scholarly journals Target joint resolution in patients with haemophilia A receiving long‐term prophylaxis with BAY 94‐9027

Haemophilia ◽  
2020 ◽  
Vol 26 (4) ◽  
Author(s):  
Mark T. Reding ◽  
Ingrid Pabinger ◽  
Shadan Lalezari ◽  
Elena Santagostino ◽  
Maria Elisa Mancuso
Keyword(s):  
Haemophilia A ◽  
Joint Resolution

scholarly journals The effect of emicizumab prophylaxis on long‐term, self‐reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies

Haemophilia ◽  
2021 ◽  
Author(s):  
Mark W. Skinner ◽  
Claude Négrier ◽  
Ido Paz‐Priel ◽  
Sammy Chebon ◽  
Victor Jiménez‐Yuste ◽  
...  
Keyword(s):  
Physical Health ◽  
Factor Viii ◽  
Haemophilia A

Relapse pattern and long‐term outcomes in subjects with acquired haemophilia A

Haemophilia ◽  
2019 ◽  
Vol 25 (2) ◽  
pp. 252-257 ◽  
Author(s):  
Terry Mizrahi ◽  
Karine Doyon ◽  
Evemie Dubé ◽  
Arnaud Bonnefoy ◽  
Margaret Warner ◽  
...  
Keyword(s):  
Haemophilia A ◽  
Long Term Outcomes ◽  
Acquired Haemophilia ◽  
Relapse Pattern

scholarly journals Long‐term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A

Haemophilia ◽  
2015 ◽  
Vol 22 (1) ◽  
pp. 72-80 ◽  
Author(s):  
B. Nolan ◽  
J. Mahlangu ◽  
D. Perry ◽  
G. Young ◽  
R. Liesner ◽  
...  
Keyword(s):  
Fusion Protein ◽  
Factor Viii ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Safety And Efficacy ◽  
Fc Fusion Protein

Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

2016 ◽  
Vol 115 (05) ◽  
pp. 931-938 ◽  
Author(s):  
Annelies Nijdam ◽  
Wouter Foppen ◽  
Piet de Kleijn ◽  
Evelien P. Mauser-Bunschoten ◽  
Goris Roosendaal ◽  
...  
Keyword(s):  
Functional Limitations ◽  
Objective Assessment ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
On Demand ◽  
Recommended Treatment ◽  
Objective Monitoring ◽  
Joint Health ◽  
Observational Cohort

SummaryProphylaxis is the recommended treatment for children with severe haemophilia A, but whether prophylaxis should be continued in adulthood is still under debate. Previous studies with limited follow-up have suggested that some patients may be able to stop prophylaxis in adulthood, while maintaining good joint health. This single-centre observational cohort study examined patients with severe haemophilia A born 1970–1988 without inhibitor development, and assessed the long-term consequences of discontinuing prophylaxis. Patient-initiated changes in prophylaxis, including all switches to on-demand treatment lasting a minimum of two consecutive weeks, were recorded from the time self-infusion began until the last evaluation. Sixty-six patients were evaluated at a median age of 32.4 years: 26 of patients had stopped prophylaxis for a median of 10 years, 15 had interrupted prophylaxis and 59 had continued prophylaxis. Annual joint bleeding rate (AJBR), Haemophilia Joint Health Score (HJHS-2.1; 0–124 points), radiological Pettersson score (0–78 points) and Haemophilia Activities List score (HAL; 100–0 points) were compared between patients who stopped and patients who continued prophylaxis. Although self-reported bleeding rates and functional limitations were similar in both groups (AJBR: 1.5 vs 1.2 and HAL: 84 vs 84 for those who stopped and continued prophylaxis, respectively), objective assessment of joint status showed increased arthropathy after 10 years of on-demand treatment in patients who stopped prophylaxis compared with those who continued (HJHS: 23 vs. 14 and Pettersson: 16 vs 5, respectively; P< 0.01). These results support continuation of long-term prophylaxis in adults and demonstrate the need for objective monitoring of joint status.Trial registration: Dutch Trial Registry number 3098; UTN U1111–1121–7069.

The long-term course of factor VIII inhibitors in patients with congenital haemophilia A without immune tolerance induction

2011 ◽  
Vol 105 (01) ◽  
pp. 59-65 ◽  
Author(s):  
Camila Caram ◽  
Roberta Grazielle de Souza ◽  
Júlio Carepa de Sousa ◽  
Tatiana Araújo Pereira ◽  
Ana Maria do Amaral Cerqueira ◽  
...  
Keyword(s):  
Confidence Interval ◽  
Factor Viii ◽  
Immune Tolerance ◽  
Tolerance Induction ◽  
Clotting Factor ◽  
Haemophilia A ◽  
Immune Tolerance Induction ◽  
Inhibitor Titre ◽  
Very High

SummaryThe development of alloantibodies that inhibit or neutralise the function of factor VIII is considered the most serious complication of the treatment of congenital haemophilia A. In order to describe their course without immune tolerance induction (ITI), we documented data on all performed inhibitor tests with dates as well as on clotting factor infusions of all consecutive patients who were treated in our centre between 1993 and 2006. Patients were tested every 7.1 months (95% confidence interval [CI], 6.6–7.8). A ‘sustained negative inhibitor status’ was defined as consistent non-positive inhibitor measurements for two years or longer. A total of 60/486 (12%) patients tested had a positive inhibitor titre in two or more occasions. Most of the patients (56%) with a maximum inhibitor titre of < 5 Bethesda unit (BU)/ml (named “low titre inhibitor”) developed a sustained negative inhibitor status. Among patients with high (5–9.9 BU/ml) and very high (≥ 10 BU/ ml) inhibitor titres, the proportions were 50% and 3%, respectively. Our findings suggest that ITI might not be needed for all patients with non-transient inhibitors, especially when their maximum inhibitor titre is below 10 BU/ml. Further studies in countries where ITI is not available are needed to examine predictors of the natural sustained negative inhibitor status.

Prevalence and risk factors associated with hepatitis C among Brazilian male patients with haemophilia: A long‐term follow‐up

Haemophilia ◽  
2019 ◽  
Vol 25 (3) ◽  
pp. 447-455 ◽  
Author(s):  
Ricardo Andrade Carmo ◽  
Marina Lobato Martins ◽  
Daniel Gonçalves Chaves ◽  
Lorenza Nogueira Campos Dezanet
Keyword(s):  
Risk Factors ◽  
Hepatitis C ◽  
Haemophilia A ◽  
Factors Associated ◽  
Long Term Follow Up ◽  
Male Patients

scholarly journals Gene therapy for haemophilia: a very modern success story

2015 ◽  
Vol 2 (2) ◽  
pp. 26-28
Author(s):  
Amit Nathwani
Keyword(s):  
Gene Therapy ◽  
Clotting Factor ◽  
Haemophilia A ◽  
Single Administration ◽  
Success Story ◽  
Spontaneous Bleeding ◽  
Bleeding Episodes ◽  
Paradigm Shifting

Abstract AAV-mediated gene therapy has the potential to be paradigm shifting as a treatment for haemophilia. A single administration of AAV vector can result in safe and consistent long-term expression of transgene (>5 years), reduction in spontaneous bleeding episodes, reduction in clotting factor usage and an improvement in quality of life. There is huge commercial interest in this approach, with the expectation that an AAV gene therapy product for haemophilia B will be licensed by 2020.

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