Blood: Adolescents and adults with congenital immunodeficiency may also benefit from HSCT treatment

Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard treatment for infants and children with congenital immunodeficiency (IEI), but transplantation is
rarely recommended for adolescents and adults with IEI.
In addition to small, single-center studies, the lack of published prognostic data on HSCT in adolescents and adults with IEI and the high risk of transplant-related death have delayed the use of HSCT in patients with IEI who develop or develop severe organ damage later in life
.

A large retrospective, multicenter HSCT prognostic study published in the journal Blood reported the prognosis
of 329 patients with IEI treated with HSCT (age at HSCT: 15-62.
5 years).

The patient received his first hematopoietic stem cell transplant
between 2000 and 2019.
The primary endpoints are overall survival (OS) and event-free lifetime (EFS).

The researchers also evaluated the effects of IEI subgroups and IEI-specific risk factors at HSCT, including infection, bronchiectasis, colitis, malignancy, inflammatory lung disease, splenectomy, liver dysfunction, and systemic immunosuppression
.

Overall survival after HSCT

With a median follow-up of 44.
3 months, the estimated overall survival rates for all patients at 1 and 5 years after HSCT were 78% and 71%, EFS, respectively, and 65% and 62%, respectively, with a lower
incidence of severe acute (8%) or extensive chronic (7%) graft-versus-host disease.
In univariate analysis, OS and EFS were relatively low
in patients with primary antibody deficiency, bronchiectasis, previous splenectomy, hepatic comorbidities, and higher hematopoietic cell transplant comorbidity index scores.
In multivariate analyses, EFS was worse
in patients with more IEI-related complications.
Age and donor had no significant effect on
OS or EFS.

In summary, the study showed that adolescents and adults with congenital immunodeficiency can also benefit significantly from allogeneic hematopoietic stem cell transplantation, and age is not a risk factor
for poor prognosis.
The findings provide much-needed evidence
to determine which patients are most likely to benefit from HSCT.

Original source:

Michael H.
Albert, et al.
Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study.
Blood (2022) 140 (14): 1635–1649.
https://doi.
org/10.
1182/blood.
2022015506