. Author manuscript; available in PMC: 2023 Jan 28.

Published in final edited form as: Haemophilia. 2019 Jul;25(4):581–589. doi: 10.1111/hae.13717

TABLE 4.

Inhibitor trials able to be supported within HTCs

HA PUP Inhibitor Prevention Trial
PUPs (HA): Estimate approximately 257/y
HTCs: N = 141
Newborn severe HA PUPs: 1.82/y/centre¹⁰
Newborn severe HA PUPs in 141 HTC: N = 257
Treatment with FVIII: Median age 1st bleed, start FVIII =7 mo
New inhibitors in severe HA PUPs: 30% (N = 77)¹
Proportion joining clinical trial: 25% (N = 64)
Proportion not joining clinical trial: 75% (N = 193)
For past factor; bleed; refusal; travel/time barriers; competing trials²⁴
HA-I Inhibitor Eradication Trial
Refractory Inhibitor (HA-I): Estimate approximately 1000 total
Estimated prevalence HA-I (12% severe HA)²⁵
Likely to enroll, as poor response to Rx, and ineligible for gene therapy
ESTIMATE: trials that could be supported in HTCs
2-3 HA PUP inhibitor prevention trials
2-3 HA-I PUP inhibitor eradication trials

HA, haemophilia A; HA-I, haemophilia A with inhibitor; HTC, haemophilia treatment centre; PUP, previously untreated patient.