Gonadal function in pediatric female post-Allo-HSCT: a real-life prospective study from China.

Objective: While advances in allogeneic hematopoietic stem cell transplantation (allo-HSCT) have improved survival rates, the impact on reproductive function in Chinese female pediatric survivors remains underexplored. This study evaluated the incidence and factors influencing ovarian function impairment in these survivors.

Methods: A prospective study was conducted on 104 children who survived allo-HSCT between 2018 and 2021 at Beijing Children's Hospital. Data on hematologic disease, age, and ovarian function were collected. Statistical analyses assessed changes in sex hormone levels and identified factors linked to gonadal damage.

Results: Post-transplant, follicle-stimulating hormone levels rose significantly (p = 0.013 for girls ≥10 years old), while anti-Müllerian hormone levels decreased (p < 0.001). The incidence of premature ovarian insufficiency (POI) was 26.92% at 1 year, 47.44% at 2 years, and 70.18% at 3 years post-transplant. Risk factors for POI included transplantation age ≥10 years, weight >20 kg at transplantation, myeloablative conditioning, and total body irradiation (OR = 6.76, p = 0.005; OR = 6.63, p = 0.003; OR = 17, p = 0.017; OR = 15.03, p = 0.026). Only 3.85% of patients received ovarian function protection before transplantation. Among patients aged ≥12 years at follow-up, 86.36% developed POI, with 26.32% receiving oral estrogen replacement therapy.

Conclusions: Hypogonadal function significantly declines in pediatric patients after allo-HSCT. Fertility protection and hormone replacement therapy were rarely used.