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Successful Targeted Treatment of Mast Cell Activation Syndrome with Tofacitinib.

Discussion in 'Mast Cell Disorders/Mastocytosis' started by impossible, Apr 20, 2017.

  1. impossible

    impossible

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    Another one from Afrin:

    Abstract
    Mast cell (MC) activation syndrome (MCAS) is a collection of illnesses of inappropriate MC activation with little to no neoplastic MC proliferation, distinguishing it from mastocytosis. MCAS presents as chronic, generally inflammatory multisystem polymorbidity likely driven in most by heterogeneous patterns of constitutively activating mutations in MC regulatory elements, posing challenges for identifying optimal mutation-targeted treatment in individual patients. Targeting commonly affected downstream effectors may yield clinical benefit independent of upstream mutational profile. For example, both activated KIT and numerous cytokine receptors activate the Janus kinases (JAKs). Thus, JAK-inhibiting therapies may be useful against the downstream inflammatory effects of MCAS. The oral JAK1/JAK3 inhibitor, tofacitinib, is currently approved for rheumatoid arthritis and is in clinical trials for other chronic inflammatory disorders. Herein, we report two MCAS patients who rapidly gained substantial symptomatic response to tofacitinib. Their improvement suggests need for further evaluation of this class of drugs in MCAS treatment. This article is protected by copyright. All rights reserved.

    This one seems like it has the potential to help alot of mcas patients.
     
    zenzen, bspg, Jesse2233 and 4 others like this.
  2. Jesse2233

    Jesse2233 Senior Member

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    Southern California
    Has there been any more information on this?
     
  3. impossible

    impossible

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    Ruxolitinib improves symptoms and quality of life in a patient with systemic mastocytosis.
    Yacoub A1, Prochaska L1.
    Author information

    Abstract
    BACKGROUND:
    Systemic mastocytosis is a clonal myeloproliferative neoplasm associated with constitutional symptoms from mast cell mediated chemical and cytokine release. According to the literature, Ruxolitinib, a JAK1/JAK2 inhibitor, has been shown to reduce symptoms related to proinflammatory cytokine release in other myeloproliferative neoplasms.

    CASE PRESENTATION:
    Here we present a case using Ruxolitinib for disabling constitutional symptoms despite complete bone marrow response in a patient with aggressive systemic mastocytosis. Assessment tools used to monitor symptoms in previously published Ruxolitinib trials were adopted to track symptom improvement and quality of life.

    CONCLUSIONS:
    Ruxolitinib significantly improved symptoms and quality of life in our patient with systemic mastocytosis.



    I read the complete study and it seemed like she had a pretty substantial improvement
     
    zenzen likes this.
  4. impossible

    impossible

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    Ive been using artemisinin for a little over a month, for its jak inhibiting/socs boosting properties, and its been very helpful so far.
     
    zenzen likes this.

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