博元婦產科

44個救命寶寶的經驗rescue baby

有一份來自於以色列特拉維夫大學的研究，他們針對10種疾病去使用胚胎著床前配對HLA人類淋巴球注射抗原配對成功，讓這個胚胎懷孕之後用臍帶血來救寶寶，稱之為救命寶寶叫做rescue baby，有哪10種病適合救命寶寶呢？following 10 indications; beta-thalassaemia, Wiskott-Aldrich syndrome, Fanconi anaemia, sickle cell anaemia, acute myeloid leukaemia, acute lymphoblastic leukaemia, Glanzmann's thrombasthaenia, Diamond-Blackfan anaemia, X-linked adrenoleukodystrophy and mucopolysaccharidosis type I.

1.乙型地中海型貧血beta-thalassaemia

2.為可特症候群

3.法科寧貧血症

4.鐮刀貧血

5.AML急性骨血性白血病

6.ALL急性的淋巴球白血病ALL

7.格雷曼士紫斑症GT

8.帶蒙貧血

9.X性連遺傳的腎上腺白質第一型疾病

在這44個救命寶寶病沒有嚴重的合併症，對於捐卵者或捐贈者或受贈者，而這44個裡面有44個用臍帶血去救，有4個是治療失敗的，這4個是乙型地中海型貧血，而胚胎著床前進行HLA配對是一個相當可信賴的技術。

Reprod Biomed Online. 2014 Jun 12. pii: S1472-6483(14)00308-3. doi: 10.1016/j.rbmo.2014.05.010. [Epub ahead of print]

Successful haematopoietic stem cell transplantation in 44 children from healthy siblings conceived after preimplantation HLA matching.

Kahraman S1, Beyazyurek C2, Yesilipek MA3, Ozturk G4, Ertem M5, Anak S4, Kansoy S6, Aksoylar S6, Kuşkonmaz B7, OOniz H8, Slavin S9, Karakas Z4, Tac HA1, Gulum N1, Ekmekci GC1.

Author information

Abstract

Haematopoietic stem cell transplantation (HSCT) remains the best therapeutic option for many acquired and inherited paediatric haematological disorders. Unfortunately, the probability of finding an HLA matched donor is limited. An alternative technique is PGD combined with HLA matching, which offers the possibility of selecting unaffected embryos that are HLA compatible with the sick child, with the aim of possible use of stem cells from the resulting baby in future. Since the first successful report for Fanconi anaemia a decade ago, the therapeutic success of this technique was reported in a few cases and for a limited number of disorders. Here, we report full recovery of 44 sick children who received HSCT from healthy infants conceived after pre-implantation HLA matching for the following 10 indications; beta-thalassaemia, Wiskott-Aldrich syndrome, Fanconi anaemia, sickle cell anaemia, acute myeloid leukaemia, acute lymphoblastic leukaemia, Glanzmann's thrombasthaenia, Diamond-Blackfan anaemia, X-linked adrenoleukodystrophy and mucopolysaccharidosis type I. No serious complications were observed among recipients and donors. Graft failure occurred in four children with beta-thalassaemia where a second HSCT was planned. Preimplantation HLA matching is a reliable technique and provides a realistic option for couples seeking treatment for an affected child when no HLA-matched donor is available.

KEYWORDS:

cord blood; hematopoietic stem cell transplantation (HSCT); human leukocyte antigen (HLA) matching; in vitro fertilization (IVF); inherited and acquired hematological disorders; preimplantation genetic diagnosis (PGD)