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New research suggests that starting prophylactic factor VIII (FVIII) therapy early in life can preserve joint health in patients with hemophilia A.
In studying data on more than 6000 patients, researchers found the use of FVIII prophylaxis increased from 1999 to 2010.
And the rate of bleeding events, including joint bleeds, decreased over this time period.
The study also indicated that starting FVIII prophylaxis before age 4 decreases a patient’s risk of losing normal range of motion (ROM) in his joints.
Marilyn J. Manco-Johnson, MD, of the University of Colorado Denver in Aurora, Colorado, and her colleagues reported these findings in Blood.
The team studied information collected from 6196 males between 2 and 69 years of age with severe hemophilia A. Altogether, these patients had 26,614 visits to US hemophilia treatment centers between 1999 and 2010.
The data collected from the clinic visits were used to examine trends in FVIII prophylaxis use and changes in the patients’ health over the study period.
Prophylaxis and bleeds
Overall, prophylaxis use increased from 31% in 1999 to 59% in 2010. Three-quarters of patients younger than 20 years of age were on prophylaxis by 2010.
The rate of total bleeds fell 17% from 1999 to 2010 for patients receiving prophylaxis—from a mean of 4.91 bleeds every 6 months to a mean of 4.07 bleeds every 6 months. For patients not on prophylaxis, the rate of total bleeds fell 30%, from 14.2 to 9.87 bleeds.
For patients on prophylaxis, the rate of joint bleeding fell 22%, from a mean of 3.03 bleeds every 6 months in 1999 to a mean of 2.36 bleeds every 6 months in 2010. Among patients not on prophylaxis, the rate of joint bleeding fell 23%, from 9.42 to 7.25 bleeds.
The researchers noted that rates of joint bleeding and total bleeding events in patients not on prophylaxis were roughly twice the rates for patients who were using prophylaxis.
Joint ROM
The researchers also conducted longitudinal analyses to assess joint ROM on 3078 patients who had 14,130 visits to hemophilia treatment centers during the study period.
The team found a few factors that were significantly associated with a decrease in overall joint ROM at the patients’ initial visit. This included advancing age (P<0.001), non-white race (P<0.001), and obesity (P=0.003).
Obesity was associated with a significant increase in loss of joint ROM over time as well (P<0.001).
On the other hand, starting prophylaxis before age 4 was associated with a significant decrease in loss of joint ROM over time (P=0.03).
Questions and next steps
The researchers said it isn’t clear why prophylaxis is best able to protect joint ROM when treatment is started at a very young age. And it’s not clear why hemophilia patients who might benefit from prophylaxis aren’t using it.
The team said studies are needed to understand why some hemophilia patients don’t use prophylaxis and to develop strategies that are successful in changing this behavior.
In addition, more work is needed to understand the factors that may lead to joint bleeds and to develop treatment strategies for hemophilia patients who have a higher risk for joint disease. 
New research suggests that starting prophylactic factor VIII (FVIII) therapy early in life can preserve joint health in patients with hemophilia A.
In studying data on more than 6000 patients, researchers found the use of FVIII prophylaxis increased from 1999 to 2010.
And the rate of bleeding events, including joint bleeds, decreased over this time period.
The study also indicated that starting FVIII prophylaxis before age 4 decreases a patient’s risk of losing normal range of motion (ROM) in his joints.
Marilyn J. Manco-Johnson, MD, of the University of Colorado Denver in Aurora, Colorado, and her colleagues reported these findings in Blood.
The team studied information collected from 6196 males between 2 and 69 years of age with severe hemophilia A. Altogether, these patients had 26,614 visits to US hemophilia treatment centers between 1999 and 2010.
The data collected from the clinic visits were used to examine trends in FVIII prophylaxis use and changes in the patients’ health over the study period.
Prophylaxis and bleeds
Overall, prophylaxis use increased from 31% in 1999 to 59% in 2010. Three-quarters of patients younger than 20 years of age were on prophylaxis by 2010.
The rate of total bleeds fell 17% from 1999 to 2010 for patients receiving prophylaxis—from a mean of 4.91 bleeds every 6 months to a mean of 4.07 bleeds every 6 months. For patients not on prophylaxis, the rate of total bleeds fell 30%, from 14.2 to 9.87 bleeds.
For patients on prophylaxis, the rate of joint bleeding fell 22%, from a mean of 3.03 bleeds every 6 months in 1999 to a mean of 2.36 bleeds every 6 months in 2010. Among patients not on prophylaxis, the rate of joint bleeding fell 23%, from 9.42 to 7.25 bleeds.
The researchers noted that rates of joint bleeding and total bleeding events in patients not on prophylaxis were roughly twice the rates for patients who were using prophylaxis.
Joint ROM
The researchers also conducted longitudinal analyses to assess joint ROM on 3078 patients who had 14,130 visits to hemophilia treatment centers during the study period.
The team found a few factors that were significantly associated with a decrease in overall joint ROM at the patients’ initial visit. This included advancing age (P<0.001), non-white race (P<0.001), and obesity (P=0.003).
Obesity was associated with a significant increase in loss of joint ROM over time as well (P<0.001).
On the other hand, starting prophylaxis before age 4 was associated with a significant decrease in loss of joint ROM over time (P=0.03).
Questions and next steps
The researchers said it isn’t clear why prophylaxis is best able to protect joint ROM when treatment is started at a very young age. And it’s not clear why hemophilia patients who might benefit from prophylaxis aren’t using it.
The team said studies are needed to understand why some hemophilia patients don’t use prophylaxis and to develop strategies that are successful in changing this behavior.
In addition, more work is needed to understand the factors that may lead to joint bleeds and to develop treatment strategies for hemophilia patients who have a higher risk for joint disease.
New research suggests that starting prophylactic factor VIII (FVIII) therapy early in life can preserve joint health in patients with hemophilia A.
In studying data on more than 6000 patients, researchers found the use of FVIII prophylaxis increased from 1999 to 2010.
And the rate of bleeding events, including joint bleeds, decreased over this time period.
The study also indicated that starting FVIII prophylaxis before age 4 decreases a patient’s risk of losing normal range of motion (ROM) in his joints.
Marilyn J. Manco-Johnson, MD, of the University of Colorado Denver in Aurora, Colorado, and her colleagues reported these findings in Blood.
The team studied information collected from 6196 males between 2 and 69 years of age with severe hemophilia A. Altogether, these patients had 26,614 visits to US hemophilia treatment centers between 1999 and 2010.
The data collected from the clinic visits were used to examine trends in FVIII prophylaxis use and changes in the patients’ health over the study period.
Prophylaxis and bleeds
Overall, prophylaxis use increased from 31% in 1999 to 59% in 2010. Three-quarters of patients younger than 20 years of age were on prophylaxis by 2010.
The rate of total bleeds fell 17% from 1999 to 2010 for patients receiving prophylaxis—from a mean of 4.91 bleeds every 6 months to a mean of 4.07 bleeds every 6 months. For patients not on prophylaxis, the rate of total bleeds fell 30%, from 14.2 to 9.87 bleeds.
For patients on prophylaxis, the rate of joint bleeding fell 22%, from a mean of 3.03 bleeds every 6 months in 1999 to a mean of 2.36 bleeds every 6 months in 2010. Among patients not on prophylaxis, the rate of joint bleeding fell 23%, from 9.42 to 7.25 bleeds.
The researchers noted that rates of joint bleeding and total bleeding events in patients not on prophylaxis were roughly twice the rates for patients who were using prophylaxis.
Joint ROM
The researchers also conducted longitudinal analyses to assess joint ROM on 3078 patients who had 14,130 visits to hemophilia treatment centers during the study period.
The team found a few factors that were significantly associated with a decrease in overall joint ROM at the patients’ initial visit. This included advancing age (P<0.001), non-white race (P<0.001), and obesity (P=0.003).
Obesity was associated with a significant increase in loss of joint ROM over time as well (P<0.001).
On the other hand, starting prophylaxis before age 4 was associated with a significant decrease in loss of joint ROM over time (P=0.03).
Questions and next steps
The researchers said it isn’t clear why prophylaxis is best able to protect joint ROM when treatment is started at a very young age. And it’s not clear why hemophilia patients who might benefit from prophylaxis aren’t using it.
The team said studies are needed to understand why some hemophilia patients don’t use prophylaxis and to develop strategies that are successful in changing this behavior.
In addition, more work is needed to understand the factors that may lead to joint bleeds and to develop treatment strategies for hemophilia patients who have a higher risk for joint disease.