Table 1.
Comparisons of indications and outcomes of allogeneic hematopoeitic transplantation and CAR T-cell therapy.
| Allogeneic HCT | CAR-T | |
|---|---|---|
| B-ALL | ||
| Indications | High-risk CR1, ≥CR2, Post CAR-T (especially early loss of B-cell aplasia, no prior alloHCT), controversial in active disease | Refractory or 2nd or greater relapse in ≤25 years-old (tisagenlecleucel) Efficacy seen in post alloHCT In clinical development for adult patients: dual-targeting CAR, relapse post CD19 CAR, “off-the-shelf” allogeneic CAR T |
| CR | N/A | 60%–80% (adults) 70%–90% (pediatrics) |
| OS | 30%–60% at 3 years (adults) 60%–75% at 3 years (pediatrics) 20% at 3 years (alloHCT with active disease) |
40%–70% at 2 years |
| Toxicity | aGVHD, cGVHD, graft failure and prolonged cytopenias, infections | CRS, ICANS, prolonged cytopenias, infection |
| References | (31–34) | See Tables 2 and 3 |
| AML | ||
| Indications | Intermediate or unfavorable risk in CR1, ≥CR2, active disease (usually on a clinical trial) | Currently in clinical development for rel/ref active disease CAR Targets: NKG2D, CD123, CLL-1, and CD33 |
| CR/CRi | N/A | 50% (early phase I data) |
| OS | 25%–60% at 3 years (adults) 30%–70% at 3 years (pediatrics) 10%–20% at 3 years (active disease) |
N/A |
| Toxicity | aGVHD, cGVHD, graft failure and prolonged cytopenias, infections | CRS, ICANS, marrow ablation (theoretical) |
| References | (33, 35) | (36–38) |
| DLBCL | ||
| Indications | Relapse after ASCT – best in patients with >12 mo remission after ASCT, chemosensitive disease, lower NRM with RIC | Rel/ref after two lines of therapy (FDA indications for tisagenlecleucel and axi-cel) Allogeneic CAR T, dual-targeting CAR T, relapse post-CD19 CAR T in clinical development |
| CR | N/A | 40%–60% |
| PFS | 40% at 3 years | Axi-cel: 75% at 2 years in responders, 22% at 2 years in patients with SD Tisagenlecleucel: 83% at 1 year in responders |
| OS | 54% at 3 years | Axi-cel: 50% at 2 years (ITT) Tisagenlecleucel: 40% at 1 year (ITT) |
| NRM | 25%–30% | 4% (axi-cel), 0% (tisagenlecleucel) |
| References | (39) | See Table 4 |
| FL | ||
| Indications | Rel/ref FL – better outcomes with chemosensitive disease and RIC | Rel/ref FL (axi-cel, in clinical development) |
| CR | N/A | 80% |
| PFS | 50% at 5 years | 50% at 2 years |
| OS | 60% at 5 years | 90% at 2 year |
| NRM | 20% | 3% |
| References | (40, 41) | (42, 43) |
| MCL | ||
| Indications | Rel/ref MCL – better outcomes with chemosensitive disease, RIC, earlier in disease course although controversial | Rel/ref MCL having received at least 2 lines of therapy (brexucabtagene autoleucel, approved indication) |
| CR | N/A | 67% |
| PFS | 40%–50% at 3 years | 61% at 1 year |
| OS | 40%–60% at 3 years | 83% at 1 year |
| NRM | 15%–25% | 3% |
| References | (44) | (45) |
| MM | ||
| Indications | Rarely indicated – usually in a younger patient or high-risk disease as part of a clinical trial | In clinical development for triple-class refractory disease and suboptimal response to ASCT |
| CR | N/A | 30%–90% |
| PFS | 30%–40% at 3 years | 40%–50% at 1 year |
| OS | 50% at 3 years | 75%–90% at 1 year |
| NRM | 20%–25% | 2%–5% |
| References | (12, 46) | (47–49) |
Allo-HCT, allogeneic hematopoietic cell transplantation; aGVHD, acute graft-vs-host disease; AML, acute myeloid leukemia; B-ALL, B-cell acute lymphoblastic leukemia; CR, complete response; CRS, cytokine release syndrome; DLBCL, diffuse large B-cell lymphoma; FL, follicular lymphoma; MCL, mantle cell lymphoma; N/A, not available; NRM, non-relapse mortality; OS, overall survival; PFS, progression-free survival; PR, partial response; rel/ref, relapsed/refractory.