Evidence map›Paper›PMID 28945834›Full record

ReviewAnnals of oncology : official journal of the European Society for Medical Oncology2017

Midostaurin: a magic bullet that blocks mast cell expansion and activation.

P Valent, C Akin, K Hartmann, T I George, K Sotlar, B Peter, K V Gleixner, K Blatt, W R Sperr, P W Manley and 6 more

Open access · bronzeAbstract readReview
In one paragraph

Review in Annals of oncology : official journal of the European Society for Medical Oncology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 papers.

0numbers the graph read from it
0cells of the map it votes in
44citing papers in PubMed
3.5field-weighted citation impact, top 7% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

44 citing papers in PubMed, 77 citations in OpenAlex.

  1. Article
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  3. Review
  4. Review
  5. Review
  6. Article
  7. Review
  8. Article
  9. Observational
  10. Review
  11. Mastocytosis.Nature reviews. Disease primers · 2025
    Review
  12. Mast cell activation syndrome: An up-to-date review of literature.World journal of clinical pediatrics · 2024
    Review
  13. Article
  14. Review
  15. Review
  16. Review
  17. Article
  18. Review
  19. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors at 13 institutions in 6 countries.

P ValentLudwig Boltzmann Cluster Oncology, Medical University of Vienna, Vienna, Austria;; Division of Hematology & Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria;. Electronic address: peter.valent@meduniwien.ac.at.
C AkinDivision of Allergy and Clinical Immunology, University of Michigan, Ann Arbor, USA.
K HartmannDepartment of Dermatology, University of Luebeck, Luebeck, Germany.
T I GeorgeDepartment of Pathology, University of New Mexico, Albuquerque, USA.
K SotlarInstitute of Pathology, Paracelsus Medical University Salzburg, Salzburg, Austria.
B PeterLudwig Boltzmann Cluster Oncology, Medical University of Vienna, Vienna, Austria;; Division of Hematology & Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria.
K V GleixnerLudwig Boltzmann Cluster Oncology, Medical University of Vienna, Vienna, Austria;; Division of Hematology & Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria.
K BlattLudwig Boltzmann Cluster Oncology, Medical University of Vienna, Vienna, Austria;; Division of Hematology & Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria.
W R SperrLudwig Boltzmann Cluster Oncology, Medical University of Vienna, Vienna, Austria;; Division of Hematology & Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria.
P W ManleyNovartis Oncology, Basel, Switzerland.
O HermineCentre National de Référence des Mastocytoses, Imagine Institute Université Paris Descartes, Sorbonne Paris Cité, Paris, France.
H C Kluin-NelemansDepartment of Hematology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
M ArockLBPA CNRS UMR8113, Ecole Normale Supérieure de Cachan, Cachan, France.
H-P HornyInstitute of Pathology, Ludwig-Maximilians-University, Munich, Germany.
A ReiterDepartment of Hematology and Oncology, University Medical Center Mannheim, Heidelberg University, Mannheim, Germany.
J GotlibStanford University School of Medicine/Stanford Cancer Institute, Stanford, USA.
Ludwig Boltzmann Cluster for Cardiovascular Research · ATMedical University of Vienna · ATÉcole Normale Supérieure Paris-Saclay · FRLudwig-Maximilians-Universität München · DENovartis (Switzerland) · CHParacelsus Medical University · ATSorbonne Paris Cité · FRStanford Cancer InstituteUniversity Hospital Heidelberg · DEUniversity Medical Center Groningen · NLUniversity of Lübeck · DEUniversity of Michigan–Ann Arbor · USUniversity of New Mexico · US

Funding

Austrian Science Fund FWF F 4704
6 · The paper itself

Abstract

Clinically relevant features in patients with systemic mastocytosis (SM) include the cosmetic burden of lesional skin, mediator-related symptoms, and organ damage resulting from mast cell (MC) infiltration in advanced forms of SM. Regardless of the SM variant, expansion of neoplastic MC in the skin and other organs is triggered by mutant forms of KIT, the most prevalent being D816V. Activation of MC with subsequent release of chemical mediators is often caused by IgE-dependent mechanisms in these patients. Midostaurin, also known as PKC412, blocks the kinase activity of wild-type KIT and KIT D816V, counteracts KIT-dependent growth of neoplastic MC, and inhibits IgE-dependent mediator secretion. Based on this activity-profile, the drug has been used for treatment of patients with advanced SM. Indeed, encouraging results have been obtained with the drug in a recent multi-center phase II trial in patients with advanced SM, with an overall response rate of 60% and a substantial decrease in the burden of neoplastic MC in various organs. Moreover, midostaurin improved the overall survival and relapse-free survival in patients with advanced SM compared with historical controls. In addition, midostaurin was found to improve mediator-related symptoms and quality of life, suggesting that the drug may also be useful in patients with indolent SM suffering from mediator-related symptoms resistant to conventional therapies or those with MC activation syndromes. Ongoing and future studies will determine the actual value of midostaurin-induced MC depletion and MC deactivation in these additional indications.

Indexed as

Antineoplastic AgentsClinical Trials, Phase II as TopicDrug Resistance, NeoplasmHumansMast CellsMastocytosis, SystemicMulticenter Studies as TopicStaurosporineAntineoplastic AgentsmidostaurinStaurosporineIgEmast cell activationmast cell leukemiamast cellstargeted drugs

Identifiers

PMID28945834
PMCPMC7115852
OpenAlexW2753355630

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.