Outcomes of pediatric patients with therapy-related myeloid neoplasms.
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
12 2021
12 2021
Historique:
received:
19
05
2021
accepted:
20
08
2021
revised:
06
08
2021
pubmed:
5
9
2021
medline:
11
3
2022
entrez:
4
9
2021
Statut:
ppublish
Résumé
Long-term outcomes after allogeneic hematopoietic cell transplantation (HCT) for therapy-related myeloid neoplasms (tMNs) are dismal. There are few multicenter studies defining prognostic factors in pediatric patients with tMNs. We have accumulated the largest cohort of pediatric patients who have undergone HCT for a tMN to perform a multivariate analysis defining factors predictive of long-term survival. Sixty-eight percent of the 401 patients underwent HCT using a myeloablative conditioning (MAC) regimen, but there were no statistically significant differences in the overall survival (OS), event-free survival (EFS), or cumulative incidence of relapse and non-relapse mortality based on the conditioning intensity. Among the recipients of MAC regimens, 38.4% of deaths were from treatment-related causes, especially acute graft versus host disease (GVHD) and end-organ failure, as compared to only 20.9% of deaths in the reduced-intensity conditioning (RIC) cohort. Exposure to total body irradiation (TBI) during conditioning and experiencing grade III/IV acute GVHD was associated with worse OS. In addition, a diagnosis of therapy-related myelodysplastic syndrome and having a structurally complex karyotype at tMN diagnosis were associated with worse EFS. Reduced-toxicity (but not reduced-intensity) regimens might help to decrease relapse while limiting mortality associated with TBI-based HCT conditioning in pediatric patients with tMNs.
Identifiants
pubmed: 34480120
doi: 10.1038/s41409-021-01448-x
pii: 10.1038/s41409-021-01448-x
pmc: PMC9260859
mid: NIHMS1820140
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
2997-3007Subventions
Organisme : NCI NIH HHS
ID : K12 CA090625
Pays : United States
Organisme : NCI NIH HHS
ID : P30 CA008748
Pays : United States
Informations de copyright
© 2021. The Author(s), under exclusive licence to Springer Nature Limited.
Références
Davies SM. Subsequent malignant neoplasms in survivors of childhood cancer: childhood cancer survivor study (CCSS) studies. Pediatr Blood Cancer. 2007;48:727–730. https://doi.org/10.1002/pbc.21113
doi: 10.1002/pbc.21113
pubmed: 17243132
Aguilera DG, Vaklavas C, Tsimberidou AM, Wen S, Medeiros LJ, Corey SJ. Pediatric therapy-related myelodysplastic syndrome/acute myeloid leukemia: the MD Anderson Cancer Center experience. J Pediatr Hematol Oncol. 2009;31:803–11. https://doi.org/10.1097/MPH.0b013e3181ba43dc . e-pub ahead of print 2009/10/06
doi: 10.1097/MPH.0b013e3181ba43dc
pubmed: 19801947
Bhatia S, Krailo MD, Chen Z, Burden L, Askin FB, Dickman PS, et al. Therapy-related myelodysplasia and acute myeloid leukemia after Ewing sarcoma and primitive neuroectodermal tumor of bone: a report from the Children’s Oncology Group. Blood. 2007;109:46–51. https://doi.org/10.1182/blood-2006-01-023101
doi: 10.1182/blood-2006-01-023101
pubmed: 16985182
pmcid: 1785079
Schmiegelow K, Levinsen MF, Attarbaschi A, Baruchel A, Devidas M, Escherich G, et al. Second malignant neoplasms after treatment of childhood acute lymphoblastic leukemia. J Clin Oncol. 2013;31:2469–76. https://doi.org/10.1200/JCO.2012.47.0500
doi: 10.1200/JCO.2012.47.0500
pubmed: 23690411
pmcid: 3807139
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 . e-pub ahead of print 2010/12/04
doi: 10.1182/blood-2010-08-301713
pubmed: 21127174
Bhatia S. Therapy-related myelodysplasia and acute myeloid leukemia. Semin Oncol. 2013;40:666–75. https://doi.org/10.1053/j.seminoncol.2013.09.013 . e-pub ahead of print 2013/12/18
doi: 10.1053/j.seminoncol.2013.09.013
pubmed: 24331189
Schoch C, Kern W, Schnittger S, Hiddemann W, Haferlach T. Karyotype is an independent prognostic parameter in therapy-related acute myeloid leukemia (t-AML): an analysis of 93 patients with t-AML in comparison to 1091 patients with de novo AML. Leukemia. 2004;18:120–5. https://doi.org/10.1038/sj.leu.2403187 . e-pub ahead of print 2003/10/31
doi: 10.1038/sj.leu.2403187
pubmed: 14586477
Finke J, Schmoor C, Bertz H, Marks R, Wasch R, Zeiser R, et al. Long-term follow-up of therapy-related myelodysplasia and AML patients treated with allogeneic hematopoietic cell transplantation. Bone Marrow Transpl. 2016;51:771–7. https://doi.org/10.1038/bmt.2015.338 . e-pub ahead of print 2016/01/12
doi: 10.1038/bmt.2015.338
Nilsson C, Hulegardh E, Garelius H, Mollgard L, Brune M, Wahlin A, et al. Secondary acute myeloid leukemia and the role of allogeneic stem cell transplantation in a population-based setting. Biol Blood Marrow Transpl. 2019;25:1770–8. https://doi.org/10.1016/j.bbmt.2019.05.038 . e-pub ahead of print 2019/06/10
doi: 10.1016/j.bbmt.2019.05.038
Kroger N, Brand R, van Biezen A, Zander A, Dierlamm J, Niederwieser D, et al. Risk factors for therapy-related myelodysplastic syndrome and acute myeloid leukemia treated with allogeneic stem cell transplantation. Haematologica. 2009;94:542–9. https://doi.org/10.3324/haematol.2008.000927
doi: 10.3324/haematol.2008.000927
pubmed: 19278968
pmcid: 2663618
Litzow MR, Tarima S, Perez WS, Bolwell BJ, Cairo MS, Camitta BM, et al. Allogeneic transplantation for therapy-related myelodysplastic syndrome and acute myeloid leukemia. Blood. 2010;115:1850–7. https://doi.org/10.1182/blood-2009-10-249128
doi: 10.1182/blood-2009-10-249128
pubmed: 20032503
pmcid: 2832815
Kida M, Usuki K, Uchida N, Fukuda T, Katayama Y, Kondo T, et al. Outcome and risk factors for therapy-related myeloid neoplasms treated with allogeneic stem cell transplantation in Japan. Biol Blood Marrow Transpl. 2020;26:1543–51. https://doi.org/10.1016/j.bbmt.2020.04.004 . e-pub ahead of print 2020/04/23
doi: 10.1016/j.bbmt.2020.04.004
Brown CA, Youlden DR, Aitken JF, Moore AS. Therapy-related acute myeloid leukemia following treatment for cancer in childhood: a population-based registry study. Pediatr Blood Cancer. 2018;65:e27410 https://doi.org/10.1002/pbc.27410 . e-pub ahead of print 2018/09/06
doi: 10.1002/pbc.27410
pubmed: 30183136
Gassas A, Sivaprakasam P, Cummins M, Breslin P, Patrick K, Slatter M, et al. High transplant-related mortality associated with haematopoietic stem cell transplantation for paediatric therapy-related acute myeloid leukaemia (t-AML). A study on behalf of the United Kingdom Paediatric Blood and Bone Marrow Transplant Group. Bone Marrow Transpl. 2018;53:1165–9. https://doi.org/10.1038/s41409-018-0157-x . e-pub ahead of print 2018/03/17
doi: 10.1038/s41409-018-0157-x
Imamura T, Taga T, Takagi M, Kawasaki H, Koh K, Taki T, et al. Nationwide survey of therapy-related leukemia in childhood in Japan. Int J Hematol. 2018;108:91–97. https://doi.org/10.1007/s12185-018-2439-x . e-pub ahead of print 2018/03/27
doi: 10.1007/s12185-018-2439-x
pubmed: 29574603
Kobos R, Steinherz PG, Kernan NA, Prockop SE, Scaradavou A, Small TN, et al. Allogeneic hematopoietic stem cell transplantation for pediatric patients with treatment-related myelodysplastic syndrome or acute myelogenous leukemia. Biol Blood Marrow Transpl. 2012;18:473–80. https://doi.org/10.1016/j.bbmt.2011.11.009 . e-pub ahead of print 2011/11/15
doi: 10.1016/j.bbmt.2011.11.009
Woodard P, Barfield R, Hale G, Horwitz E, Leung W, Ribeiro R, et al. Outcome of hematopoietic stem cell transplantation for pediatric patients with therapy-related acute myeloid leukemia or myelodysplastic syndrome. Pediatr Blood Cancer. 2006;47:931–5. https://doi.org/10.1002/pbc.20596
doi: 10.1002/pbc.20596
pubmed: 16155933
Swerdlow SH, Campo E, Harris NL, Jaffe ES, Pileri SA, Stein H et al. WHO classification of tumours of haematopoietic and lymphoid tissue. Lyon, France: International Agency for Research on Cancer; 2017.
Godley LA, Larson RA. Therapy-related myeloid leukemia. Semin Oncol. 2008;35:418–29. https://doi.org/10.1053/j.seminoncol.2008.04.012 . e-pub ahead of print 2008/08/12
doi: 10.1053/j.seminoncol.2008.04.012
pubmed: 18692692
pmcid: 2600445
Lee CJ, Labopin M, Beelen D, Finke J, Blaise D, Ganser A, et al. Comparative outcomes of myeloablative and reduced-intensity conditioning allogeneic hematopoietic cell transplantation for therapy-related acute myeloid leukemia with prior solid tumor: a report from the acute leukemia working party of the European society for blood and bone marrow transplantation. Am J Hematol. 2019;94:431–8. https://doi.org/10.1002/ajh.25395 . e-pub ahead of print 2019/01/01
doi: 10.1002/ajh.25395
pubmed: 30597620
Sengsayadeth S, Gatwood KS, Boumendil A, Labopin M, Finke J, Ganser A, et al. Conditioning intensity in secondary AML with prior myelodysplastic syndrome/myeloproliferative disorders: an EBMT ALWP study. Blood Adv. 2018;2:2127–35. https://doi.org/10.1182/bloodadvances.2018019976 . e-pub ahead of print 2018/08/26
doi: 10.1182/bloodadvances.2018019976
pubmed: 30143527
pmcid: 6113606
Gatwood KS, Labopin M, Savani BN, Finke J, Socie G, Beelen D, et al. Transplant outcomes for patients with therapy-related acute myeloid leukemia with prior lymphoid malignancy: an ALWP of EBMT study. Bone Marrow Transpl. 2020;55:224–32. https://doi.org/10.1038/s41409-019-0673-3 . e-pub ahead of print 2019/09/19
doi: 10.1038/s41409-019-0673-3
Kroger N. Induction, bridging, or straight ahead: the ongoing dilemma of allografting in advanced myelodysplastic syndrome. Biol Blood Marrow Transpl. 2019;25:e247–e249. https://doi.org/10.1016/j.bbmt.2019.06.016 . e-pub ahead of print 2019/06/24
doi: 10.1016/j.bbmt.2019.06.016
McDonald GB, Sandmaier BM, Mielcarek M, Sorror M, Pergam SA, Cheng GS, et al. Survival, nonrelapse mortality, and relapse-related mortality after allogeneic hematopoietic cell transplantation: comparing 2003–2007 versus 2013–2017 cohorts. Ann Intern Med. 2020;172:229–39. https://doi.org/10.7326/M19-2936 . e-pub ahead of print 2020/01/21
doi: 10.7326/M19-2936
pubmed: 31958813
pmcid: 7847247
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. 2017;35:1154–61. https://doi.org/10.1200/JCO.2016.70.7091 . e-pub ahead of print 2017/04/06
doi: 10.1200/JCO.2016.70.7091
pubmed: 28380315
pmcid: 5455603
Singhal D, Hahn CN, Feurstein S, Wee LYA, Moma L, Kutyna MM et al. Targeted gene panels identify a high frequency of pathogenic germline variants in patients diagnosed with a hematological malignancy and at least one other independent cancer. Leukemia. 2021. e-pub ahead of print 2021/04/15; https://doi.org/10.1038/s41375-021-01246-w
Gibson CJ, Lindsley RC, Tchekmedyian V, Mar BG, Shi J, Jaiswal S, et al. Clonal hematopoiesis associated with adverse outcomes after autologous stem-cell transplantation for lymphoma. J Clin Oncol. 2017;35:1598–605. https://doi.org/10.1200/JCO.2016.71.6712 . e-pub ahead of print 2017/01/10
doi: 10.1200/JCO.2016.71.6712
pubmed: 28068180
pmcid: 5455707
Wong TN, Ramsingh G, Young AL, Miller CA, Touma W, Welch JS, et al. Role of TP53 mutations in the origin and evolution of therapy-related acute myeloid leukaemia. Nature. 2015;518:552–5. https://doi.org/10.1038/nature13968 . e-pub ahead of print 2014/12/10
doi: 10.1038/nature13968
pubmed: 25487151
Berger G, Kroeze LI, Koorenhof-Scheele TN, de Graaf AO, Yoshida K, Ueno H, et al. Early detection and evolution of preleukemic clones in therapy-related myeloid neoplasms following autologous SCT. Blood. 2018;131:1846–57. https://doi.org/10.1182/blood-2017-09-805879 . e-pub ahead of print 2018/01/10
doi: 10.1182/blood-2017-09-805879
pubmed: 29311096
Schwartz JR, Ma J, Kamens J, Westover T, Walsh MP, Brady SW, et al. The acquisition of molecular drivers in pediatric therapy-related myeloid neoplasms. Nat Commun. 2021;12:985 https://doi.org/10.1038/s41467-021-21255-8 . e-pub ahead of print 2021/02/14
doi: 10.1038/s41467-021-21255-8
pubmed: 33579957
pmcid: 7880998
Waanders E, Gu Z, Dobson SM, Antic Z, Crawford JC, Ma X, et al. Mutational landscape and patterns of clonal evolution in relapsed pediatric acute lymphoblastic leukemia. Blood Cancer Discov. 2020;1:96–111. https://doi.org/10.1158/0008-5472.BCD-19-0041 . e-pub ahead of print 2020/08/15
doi: 10.1158/0008-5472.BCD-19-0041
pubmed: 32793890
pmcid: 7418874
Myllymaki M, Redd R, Reilly CR, Saber W, Spellman SR, Gibson CJ, et al. Short telomere length predicts nonrelapse mortality after stem cell transplantation for myelodysplastic syndrome. Blood. 2020;136:3070–81. https://doi.org/10.1182/blood.2020005397 . e-pub ahead of print 2020/12/29
doi: 10.1182/blood.2020005397
pubmed: 33367544
Baron F, Labopin M, Savani BN, Beohou E, Niederwieser D, Eder M, et al. Graft-versus-host disease and graft-versus-leukaemia effects in secondary acute myeloid leukaemia: a retrospective, multicentre registry analysis from the Acute Leukaemia Working Party of the EBMT. Br J Haematol. 2020;188:428–37. https://doi.org/10.1111/bjh.16185 . e-pub ahead of print 2019/10/16
doi: 10.1111/bjh.16185
pubmed: 31612473
Kongtim P, Parmar S, Milton DR, Perez JMR, Rondon G, Chen J, et al. Impact of a novel prognostic model, hematopoietic cell transplant-composite risk (HCT-CR), on allogeneic transplant outcomes in patients with acute myeloid leukemia and myelodysplastic syndrome. Bone Marrow Transpl. 2019;54:839–48. https://doi.org/10.1038/s41409-018-0344-9 . e-pub ahead of print 2018/09/28
doi: 10.1038/s41409-018-0344-9
Armand P, Kim HT, Logan BR, Wang Z, Alyea EP, Kalaycio ME, et al. Validation and refinement of the disease risk index for allogeneic stem cell transplantation. Blood. 2014;123:3664–71. https://doi.org/10.1182/blood-2014-01-552984 . e-pub ahead of print 2014/04/20
doi: 10.1182/blood-2014-01-552984
pubmed: 24744269
pmcid: 4047501
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 . e-pub ahead of print 2005/07/05
doi: 10.1182/blood-2005-05-2004
pubmed: 15994282
pmcid: 1895304
Broglie L, Ruiz J, Jin Z, Kahn JM, Bhatia M, George D et al. Limitations of applying the hematopoietic cell transplantation comorbidity index in pediatric patients receiving allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transpl. 2020. e-pub ahead of print 2020/10/12; https://doi.org/10.1016/j.bbmt.2020.10.003