Outcome
Median follow-up was 48 months (IQR 17-101). Three-year EFS and OS were
62% ± SE 7% (95% CI 49-77) and 77% ± SE 6% (95% CI 66-89),
respectively. There was no significant difference in EFS or OS for
children who received LMB-96 vs CPR as their chemotherapy regimen.
Patients treated as per LMB-96 had a 3-year EFS of 69% ± SE 11% (95%
CI 51-93) and those treated with CPR had a 3-year EFS of 76% ± SE 12%
(95% CI 55-100), p=0.991, Figure 1. At last follow-up, 38 (69%)
children were alive without evidence of PTLD, 1 (2%) was alive with
PTLD. Sixteen (29%) children had died; 10 (18%) of PTLD, 4 (7%) of
allograft dysfunction, 1 (2%) of multiorgan failure and 1 (2%) of
unknown cause. Of those alive, 9 (23%) had allograft dysfunction and 2
(5%) required second transplant or organ replacement therapy.