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First-line allogeneic hematopoietic stem cell transplantation of HLA-matched sibling donors compared with first-line ciclosporin and/or antithymocyte or antilymphocyte globulin for acquired severe aplastic anemia

  1. Frank Peinemann1,*,
  2. Carmen Bartel2,
  3. Ulrich Grouven3

Editorial Group: Cochrane Haematological Malignancies Group

Published Online: 23 JUL 2013

Assessed as up-to-date: 22 APR 2013

DOI: 10.1002/14651858.CD006407.pub2


How to Cite

Peinemann F, Bartel C, Grouven U. First-line allogeneic hematopoietic stem cell transplantation of HLA-matched sibling donors compared with first-line ciclosporin and/or antithymocyte or antilymphocyte globulin for acquired severe aplastic anemia. Cochrane Database of Systematic Reviews 2013, Issue 7. Art. No.: CD006407. DOI: 10.1002/14651858.CD006407.pub2.

Author Information

  1. 1

    Children's Hospital, University of Cologne, Cologne, NW, Germany

  2. 2

    Cologne, Germany

  3. 3

    Hannover Medical School, Hannover, Germany

*Frank Peinemann, Children's Hospital, University of Cologne, Kerpener Str. 62, Cologne, NW, 50937, Germany. pubmedprjournal@gmail.com.

Publication History

  1. Publication Status: New
  2. Published Online: 23 JUL 2013

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References

References to studies included in this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
Bayever 1984 {published data only}
  • Bayever E, Champlin R, Ho W, Lenarsky C, Storch S, Ladisch S, et al. Comparison between bone marrow transplantation and antithymocyte globulin in treatment of young patients with severe aplastic anemia. Journal of Pediatrics 1984;105:920-5.
Führer 1998 {published data only}
  • Führer M, Bender-Götze C, Ebell W, Friedrich W, Kohne E. Treatment of aplastic anemia--aims and development of the SAA 94 pilot protocol [Die Behandlung der Aplastischen Anamie--Zielsetzung und Aufbau des Pilotprotokolls SAA 94]. Klinische Pädiatrie 1994;206:289-95.
  • Führer M, Burdach S, Ebell W, Gadner H, Haas R, Harbott J, et al. Relapse and clonal disease in children with aplastic anemia (AA) after immunosuppressive therapy (IST): The SAA 94 experience. Klinische Pädiatrie 1998;210:173-9.
  • Führer M, Rampf U, Baumann I, Faldum A, Niemeyer C, Janka-Schaub G, et al. Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. Blood 2005;106:2102-4.
  • Führer M, Rampf U, Burdach S. Immunosuppressive therapy (IST) and bone marrow transplantation (BMT) for aplastic anemia (AA) in children. Blood 1998;92 Suppl 1:156a, Abstract 631.
  • Führer M, Rampf U, Niemeyer CM, Faldum A, Baumann I, Friedrich W, et al. Bone marrow transplantation and immunosuppressive therapy in children with aplastic anemia: data from a prospective multinational trial in Germany, Austria and Switzerland. Blood 2004;104:Abstract 1439.
Gratwohl 1981 {published data only}
  • Gratwohl A, Osterwalder B, Nissen C, Leibundgut U, Signer E, Luthy A, et al. Treatment of severe aplastic anemia [Behandlung der schweren aplastischen Anämie]. Schweizerische Medizinische Wochenschrift 1981;111:1520-2.

References to studies excluded from this review

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
ACS/NIH 1976 {published data only}
  • ACS/NIH. Bone marrow transplantation from donors with aplastic anemia. A report from the ACS/NIH bone marrow transplant registry. JAMA 1976;236(10):1131-5.
Ahn 2003 {published data only}
  • Ahn MJ, Choi JH, Lee YY, Choi IY, Kim IS, Yoon SS, et al. Outcome of adult severe or very severe aplastic anemia treated with immunosuppressive therapy compared with bone marrow transplantation: multicenter trial. International Journal of Hematology 2003;78:133-8.
Arranz 1994 {published data only}
  • Arranz R, Otero MJ, Ramos R, Steegmann JL, Lamana ML, Tomas JF, et al. Clinical results in 50 multiply transfused patients with severe aplastic anemia treated with bone marrow transplantation or immunosuppressive therapy. Bone Marrow Transplantation 1994;13:383-7.
  • Arranz R, Steegmann JL, Fernandez-Garesse D, Figuera A, Vazquez L, Fernandez-Ranada JM. Treatment of severe aplastic anemia with bone marrow transplants or with antithymocyte globulin. Evaluation of 27 patients [Tratamiento de la anemia aplasica severa con trasplante de medula osea o con globulina antitimocitica. Evaluacion de 27 pacientes]. Revista Clínica Espanola 1989;184:121-4.
Bacigalupo 1988 {published data only}
Bacigalupo 1996 {published data only}
  • Bacigalupo A. Aetiology of severe aplastic anaemia and outcome after allogeneic bone marrow transplantation or immunosuppression therapy. Working Party on Severe Aplastic Anaemia of the European Blood and Marrow Transplantation Group. European Journal of Haematology 1996;60:16-9.
Bacigalupo 2000a {published data only}
  • Bacigalupo A, Oneto R, Bruno B, Socie G, Passweg J, Locasciulli A, et al. Current results 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 Haematologica 2000;103:19-25.
Bacigalupo 2000b {published data only}
  • Bacigalupo A, Brand R, Oneto R, Bruno B, Socie G, Passweg J, et al. Treatment of acquired severe aplastic anemia: bone marrow transplantation compared with immunosuppressive therapy--The European Group for Blood and Marrow Transplantation experience. Seminars in Hematology 2000;37:69-80.
Camitta 1976 {published data only}
  • Camitta BM, Thomas ED, Nathan DG, Santos G, Gordon-Smith EC, Gale RP, et al. Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality. Blood 1976;48:63-70.
Camitta 1978 {published data only}
  • Camitta BM, Thomas ED. Severe aplastic anaemia: a prospective study of the effect of androgens or transplantation on haematological recovery and survival. Clinical Haematology 1978;7:587-95.
Camitta 1979 {published data only}
  • 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;53:504-14.
Cesaro 2010 {published data only}
  • Cesaro S, Marsh J, Tridello G, Rovo A, Maury S, Montante B, et al. Retrospective survey on the prevalence and outcome of prior autoimmune diseases in patients with aplastic anemia reported to the registry of the European group for blood and marrow transplantation (Conference abstract). Acta Haematologica 2010;124(1):19-20.
Champlin 1984 {published data only}
  • Champlin R, Ho W, Bayever E, Winston DJ, Lenarsky C, Feig SA, et al. Treatment of aplastic anemia: results with bone marrow transplantation, antithymocyte globulin, and a monoclonal anti-T cell antibody. Progress in Clinical Biological Research 1984;148:227-38.
Chen 1992 {published data only}
  • Chen RL, Lin KH, Chen BW, Su S, Lin DT, Chuu WM, et al. Long-term observation of pediatric aplastic anemia. Journal of the Formosan Medical Association 1992;91:390-5.
Crump 1992 {published data only}
  • Crump M, Larratt LM, Maki E, Curtis JE, Minden MD, Meharchand JM, et al. Treatment of adults with severe aplastic anemia: primary therapy with antithymocyte globulin (ATG) and rescue of ATG failures with bone marrow transplantation. American Journal of Medicine 1992;92:596-602.
de Planque 1990 {published data only}
  • de Planque MM, Richel DJ, Fibbe WE, den Ottolander GJ, Guiot HF, Zwaan FE. Acquired severe aplastic anaemia in adults--a single centre study with 13 years follow-up. The Netherlands Journal of Medicine 1990;37:103-10.
Deyell 2009 {published data only}
  • Deyell RJ, Shereck EB, Schultz KR. Immunosuppressive therapy without granulocyte-colony stimulating factor in pediatric acquired aplastic anemia (Conference abstract). Pediatric Blood and Cancer 2009 2009;52(6):705.
Doney 1981 {published data only}
  • Doney KC, Weiden PL, Buckner CD, Storb R, Thomas ED. Treatment of severe aplastic anemia using antithymocyte globulin with or without an infusion of HLA haploidentical marrow. Experimental Hematology 1981;9(8):829-34.
Doney 1997 {published data only}
  • Doney K, Leisenring W, Storb R, Appelbaum FR. Primary treatment of acquired aplastic anemia: outcomes with bone marrow transplantation and immunosuppressive therapy. Seattle Bone Marrow Transplant Team. Annals of Internal Medicine 1997;126(2):107-15.
Fouladi 2000 {published data only}
  • Fouladi M, Herman R, Rolland-Grinton M, Jones-Wallace D, Blanchette V, Calderwood S, et al. Improved survival in severe acquired aplastic anemia of childhood. Bone Marrow Transplantation 2000;26:1149-56.
Ganapiev 2010 {published data only}
  • Ganapiev AA, Golubovskaia IK, Zalialov IR, Estrina MA, Afanas'ev BV. The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia [Russian]. Terapevticheskii Arkhiv 2010;82(7):48-52.
Garanito 2009 {published data only}
  • Garanito MP, Filho VO, Dos Santos MV, Velloso E, Dulley FL, Carneiro JDA. Long-term clinical outcome of children with acquired aplastic anemia in Brazil (Conference abstract). Blood 2009;114(22):4216.
George 2010 {published data only}
  • George B, Mathews V, Viswabandya A, Lakshmi KM, Srivastava A, Chandy M. Allogeneic hematopoietic stem cell transplantation is superior to immunosuppressive therapy in Indian children with aplastic anemia single-center analysis of 100 patients. Pediatric Hematology and Oncology 2010;27(2):122-31.
Ghavamzadeh 2004 {published data only}
  • Ghavamzadeh A, Iravani M, Vafaiezadeh F, Jahani M, Mousavi A. Bone marrow transplantation versus immunosuppressive therapy in severe aplastic anemia, 1990 - 2001. Archives of Iranian Medicine 2004;7:272-8.
Gillio 1997 {published data only}
  • Gillio AP, Boulad F, Small TN, Kernan NA, Reyes B, Childs BH, et al. Comparison of long-term outcome of children with severe aplastic anemia treated with immunosuppression versus bone marrow transplantation. Biology of Blood and Marrow Transplantation 1997;3:18-24.
Gluckman 1979 {published data only}
  • Gluckman E, Devergie A, Faille A, Bussel A, Benbunan M, Bernard J. Antilymphocyte globulin treatment in severe aplastic anemia--comparison with bone marrow transplantation. Report of 60 cases. Haematology and Blood Transfusion 1979;24:171-9.
Gluckman 1981 {published data only}
Gluckman 1987 {published data only}
  • Gluckman E. Current status of bone marrow transplantation for severe aplastic anemia: a preliminary report from the International Bone Marrow Transplant Registry. Transplantation Proceedings 1987;19:2597-9.
Golubovskaya 2010 {published data only}
  • Golubovskaya I, Zalyalov Y, Ganapiev A, Afanasyev B. Treatment of acquired aplastic anaemia by bone marrow transplantation and antithymocyte globulin: A 5-year single-centre experience (Conference abstract). Bone Marrow Transplantation 2010;45:S171.
Halperin 1989 {published data only}
  • Halperin DS, Grisaru D, Freedman MH, Saunders EF. Severe acquired aplastic anemia in children: 11-year experience with bone marrow transplantation and immunosuppressive therapy. American Journal of Pediatric Hematology/Oncology 1989;11:304-9.
Hirabayashi 1995 {published data only}
  • Hirabayashi N, Kodera Y, Matsuyama T, Tanimoto M, Horibe K, Naoe T, et al. Bone marrow transplantation in 614 patients: Twenty year experience of the Nagoya Bone Marrow Transplantation Group (NBMTG). Transplantation Proceedings 1995;27:1380-2.
Horowitz 1989 {published data only}
Horowitz 1997 {published data only}
  • Horowitz MM, Rowlings PA. An update from the International Bone Marrow Transplant Registry and the Autologous Blood and Marrow Transplant Registry on current activity in hematopoietic stem cell transplantation. Current Opinion in Hematology 1997;4:395-400.
Howard 2004 {published data only}
Höcker 1987 {published data only}
  • Höcker P, Hinterberger W, Gadner H, Hajek-Rosenmayr A, Fischer-Hinterberger M, Kos M, et al. Therapy of severe aplastic anemia (SAA) by bone marrow transplantation (BMT) or immunosuppression? [Therapie der schweren aplastischen Anämie (SSA) durch Knochenmarktransplantation (KMT) oder Immunsuppression?]. Beiträge zu Infusiontherapie und Klinischer Ernährung 1987;18:329-31.
Ilhan 2001 {published data only}
  • Ilhan O, Bakanay SM, Arat M, Ozcan M, Celebi H, Gurman G, et al. Treatment of aplastic anemia: Experience of Ibn-i Sina Hospital. Turkish Journal of Haematology 2001;18:33-40.
Kahn 2002 {published data only}
  • Kahn Q, Ellis RJ, Skikne BS, Mayo MS, Allgood JW, Bodensteiner DM, et al. A retrospective analysis of long-term survival in severe aplastic anemia patients treated with allogeneic bone marrow transplantation or immunosuppressive therapy with antithymocyte globulin and cyclosporin A at a single institution. Military Medicine 2002;167:541-5.
Kim 1994 {published data only}
  • Kim CC, Jin JY, Kim DJ. Treatment of aplastic anemia: Allogeneic bone marrow transplantation versus immunomodulation therapy with antilymphocyte globulin. Chinese Medical Journal 1994;107:735-6.
Kim 2003 {published data only}
  • Kim I, Yoon SS, Park S, Kim BK, Kim NK. The treatment of severe aplastic anemia: outcomes of bone marrow transplantation and immunosuppressive therapy in a single institution of Korea. Journal of Korean Medical Science 2003;18:365-71.
Kodera 1999 {published data only}
  • Kodera Y, Morishima Y, Kato S, Akiyama Y, Sao H, Matsuyama T, et al. Analysis of 500 bone marrow transplants from unrelated donors (UR-BMT) facilitated by the Japan Marrow Donor Program: confirmation of UR-BMT as a standard therapy for patients with leukemia and aplastic anemia. Bone Marrow Transplantation 1999;24:995-1003.
Kojima 1988 {published data only}
  • Kojima S, Fukuda M, Horibe K, Matsuyama K, Takeyama K, Matsushita T, et al. Comparison between bone marrow transplantation and immuno-suppressive therapy in treatment of patients younger than 20 years with severe aplastic anemia. Acta Haematologica Japonica 1988;51(1):28-35.
Kojima 2000 {published data only}
  • Kojima S, Horibe K, Inaba J, Yoshimi A, Takahashi Y, Kudo K, et al. Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation. British Journal of Haematology 2000;111:321-8.
Kosaka 2004 {published data only}
  • Kosaka Y, Kobayashi R, Ayukawa H, Kato K, Kaneko T, Kigasawa H, et al. Prospective multicenter trial comparing repeated immunosuppressive therapy (IST) with stem cell transplantation (SCT) from an alternative donor as a second-line treatment for children with acquired aplastic anemia (AA). Blood (ASH Annual Meeting Abstracts) 2004;104:Abstract 2815.
Kulagin 2006 {published data only}
  • Kulagin AD, Lisukov IA, Kriuchkova IV, Sizikova SA, Gilevich AV, Denisova VV, et al. Diagnosis and treatment of acquired aplastic anaemia. Terapevticheskii Arkhiv 2006;78(11):48-54.
Lawlor 1997 {published data only}
  • Abella E, Ravindranth Y. Immunosuppressive therapy vs. bone marrow transplant for severe aplastic anemia. Journal of Pediatric Hematology/Oncology 1997;19:533-4.
  • Lawlor ER, Anderson RA, Davis JH, Fryer CJ, Pritchard SL, Rogers PC, et al. Immunosuppressive therapy: a potential alternative to bone marrow transplantation as initial therapy for acquired severe aplastic anemia in childhood?. Journal of Pediatric Hematology/Oncology 1997;19:115-23.
Ljungman 1991 {published data only}
  • Ljungman P, Aschan J, Bolme P, Lindqvist R, Lonnqvist B, Ringden O, et al. Bone marrow transplantation or antithymocyte globulin in aplastic anemia? Both therapeutic alternatives result in good quality of life [Benmärgstransplantation eller antitymocytglobulin vid aplastisk anemi? Bada behandlingsalternativen ger god livskvalitet]. Läkartidningen 1991;88:2308-11.
Locasciulli 1990 {published data only}
  • Locasciulli A, van't Veer L, Bacigalupo A, Hows J, Van Lint MT, Gluckman E, et al. Treatment with marrow transplantation or immunosuppression of childhood acquired severe aplastic anemia: a report from the EBMT SAA Working Party. Bone Marrow Transplantation 1990;6:211-7.
Locasciulli 2007 {published data only}
  • Locasciulli A, Oneto R, Bacigalupo A, Socié G, Korthof E, Bekassy A, et al. 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. Haematologica 2007;92:11-8.
McCann 1994 {published data only}
  • McCann SR, Bacigalupo A, Gluckman E, Hinterberger W, Hows J, Ljungman P, et al. Graft rejection and second bone marrow transplants for acquired aplastic anaemia: a report from the Aplastic Anaemia Working Party of the European Bone Marrow Transplant Group. Bone Marrow Transplantation 1994;13:233-7.
Milosevic 1998 {published data only}
  • Milosevic R, Antonijevic N, Jankovic G, Babic D, Colovic M. Aplastic anemia--clinical characteristics and survival analysis [Aplasticna anemija--klinicke odlike i analiza prezivnjavanja]. Srpski Arhiv za Celokupno Lekarstvo 1998;126:234-8.
Mollee 2001 {published data only}
Montante 2009 {published data only}
  • Montante B, Pinazzi MB, Severino A, Proia A, Majolino I, Locasciulli A. Cyclosporin monitoring at 2 hours from oral intake leads to less toxicity and better compliance in patients undergoing hematopoietic stem cells transplantation and immunosuppressive treatment for severe aplastic anemia (Conference abstract). Haematologica 2009;94:219-20.
Mrsic 2009 {published data only}
  • Mrsic M, Serventi Seiwerth R, Labar B, Bogdanic V, Nemet D, Durakovic N, et al. Twenty years of severe aplastic anemia treatment at Department of Hematology, University Department of Medicine, Zagreb University Hospital Center, Zagreb, Croatia [Dvadeset godina lijecenja teske aplasticne anemije u Zavodu za Hematologiju Klinike za Unutrasnje Bolesti Klinickog Bolnickog Centra Zagreb]. Acta Medica Croatica 2009;63(3):209-14.
Nagler 2001 {published data only}
  • Nagler A, Aker M, Or R, Naparstek E, Varadi G, Brautbar C, et al. Low-intensity conditioning is sufficient to ensure engraftment in matched unrelated bone marrow transplantation. Experimental Hematology 2001;29(3):362-70.
Paquette 1995 {published data only}
  • Paquette RL, Tebyani N, Frane M, Ireland P, Ho WG, Champlin RE, et al. Long-term outcome of aplastic anemia in adults treated with antithymocyte globulin: comparison with bone marrow transplantation. Blood 1995;85:283-90.
Pitcher 1999 {published data only}
Podesta 1998 {published data only}
  • Podesta M, Piaggio G, Frassoni F, Pitto A, Zikos P, Sessarego M, et al. The assessment of the hematopoietic reservoir after immunosuppressive therapy or bone marrow transplantation in severe aplastic anemia. Blood 1998;91:1959-65.
Pokorski 1998 {published data only}
  • Pokorski RJ. 5-year survival after bone marrow transplantation for aplastic anemia. Journal of Insurance Medicine 1998;30:237-9.
Shaw 1999 {published data only}
  • Shaw PH, Haut PR, Olszewski M, Kletzel M. Hematopoietic stem-cell transplantation using unrelated cord-blood versus matched sibling marrow in pediatric bone marrow failure syndrome: one center's experience. Pediatric Transplantation 1999;3:315-21.
Socié 1993 {published data only}
  • Socie G, Henry-Amar M, Bacigalupo A, Hows J, Tichelli A, Ljungman P, et al. Malignant tumors occurring after treatment of aplastic anemia. European Bone Marrow Transplantation-Severe Aplastic Anaemia Working Party. New England Journal of Medicine 1993;329:1152-7.
Sovinz 2010 {published data only}
  • Sovinz P, Lackner H, Schwinger W, Benesch M, Nebl A, Urban C. Management of severe aplastic anaemia in childhood: A single-centre experience over 30 years (Conference abstract). Bone Marrow Transplantation. 2010; Vol. 45:S272.
Stalder 2009 {published data only}
Starý 1998 {published data only}
  • Stary J, Sedlacek P, Kobylka P, Komrska V, Syruckova Z, Smisek P, et al. Significant progress in the treatment of acquired aplastic anemia in children in 1990s [Vyznamny pokrok v lecbe ziskane aplasticke anemie u deti v 90. Letech]. Ceskoslovenska Pediatrie 1998;53:260-6.
Tichelli 1994 {published data only}
  • Speck B, Gratwohl A, Nissen C. Severe aplastic anemia: A prospective study on the value of different therapeutic approaches in 37 successive patients. Blut 1980;41:160-3.
  • Speck B, Gratwohl A, Nissen C, Leibundgut U, Ruggero D, Osterwalder B, et al. Treatment of severe aplastic anaemia with antilymphocyte globulin or bone-marrow transplantation. British Medical Journal 1981;282:860-3.
  • Speck B, Gratwohl A, Nissen C, Osterwalder B, Signer E, Jeannet M. Bone marrow graft versus ALG in patients with aplastic anaemia. Biomedicine & Pharmacotherapy 1983;37:139-43.
  • Speck B, Gratwohl A, Nissen C, Osterwalder B, Signer E, Jeannet M. Treatment of severe aplastic anemia: a prospective study of antilymphocyte globulin versus bone marrow transplantation. Progress in Clinical Biological Research 1984;148:249-58.
  • Speck B, Gratwohl A, Nissen C, Osterwalder B, Wursch A, Tichelli A, et al. A comparison between ALG and bone marrow transplantation in treatment of severe aplastic anemia. Thymus 1987;10:147-58.
  • Speck B, Gratwohl A, Nissen C, Osterwalder B, Wursch A, Tichelli A, et al. Treatment of severe aplastic anemia. Experimental Hematology 1986;14:126-32.
  • Speck R, Tichelli A, Gratwohl A, Nissen C. Treatment of severe aplastic anemia: A longterm followup of 175 patients on antilymphocyte globulin or bone marrow transplantation. Chinese Medical Journal 1994;107:739.
  • Tichelli A, Gratwohl A, Nissen C, Speck B. Late clonal complications in severe aplastic anemia. Leukemia & Lymphoma 1994;12:167-75.
  • Tichelli A, Gratwohl A, Nissen C, Wursch A, Signer E, Speck B. Late complications in patients with aplastic anemia [Spätkomplikationen bei Patienten mit aplastischer Anämie]. Schweizerische Medizinische Wochenschrift 1988;118:1528-32.
  • Tichelli A, Gratwohl A, Wursch A, Nissen C, Speck B. Late haematological complications in severe aplastic anaemia. British Journal of Haematology 1988;69(3):413-8.
Tschiedel 2010 {published data only}
  • Tschiedel E, Gierenz N, Wieland R, Wulff B, Ballauff A. Severe aplastic anaemia in six children after non-A-E hepatitis without hepatic failure [Schwere aplastische Anamie bei 6 Kindern nach Non-A-E-Hepatitis ohne Leberversagen]. Zeitschrift fur Gastroenterologie 2010;48(8):825-8.
Tsukimoto 1989 {published data only}
  • Tsukimoto I, Tsuchida M, Ohara A, Akabane T, Nakahata T, Akatsuka J, et al. Long-term prognosis and residual abnormalities of idiopathic acquired aplastic anemia in children. Nippon Ketsueki Gakkai Zasshi 1989;52:1370-8.
Tukic 2009 {published data only}
  • Tukic L, Stamatovic D, Tarabar O, Elez M, Tatomirovic Z, Radic-Tasic O, et al. Long-term outcome of acquired aplastic anemia: Comparation between immunosuppressive therapy and bone marrow transplantation (Conference abstract). Haematologica. 2009; Vol. 94:241.
Tzeng 1989 {published data only}
  • Tzeng CH, Chen PM, Chuang MW, Liu JH, Hsieh RK, Liu CJ, et al. Treatment of severe aplastic anemia: comparison of bone marrow transplantation to immunotherapy. Chung Hua I Hsueh Tsa Chih 1989;43:21-8.
UCLA 1976 {published data only}
  • UCLA Bone Marrow Transplant Team. Bone-marrow transplantation in severe aplastic anaemia. Lancet 1976;308(7992):921-3.
Uss 1999 {published data only}
  • Uss AL, Zmachinskii VA, Milanovich NF, Skriagin AE, Batan ZE, Dziuba EV, et al. Experience in the use of allogeneic bone marrow transplantation in severe forms of aplastic anemia at the Byelorussian hematological center [Opyt primeneniia transplantatsii allogennogo kostnogo mozga pri tiazheloi forme aplasticheskoi anemii v Belorusskom gematologicheskom tsentre]. Terapevticheskii Arkhiv 1999;71:69-72.
Viollier 2005 {published data only}
  • Viollier R, Passweg J, Gregor M, Favre G, Kühne T, Nissen C, et al. Quality-adjusted survival analysis shows differences in outcome after immunosuppression or bone marrow transplantation in aplastic anemia. Annals of Hematology 2005;84:47-55.
Wagner 1996 {published data only}
  • Wagner JE, Rosenthal J, Sweetman R, Shu XO, Davies SM, Ramsay NK, et al. Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease. Blood 1996;88:795-802.
Webb 1991 {published data only}
Werner 1989 {published data only}
  • Werner EJ, Stout RD, Valdez LP, Harris RE. Immunosuppressive therapy versus bone marrow transplantation for children with aplastic anemia. Pediatrics 1989;83:61-5.
Windass 1987 {published data only}
Yan 2011 {published data only}
  • Yan HM, Liu J, Xue M, Wang ZD, Zhu L, Ding L, et al. Comparison of hematopoietic stem cell transplantation and non-transplantation for treatment of severe aplastic anemia [Chinese]. Journal of Clinical Rehabilitative Tissue Engineering Research 2011;15(1):63-7.
Yoshida 2011 {published data only}
  • Yoshida N, Kikuchi A, Kobayashi R, Yabe H, Kosaka Y, Muramatsu H, et al. Outcomes in children with severe aplastic anemia receiving bone marrow transplantation from an HLA-matched family donor or intensive immunosuppressive therapy as first-line treatment. Blood (ASH Annual Meeting Abstracts). Washington, DC: American Society of Hematology, 2011; Vol. 118:Abstract 3421.

Additional references

  1. References to studies included in this review
  2. References to studies excluded from this review
  3. Additional references
ASH meeting abstracts 2012
  • American Society of Hematology. ASH meeting abstracts. Blood. Washington, DC: American Society of Hematology, 2012 (http://bloodjournal.hematologylibrary.org/site/misc/ASH_Meeting_Abstracts_Info.xhtml).
Bacigalupo 2000
  • Bacigalupo A, Brand R, Oneto R, Bruno B, Socie G, Passweg J, et al. Treatment of acquired severe aplastic anemia: bone marrow transplantation compared with immunosuppressive therapy--The European Group for Blood and Marrow Transplantation experience. Seminars in Hematology 2000;37:69-80.
Bacigalupo 2008
Bacigalupo 2012
  • Bacigalupo A, Socie G, Schrezenmeier H, Tichelli A, Locasciulli A, Führer M, et al. Bone marrow versus peripheral blood sibling transplants in acquired aplastic anemia: survival advantage for marrow in all age groups. Haematologica 2012;97(8):1142-8.
Battiwalla 2012
  • Battiwalla M, Wang T, Carreras J, Deeg HJ, Ayas M, Bajwa RPS, et al. HLA-matched sibling transplantation for severe aplastic anemia: Impact of HLA DR15 antigen status on engraftment, graft-versus-host disease, and overall survival. Biology of Blood and Marrow Transplantation 2012;18(9):1401-6.
Brodsky 2005
Buchbinder 2013
  • Buchbinder D, Hsieh L, Puthenveetil G, Soni A, Stites J, Huynh V, et al. Successful autologous cord blood transplantation in a child with acquired severe aplastic anemia. Pediatric Transplantation 2013;17(3):E104-7.
Burroughs 2012
Camitta 1975
Chybicka 2008
  • Chybicka A. Quality of life and ethical and legal dilemmas in children during and after hematopoietic SCT procedure. Bone Marrow Transplantation 2008;42 Suppl 2:S87-9.
ClinicalTrials.gov 2012
  • NIH National Institutes of Health. ClinicalTrials.gov. Department of Health and Human Services (http://clinicaltrials.gov/) 2012.
Deeks 2011
  • Deeks JJ, Higgins JPT, Altman DG. Chapter 9: Analysing data and undertaking meta-analyses. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org 2011.
DerSimonian 1986
Deyell 2011
  • Deyell RJ, Shereck EB, Milner RA, Schultz KR. Immunosuppressive therapy without hematopoietic growth factor exposure in pediatric acquired aplastic anemia. Pediatric Hematology and Oncology 2011;28(6):469-78.
Eapen 2012
  • Eapen M. Allogeneic transplantation for aplastic anemia. Hematology 2012;17 Suppl 1:15-7.
EBMT meeting abstracts 2012
  • European Group of Blood and Marrow Transplantation. EBMT meeting abstracts. EBMT conference proceedings. London: European Group of Blood and Marrow Transplantation, 2012 (http://www.ebmt.org/Contents/Annual-Meeting/PastEBMTAnnualMeetings/Pages/Past-EBMT-Annual-Meetings.aspx).
Frickhofen 2003
  • Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H. German Aplastic Anemia Study Group. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood 2003;101:1236-42.
GARD 2012
  • Genetic and Rare Diseases Information Center (GARD). Aplastic anemia. The Office of Rare Diseases Research (ORDR) and the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH) (http://rarediseases.info.nih.gov/GARD/Condition/5836/Aplastic_anemia.aspx) 2012.
GCCR 2006
  • Annual Report 2006. Routine Analyses: diagnoses and cases, time trends, regional differences, survival probabilities, and mortality. Summary data for selected diagnoses not defined in ICCC-3 of patients under 15 in Germany (2001-2005). German Childhood Cancer Registry (GCCR) (http://www.kinderkrebsregister.de/external/publications/annual-reports/jb2006gb/index.html?L=1) 2006.
Google Translate 2012 [Computer program]
  • Google, Inc. Google Translate (http://translate.google.com/). Mountain View: Google, Inc, 2012.
GRADEpro 2008 [Computer program]
  • Brozek J, Oxman A, Schünemann H. GRADEpro (GRADE profiler) (http://ims.cochrane.org/gradepro). Version Version 3.2 for Windows. Copenhagen: Information Management System (IMS), Nordic Cochrane Centre, The Cochrane Collaboration, 2008.
Gray 1991
  • Gray R, Wheatley K. How to avoid bias when comparing bone marrow transplantation with chemotherapy. Bone Marrow Transplantation 1991;7(Suppl 3):9-12.
Guinan 2009
Gupta 2010
  • Gupta V, Eapen M, Brazauskas R, Carreras J, Aljurf M, Gale RP, et al. Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors. Haematologica 2010;95(12):2119-25.
Higgins 2003
Higgins 2011a
  • Higgins JPT, Altman DG, Sterne JAC. Table 8.5.a The Cochrane Collaboration's tool for assessing risk of bias. Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
Higgins 2011b
  • Higgins JPT, Altman DG, Sterne JAC. Table 8.5.d Criteria for judging risk of bias in the 'Risk of bias' assessment tool. Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
Higgins 2011c
  • Higgins JPT, Deeks JJ, Altman DG. 16.1 Missing data. Chapter 16: Special topics in statistics. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
ICTRP 2012
  • ICTRP International Clinical Trials Registry Platform. ICTRP Search Platform. WHO World Health Organization (http://www.who.int/ictrp/en/) 2012.
Kamio 2011
  • Kamio T, Ito E, Ohara A, Kosaka Y, Tsuchida M, Yagasaki H, et al. Relapse of aplastic anemia in children after immunosuppressive therapy: a report from the Japan Childhood Aplastic Anemia Study Group. Haematologica 2011;96(6):814-9.
Karnofsky 1949
  • Karnofsky DA, Burchenal JH. The clinical evaluation of chemotherapeutic agents in cancer. In: MacLeod CM editor(s). Evaluation of Chemotherapeutic Agents. New York City: Columbia University Press, 1949:191.
Katan 1986
  • Katan MB. Apolipoprotein E isoforms, serum cholesterol, and cancer. Lancet 1986;327(8479):507-8.
Kaufman 2006
Killick 2000
Kim 2012
  • Kim H, Lee KH, Yoon SS, Sohn SK, Joo YD, Kim SH, et al. Allogeneic hematopoietic stem cell transplant for adults over 40 years old with acquired aplastic anemia. Biology of Blood and Marrow Transplantation 2012;18(10):1500-8.
Kojima 2011
  • Kojima S, Nakao S, Young N, Bacigalupo A, Gerard G, Hirano N, et al. The Third Consensus Conference on the treatment of aplastic anemia. International Journal of Hematology 2011;93(6):832-7.
Lefebvre 2011
  • Lefebvre C, Manheimer E, Glanville J. Chapter 6: Searching for studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
Lewis 2010
  • Lewis SJ. Mendelian randomization as applied to coronary heart disease, including recent advances incorporating new technology. Circulation Cardiovascular Genetics 2010;3(1):109-17.
Marsh 2009
Marsh 2011
  • Marsh JCW, Cavenagh J, Darbyshire P, Dokal I, Gordon-Smith EC, Keidan J, et al. Guidance from the BCSH Aplastic Anaemia Writing Committee relating to recent prospective studies of rabbit antithymocyte globulin (ATG) used for a first course of immunosuppressive therapy (IST) in aplastic anaemia. British Committee for Standards in Haematology (BCSH), London, UK (http://www.bcshguidelines.com/documents/Use_of_rabbit_ATG_July_2011.pdf) 2011.
Moher 2009
Nitsch 2006
  • Nitsch D, Molokhia M, Smeeth L, DeStavola BL, Whittaker JC, Leon DA. Limits to causal inference based on Mendelian randomization: a comparison with randomized controlled trials. American Journal of Epidemiology 2006;163(5):397-403.
NPCRC 2012
  • Karnofsky performance status scale definitions rating (%) criteria. National Palliative Care Research Center (NPCRC) (http://www.npcrc.org/usr_doc/adhoc/functionalstatus/Karnofsky%20Performance%20Scale.pdf) 2012.
Parmar 1998
Passweg 2010
  • Passweg JR, Marsh JCW. Aplastic Anemia: First-line treatment by immunosuppression and sibling marrow transplantation. Hematology American Society of Hematology Education Program 2010;2010:36-42.
Peinemann 2011
  • Peinemann F, Grouven U, Kroger N, Bartel C, Pittler MH, Lange S. First-line matched related donor hematopoietic stem cell transplantation compared to immunosuppressive therapy in acquired severe aplastic anemia. PLoS One 2011;6(4):e18572.
Review Manager 2011 [Computer program]
  • The Nordic Cochrane Centre, The Cochrane Collaboration. Review Manager (RevMan). Version 5.1. Copenhagen: The Nordic Cochrane Centre, The Cochrane Collaboration, 2011.
Risitano 2013
  • Risitano AM, Schrezenmeier H. Alternative immunosuppression in patients failing immunosuppression with ATG who are not transplant candidates: Campath (Alemtuzumab). Bone Marrow Transplantation 2013;48(2):186-90.
Sangiolo 2010
  • Sangiolo D, Storb R, Deeg HJ, Flowers MED, Martin PJ, Sandmaier BM, et al. Outcome of allogeneic hematopoietic cell transplantation from HLA-identical siblings for severe aplastic anemia in patients over 40 years of age. Biology of Blood and Marrow Transplantation 2010;16(10):1411-8.
Scheinberg 2011
  • Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. New England Journal of Medicine 2011;365(5):430-8.
Scheinberg 2012a
  • Scheinberg P. Aplastic anemia: therapeutic updates in immunosuppression and transplantation. Hematology / the Education Program of the American Society of Hematology 2012;2012:292-300.
Scheinberg 2012b
Schrezenmeier 2000
  • Schrezenmeier H, Bacigalupo A, Aglietta M, Frickhofen N, Führer M, Gluckman E, et al. Guidelines for treatment of aplastic anemia. Consensus document of a group of international experts. In: Schrezenmeier H, Bacigalupo A editor(s). Aplastic Anemia. Pathophysiology and Treatment. Cambridge: Cambridge University Press, 2000:308-15.
Schrezenmeier 2007
  • Schrezenmeier H, Passweg JR, Marsh JCW, Bacigalupo A, Bredeson CN, Bullorsky E, et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA-matched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood 2007;110(4):1397-400.
Schünemann 2011
  • Schünemann HJ, Oxman AD, Vist GE, Higgins JPT, Deeks JJ, Glasziou P, et al. Chapter 12: Interpreting results and drawing conclusions. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
Sterne 2011
  • Sterne JAC, Egger M, Moher D. Chapter 10: Addressing reporting biases. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
Thomson Reuters Corp 2012 [Computer program]
  • Thomson Reuters. EndNote. Version X3. New York City: Thomson Reuters, 2012.
Tierney 2007
Wheatley 2004
  • Wheatley K, Gray R. Commentary: Mendelian randomization: an update on its use to evaluate allogeneic stem cell transplantation in leukaemia. International Journal of Epidemiology 2004;33:15-7.
Young 2006