keyword
MENU ▼
Read by QxMD icon Read
search

Inhibitor haemophilia

keyword
https://www.readbyqxmd.com/read/28900904/lonoctocog-alfa-a-review-in-haemophilia-a
#1
Zaina T Al-Salama, Lesley J Scott
Lonoctocog alfa (rVIII-SingleChain; Afstyla(®)) is a novel single-chain recombinant factor VIII (FVIII) molecule, with a truncated B-domain and the heavy and light chains covalently linked to form a stable and homogenous drug that binds with high affinity to von Willebrand factor (VWF). Intravenous lonoctocog alfa is approved for the prophylaxis and treatment of bleeding in patients with haemophilia A in several countries worldwide. In two pivotal, multicentre trials, lonoctocog alfa was effective in the treatment of bleeding episodes and as prophylaxis, including for perioperative management in adults, adolescents and children...
September 12, 2017: Drugs
https://www.readbyqxmd.com/read/28873263/continuous-infusion-of-coagulation-factor-concentrates-during-intensive-treatment
#2
REVIEW
P A Holme, G E Tjønnfjord, A Batorova
In clinical management of bleeds and surgical procedures in patients suffering from bleeding disorders either repetitive bolus injections (BI) or continuous infusion (CI) can be used for coagulation factor replacement. Continuous infusion seems to be an attractive route of administration and may be considered if replacement therapy is required for more than 3 days. The strongest argument favouring continuous infusion is its superiority in providing the patient with a safe and constant level of the deficient coagulation factor by balancing input with clearance...
September 5, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28836262/immune-status-of-patients-with-haemophilia-a-before-exposure-to-factor-viii-first-results-from-the-hemfil-study
#3
Letícia L Jardim, Daniel G Chaves, Amanda C O Silveira-Cassette, Ana Cristina Simões E Silva, Marcio P Santana, Monica H Cerqueira, Alessandra Prezotti, Claudia Lorenzato, Vivian Franco, Johanna G van der Bom, Suely M Rezende
Previous cross-sectional studies showed that some patients with haemophilia A (HA) without inhibitor presented a pro-inflammatory profile during factor VIII (FVIII) replacement therapy. Furthermore, an anti-inflammatory/regulatory state was described in HA patients after inhibitor development. However, no study investigated the levels of these biomarkers before exposure to exogenous FVIII. This study investigated the immunological profile of previously untreated patients (PUPs) with HA in comparison with non-haemophiliac boys...
August 23, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28833808/pharmacokinetics-safety-and-efficacy-of-a-recombinant-factor-ix-product-trenonacog-alfa-in-previously-treated-haemophilia-b-patients
#4
P W Collins, D V K Quon, M Makris, P Chowdary, C L Kempton, S J Apte, M V Ramanan, C R M Hay, B Drobic, Y Hua, T J Babinchak, E D Gomperts
INTRODUCTION: Trenonacog alfa (IB1001) is a recombinant factor IX (rFIX) manufactured in Chinese hamster ovary (CHO) cells. IB1001 was evaluated in a multicentre clinical trial with haemophilia B patients. AIM: The aim was to establish IB1001 pharmacokinetic non-inferiority to comparator rFIX, safety and efficacy in previously treated patients (PTPs) with haemophilia B. METHODS: Subjects were severe or moderately severe haemophilia B adult and adolescent PTPs with no history of FIX inhibitors...
August 17, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28826659/haemophilia-b-where-are-we-now-and-what-does-the-future-hold
#5
REVIEW
Gerry Dolan, Gary Benson, Anne Duffy, Cedric Hermans, Victor Jiménez-Yuste, Thierry Lambert, Rolf Ljung, Massimo Morfini, Silva Zupančić Šalek
Research has been lacking on the natural history, complications, and treatment of haemophilia B, which is less common than haemophilia A and was recognized as a distinct clinical entity in 1947. Although the two diseases share the same clinical manifestations, they differ in causative mutation, risk of inhibitor development, and patient quality of life. Frequently debated is whether haemophilia B is as clinically severe as haemophilia A, with much of the published data on overall and haemophilia-specific health outcomes suggesting that haemophilia B may have a less severe clinical phenotype...
August 16, 2017: Blood Reviews
https://www.readbyqxmd.com/read/28815880/immunogenicity-efficacy-and-safety-of-nuwiq-%C3%A2-human-cl-rhfviii-in-previously-untreated-patients-with-severe-haemophilia-a-interim-results-from-the-nuprotect-study
#6
R J Liesner, M Abashidze, O Aleinikova, C Altisent, M J Belletrutti, A Borel-Derlon, M Carcao, H Chambost, A K C Chan, L Dubey, J Ducore, N A Fouzia, M Gattens, Y Gruel, B Guillet, N Kavardakova, M El Khorassani, A Klukowska, T Lambert, S Lohade, M Sigaud, V Turea, J K M Wu, V Vdovin, A Pavlova, M Jansen, L Belyanskaya, O Walter, S Knaub, E J Neufeld
INTRODUCTION: Nuwiq(®) (Human-cl rhFVIII) is a fourth generation recombinant FVIII, produced in a human cell line, without chemical modification or protein fusion. No inhibitors developed in studies with Nuwiq(®) in 201 previously treated patients with haemophilia A (HA). The immunogenicity, efficacy and safety of Nuwiq(®) in previously untreated patients (PUPs) with severe HA are being assessed in the ongoing NuProtect study. METHODS: The study, conducted across 38 centres worldwide, is evaluating 110 true PUPs of all ages and ethnicities enrolled for study up to 100 exposure days (EDs) or 5 years maximum...
August 16, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28806864/study-of-physical-function-in-adolescents-with-haemophilia-the-so-fit-study
#7
K Khair, M Holland, M Bladen, A Griffioen, P McLaughlin, S von Mackensen
INTRODUCTION: Contemporary haemophilia care demands Patient-Reported Outcomes. SO-FIT is a UK multi-centre study, assessing self-reported function, health-related quality of life (HRQoL) and joint health in boys with severe haemophilia. METHODS: Subjective physical function (PedHAL, HEP-Test-Q) and HRQoL (Haemo-QoL Short Form [SF]) were assessed alongside joint health using the objective Haemophilia Joint Health Score (HJHS v2.1). Demographic and clinical data were collected...
August 14, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28776894/persept-1-a-phase-3-trial-of-activated-eptacog-beta-for-on-demand-treatment-of-haemophilia-inhibitor-related-bleeding
#8
M Wang, J B Lawrence, D V Quon, J Ducore, M L Simpson, L N Boggio, I S Mitchell, G Yuan, W A Alexander, J-F Schved
INTRODUCTION: Haemophilia A or B patients with inhibitors have been treated with FVIIa-containing bypassing agents for over 20 years. However, due to uncertainty regarding dose response and thrombotic risk, the use of a gradual, titrated, minimal dosing strategy remains prevalent, potentially hampering early haemostasis. AIM: Evaluate the dose-dependent efficacy, safety and immunogenicity of activated eptacog beta (rhFVIIa), a new recombinant inhibitor bypassing agent for the treatment of bleeding episodes (BEs)...
August 3, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28764183/acquired-inhibitor-of-factor-viii-presenting-as-delayed-wound-healing
#9
Sushma Nayar, Kishore Babu Esakkimuthu Parvathi, Mayilananthi Kaliannan, Premlatha Sivasailam
Acquired coagulation factor VIII inhibitor leads to a rare disease i.e., acquired haemophilia which is idiopathic in majority of cases and is seen with autoimmune diseases, haematologic and solid tumours, infections, in the post-partum period and also with certain long-term use of drugs like penicillin and its derivatives, phenytoin, sulfa antibiotics, chloramphenicol, methyldopa, chlorpromazine, levodopa, interferon-α, fludarabine, clopidogrel. We report a case here, with acquired Factor VIII (FVIII) inhibitor acquisition which presented with delayed wound healing as a result of protracted bleeding into the wound...
June 2017: Journal of Clinical and Diagnostic Research: JCDR
https://www.readbyqxmd.com/read/28758324/surgery-and-survival-in-birth-cohorts-with-severe-haemophilia-and-differences-in-access-to-replacement-therapy-the-malm%C3%A3-experience
#10
M Osooli, K Steen Carlsson, J Astermark, E Berntorp
BACKGROUND: Persons with severe haemophilia require lifelong replacement therapy, prophylaxis, to prevent bleeding. Data describing long-term outcomes of prophylactic treatment are scarce. The aim of this study was to investigate joint surgery and survival among persons with severe haemophilia with special attention to access to prophylaxis in the early years of life. METHODS: Eligible participants had severe haemophilia A or B and were treated at the Malmö centre from the 1960s onward...
July 31, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28756381/acquired-haemophilia-a-with-a-recalcitrant-high-titre-factor-viii-inhibitor-in-the-setting-of-interstitial-lung-disease
#11
Lova Sun, David B Sykes
Acquired haemophilia A (AHA) is a bleeding disorder that results from autoantibodies against factor VIII (FVIII). A 70-year-old man with a history of interstitial lung disease presented with spontaneous bleeding into his thigh. He had undetectable FVIII levels and a high-titre FVIII inhibitor (>2000ââ'¬â€°Bethesda units/mL) and was diagnosed with AHA. He had several relapses, required multiple haemostatic and immunosuppressive treatments but eventually achieved a stable remission after 2ââ'¬â€°years of therapy...
July 28, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28752639/perioperative-management-of-haemophilia-b-a-critical-appraisal-of-the-evidence-and-current-practices
#12
REVIEW
E J Neufeld, L Solimeno, D Quon, C Walsh, S Seremetis, D Cooper, N N Iyer, C S Hoxer, P Giangrande
BACKGROUND: While there is substantial literature addressing the principles of general management of haemophilia, literature on perioperative management of haemostasis is scarce. OBJECTIVE: The aim of this study was to better understand perioperative management among congenital haemophilia B patients (without inhibitors) and to gain insights into real-world surgical practices. METHOD: A systematic literature review, with an emphasis on haemophilia B, was conducted using EMBASE(®) , Medline(®) and the Cochrane Library...
July 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28750470/clot-waveform-analysis-using-cs-2000i%C3%A2-distinguishes-between-very-low-and-absent-levels-of-factor-viii-activity-in-patients-with-severe-haemophilia-a
#13
T Matsumoto, K Nogami, Y Tabuchi, K Yada, K Ogiwara, H Kurono, N Arai, M Shima
INTRODUCTION: A recently developed method to assess comprehensive coagulation function, clot waveform analysis (CWA), accurately detect low levels (<1 IU/dL) of factor VIII activity (FVIII:C) in haemophilia A patients (HA-pts). Improvements are needed, however, to differentiate patients with very low from absent levels of FVIII:C. AIM: We attempted to optimize CWA using the coagulation analyser CS-2000i™ to distinguish between very low levels and absent FVIII:C in severe HA-pts...
July 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28699675/intracranial-haemorrhage-in-children-and-adolescents-with-severe-haemophilia-a-or-b-the-impact-of-prophylactic-treatment
#14
Nadine G Andersson, Günter Auerswald, Chris Barnes, Manuel Carcao, Amy L Dunn, Karin Fijnvandraat, Marianne Hoffmann, Kaan Kavakli, Gili Kenet, Rainer Kobelt, Karin Kurnik, Ri Liesner, Anne Mäkipernaa, Marilyn J Manco-Johnson, Maria E Mancuso, Angelo C Molinari, Beatrice Nolan, Rosario Perez Garrido, Pia Petrini, Helen E Platokouki, Amy D Shapiro, Runhui Wu, Rolf Ljung
The discussion of prophylactic therapy in haemophilia is largely focused on joint outcomes. The impact of prophylactic therapy on intracranial haemorrhage (ICH) is less known. This study aimed to analyse ICH in children with haemophilia, with a focus on different prophylaxis regimens and sequelae of ICH. We conducted a multicentre retrospective and prospective study that included 33 haemophilia centres from 20 countries. Inclusion criteria were children and adolescents born between 1993 and 2014, with severe haemophilia A or B without inhibitors...
July 12, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28692108/safety-and-efficacy-of-a-glycopegylated-rfviii-turoctocog-alpha-pegol-n8-gp-in-paediatric-patients-with-severe-haemophilia-a
#15
Sandrine Meunier, Jayanthi Alamelu, Silke Ehrenforth, Hideji Hanabusa, Faraizah Abdul Karim, Kaan Kavakli, Melanie Khodaie, Janice Staber, Oleksandra Stasyshyn, Donald L Yee, Lina Rageliene
Turoctocog alfa pegol (N8-GP, Novo Nordisk, Bagsværd, Denmark), an extended half-life glycoPEGylated recombinant factor VIII (rFVIII), is being developed for prophylaxis and treatment of bleeds in haemophilia A patients. pathfinder™5 is a multinational, open-label, single-arm trial to assess safety, efficacy and pharmacokinetics of N8-GP in paediatric (<12 years), previously treated patients. Boys with severe haemophilia A (<1 % FVIII), no history of inhibitors and previously treated with FVIII products (>50 exposure days [ED] for patients aged 0-5 years [younger cohort]; >150 ED for patients aged 6-11 years [older cohort]) were included...
August 30, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28686157/the-role-of-recombinant-activated-factor-vii-in-the-haematological-management-of-elective-orthopaedic-surgery-in-haemophilia-a-patients-with-inhibitors
#16
REVIEW
Giancarlo Castaman
The clinical profile and expectations of haemophilic patients with inhibitors have changed over the last three decades, mainly because of the prolongation of life-expectancy, often resulting in an increase of the orthopaedic burden. Recombinant activated factor VII (rFVIIa) is the most frequently used bypassing agent in haemophilia patients with inhibitors during elective orthopaedic surgery. For nearly 30 years, rFVIIa has been successfully used to control haemostasis in several major and minor surgical procedures...
September 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28686156/high-titre-inhibitors-in-previously-untreated-patients-with-severe-haemophilia-a-receiving-recombinant-or-plasma-derived-factor-viii-a-budget-impact-analysis
#17
Andrea Messori, Flora Peyvandi, Sabrina Trippoli, Roberta Palla, Frits R Rosendaal, Pier Mannuccio Mannucci
No abstract text is available yet for this article.
May 15, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28678027/meta-analysis-on-incidence-of-inhibitors-in-patients-with-haemophilia-a-treated-with-recombinant-factor-viii-products
#18
Matteo Rota, Paolo A Cortesi, Katharina N Steinitz-Trost, Armin J Reininger, Alessandro Gringeri, Lorenzo G Mantovani
: Recent cohort studies showed differences in inhibitor incidence in previously untreated patients (PUPs) with haemophilia A treated with recombinant factor VIII (rFVIII) concentrates. We carried out a systematic literature search and meta-analysis for all randomized clinical trials and observational studies published from 1 January 1988 to 31 August 2015, to assess the incidence of inhibitor development and the relationship with rFVIII product used in PUPs and minimally treated patients (MTPs, ≤5 previous exposure days), with severe haemophilia...
July 3, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28646426/eftrenonacog-alfa-a-review-in-haemophilia-b
#19
Sheridan M Hoy
Eftrenonacog alfa (Alprolix™) is a recombinant fusion protein comprising human factor IX (FIX) covalently linked to the constant region (Fc) domain of human IgG1 (i.e. rFIXFc). The presence of the Fc domain extends the terminal half-life (t½) of rFIXFc, permitting prolonged treatment intervals. rFIXFc is available for intravenous use for the prophylaxis and treatment of bleeding in patients with haemophilia B. In two multinational, phase III studies in previously treated children, adolescents and adults with severe haemophilia B, rFIXFc prophylaxis resulted in low median annualized bleeding rates (ABRs), and was associated with reductions in median weekly factor consumption and dosing frequency compared with pre-study FIX regimens...
July 2017: Drugs
https://www.readbyqxmd.com/read/28594467/biochemical-characterization-of-lr769-a-new-recombinant-factor-viia-bypassing-agent-produced-in-the-milk-of-transgenic-rabbits
#20
G Chevreux, N Tilly, Y Leblanc, C Ramon, V Faid, M Martin, F Dhainaut, N Bihoreau
BACKGROUND: The bypassing agent factor VII (FVIIa) is a first-line therapy for the treatment of acute bleeding episodes in patients with haemophilia and high-titre inhibitors. FVIIa is a highly post-translationally modified protein that requires eukaryotic expression systems to produce a fully active molecule. A recombinant FVIIa was produced in the milk of transgenic rabbits to increase expression and provide an efficient, safe and affordable product after purification to homogeneity (LR769)...
June 8, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
keyword
keyword
53941
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"