Patient-reported Outcome Scale in Acute Pancreatitis

NCT ID: NCT03650062

Last Updated: 2019-02-05

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 525 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-05-30
• Study Completion Date: 2019-02-03

Brief Summary

The PAN-PROMISE study (PAtieNt-rePoRted OutcoMe scale in acute pancreatItis-an international proSpEctive cohort study) aims to measure an outcome variable in acute pancreatitis (AP) based in the patient´s experience. PAN-PROMISE is a cohort study involving patients with AP. The patient´s symptom perception will be compared with the opinion of the clinicians and with clinical outcomes.

Detailed Description

Currently, the most important outcome variables in Acute Pancreatitis (AP) are persistent organ failure (POF) and mortality, according to the physician point of view. These variables occur infrequently (<10 and <5% respectively) (Sternby et all, see "References"), therefore, studies designed to assess the effectiveness of new treatments for AP to decrease POF or mortality require a very large sample of patients, which is unfeasible. Currently, clinical trials addressing the management of AP do not take into account Patient Reported Outcome Measures.

PROMS (Patient Reported Outcome Measures) have been defined as any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else (first link in "References"). PROMS are gaining importance as a tool to design outcome variables. This kind of measurement has been recently included in the quality assesment approaches (Crossing the Quality Chasm: A New Health System for the 21st Century, see "References"), being PROMS a useful tool to improve the triple aim framework of better quality, appropriate utilization, and enhanced patient experience (second link in "References"). The aim of PAN-PROMISE is to incorporate an outcome variable in AP based in the patient´s experience. For this purpose the investigators involved in this project have developed a new instrument based on symptoms found to be important for the patient. This new instrument is intended to be a simple quantitative variable, relevant for the patient, that can be useful to test new treatments for AP without the need for a very large sample size, fulfilling the requirements of the triple aim framework by incorporating an outcome measurement defined by the patient.

Hypothesis:

• PROMS must be incorporated to patient care and research in AP to improve the triple aim framework of better quality, appropriate utilization, and enhanced patient experience
• A PROMS scale is a useful outcome variable to investigate new treatments in AP
• The different severity categories of the revision of the Atlanta Classification (RAC) have different scores on the PROMS scale in acute pancreatitis: severe greater than moderate and mild, moderate greater than mild.
• Increasing scores on the PROMS scale are associated to increased morbidity

Aims:

• Main aim 1: to measure patient´s symptoms during an episode of AP and to correlate the intensity of these symptoms to clinical outcomes
• Main aim 2: to design and validate a PROMS scale in AP intended to be an suitable outcome variable in future trials addressing the management of AP

Methods

Part 1: development of the PAN-PROMISE instrument The PAN-PROMISE research team developed a qualitative study in which three nominal groups were conducted, two with patients who had suffered an acute pancreatitis (AP) in hospitals in Alicante (Universitarian General Hospital)), and Valencia (Clinic Hospital), Spain and another with clinicians from the Alicante region with experience in treating patients with AP. Participants were asked about the symptoms that cause the most discomfort and concern to patients at three specific times: before receiving treatment for AP, during hospital admission and after discharge. The perspectives of patients and professionals were compared. From the information gathered in the nominal groups, a first version of a PROMS questionnaire was elaborated and shared with an international group of gastroenterologists, experienced in the management of pancreatitis, from 4 countries (Germany, Spain, USA, India and Turkey) through an online application to determine to what extent these experiences were shared in frequency and intensity by patients in these different countries. With all this information, a PROMS instrument was generated.

Part 2: measurement of patient´s symptoms in an international cohort study

2.1 Design. The PAN-PROMISE instrument is being measured in patients participating in a multicenter international prospective cohort study. Consecutive adult patients with AP are eligible after signed informed consent.

2.2 Center recruitment The PAN-PROMISE study has been endorsed by the Spanish Association of Pancreatology (AESPANC), the Spanish Association of Gastroenterology (AEG) and the International Association of Pancreatology (IAP); these associations shared the project with their members and through their social networks, calling for researchers and centers to join this initiative. PAN-PROMISE has also a webpage www.promisepancreatitis.com to promote center recruitment.

2.3 Patient registry PAN-PROMISE measurement will rely on a patient registry hosted in the Spanish Association of Gastroenterology (AEG) REDCap node. AEG offered this REDCap web-based online patient registry tool at no cost. Claudia Sánchez-Marin, a gastroenterologist, is on charge of quality control (data validation, data checks, contact with researchers to solve issues). The REDCap online registry was designed with logical rules to ensure good quality data entry, including mandatory fields to enter, logical ranges for quantitative variables, alarms if logical rules are not coherent (for example to have persistent organ failure but to classify the episode as mild or moderate), expanding fields only available in the presence of certain circumstances (for example, detailed organ failure description is only available if the patient had organ failure).

Conditions

Acute Pancreatitis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Patients ≥18 and <80 years.
• Karnofsky performance status previous to AP 100 (normal, no complaints, no evidence of disease), 90 (able to carry on normal activity; minor signs or symptoms of disease) or 80 (normal activity with effort; some signs or symptoms of disease).
• Acute pancreatitis defined as at least 2 of the following 3 criteria: A) Typical abdominal pain, B) Elevation of amylase and/or lipase more than 3 times the upper limit of normality, and C) Imaging (preferably CT and/or MR) compatible with AP.
• Written informed consent.

Exclusion Criteria

• Time between onset of symptoms and presentation in the emergency room (ER) greater than 48 hours (we do not consider as onset of symptoms previous self-limited heraldic biliary pain)
• Recruitment >24h after presentation in the ER
• Karnofsky performance status previous to AP 70 (Cares for self; unable to carry on normal activity or to do active work) or less
• Inability to express or understand the instructions of the study (severe congenital or acquired intellectual deficit).
• More than 1 previous episode of AP.
• Chronic pancreatitis.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 79 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Enrique de-Madaria

OTHER

Sponsor Role: lead

Responsible Party

Enrique de-Madaria

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

José Joaquín Mira, PsyD PhD

Role: STUDY_DIRECTOR

Universidad Miguel Hernandez de Elche

Enrique de-Madaria, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Hospital General Universitario de Alicante

Locations

Johns Hopkins Medical Center

Baltimore, Maryland, United States

Hospital Universitario San Ignacio

Bogotá, , Colombia

Attikon University Hospital

Athens, , Greece

Centre for Translational Medicine, University of Pécs

Pécs, , Hungary

University of Szeged

Szeged, , Hungary

Dip.Medicina-Università degli Studi di Verona

Verona, , Italy

Vilnius University

Vilnius, , Lithuania

Servicio de Cirugía General, IMSS, León

Guanajuato City, , Mexico

Medical University of Łódź

Lodz, , Poland

Fernando Fonseca Hospital

Amadora, , Portugal

Centro Hospitalar Tondela-Viseu

Viseu, , Portugal

Emergency Hospital of Bucharest

Bucharest, , Romania

Iuliu Hatieganu University of Medicine and Pharmacy

Cluj-Napoca, , Romania

Immanuel Kant Baltic Federal University

Kaliningrad, , Russia

Konkuk University School of Medicine

Seoul, , South Korea

Hospital General Universitario de Alicante

Alicante, , Spain

Hospital Santa María

Lleida, , Spain

Hospital San Pedro

Logroño, , Spain

Hospital Central de Asturias

Oviedo, , Spain

Complejo Hospitalario de Navarra

Pamplona, , Spain

Complejo Hospitalario Universitario de Santiago

Santiago de Compostela, , Spain

Hospital Clínico Universitario de Valladolid

Valladolid, , Spain

Hospital Alvaro Cunqueiro

Vigo, , Spain

Hospital Lozano Blesa

Zaragoza, , Spain

Hospital Miguel Servet

Zaragoza, , Spain

Medical Institute Lviv

Lviv, , Ukraine

Countries

United States; Colombia; Greece; Hungary; Italy; Lithuania; Mexico; Poland; Portugal; Romania; Russia; South Korea; Spain; Ukraine

References

Institute of Medicine (US) Committee on Quality of Health Care in America. Crossing the Quality Chasm: A New Health System for the 21st Century. Washington (DC): National Academies Press (US); 2001. Available from http://www.ncbi.nlm.nih.gov/books/NBK222274/

Reference Type: BACKGROUND

PMID: 25057539 (View on PubMed)

Sternby H, Bolado F, Canaval-Zuleta HJ, Marra-Lopez C, Hernando-Alonso AI, Del-Val-Antonana A, Garcia-Rayado G, Rivera-Irigoin R, Grau-Garcia FJ, Oms L, Millastre-Bocos J, Pascual-Moreno I, Martinez-Ares D, Rodriguez-Oballe JA, Lopez-Serrano A, Ruiz-Rebollo ML, Viejo-Almanzor A, Gonzalez-de-la-Higuera B, Orive-Calzada A, Gomez-Anta I, Pamies-Guilabert J, Fernandez-Gutierrez-Del-Alamo F, Iranzo-Gonzalez-Cruz I, Perez-Munante ME, Esteba MD, Pardillos-Tome A, Zapater P, de-Madaria E. Determinants of Severity in Acute Pancreatitis: A Nation-wide Multicenter Prospective Cohort Study. Ann Surg. 2019 Aug;270(2):348-355. doi: 10.1097/SLA.0000000000002766.

Reference Type: RESULT

PMID: 29672416 (View on PubMed)

de-Madaria E, Sanchez-Marin C, Carrillo I, Vege SS, Chooklin S, Bilyak A, Mejuto R, Mauriz V, Hegyi P, Marta K, Kamal A, Lauret-Brana E, Barbu ST, Nunes V, Ruiz-Rebollo ML, Garcia-Rayado G, Lozada-Hernandez EE, Pereira J, Negoi I, Espina S, Hollenbach M, Litvin A, Bolado-Concejo F, Vargas RD, Pascual-Moreno I, Singh VK, Mira JJ. Design and validation of a patient-reported outcome measure scale in acute pancreatitis: the PAN-PROMISE study. Gut. 2021 Jan;70(1):139-147. doi: 10.1136/gutjnl-2020-320729. Epub 2020 Apr 3.

Reference Type: DERIVED

PMID: 32245906 (View on PubMed)

Related Links

https://www.fda.gov/downloads/drugs/guidances/ucm193282.pdf

Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims

http://www.ihi.org/Engage/Initiatives/TripleAim/Pages/default.aspx

Institute for Healthcare Improvement (IHI): The IHI Triple Aim

Other Identifiers

PI2016/69

Identifier Type: -

Identifier Source: org_study_id