234
Views
0
CrossRef citations to date
0
Altmetric
Review

Assessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies

& ORCID Icon
Pages 2221-2229 | Published online: 01 Oct 2021

References

  • Eaton WA, Bunn HF. Treating sickle cell disease by targeting HbS polymerization. Blood. 2017;129(20):2719–2726. doi:10.1182/blood-2017-02-765891
  • Pecker LH, Lanzkron S. Sickle cell disease. Ann Intern Med. 2021;174(1):Itc1–Itc16. doi:10.7326/aitc202101190
  • Pecker LH, Naik RP. The current state of sickle cell trait: implications for reproductive and genetic counseling. Blood. 2018;132(22):2331–2338. doi:10.1182/blood-2018-06-848705
  • Niihara Y, Miller ST, Kanter J, et al. A phase 3 trial of l-glutamine in sickle cell disease. N Engl J Med. 2018;379(3):226–235. doi:10.1056/NEJMoa1715971
  • Vichinsky E, Hoppe CC, Ataga KI, et al. A phase 3 randomized trial of voxelotor in sickle cell disease. N Engl J Med. 2019;381(6):509–519. doi:10.1056/NEJMoa1903212
  • Su ZT, Segal JB, Lanzkron S, Ogunsile FJ. National trends in hydroxyurea and opioid prescribing for sickle cell disease by office-based physicians in the United States, 1997–2017. Pharmacoepidemiol Drug Saf. 2019;28(9):1246–1250. doi:10.1002/pds.4860
  • Gluckman E, Cappelli B, Bernaudin F, et al. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood. 2017;129(11):1548–1556. doi:10.1182/blood-2016-10-745711
  • Krishnamurti L. Hematopoietic cell transplantation for sickle cell disease: state of the art. Expert Opin Biol Ther. 2007;7(2):161–172. doi:10.1517/14712598.7.2.161
  • Vrettou C, Kakourou G, Mamas T, Traeger-Synodinos J. Prenatal and preimplantation diagnosis of hemoglobinopathies. Int J Lab Hematol. 2018;40(S1):74–82. doi:10.1111/ijlh.12823
  • Thompson WE, Eriator I. Pain control in sickle cell disease patients: use of complementary and alternative medicine. Pain Med. 2014;15(2):241–246. doi:10.1111/pme.12292
  • Harris KM, Dadekian JN, Abel RA, et al. Increasing educational attainment in adolescents with sickle cell disease. Soc Work Public Health. 2019;34(6):468–482. doi:10.1080/19371918.2019.1629142
  • Ross D, Bakshi N, Khemani K, Sinha C, Loewenstein G, Krishnamurti L. What are the expectations of patients in decision making process for disease modifying therapies for sickle cell disease: do they care about shared decision making? Blood. 2016;128(22):5968. doi:10.1182/blood.V128.22.5968.5968
  • Bakshi N, Sinha CB, Ross D, Khemani K, Loewenstein G, Krishnamurti L. Proponent or collaborative: physician perspectives and approaches to disease modifying therapies in sickle cell disease. PLoS One. 2017;12(7):e0178413. doi:10.1371/journal.pone.0178413
  • Badawy SM, Thompson AA, Liem RI. Technology access and smartphone app preferences for medication adherence in adolescents and young adults with sickle cell disease. Pediatr Blood Cancer. 2016;63(5):848–852. doi:10.1002/pbc.25905
  • Britto MT, DeVellis RF, Hornung RW, DeFriese GH, Atherton HD, Slap GB. Health care preferences and priorities of adolescents with chronic illnesses. Pediatrics. 2004;114(5):1272–1280. doi:10.1542/peds.2003-1134-L
  • Britto MT, Slap GB, DeVellis RF, et al. Specialists understanding of the health care preferences of chronically ill adolescents. J Adolesc Health. 2007;40(4):334–341. doi:10.1016/j.jadohealth.2006.10.020
  • Fertleman CR, Gallagher A, Rossiter MA. Evaluation of fast track admission policy for children with sickle cell crises: questionnaire survey of parents’ preferences. BMJ. 1997;315(7109):650. doi:10.1136/bmj.315.7109.650
  • Junghans-Rutelonis AN, Moquist KL, Blaylark RM, Anderson N, Brown ML. Incorporating integrative medicine and patient preferences into a pilot interdisciplinary sickle cell wellness clinic. Complement Ther Med. 2020;49:102333. doi:10.1016/j.ctim.2020.102333
  • Goetz AM, Wagener MM, Miller JM, Muder RR. Risk of infection due to central venous catheters: effect of site of placement and catheter type. Infect Control Hosp Epidemiol. 1998;19(11):842–845. doi:10.2307/30141562
  • Lorente L, Henry C, Martin MM, Jimenez A, Mora ML. Central venous catheter-related infection in a prospective and observational study of 2595 catheters. Crit Care. 2005;9(6):R631–R635. doi:10.1186/cc3824
  • Trottier SJ, Veremakis C, Obrien J, Auer AI. Femoral deep-vein thrombosis associated with central venous catheterization - results from a prospective, randomized trial. Crit Care Med. 1995;23(1):52–59. doi:10.1097/00003246-199501000-00011
  • Yeral M, Boga C, Oguzkurt L, Asma S, Kasar M, Kozanoglu I. Short-term central venous catheter complications in patients with sickle cell disease who undergo apheresis. J Thromb Thrombolysis. 2014;37(2):97–101. doi:10.1007/s11239-013-0914-z
  • Park PJ, Cole SS, Bercu ZL, Martin J, Newsome J. Understanding patient preferences for femoral central venous catheterization among patients with sickle cell disease. J Vasc Access. 2018;19(2):199–200. doi:10.5301/jva.5000794
  • Kodish E, Lantos J, Stocking C, Singer PA, Siegler M, Johnson FL. Bone marrow transplantation for sickle cell disease. A study of parents’ decisions. N Engl J Med. 1991;325(19):1349–1353. doi:10.1056/NEJM199111073251905
  • Bai F, Ling J, Esoimeme G, Yao L, Wang M, Huang J, et al. A systematic review of questionnaires about patient's values and preferences in clinical practice guidelines. Patient Prefer Adherence. 2018;12:2309–23. doi:10.2147/PPA.S177540
  • van Besien K, Koshy M, Anderson-Shaw L, et al. Allogeneic stem cell transplantation for sickle cell disease. A study of patients’ decisions. Bone Marrow Transplant. 2001;28(6):545–549. doi:10.1038/sj.bmt.1703208
  • Meier ER, Dioguardi JV, Kamani N. Current attitudes of parents and patients toward hematopoietic stem cell transplantation for sickle cell anemia. Pediatr Blood Cancer. 2015;62(7):1277–1284. doi:10.1002/pbc.25446
  • Chang EM, Saigal CS, Raldow AC. Explaining health state utility assessment. JAMA. 2020;323(11):1085–1086. doi:10.1001/jama.2020.0656
  • Stiggelbout AM, de Haes JC. Patient preference for cancer therapy: an overview of measurement approaches. J Clin Oncol. 2001;19(1):220–230. doi:10.1200/jco.2001.19.1.220
  • Brett Hauber A, Fairchild AO, Reed johnson F. Quantifying benefit-risk preferences for medical interventions: an overview of a growing empirical literature. Appl Health Econ Health Policy. 2013;11(4):319–329. doi:10.1007/s40258-013-0028-y
  • Ojelabi AO, Bamgboye AE, Ling J, Stillwaggon E. Preference-based measure of health-related quality of life and its determinants in sickle cell disease in Nigeria. PLoS One. 2019;14(11):e0223043. doi:10.1371/journal.pone.0223043
  • Oluwole EO, Adeyemo TA, Osanyin GE, Odukoya OO, Kanki PJ, Afolabi BB. Feasibility and acceptability of early infant screening for sickle cell disease in Lagos, Nigeria-a pilot study. PLoS One. 2020;15(12):e0242861. doi:10.1371/journal.pone.0242861
  • O’Brien SH, Hankins JS. Decision analysis of treatment strategies in children with severe sickle cell disease. J Pediatr Hematol Oncol. 2009;31(11):873–878. doi:10.1097/MPH.0b013e3181b83cab
  • Nietert PJ, Abboud MR, Silverstein MD, Jackson SM. Bone marrow transplantation versus periodic prophylactic blood transfusion in sickle cell patients at high risk of ischemic stroke: a decision analysis. Blood. 2000;95(10):3057–3064. doi:10.1182/blood.V95.10.3057
  • Neumann PJ, Goldie SJ, Weinstein MC. Preference-based measures in economic evaluation in health care. Annu Rev Public Health. 2000;21:587–611. doi:10.1146/annurev.publhealth.21.1.587
  • Oyeku SO, Driscoll MC, Cohen HW, et al. Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease. Pediatr Blood Cancer. 2013;60(4):653–658. doi:10.1002/pbc.24381
  • Creary S, Zickmund S, Ross D, Krishnamurti L, Bogen DL. Hydroxyurea therapy for children with sickle cell disease: describing how caregivers make this decision. BMC Res Notes. 2015;8:372. doi:10.1186/s13104-015-1344-0
  • Hawkins LM, Sinha CB, Ross D, et al. Patient and family experience with chronic transfusion therapy for sickle cell disease: a qualitative study. BMC Pediatr. 2020;20(1):172. doi:10.1186/s12887-020-02078-w
  • Khemani K, Ross D, Sinha C, Haight A, Bakshi N, Krishnamurti L. Experiences and decision making in hematopoietic stem cell transplant in sickle cell disease: patients’ and caregivers’ perspectives. Biol Blood Marrow Transplant. 2018;24(5):1041–1048. doi:10.1016/j.bbmt.2017.11.018
  • Schulz GL, Kelly KP, Holtmann M, Armer JM. Navigating decisional conflict as a family when facing the decision of stem cell transplant for a child or adolescent with sickle cell disease. Patient Educ Couns. 2020;104(5):1086–1093. doi:10.1016/j.pec.2020.10.011
  • Krishnamurti L. Hematopoietic CellTransplantation for Sickle Cell Disease. Review. Frontiers in Pediatrics. 2021-January-05 2021;8(846). doi:10.3389/fped.2020.551170
  • Sinha CB, Bakshi N, Ross D, Loewenstein G, Krishnamurti L. Primary caregiver decision-making in hematopoietic cell transplantation and gene therapy for sickle cell disease. Pediatr Blood Cancer. 2021;68(1):e28749. doi:10.1002/pbc.28749
  • Utrankar A, Mayo-Gamble TL, Allen W, et al. Technology use and preferences to support clinical practice guideline awareness and adherence in individuals with sickle cell disease. J Am Med Inform Assoc. 2018;25(8):976–988. doi:10.1093/jamia/ocy036
  • Gooberman-Hill R. Qualitative approaches to understanding patient preferences. Patient. 2012;5(4):215–223. doi:10.2165/11633720-000000000-00000
  • Rolfe DE, Ramsden VR, Banner D, Graham ID. Using qualitative health research methods to improve patient and public involvement and engagement in research. Res Invol Engage. 2018;4:49. doi:10.1186/s40900-018-0129-8
  • Bastemeijer CM, Voogt L, van Ewijk JP, Hazelzet JA. What do patient values and preferences mean? A taxonomy based on a systematic review of qualitative papers. Patient Educ Couns. 2017;100(5):871–881. doi:10.1016/j.pec.2016.12.019