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Review

Haematopoietic stem cell transplantation to treat aplastic anaemia

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Pages 617-626 | Published online: 24 Nov 2005

Bibliography

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  • YOUNG NS, MACIEJEWSKI J: The pathophysiology of acquired aplastic anemia. N EngL J. Med. (1997) 336:1365–1372.
  • •Excellent position paper regarding the current understanding of mechanisms of autoimmunity leading to acquired AA.
  • BACIGALUPO A, CHAPLE M, HOWS J 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) 83:145–151.
  • CAMITTA BM, THOMAS ED, NATHAN DG et aL: Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality. Blood (1976) 48:63–70.
  • •Classic paper showing the preponderance that PBSCT would have in treating SAA patients during the following decades.
  • BACIGALUPO A, HOWS J, GLUCKMAN E et al.: Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anemia (SAA): a report of the EBMT SAA working party. Br. J. Haematol. (1988) 70:177–182.
  • FRICKHOFEN N, HEIMPEL H, KALTWASSER JP et aL: Antithymocyte globulin with or without cyclosporine A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood (2003) 101:1236–1242.
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  • 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 (EMBT) Working Party on Severe Aplastic Anemia and the Gruppo Italiano Trapianti de Midolio Osseo (GITMO). Blood (2000) 95:1931–1934.
  • MARSH JC, BALL SE, DARBYSHIRE P et al.: Guidelines for the diagnosis and management of acquired aplastic anaemia. Br. J. Haematol. (2003) 123:782–801.
  • ••Excellent review and guidelines fromEngland on current aspects of the disease.
  • BACIGALUPO A, BRAND R, ONETO R et al.: Treatment of acquired severe aplastic anaemia: bone marrow transplantation compared with immunosuppressive therapy - the European Group for Blood and Marrow transplantation experience. Semin. HematoL (2000) 37:69–80.
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  • ••Excellent perspective of long-term resultsof transplantation in AA.
  • RUIZ-ARGOELLES GJ, GOMEZ-RANGEL D: Long term results of the immunosuppressive treatment of patients with severe, acquired aplastic anemia: a single institution study. Acta Haematol. (2003) 110:184–187.
  • MCSWEENEY PA, NIEDERWIESER D, SHIZURU A et al.: Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versus-tumor effects. Blood (2001) 97:3390–3400.
  • 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 liwnspL (1999) 5:243–252.
  • KOJIMA S, INABA J, YOSHIMI A et al.:Unrelated donor marrow transplantation in children with severe aplastic anaemia using cyclophosphamide, anti-thymocyte globulin and total body irradiation. Br. J. HaematoL (2001) 114:706–711.
  • VASSILIOU GS, WEBB DK, PAMPHILON D et al.: Improved outcome of alternative donor bone marrow transplantation in children with severe aplastic anaemia using a conditioning regimen containing low-dose total body irradiation, cyclophosphamide and Campath. Br. J. HaematoL (2001) 114:701–705.
  • •A provocative study. New conditioning regimen using alternative donors.
  • BARON F, STORB R, LITTLE M: Hematopoietic stem cell transplantation: five decades of progress. Arch. Med. Res. (2003) 34:528–544.
  • HERRERA-GARZA JL, JAIME-PEREZ JC, IBARRA-PEART R, MONTEMAYOR JL, GOMEZ-ALMAGUER D: High-dose peripheral blood stem cell transplant for multitransfused severe aplastic anemia patients without anti-thymocyte globulin in the conditioning regimen. Bone Marrow lianspL (1999) 24:845–848.
  • •One of the first papers demonstrating the feasibility of PBSCT for SAA and the importance of infusing high numbers of haematopoietic cells to circumvent the problem of alloimmunisation to HLA antigens.
  • MIN CK, KIM DW, LEE JW, HAN CW, MIN WS, KIM CC: Hematopoietic stem cell transplantation for high-risk adult patients with severe aplastic anemia; reduction of graft failure by enhancing stem cell dose. Haematologica (2000) 86:303–310.
  • •Stem cell dose importance is highlighted in this very interesting study.
  • HELDAL D, TJONNFJORD G, BRINCH Let al.: A randomized study of allogeneic transplantation with stem cells from blood or bone marrow. Bone Marrow lianspL (2000) 25:1129–1136.
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  • •This article underscores the finding of a higher risk when infusing peripheral stem cells instead of bone marrow-derived progenitors.
  • PAVLETIC SZ, SMITH LM, BISHOP MR et al.: Prognostic factors of chronic graft-versus-host disease after allogeneic blood stem-cell transplantation. Am. J. HematoL (2005) 78:265–274.
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  • ••Depicts the current status of multiplefactors affecting HSCT.
  • WALL DA: Allogeneic peripheral blood versus bone marrow transplantation: children are small adults and more. J. Clin. OncoL (2004) 22:4865–4866.
  • KROGER N, ZANDER AR: Dose and schedule effect of G-GSF for stem cell mobilization in healthy donors for allogeneic transplantation. Leuk. Lymphoma (2002) 43:1391–1394.
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  • ••Excellent study analysing the different outcomes observed in younger patients when PBSC are used instead of bone marrow.
  • 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) 111:321–328.
  • EAPEN M, RAMSAY MK, MERTENS AC, ROBINSON LL, DEFOR T, DAVIES SA: Late outcomes after bone marrow transplant for aplastic anemia. Br. J. HaematoL (2000) 111:754–760.
  • 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) 84:941–949.
  • LOCASCIULLI A, BRUNO B, RAMBALDI A: Treatment of severe aplastic anemia with antilymphocyte globulin, cyclosporine and two different granulocyte colony-stimulating factor regimens: a GITMO prospective randomized study. Haematologica (2004) 89:1054–1061.
  • •Interesting study aimed at defining the contribution of G-CSF to haematopoietic recovery as complement of 1ST for SAA.
  • KARANES C, CONFER D, WALKER T, ASKREN A, KELLER C: Unrelated donor stem cell transplantation: the role of the National Marrow Donor Program. Oncology (Huntingt.) (2003) 17:1036–1038.
  • BACIGALUPO A, ONETO R, BRUNO B et al.: Current status of bone marrow transplantation in patients with acquired severe aplastic anemia. Report of the European Group for Blood and Marrow Transplantation. On behalf of the Working Party on Severe Aplastic Anemia of the European Group for Blood and Marrow Transplantation. Acta HaematoL (2000) 103:19–25.
  • ••Excellent overview of transplant activity inEurope and its perspectives.
  • HANDGRETINER R, KLINGEBIEL T, LANG P, GORDON P, NIETHAMMER D: Megadose transplantation of highly purified haploidentical stem cells: current results and future prospects. Pediatr. Transpl. (2003) 7:51–55.
  • BENESCH M, URBAN C, SYKORA KW et al.: Transplantation of highly purified CD34+ progenitor cells from alternative donors in children with refractory severe aplastic anemia. Br. J. HaematoL (2004) 125:58–63.
  • BUNIN N, APLENC R, IANNONE R et al.: Unrelated donor bone marrow transplantation for children with severe aplastic anemia: minimal GVHD and durable engraftment with partial T cell depletion. Bone Marrow TranspL (2005) 35:369–373.
  • ••Very good report of initial results ofUR-SCT.
  • MUDUR G: Indian researchers accused of violating ethical guidelines. Br. Med. J. (2005) 330:60.
  • COHEN Y, NAGLER A: Hematopoietic stem cell transplantation using umbilical cord blood. Leuk. Lymphoma (2003) 44:1287–1299.
  • YU LC, WALL DA, SANDLER E, CHAN KW, GRAYSON G, KLETZEL M: Unrelated cord blood transplant experience by the pediatric blood and marrow transplant consortium. Pediatr. HematoL OncoL (2001) 18:235–245.
  • MAO P, WANG S, WANG S et al.: Umbilical cord blood transplant for adult patients with severe aplastic anemia using anti-lymphocyte globulin and cyclophosphamide as conditioning regimen. Bone Marrow TranspL (2004) 33:33–38.
  • •This paper was one of the first to show that a reasonable good result can be obtained when transplanting adult patients with SAA employing URJCB-SCT.
  • STORB R, BLUME KG, O'DONELL MR et al.: Cyclophosphamide and antithymocyte globulin to condition patients with aplastic anemia for allogeneic marrow transplantations: the experience in four centers. Biol. Blood Marrow TranspL (2001) 7:39–44.
  • STORB R, PRENTICE RL, THOMAS ED et al.: Marrow transplantation for treatment of aplastic anemia: an analysis of factors associated with graft rejection. N Engl. J. Med. (1977) 296:61–66.
  • LOCATELLI F, BRUNO B, ZECCA M et al.: Cyclosporin A and short term methotrexate versus cyclosporin A as graft versus host disease prophylaxis in patients with severe aplastic anemia given allogeneic bone marrow transplantation from HLA identical siblings: result of a GITMO/ EBMT randomized trial. Blood (2000) 96:1690–1697.
  • GLUCKMAN E, HOROWITZ MM, CHAMPLIN RE et al.: Bone marrow transplantation for severe aplastic anemia: influence of conditioning and graft-versus-host disease regimens on outcome. Blood (1992) 79:269–275.
  • REMBERGER M, BEELEN DW, FAUSER A, BASARA N, BASU O, RINGDEN O: Increased risk of extensive chronic graft-versus-host disease after allogeneic peripheral blood stem cell transplantation using unrelated donors. Blood (2005) 105:548–551.
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  • STORB R, LONGTON G, ANASETTI C et al.: Changing trends in marrow transplantation for aplastic anemia. Bone Marrow TranspL (1992) 10:45–52.
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  • TZENG CH, CHEN PM, FANS et al.: CY/TBI-800 as a pretransplant regimen for allogeneic bone barrow transplantation for severe aplastic anemia using HLA-haploidentical family donors. Bone Marrow lianspL (1996) 18:273–277.
  • DEEG HJ, AMYLON ID, HARRRIS RE et al.: Marrow transplants from unrelated donors for patients with aplastic anemia: minimum effective dose of total body irradiation. Biol. Blood Marrow TranspL (2001) 7:208–215.
  • KOJIMA S, MATSUYAMA T, KATO S et al.: Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program. Blood (2002) 100:799–803.
  • LAUGHLIN MJ, BARKER J, BAMBACH B et al.: Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N Engl J. Med. (2001) 344:1815–1822.
  • BARR RD: The importance of lowering costs of stem cell transplantation in developing countries. Int. J. Hematol (2002) 76\(Suppl. 1):365–367.
  • GOMEZ-ALMAGUER D, VELA-OJEDA J, JAIME-PEREZ JC et al.: Allografting in patients with severe, refractory aplastic anemia using peripheral blood stem cells and a fludarabine-based conditioning regimen: the Mexican experience. Am. J. Hematol (2005) (In Press).
  • CHAMPLIN R, PEREZE WS, PASSWEG J et al.: Addition of antithymocyte globulin (ATG) to cyclophosphamide (Cy) for HLA-identical allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA). Result of a randomized controlled trial (abstract). Blood (2003) 102:269.
  • CHAN KW, LI CK, WORTH LL et al: A fluciarabine-based conditioning regimen for severe aplastic anemia. Bone Marrow Transpl (2001) 27:125–128.
  • GUPTA V BALL SE, SAGE D et al: Marrow transplants from matched unrelated donors for aplastic anemia using alemtuzumab, fludarabine and cyclophosphamide based conditioning. Bone Marrow Transpl (2005) 35:467–471.
  • DULLEY FL, VIGORITO AC, ARANHA FJ et al: Addition of low-dose busulfan to cyclophosphamide in aplastic anemia patients prior to allogeneic bone marrow transplantation to reduce rejection. Bone Marrow Transpl (2004) 33:9–13.
  • DEVETTEN MP, VOSE JM: Graft-versus-host disease: how to translate new insights into new therapeutic strategies. Biol. Blood Marrow Transpl (2004) 10:815–825.
  • ••Excellent state of the art paper.
  • RUIZ-ARGOELLES GJ, GOMEZ-ALMAGUER D: Breaking dogmata to help patients: non-myeloablative hematopoietic stem cell transplantation. Expert Opin. Biol. Ther. (2004) 4:1693–1699.
  • COURIEL DR, SALIBA RM, GIRALT S et al: Acute and chronic graft versus-host-disease after ablative and nonmyeloablative conditioning for allogeneic hematopoietic transplantation. Biol. Blood Marrow Transpl. (2004) 10:178–185.
  • VELA-OJEDA J, GARCIA-RUIZ ESPARZA MA, TRIPP-VILLANUEVA F: Allogeneic peripheral blood stem cell transplantation using reduced intensity versus myeloablative conditioning regimens for the treatment of leukemia. Stem Cells Dev. (2004) 13:571–579.
  • DEEG HJ, LEISERING W, STORB R et al.: Long- term outcome after marrow transplantation for severe aplastic anemia. Blood (1998) 10:3637–3645.
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  • GOMEZ-ALMAGUER D: The simplification of SCT in developing countries has resulted in cost-lowering and availability to more patients. Int. J. Hematol (2002) 76\(Suppl. 1):380–382.
  • RUIZ-ARGUELLES GJ: Allogeneic stem cell transplantation using non-myeloablative conditioning regimens: Results of the Mexican approach. Int. J. Hematol (2002) 76\(Suppl. 1):376–379.
  • MACIEJEWSKI JP, HIBBS JR, ANDERSON S, KATEVAS P, YOUNG NS: Bone marrow and peripheral blood lymphocyte phenotype in patients with bone marrow failure. Exp. Hematol (1994) 22:1102–1110.
  • MACIEJEWSKI J, SELLERI C, SATO T, ANDERSON S, YOUNG NS: Increased expression of Fas antigen on bone marrow CD34+ cells of patients with aplastic anaemia. Br. J. Haematol. (1995) 91:245–252.
  • RISITANO AM, MACIEJEWSKI JP, GREEN S, PLASILOVA M, ZENG W YOUNG NS: In-vivo dominant immune responses in aplastic anaemia: molecular tracking of putatively pathogenetic T-cell clones by TCR beta-CDR3 sequencing. Lancet (2004) 364:355-364. Excellent perspective paper on the molecular mechanisms involved in AA.
  • NISSEN C, SCHUBERT J: Seeing thegood and bad in aplastic anemia: is autoimmunity in AA dysregulated or antineoplastic? Hematol J. (2002) 3:169–175.
  • ••Provocative hypothesis on an alternative role of autoimmunity in AA.

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