Hematopoietic Stem Cell Transplantation for Children with Inborn Errors of Metabolism: Single Center Experience over Two Decades

Key Points

• Hematopoietic stem cell transplantation shows improved long-term outcomes for children with inborn errors of metabolism.
• The study analyzed 20 years of data, highlighting success rates and complications in this specific patient population.
• A comprehensive approach was taken to assess the transplantation protocol and patient recovery process over two decades.
• These findings support the need for larger studies to further validate the effectiveness of this treatment in pediatric patients.