There is currently no cure for complex lymphatic anomalies (CLAs). Treatment focuses on controlling symptoms, preventing serious complications, and improving overall quality of life.
Because CLAs can affect multiple organ systems — including bones, lungs, chest cavity, abdomen, and lymphatic flow — care is typically coordinated through a multidisciplinary team.
This team may include specialists in vascular anomalies, interventional radiology, pulmonology, cardiology, surgery, nutrition, genetics, and pain management.
Sclerotherapy
Sclerotherapy is a minimally invasive procedure in which fluid is drained from a lymphatic cyst and a medication (called a sclerosant) is injected to irritate the cyst lining. This causes the cyst to shrink or close. Most commonly requires multiple treatments and not everyone responds.
Surgery
Surgery (sometimes called debulking) may be used to reduce the size of lymphatic cyst and relieve symptoms such as pressure on the airway or organs, pain, difficulty breathing, or repeated infections.
Because the cysts often involve multiple tissue layers and may surround important structures—such as nerves, blood vessels, or organs—surgery is not always recommended or possible. Even when surgery is performed, some abnormal lymphatic tissue often remains, and regrowth often occurs over time.
Medications
Medications may be used to help reduce abnormal lymphatic growth, inflammation, or complications. Examples include:
- Sirolimus (oral or topical)
Helps calm an overactive cell-growth pathway (mTOR), which may reduce lymphatic vessel growth and inflammation. Topical sirolimus may help skin-based lesions that bleed or leak easily. - Alpelisib
Targets the PI3K protein, which is overactive in people with PIK3CA gene mutations. It may be used in selected patients to reduce abnormal tissue growth and related complications. - Trametinib
Targets the MEK pathway, which is overactive in some people with MAPK-pathway gene mutations (such as KRAS or BRAF). It may be used in selected patients to help reduce abnormal lymphatic growth and related symptoms. - Bisphosphonates
Medications that help slow bone breakdown by reducing osteoclast activity and may be used when bone involvement is present.
Important note:
This is not a complete list of all possible medications. Treatment plans are individualized, and other medications may be used based on diagnosis, symptoms, overall health, and response to previous therapies.
Nutrition
Nutrition plays an important role, particularly for patients with chyle leaks.
- Low-fat diets, often supplemented with medium-chain triglycerides (MCTs), can reduce fat flow through the lymphatic system and decrease chyle production.
- These diets must be closely supervised by a healthcare provider and dietitian to avoid vitamin and mineral deficiencies.
Additional considerations:
- A low-carbohydrate diet may be recommended for patients taking sirolimus or alpelisib, as these medications can raise blood sugar levels.
- Patients with CLA-related bone involvement may benefit from vitamin D and calcium supplementation, under medical guidance.
Additional Supportive Options
Other treatments and supportive strategies that may be helpful include:
- Compression garments (sleeves, stockings, or bandages) to help manage swelling
- Manual lymphatic drainage therapy to support lymphatic flow
- Laser therapy or radiofrequency ablation for CLA patients with microcystic lymphatic malformations
- Clinical trials, which may offer access to new and emerging therapies
