From 1995 to 2001, five children received 6 cord blood transplantations (CBT) for their diseases including 3 with acute lymphoblastic leukemia (ALL), 1 severe aplastic anemia (SAA) and 1 mucopolysaccharidosis type 11 (MPS -11). Patient characteristics and transplantation summary are shown in the following table. Among 4 sibling donor’s CBT, complete chimerism was documented after CBT, although 2 of them also received stem cells of other sources from the same donors.. Three ALL children died of refractory leukemia. The child with SAA died of sepsis following a late graft failure. Among 2 unrelated donor’s CBT, the MPS 11 child failed the first CBT with autologous recovery. The stem cells of the 2nd CBT from sun Yat-Sen Cancer Center Cord Blood Bank survived over 18 months after CBT for this MPS 11 Child. The glycosaminoglycans (GAG) in urine decreased from 469 to 202 mg GAG/gm Cr. And the iduronate sulfatase (IDS) activity increased from 2 to 214 nmole/ml/h after CBT. The preliminary data show that the stored cord blood unit is of suitable for clinical use to correct the deficiency of IDS activity required in the degradative pathways of sulfated GAGs.
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