Consensus in stem cell transplantation for pediatric solid tumors
DOI:
https://doi.org/10.46765/2675-374X.2025v6n1e305Keywords:
Stem Cell Transplantation, Ewing Sarcoma, Wilms Tumor, Solid tumors, ChildAbstract
Solid tumors in the pediatric population are preferred treated in clinical national or international protocols, in which the work of pediatric oncology cooperative groups guides the therapeutic standards and advancements. The role of high-dose chemotherapy (HDC) and hematopoietic cell transplant (HCT) in the pediatric population is usually reserved for patients or subgroups of patients who have diseases with poor prognosis and sometimes are refractory to the best available treatment, in combination to conventional chemotherapy, surgical procedure, and radiation. The Pediatric Group of the Brazilian Society of Bone Marrow Transplantation and Cellular Therapy has revised its current consensus, providing updated recommendations for indications and conditioning protocols. This updated guidance emphasizes that, while HDC/HCT is an important tool for specific high-risk malignancies, several tumors continue to have no indication for autologous transplantation based on current evidence.
References
1. Snowden JA, Sánchez-Ortega I, Corbacioglu S, Basak GW, Chabannon C, de la Camara R, Dolstra H, Duarte RF, Glass B, Greco R, Lankester AC, Mohty M, Neven B, de Latour RP, Pedrazzoli P, Peric Z, Yakoub-Agha I, Sureda A, Kröger N; European Society for Blood and Marrow Transplantation (EBMT). Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022. Bone Marrow Transplant. 2022;57(8):1217–39. https://doi.org/10.1038/s41409-022-01691-w DOI: https://doi.org/10.1038/s41409-022-01691-w
2. Mello CA, Campos FAB, Santos TG, Silva MLG, Torrezan GT, Costa FD, Formiga MN, Nicolau U, Nascimento AG, Silva C, Curado MP, Nakagawa SA, Lopes A, Aguiar S Jr. Desmoplastic small round cell tumor: a review of main molecular abnormalities and emerging therapy. Cancers (Basel). 2021;13(3):498. https://doi.org/10.3390/cancers13030498 DOI: https://doi.org/10.3390/cancers13030498
3. Hovsepyan S, Giani C, Pasquali S, Di Giannatale A, Chiaravalli S, Colombo C, Orbach D, Bergamaschi L, Vennarini S, Gatz SA, Gasparini P, Berlanga P, Casanova M, Ferrari A. Desmoplastic small round cell tumor: from state of the art to future clinical prospects. Expert Rev Anticancer Ther. 2023;23(5):471–84. https://doi.org/10.1080/14737140.2023.2200171 DOI: https://doi.org/10.1080/14737140.2023.2200171
4. Stiles ZE, Dickson PV, Glazer ES, Murphy AJ, Davidoff AM, Behrman SW, Bishop MW, Martin MG, Deneve JL. Desmoplastic small round cell tumor: A nationwide study of a rare sarcoma. J Surg Oncol. 2018;117(8):1759–67. https://doi.org/10.1002/jso.25071 DOI: https://doi.org/10.1002/jso.25071
5. Bulbul A, Fahy BN, Xiu J, Rashad S, Mustafa A, Husain H, Hayes-Jordan A. Desmoplastic small round blue cell tumor: a review of treatment and potential therapeutic genomic alterations. Sarcoma. 2017;2017:1278268. https://doi.org/10.1155/2017/1278268 DOI: https://doi.org/10.1155/2017/1278268
6. Cook RJ, Wang Z, Arora M, Lazarus HM, Kasow KA, Champagne MA, Saber W, van Besien KM, Hale GA, Copelan EA, Elmongy M, Ueno NT, Horn BN, Slavin S, Bishop MR, Stadtmauer EA. Clinical outcomes of patients with desmoplastic small round cell tumor of the peritoneum undergoing autologous HCT: a CIBMTR retrospective analysis. Bone Marrow Transplant. 2012;47(11):1455–8. https://doi.org/10.1038/bmt.2012.57 DOI: https://doi.org/10.1038/bmt.2012.57
7. Gupta A, Riedel RF, Shah C, Borinstein SC, Isakoff MS, Chugh R, Rosenblum JM, Murphy ES, Campbell SR, Albert CM, Zahler S, Thomas SM, Trucco M. Consensus recommendations in the management of Ewing sarcoma from the National Ewing Sarcoma Tumor Board. Cancer. 2023;129(21):3363–71. https://doi.org/10.1002/cncr.34942 DOI: https://doi.org/10.1002/cncr.34942
8. Dirksen U, Brennan B, Le Deley MC, Cozic N, van den Berg H, Bhadri V, Brichard B, Claude L, Craft A, Amler S, Gaspar N, Gelderblom H, Goldsby R, Gorlick R, Grier HE, Guinbretiere JM, Hauser P, Hjorth L, Janeway K, Juergens H, Judson I, Krailo M, Kruseova J, Kuehne T, Ladenstein R, Lervat C, Lessnick SL, Lewis I, Linassier C, Marec-Berard P, Marina N, Morland B, Pacquement H, Paulussen M, Randall RL, Ranft A, Le Teuff G, Wheatley K, Whelan J, Womer R, Oberlin O, Hawkins DS; Euro-E.W.I.N.G. 99 and Ewing 2008 Investigators. High-dose chemotherapy compared with standard chemotherapy and lung radiation in Ewing sarcoma with pulmonary metastases: results of the European Ewing Tumour Working Initiative of National Groups, 99 and EWING 2008. J Clin Oncol. 2019;37(34):3129–202. https://doi.org/10.1200/jco.19.00915 DOI: https://doi.org/10.1200/JCO.19.00915
9. Whelan J, Le Deley MC, Dirksen U, Le Teuff G, Brennan B, Gaspar N, Hawkins DS, Amler S, Bauer S, Bielack S, Blay JY, Burdach S, Castex MP, Dilloo D, Eggert A, Gelderblom H, Gentet JC, Hartmann W, Hassenpflug WA, Hjorth L, Jimenez M, Klingebiel T, Kontny U, Kruseova J, Ladenstein R, Laurence V, Lervat C, Marec-Berard P, Marreaud S, Michon J, Morland B, Paulussen M, Ranft A, Reichardt P, van den Berg H, Wheatley K, Judson I, Lewis I, Craft A, Juergens H, Oberlin O. High-dose chemotherapy and blood autologous stem-cell rescue compared with standard chemotherapy in localized high-risk Ewing sarcoma: results of Euro-E.W.I.N.G.99 and Ewing-2008. J Clin Oncol. 2018;36(31):3110–9. https://doi.org/10.1200/JCO.2018.78.2516 DOI: https://doi.org/10.1200/JCO.2018.78.2516
10. Drabko K, Raciborska A, Bilska K, Styczynski J, Ussowicz M, Choma M, Wojcik B, Zaucha-Prazmo A, Gorczynska E, Skoczen S, Wozniak W, Chybicka A, Wysocki M, Gozdzik J, Kowalczyk J. Consolidation of first-line therapy with busulphan and melphalan, and autologous stem cell rescue in children with Ewing’s sarcoma. Bone Marrow Transplant. 2012;47(12):1530–4. https://doi.org/10.1038/bmt.2012.78 DOI: https://doi.org/10.1038/bmt.2012.78
11. Choderlos de Laclosa X, Risbourg S, Brennan B, Bertucci F, Gaspar N, Gelderblom H, Hawkins DS, Janeway K, Juergens H, Kasper B, Krailo MD, Le Deley MC, Marec-Bérard P, McCabe MG, Metzler M, Ranft A, Strauss S, Tabone MD, Windsor R, Dirksen U, Gandemer V. Impact of age on safety of Busulfan Melphalan followed by autologous hematopoietic stem-cell transplantation versus standard chemotherapy in the patients of the EURO-E.W.I.N.G. 99 and Ewing 2008 clinical trials. Eur J Cancer. 2024;208:114229. https://doi.org/10.1016/j.ejca.2024.114229 DOI: https://doi.org/10.1016/j.ejca.2024.114229
12. Koch R, Gelderblom H, Haveman L, Brichard B, Jürgens H, Cyprova S, van den Berg H, Hassenpflug W, Raciborska A, Ek T, Baumhoer D, Egerer G, Eich HT, Renard M, Hauser P, Burdach S, Bovee J, Bonar F, Reichardt P, Kruseova J, Hardes J, Kühne T, Kessler T, Collaud S, Bernkopf M, Butterfaß-Bahloul T, Dhooge C, Bauer S, Kiss J, Paulussen M, Hong A, Ranft A, Timmermann B, Rascon J, Vieth V, Kanerva J, Faldum A, Metzler M, Hartmann W, Hjorth L, Bhadri V, Dirksen U. High‐dose treosulfan and melphalan as consolidation therapy versus standard therapy for high‐risk (metastatic) Ewing sarcoma. J Clin Oncol. 2022;40(21):2307–20. https://doi.org/10.1200/jco.21.01942 DOI: https://doi.org/10.1200/JCO.21.01942
13. Gupta A, Dietz MS, Riedel RF, Dhir A, Borinstein SC, Isakoff MS, Aye JM, Rainusso N, Armstrong AE, DuBois SG, Wagner LM, Rosenblum JM, Cohen-Gogo S, Albert CM, Zahler S, Chugh R, Trucco M. Consensus recommendations for systemic therapies in the management of relapsed Ewing sarcoma: A report from the National Ewing Sarcoma Tumor Board. Cancer. 2024;130(23):4028–39. https://doi.org/10.1002/cncr.35537 DOI: https://doi.org/10.1002/cncr.35537
14. Windsor R, Hamilton A, McTiernan A, Dileo P, Michelagnoli M, Seddon B, Strauss SJ, Whelan J. Survival after high-dose chemotherapy for refractory and recurrent Ewing sarcoma. Eur J Cancer. 2022;170:131–9. https://doi.org/10.1016/j.ejca.2022.04.007 DOI: https://doi.org/10.1016/j.ejca.2022.04.007
15. Presson A, Moore TB, Kempert P. Efficacy of high-dose chemotherapy and autologous stem cell transplant for recurrent Wilms’ tumor: a meta-analysis. J Pediatr Hematol Oncol. 2010;32(6): 454–61. https://doi. org/10.1097/mph.0b013e3181e001c2 DOI: https://doi.org/10.1097/MPH.0b013e3181e001c2
16. Malogolowkin MH, Hemmer MT, Le-Rademacher J, Hale GA, Mehta PA, Smith AR, Kitko C, Abraham A, Abdel-Azim H, Dandoy C, Angel Diaz M, Gale RP, Guilcher G, Hayashi R, Jodele S, Kasow KA, MacMillian ML, Thakar M, Wirk BM, Woolfrey A, Thiel EL. Outcomes following autologous hematopoietic stem cell transplant for patients with relapsed Wilms’ tumor: a CIBMTR retrospective analysis. Bone Marrow Transplant. 2017;52(11):1549–55. https://doi.org/10.1038/bmt.2017.178 DOI: https://doi.org/10.1038/bmt.2017.178
17. Ha TC, Spreafico F, Graf N, Dallorso S, Dome JS, Malogolowkin M, Furtwängler R, Hale JP, Moroz V, Machin D, Pritchard-Jones K. An international strategy to determine the role of high dose therapy in recurrent Wilms’ tumour. Eur J Cancer. 2013;49(1):194–210. https://doi.org/10.1016/j.ejca.2012.07.010 DOI: https://doi.org/10.1016/j.ejca.2012.07.010
18. Spreafico F, Dalissier A, Pötschger U, Locatelli F, Michon JM, Peters C, Bader P, Bisogno G, Yeomanson D, Willasch A, van den Heuvel Eibrink M, Graf N, Dallorso S; EBMT Paediatric Diseases Working Party. High dose chemotherapy and autologous hematopoietic cell transplantation for Wilms tumor: a study of the European Society for Blood and Marrow Transplantation. Bone Marrow Transplant. 2020;55(2):376–83. https://doi.org/10.1038/s41409-019-0661-7 DOI: https://doi.org/10.1038/s41409-019-0661-7
19. Kobayashi R, Inoue M, Takahashi Y, Kikuta A, Ogawa A, Koga Y, Koh K, Hashii Y, Hara J, Kato K, Tabuchi K, Matsumoto K. Autologous stem cell transplantation for children with renal tumors, and adults with Wilms tumor: retrospective analysis of the Japanese Transplant Registry Unified Management Program. J Pediatr Hematol Oncol. 2020;42(4):251–5. https://doi.org/10.1097/mph.0000000000001779 DOI: https://doi.org/10.1097/MPH.0000000000001779
20. Garaventa A, Hartmann O, Bernard JL, Zucker JM, Pardo N, Castel V, Dallorso S, Adelbost Z, Ladenstein R, Chauvin F, Phillip T. Autologous bone marrow transplantation for pediatric Wilms’ tumor: the experience of the European Bone Marrow Transplantation Solid Tumor Registry. Med Pediatr Oncol. 1994;22(1):11–4. https://doi.org/10.1002/mpo.2950220103 DOI: https://doi.org/10.1002/mpo.2950220103
21. Hale J, Hobson R, Moroz V, Sartori P. Results of UK Children’s Cancer and Leukemia Group (CCLG) protocol for relapsed Wilms tumor (UKWR): unified relapse strategy improves outcome. 40th meeting of International Society of Paediatric Oncology. 2008.
22. Ortiz MV, Koenig C, Armstrong AE, Brok J, de Camargo B, Mavinkurve-Groothuis AMC, Herrera TBV, Venkatramani R, Woods AD, Dome JS, Spreafico F. Advances in the clinical management of high-risk Wilms tumors. Pediatr Blood Cancer. 2023;70(3):e30153. https://doi.org/10.1002/pbc.30153 DOI: https://doi.org/10.1002/pbc.30153
23. Castro Jr CG. Consensus on indications for hematopoietic stem cell transplantation in pediatrics. Update 2020: Sarcomas, Ewing Family Tumor, Osteosarcoma and Hepatoblastoma. J Bone Marrow Transplant Cell Ther. 2021;2(4):143. https://doi.org/10.46765/2675-374X.2021v2n2p143 DOI: https://doi.org/10.46765/2675-374X.2021v2n4p143
24. Brok J, Ramírez-Villar GL. Treatment of renal tumours: a summary of the SIOP-RTSG 2016 Umbrella Guidelines (Version II). SIOP-Europe. Available at: https://siope.eu/media/documents/escp-treatment-ofrenal-tumours.pdf
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