Treatment Outcomes
| Parameter . | GIS BMT . | HIR BMT . |
|---|---|---|
| No. of patients | 28* | 26 |
| Engraftment | ||
| Complete/partial/autologous (n) | 15/7/4 | 16/1/9 |
| α-l-Iduronidase enzyme activity† | ||
| Median, range, n‡ | 21.0, 14.3-41.0, 14 | 14.4, 8.8-21.0, 6 |
| Other§ | ||
| Acute GVHD (n) | ||
| Grade 0 to I/II to IV | 17/8 | 10/12 |
| Not applicable or assessable | 3 | 4 |
| Chronic GVHD (n) | ||
| None/limited/extensive | 25/0/0 | 13/4/5 |
| Not applicable or assessable | 3 | 4 |
| Survival (n) | ||
| Alive/alive and engrafted | 21/21 | 14/9 |
| Follow-up of survivors (yr) | ||
| Median (range) | 8.7 (2.2-13.9) | 7.2 (2.0-10.2) |
| Cause of death (n) | ||
| Cardiac | 2 | 4 |
| GVHD | 1 | 5 |
| Sepsis | 1 | 1 |
| Hemorrhage | 1 | 0 |
| MPSIH | 1 | 0 |
| Organ failure | 0 | 1 |
| Pneumonitis | 1 | 0 |
| Unknown | 0 | 1 |
| Parameter . | GIS BMT . | HIR BMT . |
|---|---|---|
| No. of patients | 28* | 26 |
| Engraftment | ||
| Complete/partial/autologous (n) | 15/7/4 | 16/1/9 |
| α-l-Iduronidase enzyme activity† | ||
| Median, range, n‡ | 21.0, 14.3-41.0, 14 | 14.4, 8.8-21.0, 6 |
| Other§ | ||
| Acute GVHD (n) | ||
| Grade 0 to I/II to IV | 17/8 | 10/12 |
| Not applicable or assessable | 3 | 4 |
| Chronic GVHD (n) | ||
| None/limited/extensive | 25/0/0 | 13/4/5 |
| Not applicable or assessable | 3 | 4 |
| Survival (n) | ||
| Alive/alive and engrafted | 21/21 | 14/9 |
| Follow-up of survivors (yr) | ||
| Median (range) | 8.7 (2.2-13.9) | 7.2 (2.0-10.2) |
| Cause of death (n) | ||
| Cardiac | 2 | 4 |
| GVHD | 1 | 5 |
| Sepsis | 1 | 1 |
| Hemorrhage | 1 | 0 |
| MPSIH | 1 | 0 |
| Organ failure | 0 | 1 |
| Pneumonitis | 1 | 0 |
| Unknown | 0 | 1 |
*Two patients died during preparative therapy.
†Leukocyte α-l-iduronidase enzyme activity in engrafted survivors 0.7 to 10.9 years following BMT.
‡Normal: 39 ± 15 nmol/mg protein/h.
§GIS BMT, n = 1 for each of the following: 4.1 (normal, 11.7 to 20.6 nmol/mg protein/h); 9.7 (normal, 14.5 to 23.3 nmol/mg protein/h); 17.4 (normal, 15.1 to 50.9 nmol/mg protein/h); 1.9 (normal, 21.3 to 22.6 nmol/mg protein/h); 21.8 (normal, 21.8 to 39.4 nmol/mg protein/h); 23.2 (normal, 39.6 to 50.9 nmol/mg protein/h); HIR BMT, n = 1 for each of the following: 188.0 (normal, 615.4 pmol/mg protein/min); 527.1 (normal, 558.1 pmol/mg protein/min).