Validity of the Patient Experiences and Satisfaction with Medications (PESaM) Questionnaire

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Abstract

Background

This study assessed the validity and reliability of the generic module of the recently developed Patient Experiences and Satisfaction with Medications (PESaM) questionnaire in a sample of patients in the Netherlands.

Methods

The generic module of the PESaM questionnaire consists of 18 items related to the domains effectiveness, side effects and ease of use of medications. It assesses patients’ experiences regarding the impact of the medication on daily life, health and satisfaction. In 2017, the PESaM questionnaire was sent out to idiopathic pulmonary fibrosis patients using pirfenidone or nintedanib, atypical haemolytic uraemic syndrome patients receiving eculizumab and patients using tacrolimus after kidney transplantation. Mean scores for each domain were calculated applying a scoring algorithm. Construct validity and reliability were assessed using recommended methods.

Results

188 participants completed the generic module, of whom 48% used pirfenidone, 36% nintedanib, 11% tacrolimus and 5% eculizumab. The generic module has good structural properties. Internal consistency values of the domains were satisfactory (i.e. Cronbach’s coefficient alpha above 0.7). Confirmatory factor analysis provided further evidence for construct validity, with good convergent and discriminant validity. The PESaM questionnaire also showed different scores for patients using different medications, in line with expectations, and was therefore able to differentiate between patient groups. Test–retest reliability of the items and domains were rated as moderate to fair (i.e. intraclass coefficients ranged between 0.18 and 0.76).

Conclusions

The PESaM questionnaire is a unique patient-reported outcome measure evaluating patient experiences and satisfaction with medications. It has been developed in conjunction with patients, ensuring coverage of domains and issues relevant from the patient’s perspective. This study has shown promising validity of the generic module of the PESaM questionnaire. Further research is recommended to assess reliability in greater detail as well as the responsiveness of the measure.

Trial registration

The study is registered in The Netherlands National Trial Register (Trial Code 5860).

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Most cited references 26

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Individual-patient monitoring in clinical practice: are available health status surveys adequate?

C A McHorney, A. Tarlov (1995)

Interest has increased in recent years in incorporating health status measures into clinical practice for use at the individual-patient level. We propose six measurement standards for individual-patient applications: (1) practical features, (2) breadth of health measured, (3) depth of health measured, (4) precision for cross-sectional assessment, (5) precision for longitudinal monitoring and (6) validity. We evaluate five health status surveys (Functional Status Questionnaire, Dartmouth COOP Poster Charts, Nottingham Health Profile, Duke Health Profile, and SF-36 Health Survey) that have been proposed for use in clinical practice. We conducted an analytical literature review to evaluate the six measurement standards for individual-patient applications across the five surveys. The most problematic feature of the five surveys was their lack of precision for individual-patient applications. Across all scales, reliability standards for individual assessment and monitoring were not satisfied, and the 95% CIs were very wide. There was little evidence of the validity of the five surveys for screening, diagnosing, or monitoring individual patients. The health status surveys examined in this paper may not be suitable for monitoring the health and treatment status of individual patients. Clinical usefulness of existing measures might be demonstrated as clinical experience is broadened. At this time, however, it seems that new instruments, or adaptation of existing measures and scaling methods, are needed for individual-patient assessment and monitoring.

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Factors affecting therapeutic compliance: A review from the patient’s perspective

Jing Jin, Grant Sklar, Vernon Min Sen Oh … (2008)

Objective To explore and evaluate the most common factors causing therapeutic non-compliance. Methods A qualitative review was undertaken by a literature search of the Medline database from 1970 to 2005 to identify studies evaluating the factors contributing to therapeutic non-compliance. Results A total of 102 articles was retrieved and used in the review from the 2095 articles identified by the literature review process. From the literature review, it would appear that the definition of therapeutic compliance is adequately resolved. The preliminary evaluation revealed a number of factors that contributed to therapeutic non-compliance. These factors could be categorized to patient-centered factors, therapy-related factors, social and economic factors, healthcare system factors, and disease factors. For some of these factors, the impact on compliance was not unequivocal, but for other factors, the impact was inconsistent and contradictory. Conclusion There are numerous studies on therapeutic noncompliance over the years. The factors related to compliance may be better categorized as “soft” and “hard” factors as the approach in countering their effects may differ. The review also highlights that the interaction of the various factors has not been studied systematically. Future studies need to address this interaction issue, as this may be crucial to reducing the level of non-compliance in general, and to enhancing the possibility of achieving the desired healthcare outcomes.

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Atypical hemolytic uremic syndrome

Chantal Loirat, Veronique Fremeaux-Bacchi (2011)

Hemolytic uremic syndrome (HUS) is defined by the triad of mechanical hemolytic anemia, thrombocytopenia and renal impairment. Atypical HUS (aHUS) defines non Shiga-toxin-HUS and even if some authors include secondary aHUS due to Streptococcus pneumoniae or other causes, aHUS designates a primary disease due to a disorder in complement alternative pathway regulation. Atypical HUS represents 5 -10% of HUS in children, but the majority of HUS in adults. The incidence of complement-aHUS is not known precisely. However, more than 1000 aHUS patients investigated for complement abnormalities have been reported. Onset is from the neonatal period to the adult age. Most patients present with hemolytic anemia, thrombocytopenia and renal failure and 20% have extra renal manifestations. Two to 10% die and one third progress to end-stage renal failure at first episode. Half of patients have relapses. Mutations in the genes encoding complement regulatory proteins factor H, membrane cofactor protein (MCP), factor I or thrombomodulin have been demonstrated in 20-30%, 5-15%, 4-10% and 3-5% of patients respectively, and mutations in the genes of C3 convertase proteins, C3 and factor B, in 2-10% and 1-4%. In addition, 6-10% of patients have anti-factor H antibodies. Diagnosis of aHUS relies on 1) No associated disease 2) No criteria for Shigatoxin-HUS (stool culture and PCR for Shiga-toxins; serology for anti-lipopolysaccharides antibodies) 3) No criteria for thrombotic thrombocytopenic purpura (serum ADAMTS 13 activity > 10%). Investigation of the complement system is required (C3, C4, factor H and factor I plasma concentration, MCP expression on leukocytes and anti-factor H antibodies; genetic screening to identify risk factors). The disease is familial in approximately 20% of pedigrees, with an autosomal recessive or dominant mode of transmission. As penetrance of the disease is 50%, genetic counseling is difficult. Plasmatherapy has been first line treatment until presently, without unquestionable demonstration of efficiency. There is a high risk of post-transplant recurrence, except in MCP-HUS. Case reports and two phase II trials show an impressive efficacy of the complement C5 blocker eculizumab, suggesting it will be the next standard of care. Except for patients treated by intensive plasmatherapy or eculizumab, the worst prognosis is in factor H-HUS, as mortality can reach 20% and 50% of survivors do not recover renal function. Half of factor I-HUS progress to end-stage renal failure. Conversely, most patients with MCP-HUS have preserved renal function. Anti-factor H antibodies-HUS has favourable outcome if treated early.

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Author and article information

Contributors

Merel L. Kimman: +31 43 3874483 , merel.kimman@mumc.nl

Marlies S. Wijsenbeek: m.wijsenbeek-lourens@erasmusmc.nl

Sander M. J. van Kuijk: sander.van.kuijk@mumc.nl

Kioa L. Wijnsma: Kioa.Wijnsma@radboudumc.nl

Nicole C. A. J. van de Kar: Nicole.vandeKar@radboudumc.nl

Marjolein Storm: storm@nvn.nl

Xana van Jaarsveld: XanavanJaarsveld@longfonds.nl

Carmen D. Dirksen: c.dirksen@mumc.nl

Journal

Journal ID (nlm-ta): Patient

Journal ID (iso-abbrev): Patient

Title: The Patient

Publisher: Springer International Publishing (Cham )

ISSN (Print): 1178-1653

ISSN (Electronic): 1178-1661

Publication date (Electronic): 26 October 2018

Publication date PMC-release: 26 October 2018

Publication date (Print): 2019

Volume: 12

Issue: 1

Pages: 149-162

Affiliations

[1 ]ISNI 0000 0004 0480 1382, GRID grid.412966.e, Department of Clinical Epidemiology and Medical Technology Assessment, , Maastricht University Medical Centre, ; Maastricht, The Netherlands

[2 ]ISNI 000000040459992X, GRID grid.5645.2, Department of Respiratory Medicine, Erasmus Medical Centre, , University Hospital Rotterdam, ; Rotterdam, The Netherlands

[3 ]GRID grid.461578.9, Department of Pediatric Nephrology, Radboud University Medical Center, Radboud Institute for Health Sciences, , Amalia Children’s Hospital, ; Nijmegen, The Netherlands

[4 ]Kidney Patients Association, Bussum, The Netherlands

[5 ]GRID grid.483832.6, Lung Foundation Netherlands, ; Amersfoort, The Netherlands

[6 ]ISNI 0000 0004 0480 1382, GRID grid.412966.e, Department of Clinical Epidemiology and Medical Technology Assessment, Care and Public Health Research Institute (CAPHRI), , Maastricht University Medical Centre, ; Maastricht, The Netherlands

Article

Publisher ID: 340

DOI: 10.1007/s40271-018-0340-6

PMC ID: 6335379

PubMed ID: 30367435

SO-VID: 9b5683bd-442d-4d96-b25a-d030ea4a2762

License:

Open AccessThis article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/), which permits any noncommercial use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.

History

Funding

Funded by: FundRef http://dx.doi.org/10.13039/501100001826, ZonMw;

Award ID: 15200095001

Award Recipient : Merel L. Kimman

Funded by: The Federation of Patients and Consumer Organisations in the Netherlands (NPCF)

Funded by: Lung Foundation Netherlands

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Validity of the Patient Experiences and Satisfaction with Medications (PESaM) Questionnaire

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Abstract

Background

Methods

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Conclusions

Trial registration

Related collections

AboutScience

Most cited references 26

Individual-patient monitoring in clinical practice: are available health status surveys adequate?

Factors affecting therapeutic compliance: A review from the patient’s perspective

Atypical hemolytic uremic syndrome

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Cited by 7

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