BackgroundAlthough the quality of life (QoL) plays an important role in treatment decision making and clinical management of mycosis fungoides (MF) or Sézary syndrome (SS) subtypes of cutaneous T-cell lymphomas (MF/SS-CTCLs), an MF- or SS-specific measure of QoL does not exist.ObjectiveThe objective of this research was to develop and validate the first QoL instrument for MF/SS-CTCL using a patient-centered approach.MethodsA conceptual framework for the MF/SS-CTCL QoL was developed through a literature review and interviews with key opinion leaders. Concept elicitation with patients was utilized to refine the conceptual model and generate preliminary items. The items were then revised based on qualitative and quantitative feedback obtained through cognitive debriefing surveys and interviews with patients. Next, participants (N=126) completed the preliminary MF/SS-CTCL QoL and a comparator measure of health-related QoL (Skindex-29) through the PatientsLikeMe Open Research Exchange. The MF/SS-CTCL QoL was completed again 5 days later by 66 participants for the purposes of evaluating test-retest reliability. The MF/SS-CTCL QoL was finalized based on results from an empirical evaluation, which included both classical and modern test theory approaches. Specifically, this included evaluation of (1) the optimal item response theory measurement model; (2) item fit; (3) unidimensionality; (4) rating scale performance; (5) reliability; (6) test information (precision); (7) person-to-item map; (8) convergent and discriminant validity; and (9) presence of bias via differential item function.ResultsResults from the comprehensive psychometric evaluation utilizing a Rasch-Grouped Rating Scale model yielded a final 12-item instrument. The rating scale functioned as expected, and the instrument exhibited adequate person reliability (.87), good to excellent test-retest reliability (r=.89, P<.001), high levels of measurement precision, and good person-to-item targeting. The correlation between the MF/SS-CTCL QoL and the Skindex-29 (r=.852, P<.001) was significantly greater than the correlation between the MF/SS-CTCL QoL and syndrome stage (r=.260, P<.001), providing support for convergent and discriminant validity. Items did not show significant bias based on gender, age, or race. Rasch scores were converted to scaled scores with qualitative descriptive categories for ease of interpretation.ConclusionsEmpirical evaluation demonstrated strong evidence of excellent psychometric properties. Utilizing a patient-centered measure development approach ensures that this QoL instrument captures the information that is most meaningful and clinically relevant to patients.
Objectives: Patients' experience of MF/SS-CTCL remains understudied due to the rarity of the condition. Therefore, PatientsLikeMe (PLM) aimed to harness its internal platform, Open Research Exchange (ORE), to explore a novel method of data collection for the concept elicitation phase of PRO development. MethOds: Members of the MF/SS-CTCL community on PLM were invited to complete an online survey to share their experiences as patients. The 11 open-ended questions intended to elicit descriptions of MF/SS-CTCL quality of life impact, ability to cope and manage their condition, perception of symptoms and treatments, and sources of distress were administered to 21 patients. Of those, 10 participated in a follow-up interview. The data was analyzed by two trained raters and inter-rater reliability was used to validate and confirm their agreement. Results: The sample (N= 21) was predominantly female (67%), all white, and mostly reported MF (76%) rather than SS. The two raters achieved moderate to high inter-rater agreement for the qualitative analysis (0.68-0.80 Cohen's kappa for the 43 validated codes). Saturation for the sample was reached after 15 patients. Dominant themes that were elicited from the data were social impact (f= 27), emotional impact (f= 30), impact on daily activities (f= 20), coping/management (f= 64), severity and burden of symptoms and treatments (f= 377, f= 291), and overall treatment satisfaction (f= 291). cOnclusiOns: The online method proved to be an effective tool for understanding the experiences of a population living with a rare condition and complimented the interview data in eliciting dominant themes that were present in the literature. Utilizing ORE as the primary method of data collection, recruitment for this rare condition was found to be easier and faster than traditional methods and the sample size recruited was validated by concept saturation. Therefore, together, the survey and the follow-up interviews proved to be successful in eliciting relevant concepts from patients.Objectives: To develop a patient-reported outcome (PRO) instrument that assesses important and relevant signs, symptoms, and impacts for adult patients with pancreatic cancer; and to evaluate patient comprehension and usability of the draft Pancreatic Cancer Symptom Assessment Form (PC-SAF) administered via electronic diary. MethOds: Concept elicitation (CE) interviews were conducted with 18 adult patients with pancreatic cancer from one of six US clinic sites to identify the most important and relevant signs, symptoms, and impacts of pancreatic cancer. Results from patient interviews, clinical expert interviews, and a targeted concept literature review informed the development of the draft PC-SAF, which was evaluated in cognitive interviews (CI) with N= 14 adult patients with pancreatic cancer from one of two US clinic sites to assess patient comprehension and usability of the electronic instrument. Results: Gastrointestinal (GI) symptoms (loss of appetite, early satiety, gas/bloating, constipation, diarrhea, naus...
BACKGROUND Although the quality of life (QoL) plays an important role in treatment decision making and clinical management of mycosis fungoides (MF) or Sézary syndrome (SS) subtypes of cutaneous T-cell lymphomas (MF/SS-CTCLs), an MF- or SS-specific measure of QoL does not exist. OBJECTIVE The objective of this research was to develop and validate the first QoL instrument for MF/SS-CTCL using a patient-centered approach. METHODS A conceptual framework for the MF/SS-CTCL QoL was developed through a literature review and interviews with key opinion leaders. Concept elicitation with patients was utilized to refine the conceptual model and generate preliminary items. The items were then revised based on qualitative and quantitative feedback obtained through cognitive debriefing surveys and interviews with patients. Next, participants (N=126) completed the preliminary MF/SS-CTCL QoL and a comparator measure of health-related QoL (Skindex-29) through the PatientsLikeMe Open Research Exchange. The MF/SS-CTCL QoL was completed again 5 days later by 66 participants for the purposes of evaluating test-retest reliability. The MF/SS-CTCL QoL was finalized based on results from an empirical evaluation, which included both classical and modern test theory approaches. Specifically, this included evaluation of (1) the optimal item response theory measurement model; (2) item fit; (3) unidimensionality; (4) rating scale performance; (5) reliability; (6) test information (precision); (7) person-to-item map; (8) convergent and discriminant validity; and (9) presence of bias via differential item function. RESULTS Results from the comprehensive psychometric evaluation utilizing a Rasch-Grouped Rating Scale model yielded a final 12-item instrument. The rating scale functioned as expected, and the instrument exhibited adequate person reliability (.87), good to excellent test-retest reliability (r=.89, P<.001), high levels of measurement precision, and good person-to-item targeting. The correlation between the MF/SS-CTCL QoL and the Skindex-29 (r=.852, P<.001) was significantly greater than the correlation between the MF/SS-CTCL QoL and syndrome stage (r=.260, P<.001), providing support for convergent and discriminant validity. Items did not show significant bias based on gender, age, or race. Rasch scores were converted to scaled scores with qualitative descriptive categories for ease of interpretation. CONCLUSIONS Empirical evaluation demonstrated strong evidence of excellent psychometric properties. Utilizing a patient-centered measure development approach ensures that this QoL instrument captures the information that is most meaningful and clinically relevant to patients.
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