A LONGITUDINAL OBSERVATIONAL STUDY TO EVALUATE THE PSYCHOMETRIC PROPERTIES OF A DAILY DIARY FOR ADOLESCENTS AND ADULTS WITH SICKLE CELL DISEASE
Author(s): ,
Robert Clark Brown
Affiliations:
Aflac Cancer and Blood Disorders Center, Emory - Children’s Pediatric Institute,Atlanta,United States
,
Xiaochen Lin
Affiliations:
QualityMetric Incorporated LLC,Johnston,United States
,
Avery A. Rizio
Affiliations:
QualityMetric Incorporated LLC,Johnston,United States
,
Jackie Han
Affiliations:
Novartis Pharmaceuticals Corporation,East Hanover,United States
,
Emerenziana Marturano
Affiliations:
Novartis Pharma AG,Basel,Switzerland
Kenneth I. Ataga
Affiliations:
UTHSC Center for Sickle Cell Disease,Memphis,United States
EHA Library. Clark Brown R. 06/09/21; 324940; EP1219
Robert Clark Brown
Robert Clark Brown
Contributions
Abstract
Presentation during EHA2021: All e-poster presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: EP1219

Type: E-Poster Presentation

Session title: Sickle cell disease

Background
Vaso-occlusive crises (VOCs) are one of the most serious clinical features of sickle cell disease (SCD), impacting mortality, morbidity, and health-related quality of life (HRQoL). Existing patient-reported outcome measures (PROs) aim to assess the overall impact of SCD, and are typically administered at fixed intervals several days or weeks apart. However, such PROs are unable to accurately capture the full impact of VOCs, which occur sporadically and unpredictably. The Sickle Cell Pain Diary – Self Report (SCPD-S) was developed to address this measurement gap, assessing the impact of VOCs in adolescents and adults. Administered daily, the diary was designed with patient input to evaluate the severity of SCD-related pain during and outside of a VOC, and to capture the impact of pain on patient-relevant HRQoL concepts.

Aims
To develop a longitudinal observational study to assess the psychometric properties of the SCPD-S.

Methods
Study plans include enrolling 328 patients from 3 countries with a high burden of SCD: the United States (US), United Kingdom (UK), and Kingdom of Saudi Arabia. Participants with a verified diagnosis of SCD who were ≥12 years old were recruited through participant panels, advocacy groups, and clinician referrals. Participants were asked to complete the SCPD-S every day through a mobile application. On days that a VOC was experienced, participants reported on the impact of the VOC; if a VOC was not experienced, they reported on the impact of SCD on their daily life. The assessments occurred for at least 3 months, increasing the likelihood that participants would experience at least 1 VOC during the study. Anticipating that participants may not have perfect diary completion, it was determined that to be included in the planned analyses, participants must complete at least 25 of 30 daily assessments for at least one month and experience a VOC during that month. One criterion measure was administered daily (single item global impression of severity), while the remainder were administered the day after a VOC in order to reduce respondent burden during a crisis. Planned analyses include factor analysis to confirm the structure of the SCPD-S, which is hypothesized to assess a unidimensional construct of VOC impact. Multiple scoring systems will be explored to provide users flexibility in evaluating diary responses. Psychometric analyses will be conducted to examine the reliability (test-retest and internal consistency reliability), validity (convergent, divergent, and known-groups validity), and responsiveness of the instrument.

Results
After 11 weeks of recruitment in the UK, 81 participants have been enrolled. After 30 weeks of recruitment in the US, a total of 135 participants have been enrolled in the study. Of these, 9 were lost to follow-up, leaving 126 active participants. Only 1 participant missed more than 5 daily assessments for each month of their participation; 8 participants are still in their first month of the study. One hundred nine (93%) of the 117 remaining participants who have completed at least one month of the study have reported at least 1 VOC throughout their participation.

Conclusion
This study takes a unique approach to the psychometric validation of an SCD-specific PRO that was designed to collect information on sporadic and unpredictable events. Once the psychometric properties of the SCPD-S have been documented, the diary could be used in clinical practice to describe patients’ experiences during VOCs and capture responses to treatment.

Keyword(s): Outcome, Quality of life, Sickle cell disease

Presentation during EHA2021: All e-poster presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: EP1219

Type: E-Poster Presentation

Session title: Sickle cell disease

Background
Vaso-occlusive crises (VOCs) are one of the most serious clinical features of sickle cell disease (SCD), impacting mortality, morbidity, and health-related quality of life (HRQoL). Existing patient-reported outcome measures (PROs) aim to assess the overall impact of SCD, and are typically administered at fixed intervals several days or weeks apart. However, such PROs are unable to accurately capture the full impact of VOCs, which occur sporadically and unpredictably. The Sickle Cell Pain Diary – Self Report (SCPD-S) was developed to address this measurement gap, assessing the impact of VOCs in adolescents and adults. Administered daily, the diary was designed with patient input to evaluate the severity of SCD-related pain during and outside of a VOC, and to capture the impact of pain on patient-relevant HRQoL concepts.

Aims
To develop a longitudinal observational study to assess the psychometric properties of the SCPD-S.

Methods
Study plans include enrolling 328 patients from 3 countries with a high burden of SCD: the United States (US), United Kingdom (UK), and Kingdom of Saudi Arabia. Participants with a verified diagnosis of SCD who were ≥12 years old were recruited through participant panels, advocacy groups, and clinician referrals. Participants were asked to complete the SCPD-S every day through a mobile application. On days that a VOC was experienced, participants reported on the impact of the VOC; if a VOC was not experienced, they reported on the impact of SCD on their daily life. The assessments occurred for at least 3 months, increasing the likelihood that participants would experience at least 1 VOC during the study. Anticipating that participants may not have perfect diary completion, it was determined that to be included in the planned analyses, participants must complete at least 25 of 30 daily assessments for at least one month and experience a VOC during that month. One criterion measure was administered daily (single item global impression of severity), while the remainder were administered the day after a VOC in order to reduce respondent burden during a crisis. Planned analyses include factor analysis to confirm the structure of the SCPD-S, which is hypothesized to assess a unidimensional construct of VOC impact. Multiple scoring systems will be explored to provide users flexibility in evaluating diary responses. Psychometric analyses will be conducted to examine the reliability (test-retest and internal consistency reliability), validity (convergent, divergent, and known-groups validity), and responsiveness of the instrument.

Results
After 11 weeks of recruitment in the UK, 81 participants have been enrolled. After 30 weeks of recruitment in the US, a total of 135 participants have been enrolled in the study. Of these, 9 were lost to follow-up, leaving 126 active participants. Only 1 participant missed more than 5 daily assessments for each month of their participation; 8 participants are still in their first month of the study. One hundred nine (93%) of the 117 remaining participants who have completed at least one month of the study have reported at least 1 VOC throughout their participation.

Conclusion
This study takes a unique approach to the psychometric validation of an SCD-specific PRO that was designed to collect information on sporadic and unpredictable events. Once the psychometric properties of the SCPD-S have been documented, the diary could be used in clinical practice to describe patients’ experiences during VOCs and capture responses to treatment.

Keyword(s): Outcome, Quality of life, Sickle cell disease

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