Long-term Consequences of Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Patients with Primary Immunodeficiencies
Overview
This systematic review identifies and summarizes late effects following allogeneic HSCT in pediatric patients with primary immunodeficiencies (PIDs).
Background
Allogeneic hematopoietic stem cell transplantation (HSCT) has improved survival in children with primary immunodeficiencies (PIDs). As survival rates improve, attention has shifted to the late effects (LEs) that can emerge years after transplantation.
Data Highlights
Late Effect
Number of Patients
Percentage
Growth and Developmental Delay
280
12.2%
Neurological Issues
173
7.5%
Autoimmune or Hematologic Issues
161
7.0%
Pulmonary Complications
150
6.5%
Skin Complications
150
6.5%
Dental and Skeletal Impairments
112
4.9%
Endocrine Issues
99
4.3%
Gastrointestinal or Liver Impairments
75
3.3%
Key Findings
24% of pediatric HSCT survivors with PIDs reported at least one late effect.
Growth and developmental delays were the most frequently reported late effects, affecting 12.2% of patients.
Neurological issues were noted in 7.5% of patients.
Autoimmune or hematologic complications occurred in 7.0% of patients.
Pulmonary and skin complications were each reported in 6.5% of patients.
Endocrine issues were documented in 4.3% of patients.
Clinical Implications
The findings emphasize the importance of long-term monitoring for pediatric HSCT survivors to identify and manage late effects effectively. A multidisciplinary follow-up approach is essential to optimize health outcomes and quality of life for these patients.
Conclusion
A significant proportion of pediatric HSCT survivors with PIDs experience late effects that can impact various organ systems.
