Favorable overall survival with imetelstat in relapsed/refractory myelofibrosis patients compared with real-world data.
Aged
Female
Humans
Janus Kinases
/ antagonists & inhibitors
Male
Middle Aged
Nitriles
/ adverse effects
Oligonucleotides
/ adverse effects
Primary Myelofibrosis
/ drug therapy
Propensity Score
Protein Kinase Inhibitors
/ adverse effects
Pyrazoles
/ adverse effects
Pyrimidines
/ adverse effects
Secondary Prevention
Survival Analysis
Imetelstat
Janus kinase inhibitor
Myelofibrosis
Real-world data
Ruxolitinib
Survival
Journal
Annals of hematology
ISSN: 1432-0584
Titre abrégé: Ann Hematol
Pays: Germany
ID NLM: 9107334
Informations de publication
Date de publication:
Jan 2022
Jan 2022
Historique:
received:
15
09
2021
accepted:
26
09
2021
pubmed:
9
10
2021
medline:
14
1
2022
entrez:
8
10
2021
Statut:
ppublish
Résumé
In the MYF2001 trial, treatment of Janus kinase (JAK) inhibitor-relapsed/refractory intermediate-2 or high-risk myelofibrosis (MF) with imetelstat 9.4 mg/kg every 3 weeks demonstrated encouraging median overall survival of 29.9 months. To provide historical context, external real-world data (RWD) were collected from a study of 96 patients who had discontinued ruxolitinib and were subsequently treated with best available therapy (BAT) at Moffitt Cancer Center. A closely matched cohort was identified using the MYF2001 eligibility criteria, including patients with MF who had discontinued ruxolitinib due to lack or loss of response. Overall survival was measured from time of JAK inhibitor discontinuation to death or censored at last follow-up. To improve comparability, propensity score weighting approaches using average treatment effect for overlap population (ATO) and stabilized inverse probability treatment weighting (sIPTW) were used for 10 critical baseline covariates. Fifty-seven patients treated with imetelstat 9.4 mg/kg from MYF2001 and 38 patients treated with BAT from RWD were analyzed with improved balanced baseline covariates after propensity score adjustment, showing significantly lower risk of death with imetelstat compared with BAT (hazard ratio: 0.35; p = 0.0019). With sIPTW, results were similar. Results of sensitivity analyses were consistent with the primary analysis. In conclusion, treatment with imetelstat was associated with longer overall survival compared to BAT (30 vs 12 months, respectively) in closely matched patients with MF after JAK inhibitor failure, warranting further evaluation of imetelstat in this poor-prognosis patient population.
Identifiants
pubmed: 34622316
doi: 10.1007/s00277-021-04683-w
pii: 10.1007/s00277-021-04683-w
doi:
Substances chimiques
Nitriles
0
Oligonucleotides
0
Protein Kinase Inhibitors
0
Pyrazoles
0
Pyrimidines
0
ruxolitinib
82S8X8XX8H
Janus Kinases
EC 2.7.10.2
imetelstat
F60NE4XB53
Types de publication
Clinical Trial
Comparative Study
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
139-146Informations de copyright
© 2021. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.
Références
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