Optimizing the individualized hemophilia treatment

Every person with hemophilia is different and this demands tailor-made treatments, both in the preparations of dosage factors to be substituted and in the treatment prevention of disease-related changes in joints.

Specialized physiotherapy and weight-adjusted factor dosing may optimize the treatment

Every person with hemophilia is different and this demands tailor-made treatments, both in the preparations of dosage factors to be substituted and in the treatment prevention of disease-related changes in joints.

Experts discussed the opportunities of individualized hemophilia therapy during a Novo Nordisk satellite symposium at the 61st Annual Meeting of the Society for Thrombosis and Hemostasis Research (GTH). They emphasized the importance of intensive interdisciplinary cooperation between haemostaseologists, orthopedists, and physiotherapists. The individualized weight-based dosing strategies for factor dosage has been argued to contribute to the optimization of hemophilia treatments in the future. Findings are expected from an ongoing study looking into the pharmacokinetics of Turoctocog-alfa pertaining to a patient's Body Mass Index in hemophilia type A cases (1).

"In addition to drug therapy, people with hemophilia need an optimal multidisciplinary treatment design with curative and preventive measures that can also improve the quality of life," said Dr. Susan Halimeh, a Duisburg-based hemostaseologist. Haemophilia often causes hemorrhages in the joints, which can eventually lead to arthropathies with extensive physical disabilities and an impaired quality of life. For this situation to be avoided, according to the experts, three fields of specialization should work closely together. First, the haemostaseologist should adequately substitute coagulation factors for the prevention and treatment of hemorrhage. Second, the orthopedist should apply an examination of the joints and their functionality, and initiate targeted treatment when needed. Finally, the physiotherapist can contribute to the preservation of joint functionality with early regular measures, reduce the frequency of bleeding and pain, and prevent eventual damage (2).

However, the specialized physiotherapeutic care for people with hemophilia is not yet available in some countries. To fill this need gap, the Haem Academy for Physiotherapists was launched in 2013 where interested physiotherapists have the opportunity to further their education in the field of hemophilia patient treatments in theory and practice.

The weight-based strategies for factor dosage could further improve the hemophilia treatment in the future, said Dr. Georg Goldmann, based in Bonn. This line of analysis will be developed on the GuardianTM9 multicenter open-label study launched in October 2016 (1). This study investigates the pharmacokinetics of Turoctocog-alfa in relation to body mass index (BMI) and body mass in people with severe hemophilia A. Today - Goldman explained - more people diagnosed with coagulation disorders are, just like the healthy population, overweight.

Management of hemophilia in overweight (BMI 25-29.9 kg / m2) or obesity (BMI ≥30 kg / m2) is a major challenge, Goldmann continued. The plasma volume per kilogram of body weight is smaller in obese than in non-obese patients (3), resulting in a higher rate of recovery of the substituted coagulation factors (4). Factor VIII (FVIII) compositions are usually dosed by body weight, which may result in higher levels of FVIII activity in overweight or obese people than in healthy weight subjects after an injection (4-6). The GuardianTM9 results are intended to provide further insight into the preparations for factor dosage adjustment needed to cater for overweight cases. "We hope that the data will ultimately result in a workable model for weight-adjusted factor dosing," concluded Goldmann.

1. Persson P et al. Poster presented at the European Association of Haemophilia and Allied Disorders (EAHAD) 10th Annual Congress, February 1-3, 2017, Paris, France. P144.
2. Halimeh S et al. Haemophilia 2013; 19 (Suppl 2): 10-82.
3. Gibson JG, Evans WA. J Clin Invest 1937; 16 (3): 317-328.
4. Henrard S et al. Haematologica 2013; 98 (9): 1481-1486.
5. Wong TE et al. At J Prev Med 2011; 41 (6 Suppl 4): S369-375.
6. Young G. Hematology Am Soc Hematol Educ Program 2012; 2012: 362-368.

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