Rickets is a condition primarily affecting children, causing bones to soften and weaken. Living with Rickets can lead to bone pain, muscle weakness, and, if untreated, visible skeletal deformities such as bowed legs or thickened ankles. These symptoms often limit a child’s mobility and can be alarming for parents. While once common, Rickets remains a serious concern that demands prompt medical attention.

Treatment is essential to reverse the bone softening process, promote healthy growth, and prevent permanent physical abnormalities. Rickets occurs when the body lacks enough Vitamin D, calcium, or phosphate, the minerals necessary to harden the growing ends of bones. Because the underlying cause can be nutritional (a lack of Vitamin D/calcium) or genetic, medication choices depend entirely on the specific diagnosis. Treatment aims to restore the necessary mineral balance to allow the bones to heal and strengthen (Mayo Clinic, 2024).

Overview of treatment options for Rickets

The primary goal of Rickets treatment is to replace the deficient nutrient, which targets the root cause of the disorder. For the vast majority of cases known as nutritional Rickets, treatment is straightforward and focuses on simple, high-dose supplementation.

Treatment strategies vary significantly between common and rare forms. Nutritional Rickets is treated acutely with high doses of Vitamin D, which is then continued at a maintenance level, often coupled with calcium. For rarer forms, such as hereditary or X-linked hypophosphatemic Rickets (XLH), the problem is not a lack of nutrients but the kidney’s inability to retain them, requiring specialized pharmacological agents. While medication is the central therapy, severe, long-standing skeletal deformities may eventually require surgical intervention once the bones have been strengthened.

Medications used for Rickets

For nutritional Rickets, the first line of treatment involves high-dose supplements. The main drug class is Vitamin D (calciferol), often administered as a short, intensive course followed by a daily maintenance dose. This is almost always paired with Calcium supplements, as calcium is the primary mineral used to harden the bone tissue. Improvement in bone pain and muscle weakness is typically seen within days, and signs of healing appear on X-rays within a few months (Centers for Disease Control and Prevention, 2022).

For genetic Rickets, specialized drug classes are required:

1. Phosphate and Active Vitamin D Analogs: For hereditary forms involving kidney loss, oral phosphate salts are required, often alongside specialized, active Vitamin D analogs like calcitriol.
2. Monoclonal Antibodies: For XLH, a newer drug, burosumab, is available. This injectable medication directly addresses the underlying genetic fault and represents a targeted shift in therapy.

How these medications work

In nutritional Rickets, the Vitamin D provided by the medication is crucial because its active form acts like a hormone. It significantly increases the absorption of calcium and phosphate from the food in the intestines. This ensures enough of these vital minerals are delivered to the growth plates of the bones, allowing the soft, rubbery tissue to become properly mineralized and hard.

For specialized treatments, the mechanism is often more complex. Active Vitamin D analogs bypass the body’s need to convert Vitamin D into its usable form. Burosumab, used for XLH, is a monoclonal antibody that targets and neutralizes a specific hormone called fibroblast growth factor 23 (FGF23). In XLH, excessive FGF23 causes the kidneys to waste phosphate; by blocking it, the drug helps the body retain phosphate, providing the essential mineral needed for bone strengthening.

Side effects and safety considerations

Regardless of the form of Rickets being treated, careful monitoring is essential. The primary safety concern with all high-dose Vitamin D and calcium supplements is hypercalcemia (excessive calcium in the blood). This can cause symptoms like nausea, vomiting, fatigue, and can lead to kidney stones if levels remain too high.

Oral phosphate salts can commonly cause gastrointestinal distress, such as diarrhea, and may also increase the risk of kidney stones. Patients receiving active treatment for any form of Rickets require regular blood tests to check levels of calcium, phosphate, and other markers. Patients should seek immediate medical care if they experience signs of high calcium, such as confusion or severe, persistent vomiting (National Institutes of Health, 2021).

Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care.

References

1. Mayo Clinic. https://www.mayoclinic.org
2. Centers for Disease Control and Prevention. https://www.cdc.gov
3. National Institutes of Health. https://www.nih.gov
4. MedlinePlus. https://medlineplus.gov