Haemophilia clinical outcomes

Clinical outcomes are broadly agreed, measurable changes in health 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 on home 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. So, in order to minimise the disruption to patients and their families’ lives, the centre facilitates home treatment, where the patient or their parents will carry out the infusion safely at home.

Numerator: Number of severe Haemophilia A or B patients on active treatment who accessed 95% or more of their treatment at home.

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

Table 1 Number of severe Haemophilia A or B patients on home treatment

Number of severe Haemophilia A or B patients on home treatment

2013/14

2014/15

Number of patients on prophylaxis treated at home  131 129
Number of patients on prophylaxis 137 135
Percentage of patients on prophylaxis treated at home 95.6% 95.6%
 

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, who attended this service.

Numerator: Number of patients with severe Haemophilia A or B registered at the Centre, who were seen within the previous six months.

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
2013/14 2014/15
Number of severe patients seen for a review 132 132
Number of severe patients  137 135
Percentage of severe patients seen for a review 96.4% 97.8%

3. Number of breakthrough bleeds

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. Therefore, this measure does not include bleeds caused through trauma e.g. a fall or football tackle. A breakthrough bleed is recorded per bleed (ie not per patient, so if one patient had two simultaneous bleeds, that would be recorded as two bleeds).

Numerator: Number of breakthrough bleeds in the previous three months, in patients with severe Haemophilia A or B who are on regular prophylaxis.

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

Table 3 Number of breakthrough bleeds*

Number of breakthrough bleeds in severe Haemophilia A or B patients on regular prophylaxis
2013/14 2014/15
Total bleeds

≤ 5

6
Total patients on prophylaxis 137 135

* To prevent the identification of individual patients, where there are low numbers of patients, ie fewer than five, exact numbers are not shown.

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.

Numerator: Number of patients with severe or moderate Haemophilia A or B who have a joint score recorded.

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

Table 4 Number of patients with a recorded joint score

Number of patients with severe or moderate Haemophilia A or B patients who have a joint score recorded

2014/15

Number of severe or moderate patients who have a joint score recorded 154
Number of severe or moderate patients 155
Percentage of severe or moderate patients who have a joint score recorded 99%

This information was published in April 2017.