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Inhibitor haemophilia

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https://www.readbyqxmd.com/read/29149550/a-promising-on-demand-treatment-option-for-bleeding-events-in-haemophilia-patients-with-inhibitors
#1
Amy D Shapiro
No abstract text is available yet for this article.
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29135476/thrombogenicity-evaluation-in-221-patients-with-haemophilia-b-treated-with-nonacog-alfa
#2
Pablo Rendo, Janice Lamb, Lynne Smith, Joanne Fuiman, Joan M Korth-Bradley
: Risk for thrombotic events with factor IX replacement therapy in patients with haemophilia B remains a concern for patients, those who treat them, and regulatory agencies, based on experience with early use of prothrombin complex concentrates. The current post hoc analysis assessed the incidence of thrombotic events and changes in prothrombin fragment 1 + 2, thrombin-antithrombin complex, and D-dimer in 221 patients with haemophilia B who received nonacog alfa in clinical studies. Thrombotic event and coagulation marker data were collected from 8 interventional studies utilizing on-demand, prophylactic, and preventive regimens in patients with haemophilia B...
November 10, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/29124682/nonacog-beta-pegol-a-review-in-haemophilia-b
#3
Yahiya Y Syed
Nonacog beta pegol [Refixia(®) (EU)] is an intravenously-administered, glycoPEGylated recombinant factor IX (FIX), with an extended terminal half-life. It is approved in the EU for the treatment and prophylaxis of bleeding in patients aged ≥ 12 years with haemophilia B. The therapeutic efficacy and safety of nonacog beta pegol was demonstrated in the phase 3 Paradigm trials in previously treated adolescents and adults with haemophilia B. In Paradigm 2, nonacog beta pegol showed good haemostatic effects when treating bleeds on-demand, and reduced annualized bleeding rates when used as a once-weekly prophylaxis...
November 9, 2017: Drugs
https://www.readbyqxmd.com/read/29094008/total-joint-replacement-in-inhibitor-positive-haemophilia-long-term-outcome-analysis-in-fifteen-patients
#4
Heidi Danielson, Riitta Lassila, Pekka Ylinen, Timo Yrjönen
AIM: To collect data from joint replacement in inhibitor patients, evaluate haemostatic and patient outcomes, and analyse the costs. METHODS: We report our 21-year, single-centre cumulative experience of 15 joint arthroplasties in six inhibitor patients. RESULTS: Two low responder inhibitor patients were in the early days treated with FVIII, whereas bypassing agents were used in the rest of the high responder patients. The primary haemostatic outcome was good in 8/15, fair in 4/15 and poor in 3/15 operations...
October 18, 2017: World Journal of Orthopedics
https://www.readbyqxmd.com/read/29080391/timing-and-severity-of-inhibitor-development-in-recombinant-versus-plasma-derived-factor-viii-concentrates-a-sippet-analysis
#5
F Peyvandi, A Cannavò, I Garagiola, R Palla, P M Mannucci, F R Rosendaal
BACKGROUND: The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is the most severe complication in the early phases of treatment of severe haemophilia A. Recently a randomized trial, the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET) demonstrated a two-fold higher risk of inhibitors development in children treated with recombinant FVIII (rFVIII) products than with plasma-derived FVIII (pdFVIII) during the first 50 exposure days (EDs). OBJECTIVE/METHODS: In this post-hoc SIPPET analysis we evaluated the rate of inhibitor incidence over time by every 5 EDs (from 0 to 50 EDs) in patients treated with different classes of FVIII product, made possible by a frequent testing regime...
October 28, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29080389/sippet-insights-into-factor-viii-immunogenicity
#6
Padraic G Fallon, Michelle Lavin, James S O'Donnell
In this issue of the Journal of Thrombosis and Haemostasis, Peyvandi et al present further insights from the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET) study regarding the relative immunogenicity of recombinant- (rFVIII) versus plasma-derived FVIII (pdFVIII) in patients with haemophilia A [1]. In a post-hoc analysis of this prospective randomised controlled trial, the timing and severity of inhibitor development for previously untreated patients (PUPs) treated with rFVIII was compared with that in PUPs treated with pdFVIII...
October 28, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29048712/efficacy-and-safety-of-nuwiq-%C3%A2-human-cl-rhfviii-in-patients-with-severe-haemophilia-a-undergoing-surgical-procedures
#7
N Zozulya, C M Kessler, A Klukowska, M von Depka, K Hampton, C R M Hay, M Jansen, J Bichler, S Knaub, S Rangarajan
INTRODUCTION: Haemophilia A patients are at a high risk of excess bleeding during surgeries. The aim of haemostatic therapy during the perioperative period is to normalize FVIII level perioperatively and postoperatively to maintain normal haemostasis until wound healing is complete. AIMS/METHODS: To examine the efficacy of Nuwiq(®) (simoctocog alfa, human-cl rhFVIII), a 4(th) generation recombinant FVIII produced in a human cell line, for surgical prophylaxis in patients with severe haemophilia A...
October 19, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29044825/pattern-of-bleeding-in-a-large-prospective-cohort-of-haemophilia-a-patients-a-three-year-follow-up-of-the-ahead-advate-in-haemophilia-a-outcome-database-study
#8
K Khair, M G Mazzucconi, R Parra, E Santagostino, D A Tsakiris, C Hermans, J Oldenburg, G Spotts, K Steinitz-Trost, A Gringeri
INTRODUCTION: Outcome data on treatment of patients with haemophilia A spanning several years of real-world evidence collection are currently very limited. AIM AND METHODS: The global prospective long-term Advate(®) Haemophilia A Outcome Database (AHEAD) cohort study collects real-world data from patients with severe and moderate haemophilia. We report an interim data read-out after three years of observation. RESULTS: A total of 522 patients were enrolled from 21 countries: 334 completed year 1 follow-up, 238 completed year 2 and 136 completed year 3, with an overall follow-up of 811 patient-years...
October 17, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28984010/safety-and-dose-dependency-of-eptacog-beta-activated-in-a-dose-escalation-study-of-non-bleeding-congenital-haemophilia-a-or-b-patients-with-or-without-inhibitors
#9
J Ducore, J B Lawrence, M Simpson, L Boggio, A Bellon, J Burggraaf, J Stevens, M Moerland, J Frieling, J Reijers, M Wang
INTRODUCTION: Varying initial doses of activated eptacog beta (recombinant human FVIIa, rhFVIIa) may provide therapeutic options when treating bleeding in patients with congenital haemophilia who have developed inhibitory antibodies to factor VIII (FVIII) or factor IX (FIX). This study evaluated escalated doses of a new rhFVIIa product as a prelude to selecting the doses for clinical efficacy evaluation in haemophilia patients. AIM: To assess the safety, pharmacokinetics, and laboratory pharmacodynamics of 3 doses of rhFVIIa in non-bleeding patients with congenital haemophilia A or B with or without inhibitors...
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28960809/acquired-haemophilia-in-cancer-a-systematic-and-critical-literature-review
#10
REVIEW
M Napolitano, S Siragusa, S Mancuso, C M Kessler
AIM: There is a paucity of data on the clinical presentation and management of cancer patients with acquired haemophilia (AH), we here report a systematic literature review on acquired haemophilia in the context of cancer. METHODS: Treatment outcomes of AH were defined as complete response (CR), partial response (PR) or no response (NR), based on inhibitor eradication, coagulation factor VIII levels and bleeding control. Reported deaths were either related to cancer or bleeding...
September 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28944952/bypassing-agent-prophylaxis-in-people-with-hemophilia-a-or-b-with-inhibitors
#11
REVIEW
Chatree Chai-Adisaksopha, Sarah J Nevitt, Mindy L Simpson, Maissaa Janbain, Barbara A Konkle
BACKGROUND: People with hemophilia A or B with inhibitors are at high risk of bleeding complications. Infusion of bypassing agents, such as recombinant activated FVII (rFVIIa) and plasma-derived activated prothrombin complex concentrate, are suggested as alternative therapies to factor VIII (haemophilia A) or IX (haemophilia B) for individuals who no longer respond to these treatments because they develop inhibitory antibodies. The ultimate goal of treatment is to preserve the individual's joints, otherwise destroyed by recurrent bleeds...
September 25, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28943040/treatment-of-musculo-skeletal-pain-in-haemophilia
#12
REVIEW
E Carlos Rodriguez-Merchan
Musculo-skeletal pain treatment is inadequate in many haemophilic patients. Analgesics are used only by 36% of adult patients. FVIII/FIX intravenous infusion is mainly used to lessen pain, followed in frequency by usage of NSAIDS (primarily COX-2 inhibitors). In about 30% of patients, pain continues after infusion of F VIII/IX. In acute haemarthroses pain treatment must continue until total disappearance (checked by ultrasonography) and include haematologic treatment, short-term rest of the involved joint, cryotherapy, joint aspiration and analgesic medication (paracetamol in mild pain, metamizole for more intense pain, and in a few precise patients, soft opioids such as codeine or tramadol)...
September 19, 2017: Blood Reviews
https://www.readbyqxmd.com/read/28942686/fibrin-glue-for-local-haemostasis-in-haemophilia-surgery
#13
E Carlos Rodriguez-Merchan
INTRODUCTION: Local fibrin glue (FG) appears to be a useful local haemostatic agent for severe haemorrhage in people with haemophilia (PWH) undergoing surgical procedures. AIM: To evaluate the role of local FG in PWH. METHODS: A review of the literature on the topic has been performed. RESULTS: Local FG is not always necessary to achieve haemostasis in all surgical procedures performed in PWH. However, it could be a good adjunct therapy, primarily when a surgical field will bleed more than expected (e...
October 3, 2017: Hospital Practice (Minneapolis)
https://www.readbyqxmd.com/read/28900904/lonoctocog-alfa-a-review-in-haemophilia-a
#14
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...
October 2017: Drugs
https://www.readbyqxmd.com/read/28873263/continuous-infusion-of-coagulation-factor-concentrates-during-intensive-treatment
#15
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
#16
MULTICENTER STUDY
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...
September 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
#17
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
#18
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
#19
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
#20
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...
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
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