Haemophilia clinical outcomes

Clinical outcomes are measurable changes in health, function or quality of life that result from our care. Constant review of our clinical outcomes establishes standards against which to continuously improve all aspects of our practice.

About the Haemophilia Service

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.

Clinical outcome measures

1. Number of patients prescribed prophylactic treatment

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, 2020/21

Number of severe Haemophilia A or B patients on active treatment Quarter 1 Quarter 2 Quarter 3 Quarter 4
Number of patients prescribed a prophylactic regimen 132 133 135 141
Number of patients on active treatment 136 134 137 143
Percentage of patients prescribed a prophylaxis regimen 97.1% 99.3% 98.5% 98.6%

2. Number of severe patients seen for review

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. All patients were offered at least two reviews within this period but due to the COVID epidemic, attendance was not possible for all families.

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

Number of severe Haemophilia A or B patients seen for a review 2020/21
Number of severe patients reviewed twice or more within annual review cycle 136
Number of severe patients 140
Percentage of severe patients reviewed twice or more within annual review cycle 97.1%

3. Patients regularly reporting treatment and outcomes

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

Number of severe Haemophilia A or B patients  reporting treatment and outcomes 2020/21
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 123
Number of severe patients on prophylaxis 138
Percentage 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 89.1%

4. Number of patients with a recorded joint score

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

Number of severe or moderate Haemophilia A or B patients over 4 years old with a recorded joint score 2020/21
Number of severe or moderate patients with a joint score recorded at the most recent clinical review within the annual review cycle 106
Number of severe or moderate patients 113
Percentage of severe or moderate patients with a joint score recorded at the most recent clinical review within the annual review cycle 93.8%

This information was published in September 2021.