RARE DISEASE
Pouchitis
Pouchitis
Pouchitis
Drug discovery
15
drugs
With orphan designations
Overview
Pouchitis is an inflammatory complication affecting the ileal pouch after restorative proctocolectomy with ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC). Characterized by increased stool frequency, urgency, abdominal pain, and bleeding, it is diagnosed through clinical, endoscopic, and histologic evaluation [1][16]. Most cases respond to antibiotics, but chronic forms require advanced immunosuppressive therapies [1][3][19].
Categories: rare gastroenterological diseases
Research Papers
1,526 drug discovery papers about Pouchitis, with 3 first-in-class and 8 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,526 drug discovery papers about Pouchitis, with 3 first-in-class and 8 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-14 | Continued concomitant antibiotic or immunosuppression therapies reduce the risk of pouchitis among patients with ileal pouch-anal anastomosis and primary sclerosing cholangitis.
Complications such as intermittent pouchitis (IP) and chronic inflammatory disorders of the pouch (CP) are common after ileal pouch-anal anastomosis (IPAA), particularly among patients with both ulcerative colitis and primary sclerosing cholangitis (PSC). There are no standardized approaches for prophylaxis against these complications for patients with PSC undergoing IPAA. We compared outcomes among patients with PSC and IPAA who received antibiotic or immunosuppressive therapies to those who did not. We designed a retrospective cohort study that included patients with ulcerative colitis and PSC from 7 tertiary care centers that underwent IPAA between 1994 and 2023. Patients were categorized on whether they received medications following IPAA that may provide primary prophylactic benefit, such as antibiotics, probiotics, immunomodulators, or biologic medications; and the proportion of patients developing IP and CP was evaluated. A total of 172 patients were included in the cohort, and 13 (7.6%) patients received antibiotics or immunosuppressant medications. Antibiotics and probiotics were the most common therapy (5 patients, 2.9%), followed by biologics, small-molecule inhibitors, immunomodulators, or mesalamine (all 1 patient, 0.6%). Compared to patients not receiving antibiotics or immunosuppression, patients receiving antibiotics or immunosuppression were significantly less likely to develop IP (69.2% vs 91.2%; P = .03; odds ratio 0.22; 95% CI, 0.06-0.80) and CP (23.1% vs 56.0%, P = 0.04; OR 0.24, 95% CI, 0.06-0.89). In this retrospective cohort of patients with ulcerative colitis and PSC undergoing IPAA, usage of concomitant antibiotics or immunosuppression was low. However, use of concomitant antibiotics or immunosuppression may decrease the burden of inflammatory conditions of the pouch in these high-risk patients.
2026-07-18 | Study of TOfacitinib for the treatment of chronic PouchITis (STOPit): a multi-centre open label induction with randomised, double blind, placebo-controlled maintenance trial.
Study of TOfacitinib for the treatment of chronic PouchITis (STOPit) is a national, investigator-led, multicentre, open-label induction with randomised, double-blind, placebo-controlled maintenance trial to determine the effectiveness of tofacitinib to induce and maintain clinical remission in chronic pouchitis patients. The primary objective of STOPit is to evaluate the clinical response to tofacitinib, compared with placebo, using the validated modified Pouchitis Disease Activity Index score. Secondary objectives include clinical response and remission rates, endoscopic response and mucosal healing rates in induction and maintenance phases; rates of flare and recapturing response in open label extension phase; the effect of tofacitinib on clinical biomarkers and quality of life; and safety of tofacitinib use for treatment of chronic pouchitis. Study period is up to ten months, with five visits and three pouchoscopies. Adults with ulcerative colitis who have undergone total proctocolectomy and ileal pouch-anal anastomosis and have chronic pouchitis are eligible. Participants receive open-label 10 mg two times per day tofacitinib for 8 weeks. Clinical responders at the end of week 8 will be randomised to either tofacitinib or placebo in the maintenance phase. Non-responders will receive extended induction dosing for a further 8 weeks. Persistent non-responders exit the study and responders will proceed to maintenance randomisation. Randomised individuals will be followed for 24 weeks. Patients experiencing a flare of symptoms during the maintenance phase will be eligible to receive open-label tofacitinib. Efficacy and safety data will be evaluated at 12 weeks after termination from the study. Target sample size is 72 patients to be randomised into maintenance to achieve sufficient power. Data analysis is planned to commence in October 2026. Ethics approval was granted (Project ID 66809) and the trial was notified (CTN: CT-2020-CTN-04866-1; ANZCTR: 381128) prior to commencement. The study will be conducted in accordance with National Health and Medical Research Council (NHMRC) guidelines, and findings will be published in a peer-reviewed journal. CTN: CT-2020-CTN-04866-1.
2026-07-10 | Impact of Diet and Body Mass Index on the Development of Pouchitis in the First Year After Ileal Pouch-Anal Anastomosis.
Although pouchitis is the most common complication after ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC), the influence of diet on pouchitis is poorly understood. Thus, evidence-based dietary recommendations after IPAA are limited. We evaluated dietary intake in the period after the final stage of IPAA for UC and Body Mass Index (BMI) to identify potential associations with pouchitis. We used the Dietary Screener Questionnaire (DSQ) to assess dietary intake among patients in a prospective cohort enrolled within 2 weeks of the final stage of IPAA for UC. We compared food group and fiber intake of those who developed pouchitis within 12 months of the final stage of IPAA with those who did not. These were also compared to the United States Department of Agriculture (USDA) recommended daily goals. Among 89 patients, there was no significant difference in individual fiber intake when comparing those who developed pouchitis and those who did not. In comparison with USDA guidelines, both groups (pouchitis and no pouchitis) exhibited decreased intake of several food groups, such as fruits, vegetables, fiber, and dairy. Patients who developed pouchitis had a significantly higher median body mass index (BMI) at the time of surgery compared to patients who did not develop pouchitis (median 29.2 vs. 24.8, P = 0.022). In a prospective cohort, we identified no association between dietary intake and development of pouchitis within the first year after IPAA. However, elevated BMI may represent a unique target for intervention to prevent pouchitis occurrence.
2026-07-01 | The efficacy and safety of hyoscyamine, dicyclomine, and desipramine in the treatment of irritable pouch syndrome-a retrospective cohort study.
Patients with ileal pouch-anal anastomosis (IPAA) frequently experience high bowel frequency and abdominal pain that are disproportionate to endoscopic inflammation, a condition often termed irritable pouch syndrome. Although antispasmodic agents and neuromodulators are widely used in irritable bowel syndrome, real-world data supporting their effectiveness and durability in patients with pouches remain limited. We performed a retrospective cohort study of IPAA patients followed at a pouch clinic between 2014 and 2025. Patients treated with hyoscyamine extended release (ER), dicyclomine, or desipramine for high bowel frequency or abdominal pain were included. Patients could receive multiple therapies sequentially over time. Clinical response was determined by physician-documented symptom improvement. Treatment persistence was assessed using electronic medical record prescription data. Among 219 eligible patients, 329 treatment courses were analyzed: 143 hyoscyamine ER, 80 dicyclomine, and 106 desipramine. In patients with high bowel frequency, 235 treatment courses resulted in clinical improvement in 68.5% of patients receiving hyoscyamine ER, 65.0% of those receiving dicyclomine, and 63.5% of those receiving desipramine. For abdominal pain (94 treatment courses), improvement for hyoscyamine ER, dicyclomine, and desipramine was observed in 61.5%, 68.3%, and 76.2% of courses, respectively. Approximately 60% of treatment courses persisted beyond one year. Long-term persistence (≥3 years) was highest with dicyclomine (48.7%), followed by desipramine (38.7%) and hyoscyamine ER (37.8%). Adverse events were infrequent and consistent with known safety profiles. Antispasmodic agents and low-dose neuromodulators are associated with meaningful symptom improvement in patients with irritable pouch syndrome, supporting their role as adjunctive therapies for pouch-related functional symptoms.
2026-07-01 | Patients with Ileal Pouch-Anal Anastomosis Have Decreased Bowel Frequency on Glucagon-like Peptide-1 Receptor Agonist Therapy.
High bowel frequency after ileal pouch-anal anastomosis (IPAA) causes significant symptom burden. We aimed to evaluate the impact of GLP-1 receptor agonists (GLP1RAs) in this setting. We conducted a retrospective cohort study of patients with prior IPAA for ulcerative colitis who were treated with a GLP1RA (n=20). Daily bowel frequency at baseline and 12 weeks after GLP1RA initiation was assessed. We also assessed the proportion of patients achieving ≥30% reduction in bowel frequency and ≤8 bowel movements per day. Median daily bowel frequency decreased from 9.0 (IQR, 6.0-12.5) at baseline to 6.0 (IQR, 5.0-8.1) at the 12-week follow-up (p<0.01). Overall, 7 of 20 patients achieved a ≥30% reduction in bowel frequency, and 15 of 20 had ≤8 bowel movements per day. In this retrospective cohort, we provide further evidence for the potential role of GLP1RAs in the management of high bowel frequency after IPAA.
2026-08-14 | Continued concomitant antibiotic or immunosuppression therapies reduce the risk of pouchitis among patients with ileal pouch-anal anastomosis and primary sclerosing cholangitis.
Complications such as intermittent pouchitis (IP) and chronic inflammatory disorders of the pouch (CP) are common after ileal pouch-anal anastomosis (IPAA), particularly among patients with both ulcerative colitis and primary sclerosing cholangitis (PSC). There are no standardized approaches for prophylaxis against these complications for patients with PSC undergoing IPAA. We compared outcomes among patients with PSC and IPAA who received antibiotic or immunosuppressive therapies to those who did not. We designed a retrospective cohort study that included patients with ulcerative colitis and PSC from 7 tertiary care centers that underwent IPAA between 1994 and 2023. Patients were categorized on whether they received medications following IPAA that may provide primary prophylactic benefit, such as antibiotics, probiotics, immunomodulators, or biologic medications; and the proportion of patients developing IP and CP was evaluated. A total of 172 patients were included in the cohort, and 13 (7.6%) patients received antibiotics or immunosuppressant medications. Antibiotics and probiotics were the most common therapy (5 patients, 2.9%), followed by biologics, small-molecule inhibitors, immunomodulators, or mesalamine (all 1 patient, 0.6%). Compared to patients not receiving antibiotics or immunosuppression, patients receiving antibiotics or immunosuppression were significantly less likely to develop IP (69.2% vs 91.2%; P = .03; odds ratio 0.22; 95% CI, 0.06-0.80) and CP (23.1% vs 56.0%, P = 0.04; OR 0.24, 95% CI, 0.06-0.89). In this retrospective cohort of patients with ulcerative colitis and PSC undergoing IPAA, usage of concomitant antibiotics or immunosuppression was low. However, use of concomitant antibiotics or immunosuppression may decrease the burden of inflammatory conditions of the pouch in these high-risk patients.
2026-07-18 | Study of TOfacitinib for the treatment of chronic PouchITis (STOPit): a multi-centre open label induction with randomised, double blind, placebo-controlled maintenance trial.
Study of TOfacitinib for the treatment of chronic PouchITis (STOPit) is a national, investigator-led, multicentre, open-label induction with randomised, double-blind, placebo-controlled maintenance trial to determine the effectiveness of tofacitinib to induce and maintain clinical remission in chronic pouchitis patients. The primary objective of STOPit is to evaluate the clinical response to tofacitinib, compared with placebo, using the validated modified Pouchitis Disease Activity Index score. Secondary objectives include clinical response and remission rates, endoscopic response and mucosal healing rates in induction and maintenance phases; rates of flare and recapturing response in open label extension phase; the effect of tofacitinib on clinical biomarkers and quality of life; and safety of tofacitinib use for treatment of chronic pouchitis. Study period is up to ten months, with five visits and three pouchoscopies. Adults with ulcerative colitis who have undergone total proctocolectomy and ileal pouch-anal anastomosis and have chronic pouchitis are eligible. Participants receive open-label 10 mg two times per day tofacitinib for 8 weeks. Clinical responders at the end of week 8 will be randomised to either tofacitinib or placebo in the maintenance phase. Non-responders will receive extended induction dosing for a further 8 weeks. Persistent non-responders exit the study and responders will proceed to maintenance randomisation. Randomised individuals will be followed for 24 weeks. Patients experiencing a flare of symptoms during the maintenance phase will be eligible to receive open-label tofacitinib. Efficacy and safety data will be evaluated at 12 weeks after termination from the study. Target sample size is 72 patients to be randomised into maintenance to achieve sufficient power. Data analysis is planned to commence in October 2026. Ethics approval was granted (Project ID 66809) and the trial was notified (CTN: CT-2020-CTN-04866-1; ANZCTR: 381128) prior to commencement. The study will be conducted in accordance with National Health and Medical Research Council (NHMRC) guidelines, and findings will be published in a peer-reviewed journal. CTN: CT-2020-CTN-04866-1.
2026-07-10 | Impact of Diet and Body Mass Index on the Development of Pouchitis in the First Year After Ileal Pouch-Anal Anastomosis.
Although pouchitis is the most common complication after ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC), the influence of diet on pouchitis is poorly understood. Thus, evidence-based dietary recommendations after IPAA are limited. We evaluated dietary intake in the period after the final stage of IPAA for UC and Body Mass Index (BMI) to identify potential associations with pouchitis. We used the Dietary Screener Questionnaire (DSQ) to assess dietary intake among patients in a prospective cohort enrolled within 2 weeks of the final stage of IPAA for UC. We compared food group and fiber intake of those who developed pouchitis within 12 months of the final stage of IPAA with those who did not. These were also compared to the United States Department of Agriculture (USDA) recommended daily goals. Among 89 patients, there was no significant difference in individual fiber intake when comparing those who developed pouchitis and those who did not. In comparison with USDA guidelines, both groups (pouchitis and no pouchitis) exhibited decreased intake of several food groups, such as fruits, vegetables, fiber, and dairy. Patients who developed pouchitis had a significantly higher median body mass index (BMI) at the time of surgery compared to patients who did not develop pouchitis (median 29.2 vs. 24.8, P = 0.022). In a prospective cohort, we identified no association between dietary intake and development of pouchitis within the first year after IPAA. However, elevated BMI may represent a unique target for intervention to prevent pouchitis occurrence.
2026-07-01 | The efficacy and safety of hyoscyamine, dicyclomine, and desipramine in the treatment of irritable pouch syndrome-a retrospective cohort study.
Patients with ileal pouch-anal anastomosis (IPAA) frequently experience high bowel frequency and abdominal pain that are disproportionate to endoscopic inflammation, a condition often termed irritable pouch syndrome. Although antispasmodic agents and neuromodulators are widely used in irritable bowel syndrome, real-world data supporting their effectiveness and durability in patients with pouches remain limited. We performed a retrospective cohort study of IPAA patients followed at a pouch clinic between 2014 and 2025. Patients treated with hyoscyamine extended release (ER), dicyclomine, or desipramine for high bowel frequency or abdominal pain were included. Patients could receive multiple therapies sequentially over time. Clinical response was determined by physician-documented symptom improvement. Treatment persistence was assessed using electronic medical record prescription data. Among 219 eligible patients, 329 treatment courses were analyzed: 143 hyoscyamine ER, 80 dicyclomine, and 106 desipramine. In patients with high bowel frequency, 235 treatment courses resulted in clinical improvement in 68.5% of patients receiving hyoscyamine ER, 65.0% of those receiving dicyclomine, and 63.5% of those receiving desipramine. For abdominal pain (94 treatment courses), improvement for hyoscyamine ER, dicyclomine, and desipramine was observed in 61.5%, 68.3%, and 76.2% of courses, respectively. Approximately 60% of treatment courses persisted beyond one year. Long-term persistence (≥3 years) was highest with dicyclomine (48.7%), followed by desipramine (38.7%) and hyoscyamine ER (37.8%). Adverse events were infrequent and consistent with known safety profiles. Antispasmodic agents and low-dose neuromodulators are associated with meaningful symptom improvement in patients with irritable pouch syndrome, supporting their role as adjunctive therapies for pouch-related functional symptoms.
2026-07-01 | Patients with Ileal Pouch-Anal Anastomosis Have Decreased Bowel Frequency on Glucagon-like Peptide-1 Receptor Agonist Therapy.
High bowel frequency after ileal pouch-anal anastomosis (IPAA) causes significant symptom burden. We aimed to evaluate the impact of GLP-1 receptor agonists (GLP1RAs) in this setting. We conducted a retrospective cohort study of patients with prior IPAA for ulcerative colitis who were treated with a GLP1RA (n=20). Daily bowel frequency at baseline and 12 weeks after GLP1RA initiation was assessed. We also assessed the proportion of patients achieving ≥30% reduction in bowel frequency and ≤8 bowel movements per day. Median daily bowel frequency decreased from 9.0 (IQR, 6.0-12.5) at baseline to 6.0 (IQR, 5.0-8.1) at the 12-week follow-up (p<0.01). Overall, 7 of 20 patients achieved a ≥30% reduction in bowel frequency, and 15 of 20 had ≤8 bowel movements per day. In this retrospective cohort, we provide further evidence for the potential role of GLP1RAs in the management of high bowel frequency after IPAA.
Access all drug discovery papers and probability of success in trials forecasts:
Access all drug discovery papers and probability of success in trials forecasts:
Drug Discovery Landscape
15 orphan drug designations for Pouchitis.
15 orphan drug designations for Pouchitis.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Lyophilized consortium of Faecalibacterium sp., strain PD09; Anaerostipes sp., strain PD10; Akkermansia sp., strain PD11; Butyricicoccus sp., strain PD12; Roseburia sp., strain PD14; and Lactiplantibacillus sp., strain PD15 | other | EMA | 2026-07-24 | — | Microbial Resource Management Health |
(-)-Epigallocatechin 3-gallate | small molecules | FDA | 2024-04-29 | — | PharmassetX LLC |
recombinant human interleukin-10 fusion protein | proteins | FDA | 2022-11-08 | — | Applied Molecular Transport, Inc. |
Rifamycin | small molecules | FDA | 2022-10-18 | — | Cosmo Technologies, Ltd. |
vedolizumab | antibodies | FDA | 2021-03-15 | — | Takeda Development Center Americas, Inc. |
EXE-346, a Live Biotherapeutic Product (LBP), contains eight strains of probiotic bacteria. | other | FDA | 2020-11-02 | — | ExeGi Pharma, LLC. |
Metronidazole | small molecules | EMA | 2011-06-21 | — | Avivia Projects BV |
metronidazole | small molecules | FDA | 2011-04-26 | — | S.L.A. Pharma Limited (UK) |
Alicaforsen | oligonucleotides | EMA | 2009-05-15 | — | Atlantic Healthcare Europe B.V. |
metronidazole | small molecules | FDA | 2008-09-17 | — | Avivia Project BV |
alicaforsen | oligonucleotides | FDA | 2008-06-24 | — | Bensen Therapeutics Limited |
spherical carbon adsorbent | other | FDA | 2007-12-19 | — | Ocera Therapeutics, Inc. |
Clotrimazole | small molecules | FDA | 2005-06-14 | — | AesRx, LLC |
lactic acid bacteria (Lactobacilli, Bifidobacteria, and Steptococci) | other | FDA | 2002-01-15 | — | VSL Pharmaceuticals, Inc. |
lactic acid bacteria (Lactobacilli, Bifidobacteria, and Streptococcus species) | other | FDA | 2002-01-15 | — | VSL Pharmaceuticals, Inc. |
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