Journal of Clinical Pediatrics ›› 2024, Vol. 42 ›› Issue (7): 573-577.doi: 10.12372/jcp.2024.24e0578
• Expert Review • Previous Articles Next Articles
LI Benshang, YANG Liu
Received:2024-06-05
Published:2024-07-15
Online:2024-07-08
LI Benshang, YANG Liu. Challenges and prospects of CAR-T treatment for pediatric disease[J].Journal of Clinical Pediatrics, 2024, 42(7): 573-577.
| [1] | Daei Sorkhabi A, Mohamed Khosroshahi L, Sarkesh A, et al. The current landscape of CAR T-cell therapy for solid tumors: mechanisms, research progress, challenges, and counterstrategies[J]. Front Immunol, 2023, 14: 1113882. |
| [2] | Pui CH, Yang JJ, Hunger SP, et al. Childhood acute lymphoblastic leukemia: progress through collaboration[J]. J Clin Oncol, 2015, 33(27): 2938-2948. |
| [3] | Gupta A, Cripe TP. Immunotherapies for pediatric solid tumors: a targeted update[J]. Paediatr Drugs, 2022, 24(1): 1-12. |
| [4] |
Wang F, Fu K, Wang Y, et al. Small-molecule agents for cancer immunotherapy[J]. Acta Pharm Sin B, 2024, 14(3): 905-952.
doi: 10.1016/j.apsb.2023.12.010 pmid: 38486980 |
| [5] |
Cuesta-Mateos C, Alcaraz-Serna A, Somovilla-Crespo B, et al. Monoclonal antibody therapies for hematological malignancies: not just lineage-specific targets[J]. Front Immunol, 2017, 8: 1936.
doi: 10.3389/fimmu.2017.01936 pmid: 29387053 |
| [6] |
Cappell KM, Kochenderfer JN. Long-term outcomes following CAR T cell therapy: what we know so far[J]. Nat Rev Clin Oncol, 2023, 20(6): 359-371.
doi: 10.1038/s41571-023-00754-1 pmid: 37055515 |
| [7] | Talleur AC, Qudeimat A, Métais JY, et al. Preferential expansion of CD8+ CD19-CAR T cells postinfusion and the role of disease burden on outcome in pediatric B-ALL[J]. Blood Adv, 2022, 6(21): 5737-5749. |
| [8] | Zhu M, Wu B, Brandl C, et al. Blinatumomab, a bispecific T-cell engager (BiTE®) for CD19- targeted cancer immunotherapy: clinical pharmacology and its implications[J]. Clin Pharmacokinet, 2016, 55(10): 1271-1288. |
| [9] | Wang T, Tang Y, Cai J, et al. Coadministration of CD19- and CD22-directed chimeric antigen receptor T-Cell therapy in childhood B-cell acute lymphoblastic leukemia: a single-arm, multicenter, phase Ⅱ trial[J]. J Clin Oncol, 2023, 41(9): 1670-1683. |
| [10] |
Xiao X, Huang S, Chen S, et al. Mechanisms of cytokine release syndrome and neurotoxicity of CAR T-cell therapy and associated prevention and management strategies[J]. J Exp Clin Cancer Res, 2021, 40(1): 367.
doi: 10.1186/s13046-021-02148-6 pmid: 34794490 |
| [11] |
Siegler EL, Kenderian SS. Neurotoxicity and cytokine release syndrome after chimeric antigen receptor T cell therapy: insights into mechanisms and novel therapies[J]. Front Immunol, 2020, 11: 1973.
doi: 10.3389/fimmu.2020.01973 pmid: 32983132 |
| [12] | Hines MR, Knight TE, McNerney KO, et al. Immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome[J]. Transplant Cell Ther, 2023, 29(7): 438. |
| [13] | Wat J, Barmettler S. Hypogammaglobulinemia after chimeric antigen receptor CAR T-cell therapy: characteristics, management, and future directions[J]. J Allergy Clin Immunol Pract, 2022, 10(2): 460-466. |
| [14] | Jain MD, Smith M, Shah NN. How I treat refractory CRS and ICANS after CAR T-cell therapy[J]. Blood, 2023, 141(20): 2430-2442. |
| [15] | Zhang Y, Zhou F, Wu Z, et al. Timing of tocilizumab administration under the guidance of IL-6 in CAR-T therapy for R/R acute lymphoblastic leukemia[J]. Front Immunol, 2022, 13:914959. |
| [16] | Rainone M, Ngo D, Baird JH, et al. Interferon-γ blockade in CAR T-cell therapy-associated macrophage activation syndrome/hemophagocytic lymphohistiocytosis[J]. Blood Adv, 2023, 7(4): 533-536. |
| [17] |
Manni S, Del Bufalo F, Merli P, et al. Neutralizing IFNγ improves safety without compromising efficacy of CAR-T cell therapy in B-cell malignancies[J]. Nat Commun, 2023, 14(1): 3423.
doi: 10.1038/s41467-023-38723-y pmid: 37296093 |
| [18] | Xu N, Yang XF, Xue SL, et al. Ruxolitinib reduces severe CRS response by suspending CAR-T cell function instead of damaging CAR-T cells[J]. Biochem Biophys Res Commun, 2022, 595: 54-61. |
| [19] | Mestermann K, Giavridis T, Weber J, et al. The tyrosine kinase inhibitor dasatinib acts as a pharmacologic on/off switch for CAR T cells[J]. Sci Transl Med, 2019, 11(499): eaau5907. |
| [20] | Ventin M, Cattaneo G, Maggs L, et al. Implications of high tumor burden on chimeric antigen receptor T-Cell immunotherapy: a review[J]. JAMA Oncol, 2024, 10(1): 115-121. |
| [21] | Sterner RC, Sterner RM. Immune effector cell associated neurotoxicity syndrome in chimeric antigen receptor-T cell therapy[J]. Front Immunol, 2022, 13:879608. |
| [22] | Gu T, Hu K, Si X, et al. Mechanisms of immune effector cell-associated neurotoxicity syndrome after CAR-T treatment[J]. WIREs Mech Dis, 2022, 14(6): e1576. |
| [23] | Sheth VS, Gauthier J. Taming the beast: CRS and ICANS after CAR T-cell therapy for ALL[J]. Bone Marrow Transplant, 2021, 56(3): 552-566. |
| [24] | Gatto L, Ricciotti I, Tosoni A, et al. CAR-T cells neurotoxicity from consolidated practice in hematological malignancies to fledgling experience in CNS tumors: fill the gap[J]. Front Oncol, 2023, 13: 1206983. |
| [25] |
Nix MA, Mandal K, Geng H, et al. Surface proteomics reveals CD72 as a target for in vitro-evolved nanobody-based CAR-T cells in KMT2A/MLL1-rearranged B-ALL[J]. Cancer Discov, 2021, 11(8): 2032-2049.
doi: 10.1158/2159-8290.CD-20-0242 pmid: 33727310 |
| [26] |
Leung KT, Cai J, Liu Y, et al. Prognostic implications of CD9 in childhood acute lymphoblastic leukemia: insights from a nationwide multicenter study in China[J]. Leukemia, 2024, 38(2): 250-257.
doi: 10.1038/s41375-023-02089-3 pmid: 38001171 |
| [27] |
Zhao Z, Sadelain M. CAR T cell design: approaching the elusive AND-gate[J]. Cell Res, 2023, 33(10): 739-740.
doi: 10.1038/s41422-023-00828-w pmid: 37221269 |
| [28] | Coorens THH, Collord G, Treger TD, et al. Clonal origin of KMT2A wild-type lineage-switch leukemia following CAR-T cell and blinatumomab therapy[J]. Nature Cancer, 2023, 4(8): 1095-1101. |
| [29] | Gu R, Liu W, Mei Y, et al. The impact of ZNF384 rearranged on antigen editing during treatment-specific selective pressures in adult B cell acute lymphoid leukemia[J]. Blood, 2023, 142: 4209. |
| [30] | De Bie J, Demeyer S, Gielen O, et al. BCR-ABL1 positive B-ALL can undergo T-cell lineage shift to become CD19 negative T-ALL[J]. HemaSphere, 2018, 2(3): e42. |
| [31] | Liang M, Gong D, Wang L, et al. PAX5 haploinsufficiency induced CD8+ T cells dysfunction or exhaustion by high expression of immune inhibitory-related molecules[J]. Cancer Treat Res Commun, 2021, 28: 100437. |
| [32] | Majzner RG, Ramakrishna S, Yeom KW, et al. GD2-CAR T cell therapy for H3K27M-mutated diffuse midline gliomas[J]. Nature, 2022, 603(7903): 934-941. |
| [33] | Bufalo FD, Angelis BD, Caruana I, et al. GD2-CART01 for relapsed or refractory high-risk neuroblastoma[J]. N Engl J Med, 2023, 388(14): 1284-1295. |
| [34] |
Maalej KM, Merhi M, Inchakalody VP, et al. CAR-cell therapy in the era of solid tumor treatment: current challenges and emerging therapeutic advances[J]. Mol Cancer, 2023, 22(1): 20.
doi: 10.1186/s12943-023-01723-z pmid: 36717905 |
| [35] | Ayala Ceja M, Khericha M, Harris CM, et al. CAR-T cell manufacturing: major process parameters and next-generation strategies[J]. J Exp Med, 2024, 221(2): e20230903. |
| [36] |
Yang J, He J, Zhang X, et al. Next-day manufacture of a novel anti-CD19 CAR-T therapy for B-cell acute lymphoblastic leukemia: first-in-human clinical study[J]. Blood Cancer J, 2022, 12(7): 104.
doi: 10.1038/s41408-022-00694-6 pmid: 35798714 |
| [37] | Lin H, Cheng J, Mu W, et al. Advances in universal CAR-T cell therapy[J]. Front Immunol, 2021, 12:744823. |
| [38] |
Jo S, Das S, Williams A, et al. Endowing universal CAR T-cell with immune-evasive properties using TALEN-gene editing[J]. Nat Commun, 2022, 13(1): 3453.
doi: 10.1038/s41467-022-30896-2 pmid: 35773273 |
| [39] | Muller F, Taubmann J, Bucci L, et al. CD19 CAR T-cell therapy in autoimmune disease - a case series with follow-up[J]. N Engl J Med, 2024, 390(8): 687-700. |
| [40] |
Schett G, Mackensen A, Mougiakakos D. CAR T-cell therapy in autoimmune diseases[J]. Lancet, 2023, 402(10416): 2034-2044.
doi: 10.1016/S0140-6736(23)01126-1 pmid: 37748491 |
| [1] | LIN Jinfeng, CHEN Yi, DENG Yan, FENG Ai, HUANG Juan, CHEN Lizhu, XIE Jingqi, NIE Xiaojing. A comparative study on the detection of anti-Nephrin antibodies in children's nephrotic syndrome by chemiluminescence method and cell-based assay [J]. Journal of Clinical Pediatrics, 2026, 44(8): 682-689. |
| [2] | ZHENG Fangyuan, WANG Miao, DING Mingming, LU Aidong, JIA Yueping, ZENG Huimin, ZHANG Leping. Next-generation sequencing identified gene variants and their prognostic impact in pediatric Philadelphia chromosome positive acute lymphoblastic leukemia [J]. Journal of Clinical Pediatrics, 2026, 44(8): 690-697. |
| [3] | ZHANG Xiao, FANG Hongjun, KUANG Xiaojun, WANG Lijuan, WU Zhao, JIANG Sha, LI Pei, WU Liwen. Analysis of the clinical and electrophysiological characteristics of atonic seizures in children [J]. Journal of Clinical Pediatrics, 2026, 44(8): 698-704. |
| [4] | WANG Mengqian, YANG Hui. Case series analysis of 13 intestinal Behcet’s syndrome in children [J]. Journal of Clinical Pediatrics, 2026, 44(8): 705-711. |
| [5] | WU Xirong, GAO Qi, GUO Siyuan, YIN Qingqin, QIN Qiang, YIN Ju, JIAO Anxia, CHEN Chenghao, WU Di, WANG Bei, DUAN Xiaomin, PENG Yun, XU Baoping, GAO Liwei. Clinical characteristics and prognosis of pulmonary mucormycosis in children with diabetes mellitus [J]. Journal of Clinical Pediatrics, 2026, 44(8): 712-720. |
| [6] | HAN Yanan, GE Lanlan, CUI Jieyuan. A case report of branchio-oto-renal syndrome caused by EYA1 gene variant with a 6-year follow-up [J]. Journal of Clinical Pediatrics, 2026, 44(8): 721-726. |
| [7] | SHI Jiayi, GAO Chunlin, JIA Lili, LU Yunyun, SUN Tao, ZHU Xiaodong, XIA Zhengkun, ZHANG Pei. Phenotypic characteristics and prognostic analysis of a pedigree with childhood isolated nephrotic syndrome caused by compound heterozygous mutations in the COQ2 gene [J]. Journal of Clinical Pediatrics, 2026, 44(8): 727-732. |
| [8] | Rachel Rosen, Osvaldo Borrelli, Christophe Faure, Katja Karrento, Usha Krishnan, Samuel Nurko, Nathalie Rommel, Alan Silverman, Michiel van Wijk, Marc Benninga (Author), Chinese Medical Association Parenteral and Enteral Nutrition Branch Pediatrics Group, The Subspecialty Group of Gastroenterology, The Society of Pediatric, Chinese Medical Association, Gastroenterology Group, Pediatrician Branch, Chinese Medical Doctor Association (Translator). Core interpretation and explanation of Rome Ⅴ diagnostic criteria: upper gastrointestinal disorders of gut-brain interaction in children and adolescents [J]. Journal of Clinical Pediatrics, 2026, 44(7): 595-608. |
| [9] | Carlo Di Lorenzo, Miguel Saps, Bruno P. Chumpitazi, Shaman Rajindrajith, Annamaria Staiano, Nikhil Thapar, Miranda van Tilburg, Carlos Velasco-Benítez, Arine Vlieger (Author), Chinese Medical Association Parenteral and Enteral Nutrition Branch Pediatrics Group, The Subspecialty Group of Gastroenterology, The Society of Pediatric, Chinese Medical Association, Gastroenterology Group, Pediatrician Branch, Chinese Medical Doctor Association (Translator). Core interpretation and explanation of Rome Ⅴ diagnostic criteria: lower and biliary disorders of gut-brain interaction in children and adolescents [J]. Journal of Clinical Pediatrics, 2026, 44(7): 609-621. |
| [10] | LI Xufeng, TANG Haoxun, LI Xingjun, ZHANG Lize, GAO Chenqing, ZHAO Shunying, LIU Jianhua. Clinical characteristics, chest CT imaging phenotypes, and their correlation with prognosis in 76 children with Chlamydia pneumoniae pneumonia [J]. Journal of Clinical Pediatrics, 2026, 44(7): 622-627. |
| [11] | WANG Zhen, SHAO Jingbo, ZHANG Na, XIA Min, ZHU Jiashi, DU Chengkan, LI Hong. Factors influencing long-term recurrence in pediatric acute lymphoblastic leukemia patients with low-risk disease at initial diagnosis: a five-year follow-up analysis of 254 cases from a single center [J]. Journal of Clinical Pediatrics, 2026, 44(7): 628-635. |
| [12] | GUO Xuemei, XUE Yao, WANG Yongren, RONG Liucheng, FANG Yongjun, LIN Rufeng. Clinical features and prognosis of childhood acute myeloid leukemia with CBFA2T3::GLIS2 fusion [J]. Journal of Clinical Pediatrics, 2026, 44(7): 636-643. |
| [13] | YANG Lijun, FU Dongxia, CUI Yan, YANG Junmei, ZHANG Liming. Clinical manifestation and genetic analysis of CHARGE syndrome caused by CHD7 gene variation [J]. Journal of Clinical Pediatrics, 2026, 44(7): 644-650. |
| [14] | HU Yan, YANG Fan, LIANG Aimin, MAO Meng. Identification and management of faltering growth in children: insights from the 13th International Conference on Nutrition and Growth [J]. Journal of Clinical Pediatrics, 2026, 44(6): 485-488. |
| [15] | LIU Ziyao, WANG Congying, WANG Hongmao, ZHANG Mingming, XU Yingjie, LAI Jianming, NIU Wenquan, LI Xiaohui. Early differentiation of Kawasaki disease with joint symptoms and systemic juvenile idiopathic arthritis with Kawasaki disease-like symptoms: a single-center retrospective study [J]. Journal of Clinical Pediatrics, 2026, 44(6): 489-498. |
|
||