Clinical Study

Risk Factors for High-Titer Inhibitor Development in Children with Hemophilia A: Results of a Cohort Study

Table 1

Characteristics of patients enrolled in the database.

Parameter of interestTotal 

Years of birth1980–2011
Ethnicity: caucasian (%)100
Factor concentrates used ( )
 pdFVIII177
 rFVIII111
Median (min–max) single dose FVIII (IU/kg/bw)35 (15–100)
Median (min–max) weekly substitution intervals3 (1–3)
Persistent high-titer inhibitor71/288 (24.7%)
 pdFVIII*29/177 (16.38%)
 rFVIII41/111 (36.9%)
  (i) First generation:9/46 (19.5%)
    CHO; full-length; human albumin8/38 (12.5%)
    BHK; full-length; human albumin1/8 (21.0%)
  (ii) Second generation:32/63 (50.7%)
    CHO; B-domain-deleted5/14 (35.7%)
    BHK; full-length; sucrose 27/49 (55.1%)
  (iii) Third generation1/2
    CHO; full-length; trehalose1/2 (50.0%)
Indications for intensified treatment
 Total: number 28
  Neonatal ICH6
  Cephalhematoma6
  Liver rupture1
  Head/spinal trauma4
  Knee or ankle bleed4
  Tongue bleed4
  Appendectomy1
  Meatotomy1
  Nephroblastoma surgery1

BHK: baby hamster kidney; BU: Bethesda units; CHO: Chinese hamster ovary; ICH: intracranial hemorrhage; min–max: minimum–maximum; kg bw: kilogram bodyweight; pd: plasma derived; r: recombinant.
*Beriate P (11/50: 22.0%); Hemophil M (8/39: 20.5%); Humate P (10/43: 23.0%).