Development of a validated patient-reported symptom metric for pediatric eosinophilic esophagitis: qualitative methods

James P Franciosi, Kevin A Hommel, Charles W DeBrosse, Allison B Greenberg, Alexandria J Greenler, J Pablo Abonia, Marc E Rothenberg, James W Varni, James P Franciosi, Kevin A Hommel, Charles W DeBrosse, Allison B Greenberg, Alexandria J Greenler, J Pablo Abonia, Marc E Rothenberg, James W Varni

Abstract

Background: Previous attempts to measure symptoms in pediatric Eosinophilic Esophagitis (EoE) have not fully included patients and parents in the item development process. We sought to identify and validate key patient self-reported and parent proxy-reported outcomes (PROs) specific to EoE.

Methods: We developed methodology for focus and cognitive interviews based on the Food and Drug Administration (FDA) guidelines for PROs, the validated generic PedsQL™ guidelines, and the consolidated criteria for reporting qualitative research (COREQ). Both child (ages 8-12 and 13-18) and parent-proxy (ages 2-4, 5-7, 8-12, and 13-18) interviews were conducted.

Results: We conducted 75 interviews to construct the new instrument. Items were identified and developed from individual focus interviews, followed by cognitive interviews for face and content validation. Initial domains of symptom frequency and severity were developed, and open-ended questions were used to generate specific items during the focus interviews. Once developed, the instrument construct, instructions, timeframe, scoring, and specific items were systematically reviewed with a separate group of patients and their parents during the cognitive interviews.

Conclusions: To capture the full impact of pediatric EoE, both histologic findings and PROs need to be included as equally important outcome measures. We have developed the face and content validated Pediatric Eosinophilic Esophagitis Symptom Score (PEESS™ v2.0). The PEESS™ v2.0 metric is now undergoing multisite national field testing as the next iterative instrument development phase.

Figures

Figure 1
Figure 1
Summary of content validation methodology. Focus interview transcripts of pediatric patients with EoE and their parents were used to develop the items and domains for the PEESS™ v2.0. Cognitive interviewing was conducted with separate cohorts of pediatric patients and their parents in the 8-12 and 13-18 year old age groups, while parent proxy-reports were also obtained in the 2-4 year old and 5-7 year old age groups.
Figure 2
Figure 2
From the PEESS v1.0 to the validated PEESS™ v 2.0. The instrument layout and overall design went through several iterative drafts based on patient and parent-proxy feedback through focus interviews and separate cognitive interviews. For example, participants favored presenting frequency and severity of particular items side by side rather than on two separate pages.

References

    1. Furuta GT, Liacouras CA, Collins MH, Gupta SK, Justinich C, Putnam PE, Bonis P, Hassall E, Straumann A, Rothenberg ME. Eosinophilic esophagitis in children and adults: a systematic review and consensus recommendations for diagnosis and treatment. Gastroenterology. 2007;133(4):1342–1363. doi: 10.1053/j.gastro.2007.08.017.
    1. Konikoff MR, Noel RJ, Blanchard C, Kirby C, Jameson SC, Buckmeier BK, Akers R, Cohen MB, Collins MH, Assa'ad AH. et al.A randomized, double-blind, placebo-controlled trial of fluticasone propionate for pediatric eosinophilic esophagitis. Gastroenterology. 2006;131(5):1381–1391. doi: 10.1053/j.gastro.2006.08.033.
    1. Aceves SS, Newbury RO, Dohil MA, Bastian JF, Dohil R. A symptom scoring tool for identifying pediatric patients with eosinophilic esophagitis and correlating symptoms with inflammation. Ann Allergy Asthma Immunol. 2009;103(5):401–406. doi: 10.1016/S1081-1206(10)60359-6.
    1. Klinnert MD. Psychological impact of eosinophilic esophagitis on children and families. Immunol Allergy Clin North Am. 2009;29(1):99–107. doi: 10.1016/j.iac.2008.09.011. x.
    1. Rothenberg ME, Pentiuk S, Putnam PE, Collins MH. Dissociation Between Symptoms and Histological Severity in Pediatric Eosinophilic Esophagitis. J Pediatr Gastr Nutr. 2009;48(2):152–160. doi: 10.1097/MPG.0b013e31817f0197.
    1. Pedersen S, Potter P, Dachev S, Bosheva M, Kaczmarek J, Springer E, Dunkel J, Engelstatter R. Efficacy and safety of three ciclesonide doses vs placebo in children with asthma: the RAINBOW study. Respir Med. 2010;104(11):1618–1628. doi: 10.1016/j.rmed.2010.06.012.
    1. Cella D, Yount S, Rothrock N, Gershon R, Cook K, Reeve B, Ader D, Fries JF, Bruce B, Rose M. The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years. Med Care. 2007;45(5 Suppl 1):S3–S11.
    1. DeWalt DA, Rothrock N, Yount S, Stone AA. Evaluation of item candidates: the PROMIS qualitative item review. Med Care. 2007;45(5 Suppl 1):S12–21.
    1. Aletaha D. From the item to the outcome: the promising prospects of PROMIS. Arthritis Res Ther. 2010;12(1):104.. doi: 10.1186/ar2910.
    1. Einav S, Hersch M. The "PrOMIS" of things to come. Crit Care Med. 2007;35(4):1193–1194. doi: 10.1097/.
    1. Fries JF, Krishnan E, Bruce B. Items, Instruments, Crosswalks, and PROMIS. J Rheumatol. 2009;36(6):1093–1095. doi: 10.3899/jrheum.090320.
    1. Walsh TR, Irwin DE, Meier A, Varni JW, DeWalt DA. The use of focus groups in the development of the PROMIS pediatrics item bank. Quality of Life Research. 2008;17(5):725–735. doi: 10.1007/s11136-008-9338-1.
    1. Irwin DE, Varni JW, Yeatts K, DeWalt DA. Cognitive interviewing methodology in the development of a pediatric item bank: a patient reported outcomes measurement information system (PROMIS) study. Health and Quality of Life Outcomes. 2009;7
    1. Reeve BB, Hays RD, Bjorner JB, Cook KF, Crane PK, Teresi JA, Thissen D, Revicki DA, Weiss DJ, Hambleton RK. et al.Psychometric evaluation and calibration of health-related quality of life item banks: plans for the Patient-Reported Outcomes Measurement Information System (PROMIS) Med Care. 2007;45(5 Suppl 1):S22–31.
    1. Flood EM, Beusterien KM, Amonkar MM, Jurgensen CH, Dewit OE, Kahl LP, Matza LS. Patient and caregiver perspective on pediatric eosinophilic esophagitis and newly developed symptom questionnaires*. Curr Med Res Opin. 2008;24(12):3369–3381. doi: 10.1185/03007990802536900.
    1. Ronen GM, Rosenbaum P, Law M, Streiner DL. Health-related quality of life in childhood disorders: a modified focus group technique to involve children. Quality of Life Research. 2001;10(1):71–79. doi: 10.1023/A:1016659917227.
    1. Tong A, Sainsbury P, Craig J. Consolidated criteria for reporting qualitative research (COREQ): a 32-item checklist for interviews and focus groups. Int J Qual Health Care. 2007;19(6):349–357. doi: 10.1093/intqhc/mzm042.
    1. Pentiuk S, Putnam PE, Collins MH, Rothenberg ME. Dissociation between symptoms and histological severity in pediatric eosinophilic esophagitis. J Pediatr Gastroenterol Nutr. 2009;48(2):152–160. doi: 10.1097/MPG.0b013e31817f0197.
    1. Schurman JV, Wu YP, Grayson P, Friesen CA. A pilot study to assess the efficacy of biofeedback-assisted relaxation training as an adjunct treatment for pediatric functional dyspepsia associated with duodenal eosinophilia. J Pediatr Psychol. 2010;35(8):837–847. doi: 10.1093/jpepsy/jsq010.
    1. Southwell BR, Clarke MCC, Chase JW, Gibb S, Hutson JM. Improvement of quality of life in children with slow transit constipation after treatment with transcutaneous electrical stimulation. J Pediatr Surg. 2009;44(6):1268–1273. doi: 10.1016/j.jpedsurg.2009.02.031.
    1. Nuboer R, Borsboom GJJM, Zoethout JA, Koot HM, Bruining J. Effects of insulin pump vs. injection treatment on quality of life and impact of disease in children with type 1 diabetes mellitus in a randomized, prospective comparison. Pediatr Diabetes. 2008;9(4):291–296. doi: 10.1111/j.1399-5448.2008.00396.x.
    1. Schwimmer JB, Middleton MS, Deutsch R, Lavine JE. A phase 2 clinical trial of metformin as a treatment for non-diabetic paediatric non-alcoholic steatohepatitis. Aliment Pharmacol Ther. 2005;21(7):871–879. doi: 10.1111/j.1365-2036.2005.02420.x.
    1. Zeller MH, Reiter-Purtill J, Ratcliff MB, Inge TH, Noll JG. Two-year trends in psychosocial functioning after adolescent Roux-en-Y gastric bypass. Surg Obes Relat Dis. 2011.
    1. Parekh AD, Thomas JC, Trusler L, Ankerst DP, Deshpande JK, Adams MC, Pope JCt, Brock JW. Prospective evaluation of health related quality of life for pediatric patients with ureteropelvic junction obstruction. J Urol. 2008;180(5):2171–2175. doi: 10.1016/j.juro.2008.07.064. discussion 2175-2176.
    1. Thornburg CD, Calatroni A, Panepinto JA. Differences in health-related quality of life in children with sickle cell disease receiving hydroxyurea. J Pediatr Hematol Oncol. 2011;33(4):251–254. doi: 10.1097/MPH.0b013e3182114c54.
    1. Fullerton G, Tyler C, Johnston CA, Vincent JP, Harris GE, Foreyt JP. Quality of life in Mexican-American children following a weight management program. Obesity (Silver Spring) 2007;15(11):2553–2556. doi: 10.1038/oby.2007.306.
    1. McElhiney J, Lohse MR, Arora AS, Peloquin JM, Geno DM, Kuntz MM, Enders FB, Fredericksen M, Abdalla AA, Khan Y. et al.The Mayo Dysphagia Questionnaire-30: documentation of reliability and validity of a tool for interventional trials in adults with esophageal disease. Dysphagia. 2010;25(3):221–230. doi: 10.1007/s00455-009-9246-8.
    1. Dohil R, Newbury R, Fox L, Bastian J, Aceves S. Oral viscous budesonide is effective in children with eosinophilic esophagitis in a randomized, placebo-controlled trial. Gastroenterology. 2010;139(2):418–429. doi: 10.1053/j.gastro.2010.05.001.
    1. Walsh GM. Reslizumab for pediatric eosinophilic esophagitis. Immunotherapy-Uk. 2010;2(4):461–465. doi: 10.2217/imt.10.41.

Source: PubMed

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