Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood

Purpose Allogeneic hematopoietic stem cell transplant (HSCT) remains the treatment of choice for patients with inborn errors of immunity (IEI). There is little published medical outcome data assessing late medical complications following transition to adult care. We sought to document event-free sur...

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Bibliographic Details
Published in:Journal of clinical immunology 2022-08, Vol.42 (6), p.1230-1243
Main Authors: Day, James W., Elfeky, Reem, Nicholson, Bethany, Goodman, Rupert, Pearce, Rachel, Fox, Thomas A., Worth, Austen, Booth, Claire, Veys, Paul, Carpenter, Ben, Hough, Rachael, Gaspar, H. Bobby, Titman, Penny, Ridout, Deborah, Workman, Sarita, Hernandes, Fernando, Sandford, Kit, Laurence, Arian, Campbell, Mari, Burns, Siobhan O., Morris, Emma C.
Format: Article
Language:English
Subjects:
Adults
Age
Autoimmune diseases
Biomedical and Life Sciences
Biomedicine
Children
Chimerism
Chronic infection
Graft rejection
Graft versus host disease
Graft-versus-host reaction
Grafts
Hematopoietic stem cells
Hospitals
Immune reconstitution
Immune system
Immunology
Infections
Infectious Diseases
Infertility
Internal Medicine
Malignancy
Medical Microbiology
Morbidity
Original
Original Article
Patients
Pediatrics
Recurrent infection
Stem cell transplantation
Stem cells
Transplants & implants
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Summary:Purpose Allogeneic hematopoietic stem cell transplant (HSCT) remains the treatment of choice for patients with inborn errors of immunity (IEI). There is little published medical outcome data assessing late medical complications following transition to adult care. We sought to document event-free survival (EFS) in transplanted IEI patients reaching adulthood and describe common late-onset medical complications and factors influencing EFS. Methods In this landmark analysis, 83 adults surviving 5 years or more following prior HSCT in childhood for IEI were recruited. The primary endpoint was event-free survival, defined as time post-first HSCT to graft failure, graft rejection, chronic infection, life-threatening or recurrent infections, malignancy, significant autoimmune disease, moderate to severe GVHD or major organ dysfunction. All events occurring less than 5 years post-HSCT were excluded. Results EFS was 51% for the whole cohort at a median of 20 years post HSCT. Multivariable analysis identified age at transplant and whole blood chimerism as independent predictors of long-term EFS. Year of HSCT, donor, conditioning intensity and underlying diagnosis had no significant impact on EFS. 59 events occurring beyond 5 years post-HSCT were documented in 37 patients (45% cohort). A total of 25 patients (30% cohort) experienced ongoing significant complications requiring active medical intervention at last follow-up. Conclusion Although most patients achieved excellent, durable immune reconstitution with infrequent transplant-related complications, very late complications are common and associated with mixed chimerism post-HSCT. Early intervention to correct mixed chimerism may improve long-term outcomes and adult health following HSCT for IEI in childhood.
ISSN:0271-9142
1573-2592
DOI:10.1007/s10875-022-01278-6