Inherited bleeding disorders 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. 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, psychologists 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 children and young people with a severe inherited bleeding disorder prescribed a prophylactic regimen.

Denominator: Number of children and young people with a severe inherited bleeding disorder.

Table 1 Children and young people with a severe inherited bleeding disorder prescribed a prophylactic regimen

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 6 months depending on age. 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 children with a severe inherited bleeding disorder registered at the Centre, with at least two documented clinical reviews in each 12-month review period.

Numerator: Number of children and young people with a severe inherited bleeding disorder registered at the Centre reviewed at least twice within each annual review cycle.

Denominator: Number of children and young people with a severe inherited bleeding disorder registered at the Centre.

Table 2 Children and young people with a severe inherited bleeding disorder seen for 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 children and young people with severe Haemophilia A or B or Factor X deficiency 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 children and young people with severe Haemophilia A or B or Factor X deficiency on regular prophylaxis.

Table 3 Children and young people with severe Haemophilia A or B or FX 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 children and young people 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 children and young people with severe or moderate Haemophilia A or B registered at the centre.

Table 4 Severe or moderate children and young people with a recorded joint score

Numerator: Number of children and young people who have received a new psychological intervention or episode of care relating to their bleeding disorder

Denominator: Number of children and young people, or their family members or carers, who have been referred for, or have otherwise received, a new psychology intervention or episode of care

Table 5 Children and young people, or their families, with an inherited bleeding disorder who received clinical psychology support

Numerator: Number of children and young people reporting a 'good' or 'excellent' experience of care

Denominator: Number of survey responses during the reporting period

Table 6 Children and people reporting a 'good' or 'excellent' experience of care

The outcomes above include all children with severe inherited bleeding disorders (not only haemophilia). This means the denominator includes children and young people with rare bleeding disorders, some of whom we cannot give prophylaxis to (as it is not currently possible). It provides a target for us to improve the total proportion on prophylaxis, as new treatment choices become available in clinical trials.

Access to psychological support is supported well via referral. However, our target is to have access to routine psychological input as part of our MDT care, which is not currently possible.