Successful hematopoietic stem cell transplantation for Fanconi anemia from an unaffected HLA-genotype-identical sibling selected using preimplantation genetic diagnosis

Blood

Volumn 103, Issue 3, 2004, Pages 1147-1151

  Grewal, Satkiran S ^a   Kahn, Jeffrey P ^a   MacMillan, Margaret L ^a   Ramsay, Norma K C ^a   Wagner, John E ^a  

^a UNIVERSITY OF MINNESOTA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOPHOSPHAMIDE; CYCLOSPORIN A; MESNA; OXYMETHOLONE; PREDNISONE;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; CASE REPORT; CLINICAL FEATURE; DISEASE COURSE; EMBRYO TRANSFER; FANCONI ANEMIA; FEMALE; FERTILIZATION IN VITRO; GASTROENTERITIS; GENETIC ANALYSIS; GRAFT SURVIVAL; GRAFT VERSUS HOST REACTION; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA MATCHING; HUMAN; LEGAL ASPECT; MEDICAL ETHICS; MYELODYSPLASIA; PRENATAL DIAGNOSIS; PRESCHOOL CHILD; PRIORITY JOURNAL; SIBLING; SURVIVAL RATE; TREATMENT OUTCOME; UMBILICAL CORD BLOOD; WHOLE BODY RADIATION;

EID: 1642500289     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2003-02-0587     Document Type: Article

References (27)

1. Wagner JE, Davies SM, Auerbach AD. Hematopoietic cell transplantation in the treatment of Fanconi anemia. In: Thomas ED, Blume KG, Forman SJ, eds. Hematopoietic Cell Transplantation. Maiden, MA: Blackwell Science; 1999:1204-1222.
2. Auerbach AD, Rogatko A, Schroeder-Kurth TM. International Fanconi Anemia Registry: relation of clinical symptoms to diepoxybutane sensitivity. Blood. 1989;73:391-396.
3. Butturini A, Gale RP, Verlander PC, et al. Hematologic abnormalities in Fanconi anemia: an International Fanconi Anemia Registry study. Blood. 1994;84:1650-1655.
4. Auerbach AD, Allen RG. Leukemia and preleukemia in Fanconi anemia patients: a review of the literature and report of the International Fanconi Anemia Registry. Cancer Genet Cytogenet. 1991;51:1-12.
5. Davies SM, Khan S, Wagner JE, et al. Unrelated donor bone marrow transplantation for Fanconi anemia. Bone Marrow Transplant. 1996;17:43-47.
6. MacMillan ML, Auerbach AD, Davies SM, et al. Haematopoietic cell transplantation in patients with Fanconi anaemia using alternate donors: results of a total body irradiation dose escalation trial. Br J Haematol. 2000;109:121-129.
7. Gluckman E, Auerbach AD, Horowitz MM, et al. Bone marrow transplantation for Fanconi anemia. Blood. 1995;86:2856-2862.
8. Guardiola P, Pasquini R, Dokal I, et al. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation. Blood. 2000;95:422-429.
9. Kohli-Kumar M, Morris C, DeLaat C, et al. Bone marrow transplantation in Fanconi anemia using matched sibling donors. Blood. 1994;84:2050-2054.
10. Wagner JE, Auerbach AD, Gandham S, et al. Unrelated donor hematopoietic cell transplantation for Fanconi anemia: impact of patient age, pretransplant performance status, prior androgen and fludarabine based conditioning on survival [abstract]. Blood. 2001;98:814.
11. Handyside AH, Pattinson JK, Penketh RJ, et al. Biopsy of human preimplantation embryos and sexing by DNA amplification. Lancet. 1989;1:347-349.
12. Coutelle C, Williams C, Handyside A, et al. Genetic analysis of DNA from single human oocytes: a model for preimplantation diagnosis of cystic fibrosis. BMJ. 1989;299:22-24.
13. Auerbach AD, Wolman SR. Susceptibility of Fanconi's anaemia fibroblasts to chromosome damage by carcinogens. Nature. 1976;261:494-496.
14. Verlinsky Y, Rechitsky S, Schoolcraft W, Strom C, Kuliev A. Preimplantation diagnosis for Fanconi anemia combined with HLA matching. JAMA. 2001;285:3130-3133.
15. Blasczyk R, Hahn U, Wehling J, Huhn D, Salama A. Complete subtyping of the HLA-A focus by sequence-specific amplification followed by direct sequencing or single-strand conformation polymorphism analysis. Tissue Antigens. 1995;46:86-95.
16. Yoshida M, Kimura A, Numano F, Sasazuki T. Polymerase-chain-reaction-based analysis of polymorphism in the HLA-B gene. Hum Immunol. 1992;34:257-266.
17. Rubinstein P, Dobrila L, Rosenfield RE, et al. Processing and cryopreservation of placenta/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci U S A. 1995;92:10119-10122.
18. Scharf SJ, Smith AG, Hansen JA, McFarland C, Erlich HA. Quantitative determination of bone marrow transplant engraftment using fluorescent polymerase chain reaction primers for human identity markers. Blood. 1995;85:1954-1963.
19. Handyside AH, Lesko JG, Tarin JJ, Winston RM, Hughes MR. Birth of a normal girl after in vitro fertilization and preimplantation diagnostic testing for cystic fibrosis. N Engl J Med. 1992;327:905-909.
20. Sermon K, Lissens W, Joris H, et al. Clinical application of preimplantation diagnosis for myotonic dystrophy. Prenat Diagn. 1997;17:925-932.
21. Xu K, Shi ZM, Veeck LL, Hughes MR, Rosenwaks Z. First unaffected pregnancy using preimplantation genetic diagnosis for sickle cell anemia. JAMA. 1999;281:1701-1706.
22. Lucarelli G, Galimberti M, Polchi P, et al. Bone marrow transplantation in thalassemia. Hematol Oncol Clin North Am. 1991;5:549-556.
23. MacMillan ML, Nagler A, Weisdorf DJ, Wagner JE. Successful engraftment without radiation after fludarabine based regimen in patients with Fanconi anemia undergoing related donor hematopoietic cell transplantation [abstract]. Blood. 2001;98:419.
24. Pub L No. 105-277, 112 Stat 2681.
25. Hoffman DI, Zellman GL, Fair CC, et al. Cryopreserved embryos in the United States and their availability for research. Fertil Steril. 2003;79:1063-1069.
26. Dickson v St. Mary's Hospital, No. CV-97-0138599-S (Waterbury, Conn, 2002).
27. Ferrell v Rosenbaum, 691 A.2d641 (D.C. 1997).