近期治疗方面的创新能否弥补自给自足政策中明显的不足？

Will recent innovations in therapy save perceived deficiencies in self-sufficiency policies.

作者信息

Limentani S A

机构信息

Hematology-Oncology Department, New England Medical Center, Boston, MA 02111, USA.

出版信息

Blood Coagul Fibrinolysis. 1994 Dec;5 Suppl 4:S81-4. doi: 10.1097/00001721-199412004-00021.

DOI:10.1097/00001721-199412004-00021

PMID:7795145

Abstract

Future treatment for patients with haemophilia may include the use of either gene therapy, recombinant factor VIII, recombinant factor IX, or high-purity factor IX. Studies on gene therapy are still at the pre-clinical stage, while clinical trials of recombinant factor IX are expected to start by mid-1995. High-purity factor IX concentrates are available and are the treatment of choice for patients with haemophilia B in the absence of a source of recombinant factor IX. Recombinant factor VIII provides a renewable and unlimited source of factor VIII, and is a safe and effective treatment for haemophilia A.

摘要

血友病患者未来的治疗方法可能包括使用基因疗法、重组凝血因子VIII、重组凝血因子IX或高纯度凝血因子IX。基因疗法的研究仍处于临床前阶段，而重组凝血因子IX的临床试验预计在1995年年中开始。高纯度凝血因子IX浓缩剂已有供应，在没有重组凝血因子IX来源的情况下，是B型血友病患者的首选治疗方法。重组凝血因子VIII提供了可再生且无限的凝血因子VIII来源，是治疗A型血友病的一种安全有效的方法。