Prognostic value of paroxysmal nocturnal haemoglobinuria clone presence in aplastic anaemia patients treated with combined immunosuppression: Results of two-centre prospective study

British Journal of Haematology

Volumn 164, Issue 4, 2014, Pages 546-554

  Kulagin, Alexander ^a   Lisukov, Igor ^a   Ivanova, Maria ^a   Golubovskaya, Irina ^a   Kruchkova, Irina ^b   Bondarenko, Sergey ^a   Vavilov, Vladimir ^a   Stancheva, Natalia ^a   Babenko, Elena ^a   Sipol, Alexandra ^a   Pronkina, Natalia ^b   Kozlov, Vladimir ^b   Afanasyev, Boris ^a  

^a PAVLOV FIRST SAINT PETERSBURG STATE MEDICAL UNIVERSITY   (Russian Federation)

^b INSTITUTE OF CLINICAL IMMUNOLOGY   (Russian Federation)

Author keywords

Aplastic anaemia; Bone marrow failure; Flow cytometry; Immunosuppressive therapy; Paroxysmal nocturnal haemoglobinuria

Indexed keywords

CYCLOSPORIN A; IMMUNOSUPPRESSIVE AGENT; PREDNISONE; THYMOCYTE ANTIBODY;

ADULT; AGED; APLASTIC ANEMIA; ARTICLE; CANCER SURVIVAL; CHILD; CLINICAL EVALUATION; DISEASE SEVERITY; DRUG DOSE REDUCTION; EOSINOPHIL COUNT; FAILURE FREE SURVIVAL; FEMALE; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; LYMPHOCYTE COUNT; MAJOR CLINICAL STUDY; MALE; MIDDLE AGED; MULTICENTER STUDY; OUTCOME ASSESSMENT; OVERALL SURVIVAL; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PRESCHOOL CHILD; PRIORITY JOURNAL; PROGNOSIS; PROSPECTIVE STUDY; RETICULOCYTE COUNT; TREATMENT RESPONSE; VERY ELDERLY;

APLASTIC ANAEMIA; BONE MARROW FAILURE; FLOW CYTOMETRY; IMMUNOSUPPRESSIVE THERAPY; PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA;

ADOLESCENT; ADULT; AGED; ANEMIA, APLASTIC; ANTILYMPHOCYTE SERUM; CHILD; CHILD, PRESCHOOL; CYCLOSPORINE; FEMALE; FLOW CYTOMETRY; HEMOGLOBINURIA, PAROXYSMAL; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFANT; MALE; MIDDLE AGED; PROGNOSIS; PROSPECTIVE STUDIES; SURVIVAL ANALYSIS; YOUNG ADULT;

EID: 84893024474     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.12661     Document Type: Article

References (38)

1. Afable, M.G. II, Shaik, M., Sugimoto, Y., Elson, P., Clemente, M., Makishima, H., Sekeres, M.A., Lichtin, A., Advani, A., Kalaycio, M., Tiu, R.V., O'Keefe, C.L. & Maciejewski, J.P. (2011) Efficacy of rabbit anti-thymocyte globulin in severe aplastic anemia. Haematologica, 96, 1269-1275.
2. Bacigalupo, A., Hows, J.M., Gluckman, E., Nissen, C., Marsh, J., Van Lint, M.T., Congiu, M., De Planque, M.M., Ernst, P. & McCann, S. (1988) Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT SAA Working Party. British Journal of Haematology, 70, 177-182.
3. Bacigalupo, A., Socie, G., Lanino, E., Prete, A., Locatelli, F., Locasciulli, A., Cesaro, S., Shimoni, A., Marsh, J., Brune, M., Van Lint, M.T., Oneto, R. & Passweg, J.; Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. (2010) Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA working party. Haematologica, 95, 976-982.
4. Borowitz, M.J., Craig, F.E., DiGiuseppe, J.A., Illingworth, A.J., Rosse, W., Sutherland, D.R., Wittwer, C.T. & Richards, S.J.; On behalf of the Clinical Cytometry Society. (2010) Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Cytometry Part B (Clinical Cytometry), 78, 211-230.
5. Camitta, B.M. (2000) What is the definition of cure for aplastic anemia? Acta Haematologica, 103, 16-18.
6. Camitta, B.M., Rappeport, J.M., Parkman, R. & Nathan, D.G. (1975) Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood, 45, 355-363.
7. Chang, M.H., Kim, K.H., Kim, H.S., Jun, H.J., Kim, D.H., Jang, J.H., Kim, K. & Jung, C.W. (2010) Predictors of response to immunosuppressive therapy with antithymocyte globulin and cyclosporine and prognostic factors for survival in patients with severe aplastic anemia. European Journal of Haematology, 84, 154-159.
8. Dunn, D.E., Tanawattanacharoen, P., Boccuni, P., Nagakura, S., Green, S.W., Kirby, M.R., Kumar, M.S., Rosenfeld, S. & Young, N.S. (1999) Paroxysmal nocturnal hemoglobinuria cells in patients with bone marrow failure syndromes. Annals of Internal Medicine, 131, 401-408.
9. Fine, J.P. & Gray, R.J. (1999) A proportional hazards model for the subdistribution of a competing risk. Journal of the American Statistical Association, 94, 496-509.
10. Frickhofen, N., Heimpel, H., Kaltwasser, J.P. & Schrezenmeier, H. (2003) Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood, 101, 1236-1242.
11. Griscelli-Bennaceur, A., Gluckman, E., Scrobohaci, M.L., Jonveaux, P., Vu, T., Bazarbachi, A., Carosella, E.D., Sigaux, F. & Socie, G. (1995) Aplastic anemia and paroxysmal nocturnal hemoglobinuria: search for a pathogenetic link. Blood, 85, 1354-1363.
12. International Agranulocytosis and Aplastic Anaemia Study. (1987) Incidence of aplastic anaemia: relevance of diagnosis criteria. Blood, 70, 1718-1721.
13. Kulagin, A., Golubovskaya, I., Kruchkova, I., Bondarenko, S., Stancheva, N., Lisukov, I., Kozlov, V. & Afanasyev, B. (2011) Long-term outcomes after immunosuppressive treatment for moderate aplastic anemia. Haematologica, 96, 351.
14. Kulagin, A., Ivanova, M., Golubovskaya, I., Babenko, E., Kruchkova, I., Pronkina, N., Bondarenko, S., Vavilov, V., Stancheva, N., Lisukov, I., Kozlov, V. & Afanasyev, B. (2013) The horse ATG (ATGAM) total dose of 160 mg/kg as compared with 100 mg/kg is superior in achieving haematologic response in aplastic anaemia patients. Bone Marrow Transplantation, 48, 43.
15. Locasciulli, A., Oneto, R., Bacigalupo, A., Socié, G., Korthof, E., Bekassy, A., Schrezenmeier, H., Passweg, J. & Führer, M.; Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplant Group. (2007) Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). Haematologica, 92, 11-18.
16. Maciejewski, J., Follmann, D., Nakamura, R., Saunthararajah, Y., Rivera, C., Simonis, T., Brown, K., Barrett, J. & Young, N. (2001a) Increased frequency of HLA-DR2 in patients with paroxysmal nocturnal hemoglobinuria and the PNH/aplastic anemia syndrome. Blood, 98, 3513-3519.
17. Maciejewski, J.P., Rivera, C., Kook, H., Dunn, D. & Young, N.S. (2001b) Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein deficient clones. British Journal of Haematology, 115, 1015-1022.
18. Marsh, J., Schrezenmeier, H., Marin, P., Ilhan, O., Ljungman, P., McCann, S., Socié, G., Tichelli, A., Passweg, J., Hows, J., Raghavachar, A., Locascuilli, A. & Bacigalupo, A. (1999) A prospective randomised multicentre study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with non-severe aplastic anaemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood, 93, 2191-2195.
19. Marsh, J.C., Ball, S.E., Cavenagh, J., Darbyshire, P., Dokal, I., Gordon-Smith, E.C., Keidan, J., Laurie, A., Martin, A., Mercieca, J., Killick, S.B., Stewart, R., Yin, J.A. & Committee, B.; for Standards in Haematology. (2009) Guidelines for the diagnosis and management of aplastic anaemia. British Journal of Haematology, 147, 43-70.
20. Marsh, J.C., Bacigalupo, A., Schrezenmeier, H., Tichelli, A., Risitano, A.M., Passweg, J.R., Killick, S.B., Warren, A.J., Foukaneli, T., Aljurf, M., Al-Zahrani, H.A., Schafhausen, P., Roth, A., Franzke, A., Brummendorf, T.H., Dufour, C., Oneto, R., Sedgwick, P., Barrois, A., Kordasti, S., Elebute, M.O., Mufti, G.J. & Socie, G. (2012) Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. Blood, 119, 5391-5396.
21. Nissen, C., Gratwohl, A., Tichelli, A., Stebler, C., Wursch, A., Moser, Y., Dalle Carbonare, V., Signer, E., Buser, M., Ritz, R. & Speck, B. (1993) Gender and response to antilymphocyte globulin (ALG) for severe aplastic anaemia. British Journal of Haematology, 83, 319-325.
22. Parker, C., Omine, M., Richards, S., Nishimura, J.I., Bessler, M., Ware, R., Hillmen, P., Luzzato, L., Young, N., Kinoshita, T., Rosse, W. & Socié, G.; for the International PNH Interest Group. (2005) Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood, 106, 3699-3709.
23. Passweg, J.R. & Tichelli, A. (2009) Immunosuppressive treatment for aplastic anemia: are we hitting the ceiling? Haematologica, 94, 310-312.
24. Scheinberg, P. & Young, N.S. (2012) How I treat acquired aplastic anemia. Blood, 120, 1185-1196.
25. Scheinberg, P., Wu, C.O., Nunez, O. & Young, N.S. (2009) Predicting response to immunosuppressive therapy and survival in severe aplastic anaemia. British Journal of Haematology, 144, 206-216.
26. Scheinberg, P., Marte, M., Nunez, O. & Young, N.S. (2010) Paroxysmal nocturnal hemoglobinuria clones in severe aplastic anaemia patients treated with horse anti-thymocyte globulin plus cyclosporine. Haematologica, 95, 1075-1080.
27. Scheinberg, P., Nunez, O., Weinstein, B., Scheinberg, P., Biancotto, A., Wu, C.O. & Young, N.S. (2011) Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. The New England Journal of Medicine, 365, 430-438.
28. Sugimori, C., Chuhjo, T., Feng, X., Yamazaki, H., Takami, A., Teramura, M., Mizoguchi, H., Omine, M. & Nakao, S. (2006) Minor populations of CD55-CD59- blood cells predicts response to immunosuppressive therapy and prognosis in patients with aplastic anaemia. Blood, 107, 1308-1314.
29. Sugimori, C., Yamazaki, H., Feng, X., Mochizuki, K., Kondo, Y., Takami, A., Chuhjo, T., Kimura, A., Teramura, M., Mizoguchi, H., Omine, M. & Nakao, S. (2007) Roles of DRB1 *1501 and DRB1 *1502 in the pathogenesis of aplastic anemia. Experimental Hematology, 35, 13-20.
30. Sugimori, C., Mochizuki, K., Qi, Z., Sugimori, N., Ishiyama, K., Kondo, Y., Yamazaki, H., Takami, A., Okumura, H. & Nakao, S. (2009) Origin and fate of blood cells deficient in glycosylphosphatidylinositol-anchored protein among patients with bone marrow failure. British Journal of Haematology, 147, 102-112.
31. Timeus, F., Crescenzio, N., Lorenzati, A., Doria, A., Foglia, L., Pagliano, S., Quarello, P., Ramenghi, U. & Saracco, P. (2010) Paroxysmal nocturnal haemoglobinuria clones in children with acquired aplastic anaemia: a prospective single centre study. British Journal of Haematology, 150, 480-497.
32. Viollier, R., Socié, G., Tichelli, A., Bacigalupo, A., Korthof, E.T., Marsh, J., Cornish, J., Ljungman, P., Oneto, R., Be′ka′ssy, A.N., Fuehrer, M., Maury, S., Schrezenmeier, H., van Lint, M.T., Wojcik, D., Locasciulli, A. & Passweg, J.R. (2008) Recent improvement in outcome of unrelated donor transplantation for aplastic anemia. Bone Marrow Transplantation, 41, 45-50.
33. Wang, H., Chuhjo, T., Yasue, S., Omine, M. & Nakao, S. (2002) Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood, 100, 3897-3902.
34. Yamazaki, H., Saito, C., Sugimori, N., Hosokawa, K., Kiyu, Y., Mochizuki, K., Takamatsu, H. & Nakao, S. (2011) Thymoglobuline is as effective as lymphoglobuline in Japanese patients with aplastic anemia possessing increased glycosylphosphatidylinositol-anchored protein (GPI-AP) deficient cells. Blood (ASH Annual Meeting Abstracts), 118, 1339.
35. Yoshida, N., Yagasaki, H., Takahashi, Y., Yamamoto, T., Liang, J., Wang, Y., Tanaka, M., Hama, A., Nishio, N., Kobayashi, R., Hotta, N., Asami, K., Kikuta, A., Fukushima, T., Hirano, N. & Kojima, S. (2008) Clinical impact of HLA-DR15, a minor population of paroxysmal nocturnal haemoglobinuria-type cells, and an aplastic anaemia-associated autoantibody in children with acquired aplastic anaemia. British Journal of Haematology, 142, 427-435.
36. Yoshida, N., Yagasaki, H., Hama, A., Takahashi, Y., Kosaka, Y., Kobayashi, R., Yabe, H., Kaneko, T., Tsuchida, M., Ohara, A., Nakahata, T. & Kojima, S. (2011) Predicting response to immunosuppressive therapy in childhood aplastic anemia. Haematologica, 96, 771-774.
37. Young, N.S. (2009) Paroxysmal nocturnal hemoglobinuria and myelodysplastic sydromes: clonal expansion of PIG-A-mutant hematopoietic cells in bone marrow failure. Haematologica, 94, 3-7.
38. Young, N.S., Calado, R.T. & Scheinberg, P. (2006) Current concepts in the pathophysiology and treatment of aplastic anemia. Blood, 108, 2509-2519.