메뉴 건너뛰기




Volumn 61, Issue 9, 2014, Pages 1544-1550

Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria

Author keywords

Children; Complement inhibition; Eculizumab; Paroxysmal nocturnal hemoglobinuria; Phase I II

Indexed keywords

COMPLEMENT; ECULIZUMAB; HEMOGLOBIN; LACTATE DEHYDROGENASE;

EID: 84904438509     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.25068     Document Type: Article
Times cited : (34)

References (43)
  • 1
    • 0027310539 scopus 로고
    • Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria
    • Takeda J, Miyata T, Kawagoe K, et al. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell 1993; 73:703-711.
    • (1993) Cell , vol.73 , pp. 703-711
    • Takeda, J.1    Miyata, T.2    Kawagoe, K.3
  • 2
    • 0027412627 scopus 로고
    • The cloning of PIG-A, a component in the early step of GPI-anchor biosynthesis
    • Miyata T, Takeda J, Iida Y, et al. The cloning of PIG-A, a component in the early step of GPI-anchor biosynthesis. Science 1993; 259:1318-1320.
    • (1993) Science , vol.259 , pp. 1318-1320
    • Miyata, T.1    Takeda, J.2    Iida, Y.3
  • 3
    • 0025233715 scopus 로고
    • The complement-inhibitory activity of CD59 resides in its capacity to block incorporation of C9 into membrane C5b-9
    • Rollins SA, Sims PJ. The complement-inhibitory activity of CD59 resides in its capacity to block incorporation of C9 into membrane C5b-9. J Immunol 1990; 144:3478-3483.
    • (1990) J Immunol , vol.144 , pp. 3478-3483
    • Rollins, S.A.1    Sims, P.J.2
  • 4
    • 0021713655 scopus 로고
    • Inhibition of complement activation on the surface of cells after incorporation of decay-accelerating factor (DAF) into their membranes
    • Medof ME, Kinoshita T, Nussenzweig V. Inhibition of complement activation on the surface of cells after incorporation of decay-accelerating factor (DAF) into their membranes. J Exp Med 1984; 160:1558-1578.
    • (1984) J Exp Med , vol.160 , pp. 1558-1578
    • Medof, M.E.1    Kinoshita, T.2    Nussenzweig, V.3
  • 5
    • 15944398355 scopus 로고    scopus 로고
    • The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: A novel mechanism of human disease
    • Rother RP, Bell L, Hillmen P, et al. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: A novel mechanism of human disease. JAMA 2005; 293:1653-1662.
    • (2005) JAMA , vol.293 , pp. 1653-1662
    • Rother, R.P.1    Bell, L.2    Hillmen, P.3
  • 6
    • 84882343523 scopus 로고    scopus 로고
    • Thrombosis in paroxysmal nocturnal hemoglobinuria
    • Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood 2013; 121:4985-4996.
    • (2013) Blood , vol.121 , pp. 4985-4996
    • Hill, A.1    Kelly, R.J.2    Hillmen, P.3
  • 7
    • 84855362194 scopus 로고    scopus 로고
    • Mechanisms and clinical implications of thrombosis in paroxysmal nocturnal hemoglobinuria
    • van Bijnen ST, Van Heerde WL, Muus P. Mechanisms and clinical implications of thrombosis in paroxysmal nocturnal hemoglobinuria. J Thromb Haemost 2012; 10:1-10.
    • (2012) J Thromb Haemost , vol.10 , pp. 1-10
    • van Bijnen, S.T.1    Van Heerde, W.L.2    Muus, P.3
  • 8
    • 0029998036 scopus 로고    scopus 로고
    • Megakaryocyte progenitors in paroxysmal nocturnal haemoglobinuria are sensitive to complement
    • Basara N, Antunovic P, Sefer D, et al. Megakaryocyte progenitors in paroxysmal nocturnal haemoglobinuria are sensitive to complement. Eur J Haematol 1996; 57:227-229.
    • (1996) Eur J Haematol , vol.57 , pp. 227-229
    • Basara, N.1    Antunovic, P.2    Sefer, D.3
  • 9
    • 0023806978 scopus 로고
    • Increased sensitivity to complement of megakaryocyte progenitors in paroxysmal nocturnal haemoglobinuria
    • Dessypris EN, Gleaton JH, Clark DA. Increased sensitivity to complement of megakaryocyte progenitors in paroxysmal nocturnal haemoglobinuria. Br J Haematol 1988; 69:305-309.
    • (1988) Br J Haematol , vol.69 , pp. 305-309
    • Dessypris, E.N.1    Gleaton, J.H.2    Clark, D.A.3
  • 10
    • 0028802443 scopus 로고
    • Natural history of paroxysmal nocturnal hemoglobinuria
    • Hillmen P, Lewis SM, Bessler M, et al. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med 1995; 333:1253-1258.
    • (1995) N Engl J Med , vol.333 , pp. 1253-1258
    • Hillmen, P.1    Lewis, S.M.2    Bessler, M.3
  • 11
    • 16044365965 scopus 로고    scopus 로고
    • Paroxysmal nocturnal haemoglobinuria: Long-term follow-up and prognostic factors. French Society of Haematology
    • Socie G, Mary JY, de GA, et al. Paroxysmal nocturnal haemoglobinuria: Long-term follow-up and prognostic factors. French Society of Haematology. Lancet 1996; 348:573-577.
    • (1996) Lancet , vol.348 , pp. 573-577
    • Socie, G.1    Mary, J.Y.2    de, G.A.3
  • 12
    • 2442532784 scopus 로고    scopus 로고
    • Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan
    • Nishimura J, Kanakura Y, Ware RE, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine (Baltimore) 2004; 83:193-207.
    • (2004) Medicine (Baltimore) , vol.83 , pp. 193-207
    • Nishimura, J.1    Kanakura, Y.2    Ware, R.E.3
  • 13
    • 3042789125 scopus 로고    scopus 로고
    • Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays
    • Moyo VM, Mukhina GL, Garrett ES, et al. Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays. Br J Haematol 2004; 126:133-138.
    • (2004) Br J Haematol , vol.126 , pp. 133-138
    • Moyo, V.M.1    Mukhina, G.L.2    Garrett, E.S.3
  • 14
    • 28444483571 scopus 로고    scopus 로고
    • Diagnosis and management of paroxysmal nocturnal hemoglobinuria
    • Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 2005; 106:3699-3709.
    • (2005) Blood , vol.106 , pp. 3699-3709
    • Parker, C.1    Omine, M.2    Richards, S.3
  • 15
    • 33947227491 scopus 로고    scopus 로고
    • The pathophysiology of paroxysmal nocturnal hemoglobinuria
    • Parker CJ. The pathophysiology of paroxysmal nocturnal hemoglobinuria. Exp Hematol 2007; 35:523-533.
    • (2007) Exp Hematol , vol.35 , pp. 523-533
    • Parker, C.J.1
  • 16
    • 0025999919 scopus 로고
    • Paroxysmal nocturnal hemoglobinuria with onset in childhood and adolescence
    • Ware RE, Hall SE, Rosse WF. Paroxysmal nocturnal hemoglobinuria with onset in childhood and adolescence. N Engl J Med 1991; 325:991-996.
    • (1991) N Engl J Med , vol.325 , pp. 991-996
    • Ware, R.E.1    Hall, S.E.2    Rosse, W.F.3
  • 17
    • 51349161060 scopus 로고    scopus 로고
    • Paroxysmal nocturnal hemoglobinuria in childhood and adolescence-A retrospective analysis of 18 cases
    • Naithani R, Mahapatra M, Dutta P, et al. Paroxysmal nocturnal hemoglobinuria in childhood and adolescence-A retrospective analysis of 18 cases. Ind J Pediatr 2008; 75:575-578.
    • (2008) Ind J Pediatr , vol.75 , pp. 575-578
    • Naithani, R.1    Mahapatra, M.2    Dutta, P.3
  • 18
    • 84862263225 scopus 로고    scopus 로고
    • Paroxysmal nocturnal hemoglobinuria in pediatric patients
    • Curran KJ, Kernan NA, Prockop SE, et al. Paroxysmal nocturnal hemoglobinuria in pediatric patients. Pediatr Blood Cancer 2012; 59:525-529.
    • (2012) Pediatr Blood Cancer , vol.59 , pp. 525-529
    • Curran, K.J.1    Kernan, N.A.2    Prockop, S.E.3
  • 19
    • 14144255205 scopus 로고    scopus 로고
    • Childhood paroxysmal nocturnal haemoglobinuria (PNH), a report of 11 cases in the Netherlands
    • van den Heuvel-Eibrink MM, Bredius RG, te Winkel ML, et al. Childhood paroxysmal nocturnal haemoglobinuria (PNH), a report of 11 cases in the Netherlands. Br J Haematol 2005; 128:571-577.
    • (2005) Br J Haematol , vol.128 , pp. 571-577
    • van den Heuvel-Eibrink, M.M.1    Bredius, R.G.2    te Winkel, M.L.3
  • 20
    • 84904433923 scopus 로고    scopus 로고
    • Clinical characteristics of classic paroxysmal nocturnal hemoglobinuria (PNH) in pediatric patients: A comparison with classic PNH in adults. An International PNH registry study
    • Urbano-Ispizua A, Schrezenmeier H, Muus P, et al. Clinical characteristics of classic paroxysmal nocturnal hemoglobinuria (PNH) in pediatric patients: A comparison with classic PNH in adults. An International PNH registry study. Blood 2011; 118:2102.
    • (2011) Blood , vol.118 , pp. 2102
    • Urbano-Ispizua, A.1    Schrezenmeier, H.2    Muus, P.3
  • 21
    • 84868518322 scopus 로고    scopus 로고
    • Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria
    • Peffault de Latour R, Schrezenmeier H, Bacigalupo A, et al. Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica 2012; 97:1666-1673.
    • (2012) Haematologica , vol.97 , pp. 1666-1673
    • Peffault de Latour, R.1    Schrezenmeier, H.2    Bacigalupo, A.3
  • 22
    • 54049098831 scopus 로고    scopus 로고
    • Paroxysmal nocturnal hemoglobinuria: Natural histroy of disease subcategories
    • Peffault de Latour R, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria: Natural histroy of disease subcategories. Blood 2008; 3099-3106.
    • (2008) Blood , pp. 3099-3106
    • Peffault de Latour, R.1    Mary, J.Y.2    Salanoubat, C.3
  • 23
    • 0030441149 scopus 로고    scopus 로고
    • Inhibition of complement activity by humanized anti-C5 antibody and single-chain Fv
    • Thomas TC, Rollins SA, Rother RP, et al. Inhibition of complement activity by humanized anti-C5 antibody and single-chain Fv. Mol Immunol 1996; 33:1389-1401.
    • (1996) Mol Immunol , vol.33 , pp. 1389-1401
    • Thomas, T.C.1    Rollins, S.A.2    Rother, R.P.3
  • 24
    • 41349089713 scopus 로고    scopus 로고
    • Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria
    • Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood 2008; 111:1840-1847.
    • (2008) Blood , vol.111 , pp. 1840-1847
    • Brodsky, R.A.1    Young, N.S.2    Antonioli, E.3
  • 25
    • 27144440502 scopus 로고    scopus 로고
    • Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria
    • Hill A, Hillmen P, Richards SJ, et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood 2005; 106:2559-2565.
    • (2005) Blood , vol.106 , pp. 2559-2565
    • Hill, A.1    Hillmen, P.2    Richards, S.J.3
  • 26
    • 10744228553 scopus 로고    scopus 로고
    • Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria
    • Hillmen P, Hall C, Marsh JC, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 2004; 350:552-559.
    • (2004) N Engl J Med , vol.350 , pp. 552-559
    • Hillmen, P.1    Hall, C.2    Marsh, J.C.3
  • 27
    • 33748904355 scopus 로고    scopus 로고
    • The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria
    • Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006; 355:1233-1243.
    • (2006) N Engl J Med , vol.355 , pp. 1233-1243
    • Hillmen, P.1    Young, N.S.2    Schubert, J.3
  • 28
    • 35948962168 scopus 로고    scopus 로고
    • Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria
    • Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood 2007; 110:4123-4128.
    • (2007) Blood , vol.110 , pp. 4123-4128
    • Hillmen, P.1    Muus, P.2    Duhrsen, U.3
  • 29
    • 77955486794 scopus 로고    scopus 로고
    • Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria
    • Hillmen P, Elebute M, Kelly R, et al. Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol 2010; 85:553-559.
    • (2010) Am J Hematol , vol.85 , pp. 553-559
    • Hillmen, P.1    Elebute, M.2    Kelly, R.3
  • 30
    • 84879121370 scopus 로고    scopus 로고
    • Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria
    • Hillmen P, Muus P, Roth A, et al. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol 2013; 162:62-73.
    • (2013) Br J Haematol , vol.162 , pp. 62-73
    • Hillmen, P.1    Muus, P.2    Roth, A.3
  • 31
    • 84886803820 scopus 로고    scopus 로고
    • Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial
    • Kanakura Y, Ohyashiki K, Shichishima T, et al. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol 2013; 98:406-416.
    • (2013) Int J Hematol , vol.98 , pp. 406-416
    • Kanakura, Y.1    Ohyashiki, K.2    Shichishima, T.3
  • 32
    • 79958849218 scopus 로고    scopus 로고
    • Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: Sustained efficacy and improved survival
    • Kelly RJ, Hill A, Arnold LM, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: Sustained efficacy and improved survival. Blood 2011; 117:6786-6792.
    • (2011) Blood , vol.117 , pp. 6786-6792
    • Kelly, R.J.1    Hill, A.2    Arnold, L.M.3
  • 33
    • 84904444835 scopus 로고    scopus 로고
    • Eculizumab weight-based dosing strategies in pediatric aHUS patients between neonates and 12 years of age and dosing strategies of eculizumab in pediatric aHUS patients undergoing plasma exchange-Abbreviated report. 6-22-2009 Pharsight.
    • Mouksassi S, Kassir N, Marier J. Eculizumab weight-based dosing strategies in pediatric aHUS patients between neonates and 12 years of age and dosing strategies of eculizumab in pediatric aHUS patients undergoing plasma exchange-Abbreviated report. 6-22-2009 Pharsight.
    • Mouksassi, S.1    Kassir, N.2    Marier, J.3
  • 34
    • 0029157015 scopus 로고
    • Blockade of C5a and C5b-9 generation inhibits leukocyte and platelet activation during extracorporeal circulation
    • Rinder CS, Rinder HM, Smith BR, et al. Blockade of C5a and C5b-9 generation inhibits leukocyte and platelet activation during extracorporeal circulation. J Clin Invest 1995; 96:1564-1572.
    • (1995) J Clin Invest , vol.96 , pp. 1564-1572
    • Rinder, C.S.1    Rinder, H.M.2    Smith, B.R.3
  • 35
    • 78751581550 scopus 로고    scopus 로고
    • Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: The AEGIS clinical trial
    • Kanakura Y, Ohyashiki K, Shichishima T, et al. Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: The AEGIS clinical trial. Int J Hematol 2011; 93:36-46.
    • (2011) Int J Hematol , vol.93 , pp. 36-46
    • Kanakura, Y.1    Ohyashiki, K.2    Shichishima, T.3
  • 36
    • 84868633568 scopus 로고    scopus 로고
    • Successful use of eculizumab in a pediatric patient treated for paroxysmal nocturnal hemoglobinuria
    • Bauters T, Bordon V, Robays H, et al. Successful use of eculizumab in a pediatric patient treated for paroxysmal nocturnal hemoglobinuria. J Pediatr Hematol Oncol 2012; 34:e346-e348.
    • (2012) J Pediatr Hematol Oncol , vol.34
    • Bauters, T.1    Bordon, V.2    Robays, H.3
  • 37
    • 84861209452 scopus 로고    scopus 로고
    • Eculizumab treatment of paroxysmal nocturnal hemoglobinuria presenting as moyamoya syndrome in a 9-year-old male
    • Yen TA, Lu MY, Kuo MF, et al. Eculizumab treatment of paroxysmal nocturnal hemoglobinuria presenting as moyamoya syndrome in a 9-year-old male. Pediatr Blood Cancer 2012; 59:203-204.
    • (2012) Pediatr Blood Cancer , vol.59 , pp. 203-204
    • Yen, T.A.1    Lu, M.Y.2    Kuo, M.F.3
  • 38
    • 65449188713 scopus 로고    scopus 로고
    • Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab
    • Risitano AM, Notaro R, Marando L, et al. Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood 2009; 113:4094-4100.
    • (2009) Blood , vol.113 , pp. 4094-4100
    • Risitano, A.M.1    Notaro, R.2    Marando, L.3
  • 39
    • 77950632086 scopus 로고    scopus 로고
    • Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization
    • Hill A, Rother RP, Arnold L, et al. Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization. Haematologica 2010; 95:567-573.
    • (2010) Haematologica , vol.95 , pp. 567-573
    • Hill, A.1    Rother, R.P.2    Arnold, L.3
  • 40
    • 1642301159 scopus 로고    scopus 로고
    • A novel mechanism of complement-independent clearance of red cells deficient in glycosyl phosphatidylinositol-linked proteins
    • Jasinski M, Pantazopoulos P, Rother RP, et al. A novel mechanism of complement-independent clearance of red cells deficient in glycosyl phosphatidylinositol-linked proteins. Blood 2004; 103:2827-2834.
    • (2004) Blood , vol.103 , pp. 2827-2834
    • Jasinski, M.1    Pantazopoulos, P.2    Rother, R.P.3
  • 41
    • 84893799889 scopus 로고    scopus 로고
    • Genetic variants in C5 and poor response to eculizumab
    • Nishimura J, Yamamoto M, Hayashi S, et al. Genetic variants in C5 and poor response to eculizumab. New Engl J Med 2014; 370:632-639.
    • (2014) New Engl J Med , vol.370 , pp. 632-639
    • Nishimura, J.1    Yamamoto, M.2    Hayashi, S.3
  • 42
    • 67349143377 scopus 로고    scopus 로고
    • Current status of allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria
    • Matos-Fernandez NA, Abou Mourad YR, Caceres W, et al. Current status of allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria. Biol Blood Marrow Transplant 2009; 15:656-661.
    • (2009) Biol Blood Marrow Transplant , vol.15 , pp. 656-661
    • Matos-Fernandez, N.A.1    Abou Mourad, Y.R.2    Caceres, W.3
  • 43
    • 0032936695 scopus 로고    scopus 로고
    • Bone marrow transplants for paroxysmal nocturnal haemoglobinuria
    • Saso R, Marsh J, Cevreska L, et al. Bone marrow transplants for paroxysmal nocturnal haemoglobinuria. Br J Haematol 1999; 104:392-396.
    • (1999) Br J Haematol , vol.104 , pp. 392-396
    • Saso, R.1    Marsh, J.2    Cevreska, L.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.