Relapse of aplastic anemia in children after immunosuppressive therapy: A report from the Japan childhood aplastic anemia study group

Haematologica

Volumn 96, Issue 6, 2011, Pages 814-819

  Kamio, Takuya ^a   Ito, Etsuro ^a   Ohara, Akira ^b   Kosaka, Yoshiyuki ^c   Tsuchida, Masahiro ^d   Yagasaki, Hiroshi ^e   Mugishima, Hideo ^e   Yabe, Hiromasa ^f   Morimoto, Akira ^g   Ohga, Shouichi ^h   Muramatsu, Hideki ⁱ   Hama, Asahito ⁱ   Kaneko, Takashi ^j   Nagasawa, Masayuki ^k   Kikuta, Atsushi ^l   Osugi, Yuko ^m   Bessho, Fumio ⁿ   Nakahata, Tatsutoshi ^o   Tsukimoto, Ichiro ^b   Kojima, Seiji ⁱ  

^a HIROSAKI UNIVERSITY   (Japan)

^b TOHO UNIVERSITY   (Japan)

^c Hyogo Children's Hospital   (Japan)

^d Ibaraki Children's Hospital   (Japan)

^e NIHON UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^f TOKAI UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^g KYOTO PREFECTURAL UNIVERSITY OF MEDICINE   (Japan)

^h KYUSHU UNIVERSITY   (Japan)

ⁱ NAGOYA UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^j TOKYO METROPOLITAN CHILDREN'S MEDICAL CENTER   (Japan)

^k TOKYO MEDICAL AND DENTAL UNIVERSITY   (Japan)

^l FUKUSHIMA MEDICAL UNIVERSITY   (Japan)

^m OSAKA CITY GENERAL HOSPITAL   (Japan)

ⁿ KYORIN UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^o KYOTO UNIVERSITY   (Japan)

more..

Author keywords

Aplastic anemia; Children; Immunosuppressive therapy; Relapse; Risk factors

Indexed keywords

CYCLOSPORIN; DANAZOL; IMMUNOSUPPRESSIVE AGENT; LYMPHOCYTE ANTIBODY; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; THYMOCYTE ANTIBODY;

ADOLESCENT; ADULT; APLASTIC ANEMIA; ARTICLE; CHILD; DISEASE SEVERITY; DRUG EFFICACY; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INFANT; JAPAN; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; NEWBORN; OVERALL SURVIVAL; PRESCHOOL CHILD; PROGNOSIS; PROSPECTIVE STUDY; RECURRENCE RISK; RELAPSE; RISK FACTOR; SCHOOL CHILD; SURVIVAL RATE; TREATMENT RESPONSE; TREND STUDY;

ADOLESCENT; ANEMIA, APLASTIC; ANTILYMPHOCYTE SERUM; CHILD; CHILD, PRESCHOOL; CYCLOSPORINE; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IMMUNOSUPPRESSION; IMMUNOSUPPRESSIVE AGENTS; INCIDENCE; INFANT; INFANT, NEWBORN; JAPAN; MALE; PROSPECTIVE STUDIES; RECURRENCE; RISK FACTORS;

EID: 79958021197     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2010.035600     Document Type: Article

References (27)

1. Storb R. Bone marrow transplantation for aplastic anemia. Cell Transplant. 1993;2(5): 365-379.
2. Horowitz MM. Current status of allogeneic bone marrow transplantation in acquired aplastic anemia. Semin Hematol. 2000;37(1): 30-42.
3. Ades L, Mary JY, Robin M, Ferry C, Porcher R, Esperou H, et al. Long-term outcome after bone marrow transplantation for severe aplastic anemia. Blood. 2004;103(7):2490-2497.
4. Davies JK, Guinan EC. An update on the management of severe idiopathic aplastic anaemia in children. Br J Haematol. 2007; 136(4):549-564.
5. Kojima S, Hibi S, Kosaka Y, Yamamoto M, Tsuchida M, Mugishima H, et al. Immuno -suppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood. 2000;96(6):2049-2054.
6. Bacigalupo A, Bruno B, Saracco P, Di Bona E, Locasciulli A, Locatelli F, et al. Anti -lymphocyte 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 Midollo Osseo (GITMO). Blood. 2000;95(6):1931-1934.
7. Führer M, Rampf U, Baumann I, Faldum A, Niemeyer C, Janka-Schaub G, et al. Immu -no suppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. Blood. 2005;106(6): 2102-2104.
8. Kojima S, Ohara A, Tsuchida M, Kudoh T, Hanada R, Okimoto Y, et al. Risk factors for evolution of acquired aplastic anemia into myelodysplastic syndrome and acute myeloid leukemia after immunosuppressive therapy in children. Blood. 2002;100(3):786-790.
9. Schrezenmeier H, Marin P, Raghavachar A, McCann S, Hows J, Gluckman E, et al. Relapse of aplastic anaemia after immunosuppressive treatment: a report from the European Bone Marrow Transplantation Group SAA Working Party. Br J Haematol. 1993;85(2):371-377.
10. Saracco P, Quarello P, Iori AP, Zecca M, Longoni D, Svahn J, et al. Cyclosporin A response and dependence in children with acquired aplastic anaemia: a multicentre retrospective study with long-term observation follow-up. Br J Haematol. 2008;140(2):197-205.
11. Scheinberg P, Wu CO, Nunez O, Young NS. Long-term outcome of pediatric patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine. J Pediatr. 2008;153(6):814-819.
12. Kosaka Y, Yagasaki H, Sano K, Kobayashi R, Ayukawa H, Kaneko T, et al. Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia. Blood. 2008;111(3):1054-1059.
13. Camitta BM, Thomas ED, Nathan DG, Gale RP, Kopecky KJ, Rappeport JM, et al. A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia. Blood. 1979;53(3): 504-514.
14. Bacigalupo A, Chaple M, Hows J, Van Lint MT, McCann S, Milligan D, et al. Treatment of aplastic anaemia (AA) with antilymphocyte globulin (ALG) and methylprednisolone (MPred) with or without androgens: a randomized trial from the EBMT SAA working party. Br J Haematol. 1993; 83(1):145-151.
15. Fine JP, Gray RJ. A proportional hazards model for the subdistribution of a competing risk. J Am Stat Assoc. 1999;94(446): 496-509.
16. Schrezenmeier H, Marin P, Raghavachar A, McCann S, Hows J, Gluckman E, et al. Relapse of aplastic anaemia after immunosuppressive treatment: a report from the European Bone Marrow Transplantation Group SAA Working Party. Br J Haematol. 1993;85(2):371-377.
17. 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.
18. Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclo -sporine for severe aplastic anemia; association between hematologic response and long-term outcome. JAMA. 2003;289 (3): 1130-1135.
19. Vulliamy T, Marrone A, Dokal I, Mason PJ. Association between aplastic anaemia and mutations in telomerase RNA. Lancet. 2002;359(9324):2168-2170.
20. Yamaguchi H, Baerlocher GM, Lansdorp PM, Chanock SJ, Nunez O, Sloand E, et al. Mutations of the human telomerase RNA gene (TERC) in aplastic anemia and myelodysplastic syndrome. Blood. 2003;102 (3):916-918.
21. Yamaguchi H, Calado RT, Ly H, Kajigaya S, Baerlocher GM, Chanock SJ, et al. Mutations in TERT, the gene for telomerase reverse transcriptase, in aplastic anemia. N Engl J Med. 2005;352(14):1413-1424.
22. Fogarty PF, Yamaguchi H, Wiestner A, Baerlocher GM, Sloand E, Zeng WS, et al. Late presentation of dyskeratosis congenital as apparently acquired aplastic anaemia due to mutations in telomerase RNA. Lancet. 2003;362(9396):1628-1630.
23. Liang J, Yagasaki H, Kamachi Y, Hama A, Matsumoto K, Kato K, et al. Mutations in telomerase catalytic protein in Japanese children with aplastic anemia. Haematologica. 2006;91(5):656-658.
24. Howard SC, Naidu PE, Hu XJ, Jeng MR, Rodriguez-Galindo C, Rieman MD, et al. Natural history of moderate aplastic anemia in children. Pediatr Blood Cancer. 2004; 43(5):545-551.
25. Teramura M, Kimura A, Iwase S, Yonemura Y, Nakao S, Urabe A, et al. Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan. Blood. 2007;110(6):1756-1761.
26. Gurion R, Gafter-Gvili A, Paul M, Vidal L, Ben-Bassat I, Yeshurun M, et al. Hemato -poietic growth factors in aplastic anemia patients treated with immunosuppressive therapy-systematic review and metaanalysis. Haematologica. 2009;94(5): 712-719.
27. Scheinberg P, Nunez O, Young NS. Retreatment with rabbit anti-thymocyte globulin and cyclosporin for patients with relapsed or refractory severe aplastic anaemia. Br J Haematol. 2006;133(6):622-627.