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.