Unfortunately there is no cure for LAM as yet. However there is now evidence to show that one drug (sirolimus or Rapamycin) can slow the rate of decline in lung function and reduce the size of the kidney tumours. Other treatment may be given to deal with particular symptoms or complications (supportive treatment).
- Treatment to prevent LAM progressing: Recently, a number of studies have shown that the drug Rapamycin (sirolimus) can slow the rate of loss of lung function and shrink kidney tumours in women with LAM. However, use of Rapamycin and similar drugs for LAM is still new and a number of important questions remain, including what is the safest effective dose, when should we start treatment and what is the best way to monitor treatment? Rapamycin commonly causes side effects and needs careful monitoring and it is not appropriate for all those with LAM. At present Rapamycin is mainly prescribed for women with advanced or rapidly progressive disease and those with chylous pleural effusions. In the past, various hormone treatments have been tried for LAM but because LAM is rare they have not been studied in the normal way i.e. by comparing treatment with a dummy treatment (placebo) in a controlled trial. These treatments, such as progesterone, tamoxifen and hormones that reduce the release of oestrogen, are used less often now since the evidence available does not suggest that they are useful.
- Supportive treatment: For breathlessness. Treatment for breathlessness depends on the cause of the breathlessness. For example, if there is a pneumothorax or a pleural effusion treating these should help the breathlessness. Some women with LAM benefit from the ß agonist inhalers used for asthma such as Ventolin and Bricanyl.
- Fluid on the chest (pleural effusion). If this is large it may help to remove the fluid, though in the long term it is better to prevent it accumulating. This may be helped by Rapamycin or, if that isn’t possible, by a low fat diet or by progesterone treatment (see below). If it continues to build up an operation may be needed to stick the outside of the lung to the inside of the ribcage. This is known as a pleurodesis and is usually carried out with a general anaesthetic.
- Pneumothorax. This is usually treated initially by sucking the air out of the space around the lung with a needle or tube inserted under a local anaesthetic. If it recurs it may also be treated by a pleurodesis so that the lung can’t collapse again.
- Oxygen. When breathlessness becomes more troublesome breathing additional oxygen may help. Oxygen can be given from oxygen cylinders or from a machine called a concentrator which extracts oxygen from the air. Having a concentrator is more convenient if you need oxygen for several hours a day and means you don’t need to keep replacing oxygen cylinders which may only last for 6 hours. Portable oxygen systems are also available. There are no hard and fast rules as to when oxygen should be started but patients who have to stop after walking 100 to 200 yards are likely to benefit.
- Lung transplant. Lung transplantation is a possibility for those with severe LAM. More than 100 women with LAM have had a lung transplant worldwide and overall the outcome in those with LAM appears to be at least as good as the outcome in people who have had a lung transplant for other conditions. A lung transplant is a major undertaking, however, and the results are not yet as good as those for a kidney transplant. It is only considered therefore when LAM has become very severe.
- Treatment of kidney tumours: Some women with LAM have a benign tumour in the kidney (called an angiomyolipoma). Most kidney tumours in LAM are small, do not cause symptoms and do not need treatment as they are benign. Women with LAM should have a kidney scan to see if angiomyolipomas are present, and if they are present, they should be monitored to ensure they are not growing. Occasionally larger tumours cause pain or bleeding and may need to be treated. This is done either by removing the tumour with an operation or blocking its blood supply (embolisation) which causes it to shrink. Embolisation is done through a catheter inserted into the artery to the kidney and it does not normally need a general anaesthetic. Patients recover more quickly from embolisation. It is not possible to treat all tumours in this way, however, and the procedure is not performed in all hospitals. The aim of treatment is to preserve as much normal kidney as possible, and avoid removing a kidney unless there is no alternative.