Clinical characteristics and treatment outcome in 65 cases with refractory cytopenia of childhood defined according to the WHO 2008 classification

British Journal of Haematology

Volumn 166, Issue 5, 2014, Pages 758-766

  Hasegawa, Daisuke ^a   Chen, Xiaojuan ^a,b   Hirabayashi, Shinsuke ^a,c   Ishida, Yasushi ^a   Watanabe, Shizuka ^a   Zaike, Yuji ^d   Tsuchida, Masahiro ^e   Masunaga, Atsuko ^f   Yoshimi, Ayami ^c   Hama, Asahito ^g   Kojima, Seiji ^g   Ito, Masafumi ^h   Nakahata, Tatsutoshi ⁱ   Manabe, Atsushi ^a  

^a ST LUKE'S INTERNATIONAL HOSPITAL   (Japan)

^b INSTITUTE OF HEMATOLOGY AND BLOOD DISEASES HOSPITAL   (China)

^c UNIVERSITY OF FREIBURG   (Germany)

^d UNIVERSITY OF TOKYO   (Japan)

^e Ibaraki Children's Hospital   (Japan)

^f SHOWA UNIVERSITY FUJIGAOKA HOSPITAL   (Japan)

^g NAGOYA UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^h JAPANESE RED CROSS NAGOYA FIRST HOSPITAL   (Japan)

ⁱ KYOTO UNIVERSITY   (Japan)

Author keywords

Disease progression; Haematopoietic stem cell transplantation; Immunosuppressive therapy; Myelodysplastic syndrome; Refractory cytopenia of childhood

Indexed keywords

CYCLOSPORIN; IMMUNOSUPPRESSIVE AGENT; LYMPHOCYTE ANTIBODY;

ADOLESCENT; CHILD; DISEASE COURSE; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; INFANT; MALE; MYELODYSPLASTIC SYNDROMES; NEWBORN; PANCYTOPENIA; PRESCHOOL CHILD; PROSPECTIVE STUDY; TREATMENT OUTCOME; WORLD HEALTH ORGANIZATION;

ADOLESCENT; ANTILYMPHOCYTE SERUM; CHILD; CHILD, PRESCHOOL; CYCLOSPORINE; DISEASE PROGRESSION; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFANT; INFANT, NEWBORN; MALE; MYELODYSPLASTIC SYNDROMES; PANCYTOPENIA; PROSPECTIVE STUDIES; TREATMENT OUTCOME; WORLD HEALTH ORGANIZATION;

EID: 84906051963     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.12955     Document Type: Article

References (35)

1. Baumann, I., Niemeyer, C.M. & Benett, J. (2008) Childhood myelodysplastic syndrome. In: WHO Classification of Tumours of Haematopoietic and Lymphoid tissues (eds by Swerdlow, S., Campo, E., Harris, N., Jaffe, E.S., Pileri, S.A., Stein, H., Thiele, J. & Vardiman, J.W.), pp. 104-107. IARC Press, Lyon.
2. Baumann, I., Führer, M., Behrendt, S., Campr, V., Csomor, J., Furlan, I., de Haas, V., Kerndrup, G., Leguit, R.J., De Paepe, P., Noellke, P., Niemeyer, C. & Schwarz, S. (2012) Morphological differentiation of severe aplastic anaemia from hypocellular refractory cytopenia of childhood: reproducibility of histopathological diagnostic criteria. Histopathology, 61, 10-17.
3. Calado, R.T. (2011) Immunologic aspects of hypoplastic myelodysplastic syndrome. Seminars in Oncology, 38, 667-672.
4. Cantù Rajnoldi, A., Fenu, S., Kerndrup, G., van Wering, E.R., Niemeyer, C.M. & Baumann, I. (2005) Evaluation of dysplastic features in myelodysplastic syndromes: experience from the morphology group of the European Working Group of MDS in Childhood (EWOG-MDS). Annals of Hematology, 84, 429-433.
5. Cutler, C.S., Lee, S.J., Greenberg, P., Deeg, H.J., Pérez, W.S., Anasetti, C., Bolwell, B.J., Cairo, M.S., Gale, R.P., Klein, J.P., Lazarus, H.M., Liesveld, J.L., McCarthy, P.L., Milone, G.A., Rizzo, J.D., Schultz, K.R., Trigg, M.E., Keating, A., Weisdorf, D.J., Antin, J.H. & Horowitz, M.M. (2004) A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood, 104, 579-585.
6. Filipovich, A.H., Weisdorf, D., Pavletic, S., Socie, G., Wingard, J.R., Lee, S.J., Martin, P., Chien, J., Przepiorka, D., Couriel, D., Cowen, E.W., Dinndorf, P., Farrell, A., Hartzman, R., Henslee-Downey, J., Jacobsohn, D., McDonald, G., Mittleman, B., Rizzo, J.D., Robinson, M., Schubert, M., Schultz, K., Shulman, H., Turner, M., Vogelsang, G. & Flowers, M.E. (2005) National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biology of Blood and Marrow Transplantation, 11, 945-956.
7. Germing, U., Strupp, C., Kuendgen, A., Isa, S., Knipp, S., Hildebrandt, B., Giagounidis, A., Aul, C., Gattermann, N. & Haas, R. (2006) Prospective validation of the WHO proposals for the classification of myelodysplastic syndromes. Haematologica, 91, 1596-1604.
8. Haase, D., Germing, U., Schanz, J., Pfeilstöcker, M., Nösslinger, T., Hildebrandt, B., Kundgen, A., Lübbert, M., Kunzmann, R., Giagounidis, A.A., Aul, C., Trümper, L., Krieger, O., Stauder, R., Müller, T.H., Wimazal, F., Valent, P., Fonatsch, C. & Steidl, C. (2007) New insights into the prognostic impact of the karyotype in MDS and correlation with subtypes: evidence from a core dataset of 2124 patients. Blood, 110, 4385-4395.
9. Hasegawa, D., Manabe, A., Yagasaki, H., Ohtsuka, Y., Inoue, M., Kikuchi, A., Ohara, A., Tsuchida, M., Kojima, S. & Nakahata, T. (2009) Treatment of children with refractory anemia: the Japanese Childhood MDS Study Group Trial (MDS99). Pediatric Blood & Cancer, 53, 1011-1015.
10. Hasle, H., Niemeyer, C.M., Chessells, J.M., Baumann, I., Bennett, J.M., Kerndrup, G. & Head, D.R. (2003) A pediatric approach to the WHO classification of myelodysplastic and myeloproliferative diseases. Leukemia, 17, 277-282.
11. Hasle, H., Baumann, I., Bergstrasser, E., Fenu, S., Fischer, A., Kardos, G., Kerndrup, G., Locatelli, F., Rogge, T., Schultz, K.R., Stary, J., Trebo, M., van den Heuvel-Eibrink, M.M., Harbott, J., Nollke, P. & Niemeyer, C.M. (2004) The International Prognostic Scoring System (IPSS) for childhood myelodysplastic syndrome (MDS) and juvenile myelomonocytic leukemia (JMML). Leukemia, 18, 2008-2014.
12. Howard, S.C., Naidu, P.E., Hu, X.J., Jeng, M.R., Rodriguez-Galindo, C., Rieman, M.D. & Wang, W.C. (2004) Natural history of moderate aplastic anemia in children. Pediatric Blood & Cancer, 43, 545-551.
13. Jonásova, A., Neuwirtová, R., Cermák, J., Vozobulová, V., Mociková, K., Sisková, M. & Hochová, I. (1998) Cyclosporin A therapy in hypoplastic MDS patients and certain refractory anaemias without hypoplastic bone marrow. British Journal of Haematology, 100, 304-309.
14. Kardos, G., Baumann, I., Passmore, S.J., Locatelli, F., Hasle, H., Schultz, K.R., Stary, J., Schmitt-Graeff, A., Fischer, A., Harbott, J., Chessells, J.M., Hann, I., Fenu, S., Rajnoldi, A.C., Kerndrup, G., Van Wering, E., Rogge, T., Nollke, P. & Niemeyer, C.M. (2003) Refractory anemia in childhood: a retrospective analysis of 67 patients with particular reference to monosomy 7. Blood, 102, 1997-2003.
15. Kojima, S., Hibi, S., Kosaka, Y., Yamamoto, M., Tsuchida, M., Mugishima, H., Sugita, K., Yabe, H., Ohara, A. & Tsukimoto, I. (2000) Immunosuppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood, 96, 2049-2054.
16. Luger, S.M., Ringdén, O., Zhang, M.J., Pérez, W.S., Bishop, M.R., Bornhauser, M., Bredeson, C.N., Cairo, M.S., Copelan, E.A., Gale, R.P., Giralt, S.A., Gulbas, Z., Gupta, V., Hale, G.A., Lazarus, H.M., Lewis, V.A., Lill, M.C., McCarthy, P.L., Weisdorf, D.J. & Pulsipher, M.A. (2012) Similar outcomes using myeloablative vs reduced-intensity allogeneic transplant preparative regimens for AML or MDS. Bone Marrow Transplantation, 47, 203-211.
17. Malcovati, L., Germing, U., Kuendgen, A., Della Porta, M.G., Pascutto, C., Invernizzi, R., Giagounidis, A., Hildebrandt, B., Bernasconi, P., Knipp, S., Strupp, C., Lazzarino, M., Aul, C. & Cazzola, M. (2007) Time-dependent prognostic scoring system for predicting survival and leukemic evolution in myelodysplastic syndromes. Journal of Clinical Oncology, 25, 3503-3510.
18. Matsuda, A., Germing, U., Jinnai, I., Iwanaga, M., Misumi, M., Kuendgen, A., Strupp, C., Miyazaki, Y., Tsushima, H., Sakai, M., Bessho, M., Gattermann, N., Aul, C. & Tomonaga, M. (2007) Improvement of criteria for refractory cytopenia with multilineage dysplasia according to the WHO classification based on prognostic significance of morphological features in patients with refractory anemia according to the FAB classification. Leukemia, 21, 678-686.
19. Mehta, P.A., Harris, R.E., Davies, S.M., Kim, M.O., Mueller, R., Lampkin, B., Mo, J., Myers, K. & Smolarek, T.A. (2010) Numerical chromosomal changes and risk of development of myelodysplastic syndrome-acute myeloid leukemia in patients with Fanconi anemia. Cancer Genetics and Cytogenetics, 203, 180-186.
20. Molldrem, J.J., Caples, M., Mavroudis, D., Plante, M., Young, N.S. & Barrett, A.J. (1997) Antithymocyte globulin for patients with myelodysplastic syndrome. British Journal of Haematology, 99, 699-705.
21. Molldrem, J.J., Leifer, E., Bahceci, E., Saunthararajah, Y., Rivera, M., Dunbar, C., Liu, J., Nakamura, R., Young, N.S. & Barrett, A.J. (2002) Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Annals of Internal Medicine, 137, 156-163.
22. Niemeyer, C.M. & Kratz, C.P. (2008) Paediatric myelodysplastic syndromes and juvenile myelomonocytic leukaemia: molecular classification and treatment options. British Journal of Haematology, 140, 610-624.
23. Nishio, N., Yagasaki, H., Takahashi, Y., Muramatsu, H., Hama, A., Yoshida, N., Kudo, K. & Kojima, S. (2009) Natural history of transfusion-independent non-severe aplastic anemia in children. International Journal of Hematology, 89, 409-413.
24. Passmore, S.J., Chessells, J.M., Kempski, H., Hann, I.M., Brownbill, P.A. & Stiller, C.A. (2003) Paediatric myelodysplastic syndromes and juvenile myelomonocytic leukaemia in the UK: a population-based study of incidence and survival. British Journal of Haematology, 121, 758-767.
25. Przepiorka, D., Weisdorf, D., Martin, P., Klingemann, H.G., Beatty, P., Hows, J. & Thomas, E.D. (1995) 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplantation, 15, 825-828.
26. Sasaki, H., Manabe, A., Kojima, S., Tsuchida, M., Hayashi, Y., Ikuta, K., Okamura, I., Koike, K., Ohara, A., Ishii, E., Komada, Y., Hibi, S. & Nakahata, T. (2001) Myelodysplastic syndrome in childhood: a retrospective study of 189 patients in Japan. Leukemia, 15, 1713-1720.
27. Shimamoto, T., Tohyama, K., Okamoto, T., Uchiyama, T., Mori, H., Tomonaga, M., Asano, Y., Niho, Y., Teramura, M., Mizoguchi, H., Omine, M. & Ohyashiki, K. (2003) Cyclosporin A therapy for patients with myelodysplastic syndrome: multicenter pilot studies in Japan. Leukemia Research, 27, 783-788.
28. Sloand, E.M. (2009) Hypocellular myelodysplasia. Hematology/Oncology Clinics of North America, 23, 347-360.
29. Sloand, E.M. & Rezvani, K. (2008) The role of the immune system in myelodysplasia: implications for therapy. Seminars in Hematology, 45, 39-48.
30. Sloand, E.M., Wu, C.O., Greenberg, P., Young, N. & Barrett, J. (2008) Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. Journal of Clinical Oncology, 26, 2505-2511.
31. Strahm, B., Locatelli, F., Bader, P., Ehlert, K., Kremens, B., Zintl, F., Fuhrer, M., Stachel, D., Sykora, K.W., Sedlacek, P., Baumann, I. & Niemeyer, C.M. (2007) Reduced intensity conditioning in unrelated donor translantation for refractory cytopenia in childhood. Bone Marrow Transplantation, 40, 329-333.
32. Vardiman, J.W., Harris, N.L. & Brunning, R.D. (2002) The World Health Organization (WHO) classification of the myeloid neoplasms. Blood, 100, 2292-2302.
33. Verburgh, E., Achten, R., Louw, V.J., Brusselmans, C., Delforge, M., Boogaerts, M., Hagemeijer, A., Vandenberghe, P. & Verhoef, G. (2007) A new disease categorization of low-grade myelodysplastic syndromes based on the expression of cytopenia and dysplasia in one versus more than one lineage improves on the WHO classification. Leukemia, 21, 668-677.
34. de Vries, A.C., Langerak, A.W., Verhaaf, B., Niemeyer, C.M., Stary, J., Schmiegelow, K., van Wering, E.R., Zwaan, C.M., Beishuizen, A., Pieters, R. & van den Heuvel-Eibrink, M.M. (2008) T-cell receptor Vbeta CDR3 oligoclonality frequently occurs in childhood refractory cytopenia (MDS-RC) and severe aplastic anemia. Leukemia, 22, 1170-1174.
35. Yoshimi, A., Baumann, I., Führer, M., Bergsträsser, E., Göbel, U., Sykora, K.W., Klingebiel, T., Gross-Wieltsch, U., van den Heuvel-Eibrink, M.M., Fischer, A., Nöllke, P. & Niemeyer, C. (2007) Immunosuppressive therapy with anti-thymocyte globulin and cyclosporine A in selected children with hypoplastic refractory cytopenia. Haematologica, 92, 397-400.