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Reprod Biomed Online. 2014 Sep;29(3):340-51. doi: 10.1016/j.rbmo.2014.05.010. Epub 2014 Jun 12.

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

Author information

1
ART and Reproductive Genetics Center, Istanbul Memorial Hospital, Istanbul, Turkey.
2
ART and Reproductive Genetics Center, Istanbul Memorial Hospital, Istanbul, Turkey. Electronic address: cagribeyazyurek@gmail.com.
3
School of Medicine, Pediatric Hematology and Oncology, Akdeniz University, Antalya, Turkey.
4
Faculty of Medicine, Pediatric Hematology and Oncology, Istanbul University, Istanbul, Turkey.
5
Faculty of Medicine, Pediatric Hematology and Oncology, Ankara University, Istanbul, Turkey.
6
Faculty of Medicine, Pediatric Hematology and Oncology, Ege University, Izmir, Turkey.
7
Faculty of Medicine, Pediatric Bone Marrow Transplantation Unit, Hacettepe University, Ankara, Turkey.
8
Pediatric Hematology and Oncology, Izmir Tepecik Hospital, Izmir, Turkey.
9
Scientific and Medical Director, The International Center for Cell Therapy and Cancer Immunotherapy (CTCI), Tel Aviv, Israel.

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)

PMID:
25066893
DOI:
10.1016/j.rbmo.2014.05.010
[Indexed for MEDLINE]
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