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. Author manuscript; available in PMC: 2020 May 1.
Published in final edited form as: Health Psychol. 2019 May;38(5):386–390. doi: 10.1037/hea0000696

A Preliminary Investigation of the Psychometric Properties of PROMIS® Scales in Emerging Adults with Sickle Cell Disease

Aimee K Hildenbrand 1,2,3, Charles T Quinn 4, Constance A Mara 5,6, James L Peugh 5,6, Emily A McTate 5,6, Maria T Britto 6,7, Lori E Crosby 5,6
PMCID: PMC6501803  NIHMSID: NIHMS1015515  PMID: 31045421

Abstract

Objective:

To report preliminary psychometric properties of PROMIS assessments among emerging adults with sickle cell disease (SCD).

Methods:

Forty-five emerging adults with SCD ages 18–24 (M=20.81, SD=1.73) completed PROMIS Pain Interference and Fatigue short forms and the Pediatric Quality of Life Inventory (PedsQL). Chart review was used to obtain information regarding SCD complications and healthcare utilization in the three years prior to study enrollment. Multivariate path analysis was used to compare PROMIS scores by disease severity and use of disease-modifying therapies. Cronbach’s alpha was used to evaluate internal consistency, and Pearson correlations assessed convergent validity between PROMIS measures and the PedsQL.

Results:

Internal consistency was excellent for PROMIS Pain Interference (α=.95) and good for PROMIS Fatigue (α=.85). Associations between PROMIS measures and PedsQL subscales were in the expected direction and large in magnitude (all p-values < .001). There were no differences in PROMIS scores based on disease severity or use of disease-modifying therapy (p-values = .14 – .86).

Conclusions:

Findings provide preliminary evidence of validity and reliability of the PROMIS Pain Interference and Fatigue short forms among emerging adults with SCD. Additional research is needed to replicate these findings in larger samples and assess sensitivity to change over time.

Keywords: sickle cell disease, pain, fatigue, quality of life, young adults, emerging adults

For individuals with sickle cell disease (SCD), routine assessment of patient-reported health status and symptoms is recommended to facilitate optimal prevention and treatment efforts (National Heart Lung and Blood Institute [NHLBI], 2014). SCD is a group of related multisystem genetic diseases of hemoglobin characterized by red blood cells that are rigid, abnormally adhesive, and dehydrated, which can impede blood flow to tissues and organs. SCD is associated with a number of medical complications, including chronic hemolytic anemia, recurrent vaso-occlusive painful episodes, stroke, and acute and chronic injury to the heart, lung, kidney, bones, spleen, and other systems (NHLBI, 2014). As a result, individuals with SCD demonstrate impaired health-related quality of life (HRQoL) relative to healthy peers and comparable or worse HRQoL relative to other chronic conditions (Dampier et al., 2011; McClish et al., 2005; Panepinto & Bonner, 2012). In particular, pain and fatigue are prominent symptoms that can significantly impair physical and psychosocial functioning and may increase healthcare utilization among those with SCD (Ameringer, Elswick, & Smith, 2014; Dampier et al., 2011; McClish et al., 2005).

Although SCD-related complications increase over the lifespan (Serjeant, 2013), emerging adulthood (i.e., late teens through the twenties; Arnett, 2000) is a particularly vulnerable time given multifaceted challenges related to transition to adult-centered healthcare (Jordan, Swerdlow, & Coates, 2013). These challenges contribute to high rates of emergency care visits, hospitalizations, and re-hospitalizations (Brousseau et al., 2010) as well as premature death among emerging adults who transfer from pediatric to adult medical care (Hamideh & Alvarez, 2013). As such, careful monitoring of health and functioning in this subgroup is essential. However, the existing literature largely focuses on either youth or adults of all ages, and thus HRQoL in emerging adults with SCD remains understudied and poorly understood. Furthermore, few patient-reported outcome assessment tools have been validated in emerging adults with SCD (Sarri et al., 2018).

The Patient Reported Outcomes Measurement Information System (PROMIS®) is a National Institutes of Health initiative that aims to standardize the assessment of broad aspects of health status in clinical practice and research. To this end, PROMIS offers brief yet precise patient-report measures of clinically relevant outcomes applicable to a range of chronic illness groups (Cella et al., 2010). Very few studies have examined the utility of PROMIS scales in SCD. Among youth with SCD, Dampier and colleagues demonstrated that PROMIS measures captured expected group differences (Dampier, Barry, et al., 2016) and were sensitive to changes in health status during hospitalization (Dampier, Jaeger, et al., 2016). Keller et al. (2017) found that PROMIS scales were robustly related to SCD severity and performed comparably to a SCD-specific measure of quality of life in adults. However, the psychometric properties of PROMIS tools have yet to be investigated in emerging adults with SCD. This pilot study aimed to examine reliability and validity for PROMIS Pain Interference and Fatigue scales in this unique, understudied, and at-risk group. We hypothesized that PROMIS scales would demonstrate high internal consistency and strong associations with a legacy measure of HRQoL.

Methods

Participants

This study utilizes baseline data from a larger investigation of SCD self-management interventions (Crosby, Hudepohl, et al., 2013; Crosby, Joffe, et al., 2013). Participants included 45 emerging adults with SCD receiving care in a tertiary children’s hospital or affiliated adult hospital in the Midwestern United States. Eligibility criteria included a primary diagnosis of SCD and ages 18–24 years at recruitment. Those with significant cognitive limitations precluding completion of study instruments based on medical chart review or physician report were excluded.

Procedures

Eligible individuals were contacted via letter, phone, or in-person during clinic appointments or at SCD-related community events. All participants provided informed consent prior to completing study procedures. Participants completed assessments via laptop or iPad. Electronic medical records were reviewed to obtain clinical data. Participants were compensated $35. This study was approved by the institutional review board of Cincinnati Children’s Hospital Medical Center.

Measures

Demographics and Healthcare Utilization.

A self-report form was used to assess participant demographics. Electronic medical records were reviewed to obtain SCD genotype, SCD-related complications, therapies, and hospital admissions in the three years before study enrollment.

Pain Interference and Fatigue.

The PROMIS Adult Pain Interference 6-item Short Form (Cella et al., 2010) assesses the extent to which pain interferes with daily activities over the past 7 days on a 5-point scale (1 = “not at all” to 5 = “very much”). The PROMIS Adult Fatigue 7-item Short Form (Cella et al., 2010) assesses the impact of fatigue over the past 7 days on a 5-point scale (1 = “never” to 5 = “always”). Higher scores indicate greater pain interference and fatigue. PROMIS scales are available at no cost from www.healthmeasures.net.

Quality of Life.

The Pediatric Quality of Life Inventory (PedsQL™), Young Adult Version is a 23-item instrument designed to measure quality of life among young adults with acute or chronic health problems in the past month (Varni et al., 2001). Respondents rate each item on a 5-point scale (0 = “never” to 4 = “almost always”). The PedsQL includes four subscales: Physical, Emotional, Social, and School/ Work Functioning. Higher scores indicate better HRQoL. The validity of the generic version of the PedsQL is well-established in the broader literature and has shown validity and reliability among individuals with SCD (Panepinto et al., 2008; Panepinto & Bonner, 2012).

Data Analysis

Analyses were conducted using SPSS, version 24 and MPlus, version 8 (Muthén & Muthén, 1998–2017). Multivariate path analysis was used to compare PROMIS scores by disease severity (mild vs. severe) and use of disease-modifying therapies (i.e., hydroxyurea, chronic transfusions). To evaluate internal consistency, Cronbach’s alpha was computed for PROMIS items. Pearson correlations were computed to assess convergent validity between PROMIS scales and the PedsQL; coefficients were interpreted as small (r = .10), medium (r = .30), or large (r = .50) effect sizes (Cohen, 1992). Maximum likelihood estimation was used to handle missing data.

Results

Of the 64 patients ages 18–24 who were identified as potentially eligible, 50 (78%) agreed to participate in this study. Among those who agreed, four were unable to complete the baseline study visit due to barriers such as work schedules and distance from the hospital, and one patient was ineligible due to intellectual disability.

Forty-five emerging adults ages 18 – 24 years (M = 20.81, SD = 1.73) participated in this study. All participants identified as African American, and 24 (53%) were female. Nearly all participants (n = 44, 93%) identified as single. Nineteen (42%) achieved a high school degree, with the remainder reporting: less than a high school (n = 6, 13%), some college (n = 7, 16%), a college degree (n = 4, 9%), and a graduate or professional degree (n = 2, 4%); 7 (16%) did not provide information on education. Approximately half of the sample indicated living in a home with another adult caregiver (n = 24, 53%) and were not currently employed (n = 23, 51%). Annual family income among this sample was as follows: under $10,000 (n = 19, 42%), $10,001 – $30,000 (n = 9, 20%), and $30,001 – $75,000 (n = 6, 13%); 4 participants (9%) indicated that they were unaware of their annual family income and 7 (16%) did not provide family income data.

SCD genotypes included HbSS (n = 31, 69%), HbSC (n = 12, 27%), and Hb S/β+-thalassemia (n = 2, 4%). Seventeen participants (38%) were on hydroxyurea and ten (22%) were on chronic transfusion therapy. Consistent with previous research (Panepinto et al., 2013), patients were classified as having severe SCD if they experienced one or more of the following complications based on medical record review: (1) overt stroke; (2) acute chest syndrome; or (3) three or more hospitalizations for SCD pain in the past three years. Those who did not meet these criteria were classified as having mild disease. In this sample, 25 (56%) emerging adults exhibited severe disease.

See Table 1 for full PROMIS and PedsQL descriptive statistics. This sample endorsed higher than average levels of pain interference and fatigue relative to the general population. Scores ranged widely on these measures, with 26 (58%) participants reporting levels of pain interference greater than 1 SD above the general population mean (i.e., T-scores ≥ 60). Nearly one third (n = 14, 31%) of fatigue T-scores were equal to or greater than 1 SD above the general population average. PedsQL total scores likewise ranged widely from 7.61 – 97.83 (M = 65.66, SD = 16.94).

Table 1.

Descriptive Statistics for PROMIS Measures and PedsQL

Total Disease Severity Hydroxyurea Chronic Transfusions No disease-modifying therapy
Instrument (N = 45) M (SD) Mild (n = 20) M (SD) Severe (n = 25) M (SD) (n = 17) M (SD) (n = 10) M (SD) (n = 19) M (SD)
PROMIS Pain Interference Short Form* 59.61 (8.10) 59.07 (8.77) 60.04 (7.68) 56.80 (8.10) 62.99 (3.76) 60.55 (9.01)
PROMIS Fatigue Short Form* 55.93 (9.47) 55.20 (9.74) 56.51 (9.42) 52.68 (10.14) 58.32 (7.36) 57.55 (9.25)
PedsQL+
  Physical Functioning Subscale 64.06 (19.22) 63.59 (22.08) 64.45 (16.96) 65.26 (20.70) 62.50 (15.93) 64.06 (19.89)
  Emotional Functioning Subscale 63.30 (22.77) 66.25 (23.89) 60.83 (22.00) 69.12 (18.39) 53.50 (20.82) 63.89 (26.10)
  Social Functioning Subscale 78.41 (21.40) 80.00 (22.65) 77.08 (20.69) 82.06 (16.87) 71.50 (23.69) 78.89 (23.49)
  School/ Work Functioning Subscale 57.84 (19.06) 59.75 (19.16) 56.25 (19.24) 70.29 (17.72) 44.00 (16.63) 54.17 (14.06)
  Psychosocial Summary Score 66.52 (18.00) 68.67 (18.87) 64.72 (17.45) 73.82 (14.71) 56.33 (17.65) 65.65 (18.41)
  Total Score 65.66 (16.94) 66.90 (18.69) 64.63 (15.68) 70.84 (15.22) 58.48 (14.76) 65.10 (18.37)
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*

T-scores (M = 50, SD = 10)

+

N = 44 for all PedsQL descriptive statistics.

Note: PedsQL = Pediatric Quality of Life Inventory (PedsQL™), Young Adult Version.

Higher scores on PROMIS measures indicate greater pain interference and fatigue. Higher scores on the PedsQL indicate better HRQoL.

Internal consistency was excellent for PROMIS Pain Interference (α = .95) and good for PROMIS Fatigue (α = .85). Greater fatigue and pain interference were strongly associated with worsened PedsQL physical, social, school, and emotional functioning (all p-values < .001; see Table 2). Correlation coefficients were large in magnitude (r = −.55 to −.75). Multivariate path analysis revealed no significant differences in PROMIS Pain Interference (p-values = .17 – .86) or Fatigue scores (p-values = .14 – .71) by disease severity, hydroxyurea, or chronic transfusion treatment.

Table 2.

Pearson Correlations between PROMIS Measures and PedsQL

PedsQL Subscale PROMIS Pain Interference Short Form PROMIS Fatigue Short Form
Physical Functioning −0.55* −0.75*
Emotional Functioning −0.56* −0.65*
Social Functioning −0.61* −0.60*
School/ Work Functioning −0.58* −0.60*
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Note: N = 44 for all correlation coefficients presented above. PedsQL = Pediatric Quality of Life Inventory (PedsQL™), Young Adult Version. Higher scores on PROMIS measures indicate greater pain interference and fatigue. Higher scores on the PedsQL indicate better HRQoL.

*

p < .001

Discussion

This study sought to examine preliminary psychometric properties of PROMIS Pain Interference and Fatigue short forms during emerging adulthood. Among individuals with SCD, risk for disease complications and death sharply increases during this key developmental period (Hamideh & Alvarez, 2013), yet little attention has been directed towards advancing the measurement of health status in this vulnerable group. As hypothesized, internal consistency was high for the PROMIS Pain Interference and Fatigue short forms. Associations between PROMIS and PedsQL were strong and in the expected direction. Findings are consistent with emerging research suggesting that pain and fatigue are related to poorer functioning across various dimensions of HRQoL in youth and adults with SCD (Ameringer et al., 2014; Dampier et al., 2010, 2011; Panepinto & Bonner, 2012; Panepinto et al., 2014; Taylor et al., 2010). Although sample size precluded formal evaluation of construct validity, these findings contribute to converging evidence regarding concept inter-relationships and comparative performance of PROMIS scales by demonstrating preliminary evidence of their reliability and validity in a unique, understudied, and at-risk population.

As expected, our sample of emerging adults with SCD reported higher levels of pain interference and fatigue relative to general population norms. In particular, even those with mild disease and those receiving disease-modifying therapies endorsed significant life limitation due to pain. This sample of emerging adults also demonstrated compromised HRQoL across several domains. For instance, using cut-off scores for children and adolescents (Varni et al., 2003), this sample endorsed worse physical and school/work functioning. Compared to college students with chronic health conditions (Varni & Limbers, 2009), this sample exhibited poorer HRQoL across all domains except emotional functioning. Findings are consistent with prior research documenting impaired HRQoL among youth and adults with SCD (Panepinto & Bonner, 2012), underlining the need for continued development and dissemination of interventions to enhance HRQoL across the lifespan for this population.

Several limitations should be considered when evaluating results from this pilot study. Due to inadequate power, we were unable to formally assess construct or known groups discriminant validity; future research should examine the factor structure of PROMIS scales in emerging adults with SCD as well as their sensitivity for capturing differences in outcomes based on disease and treatment characteristics. Additionally, longitudinal research is needed to examine the utility of PROMIS measures for capturing disease- or intervention-related changes over time in emerging adults with SCD. Participants were recruited from two local institutions in the Midwestern region of the US, which may limit generalizability of findings. Replication in larger and more representative samples is needed. Given that this study focused solely on pain interference and fatigue, future research should also examine the validity and reliability of other PROMIS scales relevant to this group.

Findings from this study provide preliminary evidence of the validity and reliability of the PROMIS Pain Interference and Fatigue short forms among emerging adults with SCD. PROMIS measures are efficient, precise, and flexible and thereby represent a promising approach for standardizing the assessment of important indicators of health and wellbeing. Accurate measurement of HRQoL during and after transition from pediatric to adult healthcare settings may contribute to improved disease management and long-term outcomes for this high-risk population.

Acknowledgements:

This research was supported by a grant from the National Heart, Lung, and Blood Institute at the National Institutes of Health (grant number K07HL108720). We thank those who participated in this study as well as the medical, nursing, and psychosocial staff in the Cancer and Blood Diseases Institute at Cincinnati Children’s Hospital Medical Center. The authors would also like to acknowledge Cara Nwankwo for her assistance with this paper.

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