scholarly journals Impact of severe haemophilia A on patients' health status: results from the guardian ™ 1 clinical trial of turoctocog alfa (NovoEight ® )

Haemophilia ◽  
2015 ◽  
Vol 21 (4) ◽  
pp. 451-457 ◽  
Author(s):  
M. Ozelo ◽  
P. Chowdary ◽  
A. Regnault ◽  
A. K. Busk
Keyword(s):  
Clinical Trial ◽  
Health Status ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
The Guardian

scholarly journals Health Status in Patients with Severe Haemophilia a According to Treatment Regimen and Age

2016 ◽  
Vol 19 (7) ◽  
pp. A592-A593 ◽  
Author(s):  
C Hirst ◽  
J Black ◽  
A Elnoursi ◽  
J O'Hara
Keyword(s):  
Health Status ◽  
Treatment Regimen ◽  
Haemophilia A ◽  
Severe Haemophilia

scholarly journals Prevalence of Haemarthrosis and Clinical Impact on The Musculoskeletal System in People With Haemophilia in The United Kingdom; Evaluation of UK National Haemophilia Database and Haemtrack Patient Reported Data.

2020 ◽  
Author(s):  
Richard Wilkins ◽  
David Stephensen ◽  
Heidi Siddle ◽  
Martin Scott ◽  
Hua Xiang ◽  
...  
Keyword(s):  
Health Status ◽  
Genetic Disorder ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
The United Kingdom ◽  
Patient Reported ◽  
Joint Health ◽  
Bleeding Manifestation ◽  
Reported Data ◽  
A Current

Abstract Introduction; Severe haemophilia is a rare x-linked recessive genetic disorder characterised by bleeding into soft tissue and joints. Haemarthrosis is the most common bleeding manifestation in haemophilia, leading to haemarthropathy. The ankle joint has been reported as the most common site of bleeding, but it is unclear whether the ankle is most affected in terms of joint health status. Aims; To determine the prevalence of joint bleeding and musculoskeletal health status at an individual joint level of children and adults with severe haemophilia A and B without a current inhibitor.Methods; Application was made to the National Haemophilia Database (NHD) with a request for the following; Haemtrack patient reported treatment record and Haemophilia Joint Health Scores (HJHS) in children (<18y) and adults (≥18y) with severe haemophilia A (HA) and B (HB) (FVIII/FIX, <0.01 iu/ml) without a current inhibitor. Data were collated and reported for 1st January to 31st December 2018.Results; 2238 cases were identified, of which 273 were Haemtrack compliant with contemporaneous HJHS. The median (IQR) age of children was 10 (6-13) and adults 40 (29-50) years. Haemarthrosis prevalence in HA/HB children was 33% and 47%, respectively and 60% and 42%, respectively, in adults. The most common haemarthrosis site in children was the knee in HA and ankle in HB. In adults, the incidence of haemarthrosis at the ankles and elbows was equal. Median (IQR) total HJHS in HA/HB children were 0 (0;0). In adults with HA/HB, HJHS were 18 (6; 31) and 11 (5; 24), respectively. In adults with HA/HB, mean (SD) ankle HJHS of 3.8 (4.1) and median 4.0 (0.0; 8.0) were higher than the knee (mean 2.9 (4.1) and median 1.0 (0.0; 5.0) and elbow (mean 3.3 (4.1) and median 1.0 (0.0; 7.0) joints.Conclusion; During 2018, NHD prevalence data of haemarthrosis indicate only two-thirds of children and one-third of adults from a UK cohort compliant with prophylaxis were bleed free. HJHS of zero in children suggests joint health status is either unaffected during childhood or undetected by HJHS. In adults, higher HJHS are reported for the ankles indicating worse joint health.

scholarly journals Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A: Final results from the guardian 2 extension trial

Haemophilia ◽  
2018 ◽  
Vol 24 (6) ◽  
pp. e391-e394 ◽  
Author(s):  
Steven R. Lentz ◽  
Dragana Janic ◽  
Kaan Kavakli ◽  
Predrag Miljic ◽  
Johannes Oldenburg ◽  
...  
Keyword(s):  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy ◽  
Bleeding Episodes ◽  
The Guardian ◽  
Extension Trial

Inhibitor treatment by rituximabin congenital haemophilia A

2009 ◽  
Vol 29 (02) ◽  
pp. 151-154 ◽  
Author(s):  
Escuriola Ettingshausen ◽  
R. Linde ◽  
G. Kropshofer ◽  
L.-B. Zimmerhackl ◽  
W. Kreuz ◽  
...  
Keyword(s):  
B Cell ◽  
Immune Tolerance ◽  
High Titre ◽  
Clotting Factor ◽  
Severe Bleeding ◽  
High Morbidity ◽  
Haemophilia A ◽  
Concomitant Treatment ◽  
Bleeding Tendency ◽  
Severe Haemophilia

SummaryThe development of neutralizing alloanti-bodies (inhibitors) to factor VIII (FVIII) is one of the most serious complications in the treatment of haemophiliacs. Inhibitors occur in approximately 20 to 30% of previously untreated patients (PUPs), predominantly children, with severe haemophilia A within the first 50 exposure days (ED). Immune tolerance induction (ITI) leads to complete elimination of the inhibitor in up to 80% of the patients and offers the possibility to restore regular FVIII prophylaxis. However, patients with high titre inhibitors, in whom standard ITI fails, usually impose with high morbidity and mortality and therefore prompting physicians to alternate therapy regimens. Rituximab, an anti-CD 20 monoclonal antibody has been successfully used in children and adults for the management of B-cell mediated disorders. We report on the use of a new protocol including rituximab in two adolescents with severe haemophilia A and high titre inhibitors, severe bleeding tendency and high clotting factor consumption after failing standard ITI. Both patients received a concomitant treatment with FVIII according to the Bonn protocol, cyclosporine A and immunoglobulin. Treatment with rituximab resulted in a temporary B-cell depletion leading to the disappearance of the inhibitor. FVIII recovery and half-life turned towards normal ranges. In patient 1 the inhibitor reappeared 14 months after the last rituximab administration. In patient 2 complete immune tolerance could be achieved for 60 months. Bleeding frequency diminished significantly and clinical joint status improved in both patients. In patient 1 the treatment course was complicated by aspergillosis and hepatitis B infection. Conclusion: Rituximab may be favourable for patients with congenital haemophilia, high-titre inhibitors and a severe clinical course in whom standard ITI has failed. Prospective studies are required to determine safety, efficacy and predictors of success.

Inhibitorentwicklung nach früher hoher Exposition und Hirnblutung

2010 ◽  
Vol 30 (S 01) ◽  
pp. S115-S118
Author(s):  
C. Moorthi ◽  
A. Bade ◽  
C. Niekrens ◽  
G. Auerswald ◽  
K. Haubold
Keyword(s):  
High Titer ◽  
Cerebral Haemorrhage ◽  
High Dose ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Intron 22 Inversion ◽  
Neurosurgical Intervention

SummarySevere haemophilia A was diagnosed postpartum in a newborn. The mother was known as a conductor (intron 22 inversion) and an uncle had a persistently high titer inhibitor after failed ITI.Due to a cephalhaematoma, a high-dose pdFVIII substitution was given within the first days after birth. At the age of six month a severe cerebral haemorrhage occurred, making a high-dose pdFVIII substitution and neurosurgical intervention necessary. Several days later a porth-a-cath-system was implanted. The development of a high titer inhibitor occured six days later, an ITI was started according to the Bonn Protocol. Initially rFVIIa was given in addition to the pdFVIII substitution. Seven days after the beginning of treatment the inhibitor was no longer detectable. At monthly intervals the FVIII dosage was reduced until the dosage complied with a prophylaxis in severe haemophilia A.The duration of the ITI was nine months. A total of 30 mg rFVIIa and 276 000 IU pdFVIII were used; costs in total: 280 173.60 Euro.

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