Late effects after allogeneic hematopoietic stem cell transplantation in patients with primary immunodeficiency - Summary - MDSpire

Late effects after allogeneic hematopoietic stem cell transplantation in patients with primary immunodeficiency

  • By

  • Gintė Grubliauskaitė

  • Audronė Mulevičienė

  • Jelena Rascon

  • July 1, 2026

  • 0 min

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Objective:

To identify, categorize, and summarize late effects (LEs) following allogeneic HSCT in pediatric patients with primary immunodeficiencies (PIDs).

Approach:
  • Systematic Review: A systematic review was conducted following PRISMA guidelines, searching PubMed and EBSCOhost databases for studies on LEs in pediatric HSCT patients with PIDs who survived at least 2 years post-transplant.
Key Findings:
  • 31 studies included, covering 2,293 patients transplanted from 1981 to 2019.
  • 24% of patients reported at least one LE, with growth and developmental delay being the most common (12.2%).
  • Neurological issues were reported in 173 patients (7.5%), autoimmune or hematologic problems in 161 patients (7.0%), and pulmonary and skin complications in 150 patients each (6.5% each).
  • Endocrine issues were reported in 99 patients (4.3%), while secondary malignancies and other complications were rare.
Interpretation:

A significant proportion of pediatric HSCT survivors with PIDs experience various LEs years after transplantation.

Limitations:
  • The review included only studies published in English, which may limit the generalizability of findings.
  • Mixed cohorts of pediatric and adult patients were excluded.
Conclusion:

The findings indicate the importance of ongoing monitoring for long-term health outcomes in pediatric HSCT survivors.

Sources:

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