Haematopoietic stem cell transplantation in children with inborn errors of immunity: A single centre experience
Scandinavian Journal of Immunology, cilt.101, sa.1, 2025 (SCI-Expanded, Scopus)
- Yayın Türü: Makale / Tam Makale
- Cilt numarası: 101 Sayı: 1
- Basım Tarihi: 2025
- Doi Numarası: 10.1111/sji.13431
- Dergi Adı: Scandinavian Journal of Immunology
- Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, BIOSIS, EMBASE
- Anahtar Kelimeler: haematopoietic stem cell transplantation, inborn errors of immunity, paediatric, primary immune dysregulation disorders, severe combined immunodeficiencies
- Sağlık Bilimleri Üniversitesi Adresli: Evet
Özet
This study retrospectively analyzed the outcomes of 61 pediatric patients with inborn errors of immunity (IEI) who underwent hematopoietic stem cell transplantation (HSCT) between 2011 and 2023. Patients were categorized into primary immunodeficiency disorders (PIDD), primary immune dysregulation disorders (PIRD), and congenital defects of phagocyte number or function (CDP). Median ages at diagnosis and HSCT were 9 and 30 months, respectively. With a median follow-up of 51 months, the overall survival (OS) was 70%, with a 100-day post-transplant OS of 80%. Transplant-related mortality (TRM) was 29%, with rates of 42%, 22.5%, and 27% for PIRD, PIDD, and CDP, respectively. This study highlights the importance of early diagnosis and HSCT in improving survival for IEI patients, while also emphasizing the need for continuous improvements in transplant protocols to minimize TRM and enhance quality of life.