Psychometric validation of the dysmenorrhea daily diary (DysDD): a patient-reported outcome for dysmenorrhea

2017 ◽  
Vol 26 (8) ◽  
pp. 2041-2055 ◽  
Author(s):  
Allison M. Nguyen ◽  
Rob Arbuckle ◽  
Tjeerd Korver ◽  
Fang Chen ◽  
Beverley Taylor ◽  
...  
Keyword(s):  
Daily Diary ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Patient Reported

scholarly journals The endometriosis daily diary: qualitative research to explore the patient experience of endometriosis and inform the development of a patient-reported outcome (PRO) for endometriosis-related pain

2022 ◽  
Vol 6 (1) ◽  
Author(s):  
Yanfen Guan ◽  
Allison M. Nguyen ◽  
Samantha Wratten ◽  
Sharan Randhawa ◽  
Jessica Weaver ◽  
...  
Keyword(s):  
Qualitative Research ◽  
Conceptual Model ◽  
Daily Diary ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Handheld Device ◽  
Concept Elicitation ◽  
Chronic Disorder ◽  
Patient Reported ◽  
Valid Instrument

Abstract Purpose Endometriosis is a chronic disorder of the female reproductive system characterized by debilitating symptoms, particularly endometriosis-related pain (ERP). Patient-reported outcome (PRO) measures of symptoms and impacts are required to assess disease severity in ERP clinical studies and clinical practice. A content-valid instrument was developed by modifying the Dysmenorrhea Daily Diary (DysDD) to form the Endometriosis Daily Diary (EDD), an electronic PRO administered via handheld device. Methods Qualitative research with US females with ERP was conducted in three stages: (1) Development of an endometriosis conceptual model based on qualitative literature and conduct of concept elicitation (CE) interviews (N = 30). (2) Cognitive debriefing (CD) interviews (N = 30) conducted across two rounds to assess relevance and understanding of the EDD, with modifications between interview rounds. (3) Pilot testing to assess usability/feasibility of administrating the EDD daily on an electronic handheld device (N = 15). Clinical experts provided guidance throughout the study. Results The conceptual model provided a comprehensive summary of endometriosis to inform modifications to the DysDD, forming the EDD. CD results demonstrated that EDD items were relevant for most participants. Instructions, items, response scales, and recall period were well-understood. The resulting daily diary assesses severity of cyclic and non-cyclic pelvic pain, dyspareunia, impact of ERP on functioning and daily life, symptoms associated with ERP, and bowel symptoms. Participants were able to complete the diary daily and found the device easy to use. Conclusion The EDD demonstrated good content validity in females experiencing ERP. The next step is to perform psychometric validation in an ERP sample.

Confirmatory Psychometric Evaluation of the Axillary Sweating Daily Diary: A Vaildated Patient-Reported Outcome Measure to Assess Axillary Hyperhidrosis Sweating Severity

2017 ◽  
Vol 1 ◽  
pp. s94 ◽  
Author(s):  
Dee Anna Glaser ◽  
Adelaide A Hebert ◽  
Sheri Fehnel ◽  
Dana DiBenedetti ◽  
Lauren Nelson ◽  
...  
Keyword(s):  
Outcome Measure ◽  
Daily Diary ◽  
Psychometric Evaluation ◽  
Patient Reported Outcome ◽  
Axillary Hyperhidrosis ◽  
Patient Reported Outcome Measure ◽  
Patient Reported

Abstract Not AvailableDisclosure: Study supported by Dermira.

scholarly journals Development of a new patient-reported outcome (PRO) measure on the Impact of Nighttime Urination (INTU) in patients with nocturia-Psychometric validation

2018 ◽  
Vol 37 (5) ◽  
pp. 1678-1685 ◽  
Author(s):  
Jason B. Bennett ◽  
Kristin Khalaf Gillard ◽  
Benjamin Banderas ◽  
Steven Abrams ◽  
Linda Cheng ◽  
...  
Keyword(s):  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Patient Reported ◽  
The Impact

scholarly journals Measuring quality of life and patient satisfaction in hand conditions

2018 ◽  
Vol 1 (2) ◽  
pp. 85-99 ◽  
Author(s):  
Kyra L Sierakowski ◽  
Kathleen A Evans Sanchez ◽  
Rachael A Damarell ◽  
Nicola R Dean ◽  
Philip A Griffin ◽  
...  
Keyword(s):  
International Development ◽  
English Language ◽  
A Priori ◽  
Hand Surgery ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Surgical Interventions ◽  
Many Instruments ◽  
Patient Reported

Background: Patient reported outcomes (PROs) are important for the assessment of the effectiveness of surgical interventions. If patient reported outcome measures (PROMs) are used to prioritise resources then it is important to ensure that the instruments are scientifically valid. This review aims to assess whether the currently available PROMs in hand surgery adhere to international development guidelines and whether they incorporate the use of item response theory (IRT) or Rasch Analysis (RA). Methods: A systematic review was performed to identify all PROMs that are relevant to the field of hand surgery. An a priori protocol with strict inclusion and exclusion criteria was followed. Only instruments developed in the English language were included. A comprehensive search of nine databases was undertaken. The development methodology of the identified instruments was then analysed, followed by examination of the domain content and initial psychometric validation of each instrument. Results: A total of 3,039 article citations were retrieved, 139 citations went on to a full text review. A total of 24 patient reported outcome instruments were identified. This consisted of 10 regional upper limb, six hand and/or wrist specific and a further eight condition specific instruments. Documentation of the details of PROM development was lacking for many instruments. Conclusion: The field of hand surgery has many instruments available but few fulfil international development guidelines or use IRT or RA psychometric techniques. There are limitations in either the breadth of the domains explored or the developmental methodology used in all currently available instruments.

scholarly journals Development of Patient-Reported Outcome Measures (Symptoms and Impacts) in Adults with Pyruvate Kinase Deficiency

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3447-3447
Author(s):  
Sam Salek ◽  
Audra N. Boscoe ◽  
Chris Evans ◽  
Shayna Egan ◽  
Ted Wells ◽  
...  
Keyword(s):  
Pyruvate Kinase ◽  
Research Funding ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Specific Patient ◽  
Patient Reported ◽  
Item Wording ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
Disease Specific

Introduction: Pyruvate kinase (PK) deficiency is a rare, congenital autosomal recessive hemolytic anemia managed with supportive treatments, including transfusion, splenectomy, and iron chelation. Disease-directed treatments, including a small molecule PK activator and gene therapy, are currently in development. No disease-specific patient-reported outcome (PRO) measures have been validated for use in this patient population. The objective of this initiative was to develop PRO measures for assessing symptoms and impacts of PK deficiency and compare them to existing, non-disease-specific measures previously recommended for use in this disease area. Methods: A targeted literature review was conducted to inform the development of a preliminary hypothesized conceptual framework to identify signs, symptoms, and impacts commonly experienced by patients with PK deficiency and to inform the direction and content of interviews with such patients. Concept elicitation interviews were conducted with 21 adults with PK deficiency from the US, Netherlands, and Germany. Draft items were then tested in cognitive interviews with 20 adults with PK deficiency to further establish content validity and revised based on the results. A comparison was conducted between concepts included in the newly developed PK deficiency disease-specific measures and the domain structure and item concepts included in the EORTC QLQ-C30 and SF-36v2 to evaluate the extent of differences and conceptual overlap with instruments that had previously been recommended in this population. Specific attributes compared included face validity (i.e., conceptual coverage and inclusion of proximal symptoms and/or impacts) and measurement characteristics (i.e., item wording, recall, and response options). Results: Two measures, the PK Deficiency Diary (PKDD), a 7-item measure of the core signs and symptoms of PK deficiency, and PK Deficiency Impact Assessment (PKDIA), a 14-item measure of the impacts of PK deficiency on patients' HRQoL, were developed. A comparison of the newly drafted measures to the EORTC QLQ-C30 and SF-36v2 demonstrated minimal similarities in concepts, domains, item wording, and recall period. Of the 7 concepts in the PKDD, only 3 were common to the EORTC QLQ-C30, 4 were common to the SF36v2, and 2 were related but did not match exactly (i.e., "bone pain" and "pain"). Of the 12 distinct concepts in the PKDIA, only 5 were common to the EORTC QLQ-C30, 2 were common to the SF-36v2, and 3 were related but did not match exactly (i.e., difficulty starting things, difficulty finishing things, and difficulty performing moderate physical activity). Conclusions: This research demonstrates that the EORTC-QLQ-C30 and SF-36v2 lack the appropriate conceptual relevance and coverage of disease-specific signs, symptoms, and impacts most relevant and burdensome to patients with PK deficiency. The newly developed PKDD and PKDIA may be useful tools in clinical trials in patients with PK deficiency. Psychometric validation of these measures is currently underway. Disclosures Salek: Pfizer: Honoraria, Speakers Bureau; Merck: Consultancy; Agios Pharmaceuticals, Inc.: Consultancy, Honoraria. Boscoe:Agios Pharmaceuticals, Inc.: Employment, Equity Ownership. Evans:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Egan:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Wells:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Piantedosi:Agios Pharmaceuticals, Inc.: Employment. Grace:Agios Pharmaceuticals, Inc: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding. Storm:Agios: Employment.

scholarly journals Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

2022 ◽  
Vol 17 (1) ◽  
Author(s):  
Deborah Elstein ◽  
Nadia Belmatoug ◽  
Patrick Deegan ◽  
Özlem Göker-Alpan ◽  
Derralynn A. Hughes ◽  
...  
Keyword(s):  
Gaucher Disease ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Specific Patient ◽  
Patient Reported ◽  
Test Retest Reliability ◽  
Development And Validation ◽  
The Impact

Abstract Background Disease-specific patient-reported outcome measures (PROMs) are fundamental to understanding the impact on, and expectations of, patients with genetic disorders, and can facilitate constructive and educated conversations about treatments and outcomes. However, generic PROMs may fail to capture disease-specific concerns. Here we report the development and validation of a Gaucher disease (GD)-specific PROM for patients with type 1 Gaucher disease (GD1) a lysosomal storage disorder characterized by hepatosplenomegaly, thrombocytopenia, anemia, bruising, bone disease, and fatigue. Results and discussion The questionnaire was initially developed with input from 85 patients or parents of patients with GD1 or GD3 in Israel. Owing to few participating patients with GD3, content validity was assessed for patients with GD1 only. Content validity of the revised questionnaire was assessed in 33 patients in the US, France, and Israel according to US Food and Drug Administration standards, with input from a panel of six GD experts and one patient advocate representative. Concept elicitation interviews explored patient experience of symptoms and treatments, and a cognitive debriefing exercise explored patients’ understanding and relevance of instructions, items, response scales, and recall period. Two versions of the questionnaire were subsequently developed: a 24-item version for routine monitoring in clinical practice (rmGD1-PROM), and a 17-item version for use in clinical trials (ctGD1-PROM). Psychometric validation of the ctGD1-PROM was assessed in 46 adult patients with GD1 and re-administered two weeks later to examine test–retest reliability. Findings from the psychometric validation study revealed excellent internal consistency and strong evidence of convergent validity of the ctGD1-PROM based on correlations with the 36-item Short Form Health Survey. Most items were found to show moderate, good, or excellent test–retest reliability. Conclusions Development of the ctGD1-PROM represents an important step forward for researchers measuring the impact of GD and its respective treatment.

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