Outcome of treosulfan-based reduced-toxicity conditioning regimens for HSCT in high-risk patients with primary immune deficiencies

Haskologlu S., Bal S. K., Islamoglu C., Altun D., KENDİRLİ T., DOĞU E. F., ...Daha Fazla

PEDIATRIC TRANSPLANTATION, sa.7, 2018 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Basım Tarihi: 2018
  • Doi Numarası: 10.1111/petr.13266
  • Dergi Adı: PEDIATRIC TRANSPLANTATION
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Anahtar Kelimeler: hematopoietic stem cell transplantation, non-myeloablative conditioning, primary immune deficiencies, Treosulfan, STEM-CELL TRANSPLANTATION, VERSUS-HOST-DISEASE, NONMALIGNANT DISEASES, CHILDREN, CLASSIFICATION, FLUDARABINE, EXPERIENCE
  • Ankara Üniversitesi Adresli: Evet

Özet

Introduction HSCT is the curative therapeutic option in PIDs. Due to the increase in survival rates, reduced-toxicity conditioning regimens with treosulfan have become another alternative. The purpose of this retrospective study was to analyze the outcome of treosulfan-based conditioning before HSCT for patients with PID. MethodResultsA total of 15 patients that received a treosulfan-based conditioning regimen for HSCT were recruited. Type of diagnosis, donor and stem cell source, pretransplant organ damage, infections, engraftment, chimerism, and transplant-related toxicities were analyzed. At a median follow-up time of 32months, the overall survival was 86.7%. Following HSCT, 14 of 15 patients had engraftment, with 86.7% of the cohort having full-donor chimerism. The most common toxicity was seen on the skin (53.3%). Acute GVHD and chronic GVHD were documented in 53% and 20% of the study population, respectively. Although the cohort consisted of patients with pretransplant liver damage, SOS manifestations were documented in 20%. ConclusionTreosulfan-based conditioning regimens before HSCT are associated with lower toxicity compared to myeloablative regimens, are safe, and have high engraftment rates with full-donor chimerism in patients having PID, regardless of the specified genetic diagnosis and donor type.