scholarly journals Successful immune tolerance in two children with severe haemophilia A

2013 ◽  
Vol 1 (1) ◽  
pp. 5-7
Author(s):  
Bert Leenders
Keyword(s):  
Factor Viii ◽  
Case Studies ◽  
Immune Tolerance ◽  
Bleeding Disorder ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Igg Antibodies

Abstract Haemophilia A is an X-linked congenital bleeding disorder resulting from a deficiency of factor VIII (FVIII). Therapy to prevent or treat bleeding is by replacement of the missing FVIII. However, as a consequence of treatment, patients with HA may develop inhibitory IgG antibodies to FVIII, termed “inhibitors”. When this occurs, treatment becomes more complex and costly and morbidity increases. Inhibitor formation, occurring in up to 36% of patients with severe HA, is currently one of the most significant complications affecting patients with HA [1,2]. In these two case studies, the management of bleeds in patients with inhibitors, and the long-term inhibitor eradication will be discussed.

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.

scholarly journals Case Report: Massive epistaxis from juvenile angiofibroma in an adolescent with severe haemophilia A

F1000Research ◽  
2019 ◽  
Vol 8 ◽  
pp. 1593
Author(s):  
Jose Florencio F. Lapeña ◽  
Olivia Agnes D. Mejia
Keyword(s):  
Factor Viii ◽  
Nasal Obstruction ◽  
English Literature ◽  
Diagnostic Techniques ◽  
Bleeding Disorder ◽  
Factor Vii ◽  
Preoperative Embolization ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Juvenile Angiofibroma

Epistaxis may be profuse in individuals with normal bleeding parameters, but in an individual with haemophilia, it may be life-threatening. It is even more dangerous when epistaxis is caused by an undetected concomitant juvenile angiofibroma, and only one such case has been reported in the English literature. We report another case, of an 18-year-old Filipino adolescent with severe haemophilia A who was referred for repeated massive epistaxis. The epistaxis had been attributed to his haemophilia and managed with nasal packing, multiple blood transfusions and Factor VIII administration. After two years of unsuccessful management, nasal endoscopy was performed for the first time, revealing an intranasal mass. Imaging showed a right intranasal vascular tumour supplied mainly by the right sphenopalatine artery. He subsequently underwent preoperative embolization and endoscopic excision of the tumour with Factor VIII transfused pre-, intra-, and post-operatively, and recombinant Factor VII added post-operatively. Final histopathology was consistent with juvenile angiofibroma. There has been no nasal obstruction or recurrence of epistaxis seven years since the surgery. Clinicians should be more meticulous in assessing epistaxis in any patient with a bleeding disorder and investigate more subtle symptoms such as nasal obstruction. Verification of the source by direct visualization and ancillary diagnostic techniques (such as imaging) when indicated should be the standard of care for all patients presenting with epistaxis, whether or not a concomitant bleeding disorder exists. A high index of suspicion for juvenile angiofibroma should be maintained in adolescent males with epistaxis and nasal obstruction.

Adult severe haemophilia A patients under long-term prophylaxis with factor VIII in routine clinical practice

2015 ◽  
Vol 26 (5) ◽  
pp. 509-514 ◽  
Author(s):  
María E. Mingot-Castellano ◽  
Laura González-Díaz ◽  
Rocio Tamayo-Bermejo ◽  
Anabel I. Heiniger-Mazo
Keyword(s):  
Clinical Practice ◽  
Factor Viii ◽  
Routine Clinical Practice ◽  
Haemophilia A ◽  
Severe Haemophilia

scholarly journals Case Report: Massive epistaxis from juvenile angiofibroma in an adolescent with severe haemophilia A

F1000Research ◽  
2019 ◽  
Vol 8 ◽  
pp. 1593
Author(s):  
Jose Florencio F. Lapeña ◽  
Olivia Agnes D. Mejia
Keyword(s):  
Factor Viii ◽  
Nasal Obstruction ◽  
English Literature ◽  
Diagnostic Techniques ◽  
Bleeding Disorder ◽  
Factor Vii ◽  
Preoperative Embolization ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Juvenile Angiofibroma

Epistaxis may be profuse in individuals with normal bleeding parameters, but in an individual with haemophilia, it may be life-threatening. It is even more dangerous when epistaxis is caused by an undetected concomitant juvenile angiofibroma, and only one such case has been reported in the English literature. We report another case, of an 18-year-old Filipino adolescent with severe haemophilia A who was referred for repeated massive epistaxis. The epistaxis had been attributed to his haemophilia and managed with nasal packing, multiple blood transfusions and Factor VIII administration. After two years of unsuccessful management, nasal endoscopy was performed for the first time, revealing an intranasal mass. Imaging showed a right intranasal vascular tumour supplied mainly by the right sphenopalatine artery. He subsequently underwent preoperative embolization and endoscopic excision of the tumour with Factor VIII transfused pre-, intra-, and post-operatively, and recombinant Factor VII added post-operatively. Final histopathology was consistent with juvenile angiofibroma. There has been no nasal obstruction or recurrence of epistaxis seven years since the surgery. Clinicians should be more meticulous in assessing epistaxis in any patient with a bleeding disorder and investigate more subtle symptoms such as nasal obstruction. Verification of the source by direct visualization and ancillary diagnostic techniques (such as imaging) when indicated should be the standard of care for all patients presenting with epistaxis, whether or not a concomitant bleeding disorder exists. A high index of suspicion for juvenile angiofibroma should be maintained in adolescent males with epistaxis and nasal obstruction.

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