Clinical trial NCT04976075: ImpRovE underSTanding of Short bOwel syndRome in Latin-amErica
Official title: Multicenter Prospective Observational and Epidemiological Registry for Patients With Intestinal Failure Due to Short Bowel Syndrome, in Latin America
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Short Bowel Syndrome
Lead sponsor: La Fundacion Favaloro para la Investigacion y la Docencia Medica
Sponsor class: OTHER
Collaborators: name: Fundación de Ciencias Exactas - UNLP
First posted: 2021-07-26
Enrollment: 150
Sex: ALL
Minimum age (years): 1
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Based on the SIC prevalence calculations, we aim to include 150 patients. The inclusion of patients will be done at the national level, considering as reference centers those hospitals that have Nutritional Support and Intestinal Rehabilitation Units, both public and private. Researchers may consecutively include all patients who give their consent and meet the selection criteria.

Brief summary: Background and justification: Short bowel syndrome (SBS) is a disabling and life-threatening condition that results from a partial or total bowel resection, and has become as the main cause of Type III, intestinal failure (IF). Immediately after enterectomy there is an adaptation process consisting of structural, hormonal and metabolic changes to maximize intestinal function. These changes begin within days of resection and generally continue for several months. However, recent publications have reported even longer periods of time (up to 5 years) to achieve enteral autonomy.

It is possible to enhance the natural process of intestinal adaptation through medical or surgical treatments, called intestinal rehabilitation. During the process, complications related to intestinal failure or its treatment may arise, jeopardizing the result and even compromising survival. A better understanding of the medium and long-term results of patients under medical and / or surgical treatment with SIC is needed.

Despite the improvement recently achieved in managing IF, in most countries, pts are dispersed and seen by general health-care providers, with limited SBS or IF experience, causing increasing concern regarding the competence and equity of the care accessible to suffering pts.

The results obtained with the RESTORE project in adult patients highlight the relevance of having registries to better understand the natural history of this disease in adult patients, proving that a larger number of adult pts with SBS/III-IF can be identified and cared for than the numbers considered by recognized estimations. \[Abstract sent to TTS, 2020\].

To date, there are no data for pediatric patients with iIF secondary to SIC in Latin America, so its incidence, prevalence and evolution are unknown. Recent publications from middle-income countries, exposed the current inequality regarding the different types of therapies available within a given region.

Given the high morbidity and mortality associated with ICS-FI, there is an unmet need to create an adequate study that provides the information necessary to establish local and regional parameters and recommendations on its treatment.

Detailed description: RESTORE amedment 1project, obtained the study number DDI (1384) 1317 from the Institutional Research and Ethics committee after its approving the choices of participating centers by RESTORE's Scientific Steering Committee. For participation, those centers needed a full-time or part-time surgeon-though not necessarily offing rehabilitation surgery or transplantation or accessing enterohormone (EH) therapies-a therapist with nutritional experience, a dietitian, and specialized pharmaceutical resources. Participating teams, however, did not necessarily have to offer rehabilitation surgery or transplantation capabilities or have access to EH therapies to be elected for participation.

RESTORE's aim was assessing the long-term outcomes of SBS pts with IF, under the current standard of care in order to plumb IF's natural history, the indications for currently available therapies, and their means of modifying each course.

Study Designed: the first prospective, observational, epidemiological, multicenter study in Argentina of adult pts with SBS-caused type-III IF in Lat-America.

In adults, the inclusion of additional centers in Argentina, Mexico, Colombia, Peru, Uruguay and Chile will be required. In pediatric patients, it is proposed to add patients and centers in Argentina and, if necessary, in other Latin American countries (Mexico, Colombia, Peru, Chile and Uruguay).

Number of patients and centers:

The study aims to recruit at least 150 patients in 24 centers. The sample size is based on the expected number of subjects who may be eligible and willing to participate in the study.

Diagnosis and inclusion criteria

Inclusion criteria:

1. Patients of both sexes, diagnosed with Type III-IF due to SBS
2. Signature of informed consent and assent as appropriate
3. 8 or more consecutive weeks of PN need

Exclusion criteria:

1. Less than 8 weeks of need for PN after diagnosis of SBS / IF
2. Refusal to provide informed consent and assent as appropriate
3. FI type I and II, and type III without SBS

The data from this study will be entered into an electronic CRF and database; with visits being reinitiated at weeks 4, 8, 12, 20, and 24 and then yearly thereafter. Pts that missed 2 or more visits were considered lost from follow-up. Death, recovery of intestinal sufficiency, and intestinal transplant were considered the end points.

The results of this study will be summarized and evaluated using descriptive statistics.

Eligibility criteria: Inclusion Criteria:

1. Patients of both sexes, diagnosed with Type III intestinal failure (FI) due to SIC
2. Signature of informed consent and assent as appropriate
3. 8 or more consecutive weeks of PN need

Exclusion Criteria:

1. Less than 8 weeks of need for PN after diagnosis of SIC / FI
2. Refusal to provide informed consent and assent as appropriate
3. FI type I and II, and type III without SIC

Primary outcomes:
measure: Describe the long term outcome of Type III-IF patients with short bowel syndrome in Latinoamérica.
timeFrame: April 2023
Secondary outcomes:
measure: Describe the demographic characteristics and distribution of the disease
timeFrame: April 2023
measure: Describe the nutritional progress
timeFrame: April 2023
measure: Record the use of Enterohormones
timeFrame: April 2023
measure: Describe the use, management of HPN, and its related complications
timeFrame: April 2023
measure: Describe the surgical procedures, used, timing, outcomes and complications
timeFrame: April 2023
measure: Assess the quality of life
timeFrame: April 2023
measure: Time to obtain therapy access
timeFrame: April 2023
Other outcomes:
Locations:
city: Buenos Aires
country: Argentina
facility: University Hospital of the Favaloro Foundation
Study officials:
affiliation: University Hospital of the Favaloro Foundation
name: Gabriel Gondolesi, Doctor
role: PRINCIPAL_INVESTIGATOR
References: