Persistent Questions about the Treatment of Severe Aplastic Anemia in Children as Illustrated by Five Cases

References

• Young, N.S. (1995) "Aplastic anaemia", Lancet 346, 228–232.
   PubMed Web of Science ®Google Scholar
• Young, N.S. (1999) "Acquired aplastic anemia", JAMA 282(3), 271–278.
   PubMed Web of Science ®Google Scholar
• McGlave, P.B., Haake, R., Miller, W., Kim, T., Kersey, J. and Ramsay, N.K.C. (1987) "Therapy of severe aplastic anemia in young adults and children with allogeneic bone marrow transplantation", Blood 70(5), 1325–1330.
   PubMed Web of Science ®Google Scholar
• Gluckman, E., Socie, G., Devergie, A., Bourdeau-Esperou, H., Traineau, R. and Cosset, J.M. (1991) "Bone marrow transplantation in 107 patients with severe aplastic anemia using cyclophosphamide and thoraco-abdominal irradiation for conditioning: long-term follow-up", Blood 78(9), 2451–2455.
   PubMed Web of Science ®Google Scholar
• Kojima, S., Horibe, K., Inaba, J., Yoshima, A., Takahashi, Y., Kudo, K., Kato, K. and Matsuyama, T. (2000) "Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation", Br. J. Haematol. 111(1), 321–328.
   PubMed Web of Science ®Google Scholar
• Kojima, S., Hibi, S., Kosaka, Y., Yamamoto, M., Tsuchida, M., Mugishima, H., Sugita, K., Yabe, H., Ohara, A. and Tsukimoto, I. (2000) "Immunosuppressive therapy using anti-thymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony stimulating factor in children with acquired aplastic anemia", Blood 96(6), 2049–2054.
   PubMed Web of Science ®Google Scholar
• Fruchtman, S., Isola, L., Hurlet, A., Dracker, R., Shanbavi, R., Singh, H., Goldman, B. and Emre, S. (2001) "Autologous cord blood infusion for severe aplastic anemia (SAA)", Blood 98(1), 687a.
   Google Scholar
• Trigg, M.E., Morgan, D., Burns, T., Kook, H., Rumelhart, S., Holida, M.D. and Giller, R. (1996) "Successful program to prevent aspergillus infections in children undergoing marrow transplantation: Use of nasal amphotericin", Bone Marrow Transplant. 19, 43–47.
   Web of Science ®Google Scholar
• Hendrickson, K.J., Axtell, R.A., Hoover, S.M., Kuhn, S.M., Pritchett, J., Kehl, S.C. and Klein, J.P. (2000) "Prevention of central venous catheter-related infections and thrombotic events in immunocompromised children by the use of vancomycin/ciprofloxacin/heparin flush solution: a ran-domized, multicenter, double-blind trial", J. Clin. Oncol., 1269–1278.
   PubMed Web of Science ®Google Scholar
• Bond, R., Walter, A. and Trigg, M.E. (2002) "Severe aplastic anemia associated with hepatitis and complicated by pulmonary aspergillosis: response to immunosuppression and antifungal therapy", Del. Med. J. 74(8), 339–343.
   PubMedGoogle Scholar
• Maciejewski, J.P., Anderson, S., Katevas, P. and Young, N.S. (1994) "Phenotypic and functional analysis of bone marrow progenitor cell compartment in bone marrow failure", Br. J. Haematol. 87, 227–234.
   PubMed Web of Science ®Google Scholar
• Maciejewski, J.P., Risitano, A., Kook, H., Zeng, W., Chen, G. and Young, N.S. (2002) "Immune pathophysiology of aplastic anemia", Int. J. Hematol., 207–214.
   PubMed Web of Science ®Google Scholar
• Storb, R. (1997) "Aplastic anemia", J. IV Nurs. 20(6), 317–322.
   Google Scholar
• Rosenfeld, S., Follmann, D., Nunez, O. and Young, N.S. (2003) "Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome", JAMA 289(9), 1130–1135.
   PubMed Web of Science ®Google Scholar
• Kojima, S., Nakao, S., Tomonaga, M., Hows, J., Marsh, J., Gerard, S., Bacigalupo, A. and Mizoguchi, H. (2000) "Consensus conference on the treatment of aplastic anemia", Int. J. Hematol. 72, 118–123.
   PubMed Web of Science ®Google Scholar
• Matloub, Y.H., Smith, C., Bostrom, B., Koerper, M.A., O'Leary, M.O., Khuder, S., Smithson, W.A., Nickerson, H.J., Silberman, T., Hilden, J., Moertel, CL., Month, S., Mon-teleone, P. and Ramsay, N.K.C. (1997) "One course versus two courses of antithymocyte globulin for the treatment of severe aplastic anemia in children", J. Pediatr. Hematol./Oncol. 19(2), 110–114.
   Web of Science ®Google Scholar
• Di Bona, E., Rodeghiero, E, Bruno, B., Gabbas, T., Foa, P., Locasciulli, A., Rosanelli, C., Camba, L., Saracco, P., Lippi, A., Ion, A.P., Porta, E, De Rossi, G., Comotti, B., Iacopino, P., Dufour, C., Bacigalupo, A. and De Rossi, V. (1999) "Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulo-cyte stimulating factor is an effective treatment for aplastic anemia in patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO)", Br. J. Hematol. 107(2), 330–334.
   PubMed Web of Science ®Google Scholar
• Kojima, S., Matsuyama, T., Kato, S., Kigasawa, H., Kobayashi, R., Kikuta, A., Sakamaki, H., Ikuta, K., Tsuchida, M., Hoshi, Y., Morishima, Y. and Kodera, Y. (2002) "Outcome of 154 patients with severe aplastic anemia who received transplant from unrelated donors: the Japan Marrow Donor Program", Blood 100, 799–803.
   PubMed Web of Science ®Google Scholar
• Frickhofen, N., Heimpel, H., Kaltwasser, J.P. and Schrezen-meier, H. (2003) "Antithymocyte globulin with or without cyclosporine A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia", Blood 101(4), 1236–1242.
   PubMed Web of Science ®Google Scholar
• Deeg, H.J., Leisenring, W., Storb, R., Nims, J., Flowers, MED., Witherspoon, R.P., Sanders, J. and Sullivan, K.M. (1998) "Long-term outcome after marrow transplantation for severe aplastic anemia", Blood 91(10), 3637–3645.
   PubMed Web of Science ®Google Scholar
• Passweg, JR., Socie, G., Hinterberger, W., Bacigalupo, A., Biggs, J.C., Camitta, B.M., Champlin, RE., Gale, R.P., Gluckman, E., Gordon-Smith, E.C, Hows, J.M., Klein, J.P., Nugent, MI., Pasquini, R., Rowlings, P.A., Speck, B., Tichelli, A., Zhang, M-J., Horowitz, MM. and Bortin, UM. (1997) "Bone marrow transplantation for severe aplastic anemia: Has outcome improved?", Blood 90(2), 858–864.
   PubMed Web of Science ®Google Scholar
• Gillio, A.P., Boulad, F., Small, TN., Kernan, N.A., Reyes, B., Childs, B.H., Brochstein, J.A., Laver, J., Castro-Malaspina, H. and O'Reilly, J.F. (1997) "Comparison of long-term outcome of children with severe aplastic anemia treated with immuno-suppression versus bone marrow transplantation", Biol. Blood Marrow Transplant. 3, 18–24.
   PubMedGoogle Scholar
• Gordon-Smith, E.C., Marsh, J.C. and Gibson, F.M. (2002) "Views on the pathophysiology of aplastic anemia", Int. J. Hematol. 76(Supplement II), 163–166.
   PubMedGoogle Scholar
• Bunin, A., Leahey, A., Kamani, N. and August, C. (1996) "Bone marrow transplantation in pediatric patients with severe aplastic anemia: Cyclophosphamide and antithymo-cyte globulin conditioning followed by recombinant human granulocyte-macrophage colony stimulating factor", J. Pediatr. Hematol./Oncol. 18(1), 68–71.
   Web of Science ®Google Scholar
• Yamazaki, E., Kanamori, H. and Taguchi, J. (1997) "The evidence of clonal evolution with monosomy 7 in aplastic anemia following granulocyte colony stimulating factor using the polymerase chain reaction", Blood Cells Mol. Dis. 23, 213–218.
   PubMed Web of Science ®Google Scholar
• Vulliamy, T., Marrone, A., Dokal, I. and Mason, P.J. (2002) "Association between aplastic anemia and mutations in telomerase RNA", Lancet 359(9324), 2168–2170.
   PubMed Web of Science ®Google Scholar
• Maciewjewski, J.P., Risitano, A., Sloand, EM., Nunez, O. and Young, N.S. (2002) "Distinct clinical outcomes for cytogenetic abnormalities evolving from aplastic anemia", Blood 99, 3129–3135.
   PubMed Web of Science ®Google Scholar
• Ohara, A., Kojima, S., Hamajima, N., Tsuchida, M., Imashuku, S., Ohta, S., Sasaki, H., Okamura, J., Sugita, K., Kigasawa, H., Kiriyama, Y., Akatsuka, J. and Tsukimoto, I. (1997) "Myelodysplastic syndrome and acute myelogenous leuke-mia as a late clonal complication in children with acquired aplastic anemia", Blood 90(3), 1009–1013.
   PubMed Web of Science ®Google Scholar
• Henslee-Downey, P.J. and Gluckman, E. (1999) "Allogeneic transplant from donors other than HLA-identical siblings", Hematol./Oncol. Clin. North Am. 13(5), 1017–1039.
   PubMed Web of Science ®Google Scholar
• Goerner, M., Gooley, T., Flowers, MED., Sullivan, KM., Kiem, H-P., Sanders, J.E., Martin, P.J. and Storb, R. (2002) "Morbidity and mortality of chronic GVHD after hemato-poietic stem cell transplantation from HLA-identical siblings for patients with aplastic or refractory anemias", Biol. Blood Marrow Transplant. 8, 47–56.
   PubMed Web of Science ®Google Scholar