Baseline characteristics of patients, and extent of tabelecleucel exposure
| Characteristics . | HCT (n = 14) . | SOT (n = 12) . | Total (N = 26) . |
|---|---|---|---|
| Median age, y (range) | 46.0 (2-74) | 27.5 (7-66) | 36.0 (2-74) |
| Age category, n (%) | |||
| <16 y | 2 (14.3) | 4 (33.3) | 6 (23.1) |
| ≥16 y | 12 (85.7) | 8 (66.7) | 20 (76.9) |
| Male, n (%) | 7 (50.0) | 6 (50.0) | 13 (50.0) |
| Ethnicity, n (%) | |||
| Hispanic/Latino | 1 (7.1) | 2 (16.7) | 3 (11.5) |
| Not Hispanic/Latino | 11 (78.6) | 8 (66.7) | 19 (17.3) |
| Not given | 2 (14.3) | 2 (16.7) | 4 (15.4) |
| Race, n (%) | |||
| Caucasian | 10 (71.4) | 8 (66.7) | 18 (69.2) |
| Black | 1 (7.1) | 0 | 1 (3.8) |
| Asian | 2 (14.3) | 1 (8.3) | 3 (11.5) |
| Other/unknowns | 1 (7.1) | 3 (25.0) | 4 (15.4) |
| Median ECOG PS score (range)∗ | 1.5 (0-4) | 1.0 (0-3) | 1.0 (0-4) |
| Median Lansky score (range)† | 55.0 (50-60) | 80.0 (20-90) | 65.0 (20-90) |
| Disease risk parameters, n (%)∗ | |||
| Age of ≥60 y | 2 (16.7) | 1 (12.5) | 3 (15.0) |
| ECOG PS score of ≥2 | 6 (50.0) | 3 (37.5) | 9 (45.0) |
| Elevated serum LDH | 7 (58.3) | 4 (50.0) | 11 (55.0) |
| Risk score∗,,‡ | |||
| High | 3 (25.0) | 2 (25.0) | 5 (25.0) |
| Intermediate | 8 (66.7) | 4 (50.0) | 12 (60.0) |
| Low | 1 (8.3) | 2 (25.0) | 3 (15.0) |
| Disease morphology/histology, n (%)§ | |||
| Diffuse large B-cell lymphoma | 4 (28.6) | 8 (66.7) | 12 (46.2) |
| PTLD NOS | 6 (42.9) | 0 | 6 (23.1) |
| Polymorphic PTLD | 2 (14.3) | 1 (8.3) | 3 (11.5) |
| Hodgkin lymphoma | 0 | 1 (8.3) | 1 (3.8) |
| Infectious mononucleosis–like PTLD | 0 | 1 (8.3) | 1 (3.8) |
| Lymphoproliferative disorder NOS | 1 (7.1) | 0 | 1 (3.8) |
| Monomorphic B-cell PTLD | 0 | 1 (8.3) | 1 (3.8) |
| Transplanted organ, n (%) | |||
| Kidney | N/A | 6 (50.0) | N/A |
| Heart | N/A | 2 (16.7) | N/A |
| Lung | N/A | 2 (16.7) | N/A |
| Intestine | N/A | 2 (16.7) | N/A |
| Median time | |||
| Median time from transplant to diagnosis of EBV+ PTLD, mo (range) | 4.4 (1.4-198.4) | 7.2 (2.1-275.9) | 5.1 (1.4-275.9) |
| Median time from transplant to first dose of tabelecleucel, mo (range) | 6.4 (2.3-202.2) | 20.5 (2.3-281.3) | 9.3 (2.3-281.3) |
| Median time from initial EBV-related disease diagnosis to first tabelecleucel dose, mo (range) | 1.4 (0.2-8.2) | 5.0 (0.2-67.6) | 2.3 (0.2-67.6) |
| Baseline CNS PTLD involvement, n (%)‖ | 1 (7.1) | 1 (8.3) | 2 (7.7) |
| Baseline extranodal PTLD (including bone marrow), n (%)¶ | 1 (7.1) | 3 (25.0) | 4 (15.4) |
| Prior rituximab therapy, n (%)# | 14 (100) | 11 (91.7) | 25 (96.2) |
| Prior chemotherapy, n (%) | 1 (7.1) | 7 (58.3) | 8 (30.8) |
| Median number of lines of prior systemic therapies (range) | 1.0 (1-3) | 1.5 (1-3) | 1.0 (1-3) |
| Use of immunosuppressive medications at start of tabelecleucel, n (%) | 1 (7.1) | 11 (91.7) | 12 (46.2) |
| Treatment on trial | |||
| Median of average cells administered per dose (×106 cells per kg) (range) | 1.98 (1.6-2.0) | 1.98 (1.6-2.0) | 1.98 (1.6-2.0) |
| Median duration of tabelecleucel treatment, mo (range) | 1.3 (0.03-3.1) | 2.5 (1.2-10.4) | 1.8 (0.03-10.4) |
| Median no. of tabelecleucel doses received (range) | 4.0 (1-9) | 7.0 (4-27) | 6.0 (1-27) |
| Median no. of tabelecleucel cycles received (range) | 2.0 (1-4) | 2.5 (2-9) | 2.0 (1-9) |
| Reason for treatment discontinuation | |||
| Death | 3 (21.4) | 1 (8.3) | 4 (15.4) |
| Disease progression∗∗ | 3 (21.4) | 1 (8.3) | 4 (15.4) |
| AEs other than disease progression†† | 0 | 0 | 0 |
| Required subsequent EBV therapy‡‡ | 2 (14.3) | 1 (8.1) | 3 (11.5) |
| Received maximum available tabelecleucel cell products | 1 (7.1) | 1 (8.3) | 2 (7.7) |
| Physician decision | 1 (7.1) | 1 (8.3) | 2 (7.7) |
| Patient preference | 2 (14.3) | 1 (8.3) | 3 (11.5) |
| Other§§ | 1 (7.1) | 0 | 1 (3.8) |
| No. of tabelecleucel lots received, n (%) | |||
| 1 | 14 (100) | 8 (66.7) | 22 (84.6) |
| 2 | 0 | 3 (25.0) | 3 (11.5) |
| 3 | 0 | 0 | 0 |
| 4 | 0 | 1 (8.3) | 1 (3.8) |
| Characteristics . | HCT (n = 14) . | SOT (n = 12) . | Total (N = 26) . |
|---|---|---|---|
| Median age, y (range) | 46.0 (2-74) | 27.5 (7-66) | 36.0 (2-74) |
| Age category, n (%) | |||
| <16 y | 2 (14.3) | 4 (33.3) | 6 (23.1) |
| ≥16 y | 12 (85.7) | 8 (66.7) | 20 (76.9) |
| Male, n (%) | 7 (50.0) | 6 (50.0) | 13 (50.0) |
| Ethnicity, n (%) | |||
| Hispanic/Latino | 1 (7.1) | 2 (16.7) | 3 (11.5) |
| Not Hispanic/Latino | 11 (78.6) | 8 (66.7) | 19 (17.3) |
| Not given | 2 (14.3) | 2 (16.7) | 4 (15.4) |
| Race, n (%) | |||
| Caucasian | 10 (71.4) | 8 (66.7) | 18 (69.2) |
| Black | 1 (7.1) | 0 | 1 (3.8) |
| Asian | 2 (14.3) | 1 (8.3) | 3 (11.5) |
| Other/unknowns | 1 (7.1) | 3 (25.0) | 4 (15.4) |
| Median ECOG PS score (range)∗ | 1.5 (0-4) | 1.0 (0-3) | 1.0 (0-4) |
| Median Lansky score (range)† | 55.0 (50-60) | 80.0 (20-90) | 65.0 (20-90) |
| Disease risk parameters, n (%)∗ | |||
| Age of ≥60 y | 2 (16.7) | 1 (12.5) | 3 (15.0) |
| ECOG PS score of ≥2 | 6 (50.0) | 3 (37.5) | 9 (45.0) |
| Elevated serum LDH | 7 (58.3) | 4 (50.0) | 11 (55.0) |
| Risk score∗,,‡ | |||
| High | 3 (25.0) | 2 (25.0) | 5 (25.0) |
| Intermediate | 8 (66.7) | 4 (50.0) | 12 (60.0) |
| Low | 1 (8.3) | 2 (25.0) | 3 (15.0) |
| Disease morphology/histology, n (%)§ | |||
| Diffuse large B-cell lymphoma | 4 (28.6) | 8 (66.7) | 12 (46.2) |
| PTLD NOS | 6 (42.9) | 0 | 6 (23.1) |
| Polymorphic PTLD | 2 (14.3) | 1 (8.3) | 3 (11.5) |
| Hodgkin lymphoma | 0 | 1 (8.3) | 1 (3.8) |
| Infectious mononucleosis–like PTLD | 0 | 1 (8.3) | 1 (3.8) |
| Lymphoproliferative disorder NOS | 1 (7.1) | 0 | 1 (3.8) |
| Monomorphic B-cell PTLD | 0 | 1 (8.3) | 1 (3.8) |
| Transplanted organ, n (%) | |||
| Kidney | N/A | 6 (50.0) | N/A |
| Heart | N/A | 2 (16.7) | N/A |
| Lung | N/A | 2 (16.7) | N/A |
| Intestine | N/A | 2 (16.7) | N/A |
| Median time | |||
| Median time from transplant to diagnosis of EBV+ PTLD, mo (range) | 4.4 (1.4-198.4) | 7.2 (2.1-275.9) | 5.1 (1.4-275.9) |
| Median time from transplant to first dose of tabelecleucel, mo (range) | 6.4 (2.3-202.2) | 20.5 (2.3-281.3) | 9.3 (2.3-281.3) |
| Median time from initial EBV-related disease diagnosis to first tabelecleucel dose, mo (range) | 1.4 (0.2-8.2) | 5.0 (0.2-67.6) | 2.3 (0.2-67.6) |
| Baseline CNS PTLD involvement, n (%)‖ | 1 (7.1) | 1 (8.3) | 2 (7.7) |
| Baseline extranodal PTLD (including bone marrow), n (%)¶ | 1 (7.1) | 3 (25.0) | 4 (15.4) |
| Prior rituximab therapy, n (%)# | 14 (100) | 11 (91.7) | 25 (96.2) |
| Prior chemotherapy, n (%) | 1 (7.1) | 7 (58.3) | 8 (30.8) |
| Median number of lines of prior systemic therapies (range) | 1.0 (1-3) | 1.5 (1-3) | 1.0 (1-3) |
| Use of immunosuppressive medications at start of tabelecleucel, n (%) | 1 (7.1) | 11 (91.7) | 12 (46.2) |
| Treatment on trial | |||
| Median of average cells administered per dose (×106 cells per kg) (range) | 1.98 (1.6-2.0) | 1.98 (1.6-2.0) | 1.98 (1.6-2.0) |
| Median duration of tabelecleucel treatment, mo (range) | 1.3 (0.03-3.1) | 2.5 (1.2-10.4) | 1.8 (0.03-10.4) |
| Median no. of tabelecleucel doses received (range) | 4.0 (1-9) | 7.0 (4-27) | 6.0 (1-27) |
| Median no. of tabelecleucel cycles received (range) | 2.0 (1-4) | 2.5 (2-9) | 2.0 (1-9) |
| Reason for treatment discontinuation | |||
| Death | 3 (21.4) | 1 (8.3) | 4 (15.4) |
| Disease progression∗∗ | 3 (21.4) | 1 (8.3) | 4 (15.4) |
| AEs other than disease progression†† | 0 | 0 | 0 |
| Required subsequent EBV therapy‡‡ | 2 (14.3) | 1 (8.1) | 3 (11.5) |
| Received maximum available tabelecleucel cell products | 1 (7.1) | 1 (8.3) | 2 (7.7) |
| Physician decision | 1 (7.1) | 1 (8.3) | 2 (7.7) |
| Patient preference | 2 (14.3) | 1 (8.3) | 3 (11.5) |
| Other§§ | 1 (7.1) | 0 | 1 (3.8) |
| No. of tabelecleucel lots received, n (%) | |||
| 1 | 14 (100) | 8 (66.7) | 22 (84.6) |
| 2 | 0 | 3 (25.0) | 3 (11.5) |
| 3 | 0 | 0 | 0 |
| 4 | 0 | 1 (8.3) | 1 (3.8) |
LDH, lactate dehydrogenase; N/A, not applicable; NOS, not otherwise specified; PS, performance status.
For patients aged >16 years.
For patients aged ≤16 years.
Scored using PTLD–adapted prognostic index. Per Choquet et al,24 high-risk patients had ≥2 of the following: age ≥60 years, ECOG PS score of ≥2, and/or elevated LDH at, or before, first dose of tabelecleucel.
Disease morphology/histology was collected for 25 of 26 patients.
Baseline CNS disease was not officially evaluated by imaging because of low clinical suspicion in 21 of 26 patients (10/12 of SOT and 11/14 of HCT).
Baseline extranodal disease was missing in 1 patient and not evaluable in 2 patients.
Administered as a monotherapy; however, patients may have received other prior treatments for PTLD.
Includes 1 patient with primary disease progression.
All AEs that led to treatment discontinuation were unrelated to tabelecleucel.
Subsequent EBV therapies included immunotherapy, chemotherapy, or radiotherapy.
Initiation of non-protocol CTL treatment for cytomegalovirus disease.