PulseExploreJournal ClubDebatesTrendingResearchersJournals
Instagram
HomeExploreJournal ClubTrending
Synapse
⌘+K
Synapse
February 26, 2015Blood375 citationsOpen Access

Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens

JOJohannes Oldenburg

Key Points

  • The aim is to evaluate the effectiveness of prophylactic regimens in preventing joint disease in hemophilia.
  • Evaluated prophylactic application of clotting factor concentrates in children and adults.
  • Assessed outcomes such as number of bleeds and joint health over time in patients on primary, secondary, and tertiary prophylaxis.
  • Utilized ultrasound and magnetic resonance imaging for monitoring joint condition.
  • Adult patients on tertiary prophylaxis show significantly reduced bleeding episodes and fewer target joints.
  • Primary prophylaxis is linked to the gradual development of joint arthropathy over many years.
  • Individualized therapy based on patient-specific factors is expected to optimize treatment outcomes.

Abstract

Prophylactic application of clotting factor concentrates is the basis of modern treatment of severe hemophilia A. In children, the early start of prophylaxis as primary or secondary prophylaxis has become the gold standard in most countries with adequate resources. In adults, prophylaxis is reasonably continued when started as primary or secondary prophylaxis in childhood to maintain healthy joint function. Initial data support that adult patients with already existing advanced joint arthropathy benefit from tertiary prophylaxis with significantly lowered number of bleeds, almost complete absence of target joints, and less time off from work. Current prophylactic regimens, although very effective, do not completely prevent joint disease in a long-term perspective. Joint arthropathy in primary prophylaxis develops over many years, sometimes over a decade or even longer time periods. The ankle joints are the first and most severely affected joints in those patients and thus may serve in outcome assessment as an indicator of early joint arthropathy when followed by ultrasound or magnetic resonance imaging. Optimized outcome and best use of available resources is expected from individualization of therapy regimens, which comprises the individual's bleeding pattern, condition of the musculoskeletal system, level of physical activity and the pharmacokinetic profile of the substituted coagulation factor, and most recently includes novel products with extended half-lives.

Ask AI
Helpful
Bookmark
Share
View Full Paper

Cite This Study

Johannes Oldenburg (2015) studied this question.

synapsesocial.com/papers/69db1dc7498b35d3e6a3c7b7https://doi.org/10.1182/blood-2015-01-528414
Ask AI
Helpful
Bookmark
Share
View Full Paper