Background and Aims

Haematopoietic cell transplantation (HCT) is the only curative therapy for MHC class II expression deficiency.

Methods

We examined the outcome of 25 children with MHC class II deficiency who received a HCT at the Great North Children’s Hospital between 1995 and 2018. Outcomes included overall survival (OS), toxicity and long-term outcome. Log rank test was used to analyse predictors of OS.

Results

Median age of transplant was 21.4 months (range, 0.9-93.3). The 3-year OS for the entire cohort was 78%, increasing to 94% for HCT after 2008 (n=19) vs 33% for HCT before 2008 (n=6) (Figure1). After 2008, the OS was comparable between matched family (100%), unrelated (100%) and parental haploidentical donor recipients (85%) (Figure2). Three had graft failure and received a second transplant but died. 9 patients had grade 1 acute GvHD (36%), 4 (14%) grade II, none grade III-IV and none had chronic GvHD.

Of 14 long-term survivors with evaluable data, the median age at last follow-up was 5.2 years (range, 2.2 to 14) with the median duration of follow-up 3.5 years (range, 1.1 to 7.2). All were off immunoglobulin. Median myeloid chimerism was 100% (range, 0-100) and median T-lymphocyte chimerism was 100 (range, 59-100). The median CD count was 754 x109/L (range, 201 to 1900). 4 had CD4 lymphopenia.

Conclusions

Transplant survival has improved and long-term disease outcome of survivors is good.

Background and Aims

Parental donors provide rapid access to children with PID who lack of matched donors, which enables early transplant and improves survival.

Methods

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.

Results

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.

Conclusions

Parental HIT using TCR-ab/CD19-depleted-PBSC is a safe alternative donor procedure and leading to successful outcome even in non-SCID PID.

Background and Aims

Rotavirus vaccines have been reported to cause enteropathy in infants who received vaccine prior to a diagnosis of SCID.

Methods

We studied the impact of rotavirus vaccine on transplant outcome in 24 infants with SCID transplanted at the Great North Children’s Hospital between 2013 and 2018.

Results

7 patients diagnosed at birth did not receive rotavirus vaccine. Rotavirus vaccine was associated with increased incidence of acute GvHD (p=0.03), increased need of PN (p=0.01) and increased need for ICU (p=0.03).

Conclusions

Rotavirus vaccine is associated with significant transplant morbidity for infants with SCID

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.

Conclusions

HID is a safe alternative donor source for infants with SCID