Treatments & Interventions

While there is currently no cure for LAM, clinicians and patients have access to therapies and diagnostic tests that improve quality of life and reduce or delay the need for invasive surgical interventions. Many of these advances have occurred in the last 15 years, thanks to the work of The LAM Foundation and a global network of dedicated scientists and clinicians.

Many patients will benefit from sirolimus, the FDA approved treatment for LAM. Others will be faced with decisions about additional medical and surgical interventions depending on the progression of their disease. Below is useful information about how common complications of LAM are treated.

Medications

RAPAMUNE^® (sirolimus)

Sirolimus (also known as rapamycin, brand name Rapamune) is the first-line treatment option for managing symptoms and complications of LAM. The goal of this therapy is to stabilize or slow the rate of lung function decline, address substantial disease burden, as well as complications such as lung collapses (spontaneous pneumothorax), kidney tumors (renal angiomyolipomas) and accumulation of fluid around the lungs and in the abdomen (lymphatic manifestations such as chylous effusions).

RAPAMUNE^® (sirolimus) was the first drug to be approved by the FDA for the treatment of LAM. It is a highly specific inhibitor of the mTOR signaling pathway that is dysregulated in LAM cells. Treatment with the drug suppresses the growth of LAM cells. The efficacy of sirolimus was proven by the Multicenter International Safety and Efficacy of Sirolimus in LAM (MILES) trial. Click here to learn more. The MILES trial was a phase 3, double-blind, placebo-controlled randomized trial that showed that treatment with sirolimus can stabilize lung function decline, reduce serum VEGF-D levels, and improve some measures of quality of life in women with moderate to severe LAM. Treatment with sirolimus is suppressive rather than remission-inducing, and durable disease control requires long-term treatment with sirolimus. An ongoing trial is evaluating the role of pre-emptive treatment with sirolimus in patients with preserved lung function.

Afinitor (everolimus)

Afinitor is also used in the treatment of LAM, although it is not FDA-approved for that purpose. Everolimus has been shown to be effective for the treatment of angiomyolipomas, and epilepsy control in patients with TSC. It is a derivative of sirolimus and has very similar functions and side effects.

Patients with suspected LAM or a confirmed diagnosis should be referred to a pulmonary specialist or LAM Clinic with experience using sirolimus for appropriate assessment, treatment, and monitoring.

See Living Well with LAM for additional information about side effects and things to consider when your physician prescribes sirolimus.

Supportive Measures

Inhalers

Many women with LAM find that bronchodilator inhalers such as albuterol help to reduce breathlessness. Longer-acting bronchodilators are often prescribed. Some inhalers contain two long-acting bronchodilator drugs with different mechanisms of action; one benefit is that such inhalers only need to be taken once a day.

Supplemental Oxygen

When breathlessness becomes more troublesome or oxygen levels fall upon exertion or while sleeping, supplemental oxygen may be prescribed. Supplemental oxygen can be delivered from oxygen cylinders or from a machine called a portable oxygen concentrator (POC), which extracts oxygen from the air. Small, easily portable oxygen systems are also available which help people remain active.

TREATMENT FOR Renal Angiomyolipomas

Kidney tumors, known as renal angiomyolipomas or AMLs) occur in about one-third of patients with sporadic LAM and the majority (80-90%) of patients with TSC-LAM. The most serious complication is the risk of bleeding which is increased in larger tumors (≥4cm).

Treatment with mTOR inhibitors (sirolimus) can shrink kidney tumors associated with LAM and should be considered for patients with large (>3-4cm) or growing tumors. Embolization can also be performed to reduce bleeding risk, especially in patients with large aneurysmal burden or in patients where renal AML would otherwise be the sole indication to treat with mTOR inhibitors.

Surgical resection should be the last resort for the management of AMLs and should generally be undertaken with nephron-sparing approaches rather than a complete nephrectomy.

TREATMENT FOR pneumothoraces

Pleurodesis

Pleurodesis is a procedure that adheres the outside of the lung to the inside of the chest cavity to prevent the lung from collapsing. If you have had a pneumothorax previously and/or have a persistent leak, your doctor may ask you to consider pleurodesis as a preventative measure. Pleurodesis is usually recommended after the first incidence of lung collapse in women with LAM because patients who have one lung collapse are likely to have additional lung collapses.

Although pleurodesis isn’t a foolproof remedy against future pneumothoraces, it generally diminishes their likelihood. Also, if you have a pneumothorax after you’ve had pleurodesis, the collapse is more likely to be partial, and intervention may not be required. The pleurodesis procedure may be done by a number of different techniques.

Mechanical Pleurodesis

A mechanical pleurodesis is performed when a surgeon manually strokes the pleura (membrane enveloping the lung) with a piece of gauze. Mechanical pleurodesis roughens up the pleura so that when the abrasion heals, the lung adheres to the chest wall. Sometimes the procedure is done in combination with some type of chemical pleurodesis.

Chemical Pleurodesis

Chemical pleurodesis involves instilling a chemical irritant into the pleural space which causes adhesion between the chest wall and the lining of the lung. Chemical pleurodesis can be done either through a chest tube while you’re awake in a hospital room or it can be done under general anesthesia via surgery. Sometimes a combination of chemical and mechanical pleurodesis is used.

One of the most common methods of chemical pleurodesis is performed using surgical talc and a chest tube. If a patient already has a chest tube in place, a talc procedure is often recommended. Once the air has been removed from the chest cavity, a talc slurry is instilled through the chest tube into the chest cavity. Alternatively, during surgery talc can be blown into the chest cavity using a bulb syringe, a method called talc poudrage. Suction via a chest tube is used after this procedure to remove any remaining air in the pleural space. Talc pleurodesis or poudrage often produces a burning sensation in the chest as the area heals, but this procedure tends to be highly effective. In addition to talc, other chemical irritants such as doxycycline can be used.

Pleurectomy

If pleurodesis does not work or if there are recurrent pneumothoraces, a patient may be a candidate for a pleurectomy. This surgery involves stripping off pleura from the inside of the chest wall to promote the fusion of the lung and the chest wall.

To perform the procedures mentioned above, most doctors use one of the following two procedures to gain access to your lungs:

Thoracoscopy or VATS

Video-assisted Thorascopic Surgery (VATS) is performed using a tiny fiber-optic camera (called a thorascope) and is a much less invasive procedure than a thoracotomy. Instead of using one large incision to gain access to the chest, one or more small finger-sized incisions, or ports, are made in the side and used to insert the scope and other surgical instruments. The thorascope transmits images of the inside of your chest onto a video monitor, guiding the surgeon in maneuvering the instruments to complete the procedure. These smaller incisions, unlike a larger surgical incision, typically result in less pain and faster recovery time. For these reasons, VATS has become the preferred method for surgical lung biopsies and other lung surgeries.

Thoracotomy

Thoracotomy is a general term for open-chest surgery. While the patient is under general anesthesia, the surgeon makes an incision that runs approximately from the front to the back (by the shoulder blades) of the chest in between two ribs. This incision allows the doctor access to the lung. A thoracotomy is used for mechanical pleurodesis, for some chemical pleurodesis procedures and for pleurectomy.

A patient usually requires about a weeklong stay in the hospital after a thoracotomy, but full recovery can take several months. During recovery, the patient should practice deep-breathing techniques to help prevent pneumonia. As with any surgery, bleeding and infection are always risks.

Recovery From Pleurodesis

Proper pain management is important following any of these procedures. The doctor may prescribe pain medication to help reduce the pain. It is important to stay proactive with your pain management because once pain becomes intense, it can be difficult to manage.

Pain and discomfort with these procedures will likely be intense for the first few days but will generally decrease after several weeks of recovery. It is also normal to experience shortness of breath and fatigue following any of these procedures. Resting during this time of healing is extremely important. It may be necessary to take time off work and be restricted from driving and other activities for a period of time.

Once the healing is complete, there are usually no residual effects. Your breathing, however, may feel strange at first, as if something has changed. Unfortunately, some discomfort or a pulling sensation in the chest area can continue for months after pleurodesis.

Transplant After Pleurodesis

Many women with LAM have undergone successful lung transplants after experiencing pleurodesis. While there is a risk of bleeding complications for patients who had pleurodesis prior to lung transplantation surgery, studies have shown that these complications do not have any adverse effects on the outcome of the transplant. Experts recommend that a pleurodesis procedure is performed for women with LAM after their first episode of pneumothorax.

Lung Transplantation

Although LAM develops differently in each patient, a number of women have aggressive, fast-growing disease. Some eventually need oxygen therapy. In time, even extra oxygen may not improve a patient’s condition and she may need to consider a lung transplant.

A lung transplant is a surgery to replace one or both of the diseased lungs with healthy lungs from a human donor. This is usually considered the last resort for irreversible lung failure.

There are strict guidelines to determine which patients are eligible for transplantation. A national waiting list run by the United Network for Organ Sharing (UNOS) matches donors to potential recipients based on shared blood type, lung size, the severity of the patient’s disease, and likelihood that a new lung will improve the patient’s survival.

Benefits versus Risks of Lung Transplantation

Choosing to have a lung transplant is a major decision. It is important to educate oneself about the risks and benefits of this life-changing and potentially life-saving procedure. While lung transplantation will not treat the underlying causes of LAM, it holds the promise of giving patients longer and improved quality of life.

Lung transplantation has the same risks as any other major surgery. These may include major bleeding, pneumonia, fluid in the lungs and painful recovery. In addition, transplant patients may reject the new lung(s) and be vulnerable to infection. Other potential problems include blood clots, side effects to medications, and an increased risk of certain cancers.

Rejection

The body’s immune system will identify the new lung as a foreign invader and may try to attack them just as it would the flu or other viruses. To prevent this response, patients are given immunosuppressive (anti-rejection) medications. Rejection of the donated organ is most likely to occur within the first three months. Symptoms of lung rejection may include fever, chills, flu-like aches, shortness of breath, and infections. Patients taking immunosuppressive medications following transplantation are less able to fight off infections and viruses. Because the body’s natural ability to fight off infections is reduced, it is critical to report any signs of infection to your treating physician right away. Regular follow-up visits, breathing tests, X-rays, and other tests are needed to keep an eye on how the body is responding to, and healing from the transplant.

Tissue Donation

LAM patients who undergo transplant can help LAM researchers and scientists become more knowledgeable about LAM by donating lung tissue for LAM-related studies. Access to fresh tissue is especially important to scientists with open protocols and requires coordination many months in advance. A patient facing a procedure in which tissue will be removed may be able to donate those tissue samples to our LAM scientists for research. Please contact the LAM Foundation for more information.