A Markov model to analyze cost-effectiveness of screening for severe combined immunodeficiency (SCID)

Molecular Genetics and Metabolism

Volumn 104, Issue 3, 2011, Pages 383-389

  Chan, Kee ^a,j   Davis, Joie ^b,j   Pai, Sung Yun ^c,d,e   Bonilla, Francisco A ^c,d   Puck, Jennifer M ^f,g,j,l   Apkon, Michael ^h,i,k,l  

^a BOSTON UNIVERSITY   (United States)

^b NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d BOSTON CHILDREN S HOSPITAL   (United States)

^e DANA FARBER CANCER INSTITUTE   (United States)

^f UNIVERSITY OF CALIFORNIA   (United States)

^g UNIVERSITY OF CALIFORNIA   (United States)

^h UNIVERSITY OF PENNSYLVANIA   (United States)

ⁱ CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^j NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^k YALE UNIVERSITY   (United States)

^l YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

more..

Author keywords

Cost effectiveness; Hematopoietic cell transplant; Markov models; Newborn screening; Severe combined immunodeficiency (SCID); T cell receptor excision circle (TREC)

Indexed keywords

ARTICLE; COHORT ANALYSIS; COST EFFECTIVENESS ANALYSIS; EARLY DIAGNOSIS; HEALTH CARE UTILIZATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LIFE EXPECTANCY; MAJOR CLINICAL STUDY; MARKOV MODEL; NEWBORN; NEWBORN SCREENING; PEDIATRICS; PRIORITY JOURNAL; QUALITY OF LIFE; SENSITIVITY AND SPECIFICITY; SEVERE COMBINED IMMUNODEFICIENCY; T LYMPHOCYTE;

COST-BENEFIT ANALYSIS; DECISION TREES; HUMANS; INFANT, NEWBORN; LYMPHOPENIA; MARKOV CHAINS; MODELS, ECONOMIC; NEONATAL SCREENING; QUALITY-ADJUSTED LIFE YEARS; SENSITIVITY AND SPECIFICITY; SEVERE COMBINED IMMUNODEFICIENCY; T-LYMPHOCYTES;

EID: 80955145637     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2011.07.007     Document Type: Article

References (39)

1. Buckley R.H. Primary cellular immunodeficiencies. J. Allergy Clin. Immunol. 2002, 109:747-757.
2. Campbell E., Ross L.F. Parental attitudes regarding newborn screening of PKU and DMD. Am. J. Med. Genet A 2003, 120:209-214.
3. Howell R.R. Introduction: newborn screening. Ment. Retard. Dev. Disabil. Res. Rev. 2006, 12:229.
4. Brown L., Xu-Bayford J., Allwood Z., Slatter M., Cant A., Davies E.G., Veys P., Gennery A.R., Gaspar H.B. Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. Blood 2011, 117:3243-3246.
5. Buckley R.H. Advances in the understanding and treatment of human severe combined immunodeficiency. Immunol. Res. 2000, 22:237-251.
6. Myers L.A., Patel D.D., Puck J.M., Buckley R.H. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 2002, 99:872-878.
7. Chan K., Puck J.M. Development of population-based newborn screening for severe combined immunodeficiency. J. Allergy. Clin. Immunol. 2005, 115:391-398.
8. Baker M.W., Laessig R.H., Katcher M.L., Routes J.M., Grossman W.J., Verbsky J., Kurtycz D.F., Brokopp C.D. Implementing routine testing for severe combined immunodeficiency within Wisconsin's newborn screening program. Public Health Rep. Nov 2010, 125(Suppl. 2):88-95.
9. H.J., Comeau A.M., Pai S.Y., Bonilla F.A., Notarangelo L.D., Pasternack M.S., Meissner H.C., Cooper E.R., DeMaria A., Sahai I., Eaton R.B. Guidelines for implementation of population-based newborn screening for severe combined immunodeficiency. J. Inherit. Metab. Dis. 2010, 33:S273-S281.
10. Hale J.E., Bonilla F.A., Pai S.Y., Gerstel-Thompson J.L., Notarangelo L.D., Eaton R.B., Comeau A.M. Identification of an infant with severe combined immunodeficiency by newborn screening. J. Allergy Clin. Immunol. 2010, 126:1073-1074.
11. W.J., Gerstel-Thompson J.L., Baptiste J.C., Navas J.S., Pai S.Y., Pass K.A., Eaton R.B., Comeau A.M. High-throughput multiplexed T-cell-receptor excision circle quantitative PCR assay with internal controls for detection of severe combined immunodeficiency in population-based newborn screening. Clin. Chem. 2010, 56:1466-1474.
12. G.W., Baker M.W., Laessig R.H., Hoffman G.L., Brokopp C.D., Kurtycz D.F., Cogley M.F., Litsheim T.J., Katcher M.L., Routes J.M. Development of a routine newborn screening protocol for severe combined immunodeficiency. J. Allergy Clin. Immunol. 2009, 124:522-527.
13. McGhee S.A., Stiehm E.R., McCabe E.R. Potential costs and benefits of newborn screening for severe combined immunodeficiency. J. Pediatr. 2005, 147:603-608.
14. Russell L.B., Gold M.R., Siegel J.E., Daniels N., Weinstein M.C. The role of cost-effectiveness analysis in health and medicine. Panel on Cost-Effectiveness in Health and Medicine. JAMA 1996, 276:1172-1177.
15. Buckley R.H., Schiff S.E., Schiff R.I., Markert L., Williams L.W., Roberts J.L., Myers L.A., Ward F.E. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N. Engl. J. Med. 1999, 340:508-516.
16. Griffith L.M., Cowan M.J., Kohn D.B., Notarangelo L.D., Puck J.M., Schultz K.R., Buckley R.H., Eapen M., Kamani N.R., O'Reilly R.J., Parkman R., Roifman C.M., Sullivan K.E., Filipovich A.H., Fleisher T.A., Shearer W.T. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J. Allergy Clin. Immunol. 2008, 122:1087-1096.
17. Weinstein M.C., Siegel J.E., Gold M.R., Kamlet M.S., Russell L.B. Recommendations of the Panel on Cost-effectiveness in Health and Medicine. JAMA 1996, 276:1253-1258.
18. U.S.N.S.S.R.A.T. 2004.
19. US Census Bureau Statistical Abstract of the United States 2006, US Census Bureau, Washington DC.
20. Venditti L.N., Venditti C.P., Berry G.T., Kaplan P.B., Kaye E.M., Glick H., Stanley C.A. Newborn screening by tandem mass spectrometry for medium-chain Acyl-CoA dehydrogenase deficiency: a cost-effectiveness analysis. Pediatrics 2003, 112:1005-1015.
21. Panepinto J.A., O'Mahar K.M., DeBaun M.R., Loberiza F.R., Scott J.P. Health-related quality of life in children with sickle cell disease: child and parent perception. Br. J. Haematol. 2005, 130:437-444.
22. Haberman M., Bush N., Young K., Sullivan K.M. Quality of life of adult long-term survivors of bone marrow transplantation: a qualitative analysis of narrative data. Oncol. Nurs. Forum 1993, 20:1545-1553.
23. Koscik R.L., Douglas J.A., Zaremba K., Rock M.J., Splaingard M.L., Laxova A., Farrell P.M. Quality of life of children with cystic fibrosis. J. Pediatr. 2005, 147:S64-S68.
24. Sonnenberg F.A., Beck J.R. Markov models in medical decision making: a practical guide. Med. Decis. Making 1993, 13:322-338.
25. Beck J.R., Pauker S.G. The Markov process in medical prognosis. Med. Decis. Making 1983, 3:419-458.
26. Fenwick E., Claxton K., Sculpher M. Representing uncertainty: the role of cost-effectiveness acceptability curves. Health Econ. 2001, 10:779-787.
27. Weinstein M.C., Stason W.B. Foundations of cost-effectiveness analysis for health and medical practices. N. Engl. J. Med. 1977, 296:716-721.
28. Tsevat J., Wong J.B., Pauker S.G., Steinberg M.H. Neonatal screening for sickle cell disease: a cost-effectiveness analysis. J. Pediatr. 1991, 118:546-554.
29. Panepinto J.A., Magid D., Rewers M.J., Lane P.A. Universal versus targeted screening of infants for sickle cell disease: a cost-effectiveness analysis. J. Pediatr. 2000, 136:201-208.
30. McGhee S.A., Stiehm E.R., Cowan M., Krogstad P., McCabe E.R. Two-tiered universal newborn screening strategy for severe combined immunodeficiency. Mol. Genet. Metab. 2005, 86:427-430.
31. Roifman C.M., Somech R., Grunebaum E. Matched unrelated bone marrow transplant for T+ combined immunodeficiency. Bone Marrow Transplant. 2008, 41:947-952.
32. Sarzotti-Kelsoe M., Win C.M., Parrott R.E., Cooney M., Moser B.K., Roberts J.L., Sempowski G.D., Buckley R.H. Thymic output, T cell diversity and T cell function in long-term human SCID chimeras. Blood 2009, 114:1445-1453.
33. Titman P., Pink E., Skucek E., O'Hanlon K., Cole T.J., Gaspar J., Xu-Bayford J., Jones A., Thrasher A.J., Davies E.G., Veys P.A., Gaspar H.B. Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood 2008, 112:3907-3913.
34. Benoit R.M., Gronberg H., Naslund M.J. A quantitative analysis of the costs and benefits of prostate cancer screening. Prostate Cancer Prostatic Dis. 2001, 4:138-145.
35. G.W., Routes J.M., Verbsky J., Laessig R.H., Hoffman G.L., Brokopp C.D., Baker M.W. Statewide newborn screening for severe T-cell lymphopenia. JAMA 2009, 302:2465-2470.
36. Puck J.M. Population-based newborn screening for severe combined immunodeficiency: steps toward implementation. J. Allergy Clin. Immunol. 2007, 120:760-768.
37. Grosse S.D., Boyle C.A., Kenneson A., Khoury M.J., Wilfond B.S. From public health emergency to public health service: the implications of evolving criteria for newborn screening panels. Pediatrics 2006, 117:923-929.
38. Baker M.W., Grossman W.J., Laessig R.H., Hoffman G.L., Brokopp C.D., Kurtycz D.F., Cogley M.F., Litsheim T.J., Katcher M.L., Routes J.M. Development of a routine newborn screening protocol for severe combined immunodeficiency. J. Allergy Clin. Immunol. 2009, 124:522-527.
39. V.S., Lipstein E.A., Browning M.F., Green N.S., Kemper A.R., Knapp A.A., Prosser L.A., Perrin J.M. Systematic evidence review of newborn screening and treatment of severe combined immunodeficiency. Pediatrics 2010, 125:1226-1235.