UPSC MainsMEDICAL-SCIENCE-PAPER-I202410 Marks
Q14.

Enumerate the major hormonal causes of dwarfism. Give their characteristic features.

How to Approach

This question requires a detailed understanding of pediatric endocrinology. The approach should involve listing the major hormonal deficiencies causing dwarfism, followed by a description of the characteristic features of each. Focus on growth hormone deficiency, hypothyroidism, glucocorticoid excess (Cushing's syndrome), Turner syndrome, and skeletal dysplasias affecting hormonal pathways. Structure the answer by first defining dwarfism, then discussing each hormonal cause separately, including clinical manifestations, diagnostic tests, and potential treatments. A tabular format can be used for concise comparison.

Model Answer

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Introduction

Dwarfism is generally defined as an adult height of 4 feet 10 inches (147 cm) or less. It arises from a variety of genetic and medical conditions, many of which involve hormonal imbalances impacting growth and development. While skeletal dysplasias are the most common cause, significant proportions of dwarfism are attributable to hormonal deficiencies or excesses. Early diagnosis and appropriate hormonal intervention are crucial for optimizing growth potential and improving the quality of life for affected individuals. This answer will enumerate the major hormonal causes of dwarfism and detail their characteristic features.

Growth Hormone Deficiency (GHD)

GHD is the most common hormonal cause of proportionate dwarfism. It can be congenital (present at birth) or acquired due to pituitary gland damage from tumors, trauma, or infection.

  • Characteristic Features: Slowed growth rate (typically after the first year of life), delayed bone age, increased fat mass, decreased muscle mass, youthful facial features, and delayed puberty.
  • Diagnosis: Growth hormone stimulation tests (e.g., insulin tolerance test, arginine stimulation test), measurement of Insulin-like Growth Factor-1 (IGF-1).
  • Treatment: Growth hormone replacement therapy.

Hypothyroidism

Severe, untreated congenital hypothyroidism (cretinism) can lead to growth retardation and dwarfism. This occurs due to insufficient thyroid hormone production.

  • Characteristic Features: Short stature, delayed bone age, sluggishness, constipation, dry skin, puffy face, and intellectual disability if untreated.
  • Diagnosis: Measurement of thyroid hormone levels (T4, T3) and thyroid-stimulating hormone (TSH). Neonatal screening programs are crucial for early detection.
  • Treatment: Thyroid hormone replacement therapy (levothyroxine).

Glucocorticoid Excess (Cushing's Syndrome)

Prolonged exposure to high levels of glucocorticoids (cortisol) can suppress growth hormone secretion and inhibit bone growth, leading to dwarfism.

  • Characteristic Features: Slowed growth, weight gain (especially in the trunk), moon face, buffalo hump, thin skin, easy bruising, and hypertension.
  • Diagnosis: 24-hour urinary free cortisol measurement, dexamethasone suppression test, salivary cortisol levels.
  • Treatment: Addressing the underlying cause of cortisol excess (e.g., tumor removal, medication adjustment).

Turner Syndrome (Monosomy X)

This genetic condition affects females and results in the absence of one X chromosome. It leads to ovarian dysgenesis and reduced estrogen production, impacting growth.

  • Characteristic Features: Short stature, ovarian failure (leading to infertility), webbed neck, broad chest, lymphedema, and congenital heart defects.
  • Diagnosis: Karyotyping (chromosome analysis).
  • Treatment: Growth hormone therapy and estrogen replacement therapy.

Skeletal Dysplasias Affecting Hormonal Pathways

Certain skeletal dysplasias, while primarily affecting bone and cartilage development, can also impact hormonal signaling pathways involved in growth.

  • Achondroplasia: The most common form of skeletal dysplasia, caused by a mutation in the FGFR3 gene. While not a primary hormonal deficiency, it can affect growth hormone signaling.
  • Pseudoachondroplasia: Another skeletal dysplasia affecting cartilage development.
Hormonal Cause Characteristic Features Diagnostic Test Treatment
Growth Hormone Deficiency Slowed growth, delayed bone age, increased fat mass Growth hormone stimulation tests, IGF-1 Growth hormone replacement therapy
Hypothyroidism Short stature, delayed bone age, sluggishness T4, T3, TSH levels Thyroid hormone replacement therapy
Cushing's Syndrome Slowed growth, weight gain, moon face 24-hour urinary free cortisol, dexamethasone suppression test Address underlying cause of cortisol excess
Turner Syndrome Short stature, ovarian failure, webbed neck Karyotyping Growth hormone and estrogen replacement therapy

Conclusion

In conclusion, hormonal causes represent a significant proportion of dwarfism cases, each presenting with distinct clinical features and requiring specific diagnostic approaches. Early identification through newborn screening and comprehensive endocrine evaluation is paramount. Treatment strategies, ranging from hormone replacement therapy to addressing underlying causes of hormonal imbalances, can significantly improve growth outcomes and overall quality of life for individuals affected by these conditions. Continued research into the complex interplay between hormones and growth remains crucial for optimizing care.

Answer Length

This is a comprehensive model answer for learning purposes and may exceed the word limit. In the exam, always adhere to the prescribed word count.

Additional Resources

Key Definitions

IGF-1
Insulin-like Growth Factor-1 is a hormone similar in structure to insulin, which plays a crucial role in childhood growth and development. It is produced primarily by the liver in response to growth hormone stimulation.
Bone Age
Bone age refers to the maturity of a child's skeletal system, as determined by X-ray of the left hand and wrist. It is used to assess growth and development and can help identify hormonal imbalances affecting growth.

Key Statistics

The prevalence of GHD is estimated to be 1 in 4,000 to 1 in 10,000 live births.

Source: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) - Knowledge cutoff 2023

Turner syndrome affects approximately 1 in 2,000 to 1 in 2,500 female births.

Source: National Human Genome Research Institute - Knowledge cutoff 2023

Examples

Case of Congenital Hypothyroidism

A newborn screened positive for hypothyroidism via heel prick testing. Early initiation of levothyroxine therapy prevented severe intellectual disability and allowed for near-normal growth and development.

Frequently Asked Questions

Can growth hormone therapy completely normalize height in children with GHD?

While growth hormone therapy can significantly improve growth velocity and final adult height, it may not always result in a completely normal height. The extent of catch-up growth depends on factors such as the severity of the deficiency, age at initiation of treatment, and genetic potential.

Topics Covered

EndocrinologyPediatricsGrowth DisordersHormonesDwarfism