Gonadal function after Busulfan compared to Treosulfan in children and adolescents undergoing allogeneic hematopoietic stem cell transplantation. On Behalf of the Pediatric and Transplant Complications Working Parties of EBMT

Maura Faraci, Tamara Diesch, Myriam Labopin, Arnaud Dalissier, Arian Lankester, Andrew Gennery, Mikael Sundin, Duygu Uckan-Cetinkaya, Marc Bierings, Anke M J Peters, Martina Garwer, Ansgar Schulz, Gerard Michel, Giovanna Giorgiani, Bernd Gruhn, Franco Locatelli, Stefano Giardino, Anne Uyttebroeck, Fanny Rialland, Maija Itäla-RemesPeter Dreger, Peter J Shaw, Victoria Bordon, Paul G Schlegel, Karin Mellgren, Jose M Moraleda, Katharine Patrick, Pascale Schneider, Charlotte Jubert, Anita Lawitschka, Nina Salooja, Grzegorz W Basak, Selim Corbaciuoglu, Rafael Duarte, Peter Bader

Research output: Contribution to journalArticle

Abstract

INTRODUCTION: Gonadal impairment is an important late effect having a significant impact on quality of life of transplanted patients. The aim of this study was to compare gonadal function after Busulfan (Bu) or Treosulfan (Treo) conditioning regimens in pre and post-pubertal children.

MATERIAL AND METHODS: This is a retrospective, multicenter study including children transplanted in pediatric European Society for Blood and Marrow Transplantation (EBMT) centers between 1992 and 2012 who did not receive gonadotoxic chemo-radiotherapy before the transplant.

RESULTS: We evaluated 137 patients transplanted in 25 pediatric EBMT centers. Median age at transplant was 11.04 years (range 5-18); 89 patients were males and 48 were females. Eighty-nine patients were pre-pubertal at transplant, while 48 were post-pubertal. One hundred eighteen children received Bu and 19 Treo. A higher proportion of females treated with Treo in pre-pubertal stage reached spontaneous puberty compared to those treated with Bu (p=0.02). Spontaneous menarche was more frequent after Treo than after Bu (p< 0.001). Post-pubertal males and females treated with Treo had significantly lower luteinizing hormone (LH) levels (p=0.03 and p=0.04, respectively) compared to the Bu group.

CONCLUSIONS: Frequency of gonadal damage associated with Treo was significantly lower than that observed after Bu. These results need to be confirmed in a larger population.

Original languageEnglish
JournalBiology of Blood and Marrow Transplantation
DOIs
Publication statusE-pub ahead of print - May 10 2019

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