Rodeghiero F, Tosetto A, Di Bona E, Castaman G
Department of Hematology and Hemophilia, San Bortolo Hospital, Vicenza, Italy.
Am J Hematol. 1991 Jan;36(1):30-4. doi: 10.1002/ajh.2830360107.
Limited data are available about the pharmacokinetics of placenta-derived factor XIII (FXIII) concentrate in patients with FXIII deficiency. This concentrate contains only the active subunit A but not the carrier subunit B of the factor, and perplexities have been raised about its clinical use. Moreover, no data are available on its use in the rare patients completely lacking both subunit A and subunit B. Therefore, we evaluated the pharmacokinetics of a commercial placenta concentrate in three patients with FXIII deficiency: two lacking subunit A (type II) and one lacking both subunits (type I). The elimination half-life of the infused placenta subunit A in the three patients was very similar (280, 283, and 272 hr) and was also consistent with the previously reported data for plasma-derived FXIII. No thrombin-independent activity was observed in our concentrate batches. The recovery was significantly lower in the type I patient, in whom infusion of subunit A was not able to elicit a monthly increment of subunit B, as usually observed in type II patients. Monthly infusions of placenta concentrate (at higher dosage in type I patient) have been administered to our patients for two to three years and no evidence of inhibitor against factor XIII activity has been observed. We conclude that placenta concentrates may be as effective as plasma derivatives in replacement therapy of factor XIII deficiency, even in patients who lack subunit B.
关于胎盘源性凝血因子 XIII（FXIII）浓缩物在 FXIII 缺乏症患者中的药代动力学数据有限。这种浓缩物仅含有活性亚基 A，而不含有该因子的载体亚基 B，其临床应用引发了诸多困惑。此外，对于其在罕见的同时缺乏亚基 A 和亚基 B 的患者中的应用尚无数据。因此，我们评估了一种市售胎盘浓缩物在 3 例 FXIII 缺乏症患者中的药代动力学：2 例缺乏亚基 A（II 型），1 例同时缺乏两个亚基（I 型）。3 例患者中输注的胎盘亚基 A 的消除半衰期非常相似（280、283 和 272 小时），也与先前报道的血浆源性 FXIII 的数据一致。在我们的浓缩物批次中未观察到非凝血酶依赖性活性。I 型患者的回收率显著较低，在该患者中输注亚基 A 未能像在 II 型患者中通常观察到的那样引起亚基 B 的每月增加。我们的患者已接受胎盘浓缩物每月输注（I 型患者剂量更高）两到三年，未观察到针对因子 XIII 活性的抑制剂证据。我们得出结论，胎盘浓缩物在 FXIII 缺乏症的替代治疗中可能与血浆衍生物一样有效，即使在缺乏亚基 B 的患者中也是如此。
