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[重型β地中海贫血与骨髓同种异体移植。两例病例及文献研究]

[Major beta-thalassemia and bone marrow allograft. Two cases and a study of the literature].

作者信息

Frappaz D, Freycon F, Richard O, Galacteros F, Bertheas M F, Dutou L, Bouteille M

机构信息

Service de pédiatrie, Hôpital Nord, Saint-Priest-en Jarez, France.

出版信息

Pediatrie. 1987;42(8):637-44.

PMID:3329338
Abstract

A 5 year 9 month-old boy has received a bone marrow allograft for beta-thalassaemia major. Conditioning included busulfan: 16 mg/kg, cyclophosphamide 200 mg/kg and a (6 Gy) thoracoabdominal irradiation. After a 16 months follow-up, the child is currently in complete remission without treatment with all the markers of his donor. His 9 year-old sister has been allografted for beta-thalassaemia major, with the same conditioning regimen. After engraftment, rejection occurred at day 85 with severe aplastic anaemia. A second graft was performed with the same donor without engraftment and the patient died at day 18 of pneumonitis. A review of the literature is proposed and the ethical choices are discussed.

摘要

一名5岁9个月大的男孩因重型β地中海贫血接受了骨髓同种异体移植。预处理方案包括白消安:16mg/kg、环磷酰胺200mg/kg以及一次(6Gy)胸腹照射。经过16个月的随访,该患儿目前处于完全缓解状态,无需接受治疗,且具有其供体的所有标记物。他9岁的姐姐也因重型β地中海贫血接受了同种异体移植,采用了相同的预处理方案。移植后,在第85天发生排斥反应,伴有严重再生障碍性贫血。使用同一供体进行了第二次移植,但未成功植入,患者在第18天死于肺炎。本文对相关文献进行了综述,并讨论了伦理选择。

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