{"doi":"10.1016/j.leukres.2022.106928","title":"CD123-directed allogeneic chimeric-antigen receptor T-cell therapy (CAR-T) in blastic plasmacytoid dendritic cell neoplasm (BPDCN): Clinicopathological insights","abstract":null,"journal":"Leukemia Research","year":2022,"id":639715,"datarank":0.6438263988345359,"base_score":3.044522437723423,"endowment":3.044522437723423,"self_citation_contribution":0.4566783656585135,"citation_network_contribution":0.18714803317602233,"self_endowment_contribution":0.4566783656585135,"citer_contribution":0.18714803317602233,"corpus_percentile":null,"corpus_rank":null,"citation_count":20,"citer_count":17,"citers_with_citation_signal":11,"citers_with_endowment":11,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":689106,"name":"Nathaniel R. Wilson","orcid":"0000-0002-9123-1781","position":1,"is_corresponding":false},{"id":1060773,"name":"Jayastu Senapati","orcid":"0000-0002-6831-2567","position":2,"is_corresponding":false},{"id":1251021,"name":"Minas P. Economides","orcid":"0000-0003-4625-421X","position":3,"is_corresponding":false},{"id":1662271,"name":"Monica L. Guzman","orcid":null,"position":4,"is_corresponding":false},{"id":11096,"name":"Sattva S. Neelapu","orcid":"0000-0003-1045-4914","position":5,"is_corresponding":false},{"id":1662272,"name":"Rossana Kazemimood","orcid":null,"position":6,"is_corresponding":false},{"id":1662273,"name":"Richard Eric Davis","orcid":null,"position":7,"is_corresponding":false},{"id":232850,"name":"Nitin Jain","orcid":"0000-0003-0114-8384","position":8,"is_corresponding":false},{"id":304997,"name":"Joseph D. Khoury","orcid":"0000-0003-2621-3584","position":9,"is_corresponding":false},{"id":468177,"name":"Mayumi Sugita","orcid":"0000-0001-8510-7154","position":10,"is_corresponding":false},{"id":691208,"name":"Tianyu Cai","orcid":"0000-0002-5271-3235","position":11,"is_corresponding":false},{"id":488777,"name":"Julianne Smith","orcid":"0000-0002-2536-5933","position":12,"is_corresponding":false},{"id":555152,"name":"Mark G. Frattini","orcid":null,"position":13,"is_corresponding":false},{"id":1117099,"name":"Andrew Garton","orcid":"0000-0003-4206-9084","position":14,"is_corresponding":false},{"id":1542214,"name":"Gail Roboz","orcid":null,"position":15,"is_corresponding":false},{"id":18373,"name":"Marina Konopleva","orcid":"0000-0002-9347-2212","position":16,"is_corresponding":false},{"id":232838,"name":"Naveen Pemmaraju","orcid":"0000-0002-1670-6513","position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"CD123-directed allogeneic chimeric-antigen receptor T-cell therapy (CAR-T) in blastic plasmacytoid dendritic cell neoplasm (BPDCN): Clinicopathological insights","abstract":"<h4>Purpose</h4>Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a hematologic malignancy associated with overexpression of CD123. Allogeneic chimeric antigen receptor T cells (CAR-T) directed against CD123 in BPDCN have been studied in clinical trials. We performed post-mortem analysis of a patient treated with anti-CD123 CAR-T to elucidate cause of death, development of cytokine release syndrome (CRS), and tissue distribution of UCART123 cells.<h4>Methods</h4>A post-mortem multidisciplinary clinicopathologic analysis was performed with digital droplet polymerase chain reaction of isolated blood and tissue ribonucleic acid (RNA) to evaluate tissue distribution of infused CAR-T. Multiparameter flow cytometry for detection of CAR-T was used for whole blood samples. Cytokine levels in plasma were measured using multiplex bead assay. Gene expression profiling on isolated RNA was performed using semi-custom Nanostring immune gene panel and RNA-sequence method. RNA in situ hybridization was performed using CAR-specific probe.<h4>Results</h4>The patient developed severe clinical CRS refractory to corticosteroids, tocilizumab, and lymphodepletion. Despite significant reduction in BPDCN lesions, the patient passed away on day 9 of CAR-T. Autopsy results show that following lymphodepletion and UCART123 administration, the patient remained severely lymphopenic with few UCART123 cells detected, predominantly localized to spleen.<h4>Conclusions</h4>No definitive cause of death was determined, but we hypothesized that the patient may have succumbed to CAR-T-mediated cardiopulmonary toxicity. UCART123 cells displayed low overall distribution, with predominance in immune organs and tissues. Mechanism of CRS development is still poorly understood in patients receiving CAR-T therapy. Future directions in the field developing CD123-targeted agents in BPDCN are discussed.","is_dataset_classified":null,"base_score":0.0,"endowment":0.0,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"35963025","pmcid":null,"openalex_id":null,"authors":[],"funders":[{"funder_name":"University of Texas MD Anderson Cancer Center","grant_id":"CA016672","title":null},{"funder_name":"University of Texas MD Anderson Cancer Center","grant_id":"CA100632","title":null}],"total_grants":2,"fwci":null,"citation_percentile":null,"influential_citations":0,"citation_trend":[],"oa_status":"hybrid","license":"cc-by-nc-nd","oa_locations":[{"url":"https://doi.org/10.1016/j.leukres.2022.106928","host_type":"publisher"},{"url":"https://api.elsevier.com/content/article/PII:S0145212622001540?httpAccept=text/xml","host_type":"publisher"},{"url":"https://api.elsevier.com/content/article/PII:S0145212622001540?httpAccept=text/plain","host_type":"publisher"}],"fields_of_study":[],"mesh_terms":["Dendritic Cells","Humans","Hematologic Neoplasms","Skin Neoplasms","Myeloproliferative Disorders","Acute Disease","RNA","Cytokines","Hematopoietic Stem Cell Transplantation","Interleukin-3 Receptor alpha Subunit","Receptors, Chimeric Antigen"],"keywords":["Autopsy","Cytokine Release Syndrome","Bpdcn","Allogeneic Car-t"],"sdg_mappings":[],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-07T01:21:49.226509Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}