title: Successful haematopoietic stem cell transplantation in 44 children from healthy siblings conceived after preimplantation HLA matching.
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.
authors: ART and Reproductive Genetics Center, Istanbul Memorial Hospital, Istanbul, Turkey.
authors: Semra
authors: S
authors: Kahraman
authors: ART and Reproductive Genetics Center, Istanbul Memorial Hospital, Istanbul, Turkey. Electronic address: cagribeyazyurek@gmail.com.
authors: Cagri
authors: C
authors: Beyazyurek
authors: School of Medicine, Pediatric Hematology and Oncology, Akdeniz University, Antalya, Turkey.
authors: Mehmet Akif
authors: MA
authors: Yesilipek
authors: Faculty of Medicine, Pediatric Hematology and Oncology, Istanbul University, Istanbul, Turkey.
authors: Gulyuz
authors: G
authors: Ozturk
authors: Faculty of Medicine, Pediatric Hematology and Oncology, Ankara University, Istanbul, Turkey.
authors: Mehmet
authors: M
authors: Ertem
authors: Faculty of Medicine, Pediatric Hematology and Oncology, Istanbul University, Istanbul, Turkey.
authors: Sema
authors: S
authors: Anak
authors: Faculty of Medicine, Pediatric Hematology and Oncology, Ege University, Izmir, Turkey.
authors: Savas
authors: S
authors: Kansoy
authors: Faculty of Medicine, Pediatric Hematology and Oncology, Ege University, Izmir, Turkey.
authors: Serap
authors: S
authors: Aksoylar
authors: Faculty of Medicine, Pediatric Bone Marrow Transplantation Unit, Hacettepe University, Ankara, Turkey.
authors: Barış
authors: B
authors: Kuşkonmaz
authors: Pediatric Hematology and Oncology, Izmir Tepecik Hospital, Izmir, Turkey.
authors: Haldun
authors: H
authors: Oniz
authors: Scientific and Medical Director, The International Center for Cell Therapy and Cancer Immunotherapy (CTCI), Tel Aviv, Israel.
authors: Shimon
authors: S
authors: Slavin
authors: Faculty of Medicine, Pediatric Hematology and Oncology, Istanbul University, Istanbul, Turkey.
authors: Zeynep
authors: Z
authors: Karakas
authors: ART and Reproductive Genetics Center, Istanbul Memorial Hospital, Istanbul, Turkey.
authors: Huseyin Avni
authors: HA
authors: Tac
authors: ART and Reproductive Genetics Center, Istanbul Memorial Hospital, Istanbul, Turkey.
authors: Nese
authors: N
authors: Gulum
authors: ART and Reproductive Genetics Center, Istanbul Memorial Hospital, Istanbul, Turkey.
authors: Gokhan Cumhur
authors: GC
authors: Ekmekci
mesh_terms: D020739:Brain Diseases, Metabolic, Inborn / Q000175:diagnosis / Q000628:therapy*; D006680:HLA Antigens; D006402:Hematologic Diseases / Q000151:congenital / Q000175:diagnosis / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D019836:Preimplantation Diagnosis; D035781:Siblings
chemical_list: D006680:HLA Antigens
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)