Evidence map›Paper›PMID 31606419›Full record

Trial reportThe Lancet. Oncology2019

Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial.

Theodore W Laetsch, Gary Douglas Myers, André Baruchel, Andrew C Dietz, Michael A Pulsipher, Henrique Bittencourt, Jochen Buechner, Barbara De Moerloose, Kara L Davis, Eneida Nemecek and 16 more

Registry-linked trialOpen access · greenAbstract readClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The Lancet. Oncology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06879340 (A Phase 1 Multicenter, Open Label Trial Evaluating the Safety and Efficacy of DuoCAR20.19.22-D95 in Adult Patients With Relapsed or Refractory B-cell Malignancies), which is not on this map. Cited by 56 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
56citing papers in PubMed, 7 pooled it
10.2field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

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.

NCT06879340 phase1recruitingnot on this mapstarted 2025, after this paper: background citation

A Phase 1 Multicenter, Open Label Trial Evaluating the Safety and Efficacy of DuoCAR20.19.22-D95 in Adult Patients With Relapsed or Refractory B-cell Malignancies

TypeinterventionalSponsorUniversity of Kansas Medical CenterRan2025 to 2040Enrolled54ConditionsB-Cell Non-Hodgkin Lymphoma, B-cell Acute Lymphoblastic LeukemiaArmsDuoCAR20.19.22-D95, Fludarabine (Conditional therapy), Cyclophosphamide (Conditional therapy)
3 · Its place in the literature

Who cites it

56 citing papers in PubMed, 7 syntheses or guidelines pooled it, 86 citations in OpenAlex.

  1. Pooled it
  2. Guideline
  3. Pooled it
  4. Pooled it
  5. Pooled it
  6. Guideline
  7. Pooled it
  8. Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023
    Trial
  9. Quality of Life in CAR-T Cell Therapy.Hematology reports · 2026
    Review
  10. Article
  11. Article
  12. Review
  13. Review
  14. Article
  15. Review
  16. Review
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  18. Review
  19. Long-term follow-up of CD19-CAR T-cell therapy in children and young adults with B-ALL.Hematology. American Society of Hematology. Education Program · 2023
    Review
  20. Early and Late Toxicities of Chimeric Antigen Receptor T-Cells.Hematology/oncology clinics of North America · 2023
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

26 authors at 20 institutions in 10 countries.

Theodore W LaetschDepartment of Pediatrics and Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern, Dallas, TX, USA; Pauline Allen Gill Center for Cancer and Blood Disorders, Children's Health, Dallas, TX, USA. Electronic address: ted.laetsch@utsouthwestern.edu.
Gary Douglas MyersDivision of BMT/Hematology/Oncology, Children's Mercy Hospital Kansas City, Kansas City, MO, USA.
André BaruchelPediatric Hemato-Immunology Department, Robert Debré University Hospital (APHP) and University Paris Diderot, Paris, France.
Andrew C DietzChildren's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Michael A PulsipherChildren's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Henrique BittencourtHematology-Oncology Division, CHU Sainte-Justine, Université de Montréal, Montréal, QC, Canada.
Jochen BuechnerDepartment of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway.
Barbara De MoerlooseDepartment of Pediatrics, Ghent University Hospital, Ghent, Belgium.
Kara L DavisBass Center for Childhood Cancer and Blood Disorders, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
Eneida NemecekBone Marrow Transplantation Program, Oregon Health & Science University, Portland, OR, USA.
Timothy DriscollDivision of Pediatric Blood and Marrow Transplant, Children's Health Center, Duke University Medical Center, Durham, NC, USA.
Francoise MechinaudChildren's Cancer Centre, The Royal Children's Hospital Melbourne, Melbourne, VIC, Australia.
Nicolas BoisselHematology Adolescent and Young Adult Unit, Saint-Louis Hospital, Paris, France.
Susana RivesDepartment of Pediatric Hematology and Oncology Hospital Sant Joan de Déu de Barcelona, Institut de Recerca Sant Joan de Déu, Barcelona Spain.
Peter BaderDivision for Stem Cell Transplantation and Immunology, Goethe University, Hospital for Children and Adolescents, Frankfurt, Germany.
Christina PetersStem Cell Transplant Unit, St. Anna Children's Hospital, Medical University, Vienna, Austria.
Himalee S SabnisAflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA, USA.
Stephan A GruppDivision of Oncology, Children's Hospital of Philadelphia, and Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Gregory A YanikDepartment of Pediatrics, University of Michigan Medical Center, Ann Arbor, MI, USA.
Hidefumi HiramatsuDepartment of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Heather E StefanskiDepartment of Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, MN, USA.
Lawrence RasouliyanRTI Health Solutions, Barcelona, Spain.
Lan YiNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Sweta ShahNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Jie ZhangNovartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Andrew C HarrisDepartment of Pediatrics, University of Utah, Salt Lake City, UT, USA.
Novartis (United States) · USAflac (United States) · USCenter for Cancer and Blood Disorders · USCentre Hospitalier Universitaire Sainte-Justine · CAChildren's Center · USChildren's Mercy Hospital · USDuke Medical Center · USGhent University Hospital · BEGoethe University Frankfurt · DEHôpital Saint-Louis · FRInstitut de Recerca Sant Joan de DéuKyoto University · JPOregon Health & Science University · USOslo University Hospital · NORoyal Children's Hospital · AUStanford University · USSt Anna Children's Hospital · ATUniversité Paris Cité · FRUniversity of Michigan–Ann Arbor · USUniversity of Minnesota · US

Funding

USC/NORRIS COMPREHENSIVE CANCER CENTER (CORE) SUPPORTP30CA014089 · NCI · UNIVERSITY OF SOUTHERN CALIFORNIA · PI MYLES G COCKBURN · 1985 to 2026
$181.4M
Project 3: Combinatorial and gene-editing approaches to enhance the efficacy of CAR T cell therapy of multiple myeloma.P01CA214278 · NCI · UNIVERSITY OF PENNSYLVANIA · PI Joseph Anthony Fraietta · 2017 to 2026
$26.8M
NCI NIH HHS P01 CA214278NCI NIH HHS P30 CA014089
6 · The paper itself

Abstract

backgroundThe ELIANA trial showed that 61 (81%) of 75 paediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukaemia achieved overall remission after treatment with tisagenlecleucel, a chimeric antigen receptor targeted against the CD19 antigen. We aimed to evaluate patient-reported quality of life in these patients before and after tisagenlecleucel infusion.

methodsELIANA, a global, single-arm, open-label, phase 2 trial, was done in 25 hospitals across Australia, Austria, Belgium, Canada, France, Germany, Italy, Japan, Norway, Spain, and the USA. Patients with B-cell acute lymphoblastic leukaemia aged at least 3 years at the time of screening and 21 years or younger at the time of initial diagnosis who were in second or greater bone marrow relapse, chemorefractory, relapsed after allogeneic stem-cell transplantation, or were otherwise ineligible for allogeneic stem-cell transplantation were enrolled. Patients received a single intravenous administration of a target dose of 0·2-5 × 10

findingsBetween April 8, 2015, and April 25, 2017, 107 patients were screened, 92 were enrolled, and 75 received tisagenlecleucel. 58 patients aged 8-23 years were included in the analysis of quality of life. At baseline, 50 (86%) patients had completed the PedsQL questionnaire and 48 (83%) had completed the EQ-5D VAS. Improvements in patient-reported quality-of-life scores were observed for all measures at month 3 after tisagenlecleucel infusion (mean change from baseline to month 3 was 13·3 [95% CI 8·9-17·6] for the PedsQL total score and 16·8 [9·4-24·3] for the EQ-5D visual analogue scale). 30 (81%) of 37 patients achieved the minimal clinically important difference at month 3 for the PedsQL total score and 24 (67%) of 36 patients achieved this for the EQ-5D visual analogue scale.

interpretationThese findings, along with the activity and safety results of ELIANA, suggest a favourable benefit-risk profile of tisagenlecleucel in the treatment of paediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukaemia.

fundingNovartis.

Indexed as

Drug Resistance, NeoplasmPatient Reported Outcome MeasuresQuality of LifeSalvage TherapyAdolescentAdultCell- and Tissue-Based TherapyChildFemaleFollow-Up StudiesHumansImmunotherapyInfusions, IntravenousMaleNeoplasm Recurrence, LocalPrecursor B-Cell Lymphoblastic Leukemia-LymphomaReceptors, Antigen, T-Celltisagenlecleucel

Identifiers

PMID31606419
PMCPMC7480957
OpenAlexW2979548369

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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.