Comparative study of treosulfan plus Fludarabine (FT14) with busulfan plus Fludarabine (FB4) for acute myeloid leukemia in first or second complete remission: An analysis from the European Society for Blood and Marrow Transplantation (EBMT) Acute Leukemia Working Party (ALWP).
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
12 2022
12 2022
Historique:
received:
08
10
2021
accepted:
08
09
2022
revised:
26
08
2022
pubmed:
23
9
2022
medline:
6
12
2022
entrez:
22
9
2022
Statut:
ppublish
Résumé
Different doses of treosulfan plus fludarabine have shown advantage over reduced intensity regimens. However, data comparing higher doses of treosulfan to myeloablative busulfan are limited. Thus, we compared outcomes between FT14 (fludarabine 150/160 mg/m
Identifiants
pubmed: 36138068
doi: 10.1038/s41409-022-01830-3
pii: 10.1038/s41409-022-01830-3
doi:
Substances chimiques
treosulfan
CO61ER3EPI
Busulfan
G1LN9045DK
fludarabine
P2K93U8740
Vidarabine
FA2DM6879K
Types de publication
Multicenter Study
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1803-1809Informations de copyright
© 2022. The Author(s), under exclusive licence to Springer Nature Limited.
Références
Thein MS, Ershler WB, Jemal A, Yates JW, Baer MR. Outcome of older patients with acute myeloid leukemia: an analysis of SEER data over 3 decades. Cancer. 2013;119:2720–7. https://doi.org/10.1002/cncr.28129 .
doi: 10.1002/cncr.28129
pubmed: 23633441
Alam N, Atenafu EG, Kuruvilla J, Uhm J, Lipton JH, Messner HA, et al. Outcomes of patients with therapy-related AML/myelodysplastic syndrome (t-AML/MDS) following hematopoietic cell transplantation. Bone marrow Transplant. 2015;50:1180–6. https://doi.org/10.1038/bmt.2015.151 .
doi: 10.1038/bmt.2015.151
pubmed: 26121109
Kayser S, Dohner K, Krauter J, Kohne CH, Horst HA, Held G, et al. The impact of therapy-related acute myeloid leukemia (AML) on outcome in 2853 adult patients with newly diagnosed AML. Blood 2011;117:2137–45. https://doi.org/10.1182/blood-2010-08-301713 .
doi: 10.1182/blood-2010-08-301713
pubmed: 21127174
Jethava YS, Sica S, Savani B, Socola F, Jagasia M, Mohty M, et al. Conditioning regimens for allogeneic hematopoietic stem cell transplants in acute myeloid leukemia. Bone marrow Transplant. 2017;52:1504–11. https://doi.org/10.1038/bmt.2017.83 .
doi: 10.1038/bmt.2017.83
pubmed: 28504666
Bacigalupo A, Ballen K, Rizzo D, Giralt S, Lazarus H, Ho V, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2009;15:1628–33. https://doi.org/10.1016/j.bbmt.2009.07.004 .
doi: 10.1016/j.bbmt.2009.07.004
Slavin S, Nagler A, Naparstek E, Kapelushnik Y, Aker M, Cividalli G, et al. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood. 1998;91:756–63.
doi: 10.1182/blood.V91.3.756
pubmed: 9446633
de Lima M, Anagnostopoulos A, Munsell M, Shahjahan M, Ueno N, Ippoliti C, et al. Nonablative versus reduced-intensity conditioning regimens in the treatment of acute myeloid leukemia and high-risk myelodysplastic syndrome: dose is relevant for long-term disease control after allogeneic hematopoietic stem cell transplantation. Blood. 2004;104:865–72. https://doi.org/10.1182/blood-2003-11-3750 .
doi: 10.1182/blood-2003-11-3750
pubmed: 15090449
Shimoni A, Hardan I, Shem-Tov N, Yeshurun M, Yerushalmi R, Avigdor A, et al. Allogeneic hematopoietic stem-cell transplantation in AML and MDS using myeloablative versus reduced-intensity conditioning: the role of dose intensity. Leukemia. 2006;20:322–8. https://doi.org/10.1038/sj.leu.2404037 .
doi: 10.1038/sj.leu.2404037
pubmed: 16307018
Oliansky DM, Appelbaum F, Cassileth PA, Keating A, Kerr J, Nieto Y, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute myelogenous leukemia in adults: an evidence-based review. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2008;14:137–80. https://doi.org/10.1016/j.bbmt.2007.11.002 .
doi: 10.1016/j.bbmt.2007.11.002
Nagler A, Labopin M, Shimoni A, Niederwieser D, Mufti GJ, Zander AR, et al. Mobilized peripheral blood stem cells compared with bone marrow as the stem cell source for unrelated donor allogeneic transplantation with reduced-intensity conditioning in patients with acute myeloid leukemia in complete remission: an analysis from the Acute Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2012;18:1422–9. https://doi.org/10.1016/j.bbmt.2012.02.013 .
doi: 10.1016/j.bbmt.2012.02.013
Danylesko I, Shimoni A, Nagler A. Treosulfan-based conditioning before hematopoietic SCT: More than a BU look-alike. Bone marrow Transplant. 2012;47:5–14. https://doi.org/10.1038/bmt.2011.88 .
doi: 10.1038/bmt.2011.88
pubmed: 21478921
Slatter MA, Boztug H, Potschger U, Sykora KW, Lankester A, Yaniv I, et al. Treosulfan-based conditioning regimens for allogeneic haematopoietic stem cell transplantation in children with non-malignant diseases. Bone marrow Transplant. 2015;50:1536–41. https://doi.org/10.1038/bmt.2015.171 .
doi: 10.1038/bmt.2015.171
pubmed: 26259076
Strocchio L, Zecca M, Comoli P, Mina T, Giorgiani G, Giraldi E, et al. Treosulfan-based conditioning regimen for -logeneic haematopoietic stem cell transplantation in children with sickle cell disease. Br urinal Haematol. 2015;169:726–36. https://doi.org/10.1111/bjh.13352 .
doi: 10.1111/bjh.13352
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. https://doi.org/10.1002/pbc.25764 .
doi: 10.1002/pbc.25764
pubmed: 26398915
Ruutu T, Volin L, Beelen DW, Trenschel R, Finke J, Schnitzler M, et al. Reduced-toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes: final results of an international prospective phase II trial. Haematologica. 2011;96:1344–1350. https://doi.org/10.3324/haematol.2011.043810 .
doi: 10.3324/haematol.2011.043810
pubmed: 21659356
pmcid: 3166105
Sjoo F, Hassan Z, Abedi-Valugerdi M, Griskevicius L, Nilsson C, Remberger M, et al. Myeloablative and immunosuppressive properties of treosulfan in mice. Exp Hematol. 2006;34:115–21. https://doi.org/10.1016/j.exphem.2005.09.015 .
doi: 10.1016/j.exphem.2005.09.015
pubmed: 16413398
Glowka F, Kasprzyk A, Romanski M, Wrobel T, Wachowiak J, Szpecht D, et al. Pharmacokinetics of treosulfan and its active monoepoxide in pediatric patients after intravenous infusion of high-dose treosulfan prior to HSCT. Eur J Pharm Sci: Off J Eur Federation Pharm Sci. 2015;68:87–93. https://doi.org/10.1016/j.ejps.2014.12.010 .
doi: 10.1016/j.ejps.2014.12.010
Kroger N, Shimoni A, Zabelina T, Schieder H, Panse J, Ayuk F. et al.Reduced-toxicity conditioning with treosulfan, fludarabine and ATG as preparative regimen for allogeneic stem cell transplantation (alloSCT) in elderly patients with secondary acute myeloid leukemia (sAML) or myelodysplastic syndrome (MDS).Bone Marrow Transpl.2006;37:339–44. https://doi.org/10.1038/sj.bmt.1705259 .
doi: 10.1038/sj.bmt.1705259
Shimoni A, Hardan I, Shem-Tov N, Rand A, Yerushalmi R, Nagler A. Fludarabine and treosulfan: a novel modified myeloablative regimen for allogeneic hematopoietic stem-cell transplantation with effective antileukemia activity in patients with acute myeloid leukemia and myelodysplastic syndromes. Leuk Lymphoma. 2007;48:2352–9. https://doi.org/10.1080/10428190701671051 .
doi: 10.1080/10428190701671051
pubmed: 18067010
Nemecek ER, Guthrie KA, Sorror ML, Wood BL, Doney KC, Hilger RA, et al. Conditioning with treosulfan and fludarabine followed by allogeneic hematopoietic cell transplantation for high-risk hematologic malignancies. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2011;17:341–50. https://doi.org/10.1016/j.bbmt.2010.05.007 .
doi: 10.1016/j.bbmt.2010.05.007
Casper J, Holowiecki J, Trenschel R, Wandt H, Schaefer-Eckart K, Ruutu T, et al. Allogeneic hematopoietic SCT in patients with AML following treosulfan/fludarabine conditioning. Bone marrow Transplant. 2012;47:1171–7. https://doi.org/10.1038/bmt.2011.242 .
doi: 10.1038/bmt.2011.242
pubmed: 22158386
Gyurkocza B, Gutman J, Nemecek ER, Bar M, Milano F, Ramakrishnan A, et al. Treosulfan, fludarabine, and 2-Gy total body irradiation followed by allogeneic hematopoietic cell transplantation in patients with myelodysplastic syndrome and acute myeloid leukemia. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2014;20:549–55. https://doi.org/10.1016/j.bbmt.2014.01.009 .
doi: 10.1016/j.bbmt.2014.01.009
Casper J, Wolff D, Knauf W, Blau IW, Ruutu T, Volin L, et al. Allogeneic hematopoietic stem-cell transplantation in patients with hematologic malignancies after dose-escalated treosulfan/fludarabine conditioning. J Clin Oncol: Off J Am Soc Clin Oncol. 2010;28:3344–51. https://doi.org/10.1200/JCO.2009.23.3429 .
doi: 10.1200/JCO.2009.23.3429
Zhu S, Liu G, Liu J, Chen Q, Wang Z. Long-Term Outcomes of Treosulfan- vs. Busulfan-Based Conditioning Regimen for Patients With Myelodysplastic Syndrome and Acute Myeloid Leukemia Before Hematopoietic Cell Transplantation: A Systematic Review and Meta-Analysis. Front Oncol. 2020;10:591363 https://doi.org/10.3389/fonc.2020.591363 .
doi: 10.3389/fonc.2020.591363
pubmed: 33425740
pmcid: 7793760
Beelen DW, Trenschel R, Stelljes M, Groth C, Masszi T, Remenyi, et al. Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid leukaemia or myelodysplastic syndrome (MC-FludT.14/L): A randomised, non-inferiority, phase 3 trial. Lancet Haematol. 2020;7:e28–e39. https://doi.org/10.1016/S2352-3026(19)30157-7 .
doi: 10.1016/S2352-3026(19)30157-7
pubmed: 31606445
Sakellari I, Mallouri D, Gavriilaki E, Batsis I, Kaliou M, Constantinou V, et al. Survival Advantage and Comparable Toxicity in Reduced-Toxicity Treosulfan-Based versus Reduced-Intensity Busulfan-Based Conditioning Regimen in Myelodysplastic Syndrome and Acute Myeloid Leukemia Patients after Allogeneic Hematopoietic Cell Transplantation. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2017;23:445–51. https://doi.org/10.1016/j.bbmt.2016.11.023 .
doi: 10.1016/j.bbmt.2016.11.023
Shimoni A, Shem-Tov N, Volchek Y, Danylesko I, Yerushalmi R, Nagler A. Allo-SCT for AML and MDS with treosulfan compared with BU-based regimens: reduced toxicity vs reduced intensity. Bone marrow Transplant. 2012;47:1274–82. https://doi.org/10.1038/bmt.2012.4 .
doi: 10.1038/bmt.2012.4
pubmed: 22327135
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.
doi: 10.1002/(SICI)1097-0258(19990330)18:6<695::AID-SIM60>3.0.CO;2-O
pubmed: 10204198
Sakellari I, Gavriilaki E, Mallouri D, Batsis I, Varelas C, Tagara S, et al. Survival Advantage of Treosulfan Plus Fludarabine Before Allogeneic Hematopoietic Cell Transplantation for Older or Comorbid Patients With Myeloid Malignancies. Transpl Cell Ther. 2021;27:916 e911–916. https://doi.org/10.1016/j.jtct.2021.07.020 .
doi: 10.1016/j.jtct.2021.07.020
Passweg JR, Baldomero H, Basak GW, Chabannon C, Corbacioglu S, Duarte R, et al. The EBMT activity survey report 2017: A focus on allogeneic HCT for nonmalignant indications and on the use of non-HCT cell therapies. Bone Marrow Transplant. 2019;54:1575–85. https://doi.org/10.1038/s41409-019-0465-9 .
doi: 10.1038/s41409-019-0465-9
pubmed: 30728439
pmcid: 6957459
Loke J, Malladi R, Moss P, Craddock C. The role of allogeneic stem cell transplantation in the management of acute myeloid leukaemia: A triumph of hope and experience. Br J Haematol. 2020;188:129–46. https://doi.org/10.1111/bjh.16355 .
doi: 10.1111/bjh.16355
pubmed: 31823351
Fasslrinner F, Schetelig J, Burchert A, Kramer M, Trenschel R, Hegenbart U, et al. Long-term efficacy of reduced-intensity versus myeloablative conditioning before allogeneic haemopoietic cell transplantation in patients with acute myeloid leukaemia in first complete remission: Retrospective follow-up of an open-label, randomised phase 3 trial. Lancet Haematol. 2018;5:e161–e169. https://doi.org/10.1016/S2352-3026(18)30022-X .
doi: 10.1016/S2352-3026(18)30022-X
pubmed: 29550384
Scott BL, Pasquini MC, Logan BR, Wu J, Devine SM, Porter DL, et al. Myeloablative Versus Reduced-Intensity Hematopoietic Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes. J Clin Oncol: Off J Am Soc Clin Oncol. 2017;35:1154–61. https://doi.org/10.1200/JCO.2016.70.7091 .
doi: 10.1200/JCO.2016.70.7091
Rambaldi A, Grassi A, Masciulli A, Boschini C, Mico MC, Busca A, et al. Busulfan plus cyclophosphamide versus busulfan plus fludarabine as a preparative regimen for allogeneic haemopoietic stem-cell transplantation in patients with acute myeloid leukaemia: An open-label, multicentre, randomised, phase 3 trial. Lancet Oncol. 2015;16:1525–36. https://doi.org/10.1016/S1470-2045(15)00200-4 .
doi: 10.1016/S1470-2045(15)00200-4
pubmed: 26429297
Spyridonidis A, Labopin M, Savani BN, Niittyvuopio R, Blaise D, Craddock C, et al. Redefining and measuring transplant conditioning intensity in current era: A study in acute myeloid leukemia patients. Bone Marrow Transplant. 2020;55:1114–25. https://doi.org/10.1038/s41409-020-0803-y .
doi: 10.1038/s41409-020-0803-y
pubmed: 31996792
Wedge E, Sengelov H, Hansen JW, Andersen NS, Schjodt I, Petersen SL, et al. Improved Outcomes after Allogenic Hematopoietic Stem Cell Transplantation with Fludarabine/Treosulfan for Patients with Myelodysplastic Syndromes. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2020;26:1091–8. https://doi.org/10.1016/j.bbmt.2020.02.010 .
doi: 10.1016/j.bbmt.2020.02.010
Fein JA, Shimoni A, Labopin M, Shem-Tov N, Yerushalmi R, Magen H, et al. The impact of individual comorbidities on non-relapse mortality following allogeneic hematopoietic stem cell transplantation. Leukemia 2018;32:1787–94. https://doi.org/10.1038/s41375-018-0185-y .
doi: 10.1038/s41375-018-0185-y
pubmed: 29950692
Sorror ML, Maris MB, Storb R, Baron F, Sandmaier BM, Maloney DG, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: A new tool for risk assessment before allogeneic HCT. Blood 2005;106:2912–9. https://doi.org/10.1182/blood-2005-05-2004 .
doi: 10.1182/blood-2005-05-2004
pubmed: 15994282
pmcid: 1895304
Cheung E, Perissinotti AJ, Bixby DL, Burke PW, Pettit KM, Benitez LL, et al. The leukemia strikes back: A review of pathogenesis and treatment of secondary AML. Ann Hematol. 2019;98:541–59. https://doi.org/10.1007/s00277-019-03606-0 .
doi: 10.1007/s00277-019-03606-0
pubmed: 30666431
Sengsayadeth S, Labopin M, Boumendil A, Finke J, Ganser A, Stelljes M, et al. Transplant Outcomes for Secondary Acute Myeloid Leukemia: Acute Leukemia Working Party of the European Society for Blood and Bone Marrow Transplantation Study. Biol Blood Marrow Transplant: J Am Soc Blood Marrow Transplant. 2018;24:1406–14. https://doi.org/10.1016/j.bbmt.2018.04.008 .
doi: 10.1016/j.bbmt.2018.04.008