Haemophilia care in children - Benefits of early prophylaxis for inhibitor prevention

M. E. Mancuso, L. Graca, G. Auerswald, E. Santagostino

Research output: Contribution to journalArticle

26 Citations (Scopus)

Abstract

Haemophilia therapy is aimed at treating and preventing bleeding episodes and related complications and clinical studies have shown that regular prophylaxis, started at an early age, is able to reduce physical impairment from haemophilic arthropathy. Today, the development of anti-Factor VIII (FVIII) inhibitors is the most serious treatment-related complication of haemophilia therapy and a number of genetic and environmental risk factors have been identified in the past years. Clinical data show that early start of prophylaxis and the avoidance of intensive treatment periods may protect patients from inhibitor development. The mechanisms are not completely understood; yet, recent experimental data suggest that pro-inflammatory or danger signals' may be involved in inducing tolerance vs. an effector immune response. So, exposure to a factor concentrate by itself may not be enough to trigger an immune response, while an intensive exposure to FVIII in the presence of such 'danger signals' can activate antigen-presenting cells, up-regulating co-stimulatory signals for T lymphocytes and ultimately enhancing antibody production. The 'optimal' regimen for primary prophylaxis is still not identified and barriers to prophylaxis implementation remain relevant. Key issues include the optimal age at prophylaxis onset, the optimal dosage/schedule, the proper clinical and laboratory monitoring and patients' compliance. Practical approaches to early prophylaxis as implemented in the haemophilia centres in Milan and Bremen are discussed in this respect.

Original languageEnglish
Pages (from-to)8-14
Number of pages7
JournalHaemophilia
Volume15
Issue numberSUPPL. 1
DOIs
Publication statusPublished - 2009

Fingerprint

Hemophilia A
Child Care
Factor VIII
Blocking Antibodies
Joint Diseases
Antigen-Presenting Cells
Therapeutics
Patient Compliance
Age of Onset
Antibody Formation
Appointments and Schedules
Hemorrhage
T-Lymphocytes

Keywords

  • Children
  • Haemophilia A
  • Immune tolerance induction
  • Inhibitors
  • Prophylaxis

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Haemophilia care in children - Benefits of early prophylaxis for inhibitor prevention. / Mancuso, M. E.; Graca, L.; Auerswald, G.; Santagostino, E.

In: Haemophilia, Vol. 15, No. SUPPL. 1, 2009, p. 8-14.

Research output: Contribution to journalArticle

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