Current management of the hemophilic child: a demanding interlocutor. Quality of life and adequate cost-efficacy analysis

Pediatr Hematol Oncol. 2014 Nov;31(8):687-702. doi: 10.3109/08880018.2014.930768. Epub 2014 Jul 9.

Abstract

Hemophilias are the most known inherited bleeding disorders. The challenges in the management of hemophilic children are different from those in adults: prophylaxis regimen removed the hallmark of crippling disease with lifelong disabilities; individualized regimens are being implemented in order to overcome venous access problems. Presently, at least in high-income countries, advances in treatment of hemophilia resulted in continuous improvement of the patients' quality of life and life expectancy. Inhibitors remain the most severe complication of hemophilia therapy. The treatment' compliance is the key to achieve a successful management. The patient, his family, the medical and psychological team are the players of a comprehensive care system. The current management of hemophilic children is the example of huge resource investments enabling long-term benefits in particular quality of life as a primary objective of the healthcare process.

Keywords: children; cost-analysis; health-related quality of life; hemophilia; prophylaxis; replacement treatment.

Publication types

  • Review

MeSH terms

  • Child
  • Child, Preschool
  • Cost-Benefit Analysis
  • Delivery of Health Care / economics
  • Disease Management
  • Factor IX / administration & dosage
  • Factor IX / genetics
  • Factor IX / metabolism
  • Factor VIII / administration & dosage
  • Factor VIII / genetics
  • Factor VIII / metabolism
  • Hemophilia A / blood
  • Hemophilia A / complications
  • Hemophilia A / economics
  • Hemophilia A / prevention & control
  • Hemophilia A / therapy*
  • Humans
  • Quality of Life* / psychology
  • Risk Factors

Substances

  • Factor VIII
  • Factor IX