The International Immune Tolerance Induction Study and its follow-up study on Japanese hemophilia A patients with inhibitors.
The International Immune Tolerance Induction Study and its follow-up study on Japanese hemophilia A patients with inhibitors.
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国际免疫耐受诱导研究及其对日本甲型血友病患者抑制剂的后续研究。
DOI：
10.1007/s12185-015-1734-z
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发表时间：
2015
期刊：
影响因子：
--
通讯作者：
Fukutake K
中科院分区：
文献类型：
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作者：
Yoshioka A;Ishii E;Ueno T;Usami I;Kobayashi M;Kobayashi R;Sotomatsu M;Shirahata A;Suzuki T;Taki M;Ishida Y;Matsushita T;Shima M;Nogami K;Sakai M;Kigasawa H;Fukutake K
在存在抑制物的血友病A患者中进行的国际免疫耐受诱导（I-ITI）研究共纳入115例受试者中的16例日本患者。该组日本患者的结果为11例I-ITI研究结束时，3例I-ITI研究期间，2例耐受预防性治疗。低剂量组和高剂量组之间的成功率无显著差异（研究I）。在征得同意的情况下，在日本对14例病例进行了连续的独立随访调查（研究II）。在I-ITI研究结束时，10例病例退出研究。在这10例病例中，7例成功病例中的7例在随访研究结束时仍保持临床成功，1例部分成功变为完全成功，而第2例复发，1例失败随后被评估为部分成功。I-ITI研究结束时正在研究的四个病例在随访研究结束时被分类为三个成功和一个失败。结果，随访研究结束时的状态为：11例ITI成功（78.6%）; 1例部分成功; 1例失败; 1例复发。因此，ITI随访研究有助于提供抑制剂的长期预后测定。
The International Immune Tolerance Induction (I-ITI) Study in hemophilia A patients with inhibitors included 16 Japanese patients among a total of 115 test subjects. The results within this group of Japanese patients were 11 cases of I-ITI off-study, three cases of I-ITI on-study, and two cases of tolerance on prophylaxis. There was no significant difference in success rate between the low-dose and high-dose groups (Study I). Successively, independent follow-up survey in Japan was conducted in 14 cases, with consent (Study II). Ten cases were off-study at the end of the I-ITI Study. Of these 10 cases, seven of seven successful cases remained clinical successes at the end of the follow-up study, one partial success became a full success while a second relapsed, and one failure was subsequently evaluated as a partial success. Four cases that were on-study at the end of I-ITI Study were classified as three successes and one failure at the end of the follow-up study. As a result, the status at the end of follow-up study was: 11 ITI successes (78.6 %); one partial success; one failure; and one relapse. Thus, the ITI follow-up study was helpful in providing a long-term prognostic determination of inhibitors.