Pubertal outcomes of children transplanted with allogeneic stem cells after myeloablative total body irradiation or busulfan: Influence of age and sex is confirmed, while a role of chronic graft‐versus‐host disease in delayed puberty onset is revealed. (23rd July 2020)

• Record Type: Journal Article
• Authors: Weinhard, Sara; Wiedemann, Arnaud; Leheup, Bruno; Dalle, Jean‐Hugues; Lebon Labich, Béatrice; Pochon, Cécile
• Is Part Of: Pediatric transplantation. Volume 24:Number 6(2020)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2020-07-23
• Subjects: female sex -- graft‐versus‐host disease -- hematopoietic stem cell transplantation -- myeloablative conditioning regimen -- pubertal development; Transplantation of organs, tissues, etc. in children -- Periodicals; 617.95408305
• DOI: 10.1111/petr.13773
• Languages: English
• ISSNs: 1397-3142

Abstract

Abstract: Introduction: Myeloablative conditioning before allogeneic HSCT during childhood exposes to serious long‐term complications, especially gonadal dysfunction. Pubertal issues are less described than other post‐HSCT sequelae in childhood. Methods: Pubertal development and biological gonadal parameters were assessed in a retrospective monocentric cohort of prepubertal patients who underwent HSCT after myeloablative conditioning with TBI or busulfan between 1981 and 2017. Results: Seventy‐four patients (28 girls and 46 boys) were included. No spontaneous pubertal development was found in 50% of girls and 10% of boys ( P < .001), and delayed puberty or no spontaneous pubertal development was found in 57% of girls and 24% of boys ( P = .009). HRT was used in 82% of girls and 24% of boys ( P < .001). In univariate analysis, TBI conditioning ( P = .05), female sex ( P < .001), acute GVHD ( P = .05), extensive chronic GVHD ( P = .021), steroid treatment >6 months ( P = .016), and malignant diseases ( P = .016) were associated with no spontaneous pubertal development, whereas TBI conditioning ( P = .003) and extensive chronic GVHD ( P = .005) were associated with delayed puberty. In multivariate analysis, factors independently associated with no spontaneous puberty onset were female sex ( P = .001) and age >10 years ( P = .033). Factors independently associated with delayed puberty were extensive chronic GVHD ( P = .041) and age >10 years ( P = .031). Conclusion: This study highlighted the toxicity of MAC in prepubescent children: TBI did worse, but this was especially true for the most susceptible patients (girls, leukemic patients, and patients older than 10 years). It suggests a possible role of GVHD in delayed puberty.