Prognostic value of the revised International Prognostic Scoring System five-group cytogenetic abnormality classification for the outcome prediction of hematopoietic stem cell transplantation in pediatric myelodysplastic syndrome.
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:
03
06
2021
accepted:
19
08
2021
revised:
10
08
2021
pubmed:
12
9
2021
medline:
11
3
2022
entrez:
11
9
2021
Statut:
ppublish
Résumé
Cytogenetic abnormalities are a major risk factor for relapse after hematopoietic stem cell transplantation (HSCT) for myelodysplastic syndrome (MDS). We aimed to evaluate the value of the five-group cytogenetic classification according to the revised International Prognostic Scoring System (R-IPSS) for predicting the outcome after HSCT in pediatric patients with MDS. We retrospectively analyzed the Japanese registration data of 242 pediatric patients with MDS. According to the R-IPSS classification, 112 (45.5%) patients had good, 55 (22.7%) had intermediate, 64 (26.4%) had poor, and 11 (4.6%) had very poor cytogenetics. The 5-year overall survival (5yOS) was 72%, 69%, 59%, and 30% in the good, intermediate, poor, and very poor cytogenetic subgroups (p = 0.026), respectively. The very good, good, and intermediate subgroups were grouped into a "standard" subgroup and reclassified into three subgroups (standard, poor, and very poor). Patients with very poor risk had worse 5yOS (hazard ratio 2.17, 95% confidence interval (CI) 1.02-4.61; p = 0.04) and a much higher 5yCIR (hazard ratio 2.52, 95% CI 1.05-6.04; p = 0.04) than those of patients in the standard group in the multivariate analysis, indicating that very poor risk cytogenetic characteristics independently predicted worse outcome after HSCT in pediatric patients with MDS.
Identifiants
pubmed: 34508178
doi: 10.1038/s41409-021-01446-z
pii: 10.1038/s41409-021-01446-z
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
3016-3023Informations de copyright
© 2021. The Author(s), under exclusive licence to Springer Nature Limited.
Références
Tefferi A, Vardiman JW. Myelodysplastic syndromes. N Engl J Med. 2009;361:1872–85.
doi: 10.1056/NEJMra0902908
Hasle H, Wadsworth LD, Massing BG, McBride M, Schultz KR. A population-based study of childhood myelodysplastic syndrome in British Columbia, Canada. Br J Haematol. 1999;106:1027–32.
doi: 10.1046/j.1365-2141.1999.01645.x
Deeg HJ, Storer B, Slattery JT, Anasetti C, Doney KC, Hansen JA, et al. Conditioning with targeted busulfan and cyclophosphamide for hemopoietic stem cell transplantation from related and unrelated donors in patients with myelodysplastic syndrome. Blood. 2002;100:1201–7.
doi: 10.1182/blood-2002-02-0527
Armand P, Kim HT, DeAngelo DJ, Ho VT, Cutler CS, Stone RM, et al. Impact of cytogenetics on outcome of de novo and therapy-related AML and MDS after allogeneic transplantation. Biol Blood Marrow Transplant. 2007;13:655–64.
doi: 10.1016/j.bbmt.2007.01.079
Greenberg PL, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89:2079–88.
doi: 10.1182/blood.V89.6.2079
Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Solé F, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120:2454–65.
doi: 10.1182/blood-2012-03-420489
Deeg HJ, Scott BL, Fang M, Shulman HM, Gyurkocza B, Myerson D, et al. Five-group cytogenetic risk classification, monosomal karyotype, and outcome after hematopoietic cell transplantation for MDS or acute leukemia evolving from MDS. Blood. 2012;120:1398–408.
doi: 10.1182/blood-2012-04-423046
Koenecke C, Göhring G, de Wreede LC, van Biezen A, Scheid C, Volin L, et al. Impact of the revised International Prognostic Scoring System, cytogenetics and monosomal karyotype on outcome after allogeneic stem cell transplantation for myelodysplastic syndromes and secondary acute myeloid leukemia evolving from myelodysplastic syndromes: a retrospective multicenter study of the European Society of Blood and Marrow Transplantation. Haematologica. 2015;100:400–8.
doi: 10.3324/haematol.2014.116715
Della Porta MG, Alessandrino EP, Bacigalupo A, van Lint MT, Malcovati L, Pascutto C, et al. Predictive factors for the outcome of allogeneic transplantation in patients with MDS stratified according to the revised IPSS-R. Blood. 2014;123:2333–42.
doi: 10.1182/blood-2013-12-542720
Hasle H, Niemeyer CM, Chessells JM, Baumann I, Bennett JM, Kerndrup G, et al. A pediatric approach to the WHO classification of myelodysplastic and myeloproliferative diseases. Leukemia. 2003;17:277–82.
doi: 10.1038/sj.leu.2402765
Atsuta Y, Suzuki R, Yoshimi A, Gondo H, Tanaka J, Hiraoka A, et al. Unification of hematopoietic stem cell transplantation registries in Japan and establishment of the TRUMP system. Int J Hematol. 2007;86:269–74.
doi: 10.1007/BF03006932
Bennet JM, Catovsky D, Daniel MT, Flandrin G, Galton DA, Gralnick HR, et al. Proposals for the classification of the myelodysplastic syndromes. Br J Haematol. 1982;51:189–99.
doi: 10.1111/j.1365-2141.1982.tb08475.x
Vardiman JW, Thiele J, Arber DA, Brunning RD, Borowitz MJ, Porwit A, et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood. 2009;114:937–51.
doi: 10.1182/blood-2009-03-209262
Giralt S, Ballen K, Rizzo D, Bacigalupo A, Horowitz M, Pasquini M, et al. Reduced-intensity conditioning regimen workshop: defining the dose spectrum. Report of a workshop convened by the center for international blood and marrow transplant research. Biol Blood Marrow Transplant. 2009;15:367–9.
doi: 10.1016/j.bbmt.2008.12.497
Kanda Y. Investigation of the freely available easy-to-use software ‘EZR’ for medical statistics. Bone Marrow Transplant. 2013;48:452–8.
doi: 10.1038/bmt.2012.244
Andolina JR, Kletzel M, Tse WT, Jacobsohn DA, Duerst RE, Schneiderman J, et al. Allogeneic hematopoetic stem cell transplantation in pediatric myelodysplastic syndromes: improved outcomes for de novo disease. Pediatr Transplant. 2011;15:334–43.
doi: 10.1111/j.1399-3046.2011.01479.x
Gohring G, Michalova K, Beverloo HB, Betts D, Harbott J, Haas OA, et al. Complex Karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood. 2010;116:3766–9.
doi: 10.1182/blood-2010-04-280313
Kikuchi A, Hasegawa D, Ohtsuka Y, Hamamoto K, Kojima S, Okamura J, et al. Outcome of children with refractory anaemia with excess of blast (RAEB) and RAEB in transformation (RAEB-T) in the Japanese MDS99 study. Br J Haematol. 2012;158:657–61.
doi: 10.1111/j.1365-2141.2012.09210.x
Moriwaki K, Manabe A, Taketani T, Kikuchi A, Nakahata T, Hayashi Y. Cytogenetics and clinical features of pediatric myelodysplastic syndrome in Japan. Int J Hematol. 2014;100:478–84.
doi: 10.1007/s12185-014-1674-z
Luna-Fineman S, Shannon KM, Atwater SK, Davis J, Masterson M, Orgega J, et al. Myelodysplastic and myeloproliferative disorders of childhood: a study of 167 patients. Blood. 1999;93:459–66.
doi: 10.1182/blood.V93.2.459
Oran B, Kongtim P, Popat U, de Lima M, Jabbour E, Lu X, et al. Cytogenetics, donor type, and use of hypomethylating agents in myelodysplastic syndrome with allogeneic stem cell transplantation. Biol Blood Marrow Transplant. 2014;20:1618–25.
doi: 10.1016/j.bbmt.2014.06.022
Strahm B, Nolke P, Zecca M, Korthof ET, Bierings M, Furlan I, et al. Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study. Leukemia. 2011;25:455–62.
doi: 10.1038/leu.2010.297
Madureira ABM, Eapen M, Locatelli F, Teira P, Zhang MJ, Davies SM, et al. Analysis of risk factors influencing outcome in children with myelodysplastic syndrome after unrelated cord blood transplantation. Leukemia. 2011;25:449–54.
doi: 10.1038/leu.2010.285
Wlodarski MW, Hirabayashi S, Pastor V, Starý J, Hasle H, Masetti R, et al. Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents. Blood. 2016;127:1387–97.
doi: 10.1182/blood-2015-09-669937
Locatelli F, Strahm B. How I treat myelodysplastic syndromes of childhood. Blood. 2018;131:1406–14.
doi: 10.1182/blood-2017-09-765214
Basquiera AL, Pizzi S, Correas AG, Longo PG, Goldman WC, Prates MV, et al. Allogeneic hematopoietic stem cell transplantation in pediatric myelodysplastic syndromes: a multicenter experience from Argentina. Pediatr Blood Cancer. 2015;62:153–7.
doi: 10.1002/pbc.25238
Smith AR, Christiansen EC, Wagner JE, Cao Q, MacMillan ML, Stefanski HE, et al. Early hematopoietic stem cell transplant is associated with favorable outcomes in children with MDS. Pediatr Blood Cancer. 2013;60:705–10.
doi: 10.1002/pbc.24390
de Lima M, Giralt S, Thall PF, de Padua Silva L, Jones RB, Komanduri K, et al. Maintenance therapy with low-dose azacitidine after allogeneic hematopoietic stem cell transplantation for recurrent acute myelogenous leukemia or myelodysplastic syndrome: a dose and schedule finding study. Cancer. 2010;116:5420–31.
doi: 10.1002/cncr.25500
Bejar R, Stevenson K, Abdel-Wahab O, Galili N, Nilsson B, Garcia-Manero G, et al. Clinical effect of point mutations in myelodysplastic syndromes. N Engl J Med. 2011;364:2496–506.
doi: 10.1056/NEJMoa1013343
Papaemmanuil E, Cazzola M, Boultwood J, Malcovati L, Vyas P, Bowen D, et al. Somatic SF3B1 mutation in myelodysplasia with ring sideroblasts. N Engl J Med. 2011;365:1384–95.
doi: 10.1056/NEJMoa1103283
Bejar R, Stevenson K, Caughey B, Lindsley RC, Mar BG, Stojanov P, et al. Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation. J Clin Oncol. 2014;32:2691–8.
doi: 10.1200/JCO.2013.52.3381