Thromb Haemost 2016; 115(05): 913-920
DOI: 10.1160/TH15-08-0664
Review Article
Schattauer GmbH

Practical considerations in choosing a factor VIII prophylaxis regimen: Role of clinical phenotype and trough levels

Rolf Ljung
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
,
Kathelijn Fischer
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
,
Manuel Carcao
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
,
Elena Santagostino
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
,
Marilyn J. Manco-Johnson
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
,
Prasad Mathew
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
1   Lund University, Department of Clinical Sciences Lund – Pediatrics, Skåne University Hospital, Malmö, Sweden
› Author Affiliations
Further Information

Publication History

Received: 21 August 2015

Accepted after major revision: 28 January 2015

Publication Date:
06 December 2017 (online)

Summary

Current therapy for haemophilia A is guided by severity of the disease, which in turn is best reflected in patients' endogenous factor VIII activity levels. For patients with severe haemophilia (particularly children), prophylaxis with continuous routine factor replacement has become standard of care in developed countries and is gradually becoming the standard of care in developing countries. The question arises then: what is an appropriate prophylaxis regimen to prevent bleeding events and arthropathy, while also maximizing patient quality of life and taking into consideration the costs of prophylaxis? Should all patients be treated with one standard, fixed prophylaxis regimen, or should prophylaxis be individualised for each patient? If so, what factors need to be considered in choosing the appropriate dose and frequency of factor administration? If prophylaxis is tailored to the individual patient, then patient-related factors (bleeding phenotype, activity profiles, age, joint status) and product-specific factors (half-life of the replacement factor in the individual patient) will determine the choice of regimen, whether it be a fixed-regimen prophylaxis or prophylaxis that is tailored to patient activity and bleeding risk. Regardless of the choice of prophylaxis regimen, for any regimen to be effective, adherence to therapy is key to optimising outcomes.

* International Network of Pediatric Hematologists


 
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