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Open Access 01-12-2021 | Research

Content validation of observer-reported sickle cell pain diaries (SCPD-CS and SCPD-CN): results from interviews with caregivers

Authors: Michelle K. White, April M. Foster, Miranda Bailey, Denise D’Alessio, Avery Rizio, Patricia Stebbins, Danielle St. Pierre, Cory Saucier

Published in: Health and Quality of Life Outcomes | Issue 1/2021

Abstract

Background

Patients with sickle cell disease (SCD) experience daily pain and acute episodes known as sickle cell pain crises (SCPCs). The Sickle Cell Pain Diary-Caregiver Report (SCPD-C) is an observer-reported diary for use by caregivers of children ages < 12 years with SCD. This study reports on the content validity of the SCPD-C.

Methods

The SCPD-C was developed based on a literature review, measurement expert input, and a patient advisory board including clinicians. Three rounds of interviews (including both concept elicitation and cognitive debriefing methodologies) were conducted with caregivers of children with SCD aged < 12 to evaluate the content validity of the SCPD-C.

Results

Across three rounds of interviews, caregivers confirmed concepts in the SCPD-C and described observed impacts that were important and were added. Overall, caregivers evaluated the SCPD-C as easy to understand, with some minor adaptations for clarity. Additionally, the diary was split into two versions based on the child’s age and school enrollment status (SCPD-CS for school-aged and SCPD-CN for non-school age children).

Conclusions

Caregivers provided valuable input that led to important additions and changes to the measures. The SCPD-CS and SCPD-CN are appropriate and fit-for-purpose observer-reported outcome measures of SCPC-related pain frequency and severity, and impacts on health-related quality of life.

Centers for Disease Control and Prevention. Sickle Cell Disease (SCD): Data & Statistics. December 17, 2020. https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed 28 Sep 2021.

Brousseau DC, Panepinto JA, Nimmer M, Hoffmann RG. The number of people with sickle-cell disease in the United States: national and state estimates. Am J Hematol. 2010;85:77–8. https://doi.org/10.1002/ajh.21570.CrossRefPubMed

Nelson SC, Hackman HW. Race matters: perceptions of race and racism in a sickle cell center. Pediatr Blood Cancer. 2013;60:451–4. https://doi.org/10.1002/pbc.24361.CrossRefPubMed

Kanter J, Kruse-Jarres R. Management of sickle cell disease from childhood through adulthood. Blood Rev. 2013;27:279–87. https://doi.org/10.1016/j.blre.2013.09.001.CrossRefPubMed

U.S. Food and Drug Administration. The FDA Encourages New Treatments for Sickle Cell Disease. 2014. https://www.fda.gov/ForConsumers/ConsumerUpdates/ucm418232.htm. Accessed 25 Sep 2017.

Ballas SK, Kesen MR, Goldberg MF, Lutty GA, Dampier C, Osunkwo I, et al. Beyond the definitions of the phenotypic complications of sickle cell disease: an update on management. Sci World J. 2012;2012: 949535. https://doi.org/10.1100/2012/949535.CrossRef

Dampier C, Ely B, Brodecki D, Coleman C, Aertker L, Sendecki JA, et al. Pain characteristics and age-related pain trajectories in infants and young children with sickle cell disease. Pediatr Blood Cancer. 2014;61:291–6. https://doi.org/10.1002/pbc.24796.CrossRefPubMed

Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, editors. The Voice of the Patient_ Sickle Cell Report: A series of reports from the U.S. Food and Drug Administrations's (FDA's) Patient-Focused Drug Development Initiative; 2014.

Martin SR, Cohen LL, Mougianis I, Griffin A, Sil S, Dampier C. Stigma and pain in adolescents hospitalized for sickle cell vasoocclusive pain episodes. Clin J Pain. 2018;34:438–44. https://doi.org/10.1097/AJP.0000000000000553.CrossRefPubMed

10.

Wakefield EO, Popp JM, Dale LP, Santanelli JP, Pantaleao A, Zempsky WT. Perceived racial bias and health-related stigma among youth with sickle cell disease. J Dev Behav Pediatr. 2017;38:129–34. https://doi.org/10.1097/DBP.0000000000000381.CrossRefPubMed

11.

Badawy SM, Thompson AA, Lai J-S, Penedo FJ, Rychlik K, Liem RI. Adherence to hydroxyurea, health-related quality of life domains, and patients’ perceptions of sickle cell disease and hydroxyurea: a cross-sectional study in adolescents and young adults. Health Qual Life Outcomes. 2017;15:136. https://doi.org/10.1186/s12955-017-0713-x.CrossRefPubMedPubMedCentral

12.

Adeyemo TA, Ojewunmi OO, Diaku-Akinwumi IN, Ayinde OC, Akanmu AS. Health related quality of life and perception of stigmatisation in adolescents living with sickle cell disease in Nigeria: A cross sectional study. Pediatr Blood Cancer. 2015;62:1245–51. https://doi.org/10.1002/pbc.25503.CrossRefPubMed

13.

Ugwu AO, Ibegbulam OG, Nwagha TU, Madu AJ, Ocheni S, Okpala I. Clinical and Laboratory Predictors of Frequency of Painful Crises among Sickle Cell Anaemia Patients in Nigeria. J Clin Diagn Res. 2017;11:EC22–5. https://doi.org/10.7860/JCDR/2017/26446.10042.CrossRefPubMedPubMedCentral

14.

Merle NS, Paule R, Leon J, Daugan M, Robe-Rybkine T, Poillerat V, et al. P-selectin drives complement attack on endothelium during intravascular hemolysis in TLR-4/heme-dependent manner. Proc Natl Acad Sci U S A. 2019;116:6280–5. https://doi.org/10.1073/pnas.1814797116.CrossRefPubMedPubMedCentral

15.

Bender MA. Gene Reviews: Sickle Cell Disease. 1993–2017. https://www.ncbi.nlm.nih.gov/books/NBK1377/. Accessed 25 Sep 2017.

16.

Heath LE, Heeney MM, Hoppe CC, Adjei S, Agbenyega T, Badr M, et al. Successful utilization of an electronic pain diary in a multinational phase 3 interventional study of pediatric sickle cell anemia. Clin Trials. 2017;14:563–71. https://doi.org/10.1177/1740774517723307.CrossRefPubMed

17.

Lewandowski AS, Palermo TM, Kirchner HL, Drotar D. Comparing diary and retrospective reports of pain and activity restriction in children and adolescents with chronic pain conditions. Clin J Pain. 2009;25:299–306. https://doi.org/10.1097/AJP.0b013e3181965578.CrossRefPubMedPubMedCentral

18.

Brousseau DC, Owens PL, Mosso AL, Panepinto JA, Steiner CA. Acute care utilization and rehospitalizations for sickle cell disease. JAMA. 2010;303:1288–94. https://doi.org/10.1001/jama.2010.378.CrossRefPubMed

19.

Kauf TL, Coates TD, Huazhi L, Mody-Patel N, Hartzema AG. The cost of health care for children and adults with sickle cell disease. Am J Hematol. 2009;84:323–7. https://doi.org/10.1002/ajh.21408.CrossRefPubMed

20.

Shankar SM, Arbogast PG, Mitchel E, Cooper WO, Wang WC, Griffin MR. Medical care utilization and mortality in sickle cell disease: a population-based study. Am J Hematol. 2005;80:262–70. https://doi.org/10.1002/ajh.20485.CrossRefPubMed

21.

Wallen GR, Minniti CP, Krumlauf M, Eckes E, Allen D, Oguhebe A, et al. Sleep disturbance, depression and pain in adults with sickle cell disease. BMC Psychiatry. 2014;14:207. https://doi.org/10.1186/1471-244X-14-207.CrossRefPubMedPubMedCentral

22.

Dampier C, LeBeau P, Rhee S, Lieff S, Kesler K, Ballas S, et al. Health-related quality of life in adults with sickle cell disease (SCD): a report from the comprehensive sickle cell centers clinical trial consortium. Am J Hematol. 2011;86:203–5. https://doi.org/10.1002/ajh.21905.CrossRefPubMedPubMedCentral

23.

Matthie N, Hamilton J, Wells D, Jenerette C. Perceptions of young adults with sickle cell disease concerning their disease experience. J Adv Nurs. 2016;72:1441–51. https://doi.org/10.1111/jan.12760.CrossRefPubMed

24.

El-Haj N, Hoppe C. Newborn screening for SCD in the USA and Canada. IJNS. 2018;4:36. https://doi.org/10.3390/ijns4040036.CrossRefPubMedPubMedCentral

25.

Quinn CT, Rogers ZR, Buchanan GR. Survival of children with sickle cell disease. Blood. 2004;103:4023–7. https://doi.org/10.1182/blood-2003-11-3758.CrossRefPubMed

26.

Chaturvedi S, DeBaun MR. Evolution of sickle cell disease from a life-threatening disease of children to a chronic disease of adults: The last 40 years. Am J Hematol. 2016;91:5–14. https://doi.org/10.1002/ajh.24235.CrossRefPubMed

27.

Maikler VE, Broome ME, Bailey P, Lea G. Childrens’ and adolescents’ use of diaries for sickle cell pain. J Soc Pediatr Nurs. 2001;6:161–9.CrossRef

28.

Dampier C, Ely E, Eggleston B, Brodecki D, O’Neal P. Physical and cognitive-behavioral activities used in the home management of sickle pain: a daily diary study in children and adolescents. Pediatr Blood Cancer. 2004;43:674–8. https://doi.org/10.1002/pbc.20162.CrossRefPubMed

29.

Dampier C, Ely B, Brodecki D, O’Neal P. Characteristics of pain managed at home in children and adolescents with sickle cell disease by using diary self-reports. J Pain. 2002;3:461–70.CrossRef

30.

Dampier C, Ely E, Brodecki D, O’Neal P. Home management of pain in sickle cell disease: a daily diary study in children and adolescents. J Pediatr Hematol Oncol. 2002;24:643–7. https://doi.org/10.1097/00043426-200211000-00008.CrossRefPubMed

31.

Karlson CW, Baker AM, Bromberg MH, David Elkin T, Majumdar S, Palermo TM. Daily pain, physical activity, and home fluid intake in pediatric sickle cell disease. J Pediatr Psychol. 2017;42:335–44. https://doi.org/10.1093/jpepsy/jsw061.CrossRefPubMed

32.

Matza LS, Patrick DL, Riley AW, Alexander JJ, Rajmil L, Pleil AM, Bullinger M. Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO good research practices for the assessment of children and adolescents task force. Value Health. 2013;16:461–79. https://doi.org/10.1016/j.jval.2013.04.004.CrossRefPubMed

33.

Treadwell MJ, Hassell K, Levine R, Keller S. Adult sickle cell quality-of-life measurement information system (ASCQ-Me): conceptual model based on review of the literature and formative research. Clin J Pain. 2014;30:902.CrossRef

34.

Willis GB. Cognitive interviewing: a tool for improving questionnaire design. 6th ed. California: SAGE Publications, Inc.; 2005.CrossRef

35.

Assarroudi A, Heshmati Nabavi F, Armat MR, Ebadi A, Vaismoradi M. Directed qualitative content analysis: the description and elaboration of its underpinning methods and data analysis process. J Res Nurs. 2018;23:42–55. https://doi.org/10.1177/1744987117741667.CrossRefPubMedPubMedCentral

36.

Corbin JM, Strauss A. Basics of qualitative research: techniques and procedures for developing grounded theory. Thousand Oaks: SAGE Publications, Inc.; 2015.

37.

Kolb SM. Grounded theory and the constant comparative method: Valid research strategies for educators. JETERAPS. 2012;3:83–6.

38.

Turner-Bowker DM, Lamoureux RE, Stokes J, Litcher-Kelly L, Galipeau N, Yaworsky A, et al. Informing a priori sample size estimation in qualitative concept elicitation interview studies for clinical outcome assessment instrument development. Value Health. 2018;21:839–42. https://doi.org/10.1016/j.jval.2017.11.014.CrossRefPubMed

39.

U.S. Food & Drug Administration. Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 2009. https://www.fda.gov/downloads/drugs/guidances/ucm193282.pdf. Accessed 4 Jun 2018.

40.

Patrick DL, Burke LB, Gwaltney CJ, Leidy NK, Martin ML, Molsen E, Ring L. Content validity–establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 2–assessing respondent understanding. Value Health. 2011;14:978–88. https://doi.org/10.1016/j.jval.2011.06.013.CrossRefPubMed

41.

Raphael JL, Dietrich CL, Whitmire D, Mahoney DH, Mueller BU, Giardino AP. Healthcare utilization and expenditures for low income children with sickle cell disease. Pediatr Blood Cancer. 2009;52:263–7. https://doi.org/10.1002/pbc.21781.CrossRefPubMed

42.

Mvundura M, Amendah D, Kavanagh PL, Sprinz PG, Grosse SD. Health care utilization and expenditures for privately and publicly insured children with sickle cell disease in the United States. Pediatr Blood Cancer. 2009;53:642–6. https://doi.org/10.1002/pbc.22069.CrossRefPubMed

43.

Blinder MA, Vekeman F, Sasane M, Trahey A, Paley C, Duh MS. Age-related treatment patterns in sickle cell disease patients and the associated sickle cell complications and healthcare costs. Pediatr Blood Cancer. 2013;60:828–35. https://doi.org/10.1002/pbc.24459.CrossRefPubMed

44.

Panepinto JA, Torres S, Varni JW. Development of the PedsQLTM sickle cell disease module items: Qualitative methods. Qual Life Res. 2012;21:341–57.CrossRef

45.

Panepinto JA, Torres S, Bendo CB, McCavit TL, Dinu B, Sherman-Bien S, et al. PedsQLTM sickle cell disease module: feasibility, reliability, and validity. Pediatr Blood Cancer. 2013;60:1338–44. https://doi.org/10.1002/pbc.24491.CrossRefPubMedPubMedCentral

Title: Content validation of observer-reported sickle cell pain diaries (SCPD-CS and SCPD-CN): results from interviews with caregivers
Authors: Michelle K. White
April M. Foster
Miranda Bailey
Denise D’Alessio
Avery Rizio
Patricia Stebbins
Danielle St. Pierre
Cory Saucier
Publication date: 01-12-2021
Publisher: BioMed Central
Published in: Health and Quality of Life Outcomes / Issue 1/2021
Electronic ISSN: 1477-7525
DOI: https://doi.org/10.1186/s12955-021-01888-5

At a glance: The STEP trials

Springer Medicine

Content validation of observer-reported sickle cell pain diaries (SCPD-CS and SCPD-CN): results from interviews with caregivers

Abstract

Background

Methods

Results

Conclusions

At a glance: The STEP trials

Springer Medicine

Abstract

Background

Methods

Results

Conclusions

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