泰国首例亚洲单中心 HLA 配型囊胚植入前遗传学诊断用于预防地中海贫血症，并随后对 12 名受影响的同胞进行根治性造血干细胞移植。
The First Asian, Single-Center Experience of Blastocyst Preimplantation Genetic Diagnosis with HLA Matching in Thailand for the Prevention of Thalassemia and Subsequent Curative Hematopoietic Stem Cell Transplantation of Twelve Affected Siblings.
机构信息
Superior A.R.T., 1126/2 Vanit Building II, New Petchburi Road, Makkasan, Ratchathewi, Bangkok 10400, Thailand.
THAI StemLife, 566/3 Soi Ramkhamhaeng 39 (Thepleela 1), Prachaouthit Rd., Wangthonglang, Wangthonglang, Bangkok 10310, Thailand.
出版信息
Biomed Res Int. 2020 Jun 26;2020:5292090. doi: 10.1155/2020/5292090. eCollection 2020.
RESULTS
In 221 cycles from 138 patients (104 cycles requiring HLA matching), 90.5% had embryo(s) biopsied for genetic testing. There were 119 embryo transfers for thalassemia (76) and thalassemia-HLA cases (43), respectively, resulting in overall clinical pregnancy rates of 54.6%, implantation rates of 45.7%, and live birth rates of 44.1%. Our dataset included fifteen PGD-HLA live births with successful HSCT in twelve affected siblings, 67% using umbilical cord blood stem cells (UCBSC) as the only SC source.
CONCLUSIONS
We report favorable thalassemia PGD and PGD-HLA laboratory and clinical outcomes from a single center. The ultimate success in PGD-HLA is of course the cure of a thalassemia-affected sibling by HSCT. Our PGD-HLA HSCT series is the first and largest performed entirely in Asia with twelve successful and two pending cures and predominant UCBSC use.
结果
在 138 名患者的 221 个周期（104 个周期需要 HLA 匹配）中，90.5%的患者对胚胎进行了基因检测。分别有 119 个胚胎用于地中海贫血（76 个）和地中海贫血-HLA 病例（43 个）的转移，总临床妊娠率为 54.6%，着床率为 45.7%，活产率为 44.1%。我们的数据集包括 15 例 PGD-HLA 活产，12 例受影响的兄弟姐妹接受了成功的 HSCT，其中 67%使用脐带血干细胞（UCBSC）作为唯一的干细胞来源。
结论
我们报告了来自单一中心的有利的地中海贫血症 PGD 和 PGD-HLA 实验室和临床结果。PGD-HLA 的最终成功当然是通过 HSCT 治愈受地中海贫血影响的兄弟姐妹。我们的 PGD-HLA HSCT 系列是亚洲首例也是最大的系列，有 12 例成功治愈和 2 例待治愈，且主要使用 UCBSC。
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