TABLE 1
FROM:
Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease
H Church, K Tylee, A Cooper, M Thornley, J Mercer, E Wraith, T Carr, A O'Meara and R F Wynn
BACK TO ARTICLETable 1. Biochemical data from the four patient groups after 12 months of stable engraftment
| Patient Group |
Iduronidase activity  mol/g/h (range and mean)
| GAGs DS/CS ratio (range and mean) |
|---|---|---|
| Group 1 n=19 | 10.7–42.0 | 0.2–0.77 |
| MUD=8, MUC=6, | mean=24.8 | mean=0.38 |
| (Normal sib=5) | ||
| Group 2 n=8 | 5.3–20.5 | 0.5–0.87 |
| mean=10.2 | mean=0.67 | |
| Group 3 n=5 | 7.1–38 | 0.31–0.71 |
| (MUD=4, normal sib=1) | mean=17.0 | mean=0.49 |
| Group 4 n=8 | 7.4–13.2 | 0.45–1.06 |
| mean=7.1 | mean=0.77 |
Abbreviations: CS=chondroitin sulphate; DS=dermatan sulphate; GAG=glycosaminoglycan; MUC=matched unrelated cord; MUD=matched unrelated donor.
The biochemical data is averaged enzyme level following transplant and residual storage product, expressed as DS/CS ratio.
Group 1 where a normal donor is used – MUD, MUC or normal family donor – and there is full donor chimerism. No difference in engrafted enzyme level was seen between unrelated cord (n=6, range 12.5–34.6, mean=24.3) and unrelated adult donors (n=13, range 10.7–42.0, mean=25.1) with full engraftment. In Group 2, a heterozygous family donor has been used and there is full donor chimerism. In Group 3, a normal donor is used and there is mixed chimerism (range 20–90% donor, median 70%, mean 62%). No mixed chimerism was seen where in recipients of unrelated cord transplants. In group 4, there is mixed chimerism from a heterozygous donor (range 20–70% donor, median 55%, mean 50%).
