Long-term outcomes of thalidomide in pediatric Crohn's disease.
Crohn's disease
Monogenic
Safety
Thalidomide
Treatment
Journal
Journal of gastroenterology and hepatology
ISSN: 1440-1746
Titre abrégé: J Gastroenterol Hepatol
Pays: Australia
ID NLM: 8607909
Informations de publication
Date de publication:
Jul 2020
Jul 2020
Historique:
received:
10
09
2019
revised:
29
10
2019
accepted:
17
11
2019
pubmed:
24
11
2019
medline:
11
11
2020
entrez:
24
11
2019
Statut:
ppublish
Résumé
In this largest pediatric cohort to date in Asian population, we aimed to report our long-term real-life experience with thalidomide treatment in pediatric Crohn's disease (CD). A retrospective single-center analysis of pediatric CD patients treated by thalidomide was conducted. The clinical characteristics and outcomes were extracted. Primary outcomes were clinical response and remission rate at different time points, especially comparing the difference between monogenic and non-monogenic mutation patients. We also evaluated the long-term safety of thalidomide. A total of 62 patients met the inclusion criteria. The median follow-up period was 30.5 months. Among all, 19 patients (30.6%) were diagnosed with monogenic mutation during treatment. Clinical remission rate was 53.2% (33/62) at 6 months, 54.8% (34/62) at 12 months, and 33.9% (21/62) at the end of follow-up. Clinical remission rates between non-monogenic and monogenic groups at the end were statistically different (44.2% [19/43] vs 10.5% [2/19], P < 0.05). At 12 months, 66.7% (30/45) were with normalized C-reactive protein level. Most patients (95.4%, 21/22) discontinued steroids with a median time of 4.4 months. Twelve patients relapsed, but no risk factor was identified to be significantly associated with relapse. A total of 45.2% (28/62) patients experienced an adverse event, in which 22 patients stopped thalidomide due to safety concern. Cumulative dose was not associated with abnormal electromyography but with the occurrence of adverse events. Thalidomide was clinically efficacious and safe among pediatric CD. Our results suggest that it is an alternative therapy in monogenic mutation patients.
Sections du résumé
BACKGROUND AND AIM
OBJECTIVE
In this largest pediatric cohort to date in Asian population, we aimed to report our long-term real-life experience with thalidomide treatment in pediatric Crohn's disease (CD).
METHODS
METHODS
A retrospective single-center analysis of pediatric CD patients treated by thalidomide was conducted. The clinical characteristics and outcomes were extracted. Primary outcomes were clinical response and remission rate at different time points, especially comparing the difference between monogenic and non-monogenic mutation patients. We also evaluated the long-term safety of thalidomide.
RESULTS
RESULTS
A total of 62 patients met the inclusion criteria. The median follow-up period was 30.5 months. Among all, 19 patients (30.6%) were diagnosed with monogenic mutation during treatment. Clinical remission rate was 53.2% (33/62) at 6 months, 54.8% (34/62) at 12 months, and 33.9% (21/62) at the end of follow-up. Clinical remission rates between non-monogenic and monogenic groups at the end were statistically different (44.2% [19/43] vs 10.5% [2/19], P < 0.05). At 12 months, 66.7% (30/45) were with normalized C-reactive protein level. Most patients (95.4%, 21/22) discontinued steroids with a median time of 4.4 months. Twelve patients relapsed, but no risk factor was identified to be significantly associated with relapse. A total of 45.2% (28/62) patients experienced an adverse event, in which 22 patients stopped thalidomide due to safety concern. Cumulative dose was not associated with abnormal electromyography but with the occurrence of adverse events.
CONCLUSIONS
CONCLUSIONS
Thalidomide was clinically efficacious and safe among pediatric CD. Our results suggest that it is an alternative therapy in monogenic mutation patients.
Substances chimiques
Thalidomide
4Z8R6ORS6L
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1124-1129Informations de copyright
© 2019 Journal of Gastroenterology and Hepatology Foundation and John Wiley & Sons Australia, Ltd.
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