Shapiro A D, Ragni M V, Lusher J M, Culbert S, Koerper M A, Bergman G E, Hannan M M
Riley Hospital for Children, Indiana University Medical Center, Indianapolis 46202-5225, USA.
Thromb Haemost. 1996 Jan;75(1):30-5.
The safety and efficacy of a monoclonal antibody purified factor IX concentrate were evaluated in two continuing trials of 32 previously untreated patients with mild, moderate, or severe hemophilia B. Patients were evaluated every 2 weeks for 24 weeks and every 3 months thereafter for at least 1 year. No patients became positive for human immunodeficiency virus antibody or hepatitis C virus antibody during the trial. Two patients developed a false-positive hepatitis B core antibody, one transiently, but neither had elevated levels of alanine aminotransferase (ALT). None of the 25 patients evaluable for non-A, non-B, non-C hepatitis by strict International Society of Thrombosis and Hemostasis criteria developed elevated levels of ALT indicative of posttransfusion infection. Anaphylaxis occurred in one subject who also developed an inhibitor to factor IX (19.3 Bethesda units). Five of the eight adverse events reported (63%) were mild in severity, and the relationship of three of these to therapy was considered remote. Hemostasis with monoclonal antibody purified factor IX concentrate was excellent in all patients.
在两项针对32例既往未经治疗的轻、中、重度B型血友病患者的连续性试验中，评估了单克隆抗体纯化的凝血因子IX浓缩物的安全性和有效性。患者在24周内每2周接受一次评估，此后每3个月评估一次，为期至少1年。试验期间，没有患者的人类免疫缺陷病毒抗体或丙型肝炎病毒抗体呈阳性。两名患者出现乙型肝炎核心抗体假阳性，其中一名为短暂性假阳性，但两人的丙氨酸氨基转移酶（ALT）水平均未升高。按照血栓与止血国际协会的严格标准，25例可评估非甲、非乙、非丙型肝炎的患者中，没有出现ALT水平升高以提示输血后感染。一名受试者发生过敏反应，该受试者还产生了针对凝血因子IX的抑制剂（19.3贝塞斯达单位）。报告的8例不良事件中有5例（63%）为轻度，其中3例与治疗的关系被认为不密切。单克隆抗体纯化的凝血因子IX浓缩物在所有患者中均具有出色的止血效果。
