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Updated: Jun 26, 2026
08:13
Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice
Published on: September 30, 2021
Primary prophylaxis in children with haemophilia
Antonio Coppola1, Mirko Di Capua, Ciro De Simone
1Centro di Riferimento Regionale per le Emocoagulopatie, Università Federico II, Napoli, Italia. antocopp@unina.it
Blood Transfusion = Trasfusione Del Sangue
|December 25, 2008
Summary
Prophylaxis, the regular administration of clotting factors, is the recommended treatment for severe hemophilia to prevent bleeding and joint damage. Early initiation of prophylaxis in children leads to better long-term joint health outcomes.
Area of Science:
- Hematology
- Pediatrics
- Preventive Medicine
Background:
- Clinical observations in moderate hemophiliacs showed fewer bleeds and less joint deterioration.
- Pioneering prophylaxis in Sweden demonstrated its long-term benefits.
- Severe hemophilia treatment historically relied on on-demand factor replacement.
Purpose of the Study:
- To review the evidence supporting prophylaxis in severe hemophilia.
- To summarize data informing current clinical practice of primary and early secondary prophylaxis in children.
- To discuss optimal prophylaxis regimens and implementation barriers.
Main Methods:
- Review of observational studies and recent randomized trials.
- Analysis of data on joint outcomes related to prophylaxis initiation.
- Synthesis of evidence from global health organizations' recommendations.
Main Results:
- Prophylaxis significantly reduces the risk of hemophilic arthropathy compared to on-demand treatment.
- Earlier initiation of prophylaxis, especially primary prophylaxis, is associated with superior joint outcomes.
- Randomized trial evidence now supports findings from observational studies.
Conclusions:
- Current clinical practice for primary (and early secondary) prophylaxis in children with severe hemophilia is based on robust evidence.
- Prophylaxis is a cornerstone of modern hemophilia management, preventing long-term joint damage.
- Further research is needed on optimal regimens and overcoming barriers to widespread prophylaxis implementation.
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