Overview
What are Growth Disorders?
Growth disorders impact normal tissue growth, and can arise from many causes including malnutrition or poor nutrient absorption, hormone disorders, bone and collagen disorders, or genetic and metabolic diseases. Short stature associated with growth disorders is typically defined as someone who less than the third percentile of average height compared to others of the same age and birth sex. This means they are currently shorter than or projected to be shorter than 97% of their peers. Short stature can also be defined as relative to parental height, or someone who is much shorter than their expected height given their genetic growth potential. Severe short stature, or a projected adult height less than 4’10” tall, can significantly impact activities of daily living, and may require adaptations to perform tasks: such as workplace duties, driving, and household tasks.
Growth disorders with specialty medication treatments include:
- Growth hormone (GH) deficiency and growth-hormone responsive conditions
- Insulin-like growth factor-1 (IGF-1) deficiency
- Achondroplasia
More about Growth Hormone and IGF-1 related conditions:
Growth hormone (GH) is secreted from the anterior pituitary gland, which is located at the base of the brain. In response to growth hormone, the body releases Insulin-like Growth Factor-1 (IGF-1) to signal tissues to grow.1
Growth hormone-treated disorders could be a result of growth hormone deficiency or could be due to a genetic condition responsive to growth hormone treatment. Growth hormone deficiency could occur in isolation or can also be acquired at any time of life as the result of pituitary damage from brain injury, radiation treatments, brain infection, or tumors. Sometimes, growth hormone deficiency (GHD) or short stature responsive to GH treatment may have no known cause and improve after childhood. Some children may continue to be growth hormone deficient as adults, especially if they have other pituitary hormone disorders.
Short stature may occur whether or not there is growth hormone deficiency. Examples short stature diagnoses where growth hormone is used include idiopathic short stature and babies born small for gestational age, who do not catch up to average height by age 2. Some congenital causes of short stature that may improve with growth hormone treatment include Turner Syndrome, Russel-Silver Syndrome, Prader-Willi Syndrome, Noonan Syndrome, and SHOX deficiency.
Growth hormone replacement injections may be given until childhood growth stops. In patients with continued growth hormone deficiency, treatment continues into adulthood.
Growth hormone insensitivity can be a result of IGF-1 deficiency. This rare diagnosis can cause severe short stature, disproportionate body features, and developmental abnormalities. It can be treated with mecasermin, which is a twice daily injection.2
Due to the potential for abuse and misuse, federal law prohibits dispensing growth hormone for unapproved uses, such as anti-aging or cosmetic treatment, performance enhancement or sports recovery, weight loss, or to boost height in average-height children. Growth hormone does not have a proven benefit for these uses. In some states, growth hormone is scheduled as a controlled substance to deter possible abuse.
More about Achondroplasia:
Achondroplasia is a disorder of bone growth that prevents the changing of cartilage to bone. It occurs as the result of a genetic defect in the fibroblast growth factor receptor type 3 (FGFR3) gene. Most cases occur at random; however those with achondroplasia have a 50% chance of having a child with achondroplasia. Achondroplasia causes severe short stature, disproportionate body features, and complications such as spinal compression, scoliosis, sleep apnea, hearing problems and ear infections.
How common are growth disorders?
Short stature occurs in about 2.5% of children. Growth hormone deficiency is diagnosed occurs in approximately 1:4,000 to 1:10,000 children. Adult GH deficiency affects approximately 1:100,000 people per year, with approximately 6,000 new adult cases being diagnosed each year in the United States.3
Achondroplasia occurs in 1:20,000 to 1:30,000 live births. It is the most common skeletal disorder leading to severe short stature.4
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- 1Cook, David, et al. "American Association of Clinical Endocrinologists medical guidelines for clinical practice for growth hormone use in growth hormone-deficient adults and transition patients-2009 update." Endocrine practice (2010).
- 2Human Growth Foundation. Epidemiology of Growth Hormone Deficiency. Available at: Human Growth Foundation
- 3Bamba, Vaneeta. Pediatric Growth Hormone Deficiency Medscape. Last Updated: Dec 09, 2022.
- 4Pauli, R.M. Achondroplasia: a comprehensive clinical review. Orphanet J Rare Dis 14, 1 (2019).
Symptoms
What are the symptoms of growth disorders?
Childhood-onset Growth Hormone Deficiency:1 2
- Immature facial features
- Micropenis
- Slow tooth eruption
- Delayed lengthening of long bones
- Fine hair
- Poor nail growth
- Truncal obesity
- High pitched voice
- Bouts of hypoglycemia
- Low growth velocity
- Delayed puberty
- Low birth weight
Adult-onset Growth Hormone Deficiency:3 4
- Relative increase in fat mass (especially abdominal and visceral)
- Decrease in muscle mass
- Decreased energy levels
- Anxiety and/or depression
- Increase in LDL cholesterol and triglyceride levels
Achondroplasia:
- Disproportionate short stature, seen at birth
- Shorter limb to trunk ratio
- Decreased muscle tone
- Shortened fingers
- Shortened fingers
- Abnormal flexibility
- Bowed legs with growth
- Abnormal curvature of the spine
- Hearing loss (middle-ear defects)
- Frequent ear infections
- Sleep apnea
How is it diagnosed?
Medical history including gestational events, family history, and growth charts (in children) are important parts of a growth disorder work up.
Growth hormone deficiency work up also includes x-rays for bone age (in children), lab tests and may include imaging to rule out certain diagnoses. Children with GHD have delayed bone age compared to same-age peers. Laboratory tests may include IGF-1 level, IGFBP-3 level (insulin growth factor binding protein-3), thyroid hormone level, pituitary hormone levels, and tests that stimulate growth hormone release. Growth hormone stimulation tests may take several hours of monitoring and blood draws to assess the body’s ability to release GH.2
- 1Bamba, Vaneeta. Pediatric Growth Hormone Deficiency Medscape. Last Updated: Dec 09, 2022.
- 2Rieser, Patricia A. Pediatric Growth Hormone Deficiency Human Growth Foundation. Accessed February 19, 2018.
- 3Cook, David, et al. "American Association of Clinical Endocrinologists medical guidelines for clinical practice for growth hormone use in growth hormone-deficient adults and transition patients-2009 update." Endocrine practice (2010).
- 4Human Growth Foundation. (last updated 2015) Epidemiology of Growth Hormone Deficiency. Available at: Adult Growth Hormone Deficiency Accessed December 3, 2016.
Medications
Growth Hormone
The following specialty medications are available at Accredo, a specialty pharmacy for Growth Hormone.
Growth Hormone used for growth disorders:
Financing Your Care
Financial assistance may be available to help with your medication costs, including manufacturer and community programs. Accredo specialists are available to help find a program that may work for you.
- Genotropin® Savings Program | 800-645-1280
- Humatrope® Copay Program | 866-923-1953
- Increlex® IPSEN CARES® | 866-435-5677
- Norditropin® NordiSure™ | 888-668-6444
- Nutropin Copay Card Program | 866-688-7674
- Omnitrope® Copay Support | 877-456-6794
- Serostim® Copay Assistance Program | 877-714-2947
- Voxzogo® Assistance at BioMarin Rare Connections™ | 866-906-6100
- Skytrofa ASAP Program | 844-442-7236
- Zomacton ZoGo Support Program 844-944-ZOGO (9646)
Financial Guidance
Life-saving specialty medication can be expensive. Learn how the Accredo teams help individuals find ways to afford the medication they need to survive in this video.
Find Support
Growth Disorders Organizations
There are many organizations that support research and advocacy for Growth Disorders. See below for a few of those organizations.
Support organizations
- National Organization for Rare Disorders (NORD)
- Child Growth Foundation
- Human Growth Foundation
- MAGIC Foundation
- Little People of America| 714-368-3689
Government organizations
Meet the Team
Accredo’s Growth disorders team is dedicated to serving you and we understand the complexity of your condition. Our specialty-trained clinicians are available 24 hours a day, seven days a week, to answer any questions.
Why We Do It
Accredo supports patients with chronic and complex conditions and helps them live their best life. Watch our video to learn why we do what we do for our patients.