While Mastocytosis and MCAS share some similarities in terms of symptom management, their distinct underlying causes, such as excessive mast cell proliferation and instability in mastocytosis, lead to different treatment approaches. Consequently, not all treatments suitable for Mastocytosis are safe or appropriate for MCAS, and some management strategies used for MCAS, such as rigid dietary restrictions, are also not always necessary or safe for individuals with Mastocytosis.
References to medication categories and possible management methods on TAMS website do not constitute medical advice. The medications categories listed are not exhaustive, and may not be relevant to your individual management. You are urged never pursue a treatment solely because others with your condition (or similar) are known to use it.
Engaging in multiple concurrent therapies without proper medical guidance can have serious and irreversible consequences. Please always consult an informed specialist before starting new therapies and ensure you understand the intended benefits and side effects of any medication before taking it.
Please use common sense when considering treatment options and consult your doctor(s) to determine what is right for your individual situation. Your healthcare provider is your best resource for personalised medical advice and treatment planning.
Treatment for Mastocytosis
This section tells you the treatments that are the standard of care for mastocytosis. “Standard of care” means the best treatments known. When making treatment plan decisions talk to your doctor about any applicable clinical trials. A clinical trial is a research study that tests a new approach to treatment. Doctors want to learn whether the new treatment is safe, effective and possibly better than the standard treatment. Clinical trials can test a new drug, a new combination of standard treatments, or new doses of standard drugs or other treatments. Your doctor can help you consider all your treatment options.
The treatment of mastocytosis depends on the type, the symptoms of the disease, its extent and the person’s overall health. In many cases, different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called multidisciplinary care.
In Australia, multidisciplinary care can be established through the creation of a General Practitioner Management Plan (GPMP) and Team Care Arrangements (TCAs).
For further details, please refer to the Australian Institute of Health and Welfare.
There is no cure for mastocytosis, several treatments can be used to relieve symptoms. Take time to learn about all of your treatment options and be sure to ask questions about things that are unclear. Talk with your health care team about the goals of each treatment from a medical and individual (personal) perspective, ensuring quality of life goals are always supported.
An important part of treating mastocytosis is controlling a person’s symptoms. One important way to do this is to avoid anything that may cause mast cells to release mediators like histamine, one of hundreds of mediators. This may include extreme temperatures, alcohol, emotional stress, insect bites and certain medications. For example, nonsteroidal anti-inflammatory drugs (NSAIDs) should be used with caution because these drugs may trigger the release of histamine and can cause severe reactions (“flares”), or anaphylaxis in some cases.
Doctors may recommend the following treatments to help manage symptoms of mastocytosis.
Antihistamines (H1/H2): Antihistamines can help by alleviating symptoms triggered by the release of histamine, such as itching, welting, flushing, gastrointestinal discomfort, as well as respiratory, sinus and airway symptoms
Epinephrine: People with mastocytosis often carry a syringe of epinephrine, a hormone made by the adrenal glands, in case they need to treat a severe allergic reaction.2, 3
Mast Cell Stabilisers: Designed to inhibit the excessive release of chemicals from mast cells, which may assist with symptoms such as itching, hives, skin flushing and gastrointestinal issues.4
Antileukotriene therapy: Can aid in the management of symptoms such as breathing difficulties, skin rashes, gastrointestinal issues and overall mast cell stability (all mast cell activation symptoms).1 – 3
Steroids (topical): Topical corticosteroids may be used for the treatment of cutaneous disease and may be used to reduce the size of skin lesions. 1
Other therapies: Including Ultraviolet light (UV) for cutaneous symptoms, Aspirin therapy (physician-directed) for flushing, brain fog and bone pain influenced by elevated prostaglandins, corticosteroids (oral), and Interferon and Chemotherapy (typically for advanced disease), and others on a case-by-case basis. 1 – 4, 6 – 7
Before surgery, talk with your health care team about the possible side effects from the specific surgery you will have.
More information about how to prepare for surgery please see The Mast Cell Disease Society (TMS) Emergency Room Protocol. The protocol provides insights on medications often used in a hospital setting which may be considered more or less suitable for individuals with Mast Cell Diseases.
Targeted therapy is a treatment that targets the disease’s specific genes, proteins or the tissue environment unique to mastocytosis that contribute to its growth and survival. This type of treatment blocks the growth of mast cells while limiting damage to healthy cells. 7,9
Recent research studies have discovered more about specific molecular targets and new treatments directed at them.10, 11 To find the most effective treatment for an individual’s unique mastocytosis profile, doctors may run tests to identify the genes, proteins and other relevant factors.3, 8.
Tyrosine Kinase Inhibitors (TKIs) are a type of targeted therapy. For mastocytosis, the target is the unique protein called the c-kit tyrosine kinase receptor. Treatment with TKIs, including dasatinib, midostaurin (recently approved by the U.S. Food and Drug Administration for the treatment of Systemic Mastocytosis), and less commonly imatinib and nilotinib, may be considered for patients with mast cells that have a mutation in the c-kit tyrosine kinase receptor. Patients with more advanced systemic mastocytosis should be tested for this genetic mutation.11, 12
Talk with your health care team about possible targeted therapy, its side effects, and how they can be managed.
Stem cell transplantation/bone marrow transplantation
A stem cell transplant is a medical procedure in which unhealthy bone marrow is replaced by highly specialised cells, called hematopoietic stem cells, that develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the stem cells in the blood that are typically being transplanted, not the actual bone marrow tissue.
Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the results of any previous treatment and the patient’s age and general health.
There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO uses donated stem cells, while AUTO uses the patient’s own stem cells. In both types, the goal is to destroy all unhealthy cells in the marrow, blood, and other parts of the body using high doses of chemotherapy and/or radiation therapy and then allow replacement blood stem cells to create healthy bone marrow.
Stem cell transplantation is not used frequently as a treatment for mastocytosis because treatment results are inconsistent and there are significant risks associated with this treatment approach.
Chemotherapy for mastocytosis-related cancer
Chemotherapy is sometimes recommended if mastocytosis becomes cancerous. Chemotherapy is the use of drugs to destroy cancer cells, usually by ending the cancer cells’ ability to grow and divide. Chemotherapy is given by a medical oncologist, a doctor who specialises in treating cancer with medication, or a haematologist, a doctor who specialises in treating blood disorders.
Systemic chemotherapy gets into the bloodstream to reach cancer cells throughout the body. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle or in a pill or capsule that is swallowed (orally).
A chemotherapy regimen, or schedule, usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs given at the same time.
The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite and diarrhoea. These side effects usually subside after treatment is finished.
The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications.
Getting care for side effects
Mastocytosis treatment often causes side effects. In addition to treatment to manage the condition, an important part of treatment is relieving these side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.
Palliative care is any treatment that focuses on reducing symptoms (see section above) and treatment side effects, improving quality of life, and supporting patients and their families. Any person, regardless of age or type of disease, may receive palliative care. It works best when palliative care is started as early as needed in the treatment process.
People often receive treatment for the disease and treatment at the same time that they receive treatment to ease side effects. In fact, patients who receive both at the same time often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.
Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. Talk with your health care team about the goals of each treatment in your treatment plan.
Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and palliative care options. During and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem so it can be addressed as quickly as possible.
Treatment for Mast Cell Activation Syndrome (MCAS)
MCAS treatment primarily focuses on management of symptoms caused by mast cell activation (MCA) 22. Part of MCAS treatment involves identifying and avoiding various, individual triggers, such as dietary, environmental and stress-related factors. Stress reduction techniques and mental health management are often considered vital for the effective treatment of MCAS.4, 16, 17
MCAS treatment can be complex given the diverse array of symptoms and triggers, and absence of universally accepted testing for diagnosis and monitoring. Consequently, treatment plans are often tailored based on individual responsiveness.18. 19
Achieving effective personalised management is presently further complicated by the limited availability of medications with proven efficacy, or approval, for the treatment of MCAS. A substantial portion of individuals with MCAS also have comorbid conditions that \necessitate other medical treatments, which can blur the lines regarding causation or correlation of symptoms and sides, including changes in pathology.20, 21
Treatment for Hereditary Alpha Tryptasemia (HαT)
As the global medical community has not reached a consensus on the clinical significance of HαT in isolation or in relation to other conditions, there are currently no specific treatments recognised for the management of HαT.
However, TAMS advocates for individuals with clinically identifiable symptoms of Mast Cell Disease to receive appropriate medical evaluations and care, regardless of HαT involvement.
If you consider that you may have HαT please see our HαT page for more information, and speak to your doctor about whether testing is considered appropriate or beneficial in your case.
Managing side effects for mast cell disease treatments
Managing side effects is a critical component of treating individuals with Mast Cell Disease (MCD) as it has the ability to support overall quality of life.
Regular and ongoing open communication between individuals with MCDs and their health care team is critical, as this supports mutual understandings which can provide for a holistic approach, where adjustments to treatment plans address all aspects of an individual’s needs.
Managing side effects aims to alleviate the discomfort and challenges associated with both the condition itself and side effects of the prescribed treatments. The goal is to minimise the impacts of the illness and its treatments on daily functioning. 14 – 15
TAMS wish to acknowledge the American Society of Clinical Oncology and Cancer.Net as the original authors of some of the above information. View original source on Cancer.Net.
- Cardet JC, Akin C, Lee MJ. Mastocytosis: update on pharmacotherapy and future direction. Expert Opin Pharmacother. 2013 Oct;14(15):2033-45.
- Valent P, Akin C, Sperr WR, Horny HP, Arock M, et al. Diagnosis and treatment of systemic mastocytosis: state of the art. British Journal of Haematology, 122: 695-717.https://onlinelibrary.wiley.com/doi/full/10.1046/j.1365-2141.2003.04575.x
- American Academy of Allergy, Asthma & Immunology. Systemic Mastocytosis > Treatment and Management. https://www.aaaai.org/conditions-treatments/related-conditions/systemic-mastocytosis#:~:text=TREATMENT%20%26%20MANAGEMENT
- The Mast Cell Disease Society. Medications to Treat Mast Cell Diseases
- Akin C, Arock M, Valent P. Tyrosine kinase inhibitors for the treatment of indolent systemic mastocytosis: Are we there yet? J Allergy Clin Immunol. 2022 Jun;149(6):1912-1918. https://www.jacionline.org/article/S0091-6749(22)00559-0/fulltext
- Brazzelli V, Grasso V, Manna G, Barbaccia V, Merante S, Boveri E, Borroni G, et al. Indolent systemic mastocytosis treated with narrow-band UVB phototherapy: study of five cases. J Eur Acad Dermatol Venereol. 2012 Apr;26(4):465-9.
- Cancer.Net. Mastocytosis: Treatment Options. June 2012.
- National Institutes of Health – National Centre for Biotechnology Information. KIT proto-oncogene, receptor tyrosine kinase [(human)]. April 2007.
- Metcalfe DD. Targeted Therapy for the Predominant Form of Mastocytosis. Front Med (Lausanne). July 2017 20;4:110. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9229771/
- Sciumè M, De Magistris C, Galli N, Ferretti E, et al. Target Therapies for Systemic Mastocytosis: An Update. Jun 2022 11;15(6):738.
- Monaldi C, De Santis S, Mancini M, Bruno S, Cavo M. Systemic Mastocytosis: Molecular Landscape and Implications for Treatment. Front Med (Lausanne). Jul 2017. 1;13(1):e2021046. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8265368/
- Akin C, Metcalfe DD. The biology of Kit in disease and the application of pharmacogenetics. J Allergy Clin Immunol. 2004;114(1):13-20.
- Ustun C, Arock M, Kluin-Nelemans HC, Reiter A, et al. Advanced systemic mastocytosis: from molecular and genetic progress to clinical practice. Haematologica. 2016;101(10):1133-1143.https://haematologica.org/article/view/7840.
- Siebenhaar F, von Tschirnhaus E, Hartmann K, Rabenhorst A, Staubach P, et al. Development and validation of the mastocytosis quality of life questionnaire: MC-QoL. 2016 Allergy, 71(6), 869–877. https://onlinelibrary.wiley.com/doi/10.1111/all.12842
- Schmidt TJ, Sellin J, Molderings GJ, Conrad R, Mücke M, et al.. Health-related quality of life and health literacy in patients with systemic mastocytosis and mast cell activation syndrome. Orphanet journal of rare diseases, July 2022 17(1), 295. https://ojrd.biomedcentral.com/articles/10.1186/s13023-022-02439-x
- American Academy of Allergy, Asthma & Immunology. Mast Cell Activation Syndrome (MCAS). https://www.aaaai.org/conditions-treatments/related-conditions/mcas
- SantaBarbara JN, Carroll J, Lobel M. Coping, social support, and anxiety in people with Mast Cell Disorders. Ann Allergy Asthma Immunol. 2021;127(4):435-440. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9909465/
- Akin C, Valent P, Metcalfe DD. Mast Cell Activation Syndrome: Proposed Diagnostic Criteria. J Allergy Clin Immunol. 2010;126(6):1099-104.e4.
- Gülen T, Akin C, Bonadonna P, Siebenhaar F, et al. Selecting the Right Criteria and Proper Classification to Diagnose Mast Cell Activation Syndromes: A Critical Review. J Allergy Clin Immunol Pract. 2021;9(11):3918-3928.
- Mast Cell Disorders in Ehlers-Danlos Syndrome (for Non-experts). The Ehlers DanlosSociety 2017. .https://www.ehlers-danlos.com/2017-eds-classification-non-experts/mast-cell-disorders-ehlers-danlos-syndrome-2/
- Kohno R, Cannom DS, Olshansky B, Xi SC, Krishnappa D, et al. Mast Cell Activation Disorder and Postural Orthostatic Tachycardia Syndrome: A Clinical Association. J Am Heart Assoc. 2021;10(17):e021002. https://www.ahajournals.org/doi/10.1161/JAHA.121.021002
- Valent P, Hartmann K, Bonadonna P, et al. Mast Cell Activation Syndromes: Collegium Internationale Allergologicum Update 2022. Int Arch Allergy Immunol. 2022;183(7):693-705. https://karger.com/iaa/article-pdf/183/7/693/3721896/000524532.pdf
- NPS Medicinewise. Medicine and Side Effects. https://www.nps.org.au/consumers/medicine-and-side-effects#
- Lyons JJ. Inherited and acquired determinants of serum tryptase levels in humans. Ann Allergy Asthma Immunol. Oct 2021;127(4):420–6. https://pubmed.ncbi.nlm.nih.gov/34175497/
- Kačar M, Rijavec M, Šelb J, Korošec P. Clonal mast cell disorders and hereditary α-tryptasemia as risk factors for anaphylaxis. Clin Exp Allergy. April 2023;53(4):392-404. https://pubmed.ncbi.nlm.nih.gov/36654513/
- Chollet MB, Akin C. Hereditary alpha tryptasemia is not associated with specific clinical phenotypes. J Allergy Clin Immunol. Feb 2022;149(2):728-735.e2. https://pubmed.ncbi.nlm.nih.gov/34174297/
- Glover SC, Carter MC, Korošec P, et al. Clinical relevance of inherited genetic differences in human tryptases: Hereditary alpha-tryptasemia and beyond. Ann Allergy Asthma Immunol. 2021;127(6):638-647. https://pubmed.ncbi.nlm.nih.gov/34400315/