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https://www.readbyqxmd.com/read/29326244/gene-therapy-comes-of-age
#1
REVIEW
Cynthia E Dunbar, Katherine A High, J Keith Joung, Donald B Kohn, Keiya Ozawa, Michel Sadelain
After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engineered T cells or hematopoietic stem cells...
January 12, 2018: Science
https://www.readbyqxmd.com/read/29296916/thromboembolic-event-rate-in-patients-exposed-to-anti-inhibitor-coagulant-complex-a-meta-analysis-of-40-year-published-data
#2
Matteo Rota, Paolo A Cortesi, Roberto Crea, Alessandro Gringeri, Lorenzo G Mantovani
Anti-inhibitor coagulant complex (AICC), an activated prothrombin complex concentrate, has been available for the treatment of patients with inhibitors since 1977, and thromboembolic events (TEEs) have been reported after infusion of AICC in patients with congenital or acquired hemophilia. With the aim of estimating the TEE incidence rate (IR) related to AICC exposure in these patients, a systematic review of the literature was carried out in Medline, according to PRISMA guidelines, from inception date to March 2017...
December 12, 2017: Blood Advances
https://www.readbyqxmd.com/read/29296912/hemophilia-gene-therapy-comes-of-age
#3
REVIEW
Lindsey A George
Concurrent with the development of recombinant factor replacement products, the characterization of the F9 and F8 genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first "cure" in a large animal model of hemophilia B in 1998, first in human sustained vector-derived factor IX activity in 2011, and our clinical trial results reporting sustained vector-derived factor IX activity well into the mild or normal range in 2016...
December 12, 2017: Blood Advances
https://www.readbyqxmd.com/read/29284760/the-concept-of-biosimilars-from-characterization-to-evolution-a-narrative-review
#4
REVIEW
Fadi Farhat, Alfredo Torres, Wungki Park, Gilberto de Lima Lopes, Raja Mudad, Chukwuemeka Ikpeazu, Simon Abi Aad
Biologic agents are currently the fastest emerging segment of drug expenditure. Unlike chemically synthesized small-molecule drugs, biologics are more complex, medicinal products produced by a living organism. They have become part of the standard of care in the treatment of a large variety of diseases, such as growth disorders, autoimmune diseases, cancer, cardiovascular illnesses, hemophilia, and rare genetic conditions, to name a few. Biosimilars, which are copies of biologics that are highly similar, were introduced in the market with an aim to offer efficacy that is not clinically different from the originator or reference product, at lower prices...
December 28, 2017: Oncologist
https://www.readbyqxmd.com/read/29274196/musculoskeletal-ultrasound-for-intra-articular-bleed-detection-a-highly-sensitive-imaging-modality-compared-to-conventional-magnetic-resonance-imaging
#5
S Nguyen, X Lu, Y Ma, J Du, E Y Chang, A von Drygalski
BACKGROUND: There is increasing demand for musculoskeletal ultrasound (MSKUS) to detect hemophilic joint bleeding, but there is uncertainty regarding blood detection concentration thresholds or if magnetic resonance imaging (MRI) is more accurate. AIMS: Compare the sensitivity of blood detection by MSKUS and MRI. METHODS: Increasing blood concentrations in plasma were imaged with MSKUS and MRI 1-2 hours, 3-4 days and 7 days after blood withdrawal in vitro, and after injection into cadaveric pig joints...
December 23, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29259958/bone-density-status-in-bleeding-disorders-where-are-we-and-what-needs-to-be-done
#6
REVIEW
Hassan Mansouritorghabeh, Zahra Rezaieyazdi
Bleeding disorders, including hemophilia, can be seen in every ethnic population in the world. Among various bleeding disorders, reduced bone density has been addressed in hemophilia A. In recent years, there has been an increasing interest in addressing osteopenia and osteoporosis in hemophilia A. There is little or no study about the possible susceptibility of other individuals with bleeding disorders to reduced bone density. Questions have been raised about the role of blood coagulation factors in bone mineralization...
November 2017: Journal of Bone Metabolism
https://www.readbyqxmd.com/read/29258406/a-contemporary-look-at-fviii-inhibitor-development-still-a-great-influence-on-the-evolution-of-hemophilia-therapies
#7
Elena Santagostino, Guy Young, Manuel Carcao, Pier Mannuccio Mannucci, Susan Halimeh, Steve Austin
The development of inhibitors against factor VIII (FVIII) replacement therapy remains the most important challenge for clinicians in the treatment of hemophilia patients. This review focusses on risk factors and management of FVIII inhibitors, particularly in light of SIPPET study findings and subsequent analyses. Areas covered: A brief history and evolution of hemophilia therapies is provided, including an overview of conventional and new (including investigational) therapeutic approaches for the treatment of hemophilia...
December 19, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29222310/novel-alternate-hemostatic-agents-for-patients-with-inhibitors-beyond-bypass-therapy
#8
REVIEW
Margaret V Ragni
Inhibitor formation is among the most severe complications of hemophilia treatment. With a cumulative incidence of ∼30% in those with severe hemophilia A and ∼3% in those with severe hemophilia B, inhibitors are caused by a T-cell response directed against infused coagulation factor; these inhibitors neutralize factor VIII or IX activity and disrupt normal hemostasis. Inhibitor patients become unresponsive to standard factor treatment and, as an alternative, use bypass treatment (eg, recombinant factor VIIa or factor VIII inhibitor bypass activity)...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29222308/hemophilia-gene-therapy-comes-of-age
#9
REVIEW
Lindsey A George
Concurrent with the development of recombinant factor replacement products, the characterization of the F9 and F8 genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first "cure" in a large animal model of hemophilia B in 1998, first in human sustained vector-derived factor IX activity in 2011, and our clinical trial results reporting sustained vector-derived factor IX activity well into the mild or normal range in 2016...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29178638/living-with-a-rare-disorder-a-systematic-review-of-the-qualitative-literature
#10
REVIEW
Charlotte von der Lippe, Plata S Diesen, Kristin B Feragen
BACKGROUND: Individuals with rare diseases may face challenges that are different from those experienced in more common medical conditions. A wide range of different rare conditions has resulted in a myriad of studies investigating the specificities of the diagnosis in focus. The shared psychological experiences of individuals with a rare condition, however, have not been reviewed systematically. METHODS: We performed a systematic review, including qualitative studies on adults, published between 2000 and 2016...
November 2017: Molecular Genetics & Genomic Medicine
https://www.readbyqxmd.com/read/29178325/the-spectrum-of-bleeding-in-women-and-girls-with-haemophilia-b
#11
REVIEW
J Staber, S E Croteau, J Davis, E F Grabowski, P Kouides, R F Sidonio
Although hemophilia B affects 1 in 25,000 males there may be 3 female hemophilia B carriers per affected male. This clinical review highlights the unique challenges faced by hemophilia B carriers including the under-recognition of bleeding symptoms associated with and without FIX deficiency, discrepancies in correlation between genotype and bleeding phenotype and therapeutic considerations utilizing clinical vignettes of common scenarios.
November 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29178312/total-hip-arthroplasty-in-hemophilia-patients-a-mid-term-to-long-term-follow-up
#12
Guo-Liang Wu, Ji-Liang Zhai, Bin Feng, Yan-Yan Bian, Chi Xu, Xi-Sheng Weng
OBJECTIVE: To report the results of mid-term to long-term follow-up after primary total hip arthroplasty (THA) in hemophiliacs and to hypothesize that THA can provide satisfactory outcomes in these patients. METHODS: Twenty-four primary THA performed in 21 hemophilia patients between 2002 and 2012 were reviewed retrospectively, including 20 cases of hemophilia A and 1 case of hemophilia B. The standard lateral approach was used for all implantations. Substitution therapy for factor VIII and activated prothrombin complex concentrates were administered to patients with hemophilia A and B, respectively...
November 2017: Orthopaedic Surgery
https://www.readbyqxmd.com/read/29172774/trenonacog-alfa-for-prophylaxis-on-demand-and-perioperative-management-of-hemophilia-b
#13
Yvonne Brennan, Jennifer Curnow, Emmanuel J Favaloro
Current treatment for hemophilia B involves replacing the missing coagulation factor IX (FIX) with either plasma-derived or recombinant (r) FIX. Trenonacog alfa is the third normal half-life rFIX that has been granted FDA approval. Area covered: In this review, the authors examine trenonacog alfa for the treatment of hemophilia B including prophylaxis, on-demand and perioperative hemostasis. They compare the PK profile to nonacog alfa and evaluate the drug's efficacy and safety from published studies. Expert opinion: Trenonacog alfa appears to be an effective and safe treatment option for patients with hemophilia B with a PK profile similar to that of nonacog alfa...
November 26, 2017: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/29165739/inhibitors-in-patients-with-congenital-bleeding-disorders-other-than-hemophilia
#14
Massimo Franchini, Giuseppe Marano, Carlo Mengoli, Vanessa Piccinini, Simonetta Pupella, Stefania Vaglio, Giancarlo Maria Liumbruno
The most worrying complication of replacement therapy for severe hemophilia A and B is currently the occurrence of inhibitory alloantibodies against infused factor VIII and factor IX, respectively. Inhibitors compromise the management of hemorrhage in affected patients, with a considerable increase in complications, disability, and costs. While these alloantibodies have been extensively studied in the past years in hemophilia A and B, those occurring in patients with other inherited bleeding disorders are less well characterized and still poorly understood, mostly due to the rarity of these hemorrhagic conditions...
November 17, 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/29165738/alloantibodies-in-von-willebrand-disease
#15
Massimo Franchini, Pier Mannuccio Mannucci
von Willebrand disease (VWD), the most commonly known inherited bleeding disorder, is caused by a partial (type 1) or total (type 3) deficiency or dysfunction (type 2) of von Willebrand factor (VWF). Its management encompasses the prevention or treatment of bleeding by raising endogenous VWF levels using a synthetic agent, such as desmopressin, or providing exogenous VWF concentrates. The development of inhibitory alloantibodies against VWF is a rare but often severe complication encountered during the treatment of type 3 VWD, which is associated with a lack of hemostatic response to infused VWF concentrates and more rarely with allergic, even anaphylactic, reactions...
November 17, 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/29165342/knee-and-ankle-arthroplasty-in-hemophilia
#16
REVIEW
Luigi Piero Solimeno, Gianluigi Pasta
Today, major surgical procedures can be safely performed in hemophilic patients with chronic arthropathy, using available factor concentrates. In this setting, total knee replacement is considered the "gold standard", while the use of total ankle replacement is still debated. Indeed, the unsatisfactory results obtained with the previous available design of implants did not raise enthusiasm as knee or hip replacement. Recently, the introduction of new implant designs and better reported outcomes have renewed the interest in total ankle replacement in people with hemophilia...
November 22, 2017: Journal of Clinical Medicine
https://www.readbyqxmd.com/read/29159733/management-of-hemophilia-in-older-patients
#17
REVIEW
Massimo Franchini, Pier Mannuccio Mannucci
In recent decades, several improvements in hemophilia care have resulted in increased quality of life and life expectancy for those affected by this inherited hemorrhagic condition. Nowadays, individuals with hemophilia enjoy a life expectancy at birth close to that of males in the general population. As a consequence of the increasing age of the hemophilia population, a growing number of these patients develop age-related co-morbidities, such as cardiovascular disease and cancer, the management of which represents a new challenge for caregivers at hemophilia treatment centers...
November 20, 2017: Drugs & Aging
https://www.readbyqxmd.com/read/29153709/cytokines-in-the-pathogenesis-of-hemophilic-arthropathy
#18
REVIEW
Piotr Wojdasiewicz, Łukasz A Poniatowski, Paweł Nauman, Tomasz Mandat, Agnieszka Paradowska-Gorycka, Katarzyna Romanowska-Próchnicka, Dariusz Szukiewicz, Andrzej Kotela, Łukasz Kubaszewski, Ireneusz Kotela, Iwona Kurkowska-Jastrzębska, Robert Gasik
Hemophilic arthropathy (HA) is one of the most common and typical manifestation in the course of recurrent bleeding episodes in patients with hemophilia. Clinical and subclinical joint bleeding episodes gradually lead to irreversible changes manifesting themselves as pain, progressing ankylosis, marked limitation of the range of motion, muscle atrophy and osteoporosis commonly concomitant with joint deformity resulting from chronic proliferative synovitis and both cartilage and bone degeneration leading to the final functional impairment of the joint...
November 13, 2017: Cytokine & Growth Factor Reviews
https://www.readbyqxmd.com/read/29147591/upper-limb-deep-vein-thrombosis-in-patient-with-hemophilia-a-and-heterozygosity-for-prothrombin-g20210a-a-case-report-and-review-of-the-literature
#19
Fares Darawshy, Yosef Kalish, Issam Hendi, Ayman Abu Rmelieh, Tawfik Khoury
Deep vein thrombosis (DVT) is a rare disease in patients with hemophilia A. We report a case of 22-year-old male with severe hemophilia A who presented to the emergency room with 5-day history of right arm pain that was attributed initially to bleeding event. In the absence of external signs of bleeding or hematoma and normal hemoglobin level, we suspected an underlying DVT. Doppler ultrasonography of the right upper limb revealed thrombosis of the subclavian vein and this was confirmed by CT venography. The d-dimer level was normal and investigations for prothrombotic state revealed heterozygosity for prothrombin G20210A mutation...
2017: Case Reports in Hematology
https://www.readbyqxmd.com/read/29145340/open-repair-of-intraoperative-popliteal-artery-injury-during-total-knee-arthroplasty-in-a-patient-with-severe-hemophilia-a-a-case-report-and-literature-review
#20
Bin Feng, Ke Xiao, Jiang Shao, Yu Fan, Xisheng Weng
RATIONALE: Arterial injuries during total knee arthroplasty (TKA) though rare may occur. Hemophilia may further challenge the management of such injury. PATIENT CONCERNS: A 48-year-old male patient with severe hemophilia type A and stiff knee arthropathy underwent bilateral TKAs. Left popliteal artery injury was detected at the end of the left TKA. DIAGNOSES: Urgent angiography confirmed the diagnosis of the left popliteal artery transection...
November 2017: Medicine (Baltimore)
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