Characteristics of first definitive cellular therapy
| Characteristics of therapy . | Treatment groups . | P . | ||
|---|---|---|---|---|
| HCT with no preceding ERT (HCT) (N = 56) . | ERT followed by HCT (ERT-HCT) (N = 31) . | ERT followed by gene therapy (ERT-GT) (N = 33) . | ||
| Duration of ERT before first definitive cellular therapy occurred (d) | .514 | |||
| N with data | N/A | 31 | 33 | |
| Median (min-max) | N/A | 180 (20-5114) | 202 (67-5268) | |
| Duration of ERT before first definitive cellular therapy occurred (mo) | ||||
| 0-3 | N/A | 10 (32.3) | 5 (15.2%) | .066*,† |
| 3-12 | N/A | 10 (32.3) | 20 (60.6%) | |
| >12 | N/A | 11 (35.5) | 8 (24.2%) | |
| Year of first cellular therapy | <.001*,† | |||
| 1982-1989 | 13 (23.2%) | 3 (9.7%) | 0 (0.0%) | |
| 1990-1999 | 16 (28.6%) | 5 (16.1%) | 0 (0.0%) | |
| 2000-2009 | 16 (28.6%) | 8 (25.8%) | 7 (21.2%) | |
| 2010-2017 | 11 (19.6%) | 15 (48.4%) | 26 (78.8%) | |
| Age at first cellular therapy (days) | <.001‡ | |||
| N with data | 56 | 31 | 33 | |
| Median (min-max) | 131.5 (8-4783) | 361 (48-5318) | 317 (98-5601) | |
| Infection at time of first cellular therapy | <.001* | |||
| Active | 22 (40.7%) | 5 (21.7%) | 0 (0.0%) | |
| None or resolved (not active) | 32 (59.3%) | 18 (78.3%) | 33 (100%) | |
| Missing | 2 | 8 | 0 | |
| Height percentile at time of first cellular therapy | .025* | |||
| <5th | 26 (63.4%) | 7 (35.0%) | 10 (34.5%) | |
| ≥5th | 15 (36.6%) | 13 (65.0%) | 19 (65.5%) | |
| Missing | 15 | 11 | 4 | |
| Weight percentile at time of first cellular therapy | <.001* | |||
| <5th | 44 (81.5%) | 11 (40.7%) | 10 (31.3%) | |
| ≥5th | 10 (18.5%) | 16 (59.3%) | 22 (68.8%) | |
| Missing | 2 | 4 | 1 | |
| Donor type (first HCT) | <.001* | |||
| HLA-identical sibling | 9 (16.1%) | 6 (19.4%) | N/A | |
| HLA-matched family | 6 (10.7%) | 0 (0.0%) | N/A | |
| HLA-mismatched other relative (haploidentical) | 36 (64.3%) | 6 (19.4%) | N/A | |
| Unrelated donor | 5 (8.9%) | 19 (61.3%) | N/A | |
| Graft type (first HCT) | .008*,† | |||
| Bone marrow | 50 (89.3%) | 19 (61.3%) | N/A | |
| Cord blood | 3 (5.4%) | 7 (22.6%) | N/A | |
| PBSC | 3 (5.4%) | 5 (16.1%) | N/A | |
| Product type (first GT) | N/A | |||
| Bone marrow CD34+ cells | N/A | N/A | 33 (100%) | |
| Vector type (first GT) | N/A | |||
| Lentiviral | N/A | N/A | 21 (63.6%) | |
| Retroviral | N/A | N/A | 12 (36.4%) | |
| Conditioning intensity (first CT) | <.001*,† | |||
| None | 39 (69.6%) | 6 (20.0%) | 0 (0.0%) | |
| Immune suppression only | 4 (7.1%) | 2 (6.7%) | 0 (0.0%) | |
| Reduced intensity | 5 (8.9%) | 8 (26.7%) | 33 (100%) | |
| Myeloablative | 8 (14.3%) | 14 (46.7%) | 0 (0.0%) | |
| Missing | 0 | 1 | 0 | |
| Serotherapy in conditioning | <.001*,† | |||
| ATG | 6 (10.7%) | 12 (38.7%) | 0 (0.0%) | |
| Alemtuzumab | 2 (3.6%) | 4 (12.9%) | 0 (0.0%) | |
| None | 48 (85.7%) | 15 (48.4%) | 33 (100%) | |
| GVHD prophylaxis (first HCT) | <.001*,† | |||
| TCD w/soybean lectin | 32 (57.1%) | 7 (22.6%) | N/A | |
| Other/unknown TCD | 3 (5.4%) | 0 (0.0%) | N/A | |
| CD34 selection ± TCD | 3 (5.4%) | 1 (3.2%) | N/A | |
| IS + ATG/alemtuzumab | 3 (5.4%) | 11 (35.5%) | N/A | |
| IS only | 10 (17.9%) | 8 (25.8%) | N/A | |
| None | 5 (8.9%) | 2 (6.5%) | N/A | |
| Other | 0 (0.0%) | 2 (6.5%) | N/A | |
| Need for subsequent therapy following first definitive cellular therapy | .054*,† | |||
| ERT | 6 (10.7%) | 0 (0.0) | 4 (12.1%) | |
| GT | 2 (3.6%) | 0 (0.0) | 0 (0.0%) | |
| HCT | 8 (14.3%) | 4 (12.9%) | 0 (0.0%) | |
| None | 40 (71.4%) | 27 (87.1%) | 29 (87.9%) | |
| Total number of definitive cellular therapies performed over patient lifetime | .043*,† | |||
| 1 (initial one only) | 46 (82.1%) | 27 (87.1%) | 33 (100%) | |
| 2 (need for second CT) | 10 (17.9%) | 4 (12.9%) | 0 (0.0%) | |
| Characteristics of therapy . | Treatment groups . | P . | ||
|---|---|---|---|---|
| HCT with no preceding ERT (HCT) (N = 56) . | ERT followed by HCT (ERT-HCT) (N = 31) . | ERT followed by gene therapy (ERT-GT) (N = 33) . | ||
| Duration of ERT before first definitive cellular therapy occurred (d) | .514 | |||
| N with data | N/A | 31 | 33 | |
| Median (min-max) | N/A | 180 (20-5114) | 202 (67-5268) | |
| Duration of ERT before first definitive cellular therapy occurred (mo) | ||||
| 0-3 | N/A | 10 (32.3) | 5 (15.2%) | .066*,† |
| 3-12 | N/A | 10 (32.3) | 20 (60.6%) | |
| >12 | N/A | 11 (35.5) | 8 (24.2%) | |
| Year of first cellular therapy | <.001*,† | |||
| 1982-1989 | 13 (23.2%) | 3 (9.7%) | 0 (0.0%) | |
| 1990-1999 | 16 (28.6%) | 5 (16.1%) | 0 (0.0%) | |
| 2000-2009 | 16 (28.6%) | 8 (25.8%) | 7 (21.2%) | |
| 2010-2017 | 11 (19.6%) | 15 (48.4%) | 26 (78.8%) | |
| Age at first cellular therapy (days) | <.001‡ | |||
| N with data | 56 | 31 | 33 | |
| Median (min-max) | 131.5 (8-4783) | 361 (48-5318) | 317 (98-5601) | |
| Infection at time of first cellular therapy | <.001* | |||
| Active | 22 (40.7%) | 5 (21.7%) | 0 (0.0%) | |
| None or resolved (not active) | 32 (59.3%) | 18 (78.3%) | 33 (100%) | |
| Missing | 2 | 8 | 0 | |
| Height percentile at time of first cellular therapy | .025* | |||
| <5th | 26 (63.4%) | 7 (35.0%) | 10 (34.5%) | |
| ≥5th | 15 (36.6%) | 13 (65.0%) | 19 (65.5%) | |
| Missing | 15 | 11 | 4 | |
| Weight percentile at time of first cellular therapy | <.001* | |||
| <5th | 44 (81.5%) | 11 (40.7%) | 10 (31.3%) | |
| ≥5th | 10 (18.5%) | 16 (59.3%) | 22 (68.8%) | |
| Missing | 2 | 4 | 1 | |
| Donor type (first HCT) | <.001* | |||
| HLA-identical sibling | 9 (16.1%) | 6 (19.4%) | N/A | |
| HLA-matched family | 6 (10.7%) | 0 (0.0%) | N/A | |
| HLA-mismatched other relative (haploidentical) | 36 (64.3%) | 6 (19.4%) | N/A | |
| Unrelated donor | 5 (8.9%) | 19 (61.3%) | N/A | |
| Graft type (first HCT) | .008*,† | |||
| Bone marrow | 50 (89.3%) | 19 (61.3%) | N/A | |
| Cord blood | 3 (5.4%) | 7 (22.6%) | N/A | |
| PBSC | 3 (5.4%) | 5 (16.1%) | N/A | |
| Product type (first GT) | N/A | |||
| Bone marrow CD34+ cells | N/A | N/A | 33 (100%) | |
| Vector type (first GT) | N/A | |||
| Lentiviral | N/A | N/A | 21 (63.6%) | |
| Retroviral | N/A | N/A | 12 (36.4%) | |
| Conditioning intensity (first CT) | <.001*,† | |||
| None | 39 (69.6%) | 6 (20.0%) | 0 (0.0%) | |
| Immune suppression only | 4 (7.1%) | 2 (6.7%) | 0 (0.0%) | |
| Reduced intensity | 5 (8.9%) | 8 (26.7%) | 33 (100%) | |
| Myeloablative | 8 (14.3%) | 14 (46.7%) | 0 (0.0%) | |
| Missing | 0 | 1 | 0 | |
| Serotherapy in conditioning | <.001*,† | |||
| ATG | 6 (10.7%) | 12 (38.7%) | 0 (0.0%) | |
| Alemtuzumab | 2 (3.6%) | 4 (12.9%) | 0 (0.0%) | |
| None | 48 (85.7%) | 15 (48.4%) | 33 (100%) | |
| GVHD prophylaxis (first HCT) | <.001*,† | |||
| TCD w/soybean lectin | 32 (57.1%) | 7 (22.6%) | N/A | |
| Other/unknown TCD | 3 (5.4%) | 0 (0.0%) | N/A | |
| CD34 selection ± TCD | 3 (5.4%) | 1 (3.2%) | N/A | |
| IS + ATG/alemtuzumab | 3 (5.4%) | 11 (35.5%) | N/A | |
| IS only | 10 (17.9%) | 8 (25.8%) | N/A | |
| None | 5 (8.9%) | 2 (6.5%) | N/A | |
| Other | 0 (0.0%) | 2 (6.5%) | N/A | |
| Need for subsequent therapy following first definitive cellular therapy | .054*,† | |||
| ERT | 6 (10.7%) | 0 (0.0) | 4 (12.1%) | |
| GT | 2 (3.6%) | 0 (0.0) | 0 (0.0%) | |
| HCT | 8 (14.3%) | 4 (12.9%) | 0 (0.0%) | |
| None | 40 (71.4%) | 27 (87.1%) | 29 (87.9%) | |
| Total number of definitive cellular therapies performed over patient lifetime | .043*,† | |||
| 1 (initial one only) | 46 (82.1%) | 27 (87.1%) | 33 (100%) | |
| 2 (need for second CT) | 10 (17.9%) | 4 (12.9%) | 0 (0.0%) | |
Two gene therapy patients, receiving autologous umbilical cord blood gene therapy in the 1990s, had only baseline characteristics evaluated and were then excluded from further survival analyses.
ATG, antithymocyte globulin; IS, immune suppression; N/A, not applicable; PBSC, peripheral blood stem cells; TCD, T-cell depletion.
χ2 test.
Exact test.
Kruskal-Wallis test.