Yazarlar : Iorio A, Puccetti P, Makris M.

Yayın : Blood.

Yayın Yılı : 2012

Pubmed Linki : http://www.ncbi.nlm.nih.gov/pubmed/22692511

Konu : Hemofili

Literatür İçeriği :  

Abstract

The development of alloantibodies or inhibitors is now the most serious complication a patient with severe hemophilia can experience as a result of treatment with clotting factor concentrates. Although common in previously untreated patients, inhibitor development is rare in multiply exposed, well-tolerized individuals. There has been a non-evidence-based reluctance to change concentrate, due to a perceived higher inhibitor risk following the switch, even though most patients are now likely to be on a concentrate they have not started with. Inhibitors in previously treated patients are seen in approximately 2 per 1,000 patient/years, which makes it difficult to study and compare rates among different products. Because the baseline inhibitor risk in previously treated patients may vary over time, it is important to compare the risk in patients switching to a new product with that in a parallel control group of non- switching individuals, or within a case-controlled study. The study designs imposed by regulators are suboptimal in detecting immunogenicity signals. The issue of immunogenicity of new products is likely to gain more relevance in the near future, with a call for effective postmarketing surveillance studies for all of the new engineered FVIII concentrates with prolonged half-lives that are likely to enter clinical practice.

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