Translating scientific advances to improved outcomes for children with sickle cell disease: a timely opportunity† ‡
Jean L. Raphael MD, MPH
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Search for more papers by this authorCorresponding Author
Patricia L. Kavanagh MD
Department of Pediatrics, Boston University School of Medicine, Boston Medical Center, Boston, Massachusetts
Division of General Pediatrics, Boston University School of Medicine/Boston Medical Center, E. Newton Street, Vose Hall, 3rd Floor, Boston, MA 02118.===Search for more papers by this authorC. Jason Wang MD, PhD
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Department of Community Health Sciences, Boston University School of Public Health, Boston, Massachusetts
Search for more papers by this authorBrigitta U. Mueller MD, MHCM
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Search for more papers by this authorBarry Zuckerman MD
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Search for more papers by this authorJean L. Raphael MD, MPH
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Search for more papers by this authorCorresponding Author
Patricia L. Kavanagh MD
Department of Pediatrics, Boston University School of Medicine, Boston Medical Center, Boston, Massachusetts
Division of General Pediatrics, Boston University School of Medicine/Boston Medical Center, E. Newton Street, Vose Hall, 3rd Floor, Boston, MA 02118.===Search for more papers by this authorC. Jason Wang MD, PhD
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Department of Community Health Sciences, Boston University School of Public Health, Boston, Massachusetts
Search for more papers by this authorBrigitta U. Mueller MD, MHCM
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Search for more papers by this authorBarry Zuckerman MD
Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer Center, Houston, Texas
Search for more papers by this authorConflict of interest: Nothing to declare.
Jean L. Raphael and Patricia L. Kavanagh contributed equally to this work.
Abstract
Despite the recent advances made in the care of children with sickle cell disease (SCD), premature mortality, especially among older children and young adults, remains a hallmark of this disease. The lack of survival gains highlights the translational gap of implementing innovations found efficacious in the controlled trial setting into routine clinical practice. Health services research (HSR) examines the most effective ways to finance, organize, and deliver high quality care in an equitable manner. To date, HSR has been underutilized as a means to improve the outcomes for children with SCD. Emerging national priorities in health care delivery, new sources of funding, and evolving electronic data collection systems for patients with SCD have provided a unique opportunity to overcome the translational gap in pediatric SCD. The purpose of this article is to provide a comprehensive HSR agenda to create patient-specific evidence of clinical effectiveness for interventions used in the routine care setting, understand the barriers faced by clinicians to providing high quality care, assess and improve the interactions of patients with the health care system, and measure the quality of care delivered to increase survival for all children and young adults with SCD. Pediatr Blood Cancer 2011;56:1005–1008. © 2011 Wiley-Liss, Inc.
REFERENCES
- 1 Quinn CT, Rogers ZR, McCavit TL, et al. Improved survival of children and adolescents with sickle cell disease. Blood 2010; 115: 3447–3452.
- 2 Yanni E, Grosse SD, Yang Q, et al. Trends in pediatric sickle cell disease-related mortality in the United States, 1983–2002. J Pediatr 2009; 154: 541–545.
- 3 Raphael JL, Dietrich CL, Whitmire D, et al. Healthcare utilization and expenditures for low income children with sickle cell disease. Pediatr Blood Cancer 2009; 52: 263–267.
- 4 McCarville MB, Goodin GS, Fortner G, et al. Evaluation of a comprehensive transcranial Doppler screening program for children with sickle cell anemia. Pediatr Blood Cancer 2008; 50: 818–821.
- 5 Raphael JL, Shetty PB, Liu H, et al. A critical assessment of transcranial Doppler screening rates in a large pediatric sickle cell center: Opportunities to improve healthcare quality. Pediatr Blood Cancer 2008; 51: 647–651.
- 6 Smith LA, Oyeku SO, Homer C, et al. Disease: A question of equity and quality. Pediatrics 2006; 117: 1763–1770.
- 7 Lenfant C. Clinical research to clinical practice—Lost in translation? N Engl J Med 2003; 349: 868–874.
- 8 Agency for Healthcare Research and Quality. What is AHRQ? Rockville, MD; 2002. Available at: http://www.ahrq.gov/about/whatis.htm. Accessed June 22, 2010.
- 9 Davis PB. Cystic fibrosis since 1938. Am J Respir Crit Care Med 2006; 173: 475–482.
- 10 Lai HJ, Cheng Y, Farrell PM. The survival advantage of patients with cystic fibrosis diagnosed through neonatal screening: Evidence from the United States Cystic Fibrosis Foundation Registry Data. J Pediatr 2005; 147: S57–S63.
- 11 Konstan MW, Schluchter MD, Xue W, et al. Clinical use of ibuprofen is associated with slower FEV1 decline in children with cystic fibrosis. Am J Respir Crit Care Med 2007; 176: 1084–1089.
- 12 Kraynack NC, McBride JT. Improving care at cystic fibrosis centers through quality improvement. Semin Respir Crit Care Med 2009; 30: 547–558.
- 13 Cystic Fibrosis Foundation. Cystic Fibrosis Foundation Patient Registry: Annual Data Report 2008. Bethesda, MD; 2008. Available at: http://www.cff.org/UploadedFiles/research/ClinicalResearch/2008-Patient-Registry-Report.pdf. Accessed August 26, 2010.
- 14 Nabel EG, Shurin SB. A recommitment to sickle cell disease research. Blood 2008; 111: 4852–4853.
- 15 Institute of Medicine, Committee on Quality Health Care in America. Crossing the quality chasm: A new health system for the 21st century. Washington, DC: The National Academy Press; 2001.
- 16 Institute of Medicine, Committee on Redesigning Health Insurance Performance Measures Payment and Performance Improvement Programs. Performance measurement: Accelerating improvement. Washington, DC: National Academies Press; 2006.
- 17 Institute of Medicine. Unequal treatment: Confronting racial and ethnic disparities in health care. Washington, DC: The National Academy Press; 2002.
- 18 Sox HC, Greenfield S. Comparative effectiveness research: A report from the Institute of Medicine. Ann Intern Med 2009; 151: 203–205.
- 19 Obey D, Frank B, Gordon B, et al. American Recovery and Reinvestment Act of 2009. 111th Congress of the United States; 2009.
- 20 Sung NS, Crowley WF Jr, Genel M, et al. Central challenges facing the National Clinical Research Enterprise. JAMA 2003; 289: 1278–1287.
- 21 Vichinsky EP, Neumayr LD, Earles AN, et al. Causes and outcomes of the acute chest syndrome in sickle cell disease. N Engl J Med 2000; 342: 1855–1865.
- 22 Marti-Carvajal A, Conterno L, Knight-Madden J. Antibiotics for treating acute chest syndrome in people with sickle cell disease. Cochrane Database Syst Rev 2007; Issue 2. Art. No.: CD006110. DOI: 10.1002/14651858.CD006110.pub2
- 23 Knight-Madden J, Hambleton I. Inhaled brochodilators for acute chest syndrome in people with sickle cell disease. Cochrane Database Syst Rev 2003; Issue 3. Art. No.: CD003733. DOI: 10.1002/14651858.CD003733
- 24 Dunlop R, Bennett K. Pain management for sickle cell disease in children and adults. Cochrane Database Syst Rev 2006; Issue 2. Art. No.: CD003350. DOI: 10.1002/14651858.CD003350.pub2
- 25 Okomo U, Meremikwu M. Fluid replacement therapy for acute episodes of pain in people with sickle cell disease. Cochrane Database Syst Rev 2007; Issue 2. Art. No.: CD005406. DOI: 10.1002/14651858.CD005406.pub2
- 26 Manci EA, Culberson DE, Yang Y-M, et al. Causes of death in sickle cell disease: An autopsy study. Br J Haematol 2003; 123: 359–365.
- 27 Gaston MH, Verter JI, Woods G, et al. Prophylaxis with oral penicillin in children with sickle cell anemia: A randomized trial. N Engl J Med 1986; 314: 1593–1599.
- 28 Adamkiewicz TV, Silk BJ, Howgate J, et al. Effectiveness of the 7-valent pneumococcal conjugate vaccine in children with sickle cell disease in the first decade of life. Pediatrics 2008; 121: 562–569.
- 29 Pilishvili T, Zell ER, Farley MM, et al. Risk factors for invasive pneumococcal disease in children in the era of conjugate vaccine use. Pediatrics 2010; 126: e9–e17.
- 30 NHLBI, CDC Launch Surveillance and Research Program for Inherited Blood Disorders. NIH News. February 18, 2010. Available at: http://www.nih.gov/news/health/feb2010/nhlbi-18.htm. Accessed on May 25, 2010.
- 31 Ohene-Frempong K, Weiner SJ, Sleeper LA, et al. Cerebrovascular accidents in sickle cell disease: Rates and risk factors. Blood 1998; 91: 288–294.
- 32 Adams RJ, McKie VC, Carl E, et al. Long-term stroke risk in children with sickle cell disease screened with transcranial Doppler. Ann Neurol 1997; 42: 699–704.
- 33 Adams RJ, Brambilla D. Optimizing primary stroke prevention in sickle cell anemia trial I. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease [see comment]. N Engl J Med 2005; 353: 2769–2778.
- 34 Ferster A, Vermylen C, Cornu G, et al. Hydroxyurea for treatment of severe sickle cell anemia: A pediatric clinical trial. Blood 1996; 88: 1960–1964.
- 35 Hankins JS, Ware RE, Rogers ZR, et al. Long-term hydroxyurea therapy for infants with sickle cell anemia: The HUSOFT extension study. Blood 2005; 106: 2269–2275.
- 36 Thompson BW, Miller ST, Rogers ZR, et al. The pediatric hydroxyurea phase III clinical trial (BABY HUG): Challenges of study design. Pediatr Blood Cancer 2010; 54: 250–255.
- 37 Ware RE, Rees RC, Sarnaik SA, et al. Renal function in infants with sickle cell anemia: Baseline data from the BABY HUG trial. J Pediatr 2010; 156: 66–70, e61.
- 38 Britto MT, Garrett JM, Dugliss MAJ, et al. Preventive services received by adolescents with cystic fibrosis and sickle cell disease. Arch Pediatr Adolesc Med 1999; 153: 27–32.
- 39 Telfair J, Haque A, Etienne M, et al. Rural/urban differences in access to and utilization of services among people in Alabama with sickle cell disease. Public Health Rep 2003; 118: 27–36.
- 40 National Heart Lung and Blood Institute. The management of sickle cell disease, NIH Publication No. 02-2117. Bethesda, MD: National Institutes of Health; 2002.
- 41 Section on Hematology/Oncology Committee on Genetics, American Academy of Pediatrics. Health supervision for children with sickle cell disease [see comment]. Pediatrics 2002; 109: 526–535.
- 42 Powell-Cope GM, Luther S, Neugaard B, et al. Provider-perceived barriers and facilitators for ischaemic heart disease (IHD) guideline adherence. J Eval Clin Pract 2004; 10: 227–239.
- 43 el-Kebbi I, Ziemer D, Gallina D, et al. Diabetes in urban African-Americans. XV. Identification of barriers to provider adherence to management protocols. Diabetes Care 1999; 2: 1617–1620.
- 44 Homer CJ, Klatka K, Romm D, et al. A review of the evidence for the medical home for children with special health care needs. Pediatrics 2008; 122: e922–e937.
- 45 Raphael JL, Zhang Y, Liu H, et al. Association of medical home care and disparities in emergency care utilization among children with special health care needs. Acad Pediatr 2009; 9: 242–248.
- 46 Beal AC, Doty MM, Hernandez SE, et al. Closing the divide: How medical homes promote equity in health care: Results from The Commonwealth Fund 2006. Health care quality survey. New York, NY: The Commonwealth Fund; 2007.
- 47 Wells KJ, Battaglia TA, Dudley DJ, et al. Patient navigation: State of the art or is it science? Cancer 2008; 113: 1999–2010.
- 48 Hede K. Agencies look to patient navigators to reduce cancer care disparities. J Natl Cancer Inst 2006; 98: 157–159.
- 49 Freeman HP. Poverty, culture, and social injustice: Determinants of cancer disparities. CA Cancer J Clin 2004; 54: 72–77.
- 50 Nash D, Azeez S, Vlahov D, et al. Evaluation of an intervention to increase screening colonoscopy in an urban public hospital setting. J Urban Health 2006; 83: 231–243.
- 51 Ferrante JM, Chen PH, Kim S. The effect of patient navigation on time to diagnosis, anxiety, and satisfaction in urban minority women with abnormal mammograms: A randomized controlled trial. J Urban Health 2008; 85: 114–124.
- 52 Flores G, Bridon C, Torres S, et al. Improving asthma outcomes in minority children: A randomized, controlled trial of parent mentors. Pediatrics 2009; 124: 1522–1532.
- 53 Brook R, McGlynn E, Cleary P. Measuring quality of care. N Engl J Med 1996; 335: 966.
- 54 Ashton CM, Kuykendall DH, Johnson ML, et al. The association between the quality of inpatient care and early readmission. Ann Intern Med 1995; 122: 415–421.
- 55 Cheng SH, Wang CJ, Lin J-L, et al. Adherence to quality indicators and survival in breast cancer patients. Med Care 2008; 47: 217–225.
- 56 Wang CJ, Kavanagh PL, Little AL, et al. Development of quality indicators for the management of sickle cell disease in children: Results of an expert panel process. Pediatrics 2011; In press.
