Syngeneic bone marrow transplantation without conditioning in a patient with paroxysmal nocturnal hemoglobinuria: In vivo evidence that the mutant stem cells have a survival advantage

Blood

Volumn 88, Issue 2, 1996, Pages 742-750

  Endo, Morito ^a   Beatty, Patrick G ^a   Vreeke, Teresa M ^a   Wittwer, Carl T ^a   Singh, Shashi P ^a   Parker, Charles J ^a,b  

^a UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^b VETERANS AFFAIRS MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; BONE MARROW TRANSPLANTATION; CELL SURVIVAL; GENE MUTATION; GRAFT SURVIVAL; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL; HEMOLYSIS; HUMAN; HUMAN CELL; PANCYTOPENIA; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PRIORITY JOURNAL; THROMBOSIS;

EID: 0029899213     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v88.2.742.bloodjournal882742     Document Type: Article

References (29)

1. Rosse WF, Ware RE: The molecular basis of paroxysmal nocturnal hemoglobinuria. Blood 86:3277, 1995
2. Miyata T, Junji T, Iida Y, Yamada N, Inoue N, Takahashi M, Maeda K, Kitani T, Kinoshita T. The cloning of PIG-A, a component in the early step of GPI-anchor biosynthesis. Science 259:1318, 1993
3. Takeda J, Miyata T, Kawagoe K, Iida Y, Endo Y, Fujita T, Takahashi M, Kitani T, Kinoshita T: Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell 73:703, 1993
4. Rosse WF: Paroxysmal nocturnal hemoglobinuria, in Rosse WF (ed): Clinical Immunohematology: Basic Concepts and Clinical Applications. Boston, MA, Blackwell Scientific, 1990, p 593
5. Rosse WF: Paroxysmal nocturnal hemoglobinuria, in Hoffman R, Benz EF Jr, Shattil SJ, Furie B, Cohen HJ, Silberstein LE (eds): Hematology Principles and Practice (ed 2). New York NY, Churchill Livingstone, 1995, p 370
6. Antin JH, Ginsburg D, Smith BR, Nathan DG, Orkin SH, Rappeport JM: Bone marrow transplantation for paroxysmal nocturnal hemoglobinuria: Eradication of the PNH clone and documentation of complete lymphohematopoietic engraftment. Blood 66:1247, 1985
7. Kolb HJ, Holler E, Bender-Götze C, Walther U, Mittermüller J, Clemm C, Bauchinger M, Gerhartz HH, Brehm G, Ledderose G, Wilmanns W: Myeloablative conditioning for marrow transplantation in myelodysplastic syndromes and paroxysmal nocturnal haemoglobinuria. Bone Marrow Transplant 4:29, 1989
8. Hershko C, Ho WG, Gale RP, Cline MJ: Cure of aplastic anaemia in paroxysmal nocturnal haemoglobinuria by marrow transfusion from identical twin: failure of peripheral-leucocyte transfusion to correct marrow aplasia. Lancet 1:945, 1979
9. Champlin RE, Feig SA, Sparkes RS, Gale RP: Bone marrow transplantation from identical twins in the treatment of aplastic anaemia: implications for the pathogenesis of the disease. Br J Haematol 56:455, 1984
10. Kawahara K, Witherspoon RP, Storb R: Marrow transplantation for paroxysmal nocturnal hemoglobinuria. Am J Hematol 39:283, 1992
11. Fefer A, Freeman H, Storb R, Hill J, Singer J, Edwards A, Thomas E: Paroxysmal nocturnal hemoglobinuria and marrow failure treated by infusion of marrow from an identical twin. Ann Intern Med 84:692, 1976
12. Ware RE, Hall SE, Rosse WF: Paroxysmal nocturnal hemoglobinuria with onset in childhood and adolescence. N Engl J Med 325:991, 1991
13. Clark RA, Nauseef WM: Isolation and functional analysis of neutrophils, in Coligan JE, Kruisbeek AM, Marguilies DH, Shevach EM, Strober W (eds): Current Protocols in Immunology. New York, NY, Wiley, 1993, p 7.23.1
14. Wittwer CT, Reed GB, Ririe KM: Rapid cycle DNA amplification, in Mullis KB, Ferre F, Gibbs RA (eds): The Polymerase Chain Reaction. Boston, MA, Birkhauser, 1994, p 174
15. Holmes BJ, Fowlkes BJ: Preparation of cells and reagents for flow cytometry, in Coligan JE, Kruisbeek AM, Marguilies DH, Shevach EM, Strober W (eds): Current Protocols in Immunology. New York, NY, Wiley, 1993, p 5.3.1
16. Endo M, Ware RE, Vreeke TM, Singh SP, Howard TA, Tomita A, Holguin MH, Parker CJ: Molecular basis of the heterogeneity of expression of glycosyl phosphatidylinositol anchored proteins in paroxysmal nocturnal hemoglobinuria. Blood 87:2546, 1996
17. Miyata T, Yamada N, Iida Y, Nishimura J, Takeda J, Kitani T, Kinoshita T: Abnormalities of PIG-A transcripts in granulocytes from patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 330:249, 1994
18. Iida Y, Takeda J, Miyata T, Inoue N, Nishimura J, Kitani T, Maeda K, Kinoshita T: Characterization of genomic PIG-A gene: A gene for GPI-anchor biosynthesis and paroxysmal nocturnal hemoglobinuria. Blood 83:3126, 1994
19. Allen RC, Zoghbi HY, Moseley AB, Rosenblatt HM, Belmont JW: Methylation of HpaII and HhaI sites near the polymorphic CAG repeat in human androgen-receptor gene correlates with X chromosome inactivation. Am J Hum Genet 51:1229, 1992
20. Mandell GL, Sande MA: Antimicrobial agents, in Gilman AG, Rall TW, Nies AS, Taylor P (eds): Goodman and Gilman's The Pharmacological Basis of Therapeutics (ed 8). New York, NY, Pergamon, 1990, p 1061
21. Graham DL, Gastineau DA: Paroxysmal nocturnal hemoglobinuria as a marker for clonal myelopathy. Am J Med 93:671, 1992
22. Bessler M, Mason P, Hillmen P, Luzzatto L: Somatic mutations and cellular selection in paroxysmal nocturnal hemoglobinuria. Lancet 343:951, 1994
23. -, phosphatidylinositolglycan-anchor-deficient T lymphocytes after in vivo application of Campath-1H for refractory B-cell non-Hodgkin lymphoma. Blood 86:1487, 1995
24. van Kamp H, Landegent JE, Jansen RPM, Willemze R, Fibbe WE: Clonal hematopoiesis in patients with acquired aplastic anemia. Blood 78:3209, 1991
25. Yamada N, Miyata T, Maeda K, Kitani T, Takeda J, Kinoshita T: Somatic mutations of PIG-A gene found in Japanese patients with paroxysmal nocturnal hemoglobinuria. Blood 85:885, 1995
26. Turhan AG, Humphries RK, Phillips GL, Eaves AC, Eaves CJ: Clonal hematopoiesis demonstrated by X-linked DNA polymorphisms after allogeneic bone marrow transplantation. N Engl J Med 320:1655, 1989
27. Oni SB, Osunkoya BO, Luzzatto L: Paroxysmal nocturnal hemoglobinuria: Evidence of monoclonal origin of abnormal red cells. Blood 36:145, 1970
28. Josten KM, Tooze JA, Borthwick-Clarke C, Gordon-Smith EC, Rutherford TR: Acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria: Studies on clonality Blood 78:3162, 1991
29. Ohashi H, Hotta T, Ichikawa A, Kinoshita T, Taguchi R, Kiguchi T, Ikezawa H, Saito H: Peripheral blood cells are predominantly chimeric of affected and normal cells in patients with paroxysmal nocturnal hemoglobinuria: Simultaneous investigation of clonality and expression of glycosylphosphatidylinositol-anchored proteins. Blood 83:853, 1994