1 Guidance

1 Guidance

This guidance replaces NICE technology appraisal guidance 42 issued in May 2002.

For details, see about this guidance.

1.1 Somatropin (recombinant human growth hormone) is recommended as a treatment option for children with growth failure associated with any of the following conditions:

  • growth hormone deficiency

  • Turner syndrome

  • Prader–Willi syndrome

  • chronic renal insufficiency

  • born small for gestational age with subsequent growth failure at 4 years of age or later

  • short stature homeobox‑containing gene (SHOX) deficiency.

1.2 Treatment with somatropin should always be initiated and monitored by a paediatrician with specialist expertise in managing growth hormone disorders in children. The choice of product should be made on an individual basis after informed discussion between the responsible clinician and the patient and/or their carer about the advantages and disadvantages of the products available, taking into consideration therapeutic need and the likelihood of adherence to treatment. If, after that discussion, more than one product is suitable, the least costly product should be chosen.

1.3 Treatment with somatropin should be discontinued if any of the following apply:

  • growth velocity increases less than 50% from baseline in the first year of treatment

  • final height is approached and growth velocity is less than 2 cm total growth in 1 year

  • there are insurmountable problems with adherence

  • final height is attained.

    In Prader–Willi syndrome evaluation of response to therapy should also consider body composition.

    Treatment should not be discontinued by default. The decision to stop treatment should be made in consultation with the patient and/or carers either by:

  • a paediatrician with specialist expertise in managing growth hormone disorders in children, or

  • an adult endocrinologist, if care of the patient has been transferred from paediatric to adult services.

  • National Institute for Health and Care Excellence (NICE)