Transplant outcome
Four children underwent haploidentical HSCT 43, 76, 78, and 151 days,
respectively, after start of the first course of CloEC. Three of them
fulfilled proceed-to-transplant criteria, and one was transplanted
according to the treating physician’s decision despite not being in
morphological remission. All donors were mothers. The numbers of
transplanted CD34+ cells ranged from 4.45 to 10.6 x
106/kg and residual αβ TCR+ lymphocytes from 5.0 to
102 x 103/kg. All children showed sustained
engraftment. One patient is alive and well 7.5 years after
transplantation. Two children relapsed on days +55 and +128
post-transplant and ultimately died of progressive leukemia. One child
developed AdV-induced fulminant hepatitis and died of liver failure in
complete remission. Details of transplantation and post-transplant
follow-up are presented in Table 3.