The treatment options for mastocytosis depend on which type you have and how severe your symptoms are.
Nearly all medications used to treat mastocytosis are unlicensed. This means the manufacturers haven't applied for a licence for their medication to be used to treat mastocytosis.
In other words, the medication hasn't undergone clinical trials to see if it can treat mastocytosis effectively and safely.
Many experts will use an unlicensed medication if they think it will be effective and the benefits of treatment outweigh any associated risk.
Mild to moderate cases of cutaneous mastocytosis can be treated with a very strong steroid cream (topical corticosteroids) for a limited length of time, usually up to six weeks.
Steroid cream reduces the number of mast cells that can release histamine and trigger inflammation inside the skin.
Side effects of steroid cream when used too much include:
- thinning of the skin, which can sometimes result in permanent stretch marks
- a temporary reduction in the pigmentation of the skin
- the affected area of skin bruising easily
You should only apply the cream to areas of skin affected by lesions to reduce the risk of side effects.
Antihistamines can also be used to treat symptoms of cutaneous or indolent mastocytosis, such as itchiness and skin redness.
Antihistamines are a type of medication that block the effects of histamine. They're widely used to treat allergic conditions.
Side effects of some "classical" antihistamines include:
However, these side effects should pass quickly once you're used to the medication. Modern second generation antihistamines don't usually cause these side effects.
Sodium cromoglicate is a medication used to treat conjunctivitis, asthma and food allergy. It may also be used to treat gut symptoms of mastocytosis, but isn't absorbed well from the bowel.
Sodium cromoglicate is a mast cell stabiliser, which means it reduces the amount of chemicals released by the mast cells. This helps relieve symptoms such as diarrhoea.
A case report published in 2010 describes a patient with mastocytosis whose symptoms improved after taking antihistamines and sodium cromoglicate capsules.
Their bone pain, fatigue and headache improved further after also taking inhaled sodium cromoglicate.
Nausea and joint pain have been reported in some people taking sodium cromoglicate.
A form of sodium cromoglicate applied to the skin (topical) is available that may help with itching. However, it's not routinely available on prescription.
More severe symptoms of cutaneous mastocytosis, such as severe itchy skin, may require a type of treatment called psoralen plus ultraviolet A (PUVA).
PUVA involves taking a medication called psoralen, which makes the skin more sensitive to the effects of ultraviolet light.
The skin is then exposed to a wavelength of light called ultraviolet A (UVA), which helps reduce lesions in the skin.
You can only have a limited number of PUVA sessions as using the treatment too many times – around 150 sessions – may increase your risk of developing skin cancer over your lifetime.
If symptoms such as itchiness are particularly severe, corticosteroid tablets (oral corticosteroids) may be prescribed on a short-term basis. However, this is rare.
A short course of corticosteroid tablets may be recommended if you have bone pain caused by mastocytosis, or a severe allergic reaction (anaphylaxis).
Side effects of oral corticosteroids used on a short-term basis include:
- an increase in appetite
- weight gain
- fluid retention
- mood changes, such as feeling irritable or anxious
Bisphosphonates and calcium supplements
If you have weakened bones (osteoporosis) resulting from abnormal mast cell activity in your bones, you'll be given a type of medication called bisphosphonates.
Bisphosphonates slow the process of bone breakdown while allowing production of new bone to continue as normal, which improves your bone density.
You may also be given calcium supplements, as calcium helps strengthen bones.
Read more about treating osteoporosis.
If you have stomach pain caused by a stomach ulcer (peptic ulcer), you'll be given a medication called an H2-receptor antagonist.
This blocks the effects of histamine in the stomach – histamine stimulates the production of stomach acid, which damages the stomach lining.
Read more about treating a peptic ulcer.
Originally designed to treat cancer, interferon alpha has proved effective in treating some cases of aggressive mastocytosis.
It's not known exactly why this is, but it appears the medication reduces the production of mast cells inside the bone marrow.
Interferon alpha is given by injection. You may have flu-like symptoms, such as chills, a high temperature and joint pain, when you start taking interferon alpha.
However, your symptoms should improve over time as your body gets used to the medicine.
Imatinib is an alternative medication to interferon alpha. It's taken as a tablet and blocks the effects of an enzyme called tyrosine kinase, which helps stimulate production of mast cells.
However, imatinib should only be used for people who don't have the KIT mutation, and it doesn't work for most cases of mastocytosis.
Imatinib can also make you more vulnerable to infection. Contact your GP immediately if you develop possible signs of an infection, such as:
- a high temperature (fever) of or above 38C (100.4F)
- aching muscles
Nilotinib and dasatinib
Nilotinib or dasatinib may be recommended if you don't respond to treatment with imatinib. They work in much the same way, blocking the effects of tyrosine kinase.
The medication will make you more vulnerable to infection, so report possible symptoms of infection to your GP immediately.
Cladribine was originally designed to treat cancer of the white blood cells (leukaemia), but it's also been shown to be useful in treating aggressive systematic mastocytosis. However, cladribine hasn't been approved (licensed) to treat mastocytosis.
Cladribine suppresses the activity of your immune system. It's given by infusion, which means it's slowly released into your body through a drip in your arm over the course of two hours.
Like imatinib, nilotinib and dasatinib, cladribine will also make you more vulnerable to the effects of infection, so you should report possible symptoms of infection to your GP immediately.
Treatments for haematological disease
Systemic mastocytosis with associated blood (haematological) disease will be treated in the same way as aggressive systematic mastocytosis, with a number of additional treatments for the related haematological condition.
For more information about treating the most common haematological conditions, see:
Treating acute myeloid leukaemia
Treating chronic myeloid leukaemia
Treating acute lymphoblastic leukaemia
Treating chronic lymphocytic leukaemia
Treating multiple myeloma
Myeloproliferative neoplasms are also haematological disorders that can be associated with mastocytosis.
Using an adrenaline injection pen
Because of your increased risk of anaphylaxis, you may be given an adrenaline auto-injector to use in an emergency.
Adrenaline is a natural chemical that helps fight the effects of too much histamine, while also relieving breathing difficulties. Each pen contains a single dose of adrenaline – 0.3mg for adults or 0.15mg for children.
There are three types:
These auto-injectors release adrenaline when jabbed or pressed against the outer thigh. The injections can be given through clothing.
If you're given an adrenaline auto-injector, you need to keep an eye on its expiry date because it won't be effective beyond this date.