What is the Treatment for Laron Syndrome and Its Cost?

Recombinant IGF-1 Therapy: A Lifeline for Laron Syndrome Patients

Living with Laron syndrome can be challenging, especially when affected individuals cannot accept their short stature. It leads to low self-confidence and struggles in social and emotional well-being. About 300 to 500 people globally are diagnosed with this rare condition. It is time to raise awareness about treatment and medical care costs in India.

What is the Treatment for Laron Syndrome?

Replacement Therapy with Recombinant Human IGF-1 (rhIGF-1) is the only treatment for Laron syndrome. This therapy is done substantially twice a day through injection to reduce adiposity and improve linear growth in patients. Growth hormone therapy is ineffective in Laron Syndrome due to receptor insensitivity. Recombinant human IGF-1 (rhIGF-1) is the only effective treatment to improve linear growth and metabolic outcomes.

• Adverse effects of replacement therapy include retention of dys-electrolytemia, water, and calciuria.
• IGF-1 replacement improves growth velocity and contributes to skeletal maturity over time. GHR cannot be used therapeutically due to its genetic defect in Laron Syndrome.

Doctors usually recommend prescribing a low-fat, high-protein diet to enhance IGF-1 response. Maintaining a diet while treating this rare genetic disease can reduce the risk of obesity in future.

What is the Cost of Laron Syndrome Treatment?

Laron syndrome treatment involves two injections of rhIGF-1 and is tailored based on the child’s weight. For a child weighing 20 kg, the treatment cost is estimated at over Rs. 15 lakhs. While currently limited in availability, efforts are ongoing to improve access to this specialised therapy in India.

What are the 3 Benefits of Replacement Therapy with rhIGF-1?

Here are some benefits of replacement therapy.

• This multidisciplinary approach improves height prognosis, around 116 to 142 cm for men and 108 to 136 cm for females.
• Despite the appearance of premature ageing signs, including wrinkled and thin skin, patients usually live longer.
• IGF-1 replacement protects patients from malignancy, which is a most appreciable benefit of this therapy.

What are the 3 Side Effects of Replacement Therapy with rhIGF-1?

However, this replacement therapy also has some health risks, including:

• This therapy may increase the risk of cardiovascular disease due to the low level of IGF-1.
• Treatment of Laron syndrome can lead to local reactions and hypoglycaemia, including lymphoid tissue hypertrophy and redness.
• In rare cases, replacement therapy increases intracranial pressure and causes headaches as side effects.

What are the Strategies to Manage Laron Syndrome Effectively?

In case Laron syndrome is left untreated, it can lead to life-threatening complications, including heart disease, cancer and diabetes. Early diagnosis and proper treatment can manage the symptoms of the affected individual. However, patients can strategise to manage this rare condition through:

• Monitor metabolic health
• Focus on growth hormone therapy regularly
• Maintain dietary adjustments in daily lifestyle to support well-being

Laron syndrome causes short stature from childhood due to growth hormone deficiency. The only treatment option is the administration of regular recombinant human IGF-1 replacement therapy via injection twice a day from early childhood. This treatment with high doses of daily therapy requires a huge cost for a single injection, which is inaccessible for the majority of patients.