Haemophilia clinical outcomes

The Great Ormond Street Hospital (GOSH) Haemophilia Comprehensive Care Centre treats children and young people with inherited bleeding disorders and some thrombotic conditions. 

The centre is the largest paediatric centre in Europe and acts as the hub of the North London Paediatric Haemophilia Network. The centre manages the care of over 650 patients with a variety of inherited bleeding disorders of different severities including:

• Haemophilia A
• Haemophilia B
• Von Willebrands disease
• Factor X Deficiency
• Factor XI Deficiency
• Glanzmann's thrombasthenia

The complex nature of these lifelong conditions requires specialist care in a multi-disciplinary setting with input from consultants, clinical nurse specialists and physiotherapists. As one of the leading paediatric haemophilia centres in the world, GOSH specialises in the management of complex cases. These include patients with inhibitors or those requiring surgery.

Some patients with inherited bleeding disorders require regular infusions of factor products, known as prophylaxis, in order to prevent spontaneous bleeding. These infusions can be up to once a day.

Numerator: Number of severe Haemophilia A or B patients on active treatment prescribed a prophylactic regimen.

Denominator: Number of severe Haemophilia A or B patients on active treatment.

Prophylaxis is not necessary within the first months of life in most cases and the discrepancy between the total number of patients and those on prophylaxis reflects young babies not yet started on regular treatment.

Table 1 Number of severe Haemophilia A or B patients prescribed a prophylactic regimen, 2021/22

In order to monitor patients’ care and provide access to specialist treatment such as physiotherapy, patients are formally reviewed at the Centre every three to 12 months depending on age and severity. This process allows Centre staff to adjust treatment doses and provide advice on any clinical problems that may have arisen. The review rates seen below show the proportion of severe Haemophilia A or B patients registered at the Centre, with at least two documented clinical reviews in each 12-month review period.

Numerator: Number of patients with severe Haemophilia A or B registered at the Centre reviewed at least twice within each annual review cycle.

Denominator: Number of patients with severe Haemophilia A or B registered at the Centre.

Table 2 Number of severe patients seen for a review

Preventing breakthrough bleeds is important in maintaining healthy joints. A breakthrough bleed indicates that prophylaxis is not working optimally and may need to be adjusted. To help monitor treatment self-administration and bleeds, patients regularly report these via digital interfaces (such as Haemtrack™).

Numerator: Number of patients providing data relating to the self-administration of blood factor products, and data relating to the incidence of bleeding episodes and other related events via digital interfaces.

Denominator: Number of patients with severe Haemophilia A or B on regular prophylaxis.

Table 3 Number of severe patients reporting treatment and outcomes

The Haemophilia Joint Health Score (HJHS) is a validated measure of joint impairment. It provides information on joint health status over time, and therefore effectiveness of treatment in avoiding joint bleeds, which damage joints. All patients were offered HJHS assessments but some families preferred to defer this during the pandemic.

Numerator: Number of patients with severe or moderate Haemophilia A or B with a joint score recorded at the most recent annual review within the annual review cycle.

Denominator: Number of patients with severe or moderate Haemophilia A or B registered at the centre.

Table 4 Number of severe or moderate patients with a recorded joint score

This information was updated in November 2022.