High-dose cyclophosphamide for severe aplastic anemia: Long-term follow-up

Blood

Volumn 115, Issue 11, 2010, Pages 2136-2141

  Brodsky, Robert A ^a,b   Chen, Allen R ^b   Dorr, Donna ^a   Fuchs, Ephraim J ^b   Huff, Carol Ann ^b   Luznik, Leo ^b   Smith, B Douglas ^b   Matsui, William H ^b   Goodman, Steven N ^b   Ambinder, Richard F ^b   Jones, Richard J ^b  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b JOHNS HOPKINS UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUCONAZOLE; GRANULOCYTE COLONY STIMULATING FACTOR; MESNA; NORFLOXACIN; SULFAMETHOXAZOLE; THYMOCYTE ANTIBODY; TRIMETHOPRIM; VALACICLOVIR;

ADOLESCENT; ADULT; AGED; ANTIBIOTIC THERAPY; APLASTIC ANEMIA; ARTICLE; BONE MARROW TRANSPLANTATION; BRAIN HEMORRHAGE; CHILD; DISEASE SEVERITY; DRUG FATALITY; DRUG MEGADOSE; ERYTHROCYTE TRANSFUSION; EVENT FREE SURVIVAL; FEMALE; FOLLOW UP; HEMORRHAGIC CYSTITIS; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INTRAVASCULAR HEMOLYSIS; LONG TERM CARE; MAJOR CLINICAL STUDY; MALE; MYCOSIS; MYELODYSPLASTIC SYNDROME; OVERALL SURVIVAL; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PNEUMOCYSTIS PNEUMONIA; PRESCHOOL CHILD; PRIORITY JOURNAL; REFRACTORY ANEMIA; RELAPSE; SCHOOL CHILD; SEVERE APLASTIC ANEMIA; THERAPY EFFECT; TREATMENT RESPONSE;

EID: 77950412952     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2009-06-225375     Document Type: Article

References (34)

1. Brodsky RA, Jones RJ. Aplastic anaemia. Lancet. 2005;365(9471):1647-1656.
2. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006;108(8):2509-2519.
3. Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003;101(4):1236-1242. (Pubitemid 36182490)
4. Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003;289(9):1130-1135. (Pubitemid 37430327)
5. Doney K, Storb R, Buckner CD, et al. Treatment of aplastic anemia with antithymocyte globulin, high-dose corticosteroids, and androgens. Exp Hematol. 1987;15(3):239-242.
6. Scheinberg P, Nunez O, Wu C, Young NS. Treatment of severe aplastic anaemia with combined immunosuppression: anti-thymocyte globulin, ciclosporin and mycophenolate mofetil. Br J Haematol. 2006;133(6):606-611. (Pubitemid 43742774)
7. Viollier R, Passweg J, Gregor M, et al. Quality-adjusted survival analysis shows differences in outcome after immunosuppression or bone marrow transplantation in aplastic anemia. Ann Hematol. 2005;84(1):47-55. (Pubitemid 39618780)
8. Ohara A, Kojima S, Hamajima N, et al. Myelodys-plastic syndrome and acute myelogenous leukemia as a late clonal complication in children with acquired aplastic anemia. Blood. 1997;90(3): 1009-1013. (Pubitemid 27314134)
9. Brodsky RA, Sensenbrenner LL, Jones RJ. Complete remission in acquired severe aplastic anemia following high-dose cyclophosphamide. Blood. 1996;87(2):491-494.
10. Brodsky RA, Sensenbrenner LL, Smith BD, et al. Durable treatment-free remission following highdose cyclophosphamide for previously untreated severe aplastic anemia. Ann Intern Med. 2001; 135(7):477-483.
11. Brodsky RA, Chen AR, Brodsky I, Jones RJ. High-dose cyclophosphamide as salvage therapy for severe aplastic anemia. Exp Hematol. 2004; 32(5):435-440. (Pubitemid 38624327)
12. Jaime-Perez JC, Gonzalez-Llano O, Gomez-Almaguer D. High-dose cyclophosphamide in the treatment of severe aplastic anemia in children. Am J Hematol. 2001;66(1):71. (Pubitemid 32006334)
13. Tisdale JF, Dunn DE, Geller N, et al. High-dose cyclophosphamide in severe aplastic anaemia: a randomised trial. Lancet. 2000;356(9241):1554-1559.
14. Brodsky RA, Petri M, Smith BD, et al. Immunoablative high-dose cyclophosphamide without stem cell rescue for refractory severe autoimmune disease. Ann Intern Med. 1998;129(12):1031-1035. (Pubitemid 29005827)
15. Petri M, Jones RJ, Brodsky RA. High-dose cyclophosphamide without stem cell transplantation in systemic lupus erythematosus. Arthritis Rheum. 2003;48(1):166-173. (Pubitemid 36091660)
16. Moyo VM, Smith D, Brodsky I, Crilley P, Jones RJ, Brodsky RA. High-dose cyclophosphamide for refractory autoimmune hemolytic anemia. Blood. 2002;100(2):704-706. (Pubitemid 34761141)
17. Drachman DB, Jones RJ, Brodsky RA. Treatment of refractory myasthenia: "rebooting" with high-dose cyclophosphamide. Ann Neurol. 2003;53(1):29-34.
18. Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc. 1958;53:457-481.
19. Choudhury JB. Non-parametric confidence interval estimation for competing risks analysis: application to contraceptive data. Stat Med. 2002; 21(8):1129-1144.
20. Brodsky RA. Narrative review: paroxysmal nocturnal hemoglobinuria: the physiology of complement-related hemolytic anemia. Ann Intern Med. 2008;148(8):587-595.
21. Drachman DB, Adams RN, Hu R, Jones RJ, Brodsky RA. Rebooting the immune system with high-dose cyclophosphamide for treatment of refractory myasthenia gravis. Ann N Y Acad Sci. 2008;1132:305-314.
22. Krishnan C, Kaplin AI, Brodsky RA, et al. Reduction of disease activity and disability with highdose cyclophosphamide in patients with aggressive multiple sclerosis. Arch Neurol. 2008;65(8):1044-1051.
23. Storb R, Etzioni R, Anasetti C, et al. Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia. Blood. 1994;84(3):941-949. (Pubitemid 24223895)
24. Luznik L, O'Donnell PV, Symons HJ, et al. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008;14(6):641-650.
25. Socie G, Henry-Amar M, Bacigalupo A, et al. Malignant tumors occurring after treatment of aplastic anemia. N Engl J Med. 1993;329(16):1152-1157.
26. Maciejewski JP, Risitano A, Sloand EM, Nunez O, Young NS. Distinct clinical outcomes for cytogenetic abnormalities evolving from aplastic anemia. Blood. 2002;99(9):3129-3135. (Pubitemid 34525289)
27. Bacigalupo A, Bruno B, Saracco P, et al. Antilymphocyte globulin, cyclosporine, prednisolone, and granulocyte colony-stimulating factor for severe aplastic anemia: an update of the GITMO/EBMT study on 100 patients: European Group for Blood and Marrow Transplantation (EBMT) Working Party on Severe Aplastic Anemia and the Gruppo Italiano Trapianti di Midolio Osseo (GITMO). Blood. 2000;95(6):1931-1934.
28. Kojima S, Horibe K, Inaba J, et al. Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation. Br J Haematol. 2000;111(1):321-328.
29. de Planque MM, Bacigalupo A, Wursch A, et al. Long-term follow-up of severe aplastic anaemia patients treated with antithymocyte globulin. Br J Haematol. 1989;73(1):121-126.
30. Deeg HJ, Seidel K, Casper J, et al. Marrow transplantation from unrelated donors for patients with severe aplastic anemia who have failed immunosuppressive therapy. Biol Blood Marrow Transplant. 1999;5(4):243-252.
31. Di Bona E, Rodeghiero F, Bruno B, et al. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy: Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol. 1999; 107(2):330-334.
32. Tichelli A, Passweg J, Nissen C, et al. Repeated treatment with horse antilymphocyte globulin for severe aplastic anaemia. Br J Haematol. 1998; 100(2):393-400. (Pubitemid 28131078)
33. Bacigalupo A, Bruno B, Saracco P, et al. Antilymphocyte globulin, cyclosporine, prednisolone, and granulocyte colony-stimulating factor for severe aplastic anemia: an update of the GITMO/EBMT study on 100 patients. Blood. 2000;95(6):1931-1934.
34. Frickhofen N, Kaltwasser JP, Schrezenmeier H, et al. Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine. N Engl J Med. 1991; 324(19):1297-1304.