Beschreibung:
<jats:title>Abstract</jats:title><jats:sec><jats:title>Introduction</jats:title><jats:p><jats:styled-content style="fixed-case">HSCT</jats:styled-content> 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 <jats:styled-content style="fixed-case">HSCT</jats:styled-content> for patients with <jats:styled-content style="fixed-case">PID</jats:styled-content>.</jats:p></jats:sec><jats:sec><jats:title>Method</jats:title><jats:p>A total of 15 patients that received a treosulfan‐based conditioning regimen for <jats:styled-content style="fixed-case">HSCT</jats:styled-content> were recruited. Type of diagnosis, donor and stem cell source, pretransplant organ damage, infections, engraftment, chimerism, and transplant‐related toxicities were analyzed.</jats:p></jats:sec><jats:sec><jats:title>Results</jats:title><jats:p>At a median follow‐up time of 32 months, the overall survival was 86.7%. Following <jats:styled-content style="fixed-case">HSCT</jats:styled-content>, 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 <jats:styled-content style="fixed-case">GVHD</jats:styled-content> and chronic <jats:styled-content style="fixed-case">GVHD</jats:styled-content> were documented in 53% and 20% of the study population, respectively. Although the cohort consisted of patients with pretransplant liver damage, <jats:styled-content style="fixed-case">SOS</jats:styled-content> manifestations were documented in 20%.</jats:p></jats:sec><jats:sec><jats:title>Conclusion</jats:title><jats:p>Treosulfan‐based conditioning regimens before <jats:styled-content style="fixed-case">HSCT</jats:styled-content> are associated with lower toxicity compared to myeloablative regimens, are safe, and have high engraftment rates with full‐donor chimerism in patients having <jats:styled-content style="fixed-case">PID</jats:styled-content>, regardless of the specified genetic diagnosis and donor type.</jats:p></jats:sec>