Recent advances in treatment of aplastic anemia
1
조회
0
다운로드
2
인용
0
참고문헌
키워드
초록
Recent advances in the treatment of aplastic anemia (AA) made most of patients toexpect to achieve a long-term survival. Allogeneic stem cell transplantation (SCT)from HLA-matched sibling donor (MSD-SCT) is a preferred first-line treatmentoption for younger patients with severe or very severe AA, whereas immunosuppressivetreatment (IST) is an alternative option for others. Horse anti-thymocyteglobuline (ATG) with cyclosporin A (CsA) had been a standard IST regimen withacceptable response rate. Recently, horse ATG had been not available and replacedwith rabbit ATG in most countries. Subsequently, recent comparative studiesshowed that the outcomes of patients who received rabbit ATG/CsA were similaror inferior compared to those who received horse ATG/CsA. Therefore, furtherstudies to improve the outcomes of IST, including additional eltrombopag, arenecessary. On the other hand, the upper age limit of patients who are able toreceive MSD-SCT as first-line treatment is a current issue because of favorableoutcomes of MSD-SCT of older patients using fludarabine-based conditioning. In addition, further studies to improve the outcomes of patients who receive allogeneicSCT from alternative donors are needed. In this review, current issues andthe newly emerging trends that may improve their outcomes in near futures willbe discussed focusing the management of patients with AA.
Abstract
Recent advances in the treatment of aplastic anemia (AA) made most of patients toexpect to achieve a long-term survival. Allogeneic stem cell transplantation (SCT)from HLA-matched sibling donor (MSD-SCT) is a preferred first-line treatmentoption for younger patients with severe or very severe AA, whereas immunosuppressivetreatment (IST) is an alternative option for others. Horse anti-thymocyteglobuline (ATG) with cyclosporin A (CsA) had been a standard IST regimen withacceptable response rate. Recently, horse ATG had been not available and replacedwith rabbit ATG in most countries. Subsequently, recent comparative studiesshowed that the outcomes of patients who received rabbit ATG/CsA were similaror inferior compared to those who received horse ATG/CsA. Therefore, furtherstudies to improve the outcomes of IST, including additional eltrombopag, arenecessary. On the other hand, the upper age limit of patients who are able toreceive MSD-SCT as first-line treatment is a current issue because of favorableoutcomes of MSD-SCT of older patients using fludarabine-based conditioning. In addition, further studies to improve the outcomes of patients who receive allogeneicSCT from alternative donors are needed. In this review, current issues andthe newly emerging trends that may improve their outcomes in near futures willbe discussed focusing the management of patients with AA.
참고문헌 (96)
- Shin SH, "The optimal immunosuppressive therapy for aplastic anemia", Int J Hematol, 97, pp.564~572 (2013)
- Maciejewski J, "Fas antigen expression on CD34+ human marrow cells is induced by interferon gamma and tumor necrosis factor alpha and potentiates cytokine-mediated hematopoietic suppression in vitro", Blood, 85, pp.3183~3190 (1995)
- Risitano AM, "In-vivo dominant immune responses in aplastic anaemia: molecular tracking of putatively pathogenetic T-cell clones by TCR beta-CDR3 sequencing", Lancet, 364, pp.355~364 (2004)
- Young NS, "Current concepts in the pathophysiology and treatment of aplastic anemia", Blood, 108, pp.2509~2519 (2006)
- "Incidence of aplastic anemia: the relevance of diagnostic criteria: by the International Agranulocytosis and Aplastic Anemia Study", Blood, 70, pp.1718~1721 (1987)
- Mary JY, "Epidemiology of aplastic anemia in France: a prospective multicentric study: the French Cooperative Group for Epidemiological Study of Aplastic Anemia", Blood, 75, pp.1646~1653 (1990)
- Issaragrisil S, "Epidemiology of aplastic anemia in Thailand:Thai Aplastic Anemia Study Group", Int J Hematol, 70, pp.137~140 (1999)
- Yang C, "Incidence survey of aplastic anemia in China", Chin Med Sci J, 6, pp.203~207 (1991)
- Yong AS, "Epidemiology of aplastic anaemia in the state of Sabah, Malaysia", Med J Malaysia, 53, pp.59~62 (1998)
- Marsh JC, "Guidelines for the diagnosis and management of aplastic anaemia", Br J Haematol, 147, pp.43~70 (2009)
- Rozman C, "Criteria for severe aplastic anaemia", Lancet, 2, pp.955~957 (1987)
- Scheinberg P, "Aplastic anemia: therapeutic updates in immunosuppression and transplantation", Hematology Am Soc Hematol Educ Program, 2012, pp.292~300 (2012)
- Speck B, "Treatment of aplastic anaemia by antilymphocyte globulin with and without allogeneic bone-marrow infusions", Lancet, 2, pp.1145~1148 (1977)
- Speck B, "Treatment of severe aplastic anaemia with antilymphocyte globulin or bone-marrow transplantation", Br Med J (Clin Res Ed), 282, pp.860~863 (1981)
- Champlin R, "Antithymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial", N Engl J Med, 308, pp.113~118 (1983)
- Gluckman E, "Results of immunosuppression in 170 cases of severe aplastic anaemia: report of the European Group of Bone Marrow Transplant (EGBMT)", Br J Haematol, 51, pp.541~550 (1982)
- de Planque MM, "Longterm follow-up of severe aplastic anaemia patients treated with antithymocyte globulin: Severe Aplastic Anaemia Working Party of the European Cooperative Group for Bone Marrow Transplantation (EBMT)", Br J Haematol, 73, pp.121~126 (1989)
- Young N, "A multicenter trial of antithymocyte globulin in aplastic anemia and related diseases", Blood, 72, pp.1861~1869 (1988)
- Stryckmans PA, "Cyclosporine in refractory severe aplastic anemia", N Engl J Med, 310, pp.655~656 (1984)
- Frickhofen N, "Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine:the German Aplastic Anemia Study Group", N Engl J Med, 324, pp.1297~1304 (1991)
- Frickhofen N, "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, 101, pp.1236~1242 (2003)
- Marsh J, "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, pp.2191~2195 (1999)
- Rosenfeld S, "Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome", JAMA, 289, pp.1130~1135 (2003)
- Osugi Y, "Antithymocyte globulin and cyclosporine for treatment of 44 children with hepatitis associated aplastic anemia", Haematologica, 92, pp.1687~1690 (2007)
- Scheinberg P, "Predicting response to immunosuppressive therapy and survival in severe aplastic anaemia", Br J Haematol, 144, pp.206~216 (2009)
- Maciejewski JP, "Increased frequency of HLA-DR2 in patients with paroxysmal nocturnal hemoglobinuria and the PNH", aplastic anemia syndrome, 2001, pp.98, 3513~3519
- Sugimori C, "Minor population of CD55-CD59- blood cells predicts response to immunosuppressive therapy and prognosis in patients with aplastic anemia", Blood, 107, pp.1308~1314 (2006)
- Kim SY, "The characteristics and clinical outcome of adult patients with aplastic anemia and abnormal cytogenetics at diagnosis", Genes Chromosomes Cancer, 49, pp.844~850 (2010)
- Saracco P, "Cyclosporin A response and dependence in children with acquired aplastic anaemia: a multicentre retrospective study with long-term observation follow-up", Br J Haematol, 140, pp.197~205 (2008)
- Risitano AM, "Immunosuppressive therapies in the management of immune-mediated marrow failures in adults: where we stand and where we are going", Br J Haematol, 152, pp.127~140 (2011)
- Scheinberg P, "Association of telomere length of peripheral blood leukocytes with hematopoietic relapse, malignant transformation, and survival in severe aplastic anemia", JAMA, 304, pp.1358~1364 (2010)
- Sakaguchi H, "Peripheral blood lymphocyte telomere length as a predictor of response to immunosuppressive therapy in childhood aplastic anemia", Haematologica, 99, pp.1312~1316 (2014)
- Scheinberg P, "Horse versus rabbit antithymocyte globulin in acquired aplastic anemia", N Engl J Med, 365, pp.430~438 (2011)
- Atta EH, "Comparison between horse and rabbit antithymocyte globulin as first-line treatment for patients with severe aplastic anemia: a single-center retrospective study", Ann Hematol, 89, pp.851~859 (2010)
- Afable MG, 2nd, "Efficacy of rabbit anti-thymocyte globulin in severe aplastic anemia", Haematologica, 96, pp.1269~1275 (2011)
- Shin SH, "The efficacy of rabbit antithymocyte globulin with cyclosporine in comparison to horse antithymocyte globulin as a first-line treatment in adult patients with severe aplastic anemia:a single-center retrospective study", Ann Hematol, 92, pp.817~824 (2013)
- Marsh JC, "Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party", Blood, 119, pp.5391~5396 (2012)
- Jeong DC, "Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children", Haematologica, 99, pp.664~671 (2014)
- Scheinberg P, "Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study", Haematologica, 94, pp.348~354 (2009)
- Scheinberg P, "Treatment of severe aplastic anaemia with combined immunosuppression:anti-thymocyte globulin, ciclosporin and mycophenolate mofetil", Br J Haematol, 133, pp.606~611 (2006)
- Teramura M, "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, 110, pp.1756~1761 (2007)
- Tichelli A, "A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF:a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation", Blood, 117, pp.4434~4441 (2011)
- Kuter DJ, "The biology of thrombopoietin and thrombopoietin receptor agonists", Int J Hematol, 98, pp.10~23 (2013)
- Geddis AE, "Congenital amegakaryocytic thrombocytopenia", Pediatr Blood Cancer, 57, pp.199~203 (2011)
- Olnes MJ, "Eltrombopag and improved hematopoiesis in refractory aplastic anemia", N Engl J Med, 367, pp.11~19 (2012)
- Desmond R, "Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug", Blood, 123, pp.1818~1825 (2014)
- Kosaka Y, "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, 111, pp.1054~1059 (2008)
- Di Bona E, "Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy:Gruppo Italiano Trapianto di Midollo Osseo (GITMO)", Br J Haematol, 107, pp.330~334 (1999)
- Scheinberg P, "Retreatment with rabbit anti-thymocyte globulin and ciclosporin for patients with relapsed or refractory severe aplastic anaemia", Br J Haematol, 133, pp.622~627 (2006)
- Scheinberg P, "Horse antithymocyte globulin as salvage therapy after rabbit antithymocyte globulin for severe aplastic anemia", Am J Hematol, 89, pp.467~469 (2014)
- Risitano AM, "Alemtuzumab is safe and effective as immunosuppressive treatment for aplastic anaemia and single-lineage marrow failure: a pilot study and a survey from the EBMT WPSAA", Br J Haematol, 148, pp.791~796 (2010)
- Scheinberg P, "Activity of alemtuzumab monotherapy in treatment-naive, relapsed, and refractory severe acquired aplastic anemia", Blood, 119, pp.345~354 (2012)
- Brodsky RA, "High-dose cyclophosphamide for severe aplastic anemia: long-term follow-up", Blood, 115, pp.2136~2141 (2010)
- Tisdale JF, "High-dose cyclophosphamide in severe aplastic anaemia: a randomised trial", Lancet, 356, pp.1554~1559 (2000)
- McCann SR, "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 Transplant, 13, pp.233~237 (1994)
- Champlin RE, "Graft failure following bone marrow transplantation for severe aplastic anemia: risk factors and treatment results", Blood, 73, pp.606~613 (1989)
- Gluckman E, "Bone marrow transplantation for severe aplastic anemia: inf luence of conditioning and graft-versus-host disease prophylaxis regimens on outcome", Blood, 79, pp.269~275 (1992)
- Deeg HJ, "Malignancies after marrow transplantation for aplastic anemia and fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients", Blood, 87, pp.386~392 (1996)
- Gale RP, "Prevention of graft rejection following bone marrow transplantation", Blood, 57, pp.9~12 (1981)
- Storb R, "Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia", Blood, 84, pp.941~949 (1994)
- Champlin RE, "Bone marrow transplantation for severe aplastic anemia: a randomized controlled study of conditioning regimens", Blood, 109, pp.4582~4585 (2007)
- Ades L, "Long-term outcome after bone marrow transplantation for severe aplastic anemia", Blood, 103, pp.2490~2497 (2004)
- Kahl C, "Cyclophosphamide and antithymocyte globulin as a conditioning regimen for allogeneic marrow transplantation in patients with aplastic anaemia: a long-term follow-up", Br J Haematol, 130, pp.747~751 (2005)
- Schrezenmeier H, "Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLAmatched sibling donor transplants for young patients with severe acquired aplastic anemia", Blood, 110, pp.1397~1400 (2007)
- Locatelli F, "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 an HLA-identical sibling: results of a GITMO", EBMT randomized trial, 2000, pp.96, 1690~1697
- Gupta V, "Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors", Haematologica, 95, pp.2119~2125 (2010)
- Maury S, "Improved outcome of patients older than 30 years receiving HLA-identical sibling hematopoietic stem cell transplantation for severe acquired aplastic anemia using f ludarabine-based conditioning: a comparison with conventional conditioning regimen", Haematologica, 94, pp.1312~1315 (2009)
- Shin SH, "Influence of recipient’s age on the outcome of HLA-matched sibling transplants in adult patients with severe aplastic anemia who conditioned with f ludarabine-based regimen", Paper presented at: 54th ASH Annual Meeting and Exposition, pp.1921~1922 (2012)
- Passweg JR, "Bone marrow transplants from mismatched related and unrelated donors for severe aplastic anemia", Bone Marrow Transplant, 37, pp.641~649 (2006)
- Bacigalupo A, "Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party", Bone Marrow Transplant, 36, pp.947~950 (2005)
- Bacigalupo A, "Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA Working Party", Haematologica, 95, pp.976~982 (2010)
- Deeg HJ, "Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy", Blood, 108, pp.1485~1491 (2006)
- Maury S, "Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient", Haematologica, 92, pp.589~596 (2007)
- Viollier R, "Recent improvement in outcome of unrelated donor transplantation for aplastic anemia", Bone Marrow Transplant, 41, pp.45~50 (2008)
- Kim SY, "Unrelated donor bone marrow transplants for severe aplastic anemia with conditioning using total body irradiation and cyclophosphamide", Biol Blood Marrow Transplant, 13, pp.863~870 (2007)
- Lee JW, "The outcome of unrelated hematopoietic stem cell transplants with total body irradiation (800 cGy) and cyclophosphamide (120 mg", kg)in adult patients with acquired severe aplastic anemia, 2011, pp.17, 101~108
- Yakoub-Agha I, "Allogeneic marrow stem-cell transplantation from human leukocyte antigen-identical siblings versus human leukocyte antigen-allelic-matched unrelated donors (10", 10) in patients with standard-risk hematologic malignancy: a prospective study from the French Society of Bone Marrow Transplantation and Cell Therapy, 2006, pp.24, 5695~5702
- Yagasaki H, "Tacrolimus", Methotrexate versus cyclosporine, Biol Blood Marrow Transplant, pp.2009, 15[DOI]
- Baldomero H, "The EBMT activity survey 2009: trends over the past 5 years", Bone Marrow Transplant, 46, pp.485~501 (2011)
- Lee SE, "In vivo T-cell depletion with low-dose antithymocyte goblulin to reduce acute GVHD in urenlated donor stem cell transplant for patients with severe aplastic anemia", Paper presented at:18th EHA 2013 Annual Meeting, pp.380~381 (2013)
- Wang Y, "Long-term follow-up of haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for the treatment of leukemia: nine years of experience at a single center", Cancer, 119, pp.978~985 (2013)
- Aversa F, "Full haplotype-mismatched hematopoietic stem-cell transplantation: a phase II study in patients with acute leukemia at high risk of relapse", J Clin Oncol, 23, pp.3447~3454 (2005)
- Wagner JL, "Bone marrow transplantation for severe aplastic anemia from genotypically HLA-nonidentical relatives: an update of the Seattle experience", Transplantation, 61, pp.54~61 (1996)
- Tzeng CH, "CY", TBI-800 as a pretransplant regimen for allogeneic bone marrow transplantation for severe aplastic anemia using HLA-haploidentical family donors, 1996, pp.18, 273~277
- Xu LP, "A novel protocol for haploidentical hematopoietic SCT without in vitro T-cell depletion in the treatment of severe acquired aplastic anemia", Bone Marrow Transplant, 47, pp.1507~1512 (2012)
- Dezern AE, "Post-transplantation cyclophosphamide for GVHD prophylaxis in severe aplastic anemia", Bone Marrow Transplant, 46, pp.1012~1013 (2011)
- Im HJ, "Excellent outcome of haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia", Biol Blood Marrow Transplant, 19, pp.754~759 (2013)
- Takatoku M, "Retrospective nationwide survey of Japanese patients with transfusion-dependent MDS and aplastic anemia highlights the negative impact of iron overload on morbidity", mortality, 2007, pp.78, 487~494
- Kushner JP, "Secondary iron overload", Hematology Am Soc Hematol Educ Program, 2001, pp.47~61 (2001)
- Porter J, "Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study", Eur J Haematol, 80, pp.168~176 (2008)
- Cappellini MD, "A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia", Blood, 107, pp.3455~3462 (2006)
- Lee JW, "Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial", Blood, 116, pp.2448~2454 (2010)
- Lee JW, "Hematologic responses in patients with aplastic anemia treated with deferasirox:a post hoc analysis from the EPIC study", Haematologica, 98, pp.1045~1048 (2013)
- Lee SE, "Improvement in hematopoiesis after iron chelation therapy with deferasirox in patients with aplastic anemia", Acta Haematol, 129, pp.72~77 (2013)
- Lee JW, "Iron chelation therapy with deferasirox results in recovery of hematopoiesis in a child with aplastic anemia", Pediatr Hematol Oncol, 28, pp.718~720 (2011)
- Koh KN, "Restoration of hematopoiesis after iron chelation therapy with deferasirox in 2 children with severe aplastic anemia", J Pediatr Hematol Oncol, 32, pp.611~614 (2010)
