Mastocytosis

Treatment

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 meansthe manufacturers haven't applied for a licence for their medication to be used to treat mastocytosis.

In other words, the medication hasn't undergone Research and 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 andthe benefits of treatment outweigh any associated risk.

Steroid cream

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 creamreduces the number of mast cells that can release histamine and trigger inflammation inside the skin.

Side effects ofsteroid creamwhen 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 lesionsto reduce the risk of side effects.

Antihistamines

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 totreat allergic conditions.

Side effects of some "classical" antihistamines include:

headache
dry mouth
dry nose

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

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 meansit 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.

PUVA

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 helpsreduce lesions inthe skin.

You can only have a limited number of PUVA sessions as using the treatment too many times around 150 sessionsmay increase your risk of developing skin cancer over your lifetime.

Steroid tablets

If symptoms such as itchiness are particularly severe, corticosteroid tablets (oral corticosteroids) may be prescribed on a short-term basis. However, this is rare.

Ashort course of corticosteroid tablets may be recommended if you have bone paincaused bymastocytosis, ora severe allergic reaction (anaphylaxis) .

Side effects of oral corticosteroids used on a short-term basis include:

an increase in appetite
weight gain
insomnia
fluid retention
mood changes, such as feeling irritable or anxious

Bisphosphonates and calcium supplements

If you haveweakened bones (osteoporosis) resulting from abnormal mast cellactivity 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.

H2-receptor antagonists

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.

It's not known exactly why this is, butit 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

Imatinibis 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)
headache
aching muscles
diarrhoea
tiredness

Nilotinib and dasatinib

Nilotinibordasatinib 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

Cladribinewas originally designed to treatcancer of the white blood cells (leukaemia), but it's also been shown to be usefulin 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 treatmentsfor 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 lymphoma

Treating multiple myeloma

Myeloproliferative neoplasms arealso haematological disorders that can be associated with mastocytosis.

Using an adrenaline injection pen

Because ofyour 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: