Developing a Treatment Plan for XLH

X-linked hypophosphatemia (XLH) is a rare, progressive genetic disorder characterized by rickets-like symptoms such as osteomalacia (soft and weak bones), early osteoarthritis, fractures, pain, dental abscesses (buildup of pus in the teeth), and stunted growth.

The disease affects multiple organs, such as the bones, muscles, and teeth, and thus requires a combination of treatments. A treatment plan, which covers all aspects of the disease and its treatment, is therefore needed to effectively manage the symptoms of XLH.

Here are some pointers on what a treatment plan should include.

What is the purpose of a treatment plan?

A treatment plan is an important document that provides a comprehensive overview of the disease and its ongoing treatment. It’s designed with the following purposes:

• Guiding a patient in achieving goals that improve symptoms and overall quality of life.
• Providing an easy-to-reference document for family members, caregivers, and organizations to help the patient manage the disease. For example, if your child has XLH, his or her school can use the treatment plan to arrange for suitable accommodations or special education plans.
• Documenting goals and objectives including the use of therapies such as Crysvita (burosumab) or surgical interventions to improve XLH symptoms along with potential and reported side effects.

What goes into a treatment plan?

A treatment plan covers several aspects of the disease. These include:

• Information about the patient.
• A summary of family history and results of blood tests, urine tests, imaging tests, and genetic tests that have helped confirm an XLH diagnosis.
• A description of the symptoms in a way that is measurable, such as the extent of angular bone defects or how much body height is affected by the disease.
• Goals and objectives of the treatment, and tools to measure those objectives.
• Interventions needed to achieve the objectives.
• Progress and outcome reports.
• Duration of treatment and expected costs.
• A plan for continuing care.

How are goals set in a treatment plan?

A goal is a clinical statement describing the change a doctor wishes to see in the patient. Goals in a treatment plan are highly individualized and are decided by the clinician based on the patient’s symptoms. Effective goal-setting looks into ways to eliminate symptoms and methods to help the patient better cope with the disease.

How are the objectives set for each goal?

An objective describes a specific skill set needed to achieve a goal. For example, proper care of teeth is an objective to achieve the goal of improved dental health for children with XLH who commonly get dental abscesses.

What is my role in the development of a treatment plan?

While doctors devise treatment plans based on their expertise, proper and timely feedback from patients or their families goes a long way in ensuring that goals are met. A treatment plan will be based on your conversations with the doctors so be as comprehensive as possible.

Remember that as a patient, you have the ongoing right to develop and participate in a treatment plan and have various aspects of the plan explained and made understandable to you.

What is the importance of consent in a treatment plan?

A treatment plan is a document personalized for you or your child, developed by a licensed independent practitioner along with a multi-disciplinary team. It is an essential part of ongoing therapy. By signing and giving consent to the plan, you acknowledge that you understand and agree to its contents.

Familiarize yourself with the plan before giving consent, and communicate any opinions about it before signing. Make sure your doctor clarifies any medical terminology that you don’t understand.

How is my privacy protected?

The Health Insurance Portability and Accountability Act ensures the privacy and security of the information that goes into a treatment plan. Under this Act, your information is kept confidential and cannot be shared with third parties without explicit consent, except for a few circumstances such as when required by law enforcement or for essential government functions.

Last updated: March 2, 2020.

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XLH News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.