Medication Information

A number of medications have been used to treat generalized lymphatic anomaly (GLA)/lymphangiomatosis, Gorham-Stout disease (GSD),  as well as kaposiform lymphangiomatosis (KLA) and other complex lymphatic conditions, with varying results. There also are numerous medications that may be prescribed to help manage symptoms.

Currently there are no medications that have been approved by the Food and Drug Administration (FDA) specifically for these diseases. The use of medications that are not approved by the FDA for use for a specific disease is called off-label. Because of this, one most likely will not see the conditions listed in the drug’s literature. This is not unusual, especially for rare diseases.

Your most valuable resources for medication information are your physicians and nursing staff and your pharmacist. The internet can be a useful supplement to the information these professionals provide, but should not be viewed as a substitute for communication with your health care team.

The first three sections are definitions of medication terms that are important to understand and tips to help you organize and communicate your medication needs. The last section includes specific medications that have been reported in the medical literature as having been used for GLA/lymphangiomatosis, GSD, KLA, and other complex lymphatic anomalies.

Important Terms:

FDA Approved: The Food and Drug Administration has granted official permission allowing a drug manufacturer to market a new prescription drug for a specific health benefit.

Experimental: A drug that has been approved by the FDA for testing in humans. An experimental drug also may be one that has been approved by the FDA for use in humans for one disease or condition but is considered investigational in other diseases or conditions.

Off-Label: This is the use of a medication for a condition other than that for which it has official approval of the FDA. Sometimes after a drug has been in use for a period of time patients and physicians discover other symptoms or conditions for which the drug is effective that were not among the reasons the drug was originally approved. With the exception of some controlled drugs such as opiods, off-label use is legal, but the drug manufacturer is not allowed to advertise this use of the drug. Learn more about off-label uses of drugs here.

Side-Effect: An unwanted effect produced by a medication in addition to its desired therapeutic effect. Some side effects are irritants to the patient, such as dry mouth, while others can cause harm, such as suppression of immune response. It is important to discuss side effects with your physician and to report any unusual symptoms you experience, as these may or may not be side effects of a medication you are taking.

 

Keeping track of your medications:

  • Use weekly pill dispensers, fill them on the same day each week. This will help remind you to take the medications and is an easy way to determine if you took your pills or not.
  • For liquid medication place a magnetic dry-erase board or a piece of paper on the refrigerator door and on the day you fill your pill tray write out the day and time you are to take the liquid meds. Each time you take a dose, mark through or erase the notation.
  • Make a list of your medications, including over-the-counter drugs and vitamins, and dosages and keep it in your wallet. Include on this list the names of drugs and substances to which you are allergic.
  • Always use the same pharmacy for all your prescriptions. Using a national chain pharmacy will allow you to get refills should you run out of your medication when traveling. (This is a good practice for parents, as well.)
  • If you are prescribed a medication that you can only get from a specialty pharmacy, be sure to inform your regular pharmacy that you are taking the drug and give a copy of your list of medications to the specialty pharmacist.
  • Enroll in a service such as MedicAlert®, which will store all your medical information, such as diagnosis, medications, emergency contacts, and physician contact information that can be accessed electronically in case of emergency.

 

All drugs have side effects!

Become familiar with the listed side effects of your medications and consult your physician, nurse, and/or pharmacist with questions you have about your medications. Many side effects will go away after taking a drug for a period of time. Be sure to tell your doctor about any side effects that are severe. There may be another medication with fewer side effects or a smaller dose of the same drug may help relieve side effects.

Unfortunately, there are some side effects that are both unpleasant and unavoidable. Talk with your physician and your nurse about ways to lessen any discomfort caused by medication side effects. It may mean adding another medication to your regimen, which is frustrating for patients and doctors alike. But this is sometimes necessary. For example, some drugs may cause nausea, which interferes with eating and that leads to nutritional problems. There are medications that can relieve the nausea and help the patient eat, thus avoiding complications of poor nutrition.

 

Information About Medications

The listed medications have been reported in the medical literature as having been used for GLA/lymphangiomatosis, GSD, KLA, and other complex lymphatic anomalies. This list is not intended to replace consultation with the patient’s physicians regarding the use of these medications and is not an exhaustive list. The inclusion of a drug on this list does not in any way imply endorsement by the LGDA. As always, we recommend you consult with your personal physician regarding all decisions regarding treatments for your particular diagnosis.

 

alendronate (a len’ droe nate)

Drug Class: bisphosphonate

Administration route: oral in tablet or liquid form

Brand names: Binosto®, Fosamax®

 

interferon alfa-2b

Drug Class: antineoplastic

Administration route: injection

Brand names: Intron A®

 

octreotide (ok – tree’ – oh – tide)

Drug Class: Endocrine-Metabolic Agent

Administration route: injection or IV

Brand names: Sandostatin®, Sandostatin® LAR Depot

 

pamidronate (pa – mi – droe’ – nate)

Drug Class: calcium regulator

Administration route: IV

Brand names: Aredia®

Other names: ADP Sodium, AHPrBP Sodium

 

Prednisone (pred’ ni sone)

Drug Class: coricosteroid

Administration route: oral

Brand names: Prednisone Intensol, Sterapred® , Sterapred® DS

 

sirolimus (pronounced: sir oh’ li mus)

Drug class: immunosuppresant

Administration routes: oral in tablet or liquid form

Brand name: Rapamune®

Other names: Rapamycin

 

Thalidomide (tha li’ doe mide)

Drug Class: antineoplastic agent, leprostatic

Administration route: oral

Brand names: Thalomid®

 

vincristine (vin – kris’ – teen)

Drug Class: antineoplastic agent

Administration route: IV

Brand names: Vincasar® PFS

Other Names: Leurocristine Sulfate, LCR, VCR

 

zoledronic acid (pronounced: zoe’ le dron ik)

Drug Class: bisphosphonate

Administration route: IV

Brand names: Reclast® , Zometa®

Supplemental Medications

Total Parenteral Nutrition (TPN)

 

MCT Oil (medium chain triglycerides)

Drug Class: medical food

Administration route: oral liquid

 

Oxygen

 
These links and the information therein are not intended to replace consultation with your professional health care team, but as a supplement to that relationship. The LGDA does not endorse the use of any of these medications or warrant any information provided by the sites linked to, nor is it compensated for the inclusion of any given drug on this list.