What is the future of patient-reported outcomes in sickle-cell disease?

References

• Piel FB, Patil AP, Howes RE, et al. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates. Lancet. 2013 Jan;381(9861):142–151.
   PubMed Web of Science ®Google Scholar
• Kato GJ, Piel FB, Reid CD, et al. Sickle cell disease. Nat Rev Dis Primers. 2018;4:18010.
   PubMed Web of Science ®Google Scholar
• Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec;376(9757):2018–2031.
   PubMed Web of Science ®Google Scholar
• Ataga KI, Desai PC. Advances in new drug therapies for the management of sickle cell disease. Expert Opin Orphan Drugs. 2018;6(5):329–343.
   PubMed Web of Science ®Google Scholar
• Kalpatthi R, Novelli EM. Measuring success: utility of biomarkers in sickle cell disease clinical trials and care. Hematology Am Soc Hematol Educ Program. 2018 Nov;2018(1):482–492.
   PubMedGoogle Scholar
• Farrell AT, Panepinto J, Carroll CP, et al. End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain. Blood Adv. 2019 Dec;3(23):3982–4001.
   PubMed Web of Science ®Google Scholar
• Lavallee DC, Chenok KE, Love RM, et al. Incorporating patient-reported outcomes into health care to engage patients and enhance care. Health Aff (Millwood). 2016 Apr;35(4):575–582.
   PubMed Web of Science ®Google Scholar
• Basch E. Patient-reported outcomes - harnessing patients’ voices to improve clinical care. N Engl J Med. 2017 Jan;376(2):105–108.
   PubMed Web of Science ®Google Scholar
• Nixon A, Wild D, Muehlhausen W Patient reported outcomes: an overview. SEEd Medical Publishers.
   Google Scholar
• Vodicka E, Kim K, Devine EB, et al. Inclusion of patient-reported outcome measures in registered clinical trials: evidence from clinicaltrials.gov (2007–2013). Contemp Clin Trials. 2015 Jul;43:1–9.
   PubMed Web of Science ®Google Scholar
• Mercieca-Bebber R, King MT, Calvert MJ, et al. The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Relat Outcome Meas. 2018;9:353–367.
   PubMedGoogle Scholar
• Acquadro C, Berzon R, Dubois D, et al. Incorporating the patient’s perspective into drug development and communication: an ad hoc task force report of the patient-reported outcomes (PRO) harmonization group meeting at the food and drug administration, February 16, 2001. Value Health. 2003 Sep-Oct;6(5):522–531.
   PubMed Web of Science ®Google Scholar
• Calvert M, Kyte D, Price G, et al. Maximising the impact of patient reported outcome assessment for patients and society. BMJ. 2019;364:k5267.
   PubMed Web of Science ®Google Scholar
• Willke RJ, Burke LB, Erickson P. Measuring treatment impact: a review of patient-reported outcomes and other efficacy endpoints in approved product labels. Control Clin Trials. 2004 Dec;25(6):535–552.
   PubMedGoogle Scholar
• Howie L, Hirsch B, Locklear T, et al. Assessing the value of patient-generated data to comparative effectiveness research. Health Aff (Millwood). 2014 Jul;33(7):1220–1228.
   PubMed Web of Science ®Google Scholar
• Gnanasakthy A, Lewis S, Clark M, et al. Potential of patient-reported outcomes as nonprimary endpoints in clinical trials. Health Qual Life Outcomes. 2013;11:83.
   PubMed Web of Science ®Google Scholar
• Calvert M, Blazeby J, Altman DG, et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA. 2013 Feb;309(8):814–822.
   PubMed Web of Science ®Google Scholar
• Coons SJ, Kothari S, Monz BU, et al. The patient-reported outcome (PRO) consortium: filling measurement gaps for PRO end points to support labeling claims. Clin Pharmacol Ther. 2011 Nov;90(5):743–748.
   PubMed Web of Science ®Google Scholar
• Cella D, Yount S, Rothrock N, et al. The patient-reported outcomes measurement information system (PROMIS): progress of an NIH roadmap cooperative group during its first two years. Med Care. 2007 May;45(5 Suppl 1):S3–S11.
   PubMed Web of Science ®Google Scholar
• Fehnel S, DeMuro C, McLeod L, et al. US FDA patient-reported outcome guidance: great expectations and unintended consequences. Expert Rev Pharmacoecon Outcomes Res. 2013 Aug;13(4):441–446.
   PubMed Web of Science ®Google Scholar
• Fiero MH, Roydhouse JK, Vallejo J, et al. US food and drug administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January, 2008, and December, 2017. Lancet Oncol. 2019 Oct;20(10):e582–e589.
   PubMed Web of Science ®Google Scholar
• Cappelleri JC, Zou KH, Bushmakin AG, et al. Patient-reported outcomes: measurement, implementation and interpretation. 2014.
   Google Scholar
• Mapi research trust. [cited 2020 Jul 24]. Available from: https://eprovide.mapi-trust.org
   Google Scholar
• Emery MP, Perrier LL, Acquadro C. Patient-reported outcome and quality of life instruments database (PROQOLID): frequently asked questions. Health Qual Life Outcomes. 2005 3;Mar:12.
   Google Scholar
• HealthMeasures. [cited 2020 Jul 24]. Available from: healthmeasures.net
   Google Scholar
• Calvert M, Kyte D, Mercieca-Bebber R, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: the SPIRIT-PRO extension. JAMA. 2018 feb;319(5):483–494.
   PubMed Web of Science ®Google Scholar
• Kotronoulas G, Kearney N, Maguire R, et al. What is the value of the routine use of patient-reported outcome measures toward improvement of patient outcomes, processes of care, and health service outcomes in cancer care? A systematic review of controlled trials. J Clin Oncol. 2014 May;32(14):1480–1501.
   PubMed Web of Science ®Google Scholar
• Cella D, Hahn EA, Jensen SE, et al. Patient-reported outcomes in performance measurement. RTI Press; 2015.
   Google Scholar
• Dobrozsi S, Panepinto J. Patient-reported outcomes in clinical practice. Hematology Am Soc Hematol Educ Program. 2015;2015:501–506.
   PubMedGoogle Scholar
• Kyte D, Ives J, Draper H, et al. Inconsistencies in quality of life data collection in clinical trials: a potential source of bias? Interviews with research nurses and trialists. PLoS One. 2013;8(10):e76625.
   PubMed Web of Science ®Google Scholar
• Mercieca-Bebber R, Calvert M, Kyte D, et al. The administration of patient-reported outcome questionnaires in cancer trials: interviews with trial coordinators regarding their roles, experiences, challenges and training. Contemp Clin Trials Commun. 2018;9:23–32.
   PubMed Web of Science ®Google Scholar
• Ayilara OF, Zhang L, Sajobi TT, et al. Impact of missing data on bias and precision when estimating change in patient-reported outcomes from a clinical registry. Health Qual Life Outcomes. 2019 Jun;17(1):106.
   PubMedGoogle Scholar
• Bell ML, Fairclough DL. Practical and statistical issues in missing data for longitudinal patient-reported outcomes. Stat Methods Med Res. 2014 Oct;23(5):440–459.
   PubMed Web of Science ®Google Scholar
• Díaz-Ordaz K, Kenward MG, Cohen A, et al. Are missing data adequately handled in cluster randomised trials? A systematic review and guidelines. Clin Trials. 2014 Oct;11(5):590–600.
   PubMed Web of Science ®Google Scholar
• Mercieca-Bebber R, Palmer MJ, Brundage M, et al. Design, implementation and reporting strategies to reduce the instance and impact of missing patient-reported outcome (PRO) data: a systematic review. BMJ Open. 2016;6(6):e010938.
   PubMed Web of Science ®Google Scholar
• Coens C, Pe M, Dueck AC, et al. International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL consortium. Lancet Oncol. 2020 feb;21(2):e83–e96.
   PubMed Web of Science ®Google Scholar
• Upton P, Lawford J, Eiser C. Parent-child agreement across child health-related quality of life instruments: a review of the literature. Qual Life Res. 2008 Aug;17(6):895–913.
   PubMed Web of Science ®Google Scholar
• Jardine J, Glinianaia SV, McConachie H, et al. Self-reported quality of life of young children with conditions from early infancy: a systematic review. Pediatrics. 2014 Oct;134(4):e1129–48.
   PubMed Web of Science ®Google Scholar
• Birnie KA, Richardson PA, Rajagopalan AV, et al. Factors related to agreement between child and caregiver report of child functioning with chronic pain: PROMIS pediatric and parent proxy report. Clin J Pain. 2020 Mar;36(3):203–212.
   PubMed Web of Science ®Google Scholar
• Blake A, Guthrie-Dixon N, Grindley M, et al. Level of agreement between adolescents’ self-assessment and parent proxy report of health-related quality of life in adolescents with sickle cell disease. Pediatr Blood Cancer. 2020 Apr;67(4):e28198.
   PubMed Web of Science ®Google Scholar
• Hilari K, Owen S, Farrelly SJ. Proxy and self-report agreement on the stroke and Aphasia quality of life scale-39. J Neurol Neurosurg Psychiatry. 2007 Oct;78(10):1072–1075.
   PubMed Web of Science ®Google Scholar
• Panepinto JA, Hoffmann RG, Pajewski NM. The effect of parental mental health on proxy reports of health-related quality of life in children with sickle cell disease. Pediatr Blood Cancer. 2010 Oct;55(4):714–721.
   PubMed Web of Science ®Google Scholar
• Sarri G, Bhor M, Abogunrin S, et al. Systematic literature review and assessment of patient-reported outcome instruments in sickle cell disease. Health Qual Life Outcomes. 2018 May;16(1):99.
   PubMedGoogle Scholar
• Ballas SK. Pain management of sickle cell disease. Hematol Oncol Clin North Am. 2005 Oct;19(5):785–802, v.
   PubMed Web of Science ®Google Scholar
• Gladwin MT, Kato GJ, Weiner D, et al. Nitric oxide for inhalation in the acute treatment of sickle cell pain crisis: a randomized controlled trial. JAMA. 2011 Mar;305(9):893–902.
   PubMed Web of Science ®Google Scholar
• Dampier CD, Smith WR, Wager CG, et al. IMPROVE trial: a randomized controlled trial of patient-controlled analgesia for sickle cell painful episodes: rationale, design challenges, initial experience, and recommendations for future studies. Clin Trials. 2013 Apr;10(2):319–331.
   PubMed Web of Science ®Google Scholar
• Lopez BL, Flenders P, Davis-Moon L, et al. Clinically significant differences in the visual analog pain scale in acute vasoocclusive sickle cell crisis. Hemoglobin. 2007;31(4):427–432.
   PubMed Web of Science ®Google Scholar
• Myrvik MP, Brandow AM, Drendel AL, et al. Clinically meaningful measurement of pain in children with sickle cell disease. Pediatr Blood Cancer. 2013 Oct;60(10):1689–1695.
   PubMed Web of Science ®Google Scholar
• Myrvik MP, Drendel AL, Brandow AM, et al. A comparison of pain assessment measures in pediatric sickle cell disease: visual analog scale versus numeric rating scale. J Pediatr Hematol Oncol. 2015 Apr;37(3):190–194.
   PubMed Web of Science ®Google Scholar
• Dworkin RH, Turk DC, Wyrwich KW, et al. Interpreting the clinical importance of treatment outcomes in chronic pain clinical trials: IMMPACT recommendations. J Pain. 2008 Feb;9(2):105–121.
   PubMed Web of Science ®Google Scholar
• Collaborative health outcomes information registry [cited 2020 Jul 24]. Available from: https://choir.stanford.edu
   Google Scholar
• Bhandari RP, Feinstein AB, Huestis SE, et al. Pediatric-collaborative health outcomes information registry (Peds-CHOIR): a learning health system to guide pediatric pain research and treatment. Pain. 2016 Sept;157(9):2033–2044.
   PubMed Web of Science ®Google Scholar
• Smith WR, Penberthy LT, Bovbjerg VE, et al. Daily assessment of pain in adults with sickle cell disease. Ann Intern Med. 2008 Jan;148(2):94–101.
   PubMed Web of Science ®Google Scholar
• Dampier C, Ely B, Brodecki D, et al. Characteristics of pain managed at home in children and adolescents with sickle cell disease by using diary self-reports. J Pain. 2002 Dec;3(6):461–470.
   PubMed Web of Science ®Google Scholar
• Bakshi N, Stinson JN, Ross D, et al. Development, content validity, and user review of a web-based multidimensional pain diary for adolescent and young adults with sickle cell disease. Clin J Pain. 2015 Jun;31(6):580–590.
   PubMed Web of Science ®Google Scholar
• Cella D, Riley W, Stone A, et al. The patient-reported outcomes measurement information system (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005–2008. J Clin Epidemiol. 2010 Nov;63(11):1179–1194.
   PubMed Web of Science ®Google Scholar
• Rothrock NE, Hays RD, Spritzer K, et al. Relative to the general US population, chronic diseases are associated with poorer health-related quality of life as measured by the patient-reported outcomes measurement information system (PROMIS). J Clin Epidemiol. 2010 Nov;63(11):1195–1204.
   PubMed Web of Science ®Google Scholar
• DeWalt DA, Gross HE, Gipson DS, et al. PROMIS(®) pediatric self-report scales distinguish subgroups of children within and across six common pediatric chronic health conditions. Qual Life Res. 2015 Sep;24(9):2195–2208.
   PubMed Web of Science ®Google Scholar
• Walsh TR, Irwin DE, Meier A, et al. The use of focus groups in the development of the PROMIS pediatrics item bank. Qual Life Res. 2008 Jun;17(5):725–735.
   PubMed Web of Science ®Google Scholar
• Irwin DE, Varni JW, Yeatts K, et al. Cognitive interviewing methodology in the development of a pediatric item bank: a patient reported outcomes measurement information system (PROMIS) study. Health Qual Life Outcomes. 2009;7:3.
   PubMed Web of Science ®Google Scholar
• Varni JW, Magnus B, Stucky BD, et al. Psychometric properties of the PROMIS ® pediatric scales: precision, stability, and comparison of different scoring and administration options. Qual Life Res. 2014 May;23(4):1233–1243.
   PubMed Web of Science ®Google Scholar
• Dampier C, Jaeger B, Gross HE, et al. Responsiveness of PROMIS® pediatric measures to hospitalizations for sickle pain and subsequent recovery. Pediatr Blood Cancer. 2016 Jun;63(6):1038–1045.
   PubMed Web of Science ®Google Scholar
• Dampier C, Barry V, Gross HE, et al. Initial evaluation of the pediatric PROMIS® health domains in children and adolescents with sickle cell disease. Pediatr Blood Cancer. 2016 Jun;63(6):1031–1037.
   PubMed Web of Science ®Google Scholar
• Keller S, Yang M, Treadwell MJ, et al. Sensitivity of alternative measures of functioning and wellbeing for adults with sickle cell disease: comparison of PROMIS® to ASCQ-MeSM. Health Qual Life Outcomes. 2017 Jun;15(1):117.
   PubMedGoogle Scholar
• Singh A, DasGupta M, Simpson PM, et al. Use of the new pediatric PROMIS measures of pain and physical experiences for children with sickle cell disease. Pediatr Blood Cancer. 2019 May;66(5):e27633.
   PubMed Web of Science ®Google Scholar
• Reeve BB, Edwards LJ, Jaeger BC, et al. Assessing responsiveness over time of the PROMIS. Qual Life Res. 2018 Jan;27(1):249–257.
   PubMed Web of Science ®Google Scholar
• Singh A, Dasgupta M, Simpson PM, et al. Can PROMIS domains of pain and physical functioning detect changes in health over time for children with sickle cell disease? Pediatr Blood Cancer. 2020 May;67(5):e28203.
   Web of Science ®Google Scholar
• Singh A, Panepinto JA. Clinical meaning of PROMIS pain domains for children with sickle cell disease. Blood Adv. 2019 Aug;3(15):2244–2249.
   PubMed Web of Science ®Google Scholar
• Revicki D, Hays RD, Cella D, et al. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol. 2008 Feb;61(2):102–109.
   PubMed Web of Science ®Google Scholar
• Thissen D, Liu Y, Magnus B, et al. Estimating minimally important difference (MID) in PROMIS pediatric measures using the scale-judgment method. Qual Life Res. 2016 Jan;25(1):13–23.
   PubMed Web of Science ®Google Scholar
• Adams-Graves P, Bronte-Jordan L. Recent treatment guidelines for managing adult patients with sickle cell disease: challenges in access to care, social issues, and adherence. Expert Rev Hematol. 2016 Jun;9(6):541–552.
   PubMed Web of Science ®Google Scholar
• Treadwell MJ, Hassell K, Levine R, et al. 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 Oct;30(10):902–914.
   PubMed Web of Science ®Google Scholar
• Keller SD, Yang M, Treadwell MJ, et al. Patient reports of health outcome for adults living with sickle cell disease: development and testing of the ASCQ-Me item banks. Health Qual Life Outcomes. 2014;12:125.
   PubMed Web of Science ®Google Scholar
• Cooper O, McBain H, Tangayi S, et al. Psychometric analysis of the adult sickle cell quality of life measurement information system (ACSQ-Me) in a UK population. Health Qual Life Outcomes. 2019 Apr;17(1):74.
   PubMedGoogle Scholar
• Esham KS, Rodday AM, Smith HP, et al. Assessment of health-related quality of life among adults hospitalized with sickle cell disease vaso-occlusive crisis. Blood Adv. 2020 Jan;4(1):19–27.
   PubMed Web of Science ®Google Scholar
• Varni JW, Limbers CA. The pediatric quality of life inventory: measuring pediatric health-related quality of life from the perspective of children and their parents. Pediatr Clin North Am. 2009 Aug;56(4):843–863.
   PubMed Web of Science ®Google Scholar
• PedsQL. [cited 2020 Jul 24]. Available from: www.pedsql.org
   Google Scholar
• Varni JW, Burwinkle TM, Seid M, et al. The PedsQL 4.0 as a pediatric population health measure: feasibility, reliability, and validity. Ambul Pediatr. 2003 Nov-Dec;3(6):329–341.
   PubMedGoogle Scholar
• Panepinto JA, Pajewski NM, Foerster LM, et al. The performance of the PedsQL generic core scales in children with sickle cell disease. J Pediatr Hematol Oncol. 2008 Sep;30(9):666–673.
   PubMed Web of Science ®Google Scholar
• Thornburg CD, Calatroni A, Panepinto JA. Differences in health-related quality of life in children with sickle cell disease receiving hydroxyurea. J Pediatr Hematol Oncol. 2011 May;33(4):251–254.
   PubMed Web of Science ®Google Scholar
• Dampier C, Lieff S, LeBeau P, et al. Health-related quality of life in children with sickle cell disease: a report from the comprehensive sickle cell centers clinical trial consortium. Pediatr Blood Cancer. 2010 Sep;55(3):485–494.
   PubMed Web of Science ®Google Scholar
• Panepinto JA, Torres S, Bendo CB, et al. PedsQL™ multidimensional fatigue scale in sickle cell disease: feasibility, reliability, and validity. Pediatr Blood Cancer. 2014 Jan;61(1):171–177.
   PubMed Web of Science ®Google Scholar
• Varni JW, Burwinkle TM, Katz ER, et al. The PedsQL in pediatric cancer: reliability and validity of the pediatric quality of life inventory generic core scales, multidimensional fatigue scale, and cancer module. Cancer. 2002 Apr;94(7):2090–2106.
   PubMed Web of Science ®Google Scholar
• Varni JW, Burwinkle TM, Szer IS. The PedsQL multidimensional fatigue scale in pediatric rheumatology: reliability and validity. J Rheumatol. 2004 Dec;31(12):2494–2500.
   PubMed Web of Science ®Google Scholar
• Panepinto JA, Torres S, Varni JW. Development of the PedsQL™ sickle cell disease module items: qualitative methods. Qual Life Res. 2012 Mar;21(2):341–357.
   PubMed Web of Science ®Google Scholar
• Panepinto JA, Torres S, Bendo CB, et al. PedsQL™ sickle cell disease module: feasibility, reliability, and validity. Pediatr Blood Cancer. 2013 Aug;60(8):1338–1344.
   PubMed Web of Science ®Google Scholar
• Beverung LM, Varni JW, Panepinto JA. Clinically meaningful interpretation of pediatric health-related quality of life in sickle cell disease. J Pediatr Hematol Oncol. 2015 Mar;37(2):128–133.
   PubMed Web of Science ®Google Scholar
• Panepinto JA, Paul Scott J, Badaki-Makun O, et al. Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ sickle cell disease module. Health Qual Life Outcomes. 2017 Jun;15(1):124.
   PubMedGoogle Scholar
• Alonso J, Ferrer M, Gandek B, et al. Health-related quality of life associated with chronic conditions in eight countries: results from the international quality of life assessment (IQOLA) Project. Qual Life Res. 2004 Mar;13(2):283–298.
   PubMed Web of Science ®Google Scholar
• McClish DK, Penberthy LT, Bovbjerg VE, et al. Health related quality of life in sickle cell patients: the PiSCES project. Health Qual Life Outcomes. 2005;3:50.
   PubMedGoogle Scholar
• Dampier C, LeBeau P, Rhee 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 Feb;86(2):203–205.
   PubMed Web of Science ®Google Scholar
• Ware JE, Sherbourne CD, The MOS. 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992 Jun;30(6):473–483.
   PubMed Web of Science ®Google Scholar
• Ahmed AE, Alaskar AS, Al-Suliman AM, et al. Health-related quality of life in patients with sickle cell disease in Saudi Arabia. Health Qual Life Outcomes. 2015;13:183.
   PubMedGoogle Scholar
• Asnani MR, Lipps GE, Reid ME. Validation of the SF-36 in Jamaicans with sickle-cell disease. Psychol Health Med. 2009 Oct;14(5):606–618.
   PubMed Web of Science ®Google Scholar
• Ojelabi AO, Bamgboye AE, Ling J. Preference-based measure of health-related quality of life and its determinants in sickle cell disease in Nigeria. PLoS One. 2019;14(11):e0223043.
   PubMed Web of Science ®Google Scholar
• Schalet BD, Revicki DA, Cook KF, et al. Establishing a common metric for physical function: linking the HAQ-DI and SF-36 PF subscale to PROMIS(®) physical function. J Gen Intern Med. 2015 Oct;30(10):1517–1523.
   PubMed Web of Science ®Google Scholar
• Van Der Wees PJ, Nijhuis-Van Der Sanden MW, Ayanian JZ, et al. Integrating the use of patient-reported outcomes for both clinical practice and performance measurement: views of experts from 3 countries. Milbank Q. 2014 Dec;92(4):754–775.
   PubMed Web of Science ®Google Scholar
• Kauf TL, Coates TD, Huazhi L, et al. The cost of health care for children and adults with sickle cell disease. Am J Hematol. 2009 Jun;84(6):323–327.
   PubMed Web of Science ®Google Scholar
• Panepinto JA, Bonner M. Health-related quality of life in sickle cell disease: past, present, and future. Pediatr Blood Cancer. 2012 Aug;59(2):377–385.
   PubMed Web of Science ®Google Scholar
• Administration USFaD. The voice of the patient: sickle cell report 2014. Available from: https://www.fda.gov/industry/prescription-drug-user-fee-amendments/voice-patient-series-reports-fdas-patient-focused-drug-development-initiative
   Google Scholar
• Snyder CF, Aaronson NK, Choucair AK, et al. Implementing patient-reported outcomes assessment in clinical practice: a review of the options and considerations. Qual Life Res. 2012 Oct;21(8):1305–1314.
   PubMed Web of Science ®Google Scholar
• Appiah-Kubi A, Lipton JM. The long road to the cure of sickle cell anemia: reflections on race and medicine in America. Pediatr Blood Cancer. 2012 Apr;58(4):485–486.
   PubMed Web of Science ®Google Scholar
• Bediako SM, Lanzkron S, Diener-West M, et al. The measure of sickle cell stigma: initial findings from the improving patient outcomes through respect and trust study. J Health Psychol. 2016 May;21(5):808–820.
   PubMed Web of Science ®Google Scholar
• Scharff DP, Mathews KJ, Jackson P, et al. More than Tuskegee: understanding mistrust about research participation. J Health Care Poor Underserved. 2010 Aug;21(3):879–897.
   PubMed Web of Science ®Google Scholar
• Adeyemo TA, Ojewunmi OO, Diaku-Akinwumi IN, et al. 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 Jul;62(7):1245–1251.
   PubMed Web of Science ®Google Scholar
• Bulgin D, Tanabe P, Jenerette C. Stigma of sickle cell disease: a systematic review. Issues Ment Health Nurs. 2018 Aug;39(8):675–686.
   PubMed Web of Science ®Google Scholar
• Smith LA, Oyeku SO, Homer C, et al. Sickle cell disease: a question of equity and quality. Pediatrics. 2006 May;117(5):1763–1770.
   PubMed Web of Science ®Google Scholar
• Wailoo K. Sickle cell disease - a history of progress and peril. N Engl J Med. 2017 March;376(9):805–807.
   PubMed Web of Science ®Google Scholar
• Telen MJ, Malik P, Vercellotti GM. Therapeutic strategies for sickle cell disease: towards a multi-agent approach. Nat Rev Drug Discov. 2019 Feb;18(2):139–158.
   PubMed Web of Science ®Google Scholar
• Niihara Y, Miller ST, Kanter J, et al. A phase 3 trial of l-glutamine in sickle cell disease. N Engl J Med. 2018 Jul;379(3):226–235.
   PubMed Web of Science ®Google Scholar
• Vichinsky E, Hoppe CC, Ataga KI, et al. A phase 3 randomized trial of voxelotor in sickle cell disease. N Engl J Med. 2019 Aug;381(6):509–519.
   PubMed Web of Science ®Google Scholar
• Ataga KI, Kutlar A, Kanter J, et al. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017 feb;376(5):429–439.
   PubMed Web of Science ®Google Scholar
• Badawy SM, Thompson AA, Liem RI. Beliefs about hydroxyurea in youth with sickle cell disease. Hematol Oncol Stem Cell Ther. 2018 Sep;11(3):142–148.
   PubMedGoogle Scholar
• Walsh KE, Cutrona SL, Kavanagh PL, et al. Medication adherence among pediatric patients with sickle cell disease: a systematic review. Pediatrics. 2014 Dec;134(6):1175–1183.
   PubMed Web of Science ®Google Scholar
• Williams TN. Sickle cell disease in sub-Saharan Africa. Hematol Oncol Clin North Am. 2016 Apr;30(2):343–358.
   PubMed Web of Science ®Google Scholar
• Smart LR, Hernandez AG, Ware RE. Sickle cell disease: translating clinical care to low-resource countries through international research collaborations. Semin Hematol. 2018 April;55(2):102–112.
   PubMed Web of Science ®Google Scholar
• Farrell AT, Panepinto J, Desai AA, et al. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Adv. 2019 Dec;3(23):4002–4020.
   PubMed Web of Science ®Google Scholar