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Swati Shrivastava, Mayank Gupta, Randall Sequeira, Bhaarat Singh, Guru Kumar Sain, Sunil Mahavar, Arvind Palawat, Raman Sharma
No abstract text is available yet for this article.
July 2016: Journal of the Association of Physicians of India
Bagirath Gangadharan, Mathieu Ing, Sandrine Delignat, Ivan Peyron, Maud Teyssandier, Srinivas V Kaveri, Sébastien Lacroix-Desmazes
The development of inhibitory antibodies to therapeutic FVIII is the major complication of replacement therapy in patients with hemophilia A. The first step in the initiation of the anti-FVIII immune response is FVIII interaction with receptor(s) on antigen-presenting cells followed by endocytosis and presentation to naive CD4+ T cells. Recent studies indicate a role for the C1 domain in FVIII uptake. We investigated whether charged residues in the C2 domain participate in immunogenic FVIII uptake. Co-incubation of FVIII with BO2C11, a monoclonal C2-specific IgG, reduced FVIII endocytosis by dendritic cells and presentation to CD4+ T cells, and diminished FVIII immunogenicity in FVIII-deficient mice...
October 6, 2016: Haematologica
Clemens Feistritzer, Stefan Schmidt
During the 57(th) annual meeting of the American Society of Hematology 2015 in Orlando, Florida, various aspects in the field of hemostaseology were presented. The Choosing Wisely® campaign pointed out the importance of the critical use of diagnostic tools to rule out pulmonary embolism and questioned the relevance of thrombophilia testing in women undergoing routine infertility evaluation. Furthermore, the approval of idarucizumab, a specific antidote for the reversal of the anticoagulant effects of the direct thrombin inhibitor dabigatran, was highlighted...
2016: Memo
Claude T Tagny, Pauline N Balôgôg, Francoise Boehlen, Dora Mbanya
In Cameroon, the Yaoundé Hemophilia Treatment Center (HTC) has so far recorded 121 cases of hemophilia and only 10 cases of von Willebrand disease (VWD). We report the case of a 16-year-old girl, living in the north of Cameroon. She presented with severe meno-metrorrhagia, which had increased drastically within the past 3 months, associated more recently by gum bleeding and epistaxis. The coagulation screen showed a prolonged bleeding time. The clinical profile of this young girl and the findings of less than 5% for von Willebrand factor (VWF):Ag and 10% for VWF Ristocetin cofactor suggests a type 3 VWD...
October 2016: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
Edward Tuddenham
The declarative title is a fair summary of research described in this paper (Targeting Factor VIII expression to platelets for hemophilia A gene therapy does not induce an apparent thrombotic risk in mice)(10) , is a fair summary of research described in this paper, which is itself the 14th in a series from the group of researchers led by Montgomery who, since 2003 have progressively advanced the idea of storing factor VIII in platelets to treat hemophilia A. Why, one might ask, would one do such a thing when evolution has selected endothelial cells as both the site for synthesis and release of factor VIII complexed with Von Willebrand factor to provide the cofactor when and where needed?(1,2) The answer lies in the unfortunate fact that a high proportion of patients with hemophilia develop resistance to factor VIII due to alloimmunity to the protein, which is foreign to their immune system, leading to production of inhibitory antibodies to the cofactor...
October 17, 2016: Journal of Thrombosis and Haemostasis: JTH
G N Nguyen, L A George, J I Siner, R J Davidson, C B Zander, X L Zheng, V R Arruda, R M Camire, D E Sabatino
BACKGROUND: The major challenge for developing gene-based therapies for hemophilia A is that human factor VIII (hFVIII) has intrinsic properties that result in inefficient biosynthesis. During intracellular processing, hFVIII is predominantly cleaved at a Paired basic Amino acid Cleaving Enzyme (PACE) or furin cleavage site to yield a heterodimer that is the major form of secreted protein. Previous studies with B-domain deleted (BDD) canine FVIII and hFVIII-R1645H, both differing from hFVIII by a single amino acid at this site, suggested that these proteins are secreted mainly in a single polypeptide chain (SC) form and exhibit enhanced function...
October 17, 2016: Journal of Thrombosis and Haemostasis: JTH
Julie Ann Smith
Hemophilia will be encountered in the oral and maxillofacial surgeon's office. A thorough understanding of hemophilia is necessary to safely care for these patients. One must understand the severity of the patient's hemophilia as well as whether or not inhibitors are present. The patient's surgical management will be influenced by these two factors. In addition to the possible need to transfuse factors or desmopressin, special care must be taken perioperatively to avoid bleeding complications. This article reviews the overall management of hemophilia A and B as well as the specific perioperative management of these patients...
November 2016: Oral and Maxillofacial Surgery Clinics of North America
Kimona Issa, Todd P Pierce, Steven F Harwin, Anthony J Scillia, Anthony Festa, Michael A Mont
BACKGROUND: HIV is prevalent worldwide and numerous patients with this diagnosis ultimately may become candidates for TKA. Although some studies have suggested that complications are more common in patients with HIV who undergo TKA, these studies largely were done before the contemporary era of HIV management; moreover, it is unclear whether patients with HIV achieve lower patient-reported outcome scores or inferior implant survivorship. QUESTIONS/PURPOSES: We asked whether there were any differences in the outcomes of patients with HIV without hemophilia who undergo TKA compared with a matched control cohort in terms of: (1) patient-reported outcomes; (2) implant survivorship; and (3) complication rates...
October 14, 2016: Clinical Orthopaedics and related Research
S M Johnson, G M Garnett, R K Woo
Maintenance of central venous access in patients with chronic medical conditions such as short bowel syndrome demands forethought and ingenuity. We describe an innovative technique for re-utilizing central venous access sites in patients who have chronic central venous access needs. Records of patients undergoing this technique were reviewed between August 2012 and December 2015. The technique involves "cutting-down" to the sterile fibrous tunnel that naturally forms around tunneled catheters. The fibrous sheath is then isolated and controlled much as would be done for a venous "cut-down...
October 14, 2016: Pediatric Surgery International
Amy M Lange, Ekaterina S Altynova, Giang N Nguyen, Denise E Sabatino
Factor VIII (FVIII) is a large glycoprotein that is challenging to express both in vitro and in vivo. Several studies suggest that high levels of FVIII expression can lead to cellular stress. After gene transfer, transgene expression is restricted to a subset of cells and the increased FVIII load per cell may impact activation of the unfolded protein response. We sought to determine whether increased FVIII expression in mice after adeno-associated viral liver gene transfer would affect the unfolded protein response and/or immune response to the transgene...
2016: Molecular Therapy. Methods & Clinical Development
Irene Zolotukhin, David M Markusic, Brett Palaschak, Brad E Hoffman, Meera A Srikanthan, Roland W Herzog
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively. Gene therapy for hemophilia with adeno-associated virus vectors has shown efficacy in hemophilia B patients. Although hemophilia A patients are more prevalent, the development of therapeutic adeno-associated virus vectors has been impeded by the size of the F8 cDNA and impaired secretion of FVIII protein. Further, it has been reported that over-expression of the FVIII protein induces endoplasmic reticulum stress and activates the unfolded protein response pathway both in vitro and in hepatocytes in vivo, presumably due to retention of misfolded FVIII protein within the endoplasmic reticulum...
2016: Molecular Therapy. Methods & Clinical Development
(no author information available yet)
No abstract text is available yet for this article.
October 13, 2016: Blood
Iris van Moort, Marieke Joosten, Moniek P M de Maat, Frank W G Leebeek, Marjon H Cnossen
Measurements of factor VIII coagulation activity (FVIII:C) may vary and result in misclassification of hemophilia A with delay in initiation of prophylactic treatment. We describe two young brothers who were diagnosed as moderate hemophilia patients and therefore not prophylactically treated with factor VIII concentrate despite frequent bleeding events. These findings emphasize the importance of (i) multiple measurements of FVIII:C by certified laboratories, (ii) adjustment of treatment when test results do not correspond to clinical symptoms, (iii) relevance of additional DNA mutation analysis in patients with hemophilia A, and (iv) treatment in centers with expertise...
October 13, 2016: Pediatric Blood & Cancer
Joshua I Siner, Benjamin J Samelson-Jones, Julie M Crudele, Robert A French, Benjamin J Lee, Shanzhen Zhou, Elizabeth Merricks, Robin Raymer, Timothy C Nichols, Rodney M Camire, Valder R Arruda
Processing by the proprotein convertase furin is believed to be critical for the biological activity of multiple proteins involved in hemostasis, including coagulation factor VIII (FVIII). This belief prompted the retention of the furin recognition motif (amino acids 1645-1648) in the design of B-domain-deleted FVIII (FVIII-BDD) products in current clinical use and in the drug development pipeline, as well as in experimental FVIII gene therapy strategies. Here, we report that processing by furin is in fact deleterious to FVIII-BDD secretion and procoagulant activity...
October 6, 2016: JCI Insight
Peter S Fong, Devon M Flynn, Christopher D Evans, P Todd Korthuis
Integrase strand transfer inhibitors (INSTIs) have become integral antiretroviral therapy (ART) agents for treating HIV infection. We report the case of a 44-year-old male with a history of hemophilia A who developed diabetes mellitus four months after switching from abacavir, lamivudine, and efavirenz to abacavir, lamivudine, and raltegravir. Hemoglobin A1C normalized without further need for exogenous insulin after raltegravir was switched back to efavirenz. In this case report, we will review a possible mechanism for INSTI-induced hyperglycemia and/or diabetes mellitus...
October 12, 2016: International Journal of STD & AIDS
Tomoko Matsumoto, Keiji Nogami, Midori Shima
Patients with mild/moderate hemophilia (H)A, acquired HA (AHA) and lupus anticoagulants (LA), have prolonged aPTTs with low levels of factor (F)VIII activity, but the differentiation of these disorders is complex and time consuming. We established an approach to quickly differentiate these disorders using comprehensive coagulation tests. Patients' plasmas with mild/moderate HA, AHA, LA without anti-phospholipid syndrome [LA-APS(-)], and LA with APS [LA-APS(+)] were examined using clot waveform analysis (CWA) and thrombin generation test (TGT)...
October 11, 2016: International Journal of Hematology
Slawomir Gajda, Tomasz Szopiński, Andrzej B Szczepanik, Roman Sosnowski, Anna M Szczepanik
Surgery in patients with hemophilia is a serious challenge. It requires a comprehensive approach, as well as careful postoperative monitoring. We present here the first case of a transperitoneal laparoscopic radical nephrectomy (TLRN) for renal cell carcinoma, of the clear-cell type, performed in a hemophilia B patient. The level of factor IX clotting activity before surgery and on postoperative days 1-6 was maintained at 65-130% and at 30-40% on subsequent days until healing of the post-operative wound was achieved...
2016: Central European Journal of Urology
Elie Dolgin
No abstract text is available yet for this article.
October 11, 2016: Nature Biotechnology
Masoume Rambod, Farkhondeh Sharif, Zahra Molazem, Kate Khair
BACKGROUND: Pain, as a crucial subsequence of joint hemorrhages in hemophilia patients, is chronic, debilitating, and distracting. This study aimed to describe and interpret pain experiences of hemophilia patients in their lives. METHODS: This qualitative study with hermeneutic phenomenological approach was conducted on fourteen hemophilia patients who had been referred to a hemophilia center affiliated to Shiraz University of Medical Sciences, Shiraz, Iran. The study question was "what is the meaning of pain in hemophilia patients' lives? The data were collected through semi-structured interviews and field notes through purposeful sampling...
October 2016: International Journal of Community Based Nursing and Midwifery
Anirban Basu, Prasun Subedi, Sachin Kamal-Bahl
BACKGROUND: Financing medical breakthroughs or cures is becoming increasingly challenging in the current fiscal environment. OBJECTIVES: In this paper, we develop the precise conditions needed for a financing mechanism, HealthCoin, to work between a private payer and Medicare, to incentivize the former to invest in breakthrough therapies or cures in the US. METHODS: We illustrate the valuation of such a currency for a cure of Type 2 diabetes...
September 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
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