Unrelated cord blood transplantation using minimal-intensity conditioning in a 1.5-month-old infant with X-linked severe combined immunodeficiency.
Transpl Immunol
; : 102115, 2024 Sep 02.
Article
em En
| MEDLINE
| ID: mdl-39233094
ABSTRACT
BACKGROUND:
Severe combined immunodeficiency (SCID) is a heterogenous disorder with profound deficiency of T/B-cell functions. The best SCID therapy requires hematopoietic stem cell transplantation (HSCT) early in life. HSCT with conditioning is necessary to achieve a long-term reconstitution of B-cell functions. However, conditioning may aggravate pre-existing infection and cause transplant-related toxicity, especially in very young infants. Hence, the intensity of conditioning should be reduced to allow the reconstitution of immunity including B cells to the extent that prevents transplant-related toxicity and delayed complications.METHODS:
An infant with a family history of X-linked SCID (X-SCID) was diagnosed with X-SCID disorder soon after birth. The infant exhibited cytomegalovirus (CMV) infection despite being strictly isolated. At 1.5â¯months of age, we performed an unrelated cord blood transplantation (CBT) with a less intensity conditioning regimen fludarabine (125â¯mg/m2)â¯+â¯melphalan (80â¯mg/m2). We evaluated the efficacy of reconstitution by assessing B-cell function and growth and psychomotor development at 5â¯years and 7â¯months after CBT.RESULTS:
The clinical course after CBT was uneventful after CBT. The CMV infection was fully controlled by ganciclovir or foscavir therapy, which was discontinued at day 55 after CBT. Furthermore, immunoglobulin (Ig) replacement therapy was also discontinued at 6â¯months after CBT. A sufficient proportion of CD27+ memory B cells was developed, which was essential for an effective vaccination and prevention of infections. While the B-cell chimerism became recipient-dominant, the Ig replacement therapy was substituted by very successful post-vaccine immunity acquisition after CBT. The analysis of the general developmental parameters showed that chemotherapy did not cause any delay in growth and psychomotor development.CONCLUSIONS:
The CBT therapy with this conditioning regimen was well tolerated and induced an effective reconstitution of B-cell functions in an X-SCID infant under the 3â¯months of age.
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Coleções:
01-internacional
Base de dados:
MEDLINE
Idioma:
En
Revista:
Transpl Immunol
Ano de publicação:
2024
Tipo de documento:
Article