Pediatric hematology providers on referral for transplant evaluation for sickle cell disease: A regional perspective

Journal of Pediatric Hematology/Oncology

Volumn 36, Issue 7, 2014, Pages 566-571

  Mikles, Bethany ^a   Bhatia, Monica ^b   Oyeku, Suzette O ^d   Jin, Zhezhen ^c   Green, Nancy S ^b  

^a NAVAL MEDICAL CENTER PORTSMOUTH   (United States)

^b Department of Medicine   (United States)

^c Department of Medicine   (United States)

^d MONTEFIORE MEDICAL CENTER   (United States)

Author keywords

Provider perspectives; Sickle cell disease; Transplantation

Indexed keywords

ADOLESCENT; ADULT; AGE DISTRIBUTION; ARTICLE; BETA THALASSEMIA; CHILD; CLINICAL EVALUATION; CONTROLLED STUDY; EVENT FREE SURVIVAL; FEMALE; HEALTH CARE PERSONNEL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHY; HUMAN; MAJOR CLINICAL STUDY; MALE; MIDDLE AGED; OUTCOME ASSESSMENT; OVERALL SURVIVAL; PATIENT REFERRAL; PRESCHOOL CHILD; SIBLING; SICKLE CELL ANEMIA; SURVIVAL RATE; TREATMENT INDICATION; TREATMENT RESPONSE; YOUNG ADULT; BLOOD TRANSFUSION; CROSS-SECTIONAL STUDY; DISEASE FREE SURVIVAL; HEALTH CARE SURVEY; HEMATOLOGY; INFANT; NEWBORN; NEWBORN SCREENING; PEDIATRICS; PROFESSIONAL PRACTICE; STATISTICS; UNITED STATES; UTILIZATION REVIEW;

ADULT; ANEMIA, SICKLE CELL; BETA-THALASSEMIA; BLOOD TRANSFUSION; CHILD; CHILD, PRESCHOOL; CROSS-SECTIONAL STUDIES; DISEASE-FREE SURVIVAL; FEMALE; HEALTH CARE SURVEYS; HEMATOLOGY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; INFANT, NEWBORN; MALE; MIDDLE AGED; NEONATAL SCREENING; NEW ENGLAND; PEDIATRICS; PROFESSIONAL PRACTICE; REFERRAL AND CONSULTATION; YOUNG ADULT;

EID: 84910111432     PISSN: 10774114     EISSN: 15363678     Source Type: Journal    
DOI: 10.1097/MPH.0000000000000147     Document Type: Article

References (35)

1. Bhatia M, Walters MC. Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplant. 2008;41:109-117.
2. Bolanos-Meade J, Brodsky RA. Blood and marrow transplantation for sickle cell disease: overcoming barriers to success. Curr Opin Oncol. 2009;21:158-161.
3. Gaziev J, Lucarelli G. Stem cell transplantation for hemoglobinopathies. Curr Opin Pediatr. 2003;15:24-31.
4. Friedrich MJ. Advances reshaping sickle cell therapy. JAMA. 2011;305:239-241.
5. Hoppe CC, Walters MC. Bone marrow transplantation in sickle cell anemia. Curr Opin Oncol. 2001;13:85-90.
6. Panepinto JA, Walters MC, Carreras J, et al. Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Non-Malignant Marrow Disorders Working Committee, Center for International Blood and Marrow Transplant Research. Br J Haematol. 2007;137:479-485.
7. Johnson FL, Lock AT, Gockerman J, et al. Bone-marrow transplantation in a patient with sickle-cell anemia. N Engl J Med. 1984;311:780-783.
8. Walters MC, Patience M, Leisenring W, et al. Bone marrow transplantation for sickle cell disease. N Engl J Med. 1996;335:369-376.
9. Bernaudin F, Socie G, Kuentz M, et al. Long-term results of related, myeloablative stem cell transplantation to cure sickle cell disease. Blood. 2007;110:2749-2756.
10. Vermylen C, Cornu G, Ferster A, et al. Haematopoetic stem cell transplantation for Sickle Cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant. 1998;22:1-6.
11. Krishnamurti L, Kharbanda S, Biernacki M, et al. Stable longterm donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2008;14:1270-1278.
12. Hsieh MM, Kang EM, Fitzhugh CD, et al. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009;361:2309-2317.
13. Quinn CT, Rogers ZR, McCavit TL, et al. Improved survival of children and adolescents with sickle cell disease. Blood. 2010;115:3447-3452.
14. Lucarelli G, Gaziev J, Isgrò A, et al. Allogeneic cellular gene therapy in hemoglobinopathies-evaluation of hematopoietic SCT in sickle cell anemia. Bone Marrow Transplant. 2012;47:227-230.
15. US Transplant Data by Disease Report. Available at: http://bloodcell.transplant.hrsa.gov/research/transplant-data/us-tx-data/Data-by-Disease/national.aspx. Accessed September 15, 2013).
16. Buchanan G, Vichinsky E, Krishnamurti L, et al. Severe sickle cell disease-pathophysiology and therapy. Biol Blood Marrow Transplant. 2010;16(suppl):S64-S67.
17. Shenoy S. Hematopoietic stem cell transplantation for sickle cell disease: current practice and emerging trends. Blood. 2012;120:4285-4291.
18. Hassell KL. Population estimates of sickle cell disease in the US. Am J Prev Med. 2010;38(suppl):S512-S521. Available at: http://genes-r-us.uthscsa.edu. Accessed September 27, 2013.
19. Vichinsky EP, MacKlin EA, Waye JS, et al. Changes in the epidemiology of thalassemia in North America: a new minority disease. Pediatrics. 2005;116:e818-e825.
20. NYMAC resources. Available at: http://www.wadsworth.org/newborn/nymac/. Accessed September 27, 2013.
21. HRSA Regional genetics and Newborn Screening Collaboratives (National Newborn Screening and Global Resource Center). Available at: http://genes-r-us.uthscsa.edu/resources/genetics/StatePages/genetic-region-map.htm. Accessed September 27, 2013.
22. Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children with Sickle Cell Disease (BMT CTN 601). Available at: http://www.clinicaltrials.gov/ct2/show/NCT00745420. Accessed September 27, 2013.
23. Bolanos-Meade J, Fuchs E, Luznic L, et al. HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood. 2012;120:4285-4291.
24. Kodish E, Lantos J, Stocking C. Bone marrow transplantation for Sickle Cell Disease-a study of parents' decisions. New Engl J Med. 1991;325:1349-1353.
25. Roth M, Krystal J, Manwani D, et al. Stem cell transplantation for children with sickle cell anemia: parent and patient interest. Biol Blood Marrow Transplant. 2012;18:1709-1715.
26. 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;28:545-549.
27. Hansbury EN, Schultz WH, Ware RE, et al. Bone Marrow transplant options and preferences in a sickle cell anemia cohort on chronic transfusions. Pediatr Blood Cancer. 2012;58:611-615.
28. McLeod CC, Klaubunde CN, Willis GB, et al. Health care provider surveys in the United States, 2000-2010: a review. Eval Health Prof. 2013;36:106-126.
29. Asch DA, Jedrziewski MK, Christakis NA. Response rates to mail surveys published in medical journals. J Clin Epidemiol. 1997;50:1129-1136.
30. Cummings SM, Savitz LA, Konrad TR. Reported response rates to mailed physician questionnaires. Health Serv Res. 2001;35:1347-1355.
31. Ziegenfuss JY, Shah ND, Fan JF, et al. Patient characteristics of provider survey respondents: no evidence of nonresponse bias. Eval Health Prof. 2012;35:507-516.
32. Barton J, Bain C, Hennekens CH, et al. Characteristics of respondents and non-respondents to a mailed questionnaire. Am J Public Health. 1980;70:823-825.
33. Kellerman SE, Herold J. Physician response to surveys: a review of the literature. Am J Prev Med. 2001;20:61-67.
34. Beebe TJ, Locke GR III, Barnes SA, et al. Mixing web and mail methods in a survey of physicians. Health Serv Res. 2007;42:1219-1234.
35. Omondi NA, Ferguson SE, Majhail NS, et al. Barriers to hematopoietic cell transplantation clinical trial participation of African American and black youth with sickle cell disease and their parents. J Pediatr Hematol Oncol. 2013;35:289-298.