메뉴 건너뛰기




Volumn 29, Issue SUPPL. 3, 2014, Pages

The rare diseases clinical research network's organization and approach to observational research and health outcomes research

Author keywords

multi institutional clinical research; outcomes; patient advocacy groups; rare diseases network

Indexed keywords

COOPERATION; HUMAN; INFORMATION PROCESSING; METHODOLOGY; ORGANIZATION AND MANAGEMENT; OUTCOME ASSESSMENT; RARE DISEASE; REGISTER; UNITED STATES;

EID: 84905923468     PISSN: 08848734     EISSN: 15251497     Source Type: Journal    
DOI: 10.1007/s11606-014-2894-x     Document Type: Article
Times cited : (30)

References (28)
  • 3
    • 85007086091 scopus 로고    scopus 로고
    • Brain Vascular Malformation Consortium: Overview, progress, and future directions
    • the Brain Vascular Malformation Consortium
    • Akers A, Ball KL, Clancy M, et al. the Brain Vascular Malformation Consortium. Brain Vascular Malformation Consortium: overview, progress, and future directions. J Rare Disorders. 2013;1(1):1-15.
    • (2013) J Rare Disorders , vol.1 , Issue.1 , pp. 1-15
    • Akers, A.1    Ball, K.L.2    Clancy, M.3
  • 4
    • 79960234742 scopus 로고    scopus 로고
    • Rationale and design of the chronic GVHD cohort study: Improving outcomes assessment in chronic GVHD
    • Chronic Graft Versus Host Disease Consortium
    • Chronic Graft Versus Host Disease Consortium. Rationale and design of the chronic GVHD cohort study: improving outcomes assessment in chronic GVHD. Biol Blood Marrow Transplant. 2011;17(8):1114-1120.
    • (2011) Biol Blood Marrow Transplant , vol.17 , Issue.8 , pp. 1114-1120
  • 5
    • 84875701332 scopus 로고    scopus 로고
    • Design of the Nephrotic Syndrome Study Network (NEPTUNE) to evaluate primary glomerular nephropathy by a multidisciplinary approach
    • Gadegbeku CA, Gipson DS, Holzman LB, et al. Design of the Nephrotic Syndrome Study Network (NEPTUNE) to evaluate primary glomerular nephropathy by a multidisciplinary approach. Kidney Int. 2013;83(4):749-756.
    • (2013) Kidney Int , vol.83 , Issue.4 , pp. 749-756
    • Gadegbeku, C.A.1    Gipson, D.S.2    Holzman, L.B.3
  • 6
    • 50649105460 scopus 로고    scopus 로고
    • An automated standardized system for managing adverse events in clinical research networks
    • Richesson RL, Malloy JF, Paulus K, Cuthbertson D, Krischer JP. An automated standardized system for managing adverse events in clinical research networks. Drug Saf. 2008;31(10):807-822.
    • (2008) Drug Saf , vol.31 , Issue.10 , pp. 807-822
    • Richesson, R.L.1    Malloy, J.F.2    Paulus, K.3    Cuthbertson, D.4    Krischer, J.P.5
  • 7
    • 57649105507 scopus 로고    scopus 로고
    • An automated communications system in a contact registry for persons with rare diseases: Scalable tools for identifying and recruiting clinical research participants
    • Richesson RL, Lee HS, Cuthbertson D, Lloyd J, Young K, Krischer JP. An automated communications system in a contact registry for persons with rare diseases: scalable tools for identifying and recruiting clinical research participants. Contemp Clin Trials. 2009;30(1):55-62.
    • (2009) Contemp Clin Trials , vol.30 , Issue.1 , pp. 55-62
    • Richesson, R.L.1    Lee, H.S.2    Cuthbertson, D.3    Lloyd, J.4    Young, K.5    Krischer, J.P.6
  • 8
    • 84861532036 scopus 로고    scopus 로고
    • The rare diseases clinical research network contact registry update: Features and functionality
    • Richesson RL, Sutphen R, Shereff D, Krischer JP. The rare diseases clinical research network contact registry update: features and functionality. Contemp Clin Trials. 2012;33(4):647-656.
    • (2012) Contemp Clin Trials , vol.33 , Issue.4 , pp. 647-656
    • Richesson, R.L.1    Sutphen, R.2    Shereff, D.3    Krischer, J.P.4
  • 9
    • 84881359035 scopus 로고    scopus 로고
    • Pregnancy outcomes among patients with vasculitis
    • for the Vasculitis Clinical Research Consortium
    • Clowse ME, Richeson RL, Pieper C, Merkel PA. for the Vasculitis Clinical Research Consortium. Pregnancy outcomes among patients with vasculitis. Arthritis Care Res. 2013;65(8):1370-1374.
    • (2013) Arthritis Care Res , vol.65 , Issue.8 , pp. 1370-1374
    • Clowse, M.E.1    Richeson, R.L.2    Pieper, C.3    Merkel, P.A.4
  • 10
    • 79955510505 scopus 로고    scopus 로고
    • Efficacy and safety of sirolimus in lymphangioleiomyomatosis
    • the National Institutes of Health Rare Lung Diseases Consortium; the MILES Trial Group
    • McCormack FX, Inoue Y, Moss J, Singer LG, et al. the National Institutes of Health Rare Lung Diseases Consortium; the MILES Trial Group. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. N Engl J Med. 2011;364(17):1595-1606.
    • (2011) N Engl J Med , vol.364 , Issue.17 , pp. 1595-1606
    • McCormack, F.X.1    Inoue, Y.2    Moss, J.3    Singer, L.G.4
  • 11
    • 84866732337 scopus 로고    scopus 로고
    • Treatment of idiopathic pulmonary fibrosis with losartan: A pilot project
    • Couluris M, Kinder BW, Xu P, Gross-King M, Krischer J, Panos RJ. Treatment of idiopathic pulmonary fibrosis with losartan: a pilot project. Lung. 2012;190(5):523-527.
    • (2012) Lung , vol.190 , Issue.5 , pp. 523-527
    • Couluris, M.1    Kinder, B.W.2    Xu, P.3    Gross-King, M.4    Krischer, J.5    Panos, R.J.6
  • 12
    • 84866919087 scopus 로고    scopus 로고
    • Mexiletine for symptoms and signs of myotonia in nondystrophic myotonia: A randomized controlled trial
    • Statland JM, Bundy BN, Wang Y, et al. Mexiletine for symptoms and signs of myotonia in nondystrophic myotonia: a randomized controlled trial. JAMA. 2012;308(13):1357-1365.
    • (2012) JAMA , vol.308 , Issue.13 , pp. 1357-1365
    • Statland, J.M.1    Bundy, B.N.2    Wang, Y.3
  • 13
    • 84886402463 scopus 로고    scopus 로고
    • Primary ciliary dyskinesia. Recent advances in diagnostics, genetics, and characterization of clinical disease
    • Knowles MR, Daniels LA, Davis SD, Zariwala MA, Leigh MW. Primary ciliary dyskinesia. Recent advances in diagnostics, genetics, and characterization of clinical disease. Am J Resp. Crit Care Med. 2013;188(8):913-922.
    • (2013) Am J Resp. Crit Care Med. , vol.188 , Issue.8 , pp. 913-922
    • Knowles, M.R.1    Daniels, L.A.2    Davis, S.D.3    Zariwala, M.A.4    Leigh, M.W.5
  • 14
    • 84880211812 scopus 로고    scopus 로고
    • Implementation of timeline reforms speeds initiation of National Cancer Institute-sponsored trials
    • Abrams JS, Mooney MM, Zwiebel JA, et al. Implementation of timeline reforms speeds initiation of National Cancer Institute-sponsored trials. JNCI. 2013;105(13):954-959.
    • (2013) JNCI , vol.105 , Issue.13 , pp. 954-959
    • Abrams, J.S.1    Mooney, M.M.2    Zwiebel, J.A.3
  • 15
    • 57649109461 scopus 로고    scopus 로고
    • Clinical research for rare disease: Opportunities, challenges, and solutions
    • Griggs RC, Batshaw M, Dunkle M, et al. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab. 2009;96(1):20-26.
    • (2009) Mol Genet Metab , vol.96 , Issue.1 , pp. 20-26
    • Griggs, R.C.1    Batshaw, M.2    Dunkle, M.3
  • 16
    • 38449108850 scopus 로고    scopus 로고
    • A web-based SNOMED CT browser: Distributed and real-time use of SNOMED CT during the clinical research process
    • Richesson R, Syed A, Guillette H, Tuttle MS, Krischer J. A web-based SNOMED CT browser: distributed and real-time use of SNOMED CT during the clinical research process. Stud Health Technol Inform. 2007;129(Pt 1):631-635.
    • (2007) Stud Health Technol Inform , vol.129 , Issue.PART 1 , pp. 631-635
    • Richesson, R.1    Syed, A.2    Guillette, H.3    Tuttle, M.S.4    Krischer, J.5
  • 17
    • 77956059196 scopus 로고    scopus 로고
    • Achieving standardized medication data in clinical research studies: Two approaches and applications for implementing RxNorm
    • Richesson RL, Smith SB, Malloy J, Krischer JP. Achieving standardized medication data in clinical research studies: two approaches and applications for implementing RxNorm. J Med Syst. 2010;34(4):651-657.
    • (2010) J Med Syst , vol.34 , Issue.4 , pp. 651-657
    • Richesson, R.L.1    Smith, S.B.2    Malloy, J.3    Krischer, J.P.4
  • 18
    • 79958255131 scopus 로고    scopus 로고
    • Data standards for clinical research data collection forms: Current status and challenges
    • Richesson RL, Nadkarni P. Data standards for clinical research data collection forms: current status and challenges. J Am Med Inform Assoc. 2011;18(3):341-346.
    • (2011) J Am Med Inform Assoc , vol.18 , Issue.3 , pp. 341-346
    • Richesson, R.L.1    Nadkarni, P.2
  • 19
    • 79955975797 scopus 로고    scopus 로고
    • Patient-reported quality of life is associated with severity of chronic graft-versus-host disease as measured by NIH criteria: Report on baseline data from the Chronic GVHD Consortium
    • Pidala J, Kurland B, Chai X, et al. Patient-reported quality of life is associated with severity of chronic graft-versus-host disease as measured by NIH criteria: report on baseline data from the Chronic GVHD Consortium. Blood. 2011;117(17):4651-4657.
    • (2011) Blood , vol.117 , Issue.17 , pp. 4651-4657
    • Pidala, J.1    Kurland, B.2    Chai, X.3
  • 20
    • 80053637674 scopus 로고    scopus 로고
    • Sensitivity of changes in chronic graft-versus-host disease activity to changes in patient-reported quality of life: Results from the Chronic Graft-versus-Host Disease Consortium
    • Pidala J, Kurland BF, Chai X, et al. Sensitivity of changes in chronic graft-versus-host disease activity to changes in patient-reported quality of life: results from the Chronic Graft-versus-Host Disease Consortium. Haematologica. 2011;96(10):1528-1535.
    • (2011) Haematologica , vol.96 , Issue.10 , pp. 1528-1535
    • Pidala, J.1    Kurland, B.F.2    Chai, X.3
  • 21
    • 77957936037 scopus 로고    scopus 로고
    • Determinants of reduced health-related quality of life in pediatric inherited neuropathies
    • Burns J, Ramchandren S, Ryan MM, et al. Determinants of reduced health-related quality of life in pediatric inherited neuropathies. Neurology. 2010;75(8):726-731.
    • (2010) Neurology , vol.75 , Issue.8 , pp. 726-731
    • Burns, J.1    Ramchandren, S.2    Ryan, M.M.3
  • 22
    • 77649241209 scopus 로고    scopus 로고
    • Quality of life in children with Charcot-Marie-Tooth disease
    • Burns J, Ryan MM, Ouvrier RA. Quality of life in children with Charcot-Marie-Tooth disease. J Child Neurol. 2010;25(3):343-347.
    • (2010) J Child Neurol , vol.25 , Issue.3 , pp. 343-347
    • Burns, J.1    Ryan, M.M.2    Ouvrier, R.A.3
  • 23
    • 79959979797 scopus 로고    scopus 로고
    • Health-related quality of life in patients with newly diagnosed antineutrophil cytoplasmic antibody-associated vasculitis
    • Walsh M, Mukhtyar C, Mahr A, et al. Health-related quality of life in patients with newly diagnosed antineutrophil cytoplasmic antibody-associated vasculitis. Arthritis Care Res. 2011;63(7):1055-1061.
    • (2011) Arthritis Care Res , vol.63 , Issue.7 , pp. 1055-1061
    • Walsh, M.1    Mukhtyar, C.2    Mahr, A.3
  • 24
    • 84858044570 scopus 로고    scopus 로고
    • Assessment of health-related quality of life as an outcome measure in granulomatosis with polyangiitis (Wegener's)
    • Tomasson G, Boers M, Walsh M, et al. Assessment of health-related quality of life as an outcome measure in granulomatosis with polyangiitis (Wegener's). Arthritis Care Res. 2012;64(2):273-279.
    • (2012) Arthritis Care Res , vol.64 , Issue.2 , pp. 273-279
    • Tomasson, G.1    Boers, M.2    Walsh, M.3
  • 25
    • 82955175584 scopus 로고    scopus 로고
    • Clinical severity and quality of life in chi ldren and adolescents with Rett syndrome
    • Lane JB, Lee HS, Smith LW, et al. Clinical severity and quality of life in chi ldren and adolescents with Rett syndrome. Neurology. 2011;77(20):1812- 1818.
    • (2011) Neurology , vol.77 , Issue.20 , pp. 1812-1818
    • Lane, J.B.1    Lee, H.S.2    Smith, L.W.3
  • 26
    • 79952049632 scopus 로고    scopus 로고
    • Patient-reported outcome assessment in vasculitis may provide important data and a unique perspective
    • Herlyn K, Hellmich B, Seo P, Merkel PA. Patient-reported outcome assessment in vasculitis may provide important data and a unique perspective. Arthritis Care Res. 2010;62(11):1639-1645.
    • (2010) Arthritis Care Res , vol.62 , Issue.11 , pp. 1639-1645
    • Herlyn, K.1    Hellmich, B.2    Seo, P.3    Merkel, P.A.4
  • 27
    • 78650170260 scopus 로고    scopus 로고
    • 168th ENMC International Workshop: Outcome measures and clinical trials in Charcot-Marie-Tooth disease (CMT)
    • Reilly MM, Shy ME, Muntoni F, Pareyson D. 168th ENMC International Workshop: outcome measures and clinical trials in Charcot-Marie-Tooth disease (CMT). Neuromuscul Disord. 2010;20(12):839-846.
    • (2010) Neuromuscul Disord , vol.20 , Issue.12 , pp. 839-846
    • Reilly, M.M.1    Shy, M.E.2    Muntoni, F.3    Pareyson, D.4
  • 28
    • 84905906399 scopus 로고    scopus 로고
    • A model for collaborative clinical research in rare diseases: Experience from the Rare Disease Clinical Research Network program
    • Groft SC, Gopal-Srivastava R. A model for collaborative clinical research in rare diseases: experience from the Rare Disease Clinical Research Network program. J Clin Invest. 2013;3(11):1015-21.
    • (2013) J Clin Invest , vol.3 , Issue.11 , pp. 1015-1021
    • Groft, S.C.1    Gopal-Srivastava, R.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.