Abstract
This study analyzes the hematopoietic cell transplantation experience in patients with immune deficiency at a single institution. The objective is to comprehensively evaluate the short-term and long-term outcomes with various preparative regimens, donor grafts, and ex vivo manipulations to identify transplantation approaches that most likely favor early donor immune competency without generating excessive toxicity. Clinical outcomes were evaluated in 52 consecutive patients with immune deficiencies. Thirty-seven of the 52 patients (71%) survived with attenuation of their underlying disease. The use of a melphalan-based reduced-intensity conditioning preparative regimen and immunomagnetic CD3+ T cell depletion techniques (when T cell depletion was indicated) were associated with improved event-free survival. Survivors who received a preparative regimen other than a melphalan-based reduced-intensity regimen suffered from therapy-related morbidities or chronic/recurrent infections. Our findings indicate that melphalan-based reduced-intensity conditioning regimens and immunomagnetic CD3+ T cell depletion limit therapy-related toxicity, and demonstrate promising results for the early establishment of donor immune competency.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 1911-1920 |
| Number of pages | 10 |
| Journal | Biology of Blood and Marrow Transplantation |
| Volume | 18 |
| Issue number | 12 |
| DOIs | |
| State | Published - Dec 1 2012 |
| Externally published | Yes |
Fingerprint
Keywords
- Immune reconstitution
- Severe combined immune deficiency
ASJC Scopus subject areas
- Hematology
- Transplantation
Cite this
Effects of Conditioning Regimens and T Cell Depletion in Hematopoietic Cell Transplantation for Primary Immune Deficiency. / Triplett, Brandon M.; Wang, Chong; Yang, Jie; Dallas, Mari; Hartford, Christine; Howard, Vanessa; Pillai, Asha; Shook, David; Srinivasan, Ashok; Laver, Joseph; Leung, Wing.
In: Biology of Blood and Marrow Transplantation, Vol. 18, No. 12, 01.12.2012, p. 1911-1920.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Effects of Conditioning Regimens and T Cell Depletion in Hematopoietic Cell Transplantation for Primary Immune Deficiency
AU - Triplett, Brandon M.
AU - Wang, Chong
AU - Yang, Jie
AU - Dallas, Mari
AU - Hartford, Christine
AU - Howard, Vanessa
AU - Pillai, Asha
AU - Shook, David
AU - Srinivasan, Ashok
AU - Laver, Joseph
AU - Leung, Wing
PY - 2012/12/1
Y1 - 2012/12/1
N2 - This study analyzes the hematopoietic cell transplantation experience in patients with immune deficiency at a single institution. The objective is to comprehensively evaluate the short-term and long-term outcomes with various preparative regimens, donor grafts, and ex vivo manipulations to identify transplantation approaches that most likely favor early donor immune competency without generating excessive toxicity. Clinical outcomes were evaluated in 52 consecutive patients with immune deficiencies. Thirty-seven of the 52 patients (71%) survived with attenuation of their underlying disease. The use of a melphalan-based reduced-intensity conditioning preparative regimen and immunomagnetic CD3+ T cell depletion techniques (when T cell depletion was indicated) were associated with improved event-free survival. Survivors who received a preparative regimen other than a melphalan-based reduced-intensity regimen suffered from therapy-related morbidities or chronic/recurrent infections. Our findings indicate that melphalan-based reduced-intensity conditioning regimens and immunomagnetic CD3+ T cell depletion limit therapy-related toxicity, and demonstrate promising results for the early establishment of donor immune competency.
AB - This study analyzes the hematopoietic cell transplantation experience in patients with immune deficiency at a single institution. The objective is to comprehensively evaluate the short-term and long-term outcomes with various preparative regimens, donor grafts, and ex vivo manipulations to identify transplantation approaches that most likely favor early donor immune competency without generating excessive toxicity. Clinical outcomes were evaluated in 52 consecutive patients with immune deficiencies. Thirty-seven of the 52 patients (71%) survived with attenuation of their underlying disease. The use of a melphalan-based reduced-intensity conditioning preparative regimen and immunomagnetic CD3+ T cell depletion techniques (when T cell depletion was indicated) were associated with improved event-free survival. Survivors who received a preparative regimen other than a melphalan-based reduced-intensity regimen suffered from therapy-related morbidities or chronic/recurrent infections. Our findings indicate that melphalan-based reduced-intensity conditioning regimens and immunomagnetic CD3+ T cell depletion limit therapy-related toxicity, and demonstrate promising results for the early establishment of donor immune competency.
KW - Immune reconstitution
KW - Severe combined immune deficiency
UR - http://www.scopus.com/inward/record.url?scp=84869093665&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84869093665&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2012.07.014
DO - 10.1016/j.bbmt.2012.07.014
M3 - Article
C2 - 22842333
AN - SCOPUS:84869093665
VL - 18
SP - 1911
EP - 1920
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
SN - 1083-8791
IS - 12
ER -