Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module

Scott, John; Badaki-Makun, Oluwakemi; Darbari, Deepika S.; Chumpitazi, Corrie E.; Airewele, Gladstone; Ellison, Angela M.; Smith‐Whitley, Kim; Mahajan, Prashant; Sarnaik, Sharada A.; Casper, T. Charles; Cook, Larry; Leonard, Julie C.; Hulbert, Monica L.; Powell, Elizabeth C.; Liem, Robert I.; Hickey, Robert W.; Krishnamurti, Lakshmanan; Hillery, Cheryl A.; Brousseau, David C.

doi:10.1186/s12955-017-0700-2

Cited by 27 publications

(27 citation statements)

References 16 publications

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“…Previously, the Pediatric Quality-of-Life Inventory (PedsQL) generic and PedsQL SCD module have been shown to be valid and reliable tools Abbreviations: ICC, intraclass correlation coefficient; PRO, patient-reported outcome; PROMIS, Patient-Reported Outcomes Measurement Information System; REDCap, Research Electronic Data Capture; SCD, sickle cell disease to measure PROs for children with SCD. [4][5][6] More recently, the National Institutes of Health (NIH) has funded the development of the Patient-Reported Outcomes Measurement Information System (PROMIS) to assess patients' functioning. Prior work supports the psychometric properties of the original PROMIS domains for use in children with SCD.…”

Section: Introductionmentioning

confidence: 99%

Use of the new pediatric PROMIS measures of pain and physical experiences for children with sickle cell disease

Singh

Dasgupta

Simpson

et al. 2019

Pediatric Blood & Cancer

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Background There are new pediatric domains to measure patients’ pain and physical experiences in the Patient‐Reported Outcomes Measurement Information System (PROMIS). The objective of this study was to establish the psychometric properties of these domains for children with sickle cell disease (SCD). Procedure We conducted a cross‐sectional analysis of PROMIS assessments of children with SCD recruited from a pediatric tertiary care clinic. Validity of the new PROMIS domains was determined by comparing scores between known groups and describing their correlations with previously validated PROMIS measures. Cronbach's alpha and item response theory (IRT) reliability were used to assess internal consistency reliability. Agreement between parent‐proxy and child self‐report was determined for all domains. Results Our study included 164 subjects, of whom 117 were eligible to self‐report. The mean T‐scores for physical stress experience, strength impact, pain behavior, and pain quality sensory scores were significantly different between children who used pain medications in the prior week and those who did not. There were also differences in T‐scores across children reporting mild, moderate, and severe pain on the pain intensity scale. All measures had Cronbach's alpha and IRT reliability > 0.80. The percentage of agreement between child and parent‐proxy PROMIS domains ranged from 36% to 60% depending on the domain. Conclusions The new PROMIS domains of physical stress experience, strength impact, pain behavior, and pain quality sensory domains are valid and reliable for children with SCD. The low–moderate agreement between parent‐proxy and child self‐report scores support the complementary information provided by the two perspectives.

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Section: Introductionmentioning

confidence: 99%

Use of the new pediatric PROMIS measures of pain and physical experiences for children with sickle cell disease

Singh

Dasgupta

Simpson

et al. 2019

Pediatric Blood & Cancer

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“…The interitem reliabilities for the total scale are 0.95 for adolescent report and 0.97 for parental report, with those for the domains ranging from 0.69 to 0.90 for patient self-report and 0.83-0.97 for parentproxy reports 28 ; it has, to date, shown responsiveness to changes in HRQOL in patients with painful crises, 31 which offers further support for use of this instrument. The PedsQL-SCD module instrument has been validated for Jamaican adolescents with SCD and shows good feasibility and reliability in this population.…”

Section: Pediatric Quality Of Life Inventory-sickle Cell Disease Modulementioning

confidence: 87%

Level of agreement between adolescents’ self‐assessment and parent proxy report of health‐related quality of life in adolescents with sickle cell disease

Blake

Guthrie-Dixon²,

Grindley

et al. 2020

Pediatric Blood & Cancer

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Background We aim to assess the levels of agreement between parents, as proxies, and Jamaican adolescents living with sickle cell disease (SCD) in the reporting of the adolescent's quality of life. Procedures This cross‐sectional study assessed 102 patient/proxy pairs on quality of life of adolescents with SCD using the PedsQL‐SCD module. The level of agreement among pairs was assessed starting with broad group‐level approaches (the Wilcoxon signed‐rank test augmented by exploring percentage agreement) tapering to individual‐level approaches (intraclass correlation coefficients [ICCs] supplemented with Bland‐Altman plots). Results Most patients (76.5%) had homozygous SS disease (45.1% females; mean age 15.2 ± 1.5 years). Median total pediatric quality of life (PedsQL) scores were 79.1 (adolescent report) and 80.2 (parental report) (P = .60). There were 11.8% underestimation and 12.7% overestimation of overall health‐related quality of life (HRQOL) by parents. The highest perfect agreement existed on the “pain and hurt” domain for both male and female adolescents (85.7% and 84.4%, respectively). Overestimation was highest on the “social communication” domain for both male and female adolescents (19.6% and 34.8%, respectively). Parents exhibited good agreement on total PedsQL scores in male adolescents (ICC = 0.70), but moderate agreement (ICC = 0.43) in female adolescents. Generally, parents underestimated their male child's functioning and overestimated the female child's functioning on the various domains. Conclusions Parents and adolescents exhibit fair agreement in assessment of the adolescent's overall HRQOL but differ on subjective domains. Agreement varies by sex of the affected teen where girls’ HRQOL is generally overestimated by the parental proxy. Interventions to improve parents’ understanding of their children's psychosocial needs are needed.

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“…Similarities exist between clinical features of pain in SCD and in BERK and Townes mouse models. Increased sensitivity to heat, cold, and mechanical stimuli observed in mice has also been observed in patients with SCD using quantitative sensory testing (QST) (Bakshi, Lukombo, Belfer, & Krishnamurti, 2018;Brandow, Stucky, Hillery, Hoffmann, & Panepinto, 2013;Cain et al, 2012;Campbell et al, 2016;Darbari et al, 2017;Ezenwa et al, 2016;Hillery et al, 2011;Jacob et al, 2015;Kohli et al, 2010;Lei, Benson, Tran, Ofori-Acquah, & Gupta, 2016). For both BERK and Townes sickle mice, females show increased hyperalgesia compared to age-matched males (Kohli et al, 2010;Lei et al, 2016).…”

Section: Mouse Models Of Pain In Scdmentioning

confidence: 91%

“…Central sensitization in BERK mice based on electrophysiological recordings of nociceptors in the dorsal horn of the spinal cord has been observed (Cataldo, Rajput, Gupta, & Simone, 2015). Suggestion of central sensitization in patients with SCD has been reported following neuroimaging of the brain and QST Darbari et al, 2015Darbari et al, , 2017. Of note, BERK sickle mice and patients with SCD show higher circulating substance P, a critical mediator of chronic pain Michaels, Ohene-Frempong, Zhao, & Douglas, 1998;Vang et al, 2015;Vincent et al, 2013).…”

Section: Of 30mentioning

confidence: 99%

Mouse Models of Pain in Sickle Cell Disease

et al. 2018

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Sickle cell disease (SCD) is a genetic blood disorder that impacts millions of individuals worldwide. SCD is characterized by debilitating pain that can begin during infancy and may continue to increase throughout life. This pain can be both acute and chronic. A characteristic feature specific to acute pain in SCD occurs during vaso‐occlusive crisis (VOC) due to the blockade of capillaries with sickle red blood cells. The acute pain of VOC is intense, unpredictable, and requires hospitalization. Chronic pain occurs in a significant population with SCD. Treatment options for sickle pain are limited and primarily involve the use of opioids. However, long‐term opioid use is associated with numerous side effects. Thus, pain management in SCD remains a major challenge. Humanized transgenic mice expressing exclusively human sickle hemoglobin show features of pain and pathobiology similar to that in patients with SCD. Therefore, these mice offer the potential for investigating the mechanisms of pain in SCD and allow for development of novel targeted analgesic therapies. © 2018 by John Wiley & Sons, Inc.

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Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module

Cited by 27 publications

References 16 publications

Use of the new pediatric PROMIS measures of pain and physical experiences for children with sickle cell disease

Use of the new pediatric PROMIS measures of pain and physical experiences for children with sickle cell disease

Level of agreement between adolescents’ self‐assessment and parent proxy report of health‐related quality of life in adolescents with sickle cell disease

Mouse Models of Pain in Sickle Cell Disease

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