Mast cell tumor medical therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1];Associate Editor(s)-in-Chief: Suveenkrishna Pothuru, M.B,B.S. [2]


The mainstay of therapy for mast cell tumor is avoidance of triggering factors and symptomatic therapy. Chemotherapy is indicated for aggressive form of mast cell tumor.

Medical Therapy

The treatment of mast cell tumor is mainly focused on avoidance of triggering factors (e.g. physical stimuli such as heat or cold, alcohol, stress, sternous exercise, drugs such as aspirin and other NSAIDS) and symptomatic therapy. Commonly used medications for symptomatic relief include:[1][2][3]

Medication Indications

H1 antihistamine

H2 antihistamine

Antileukotriene drugs

  • Flushing
  • Pruritus
  • Abdominal discomfort

Mast cell stabilizers

Proton pump inhibitors

  • Peptic ulcers


  • Anaphylaxis



  • Antihistamines block receptors targeted by histamine released from mast cells. Both H1 and H2 blockers may be helpful.
  • Leukotriene antagonists block receptors targeted by leukotrienes released from mast cells.
  • Antihistamines frequently treat itching and other skin complaints.
  • Certain antihistamines work specifically against ulcers

Mast cell stabilizers

  • Mast cell stabilizers help prevent mast cells from releasing their chemical contents.
  • Cromolyn sodium oral solution (Gastrocrom® / Cromoglicate) is the only medicine specifically approved by the U.S. FDA for the treatment of mastocytosis.
  • Ketotifen is available in Canada and Europe, but is only available in the U.S. as ophthalmic drops (Zaditor®).
  • Cromolyn sodium may help reduce cramping in the abdomen.

Proton pump inhibitors

  • Proton pump inhibitors help reduce production of gastric acid, which is often increased in patients with mastocytosis.
  • Excess gastric acid can harm the stomach, esophagus, and small intestine.


  • Epinephrine constricts blood vessels and opens airways to maintain adequate circulation and ventilation when excessive mast cell degranulation has caused anaphylaxis.
  • Epinephrine treats symptom flares which occur with shock, referred to as "anaphylaxis".
  • In case of repeated life-threatening anaphylactoid episodes, the self-administration of epinephrine on demand has been recommended as an appropriate approach.[4]


  • Albuterol and other beta-2 agonists open airways that can constrict in the presence of histamine.


  • Corticosteroids can be used topically, inhaled, or systemically to reduce inflammation associated with mastocytosis.
  • Steroids treat malabsorption or impaired ability to take in nutrients.


  • Antidepressants are an important and often overlooked tool in the treatment of mastocytosis.
  • The stress and physical discomfort of any chronic disease may increase the likelihood of a patient developing depression.
  • Depression and other neurological symptoms have been noted in mastocytosis.[5] Some antidepressants such as doxepin are themselves potent antihistamines and can help relieve physical as well as cognitive symptoms.

Calcium channel blocker


  • In rare cases in which mastocytosis is cancerous or associated with a blood disorder, the patient may have to use steroids and/or chemotherapy.
  • The novel agent imatinib (Glivec® or Gleevec®) has been found to be effective in certain types of mastocytosis.[7]
  • Recent literature shows that C-Kit (D816V) lends some resistance to imatinib and sorafenib but these cells are still sensitive to nilotinib, dasatinib and rapamycin. Cladribine and interferon have also been found to be effective.


  • A difficult situation is the occurrence of life-threatening anaphylaxis in patients with mast cell tumor.[4]
  • If anaphylaxis is provoked by a known allergen, especially hymenoptera venom, immunotherapy should be considered with recognition of potential risks.

Cutaneous Mastocytosis

  • Usually, Isolated cutaneous mastocytosis needs no treatment or action to be taken.[8]
  • Cutaneous mastocytosis with cosmetic complaints should be treated by a topical therapy only in children older than 2 years.
  • When cutaneous mastocytosis is confined to about 10% of body surface, occlusive dressing is used.
  • When the extension is larger, (more than 10%) the occlusive dressing is optional, and usually the corticosteroid cream is diluted.
  • In cutaneous mastocytosis with complaints of itch, redness, and swelling, avoiding foods with a possible triggering role has to be suggested.

Diffuse cutaneous mastocytosis

  • Diffuse cutaneous mastocytosis is self-limiting, and the need for a treatment is questionable.[8]
  • If diffuse cutaneous mastocytosis causes significant distress, leads to frequent hospitalizations, or is life-threatening, treatment with imatinib is a good option.
  • Imatinib mesylate, a type II kinase inhibitor, inhibits cell proliferation and induces apoptosis by binding the adenosine triphosphate pocket and the adjacent site of the kinase.
  • The nonselectivity of imatinib also explains side effects such as cardiomyopathy and reduced growth velocity, the latter being a relevant side effect in children.
  • A dose of 200 mg/m2/day have been used in children, and reducing the dosage as soon as sustained healing of the skin lesions is noted is sufficient to obtain remission.


  1. Koenig, Martial; Morel, Jérôme; Reynaud, Jacqueline; Varvat, Cécile; Cathébras, Pascal (2008). "An unusual cause of spontaneous bleeding in the intensive care unit – mastocytosis: a case report". Cases Journal. 1 (1): 100. doi:10.1186/1757-1626-1-100. ISSN 1757-1626.
  2. Tolar J, Tope WD, Neglia JP (February 2004). "Leukotriene-receptor inhibition for the treatment of systemic mastocytosis". N. Engl. J. Med. 350 (7): 735–6. doi:10.1056/NEJM200402123500723. PMID 14960756.
  3. Worobec AS (June 2000). "Treatment of systemic mast cell disorders". Hematol. Oncol. Clin. North Am. 14 (3): 659–87, vii. PMID 10909045.
  4. 4.0 4.1 Molderings, Gerhard J; Brettner, Stefan; Homann, Jürgen; Afrin, Lawrence B (2011). "Mast cell activation disease: a concise practical guide for diagnostic workup and therapeutic options". Journal of Hematology & Oncology. 4 (1): 10. doi:10.1186/1756-8722-4-10. ISSN 1756-8722.
  5. Rogers MP, Bloomingdale K, Murawski BJ, Soter NA, Reich P, Austen KF. Mixed organic brain syndrome as a manifestation of systemic mastocytosis. Psychosom Med 1986;48:437-47. PMID 3749421
  6. Fairley JA, et al: Urticaria pigmentosa responsive to nifedipine. J Am Acad Dermatol 11:740-743, 1984.
  7. Droogendijk HJ, Kluin-Nelemans HJ, van Doormaal JJ, Oranje AP, van de Loosdrecht AA, van Daele PL. Imatinib mesylate in the treatment of systemic mastocytosis: a phase II trial. Cancer. 2006 Jul 15;107(2):345-51. PMID 16779792
  8. 8.0 8.1 Ferrante, Giuliana; Scavone, Valeria; Muscia, Maria; Adrignola, Emilia; Corsello, Giovanni; Passalacqua, Giovanni; La Grutta, Stefania (2015). "The care pathway for children with urticaria, angioedema, mastocytosis". World Allergy Organization Journal. 8 (1): 5. doi:10.1186/s40413-014-0052-x. ISSN 1939-4551.

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