-
1
-
-
0035822038
-
The hemophilias—from royal genes to gene therapy
-
1 Mannucci, PM, Tuddenham, EG, The hemophilias—from royal genes to gene therapy. N Engl J Med 344 (2001), 1773–1779.
-
(2001)
N Engl J Med
, vol.344
, pp. 1773-1779
-
-
Mannucci, P.M.1
Tuddenham, E.G.2
-
2
-
-
0025017341
-
The increasing prevalence of haemophilia
-
2 Rosendaal, FR, Briët, E, The increasing prevalence of haemophilia. Thromb Haemost, 63, 1990, 145.
-
(1990)
Thromb Haemost
, vol.63
, pp. 145
-
-
Rosendaal, F.R.1
Briët, E.2
-
3
-
-
34347359499
-
How to interpret and pursue an abnormal prothrombin time, activated partial thromboplastin time, and bleeding time in adults
-
3 Kamal, AH, Tefferi, A, Pruthi, RK, How to interpret and pursue an abnormal prothrombin time, activated partial thromboplastin time, and bleeding time in adults. Mayo Clin Proc 82 (2007), 864–873.
-
(2007)
Mayo Clin Proc
, vol.82
, pp. 864-873
-
-
Kamal, A.H.1
Tefferi, A.2
Pruthi, R.K.3
-
4
-
-
0035077234
-
Definitions in hemophilia: recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis
-
4 White, GC 2nd, Rosendaal, F, Aledort, LM, Lusher, JM, Rothschild, C, Ingerslev, J, Definitions in hemophilia: recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost, 85, 2001, 560.
-
(2001)
Thromb Haemost
, vol.85
, pp. 560
-
-
White, G.C.1
Rosendaal, F.2
Aledort, L.M.3
Lusher, J.M.4
Rothschild, C.5
Ingerslev, J.6
-
5
-
-
84885351915
-
Developmental hemostasis: age-specific differences in the levels of hemostatic proteins
-
5 Attard, C, van der Straaten, T, Karlaftis, V, Monagle, P, Ignjatovic, V, Developmental hemostasis: age-specific differences in the levels of hemostatic proteins. J Thromb Haemost 11 (2013), 1850–1854.
-
(2013)
J Thromb Haemost
, vol.11
, pp. 1850-1854
-
-
Attard, C.1
van der Straaten, T.2
Karlaftis, V.3
Monagle, P.4
Ignjatovic, V.5
-
6
-
-
85047687768
-
Comparison of three methods for measuring factor VIII levels in plasma
-
6 Chandler, WL, Ferrell, C, Lee, J, Tun, T, Kha, H, Comparison of three methods for measuring factor VIII levels in plasma. Am J Clin Pathol 120 (2003), 34–39.
-
(2003)
Am J Clin Pathol
, vol.120
, pp. 34-39
-
-
Chandler, W.L.1
Ferrell, C.2
Lee, J.3
Tun, T.4
Kha, H.5
-
7
-
-
11044238614
-
Monitoring haemophilia severity and treatment: new or old laboratory tests?
-
7 Barrowcliffe, TW, Monitoring haemophilia severity and treatment: new or old laboratory tests?. Haemophilia 10:suppl 4 (2004), 109–114.
-
(2004)
Haemophilia
, vol.10
, pp. 109-114
-
-
Barrowcliffe, T.W.1
-
8
-
-
77952766250
-
Severe hemophilia with mild bleeding phenotype: molecular characterization and global coagulation profile
-
8 Santagostino, E, Mancuso, ME, Tripodi, A, et al. Severe hemophilia with mild bleeding phenotype: molecular characterization and global coagulation profile. J Thromb Haemost 8 (2010), 737–743.
-
(2010)
J Thromb Haemost
, vol.8
, pp. 737-743
-
-
Santagostino, E.1
Mancuso, M.E.2
Tripodi, A.3
-
9
-
-
78649982927
-
Discrepancy between one-stage and chromogenic factor VIII activity assay results can lead to misdiagnosis of haemophilia A phenotype
-
9 Oldenburg, J, Pavlova, A, Discrepancy between one-stage and chromogenic factor VIII activity assay results can lead to misdiagnosis of haemophilia A phenotype. Hamostaseologie 30 (2010), 207–211.
-
(2010)
Hamostaseologie
, vol.30
, pp. 207-211
-
-
Oldenburg, J.1
Pavlova, A.2
-
10
-
-
84899768541
-
Haemophilia A mutations in patients with non-severe phenotype associated with a discrepancy between one-stage and chromogenic factor VIII activity assays
-
10 Pavlova, A, Delev, D, Pezeshkpoor, B, Müller, J, Oldenburg, J, Haemophilia A mutations in patients with non-severe phenotype associated with a discrepancy between one-stage and chromogenic factor VIII activity assays. Thromb Haemost 111 (2014), 851–861.
-
(2014)
Thromb Haemost
, vol.111
, pp. 851-861
-
-
Pavlova, A.1
Delev, D.2
Pezeshkpoor, B.3
Müller, J.4
Oldenburg, J.5
-
11
-
-
79959850420
-
Guideline on the management of haemophilia in the fetus and neonate
-
11 Chalmers, E, Williams, M, Brennand, J, et al. Guideline on the management of haemophilia in the fetus and neonate. Br J Haematol 154 (2011), 208–215.
-
(2011)
Br J Haematol
, vol.154
, pp. 208-215
-
-
Chalmers, E.1
Williams, M.2
Brennand, J.3
-
12
-
-
0037810809
-
Standardization of FVIII & FIX assays
-
12 Barrowcliffe, TW, Standardization of FVIII & FIX assays. Haemophilia 9 (2003), 397–402.
-
(2003)
Haemophilia
, vol.9
, pp. 397-402
-
-
Barrowcliffe, T.W.1
-
13
-
-
84863419863
-
F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis
-
13 Gouw, SC, van den Berg, HM, Oldenburg, J, et al. F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis. Blood 119 (2012), 2922–2934.
-
(2012)
Blood
, vol.119
, pp. 2922-2934
-
-
Gouw, S.C.1
van den Berg, H.M.2
Oldenburg, J.3
-
14
-
-
0027520025
-
Inversions disrupting the factor VIII gene are a common cause of severe haemophilia A
-
14 Lakich, D, Kazazian, HH Jr, Antonarakis, SE, Gitschier, J, Inversions disrupting the factor VIII gene are a common cause of severe haemophilia A. Nat Genet 5 (1993), 236–241.
-
(1993)
Nat Genet
, vol.5
, pp. 236-241
-
-
Lakich, D.1
Kazazian, H.H.2
Antonarakis, S.E.3
Gitschier, J.4
-
15
-
-
0036096037
-
Recurrent inversion breaking intron 1 of the factor VIII gene is a frequent cause of severe haemophilia A
-
15 Bagnall, RD, Waseem, N, Green, PM, Giannelli, F, Recurrent inversion breaking intron 1 of the factor VIII gene is a frequent cause of severe haemophilia A. Blood 99 (2002), 168–174.
-
(2002)
Blood
, vol.99
, pp. 168-174
-
-
Bagnall, R.D.1
Waseem, N.2
Green, P.M.3
Giannelli, F.4
-
16
-
-
65449138677
-
Identification of de novo deletion in the factor VIII gene by MLPA technique in two girls with isolated factor VIII deficiency
-
16 Lannoy, N, Abinet, I, Dahan, K, Hermans, C, Identification of de novo deletion in the factor VIII gene by MLPA technique in two girls with isolated factor VIII deficiency. Haemophilia 15 (2009), 797–880.
-
(2009)
Haemophilia
, vol.15
, pp. 797-880
-
-
Lannoy, N.1
Abinet, I.2
Dahan, K.3
Hermans, C.4
-
17
-
-
0035969936
-
Evaluation of DHPLC in the analysis of hemophilia A
-
17 Oldenburg, J, Ivaskevicius, V, Rost, S, et al. Evaluation of DHPLC in the analysis of hemophilia A. J Biochem Biophys Methods 47 (2001), 39–51.
-
(2001)
J Biochem Biophys Methods
, vol.47
, pp. 39-51
-
-
Oldenburg, J.1
Ivaskevicius, V.2
Rost, S.3
-
18
-
-
24644437294
-
Identification of factor VIII gene mutations in 101 patients with haemophilia A: mutation analysis by inversion screening and multiplex PCR and CSGE and molecular modelling of 10 novel missense substitutions
-
18 Jayandharan, G, Shaji, RV, Baidya, S, Nair, SC, Chandy, M, Srivastava, A, Identification of factor VIII gene mutations in 101 patients with haemophilia A: mutation analysis by inversion screening and multiplex PCR and CSGE and molecular modelling of 10 novel missense substitutions. Haemophilia 11 (2005), 481–491.
-
(2005)
Haemophilia
, vol.11
, pp. 481-491
-
-
Jayandharan, G.1
Shaji, R.V.2
Baidya, S.3
Nair, S.C.4
Chandy, M.5
Srivastava, A.6
-
19
-
-
33645730547
-
Characterisation of 96 mutations in 128 unrelated severe haemophilia A patients from France. Description of 62 novel mutations
-
19 Vinciguerra, C, Zawadzki, C, Dargaud, Y, et al. Characterisation of 96 mutations in 128 unrelated severe haemophilia A patients from France. Description of 62 novel mutations. Thromb Haemost 95 (2006), 593–599.
-
(2006)
Thromb Haemost
, vol.95
, pp. 593-599
-
-
Vinciguerra, C.1
Zawadzki, C.2
Dargaud, Y.3
-
20
-
-
33845957929
-
Spectrum of molecular defects and mutation detection rate in patients with mild and moderate hemophilia A
-
20 Bogdanova, N, Markoff, A, Eisert, R, Spectrum of molecular defects and mutation detection rate in patients with mild and moderate hemophilia A. Hum Mutat 28 (2007), 54–60.
-
(2007)
Hum Mutat
, vol.28
, pp. 54-60
-
-
Bogdanova, N.1
Markoff, A.2
Eisert, R.3
-
21
-
-
40049086790
-
Identification of 217 unreported mutations in the F8 gene in a group of 1,410 unselected Italian patients with hemophilia A
-
21 Santacroce, R, Acquila, M, Belvini, D, et al. Identification of 217 unreported mutations in the F8 gene in a group of 1,410 unselected Italian patients with hemophilia A. J Hum Genet 53 (2008), 275–284.
-
(2008)
J Hum Genet
, vol.53
, pp. 275-284
-
-
Santacroce, R.1
Acquila, M.2
Belvini, D.3
-
22
-
-
19544372311
-
Analysis of mRNA in hemophilia A patients with undetectable mutations reveals normal splicing in the factor VIII gene
-
22 El-Maarri, O, Herbiniaux, U, Graw, J, et al. Analysis of mRNA in hemophilia A patients with undetectable mutations reveals normal splicing in the factor VIII gene. J Thromb Haemost 3 (2005), 332–339.
-
(2005)
J Thromb Haemost
, vol.3
, pp. 332-339
-
-
El-Maarri, O.1
Herbiniaux, U.2
Graw, J.3
-
23
-
-
59649126675
-
Identification of deletion carriers in hemophilia B: quantitative real-time polymerase chain reaction or multiple ligation probe amplification
-
23 Casana, P, Haya, S, Cid, AR, et al. Identification of deletion carriers in hemophilia B: quantitative real-time polymerase chain reaction or multiple ligation probe amplification. Transl Res 153 (2009), 114–117.
-
(2009)
Transl Res
, vol.153
, pp. 114-117
-
-
Casana, P.1
Haya, S.2
Cid, A.R.3
-
24
-
-
84876394250
-
A CpG mutational hotspot in a ONECUT binding site accounts for the prevalent variant of hemophilia B Leyden
-
24 Funnell, AP, Wilson, MD, Ballester, B, et al. A CpG mutational hotspot in a ONECUT binding site accounts for the prevalent variant of hemophilia B Leyden. Am J Hum Genet 92 (2013), 460–467.
-
(2013)
Am J Hum Genet
, vol.92
, pp. 460-467
-
-
Funnell, A.P.1
Wilson, M.D.2
Ballester, B.3
-
25
-
-
84891161676
-
Hemophilia B Leyden and once mysterious cis-regulatory mutations
-
25 Funnell, AP, Crossley, M, Hemophilia B Leyden and once mysterious cis-regulatory mutations. Trends Genet 30 (2014), 18–23.
-
(2014)
Trends Genet
, vol.30
, pp. 18-23
-
-
Funnell, A.P.1
Crossley, M.2
-
26
-
-
0026767136
-
Recovery from hemophilia B Leyden: an androgen-responsive element in the factor IX promoter
-
26 Crossley, M, Ludwig, M, Stowell, KM, De Vos, P, Olek, K, Brownlee, GG, Recovery from hemophilia B Leyden: an androgen-responsive element in the factor IX promoter. Science 257 (1992), 377–379.
-
(1992)
Science
, vol.257
, pp. 377-379
-
-
Crossley, M.1
Ludwig, M.2
Stowell, K.M.3
De Vos, P.4
Olek, K.5
Brownlee, G.G.6
-
27
-
-
0035022048
-
Haemophilia B mutations in Sweden: a population-based study of mutational heterogeneity
-
27 Ljung, R, Petrini, P, Tengborn, L, Sjörin, E, Haemophilia B mutations in Sweden: a population-based study of mutational heterogeneity. Br J Haematol 113 (2001), 81–86.
-
(2001)
Br J Haematol
, vol.113
, pp. 81-86
-
-
Ljung, R.1
Petrini, P.2
Tengborn, L.3
Sjörin, E.4
-
28
-
-
47749087043
-
The Italian haemophilia B mutation database: a tool for genetic counselling, carrier detection and prenatal diagnosis
-
28 Tagariello, G, Belvini, D, Salviato, R, et al. The Italian haemophilia B mutation database: a tool for genetic counselling, carrier detection and prenatal diagnosis. Blood Transfus 5 (2007), 158–163.
-
(2007)
Blood Transfus
, vol.5
, pp. 158-163
-
-
Tagariello, G.1
Belvini, D.2
Salviato, R.3
-
29
-
-
84935698157
-
Hemophilia B: molecular pathogenesis and mutation analysis
-
29 Goodeve, AC, Hemophilia B: molecular pathogenesis and mutation analysis. J Thromb Haemost 13 (2015), 1184–1195.
-
(2015)
J Thromb Haemost
, vol.13
, pp. 1184-1195
-
-
Goodeve, A.C.1
-
30
-
-
0025166340
-
‘Founder’ effect in different families with haemophilia B mutation
-
30 Thompson, AR, Bajaj, SP, Chen, SH, MacGillivray, RT, ‘Founder’ effect in different families with haemophilia B mutation. Lancet, 335, 1990, 418.
-
(1990)
Lancet
, vol.335
, pp. 418
-
-
Thompson, A.R.1
Bajaj, S.P.2
Chen, S.H.3
MacGillivray, R.T.4
-
31
-
-
47649108641
-
Mutation analysis of haemophilia B in the Irish population: increased prevalence caused by founder effect
-
31 Jenkins, PV, Egan, H, Keenan, C, et al. Mutation analysis of haemophilia B in the Irish population: increased prevalence caused by founder effect. Haemophilia 14 (2008), 717–722.
-
(2008)
Haemophilia
, vol.14
, pp. 717-722
-
-
Jenkins, P.V.1
Egan, H.2
Keenan, C.3
-
32
-
-
84892404997
-
Genetic sequence analysis of inherited bleeding diseases
-
32 Peyvandi, F, Kunicki, T, Lillicrap, D, Genetic sequence analysis of inherited bleeding diseases. Blood 122 (2013), 3423–3431.
-
(2013)
Blood
, vol.122
, pp. 3423-3431
-
-
Peyvandi, F.1
Kunicki, T.2
Lillicrap, D.3
-
33
-
-
67650258731
-
Fetal loss rate after chorionic villus sampling and amniocentesis: an 11-year national registry study
-
33 Tabor, A, Vestergaard, CH, Lidegaard, Ø, Fetal loss rate after chorionic villus sampling and amniocentesis: an 11-year national registry study. Ultrasound Obstet Gynecol 34 (2009), 19–24.
-
(2009)
Ultrasound Obstet Gynecol
, vol.34
, pp. 19-24
-
-
Tabor, A.1
Vestergaard, C.H.2
Lidegaard, Ø.3
-
34
-
-
0342618532
-
Presence of foetal DNA in maternal plasma and serum
-
34 Lo, YM, Corbetta, N, Chamberlain, PF, et al. Presence of foetal DNA in maternal plasma and serum. Lancet 350 (1997), 485–487.
-
(1997)
Lancet
, vol.350
, pp. 485-487
-
-
Lo, Y.M.1
Corbetta, N.2
Chamberlain, P.F.3
-
35
-
-
80051564369
-
Noninvasive fetal sex determination using cell-free fetal DNA: a systematic review and meta-analysis
-
35 Devaney, SA, Palomaki, GE, Scott, JA, Bianchi, DW, Noninvasive fetal sex determination using cell-free fetal DNA: a systematic review and meta-analysis. JAMA 306 (2011), 627–636.
-
(2011)
JAMA
, vol.306
, pp. 627-636
-
-
Devaney, S.A.1
Palomaki, G.E.2
Scott, J.A.3
Bianchi, D.W.4
-
36
-
-
84857950472
-
Non-invasive tool for foetal sex determination in early gestational age
-
36 Mortarino, M, Garagiola, I, Lotta, LA, Siboni, SM, Semprini, AE, Peyvandi, F, Non-invasive tool for foetal sex determination in early gestational age. Haemophilia 17 (2011), 952–956.
-
(2011)
Haemophilia
, vol.17
, pp. 952-956
-
-
Mortarino, M.1
Garagiola, I.2
Lotta, L.A.3
Siboni, S.M.4
Semprini, A.E.5
Peyvandi, F.6
-
37
-
-
2442505031
-
Preimplantation genetic diagnosis
-
37 Sermon, K, Van Steirteghem, A, Liebaers, I, Preimplantation genetic diagnosis. Lancet 363 (2004), 1633–1641.
-
(2004)
Lancet
, vol.363
, pp. 1633-1641
-
-
Sermon, K.1
Van Steirteghem, A.2
Liebaers, I.3
-
38
-
-
79959561246
-
Prenatal diagnosis and preimplantation genetic diagnosis: novel technologies and state of the art of PGD in different regions of the world
-
38 Peyvandi, F, Garagiola, I, Mortarino, M, Prenatal diagnosis and preimplantation genetic diagnosis: novel technologies and state of the art of PGD in different regions of the world. Haemophilia 17:suppl 1 (2011), 14–17.
-
(2011)
Haemophilia
, vol.17
, pp. 14-17
-
-
Peyvandi, F.1
Garagiola, I.2
Mortarino, M.3
-
39
-
-
84857971879
-
Comparing bleed frequency and factor concentrate use between haemophilia A and haemophilia B
-
39 Nagel, K, Walker, I, Decker, K, Chan, HKC, Pai, MK, Comparing bleed frequency and factor concentrate use between haemophilia A and haemophilia B. Haemophilia 17 (2011), 872–874.
-
(2011)
Haemophilia
, vol.17
, pp. 872-874
-
-
Nagel, K.1
Walker, I.2
Decker, K.3
Chan, H.K.C.4
Pai, M.K.5
-
40
-
-
84885052482
-
Hemophilia A and hemophilia B: different types of diseases?
-
40 Santagostino, E, Fasulo, MR, Hemophilia A and hemophilia B: different types of diseases?. Semin Thromb Hemost 39 (2013), 697–701.
-
(2013)
Semin Thromb Hemost
, vol.39
, pp. 697-701
-
-
Santagostino, E.1
Fasulo, M.R.2
-
41
-
-
84926151321
-
Similar bleeding phenotype in young children with haemophilia A and B: a cohort study
-
41 Clausen, N, Petrini, P, Claeyssens-Donadel, S, Gouw, SC, Liesner, R, PedNet and Research of Determinants of Inhibitor development (RODIN) Study Group. Similar bleeding phenotype in young children with haemophilia A and B: a cohort study. Haemophilia 20 (2014), 747–755.
-
(2014)
Haemophilia
, vol.20
, pp. 747-755
-
-
Clausen, N.1
Petrini, P.2
Claeyssens-Donadel, S.3
Gouw, S.C.4
Liesner, R.5
-
42
-
-
0035120786
-
Perinatal management of neonates with haemophilia
-
42 Kulkarni, R, Lusher, J, Perinatal management of neonates with haemophilia. Br J Haematol 112 (2001), 264–274.
-
(2001)
Br J Haematol
, vol.112
, pp. 264-274
-
-
Kulkarni, R.1
Lusher, J.2
-
43
-
-
84855759786
-
Neonatal bleeding in haemophilia: a European cohort study
-
43 Richards, M, Lavigne Lissalde, G, Combescure, C, et al. Neonatal bleeding in haemophilia: a European cohort study. Br J Haematol 156 (2012), 374–382.
-
(2012)
Br J Haematol
, vol.156
, pp. 374-382
-
-
Richards, M.1
Lavigne Lissalde, G.2
Combescure, C.3
-
44
-
-
70449576002
-
Sites of initial bleeding episodes, mode of delivery and age of diagnosis in babies with haemophilia diagnosed before the age of 2 years: a report from The Centers for Disease Control and Prevention's (CDC) Universal Data Collection (UDC) project
-
44 Kulkarni, R, Soucie, JM, Lusher, J, et al. Sites of initial bleeding episodes, mode of delivery and age of diagnosis in babies with haemophilia diagnosed before the age of 2 years: a report from The Centers for Disease Control and Prevention's (CDC) Universal Data Collection (UDC) project. Haemophilia 15 (2009), 1281–1290.
-
(2009)
Haemophilia
, vol.15
, pp. 1281-1290
-
-
Kulkarni, R.1
Soucie, J.M.2
Lusher, J.3
-
45
-
-
84872102240
-
The diagnosis and management of congenital hemophilia
-
45 Carcao, MD, The diagnosis and management of congenital hemophilia. Semin Thromb Hemost 38 (2012), 727–734.
-
(2012)
Semin Thromb Hemost
, vol.38
, pp. 727-734
-
-
Carcao, M.D.1
-
46
-
-
34250702800
-
Phenotypic heterogeneity in severe hemophilia
-
46 van den Berg, HM, De Groot, PH, Fischer, K, Phenotypic heterogeneity in severe hemophilia. J Thromb Haemost 5:suppl 1 (2007), 151–156.
-
(2007)
J Thromb Haemost
, vol.5
, pp. 151-156
-
-
van den Berg, H.M.1
De Groot, P.H.2
Fischer, K.3
-
47
-
-
79955142222
-
Trends in bleeding patterns during prophylaxis for severe haemophilia: observations from a series of prospective clinical trials
-
47 Fischer, K, Collins, P, Björkman, S, et al. Trends in bleeding patterns during prophylaxis for severe haemophilia: observations from a series of prospective clinical trials. Haemophilia 17 (2011), 433–438.
-
(2011)
Haemophilia
, vol.17
, pp. 433-438
-
-
Fischer, K.1
Collins, P.2
Björkman, S.3
-
48
-
-
70449568470
-
Changing patterns of bleeding in patients with severe haemophilia A
-
48 Stephensen, D, Tait, RC, Brodie, N, et al. Changing patterns of bleeding in patients with severe haemophilia A. Haemophilia 15 (2009), 1210–1214.
-
(2009)
Haemophilia
, vol.15
, pp. 1210-1214
-
-
Stephensen, D.1
Tait, R.C.2
Brodie, N.3
-
49
-
-
7444264189
-
Haemophilic arthropathy
-
49 Luck, JV Jr, Silva, M, Rodriguez-Merchan, EC, Ghalambor, N, Zahiri, CA, Finn, RS, Haemophilic arthropathy. J Am Acad Orthop Surg 12 (2004), 234–245.
-
(2004)
J Am Acad Orthop Surg
, vol.12
, pp. 234-245
-
-
Luck, J.V.1
Silva, M.2
Rodriguez-Merchan, E.C.3
Ghalambor, N.4
Zahiri, C.A.5
Finn, R.S.6
-
50
-
-
77956489886
-
Blood-induced joint disease: the pathophysiology of haemophilic arthropathy
-
50 Valentino, LA, Blood-induced joint disease: the pathophysiology of haemophilic arthropathy. J Thromb Haemost 8 (2010), 1895–1902.
-
(2010)
J Thromb Haemost
, vol.8
, pp. 1895-1902
-
-
Valentino, L.A.1
-
51
-
-
34547757915
-
Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia
-
51 Manco-Johnson, MJ, Abshire, TC, Shapiro, AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 357 (2007), 535–544.
-
(2007)
N Engl J Med
, vol.357
, pp. 535-544
-
-
Manco-Johnson, M.J.1
Abshire, T.C.2
Shapiro, A.D.3
-
52
-
-
84885751905
-
Joint replacement
-
CA Lee EE Berntorp K Hoots Blackwell Publishing Chichester, UK
-
52 Goddard, N, Joint replacement. Lee, CA, Berntorp, EE, Hoots, K, (eds.) Textbook of haemophilia, 2010, Blackwell Publishing, Chichester, UK, 176–181.
-
(2010)
Textbook of haemophilia
, pp. 176-181
-
-
Goddard, N.1
-
53
-
-
84885066073
-
Arthropathy in patients with moderate hemophilia A: a systematic review of the literature
-
53 Di Minno, MN, Ambrosino, P, Franchini, M, Coppola, A, Di Minno, G, Arthropathy in patients with moderate hemophilia A: a systematic review of the literature. Semin Thromb Hemost 39 (2013), 723–731.
-
(2013)
Semin Thromb Hemost
, vol.39
, pp. 723-731
-
-
Di Minno, M.N.1
Ambrosino, P.2
Franchini, M.3
Coppola, A.4
Di Minno, G.5
-
54
-
-
84934437220
-
A cross-sectional study of bleeding phenotype in haemophilia A carriers
-
54 Paroskie, A, Gailani, D, DeBaun, MR, Sidonio, RF Jr, A cross-sectional study of bleeding phenotype in haemophilia A carriers. Br J Haematol 170 (2015), 223–228.
-
(2015)
Br J Haematol
, vol.170
, pp. 223-228
-
-
Paroskie, A.1
Gailani, D.2
DeBaun, M.R.3
Sidonio, R.F.4
-
55
-
-
84871011258
-
Guidelines for the management of hemophilia
-
55 Srivastava, A, Brewer, AK, Mauser-Bunschoten, EP, et al. Guidelines for the management of hemophilia. Haemophilia 19 (2013), e1–47.
-
(2013)
Haemophilia
, vol.19
, pp. e1-47
-
-
Srivastava, A.1
Brewer, A.K.2
Mauser-Bunschoten, E.P.3
-
56
-
-
0034814399
-
Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy
-
56 Fischer, K, Van der Bom, JG, Mauser-Bunschoten, EP, et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 7 (2001), 446–452.
-
(2001)
Haemophilia
, vol.7
, pp. 446-452
-
-
Fischer, K.1
Van der Bom, J.G.2
Mauser-Bunschoten, E.P.3
-
57
-
-
0042411064
-
Consensus perspectives on prophylactic therapy for haemophilia: summary statement
-
57 Berntorp, E, Astermark, J, Björkman, S, et al. Consensus perspectives on prophylactic therapy for haemophilia: summary statement. Haemophilia 9:suppl 1 (2003), 1–4.
-
(2003)
Haemophilia
, vol.9
, pp. 1-4
-
-
Berntorp, E.1
Astermark, J.2
Björkman, S.3
-
58
-
-
0028004222
-
A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs
-
58 Aledort, LM, Haschmeyer, RH, Pettersson, H, the Orthopaedic Outcome Study Group. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. J Intern Med 236 (1994), 391–399.
-
(1994)
J Intern Med
, vol.236
, pp. 391-399
-
-
Aledort, L.M.1
Haschmeyer, R.H.2
Pettersson, H.3
-
59
-
-
0026635406
-
Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
-
59 Nilsson, IM, Berntorp, E, Löfqvist, T, Pettersson, H, Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 232 (1992), 25–32.
-
(1992)
J Intern Med
, vol.232
, pp. 25-32
-
-
Nilsson, I.M.1
Berntorp, E.2
Löfqvist, T.3
Pettersson, H.4
-
60
-
-
0036827020
-
Prophylaxis treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen
-
60 Fischer, K, Astermark, J, van der Bom, JG, et al. Prophylaxis treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen. Haemophilia 8 (2002), 753–760.
-
(2002)
Haemophilia
, vol.8
, pp. 753-760
-
-
Fischer, K.1
Astermark, J.2
van der Bom, J.G.3
-
61
-
-
84871008917
-
Short-term low-dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi-centre pilot study in China
-
61 Tang, L, Wu, R, Sun, J, et al. Short-term low-dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi-centre pilot study in China. Haemophilia 19 (2013), 27–34.
-
(2013)
Haemophilia
, vol.19
, pp. 27-34
-
-
Tang, L.1
Wu, R.2
Sun, J.3
-
62
-
-
79953306825
-
A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)
-
62 Gringeri, A, Lundin, B, von Mackensen, S, Mantovani, L, Mannucci, PM, A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 9 (2011), 700–710.
-
(2011)
J Thromb Haemost
, vol.9
, pp. 700-710
-
-
Gringeri, A.1
Lundin, B.2
von Mackensen, S.3
Mantovani, L.4
Mannucci, P.M.5
-
63
-
-
66049119139
-
Improved treatment feasibility in children with hemophilia using arteriovenous fistulae: the results after seven years of follow-up
-
63 Mancuso, ME, Berardinelli, L, Beretta, C, Raiteri, M, Pozzoli, E, Santagostino, E, Improved treatment feasibility in children with hemophilia using arteriovenous fistulae: the results after seven years of follow-up. Haematologica 94 (2009), 687–692.
-
(2009)
Haematologica
, vol.94
, pp. 687-692
-
-
Mancuso, M.E.1
Berardinelli, L.2
Beretta, C.3
Raiteri, M.4
Pozzoli, E.5
Santagostino, E.6
-
64
-
-
84862872608
-
Personalized prophylaxis
-
64 Collins, PW, Personalized prophylaxis. Haemophilia 18:suppl 4 (2012), 131–135.
-
(2012)
Haemophilia
, vol.18
, pp. 131-135
-
-
Collins, P.W.1
-
65
-
-
24644521462
-
Variability in clinical phenotype of severe hemophilia: the role of the first joint bleed
-
65 van Dijk, K, Fischer, K, van der Bom, JG, Grobbee, DE, van den Berg, HM, Variability in clinical phenotype of severe hemophilia: the role of the first joint bleed. Haemophilia 11 (2005), 438–443.
-
(2005)
Haemophilia
, vol.11
, pp. 438-443
-
-
van Dijk, K.1
Fischer, K.2
van der Bom, J.G.3
Grobbee, D.E.4
van den Berg, H.M.5
-
66
-
-
0017336844
-
1-Deamino-8-d-arginine vasopressin: a new pharmacological approach to the management of haemophilia and von Willebrands' diseases
-
66 Mannucci, PM, Ruggeri, ZM, Pareti, FI, Capitanio, A, 1-Deamino-8-d-arginine vasopressin: a new pharmacological approach to the management of haemophilia and von Willebrands' diseases. Lancet 1 (1977), 869–872.
-
(1977)
Lancet
, vol.1
, pp. 869-872
-
-
Mannucci, P.M.1
Ruggeri, Z.M.2
Pareti, F.I.3
Capitanio, A.4
-
68
-
-
0013856638
-
Production of high-potency concentrates of antihaemophilic globulin in a closed-bag system
-
68 Pool, JG, Shannon, AE, Production of high-potency concentrates of antihaemophilic globulin in a closed-bag system. N Engl J Med 273 (1965), 1443–1447.
-
(1965)
N Engl J Med
, vol.273
, pp. 1443-1447
-
-
Pool, J.G.1
Shannon, A.E.2
-
69
-
-
0013829369
-
Clinical use of a new glycine-precipitated antihaemophilic fraction
-
69 Webster, WP, Roberts, HR, Thelin, GM, Wagner, RH, Brinkhous, KM, Clinical use of a new glycine-precipitated antihaemophilic fraction. Am J Med Sci 250 (1965), 643–651.
-
(1965)
Am J Med Sci
, vol.250
, pp. 643-651
-
-
Webster, W.P.1
Roberts, H.R.2
Thelin, G.M.3
Wagner, R.H.4
Brinkhous, K.M.5
-
70
-
-
3042704579
-
AIDS, hepatitis and hemophilia in the 1980s: memoirs from an insider
-
70 Mannucci, PM, AIDS, hepatitis and hemophilia in the 1980s: memoirs from an insider. J Thromb Haemost 1 (2003), 2065–2069.
-
(2003)
J Thromb Haemost
, vol.1
, pp. 2065-2069
-
-
Mannucci, P.M.1
-
71
-
-
84911484405
-
Innovative approach for improved rFVIII concentrate
-
71 Morfini, M, Innovative approach for improved rFVIII concentrate. Eur J Haematol 93 (2014), 361–368.
-
(2014)
Eur J Haematol
, vol.93
, pp. 361-368
-
-
Morfini, M.1
-
72
-
-
77955492929
-
Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment
-
72 Mei, B, Pan, C, Jiang, H, et al. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment. Blood 116 (2010), 270–279.
-
(2010)
Blood
, vol.116
, pp. 270-279
-
-
Mei, B.1
Pan, C.2
Jiang, H.3
-
73
-
-
80052167452
-
Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide
-
73 Ostergaard, H, Bjelke, JR, Hansen, L, et al. Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide. Blood 118 (2011), 2333–2341.
-
(2011)
Blood
, vol.118
, pp. 2333-2341
-
-
Ostergaard, H.1
Bjelke, J.R.2
Hansen, L.3
-
74
-
-
84876182114
-
Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-man trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A
-
74 Tiede, A, Brand, B, Fischer, R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-man trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost 11 (2013), 670–678.
-
(2013)
J Thromb Haemost
, vol.11
, pp. 670-678
-
-
Tiede, A.1
Brand, B.2
Fischer, R.3
-
75
-
-
84898028710
-
Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A
-
75 Coyle, TE, Reding, MT, Lin, JC, Michaels, LA, Shah, A, Powell, J, Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A. J Thromb Haemost 12 (2014), 488–496.
-
(2014)
J Thromb Haemost
, vol.12
, pp. 488-496
-
-
Coyle, T.E.1
Reding, M.T.2
Lin, J.C.3
Michaels, L.A.4
Shah, A.5
Powell, J.6
-
76
-
-
84919491430
-
Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial
-
76 Collins, PW, Young, G, Knobe, K, et al. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood 124 (2014), 3880–3886.
-
(2014)
Blood
, vol.124
, pp. 3880-3886
-
-
Collins, P.W.1
Young, G.2
Knobe, K.3
-
77
-
-
84868152441
-
Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX
-
77 Collins, PW, Moss, J, Knobe, K, Groth, A, Colberg, T, Watson, E, Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost 10 (2012), 2305–2312.
-
(2012)
J Thromb Haemost
, vol.10
, pp. 2305-2312
-
-
Collins, P.W.1
Moss, J.2
Knobe, K.3
Groth, A.4
Colberg, T.5
Watson, E.6
-
78
-
-
84893123337
-
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
-
78 Mahlangu, J, Powell, JS, Ragni, MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood 123 (2014), 317–325.
-
(2014)
Blood
, vol.123
, pp. 317-325
-
-
Mahlangu, J.1
Powell, J.S.2
Ragni, M.V.3
-
79
-
-
84889769562
-
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B
-
79 Powell, JS, Pasi, KJ, Ragni, MV, et al. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med 369 (2013), 2313–2323.
-
(2013)
N Engl J Med
, vol.369
, pp. 2313-2323
-
-
Powell, J.S.1
Pasi, K.J.2
Ragni, M.V.3
-
80
-
-
84866594053
-
Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients
-
80 Santagostino, E, Negrier, C, Klamroth, R, et al. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood 120 (2012), 2405–2411.
-
(2012)
Blood
, vol.120
, pp. 2405-2411
-
-
Santagostino, E.1
Negrier, C.2
Klamroth, R.3
-
81
-
-
84946493489
-
Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients
-
81 Martinowitz, U, Lissitchkov, T, Lubetsky, A, et al. Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients. Haemophilia 21 (2015), 784–790.
-
(2015)
Haemophilia
, vol.21
, pp. 784-790
-
-
Martinowitz, U.1
Lissitchkov, T.2
Lubetsky, A.3
-
82
-
-
60149088848
-
Origins and mechanisms of miRNAs and siRNAs
-
82 Carthew, RW, Sontheimer, EJ, Origins and mechanisms of miRNAs and siRNAs. Cell 136 (2009), 642–655.
-
(2009)
Cell
, vol.136
, pp. 642-655
-
-
Carthew, R.W.1
Sontheimer, E.J.2
-
83
-
-
77954220072
-
Aptamers as therapeutics
-
83 Keefe, AD, Pai, S, Ellington, A, Aptamers as therapeutics. Nat Rev Drug Discov 9 (2010), 537–550.
-
(2010)
Nat Rev Drug Discov
, vol.9
, pp. 537-550
-
-
Keefe, A.D.1
Pai, S.2
Ellington, A.3
-
84
-
-
84928764693
-
Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial
-
84 Chowdary, P, Lethagen, S, Friedrich, U, et al. Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial. J Thromb Haemost 13 (2015), 743–754.
-
(2015)
J Thromb Haemost
, vol.13
, pp. 743-754
-
-
Chowdary, P.1
Lethagen, S.2
Friedrich, U.3
-
85
-
-
84870302675
-
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model
-
85 Kitazawa, T, Igawa, T, Sampei, Z, et al. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med 18 (2012), 1570–1574.
-
(2012)
Nat Med
, vol.18
, pp. 1570-1574
-
-
Kitazawa, T.1
Igawa, T.2
Sampei, Z.3
-
86
-
-
84970937901
-
Long-term safety and prophylactic efficacy of once weekly subcutaneous administration of ACE910, in Japanese hemophilia A patients with and without FVIII inhibitors: interim results of the extension study of a phase 1 study
-
(abstr).
-
86 Shima, M, Hanabusa, H, Taki, M, et al. Long-term safety and prophylactic efficacy of once weekly subcutaneous administration of ACE910, in Japanese hemophilia A patients with and without FVIII inhibitors: interim results of the extension study of a phase 1 study. J Thromb Haemost, 13(suppl S2), 2015, 6 (abstr).
-
(2015)
J Thromb Haemost
, vol.13
, pp. 6
-
-
Shima, M.1
Hanabusa, H.2
Taki, M.3
-
87
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
87 Nathwani, AC, Tuddenham, EG, Rangarajan, S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 365 (2011), 2357–2365.
-
(2011)
N Engl J Med
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
-
88
-
-
84911383748
-
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
-
88 Nathwani, AC, Reiss, UM, Tuddenham, EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 371 (2014), 1994–2004.
-
(2014)
N Engl J Med
, vol.371
, pp. 1994-2004
-
-
Nathwani, A.C.1
Reiss, U.M.2
Tuddenham, E.G.3
-
89
-
-
84899533543
-
Current status of haemophilia gene therapy
-
89 High, KH, Nathwani, A, Spencer, T, Lillicrap, D, Current status of haemophilia gene therapy. Haemophilia 20:suppl 4 (2014), 43–49.
-
(2014)
Haemophilia
, vol.20
, pp. 43-49
-
-
High, K.H.1
Nathwani, A.2
Spencer, T.3
Lillicrap, D.4
-
90
-
-
84877704167
-
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
-
90 McIntosh, J, Lenting, PJ, Rosales, C, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood 121 (2013), 3335–3344.
-
(2013)
Blood
, vol.121
, pp. 3335-3344
-
-
McIntosh, J.1
Lenting, P.J.2
Rosales, C.3
-
91
-
-
84906667355
-
Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery
-
91 Wang, Q, Dong, B, Firrman, J, et al. Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery. Hum Gene Ther Methods 25 (2014), 261–268.
-
(2014)
Hum Gene Ther Methods
, vol.25
, pp. 261-268
-
-
Wang, Q.1
Dong, B.2
Firrman, J.3
-
92
-
-
79953322192
-
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of hemophilia A
-
92 Matsui, H, Hegadorn, C, Ozelo, M, et al. A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of hemophilia A. Mol Ther 19 (2011), 723–730.
-
(2011)
Mol Ther
, vol.19
, pp. 723-730
-
-
Matsui, H.1
Hegadorn, C.2
Ozelo, M.3
-
93
-
-
18344405441
-
A detailed comparison of the performance of the standard versus the Nijmegen modification of the Bethesda assay in detecting factor VIII:C inhibitors in the haemophilia A population of Canada. Association of Hemophilia Centre Directors of Canada. Factor VIII/IX Subcommittee of Scientific and Standardization Committee of International Society on Thrombosis and Haemostasis
-
93 Giles, AR, Verbruggen, B, Rivard, GE, Teitel, J, Walker, I, A detailed comparison of the performance of the standard versus the Nijmegen modification of the Bethesda assay in detecting factor VIII:C inhibitors in the haemophilia A population of Canada. Association of Hemophilia Centre Directors of Canada. Factor VIII/IX Subcommittee of Scientific and Standardization Committee of International Society on Thrombosis and Haemostasis. Thromb Haemost 79 (1998), 872–875.
-
(1998)
Thromb Haemost
, vol.79
, pp. 872-875
-
-
Giles, A.R.1
Verbruggen, B.2
Rivard, G.E.3
Teitel, J.4
Walker, I.5
-
94
-
-
9144232123
-
Inhibitors in congenital coagulation disorders
-
94 Key, NS, Inhibitors in congenital coagulation disorders. Br J Haematol 127 (2004), 379–391.
-
(2004)
Br J Haematol
, vol.127
, pp. 379-391
-
-
Key, N.S.1
-
95
-
-
73949108325
-
UK Haemophilia Centre Doctors' Organisation. The incidence of FVIII and FIX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977–1999
-
95 Darby, SC, Keeling, DM, Spooner, RJ, et al. UK Haemophilia Centre Doctors' Organisation. The incidence of FVIII and FIX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977–1999. J Thromb Haemost 2 (2004), 1047–1054.
-
(2004)
J Thromb Haemost
, vol.2
, pp. 1047-1054
-
-
Darby, S.C.1
Keeling, D.M.2
Spooner, R.J.3
-
96
-
-
84860334450
-
F8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicity
-
96 Miller, CH, Benson, J, Ellingsen, D, et al. F8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicity. Haemophilia 18 (2012), 375–382.
-
(2012)
Haemophilia
, vol.18
, pp. 375-382
-
-
Miller, C.H.1
Benson, J.2
Ellingsen, D.3
-
97
-
-
33645751563
-
Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A
-
97 Astermark, J, Oldenburg, J, Pavlova, A, Berntorp, E, Lefvert, AK, Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A. Blood 107 (2006), 3167–3172.
-
(2006)
Blood
, vol.107
, pp. 3167-3172
-
-
Astermark, J.1
Oldenburg, J.2
Pavlova, A.3
Berntorp, E.4
Lefvert, A.K.5
-
98
-
-
33845239946
-
Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A
-
98 Astermark, J, Oldenburg, J, Carlson, J, et al. Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A. Blood 108 (2006), 3739–3745.
-
(2006)
Blood
, vol.108
, pp. 3739-3745
-
-
Astermark, J.1
Oldenburg, J.2
Carlson, J.3
-
99
-
-
33846420672
-
Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A
-
99 Astermark, J, Wang, X, Oldenburg, J, Berntorp, E, Lefvert, AK, Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A. J Thromb Haemost 5 (2007), 263–265.
-
(2007)
J Thromb Haemost
, vol.5
, pp. 263-265
-
-
Astermark, J.1
Wang, X.2
Oldenburg, J.3
Berntorp, E.4
Lefvert, A.K.5
-
100
-
-
84905648933
-
The Fc gamma receptor IIa R131H polymorphism is associated with inhibitor development in severe hemophilia A
-
100 Eckhardt, CL, Astermark, J, Nagelkerke, SQ, et al. The Fc gamma receptor IIa R131H polymorphism is associated with inhibitor development in severe hemophilia A. J Thromb Haemost 12 (2014), 1294–1301.
-
(2014)
J Thromb Haemost
, vol.12
, pp. 1294-1301
-
-
Eckhardt, C.L.1
Astermark, J.2
Nagelkerke, S.Q.3
-
101
-
-
0031598278
-
Inhibitors occur more frequently in African-Americans and Latino hemophiliacs
-
101 Aledort, LM, Di Michele, DM, Inhibitors occur more frequently in African-Americans and Latino hemophiliacs. Haemophilia, 4, 1998, 68.
-
(1998)
Haemophilia
, vol.4
, pp. 68
-
-
Aledort, L.M.1
Di Michele, D.M.2
-
102
-
-
34249690732
-
Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study
-
102 Gouw, SC, van der Bom, JG, van den Berg, HM, Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood 109 (2007), 4648–4654.
-
(2007)
Blood
, vol.109
, pp. 4648-4654
-
-
Gouw, S.C.1
van der Bom, J.G.2
van den Berg, H.M.3
-
103
-
-
30344434999
-
Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A
-
103 Goudemand, J, Rothschild, C, Demiguel, V, et al. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 107 (2006), 46–51.
-
(2006)
Blood
, vol.107
, pp. 46-51
-
-
Goudemand, J.1
Rothschild, C.2
Demiguel, V.3
-
104
-
-
77954506432
-
Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review
-
104 Iorio, A, Halimeh, S, Holzhauer, S, et al. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost 8 (2010), 1256–1265.
-
(2010)
J Thromb Haemost
, vol.8
, pp. 1256-1265
-
-
Iorio, A.1
Halimeh, S.2
Holzhauer, S.3
-
105
-
-
84872450786
-
Factor VIII products and inhibitor formation in severe hemophilia A
-
105 Gouw, SC, van der Bom, JG, Ljung, R, et al. Factor VIII products and inhibitor formation in severe hemophilia A. N Engl J Med 368 (2013), 231–239.
-
(2013)
N Engl J Med
, vol.368
, pp. 231-239
-
-
Gouw, S.C.1
van der Bom, J.G.2
Ljung, R.3
-
106
-
-
84914142516
-
Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe haemophilia A, 2000–2011
-
106 Collins, PW, Palmer, BP, Chalmers, EA, et al. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe haemophilia A, 2000–2011. Blood 124 (2014), 3389–3397.
-
(2014)
Blood
, vol.124
, pp. 3389-3397
-
-
Collins, P.W.1
Palmer, B.P.2
Chalmers, E.A.3
-
107
-
-
84914140043
-
Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A
-
107 Calvez, T, Chambost, H, Claeyssens-Donadel, S, et al. Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A. Blood 124 (2014), 3398–3408.
-
(2014)
Blood
, vol.124
, pp. 3398-3408
-
-
Calvez, T.1
Chambost, H.2
Claeyssens-Donadel, S.3
-
108
-
-
84991317784
-
Source of factor VIII replacement (PLASMATIC OR RECOMBINANT) and incidence of inhibitory alloantibodies in previously untreated patients with severe hemophilia A: the multicenter randomized Sippet study. 57th Annual Meeting & Exposition of the American Society of Hematology; Orlando, FL; Dec 5–8, 2015
-
(accessed Nov 13, 2015).
-
108 Peyvandi, F, Mannucci, PM, Garagiola, I, et al. Source of factor VIII replacement (PLASMATIC OR RECOMBINANT) and incidence of inhibitory alloantibodies in previously untreated patients with severe hemophilia A: the multicenter randomized Sippet study. 57th Annual Meeting & Exposition of the American Society of Hematology; Orlando, FL; Dec 5–8, 2015. https://ash.confex.com/ash/2015/webprogram/Paper82866.html (accessed Nov 13, 2015).
-
-
-
Peyvandi, F.1
Mannucci, P.M.2
Garagiola, I.3
-
109
-
-
84931274723
-
Inhibitor development in haemophilia according to concentrate. Four-year results from the European HAemophilia Safety Surveillance (EUHASS) project
-
109 Fischer, K, Lassila, R, Peyvandi, F, et al. Inhibitor development in haemophilia according to concentrate. Four-year results from the European HAemophilia Safety Surveillance (EUHASS) project. Thromb Haemost 113 (2015), 968–975.
-
(2015)
Thromb Haemost
, vol.113
, pp. 968-975
-
-
Fischer, K.1
Lassila, R.2
Peyvandi, F.3
-
110
-
-
0842333021
-
Intensive exposure to factor VIII is a risk factor for inhibitor development in mild hemophilia A
-
110 Sharathkumar, A, Lillicrap, D, Blanchette, VS, et al. Intensive exposure to factor VIII is a risk factor for inhibitor development in mild hemophilia A. J Thromb Haemost 1 (2003), 1228–1236.
-
(2003)
J Thromb Haemost
, vol.1
, pp. 1228-1236
-
-
Sharathkumar, A.1
Lillicrap, D.2
Blanchette, V.S.3
-
111
-
-
84887363261
-
Factor VIII gene (F8) and the risk of inhibitor development in nonsevere hemophilia A
-
111 Eckhardt, CL, van Velzen, AS, Peters, M, et al. Factor VIII gene (F8) and the risk of inhibitor development in nonsevere hemophilia A. Blood 122 (2013), 1954–1962.
-
(2013)
Blood
, vol.122
, pp. 1954-1962
-
-
Eckhardt, C.L.1
van Velzen, A.S.2
Peters, M.3
-
112
-
-
0030884459
-
Loss of tolerance to exogenous and endogenous factor VIII in a mild hemophilia A patient with an Arg593 to Cys mutation
-
112 Thompson, AR, Murphy, ME, Liu, M, et al. Loss of tolerance to exogenous and endogenous factor VIII in a mild hemophilia A patient with an Arg593 to Cys mutation. Blood 90 (1997), 1902–1910.
-
(1997)
Blood
, vol.90
, pp. 1902-1910
-
-
Thompson, A.R.1
Murphy, M.E.2
Liu, M.3
-
113
-
-
0033121017
-
Antifactor VIII antibody inhibiting allogeneic but not autologous factor VIII in patients with mild hemophilia A
-
113 Peerlinck, K, Jacquemin, MG, Arnout, J, et al. Antifactor VIII antibody inhibiting allogeneic but not autologous factor VIII in patients with mild hemophilia A. Blood 93 (1999), 2267–2273.
-
(1999)
Blood
, vol.93
, pp. 2267-2273
-
-
Peerlinck, K.1
Jacquemin, M.G.2
Arnout, J.3
-
114
-
-
84943166338
-
Inhibitor development in non-severe haemophilia across Europe
-
114 Fischer, K, Iorio, A, Lassila, R, et al. Inhibitor development in non-severe haemophilia across Europe. Thromb Haemost 114 (2015), 670–675.
-
(2015)
Thromb Haemost
, vol.114
, pp. 670-675
-
-
Fischer, K.1
Iorio, A.2
Lassila, R.3
-
115
-
-
0036123458
-
Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study
-
115 Poon, MC, Lillicrap, D, Hensman, C, Card, R, Scully, MF, Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study. Thromb Haemost 87 (2002), 431–435.
-
(2002)
Thromb Haemost
, vol.87
, pp. 431-435
-
-
Poon, M.C.1
Lillicrap, D.2
Hensman, C.3
Card, R.4
Scully, M.F.5
-
116
-
-
84935756053
-
Inhibitor development and mortality in non-severe hemophilia A
-
116 Eckhardt, CL, Loomans, JI, van Velzen, AS, et al. Inhibitor development and mortality in non-severe hemophilia A. J Thromb Haemost 13 (2015), 1217–1225.
-
(2015)
J Thromb Haemost
, vol.13
, pp. 1217-1225
-
-
Eckhardt, C.L.1
Loomans, J.I.2
van Velzen, A.S.3
-
117
-
-
84928272570
-
Impact of inhibitors on hemophilia A mortality in the United States
-
117 Walsh, CE, Soucie, JM, Miller, CH, United States Hemophilia Treatment Center Network. Impact of inhibitors on hemophilia A mortality in the United States. Am J Hematol 90 (2015), 400–405.
-
(2015)
Am J Hematol
, vol.90
, pp. 400-405
-
-
Walsh, C.E.1
Soucie, J.M.2
Miller, C.H.3
-
118
-
-
34548304886
-
European study on orthopaedic status of haemophilia patients with inhibitors
-
118 Morfini, M, Haya, S, Tagariello, G, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 13 (2007), 606–612.
-
(2007)
Haemophilia
, vol.13
, pp. 606-612
-
-
Morfini, M.1
Haya, S.2
Tagariello, G.3
-
119
-
-
81355132467
-
Physical functioning in boys with hemophilia in the U.S
-
119 Monahan, PE, Baker, JR, Riske, B, Soucie, JM, Physical functioning in boys with hemophilia in the U.S. Am J Prev Med 41:6S4 (2011), S360–S368.
-
(2011)
Am J Prev Med
, vol.41
, Issue.6S4
, pp. S360-S368
-
-
Monahan, P.E.1
Baker, J.R.2
Riske, B.3
Soucie, J.M.4
-
120
-
-
84856866914
-
International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison
-
120 Hay, CR, DiMichele, DM, International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 119 (2012), 1335–1344.
-
(2012)
Blood
, vol.119
, pp. 1335-1344
-
-
Hay, C.R.1
DiMichele, D.M.2
-
121
-
-
84871666279
-
More than a decade of international experience with a pdFVIII/VWF concentrate in immune tolerance
-
121 Santagostino, E, More than a decade of international experience with a pdFVIII/VWF concentrate in immune tolerance. Haemophilia 19:suppl 1 (2013), 8–11.
-
(2013)
Haemophilia
, vol.19
, pp. 8-11
-
-
Santagostino, E.1
-
122
-
-
63049130236
-
The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy
-
122 Dimichele, D, The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia 15 (2009), 320–328.
-
(2009)
Haemophilia
, vol.15
, pp. 320-328
-
-
Dimichele, D.1
-
123
-
-
84890858676
-
Primary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrate
-
123 Oldenburg, J, Jiménez-Yuste, V, Peiró-Jordán, R, Aledort, LM, Santagostino, E, Primary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrate. Haemophilia 20 (2014), 83–91.
-
(2014)
Haemophilia
, vol.20
, pp. 83-91
-
-
Oldenburg, J.1
Jiménez-Yuste, V.2
Peiró-Jordán, R.3
Aledort, L.M.4
Santagostino, E.5
-
124
-
-
84958833925
-
First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study
-
published online July 23.
-
124 Kreuz, W, Escuriola Ettingshausen, C, Vdovin, V, et al. First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study. Haemophilia, 2015, 10.1111/hae.12774 published online July 23.
-
(2015)
Haemophilia
-
-
Kreuz, W.1
Escuriola Ettingshausen, C.2
Vdovin, V.3
-
125
-
-
84907270053
-
Rituximab for treatment of inhibitors in haemophilia A: a phase II study
-
125 Leissinger, C, Josephson, CD, Granger, S, et al. Rituximab for treatment of inhibitors in haemophilia A: a phase II study. Thromb Hemost 112 (2014), 445–458.
-
(2014)
Thromb Hemost
, vol.112
, pp. 445-458
-
-
Leissinger, C.1
Josephson, C.D.2
Granger, S.3
-
126
-
-
84900468578
-
Inhibitor eradication with rituximab in haemophilia: where do we stand?
-
126 Franchini, M, Mannucci, PM, Inhibitor eradication with rituximab in haemophilia: where do we stand?. Br J Haematol 165 (2014), 600–608.
-
(2014)
Br J Haematol
, vol.165
, pp. 600-608
-
-
Franchini, M.1
Mannucci, P.M.2
-
127
-
-
0019426632
-
The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII: a double-blind clinical trial
-
127 Sjamsoedin, LJ, Heijnen, L, Mauser-Bunschoten, EP, et al. The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII: a double-blind clinical trial. N Engl J Med 205 (1981), 717–721.
-
(1981)
N Engl J Med
, vol.205
, pp. 717-721
-
-
Sjamsoedin, L.J.1
Heijnen, L.2
Mauser-Bunschoten, E.P.3
-
128
-
-
17144437423
-
Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (Novoseven) in haemophiliacs with inhibitors
-
128 Key, NS, Aledort, LM, Beardsley, D, et al. Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (Novoseven) in haemophiliacs with inhibitors. Thromb Haemost 80 (1998), 912–918.
-
(1998)
Thromb Haemost
, vol.80
, pp. 912-918
-
-
Key, N.S.1
Aledort, L.M.2
Beardsley, D.3
-
129
-
-
33846185403
-
A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study
-
129 Astermark, J, Donfield, SM, DiMichele, DM, et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study. Blood 109 (2007), 546–551.
-
(2007)
Blood
, vol.109
, pp. 546-551
-
-
Astermark, J.1
Donfield, S.M.2
DiMichele, D.M.3
-
130
-
-
40349085780
-
−1-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison
-
−1-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison. Haemophilia 14 (2008), 287–294.
-
(2008)
Haemophilia
, vol.14
, pp. 287-294
-
-
Young, G.1
Shafer, F.E.2
Rojas, P.3
Seremetis, S.4
-
131
-
-
0030837755
-
Multicenter retrospective study on the utilization of FEIBA in France in patients with factor VIII and factor IX inhibitors
-
131 Negrier, C, Goudemand, J, Sultan, Y, Bertrand, M, Rothschild, C, Lauroua, P, the members of the French FEIBA study group. Multicenter retrospective study on the utilization of FEIBA in France in patients with factor VIII and factor IX inhibitors. Thromb Haemost 77 (1997), 1113–1119.
-
(1997)
Thromb Haemost
, vol.77
, pp. 1113-1119
-
-
Negrier, C.1
Goudemand, J.2
Sultan, Y.3
Bertrand, M.4
Rothschild, C.5
Lauroua, P.6
-
132
-
-
0018352662
-
Effect of treatment with activated prothrombin complex concentrate (FEIBA) on factor VIII-antibody level
-
132 Lechner, K, Nowotny, C, Krinninger, B, Zegner, M, Deutsch, E, Effect of treatment with activated prothrombin complex concentrate (FEIBA) on factor VIII-antibody level. Thromb Haemost 40 (1979), 478–485.
-
(1979)
Thromb Haemost
, vol.40
, pp. 478-485
-
-
Lechner, K.1
Nowotny, C.2
Krinninger, B.3
Zegner, M.4
Deutsch, E.5
-
133
-
-
84993661361
-
Factor VIII inhibitors in hemophilia A: rationale and latest evidence
-
133 Witmer, C, Young, G, Factor VIII inhibitors in hemophilia A: rationale and latest evidence. Ther Adv Hematol 4 (2013), 59–72.
-
(2013)
Ther Adv Hematol
, vol.4
, pp. 59-72
-
-
Witmer, C.1
Young, G.2
-
134
-
-
84885028310
-
FEIBA versus NovoSeven in hemophilia patients with inhibitors
-
134 Franchini, M, Coppola, A, Tagliaferri, A, Lippi, G, FEIBA versus NovoSeven in hemophilia patients with inhibitors. Semin Thromb Hemost 39 (2013), 772–778.
-
(2013)
Semin Thromb Hemost
, vol.39
, pp. 772-778
-
-
Franchini, M.1
Coppola, A.2
Tagliaferri, A.3
Lippi, G.4
-
135
-
-
34248551364
-
Sequential therapy with activated prothrombin complex concentrate and recombinant factor VIIa in patients with severe haemophilia and inhibitors: update of our previous experience
-
135 Schneiderman, J, Rubin, E, Nugent, DJ, Young, G, Sequential therapy with activated prothrombin complex concentrate and recombinant factor VIIa in patients with severe haemophilia and inhibitors: update of our previous experience. Haemophilia 13 (2007), 244–248.
-
(2007)
Haemophilia
, vol.13
, pp. 244-248
-
-
Schneiderman, J.1
Rubin, E.2
Nugent, D.J.3
Young, G.4
-
136
-
-
67649868161
-
Concomitant infusions of low doses of rFVIIa and FEIBA in haemophilia patients with inhibitors
-
136 Martinowitz, U, Livnat, T, Zivelin, A, Kenet, G, Concomitant infusions of low doses of rFVIIa and FEIBA in haemophilia patients with inhibitors. Haemophilia 15 (2009), 904–910.
-
(2009)
Haemophilia
, vol.15
, pp. 904-910
-
-
Martinowitz, U.1
Livnat, T.2
Zivelin, A.3
Kenet, G.4
-
137
-
-
80053570793
-
Parallel use of bypassing agents in haemophilia with inhibitors: a critical review
-
137 Ingerslev, J, Sorensen, B, Parallel use of bypassing agents in haemophilia with inhibitors: a critical review. Br J Haematol 155 (2011), 256–262.
-
(2011)
Br J Haematol
, vol.155
, pp. 256-262
-
-
Ingerslev, J.1
Sorensen, B.2
-
138
-
-
34548096392
-
Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors
-
138 Konkle, BA, Ebbesen, LS, Erhardtsen, E, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 5 (2007), 1904–1913.
-
(2007)
J Thromb Haemost
, vol.5
, pp. 1904-1913
-
-
Konkle, B.A.1
Ebbesen, L.S.2
Erhardtsen, E.3
-
139
-
-
80455144639
-
Anti-inhibitor coagulant complex prophylaxis in hemophilia patients with inhibitors
-
139 Leissinger, C, Gringeri, A, Antmen, B, et al. Anti-inhibitor coagulant complex prophylaxis in hemophilia patients with inhibitors. N Engl J Med 365 (2011), 1684–1692.
-
(2011)
N Engl J Med
, vol.365
, pp. 1684-1692
-
-
Leissinger, C.1
Gringeri, A.2
Antmen, B.3
-
140
-
-
84890855644
-
Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A patients with inhibitors
-
140 Antunes, SV, Tangada, S, Stasyshyn, O, et al. Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A patients with inhibitors. Haemophilia 20 (2014), 65–72.
-
(2014)
Haemophilia
, vol.20
, pp. 65-72
-
-
Antunes, S.V.1
Tangada, S.2
Stasyshyn, O.3
-
141
-
-
84928829654
-
Prophylaxis with FEIBA in paediatric patients with haemophilia A and inhibitors
-
141 Ewing, N, Escuriola-Ettingshausen, C, Kreuz, W, Prophylaxis with FEIBA in paediatric patients with haemophilia A and inhibitors. Haemophilia 21 (2015), 358–364.
-
(2015)
Haemophilia
, vol.21
, pp. 358-364
-
-
Ewing, N.1
Escuriola-Ettingshausen, C.2
Kreuz, W.3
|