Reduced final height in boys after allogeneic hematopoietic stem cell transplantation (HSCT) for childhood cancer: does pubertal growth spurt matters?

Vittorio Ferrari; Erika Cantarelli; Davide Leardini; Alessia Brandi; Rita Ortolano; Federico Baronio

P1-263

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ESPE Abstracts (2024) 98 P1-263

ESPE2024 Poster Category 1 Growth and Syndromes 4 (9 abstracts)

Reduced final height in boys after allogeneic hematopoietic stem cell transplantation (HSCT) for childhood cancer: does pubertal growth spurt matters?

Vittorio Ferrari ¹ , Erika Cantarelli ¹ , Davide Leardini ² , Alessia Brandi ² , Rita Ortolano ¹ & Federico Baronio ¹

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¹Department Hospital of Woman and Child, Pediatric Unit, IRCCS AOU di Bologna Policlinico di S. Orsola, Endo-ERN Center for Rare Endocrine Conditions, Bologna, Italy. ²Department Hospital of Woman and Child, Pediatric Hematology and Oncology Unit, IRCCS AOU di Bologna Policlinico di S. Orsola, Bologna, Italy

Introduction: Growth failure is a side effect of HSCT in children. This study aims to evaluate the growth trajectory in a series of boys who underwent allogeneic HSCT for childhood cancer in pre-puberty and reached final height (FH).

Methods: We evaluated auxological parameters, mid-parental target height (TH), serum testosterone, and insulin-like growth factor 1 (IGF-1 SDS) levels of 19 boys who underwent allogeneic HSCT with Busulphan-based conditioning regimens in all cases but two, who underwent total body irradiation. The time point evaluations considered were at the first post-HSCT visit, the beginning of puberty (serum Testosterone > 0.24 ng/ml) and FH. We subdivided patients (pts) into two groups according to ΔFH-TH SDS: Group 1 (ΔFH-TH≥-1 SDS) and Group 2 (ΔFH-TH<-1 SDS).

Results: Overall, the mean age at HSCT was 5.3 years ± 2.9, and the mean ΔFH-TH was -1.3±0.9 SDS. 17/19 pts entered puberty spontaneously between 9.9-13.8 years. The mean pubertal tempo was 4.8 years±1.6, and mean testosterone levels during puberty were 3.0 ng/ml ±1.03. 13/19 pts (68%) reached FH below TH [Group 1, (-2.1±0.9 SDS), whereas 6/19 (32%) pts near or above TH [Group 2 (-0.2±0.7 SDS)]. The mean ΔFH-TH was -1.8±0.6 SDS in Group 1 and -0.2 ±0.3 SDS in Group 2 (P = <0.001). No significant differences between the two groups were found for age at HSCT, mean pubertal tempo, serum Testosterone during growth spurt, BMI SDS, and IGF-1 SDS; although not significant, growth spurt was different in the two groups [group 1: 16.5 cm ±7.4; group 2: 23.3 cm±7.9 (P = 0.10)]. The prepubertal height trend (measured as the difference between height SDS at the start of puberty and height SDS at the first visit post-HSCT) showed a regular trajectory without significant differences among the 2 groups. The growth spurt significantly differed between Group 1(-1.0 ±0.7 SDS) and Group 2 (-0.2 ±0.5 SDS) P = 0.035. Linear regression analysis highlighted that ΔFH-TH was significantly explained by ΔSDS growth spurt (β= 0.754; P = 0.006).

Conclusions: The majority of our pts patients showed FH below TH. Significant impairment of growth spurt is the leading cause of reduced FH in our cases without significant other associated clinical or biochemical features. More extensive cases are needed to confirm our results and better characterize these patients to maximize their growth potential during puberty.