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Long-term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma-derived FVIII/VWF product: the long-term ITI study

Long-term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma-derived FVIII/VWF product: the long-term ITI study
Long-term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma-derived FVIII/VWF product: the long-term ITI study
Introduction
Immune tolerance induction (ITI) is a standard intervention to eradicate inhibitors in haemophilic patients. However, information on the long‐term condition of patients who eradicated the inhibitor totally or partially after ITI is scarce.

Aim
To perform a long‐term follow‐up to describe the status of patients reported as ITI success in the G‐ITI study.

Methods
This was an international, multicentre, observational, retrospective study of the 57 haemophilic patients who were reported as ITI success in the G‐ITI study. Demographics and post‐ITI clinical data recorded until January 2015 were extracted from the medical records. A descriptive analysis was undertaken.

Results
Forty‐four patients were evaluable. Post‐ITI follow‐up was 9.1 years in average. Thirty‐seven target joints were affected in 21 patients; 38 patients presented bleeding with a mean of 1.0 ± 1.2 episodes year−1, most of them (271/330) treated with Fanhdi® (Grifols). Around half of the patients underwent at least one surgical procedure. Most venous access complications were of expected nature, requiring hospital stay in practically all cases. Fanhdi was used in 42 patients as the regular haemophilia treatment after ITI, mainly prophylactically. Three patients (6.8%) who were being treated with Fanhdi (prophylaxis), Kogenate (prophylaxis) and Emoclot (on demand), respectively, showed inhibitor relapse (at 29, 53 and 13 months after ITI, with 0.9, 3.65 and 12.5 BU respectively). All of them were successfully tolerized after rescue ITI.

Conclusion
After ITI success, all patients continued with regular successful FVIII treatment for haemophilia for more than 9 years. The few inhibitor relapses were successfully overcome after rescue ITI.
1351-8216
859-865
Jiménez-Yuste, V.
012eb324-878f-458f-884b-272948a39e86
Oldenburg, J.
79e6962f-3ba2-47ab-aabb-b3806ac25689
Rangarajan, S.
9a5e4c7e-55ba-4a3a-b5f6-f1e269d927c3
Peiró-Jordán, R.
3f75c2fb-648a-46e1-9e3c-4923a37fd444
Santagostino, E.
43deb3c2-fa5c-4d17-a8e5-960a9119f636
Jiménez-Yuste, V.
012eb324-878f-458f-884b-272948a39e86
Oldenburg, J.
79e6962f-3ba2-47ab-aabb-b3806ac25689
Rangarajan, S.
9a5e4c7e-55ba-4a3a-b5f6-f1e269d927c3
Peiró-Jordán, R.
3f75c2fb-648a-46e1-9e3c-4923a37fd444
Santagostino, E.
43deb3c2-fa5c-4d17-a8e5-960a9119f636

Jiménez-Yuste, V., Oldenburg, J., Rangarajan, S., Peiró-Jordán, R. and Santagostino, E. (2016) Long-term outcome of haemophilia A patients after successful immune tolerance induction therapy using a single plasma-derived FVIII/VWF product: the long-term ITI study. Haemophilia, 22 (6), 859-865. (doi:10.1111/hae.12986).

Record type: Article

Abstract

Introduction
Immune tolerance induction (ITI) is a standard intervention to eradicate inhibitors in haemophilic patients. However, information on the long‐term condition of patients who eradicated the inhibitor totally or partially after ITI is scarce.

Aim
To perform a long‐term follow‐up to describe the status of patients reported as ITI success in the G‐ITI study.

Methods
This was an international, multicentre, observational, retrospective study of the 57 haemophilic patients who were reported as ITI success in the G‐ITI study. Demographics and post‐ITI clinical data recorded until January 2015 were extracted from the medical records. A descriptive analysis was undertaken.

Results
Forty‐four patients were evaluable. Post‐ITI follow‐up was 9.1 years in average. Thirty‐seven target joints were affected in 21 patients; 38 patients presented bleeding with a mean of 1.0 ± 1.2 episodes year−1, most of them (271/330) treated with Fanhdi® (Grifols). Around half of the patients underwent at least one surgical procedure. Most venous access complications were of expected nature, requiring hospital stay in practically all cases. Fanhdi was used in 42 patients as the regular haemophilia treatment after ITI, mainly prophylactically. Three patients (6.8%) who were being treated with Fanhdi (prophylaxis), Kogenate (prophylaxis) and Emoclot (on demand), respectively, showed inhibitor relapse (at 29, 53 and 13 months after ITI, with 0.9, 3.65 and 12.5 BU respectively). All of them were successfully tolerized after rescue ITI.

Conclusion
After ITI success, all patients continued with regular successful FVIII treatment for haemophilia for more than 9 years. The few inhibitor relapses were successfully overcome after rescue ITI.

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More information

Accepted/In Press date: 25 April 2016
e-pub ahead of print date: 22 June 2016
Published date: 1 November 2016

Identifiers

Local EPrints ID: 442860
URI: http://eprints.soton.ac.uk/id/eprint/442860
ISSN: 1351-8216
PURE UUID: fc71e0cf-d63c-43b4-b58e-49a416604995
ORCID for S. Rangarajan: ORCID iD orcid.org/0000-0001-7367-133X

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Date deposited: 29 Jul 2020 16:34
Last modified: 02 Jul 2022 02:07

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Contributors

Author: V. Jiménez-Yuste
Author: J. Oldenburg
Author: S. Rangarajan ORCID iD
Author: R. Peiró-Jordán
Author: E. Santagostino

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