Hematopoietic stem cell transplantation in infants

Area of Science:

• Pediatric Hematology
• Transplant Immunology
• Oncology

Background:

• Hematopoietic stem cell transplantation (HSCT) is infrequently performed in infants (<365 days old).
• This review examines the indications, survival rates, and long-term effects of HSCT in this pediatric population.

Purpose of the Study:

• To assess the safety and efficacy of HSCT in infants.
• To analyze survival outcomes and late effects in infants undergoing HSCT for various conditions.

Main Methods:

• A retrospective review of 1,363 pediatric HSCT cases between 1992 and 2010 at a single center.
• Analysis focused on 51 infants (<365 days old) who received either allogeneic (allo) or autologous (auto) HSCT.

Main Results:

• Seventeen infants received allo HSCT for genetic metabolic disorders, with a 70% survival rate after median follow-up of 8.9 years.
• Thirty-four infants received auto HSCT (n=10) or allo HSCT (n=24) for non-metabolic disorders, achieving a 76% overall survival rate after median follow-up of 8.7 years.
• Long-term survival was observed in 80% of infants receiving auto HSCT, with late effects within acceptable ranges.

Conclusions:

• Infant HSCT demonstrates encouraging survival rates across different transplant types and indications.
• The study supports the feasibility and positive outcomes of HSCT in very young children.
• Late effects appear manageable, warranting continued investigation in this cohort.