NICE Technology Appraisal Guidance - No. 42 Guidance on the use
of human growth hormone (somatropin) in children with growth
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
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
- 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
- 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
- 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:
- 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
- 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.
- 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
- 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
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.
Published Date: 24 April 2002