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Read case studies from a range of patient types

Read examples based on 5 very different people living with hemophilia A. They are representative of real patient experiences and data using prophylaxis with ADVATE [Antihemophilic Factor (Recombinant)] in a clinical study. Certain details have been changed to protect the identity of the clinical study participants. In the clinical study, 53 previously treated patients with severe or moderately severe hemophilia A FVIII level <=2 IU/dL switched from 6 months of on-demand treatment to 12 months of prophylaxis with ADVATE.1

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Prophylaxis Case Studies

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Case Studies Mentor

PAUL

38 years old | Severe hemophilia A

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Actual father. Actual results.*

Prophylaxis treatment with ADVATE helped this 38-year-old reduce his annual bleed rate (ABR) in a clinical study of prophylaxis treatment with ADVATE.

Shire conducted a clinical study where 53 previously treated patients with severe or moderately severe hemophilia A FVIII level <=2 IU/dL switched from 6 months of on-demand to 12 months of prophylaxis with ADVATE [Antihemophilic Factor (Recombinant)]. There was a 98% reduction in median annual bleed rate (ABR) from 44 to 1 when switched from on-demand to prophylaxis.
See full study results.

ADVATE [Antihemophilic Factor (Recombinant)] had a 98% reduction in ABR in clinical studies.

ON-DEMAND (6 months) ABR 44

At first Paul thought the path to reduce his bleeding episodes was to cut back on activities. Unfortunately, that made little difference.

  • Paul had frequent bleeds, which made it difficult to be his best at work.
  • He missed out on many family activities with his daughters due to bleeds.
  • Target joints: right ankle and left elbow.
  • Paul was ready to participate in the ADVATE study of prophylaxis.
  • During 6 months of on-demand treatment, Paul had 22 joint bleeds.

PROPHYLAXIS (1 year) ABR 0

Paul then received every-third-day prophylaxis with ADVATE to prevent bleeds.

  • Paul had no bleeding episodes of any type by keeping adherent (>90%) to his regimen.
  • Paul is active and devotes time to his young daughters.
  • Paul and his wife go on hiking trips.

*This story represents a real community member's experience and data using prophylaxis with ADVATE in a clinical study. Certain details have been changed to protect the identity of the clinical study participants.

Case Studies Work

GLENN

57 years old | Severe hemophilia A

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Actual businessman. Actual results.*

Prophylaxis treatment with ADVATE helped this 57-year-old reduce his annual bleed rate (ABR) in a clinical study of prophylaxis treatment with ADVATE.

Shire conducted a clinical study where 53 previously treated patients with severe or moderately severe hemophilia A FVIII level <=2 IU/dL switched from 6 months of on-demand to 12 months of prophylaxis with ADVATE [Antihemophilic Factor (Recombinant)]. There was a 98% reduction in median annual bleed rate (ABR) from 44 to 1 when switched from on-demand to prophylaxis.
See full study results.

ADVATE [Antihemophilic Factor (Recombinant)] had a 98% reduction in ABR in clinical studies.

ON-DEMAND (6 months) ABR 76

For Glenn, bleeds seemed just part of life, something to manage and move past.

  • He has been treating his bleeds on-demand for his entire life.
  • Glenn has extensive joint disease along with arthritis.
  • He was having 6 to 7 spontaneous joint bleeds a month.
  • Glenn had never been treated at an HTC.
  • He thought that prophylaxis was only for children.

PROPHYLAXIS (1 year) ABR 1

Glenn then received standard prophylaxis (every other day) with ADVATE to reduce his bleeds and bring ABR down.

  • Glenn found it easy to stay adherent (>90%) to his prophylaxis regimen.
  • 99% reduction in his ABR.
  • He only had 1 bleed the entire year.
  • After the study, Glenn worked with his healthcare professional to try personalized dosing, every-third-day.

*This story represents a real community member's experience and data using prophylaxis with ADVATE in a clinical study. Certain details have been changed to protect the identity of the clinical study participants.

Case Studies Score

AIDEN

11 years old | Severe hemophilia A

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Actual boy. Actual results.*

Prophylaxis treatment with ADVATE helped this 11-year-old reduce his annual bleed rate (ABR) in a clinical study of prophylaxis treatment with ADVATE.

Shire conducted a clinical study where 53 previously treated patients with severe or moderately severe hemophilia A FVIII level <=2 IU/dL switched from 6 months of on-demand to 12 months of prophylaxis with ADVATE [Antihemophilic Factor (Recombinant)]. There was a 98% reduction in median annual bleed rate (ABR) from 44 to 1 when switched from on-demand to prophylaxis.
See full study results.

ADVATE [Antihemophilic Factor (Recombinant)] had a 98% reduction in ABR in clinical studies.

ON-DEMAND (6 months) ABR 40

Aiden was a very active boy when entering the study, and his parents felt they had a handle on managing his bleeds.

  • Aiden was highly active and he enjoys playing soccer.
  • He had no pain associated with bleeds.
  • During the 6 months of on-demand treatment, Aiden had 4 joint bleeds and 16 soft tissue bleeds (all trauma-related).

PROPHYLAXIS (1 year) ABR 5.5

After switching to standard prophylaxis (every other day) with ADVATE, his parents discovered Aiden could continue being himself with fewer bleeds.

  • Aiden and his parents maintained excellent adherence to the prophylactic regimen (>90% adherance).
  • 86% reduction in his ABR.
  • He had only 4 joint bleeds and 1 soft tissue bleed (all trauma-related).
  • Aiden had no pain associated with bleeds and remained active.

*This story represents a real community member's experience and data using prophylaxis with ADVATE in a clinical study. Certain details have been changed to protect the identity of the clinical study participants.

Case Studies ROCK

EMILIO

18 years old | Severe hemophilia A

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Actual teen. Actual results.*

Prophylaxis treatment with ADVATE helped this 18-year-old reduce his annual bleed rate (ABR) in a clinical study of prophylaxis treatment with ADVATE.

Shire conducted a clinical study where 53 previously treated patients with severe or moderately severe hemophilia A FVIII level <=2 IU/dL switched from 6 months of on-demand to 12 months of prophylaxis with ADVATE [Antihemophilic Factor (Recombinant)]. There was a 98% reduction in median annual bleed rate (ABR) from 44 to 1 when switched from on-demand to prophylaxis.
See full study results.

ADVATE [Antihemophilic Factor (Recombinant)] had a 98% reduction in ABR in clinical studies.

ON-DEMAND (6 months) ABR 30

At the start, Emilio and his parents felt prophylaxis treatment wasn't right for them—striving for a goal of zero bleeds seemed too far off.

  • Emilio's participation in sports and other activities was limited by frequent bleeds and reduced range of motion.
  • Target joints: both ankles and elbows, and his right knee.
  • During 6 months of on-demand treatment, Emilio had 15 bleeds into target joints.

PROPHYLAXIS (1 year) ABR 1

Emilio then followed (>90% adherence) every-third-day prophylaxis with ADVATE to prevent bleeds.

  • He had only 1 bleed (left ankle) in a year.
  • 97% reduction in his ABR.
  • Emilio joined extracurricular activities, like his high school glee club.

*This story represents a real community member's experience and data using prophylaxis with ADVATE in a clinical study. Certain details have been changed to protect the identity of the clinical study participants.

Case Studies Play

JAMAL

7 years old | Severe hemophilia A

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Actual child. Actual results.*

Prophylaxis treatment with ADVATE helped this 7-year-old reduce his annual bleed rate (ABR) in a clinical study of prophylaxis treatment with ADVATE.

Shire conducted a clinical study where 53 previously treated patients with severe or moderately severe hemophilia A FVIII level <=2 IU/dL switched from 6 months of on-demand to 12 months of prophylaxis with ADVATE [Antihemophilic Factor (Recombinant)]. There was a 98% reduction in median annual bleed rate (ABR) from 44 to 1 when switched from on-demand to prophylaxis.
See full study results.

ADVATE [Antihemophilic Factor (Recombinant)] had a 98% reduction in ABR in clinical studies.

ON-DEMAND (6 months) ABR 18

Before entering the study, prophylaxis treatment to help manage bleeds seemed too time-consuming for his busy mom.

  • Jamal had frequent joint and soft tissue bleeding episodes, some spontaneous, others related to being an energetic boy.
  • When Jamal fell and bit his tongue, his mom missed 3 days of work caring for him—an attitude-changing event for both.
  • Jamal's mom was now ready to allow her son to participate in the ADVATE study of prophylaxis.
  • During 6 months of on-demand treatment, Jamal had 4 joint and 5 soft tissue bleeds.

PROPHYLAXIS (1 year) ABR 0

Jamal then received every-third-day prophylaxis with ADVATE to prevent bleeds.

  • Jamal's mom was able to follow prophylaxis schedule.
  • Jamal had no bleeding episodes in a year.
  • He enjoyed playing with his brothers.
  • His mom is looking forward to Jamal learning to self-infuse at his next summer camp.

*This story represents a real community member's experience and data using prophylaxis with ADVATE in a clinical study. Certain details have been changed to protect the identity of the clinical study participants.

Watch and share more inspiring stories from hemophilia A patients at different stages of life.

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