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 with a severe inherited bleeding disorder prescribed a prophylactic regimen.

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

Table 1 Number of children with a severe inherited bleeding disorder prescribed a prophylactic regimen, 2022/23

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 with a severe inherited bleeding disorder registered at the Centre reviewed at least twice within each annual review cycle.

Denominator: Number of patients with a severe inherited bleeding disorder 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 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 patients with severe Haemophilia A or B or Factor X deficiency 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

Numerator: Number of patients who have received a new psychological intervention or episode of care relating to their bleeding disorder

Denominator: Number of patients, adult or paediatric, 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 Number of patients, or families of patients, with an inherited bleeding disorder who received clinical psychology support

Numerator: Number of patients who have received new social care support relating to their bleeding disorder

Denominator: Number of patients, adult or paediatric, or their family members or carers, who have been referred for, or have otherwise received, new social care support relating to their bleeding disorder

Table 6 Number of patients with an inherited bleeding disorder referred for social care support

Numerator: Number of patients reporting a 'good' or 'excellent' experience of care

Denominator: Number of survey responses during the reporting period

Table 7 Number of patients reporting a 'good' or 'excellent' experience of care

The outcomes above have been adjusted over the last year to include all children with severe inherited bleeding disorders rather than just haemophilia. This has increased the denominator in some of the categories to capture children with rare bleeding disorders, some of whom we cannot give prophylaxis to (as it is not currently possible). Whilst this means that the percentage on prophylaxis is relatively lower than in previous years (100% of our children with severe haemophilia are on prophylaxis), 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 and social work support are also new additions to our national outcome measures and whilst we have been able to support these referrals at GOSH, our target is to have access to routine psychological input as part of our MDT care, which is not currently possible.