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https://www.readbyqxmd.com/read/28491266/point-of-care-ultrasonography-pocus-in-hemophilia-a-a-commentary-on-current-status-and-its-potential-role-for-improving-prophylaxis-management-in-severe-hemophilia-a
#1
REVIEW
Suchitra S Acharya, Brittny Rule, Omar McMillan, Thomas J Humphries
In patients with severe hemophilia A, recurrent bleeding into joints results in increased morbidity and reduced quality of life. Prophylaxis using replacement factor products, especially when initiated early, has established benefits in terms of reducing joint bleeds and preserving joint function. Poor adherence to prophylactic regimens is a common cause for breakthrough bleeds and resultant arthropathy. Improving prophylaxis management, especially in the transitional age group, is a challenge. Here, we discuss the current status of ultrasonography (US) in hemophilia A, challenges in its wider implementation, and the potential for use of point-of-care US (POCUS) as an adjunct in the routine management of patients with hemophilia following prophylaxis regimens...
April 2017: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/28470674/acquired-hemophilia-a-updated-review-of-evidence-and-treatment-guidance
#2
REVIEW
Rebecca Kruse-Jarres, Christine L Kempton, Francesco Baudo, Peter W Collins, Paul Knoebl, Cindy A Leissinger, Andreas Tiede, Craig M Kessler
Acquired hemophilia A (AHA) is a rare disease resulting from autoantibodies (inhibitors) against endogenous factor VIII (FVIII) that leads to bleeding, which is often spontaneous and severe. AHA tends to occur in elderly patients with comorbidities and is associated with high mortality risk from underlying comorbidities, bleeding, or treatment complications. Treatment, which consists of hemostatic management and eradication of the inhibitors, can be challenging to manage. Few data are available to guide the management of AHA-related bleeding and eradication of the disease-causing antibodies...
May 3, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28458935/successful-management-of-acquired-hemophilia-a-associated-with-bullous-pemphigoid-a-case-report-and-review-of-the-literature
#3
Quentin Binet, Catherine Lambert, Laurine Sacré, Stéphane Eeckhoudt, Cedric Hermans
Background. Acquired hemophilia A (AHA) is a rare condition, due to the spontaneous formation of neutralizing antibodies against endogenous factor VIII. About half the cases are associated with pregnancy, postpartum, autoimmune diseases, malignancies, or adverse drug reactions. Symptoms include severe and unexpected bleeding that may prove life-threatening. Case Study. We report a case of AHA associated with bullous pemphigoid (BP), a chronic, autoimmune, subepidermal, blistering skin disease. To our knowledge, this is the 25th documented case of such an association...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/28447409/factor-viii-inhibitors-advances-in-basic-and-translational-science
#4
REVIEW
J D Lai, D Lillicrap
In the treatment of hemophilia A, the 20%-30% risk of developing of anti-factor VIII (FVIII) antibodies, or inhibitors, is the dominant concern among healthcare providers. Immune tolerance induction remains the only effective method of eradicating inhibitors in approximately 75% of patients, but is accompanied by significant emotional and economical burden. While certain risk factors, such as the type of FVIII mutation, offer some insight, there remains no strategy to confidently predict the development of an inhibitor...
May 2017: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/28441921/acquired-hemophilia-a-a-review-of-recent-data-and-new-therapeutic-options
#5
Massimo Franchini, Stefania Vaglio, Giuseppe Marano, Carlo Mengoli, Sara Gentili, Simonetta Pupella, Giancarlo Maria Liumbruno
OBJECTIVES: Acquired hemophilia A (AHA) is a rare, but potentially life-threatening, bleeding disorder caused by an autoantibody against factor VIII that interferes with its coagulant function. METHODS: We performed a narrative review focusing on the diagnostic aspects of AHA and on the current treatment strategies with particular regard to new data and therapeutic developments. RESULTS: The management of this severe hemorrhagic disorder is based on the control of bleeding with the use of bypassing agents and on the utilization of a variety of immunosuppressant agents with the goal of eliminating the autoantibody permanently...
April 25, 2017: Hematology (Amsterdam, Netherlands)
https://www.readbyqxmd.com/read/28440011/intensity-of-factor-viii-treatment-and-the-development-of-inhibitors-in-nonsevere-hemophilia-a-patients-results-of-the-insight-case-control-study
#6
A S van Velzen, C L Eckhardt, M Peters, F W G Leebeek, C Escuriola-Ettingshausen, C Hermans, R Keenan, J Astermark, C Male, K Peerlinck, S le Cessie, J G van der Bom, K Fijnvandraat
BACKGROUND: Inhibitor development is a major complication of treatment with factor VIII concentrates in hemophilia. Findings from studies among severe hemophilia A patients suggest that intensive treatment episodes increase the risk to develop inhibitors. OBJECTIVES: We set out to assess whether intensive treatment is also associated with an increased risk of inhibitor development among nonsevere hemophilia A patients. PATIENTS/METHODS: We performed a nested case-control study...
April 25, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28395496/-causes-and-management-of-pediatric-spontaneous-tonsillar-haemorrhage
#7
Z B Wu, H G Pan, Y S Teng, D L Zhang, X Y Ma, S H Han, L Li
Objective: To analyze the causes and management plan of pediatric spontaneous tonsillar haemorrhage(STH). Methods: According to the criteria of STH difined by Griffies, patients with STH from December 2013 to January 2016 were included in this retrospective study. Results: A total of 11 patients were reviewed. The etiological diagnosis included 3 pediatric Epstein-Barr virus associated infectious mononucleosis(EBV-IM), 3 suspected pediatric EBV-IM to 3 acute suppurative tonsillitis, 1 acute viral tonsillitis and 1 hemophilia A...
March 7, 2017: Zhonghua Er Bi Yan Hou Tou Jing Wai Ke za Zhi, Chinese Journal of Otorhinolaryngology Head and Neck Surgery
https://www.readbyqxmd.com/read/28388863/perioperative-coagulation-management-of-a-hemophilia-a-patient-during-cardiac-surgery
#8
Patrick Odonkor, Archana Srinivas, Erik Strauss, Brittney Williams, Michael Mazzeffi, Kenichi A Tanaka
Perioperative management of cardiovascular surgical procedures requiring cardiopulmonary bypass (CPB) in patients with hemophilia A poses a clinical challenge in coagulation management. Use of CPB requires the administration of an anticoagulant, usually unfractionated heparin, and also causes dilutional coagulopathy, platelet dysfunction or platelet consumption coagulopathy. Hypothermia and activation of the inflammatory cascade also affect coagulation. The effects of CPB on circulating levels of factor VIII have not been clearly defined...
April 1, 2017: Seminars in Cardiothoracic and Vascular Anesthesia
https://www.readbyqxmd.com/read/28352491/acquired-factor-viii-deficiency-two-case-reports-and-a-review-of-literature
#9
Lan Mo, George C Bao
BACKGROUND: Acquired factor VIII (FVIII) deficiency, or acquired hemophilia A (AHA), is a rare autoimmune disorder involving antibody-mediated depletion of coagulation FVIII, leading to severe, life-threatening bleeding. The condition is often associated with other autoimmune disorders, and its treatment involves replacement of FVIII and various modes of immunosuppression. Recently, a few noteworthy therapeutic advances have been made. We present two cases of severe AHA in Chinese women...
2017: Experimental Hematology & Oncology
https://www.readbyqxmd.com/read/28332238/outcome-measures-for-adult-and-pediatric-hemophilia-patients-with-inhibitors
#10
REVIEW
Cedric Hermans, Günter Auerswald, Gary Benson, Gerry Dolan, Anne Duffy, Victor Jiménez-Yuste, Rolf Ljung, Massimo Morfini, Thierry Lambert, Mehdi Osooli, Silva Zupančić Šalek
Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15(th) Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population...
March 22, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/28323492/regulatory-and-exhausted-t-cell-responses-to-aav-capsid
#11
Gwladys Gernoux, James M Wilson, Christian Mueller
Recombinant adeno-associated viruses (AAVs) are quickly becoming the preferred viral vector for viral gene delivery for the treatment of a wide variety of genetic disorders. However, since their use in a clinical trial targeting hemophilia B patients 10 years ago, immune responses to the AAV capsid appear to have hampered some of the early clinical gene transfer efficacy. Indeed, AAV-based gene transfer has been shown to reactivate capsid-specific memory T cells, which have correlated with a decline in AAV-transduced tissue in some patients...
April 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28321438/a-case-of-hemophilia-a-presenting-in-a-neonate-and-a-review-of-the-literature
#12
Esther Kisseih, Neeraja Yerrapotu, Deepak Yadav, Melissa February
No abstract text is available yet for this article.
2017: Global Pediatric Health
https://www.readbyqxmd.com/read/28298793/perioperative-considerations-in-a-patient-with-hemophilia-a-a-case-report-and-review-of-literature
#13
Tuhin Mistry, Neelam Dogra, Kanchan Chauhan, Jigyasa Shahani
Classic hemophilia or hemophilia A is a congenital bleeding diathesis in which the affected individual may present with spontaneous hemorrhage or persistent bleeding even after minor trauma. Knowledge about the disease process, multidisciplinary team approach, and timely management can lead to favorable outcome in these patients. We report management of a child with hemophilia A for suturing of lacerated upper lip mucosa following trauma. A review of literature with recommendations for perioperative management, especially in the setting of emergency surgery, is also provided...
January 2017: Anesthesia, Essays and Researches
https://www.readbyqxmd.com/read/28295573/therapeutic-plasma-exchange-for-perioperative-management-of-patients-with-congenital-factor-xi-deficiency
#14
Mohamed S Alsammak, Aneel A Ashrani, Jeffrey L Winters, Rajiv K Pruthi
BACKGROUND: Factor XI (FXI) deficiency (hemophilia C [HEM-C]) is a bleeding disorder with unpredictable severity that correlates poorly with FXI coagulation activity (FXI:C). It poses a perioperative hemostatic management challenge. For US patients with severe disease, fresh frozen plasma (FFP) or, in current use, thawed plasma is the most readily available option but comes with risk of volume overload. We report our experience of using therapeutic plasma exchange (TPE) as an alternative perioperative management strategy...
March 15, 2017: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/28286423/oligonucleotides-targeting-coagulation-factor-mrnas-use-in-thrombosis-and-hemophilia-research-and-therapy
#15
REVIEW
Marco Heestermans, Bart J M van Vlijmen
Small interfering (si) RNAs and antisense oligonucleotides (ASOs; here for simplicity reasons, both referred to as oligonucleotides) are small synthetic RNA or DNA molecules with a sequence complementary to a (pre)mRNA. Although the basic mechanisms of action between siRNAs and ASO are distinct, a sequence-specific interaction of the both oligonucleotides with the target (pre)mRNA alters the target's fate, which includes highly effective sequence-specific blockade of translation and consequently depletion of the corresponding protein...
2017: Thrombosis Journal
https://www.readbyqxmd.com/read/28277160/production-of-recombinant-coagulation-factors-are-humans-the-best-host-cells
#16
Kamilla Swiech, Virgínia Picanço-Castro, Dimas Tadeu Covas
The main treatment option for Hemophilia A/B patients involves the administration of recombinant coagulation factors on-demand or in a prophylactic approach. Despite the safety and efficacy of this replacement therapy, the development of antibodies against the coagulation factor infused, which neutralize the procoagulant activity, is a severe complication. The production of recombinant coagulation factors in human cell lines is an efficient approach to avoid such complication. Human cell lines can produce recombinant proteins with post translation modifications more similar to their natural counterpart, reducing potential immunogenic reactions...
February 23, 2017: Bioengineered
https://www.readbyqxmd.com/read/28264199/factor-activity-assays-for-monitoring-extended-half-life-fviii-and-factor-ix-replacement-therapies
#17
REVIEW
Steve Kitchen, Stefan Tiefenbacher, Robert Gosselin
The advent of modified factor VIII (FVIII) and factor IX (FIX) molecules with extended half-lives (EHLs) compared with native FVIII and FIX represents a major advance in the field of hemophilia care, with the potential to reduce the frequency of prophylactic injections and/or to increase the trough level prior to subsequent injections. Monitoring treatment through laboratory assays will be an important part of ensuring patient safety, including any tailoring of prophylaxis. Several approaches have been used to extend half-lives, including PEGylation, and fusion to albumin or immunoglobulin...
April 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/28262242/an-extra-x-does-not-prevent-acquired-hemophilia-pregnancy-associated-acquired-hemophilia-a
#18
REVIEW
Assaf A Barg, Tami Livnat, Gili Kenet
Acquired hemophilia A (AHA) is a severe bleeding disorder caused by autoantibodies against clotting factor VIII (FVIII). With an estimated annual incidence of 1.3 to 1.5 per million, AHA is a rare disease. An extremely rare form of AHA has been described among women in the peripartum period, and may present with peripartum hemorrhage. Notably, although hemorrhagic symptoms commonly present 1-4 months around delivery, they may occur up to 1 year after parturition. When caring for a mother with AHA it is important to note that Factor VIII inhibitor may be transferred via the placenta from the mother to the fetus...
March 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28255432/response-to-treatment-and-adverse-events-associated-with-use-of-recombinant-activated-factor-vii-in-children-a-retrospective-cohort-study
#19
James D Cooper, Arthur K Ritchey
BACKGROUND: Recombinant activated factor VII (rFVIIa) is United States (US) Food and Drug Administration (FDA)-approved for patients with hemophilia with inhibitors or congenital factor VII deficiency. Initial reports of off-label use highlighted its efficacy, though newer reports have not repeated these findings. In both types of publication, though, secondary thromboses have been seen in adult patients. The data in children are less clear. METHODS: This study analyzed all rFVIIa use at a large children's hospital for characteristics and outcomes...
February 2017: Therapeutic Advances in Drug Safety
https://www.readbyqxmd.com/read/28238287/genome-editing-for-inborn-errors-of-metabolism-advancing-towards-the-clinic
#20
REVIEW
Jessica L Schneller, Ciaran M Lee, Gang Bao, Charles P Venditti
Inborn errors of metabolism (IEM) include many disorders for which current treatments aim to ameliorate disease manifestations, but are not curative. Advances in the field of genome editing have recently resulted in the in vivo correction of murine models of IEM. Site-specific endonucleases, such as zinc-finger nucleases and the CRISPR/Cas9 system, in combination with delivery vectors engineered to target disease tissue, have enabled correction of mutations in disease models of hemophilia B, hereditary tyrosinemia type I, ornithine transcarbamylase deficiency, and lysosomal storage disorders...
February 27, 2017: BMC Medicine
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