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Long-term outcomes of thalidomide in pediatric Crohn's disease
Lin Wang1, Aijuan Xue1, Cuifang Zheng1
1Pediatric Inflammatory Bowel Disease Research Center, Department of Gastroenterology, Children's Hospital of Fudan University, Shanghai, China.
Insights
Thalidomide shows efficacy in pediatric Crohn's disease (CD), achieving remission in over half of patients. It offers an alternative therapy for pediatric CD, especially for those with monogenic mutations.
Area of Science:
- Pediatric Gastroenterology
- Immunomodulatory Therapies
- Inflammatory Bowel Disease
Background:
- Crohn's disease (CD) in children requires effective long-term treatment options.
- Thalidomide's role in pediatric CD, particularly in Asian populations, needs further elucidation.
- Understanding treatment outcomes in relation to genetic mutations is crucial for personalized therapy.
Purpose of the Study:
- To evaluate the long-term efficacy and safety of thalidomide in a large pediatric Crohn's disease cohort.
- To compare treatment outcomes between pediatric CD patients with and without monogenic mutations.
- To assess thalidomide's role as an alternative therapy in pediatric CD.
Main Methods:
- Retrospective analysis of 62 pediatric CD patients treated with thalidomide.
- Extraction of clinical characteristics and treatment outcomes.
- Comparison of remission rates and safety profiles, differentiating between monogenic and non-monogenic groups.
Main Results:
- Clinical remission achieved in 33.9% at end of follow-up; rates differed significantly between non-monogenic (44.2%) and monogenic (10.5%) groups.
- C-reactive protein normalized in 66.7% at 12 months; 95.4% discontinued steroids.
- 45.2% experienced adverse events, leading to discontinuation in 22 patients; cumulative dose linked to adverse events.
Conclusions:
- Thalidomide demonstrates clinical efficacy and a manageable safety profile in pediatric Crohn's disease.
- It represents a viable alternative therapeutic option for pediatric CD patients.
- Thalidomide may be particularly beneficial for pediatric CD patients with monogenic mutations.
Background And Aim:
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:
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:
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:
Thalidomide was clinically efficacious and safe among pediatric CD. Our results suggest that it is an alternative therapy in monogenic mutation patients.
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