FACTOR VIII, STARTING PROPHYLAXIS EARLY PROTECTS JOINTS

Starting prophylaxis with Factor VIII before two and a half years of age can help protect the joints of young hemophilic patients. This is the result of a recent study, which found a correlation between the age at which prophylaxis with Factor VIII is started and the protective effect on joints.

Decreased bone mineral density (BMD) is one of the most significant complications related to hemophilia and the cause of joint and bone degeneration in these patients. Recent studies have already shown that low-dose prophylaxis with Factor VIII, in severe hemophilia, preserves BMD, compared with on-demand therapy. Therefore, continuous and prolonged low-dose treatment with Factor VIII may prevent bleeding, as well as arthropathy.

We also now know that children who begin factor VIII prophylaxis before 2½ years of age have less joint damage at 6 years of age compared with children of the same age treated with factor VIII only when bleeding occurs. Moreover, follow-up of children shows that young children treated with prophylaxis starting at 2½ years of age also have healthier joints at age 18 years. The World Federation of Hemophilia also recommends starting prophylaxis before 3 years of age and before joint bleeds occur.

 

References

  • Factor VIII: Long-established role in haemophilia A and emerging evidence beyond haemostasis, Bethany Samuelson Bannow, Michael Recht, Claude Négrier, Cédric Hermans, Erik Berntorp, Hermann Eichler, Maria Elisa Mancuso, Robert Klamroth, Jamie O’Hara, Elena Santagostino, Tadashi Matsushita, Craig Kessler, DOI: 10.1016/j.blre.2019.03.002
  • Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study. Beth Boulden Warren, Dianne Thornhill, Jill Stein, Michael Fadell, J. David Ingram, Sharon Funk, Kristi L. Norton, Heidi D. Lane, Carolyn M. Bennett, Amy Dunn, Michael Recht, Amy Shapiro, Marilyn J. Manco-Johnson. Blood Adv (2020) 4 (11): 2451–2459. https://doi.org/10.1182/bloodadvances.2019001311
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Wikiphilia

Learn more about the meaning of the words you read on this page and learn about the entire glossary on hemophilia.

A pathological process consisting in a decrease in the amount of minerals (calcium, magnesium, phosphorus, etc.) contained in bone tissue. This mineral depletion weakens the bone and leads to a consequent increase in the risk of fractures.

The amount of minerals present in a square centimetre of bone.

A protein belonging to the clotting factor category, a group of enzymes involved in the blood-clotting process.

It is encoded by a gene on the long arm of the X chromosome.

Factor VIII is also known as anti-haemophilic factor (AHF).

A ereditary genetic illness, characterised by a deficiency of clotting factor VIII, that exposes the individual to a greater risk of both internal and external bleeds.

Haemophilia A is more common in males, whereas females tend to be healthy carriers of the condition.

The typical symptoms of the condition include haemarthroses (joint bleeds) and haematomas (muscle bleeds).

Damage that occurs in the joints and prevents their normal function, thereby impairing the individual’s ability to move normally. In haemophiliac patients, joint disease is usually the consequence of recurrent haemarthroses.

Condition that occurs when factor VIII activity is between 1% and 5%.

Condition that occurs when factor VIII activity is between 1% and 5%.

Bone cells able to produce new bone tissue through the excretion of extracellular matrix.

The cells that cause the break-down (resorption) of bone tissue.

Pathological condition associated with severe bone mineral mass depletion that makes the bones frailer and more prone to fractures.

Condition that occurs when factor VIII activity is <1%.