Clinical trial NCT03650062: Patient-reported Outcome Scale in Acute Pancreatitis
Official title: Patient-reported Outcome Scale in Acute pancreatitis-an International Prospective Cohort Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Acute Pancreatitis
Keywords: Patient Reported Outcome Measures
Follow-Up Studies
Patient Outcome Assessment
Treatment Outcome
Pancreatitis, Acute Necrotizing
Lead sponsor: Hospital General Universitario de Alicante
Sponsor class: OTHER
First posted: 2018-08-28
Enrollment: 525
Sex: ALL
Minimum age (years): 18
Maximum age (years): 79
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Consecutive patients admitted with acute pancreatitis in the participating centers.

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).

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.
* Presence of diseases or conditions different from AP that may interfere with the scale: other causes of abdominal pain (especially acute cholecystitis; Note: choledocholithiasis-acute cholangitis is not an exclusion criteria), obstruction of the digestive tract (peptic pyloric stenosis, gastrointestinal anastomotic stenosis, diabetic gastroparesis, gastrointestinal neoplasia...), nausea-vomiting (brain tumor, chemotherapy...) or weakness (pre-existing anemia with Hb \<9 mg/dL, heart failure or respiratory insufficiency associated with minimal effort dyspnea, or domiciliary treatment with O2, advanced neoplasms or other debilitating diseases).

Primary outcomes:
measure: Severity of acute pancreatitis according to the revised Atlanta classification [incidence of local and/or systemic complications]
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: PAN-PROMISE patient reported outcome measurement instrument
timeFrame: From within 24h after admission to 15 days after discharge
Secondary outcomes:
measure: EORTC QLQ-C30 (European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-C30) Version 3.0
timeFrame: 15 days after hospital discharge
measure: C-reactive protein
timeFrame: 48 and 72h after recruitment
measure: Local complications (incidence of pancreatic/peripancreatic necrosis and acute peripancreatic fluid collections)
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Need for invasive treatment
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Need for nutritional support
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Admission in the Intensive Care Unit
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Hospital stay
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Organ failure
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Mortality
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Hospital readmission
timeFrame: Up to 15 days after hospital discharge from index hospital admission
measure: Systemic Inflammatory Response Syndrome (SIRS)
timeFrame: Up to 15 days after hospital discharge from index hospital admission
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins Medical Center
state: Maryland
city: Bogotá
country: Colombia
facility: Hospital Universitario San Ignacio
city: Athens
country: Greece
facility: Attikon University Hospital
city: Pécs
country: Hungary
facility: Centre for Translational Medicine, University of Pécs
city: Szeged
country: Hungary
facility: University of Szeged
city: Verona
country: Italy
facility: Dip.Medicina-Università degli Studi di Verona
city: Seoul
country: Korea, Republic of
facility: Konkuk University School of Medicine
city: Vilnius
country: Lithuania
facility: Vilnius University
city: Guanajuato
country: Mexico
facility: Servicio de Cirugía General, IMSS, León
city: Łódź
country: Poland
facility: Medical University of Łódź
city: Amadora
country: Portugal
facility: Fernando Fonseca Hospital
city: Viseu
country: Portugal
facility: Centro Hospitalar Tondela-Viseu
city: Bucharest
country: Romania
facility: Emergency Hospital of Bucharest
city: Cluj-Napoca
country: Romania
facility: Iuliu Hatieganu University of Medicine and Pharmacy
city: Kaliningrad
country: Russian Federation
facility: Immanuel Kant Baltic Federal University
city: Alicante
country: Spain
facility: Hospital General Universitario de Alicante
city: Lleida
country: Spain
facility: Hospital Santa María
city: Logroño
country: Spain
facility: Hospital San Pedro
city: Oviedo
country: Spain
facility: Hospital Central de Asturias
city: Pamplona
country: Spain
facility: Complejo Hospitalario de Navarra
city: Santiago De Compostela
country: Spain
facility: Complejo Hospitalario Universitario de Santiago
city: Valladolid
country: Spain
facility: Hospital Clínico Universitario de Valladolid
city: Vigo
country: Spain
facility: Hospital Alvaro Cunqueiro
city: Zaragoza
country: Spain
facility: Hospital Lozano Blesa
city: Zaragoza
country: Spain
facility: Hospital Miguel Servet
city: Lviv
country: Ukraine
facility: Medical Institute Lviv
Study officials:
affiliation: Universidad Miguel Hernandez de Elche
name: José Joaquín Mira, PsyD PhD
role: STUDY_DIRECTOR
affiliation: Hospital General Universitario de Alicante
name: Enrique de-Madaria, MD PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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/
citation: 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.
citation: 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.