TABLE 1.
Summary of CAR‐T‐OPENIA as reported in pivotal trials
| Clinical trial (CAR target, disease) | Anemia | Thrombocytopenia | Neutropenia | Leukopenia | Additional comments |
|---|---|---|---|---|---|
| ELIANA (CD19 CAR T‐cell, pediatric/young adult ALL) [8] | Grade 3–4: 4% | Grade 3–4: 7% | Grade 3–4: 11% | Grade 3–4: 9% | 71% patients with grade 3–4 thrombocytopenia and 80% with grade 3–4 neutropenia had improved to grade 2 or lower at the time of last assessment. |
| ZUMA‐1 (CD19 CAR T‐cell, DLBCL) [1] | Grade 3–4: 43% | Grade 3–4: 38% | Grade 3–4: 78% | Grade 3–4: 29% | At 3 months, 17% of the patients had grade 3 or higher cytopenias including anemia (3%), thrombocytopenia (7%), and neutropenia (11%). |
| JULIET (CD19 CAR T‐cell, DLBCL) [4] | Grade 3–4: 39% | Grade 3–4: 28% | Grade 3–4: 33% | Grade 3–4: 31% | At day 28, 41% patients had unresolved grade 3–4 thrombocytopenia, and 24% had unresolved grade 3–4 neutropenia. At 3 months, 38% had unresolved grade 3–4 thrombocytopenia, and no patients had unresolved grade 3–4 neutropenia. |
| TRANSCEND (CD19 CAR T‐cell, DLBCL) [10] | Grade 3–4: 37% | Grade 3–4: 27% | Grade 3–4: 60% | Grade 3–4: 14% | At day 29, 37% of the patients had grade 3 or higher cytopenia. By day 90, recovery to grade 2 or lower seen in 82% (anemia), 62% (thrombocytopenia), and 84% (neutropenia). |
| ZUMA‐2 (CD19 CAR T‐cell, MCL) [2] | Grade 3–4: 50% | Grade 3–4: 51% | Grade 3–4: 85% | N.R | At day 90, 12% had persistent anemia while 16% patients had persistent thrombocytopenia and neutropenia. |
| ZUMA‐3 (CD19 CAR T‐cell, adult ALL) [3] | Grade 3–4:49% | Grade 3–4: 30% | Grade 3–4: 27% | Grade 3–4: 23% | At day 30, 36% of patients had grade 3 or higher cytopenia: anemia 7%, thrombocytopenia 18%, neutropenia 25%. |
| KarMMa (BCMA CAR T‐cell, Multiple Myeloma) [6] | Grade 3–4: 60% | Grade 3–4: 52% | Grade 3–4: 89% | Grade 3–4: 39% |
Among patients with >1 month grade 3–4 cytopenia, recovery to grade 2 or lower occurred at a median: 2.1 months (range, 1.2‐13.8) for thrombocytopenia and 1.9 months (range, 1.2–5.6) for neutropenia. |
| CARTITUDE‐1 (BCMA CAR T‐cell, Multiple Myeloma) [7] | Grade 3–4: 68% | Grade 3–4: 60% | Grade 3–4: 95% | Grade 3–4: 61% | Patients with grade 3–4 cytopenias recovered to grade 2 or lower by day 30 in 59% (thrombocytopenia), and 70% (neutropenia). |
| Jain et al. Blood Advances 2020 [26] | Grade 3–4: 77% | Grade 3–4: 65% | Grade 3–4: 95% | Grade 3–4: 100% | Normalization at 1 month in 7% (hemoglobin), 23% (platelets), 30% (neutrophils), 13% (WBC count). |
Abbreviations: ALL, acute lymphoblastic leukemia; BCMA, B cell maturation antigen; CAR, chimeric antigen receptor; DLBCL, diffuse large B cell lymphoma; MCL, mantle cell lymphoma; NR, not reported.