INVITED COMMENTARIES
Resistance Testing for Helicobacter pylori Infection: Is It Finally Ready for Prime Time? Benjamin D. Gold See ‘‘Novel Helicobacter pylori Sequencing Test Identifies High Rate of Clarithromycin Resistance’’ by Mitui et al on page 6.
M
itui et al (1), in their article ‘‘Novel Helicobacter pylori Sequencing Test Identifies High Rate of Clarithromycin Resistance,’’ describe the relative resistance rate to clarithromycin in Helicobacter pylori–infected children. In their study, the investigators used a novel molecular assay (involving DNA polymerase chain reaction/sequencing) to detect clarithromycin resistance in H pylori–infected pediatric patients. The authors performed their retrospective investigation in a small cohort (N ¼ 38) of children ages 3 to 19 years who had been referred to a tertiary care pediatric referral center in Texas for diagnostic upper endoscopy (esophagogastroduodenoscopy [EGD]) because of their persisting gastrointestinal symptoms. Of the 38 H pylori–infected children who underwent EGD and biopsies, 19 (50%) were found to be harboring clarithromycinresistant strains. The total number of upper endoscopies performed at the institution was not provided by the authors in this article. By providing this number, the authors may have given the reader a better sense of the population size being referred for diagnostic upper endoscopy, presumably for persistent gastrointestinal symptoms. Between the years 2010 and 2012, a total of 38 H pylori–infected biopsies were evaluated by the novel assay used in this study. A clear strength of this study and the major observation of the investigation was that molecular characterization of clarithromycin resistance was performed on biopsies that were formalin-fixed paraffin-embedded. The logical extrapolation from the study’s findings is clearly that this novel methodology could be commercially used in everyday clinical practice, such as clinical microbiology laboratories in hospitals in which children are undergoing diagnostic upper endoscopy for upper gastrointestinal symptoms. An assay such as the one described by this study would allow hospital-based clinical laboratories to rapidly determine local clarithromycin resistance rates. In addition, for each individual patient, clarithromycin resistance could be determined quickly and reliably by this novel assay and thus facilitate improved eradication regimens for infecting H pylori strains and optimize the management of H pylori–associated gastroduodenal disease. The North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition–European Society for Pediatric Gastroenterology and Nutrition guidelines actually advise that antibiotic susceptibility testing for clarithromycin be recommended before initial
Received April 23, 2014; accepted May 9, 2014. From the Children’s Center for Digestive Healthcare, Atlanta, GA. Address correspondence and reprint requests to Benjamin D. Gold, MD, Children’s Center for Digestive Healthcare, 993-D Johnson Ferry Road, NE, Suite 440, Atlanta, GA 30342 (e-mail: [email protected]). The author reports no conflicts of interest. Copyright # 2014 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition DOI: 10.1097/MPG.0000000000000438
JPGN
Volume 59, Number 1, July 2014
clarithromycin-based triple therapy in areas/populations with a known high resistance rate (>20%) of H pylori strains in children, despite no readily available, commercial laboratories that perform H pylori resistance testing (2). Mitui et al provide a reasonable explanation of primer design and specificity/sensitivity testing, and give the reader a good sense of the assay stringency and robustness, including the use of other Helicobacter and Campylobacter spp in their assay validation. Thus, there are no major methodological weaknesses to the assay’s potential sensitivity and specificity—further support for its potential use in the clinical setting. According to their methodology, 23S ribosomal RNA sequencing, 50% of the 38 H pylori–infected strains were clarithromycin resistant. Although the authors could have been less ambiguous in their description of the results in the context of prior antibiotic exposure and eradication success, only 33.3% of those H pylori strains, which were clarithromycin resistant, were eradicated. The article would have been strengthened if the rates of H pylori prevalence in the population served by the authors’ institution at the University of Texas-Southwestern were provided, thereby giving some sense of the relative burden of H pylori infection in this region of Texas to provide a context to the relative number of infecting H pylori strains that were clarithromycin resistant in that region. The high rate of clarithromycin resistance in infecting H pylori strains described is concerning despite the relatively low number of subjects in the study cohort (3). In fact, this high rate of resistance has been found in a number of different countries and may be reflective of a trend in overall reduced rates of H pylori eradication (4–8). In a recently published meta-analysis, primary resistance rates for clarithromycin in H pylori isolated from pediatric populations ranged from 40% in different regions, with the average overall resistance rate being >20% (3). Furthermore, this meta-analysis reported that the rates of secondary resistance to clarithromycin were higher than primary resistance in the same population (3). Based on the authors’ results, it could be inferred that the overall poor (62.5%) cure rate in those children treated with a clarithromycin-based eradication regimens was also influenced by undetected clarithromycin resistance or other antibiotic resistance, which has yet to be determined in the region. This poor cure rate may also be explained simply because of the high rate of clarithromycin resistance (50%) found by Mitui et al (1), a clarithromycin resistance rate greater than that reported by any of the previously published national surveys or single-center studies in the United States, Europe, or Japan. Trends of eradication rates of H pylori appear to be dropping worldwide, and thus clinical practice may require the use of antimicrobial susceptibility testing before treatment (9). The North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition–European Society for Pediatric Gastroenterology and Nutrition H pylori guidelines committee (2) recommended that resistance testing with other microbial infections is likely the future for the treatment of H pylori–infected pediatric patients to maximize treatment success. Furthermore, as implied by the Mitui et al (1) study, clarithromycin resistance testing could be determined a priori to initiating antimicrobial therapy for an H pylori–infected child and thereby substantially improve eradication success similar to how most infectious organisms are treated, that is, treatment based on the knowledge of resistance/sensitivity profiles. Moreover, because the common mutations responsible for H pylori resistance to the other major antimicrobials used for eradication (ie, amoxicillin, metronidazole, and tetracycline) have been described, the assay developed by Mitui et al (1) could be modified to include a panel of antibiotics. Ultimately, H pylori treatment regimens could then be implemented based on the infecting strains antibiotic resistance profiles to optimize therapy against the H pylori strain infecting a symptomatic child with gastroduodenal disease.
3
Copyright 2014 by ESPGHAN and NASPGHAN. Unauthorized reproduction of this article is prohibited.
Invited Commentaries
JPGN
REFERENCES 1. Mitui M, Patel A, Leos NK, et al. Novel Helicobacter pylori sequencing test identifies high rate of clarithromycin resistance. J Pediatr Gastroenterol Nutr 2014;59:6–9. 2. Koletzko S, Jones NL, Goodman KJ, et al. Evidence-based guidelines from ESPGHAN and NASPGHAN for Helicobacter pylori infection in children. J Pediatr Gastroenterol Nutr 2011;53: 230–43. 3. Xiong LJ, Tong Y, Wang Z, et al. Detection of clarithromycin-resistant Helicobacter pylori by stool PCR in children: a comprehensive review of literature. Helicobacter 2013;18:89–101. 4. Iwanczak F, Iwanczak B. Treatment of Helicobacter pylori infection in the aspect of increasing antibiotic resistance. Adv Clin Exp Med 2012; 21:671–80. 5. Giorgio F, Principi M, De Francesco V, et al. Primary clarithromycin resistance to Helicobacter pylori: is this the main reason for triple therapy failure? World J Gastrointest Pathophysiol 2013;4:43–6. 6. Naserpour Farivar T, Najafipour R, Johari P. Prevalence of clarithromycin-resistant Helicobacter pylori in patients with chronic tonsillitis by allele-specific Scorpion real-time polymerase chain reaction assay. Laryngoscope 2013;123:1478–82. 7. Seo JH, Jun JS, Yeom JS, et al. Changing pattern of antibiotic resistance of Helicobacter pylori in children during 20 years in Jinju, South Korea. Pediatr Int 2013;55:332–6. 8. Camargo MC, Garcia A, Riquelme A, et al. The problem of Helicobacter pylori resistance to antibiotics: a systematic review in Latin America. Am J Gastroenterol 2014;109:485–95. 9. Zhou L, Zhang J, Chen M, et al. A comparative study of sequential therapy and standard triple therapy for Helicobacter pylori infection: a randomized multicenter trial. Am J Gastroenterol 2014;109:535–41.
PROs and CONcepts Dawn R. Ebach See ‘‘Development of a Self-Efficacy Scale for Adolescents and Young Adults With Inflammatory Bowel Disease’’ by Izaguirre on page 29.
I
n this issue of the Journal of Pediatric Gastroenterology and Nutrition the article by Izaguirre and Keefer (1) describes the process and findings in the development of a survey. Development of such tools involves qualitative processes. The following is a brief overview of this process. Guidelines for patient-reported outcomes (PRO) were developed by the US Food and Drug Administration (FDA) (2,3) and used by the authors. A PRO is ‘‘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’’ (3). A PRO can measure a symptom, sign, change, effect of an intervention, or a concept (eg, self-efficacy). The most recent guidelines outline a cycle involving 5 steps: Received March 5, 2014; accepted March 20, 2014. From the Department of Pediatrics, University of Iowa, Iowa City. Address correspondence and reprint requests to Dawn R. Ebach, MD, Department of Pediatrics, University of Iowa, 200 Hawkins Dr, Iowa City, IA 52242 (e-mail: [email protected]). The authors report no conflicts of interest. Copyright # 2014 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition DOI: 10.1097/MPG.0000000000000382
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Volume 59, Number 1, July 2014
1. Hypothesize a conceptual framework: Identify concepts and develop a framework for a target population and application. Concepts and domains important to patients are identified. The FDA requires documentation of the development process and how domains are identified. In this study, the concept of self-efficacy (belief in one’s ability to complete a task) in pediatric and young adult inflammatory bowel disease is being explored. 2. Adjust conceptual framework and draft instrument. The instrument is developed using patient input. In this study, the authors performed semistructured interviews with surveytargeted subjects. In the interviews, open-ended questions focusing on the question of self-efficacy were used. The instrument is created to address the domains identified. Four domains were identified in this study: managing medical care, managing everyday life with IBD, managing feelings, and managing the future with IBD, and questions were developed. A Likert score, which is a rating scale often measuring the level of agreement with a statement, is used to answer questions ranging from knowledge to adherence and self-management. Draft questions are piloted with subjects. The subjects may ask for clarification and are asked to think aloud their responses to ensure questions are clear, appropriate, and that directions are understood. The survey was then adapted. 3. Confirm conceptual framework and assess other measurement properties. Once a survey is developed, it must go through a process of assessing reliability (test and retest among stable subjects) and validity (the test measures what it purports to measure) as well as the ability to detect change. This step in survey development is presently ongoing by the authors’ and involves a larger group of subjects. The questions may be revised during this stage if there are findings of poor clarity, variability in responses, reproducibility, or other problems. The researchers then finalize scoring and interpretation of results. Manuals are developed during this stage directing administration and interpretation. 4. Collect, analyze, and interpret data. Once the self-efficacy instrument is found valid and reliable it can then be used in studies to determine whether interventions result in changes in self-efficacy. 5. Modify instrument if needed. For example, if it is determined that the questions are not suitable for younger patients, a new instrument aimed at this population may be developed by modifying the instrument. This new instrument would then go through the process of validation and reliability for this population. A systematic review of self-efficacy scales for chronic disease found that many of the studies did not define a specific aim (4). Izaguirre and Keefer used this scale to measure the impact of self-management interventions in improving pediatric IBD outcomes, and further studies will confirm its utility. The FDA expects that these instruments will be reliable, valid, detect change (in this example, one would expect self-efficacy to improve with time or with intervention), and detect a ‘‘minimum important difference.’’ To fulfill these requirements follow-up studies using the instrument are needed. The study by Izaguirre and Keefer meets the initial FDA recommendations for development and I expect validity and reliability studies will then allow this instrument to be more widely used.
REFERENCES 1. Izaguirre MR, Keefer L. Development of a self-efficacy scale for adolescents and young adults with inflammatory bowel disease. J Pediatr Gastroenterol Nutr 2014;59:29–32.
www.jpgn.org
Copyright 2014 by ESPGHAN and NASPGHAN. Unauthorized reproduction of this article is prohibited.
JPGN
Volume 59, Number 1, July 2014
2. US Department of Health and Human Services FDA Center for Drug Evaluation and Research; US Department of Health and Human Services FDA Center for Biologics Evaluation and Research; US Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes 2006; 4:79.
www.jpgn.org
Invited Commentaries
3. US Department of Health and Human Services Food and Drug Administration. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. http:// www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf. Accessed May 12, 2014. 4. Frei A, Svarin A, Steurer-Stey C, et al. Self-efficacy instruments for patients with chronic disease suffer from methodological limitations—a systematic review. Health Qual Life Outcomes 2009;7:86–96.
5
Copyright 2014 by ESPGHAN and NASPGHAN. Unauthorized reproduction of this article is prohibited.
Resistance Testing for Helicobacter pylori Infection: Is It Finally Ready for Prime Time? Benjamin D. Gold See ‘‘Novel Helicobacter pylori Sequencing Test Identifies High Rate of Clarithromycin Resistance’’ by Mitui et al on page 6.
M
itui et al (1), in their article ‘‘Novel Helicobacter pylori Sequencing Test Identifies High Rate of Clarithromycin Resistance,’’ describe the relative resistance rate to clarithromycin in Helicobacter pylori–infected children. In their study, the investigators used a novel molecular assay (involving DNA polymerase chain reaction/sequencing) to detect clarithromycin resistance in H pylori–infected pediatric patients. The authors performed their retrospective investigation in a small cohort (N ¼ 38) of children ages 3 to 19 years who had been referred to a tertiary care pediatric referral center in Texas for diagnostic upper endoscopy (esophagogastroduodenoscopy [EGD]) because of their persisting gastrointestinal symptoms. Of the 38 H pylori–infected children who underwent EGD and biopsies, 19 (50%) were found to be harboring clarithromycinresistant strains. The total number of upper endoscopies performed at the institution was not provided by the authors in this article. By providing this number, the authors may have given the reader a better sense of the population size being referred for diagnostic upper endoscopy, presumably for persistent gastrointestinal symptoms. Between the years 2010 and 2012, a total of 38 H pylori–infected biopsies were evaluated by the novel assay used in this study. A clear strength of this study and the major observation of the investigation was that molecular characterization of clarithromycin resistance was performed on biopsies that were formalin-fixed paraffin-embedded. The logical extrapolation from the study’s findings is clearly that this novel methodology could be commercially used in everyday clinical practice, such as clinical microbiology laboratories in hospitals in which children are undergoing diagnostic upper endoscopy for upper gastrointestinal symptoms. An assay such as the one described by this study would allow hospital-based clinical laboratories to rapidly determine local clarithromycin resistance rates. In addition, for each individual patient, clarithromycin resistance could be determined quickly and reliably by this novel assay and thus facilitate improved eradication regimens for infecting H pylori strains and optimize the management of H pylori–associated gastroduodenal disease. The North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition–European Society for Pediatric Gastroenterology and Nutrition guidelines actually advise that antibiotic susceptibility testing for clarithromycin be recommended before initial
Received April 23, 2014; accepted May 9, 2014. From the Children’s Center for Digestive Healthcare, Atlanta, GA. Address correspondence and reprint requests to Benjamin D. Gold, MD, Children’s Center for Digestive Healthcare, 993-D Johnson Ferry Road, NE, Suite 440, Atlanta, GA 30342 (e-mail: [email protected]). The author reports no conflicts of interest. Copyright # 2014 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition DOI: 10.1097/MPG.0000000000000438
JPGN
Volume 59, Number 1, July 2014
clarithromycin-based triple therapy in areas/populations with a known high resistance rate (>20%) of H pylori strains in children, despite no readily available, commercial laboratories that perform H pylori resistance testing (2). Mitui et al provide a reasonable explanation of primer design and specificity/sensitivity testing, and give the reader a good sense of the assay stringency and robustness, including the use of other Helicobacter and Campylobacter spp in their assay validation. Thus, there are no major methodological weaknesses to the assay’s potential sensitivity and specificity—further support for its potential use in the clinical setting. According to their methodology, 23S ribosomal RNA sequencing, 50% of the 38 H pylori–infected strains were clarithromycin resistant. Although the authors could have been less ambiguous in their description of the results in the context of prior antibiotic exposure and eradication success, only 33.3% of those H pylori strains, which were clarithromycin resistant, were eradicated. The article would have been strengthened if the rates of H pylori prevalence in the population served by the authors’ institution at the University of Texas-Southwestern were provided, thereby giving some sense of the relative burden of H pylori infection in this region of Texas to provide a context to the relative number of infecting H pylori strains that were clarithromycin resistant in that region. The high rate of clarithromycin resistance in infecting H pylori strains described is concerning despite the relatively low number of subjects in the study cohort (3). In fact, this high rate of resistance has been found in a number of different countries and may be reflective of a trend in overall reduced rates of H pylori eradication (4–8). In a recently published meta-analysis, primary resistance rates for clarithromycin in H pylori isolated from pediatric populations ranged from 40% in different regions, with the average overall resistance rate being >20% (3). Furthermore, this meta-analysis reported that the rates of secondary resistance to clarithromycin were higher than primary resistance in the same population (3). Based on the authors’ results, it could be inferred that the overall poor (62.5%) cure rate in those children treated with a clarithromycin-based eradication regimens was also influenced by undetected clarithromycin resistance or other antibiotic resistance, which has yet to be determined in the region. This poor cure rate may also be explained simply because of the high rate of clarithromycin resistance (50%) found by Mitui et al (1), a clarithromycin resistance rate greater than that reported by any of the previously published national surveys or single-center studies in the United States, Europe, or Japan. Trends of eradication rates of H pylori appear to be dropping worldwide, and thus clinical practice may require the use of antimicrobial susceptibility testing before treatment (9). The North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition–European Society for Pediatric Gastroenterology and Nutrition H pylori guidelines committee (2) recommended that resistance testing with other microbial infections is likely the future for the treatment of H pylori–infected pediatric patients to maximize treatment success. Furthermore, as implied by the Mitui et al (1) study, clarithromycin resistance testing could be determined a priori to initiating antimicrobial therapy for an H pylori–infected child and thereby substantially improve eradication success similar to how most infectious organisms are treated, that is, treatment based on the knowledge of resistance/sensitivity profiles. Moreover, because the common mutations responsible for H pylori resistance to the other major antimicrobials used for eradication (ie, amoxicillin, metronidazole, and tetracycline) have been described, the assay developed by Mitui et al (1) could be modified to include a panel of antibiotics. Ultimately, H pylori treatment regimens could then be implemented based on the infecting strains antibiotic resistance profiles to optimize therapy against the H pylori strain infecting a symptomatic child with gastroduodenal disease.
3
Copyright 2014 by ESPGHAN and NASPGHAN. Unauthorized reproduction of this article is prohibited.
Invited Commentaries
JPGN
REFERENCES 1. Mitui M, Patel A, Leos NK, et al. Novel Helicobacter pylori sequencing test identifies high rate of clarithromycin resistance. J Pediatr Gastroenterol Nutr 2014;59:6–9. 2. Koletzko S, Jones NL, Goodman KJ, et al. Evidence-based guidelines from ESPGHAN and NASPGHAN for Helicobacter pylori infection in children. J Pediatr Gastroenterol Nutr 2011;53: 230–43. 3. Xiong LJ, Tong Y, Wang Z, et al. Detection of clarithromycin-resistant Helicobacter pylori by stool PCR in children: a comprehensive review of literature. Helicobacter 2013;18:89–101. 4. Iwanczak F, Iwanczak B. Treatment of Helicobacter pylori infection in the aspect of increasing antibiotic resistance. Adv Clin Exp Med 2012; 21:671–80. 5. Giorgio F, Principi M, De Francesco V, et al. Primary clarithromycin resistance to Helicobacter pylori: is this the main reason for triple therapy failure? World J Gastrointest Pathophysiol 2013;4:43–6. 6. Naserpour Farivar T, Najafipour R, Johari P. Prevalence of clarithromycin-resistant Helicobacter pylori in patients with chronic tonsillitis by allele-specific Scorpion real-time polymerase chain reaction assay. Laryngoscope 2013;123:1478–82. 7. Seo JH, Jun JS, Yeom JS, et al. Changing pattern of antibiotic resistance of Helicobacter pylori in children during 20 years in Jinju, South Korea. Pediatr Int 2013;55:332–6. 8. Camargo MC, Garcia A, Riquelme A, et al. The problem of Helicobacter pylori resistance to antibiotics: a systematic review in Latin America. Am J Gastroenterol 2014;109:485–95. 9. Zhou L, Zhang J, Chen M, et al. A comparative study of sequential therapy and standard triple therapy for Helicobacter pylori infection: a randomized multicenter trial. Am J Gastroenterol 2014;109:535–41.
PROs and CONcepts Dawn R. Ebach See ‘‘Development of a Self-Efficacy Scale for Adolescents and Young Adults With Inflammatory Bowel Disease’’ by Izaguirre on page 29.
I
n this issue of the Journal of Pediatric Gastroenterology and Nutrition the article by Izaguirre and Keefer (1) describes the process and findings in the development of a survey. Development of such tools involves qualitative processes. The following is a brief overview of this process. Guidelines for patient-reported outcomes (PRO) were developed by the US Food and Drug Administration (FDA) (2,3) and used by the authors. A PRO is ‘‘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’’ (3). A PRO can measure a symptom, sign, change, effect of an intervention, or a concept (eg, self-efficacy). The most recent guidelines outline a cycle involving 5 steps: Received March 5, 2014; accepted March 20, 2014. From the Department of Pediatrics, University of Iowa, Iowa City. Address correspondence and reprint requests to Dawn R. Ebach, MD, Department of Pediatrics, University of Iowa, 200 Hawkins Dr, Iowa City, IA 52242 (e-mail: [email protected]). The authors report no conflicts of interest. Copyright # 2014 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition DOI: 10.1097/MPG.0000000000000382
4
Volume 59, Number 1, July 2014
1. Hypothesize a conceptual framework: Identify concepts and develop a framework for a target population and application. Concepts and domains important to patients are identified. The FDA requires documentation of the development process and how domains are identified. In this study, the concept of self-efficacy (belief in one’s ability to complete a task) in pediatric and young adult inflammatory bowel disease is being explored. 2. Adjust conceptual framework and draft instrument. The instrument is developed using patient input. In this study, the authors performed semistructured interviews with surveytargeted subjects. In the interviews, open-ended questions focusing on the question of self-efficacy were used. The instrument is created to address the domains identified. Four domains were identified in this study: managing medical care, managing everyday life with IBD, managing feelings, and managing the future with IBD, and questions were developed. A Likert score, which is a rating scale often measuring the level of agreement with a statement, is used to answer questions ranging from knowledge to adherence and self-management. Draft questions are piloted with subjects. The subjects may ask for clarification and are asked to think aloud their responses to ensure questions are clear, appropriate, and that directions are understood. The survey was then adapted. 3. Confirm conceptual framework and assess other measurement properties. Once a survey is developed, it must go through a process of assessing reliability (test and retest among stable subjects) and validity (the test measures what it purports to measure) as well as the ability to detect change. This step in survey development is presently ongoing by the authors’ and involves a larger group of subjects. The questions may be revised during this stage if there are findings of poor clarity, variability in responses, reproducibility, or other problems. The researchers then finalize scoring and interpretation of results. Manuals are developed during this stage directing administration and interpretation. 4. Collect, analyze, and interpret data. Once the self-efficacy instrument is found valid and reliable it can then be used in studies to determine whether interventions result in changes in self-efficacy. 5. Modify instrument if needed. For example, if it is determined that the questions are not suitable for younger patients, a new instrument aimed at this population may be developed by modifying the instrument. This new instrument would then go through the process of validation and reliability for this population. A systematic review of self-efficacy scales for chronic disease found that many of the studies did not define a specific aim (4). Izaguirre and Keefer used this scale to measure the impact of self-management interventions in improving pediatric IBD outcomes, and further studies will confirm its utility. The FDA expects that these instruments will be reliable, valid, detect change (in this example, one would expect self-efficacy to improve with time or with intervention), and detect a ‘‘minimum important difference.’’ To fulfill these requirements follow-up studies using the instrument are needed. The study by Izaguirre and Keefer meets the initial FDA recommendations for development and I expect validity and reliability studies will then allow this instrument to be more widely used.
REFERENCES 1. Izaguirre MR, Keefer L. Development of a self-efficacy scale for adolescents and young adults with inflammatory bowel disease. J Pediatr Gastroenterol Nutr 2014;59:29–32.
www.jpgn.org
Copyright 2014 by ESPGHAN and NASPGHAN. Unauthorized reproduction of this article is prohibited.
JPGN
Volume 59, Number 1, July 2014
2. US Department of Health and Human Services FDA Center for Drug Evaluation and Research; US Department of Health and Human Services FDA Center for Biologics Evaluation and Research; US Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes 2006; 4:79.
www.jpgn.org
Invited Commentaries
3. US Department of Health and Human Services Food and Drug Administration. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. http:// www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf. Accessed May 12, 2014. 4. Frei A, Svarin A, Steurer-Stey C, et al. Self-efficacy instruments for patients with chronic disease suffer from methodological limitations—a systematic review. Health Qual Life Outcomes 2009;7:86–96.
5
Copyright 2014 by ESPGHAN and NASPGHAN. Unauthorized reproduction of this article is prohibited.
