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Fox, Thomas A.; Chakraverty, Ronjon; Burns, Siobhan; Carpenter, Benjamin; Thomson, Kirsty; Lowe, David; Fielding, Adele; Peggs, Karl; Kottaridis, Panagiotis; Uttenthal, Benjamin; Bigley, Venetia; Buckland, Matthew; Grandage, Victoria; Denovan, Shari; Grace, Sarah; Dahlstrom, Julia; Workman, Sarita; Symes, Andrew; Mackinnon, Stephen; Hough, Rachael; Morris, Emma
Blood, 02/2018, Letnik: 131, Številka: 8Journal Article
The primary immunodeficiencies (PIDs), rare inherited diseases characterized by severe dysfunction of immunity, have been successfully treated by allogeneic hematopoietic stem cell transplantation (Allo-HSCT) in childhood. Controversy exists regarding optimal timing and use of Allo-HSCT in adults, due to lack of experience and previous poor outcomes. Twenty-nine consecutive adult patients, with a mean age at transplant of 24 years (range, 17-50 years), underwent Allo-HSCT. Reduced-intensity conditioning (RIC) included fludarabine (Flu)/melphalan/alemtuzumab (n = 20), Flu/busulfan (Bu)/alemtuzumab (n = 8), and Flu/Bu/antithymocyte globulin (n = 1). Stem cell donors were matched unrelated donors or mismatched unrelated donors (n = 18) and matched related donors (n = 11). Overall survival (OS), event-free survival, transplant-related mortality (TRM), acute and chronic graft-versus-host disease incidence and severity, time to engraftment, lineage-specific chimerism, immune reconstitution, and discontinuation of immunoglobulin replacement therapy were recorded. OS at 3 years for the whole cohort was 85.2%. The rarer PID patients without chronic granulomatous disease (CGD) achieved an OS at 3 years of 88.9% (n = 18), compared with 81.8% for CGD patients (n = 11). TRM was low with only 4 deaths observed at a median follow-up of 3.5 years. There were no cases of early or late rejection. In all surviving patients, either stable mixed chimerism or full donor chimerism were observed. At last follow-up, 87% of the surviving patients had no evidence of persistent or recurrent infections. Allo-HSCT is safe and effective in young adult patients with severe PID and should be considered the treatment of choice where an appropriate donor is available. •Allo-HSCT with RIC is safe and effective in younger adults with severe PID.•Referral triggers should include severe infections, autoimmunity, malignancy, and disease progression despite conservative management.
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in: SICRIS
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