ArticleBlood advances2022
Tisagenlecleucel outcomes in relapsed/refractory extramedullary ALL: a Pediatric Real World CAR Consortium Report.
Article in Blood advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 papers, 1 of them a synthesis that pooled it.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
50 citing papers in PubMed, 1 synthesis or guideline pooled it, 77 citations in OpenAlex.
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- 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.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023Trial
- B Cell Aplasia Following CAR T Cell Therapy: Incidence, Kinetics, Prognostic Implications, and Clinical Management.Cancers · 2026Review
- Successful autologous T cell apheresis in pediatric patients with hematologic malignancies, solid tumors, and brain tumors.Cytotherapy · 2026Article
- Reaching consensus in relapsed/refractory ALL in AYAs.Blood advances · 2026Article
- The evolving landscape of CAR T cell therapy in children and young adults with B cell acute lymphoblastic leukemia.Molecular therapy. Oncology · 2026Review
- CD19/CD22 bivalent CAR T cells in children, adolescents and young adults with B-ALL: final phase 1 trial results.Journal for immunotherapy of cancer · 2026Article
- CD19 CAR T cells for B-ALL with EMD.Blood advances · 2026Article
- CD19 CAR T-cell outcomes in relapsed/refractory extramedullary B-ALL: a multisite, retrospective cohort review.Blood advances · 2026Article
- The quintessential role for CAR T cell therapy in children, adolescents and young adults with cancer.Nature reviews. Clinical oncology · 2026Review
- Ten-year experience of CD22 CAR T cells for children and young adults with B-cell acute lymphoblastic leukemia.Blood advances · 2026Article
- Efficacy of chimeric antigen receptor T-cell therapy in testicular relapse of pediatric acute lymphoblastic leukemia: a multicenter retrospective study.Frontiers in immunology · 2026Article
- With BiTEs at the kiddie table, where do CARs come in for pediatric B-ALL?Hematology. American Society of Hematology. Education Program · 2025Review
- Harmonization on defining B-cell recovery post CD19-CAR T-cell therapy in B-cell acute lymphoblastic leukemia: An international consensus statement.HemaSphere · 2025Article
- Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry.Blood advances · 2025Article
- Outcomes of brexucabtagene autoleucel in patients with relapsed/refractory acute lymphoblastic leukemia with CNS involvement.Blood advances · 2025Article
- CAR T-cells for acute leukemias in children: current status, challenges, and future directions.Cancer metastasis reviews · 2025Review
- Therapies and outcomes following extramedullary relapse of pediatric and young adult ALL after CD19 CAR T-cell therapy.Blood advances · 2025Article
- Prominent efficacy and good safety of sequential CD19 and CD22 CAR-T therapy in relapsed/refractory adult B-cell acute lymphoblastic leukemia.Experimental hematology & oncology · 2025Article
- Review
Corrections and comments
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Authors and funding
31 authors at 18 institutions in 1 country.
Funding
Abstract
Chimeric antigen receptor (CAR) T cells have transformed the therapeutic options for relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia. Data for CAR therapy in extramedullary (EM) involvement are limited. Retrospective data were abstracted from the Pediatric Real World CAR Consortium (PRWCC) of 184 infused patients from 15 US institutions. Response (complete response) rate, overall survival (OS), relapse-free survival (RFS), and duration of B-cell aplasia (BCA) in patients referred for tisagenlecleucel with EM disease (both central nervous system (CNS)3 and non-CNS EM) were compared with bone marrow (BM) only. Patients with CNS disease were further stratified for comparison. Outcomes are reported on 55 patients with EM disease before CAR therapy (CNS3, n = 40; non-CNS EM, n = 15). The median age at infusion in the CNS cohort was 10 years (range, <1-25 years), and in the non-CNS EM cohort it was 13 years (range, 2-26 years). In patients with CNS disease, 88% (35 of 40) achieved a complete response vs only 66% (10 of 15) with non-CNS EM disease. Patients with CNS disease (both with and without BM involvement) had 24-month OS outcomes comparable to those of non-CNS EM or BM only (P = .41). There was no difference in 12-month RFS between CNS, non-CNS EM, or BM-only patients (P = .92). No increased toxicity was seen with CNS or non-CNS EM disease (P = .3). Active CNS disease at time of infusion did not affect outcomes. Isolated CNS disease trended toward improved OS compared with combined CNS and BM (P = .12). R/R EM disease can be effectively treated with tisagenlecleucel; toxicity, relapse, and survival rates are comparable to those of patients with BM-only disease. Outcomes for isolated CNS relapse are encouraging.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.