Long-term outcome of non-ablative booster BMT in patients with SCID.
| dc.contributor.author | Teigland, CL | |
| dc.contributor.author | Parrott, RE | |
| dc.contributor.author | Buckley, RH | |
| dc.coverage.spatial | England | |
| dc.date.accessioned | 2017-03-01T19:08:20Z | |
| dc.date.available | 2017-03-01T19:08:20Z | |
| dc.date.issued | 2013-08 | |
| dc.description.abstract | SCID is a fatal syndrome caused by mutations in at least 13 different genes. It is characterized by the absence of T cells. Immune reconstitution can be achieved through nonablative related donor BMT. However, the first transplant may not provide sufficient immunity. In these cases, booster transplants may be helpful. A prospective/retrospective study was conducted of 49 SCID patients (28.7% of 171 SCIDs transplanted over 30 years) who had received booster transplants to define the long-term outcome, factors contributing to a need for a booster and factors that predicted success. Of the 49 patients, 31 (63%) are alive for up to 28 years. Age at initial transplantation was found to have a significant effect on outcome (mean of 194 days old for patients currently alive, versus a mean of 273 days old for those now deceased, P=0.0401). Persistent viral infection was present in most deceased booster patients. In several patients, the use of two parents as sequential donors resulted in striking T-and B-cell immune reconstitution. A majority of the patients alive today have normal or adequate T-cell function and are healthy. Nonablative booster BMT can be lifesaving for SCID. | |
| dc.identifier | ||
| dc.identifier | bmt20136 | |
| dc.identifier.eissn | 1476-5365 | |
| dc.identifier.uri | ||
| dc.language | eng | |
| dc.publisher | Springer Science and Business Media LLC | |
| dc.relation.ispartof | Bone Marrow Transplant | |
| dc.relation.isversionof | 10.1038/bmt.2013.6 | |
| dc.subject | Bone Marrow Transplantation | |
| dc.subject | Female | |
| dc.subject | Graft vs Host Disease | |
| dc.subject | Humans | |
| dc.subject | Infant | |
| dc.subject | Infant, Newborn | |
| dc.subject | Male | |
| dc.subject | Prospective Studies | |
| dc.subject | Retrospective Studies | |
| dc.subject | Severe Combined Immunodeficiency | |
| dc.subject | T-Lymphocytes | |
| dc.subject | Transplantation Chimera | |
| dc.subject | Treatment Outcome | |
| dc.title | Long-term outcome of non-ablative booster BMT in patients with SCID. | |
| dc.type | Journal article | |
| pubs.author-url | ||
| pubs.begin-page | 1050 | |
| pubs.end-page | 1055 | |
| pubs.issue | 8 | |
| pubs.organisational-group | Basic Science Departments | |
| pubs.organisational-group | Clinical Science Departments | |
| pubs.organisational-group | Duke | |
| pubs.organisational-group | Duke Cancer Institute | |
| pubs.organisational-group | Immunology | |
| pubs.organisational-group | Institutes and Centers | |
| pubs.organisational-group | Pediatrics | |
| pubs.organisational-group | Pediatrics, Allergy and Immunology | |
| pubs.organisational-group | School of Medicine | |
| pubs.publication-status | Published | |
| pubs.volume | 48 |
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