High-dose therapy and autologous hematopoietic cel transplantation rescue in children with neuroblastoma and Ewing sarcoma
Keywordshematopoietic stem cell transplantation, high-dose therapy, children, neurolastoma, Ewing sarcoma
Background. High-dose therapy (HDT) with autologous stem cell rescue has been recently applied in very-poor-risk pediatric solid tumors. Promising data have become available with the use of high-dose busulfan in neuroblastoma (NBL) and Ewing sarcoma (ES), and with high-dose treosulfan in ES. HDT approach resulted in an encouraging outcome without toxic mortality for high-risk patients.
Objective. The objective of this study is to present transplant outcomes, that is disease-free-survival and overall survival in children with high-risk NBL and ES undergoing auto-HSCT.
Patients and methods. A total number of 47 NBL and 20 ES auto-HSCT performed between 2004 and 2016 in a single transplant center were included in this analysis.
Results. Probability of 3-years pOS was 0.79±0.06 and 0.46±0.14 for NBL and ES patients, respectively. Relapse incidence at 3 years after HSCT was 0.37±0.08 and 0.26±0.11 for NBL and ES patients, respectively. The number of relapses at 3 years after HSCT was 15/47 in NBL and 6/20 in ES. Busulfan-based vs treosulfan-based conditioning in ES patients resulted in lower relapse and death rates. NBL and ES patients transplanted in complete remission (CR1) had lower relapse rates and lower death rates than patients at CR>1.
Conclusion. Obtained results of auto-HSCT confirm the therapeutic benefit for children with NBL and ES. Recent reports on current practice of HSCT in Europe indicate HDT with auto-HSCT as a standard of care in pediatric patients with high risk or relapsed NBL and ES.
Jahnukainen K, Kallio P, Koivusalo A, Saarinen-Pihkala UM: High-dose thiotepa as consolidation therapy with autologous hematopoietic stem cell transplantation for high-risk Ewing family tumors: Single-institution experience. J Pediatr Hematol Oncol 2015;37:536-542.
Morgenstern DA, London WB, Stephens D, Volchenboum SL, Simon T, Nakagawara A, Shimada H, Schleiermacher G, Matthay KK, Cohn SL, Pearson AD, Irwin MS: Prognostic significance of pattern and burden of metastatic disease in patients with stage 4 neuroblastoma: A study from the International Neuroblastoma Risk Group database. Eur J Cancer 2016;65:1-10.
Yalcin B, Kremer LC, van Dalen EC: High-dose chemotherapy and autologous haematopoietic stem cell rescue for children with high-risk neuroblastoma. Cochrane Database Syst Rev 2015:CD006301.
Diaz-Beveridge R, Lorente D, Torres B, Canete A, Rodrigo E, Bruixola G, Berlanga P, Reche E, Montalar J, Verdeguer A, Aparicio J: Multimodality treatment of pediatric and adult patients with ewing sarcoma: A single-institution experience. J Pediatr Hematol Oncol 2015;37:e278-284.
Styczyński J, Dębski R, Krenska A, Górnicka H, Hulek E, Wojtylak P, Windorbska W, Drzewiecka B, Ostrowski K, Tujakowski J, Marciniak L, Kusza K, Goc M, Prokurat A, Chrupek M, Urasiński T, Peregud-Pogorzelski J, Ociepa T, Krawczuk-Rybak M, Leszczyńska E, Balcerska A, Niedźwiecki M, M. W: Transplantacje komórek hematopoetycznych w świetle 5-letnich doświadczeń Med Biol Sci 2008;22:157-163.
Styczynski J, Reusser P, Einsele H, de la Camara R, Cordonnier C, Ward KN, Ljungman P, Engelhard D: Management of HSV, VZV and EBV infections in patients with hematological malignancies and after SCT: Guidelines from the Second European Conference on Infections in Leukemia. Bone Marrow Transplant 2009;43:757-770.
Styczynski J, Gil L: Prevention of infectious complications in pediatric HSCT. Bone Marrow Transplant 2008;42 Suppl 2:S77-81.
Ljungman P, de la Camara R, Cordonnier C, Einsele H, Engelhard D, Reusser P, Styczynski J, Ward K: Management of CMV, HHV-6, HHV-7 and Kposi-Srcoma Hrpesvirus (HHV-8) infections in patients with hematological malignancies and after SCT. Bone Marrow Transplant 2008;42:227-240.
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;47:1530-1534.
Hale GA, Arora M, Ahn KW, He W, Camitta B, Bishop MR, Bitan M, Cairo MS, Chan K, Childs RW, Copelan E, Davies SM, Perez MA, Doyle JJ, Gale RP, Vicent MG, Horn BN, Hussein AA, Jodele S, Kamani NR, Kasow KA, Kletzel M, Lazarus HM, Lewis VA, Myers KC, Olsson R, Pulsipher M, Qayed M, Sanders JE, Shaw PJ, Soni S, Stiff PJ, Stadtmauer EA, Ueno NT, Wall DA, Grupp SA: Allogeneic hematopoietic cell transplantation for neuroblastoma: The CIBMTR experience. Bone Marrow Transplant 2013;48:1056-1064.
Park JR, Bagatell R, London WB, Maris JM, Cohn SL, Mattay KK, Hogarty M: Children's Oncology Group's 2013 blueprint for research: Neuroblastoma. Pediatr Blood Cancer 2013;60:985-993.
Luksch R, Tienghi A, Hall KS, Fagioli F, Picci P, Barbieri E, Gandola L, Eriksson M, Ruggieri P, Daolio P, Lindholm P, Prete A, Bisogno G, Tamburini A, Grignani G, Abate ME, Podda M, Smeland S, Ferrari S: Primary metastatic Ewing's family tumors: Results of the Italian Sarcoma Group and Scandinavian Sarcoma Group ISG/SSG iv study including myeloablative chemotherapy and total-lung irradiation. Ann Oncol 2012;23:2970-2976.
Passweg JR, Baldomero H, Peters C, Gaspar HB, Cesaro S, Dreger P, Duarte RF, Falkenburg JH, Farge-Bancel D, Gennery A, Halter J, Kroger N, Lanza F, Marsh J, Mohty M, Sureda A, Velardi A, Madrigal A: Hematopoietic SCT in Europe: Data and trends in 2012 with special consideration of pediatric transplantation. Bone Marrow Transplant 2014;49:744-750.
Sureda A, Bader P, Cesaro S, Dreger P, Duarte RF, Dufour C, Falkenburg JH, Farge-Bancel D, Gennery A, Kroger N, Lanza F, Marsh JC, Nagler A, Peters C, Velardi A, Mohty M, Madrigal A: Indications for allo- and auto-SCT for haematological diseases, solid tumours and immune disorders: Current practice in Europe, 2015. Bone Marrow Transplant 2015;50:1037-1056.
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