The New Molecules Are Changing the Course of Pediatric Chronically Active Ulcerative Colitis: A Series of Pediatric
Rafael Martín-Masot1, Pilar Ortiz Pérez1, Encarnación Torcuato Rubio1
1From the Pediatric Gastroenterology and Nutrition Unit, Hospital Regional Universitario de Málaga, Málaga, Spain.
Insights
Two pediatric ulcerative colitis (UC) cases unresponsive to standard treatments achieved remission with ustekinumab and tofacitinib. Patient and family involvement was crucial for successful multidisciplinary management, avoiding colectomy.
Area of Science:
- Pediatric Gastroenterology
- Inflammatory Bowel Disease Research
- Novel Therapeutics in UC
Background:
- Chronically active ulcerative colitis (UC) presents management challenges, especially with evolving treatment landscapes.
- Decreased colectomy experience contrasts with increased medical therapeutic options for UC.
- Patients with chronically active UC often require extended management to identify effective treatments.
Purpose of the Study:
- To report successful treatment of refractory pediatric ulcerative colitis (UC) using advanced biologics.
- To highlight the efficacy of ustekinumab and tofacitinib in pediatric UC cases.
- To emphasize the role of patient-family collaboration in treatment decision-making for pediatric UC.
Main Methods:
- Case report of two pediatric patients with chronically active UC.
- Documented lack of response to intravenous steroids and sequential therapies.
- Administration of ustekinumab and tofacitinib as rescue therapies.
Main Results:
- Both pediatric patients achieved clinical remission following treatment with ustekinumab and tofacitinib.
- These advanced therapies, though more established in adults, showed efficacy in pediatric UC.
- Multidisciplinary management and close follow-up were integral to treatment success.
Conclusions:
- Ustekinumab and tofacitinib represent viable therapeutic options for refractory pediatric ulcerative colitis (UC).
- Active patient and family participation significantly aids medical decision-making and treatment adherence.
- Successful medical management with these agents can potentially avert the need for colectomy in pediatric UC.
Abstract:
Chronically active ulcerative colitis (UC) constitutes a challenge in an era where medical therapeutic options have increased while experience with colectomies has decreased. The change in the therapeutic paradigm of the disease means that patients with chronically active UC are being managed waiting to find their therapeutic target. We present 2 cases of children with chronically active UC who did not respond to intravenous steroids nor sequential therapy. A response was obtained with ustekinumab and tofacitinib, 2 drugs widely used in adults but still with little evidence in children. Highlighting the important role of patients and their families helped decision-making, facilitating the work of the medical team. With multidisciplinary management and close follow-up, they have been able to avoid surgery entering complete clinical remission.
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