Individual Clinical Responses Pre- and Postconversion in High Titer Patients (n = 8)
| Patient . | Spleen . | Time to a/b (mo)* . | Treatment History† . | Hemoglobin . | Platelet . | Acid Phos . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| B . | C . | P . | B . | C . | P . | B . | C . | P . | ||||
| A | Intact | 7 | 120, ↓60 @ 17 mo | 11.5 | 12.8 | 13.6 | 61 | 155 | 142 | 17.9 | 6.2 | 5.3 |
| B | Total Splenectomy | 14 | 120, ↓60 @ 17 mo ↑ 117 @ 40 mo | 14.6 | 15.2 | 14.4 | 163 | 243 | 144 | 17.1 | 8.9 | 9.4 |
| C | Intact | 9 | 120 × 29 mo | 9.9 | 12.0 | 13.4 | 21 | 22 | 37 | 13.3 | 6.8 | 5.4 |
| D | Intact | 10 | 120,‡ 100 @ 9 mo | 11.8 | 12.4 | 12.3 | 107 | 139 | 150 | 7.6 | 4.3 | 4.4 |
| E | Intact | 12 | 120, ↓60 @ 11 mo | 11.6 | 14.2 | 13.4 | 97 | 142 | 141 | 1.5 | 0.7 | 0.7 |
| F | Intact | 3 | 320 × 14 mo2-153 | 5.3 | 11.0 | 12.9 | 53 | 97 | 143 | NA | ||
| G | Intact | 19 | 120, ↓60 @ 4 mo2-155 | 10.2 | 9.2 | 10.7 | 61 | 90 | 87 | 88.7 | 70.3 | 74.1 |
| H | Intact | 10 | 120, ↑180 @ 14 mo2-154 | NA | 61 | 140 | 97 | NA | ||||
| Patient . | Spleen . | Time to a/b (mo)* . | Treatment History† . | Hemoglobin . | Platelet . | Acid Phos . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| B . | C . | P . | B . | C . | P . | B . | C . | P . | ||||
| A | Intact | 7 | 120, ↓60 @ 17 mo | 11.5 | 12.8 | 13.6 | 61 | 155 | 142 | 17.9 | 6.2 | 5.3 |
| B | Total Splenectomy | 14 | 120, ↓60 @ 17 mo ↑ 117 @ 40 mo | 14.6 | 15.2 | 14.4 | 163 | 243 | 144 | 17.1 | 8.9 | 9.4 |
| C | Intact | 9 | 120 × 29 mo | 9.9 | 12.0 | 13.4 | 21 | 22 | 37 | 13.3 | 6.8 | 5.4 |
| D | Intact | 10 | 120,‡ 100 @ 9 mo | 11.8 | 12.4 | 12.3 | 107 | 139 | 150 | 7.6 | 4.3 | 4.4 |
| E | Intact | 12 | 120, ↓60 @ 11 mo | 11.6 | 14.2 | 13.4 | 97 | 142 | 141 | 1.5 | 0.7 | 0.7 |
| F | Intact | 3 | 320 × 14 mo2-153 | 5.3 | 11.0 | 12.9 | 53 | 97 | 143 | NA | ||
| G | Intact | 19 | 120, ↓60 @ 4 mo2-155 | 10.2 | 9.2 | 10.7 | 61 | 90 | 87 | 88.7 | 70.3 | 74.1 |
| H | Intact | 10 | 120, ↑180 @ 14 mo2-154 | NA | 61 | 140 | 97 | NA | ||||
| Patient . | Liver Volume . | Spleen Volume . | Bone . | ||||
|---|---|---|---|---|---|---|---|
| B . | C . | P . | B . | C . | P . | ||
| A | NA | NA | NA | ||||
| B | NA | NA | ↑Uptake at the 16-month evaluation | ||||
| C | 1.8x | 1.11 | 1.17 | 23.6x | 18.6x | 12.5x | NA |
| D | NA | NA | Baseline: R hip infarct and pain. | ||||
| MRI improved and bone pain resolved at 18-month evaluation. | |||||||
| E | 1.17x | 1.04x | 0.9x | 6.8x | 4.49x | 3.9x | NA |
| F | NA | NA | NA | ||||
| G | NA | Improved by PE | NA | ||||
| H | NA | NA | NA | ||||
| Patient . | Liver Volume . | Spleen Volume . | Bone . | ||||
|---|---|---|---|---|---|---|---|
| B . | C . | P . | B . | C . | P . | ||
| A | NA | NA | NA | ||||
| B | NA | NA | ↑Uptake at the 16-month evaluation | ||||
| C | 1.8x | 1.11 | 1.17 | 23.6x | 18.6x | 12.5x | NA |
| D | NA | NA | Baseline: R hip infarct and pain. | ||||
| MRI improved and bone pain resolved at 18-month evaluation. | |||||||
| E | 1.17x | 1.04x | 0.9x | 6.8x | 4.49x | 3.9x | NA |
| F | NA | NA | NA | ||||
| G | NA | Improved by PE | NA | ||||
| H | NA | NA | NA | ||||
BCP denotes Baseline (B), Conversion (C), and Postchange (P) values. Postvalues are 4 to 8 months postconversion, unless otherwise indicated.
Abbreviations: NA, not applicable; PE, physical exam.
Time to detection of inhibiting antibodies.
Treatment history indicates initial dose in U/kg/4 weeks followed by dose adjustment and timing of dose adjustment.
Patient (D) had a dose interruption for 1 month after 4 months of ERT. On resuming therapy, the patient received 34 to 80 U/kg/4 weeks during months 6 to 8. The dose was then increased to 100 U/kg/4 weeks. Spleen volume was 3 cm below costal margin at pretreatment. After 19 months on ERT, spleen was nonpalpable. Bone involvement improved on MRI and bone pain resolved.
Patient F has Type III disease.
Patient G interrupted therapy after 5 months on ERT. The interruption lasted for 8 months. The dose at the time of therapy resumption was 60 × 2.
Patient H has platelet count data available only. Patient refuses other evaluations. Dose increase at 14 months was reported to be due to inadequate response.