Risk Factors for Hematopoietic Stem Cell Transplantation in Inborn Errors of Immunity.

Area of Science:

• Immunology
• Hematology
• Pediatrics

Background:

• Hematopoietic stem cell transplantation (HSCT) is a vital treatment for inborn errors of immunity (IEI).
• However, complications following HSCT can significantly impact survival in IEI patients.
• Risk factors associated with HSCT outcomes in IEI remain underexplored.

Purpose of the Study:

• To identify and analyze the key risk factors influencing overall survival (OS) after HSCT in patients with IEI.
• To provide a comprehensive understanding of factors affecting post-transplant outcomes in this vulnerable population.

Main Methods:

• A retrospective analysis of 312 patients with various IEI who underwent their first allogeneic HSCT between 2012 and 2020.
• Evaluation of risk factors including active infection, autoimmunity/inflammation, malignancy, genetic diagnosis status, nutritional status (BMI), age at HSCT, and pre-existing organ damage.
• Statistical analysis to determine the association between these factors and overall survival (OS).

Main Results:

• Overall survival (OS) after HSCT was 74%, but significantly lower in Severe Combined Immunodeficiency (SCID) patients (49%) compared to other IEI (78%).
• Factors significantly decreasing OS included active infection (43%), autoimmunity/inflammation (61%), undernutrition (52%), obesity (64%), age >12 years (61%), and organ damage (59%).
• Unknown genetic defect did not significantly affect survival, while malignancy showed a non-significant trend (67%).

Conclusions:

• Active infection, organ damage, and older age at HSCT are critical risk factors impacting survival in IEI patients post-transplant.
• The study highlights the need for improved predictive tools to assess post-HSCT survival, particularly in pediatric IEI.
• Further research is warranted to develop strategies mitigating these identified risk factors.