Treosulfan-based Reduced-intensity Allogeneic Hematopoietic Cell Transplantation in Adults With Primary Immunodeficiency: A Case Series

• Published in: Anticancer research Vol. 42; no. 9; pp. 4505 - 4509
• Main Authors: Dalla Via, Vera, Stelmes, Anne, Recher, Mike, Arranto, Christian, Junker, Till, Matteazzi, Francesca, Heim, Dominik, Passweg, Jakob R, Medinger, Michael, van den Berg, Jana
• Format: Report
• Language: English
• Published: 01.09.2022
• ISSN: 1791-7530
• DOI: 10.21873/anticanres.15952

BACKGROUND/AIMWe report three adult patients with primary immunodeficiency (PID) treated with reduced-intensity allogenic hematopoietic cell transplantation (HCT) with fludarabine/treosulfan conditioning and graft-versus-host disease (GvHD) prophylaxis with alemtuzumab and a calcineurin inhibitor. CASE REPORTPatient 1, a 51-year-old male, had common variable immunodeficiency (CVID) with protein-losing enteropathy. Patient 2 was a 29-year-old woman with STAT3 (signal transducer and activator of transcription 3)-dependent hyper-IgE syndrome (HIES). Patient 3 was a 25-year-old male with XIAP (X-linked inhibitor of apoptosis)-deficiency presenting as treatment-refractory granulomatous enteropathy. Engraftment occurred in all three patients, with 100% donor chimerism in blood. Two patients survived, whereas the patient with CVID died due to infection. CONCLUSIONThis series highlights issues of transplantation for PID in adults and treosulfan-based conditioning, which is feasible for PID patients; infectious complications are the major issue of concern.