HAPLOIDENTICAL DONOR OR UNRELATED DONOR FOR INFANTS WITH SEVERE COMBINED IMMUNODEFICIENCY?
- Su Han Lum, United Kingdom
Abstract
Background and Aims
Severe combined immunodeficiency (SCID) is a paediatric emergency and early haematopoeitic cell transplantation is the only curative therapy
Methods
We studyed transplant outcomes in 35 infants with SCID who were transplanted at Great North Children’s Hospital from 2013 to 2018.
Results
Stem cell sources were: Haploidentical donor (HID) (TCR ab/CD19, 9), matched family donor (MFD) (marrow, 7; PBSC 4), matched unrelated donor (MUD) (marrow, 3; cord, 8; PBSC, 4). Fludarabine and Treosulfan were used for patients who received conditioni ng. 2-year OS was 100% for HID, 79% for MSD and 84% for MUD. CD4 recovery at 6 months post-HCT was comparable between stem cell sources.
| HID(n=9) | MFD(n=11) | MUD(n=15) | p-value | |
| Newborn SCID | 3 | 3 | 4 | 0.93 |
| Median age at diagnosis (range), months | 2.8(at birth-6.5) | 3.0(at birth-0.47) | 2.3(at birth-13.6) | 0.59 |
| Median age at transplant (range), months | 6.0(1.7-9.7) | 4.3(1.0-7.1) | 5.3(0.8-16.3) | 0.31 |
| Median interval between diagnosis and transpant (range), months | 2.3(0.6-9.7) | 1.6(1.0-2.8) | 2.0(0.3-14.3) | 0.33 |
| PCP | 2 | 2 | 3 | 0.98 |
| Disseminated BCG | 2 | 2 | 2 | 0.47 |
| Pre-transplant viraemia | 3 | 2 | 1 | 0.24 |
| Pre-transplant gut viraemia | 6 | 5 | 7 | 0.57 |
| Conditioned transplant | 7 | 7 | 12 | 0.61 |
| Median TNC (range), 108/kg | 12 (3.7-40.0) | 9.7 (5.7-21.0) | 3.4 (0.93-20.4) | 0.03 |
| Median CD34 (range), 106/kg | 20.7 (4.0-53.0) | 11.7 (5.0-25.7) | 1.5 (0.21-24.2) | 0.02 |
| Median day of neutrophil engraftment (range) | 15(9-27) | 14(6-20) | 20(11-33) | 0.10 |
| aGvHD | 2 | 0 | 3 | 0.26 |
| Post-transplant viraemia | 3 | 3 | 2 | 0.48 |
| Median days of inpatient stay (range) | 87(38-260) | 88(36-190) | 105(43-263) | 0.29 |
Conclusions
HID is a safe alternative donor source for infants with SCID