Tisagenlecleucel vs. historical standard of care in children and young adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia.
Journal
Leukemia
ISSN: 1476-5551
Titre abrégé: Leukemia
Pays: England
ID NLM: 8704895
Informations de publication
Date de publication:
Dec 2023
Dec 2023
Historique:
received:
10
05
2023
accepted:
19
09
2023
revised:
10
08
2023
medline:
29
11
2023
pubmed:
26
10
2023
entrez:
25
10
2023
Statut:
ppublish
Résumé
In the absence of randomized controlled trials comparing tisagenlecleucel vs. standard of care (SOC) in pediatric and young adult patients with relapsed or refractory acute lymphoblastic leukemia (r/r ALL), the objective was to compare the efficacy of tisagenlecleucel with historical controls from multiple disease registries using patient-level adjustment of the historical controls. The analysis is based on patient-level data of three tisagenlecleucel studies (ELIANA, ENSIGN and CCTL019B2001X) vs. three registries in Germany/Austria. Statistical analyses were fully pre-specified and propensity score weighting of the historical controls by fine stratification weights was used to adjust for relevant confounders identified by systematic literature review. Results showed high comparability of cohorts after adjustment with absolute SMD ≤ 0.1 for all pre-specified confounders and favorable outcomes for tisagenlecleucel compared to SOC for all examined endpoints. Hazard ratios for OS
Identifiants
pubmed: 37880478
doi: 10.1038/s41375-023-02042-4
pii: 10.1038/s41375-023-02042-4
pmc: PMC10681894
doi:
Substances chimiques
tisagenlecleucel
Q6C9WHR03O
Receptors, Antigen, T-Cell
0
Types de publication
Systematic Review
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2346-2355Subventions
Organisme : Deutsche Kinderkrebsstiftung (German Childhood Cancer Foundation)
ID : DKS 2016.08, 2018.13, 2021.06
Informations de copyright
© 2023. The Author(s).
Références
Blood Adv. 2022 Apr 26;6(8):2536-2547
pubmed: 35030634
Clin Pharmacol Ther. 2022 Jan;111(1):90-97
pubmed: 34689339
Crit Rev Oncol Hematol. 2004 Jun;50(3):223-61
pubmed: 15182827
Value Health. 2012 Sep-Oct;15(6):940-7
pubmed: 22999145
J Clin Oncol. 2021 Feb 1;39(4):295-307
pubmed: 33332189
Med Decis Making. 2018 Feb;38(2):200-211
pubmed: 28823204
Internist (Berl). 2015 Apr;56(4):344-53
pubmed: 25776794
Blood. 2012 Oct 4;120(14):2807-16
pubmed: 22896001
Leukemia. 2020 Sep;34(9):2473-2478
pubmed: 32094465
BMJ. 2019 Oct 23;367:l5657
pubmed: 31645336
J Clin Oncol. 2015 Apr 10;33(11):1265-74
pubmed: 25753432
Curr Oncol Rep. 2016 Jun;18(6):39
pubmed: 27207612
J Comp Eff Res. 2020 Aug;9(12):849-860
pubmed: 32602756
Adv Ther. 2019 Apr;36(4):950-961
pubmed: 30758745
J Clin Epidemiol. 2013 Aug;66(8 Suppl):S84-S90.e1
pubmed: 23849158
Blood Adv. 2021 Dec 14;5(23):5387-5395
pubmed: 34597381