Background
Methods
Identification of studies
Study selection
Data extraction
Quality assessment
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Reliability is the degree to which the measurement is free from measurement error, as indicated by the extent to which scores for patients who have not changed are the same for repeated measurement under several conditions [14]. Studies were considered to have strong internal consistency reliability when Cronbach’s alpha was ≥0.80;
Results
Study inclusion
PRO instruments
Quality assessment results
Psychometric Properties | ||||||||
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Instrument | Aim | Items and Scoring | Developed for SCD Patients | Content Validity (SCD-specific) | Internal Reliability (Cronbach’s α) | Test-retest Reliability | Construct Validity | Responsiveness |
Coping, Self-efficacy, Self-esteem | ||||||||
Multidimensional Health Locus of Control Scale (MHLC) [18] | To assess respondents’ beliefs about whether their health status is determined by their own actions, the actions of other individuals, or is determined by fate or chance | 18 items across 3 subscales (internal, chance, and external) rated on a 6-point Likert scale; scoring system not reported | No | Unclear | Strong (0.82) | Unclear | Unclear | Unclear |
Rosenberg’s Self-esteem Scale (SES) [18] | To measure global self-esteem | 10 items scored on a 4-point Likert scale; higher scores correspond to higher self-esteem | No | Unclear | Strong (0.85) | Unclear | Unclear | Unclear |
Sense of Mastery Scale (SOM) [18] | To assess general sense of life control and mastery | 7-item instrument on a 4-point Likert scale; scoring system not reported | No | Unclear | Good (0.77) | Unclear | Unclear | Unclear |
To assess respondent’s ability to function on a day-to-day basis and to manage SCD symptomatology | 9 items ranked on a 5-point Likert scale; higher scores indicate greater self-efficacy | Yes | Unclear | Strong (0.80–0.89) | Unclear | Unclear | Unclear | |
Sickle Cell Transition Intervention Programs Skills Checklists [25] | To assess the transition readiness of patients with SCD | 5 knowledge skills sets (medical, educational/vocational, health benefits, social, independent) and 3 psychological checklists (self-efficacy, sickle cell stress, feelings about transition); scoring system not reported | Yes | Good | Good (0.46–0.86) | Unclear | Unclear | Unclear |
Simple Rathus Assertiveness Scale-Short Form (SRAS-SF) [21] | To measure a patient’s assertiveness in the health care setting | 19 items ranked on a 6-point Likert scale; higher scores indicate higher levels of assertiveness | No | Unclear | Strong (0.85) | Unclear | Unclear | Unclear |
Pain | ||||||||
PAINReportIt [23] | To help clinicians better understand the experience and etiology of pain in their patients | Patient marks areas of pain on a body outline drawing, circles words to describe pain quality and pattern, and writes narrative text to indicate activities that increase or reduce the pain, and selects pain severity indicators; scoring system not reported | No | Unclear | Unclear | Unclear | Unclear | Unclear |
Sickle Cell Disease Pain Burden Interview-Youtha (ages 7–21) (SCPBI-Y) [26] | To assess the impact of pain on physical, social/community, and emotional aspects of daily function | 7 items ranked on a 5-point Likert scale; higher scores indicate higher pain burden | Yes | Good | Strong (0.89) | Good | Good | Unclear |
West Haven-Yale Multidimensional Pain Inventory (WHYMPI) [17] | To assess patients’ ability to cope with chronic pain | 52 items ranked on a 7-point Likert scale; scoring system not reported | No | Unclear | Good (0.64–0.86) | Unclear | Unclear | Unclear |
Depression | ||||||||
Beck Depression Inventory-Fast Screen (BDI-FS) [20] | To measure symptoms of depression | 7 items ranked on a 4-point Likert scale, indicating frequency of symptom experienced over previous week; higher scores indicate more depressive symptoms | No | Unclear | Strong (0.81) | Unclear | Unclear | Unclear |
Family Impact | ||||||||
Parental Bonding Instrument (PBI) – Maternal Version [22] | To assess adults’ perceptions of maternal overprotection and care prior to the age of 16 years | 25 items; score system not reported | No | Unclear | Strong (0.83–0.89) | Unclear | Weak | Unclear |
Quality of Life | ||||||||
Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) [24] | To assess HRQL in adult patients with SCD | 4 item sets (cognitive, emotional, social functioning, and physical impact) and 5 additional items assessing pain episode severity and frequency; scoring system not reported | Yes | Strong | Strong (0.92–0.96) | Unclear | Good | Unclear |
Spirituality | ||||||||
Spiritual Well-being Scale [17] | To assess the general spirituality pertaining to existential and religious well-being | 20 items scored on a 6-point Likert scale; scoring system not reported | No | Unclear | Strong (0.82–0.88) | Unclear | Unclear | Unclear |
Stigma | ||||||||
Sickle Cell Disease Health-related Stigma Scale (SCD-HRSS) [20] | To assess the stigma patients with SCD perceive from the general public, physicians, and family | 30 items ranked on a 6-point Likert scale; higher scores indicate higher perceived stigma | No | Unclear | Strong (0.84) | Unclear | Unclear | Unclear |
Treatment Satisfaction | ||||||||
Adult Sickle Cell Quality of Life Measurement Quality of Care (ASCQ-Me QOC) [19] | To measure patients’ perceived quality of received health care services | 27 items on four domains (access, provider communication, ED care, and ED pain treatment); scoring system not reported | Yes | Good | Good (0.70–0.86) | Unclear | Unclear | Unclear |
Psychometric Properties | ||||||||
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Instrument | Aim | Items and Scoring | Developed for SCD Patients | Content Validity (SCD-specific) | Internal Reliability (Cronbach’s α) | Test-retest Reliability | Construct Validity | Responsiveness |
Health-related Quality of Life | ||||||||
To assess HRQL of children as young as 2 years (proxy report) and children as young as 5 years (self-report) | 23 items rated on a 5-point Likert scale; higher scores indicate more problems | No | Unclear | Strong (0.80–0.90 parent-report; 0.93 self-report) | Unclear | Unclear | Unclear | |
PedsQL™ SCD Module [36] | To assess quality of life that is specific to assessing the health issues relevant to pediatric patients with SCD | 43 items rated by a 5-point Likert scale; higher scores represent better quality of life | Yes | Unclear | Strong (0.95–0.97) | Unclear | Unclear | Unclear |
To measure fatigue experienced during the past 1 month across a variety of pediatric populations | 18 items rated on a 5-point Likert scale; higher scores represent better quality of life | No | Unclear | Strong (0.95 parent-report; 0.90 self-report) | Unclear | Unclear | Unclear | |
Patient Reported Outcomes Measurement Information System (PROMIS) [29] | To measure a pediatric patient’s health attributes of depressive symptoms over the previous 7 days | 156 items over 8 domains (physical functioning mobility, physical functioning upper extremity, pain interference, fatigue, depressive symptoms, anxiety, peer relationships, and anger) rated on a 5-point Likert scale; higher scores signify worse severity for depression, anxiety, anger, fatigue, and pain interference and better functioning for physical functioning mobility, physical functioning upper extremity, and peer relationships | No | Unclear | Unclear | Unclear | Good | Unclear |
Pain | ||||||||
Faces Pain Scale-Revised (FPS-R) [31] | To assess pain intensity in pediatric populations experiencing acute pain | 6 faces depicting different levels of increasing pain intensity | No | Good | Unclear | Unclear | Unclear | Unclear |
Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) [26] | To assess the pain burden among children and adolescents with SCD over the previous month | 7 items ranked on a 5-point Likert scale; higher scores indicate higher pain burden | Yes | Good | Strong (0.89) | Good | Good | Unclear |
Coping, Self-efficacy | ||||||||
Coping Strategy Questionnaire (CSQ) [30] | To measure coping with pain in adults, adolescents, and young children | 13 subscales, each with 6 items rated on a 7-point Likert-type scale; higher scores indicate greater pain | No | Unclear | Unclear | Unclear | Unclear | Unclear |
Sickle Cell Self-efficacy Scale (SCSES) [27] | To assess adults and adolescent’s self-appraisals of their ability to engage in daily functional activities despite having SCD | 9 items rated on a 5-point Likert scale; higher scores indicate greater self-efficacy | Yes | Unclear | Strong (0.87) | Unclear | Weak | Unclear |
Functioning | ||||||||
Youth Acute Pain Functional Ability Questionnaire (YAPFAQ) [37] | To assess physical function and functional recovery in youth undergoing acute and procedural pain | 40 items rated on a 5-point Likert scale; higher scores indicate greater difficulty performing functional activities | No | Unclear | Strong (0.92) | Unclear | Unclear | Unclear |
Family Impact | ||||||||
Psychosocial Assessment Tool 2.0 (PAT2.0) [32] | To measure psychosocial risk in families of a child newly diagnosed with cancer | 7 subscales; higher scores indicate many stressors | No | Unclear | Strong (0.84) | Unclear | Unclear | Unclear |
PRO instruments in adults with SCD
Validity
Reliability
Responsiveness
PRO instruments in children with SCD
Validity
Reliability
Responsiveness
Discussion
Guidance for PRO use in SCD populations based on SLR findings
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Consider using the PedsQL™ SCD Module to assess SCD-specific impact in children. This instrument allows for evaluation of various concepts, including pain, fatigue, productivity, activity, and emotion.
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Consider using the Sickle Cell Disease Pain Burden Interview-Youth (SCPBI-Y) to assess the impact of painful crises in children aged over 7 years.
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Use a short generic pain assessment tool, such as the Brief Pain Inventory, or numerical rating scales for assessing pain intensity for adults.
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Use the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) to assess patient-reported HRQL in adults. This instrument allows for evaluation of patients’ pain, fatigue, productivity, activity, and emotion.
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Use the ASCQ-Me Quality of Care (QOC) instrument to assess patient perceptions of accessibility of care and the quality of interactions with health care providers.
Recommendations for further PRO research in SCD populations
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There should be validation of a generic preference-based PRO instrument to evaluate general aspects of HRQL.
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There should be validation of specific instruments for younger children (ages 5–12 years) (caregiver report version of the SCPBI-Y and Psychosocial Assessment Tool 2.0) and for adults (ASCQ-Me, SCIPBI-Y).
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For relevant outcomes of interest (function, psychological well-being), there should be validation of the PedsQL™ 4.0 Generic Core Scales for children and the ASCQ-Me for adults.
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There should be validation (or, if none exist, development) of instruments that assess other outcomes of interest (e.g., cognitive impairment, school/work performance and attendance, treatment satisfaction).
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There should be piloting of administration of identified instruments using electronic devices (e.g., tablets, phone apps).