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The Role of Hematopoietic Growth Factors in Aplastic Anemia: An Evidence-Based Perspective

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Twenty Years of G-CSF

Part of the book series: Milestones in Drug Therapy ((MDT))

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Abstract

Evidence-based medicine, which tries to directly base clinical practice and policy decision on supporting evidence, is becoming more and more popular in the establishment of practice guidelines, and is now part of the process of decision making in medicine [1]. Evidence-based medicine aims to apply the best available evidence gained from the scientific research to medical decision making. Evidence-based medicine uses, in a balanced manner, the most updated and best evidence for treating the patient, and represents the transition from obtaining clinical decisions based on previous clinical experience and physicians’ “gut feeling” to decision making based on relevant clinical trials with precise research objectives, ranked by their relevance and validity according to established criteria formed specifically for the use of evidence-based medicine.

Please note the Erratum to this table at the end of the book

An erratum to this chapter can be found at http://dx.doi.org/10.1007/978-3-0348-0218-5_25

An erratum to this chapter can be found at http://dx.doi.org/10.1007/978-3-0348-0218-5_25

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References

  1. Woolf SH (2000) Evidence-based medicine and practice guidelines: an overview. Cancer Control 7:362–367

    PubMed  CAS  Google Scholar 

  2. Gale RP, Lazarus HM (2011) How helpful are meta-analyses in determining the best therapy of blood diseases? Acta Haematologica 125:91–101

    Article  PubMed  Google Scholar 

  3. Harbour R, Miller J (2001) A new system for grading recommendations in evidence based guidelines. Br Med J 323:334–336

    Article  CAS  Google Scholar 

  4. Marsh JC, Ball SE, Cavenagh J et al (2009) Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol 147:43–70

    Article  PubMed  CAS  Google Scholar 

  5. Tichelli A, Schrezenmeier H, Socie G et al (2009) Use of G-CSF in patients with severe aplastic anemia treated with ATG and cyclosporine increases neutrophils and decreases infection rates and hospitalization days but does not improve long-term outcome: results of a prospective, randomized clinical trial of the EBMT. Blood 114:205

    Google Scholar 

  6. Rothwell PM (1995) Can overall results of clinical trials be applied to all patients? Lancet 345:1616–1619

    Article  PubMed  CAS  Google Scholar 

  7. Mant D (1999) Can randomised trials inform clinical decisions about individual patients? Lancet 353:743–746

    Article  PubMed  CAS  Google Scholar 

  8. Kunz R, Oxman AD (1998) The unpredictability paradox: review of empirical comparisons of randomised and non-randomised clinical trials. Br Med J 317:1185–1190

    Article  CAS  Google Scholar 

  9. Bohlius J, Schwarzer G (2011) Twist and shout: one decade of meta-analyses on erythropoiesis-stimulating agents in cancer patients. Acta Haematologica 125:55–67

    Article  PubMed  Google Scholar 

  10. Rennie D (1996) How to report randomized controlled trials. The CONSORT statement. JAMA 276:649

    Article  PubMed  CAS  Google Scholar 

  11. Begg C, Cho M, Eastwood S et al (1996) Improving the quality of reporting of randomized controlled trials. The CONSORT statement. JAMA 276:637–639

    Article  PubMed  CAS  Google Scholar 

  12. Moher D, Cook DJ, Eastwood S, Olkin I, Rennie D, Stroup DF (1999) Improving the quality of reports of meta-analyses of randomised controlled trials: the QUOROM statement. Lancet 354:1896–1900

    Article  PubMed  CAS  Google Scholar 

  13. Moher D, Liberati A, Tetzlaff J, Altman DG, and the PRISMA Group (2009) Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement. Ann Intern Med 151:264–269

    PubMed  Google Scholar 

  14. Brodsky RA (1998) Biology and management of acquired severe aplastic anemia. Curr Opin Oncol 10:95–99

    Article  PubMed  CAS  Google Scholar 

  15. Montane E, Ibanez L, Vidal X et al (2008) Epidemiology of aplastic anemia: a prospective multicenter study. Haematologica 93:518–523

    Article  PubMed  Google Scholar 

  16. Issaragrisil S, Kaufman DW, Anderson T et al (2006) The epidemiology of aplastic anemia in Thailand. Blood 107:1299–1307

    Article  PubMed  CAS  Google Scholar 

  17. Young NS, Calado RT, Scheinberg P (2006) Current concepts in the pathophysiology and treatment of aplastic anemia. Blood 108:2509–2519

    Article  PubMed  CAS  Google Scholar 

  18. Camitta BM, Thomas ED, Nathan DG et al (1979) A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia. Blood 53:504–514

    PubMed  CAS  Google Scholar 

  19. Bacigalupo A, Hows J, Gluckman E et al (1998) Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT SAA working party. Br J Haematol 70:177–182

    Article  Google Scholar 

  20. Ades L, Mary JY, Robin M et al (2004) Long-term outcome after bone marrow transplantation for severe aplastic anemia. Blood 103:2490–2497

    Article  PubMed  CAS  Google Scholar 

  21. Storb R, Leisenring W, Anasetti C et al (1997) Long-term follow-up of allogeneic marrow transplants in patients with aplastic anemia conditioned by cyclophosphamide combined with antithymocyte globulin. Blood 89:3890–3891

    PubMed  CAS  Google Scholar 

  22. Anasetti C, Doney KC, Storb R et al (1986) Marrow transplantation for severe aplastic anemia. Long-term outcome in fifty “untransfused” patients. Ann Intern Med 104:461–466

    PubMed  CAS  Google Scholar 

  23. Horowitz MM (2000) Current status of allogeneic bone marrow transplantation in acquired aplastic anemia. Semin Hematol 37:30–42

    Article  PubMed  CAS  Google Scholar 

  24. Armand P, Antin JH (2007) Allogeneic stem cell transplantation for aplastic anemia. Biol Blood Marrow Transplant 13:505–516

    Article  PubMed  CAS  Google Scholar 

  25. Locasciulli A, Oneto R, Bacigalupo A et al (2007) 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 (EBMT). Haematologica 92:11–18

    Article  PubMed  CAS  Google Scholar 

  26. Fuhrer M, Rampf U, Baumann I et al (2005) Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. Blood 106:2102–2104

    Article  PubMed  Google Scholar 

  27. Bacigalupo A, Bruno B, Saracco P et al (2000) 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 (EBMT) Working Party on Severe Aplastic Anemia and the Gruppo Italiano Trapianti di Midolio Osseo (GITMO). Blood 95:1931–1934

    PubMed  CAS  Google Scholar 

  28. Gafter-Gvili A, Ram R, Gurion R et al (2008) ATG plus cyclosporine reduces all-cause mortality in patients with severe aplastic anemia – systematic review and meta-analysis. Acta Haematol 120:237–243

    Article  PubMed  CAS  Google Scholar 

  29. Marsh J, Schrezenmeier H, Marin P et al (1999) Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood 93:2191–2195

    PubMed  CAS  Google Scholar 

  30. Marsh J, Socie G, Tichelli A et al (2010) Should irradiated blood products be given routinely to all patients with aplastic anaemia undergoing immunosuppressive therapy with antithymocyte globulin (ATG)? A survey from the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party. Br J Haematol 150:377–379

    Article  PubMed  Google Scholar 

  31. Gafter-Gvili A, Paul M, Fraser A, Leibovici L (2007) Antibiotic prophylaxis in neutropenic patients. Isr Med Assoc J 9:460–462

    PubMed  CAS  Google Scholar 

  32. Robenshtok E, Gafter-Gvili A, Goldberg E et al (2007) Antifungal prophylaxis in cancer patients after chemotherapy or hematopoietic stem-cell transplantation: systematic review and meta-analysis. J Clin Oncol 25:5471–5489

    Article  PubMed  CAS  Google Scholar 

  33. Vadhan-Raj S, Broxmeyer HE, Hittelman WN (1992) Use of granulocyte-macrophage colony-stimulating factor in hematopoietic disorders: biology and nature of response. Semin Hematol 29:4–13

    PubMed  CAS  Google Scholar 

  34. Hord JD, Gay JC, Whitlock JA et al (1995) Long-term granulocyte-macrophage colony-stimulating factor and immunosuppression in the treatment of acquired severe aplastic anemia. J Pediatr Hematol Oncol 7:140–144

    Article  Google Scholar 

  35. Locasciulli A, Arcese W, Locatelli F, Di Bona E, Bacigalupo A (2001) Treatment of aplastic anaemia with granulocyte-colony stimulating factor and risk of malignancy. Italian Aplastic Anaemia Study Group. Lancet 357:43–44

    Article  PubMed  CAS  Google Scholar 

  36. Symeonidis A, Kouraklis-Symeonidis A, Seimeni U et al (2002) Ticlopidine-induced aplastic anemia: two new case reports, review, and meta-analysis of 55 additional cases. Am J Hematol 71:24–32

    Article  PubMed  CAS  Google Scholar 

  37. Marsh JC, Ball SE, Darbyshire P et al (2003) Guidelines for the diagnosis and management of acquired aplastic anaemia. Br J Haematol 123:782–801

    Article  PubMed  CAS  Google Scholar 

  38. Negrin RS, Stein R, Doherty K et al (1996) Maintenance treatment of the anemia of myelodysplastic syndromes with recombinant human granulocyte colony-stimulating factor and erythropoietin: evidence for in vivo synergy. Blood 87:4076–4081

    PubMed  CAS  Google Scholar 

  39. Gordon-Smith EC, Yandle A, Milne A et al (1991) Randomised placebo controlled study of RH-GM-CSF following ALG in the treatment of aplastic anaemia. Bone Marrow Transplant 7(Suppl 2):78–80

    PubMed  Google Scholar 

  40. Shao Z, Chu Y, Zhang Y, Chen G, Zheng Y (1998) Treatment of severe aplastic anemia with an immunosuppressive agent plus recombinant human granulocyte-macrophage colony-stimulating factor and erythropoietin. Am J Hematol 59:185–191

    Article  PubMed  CAS  Google Scholar 

  41. Kojima S, Hibi S, Kosaka Y et al (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

    PubMed  CAS  Google Scholar 

  42. Gluckman E, Rokicka-Milewska R, Hann I et al (2002) Results and follow-up of a phase III randomized study of recombinant human-granulocyte stimulating factor as support for immunosuppressive therapy in patients with severe aplastic anaemia. Br J Haematol 119:1075–1082

    Article  PubMed  CAS  Google Scholar 

  43. Zheng Y, Liu Y, Chu Y (2006) Immunosuppressive therapy for acquired severe aplastic anemia (SAA): a prospective comparison of four different regimens. Exp Hematol 34:826–831

    Article  PubMed  CAS  Google Scholar 

  44. Teramura M, Kimura A, Iwase S et al (2007) 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 10:1756–1761

    Article  Google Scholar 

  45. Gurion R, Gafter-Gvili A, Paul M et al (2009) Hematopoietic growth factors in aplastic anemia patients treated with immunosuppressive therapy-systematic review and meta-analysis. Haematologica 94:712–719

    Article  PubMed  CAS  Google Scholar 

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Author information

Authors and Affiliations

  1. Hemato-oncology Hospitalization Unit, Institute of Hematology, Davidoff Cancer Center, Beilinson Hospital, Rabin Medical Center, Petah-Tikva, 49100, Israel

    Pia Raanani, Ronit Gurion, Anat Gafter-Gvili, Isaac Ben-Bassat & Ofer Shpilberg

Authors
  1. Pia Raanani
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  2. Ronit Gurion
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  3. Anat Gafter-Gvili
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  4. Isaac Ben-Bassat
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  5. Ofer Shpilberg
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Corresponding author

Correspondence to Pia Raanani .

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Editors and Affiliations

  1. Amgen Inc., One Amgen Center Drive, Thousand Oaks, 91320, California, USA

    Graham Molineux

  2. MA Foote Associates, Par Five Court 4284, Westlake Village, 91362, California, USA

    MaryAnn Foote

  3. Amgen, Inc., One Amgen Center Dr. 1, Thousand Oaks, 91320, California, USA

    Tara Arvedson

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Raanani, P., Gurion, R., Gafter-Gvili, A., Ben-Bassat, I., Shpilberg, O. (2012). The Role of Hematopoietic Growth Factors in Aplastic Anemia: An Evidence-Based Perspective. In: Molineux, G., Foote, M., Arvedson, T. (eds) Twenty Years of G-CSF. Milestones in Drug Therapy. Springer, Basel. https://doi.org/10.1007/978-3-0348-0218-5_11

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