Switching to low dose of plasma-derived factor VIII/vWF concentrates with Confact®-F as salvage immune tolerance induction in haemophilia A patients with inhibitors: five case reports from Japan

Haemophilia ◽  
2015 ◽  
Vol 21 (5) ◽  
pp. e425-e427 ◽  
Author(s):  
A. Nagao ◽  
T. Oka ◽  
H. Hanabusa
Keyword(s):  
Factor Viii ◽  
Immune Tolerance ◽  
Low Dose ◽  
Case Reports ◽  
Tolerance Induction ◽  
Haemophilia A ◽  
Immune Tolerance Induction

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 Predictors of the outcome of immune tolerance induction in patients with haemophilia A and inhibitors: The Brazilian Immune Tolerance (BrazIT) Study protocol

PLoS ONE ◽  
2021 ◽  
Vol 16 (8) ◽  
pp. e0256265
Author(s):  
Ricardo Mesquita Camelo ◽  
Daniel Gonçalves Chaves ◽  
Luciana Werneck Zuccherato ◽  
Suely Meireles Rezende ◽  
Keyword(s):  
Immune Tolerance ◽  
Low Dose ◽  
Clinical Laboratory ◽  
Tolerance Induction ◽  
High Dose ◽  
Haemophilia A ◽  
Clinical Genetic ◽  
Immune Tolerance Induction ◽  
Immune Biomarkers ◽  
Treatment Centre

The development of inhibitors is the main complication of haemophilia A (HA) treatment. Immune tolerance induction (ITI) is the treatment of choice for inhibitor eradication. We describe the methodology of the Brazilian Immune Tolerance Induction (BrazIT) Study, aimed to identify clinical, genetic, and immune biomarkers associated with response to ITI and inhibitor recurrence. This cohort study includes people with HA (PwHA) and inhibitors (a) who require bypassing agents to treat and/or prevent bleeding, and (b) who are at any stage of ITI treatment. Patients are included in each haemophilia treatment centre (HTC). Factor VIII (FVIII) and inhibitor assessments are performed at local laboratories of each HTC. The ITI regimen followed the national protocol of the Brazilian Ministry of Health. All PwHA starts with low-dose ITI (50 IU/kg three times weekly); high-dose regimen (100 IU/kg daily) is used if there is lack of response to the low-dose ITI. Outcomes are classified as total or partial success, and failure. Standardized case report forms with clinical, laboratory, and treatment data are collected from medical files and interviews. Blood samples are collected for genetic and immune biomarkers at the time of inclusion in the study and at the end of ITI. The study is ongoing and, currently, 202/250 (80.8%) PwHA from 15 HTCs have been included. BrazIT Study is the largest cohort of PwHA and inhibitor under treatment with the same ITI regimen reported to date. This study is likely to contribute with novel predictors of ITI response.

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