth factor receptor variant III (EGFRvIII) mutation by a chimeric antigen receptor (CAR). We report our findings on the first 10 recurrent glioblastoma (GBM) patients treated. We found that manufacturing and infusion of CAR-modified T cell (CART)-EGFRvIII cells are feasible and safe, without evidence of off-tumor toxicity or cytokine release syndrome. One patient has had residual stable disease for over 18 months of follow-up...
July 19, 2017: Science Translational Medicine
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