What investigations are recommended for a patient with Congenital Adrenal Hyperplasia (CAH) on fludrocortisone and hydrocortisone therapy?

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Monitoring Investigations for CAH on Fludrocortisone and Hydrocortisone

Patients with congenital adrenal hyperplasia on fludrocortisone and hydrocortisone should be reviewed at least annually with assessment of health and well-being, measurement of weight, blood pressure, and serum electrolytes. 1

Core Monitoring Parameters

Clinical Assessment (At Each Visit)

  • Weight and growth velocity in children, as hydrocortisone dose adversely affects height velocity and final height 2, 3
  • Blood pressure in supine and standing positions to evaluate mineralocorticoid adequacy 1
  • Assessment for salt cravings or lightheadedness, which indicate under-replacement of mineralocorticoid 1
  • Peripheral edema, which may indicate mineralocorticoid over-replacement 1
  • Overall sense of well-being and energy levels to assess glucocorticoid adequacy 1

Laboratory Investigations

Essential Tests (Annual Minimum)

  • Serum electrolytes (sodium and potassium) to monitor mineralocorticoid replacement adequacy 1
  • Morning 17-hydroxyprogesterone (17-OHP) to assess disease control, though elevated levels may be acceptable if growth and clinical parameters are normal 4, 2
  • Plasma renin activity or direct renin concentration to guide fludrocortisone dosing 1

Additional Monitoring in Children

  • Height velocity standard deviation score at each visit, as this is adversely affected by hydrocortisone dose 2
  • Bone age assessment every 1-2 years to monitor for accelerated bone maturation 4, 5
  • Androstenedione and testosterone levels to assess androgen control 2, 6
  • ACTH levels periodically, as higher ACTH correlates with better growth outcomes and may indicate under-treatment 2

Frequency of Monitoring

  • Children: Every 3-6 months during active growth, with more frequent monitoring during puberty when hydrocortisone requirements increase 2
  • Adults: At least annually, with assessment of weight, blood pressure, and serum electrolytes 1

Screening for Complications

Glucocorticoid-Related Complications

  • Bone mineral density every 3-5 years to assess for glucocorticoid-induced osteoporosis 1
  • BMI monitoring at each visit, as overweight remains a clinical challenge in CAH 3

Autoimmune Screening

  • Occasional monitoring for development of new autoimmune disorders, particularly hypothyroidism with TSH and thyroid peroxidase antibodies 1

Important Caveats

Do not rely solely on 17-OHP levels to guide therapy. Higher 17-OHP levels may be acceptable if growth velocity, bone maturation, and clinical parameters remain normal, as excessive glucocorticoid dosing to normalize 17-OHP impairs final height 2, 3

Avoid using prednisolone in growing children, as it is associated with shorter final height and higher BMI compared to hydrocortisone alone, even at equivalent total glucocorticoid doses 3

The lowest effective hydrocortisone dose should be used, particularly during puberty, to optimize pubertal growth spurt and final height 2

Urinary steroid profiling by LC-MS/MS represents a useful non-invasive alternative to frequent plasma measurements, particularly in children, and can discriminate between controlled and uncontrolled patients 6

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

HYDROCORTISONE THERAPY AND GROWTH TRAJECTORY IN CHILDREN WITH CLASSICAL CONGENITAL ADRENAL HYPERPLASIA.

Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists, 2017

Research

Urinary steroidomic profiles by LC-MS/MS to monitor classic 21-Hydroxylase deficiency.

The Journal of steroid biochemistry and molecular biology, 2020

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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