Improvement of survival after hematopoietic stem cell transplantation and trends at a pediatric transplantation center; a three-decade journey.

event-free survival hematopoietic stem cell transplantation overall survival

Journal

Pediatric transplantation
ISSN: 1399-3046
Titre abrégé: Pediatr Transplant
Pays: Denmark
ID NLM: 9802574

Informations de publication

Date de publication:
Feb 2024
Historique:
received: 17 09 2023
accepted: 04 01 2024
medline: 6 2 2024
pubmed: 6 2 2024
entrez: 6 2 2024
Statut: ppublish

Résumé

Advances in stem cell transplantation have resulted in improved outcomes. This is a retrospective study aimed to analyze changes in patient profile, transplantation, graft characteristics, and outcome among 241 pediatric patients who received stem cell transplantation in a single center between 1993 and 2019. In the 2010-2019, compared with the 1993-2009 period, a significantly higher 5-year overall survival (60% vs. 44%, p = .022) and an event-free survival (53% vs. 34%, p = .025) were observed. Cumulative incidence of deaths due to relapse or progression between the 1993-2009 and 2010-2019 periods were 33% and 26% respectively (p = .66). Cumulative incidence of non-relapse mortality was significantly higher during the 1993-2009 period compared with the 2010-2019 period for malignant diseases (57.7% vs. 28.3%, p = .007). The overall survival from acute graft-versus-host disease between 1993 and 2009 was 11% versus 46% between 2010 and 2019 (p = .0001). The overall survival from infection in both eras did not show any difference (p = .41). Development in transplantation technology has led to a decrease in non-relapse mortality and better control of graft-versus-host disease. However, relapse and infection remained as major causes of death. Studies evaluating institutional trends in patients undergoing HSCT and analyzing their mortality profile, can improve the management of patients, leading to a reduction in transplant-related problems.

Sections du résumé

BACKGROUND BACKGROUND
Advances in stem cell transplantation have resulted in improved outcomes.
METHODS METHODS
This is a retrospective study aimed to analyze changes in patient profile, transplantation, graft characteristics, and outcome among 241 pediatric patients who received stem cell transplantation in a single center between 1993 and 2019.
RESULTS RESULTS
In the 2010-2019, compared with the 1993-2009 period, a significantly higher 5-year overall survival (60% vs. 44%, p = .022) and an event-free survival (53% vs. 34%, p = .025) were observed. Cumulative incidence of deaths due to relapse or progression between the 1993-2009 and 2010-2019 periods were 33% and 26% respectively (p = .66). Cumulative incidence of non-relapse mortality was significantly higher during the 1993-2009 period compared with the 2010-2019 period for malignant diseases (57.7% vs. 28.3%, p = .007). The overall survival from acute graft-versus-host disease between 1993 and 2009 was 11% versus 46% between 2010 and 2019 (p = .0001). The overall survival from infection in both eras did not show any difference (p = .41).
CONCLUSIONS CONCLUSIONS
Development in transplantation technology has led to a decrease in non-relapse mortality and better control of graft-versus-host disease. However, relapse and infection remained as major causes of death. Studies evaluating institutional trends in patients undergoing HSCT and analyzing their mortality profile, can improve the management of patients, leading to a reduction in transplant-related problems.

Identifiants

pubmed: 38317340
doi: 10.1111/petr.14691
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

e14691

Informations de copyright

© 2024 Wiley Periodicals LLC.

Références

Horowitz MM. Thomas' Hematopoietic Cell Transplantation. John Wiley & Sons; 2016:6.
Passweg JR, Baldomero H, Peters C, et al. Hematopoietic SCT in Europe: data and trends in 2012 with special consideration of pediatric transplantation. Bone Marrow Transplant. 2014;49(6):744-750. doi:10.1038/bmt.2014.55
Bolon YT, Atshan R, Allbee-Johnson M, Estrada-Merly N, Lee SJ. Current Use and Outcome of Hematopoietic Stem Cell Transplantation: CIBMTR Summary Slides, 2022. https://cibmtr.org/CIBMTR/Resources/Summary-Slides-Reports
Davies SM, Ramsay NK, Klein JP, et al. Comparison of preparative regimens in transplants for children with acute lymphoblastic leukemia. J Clin Oncol. 2000;18(2):340-347. doi:10.1200/JCO.2000.18.2.340
Satwani P, Jin Z, Duffy D, et al. Transplantation-related mortality, graft failure, and survival after reduced-toxicity conditioning and allogeneic hematopoietic stem cell transplantation in 100 consecutive pediatric recipients. Biol Blood Marrow Transplant. 2013;19(4):552-561. doi:10.1016/j.bbmt.2012.12.005
Pulsipher MA, Boucher KM, Wall D, et al. Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the pediatric blood and marrow transplant consortium study ONC0313. Blood. 2009;114(7):1429-1436. doi:10.1182/blood-2009-01-196303
Bitan M, He W, Zhang MJ, et al. Transplantation for children with acute myeloid leukemia: a comparison of outcomes with reduced intensity and myeloablative regimens. Blood. 2014;123(10):1615-1620. doi:10.1182/blood-2013-10-535716
Nash RA, Johnston L, Parker P, et al. A phase I/II study of mycophenolate mofetil in combination with cyclosporine for prophylaxis of acute graft-versus-host disease after myeloablative conditioning and allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2005;11(7):495-505. doi:10.1016/j.bbmt.2005.03.006
Gagliardi TA, Milner J, Cairo MS, Steinberg A. Concomitant Ruxolitinib and Ibrutinib for Graft-Versus-Host Disease (GVHD): The First Reported Use in Pediatric Patients. Cureus. 2022;14(9):e29195. doi:10.7759/cureus.29195
DiMaggio E. Acute graft-versus-host disease: emerging insights and updates into detection, prevention, and treatment. Pharmacotherapy. 2020;40(8):788-807. doi:10.1002/phar.2436
Zeiser R, Burchert A, Lengerke C, et al. Ruxolitinib in corticosteroid-refractory graft-versus-host disease after allogeneic stem cell transplantation: a multicenter survey. Leukemia. 2015;29(10):2062-2068. doi:10.1038/leu.2015.212
Rashidi A, DeFor TE, Holtan SG, Blazar BR, Weisdorf DJ, MacMillan ML. Outcomes and predictors of response in steroid-refractory acute graft-versus-host disease. Biol Blood Marrow Transplant. 2019;25(11):2297-2302. doi:10.1016/j.bbmt.2019.07.017
Jacobsohn DA, Vogelsang GB. Anti-cytokine therapy for the treatment of graft-versus-host disease. Curr Pharm des. 2004;10(11):1195-1205. doi:10.2174/1381612043452569
Lee SJ, Klein J, Haagenson M, et al. High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation. Blood. 2007;110(13):4576-4583. doi:10.1182/blood-2007-06-097386
Jardim TAP, Jardim BA, Breda GL, Bonfim C, Raboni SM. Mortality among pediatric hematopoietic stem cell transplantation patients: report from a single center in southern Brazil. Pediatr Transplant. 2021;25(5):e13944. doi:10.1111/petr.13944
Styczyński J, Tridello G, Koster L, et al. Death after hematopoietic stem cell transplantation: changes over calendar year time, infections and associated factors. Bone Marrow Transplant. 2020;55(1):126-136. doi:10.1038/s41409-019-0624-z
Brissot E, Rialland F, Cahu X, et al. Improvement of overall survival after allogeneic hematopoietic stem cell transplantation for children and adolescents: a three-decade experience of a single institution. Bone Marrow Transplant. 2016;51(2):267-272. doi:10.1038/bmt.2015.250
Hazar V, Karasu G, Uygun V, Akcan M, Küpesiz A, Yeşilipek A. Hematopoietic stem cell transplantation activity and trends at a pediatric transplantation center in Turkey during 1998-2008. Turk J Haematol. 2012;29(2):143-149. doi:10.5505/tjh.2012.78300
Khan S, Siddiqui K, ElSolh H, et al. Outcomes of blood and marrow transplantation in children less than 2-years of age: 23 years of experience at a single center. Int J Pediatr Adolesc Med. 2022;9(4):190-195. doi:10.1016/j.ijpam.2022.09.002
Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18(4):295-304. doi:10.1097/00007890-197410000-00001
Shulman HM, Sullivan KM, Weiden PL, et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med. 1980;69(2):204-217. doi:10.1016/0002-9343(80)90380-0
Ljungman P, Bregni M, Brune M, et al. Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe 2009. Bone Marrow Transplant. 2010;45(2):219-234. doi:10.1038/bmt.2009.141
Testi AM, Moleti ML, Angi A, Bianchi S, Barberi W, Capria S. Pediatric autologous hematopoietic stem cell transplantation: safety, efficacy, and patient outcomes. Literature Review. Pediatric Health Med Ther. 2023;14:197-215.
Matthay KK, Reynolds CP, Seeger RC, et al. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: a children's oncology group study [published correction appears in J Clin Oncol. 2014 Jun 10;32(17):1862-3]. J Clin Oncol. 2009;27(7):1007-1013. doi:10.1200/JCO.2007.13.8925
Guzelgul F, Seydel GS, Aksoy K. β-Globin gene mutations in pediatric patients with β-thalassemia in the region of Çukurova. Turkey Hemoglobin. 2020;44(4):249-253. doi:10.1080/03630269.2020.1792489
Boztug H, Zecca M, Sykora KW, et al. Treosulfan-based conditioning regimens for allogeneic HSCT in children with acute lymphoblastic leukaemia. Ann Hematol. 2015;94(2):297-306. doi:10.1007/s00277-014-2196-8
Willasch AM, Peters C, Sedláček P, et al. Myeloablative conditioning for Allo-HSCT in pediatric ALL: FTBI or chemotherapy? A multicenter EBMT-PDWP study [published correction appears in bone marrow transplant. 2021 Oct;56(10):2615]. Bone Marrow Transplant. 2020;55(8):1540-1551. doi:10.1038/s41409-020-0854-0
Peters C, Dalle JH, Locatelli F, et al. Total body irradiation or chemotherapy conditioning in childhood ALL: a multinational, randomized, noninferiority phase III study. J Clin Oncol. 2021;39(4):295-307. doi:10.1200/JCO.20.02529
Mateos MK, O'Brien TA, Oswald C, et al. Transplant-related mortality following allogeneic hematopoeitic stem cell transplantation for pediatric acute lymphoblastic leukemia: 25-year retrospective review. Pediatr Blood Cancer. 2013;60(9):1520-1527. doi:10.1002/pbc.24559
Gatza E, Reddy P, Choi SW. Prevention and treatment of acute graft-versus-host disease in children, adolescents, and young adults. Biol Blood Marrow Transplant. 2020;26(5):e101-e112. doi:10.1016/j.bbmt.2020.01.004
Haroun E, Agrawal K, Leibovitch J, et al. Chronic graft-versus-host disease in pediatric patients: differences and challenges. Blood Rev. 2023;60:101054. doi:10.1016/j.blre.2023.101054
Woodard P, Tong X, Richardson S, et al. Etiology and outcome of graft failure in pediatric hematopoietic stem cell transplant recipients. J Pediatr Hematol Oncol. 2003;25(12):955-959. doi:10.1097/00043426-200312000-00010
Szmit Z, Gorczyńska E, Mielcarek-Siedziuk M, Ussowicz M, Owoc-Lempach J, Kałwak K. Veno-occlusive disease in children and adolescents after hematopoietic stem cell transplantation: did the modified Seattle criteria fit the characteristics of pediatric population? Adv Clin Exp Med. 2020;29(3):339-344. doi:10.17219/acem/115070
Corbacioglu S, Carreras E, Ansari M, et al. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European Society for Blood and Marrow Transplantation. Bone Marrow Tranplant. 2017;53(2):138-145. doi:10.1038/bmt.2017.161
Yeung TW, Chan WYK, Wong SCY, Lee PPW, Cheuk DKL, Leung W. Epidemiology, risk factors and outcome of bacterial, fungal, and viral infections in pediatric allogeneic and autologous hematopoietic stem cell transplantation recipients. Blood. 2022;140(Supplement 1):12829-12830. doi:10.1182/blood-2022-168397
Number of Syrians in Turkey December 2021. Refugeees Association. https://multeciler.org.tr/eng/number-of-syrians-in-turkey-december-2021/
Gurlek Gokcebay D, Küpeli S, Gürsel O, et al. Hematopoietic stem cell transplantation activities and HRQOL of refugee or asylum seeker children in Turkey: a multicenter study. Pediatr Transplant. 2022;26(8):e14397. doi:10.1111/petr.14397

Auteurs

Gülay Sezgin (G)

Division of Pediatric Oncology & Pediatric BMT Unit, Çukurova University Medical School, Adana, Turkey.

Yasemin Sofu Öner (YS)

Department of Pediatrics, Çukurova University Medical School, Adana, Turkey.

Gül İnan (G)

Division of Pediatric Oncology & Pediatric BMT Unit, Çukurova University Medical School, Adana, Turkey.

İlker Ünal (İ)

Department of Biostatistics, Çukurova University Medical School, Adana, Turkey.

Ayşe Özkan (A)

Division of Pediatric Oncology & Pediatric BMT Unit, Çukurova University Medical School, Adana, Turkey.

Serhan Küpeli (S)

Division of Pediatric Oncology & Pediatric BMT Unit, Çukurova University Medical School, Adana, Turkey.

İbrahim Bayram (İ)

Division of Pediatric Oncology & Pediatric BMT Unit, Çukurova University Medical School, Adana, Turkey.

Atila Tanyeli (A)

Division of Pediatric Oncology & Pediatric BMT Unit, Çukurova University Medical School, Adana, Turkey.

Classifications MeSH