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DOI: 10.1055/s-0033-1337970
Stem Cell Transplantation
StammzelltransplantationPublikationsverlauf
Publikationsdatum:
22. Mai 2013 (online)
Abstract
Stem cell transplantation in pediatric patients with leukemia has been a matter of debate since the first successful transplantation in a child with aplastic anemia in Germany in 1975. Since then, there has been a long way to implement fully stem cell transplantation strategies into the treatment studies organized within BFM Groups for first line or second line treatment of acute and chronic leukemias and myelodysplastic syndrome. The role of different risk groups, different donors, stem cell sources and alternative approaches as haploidentical transplantation as well have been studied as the impact of chimerism and minimal residual disease. Finally, for all leukemias and myelosdysplastic syndromes integrated treatment pathways have been developed, which integrate stem cell transplantation on an evidence base into the treatment of the patient.
Zusammenfassung
Die Rolle der Stammzelltransplantation für die Behandlung von Kindern mit Leukämien ist Gegenstand intensiver Debatten, seit 1975 in Deutschland das erste Kind mit einer aplastischen Anämie erfolgreich mit einer Knochenmarktransplantation behandelt wurde. Seitdem ist ein langer Weg zurückgelegt worden bis zu vollständigen Integration der Stammzelltransplantation in die Behandlungsstudien, die von BFM-Gruppen für die Erst- oder Zweitlinien-therapie von Kindern mit akuten und chronischen Leukämien und myelodysplastischen Syndromen organisiert werden. In zahlreichen Studien wurden die Rolle der Risikogruppen, der möglichen verschiedenen Spender, der verschiedenen Stammzellquellen und alternativer Ansätze wie der haploidentischen Transplantation ebenso untersucht wie der Einfluss von Chimärismus und minimaler Restkrankheit auf das Ergebnis der Transplantation. Heute existieren für alle Leukämien und myelodysplatischen Syndrome im Kindesalter integrierte Behandlungspfade, die auf einer Evidenz-basierten Grundlage die Stammzelltransplantion in die Behandlung des Patienten integrieren.
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References
- 1 Al-Kasim FA, Thornley I, Rolland M et al. Single-centre experience with allogeneic bone marrow transplantation for acute lymphoblastic leukemia in childhood: similar survival after matched-related and matched-unrelated donor transplants. Br J Haematol 2002; 116: 483-490
- 2 Aridolina J, Neudorf S, Corey S. How I treat childhood CML. Blood 2012; 119: 1821-1830
- 3 Bader P, Hoelle W, Handgretinger R et al. Mixed hematopoetic chimerism after allogeneic bone marrow transplantation: the impact of quantitative PCR analysis for prediction of relapse and graft rejection in children. Bone Marrow Transplant 1997; 19: 697-702
- 4 Bader P, Stoll K, Huber S et al. Characterization of lineage-specific chimaerism in patients with acute leukemia and myelodysplastic syndrome after allogeneic stem cell transplantation before and after relapse. Brit J Haematol 2000; 108: 761-768
- 5 Bader P, Kreyenberg H, Hoelle W et al. Increasing mixed chimerism is an important prognostic factor for unfavorable outcome in children with acute lymphoblastic leukemia after allogeneic stem-cell transplantation: possible role for pre-emptive immunotherapy?. J Clin Oncol 2004; 22: 1696-1706
- 6 Bader P, Kreyenberg H, Henze G et al. Predictive value of MRD monitoring after allogeneic SCT in relapsed childhood acute lymphoblastic leukemia: analysis of the ALL-REZ BFM Group. Bone Marrow Transplant 2009; 43 (Suppl.1): 40
- 7 Barfield RC, Otto M, Houston J et al. A one-step large-scale method for T- and B-cell depletion of mobilized PBSC for allogeneic transplantation. Cytotherapy 2004; 6: 1-6
- 8 Barrios M, Jimenez-Velasco A, Roman-Gomez J et al. Chimerism status is a useful predictor of relapse after allogeneic stem cell transplantation for acute leukemia. Haematologica 2003; 88: 801-810
- 9 Benesch M, Urban C, Sykora KW et al. Transplantation of highly purified CD34+ progenitor cells from alternative donors in children with refractory severe aplastic anaemia. Br J Haematol 2004; 125: 58-63
- 10 Borgmann A, von Stackelberg A, Hartmann R et al. Unrelated donor stem cell transplantation compared with chemotherapy for children with acute lymphoblastic leukemia in second remission: a matched-pair analysis. Blood 2003; 101: 3835-3839
- 11 Chaleff S, Otto M, Barfield RC et al. A large-scale method for the selective depletion of alphabeta T lymphocytes from PBSC for allogeneic transplantation. Cytotherapy 2007; 9: 746-754
- 12 Creutzig U, van den Heuvel-Elbrink M, Gibson B et al. Diagnosis and management of acute myelogenic leukemia in children and adolescents: recommendations from an international expert panel, on behalf of the AML Committee of the international BFM Study Group. Blood 2012; Prepublished online August 9, 2012 DOI: 10.1182/blood-2012-03-362608.
- 13 Dopfer R, Henze G, Bender-Götze C et al. Allogeneic bone marrow transplantation for childhood acute lymphoblastic leukemia in second remission after intensive primary and relapse therapy according to the BFM and CoALL-protocols: Results of the German Cooperative Study. Blood 1991; 78: 2780-2784
- 14 Dufort G, Pisano S, Incoronato A et al. Feasibility and outcome of haploidentical SCT in pediatric high-risk hematologic malignancies and Fanconi anemia in Uruguay. Bone Marrow Transplant 2012; 47: 663-668
- 15 Eapen M, Rubinstein P, Zhang MJ et al. Comparable long-term survival after unrelated and HLA-matched sibling donor hematopoietic stem cell transplantations for acute leukemia in children younger than 18 months. J Clin Oncol 2006; 24: 145-151
- 16 Fernadez-Aviles F, Urbano-Ispizua A, Aymerich M et al. Serial quantification of lymphoid and myeloid mixed chimerism using multiplex PCR amplification of short tandem repeat markers predicts graft rejection and relapse, respectively, after allogeneic transplantation of CD 34+ selected cells from peripheral blood. Leukemia 2003; 17: 613-620
- 17 Formankova R, Sedlacek P, Krskova L et al. Chimerism-directed adoptive immunotherapy in prevention and treatment of post-transplant relapse of leukemia in childhood. Heamatologica 2003; 88: 117-118
- 18 Gustafsson A, Remberger M, Winiarski J et al. Unrelated bone marrow transplantation in children: outcome and a comparison with sibling donor grafting. Bone Marrow Transplant 2000; 25: 1059-1065
- 19 Handgretinger R, Klingebiel T, Lang P et al. Megadose transplantation of purified peripheral blood CD34(+) progenitor cells from HLA-mismatched parental donors in children. Bone Marrow Transplant 2001; 27: 777-783
- 20 Handgretinger R, Chen X, Pfeiffer M et al. Feasibility and outcome of reduced-intensity conditioning in haploidentical transplantation. Ann N Y Acad Sci 2007; 1106: 279-289 Epub@2007 Apr 18:279–289
- 21 Hasle H, Niemeyer CM, Chessels JM et al. A pediatric approach to the WHO classification of myelodysplastic and myeloproliferatie diseases. Leukemia 2003; 17: 277-282
- 22 Henslee-Downey PJ, Abhyankar SH, Parrish RS et al. Use of partially mismatched related donors extends access to allogeneic marrow transplant. Blood 1997; 89: 3864-3872
- 23 Kardos G, Baumann I, Passmore SJ et al. Refractory anemia in childhood: A retrospective analysis of 67 patients with particular reference to monosomy 7. Blood 2003; 102: 1997-2003
- 24 Kaspers G, Creutzig U. Pediatric acute mayeloic leukemia:international progress and future directions. Leukemia 2005; 19: 2025-2029
- 25 Knechtli C, Goulden N, Hancock J et al. Minimal residual disease status as a predictor of relapse after allogeneic bone marrow transplantation for children with acute lymphoblastic leukemia. Br J Haematol 1998; 102: 860-871
- 26 Klingebiel T, Cornish J, Labopin M et al. Results and factors influencing outcome after fully haploidentical hematopoietic stem cell transplantation in children with very high-risk acute lymphoblastic leukemia: impact of center size: an analysis on behalf of the Acute Leukemia and Pediatric Disease Working Parties of the European Blood and Marrow Transplant group. Blood 2010; 115: 3437-3446
- 27 Klusmann J-H, Reinhardt D, Zimmermann M et al. The role of matched sibling donor allogeneic stem cell transplantation in pediatric high-risk acute myelogenic leukemis: results from the AML-BFM 98 study. Haematologica 2012; 97: 21-91
- 28 Lang P, Handgretinger R, Niethammer D et al. Transplantation of highly purified CD34+ progenitor cells from unrelated donors in pediatric leukemia. Blood 2003; 101: 1630-1636
- 29 Leung W, Lyengar R, Turner V et al. Determinants of antileukemia effects of allogeneic NK cells. J Immunol 2004; 172: 644-650
- 30 Leung W. Use of NK Cell activity in cure by transplant. Br J Haematol 2011; 155: 14-29
- 31 Leung W, Campana D, Yang J et al. High success rate of hematopoietic cell transplantation regardless of donor source in children with very high-risk leukemia. Blood 2011; 118: 223-230
- 32 Luznik L, Fuchs EJ. High-dose, post-transplantation cyclophosphamide to promote graft-host tolerance after allogeneic hematopoietic stem cell transplantation. Immunol Res 2010; 47: 65-77
- 33 Luznik L, O’Donnell PV, Symons HJ et al. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant 2008; 14: 641-650
- 34 Locatelli F, Zecca M, Messina C et al. Improvement over time in outcome for children with acute lymphoblastic leukemia in second remission given hematopoietic stem cell transplantation from unrelated donors. Leukemia 2002; 16: 2228-2237
- 35 Locatelli F, Nollke P, Zecca M et al. European Working Group on Childhood MDS; European Blood and Marrow transplantation group. Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): Results of the EWOG-MDS/EBMT trial. Blood 2005; 105: 410-419
- 36 Niemeyer C, Dufffner U, Bender-Gotze C et al. AML-type intensive chemotherapy prior to stem cell transplantation (SCT) does not improve survival in children and adolescents with primary myelodysplastic syndromes (MDS). Blood 200 96: 521a
- 37 Niewerth D, Creutzig U, Bierings M et al. A review on allogeneic stem cell transplantation for newly diagnosed pediatric acute myeloid leukemia. Blood 2010; 116: 2205-2214
- 38 Oevermann L, Handgretinger R. New strategies for haploidentical transplantation. Pediatr Res 2012; 71: 418-426
- 39 Palma J, Salas L, Carrion F et al. Haploidentical stem cell transplantation for children with high-risk leukemia. Pediatr Blood Cancer 2012; 59: 895-901
- 40 Peters C, Schrauder A, Stackelberg A et al. Outcome of matched sibling donor SCT is equivalent to unrelated well matched donors HSCT. A report from the prospective international multicentre trial ALL-SCT-BFM 2003. Bone Marrow Transplant 2012; 47: S2 (abstr.)
- 41 Pfeiffer MM, Feuchtinger T, Teltschik HM et al. Reconstitution of natural killer cell receptors influences natural killer activity and relapse rate after haploidentical transplantation of T- and B-cell depleted grafts in children. Haematologica 2010; 95: 1381-1388
- 42 Ravindranath Y, Yeager A, Chang M et al. Autologous bone marrow transplantation versus intensive consolidation chemotherapy for acute myeloid leukemia in childhood. Pediatric Oncology Group. N Engl J Med 1996; 334: 1428-1434
- 43 Remberger M, Beelen DW, Fauser A et al. Increased risk of extensive chronic graft-versus-host disease after allogeneic peripheral blood stem cell transplantation using unrelated donors. Blood 2005; 105: 548-551
- 44 Sander A, Zimmermann M, Dworzak M et al. Consequent and intensified relapse therapy improved survival in pediatric AML: results of relapse treatment in 379 patients of three consecutive AML-BFM trials. Leukemia 2010; 24: 1422-1428
- 45 Möricke A, Zimmermann M, Reiter A et al. Long-term results of five consecutive trials in childhood acute lymphoblastic leukemia performed by the ALL-BFM study group from 1981 to 2000. Leukemia. 2010 24. 265-284
- 46 Schumm M, Lang P, Taylor G et al. Isolation of highly purified autologous and allogeneic peripheral CD34+cells using the CliniMACS device. J Hematother 1999; 8: 209-218
- 47 Stevens R, Hann I, Wheatly K et al. Marked improvement in outcome with chemotherapy alone in pediatric acute myeloid leukemia: results of the United Kingdom Medical Research Council’s 10th AML trial. MRC Childhood Leukemia Working Party. Brit J Haematol 1998; 101: 130-140
- 48 Suttorp M, Eckardt L, Tauer J et al. Management of chronic myeloid leukemia in childhood. Curr Hematol Malig Rep 2012; 7: 116-124
- 49 Woods W, Neudorf S, Gold S et al. A comparison of allogeneic bone marrow transplantation, autologous bone marrow transplantation, and aggressive chemotherapy in children with acute myeloid leukemia in remission. Blood 2001; 97: 56-62