Lymphatic malformation treatment options

  • Surgical treatment of lymphatic malformations

    Lymphatic malformations (LMs) are congenital vascular anomalies characterized by dilated lymphatic channels or cystic spaces. Present at birth, or manifesting in early childhood, they often appear as soft, compressible, non-pulsatile masses and most commonly affect the head and neck region. Although slow-growing and non-neoplastic, LMs can cause significant functional, aesthetic, and psychosocial challenges, especially when extensive or recurrent.

    While sclerotherapy is considered first-line therapy for most macrocystic LMs, surgery remains an essential part of treatment, especially for lesions refractory to sclerotherapy, those causing airway compromise, or for residual deformities..

     

    Indications for Surgical Treatment

    Surgery remains a cornerstone in the multidisciplinary management of lymphatic malformations (LMs). It is generally implemented in specific, well-selected cases of lymphatic malformation, often in combination with other treatment modalities. Indications include:

    1. Failure of Sclerotherapy

    • When lesions do not respond to multiple rounds of sclerotherapy, especially in macrocystic or mixed LMs, surgery may be necessary to remove persistent cystic components.

    2. Functional Impairment

    • LMs that affect airway, vision, swallowing, or speech — especially in the oral cavity, parotid, or submandibular space — often require partial or complete excision to preserve vital functions.

    3. Infection or Recurrent Inflammation

    • In cases of recurrent cellulitis, abscess formation, or intralesional hemorrhage, surgical excision of the inflamed segment may prevent repeated hospitalizations and improve quality of life.

    4. Disfiguring or Bulky Lesions

    • Surgery can be considered to improve cosmetic appearance in patients with residual facial or neck deformities, especially after stabilization or partial regression from prior treatment.

    5. Localized and Accessible Lesions

    • When an LM is well-circumscribed, superficial, and anatomically separable from critical structures, surgery offers the potential for complete removal and cure.

    6. Diagnostic Uncertainty or Suspicion of Malignancy

    • In rare cases where the diagnosis is uncertain or the lesion does not behave as expected, excisional biopsy may be indicated.

    Preoperative Evaluation

    Before surgery is planned, a comprehensive assessment is critical:

    a. Imaging

    • MRI with contrast is the imaging modality of choice.

      • Identifies extent, cystic architecture (macro- vs. microcystic), and relation to vital structures.

    • Ultrasound may be helpful in younger children for superficial lesions or as a preoperative mapping tool.

    b. Clinical Classification

    • LMs are typically classified into:

      • Macrocystic: Cysts >2 cm

      • Microcystic: Cysts <2 cm, spongy, small vessels, often infiltrative

      • Mixed-type: Combination of both

    • Macrocystic lesions are more amenable to surgical resection.

    c. Multidisciplinary Assessment

    • Include specialists in ENT, Maxillofacial, Plastic Surgery, Interventional Radiology and Hematology/Oncology.

    • Anesthesia and airway assessment in large cervicofacial lesions, especially in infants.

     

    2. Surgical Strategies by Lesion Type

    a. Macrocystic Lesions

    • These are often localized, well-encapsulated, and displace rather than infiltrate adjacent tissues.

    • Surgical excision may be curative, particularly in the neck, axilla, or buccal region.

    • Risks are generally low, though recurrence can occur if small residual cysts remain.

    b. Microcystic Lesions

    • Tend to be diffuse, infiltrative, and interdigitated with nerves, vessels, and muscles.

    • Complete excision is more challenging.

    • Surgery is typically reserved for debulking, removal of bleeding or infected tissue, or cosmetic contouring.

    c. Mixed-Type Lesions

    • May require staged procedures or a combined approach with sclerotherapy.

    • Strategy often depends on which component (macro- or microcystic) is dominant.

     

    3. Intraoperative Considerations

    Intraoperative Considerations

    Challenge

    Management Strategy

    Bleeding

    LMs have a rich lymphovascular supply, and good hemostasis techniques must be applied. Preoperative sclerotherapy is considered in select cases.

    Dissection near vital structures

    Particularly in the head and neck, nerves (e.g., facial, hypoglossal), salivary glands, and the airway must be preserved.

    Poor tissue planes

    Especially in microcystic lesions, there is a risk of incomplete resection or damage to adjacent structures.

    Postoperative fluid collection

    The use of drains and pressure dressing is often necessary.

     

    4. Pediatric vs. Adult Considerations

    Aspect

    Pediatric Patients

    Adult Patients

    Growth and development

    Surgery must consider facial growth and airway development.

    Growth is less of a concern; focus on function and appearance.

    Anesthesia risk

    Higher in infants, especially with large cervicofacial lesions.

    Typically, more manageable, though comorbidities may exist.

    Psychosocial impact

    Early intervention can prevent bullying or school avoidance.

    Adults often seek treatment for disfigurement or late complications.

    Recurrence risk

    Higher in cases of incomplete resections during infancy.

    May present with late-onset symptoms from residual LMs.

    Surgical management of lymphatic malformations (LMs) can offer meaningful improvements in function and appearance. However, due to the infiltrative nature of many lesions — especially microcystic and mixed types — outcomes can vary significantly. It is essential for patients and their families to understand the expected results, potential complications, and long-term quality-of-life implications.

     

    1. Surgical Outcomes

    a. Macrocystic Lesions

    • High success rate with complete or near-complete excision.

    • Often result in excellent functional and aesthetic outcomes when well-circumscribed.

    • Recurrence is low, particularly when residual microscopic disease is avoided.

    b. Microcystic and Mixed Lesions

    • Surgical excision is rarely curative.

    • Partial debulking may relieve:

      • Recurrent bleeding

      • Functional limitation (e.g., speech, mastication)

      • Disfigurement or mass effect

    • Outcomes often improve when surgery is used in conjunction with sclerotherapy or laser.

    c. Airway or Tongue Involvement

    • Surgery may improve breathing, feeding, or articulation, but carries the risk of postoperative edema or scarring.

    • Staged or multimodal interventions are often necessary in this region.

    Impact on Quality of Life

    Positive Outcomes

    • Improved appearance in the face or neck can significantly enhance social confidence, especially in children and adolescents.

    • Relief from functional symptoms (e.g., tongue bulk, drooling, limited movement) can improve eating, speech, and overall comfort.

    • Reduced frequency of infections or inflammatory episodes following excision of affected segments.

    Psychosocial Dimensions

    • Children with visible lesions often face stigma, bullying, or isolation, particularly in school.

    • Adults may experience social anxiety, embarrassment, or avoidance of personal relationships.

    • Surgery, even if it is partial, can be a turning point in improving self-esteem and facilitating social reintegration.

    Residual Concerns

    • Visible scars or asymmetries may still affect self-image, especially in facial lesions.

    • Recurrence can cause emotional fatigue, especially after multiple procedures.

    • Some patients may need ongoing psychological support, speech therapy, or aesthetic revision surgeries.

    Lymphatic Malformation Subtypes: Surgical Responsiveness

    Subtype: Macrocystic LM

    Surgical Responsiveness: ⭐⭐⭐⭐☆ (High)

    Surgical Role: Often well-circumscribed and excisable; may be curative in localized lesions.

    Alternative/Adjunct Treatments: Sclerotherapy (first-line or preoperative adjunct)

    Subtype: Microcystic LM

    ⭐⭐⭐☆☆ (Moderate)

    Surgical Role: Infiltrative; surgery limited to debulking, removal of infected/bleeding segments

    Alternative/Adjunct Treatments: Laser (for superficial mucosa), Sirolimus (systemic)


    Subtype: Mixed-type LM

    Surgical Responsiveness: ⭐⭐⭐☆☆ (Moderate)

    Surgical Role: Surgery is often staged or combined with sclerotherapy

    Alternative/Adjunct Treatments: Sclerotherapy + surgery; laser for superficial disease


    Subtype: Cervicofacial LM

    Surgical Responsiveness: ⭐⭐⭐☆☆ (Moderate)

    Surgical Role: Surgery for airway, swallowing, or disfigurement; high risk of nerve injury

    Alternative/Adjunct Treatments: Sclerotherapy, staged surgery, tracheostomy (if needed)


    Subtype: Intraoral / Tongue LM

    Surgical Responsiveness: ⭐⭐⭐☆☆ (Moderate)

    Surgical Role: Limited by anatomy and function, debulking may aid speech or feeding

    Alternative/Adjunct Treatments: CO₂ laser, sclerotherapy, Sirolimus


    Subtype: Axillary LM

    Surgical Responsiveness: ⭐⭐⭐⭐☆ (High)

    Surgical Role: Often amenable to complete or subtotal resection

    Sclerotherapy (first-line for large cysts)


    Subtype: Orbital/Periorbital LM

    Surgical Responsiveness: ⭐⭐⭐☆☆ (Moderate)

    Surgical Role: Surgery if vision is threatened, or if there is a bulging eye, high risk of scarring or recurrence

    Alternative/Adjunct Treatments: Sclerotherapy under image guidance


    Subtype: Retroperitoneal/Deep LM

    Surgical Responsiveness: ⭐⭐☆☆☆ (Low–Moderate)

    Surgical Role: Difficult access; surgery for mass effect or hemorrhage

    Image-guided sclerotherapy


    Subtype: Recurrent infected LM

    Surgical Responsiveness: ⭐⭐⭐☆☆ (Moderate)

    Surgical Role: Surgery to remove chronically inflamed or abscessed areas

    Alternative/Adjunct Treatments: Antibiotics, drainage, Sirolimus for control

    Always consult a vascular anomalies team for assessment and coordinated multimodal planning.

  • Sclerotherapy

    Sclerotherapy is the method of choice in the majority of lymphatic malformations. It is a minimally invasive method with high rates of clinical response and low complication rate.

    This method involves the percutaneous puncture of the vascular malformation and the injection of special pharmaceutical agents such as Doxycycline, Bleomycin, or Ethanol aiming for the permanent destruction of the abnormal lymphatic vessels. The selection of the most suitable sclerotic agent is made by the medical specialist for optimal results.

    Sclerotherapy is ideally performed by specialized interventional radiologists with the aid of an angiography unit and ultrasound machine.


    Bleomycin Electrosclerotherapy (BEST)

    Bleomycin electrosclerotherapy is the latest development in the treatment of vascular malformations.

    This new treatment modality is being used since 2019 in a few specialized vascular malformation centers. Our multidisciplinary group of clinicians was one of the first worldwide to apply electrosclerotherapy for the treatment of vascular malformations and has already treated a large number of patients with this modality.

    This method is mainly indicated for the treatment of low-flow vascular malformations (venous and lymphatic malformations) as well as selected cases of arteriovenous malformations.

    Our clinical experience so far has shown that this method significantly reduces the number of treatment sessions required in order to achieve a good clinical result.
    With electrosclerotherapy, the effectiveness of sclerotherapy is increased while at the same time the dose of the administered sclerotic agent (Bleomycin) can be significantly reduced.

    The procedure is performed under general anesthesia. First, the sclerosing agent is administered either directly into the vascular malformation, or intravenously. Subsequently, thin needles are placed into the vascular malformation, usually under ultrasonographic and fluoroscopic guidance. These needles are connected to the electric pulse generator. By applying short electric pulses, the permeability of the cell membrane of the cells that form the wall of the vascular malformation is increased, resulting in a dramatic increase in the intracellular concentration of Bleomycin.

  • Laser therapy in lymphatic malformations

    Laser therapy is a valuable adjunct in the management of superficial, mucosal, and microcystic lymphatic malformations, particularly when lesions are not amenable to surgical or sclerotherapy treatment. While not curative, lasers can significantly improve symptoms, reduce bleeding or lymphorrhea (a lymphatic fluid leak), and enhance cosmetic outcomes, especially in oral, facial, and cutaneous les

    Depending on the laser type used, Laser therapy works through tissue ablation or selective photothermolysis, in which specific wavelengths of laser energy target fluid-filled vesicles or abnormal lymphatic channels.

     

    Commonly Used Lasers for Lymphatic Malformations

    Laser Type: CO₂ Laser

    Wavelength: 10,600 nm

     Primary Use in LMs: Ablation of superficial mucosal or cutaneous vesicles

    Target Tissue: Water (used for vaporization)

    Laser Type: Nd:YAG Laser

    Wavelength: 1064 nm

    Primary Use in LMs: Coagulation of deeper microcysts or bleeding sites

    Target Tissue: Hemoglobin, deeper penetration

    Clinical Indications for Laser Use in LMs

    Laser therapy is best suited for adjunctive treatment or palliative, rather than definitive cure. Indications include:

    Superficial Microcystic Lesions

    • Especially in the oral cavity, tongue, lips, and face

    • Reduces vesicle size, oozing, bleeding, and local discomfort

    Bleeding or Lymphorrhea

    • Lasers can seal lymphatic leaks and control hemorrhagic vesicles

    Recurrent mucosal disease

    • Used to maintain patency or reduce regrowth in previously treated mucosal sites

    Cosmetic Refinement

    • May improve skin texture, reduce vesicle visibility, or correct residual discoloration

    Efficacy and Limitations

    Benefits

    • Minimally invasive

    • Can be repeated as needed

    • Useful in anatomically sensitive or surgically inaccessible areas

    • Particularly effective in children and young adults with mucosal LMs

     Limitations

    • Not curative, especially for deep or extensive lesions

    • Requires multiple treatment sessions

    • Risk of scarring, pigment changes, or mucosal contracture

    • Operator-dependent outcomes

    • Less effective for macrocystic or deep-tissue malformations

    Laser therapy provides an important non-surgical option for treating superficial and microcystic lymphatic malformations, particularly in cases involving mucosal and facial disease. It is most effective when integrated with other treatments, such as:

    • Sclerotherapy: to manage deeper components

    • Surgery: to debulk or correct structural distortion

    • Pharmacologic treatment (e.g., sirolimus): to suppress lesion activity in diffuse disease.

  • Lymphatic malformations
    Science has made great leaps and has led to many discoveries of the pathways that affect the growth and development of vascular tumors and vascular anomalies.  One of the important discoveries is that some of these pathways are the same as the ones used by some cancers.  In recent years, specialists have been repurposing the use of medications that target the abnormal pathway to treat patients with vascular anomalies.  Since most of these medications are new and are still actively studied, they are mostly used “off-label”, meaning not officially approved for this use yet.

    The two main pathways that have been explored are the PI3K/AKT/mTOR and RAS/RAF/ERK/MEK, both of which lead to cell growth.  Mutations (or genetic changes to the genetic make-up of the abnormal cells) of the PI3K/AKT/mTOR pathway tend to be seen in “slow blood flow” anomalies, such as venous or lymphatic malformations.  Common targets (or inhibitors) are against PI3KCA (Alpelisib), AKT (Miransertib), mTOR (Sirolimus). Sirolimus (Rapamune) targets (or inhibits) mTOR and was the first medication tried.  It is not unusual for the pathways to “crosstalk,” and it is always helpful to obtain a biopsy of the affected tissue to identify a mutation.  This information helps reinforce the diagnosis and may offer an additional treatment option.

    The main advantage of targeted medicinal treatment is that the entire vascular anomaly is treated at the microscopic level, without affecting normal tissue.  Most medications are taken by mouth, and many patients notice rapid improvement to pain, bleeding, and loss of function.  These medicines also tend to allow for less bleeding during surgery.  Experience so far indicates that using PI3KCA inhibitors may not only improve the vascular malformation but also the amount of overgrowth, which is often a source of complications in patients with Klippel-Trenaunay Syndrome or other PI3KCA Related Overgrowth Syndromes.  As with any medicine, side effects may occur, which include elevated blood sugar levels and growth delay with Alpelisib, although they tend to be self-limited with close monitoring and supportive care.  Sirolimus side effects include effects on the immune system and mild increase in risk of infection, elevated triglycerides, which are managed and not permanent.  We will discuss with you in detail the possible side effects. 

    The decision on which medication, and the best timing of treatment remains individualized.  There are treatment and patient factors that influence whether someone undergoes surgery only, sclerotherapy/ embolization, medication use or a combination of the above.  Your treatment team will review all relevant factors and make a recommendation on the best path.       .