Advancements in Pediatric Hematopoietic Stem Cell Transplantation: Emerging Indications for Expert Professionals

Advancements in Pediatric Hematopoietic Stem Cell Transplantation: Emerging Indications for Expert Professionals The landscape of pediatric hematopoietic stem cell transplantation (HSCT), continues to evolve with significant expansions in indications beyond traditional applications. Recent advances have refined patient selection criteria, particularly for high-risk hematologic malignancies, and broadened the therapeutic potential of HSCT for non-malignant disorders and solid tumors. Genetic profiling now guides transplantation decisions for hematologic malignancies and identifies patients who benefit most from allogeneic HSCT. HSCT is increasingly recognized as a transformative therapy for non-hematologic disorders in pediatric patients, extending beyond traditional applications in hematologic malignancies and bone marrow failure syndromes. Recent advances have demonstrated the potential of HSCT to modify the natural history of inherited metabolic disorders, severe immunodeficiencies, autoimmune conditions, and even certain solid tumors. For inherited metabolic diseases such as adrenoleukodystrophy (ALD) and mucopolysaccharidoses, allogeneic HSCT can halt disease progression by establishing donor-derived microglial engraftment and enzymatic correction, with long-term survival rates. The application of HSCT has also expanded to autoimmune and inflammatory disorders refractory to conventional therapies. Emerging evidence supports the role of autologous HSCT in severe juvenile idiopathic arthritis and systemic lupus erythematosus, where immunoablation and immune resetting can induce sustained remission in ۵۰% of cases. For neurometabolic conditions such as X-linked lymphoproliferative disease, HSCT can prevent fatal complications by restoring immune function and metabolic homeostasis. Despite these promising applications, significant challenges remain. Transplant-related toxicity and graft-versus-host disease (GVHD) contribute to non-relapse mortality rates of ۱۰-۳۰% in non-malignant disorders, necessitating careful risk-benefit assessment. Access barriers, including socioeconomic factors, perceived mortality risks, and donor availability, further limit utilization, particularly in resource-limited settings. The future integration of gene therapy approaches and international collaborative research efforts promises to further expand and optimize the application of HSCT for children with nonhematologic disorders, ultimately improving survival and quality of life for these vulnerable patients.