Post-transplant Outcomes
Five patients achieved neutrophil engraftment at the median of 17 days (range, 13–21 days), and all had sustained >95% donor chimerism without donor lymphocyte infusion. One (Patient 4) developed sinusoidal obstruction syndrome and a graft failure despite well targeted BU. She had the longest transfusion-dependent period and the highest ferritin level of all patients (Fig. 1 ). Two months later, umbilical cord blood (CB) transplantation was successfully performed after our conventional regimen FLU, melphalan (MEL) and low-dose total body irradiation (TBI). Histocompatible CB was obtained from the Japanese CB Bank Network. She is presently active in her school life with complete donor chimerism.
No grade >II acute GVHD or chronic GVHD occurred. Acute GVHD was well controlled by corticosteroids. Infectious complications occurred and were controlled in all 3 patients, including cytomegalovirus reactivation (n=2), human herpes virus-6 reactivation (n=1), non-tuberculosis mycobacteria infection (n=1), and herpes zoster (n=1). Autoimmune cytopenia occurred on treatment in one patient after complete engraftment. During the median follow-up period after HCT (42.5 months [range, 6–71 months]), no patients had neurological disability, neoplasms or pulmonary complications.