Table 2.
Functional outcomes of ILI gene transfer
| Subject | MVICT | MF size, μμm (X ±SD) | Ambulatory status pre and post gene therapy | 6MWT change BL | ||
|---|---|---|---|---|---|---|
| Right | Left | Pre | Post | |||
| E-01 | Pre 2.77 | Pre 3.33 | — | Post TAa Quad not available | Non-AMB pre gene therapy; non-AMB post gene therapy | * |
| Post 4.07 | Post 3.91 | |||||
| Δ↑47% | Δ ↑17% | |||||
| E-02 | Pre 1.87 | Pre 3.44 | — | No post Bx | AMB pre gene therapy; lost AMB post gene therapy | ** |
| Post 1.46 | Post 2.48 | |||||
| Δ ↓22% | Δ↓28% | |||||
| E-03 | Pre 36.83 | Pre 36.22 | Pre 56.19 (17.43) | Post 72.30 (26.05) | AMB pre gene therapy; AMB post gene therapy | Pre 538 m |
| Post 58.41 | Post 60.92 | Post 525 m | ||||
| Δ ↑59% | Δ ↑68% | Δ ↓2% | ||||
| E-04 | Pre 11.45 | Pre 8.43 | Pre 49.85 (16.64) | Post 35.12 (27.58) | AMB pre gene therapy; AMB post gene therapy | Pre 487 m |
| Post 7.43 | Post 4.33 | Post 366 m | ||||
| Δ ↓35% | Δ ↓49% | Δ ↓24% | ||||
| E-05 | Pre 5.14 | Pre 4.96 | Pre 62.13 (24.97) | Post 67.38 (22.84) | AMB pre gene therapy; lost AMB post gene therapy | ** |
| Post 5.07 | Post 4.74 | |||||
| Δ ↓1% | Δ↓4% | |||||
| E-06 | Pre 10.61 | Pre 11.01 | Pre 46.39 (14.56) | Post 32.09 (10.63) | AMB pre gene therapy; AMB post gene therapy | Pre 571 m |
| Post 8.14 | Post 9.45 | Post 600 m | ||||
| Δ ↓23% | Δ ↓14% | Δ ↑5% | ||||
a
Post gene-transfer muscle biopsy done on TA.
*
No ambulatory change pre and post gene therapy.
**
Lost ambulation post gene therapy.
ILI, isolated limb infusion; MVICT, maximum voluntary isometric contraction testing (reported in kilograms); 6MWT, six-minute walk test (reported as distance walked in meters); BL, baseline; AMB, ambulation; TA, tibialis anterior; Δ, percent change in 6MWT meters from baseline to end of study (all patients completed 2 years).