Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency.
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for severe combined immunodeficiency (SCID). Detailed assessment of the long-term outcome of HSCT, ie, the occurrence of clinical events and the quality and stability of immune reconstitution, is now required. We performed a single-center retrospective analysis of the long-term outcome of HSCT in 90-patient cohort followed for between 2 and 34 years (median, 14 years). Clinical events and immune reconstitution data were collected. Almost half the patients have experienced one or more significant clinical events, including persistent chronic graft-versus-host disease (GVHD), autoimmune and inflammatory manifestations, opportunistic and nonopportunistic infections, chronic human papilloma virus (HPV) infections, and a requirement for nutritional support. With the notable exception of severe HPV infection, these complications tend to become less common 15 years later after HSCT. A multivariate analysis showed that the occurrence of these events correlated with non-genoidentical donors, diagnosis of Artemis SCID, and quality of immune reconstitution. In most cases, HSCT enables long-term survival with infrequent sequelae. However, the occurrence of relatively late-onset complications is a concern that requires specific means of prevention and justifies careful patient follow-up.
Neven
Bénédicte
B
Unité d'Immuno-Hématologie et Rhumatologie Pédiatrique, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
Leroy
Sandrine
S
Decaluwe
Hélène
H
Le Deist
Francoise
F
Picard
Capucine
C
Moshous
Despina
D
Mahlaoui
Nizar
N
Debré
Marianne
M
Casanova
Jean-Laurent
JL
Dal Cortivo
Liliane
L
Madec
Yoann
Y
Hacein-Bey-Abina
Salima
S
de Saint Basile
Geneviève
G
de Villartay
Jean-Pierre
JP
Blanche
Stéphane
S
Cavazzana-Calvo
Marina
M
Fischer
Alain
A
D015331:Cohort Studies; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D016511:Severe Combined Immunodeficiency / Q000453:epidemiology* / Q000276:immunology / Q000523:psychology / Q000601:surgery*; D015996:Survival Rate; D013997:Time Factors; D016896:Treatment Outcome