Objective ：To analyze the growth, development and endocrine function of children with primary immunodeficiency disease after hematopoietic stem cell transplantation.

Methods ：214 patients diagnosed with primary immunodeficiency disease were selected. All patients met the indications for hematopoietic stem cell transplantation. Children after hematopoietic stem cell transplantation were tested for height, weight, endocrine hormones and secondary * characteristics.

Results ：After testing, the height of 3 children exceeded 2 standard deviations or more, the height of 110 patients was within the normal range, and the height of 30 patients was below 2 standard deviations. The BMI index was lower than normal in 24 cases, higher than normal in 11 cases, and the rest were within the normal range. The sex hormone test of female patients was basically inconsistent with age and Tanner stage. Female patients were divided into three groups, namely, pre-pubertal group, adolescent group, and post-pubertal group. It was found that 59% of patients in the pre-pubertal group had spontaneous puberty development, 47% of patients had spontaneous menarche, and 31% of patients in the adolescent group had spontaneous menarche.

Conclusion： Preconditioning plan before transplantation and patient age at the time of transplantation are crucial. Busulfan, high-dose cyclophosphamide and radiotherapy significantly affect the occurrence of spontaneous puberty and spontaneous menstruation.

Keywords ：growth and development; endocrine function; hematopoietic stem cell transplantation; primary immunodeficiency disease;

This content is only available as a PDF.
2025
Sign in via your Institution