Sobi Med - AltuvOct - Unmet Need

Addressing unmet needs in children and teenagers with haemophilia A

Early initiation of factor replacement therapy prophylaxis is critical to prevent joint disease¹

The World Federation of Hemophilia (WFH) recommends starting prophylaxis before joint disease begins, ideally before the age of 3.¹

This proactive approach may:

We need to be proactive about children’s bleed protection as this may support their future joint health

Joint damage may develop if early prophylaxis is suboptimal⁵

Today’s treatment decisions affect tomorrow’s outcomes⁵

In the real-world setting, prophylaxis fails to preserve joint health and paediatric patients could develop joint damage despite early prophylaxis with today’s treatments.⁵

In fact, a 12-month observational study conducted in 2018 revealed that 30% of children with severe haemophilia A (n=80) experienced haemarthrosis in their knees.⁶

Haemarthrosis is a disabling joint haemorrhage that may limit range of motion¹

These findings underscore the need for continuous improvement in treatment strategies to protect children’s joint health.⁶ By understanding and addressing these gaps, we can work towards a future where children with haemophilia can enjoy a better QOL.

It is important to further understand how treatment strategies may impact the joint health of children with haemophilia A

Children are affected by impaired joint health despite reporting no bleeding⁷

An observational study of paediatric patients with severe haemophilia (N=106 patients, 636 joints) evaluated using HEAD-US criteria showed that:⁷^*

HEAD-US evaluates joint health using ultrasound⁷

HEAD-US (Early Detection of Haemophilia Arthropathy with Ultrasound) is used for the evaluation of synovitis, cartilage and subchondral bone. It allows multiple joints to be evaluated during each study, without the need for sedation vs MRI.⁷

HEAD-US scores are determined by joint involvement, with a maximum score of eight points per joint. Higher scores indicate greater joint involvement.⁷

By addressing subclinical bleeds, we may improve joint health and QOL for people with haemophilia

Median age was 12 years. Mean age of starting prophylaxis was 2.7 years. 44.3% and 55.7% of patients received primary and secondary prophylaxis, respectively. Joint damage was defined by a HEAD-US score ≥1.⁷

Haemophilia impacts children’s lives⁸

For children and adolescents living with bleeding disorders, bleeds and treatments can affect their daily lives⁸

Several studies have revealed how young patients—and their caregivers—struggle with barriers to everyday life. Their QOL can be significantly impacted as they navigate physical limitations, persistent pain, and emotional challenges. The fear of bleeds can affect not only their bodies but also their emotions and behaviours, limiting their ability to fully live their childhood.^8-11

Haemophilia impacts QOL:^8-11

It is important to understand how bleeds and treatments may affect the everyday life of children with haemophilia A

Elevation of protection against bleeds may require FVIII levels in the non-haemophilia range²

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).² Recent studies show that sustained FVIII levels >40 IU/dL may provide freedom from bleeds.^2,4

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

FVIII, factor VIII; HEAD-US, Haemophilia Early Arthropathy Detection with Ultrasound; MRI, magnetic resonance imaging; QOL, quality of life; WFH, World Federation of Hemophilia.

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. Srivastava A, et al. Haemophilia 2020;26:1–1581.
2. Chowdary P, et al. Thromb Haemost 2020;120:728–736.
3. Skinner M, et al. Haemophilia 2020;26:17–24.
4. Mahlangu J et al. N Engl J Med 2023;388:694–705.
5. Oldenburg J. Blood 2015;125:2038–2044.
6. Wilkins RA, et al. BMJ Open 2022;12; DOI:10.1136/bmjopen-2021-052358.
7. Daffunchio C, et al. Thromb Res 2023;226:86–92.
8. Azeredo-da-Silva A, et al. Res Pract Thromb Haemost 2022;7:100008.
9. Rodriguez‑Santana I, et al. Orphanet J Rare Dis 2022;17:148.
10. Steen Carlson K, et al. Haemophilia 2022;28:557–567.
11. Elsakka E, et al. Int J Rheum Dis 2022;21:803–810.

Understanding the Unmet Needs in Haemophilia A for Adults & Children

Addressing unmet needs in children and teenagers with haemophilia A

Joint damage may develop if early prophylaxis is suboptimal5

Children are affected by impaired joint health despite reporting no bleeding7

Haemophilia impacts children’s lives8

Elevation of protection against bleeds may require FVIII levels in the non-haemophilia range2

Joint damage may develop if early prophylaxis is suboptimal⁵

Children are affected by impaired joint health despite reporting no bleeding⁷

Haemophilia impacts children’s lives⁸

Elevation of protection against bleeds may require FVIII levels in the non-haemophilia range²