Sobi Med - AltuvOct - Unmet Need

Prophylaxis is not sufficient to prevent joint bleeds in haemophilia¹

Despite significant advances in treatment, there remain opportunities to improve the lives of those living with haemophilia¹

For people with haemophilia challenges still exist: regular prophylaxis, while beneficial, does not prevent all bleeds.¹
In fact, a 12-month observational study conducted in 2018 revealed that 60% of adults with severe haemophilia A (n=157) experienced haemarthrosis in their knees.¹

Haemarthrosis is a disabling joint haemorrhage that may limit range of motion²

How could we ensure bleed protection and joint health for people with haemophilia A?

Joint bleeds persist³

Are your patients still at risk of bleeds?

A real-world study of patients with severe haemophilia, who received regular FVIII prophylaxis, showed the majority still experienced bleeds (n=139).³

Even with regular FVIII prophylaxis, many patients continue to experience bleeds and suboptimal bleed protection. These bleeds can significantly impact their daily lives, limiting physical activity and increasing the risk of long-term joint damage.³

Providing patients with more comprehensive bleed protection may help improve their QOL, allowing them to engage in everyday activities with greater confidence and ease.³

It’s time to explore the relationship between bleed protection and joint damage

Subclinical bleeds occur despite prophylaxis^4-6

Did you know:^4,5

Even when prophylactic regimens appear to prevent joint bleeds and patients don’t report symptoms, objective measures reveal the presence of subclinical bleeds.^4,5

How can we better understand the relationship between subclinical bleeds and joint bleeds?

Subclinical bleeds can cause long-term joint damage⁷

Despite prophylaxis, joint damage in the ankles and knees can develop over time, and is typically diagnosed after a median of 10 years. This progression is shown on the illustration below, using Pettersson and Gilbert scores.⁷

Using imaging techniques (like ultrasound) and scoring systems like Pettersson and Gilbert, healthcare providers can assess joint health even when there’s no pain. On average, the clinical Gilbert scores* become pathologic 1 to 2 decades later than the Pettersson scores*, highlighting the need for early detection through imaging.⁷

Real Stories, Real Impact

In a survey of 950 people with haemophilia, more than one-third said they commonly experience swelling in the joints and nearly half said they experience acute pain. These personal experiences highlight the need for effective monitoring and treatment.⁸

Early detection and monitoring are important to decrease the development of arthropathy⁷

The Gilbert score is a clinical scoring system used to assess the severity of haemophilic arthropathy. Scores ≥2 are regarded to be pathological.⁷
The Gilbert scoring system evaluates the condition of the joints by examining factors such as the range of motion, swelling, muscle atrophy, axial deformity, and pain. Each of these factors is given a score, and the total score reflects the overall severity of the joint disease.⁹ The system is designed to be simpler and quicker to use than radiological scoring systems, making it a practical tool for routine clinical assessments.^9,10
Unlike the Pettersson score, which is a detailed radiological scoring system,¹¹ the Gilbert score is based on physical examination and clinical features rather than radiographic findings.⁹
The Gilbert Score (also called World Federation of Hemophilia Physical Examination Score) is a detailed radiologic classification of haemophilic joints. It estimates joint destruction radiologically on 8 parameters scored. However, plain x-ray is too insensitive to diagnose early joint pathology, making it inappropriate to diagnose early joint damage. Ankles, as the first affected joints for the majority of patients, offer an interesting opportunity for diagnosing early joint disease and subsequently adapting clinical decisions.^9,10

Haemophilia continues to impact patients’ lives^8,12

In 2023, a global survey with 950 patients was conducted to explore the lives of individuals living with haemophilia⁸

Even with today’s haemophilia A treatments, patients are still struggling in their everyday lives. About half of the patients surveyed not only worried about sudden bleeds, but also felt guilty about how the disease could negatively affect their relationships. A further 41% revealed that fears about sudden bleeds kept them from participating in activities that were once part of their lives.⁸

Prophylaxis should be tailored to the lifestyle, ambitions and clinical status of each patient⁸

Elevation of protection against bleeds may require FVIII levels in the non-haemophilia range¹³

Adapted from Chowdary P. et al 2020, Mahlangu J et al. 2023 and Srivastava A, et al. 2020

As the severity of patients’ haemophilia is determined by their FVIII level, non-haemophilia range refers to FVIII levels above those associated with haemophilia (0–40 IU/dL).¹³ Recent studies show that sustained FVIII levels >40 IU/dL may provide freedom from bleeds.^13,14

Would your patients benefit from FVIII levels in the non-haemophilia range?

ABR, annualised bleed rate; FVIII, factor VIII; GS, Gilbert score; MRI, magnetic resonance imaging; PS, Pettersson score.

This content is focused on treatment diagnosis, disease management and its challenges. There is no intent to connect it with ALTUVOCT^® data and its potential use.

1. Wilkins RA, et al. BMJ Open 2022;12; DOI:10.1136/bmjopen-2021-052358.
2. Srivastava A, et al. Haemophilia 2020;26:1–158.
3. Chowdary P, et al. ISTH 2023. Montréal, Canada. Poster PB1249.
4. Di Minno D, et al. Haemophilia 2013;19(3):167–173.
5. De la Corte-Rodriguez, et al. Haemophilia 2022;28(1):138–144.
6. Manco-Johnson MJ, et al. N Engl J Med 2007;375(6):535–544.
7. Oldenburg J. Blood 2015;125(13):2038–2044.
8. Sanofi. Haemophilia Life Stages And Changes Global Fact Sheet. 2023. Available at: https://www.sanofi.com/assets/dotcom/content-app/articles/your-health/global-hemophilia-survey-captures-the-voice-of-patients-caregivers-and-providers/sanofi-1.pdf. Accessed November 2024.
9. World Federation of Hemophilia (WFH). The classification recommended by the orthopedic advisory committee of the World Federation of Hemophilia. Available at: https://wfh.org/wp-content/uploads/2021/12/WBDR-Gilbert-Score.pdf. Accessed November 2024.
10. World Federation of Hemophilia (WFH). WFH Physical Examination Score (aka Gilbert score). Available at: https://elearning.wfh.org/resource/wfh-physical-examination-score-aka-gilbert-score/. Accessed November 2024.
11. Foppen W et al. Eur Radiol 2016;26:1963–1970.
12. Malec L and Matino D. Haemophilia 2023;29(6):1419–1429.
13. Chowdary P, et al. Thromb Haemost 2020;120(5):728–736.
14. Mahlangu J et al. N Engl J Med 2023;388:694–705.

Understanding the Unmet Needs in Haemophilia A for Adults & Children

Prophylaxis is not sufficient to prevent joint bleeds in haemophilia1

Joint bleeds persist3

Subclinical bleeds occur despite prophylaxis4-6

Subclinical bleeds can cause long-term joint damage7

Haemophilia continues to impact patients’ lives8,12

Elevation of protection against bleeds may require FVIII levels in the non-haemophilia range13

Prophylaxis is not sufficient to prevent joint bleeds in haemophilia¹

Joint bleeds persist³

Subclinical bleeds occur despite prophylaxis^4-6

Subclinical bleeds can cause long-term joint damage⁷

Haemophilia continues to impact patients’ lives^8,12

Elevation of protection against bleeds may require FVIII levels in the non-haemophilia range¹³