CLINICAL PHARMACOKINETICS OF A PLACENTA-DERIVED FACTOR-XIII CONCENTRATE IN TYPE-I AND TYPE-II FACTOR-XIII DEFICIENCY
CLINICAL PHARMACOKINETICS OF A PLACENTA-DERIVED FACTOR-XIII CONCENTRATE IN TYPE-I AND TYPE-II FACTOR-XIII DEFICIENCY
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DOI：
10.1002/ajh.2830360107
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发表时间：
1991-01-01
影响因子：
12.8
通讯作者：
CASTAMAN, G
中科院分区：
文献类型：
--
作者：
RODEGHIERO, F;TOSETTO, A;CASTAMAN, G
关于胎盘衍生因子XIII（FXIII）浓缩物在FXIII缺乏症患者中的药代动力学数据有限。 这种浓缩物只含有因子的活性亚单位A，而不含有因子的载体亚单位B，因此对其临床应用提出了疑问。 此外，还没有关于其在完全缺乏亚基A和亚基B的罕见患者中使用的数据。 因此，我们评估了3例FXIII缺乏症患者的市售胎盘浓缩物的药代动力学：2例缺乏亚单位A（II型），1例缺乏两种亚单位（I型）。3例患者中输注胎盘亚单位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来源FXIII数据一致。 在我们的浓缩物批次中未观察到凝血酶非依赖性活性。 I型患者的恢复率显著较低，A亚单位输注不能引起B亚单位每月增加，而这通常在II型患者中观察到。 每月输注的胎盘浓缩物（在较高剂量的I型患者）已管理到我们的患者为两到三年，并没有证据表明对因子XIII活性的抑制剂已observed.We得出结论，胎盘浓缩物可能是有效的血浆衍生物在因子XIII缺乏症的替代治疗，即使在患者缺乏亚单位B。
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.