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

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....

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Published inAnticancer research Vol. 42; no. 9; pp. 4505 - 4509
Main Authors VIA, VERA DALLA, STELMES, ANNE, RECHER, MIKE, ARRANTO, CHRISTIAN, JUNKER, TILL, MATTEAZZI, FRANCESCA, HEIM, DOMINIK, PASSWEG, JAKOB R., MEDINGER, MICHAEL, VAN DEN BERG, JANA
Format Journal Article
LanguageEnglish
Published Athens International Institute of Anticancer Research 01.09.2022
Subjects
Adults
Apoptosis
Calcineurin
Calcineurin inhibitors
Chimerism
Common variable immunodeficiency
Complications
Conditioning
Fludarabine
Graft-versus-host reaction
Immunoglobulin E
Inhibitors
Job's syndrome
Males
Monoclonal antibodies
Patients
Primary immunodeficiencies
Prophylaxis
Stat3 protein
Stem cell transplantation
Transplantation
Treosulfan
XIAP protein
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Summary: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.
ISSN:0250-7005
1791-7530
DOI:10.21873/anticanres.15952