A Study of Previously Untreated Patients (PUPs) with Congenital Hemophilia


About this study

The purpose of this study is to determine the percentage of subjects with confirmed inhibitors (per CDC laboratory criteria; >0.5 Nijmegen Bethesda Units for hemophilia A and >0.3 Nijmegen Bethesda Units for hemophilia B within the first 50 Exposure Days (ED).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Congenital hemophilia A; FVIII ≤ 5% or congenital hemophilia B; FIX ≤ 5%.
  • Birth date on or after January 1, 2010.
  • Care established at one of the participating HTCs.
  • Co-enrollment in the ATHNdataset.
  • Parent or authorized guardian can provide informed consent.

Exclusion Criteria:

  • Patients who are referred to the HTC with no record of bleed and factor utilization data.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Rajiv Pruthi, M.B.B.S.

Closed for enrollment

More information


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