Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?-A multicenter EBMT-PDWP study

. 2020 Aug ; 55 (8) : 1540-1551. [epub] 20200317

Jazyk angličtina Země Velká Británie, Anglie Médium print-electronic

Typ dokumentu časopisecké články, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/pmid32203263
Odkazy

PubMed 32203263
PubMed Central PMC8376634
DOI 10.1038/s41409-020-0854-0
PII: 10.1038/s41409-020-0854-0
Knihovny.cz E-zdroje

Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2-18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective "real-world-practice" study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.

BMT Department Russian Children's Hospital Moscow Russia

BMT Unit Department of Pediatric Hematology and Oncology Comenius University Medical School Limbová Bratislava Slovak Republic

BMT Unit University Medical Centre Utrecht Pediatrics Utrecht The Netherlands

Bone Marrow Transplant Program Instituto Portugues Oncologia Lisbon Portugal

Bone Marrow Transplantation Service Portuguese Institute of Oncology Porto Portugal

Bone Marrow Transplantation Unit Pediatric Department of Milano Bicocca University Fondazione Monza e Brianza per il Bambino e la sua Mamma Foundation Monza Italy

Center of Pediatric Oncology and Hematology Vilnius University Vilnius Lithuania

Centre Pierre et Marie Curie Service Hématologie Greffe de Moelle Alger Algeria

Department for Pediatric Hematology and Hemopoietic Stem Cell Transplantation Central Hospital of Southern Pest National Institute of Hematology and Infectious Diseases United St Istvan and St László Hospital Budapest Hungary

Department of Bone Marrow Transplantation Hadassah Hebrew University Medical Center Jerusalem Israel

Department of Bone Marrow Transplantation Oncology and Hematology Cape of Hope Medical Center Wroclaw Medical University Wroclaw Poland

Department of Haematology Internal Clinic University Hospital Centre Zagreb Croatia

Department of Hematology Child Welfare Center Borsod County Teaching Hospital Miskolc Hungary

Department of Pediatric Hemato Immunology Hôpital Robert Debré and Université de Paris Paris France

Department of Pediatric Hematology and BMT IHOP and Claude Bernard University Lyon France

Department of Pediatric Hematology and Oncology and Research Unit EA 3279 Timone Enfants Hospital AP HM and Aix Marseille University Marseille France

Department of Pediatric Hematology and Oncology Cliniques Universitaires Saint Luc Brussels Belgium

Department of Pediatric Hematology and Oncology Collegium Medicum Nicolaus Copernicus University Torun Bydgoszcz Poland

Department of Pediatric Hematology and Oncology Oslo University Hospital Oslo Norway

Department of Pediatric Hematology and Oncology University Hospital Motol Prague Czech Republic

Department of Pediatric Hematology and Stem Cell Transplantation Unit Medicalpark Antalya Hastanesi Antalya Turkey

Department of Pediatric Hematology Oncology and Stem Cell Transplantation University of Regensburg Regensburg Germany

Department of Pediatric Hematology Oncology King Faisal Specialist Hospital and Research Center Riyadh Saudi Arabia

Department of Pediatric Hematology Oncology School of Medicine Akdeniz University Antalya Turkey

Department of Pediatric Oncology Hematology and Transplantology University of Medical Sciences Poznan Poland

Department of Pediatrics Copenhagen University Hospital Rigshospitalet Copenhagen Denmark

Department of Pediatrics Leiden University Medical Center Leiden The Netherlands

Department of Pediatrics Onco Hematology Unit Geneva University Hospital Geneva University Geneva Switzerland

Dipartimento di Onco Ematologia e Terapia Cellulare e Genica IRCCS Ospedale Pediatrico Bambino Gesù Rome Italy

Dipartimento Materno Infantile e Scienze Urologiche Sapienza University of Rome Rome Italy

Division for Stem Cell Transplantation and Immunology Department for Children and Adolescents University Hospital Goethe University Frankfurt Frankfurt Germany

Division of Pediatrics CLINTEC Karolinska Institutet and Pediatric Hematology Immunology and HCT Astrid Lindgren Children's Hospital Karolinska University Hospital Stockholm Sweden

Division of Stem Cell Transplantation and Regenerative Medicine Department of Pediatrics School of Medicine Stanford University Stanford CA USA

Division of Stem Cell Transplantation Children's Research Center University Children's Hospital Zurich Switzerland

EBMT Paediatric Diseases Working Party Paris France

EBMT Paris Study Office Paris France

Great Ormond Street Hospital for Children NHS Foundation Trust London UK

Hematology Oncology and BMT Research Shariati Hospital Tehran Iran

Hematology Oncology Unit Lalla Seràgnoli Department of Pediatrics University of Bologna Bologna Italy

Hospital for Children and Adolescents University of Helsinki Helsinki Finland

Paediatric Onco Haematology City of Science and Health of Turino Regina Margherita Children's Hospital Torino Italy

Pediatric Cell Therapy Research Center Tehran University of Medical Sciences Tehran Iran

Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology Haematology and Transplantation Saint Petersburg State Medical 1 P Pavlov University Saint Petersburg Russia

Republic Clinical Research Centre for Pediatric Oncology and Hematology Minsk Belarus

Servicio de Hematologia y Oncologia Pediátricas Hospital Universitario Vall d'Hebron Barcelona Spain

St Anna Children's Hospital Department of Pediatrics Medical University of Vienna Vienna Austria

Stem Cell Transplant Unit Aghia Sophia Children's Hospital Thivon and Papadiamantopoulou Athens Greece

Tartu University Hospital Tartu Estonia

University Hospitals Bristol NHS Foundation Trust Bristol UK

Erratum v

PubMed

Zobrazit více v PubMed

Peters C, Schrappe M, von Stackelberg A, Schrauder A, Bader P, Ebell W, et al. Stem-cell transplantation in children with acute lymphoblastic leukemia: a prospective international multicenter trial comparing sibling donors with matched unrelated donors—The ALL-SCT-BFM-2003 Trial. J Clin Oncol Off J Am Soc Clin Oncol. 2015;33:1265–74. PubMed

Bunin N, Aplenc R, Kamani N, Shaw K, Cnaan A, Simms S. Randomized trial of busulfan vs total body irradiation containing conditioning regimens for children with acute lymphoblastic leukemia: a Pediatric Blood and Marrow Transplant Consortium study. Bone Marrow Transpl. 2003;32:543–8. PubMed

Marks DI, Forman SJ, Blume KG, Perez WS, Weisdorf DJ, Keating A, et al. A comparison of cyclophosphamide and total body irradiation with etoposide and total body irradiation as conditioning regimens for patients undergoing sibling allografting for acute lymphoblastic leukemia in first or second complete remission. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2006;12:438–53. PubMed

Litzow MR, Perez WS, Klein JP, Bolwell BJ, Camitta B, Copelan EA, et al. Comparison of outcome following allogeneic bone marrow transplantation with cyclophosphamide-total body irradiation versus busulphan-cyclophosphamide conditioning regimens for acute myelogenous leukaemia in first remission. Br J Haematol. 2002;119:1115–24. PubMed

Jamieson CH, Amylon MD, Wong RM, Blume KG. Allogeneic hematopoietic cell transplantation for patients with high-risk acute lymphoblastic leukemia in first or second complete remission using fractionated total-body irradiation and high-dose etoposide: a 15-year experience. Exp Hematol. 2003;31:981–6. PubMed

Dopfer R, Henze G, Bender-Gotze C, Ebell W, Ehninger G, Friedrich W, 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;78:2780–4. PubMed

Bader P, Kreyenberg H, Henze GHR, Eckert C, Reising M, Willasch A, et al. Prognostic value of minimal residual disease quantification before allogeneic stem-cell transplantation in relapsed childhood acute lymphoblastic leukemia: The ALL-REZ BFM Study Group. J Clin Oncol. 2009;27:377–84. PubMed

Zhang MJ, Davies SM, Camitta BM, Logan B, Tiedemann K, Eapen M, et al. Comparison of outcomes after HLA-matched sibling and unrelated donor transplantation for children with high-risk acute lymphoblastic leukemia. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2012;18:1204–10. PubMed PMC

Pulsipher MA, Langholz B, Wall DA, Schultz KR, Bunin N, Carroll WL, et al. The addition of sirolimus to tacrolimus/methotrexate GVHD prophylaxis in children with ALL: a phase 3 Children’s Oncology Group/Pediatric Blood and Marrow Transplant Consortium trial. Blood. 2014;123:2017–25. PubMed PMC

Hahn T, Wall D, Camitta B, Davies S, Dillon H, Gaynon P, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute lymphoblastic leukemia in children: an evidence-based review. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2005;11:823–61. PubMed

Oliansky DM, Camitta B, Gaynon P, Nieder ML, Parsons SK, Pulsipher MA, et al. Role of cytotoxic therapy with hematopoietic stem cell transplantation in the treatment of pediatric acute lymphoblastic leukemia: update of the 2005 evidence-based review. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2012;18:505–22. PubMed

Simonin M, Dalissier A, Labopin M, Willasch A, Zecca M, Mouhab A, et al. More chronic GvHD and non-relapse mortality after peripheral blood stem cell compared with bone marrow in hematopoietic transplantation for paediatric acute lymphoblastic leukemia: a retrospective study on behalf of the EBMT Paediatric Diseases Working Party. Bone Marrow Transpl. 2017;52:1071–3. PubMed

Tracey J, Zhang MJ, Thiel E, Sobocinski KA, Eapen M. Transplantation conditioning regimens and outcomes after allogeneic hematopoietic cell transplantation in children and adolescents with acute lymphoblastic leukemia. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2013;19:255–9. PubMed PMC

Dalle JH, Balduzzi A, Bader P, Lankester A, Yaniv I, Wachowiak J, et al. Allogeneic stem cell transplantation from HLA-mismatched donors for pediatric patients with acute lymphoblastic leukemia treated according to the 2003 BFM and 2007 international BFM studies: impact of disease risk on outcomes. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2018;24:1848–55. PubMed

Wachowiak J, Bettoni C, Lange A, Malicki J, Kaczmarek-Kanold M, Gluszak B, et al. Can busulfan replace fractionated total body irradiation as conditioning regimen for allogeneic bone marrow transplantation in children with acute lymphoblastic leukemia. Acta Haematol Pol. 1995;26:377–84. PubMed

Bader P, Salzmann-Manrique E, Balduzzi A, Dalle JH, Woolfrey AE, Bar M, et al. More precisely defining risk peri-HCT in pediatric ALL: pre- vs post-MRD measures, serial positivity, and risk modeling. Blood Adv. 2019;3:3393–405. PubMed PMC

Friend BD, Bailey-Olson M, Melton A, Shimano KA, Kharbanda S, Higham C, et al. The impact of total body irradiation-based regimens on outcomes in children and young adults with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation. Pediatr Blood Cancer. 2020;67:e28079. PubMed

Socie G, Curtis RE, Deeg HJ, Sobocinski KA, Filipovich AH, Travis LB, et al. New malignant diseases after allogeneic marrow transplantation for childhood acute leukemia. J Clin Oncol Off J Am Soc Clin Oncol. 2000;18:348–57. PubMed

Baker KS, Bresters D, Sande JE. The burden of cure: long-term side effects following hematopoietic stem cell transplantation (HSCT) in children. Pediatr Clin North Am. 2010;57:323–42. PubMed

Bresters D, Emons JA, Nuri N, Ball LM, Kollen WJ, Hannema SE, et al. Ovarian insufficiency and pubertal development after hematopoietic stem cell transplantation in childhood. Pediatr Blood Cancer. 2014;61:2048–53. PubMed

Bresters D, Lawitschka A, Cugno C, Potschger U, Dalissier A, Michel G, et al. Incidence and severity of crucial late effects after allogeneic HSCT for malignancy under the age of 3 years: TBI is what really matters. Bone Marrow Transpl. 2016;51:1482–9. PubMed

Overbeek A, van den Berg MH, Kremer LC, van den Heuvel-Eibrink MM, Tissing WJ, Loonen JJ, et al. A nationwide study on reproductive function, ovarian reserve, and risk of premature menopause in female survivors of childhood cancer: design and methodological challenges. BMC Cancer. 2012;12:363. PubMed PMC

Dalle JH, Lucchini G, Balduzzi A, Ifversen M, Jahnukainen K, Macklon KT, et al. State-of-the-art fertility preservation in children and adolescents undergoing haematopoietic stem cell transplantation: a report on the expert meeting of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) in Baden, Austria, 29-30 September 2015. Bone Marrow Transpl. 2017;52:1029–35. PubMed

Cross NC, Hughes TP, Feng L, O’Shea P, Bungey J, Marks DI, et al. Minimal residual disease after allogeneic bone marrow transplantation for chronic myeloid leukaemia in first chronic phase: correlations with acute graft-versus-host disease and relapse. Br J Haematol. 1993;84:67–74. PubMed

Childhood Acute Lymphoblastic Leukaemia Collaborative Group. Beneficial and harmful effects of anthracyclines in the treatment of childhood acute lymphoblastic leukaemia: a systematic review and meta-analysis. Br J Haematol. 2009;145:376–88. PubMed PMC

Davies SM, Ramsay NK, Klein JP, Weisdorf DJ, Bolwell B, Cahn JY, et al. Comparison of preparative regimens in transplants for children with acute lymphoblastic leukemia. J Clin Oncol Off J Am Soc Clin Oncol. 2000;18:340–7. PubMed

Iacobelli S, Committee ES. Suggestions on the use of statistical methodologies in studies of the European Group for Blood and Marrow Transplantation. Bone Marrow Transpl. 2013;48(Suppl 1):S1–37. PubMed

Bull K, Spiegelhalter DJ. Survival analysis in observational studies. Stat Med. 1997;16:1041–74. PubMed

Klein JP, Rizzo JD, Zhang MJ, Keiding N. Statistical methods for the analysis and presentation of the results of bone marrow transplants. Part I: unadjusted analysis. Bone Marrow Transpl. 2001;28:909–15. PubMed

Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med. 1999;18:695–706. PubMed

McCaffrey DF, Griffin BA, Almirall D, Slaughter ME, Ramchand R, Burgette LF. A tutorial on propensity score estimation for multiple treatments using generalized boosted models. Stat Med. 2013;32:3388–414. PubMed PMC

Andersen PK, Klein JP, Zhang MJ. Testing for centre effects in multi-centre survival studies: a Monte Carlo comparison of fixed and random effects tests. Stat Med. 1999;18:1489–500. PubMed

Ridgeway G, McCaffrey DF, Morral AR, Burgette LF, Griffin BA. Toolkit for Weighting and Analysis of Nonequivalent Groups: A Tutorial for the R TWANG Package, Santa Monica, Calif.: RAND Corporation, TL-136/1-NIDA, 2014. As of March 09, 2020: https://www.rand.org/pubs/tools/TL136z1.html.

Pulsipher MA, Wayne AS, Schultz KR. New frontiers in pediatric Allo-SCT: novel approaches for children and adolescents with ALL. Bone Marrow Transpl. 2014;49:1259–65. PubMed

Hamidieh A, Kargar M, Jahani M, Alimoghaddam K, Bahar B, Mousavi SA, et al. The outcome of allogeneic hematopoietic stem cell transplants without total body irradiation in pediatric patients with acute lymphoblastic leukemia: single centre experience. J Pediatr Hematol Oncol. 2012;34:101–7. PubMed

Shah AJ, Lenarsky C, Kapoor N, Crooks GM, Kohn DB, Parkman R, et al. Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation. J Pediatr Hematol Oncol. 2004;26:91–7. PubMed

Hamidieh AA, Monzavi SM, Kaboutari M, Behfar M, Esfandbod M. Outcome analysis of pediatric patients with acute lymphoblastic leukemia treated with total body irradiation-free allogeneic hematopoietic stem cell transplantation: comparison of patients with and without central nervous system involvement. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2017;23:2110–7. PubMed

Shimoni A, Bielorai B, Toren A, Hardan I, Avigdor A, Yeshurun M, et al. Intravenous busulfan-based conditioning prior to allogeneic hematopoietic stem cell transplantation: myeloablation with reduced toxicity. Exp Hematol. 2003;31:428–34. PubMed

Aschan J. Risk assessment in haematopoietic stem cell transplantation: conditioning. Best Pract Res Clin Haematol. 2007;20:295–310. PubMed

Peters C, Schrauder A, Schrappe M, von Stackelberg A, Stary J, Yaniv I, et al. Allogeneic haematopoietic stem cell transplantation in children with acute lymphoblastic leukaemia: the BFM/IBFM/EBMT concepts. Bone Marrow Transpl. 2005;35(Suppl 1):S9–11. PubMed

Balduzzi A, Dalle JH, Wachowiak J, Yaniv I, Yesilipek A, Sedlacek P, et al. Transplantation in children and adolescents with acute lymphoblastic leukemia from a matched donor versus an HLA-identical sibling: is the outcome comparable? Results from the International BFM ALL SCT 2007 Study. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. 2019;25:2197–210. PubMed

Driessen EM, de Lorenzo P, Campbell M, Felice M, Ferster A, Hann I, et al. Outcome of relapsed infant acute lymphoblastic leukemia treated on the interfant-99 protocol. Leukemia. 2016;30:1184–7. PubMed

Mann G, Attarbaschi A, Schrappe M, De Lorenzo P, Peters C, Hann I, et al. Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study. Blood. 2010;116:2644–50. PubMed

von Bueltzingsloewen A, Esperou-Bourdeau H, Souillet G, Demeocq F, Mechinaud-Lacroix F, Michel G, et al. Allogeneic bone marrow transplantation following a busulfan-based conditioning regimen in young children with acute lymphoblastic leukemia: a Cooperative Study of the Societe Francaise de Greffe de Moelle. Bone Marrow Transpl. 1995;16:521–7. PubMed

Zander AR, Berger C, Kroger N, Stockshlader M, Kruger W, Horstmann M, et al. High dose chemotherapy with busulfan, cyclophosphamide, and etoposide as conditioning regimen for allogeneic bone marrow transplantation for patients with acute myeloid leukemia in first complete remission. Clin Cancer Res: Off J Am Assoc Cancer Res. 1997;3:2671–5. PubMed

Horstmann M, Kroschke G, Stockschlader M, Betker R, Kruger W, Erttmann R, et al. Early toxicity of intensified conditioning with etoposide combined with total body irradiation/cyclophosphamide or busulfan/cyclophosphamide in children undergoing autologous or allogeneic bone marrow transplantation. Pediatr Hematol Oncol. 1996;13:45–53. PubMed

Sandler ES, Hagg R, Coppes MJ, Mustafa MM, Gamis A, Kamani N, et al. Hematopoietic stem cell transplantation (HSCT) with a conditioning regimen of busulfan, cyclophosphamide, and etoposide for children with acute myelogenous leukemia (AML): a phase I study of the Pediatric Blood and Marrow Transplant Consortium. Med Pediatr Oncol. 2000;35:403–9. PubMed

Boztug H, Sykora KW, Slatter M, Zecca M, Veys P, Lankester A, et al. European society for blood and marrow transplantation analysis of treosulfan conditioning before hematopoietic stem cell transplantation in children and adolescents with hematological malignancies. Pediatr Blood Cancer. 2016;63:139–48. PubMed

Locatelli F, Schrappe M, Bernardo ME, Rutella S. How I treat relapsed childhood acute lymphoblastic leukemia. Blood. 2012;120:2807–16. PubMed

Najít záznam

Citační ukazatele

Nahrávání dat ...

Možnosti archivace

Nahrávání dat ...