Anticancer research · 2022
Treosulfan-based Reduced-intensity Allogeneic Hematopoietic Cell Transplantation in Adults With Primary Immunodeficiency: A Case Series.
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Dalla Via Vera, Stelmes Anne, Recher Mike, Arranto Christian, Junker Till, Matteazzi Francesca, Heim Dominik, Passweg Jakob R, Medinger Michael, van den Berg Jana
Аннотация
BACKGROUND/AIM: We 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 REPORT: Patient 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.
CONCLUSION: This 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.
CASE REPORT: Patient 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.
CONCLUSION: This 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.