Table 2

Patient characteristics according to the first recombinant VIII product received

CharacteristicsFirst recombinant FVIII product received
Product E (n = 97)Product D (n = 111)Product A (n = 48)Product C (n = 27)All rFVIII products* (n = 303)
No.%MIQRNo.%MIQRNo.%MIQRNo.%MIQRNo.%MIQR
Fixed risk factors                     
 F8 gene defect                     
  Low risk 30 30.9   19 17.1   11 22.9   6 22.2   68 22.4   
  High risk † 62 63.9   85 76.6   35 72.9   21 77.8   214 70.6   
  Undetermined (eg, untested, unidentified) 5 5.2   7 6.3   2 4.2   0 0.0   21 6.9   
 Family history                     
  Hemophilia without inhibitor 45 46.4   37 33.3   13 27.1   9 33.3   110 36.3   
  Hemophilia with inhibitor 9 9.3   15 13.5   6 12.5   3 11.1   36 11.9   
  No family history of hemophilia 43 44.3   59 53.2   29 60.4   15 55.6   157 51.8   
 Ethnic origin‡                     
  White only (both parents) 74 76.3   85 76.6   37 77.1   20 74.1   233 76.9   
  Others not African or African-American 17 17.5   23 20.7   7 14.6   6 22.2   55 18.2   
  African or African-American (at least one grandparent) 6 6.2   3 2.7   4 8.3   1 3.7   15 5.0   
 Calendar period of first exposure to rFVIII                     
  Before 2000 0 0.0   19 17.1   35 72.9   19 70.4   83 27.4   
  2000-2003 24 24.7   31 27.9   12 25.0   7 25.9   74 24.4   
  2004-2007 35 36.1   31 27.9   1 2.1   1 3.7   69 22.8   
  2008 and after 38 39.2   30 27.0   0 0.0   0 0.0   77 25.4   
 Age at first exposure to rFVIII, months   9.5 3.6-13.3   11.6 7.4-14.5   10.1 5.3-16.8   10.6 4.7-16.7   10.4 5.6-14.3 
  <6 30 30.9   23 20.7   14 29.2   7 25.9   78 25.7   
  6-11 35 36.1   35 31.5   15 31.3   8 29.6   102 33.7   
  ≥12 32 33.0   53 47.7   19 39.6   12 44.4   123 40.6   
Time-varying risk factors                     
 Initiation of regular prophylaxis within first 50 EDs§ 56 57.7   49 44.1   19 39.6   9 33.3   145 47.9   
 Cumulative EDs at start of prophylaxis   15 7-26   20 10-33   22 16-47   30 20-54   20 11-33 
 Peak treatment episode at first exposure, consecutive EDs                     
  ≥3 31 32.0   35 31.5   12 25.0   5 18.5   91 30.0   
  ≥5 14 14.4   21 18.9   8 16.7   3 11.1   50 16.5   
  ≥10 8 8.2   11 9.9   2 4.2   1 3.7   23 7.6   
 History of peak treatment episodes (≥1 during follow-up), consecutive EDs                     
  ≥3 63 64.9   75 67.6   37 77.1   21 77.8   210 69.3   
  ≥5 35 36.1   44 39.6   22 45.8   12 44.4   121 39.9   
  ≥10 14 14.4   17 15.3   5 10.4   4 14.8   41 13.5   
 First exposure linked to surgical procedure (with ≥3 EDs) 2 2.1   1 0.9   0 0.0   0 0.0   5 1.7   
 History of surgical procedures (with ≥3 EDs) during follow-up 10 10.3   8 7.2   10 20.8   5 18.5   38 12.5   
 First exposure linked to severe bleeding episode 10 10.3   10 9.0   2 4.2   0 0.0   22 7.3   
 History of severe bleeding episodes during follow-up 13 13.4   17 15.3   3 6.3   2 7.4   35 11.6   
Outcomes                     
 All inhibitors 33 34.0   55 49.5   13 27.1   7 25.9   114 37.6   
 High-titer inhibitors 20 20.6   28 25.2   7 14.6   5 18.5   63 20.8   
 Inhibitors occurring during the first 75 EDs and treated at any time during FranceCoag follow-up                     
   Cases treated with bypassing agents 24 24.7   38 34.2   6 12.5   6 22.2   77 25.4   
   Cases treated with ITI 23 23.7   40 36.0   7 14.6   5 18.5   79 26.1   
   Cases treated with bypassing agents and/or ITI 29 29.9   47 42.3   8 16.7   7 25.9   95 31.4   
CharacteristicsFirst recombinant FVIII product received
Product E (n = 97)Product D (n = 111)Product A (n = 48)Product C (n = 27)All rFVIII products* (n = 303)
No.%MIQRNo.%MIQRNo.%MIQRNo.%MIQRNo.%MIQR
Fixed risk factors                     
 F8 gene defect                     
  Low risk 30 30.9   19 17.1   11 22.9   6 22.2   68 22.4   
  High risk † 62 63.9   85 76.6   35 72.9   21 77.8   214 70.6   
  Undetermined (eg, untested, unidentified) 5 5.2   7 6.3   2 4.2   0 0.0   21 6.9   
 Family history                     
  Hemophilia without inhibitor 45 46.4   37 33.3   13 27.1   9 33.3   110 36.3   
  Hemophilia with inhibitor 9 9.3   15 13.5   6 12.5   3 11.1   36 11.9   
  No family history of hemophilia 43 44.3   59 53.2   29 60.4   15 55.6   157 51.8   
 Ethnic origin‡                     
  White only (both parents) 74 76.3   85 76.6   37 77.1   20 74.1   233 76.9   
  Others not African or African-American 17 17.5   23 20.7   7 14.6   6 22.2   55 18.2   
  African or African-American (at least one grandparent) 6 6.2   3 2.7   4 8.3   1 3.7   15 5.0   
 Calendar period of first exposure to rFVIII                     
  Before 2000 0 0.0   19 17.1   35 72.9   19 70.4   83 27.4   
  2000-2003 24 24.7   31 27.9   12 25.0   7 25.9   74 24.4   
  2004-2007 35 36.1   31 27.9   1 2.1   1 3.7   69 22.8   
  2008 and after 38 39.2   30 27.0   0 0.0   0 0.0   77 25.4   
 Age at first exposure to rFVIII, months   9.5 3.6-13.3   11.6 7.4-14.5   10.1 5.3-16.8   10.6 4.7-16.7   10.4 5.6-14.3 
  <6 30 30.9   23 20.7   14 29.2   7 25.9   78 25.7   
  6-11 35 36.1   35 31.5   15 31.3   8 29.6   102 33.7   
  ≥12 32 33.0   53 47.7   19 39.6   12 44.4   123 40.6   
Time-varying risk factors                     
 Initiation of regular prophylaxis within first 50 EDs§ 56 57.7   49 44.1   19 39.6   9 33.3   145 47.9   
 Cumulative EDs at start of prophylaxis   15 7-26   20 10-33   22 16-47   30 20-54   20 11-33 
 Peak treatment episode at first exposure, consecutive EDs                     
  ≥3 31 32.0   35 31.5   12 25.0   5 18.5   91 30.0   
  ≥5 14 14.4   21 18.9   8 16.7   3 11.1   50 16.5   
  ≥10 8 8.2   11 9.9   2 4.2   1 3.7   23 7.6   
 History of peak treatment episodes (≥1 during follow-up), consecutive EDs                     
  ≥3 63 64.9   75 67.6   37 77.1   21 77.8   210 69.3   
  ≥5 35 36.1   44 39.6   22 45.8   12 44.4   121 39.9   
  ≥10 14 14.4   17 15.3   5 10.4   4 14.8   41 13.5   
 First exposure linked to surgical procedure (with ≥3 EDs) 2 2.1   1 0.9   0 0.0   0 0.0   5 1.7   
 History of surgical procedures (with ≥3 EDs) during follow-up 10 10.3   8 7.2   10 20.8   5 18.5   38 12.5   
 First exposure linked to severe bleeding episode 10 10.3   10 9.0   2 4.2   0 0.0   22 7.3   
 History of severe bleeding episodes during follow-up 13 13.4   17 15.3   3 6.3   2 7.4   35 11.6   
Outcomes                     
 All inhibitors 33 34.0   55 49.5   13 27.1   7 25.9   114 37.6   
 High-titer inhibitors 20 20.6   28 25.2   7 14.6   5 18.5   63 20.8   
 Inhibitors occurring during the first 75 EDs and treated at any time during FranceCoag follow-up                     
   Cases treated with bypassing agents 24 24.7   38 34.2   6 12.5   6 22.2   77 25.4   
   Cases treated with ITI 23 23.7   40 36.0   7 14.6   5 18.5   79 26.1   
   Cases treated with bypassing agents and/or ITI 29 29.9   47 42.3   8 16.7   7 25.9   95 31.4   

M, median.

*

Product B (n = 10) and Product F (n = 10) are included in all rFVIII products.

†

High-risk gene defects include large deletions (≥1 exon), intron 1 and 22 inversions, small deletions/insertions with stop codon (out of A-run), and nonsense mutations.

‡

Up to four ethnic origins per patient could be recorded (one for each grandparent).

§

The initiation of regular prophylaxis was defined as the moment at which at least 3 consecutive prophylactic infusions of rFVIII were given within a period of at least 15 days (RODIN definition).14 

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