State of care for hemophilia in pediatric patients

Elena Santagostino, Alessandro Gringeri, Pier M. Mannucci

Research output: Contribution to journalArticle

18 Citations (Scopus)

Abstract

On demand treatment of bleeding episodes is still the main approach to hemophilia care of patients of any age. Prompt infusion of coagulation factor concentrate in the home setting allows treatment of hemorrhages at early onset, reducing the incidence of complications and improving the quality of life. Nevertheless, the technological evolution and progressive improvement in the safety of therapeutic products have changed the management of the disease, particularly in children. The current availability of safer concentrates has drastically reduced the risk of transmission of blood-borne infections. Innovative approaches, such as early primary prophylaxis and immune tolerance induction, have become feasible and their introduction represents a major advance in the achievement of the main therapeutic goals: control of the bleeding diathesis and elimination of inhibitors. Prophylactic regimens have been shown to be effective in preventing the occurrence not only of joint bleeding but also of arthropathy when started early in children with severe hemophilia. Inhibitor development still represents the main complication of hemophilia treatment, making concentrate administration ineffective. Immune tolerance induction by daily infusion of coagulation factor concentrate was shown to eradicate the inhibitors in 63 to 83% of patients. These intensive treatment regimens are administered at home to very young children and create the problem of adequate venous access. Subcutaneous venous ports have been used in patients with hemophilia if peripheral veins could not be frequently accessed; however, the risk of infection is an important limit to their use.

Original languageEnglish
Pages (from-to)149-157
Number of pages9
JournalPediatric Drugs
Volume4
Issue number3
Publication statusPublished - 2002

Fingerprint

Hemophilia A
Pediatrics
Hemorrhage
Immune Tolerance
Blood Coagulation Factors
Therapeutics
Joint Diseases
Disease Susceptibility
Disease Management
Infection
Veins
Patient Care
Joints
Quality of Life
Safety
Incidence

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pharmacology

Cite this

State of care for hemophilia in pediatric patients. / Santagostino, Elena; Gringeri, Alessandro; Mannucci, Pier M.

In: Pediatric Drugs, Vol. 4, No. 3, 2002, p. 149-157.

Research output: Contribution to journalArticle

@article{c524338ebf0243cb8c776cea0491c1cc,
title = "State of care for hemophilia in pediatric patients",
abstract = "On demand treatment of bleeding episodes is still the main approach to hemophilia care of patients of any age. Prompt infusion of coagulation factor concentrate in the home setting allows treatment of hemorrhages at early onset, reducing the incidence of complications and improving the quality of life. Nevertheless, the technological evolution and progressive improvement in the safety of therapeutic products have changed the management of the disease, particularly in children. The current availability of safer concentrates has drastically reduced the risk of transmission of blood-borne infections. Innovative approaches, such as early primary prophylaxis and immune tolerance induction, have become feasible and their introduction represents a major advance in the achievement of the main therapeutic goals: control of the bleeding diathesis and elimination of inhibitors. Prophylactic regimens have been shown to be effective in preventing the occurrence not only of joint bleeding but also of arthropathy when started early in children with severe hemophilia. Inhibitor development still represents the main complication of hemophilia treatment, making concentrate administration ineffective. Immune tolerance induction by daily infusion of coagulation factor concentrate was shown to eradicate the inhibitors in 63 to 83{\%} of patients. These intensive treatment regimens are administered at home to very young children and create the problem of adequate venous access. Subcutaneous venous ports have been used in patients with hemophilia if peripheral veins could not be frequently accessed; however, the risk of infection is an important limit to their use.",
author = "Elena Santagostino and Alessandro Gringeri and Mannucci, {Pier M.}",
year = "2002",
language = "English",
volume = "4",
pages = "149--157",
journal = "Paediatric Drugs",
issn = "1174-5878",
publisher = "Adis International Ltd",
number = "3",

}

TY - JOUR

T1 - State of care for hemophilia in pediatric patients

AU - Santagostino, Elena

AU - Gringeri, Alessandro

AU - Mannucci, Pier M.

PY - 2002

Y1 - 2002

N2 - On demand treatment of bleeding episodes is still the main approach to hemophilia care of patients of any age. Prompt infusion of coagulation factor concentrate in the home setting allows treatment of hemorrhages at early onset, reducing the incidence of complications and improving the quality of life. Nevertheless, the technological evolution and progressive improvement in the safety of therapeutic products have changed the management of the disease, particularly in children. The current availability of safer concentrates has drastically reduced the risk of transmission of blood-borne infections. Innovative approaches, such as early primary prophylaxis and immune tolerance induction, have become feasible and their introduction represents a major advance in the achievement of the main therapeutic goals: control of the bleeding diathesis and elimination of inhibitors. Prophylactic regimens have been shown to be effective in preventing the occurrence not only of joint bleeding but also of arthropathy when started early in children with severe hemophilia. Inhibitor development still represents the main complication of hemophilia treatment, making concentrate administration ineffective. Immune tolerance induction by daily infusion of coagulation factor concentrate was shown to eradicate the inhibitors in 63 to 83% of patients. These intensive treatment regimens are administered at home to very young children and create the problem of adequate venous access. Subcutaneous venous ports have been used in patients with hemophilia if peripheral veins could not be frequently accessed; however, the risk of infection is an important limit to their use.

AB - On demand treatment of bleeding episodes is still the main approach to hemophilia care of patients of any age. Prompt infusion of coagulation factor concentrate in the home setting allows treatment of hemorrhages at early onset, reducing the incidence of complications and improving the quality of life. Nevertheless, the technological evolution and progressive improvement in the safety of therapeutic products have changed the management of the disease, particularly in children. The current availability of safer concentrates has drastically reduced the risk of transmission of blood-borne infections. Innovative approaches, such as early primary prophylaxis and immune tolerance induction, have become feasible and their introduction represents a major advance in the achievement of the main therapeutic goals: control of the bleeding diathesis and elimination of inhibitors. Prophylactic regimens have been shown to be effective in preventing the occurrence not only of joint bleeding but also of arthropathy when started early in children with severe hemophilia. Inhibitor development still represents the main complication of hemophilia treatment, making concentrate administration ineffective. Immune tolerance induction by daily infusion of coagulation factor concentrate was shown to eradicate the inhibitors in 63 to 83% of patients. These intensive treatment regimens are administered at home to very young children and create the problem of adequate venous access. Subcutaneous venous ports have been used in patients with hemophilia if peripheral veins could not be frequently accessed; however, the risk of infection is an important limit to their use.

UR - http://www.scopus.com/inward/record.url?scp=0036214098&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0036214098&partnerID=8YFLogxK

M3 - Article

C2 - 11909007

AN - SCOPUS:0036214098

VL - 4

SP - 149

EP - 157

JO - Paediatric Drugs

JF - Paediatric Drugs

SN - 1174-5878

IS - 3

ER -