Treatment - Restricted growth (dwarfism)

Some people with restricted growth (dwarfism) may be able to have growth hormone treatment or leg-lengthening surgery.

Growth hormone medication

Somatropin

The National Institute for Health and Care Excellence (NICE) recommends a human growth hormone treatment called somatropin as an option for children whose poor growth is linked to:

• growth hormone deficiency
• Turner syndrome
• Prader-Willi syndrome
• chronic kidney disease
• a genetic condition called SHOX deficiency

NICE also recommends it for some children who are born small and fail to catch up in growth by the age of 4 or later.

Somatropin treatment should be started and monitored by a child health specialist with expertise in managing growth hormone disorders in children (paediatric endocrinologist).

The hormone is given as a single daily injection, which can usually be done by a parent, carer, or by the child when they're old enough.

Skin reactions are the most commonly reported side effect. In very rare cases, the treatment is associated with persistent severe headaches, vomiting and vision problems.

Children with growth hormone deficiency respond very well to somatropin and may be able to reach a relatively normal adult height. The effectiveness in children with other conditions can vary.

Somatropin treatment may stop once your child stops growing, but in some cases it may be given into adulthood because it can sometimes help prevent problems such as weak bones (osteoporosis).

Somatrogon

NICE also recommends a growth hormone treatment called somatrogon for children and young people aged 3 to 17 years old with poor growth caused by growth hormone deficiency.

It works in a similar way to somatropin but is given as a weekly injection instead of a daily one.

Common side effects of somatrogon include headaches, a high temperature and anaemia. It may not be a suitable treatment for all children. A child health specialist will advise you.

Leg-lengthening surgery

If you have particularly short legs, a leg-lengthening process known as distraction may be an option.

Using surgery, the leg bone is broken and fixed to a special frame. With the support of the frame, the leg is stretched, allowing new bone to form in between the two broken ends of bone.

Over time, this bone gets stronger until it's able to support your weight.

Distraction can sometimes result in a significant increase in height, but it's a lengthy treatment and has a risk of complications, so isn't always recommended.

Some people have pain during or after the limb-lengthening procedure.

Other possible complications include:

• poor bone formation
• fracture
• infection
• bone lengthening at an inappropriate rate
• blood clots

There's also some uncertainty about the safety and effectiveness of the procedure.

It's therefore important that you understand exactly what it involves and talk to your surgeon or doctor about the risks if it's suggested as a possible treatment for you or your child.

Support and treatment for related problems

As restricted growth can be associated with several related conditions and problems, affected children and adults will be cared for by a multidisciplinary team of healthcare professionals.

This team may include:

• a paediatrician (child health specialist)
• a nurse specialist
• a physiotherapist
• an occupational therapist
• a dietitian
• an audiologist (hearing specialist)
• a speech and language therapist
• a neurologist (specialist in nervous system disorders)
• an orthopaedic surgeon
• a geneticist

You may have regular check-ups with members of your care team so your health can be monitored and any problems that develop can be treated.

Help and support

If you or your child has restricted growth the Restricted Growth Association website has information for parents.