Growth Hormone Therapy: Is Your Child Getting the Most Out of Treatment?
"New research highlights the critical role of consistent adherence to growth hormone regimens in achieving optimal catch-up growth for children with growth hormone deficiency."
For parents of children diagnosed with growth hormone deficiency (GHD), the prospect of growth hormone (GH) therapy offers a beacon of hope. These treatments can stimulate significant catch-up growth, helping children achieve heights closer to their genetic potential. However, the effectiveness of GH therapy isn't solely dependent on the medication itself; consistent adherence to the prescribed treatment plan is equally vital.
While the medical community recognizes the importance of adherence, accurately measuring its impact has been challenging. Traditional methods often rely on self-reporting, which can be subjective. Moreover, isolating the effect of adherence from other factors influencing growth, such as the severity of GHD, age, and overall health, adds another layer of complexity.
Recent research published in PLOS One has shed new light on this critical area, providing compelling evidence of how adherence to growth hormone treatment directly affects growth outcomes in children. By using data from the easypod connect observational study (ECOS), researchers were able to continuously assess real-world adherence through an electronic drug-delivery device. This innovative approach allowed for a more objective and detailed analysis of the relationship between adherence and growth.
Adherence Shapes Outcomes
Growth hormone therapy for childhood growth disorders requires daily injections over many years, making sustained compliance difficult. Multiple studies identify long-term adherence as a key factor in treatment success and growth outcomes. A clinical registry study specifically examined the association between adherence to daily GH therapy and two-year growth outcomes in paediatric patients.
From Discovery to Modern Therapy
The search for growth hormone treatment for deficient children began in the 1940s. Early researchers found that GH was species specific, meaning it had to be obtained from another human brain. Later developments expanded the approved uses of GH beyond classical growth hormone deficiency. Reviews of its history also emphasize the need for continuous follow-up to better understand long-term treatment safety, including mortality, cardiovascular morbidity, and cancer risks.
Why Does Adherence Matter for Growth Hormone Therapy?
The study's findings underscore the significance of consistent adherence to GH therapy during the initial years of treatment. When children adhere to their GH treatment plans, they experience more significant improvements in height. This early catch-up growth is crucial for their overall development and well-being.
- Objective Measurement: The use of the easypod device provided continuous, objective data on adherence, overcoming the limitations of self-reported measures.
- Comprehensive Analysis: The study considered various outcome measures, including changes in height standard deviation score (ΔHSDS) and index of responsiveness (IoR), offering a holistic view of growth patterns.
- Control for Confounders: The researchers adjusted for potential clinical confounders such as age, birth weight, and GH dose, isolating the true impact of adherence.
Evidence on Adherence and Newer Treatments
A prospective multicenter study reported that more than one-third of patients with permanent congenital organic growth hormone deficiency during transition had suboptimal adherence, alongside adverse metabolic markers and persistently lower IGF-1 levels. In a three-year extension of a phase 3 trial, children with growth hormone deficiency receiving somatrogon showed a persistent increase in linear growth velocity compared with baseline. Separately, registry findings associated sustained adherence of at least 85% to daily GH therapy with clinically meaningful growth improvement over two years.
Why Treatment May Underperform
Poor adherence has been associated with worse clinical outcomes, including reduced linear growth and loss of quality of life. Adherence to GH therapy remains variable and can affect treatment response, future health, and economic consequences. However, treatment response is not predictable at the start and depends on several factors, with diagnosis identified as the main one in one review. One study limitation was the absence of IGF-I measurements to assess GH status and predict growth response and adherence.
Taking Control: How to Support Adherence to Growth Hormone Therapy
Understanding the critical role of adherence is the first step. For parents and caregivers, this study underscores the importance of creating a supportive environment that encourages consistent treatment. Here are some practical strategies to help your child adhere to their growth hormone therapy:
Check Adherence First
A multicenter study reported that poor adherence to GH therapy was common and was one factor underlying suboptimal growth during treatment. Its conclusion recommends that clinicians confirm good adherence before considering other problems that may affect growth. This makes adherence assessment an important first step when a child's response appears less than expected.
Long-Acting and Digital Directions
Emerging non-injectable modalities are under investigation and could influence future substitution trends, although human growth hormone remains the standard for approved growth hormone deficiency therapy. Market estimates differ substantially: one report places the global human growth hormone market at USD 7.20 billion in 2024 and projects it to surpass USD 23.38 billion by 2034, while another estimates the growth hormone therapy market at USD 5.3 billion in 2024 and USD 9.9 billion by 2033. A separate industry commentary reports a digital transformation market value of USD 3.41 billion in 2025 and projects further growth.