A prospective, multicenter registry for patients with short bowel syndrome (SBS Registry): Methodology, baseline characteristics, and incidence of colorectal cancer in the context of teduglutide treatment

Authors: JEPPESEN, PALLE - Rigshospitalet - Copenhagen University Hospital; ALLARD, JOHANE - University Of Toronto; GONDOLESI, GABRIEL - Favaloro Foundation University Hospital; JOLY, FRANCISCA - Beaujon Hospital; KEMENYASH, NATALIYA - Takeda Pharmaceutical; MASON, JOEL - Jean Mayer Human Nutrition Research Center On Aging At Tufts University; PAPE, ULRICH-FRANK - Charite' University Hospital Berlin; PIRONI, LORIS - University Of Bologna; PLEWA, ADAM - Takeda Pharmaceutical; ZHANG, PINGGAO - Takeda Pharmaceutical; SCHWARTZ, LAUREN - Nyu Langone Health

Submitted to: Journal of Parenteral and Enteral Nutrition
Publication Type: Abstract Only
Publication Acceptance Date: 2/21/2020
Publication Date: 2/21/2020
Citation: Jeppesen, P., Allard, J.P., Gondolesi, G., Joly, F., Kemenyash, N., Mason, J.B., Pape, U., Pironi, L., Plewa, A., Zhang, P., Schwartz, L.K. 2020. A prospective, multicenter registry for patients with short bowel syndrome (SBS Registry): Methodology, baseline characteristics, and incidence of colorectal cancer in the context of teduglutide treatment [abstract]. Journal of Parenteral and Enteral Nutrition. 44(2): 209-211. https://doi.org/10.1002/jpen.1813.
DOI: https://doi.org/10.1002/jpen.1813

Interpretive Summary:

Technical Abstract: Purpose: Patients with short bowel syndrome (SBS) are heterogeneous in their clinical presentation, disease progression, and treatment needs. They may require long-term parenteral support (PS). Teduglutide (TED) has been approved in the United States, Canada, and Europe for the treatment of PS-dependent patients >=1 year of age with SBS. This ongoing prospective, observational, multinational registry evaluates long-term safety and clinical outcomes of patients with SBS in a routine clinical setting, and includes both TED-treated and untreated patients. Here, we report on baseline characteristics and occurrences of malignancies in patients included in the registry. Methods: The registry (NCT01990040; EUPAS7973) includes patients of any age with SBS. Patients who have never received TED treatment ("never treated" patients) must have been on PS for >=6 months at the time of enrollment; patients who have never been on PS are excluded. The primary outcome is the occurrence of colorectal cancer (CRC) in the remnant colon in patients who have ever been treated with teduglutide ("ever-treated" patients) compared with never-treated patients. Study start date was June 23, 2014; data cut-off for this analysis was June 30, 2018. Results are reported for adult patients with remnant colon (primary outcome analysis set) as mean +/- SD unless otherwise indicated. Results: This present analysis included 170 ever-treated patients and 212 never-treated patients with remnant colon, enrolled from 7 countries. Demographics and baseline characteristics are reported in Table 1. Length of remaining small intestine was 69.2+/-47.90 cm for ever-treated patients (n=147) and 86.2+/-57.44 cm for never-treated patients (n=174). Among ever-treated and never-treated patients, 32.4% and 29.2% had intact colon, and 67.6% and 70.8% had colon remnant, respectively. Percent of remaining colon was 53.3%+/-25.96% in ever-treated patients (n=53) and 51.8%+/-24.72% in never-treated patients (n=59). Cause of major intestinal resection was intestinal ischemia, Crohn's disease, or "other" in 27.8%, 26.0%, and 46.2% of ever-treated patients (n=169) and 23.2%, 19.0%, and 57.8% of never-treated patients (n=211), respectively. The percentage of patients receiving PS at baseline was 77.4% for ever-treated (n=164) and 100% for never-treated patients (n=204); PS volume at baseline was 9.1+/-6.75 L/week (n=115) and 11.2+/-7.31 L/week (n=203), respectively. As of data cut-off, colonoscopies had been performed and captured in the registry for 32 ever-treated (49 events) and 10 never-treated patients (10 events). No cases of CRC were reported over a follow-up time of 1.5+/-0.86 years (range, 0.1-4.0) for ever-treated patients and 1.3+/-0.83 years (range, 0.0-4.0) for never-treated patients; TED exposure was 21.5+/-17.30 months (range, 0.2-96.7) in the ever-treated group. During follow-up, new or worsening diagnosis of any type of cancer was reported for 6 ever-treated and 7 never-treated patients (detailed information provided in Table 2). New or worsening colorectal polyps occurred in 2 ever-treated patients; 1 patient developed 1 polyp in the ascending colon, and 1 patient developed 2 polyps in the sigmoid colon. No new/worsening polyps occurred in the never-treated patients. Limitations include the short duration of follow-up time and low number of colonoscopies performed. Conclusion: Observed differences in some baseline characteristics between ever-treated and never-treated groups likely reflect the heterogeneity of the SBS patient population and selection biases related to the indication for TED treatment. No cases of CRC were reported in registry patients treated with TED from June 23, 2014 until June 30, 2018. This ongoing registry will continue to yield data on the incidence of CRC in patients with SBS.