Publications by authors named "Katarina Halling"

24 Publications

  • Page 1 of 1

"Lower abdominal pains, as if I was being squeezed…in a clamp": A Qualitative Analysis of Symptoms, Patient-Perceived Side Effects and Impacts of Ovarian Cancer.

Patient 2020 04;13(2):189-200

University of Chichester, Chichester, PO19 6PE, UK.

Background: Understanding the patient's perception of their disease is vital for guiding care decisions. The current study aimed to identify the most predominant experiences in women diagnosed with, and treated for, ovarian cancer in terms of disease-related symptoms, treatment-attributed side effects and their impacts.

Methods: Semi-structured qualitative interviews about disease-related symptoms, treatment-attributed side effects and their impacts were conducted with women who were being treated for ovarian cancer in Europe (n = 55) or in the USA (n = 9). The women were also asked to rate the bothersomeness of the symptoms, side effects and impacts that they mentioned during the interview. Symptoms, side effects and impacts were identified from coded interview transcripts using an iterative coding framework.

Results: Bloating, abdominal pain, tiredness and frequent urination were the most frequently expressed symptoms, and were reported by 72%, 67%, 64% and 55% of women, respectively, which together constituted approximately 30% of all symptom expressions. The most bothersome symptoms were reported as bloating, abdominal pain, pain in the side, tiredness and fatigue. The most frequently expressed side effects were hair loss, neuropathy, tiredness and nausea, which were reported by 84%, 63%, 61% and 61% of women, respectively. The most bothersome reported side effects were constipation, nausea, diarrhoea, pain in general, fatigue, weakness, reduced sleep quality and hair loss. Feelings of anxiety, concerns about the future, physical functioning, work limitations and the adoption of coping strategies were the most frequently expressed impacts and were reported by 72-80% of women. Impacts reported as the most difficult to deal with were concerns about the future, emotional difficulties in general, physical functioning, sexual functioning, negative self-image, fatigue, sleep difficulties, financial burden and work limitations.

Conclusions: In our qualitative study, the most common and most bothersome experiences reported by women treated for ovarian cancer were symptoms of bloating, abdominal pain and tiredness; side effects of hair loss, nausea and tiredness/fatigue; and impacts relating to concerns about the future, physical functioning and work limitations. We suggest that clinicians measure these experiences consistently and take them into consideration when making treatment decisions.
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April 2020

Patients' experience of recurrent/metastatic head and neck squamous cell carcinoma and their perspective on the EORTC QLQ-C30 and QLQ-H&N35 questionnaires: a qualitative study.

J Patient Rep Outcomes 2017 1;2:33. Epub 2018 Aug 1.

6Aintree University Hospital, Liverpool, UK.

Background: Head and neck squamous cell carcinoma (HNSCC) and its associated treatments may affect all aspects of patients' health-related quality of life (HRQoL). Although the EORTC QLQ-H&N35 is regularly administered to patients with HNSCC, there is a paucity of studies re-assessing the conceptual relevance of this patient-reported outcome (PRO) measure from a patient perspective. Furthermore, the content validity of the EORTC QLQ-C30 has not been widely documented in patients with recurrent and/or metastatic HNSCC. The objectives of this study were to understand patients' experiences of recurrent/metastatic HNSCC and its treatments, and to evaluate the conceptual relevance and acceptability of the EORTC QLQ-C30 and QLQ-H&N35 from a patient perspective for use in clinical trials.

Methods: A literature review and clinician interviews were conducted to inform in-depth semi-structured telephone interviews with US patients who had received treatment for recurrent and/or metastatic HNSCC in the preceding 12 months. Interview transcripts were analysed thematically using ATLAS.ti v7; patient quotes were coded to identify concepts and themes to develop a conceptual model of HNSCC experience.

Results: Fourteen patients were interviewed (71% male, aged 35-84 years). Patients reported few symptoms pre-diagnosis including neck lump/swelling ( = 7/14, 50%) and/or difficulty swallowing ( = 3/14, 21%). Treatments generally comprised surgery and chemotherapy and/or radiotherapy. A number of side effects from all treatments were reported. Numbness, difficulty speaking and pain were the most reported side effects of surgery ( = 4/8, 50%); weight loss and fatigue were the most reported side effects of chemotherapy and/or radiotherapy ( = 8/13, 61%). All side effects negatively impacted patients' HRQoL. Patients generally found the QLQ-C30 and QLQ H&N35 content to be understandable and conceptually relevant; excessive mucous production and neuropathic symptoms were among the suggested additions.

Conclusions: HNSCC and its diverse symptoms and treatments have a negative impact on many aspects of patients' lives. A number of reported symptoms including difficulty speaking and swallowing, localised pain and fatigue may be important for treatment benefit evaluation in clinical trials from a patient perspective. The QLQ-C30 and QLQ-H&N35 are generally relevant and suitable for use in clinical trials. However, some items could be amended/added to ensure conceptual comprehensiveness of these measures.
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August 2018

Content validity and electronic PRO (ePRO) usability of the Lung Cancer Symptom Scale-Mesothelioma (LCSS-Meso) in mesothelioma patients.

Support Care Cancer 2018 Jul 1;26(7):2229-2238. Epub 2018 Feb 1.

Evidera, 7101 Wisconsin Avenue, Suite 1400, Bethesda, MD, 20814, USA.

Purpose: Obtaining qualitative data directly from the patient perspective enhances the content validity of patient-reported outcome (PRO) instruments. The objective of this qualitative study was to evaluate the content validity of the Lung Cancer Symptom Scale for Mesothelioma (LCSS-Meso) and its usability on an electronic device.

Methods: A cross-sectional methodological study, using a qualitative approach, was conducted among patients recruited from four clinical sites. The primary target population included patients with pleural mesothelioma; data were also collected from patients with peritoneal mesothelioma on an exploratory basis. Semi-structured interviews were conducted consisting of concept elicitation, cognitive interviewing, and evaluation of electronic patient-reported outcome (ePRO) usability.

Results: Participants (n = 21) were interviewed in person (n = 9) or by telephone (n = 12); 71% were male with a mean age of 69 years (SD = 14). The most common signs and symptoms experienced by participants with pleural mesothelioma (n = 18) were shortness of breath, fluid build-up, pain, fatigue, coughing, and appetite loss. The most commonly described symptoms for those with peritoneal mesothelioma (n = 4) were bloating, changes in appetite, fatigue, fluid build-up, shortness of breath, and pain. Participants with pleural mesothelioma commonly described symptoms assessed by the LCSS-Meso in language consistent with the questionnaire and a majority understood and easily completed each of the items. The ePRO version was easy to use, and there was no evidence that the electronic formatting changed the way participants responded to the questions.

Conclusions: Results support the content validity of the LCSS-Meso and the usability of the electronic format for use in assessing symptoms among patients with pleural mesothelioma.
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July 2018

Understanding polycystic ovary syndrome from the patient perspective: a concept elicitation patient interview study.

Health Qual Life Outcomes 2017 Aug 18;15(1):162. Epub 2017 Aug 18.

Quintiles, 8 Skyline Dr, Hawthorne, New York, NY, 10532, USA.

Background: The aim of this study was to explore the need for a new disease-specific patient reported outcome (PRO) measure for use in clinical trials of drugs designed to target the underlying causes of polycystic ovary syndrome (PCOS), and in the process contribute to our understanding of the symptoms and impacts that define the patient experience with PCOS.

Methods: Semi-structured interviews were conducted in 20 women diagnosed with PCOS according to the Rotterdam criteria who had not menstruated in the previous month. The relative importance of PCOS symptoms and impact concepts to patients was determined by analyzing the frequency of their expression in the interview transcripts. These insights were compared to clinicians' perceptions of PCOS.

Results: Pain- and discomfort-related symptoms accounted for the highest proportion (27.6%) of the 735 patient expressions, although clinicians did not consider pain to be important to patients with PCOS. The most frequently expressed individual symptoms were cramping (70% of patients; 14.7% of concepts), irregular menstruation (95% of patients; 12.2% of concepts), facial hair growth (75% of patients; 10.6% of concepts), heavy bleeding (70% of patients; 8.8% of concepts), infertility (70% of patients; 5.4% of concepts), and bloating (60% of patients; 5.2% of concepts). Cramping, heavy bleeding, and bloating were not identified by clinicians as being important to patients with PCOS. The impacts most frequently reported by patients with PCOS related to emotional well-being (e.g. anxiety/stress) and coping behaviors (e.g. acne medication, hair removal).

Conclusions: The only validated PCOS-specific PRO, the PCOSQ, does not capture some key PCOS symptoms and impacts expressed by patients with PCOS, most notably those related to pain and discomfort, bleeding intensity and coping behaviours. Furthermore, some key PCOS symptoms may be under-recognized in the clinic.
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August 2017

Patient Centricity and Pharmaceutical Companies: Is It Feasible?

Ther Innov Regul Sci 2017 Jul 28;51(4):460-467. Epub 2017 Mar 28.

6 Neil Bertelsen Consulting, Berlin, Germany.

Various health care bodies (regulatory, health technology assessment, academia, health care providers, scientific journals) request patient input in their decision-making processes. This represents a shift from disease-centered to patient-centered approaches to health care. What does this "patient centricity" mean for the pharmaceutical industry? A panel of senior pharmaceutical industry representatives discussed the following key issues: why the pharmaceutical industry needs to be part of the patient-centric movement; how the industry can become patient-centric; and what a patient-centric company actually does. We summarize the panel's point of view on these key questions. The industry's role has been to develop the science and medicines for prevention or treatment of disease. In response to changes in the current health care environment, the industry should focus its efforts on initiatives that will improve impact and value for patients and carers. True patient centricity requires a change in the industry's cultural mindset, an increase in public trust, clearer roles and responsibilities within pharmaceutical organizations, openness to learn from others, and a framework to measure success. There are examples of industry engagement with patients throughout the drug discovery and development process. Patient-reported outcomes are becoming increasingly important endpoints in trials; they capture information of relevance to patients, identify preferences, and better inform treatment decision making. Understanding the patient experience can improve disease management at critical points in the disease course. The future of patient centricity lies in coordinated efforts by and alignment of multiple health care stakeholders, which can only be achieved through collaborations and consortia, with the industry playing a key role.
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July 2017

Content validity and ePRO usability of the BPI-sf and "worst pain" item with pleural and peritoneal mesothelioma.

J Patient Rep Outcomes 2017 27;2(1):16. Epub 2018 Mar 27.

Evidera - Evidence, Value & Access by PPD, 7101 Wisconsin Ave, Suite 1400, Bethesda, MD 20814 USA.

Background: The Brief Pain Inventory-short form (BPI-sf) is widely used in self-reported pain assessment, incorporates pain numeric rating scales (NRS) and is commonly utilized in electronic format in clinical trials, however, there is no published information about its usability as an electronic patient-reported outcome (ePRO) measure. The objective of this qualitative study was threefold: 1) to better understand pain experiences among patients with pleural or peritoneal mesothelioma; 2) to assess the interpretability of the instructions, item stem, recall period, and response option of the "worst pain" item of the BPI-sf; and 3) to examine the usability of the TrialMax Touch™ (CRF Health, Inc., Plymouth Meeting, PA) screen-based handheld device and the electronic format of the BPI-sf in a sub-sample of pleural mesothelioma patients.

Methods: A cross-sectional qualitative study was conducted among participants with pleural and peritoneal mesothelioma recruited from 4 clinical sites in the US. Semi-structured telephone or in-person interviews were conducted consisting of concept elicitation, cognitive interviewing of the 11-item BPI-sf, and in-person interview evaluation of ePRO assessment usability in pleural mesothelioma patients.

Results: Twenty-one participants recruited from 4 clinical sites in the US were interviewed in-person ( = 9) and by telephone ( = 12); 71% male; mean age 68.7 ± 13.6 years. Pleural and peritoneal patients described pain as ranging from discomfort to intense pain and reported being able to distinguish tumor pain from treatment pain. The BPI-sf "worst pain" item was relevant to, and easily understood by, study participants with pleural and peritoneal mesothelioma. The ePRO version was found to be easy to use, but readability of small font may be an issue. Participants reported minimal differences between their responses on the paper and ePRO version for all of the pain severity and pain interference items.

Conclusions: Results support the relevance and ease of understanding of the "worst pain" item and provide support for its content validity in patients with pleural and peritoneal mesothelioma. Usability of the ePRO format of the BPI-sf was confirmed for use in clinical trials among patients with pleural mesothelioma.
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March 2018

Comparison of Daily versus Weekly Recording of Gastroesophageal Reflux Disease Symptoms in Patients with a Partial Response to Proton Pump Inhibitor Therapy.

Value Health 2016 Sep - Oct;19(6):829-833. Epub 2016 Jun 29.

Department of Psychology and the USC Dornsife Center for Self-Report Science, University of Southern California, Los Angeles, CA, USA.

Background: The Reflux Symptom Questionnaire electronic Diary (RESQ-eD) and the Reflux Symptom Questionnaire 7-day recall (RESQ-7) are versions of a patient-reported outcome instrument that was developed and validated for measuring the frequency and intensity of symptoms in patients with gastroesophageal reflux disease (GERD) who have a partial response to proton pump inhibitor (PPI) therapy.

Objective: The aim of these analyses was to assess the ability of the RESQ-7 to reproduce findings based on RESQ-eD reports of the same symptoms.

Methods: These analyses are based on data from patients with GERD with a partial response to PPI ( identifier: NCT00703534). Participants completed the RESQ-eD twice daily for 7 days and the RESQ-7 on day 7.

Results: Data from 446 patients were available for these analyses. Symptom-level analyses showed that, for intensity, mean domain scores were higher for the RESQ-7 (range 1.49-2.72) than for the RESQ-eD (range 1.45-2.57); for frequency, scores were lower for the RESQ-7 (range 2.58-4.82) than for the RESQ-eD (range 4.22-6.24). Correspondence analyses of RESQ-7 and RESQ-eD mean domain scores indicated excellent agreement for intensity (correlation-concordance coefficient 0.77-0.83) and fair agreement for frequency (correlation-concordance coefficient 0.40-0.58). Mean RESQ-eD subscale intensity scores for GERD symptoms were higher for symptoms experienced during the daytime than for those occurring at nighttime. Symptom recall was not associated with peak or recency effects.

Conclusions: Patients with GERD slightly overestimated the intensity of their reflux symptoms and markedly underestimated the frequency on weekly recall compared with twice-daily reporting.
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May 2017

What Do Ovarian Cancer Patients Expect From Treatment?: Perspectives From an Online Patient Community.

Cancer Nurs 2017 Sep/Oct;40(5):E17-E27

Author Affiliations: PatientsLikeMe, Cambridge, Massachusetts (Mss Simacek and Raja and Dr Chiauzzi); AstraZeneca, Mölndal, Sweden (Dr Eek and Ms Halling).

Background: Advances in ovarian cancer treatment have improved outcomes. However, the gap between patients' hopes and expectations and their actual outcomes remains an understudied aspect of treatment decision making. This gap has been noted to be a predictor of poorer health-related quality of life. Incorporating patient hopes and expectations for treatment into clinical care may improve patient experiences.

Objectives: The aim of this study is to describe the hopes and expectations of ovarian cancer patients at diagnosis and throughout treatment transitions.

Methods: This study sampled from an online community of patients with ovarian cancer. Thirty members completed an online qualitative survey about their diagnostic and treatment journey, including hopes and expectations around treatment.

Results: Initially, ovarian cancer patients hoped for a complete cure or removal of cancer. As they progressed through treatment, hopes and expectations centered on issues related to living with ovarian cancer. A subset of patients emphasized a lack of information about treatment side effects.

Conclusions: The shift in expectations from survival to living with ovarian cancer may demonstrate an enhanced understanding of ovarian cancer prognosis as patients learn more about their condition. Patients underscored that a more involved discussion of side effect profiles with clinicians would enhance their treatment decision making and expectation setting process.

Implications For Practice: Expectation setting may be improved by contextualizing treatment in terms of the transitions ovarian cancer patients experience. Providing relevant information that aligns with patient needs, desires, and concerns at critical transitions may improve clinical care and decision making.
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September 2017

Understanding the Patient Perspective of Seizure Severity in Epilepsy: Development of a Conceptual Model.

Patient 2016 10;9(5):419-31

Consultant, Châtel-Censoir, France.

Purpose: For patients with uncontrolled epilepsy, the severity and postictal sequelae of seizures might be more impactful than their frequency. Seizure severity is often assessed using patient-reported outcome (PRO) instruments; however, evidence of content validity for existing instruments is lacking. Our aim was to understand the real-life experiences of patients with uncontrolled epilepsy.

Methods: A preliminary conceptual model was developed. The model was refined through (1) a targeted literature review of qualitative research on seizure severity; (2) interviews with four clinical epilepsy experts to evaluate identified concepts; and (3) qualitative interviews with patients with uncontrolled epilepsy, gathering descriptions of symptoms and impacts of epilepsy, focusing on how patients experience and describe "seizure severity." Findings were summarized in a final conceptual model of seizure severity in epilepsy.

Results: Twenty-five patients (12 who experienced primary generalized tonic-clonic seizures and 13 who experienced partial-onset seizures) expressed 42 different symptoms and 26 different impacts related to seizures. The final conceptual model contained a wide range of concepts related to seizure frequency, symptoms, and duration.

Conclusion: Our model identified several new concepts that characterize the patient experience of seizure severity. A seizure severity PRO instrument should cover a wide range of seizure symptoms alongside frequency and duration of seizures. This qualitative work reinforces the notion that measuring seizure frequency is insufficient and that seizure severity is important in defining the patient's experience of epilepsy. This model could be used to assess the content validity of existing PRO instruments, or could support the development of a new one.
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October 2016

Development and validation of the AFSymp™: an atrial fibrillation-specific measure of patient-reported symptoms.

Patient 2014 ;7(3):319-27

AstraZeneca R&D, Mölndal, Sweden.

Background: Patients with atrial fibrillation (AF) can be severely incapacitated by symptoms, but validated symptom measures are lacking. The aim of this study was to develop an AF-specific symptom questionnaire (AFSymp™).

Methods: Following a literature review, qualitative interviews with 91 patients (United States [US], n = 30; United Kingdom [UK], n = 16; France, n = 15; Germany, n = 15; Japan, n = 15) with paroxysmal, persistent, and permanent AF were conducted to identify emergent concepts and to develop the items and response options for the AFSymp™. Clinical experts (n = 21) in the US, the UK, France, Germany, and Japan provided feedback on the most clinically relevant symptoms via an email survey. Cognitive interviews with 30 patients were conducted to evaluate content validity. A prospective, observational, psychometric evaluation study (n = 313) consisting of two study visits was performed at 32 sites across the US.

Results: After item reduction, the AFSymp™ consisted of 11 items with a 1-week recall period. Exploratory and confirmatory factor analysis resulted in three subscales (heart symptoms, tiredness, chest discomfort) and two items: dizziness and shortness of breath. Internal consistency was strong across subscales (Cronbach's α 0.82-0.91). The test-retest reliability of items and subscales was acceptable (intra-class correlation [ICC] 0.58-0.78). The reproducibility of the single global score was strong (ICC 0.78). The construct and known-groups validity was acceptable.

Conclusion: The AFSymp™ demonstrates evidence of reliability and validity as a comprehensive measure of AF symptoms that can be used to assess patient outcomes in clinical and research settings. More research is needed to evaluate the instrument's responsiveness.
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October 2014

Night-time symptoms and their impact on sleep in patients with gastroesophageal reflux disease who have a partial response to proton pump inhibitors: a qualitative patient interview study.

Patient 2013 ;6(4):307-14

AstraZeneca R&D Mölndal, Pepparedsleden 1, SE-431 83, Mölndal, Sweden,

Background: Limited data exists about night-time symptoms that are generated directly from patients with gastroesophageal reflux disease (GERD) who have a partial response to proton pump inhibitor (PPI) therapy. This information is needed to select an appropriate instrument in studies in this patient population.

Objective: The objective of this qualitative interview study was to gain understanding of the night-time symptoms of patients with GERD who had a partial response to PPIs. The specific aims were (i) to evaluate whether GERD symptoms experienced during the night differ from those occurring during the day; and (ii) to understand the impact of night-time symptoms on sleep and next-day functioning.

Methods: Four US sites participated in this study of patients with GERD who, despite PPI therapy for at least 4 weeks, still experienced both daytime and night-time heartburn and/or regurgitation. Non-responders to PPIs were excluded. Patient statements were coded and grouped by concept.

Results: Twenty-nine patients were enrolled. The predominant and most troublesome symptoms during both the day and night were heartburn and regurgitation. At night-time only, expressions describing regurgitation were more frequent than those describing heartburn (62 vs. 26 %). During the daytime only, expressions describing regurgitation and heartburn occurred with similar frequency (21 vs. 27 %). Patients experienced greater severity of heartburn and regurgitation at night than during the day, and the difference was more pronounced for regurgitation. Patients focused on symptom frequency during the day but on symptom severity at night. Of expressions about the impact of night-time GERD symptoms, 46 % described impact on sleep and 41 % described compensatory behaviors when woken up by symptoms. Next-day impacts of night-time symptoms predominantly included changes in diet (53 %).

Conclusions: Partial responders to PPI therapy experience similar GERD symptoms at night and during the day. However, regurgitation is more predominant at night than during the day, and at night patients focus more on symptom severity than symptom frequency.
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April 2014

Symptom overlap between postprandial distress and epigastric pain syndromes of the Rome III dyspepsia classification.

Am J Gastroenterol 2013 May 9;108(5):767-74. Epub 2013 Apr 9.

University of Wisconsin, School of Medicine and Public Health, Madison, WI 53066, USA.

Objectives: The Rome III criteria for functional dyspepsia recognize two distinct subgroups: postprandial distress syndrome (PDS) and epigastric pain syndrome (EPS). The aim of this exploratory analysis was to evaluate the Rome III criteria and the validity of the PDS/EPS subgrouping in primary care patients with upper gastrointestinal symptoms.

Methods: Primary care patients with frequent upper gastrointestinal symptoms included in the Diamond study (NCT00291746) underwent esophageal endoscopy and 24-h pH-metry. Gastroesophageal reflux disease (GERD) was defined as the presence of at least one of the following: reflux esophagitis, pathological esophageal acid exposure, positive symptom association probability (SAP ≥95%) for association of symptoms with acid reflux. Functional dyspepsia was defined by the absence of GERD and peptic ulcer disease on investigation. PDS and/or EPS were diagnosed according to Rome III criteria.

Results: In total, 138 patients (41%) had upper gastrointestinal symptoms with normal endoscopy, pH-metry, and SAP results, consistent with the presence of functional dyspepsia. Of these patients, 130 (94%) met criteria for PDS and/or EPS: 13 (10%) had PDS alone, 31 (24%) had EPS alone, and 86 (66%) met criteria for both PDS and EPS.

Conclusions: PDS and EPS overlap in the majority of patients with functional dyspepsia. The value of dividing functional dyspepsia into the subgroups of PDS and EPS is thus questionable. A new approach to classifying functional dyspepsia is needed.
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May 2013

Validation of the reflux symptom questionnaire electronic diary in partial responders to proton pump inhibitor therapy.

Clin Transl Gastroenterol 2012 Jan 26;3:e7. Epub 2012 Jan 26.

University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.

Objectives: We aimed to develop and validate the Reflux Symptom Questionnaire electronic Diary (RESQ-eD) for use in clinical trials in patients with a partial response to proton pump inhibitor (PPI) therapy, using methods that meet US Food & Drug Administration (FDA) regulatory standards.

Methods: Patient interviews were performed to elicit new items and evaluate existing items from the Reflux Disease Questionnaire. The instrument's measurement properties were evaluated, based on data from two clinical trials of patients with gastroesophageal reflux disease (GERD) with a partial response to PPIs who received lesogaberan or placebo as an add-on to PPI therapy.

Results: The content validity phase resulted in 13 RESQ-eD items. Principal component analysis supported a four-domain structure. All domains had a high inter-item correlation (Cronbach's alpha lower 95% confidence limit: 0.87-0.95). Test-retest reliability was good to excellent (intraclass correlation coefficient: 0.65-0.85). Convergent and discriminant validity was confirmed by correlation assessments referencing the Gastrointestinal Symptom Rating Scale. The RESQ-eD demonstrated a good ability to capture change in mean intensity and proportion of symptom-free days. Confirmatory psychometric evaluation verified internal consistency reliability, test-retest reliability, and ability to capture change.

Conclusions: The RESQ-eD demonstrated good content validity and psychometric properties in the clinical trial setting in patients with GERD who have a partial response to PPI therapy. To our knowledge, the RESQ-eD is the first electronic symptom diary for use in partial responders to PPI that has been developed in line with the FDA guidance on patient-reported outcomes.
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January 2012

Systematic review of patient-reported outcome instruments for gastroesophageal reflux disease symptoms.

Eur J Gastroenterol Hepatol 2013 Jan;25(1):2-14

Department of Gastroenterology, Aurora Summit Hospital, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53066, USA.

Gastroesophageal reflux disease (GERD) symptoms are best assessed using patient-reported outcome (PRO) instruments. Guidance on developing well-defined and reliable instruments that capture optimal information from the patient's perspective was recently published by the US Food and Drug Administration and the European Medicines Agency. The aim of this systematic review was to identify and evaluate existing PRO instruments for GERD symptoms with regard to regulatory requirements. Systematic literature searches were conducted in PubMed and Embase to identify PRO instruments for GERD symptoms that have undergone psychometric evaluation. Content, construct and test-retest reliability, internal consistency, and responsiveness were evaluated in relation to regulatory recommendations. Supplementary searches were conducted to assess whether identified instruments had been used as clinical trial endpoint measures. The systematic literature searches identified 15 PRO instruments for GERD symptoms that have undergone psychometric evaluation. Eight were designed to evaluate GERD symptoms, two were to diagnose GERD, four were designed for both evaluative and diagnostic purposes, and one was designed for screening purposes. Five instruments were developed and reported to include most steps recommended by the Food and Drug Administration and European Medicines Agency, and have also been used as endpoint measures in clinical trials: the GERD Symptom Assessment Scale, the Nocturnal Gastro-oesophageal Reflux Disease Symptom Severity and Impact Questionnaire, the Reflux Questionnaire, the Reflux Disease Questionnaire, and the Proton pump inhibitor Acid Suppression Symptom test. Existing PRO instruments for GERD do not meet all the regulatory requirements for an outcome instrument in reflux trials and may need further validation.
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January 2013

Perspectives on gastroesophageal reflux disease in primary care: the REFLEX study of patient-physician agreement.

BMC Gastroenterol 2011 Mar 24;11:25. Epub 2011 Mar 24.

Gastroenterology, Trousseau Hospital, Tours, France.

Background: Physicians may be unaware of the severity and extent of gastroesophageal reflux disease (GERD) in their patients. The aim of this study was to evaluate patient-physician agreement concerning proton pump inhibitor (PPI) treatment.

Methods: 1818 French primary-care physicians and 5174 adult patients with GERD who were taking PPIs answered questions regarding symptoms and treatment satisfaction. Patient-physician agreement was scored using the Kappa (κ) method.

Results: There was moderate patient-physician agreement for PPI treatment satisfaction (κ = 0.60), PPI prescription adherence (κ = 0.57) and use of over-the-counter gastrointestinal medications (κ = 0.44-0.51). Patient satisfaction with PPI therapy and PPI treatment adherence rates were both ~90%. There was poor patient-physician agreement concerning PPI therapy expectations (κ = 0.22-0.33). Residual reflux symptoms occurred in 61% of patients. Physicians underestimated residual symptom severity compared with their patients (κ = 0.43-0.47), though there was good agreement regarding the presence (κ = 0.62-0.78) and frequency (κ = 0.61-0.66) of these symptoms and their effect on patients' daily life (κ = 0.64).

Conclusions: Patient-physician agreement regarding PPI therapy for GERD was moderate or good for the presence of residual symptoms and moderate for treatment satisfaction, but poor for treatment expectations. PPI treatment resulted in high satisfaction rates, but residual symptoms were fairly common and their severity was underestimated by physicians.
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March 2011

Impact of gastroesophageal reflux disease on daily life: the Systematic Investigation of Gastrointestinal Diseases in China (SILC) epidemiological study.

Health Qual Life Outcomes 2010 Nov 10;8:128. Epub 2010 Nov 10.

Second Military Medical University, Shanghai, China.

Background: Gastroesophageal reflux disease imposes a significant burden of illness in Western populations. However, data on the impact of reflux symptoms on daily life in Asian populations are scarce. The current study aimed to evaluate the impact of GERD (defined on the basis of symptoms) on health-related quality-of-life (HRQoL) in individuals from five regions in China, as part of the Systematic Investigation of Gastrointestinal Diseases in China (SILC) study.

Methods: In total, 18 000 residents were randomly selected from across five regions of China and asked to complete a general information questionnaire and a Chinese version of the Reflux Disease Questionnaire (RDQ). A randomly selected subsample of one-fifth of subjects (20% from each region) completed Chinese versions of the 36-item self-administered (SF-36) questionnaire and Epworth Sleepiness Scale (ESS) questionnaire. Reflux symptoms were defined as the presence of heartburn and/or regurgitation. Symptom-defined GERD was diagnosed as mild heartburn and/or regurgitation ≥2 days per week, or moderate/severe heartburn and/or regurgitation ≥1 day a week, based on the Montreal Definition of GERD for population-based studies.

Results: The response rate was 89.4% for the total sample (16 091/18 000), and for the 20% subsample (3219/3600). Meaningful impairment was observed in all 8 SF-36 dimensions in participants with symptom-defined GERD, in 7 of the 8 SF-36 dimensions in participants with troublesome reflux symptoms, and in 6 of the 8 SF-36 dimensions in participants with reflux symptoms below the threshold for symptom-defined GERD. Meaningful daytime sleepiness was also observed in each of these groups. The proportion of individuals reporting troublesome symptoms increased as reflux symptom frequency and severity approached the threshold for symptom-defined GERD, and this was associated with concomitant decreases in all HRQoL measures. Troublesome symptoms were reported by 68.2% (75/110) of individuals with symptom-defined GERD.

Conclusions: GERD diagnosed using symptom/frequency criteria (recommended for population-based studies), or based on troublesome reflux symptoms (recommended for the clinic), is associated with significantly impaired HRQoL in Chinese individuals. However, patient groups identified using these definitions do not overlap completely, suggesting that they capture slightly different, though clinically relevant, GERD populations.
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November 2010

Psychometric validation of the Dutch translation of the quality of life in reflux and dyspepsia (QOLRAD) questionnaire in patients with gastroesophageal reflux disease.

Health Qual Life Outcomes 2010 Aug 17;8:85. Epub 2010 Aug 17.

Department of Gastroenterology, Maasland Hospital, Sittard, the Netherlands.

Background: The Quality of Life in Reflux and Dyspepsia (QOLRAD) questionnaire is one of the best-characterized disease-specific instruments that captures health-related problems and symptom-patterns in patients with gastroesophageal reflux disease (GERD). This paper reports the psychometric validation of a Dutch translation of the QOLRAD questionnaire in gastroenterology outpatients with GERD.

Methods: Patients completed the QOLRAD questionnaire at visit 1 (baseline), visit 2 (after 2, 4 or 8 weeks of acute treatment with esomeprazole 40 mg once daily), and visit 4 (after 6 months with on-demand esomeprazole 40 mg once daily or continuous esomeprazole 20 mg once daily). Symptoms were assessed at each visit, and patient satisfaction was assessed at visits 2 and 4.

Results: Of the 1166 patients entered in the study, 97.3% had moderate or severe heartburn and 55.5% had moderate or severe regurgitation at baseline. At visit 2, symptoms of heartburn and regurgitation were mild or absent in 96.7% and 97.7%, respectively, and 95.3% of patients reported being satisfied with the treatment. The internal consistency and reliability of the QOLRAD questionnaire (range: 0.83-0.92) supported construct validity. Convergent validity was moderate to low. Known-groups validity was confirmed by a negative correlation between the QOLRAD score and clinician-assessed severity of GERD symptoms. Effect sizes (1.15-1.93) and standardized response means (1.17-1.86) showed good responsiveness to change. GERD symptoms had a negative impact on patients' lives.

Conclusions: The psychometric characteristics of the Dutch translation of the QOLRAD questionnaire were found to be satisfactory, with good reliability and responsiveness to change, although convergent validity was at best moderate.
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August 2010

Accuracy of the diagnosis of GORD by questionnaire, physicians and a trial of proton pump inhibitor treatment: the Diamond Study.

Gut 2010 Jun;59(6):714-21

Royal Adelaide Hospital, Adelaide, SA 5000, Australia.

Objective: The aim of this study was to determine the accuracy of the diagnosis of gastro-oesophageal reflux disease (GORD) by the Reflux Disease Questionnaire (RDQ), family practitioners, gastroenterologists and a test of esomeprazole therapy.

Methods: This was a single-blind, single-arm study over 3-4 weeks from September 2005 to November 2006. Each symptom-based diagnostic assessment was made blinded to prior diagnoses. Patients were those presenting to their family practitioner with troublesome upper gastrointestinal symptoms (n=308). The RDQ was completed and a symptom-based diagnosis was made by the family practitioner. Placebo esomeprazole was started. Gastroenterologists made a symptom-based diagnosis and then performed endoscopy with 48 h oesophageal pH and symptom association monitoring to determine the presence/absence of GORD. Symptoms were recorded during treatment with 40 mg of esomeprazole for 2 weeks. The main outcome measure was RDQ scoring for the presence of GORD compared with symptom-based diagnosis by family physicians and gastroenterologists.

Results: GORD was present in 203/308 (66%) patients. Only 49% of the patients with GORD selected either heartburn or regurgitation as the most troublesome symptom. Sensitivity and specificity, respectively, of the symptom-based diagnosis of GORD, were 62% and 67% for the RDQ, 63% and 63% for family practitioners, and 67% and 70% for gastroenterologists. Symptom response to esomeprazole was neither sensitive nor specific for the diagnosis of GORD.

Conclusions: The RDQ, family practitioners and gastroenterologists have moderate and similar accuracy for diagnosis of GORD. Symptom response to a 2 week course of 40 mg of esomeprazole does not add diagnostic precision.
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June 2010

Systematic investigation of gastrointestinal diseases in China (SILC): validation of survey methodology.

BMC Gastroenterol 2009 Nov 19;9:86. Epub 2009 Nov 19.

Department of Health Statistics, Second Military Medical University, Shanghai, China.

Background: Symptom-based surveys suggest that the prevalence of gastrointestinal diseases is lower in China than in Western countries. The aim of this study was to validate a methodology for the epidemiological investigation of gastrointestinal symptoms and endoscopic findings in China.

Methods: A randomized, stratified, multi-stage sampling methodology was used to select 18,000 adults aged 18-80 years from Shanghai, Beijing, Xi'an, Wuhan and Guangzhou. Participants from Shanghai were invited to provide blood samples and undergo upper gastrointestinal endoscopy. All participants completed Chinese versions of the Reflux Disease Questionnaire (RDQ) and the modified Rome II questionnaire; 20% were also invited to complete the 36-item Short Form Health Survey (SF-36) and Epworth Sleepiness Scale (ESS). The psychometric properties of the questionnaires were evaluated statistically.

Results: The study was completed by 16,091 individuals (response rate: 89.4%), with 3219 (89.4% of those invited) completing the SF-36 and ESS. All 3153 participants in Shanghai provided blood samples and 1030 (32.7%) underwent endoscopy. Cronbach's alpha coefficients were 0.89, 0.89, 0.80 and 0.91, respectively, for the RDQ, modified Rome II questionnaire, ESS and SF-36, supporting internal consistency. Factor analysis supported construct validity of all questionnaire dimensions except SF-36 psychosocial dimensions.

Conclusion: This population-based study has great potential to characterize the relationship between gastrointestinal symptoms and endoscopic findings in China.
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November 2009

Upper Gastrointestinal Symptoms Experienced by Users of Low-Dose Aspirin (Acetylsalicylic Acid) [75-325 mg/day] for Primary and Secondary Coronary Artery Disease Prevention: Perspectives from Patient Focus Groups.

Patient 2009 Jun;2(2):89-93

1 United BioSource Corporation, Center for Health Outcomes Research, Bethesda, Maryland, USA 2 AstraZeneca R&D, Mölndal, Sweden.

Background And Objective: : As upper gastrointestinal (GI) symptoms are common with the use of low-dose aspirin (low-dose acetylsalicylic acid [LDASA]; 75-325 mg/day), this exploratory qualitative study evaluated the upper GI symptom experience and attribution of symptoms among patients taking LDASA for coronary artery disease (CAD) or known CAD risk factors.

Methods: : Focus groups were conducted among patients aged ≥40 years with CAD or known CAD risk factors currently taking daily LDASA. Patients were recruited from primary-care clinical sites, and all had experienced upper GI symptoms the week before inclusion (including heartburn, acid reflux, and stomach or abdominal pain). The focus group discussions were designed to explore the participants' experience with upper GI symptoms, LDASA use, potential adverse effects of treatment, and physician interactions. Content analysis and descriptive statistics were used to analyze the data.

Results: : Thirty-three men and women participated in four focus group sessions in France and in the US. All participants recognized the cardioprotective benefits of LDASA and reported a high level of compliance with therapy. Although participants regarded LDASA as a necessary and valuable treatment, many participants had concerns about LDASA use, primarily because of the bleeding risk. Many participants were aware that LDASA may cause GI symptoms. Participants experienced a range of upper GI symptoms, including heartburn, regurgitation, and nausea. Almost half of the participants believed that their GI symptoms were solely due to lifestyle issues such as stress and eating spicy food rather than being caused by medication, where others reported that they were directly related to LDASA use. The GI symptoms experienced by LDASA users were cited as troublesome, causing the participants to change eating habits, avoid stress or employ stress-reduction techniques, change physical activities, and take more medication to treat the symptoms.

Conclusion: : Participants were well aware of the potential adverse effects of LDASA use and reported that GI symptoms had a high impact on several areas of their lives. To maintain the cardioprotective benefits of LDASA, participants used several strategies to deal with their upper GI symptoms, including changing their eating habits, avoiding stress, changing their physical activities, and taking medication.
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June 2009

An international comparison of the burden of illness in patients with dyspepsia.

Dig Dis 2008 7;26(3):264-73. Epub 2008 May 7.

AstraZeneca R&D Molndal, Molndal, Sweden.

Background/aims: This study investigates the symptoms and the impact of symptoms on health-related quality of life (HRQL) in patients consulting with dyspepsia.

Methods: Consecutive patients with a diagnosis of dyspepsia were recruited from primary and secondary care in Germany, Hungary, Italy, Poland, South Africa and Spain. Investigators assessed symptom frequency and severity, and subjects completed the following questionnaires: the Gastrointestinal Symptom Rating Scale (GSRS), the Quality of Life in Reflux and Dyspepsia (QOLRAD) questionnaire, the Hospital Anxiety and Depression (HAD) scale and the Short Form 36 (SF-36).

Results: 853 dyspepsia patients were included. Mean GSRS scores showed that patients were most troubled by abdominal pain and indigestion. QOLRAD scores indicated that symptoms caused emotional distress, food/drink problems and reduced vitality, with a lesser effect on sleep and physical functioning. Mean SF-36 scores were lower than mean normative values for all domains, indicating that patients had a worse HRQL than the normal population, particularly for Bodily Pain, Role Physical and Role Emotional. Of patients in each country, 18-43% were anxious and 11-21% were depressed.

Conclusions: Patients with dyspepsia have reduced HRQL because their symptoms - particularly abdominal pain and indigestion - cause emotional distress, problems with food and drink, and impaired vitality.
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July 2008

Reliability and validity of the Gastrointestinal Symptom Rating Scale (GSRS) and Quality of Life in Reflux and Dyspepsia (QOLRAD) questionnaire in dyspepsia: a six-country study.

Health Qual Life Outcomes 2008 Jan 31;6:12. Epub 2008 Jan 31.

AstraZeneca R&D, Medical Science, Mölndal, S-431 86, Sweden.

Background: Symptoms of dyspepsia significantly disrupt patients' lives and reliable methods of assessing symptom status are important for patient management. The aim of the current study was to document the psychometric characteristics of the Gastrointestinal Symptom Rating Scale (GSRS) and the Quality of Life in Reflux and Dyspepsia questionnaire (QOLRAD) in Afrikaans, German, Hungarian, Italian, Polish and Spanish patients with dyspepsia.

Methods: 853 patients with symptoms of dyspepsia completed the GSRS, the QOLRAD, the 36-item Short-Form Health Survey (SF-36) and the Hospital Anxiety and Depression scale.

Results: The internal consistency reliability of the GSRS was 0.43-0.87 and of the QOLRAD 0.79-0.95. Test-retest reliability of the GSRS was 0.36-0.75 and of the QOLRAD 0.41-0.82. GSRS Abdominal pain domain correlated significantly with all QOLRAD domains in most language versions, and with SF-36 Bodily pain in all versions. QOLRAD domains correlated significantly with the majority of SF-36 domains in most versions. Both questionnaires were able to differentiate between patients whose health status differed according to symptom frequency and severity.

Conclusion: The psychometric characteristics of the different language versions of the GSRS and QOLRAD were found to be good, with acceptable reliability and validity. The GSRS and QOLRAD were found to be useful for evaluating dyspeptic symptoms and their impact on patients' daily lives in multinational clinical trials.
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January 2008

Responsiveness of measures of heartburn improvement in non-erosive reflux disease.

Health Qual Life Outcomes 2007 Jun 11;5:32. Epub 2007 Jun 11.

Biostatistics, AstraZeneca R&D, Mölndal, Sweden.

Background: When measuring treatment effect on symptoms, the treatment success variable should be as responsive as possible. The aim of the study was to investigate the responsiveness of various treatment success variables in patients with symptoms of heartburn.

Methods: A total of 1640 patients with non-erosive reflux disease (NERD) were treated with proton pump inhibitors for 4 weeks. Treatment success variables were based on a symptom questionnaire (Gastrointestinal Symptom Rating Scale) and on investigator-assessed heartburn, measured at baseline and after 4 weeks of treatment. The rates of treatment success were compared with patients' perceived change in symptoms, assessed by the Overall Treatment Effect questionnaire.

Results: Generally, more stringent treatment success criteria (i.e., those demanding the better response) translated into more responsive treatment success variables. For example, the treatment success variable 'no heartburn' at 4 weeks was more responsive than the variable 'at most mild heartburn' at 4 weeks. Treatment success variables based on change from baseline to 4 weeks were, in general, less responsive than those based on the week 4 assessments only.

Conclusion: In patients with NERD, responsiveness varied among different treatment success definitions, with more demanding definitions (based on the 4-week assessment) giving better responsiveness.
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June 2007

Characterization of health-related quality of life in heart failure patients with preserved versus low ejection fraction in CHARM.

Eur J Heart Fail 2007 Jan 21;9(1):83-91. Epub 2006 Dec 21.

Cardiovascular Division, Department of Medicine, Brigham and Women's Hospital, 75 Francis Street, Boston, MA 02115, USA.

Background: Limited comparative studies assessing the health-related quality of life (HRQL) in heart failure (HF) patients with preserved vs. low ejection fraction (LVEF) have been disparate.

Aims: The aims of this study were a) to characterize HRQL in a large population of HF patients with preserved and low LVEF and b) to determine the factors associated with worse HRQL.

Methods: Patients with symptomatic HF (NYHA Class II-IV) enrolled in the Candesartan in Heart Failure: Assessment of Reduction in Mortality and Morbidity (CHARM) HRQL study completed the Minnesota Living with Heart Failure questionnaire at randomization. Patients were stratified into 2 HF cohorts: preserved LVEF (>40%) and low LVEF (
Results: In 2709 of the eligible 2744 (98.6%) patients, the summary scores ranged from 0 to 105 (mean 40.9). There were no differences in overall responses of HF patients with preserved vs. low LVEF (41.1 vs. 40.8). Independent factors associated with worse HRQL in both populations included female gender, younger age, higher body mass index, lower systolic blood pressure, greater symptom burden, and worse functional status.

Conclusions: In symptomatic HF patients, HRQL is equally impaired in both preserved and low LVEF populations. Targeting improvement in symptoms and HRQL is an important treatment objective in all HF patients.
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January 2007