Parental donors provide rapid access to children with PID who lack of matched donors, which enables early transplant and improves survival.
We report the outcomes of 97 children with PID who received first parental haploidentical transplantation (HIT) between 1987 and 2018 at the Great North Children’s Hospital, Newcastle upon Tyne.
Transplantation characteristics and outcomes summarised in table. There was increased use of HIT in children with non-SCID over the past 10 years (MHC class II deficiency, 6; CGD, 4; WAS, 3; DOCK8 deficiency, 3; others, 19) . Engraftment kinetics (p=0.001) improved and graft failure (p=0.01) reduced with CD3/CD19-TCD-PBSC and TCR-µb/CD19-depleted-PBSC. The incidence of acute GvHD was similar and none had chronic GvHD. Analysis by TCD methods revealed that the OS and EFS were superior for TCR-ab/CD19-depleted-PBSC compared to CD3/CD19-depleted-PBSC, CD34-selected marrow and Campath-M depleted marrow. The myeloid donor chimerism was better using the newer methods.
Campath-1M-marrow | CD34-selected-marrow | CD3/CD19-depleted-PBSC | TCR-ab/CD19-depleted-PBSC | p-value | |
Number | 27 | 28 | 7 | 35 | |
Year | 1987-1998 | 1999-2006 | 2007-2011 | 2012-2018 | |
Diagnosis | <0.001 | ||||
SCID | 26 | 24 | 5 | 12 | |
non-SCID | 1 | 4 | 2 | 23 | |
Conditioned transplant | 25 | 25 | 7 | 33 | 0.76 |
In-vivo serotherapy | 7 | 9 | 4 | 32 | <0.001 |
GvHD prophylaxis | 2 | 21 | 6 | 18 | <0.001 |
aGvHD | 6 | 4 | 0 | 7 | 0.07 |
Graft failure | 8 | 3 | 1 | 1 | 0.001 |
Lastest chimerism | |||||
Myeloid | 32(0-100) | 4(0-49) | 96(0-100) | 100(0-100) | <0.001 |
T-lymphocyte | 100(95-100) | 100(83-100) | 98(77-100) | 100(57-100) | 0.49 |
Parental HIT using TCR-ab/CD19-depleted-PBSC is a safe alternative donor procedure and leading to successful outcome even in non-SCID PID.