Nilotinib efficacy and safety as salvage treatment following imatinib intolerance and/or inefficacy in steroid refractory chronic graft-versus-host-disease (SR-cGVHD): a prospective, multicenter, phase II study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC).
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
04 2023
04 2023
Historique:
received:
18
05
2022
accepted:
07
12
2022
revised:
23
11
2022
medline:
6
4
2023
pubmed:
10
1
2023
entrez:
9
1
2023
Statut:
ppublish
Résumé
Imatinib is used for patients with SR-cGVHD. However, in 50% of cases imatinib is discontinued due to intolerance or inefficacy. In order to investigate nilotinib's role as salvage therapy in those patients, we conducted a prospective, multicenter, phase II study. (NCT02891395). Patients with SR-cGVHD were included to receive imatinib. Patients who stopped imatinib due to intolerance or inefficacy switched to Nilotinib. The primary endpoint was defined as the week-12 response rate to Nilotinib. The response was considered successful if superior to the 30% endpoint. Sixty-two patients started the IM-phase. Fourteen patients (22%) discontinued imatinib before week 12 due to: cGVHD progression (10%) or TKI-class-specific intolerance (12%). At week 12, we observed complete remission in 13 patients (21%) and partial response in 8 patients (13%). Twenty-nine patients switched to Nilotinib. Nilotinib response at week-12 was observed in 6 patients (21%) while 23 patients (79%) discontinued Nilotinib due to intolerance/cGVHD progression. The primary endpoint was not reached. This prospective study confirmed the efficacy of imatinib in patients with steroid refractory cGVHD. It failed to demonstrate the efficacy of nilotinib as a salvage therapy in patients who were intolerant/unresponsive to imatinib.
Identifiants
pubmed: 36624161
doi: 10.1038/s41409-022-01898-x
pii: 10.1038/s41409-022-01898-x
doi:
Substances chimiques
Imatinib Mesylate
8A1O1M485B
Pyrimidines
0
Steroids
0
Banques de données
ClinicalTrials.gov
['NCT02891395']
Types de publication
Clinical Trial, Phase II
Multicenter Study
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
401-406Informations de copyright
© 2023. The Author(s), under exclusive licence to Springer Nature Limited.
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