PRESENTATION
Acute Gastroenteritis. The COMMENT Working Group on Acute Diarrhea: Where Are We Now and Where Are We Going? Hania Szajewska, MD and Jacek Karas, MD
Abstract: Recently, the Consensus Group on Outcome Measures Made in Paediatric Enteral Nutrition Clinical Trials (COMMENT) was established. COMMENT agreed that consensus is needed on a core set of outcomes with agreed-upon definitions on what should be measured and reported in nutritional trials. To achieve this goal, 6 working groups were set up, including the working group on Acute Diarrhea. The following 4 steps are needed to complete the work: (1) to identify how outcomes related to acute diarrhea are reported; (2) to decide on the methodology for determining which outcomes to measure in clinical trials; (3) to develop a core outcome set for clinical trials performed in subjects with acute diarrhea; and (4) to determine how to measure the outcomes in the core set. The aim of this paper is to summarize what has been achieved and implemented so far, what is currently being done, and what we aim to achieve in the not so distant future. Key Words: acute gastroenteritis, outcomes, core outcome set, trial
(J Clin Gastroenterol 2014;48:S32–S33)
R
ecently, the Consensus Group on Outcome Measures Made in Paediatric Enteral Nutrition Clinical Trials (COMMENT) was established.1 COMMENT agreed that consensus is needed on a core set of outcomes with agreedupon definitions on what should be measured and reported in nutritional trials. To achieve this goal, 6 working groups (WGs) were set up, including the WG on Acute Diarrhea currently consisting of 6 members (Shai Ashkenazi, Alfredo Guarino, Jacek Karas, Raanan Shamir, Hania Szajewska, and Yvan Vandenplas). The aim of this paper is to summarize what has been achieved and implemented so far, what is currently being done, and what we aim to achieve in the not so distant future.
STEP 1. TO IDENTIFY HOW OUTCOMES RELATED TO ACUTE DIARRHEA ARE REPORTED The first step involved identifying how outcomes related to acute diarrhea are reported. By searching MEDLINE, EMBASE, and the Cochrane Library, we found one systematic review2 (search date: February 2009) that identified 138 randomized controlled trials reporting on Z1 primary outcomes related to pediatric acute diarrhea/diseases. The included trials used 64 unique definitions of diarrhea, 69 unique definitions of diarrhea resolution, and 46 unique primary outcomes. Overall, this systematic review documented substantial heterogeneity in acute diarrhea outcomes. Furthermore, even in what would be considered methodologically From the Department of Paediatrics, The Medical University of Warsaw, Warsaw, Poland. The authors declare no conflicts of interest for this review. Correspondence: Hania Szajewska, MD, Department of Paediatrics, The Medical University of Warsaw, 01-184 Warsaw, Dzialdowska 1, Poland (e-mail: [email protected]). Copyright r 2014 by Lippincott Williams & Wilkins
S32 | www.jcge.com
sound clinical trials, definitions of diarrhea, primary outcomes, and instruments used in randomized controlled trials of pediatric acute diarrhea lacked evidence of validity and focused on indices that may not be important to participants.
STEP 2. TO DECIDE ON THE METHODOLOGY FOR DETERMINING WHICH OUTCOMES TO MEASURE IN CLINICAL TRIALS This part of the project focused on the methodology for determining which outcomes to measure in clinical trials. A review of studies that address the process of selecting outcomes to measure in clinical trials revealed that the best strategy for selecting outcomes for clinical trials in children is currently not known.3 When deciding on our own methods, the WG had the choice of the Delphi technique, the nominal group technique, a semistructured discussion, or a questionnaire-based survey.3 The Delphi technique was eventually chosen. This is a structured method for reaching consensus in which opinions are sought from individuals and the collated results are fed back to the group as a whole to generate further discussion and finally reach an agreement.4
STEP 3. TO DEVELOP A CORE OUTCOME SET FOR CLINICAL TRIALS PERFORMED IN SUBJECTS WITH ACUTE DIARRHEA The aim of this part of the project was to identify outcomes of relevance in clinical trials on acute diarrhea from different perspectives (ie, clinicians/researchers, patients or their families, representatives from industry, and regulatory people). Recently, Sinha et al5 developed a pilot method for identifying outcomes of particular relevance when evaluating the effects of regular therapies for chronic childhood asthma from the perspectives of clinicians involved in the outpatient management of children with asthma, parents of children younger than 18 years, and young people aged between 13 and 18 years. For this, pediatricians and specialist nurses, identified through the British Paediatric Respiratory Society, completed a 2-round Delphi survey. The WG decided to adopt the methodology of the pilot study by Sinha et al.5 In brief, this will be a 2-step questionnaire study using the Delphi technique. The process will first involve identifying a long list of potential outcomes (phase 1) and then defining a short list of clinically important outcomes (phase 2).
Clinical Questionnaire An electronic questionnaire with 2 questions was created for clinicians/researchers, industry representatives, and members of regulatory bodies (Table 1). For phase 1, a clinical questionnaire was created with open-ended questions to identify potential outcomes. Members of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition
J Clin Gastroenterol
Volume 48, Supp. 1, November/December 2014
J Clin Gastroenterol
Volume 48, Supp. 1, November/December 2014
Acute Gastroenteritis
TABLE 1. Open-ended Questions Asked in the Clinicians’ and Parents’ Questionnaires Clinicians’ questionnaire Question You see a hypothetical child younger than 5 y with acute gastroenteritis (nonbloody diarrhea;
Acute Gastroenteritis. The COMMENT Working Group on Acute Diarrhea: Where Are We Now and Where Are We Going? Hania Szajewska, MD and Jacek Karas, MD
Abstract: Recently, the Consensus Group on Outcome Measures Made in Paediatric Enteral Nutrition Clinical Trials (COMMENT) was established. COMMENT agreed that consensus is needed on a core set of outcomes with agreed-upon definitions on what should be measured and reported in nutritional trials. To achieve this goal, 6 working groups were set up, including the working group on Acute Diarrhea. The following 4 steps are needed to complete the work: (1) to identify how outcomes related to acute diarrhea are reported; (2) to decide on the methodology for determining which outcomes to measure in clinical trials; (3) to develop a core outcome set for clinical trials performed in subjects with acute diarrhea; and (4) to determine how to measure the outcomes in the core set. The aim of this paper is to summarize what has been achieved and implemented so far, what is currently being done, and what we aim to achieve in the not so distant future. Key Words: acute gastroenteritis, outcomes, core outcome set, trial
(J Clin Gastroenterol 2014;48:S32–S33)
R
ecently, the Consensus Group on Outcome Measures Made in Paediatric Enteral Nutrition Clinical Trials (COMMENT) was established.1 COMMENT agreed that consensus is needed on a core set of outcomes with agreedupon definitions on what should be measured and reported in nutritional trials. To achieve this goal, 6 working groups (WGs) were set up, including the WG on Acute Diarrhea currently consisting of 6 members (Shai Ashkenazi, Alfredo Guarino, Jacek Karas, Raanan Shamir, Hania Szajewska, and Yvan Vandenplas). The aim of this paper is to summarize what has been achieved and implemented so far, what is currently being done, and what we aim to achieve in the not so distant future.
STEP 1. TO IDENTIFY HOW OUTCOMES RELATED TO ACUTE DIARRHEA ARE REPORTED The first step involved identifying how outcomes related to acute diarrhea are reported. By searching MEDLINE, EMBASE, and the Cochrane Library, we found one systematic review2 (search date: February 2009) that identified 138 randomized controlled trials reporting on Z1 primary outcomes related to pediatric acute diarrhea/diseases. The included trials used 64 unique definitions of diarrhea, 69 unique definitions of diarrhea resolution, and 46 unique primary outcomes. Overall, this systematic review documented substantial heterogeneity in acute diarrhea outcomes. Furthermore, even in what would be considered methodologically From the Department of Paediatrics, The Medical University of Warsaw, Warsaw, Poland. The authors declare no conflicts of interest for this review. Correspondence: Hania Szajewska, MD, Department of Paediatrics, The Medical University of Warsaw, 01-184 Warsaw, Dzialdowska 1, Poland (e-mail: [email protected]). Copyright r 2014 by Lippincott Williams & Wilkins
S32 | www.jcge.com
sound clinical trials, definitions of diarrhea, primary outcomes, and instruments used in randomized controlled trials of pediatric acute diarrhea lacked evidence of validity and focused on indices that may not be important to participants.
STEP 2. TO DECIDE ON THE METHODOLOGY FOR DETERMINING WHICH OUTCOMES TO MEASURE IN CLINICAL TRIALS This part of the project focused on the methodology for determining which outcomes to measure in clinical trials. A review of studies that address the process of selecting outcomes to measure in clinical trials revealed that the best strategy for selecting outcomes for clinical trials in children is currently not known.3 When deciding on our own methods, the WG had the choice of the Delphi technique, the nominal group technique, a semistructured discussion, or a questionnaire-based survey.3 The Delphi technique was eventually chosen. This is a structured method for reaching consensus in which opinions are sought from individuals and the collated results are fed back to the group as a whole to generate further discussion and finally reach an agreement.4
STEP 3. TO DEVELOP A CORE OUTCOME SET FOR CLINICAL TRIALS PERFORMED IN SUBJECTS WITH ACUTE DIARRHEA The aim of this part of the project was to identify outcomes of relevance in clinical trials on acute diarrhea from different perspectives (ie, clinicians/researchers, patients or their families, representatives from industry, and regulatory people). Recently, Sinha et al5 developed a pilot method for identifying outcomes of particular relevance when evaluating the effects of regular therapies for chronic childhood asthma from the perspectives of clinicians involved in the outpatient management of children with asthma, parents of children younger than 18 years, and young people aged between 13 and 18 years. For this, pediatricians and specialist nurses, identified through the British Paediatric Respiratory Society, completed a 2-round Delphi survey. The WG decided to adopt the methodology of the pilot study by Sinha et al.5 In brief, this will be a 2-step questionnaire study using the Delphi technique. The process will first involve identifying a long list of potential outcomes (phase 1) and then defining a short list of clinically important outcomes (phase 2).
Clinical Questionnaire An electronic questionnaire with 2 questions was created for clinicians/researchers, industry representatives, and members of regulatory bodies (Table 1). For phase 1, a clinical questionnaire was created with open-ended questions to identify potential outcomes. Members of the European Society for Paediatric Gastroenterology, Hepatology and Nutrition
J Clin Gastroenterol
Volume 48, Supp. 1, November/December 2014
J Clin Gastroenterol
Volume 48, Supp. 1, November/December 2014
Acute Gastroenteritis
TABLE 1. Open-ended Questions Asked in the Clinicians’ and Parents’ Questionnaires Clinicians’ questionnaire Question You see a hypothetical child younger than 5 y with acute gastroenteritis (nonbloody diarrhea;
