CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now?
<b>Background</b>: Patients with refractory and relapsed acute myeloid leukemia (R/R AML) face a dismal prognosis. CAR-T therapy has emerged as a potential treatment option. This study assesses the available clinical evidence on CAR-T in R/R AML, focusing on safety and efficacy outcomes....
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| Language: | English |
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MDPI AG
2025-05-01
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| Series: | Current Oncology |
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| Online Access: | https://www.mdpi.com/1718-7729/32/6/322 |
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| author | Pilar Lloret-Madrid Pedro Chorão Manuel Guerreiro Pau Montesinos |
| author_facet | Pilar Lloret-Madrid Pedro Chorão Manuel Guerreiro Pau Montesinos |
| author_sort | Pilar Lloret-Madrid |
| collection | DOAJ |
| description | <b>Background</b>: Patients with refractory and relapsed acute myeloid leukemia (R/R AML) face a dismal prognosis. CAR-T therapy has emerged as a potential treatment option. This study assesses the available clinical evidence on CAR-T in R/R AML, focusing on safety and efficacy outcomes. <b>Methods</b>: We included studies on CAR-T therapy for R/R AML published from June 2014 to January 2025. Data on patient and disease characteristics, CAR-T constructs, response rates, post-CAR-T allogeneic HSCT (allo-HSCT), and safety outcomes were analyzed. <b>Results</b>: Twenty-five CAR-T clinical trials involving 296 patients were identified. The most frequently targeted antigens were CD33, CD123, and CLL-1, while CD7, CD19, NKG2D, and CD38 were also explored. Responses were heterogeneous and often short-lived when not consolidated with allo-HSCT. Cytokine release syndrome and neurotoxicity were generally low grade and manageable. Prolonged and severe myelosuppression was a frequent limiting toxicity, often requiring allo-HSCT to restore hematopoiesis. Disease progression was the leading cause of death, followed by infections. <b>Conclusions</b>: CAR-T cell therapy may represent a feasible therapeutic strategy, particularly as bridging to allo-HSCT to mitigate myelotoxicity and improve long-term outcomes. Nevertheless, it remains in the early stages of development and faces significant efficacy and safety challenges that must be addressed in future trials to enable the expansion of this promising therapeutic approach for a population with high unmet medical needs. |
| format | Article |
| id | doaj-art-bc2d447dcda64100bf14b0b3e2e9a326 |
| institution | Kabale University |
| issn | 1198-0052 1718-7729 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Current Oncology |
| spelling | doaj-art-bc2d447dcda64100bf14b0b3e2e9a3262025-08-20T03:24:32ZengMDPI AGCurrent Oncology1198-00521718-77292025-05-0132632210.3390/curroncol32060322CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now?Pilar Lloret-Madrid0Pedro Chorão1Manuel Guerreiro2Pau Montesinos3Hematology Department, Hospital Universitari i Politècnic La Fe, 46026 Valencia, SpainHematology Department, Hospital Universitari i Politècnic La Fe, 46026 Valencia, SpainHematology Department, Hospital Universitari i Politècnic La Fe, 46026 Valencia, SpainHematology Department, Hospital Universitari i Politècnic La Fe, 46026 Valencia, Spain<b>Background</b>: Patients with refractory and relapsed acute myeloid leukemia (R/R AML) face a dismal prognosis. CAR-T therapy has emerged as a potential treatment option. This study assesses the available clinical evidence on CAR-T in R/R AML, focusing on safety and efficacy outcomes. <b>Methods</b>: We included studies on CAR-T therapy for R/R AML published from June 2014 to January 2025. Data on patient and disease characteristics, CAR-T constructs, response rates, post-CAR-T allogeneic HSCT (allo-HSCT), and safety outcomes were analyzed. <b>Results</b>: Twenty-five CAR-T clinical trials involving 296 patients were identified. The most frequently targeted antigens were CD33, CD123, and CLL-1, while CD7, CD19, NKG2D, and CD38 were also explored. Responses were heterogeneous and often short-lived when not consolidated with allo-HSCT. Cytokine release syndrome and neurotoxicity were generally low grade and manageable. Prolonged and severe myelosuppression was a frequent limiting toxicity, often requiring allo-HSCT to restore hematopoiesis. Disease progression was the leading cause of death, followed by infections. <b>Conclusions</b>: CAR-T cell therapy may represent a feasible therapeutic strategy, particularly as bridging to allo-HSCT to mitigate myelotoxicity and improve long-term outcomes. Nevertheless, it remains in the early stages of development and faces significant efficacy and safety challenges that must be addressed in future trials to enable the expansion of this promising therapeutic approach for a population with high unmet medical needs.https://www.mdpi.com/1718-7729/32/6/322acute myeloid leukemiarelapsed or refractory AMLCAR-T cell therapy |
| spellingShingle | Pilar Lloret-Madrid Pedro Chorão Manuel Guerreiro Pau Montesinos CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now? Current Oncology acute myeloid leukemia relapsed or refractory AML CAR-T cell therapy |
| title | CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now? |
| title_full | CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now? |
| title_fullStr | CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now? |
| title_full_unstemmed | CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now? |
| title_short | CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now? |
| title_sort | car t cell therapy for acute myeloid leukemia where do we stand now |
| topic | acute myeloid leukemia relapsed or refractory AML CAR-T cell therapy |
| url | https://www.mdpi.com/1718-7729/32/6/322 |
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