Lecture 5: Hemophilia and Hereditary Factor Deficiencies

Catherine McGuinn MD

Presentation Date:

October 1, 2026

About This Lecture

Hemophilia, while not the most common bleeding disorder, is widely known among the public.
It is also known as the "Royal Disease" due to its presence in the European royal families of the late 19th and early 20th Century.
The earliest records of hemophilia date to the Talmud, a Jewish religious text from over 2000 years ago.
New generations of treatment, now including gene therapy have made this a very exciting time for patients and their caregivers.

Slide Deck

Continuing Education Credit

Catherine McGuinn, MD

Weill Cornell Medicine
Pediatric Hematology-Oncology
cam9061@med.cornell.edu

Speaker Disclosures

Research support: (Last 24 Months)
Biogen/Sanofi, Roche/Genentech, Spark, Pfizer, Takeda/Shire

Medical Advisory Board (Last 24 months)
Genentech, CSL, Octapharma

I will be discussing off-label use of medications

Learning Objectives

> Accurately recognize the inheritance pattern, clinical presentation and laboratory evaluation for Hemophilia
> Understand the risks and benefits of clotting factor administration for the treatment
> Describe 3 approaches to improve the prevention of bleeding events in patients with Hemophilia