Abstract
To assess whether a tolerance-induction regimen could be applied for unrelated (MUD) HCT in SAA, we retrospectively reviewed our HCT experience using unmanipulated 10/10 HLA-matched bone marrow grafts from MSD vs. MUD donors. Conditioning was CTX 200 mg/kg (CTX) + rabbit ATG 10 mg/kg (ATG) for MSD (n = 9) and TLI (800 cGy) + CTX/ATG for MUD HCT (n = 5). Immunoprophylaxis was CSA and short-course MTX. Median patient age was 14.7 yr, median time to HCT 1.5 yr, and median follow-up 3 yr. Outcome measures included EFS, time to engraftment, and cumulative incidence of GVHD (CIN of GVHD) for MSD and MUD cohorts. EFS and stable engraftment rate were 100%. CIN of acute GVHD was: MSD, Grade I-II: 1 (11%), Grade III-IV: 0%; MUD, Grade I-II: 1 (20%), Grade III-IV: 1 (20%). CIN of chronic GVHD was: MSD, limited: 1 (11%), extensive: 0%; MUD, limited: 0%, extensive: 0%. All immunosuppressive-compliant patients successfully weaned immunosuppression. Although in limited patients, our results suggest that immunomodulatory TLI added to backbone CTX/ATG conditioning is a promising option for MUD HCT in SAA patients, which we will examine in a prospective clinical trial.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 628-634 |
| Number of pages | 7 |
| Journal | Pediatric Transplantation |
| Volume | 15 |
| Issue number | 6 |
| DOIs | |
| State | Published - Sep 1 2011 |
| Externally published | Yes |
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Keywords
- aplastic anemia engraftment
- graft-versus-host disease
- hematopoietic stem cell transplantation
- non-myeloablative
- pediatrics
- transplant tolerance
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Transplantation
Cite this
Favorable preliminary results using TLI/ATG-based immunomodulatory conditioning for matched unrelated donor allogeneic hematopoietic stem cell transplantation in pediatric severe aplastic anemia. / Pillai, Asha; Hartford, Christine; Wang, Chong; Pei, Deqing; Yang, Jie; Srinivasan, Ashok; Triplett, Brandon; Dallas, Mari; Leung, Wing.
In: Pediatric Transplantation, Vol. 15, No. 6, 01.09.2011, p. 628-634.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Favorable preliminary results using TLI/ATG-based immunomodulatory conditioning for matched unrelated donor allogeneic hematopoietic stem cell transplantation in pediatric severe aplastic anemia
AU - Pillai, Asha
AU - Hartford, Christine
AU - Wang, Chong
AU - Pei, Deqing
AU - Yang, Jie
AU - Srinivasan, Ashok
AU - Triplett, Brandon
AU - Dallas, Mari
AU - Leung, Wing
PY - 2011/9/1
Y1 - 2011/9/1
N2 - To assess whether a tolerance-induction regimen could be applied for unrelated (MUD) HCT in SAA, we retrospectively reviewed our HCT experience using unmanipulated 10/10 HLA-matched bone marrow grafts from MSD vs. MUD donors. Conditioning was CTX 200 mg/kg (CTX) + rabbit ATG 10 mg/kg (ATG) for MSD (n = 9) and TLI (800 cGy) + CTX/ATG for MUD HCT (n = 5). Immunoprophylaxis was CSA and short-course MTX. Median patient age was 14.7 yr, median time to HCT 1.5 yr, and median follow-up 3 yr. Outcome measures included EFS, time to engraftment, and cumulative incidence of GVHD (CIN of GVHD) for MSD and MUD cohorts. EFS and stable engraftment rate were 100%. CIN of acute GVHD was: MSD, Grade I-II: 1 (11%), Grade III-IV: 0%; MUD, Grade I-II: 1 (20%), Grade III-IV: 1 (20%). CIN of chronic GVHD was: MSD, limited: 1 (11%), extensive: 0%; MUD, limited: 0%, extensive: 0%. All immunosuppressive-compliant patients successfully weaned immunosuppression. Although in limited patients, our results suggest that immunomodulatory TLI added to backbone CTX/ATG conditioning is a promising option for MUD HCT in SAA patients, which we will examine in a prospective clinical trial.
AB - To assess whether a tolerance-induction regimen could be applied for unrelated (MUD) HCT in SAA, we retrospectively reviewed our HCT experience using unmanipulated 10/10 HLA-matched bone marrow grafts from MSD vs. MUD donors. Conditioning was CTX 200 mg/kg (CTX) + rabbit ATG 10 mg/kg (ATG) for MSD (n = 9) and TLI (800 cGy) + CTX/ATG for MUD HCT (n = 5). Immunoprophylaxis was CSA and short-course MTX. Median patient age was 14.7 yr, median time to HCT 1.5 yr, and median follow-up 3 yr. Outcome measures included EFS, time to engraftment, and cumulative incidence of GVHD (CIN of GVHD) for MSD and MUD cohorts. EFS and stable engraftment rate were 100%. CIN of acute GVHD was: MSD, Grade I-II: 1 (11%), Grade III-IV: 0%; MUD, Grade I-II: 1 (20%), Grade III-IV: 1 (20%). CIN of chronic GVHD was: MSD, limited: 1 (11%), extensive: 0%; MUD, limited: 0%, extensive: 0%. All immunosuppressive-compliant patients successfully weaned immunosuppression. Although in limited patients, our results suggest that immunomodulatory TLI added to backbone CTX/ATG conditioning is a promising option for MUD HCT in SAA patients, which we will examine in a prospective clinical trial.
KW - aplastic anemia engraftment
KW - graft-versus-host disease
KW - hematopoietic stem cell transplantation
KW - non-myeloablative
KW - pediatrics
KW - transplant tolerance
UR - http://www.scopus.com/inward/record.url?scp=80052097795&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=80052097795&partnerID=8YFLogxK
U2 - 10.1111/j.1399-3046.2011.01542.x
DO - 10.1111/j.1399-3046.2011.01542.x
M3 - Article
VL - 15
SP - 628
EP - 634
JO - Pediatric Transplantation
JF - Pediatric Transplantation
SN - 1397-3142
IS - 6
ER -