Growth and Endocrinological Outcomes in Patients Who Underwent or Did Not Undergo Haematopoietic Stem Cell Transplantation in Prepubertal Age
Overview
- Phase
- Not Applicable
- Status
- Not yet recruiting
- Enrollment
- 300
- Locations
- 1
- Primary Endpoint
- To assess whether pediatric oncology patients, who underwent or did not undergo prepubertal HSCT, achieve a final height (FH) within their target height (TH) range.
Overview
Brief Summary
This observational study evaluates growth and endocrine outcomes in pediatric oncology patients who underwent prepubertal HSCT compared to those who did not. The study focuses on final height, pubertal growth spurt, and sex hormone production, with data collected retrospectively and prospectively through standard clinical follow-up.
Detailed Description
Hematopoietic stem cell transplantation (HSCT) is a key treatment for pediatric oncology patients but may lead to long-term endocrine and growth complications, including thyroid dysfunction, adrenal insufficiency, impaired bone mineral density, growth deficits, and gonadal dysfunction. This observational cohort study aims to describe growth and endocrine outcomes at specific pubertal stages in pediatric oncology patients who underwent prepubertal HSCT, compared to those who did not receive HSCT in the same age period. Primary objectives include assessing attainment of final height relative to target height, evaluating pubertal growth spurt contribution, and quantifying pubertal sex hormone production (testosterone in males, estradiol in females). Secondary objectives include comparing auxological and endocrine parameters between subgroups that did or did not reach target height, and evaluating the impact of prepubertal exposure to gonadotoxic chemotherapy on growth outcomes. Data will be collected retrospectively and prospectively at key developmental time points according to standard clinical follow-up, without study-specific interventions.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Other
Eligibility Criteria
- Ages
- 5 Years to 40 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Prepubertal HSCT Group - Pediatric Oncology Patients
- •HSCT performed for pediatric oncology between January 2005 and December 2020;
- •Pubertal development not yet initiated at the time of HSCT (testicular volume \< 4 ml bilaterally, uterine volume \< 2.5 ml).
- •Non-HSCT Group - Pediatric Oncology Patients
- •Diagnosis of pediatric oncology between January 2005 and December 2020;
- •No HSCT performed before the onset of pubertal development (testicular volume \< 4 ml bilaterally, uterine volume \< 2.5 ml) during the same period.
- •For Both Groups
- •Received prepubertal chemotherapy including at least one of the following agents: cyclophosphamide, ifosfamide, procarbazine, cisplatin, carboplatin, melphalan, thiotepa, busulfan, treosulfan;
- •Spontaneous pubertal development with autonomous progression, without the need for exogenous hormone therapy (testosterone or estradiol);
- •Endocrinological follow-up to assess growth progression conducted until the end of pubertal development at the Endocrine-Metabolic Diseases Program, UOC Pediatrics, IRCCS AOUBo;
Exclusion Criteria
- •Delayed puberty (testicular volume \< 4 ml at age \> 14 years or Tanner stage 2 breast development at age \> 13 years) requiring exogenous hormone therapy with testosterone or estradiol;
- •For pediatric oncology patients not undergoing prepubertal HSCT, having received HSCT during pubertal development.
Outcomes
Primary Outcomes
To assess whether pediatric oncology patients, who underwent or did not undergo prepubertal HSCT, achieve a final height (FH) within their target height (TH) range.
Time Frame: Through study completion, an average of 10.5 years
Height (cm, SDS)
To assess whether the pubertal growth spurt, defined as the phase of maximum growth velocity, provides a sufficient contribution for pediatric oncology patients, who underwent or did not undergo prepubertal HSCT, to achieve their target height (TH).
Time Frame: Through study completion, an average of 10.5 years
Height (cm, SDS)
To quantify pubertal testosterone or estradiol production in pediatric oncology patients, who underwent or did not undergo prepubertal HSCT, in order to assess its adequacy in supporting linear growth.
Time Frame: Through study completion, an average of 10.5 years
Height (cm, SDS)
Secondary Outcomes
- To describe the impact of chemotherapy on growth outcomes in pediatric oncology patients who underwent or did not undergo prepubertal HSCT.(Through study completion, an average of 10.5 years)
Investigators
Federico Baronio
MD
IRCCS Azienda Ospedaliero-Universitaria di Bologna