DEVELOPMENT OF PATIENT-REPORTED OUTCOMES SYMPTOM MEASURE FOR PATIENTS WITH NON-TRANSFUSION-DEPENDENT THALASSEMIA (NTDT-PRO)
(Abstract release date: 05/19/16)
EHA Library. Taher A. 06/09/16; 135004; PB2104
Ali T. Taher
Contributions
Contributions
Abstract
Abstract: PB2104
Type: Publication Only
Background
Currently, there is no disease-specific Patient-Reported Outcomes (PRO) measure available to assess key symptoms experienced by patients with non-transfusion-dependent thalassemia (NTDT).
Aims
To develop a symptom outcomes measure for patients with NTDT for use in clinical trials.
Methods
A qualitative study was conducted to develop the NTDT-PRO symptom measure consistent with regulatory requirements. The instrument was developed based on concept elicitation interviews, input from clinical experts, and refined through a process of cognitive interviews. Concept elicitation interviews were conducted among a total of 25 NTDT patients recruited from three countries (Lebanon, Greece, and Canada). Findings from these interviews were used to generate an item pool, inform response options, and determine appropriate recall period. Cognitive interviews were subsequently conducted among subjects in Greece and Lebanon (N=21) to further support saturation and examine the relevance, clarity and understanding of the items among the target population. All interviews were conducted in person in the local language with written informed consent obtained, audio-recorded, transcribed and translated back into English for evaluation.
Results
Based on findings from concept elicitation interviews, saturation of important underlying concepts was obtained and a total of nine symptoms were included in the NTDT-PRO Version 1. Instructions and item stems were derived from patient language elicited during the interviews. An 11-point numeric rating scale (0-10) was used for the response options, anchored on either end by the absence of the symptom and extreme symptom. A daily recall period (“during the past 24 hours”) was selected based on the day-to-day variability in the symptom experience. Findings from the cognitive interviews indicate that subjects understood the instructions and that the daily recall period was appropriate. Subjects understood the items as intended, with a few minor exceptions. Four items were deleted as they were not considered core symptoms, and one item was added to address the symptom both with and without physical activity. The NTDT-PRO Version 2 includes six items that address three key symptoms of tiredness, weakness, and shortness of breath both during physical activity and at rest.
Conclusion
The NTDT-PRO Version 2 consists of six items to assess the key symptoms of NTDT, including tiredness, weakness, and shortness of breath using a 24-hour recall period, with plans to further evaluate the measures in an ongoing observational study and for eventual inclusion into a randomized phase 3 study.
Session topic: E-poster
Type: Publication Only
Background
Currently, there is no disease-specific Patient-Reported Outcomes (PRO) measure available to assess key symptoms experienced by patients with non-transfusion-dependent thalassemia (NTDT).
Aims
To develop a symptom outcomes measure for patients with NTDT for use in clinical trials.
Methods
A qualitative study was conducted to develop the NTDT-PRO symptom measure consistent with regulatory requirements. The instrument was developed based on concept elicitation interviews, input from clinical experts, and refined through a process of cognitive interviews. Concept elicitation interviews were conducted among a total of 25 NTDT patients recruited from three countries (Lebanon, Greece, and Canada). Findings from these interviews were used to generate an item pool, inform response options, and determine appropriate recall period. Cognitive interviews were subsequently conducted among subjects in Greece and Lebanon (N=21) to further support saturation and examine the relevance, clarity and understanding of the items among the target population. All interviews were conducted in person in the local language with written informed consent obtained, audio-recorded, transcribed and translated back into English for evaluation.
Results
Based on findings from concept elicitation interviews, saturation of important underlying concepts was obtained and a total of nine symptoms were included in the NTDT-PRO Version 1. Instructions and item stems were derived from patient language elicited during the interviews. An 11-point numeric rating scale (0-10) was used for the response options, anchored on either end by the absence of the symptom and extreme symptom. A daily recall period (“during the past 24 hours”) was selected based on the day-to-day variability in the symptom experience. Findings from the cognitive interviews indicate that subjects understood the instructions and that the daily recall period was appropriate. Subjects understood the items as intended, with a few minor exceptions. Four items were deleted as they were not considered core symptoms, and one item was added to address the symptom both with and without physical activity. The NTDT-PRO Version 2 includes six items that address three key symptoms of tiredness, weakness, and shortness of breath both during physical activity and at rest.
Conclusion
The NTDT-PRO Version 2 consists of six items to assess the key symptoms of NTDT, including tiredness, weakness, and shortness of breath using a 24-hour recall period, with plans to further evaluate the measures in an ongoing observational study and for eventual inclusion into a randomized phase 3 study.
Session topic: E-poster
Abstract: PB2104
Type: Publication Only
Background
Currently, there is no disease-specific Patient-Reported Outcomes (PRO) measure available to assess key symptoms experienced by patients with non-transfusion-dependent thalassemia (NTDT).
Aims
To develop a symptom outcomes measure for patients with NTDT for use in clinical trials.
Methods
A qualitative study was conducted to develop the NTDT-PRO symptom measure consistent with regulatory requirements. The instrument was developed based on concept elicitation interviews, input from clinical experts, and refined through a process of cognitive interviews. Concept elicitation interviews were conducted among a total of 25 NTDT patients recruited from three countries (Lebanon, Greece, and Canada). Findings from these interviews were used to generate an item pool, inform response options, and determine appropriate recall period. Cognitive interviews were subsequently conducted among subjects in Greece and Lebanon (N=21) to further support saturation and examine the relevance, clarity and understanding of the items among the target population. All interviews were conducted in person in the local language with written informed consent obtained, audio-recorded, transcribed and translated back into English for evaluation.
Results
Based on findings from concept elicitation interviews, saturation of important underlying concepts was obtained and a total of nine symptoms were included in the NTDT-PRO Version 1. Instructions and item stems were derived from patient language elicited during the interviews. An 11-point numeric rating scale (0-10) was used for the response options, anchored on either end by the absence of the symptom and extreme symptom. A daily recall period (“during the past 24 hours”) was selected based on the day-to-day variability in the symptom experience. Findings from the cognitive interviews indicate that subjects understood the instructions and that the daily recall period was appropriate. Subjects understood the items as intended, with a few minor exceptions. Four items were deleted as they were not considered core symptoms, and one item was added to address the symptom both with and without physical activity. The NTDT-PRO Version 2 includes six items that address three key symptoms of tiredness, weakness, and shortness of breath both during physical activity and at rest.
Conclusion
The NTDT-PRO Version 2 consists of six items to assess the key symptoms of NTDT, including tiredness, weakness, and shortness of breath using a 24-hour recall period, with plans to further evaluate the measures in an ongoing observational study and for eventual inclusion into a randomized phase 3 study.
Session topic: E-poster
Type: Publication Only
Background
Currently, there is no disease-specific Patient-Reported Outcomes (PRO) measure available to assess key symptoms experienced by patients with non-transfusion-dependent thalassemia (NTDT).
Aims
To develop a symptom outcomes measure for patients with NTDT for use in clinical trials.
Methods
A qualitative study was conducted to develop the NTDT-PRO symptom measure consistent with regulatory requirements. The instrument was developed based on concept elicitation interviews, input from clinical experts, and refined through a process of cognitive interviews. Concept elicitation interviews were conducted among a total of 25 NTDT patients recruited from three countries (Lebanon, Greece, and Canada). Findings from these interviews were used to generate an item pool, inform response options, and determine appropriate recall period. Cognitive interviews were subsequently conducted among subjects in Greece and Lebanon (N=21) to further support saturation and examine the relevance, clarity and understanding of the items among the target population. All interviews were conducted in person in the local language with written informed consent obtained, audio-recorded, transcribed and translated back into English for evaluation.
Results
Based on findings from concept elicitation interviews, saturation of important underlying concepts was obtained and a total of nine symptoms were included in the NTDT-PRO Version 1. Instructions and item stems were derived from patient language elicited during the interviews. An 11-point numeric rating scale (0-10) was used for the response options, anchored on either end by the absence of the symptom and extreme symptom. A daily recall period (“during the past 24 hours”) was selected based on the day-to-day variability in the symptom experience. Findings from the cognitive interviews indicate that subjects understood the instructions and that the daily recall period was appropriate. Subjects understood the items as intended, with a few minor exceptions. Four items were deleted as they were not considered core symptoms, and one item was added to address the symptom both with and without physical activity. The NTDT-PRO Version 2 includes six items that address three key symptoms of tiredness, weakness, and shortness of breath both during physical activity and at rest.
Conclusion
The NTDT-PRO Version 2 consists of six items to assess the key symptoms of NTDT, including tiredness, weakness, and shortness of breath using a 24-hour recall period, with plans to further evaluate the measures in an ongoing observational study and for eventual inclusion into a randomized phase 3 study.
Session topic: E-poster
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