Disease ID:1194
Associated with:0 targets
0 immuno targets immuno relevant
4 immuno ligands
A disease caused by mast cell hyperplasia.
Database Links
Disease Ontology: DOID:350


No target related data available for Mastocytosis


Ligand Approved Immuno References Clinical comments
brentuximab vedotin  [ FDA (2011), EMA (2012) ( ]
Clinical Use: Brentuximab vedotin is an anti-neoplastic agent used in the treatment of Hodgkin's lymphoma [2] and systemic anaplastic large cell lymphoma. Several clinical trials are evaluating the combination of brentuximab vedotin with immune checkpoint inhibitors, to assess any synergistic effects as a result of dual targeting. Checkpoint inhibitors being investigated in this way include nivolumab and pembrolizumab (both anti-PD-1), and ipilimumab (anti-CTLA4). Brentuximab vedotin + pembrolizumab (NCT02684292) and brentuximab vedotin + nivolumab (NCT03138499) are both Phase 3 studies in patients with relapsed/refractory classical Hodgkin's lymphoma.

Expanded approvals:
In November 2017, the FDA approved brentuximab vedotin for the treatment of adult patients with primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy, following review of results from the Phase 3 ALCANZA trial (NCT01578499) [1].
Immuno Disease Comments: Phase 2 clinical candidate for aggressive systemic mastocytosis, mast cell leukemia and systemic mastocytosis- see NCT01807598
midostaurin  [ FDA (2017) ( ]
Clinical Use: The FDA granted midostaurin accelerated approval in April 2017 for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive (as detected by an FDA-approved test) to be used in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. It was also approved for mastocytosis. The EMA has granted orphan designation for the treatment of AML and mastocytosis (via inhibition of KIT receptors on mast cells, which are involved in stimulating proliferation of mast cells in mastocytosis).
Click here to link to's list of Phase II midostaurin trials.
Immuno Disease Comments: FDA approved drug for aggressive systemic mastocytosis and systemic mastocytosis with associated hematological neoplasm or mast cell leukemia. EMA orphan drug for mastocytosis.
Clinical Use: The European Medicines Agency has granted this drug orphan status for treating pancreatic cancer, and it is in clinical trials for other human cancers. Under the synonym N-(methyl-diazacyclohexyl-methylbenzamide)-azaphenyl-aminothiopyrrole (a.k.a. Masican) the EMA has also granted orphan designation for the treatment of malignant gastrointestinal stromal tumours, mastocytosis and multiple myeloma. A Phase 3 trial of masitinib in combination with riluzole in amyotrophic lateral sclerosis (ALS, a.k.a. motor neurone disease, MND) is underway (NCT03127267). Click here to link to's listing of masitinib trials. Fourteen Phase 3 trials are registered with (as of Jan 2018), but many of these are not active.
Immuno Disease Comments: EMA orphan drug for mastocytosis.
cromoglicic acid  [ FDA (1984) ( ]
Clinical Use: Cromoglicic acid is available in different formulations; nasal spray is used to treat allergic rhinitis, nebulizer solution to treat asthma, eye drops to treat allergic conjunctivitis and an oral form to treat mastocytosis,dermatographic urticaria and ulcerative colitis.
Recent reports suggest that cromolyn inhibits glycogen synthase kinase 3β and may therefore have potential to treat diabetes and obesity [3] and may prove effective in treating insulin-induced lipoatrophy [4].
Immuno Disease Comments: Mast cell stabiliser used to treat mastocytosis.


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1. FDA. FDA approves Brentuximab vedotin for the treatment of adult patients with primary cutaneous anaplastic large cell lymphoma. Accessed on 10/11/2017. Modified on 10/11/2017.,

2. Moskowitz CH, Nademanee A, Masszi T, Agura E, Holowiecki J, Abidi MH, Chen AI, Stiff P, Gianni AM, Carella A et al.. (2015) Brentuximab vedotin as consolidation therapy after autologous stem-cell transplantation in patients with Hodgkin's lymphoma at risk of relapse or progression (AETHERA): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet,  [Epub ahead of print]. [PMID:25796459]

3. Motawi TM, Bustanji Y, El-Maraghy SA, Taha MO, Al Ghussein MA. (2013) Naproxen and cromolyn as new glycogen synthase kinase 3β inhibitors for amelioration of diabetes and obesity: an investigation by docking simulation and subsequent in vitro/in vivo biochemical evaluation. J. Biochem. Mol. Toxicol.27 (9): 425-36. [PMID:23784744]

4. Phua EJ, Lopez X, Ramus J, Goldfine AB. (2013) Cromolyn sodium for insulin-induced lipoatrophy: old drug, new use. Diabetes Care36 (12): e204-5. [PMID:24265375]