NICE Technology Appraisal Guidance - No. 42 Guidance on the use of human growth hormone (somatropin) in children with growth failure

NICE Technology Appraisal Guidance - No. 42 Guidance on the use of human growth hormone (somatropin) in children with growth failure.

The Health Technology Board for Scotland (HTBS) is delivering this National Institute for Clinical Excellence (NICE) Guidance to health professionals in NHSScotland with the following authoritative Comment on its use in Scotland. This HTBS Comment must be read in conjunction with the NICE Guidance.

HTBS advises that the NICE Technology Appraisal Guidance - No. 42 Guidance on the use of human growth hormone (somatropin) in children with growth failure is as valid for Scotland as for England and Wales.

The NICE recommendations are shown below.

• 1.1 Recombinant human growth hormone (somatropin) treatment is recommended for children with proven clinical diagnosis of growth hormone (GH) deficiency supported by appropriate auxological, biochemical and radiological investigations.
• 1.2 GH treatment is recommended for children with Turners syndrome (TS). The following issues should be taken into consideration in order to maximise the benefit from this treatment:
  diagnosis and treatment at earliest age possible
  appropriate timing and use of oestrogen therapy
• .3 GH treatment is recommended for pre-pubertal children with chronic renal insufficiency (CRI) providing:
  nutritional status has been optimised
  metabolic abnormalities have been optimised
  steroid therapy has been reduced to minimum.
• 1.4 GH treatment is recommended for children with Prader-Willi syndrome.
• 1.5 GH treatment should, in all circumstances, be initiated and monitored by a paediatrician with special expertise in the management of children with GH disorder. Continuation of treatment can be maintained under an agreed shared-care protocol with a general practitioner.
• 1.6 GH treatment should be re-evaluated and normally discontinued if there is a poor response to treatment, defined as an increase in growth velocity of less than 50% from baseline, in the first year of therapy. Ongoing response should be evaluated against expected growth based on standard growth charts. Therapy should normally be stopped when final height is approached and growth velocity is less than 2 cm total growth in 1 year. Persistent and uncorrectable problems with adherence to treatment should also be taken into account as part of re-evaluation of treatment. In Prader-Willi syndrome evaluation of response to therapy should also consider body composition.
• 1.7 After attainment of final height, GH therapy will normally be discontinued, but it should not be discontinued by default. The decision to stop treatment should either be made by a paediatrician with special expertise in the management of children with GH disorders in consultation with patient and carers, or therapy should be continued until re-evaluation by an adult endocrinologist has been undertaken. The transition to adult care for people with GH disorders will require a close collaboration between the responsible clinicians.
• 1.8 In children with CRI, GH treatment should be stopped after renal transplantation. It should not normally be re-started until at least 1 year after renal transplantation to allow time to ascertain whether catch-up growth will occur.
• 1.9 The use of GH therapy in children with idiopathic short stature is currently not licensed, and therefore it was not considered as part of this appraisal.

HTBS anticipates that implementing this NICE Guidance in Scotland will have the following implications for NHSScotland:

Service Issues

• NHS Boards, Trusts and health professionals with responsibility for providing care for children with growth failure should review their current practice regarding the prescription of human growth hormone in children in line with this guidance.
• To enable health professionals to audit their own compliance with this guidance it is recommended that local protocols are adapted or, if none exist, developed and implemented locally. Criteria for measuring compliance are noted in Section 7.3 and Appendix D of the NICE Guidance.
• Local protocols should define shared-care arrangements for the treatment for these children. Protocols should also include the management of children going on to adult growth hormone replacement, as the care of patients at the interface between paediatric and adult management is important.
• Section 1.5 of the NICE Guidance states that only paediatricians should initiate and monitor treatment. Clinicians in Scotland have suggested that a small sub-group of children with pituitary tumours, who present around 15 or 16 years of age, be jointly managed by a paediatrician and an adult endocrinologist. Both specialists would have expertise in growth failure in children and could be part of a managed clinical network for such children. There should be further discussion on this issue by specialists and other interested parties to determine whether this is appropriate.
• NICE will be issuing guidance on the use of growth hormone treatment in adults in summer 2002.
  

The Clinical Standards Board for Scotland (CSBS) Generic Clinical Standards published in January 2001 defines the standards to which all clinical services should be delivered in Scotland.

Impact

• In Scotland, by extrapolation from the figures published by NICE, it is estimated that the total numbers of children with GH deficiency, TS, CRI, and PWS who would be eligible for growth hormone is 500 - 700.
• The mean annual cost of treatment is estimated to be #6,103 for a child with GH deficiency, #10,126 for a child with TS, #11,132 for a child with CRI, and #9,840 for a child with PWS. These figures are derived from the economic analysis detailed in the Assessment Report commissioned by NICE and are based on the annual treatment costs of a 30 kg child.
• In Scotland there are estimated to be over 160 children currently receiving treatment for GH deficiency, TS or CRI, incurring costs of around #1.2 million to NHS Scotland annually. However, there are an estimated further 174 children with GH deficiency, 95 275 children with TS and 33 children with CRI in Scotland who may be eligible for, but are not receiving, growth hormone treatment. Treating these children would cost NHS Scotland an additional #2.4 to #4.2 million per annum.
• The number of children with PWS who are currently receiving growth hormone treatment is unknown. The cost of treating all eligible children in Scotland (around 50) would be around #500,000 per annum. However only a proportion of this cost will be incurred, depending on the number of children with PWS who currently receive growth hormone treatment.

NHSScotland should take account of this Comment from HTBS and ensure that recommended drugs or treatments are made available to meet clinical need.

This HTBS Comment is the result of a consideration of possible contextual differences in Scotland, according to the following categories:

• Principles and values of NHSScotland
• Epidemiology (frequency and distribution)
• Structure and provision of services in Scotland
• Other implications for NHSScotland.

No important differences were identified for this NICE Technology Appraisal Guidance.

An Understanding HTBS Advice is also being distributed on this topic and is available from the HTBS website or office or patient group.

HTBS would like to thank NICE for its cooperation in delivering this Comment. HTBS is also grateful to the experts in Scotland who provided input to this Comment.