Related Experiment Video

Updated: Aug 1, 2026

Catheter Ablation in Combination With Left Atrial Appendage Closure for Atrial Fibrillation
28:13

Catheter Ablation in Combination With Left Atrial Appendage Closure for Atrial Fibrillation

Published on: February 26, 2013

Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis

P S Smith1, S M Teutsch, P A Shaffer

  • 1Department of Pediatrics, Brown University, Providence 02903, USA.

The Journal of Pediatrics
|September 1, 1996
PubMed

Insights

Prophylactic care for severe hemophilia A significantly reduces bleeding events but incurs substantial costs. Cost-effectiveness improves with longer treatment duration and lower concentrate prices.

Area of Science:

  • Hematology
  • Health Economics
  • Pediatric Care

Background:

  • Severe hemophilia A requires lifelong management to prevent bleeding complications.
  • Current treatment paradigms include episodic care for bleeding events and prophylactic infusions.
  • The cost-effectiveness of these approaches requires detailed analysis.

Purpose of the Study:

  • To evaluate the incremental cost-effectiveness of prophylactic versus episodic care in boys with severe hemophilia A.
  • To compare the economic impact of different prophylactic infusion durations.

Main Methods:

  • A comparative analysis using charge data from 11 U.S. hemophilia treatment centers.
  • Data collected from 70 boys on episodic care and 27 boys on prophylactic care over two years.
  • A cost-effectiveness model developed for hypothetical patients from ages 3 to 50 years.

Main Results:

  • Prophylactic care resulted in significantly fewer bleeding events (median 3 vs 31 annually) but higher concentrate use.
  • Factor VIII concentrate constituted over 93% of costs for both care types.
  • Prophylaxis from ages 3-20 cost $1100 per bleeding event prevented; from 3-50 years, $1380.
  • Total prophylactic costs could match episodic costs if concentrate prices were halved.

Conclusions:

  • Prophylactic care substantially decreases bleeding frequency and is expected to prevent joint damage.
  • Despite significant costs, prophylaxis offers clinical benefits in managing severe hemophilia A.
  • Economic viability of prophylaxis is sensitive to Factor VIII concentrate pricing.
Abstract

More Related Videos

Rapid Point-of-Care Assay of Enoxaparin Anticoagulant Efficacy in Whole Blood11:17

Rapid Point-of-Care Assay of Enoxaparin Anticoagulant Efficacy in Whole Blood

Published on: October 12, 2012

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice08:13

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice

Published on: September 30, 2021

Related Concept Videos

Anticoagulant Drugs: Low-Molecular-Weight Heparins01:30

Anticoagulant Drugs: Low-Molecular-Weight Heparins

Hemostasis is a crucial process that prevents excessive blood loss from damaged blood vessels. It involves various mechanisms such as vasoconstriction, platelet adhesion and activation, and fibrin formation. The importance of each mechanism depends on the type of vessel injury. In contrast, thrombosis is the abnormal formation of a blood clot within the blood vessels, leading to potential complications if the clot obstructs blood flow. Thrombosis can be caused by increased coagulability of the...
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches01:23

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches

Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
Pharmaceutical Alternatives: Stability-Related Therapeutic Nonequivalence01:22

Pharmaceutical Alternatives: Stability-Related Therapeutic Nonequivalence

Generic intravenous (IV) drugs are considered bioequivalent to their branded counterparts due to their 100% bioavailability upon administration. However, variations in stability among different drug products can significantly influence their therapeutic performance, even if they are pharmaceutically equivalent.Cefuroxime, a prophylactic antimicrobial, is often used as a single-dose IV injection for patients undergoing coronary artery bypass grafting surgery. A 3 g dose typically provides...
Drug Accumulation During Multiple Dosing: Intermittent IV Infusions01:24

Drug Accumulation During Multiple Dosing: Intermittent IV Infusions

Intermittent intravenous (IV) infusion is a method of drug administration where medications are delivered over short infusion periods followed by intervals of no drug delivery. This approach helps to prevent sustained high drug concentrations in the bloodstream, reducing the risk of adverse effects associated with prolonged exposure. Unlike continuous infusion, steady-state concentrations may not be achieved during a single dosing cycle but can be reached through repeated...
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions01:15

Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions

PK–PD modeling has significantly influenced FDA regulatory decisions, particularly drug approval, dosage optimization, and labeling. These models integrate pharmacokinetics (PK) and pharmacodynamics (PD) to predict drug behavior and effects, aiding in optimizing dosing regimens and enhancing the probability of clinical trial success.One notable example is Nesiritide (Natrecor®), a recombinant human brain natriuretic peptide for treating acute decompensated congestive heart failure (CHF).
Venous Thrombosis III: Interprofessional Care01:29

Venous Thrombosis III: Interprofessional Care

Venous thrombosis requires effective prevention and treatment strategies to improve patient outcomes and reduce potential complications.Prevention StrategiesHealthcare providers must prioritize preventing venous thromboembolism (VTE) for all adult patients upon admission. Interventions depend on bleeding and thrombosis risk, medical history, current medications, diagnoses, planned procedures, and patient preferences. Patients on bed rest should change positions every two hours and, if not...