دانلود رایگان مقاله انگلیسی تنظیم مراحل برای درمان شخصی بیماری هموفیلی: نقش فارماکوکینتیک - الزویر 2018

ABSTRACT

Replacement therapy with clotting factor concentrates (CFC) is the mainstay of treatment in hemophilia. Its widespread application has led to a dramatic decrease in morbidity and mortality in patients, with concomitant improvement of quality of life. However, dosing is challenging and costs are high. This review discusses benefits and limitations of pharmacokinetic (PK)-guided dosing of replacement therapy as an alternative for current dosing regimens. Dosing of CFC is now primarily based on body weight and based on its in vivo recovery (IVR). Benefits of PK-guided dosing include individualization of treatment with better targeting, more flexible blood sampling, increased insight into association of coagulation factor levels and bleeding, and potential overall lowering of overall costs. Limitations include a slight burden for the patient, and availability of closely collaborating, experienced clinical pharmacologists.

4. Conclusion

We believe that PK-guided dosing deserves attention as a means of ensuring the individualization of treatment in hemophilia since benefits are significant and limitations can be overcome. The burden for patients and parents appears to be minimal. Accordingly, we call on patients, medical professionals, clinical pharmacologists, hemostatic laboratories and pharmaceutical companies to join hands in applying this approach for all CFCs, in hemophilia and other bleeding disorders requiring CFC replacement therapy.