Serum Cbl and metabolite levels in clinical responders
Screening parameters . | Hematologic responders, no. (%) . | Neurologic responders, no. (%) . | All responders, no. (%) . |
|---|---|---|---|
| Cbl | |||
| Less than 201 pg/mL | 3 (38) | 3 (10) | 6 (16) |
| 201 pg/mL to 300 pg/mL | 5* (62) | 7* (23) | 11* (30) |
| Greater than 300 pg/mL | 0 (0) | 20 (67) | 20 (54) |
| MMA | |||
| Less than 251 nmol/L | 2† (29) | 6 (21) | 8† (23) |
| 251 nmol/L to 376 nmol/L | 0 (0) | 14 (48) | 14 (40) |
| Greater than 376 nmol/L | 5* (71) | 9* (31) | 13* (37) |
| HCys | |||
| Less than 12.2 μmol/L | 2 (40) | 14 (48) | 17 (50) |
| 12.2 μmol/L to 13.6 μmol/L | 0 (0) | 2 (7) | 2 (6) |
| Greater than 13.6 μmol/L | 3* (60) | 13* (45) | 15* (44) |
Screening parameters . | Hematologic responders, no. (%) . | Neurologic responders, no. (%) . | All responders, no. (%) . |
|---|---|---|---|
| Cbl | |||
| Less than 201 pg/mL | 3 (38) | 3 (10) | 6 (16) |
| 201 pg/mL to 300 pg/mL | 5* (62) | 7* (23) | 11* (30) |
| Greater than 300 pg/mL | 0 (0) | 20 (67) | 20 (54) |
| MMA | |||
| Less than 251 nmol/L | 2† (29) | 6 (21) | 8† (23) |
| 251 nmol/L to 376 nmol/L | 0 (0) | 14 (48) | 14 (40) |
| Greater than 376 nmol/L | 5* (71) | 9* (31) | 13* (37) |
| HCys | |||
| Less than 12.2 μmol/L | 2 (40) | 14 (48) | 17 (50) |
| 12.2 μmol/L to 13.6 μmol/L | 0 (0) | 2 (7) | 2 (6) |
| Greater than 13.6 μmol/L | 3* (60) | 13* (45) | 15* (44) |
Pretherapy Cbl was measured in all 37 responders. Pretherapy MMA was measured in 35 responders, including 7 of 8 hematologic responders and 29 of 30 neurologic responders. Pretherapy HCys was measured in 34 responders, including 5 of 8 hematologic responders and 29 of 30 neurologic responders.
Includes the one patient who had both a hematologic and neurologic response to Cbl therapy.
Includes 2 patients with values reported as less than 400 μmol/L.