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Hemophilia A Case Study: Sean


39 year old male

Switched to ALTUVIIIO: August 2024

Sean, who had severe hemophilia A, presented for a left total knee arthroplasty (TKA) secondary to joint damage from chronic hemarthroses.

Patient Information

  • Diagnosed at age 4 but had intermittent access to factor therapy for many years, leading to multiple joint bleeds
  • Used factor on-demand until 2017 due to limited treatment access when growing up outside of the US
  • Was enrolled in a gene therapy trial in 2019
  • Currently treats with factor-mimetic prophylaxis and on-demand EHL therapy
  • Tried EHL prophylaxis prior to current regimen

  • Type 2 diabetes
  • Hypertension
  • Hyperlipidemia
  • Obesity
  • Beta thalassemia minor
  • Surgeries
    • Right TKA (2023)
    • Right knee radiofrequency ablation (2020, 2021)
    • Right knee steroid injection (2020)

  • First in family to be diagnosed with hemophilia A

  • BP: 148/95
  • BMI: 42.3
  • HR: 80
  • Significant joint deformities in both knees; left knee swollen, warm to touch, and decreased capacity for flexion; and altered gait

  • Baseline (diagnosis at age 4) factor level <1%
  • 2017: Inhibitor titer negative
  • January 2024: Chromogenic level 6.7%

Sean’s Path to High Sustained Factor Replacement Therapy, ALTUVIIIO

Diagnosis

  • Sean was diagnosed with hemophilia A (FVIII <1%) at age 4 via a bleeding disorder workup after suffering a swollen knee with bloody arthrocentesis

After diagnosis

  • Throughout his life, Sean experienced multiple joint bleeds
  • Had limited treatment access, but obtained several boxes of factor (per patient history, factor type is unclear) that he used for on-demand therapy
  • Over a span of 5 years, Sean tried multiple therapies:
    • 2017-2019: Twice-weekly EHL prophylaxis
    • 2019: Gene therapy trial enrollment
    • Early 2022: EHL prophylaxis
    • Late 2022: Factor-mimetic prophylaxis with on-demand EHL therapy
  • His limited access to prophylactic treatment earlier in life ultimately led to arthropathies, severe joint damage, and bilateral TKAs (2023 right TKA, supported by pre- and postoperative EHL therapy)

Before ALTUVIIIO

  • Sean presented for left TKA
  • Previously treating prophylactically with factor-mimetic therapy and on-demand with EHL therapy

Surgical experience

Acute management

  • 3/6/24: Underwent a left TKA
  • Infused postoperatively on Day 3 (3/9), Day 5 (3/11), and Day 15 (3/21)

Postoperative clinical course

  • 8/19/24: Experienced 2 spontaneous left knee bleeds that were resolved with ALTUVIIIO

After ALTUVIIIO

Switched to ALTUVIIIO: August 2024

  • Compared to his first total knee replacement, Sean experienced lower perioperative infusion burden with his first postoperative dose on Day 3
  • He completed PT 5 days postoperatively and continued 3 times per week for 8 weeks with no functional limitations
  • Five months after surgery, Sean switched from factor-mimetic prophylaxis to once-weekly ALTUVIIIO prophylaxis
  • He currently does PT at home and would like to be more active

Do you have patients like Sean?

If so, it may be worth it to consider the following:

How good is good enough?
What considerations would you take into account for perioperative management for a patient like Sean?

Choosing the right treatment
How would you discuss switching to a treatment option like ALTUVIIIO with patients like Sean?

Unlocking high sustained factor levels
Do you think Sean could benefit from perioperative ALTUVIIIO?

BMI=body mass index; BP=blood pressure; EHL=extended half-life; FVIII=Factor VIII; HR=heart rate; PT=physical therapy; SHL=standard half-life; TKA=total knee arthroplasty.

Other ALTUVIIIO patients who made the switch!

Meet Henry

A 14 year old student and avid baseball player with severe hemophilia A, previously treating with factor-mimetic prophylaxis and an on-demand SHL therapy.

Meet Aaron

A 13 year old student and flag-football player with severe hemophilia A previously on SHL prophylaxis therapy.

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Learn how ALTUVIIIO was studied

The XTEND-1 trial studied ALTUVIIIO in the prophylaxis, on-demand, and perioperative settings in adult and adolescent patients.

What’s next?

If you’re interested in learning more about ALTUVIIIO, why not set up a chat with one of our representatives?

Indication

ALTUVIIIO® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl] is a von Willebrand Factor (VWF) independent recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for:

  • Routine prophylaxis to reduce the frequency of bleeding episodes
  • On-demand treatment & control of bleeding episodes
  • Perioperative management of bleeding
Limitation of Use

ALTUVIIIO is not indicated for the treatment of von Willebrand disease.

Important Safety Information

Contraindications

ALTUVIIIO is contraindicated in patients who have had severe hypersensitivity reactions, including anaphylaxis, to the product or its excipients.

Warnings and Precautions
  • Allergic-type hypersensitivity reactions, including anaphylaxis, may occur with ALTUVIIIO. Allergic-type hypersensitivity reactions were not reported in the clinical trials. Advise patients to discontinue use of ALTUVIIIO if hypersensitivity symptoms occur and contact a physician and/or seek immediate emergency care.
  • Formation of neutralizing antibodies (inhibitors) to Factor VIII are possible following administration of ALTUVIIIO. Neutralizing antibodies were not reported in the clinical trials. Monitor all patients for the development of Factor VIII inhibitors by appropriate clinical observations and laboratory tests.
  • If assessment of plasma Factor VIII activity is needed, it is recommended to use a validated one-stage clotting assay. The ALTUVIIIO Factor VIII activity level is overestimated by the chromogenic assay and a specific ellagic acid-based aPTT reagent in one-stage clotting assay by approximately 2.5-fold. If these assays are used, divide the result by 2.5 to approximate the patient's ALTUVIIIO Factor VIII activity level.
Adverse Reactions

The most common adverse reactions (>10% of subjects) reported in clinical trials were headache and arthralgia.

Indication

Important Safety Information

Reference: 1. ALTUVIIIO Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA.

© 2025 Sanofi. All rights reserved. ALTUVIIIO, MyALTUVIIIO, and Sanofi are registered trademarks of Sanofi or an affiliate. MAT-US-2408466-v1.0-12/2024 Last Updated: December 2024