Late effects after allogeneic hematopoietic stem cell transplantation in patients with primary immunodeficiency.
One in four pediatric survivors of allogeneic stem cell transplant develop late effects.
Late effects after allogeneic hematopoietic stem cell transplantation in patients with primary immunodeficiency.
Allogeneic hematopoietic stem cell transplantation (hematopoietic stem cell transplantation) has markedly improved survival in children with primary immunodeficiencies (primary immunodeficiencies) transforming previously fatal diseases into curable conditions.
Following PRISMA guidelines, PubMed and EBSCOhost databases were searched without date restrictions for studies reporting late effects in patients who underwent allogeneic hematopoietic stem cell transplantation for primary immunodeficiencies at the age of 0-17 years and survived at least 2 years after hematopoietic stem cell transplantation.
nearly 1 in 4 survivors developed a lasting late effect
Growth and developmental delay were the most frequently reported late effects, observed in at least 280 patients (12.2%).
This was followed by neurological (n=173, 7.5%) and autoimmune or hematologic (n=161, 7.0%) long-term issues.
Pulmonary and skin complications were noted in 150 patients each (6.5% each), while dental and skeletal impairments affected 112 patients (4.9%).
Endocrine issues were reported in 99 patients (4.3%), and gastrointestinal or liver impairments occurred in 75 patients (3.3%).
A substantial proportion of pediatric hematopoietic stem cell transplantation survivors with primary immunodeficiencies experience late effects. They affect various organs and systems years after an immune system replacement.
These findings highlight the need for lifelong, multidisciplinary follow-up to optimize long-term health, functional outcomes, and quality of life.