Skip to main content
  • AACR Publications
    • Cancer Discovery
    • Cancer Epidemiology, Biomarkers & Prevention
    • Cancer Immunology Research
    • Cancer Prevention Research
    • Cancer Research
    • Clinical Cancer Research
    • Molecular Cancer Research
    • Molecular Cancer Therapeutics

  • Register
  • Log in
Advertisement

Main menu

  • Home
  • About
    • The Journal
    • AACR Journals
    • Subscriptions
    • Permissions and Reprints
    • Reviewing
  • Articles
    • OnlineFirst
    • Current Issue
    • Past Issues
    • Focus on Computer Resources
    • 75th Anniversary
    • Meeting Abstracts
  • For Authors
    • Information for Authors
    • Author Services
    • Best of: Author Profiles
    • Submit
  • Alerts
    • Table of Contents
    • OnlineFirst
    • Editors' Picks
    • Citations
    • Author/Keyword
  • News
    • Cancer Discovery News
  • AACR Publications
    • Cancer Discovery
    • Cancer Epidemiology, Biomarkers & Prevention
    • Cancer Immunology Research
    • Cancer Prevention Research
    • Cancer Research
    • Clinical Cancer Research
    • Molecular Cancer Research
    • Molecular Cancer Therapeutics

User menu

  • Register
  • Log in

Search

  • Advanced search
Cancer Research
Cancer Research

Advanced Search

  • Home
  • About
    • The Journal
    • AACR Journals
    • Subscriptions
    • Permissions and Reprints
    • Reviewing
  • Articles
    • OnlineFirst
    • Current Issue
    • Past Issues
    • Focus on Computer Resources
    • 75th Anniversary
    • Meeting Abstracts
  • For Authors
    • Information for Authors
    • Author Services
    • Best of: Author Profiles
    • Submit
  • Alerts
    • Table of Contents
    • OnlineFirst
    • Editors' Picks
    • Citations
    • Author/Keyword
  • News
    • Cancer Discovery News
Experimental and Molecular Therapeutics

Abstract 2082: PKC412 (Midostaurin) is safe and highly effective in Systemic Mastocytosis patients: the Bologna experience.

Cristina Papayannidis, Mariachiara Abbenante, Sarah Parisi, Stefania Paolini, Ilaria Iacobucci, Emanuela Ottaviani, Anna Ferrari, Viviana Guadagnuolo, Chiara Sartor, Simona Soverini, Caterina De Benedittis, Carmen Baldazzi, Michele Baccarani, Michele Cavo and Giovanni Martinelli
Cristina Papayannidis
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Mariachiara Abbenante
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Sarah Parisi
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Stefania Paolini
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Ilaria Iacobucci
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Emanuela Ottaviani
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Anna Ferrari
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Viviana Guadagnuolo
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Chiara Sartor
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Simona Soverini
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Caterina De Benedittis
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Carmen Baldazzi
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Michele Baccarani
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Michele Cavo
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Giovanni Martinelli
Department of Hematology/Oncology "L. and A. Seràgnoli" - University of Bologna, Bologna, Italy.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
DOI: 10.1158/1538-7445.AM2013-2082 Published April 2013
  • Article
  • Info & Metrics
Loading
Proceedings: AACR 104th Annual Meeting 2013; Apr 6-10, 2013; Washington, DC

Abstract

Introduction. Mastocytosis is a myeloid neoplasm characterized by abnormal accumulation and frequent activation of mast cells (MCs) in various organs, mostly bone marrow, skin, liver and gastrointestinal tract. In most adult patients, the systemic form of mastocytosis (SM) is diagnosed, which includes an indolent (ISM), an aggressive (ASM) and a leukemic subvariant (MCL). The c-kit mutation D816V is detectable in most adult patients with SM. Treatment of SM usually focuses on symptom relief by histamine receptor antagonists and other supportive therapy. However, in aggressive and leukemic variants, cytoreductive and targeted drugs must be applied.

Methods. From 2008, 18 patients (male/female=7/11) affected by SM, have been referred to our Institution. The median age was 49 years. All the patients underwent a bone marrow biopsy, with flow citometry and molecular biology analysis, in order to identify the presence of D816V mutation of c-Kit gene. Serum tryptase level was tested, resulting elevated in all cases. Systemic symptoms were characterized by nausea, diarrhoea, asthenia, weight loss, pruritus and serotine fever, identifying an aggressive form of the disease in 7/18 patients, due to skeletal involvement, ascitis, liver function impairment, and bone marrow disfunction. Therefore, since a first line therapy (mainly represented by IFN) and supportive care with histamine receptor antagonists werne't followed by a significant benefit, a personalized use of PKC412 was asked and obtained for 5 out of 7 ASM patients.

Results. From March 2011 5 out of 7 patients with ASM have received a prolonged period of treatment with PKC412, which was administered orally, at the dosage of 100 mg twice daily, without rest periods. The drug was well tolerated. No serious adverse events were observed. All the patients obtained a quick and prolonged improvement of clinical symptoms, in terms of weight gain, bowel function and skeletal pain. At the bone marrow evaluation, the persistence of the D816V c-kit mutation was observed, despite a significant decrease of mast cell marrow involvement. In one case we observed, after a first good response, a disease progression, characterized by the sudden reoccurrence of the same symptoms detected at diagnosis, confirmed by a relevant expansion of a pathologic mast cell population in the bone marrow. After a rest period, the drug was readministered, and a second remission was obtained.

Conclusions. PKC412 is safe and effective in patients with SM, being able to significantly improve not only the gastrointestinal and systemic symptoms, but also the haematological profile. The persistence of the D816V c-kit mutation, despite a morphologic remission, suggests that many other oncogenic factors may be responsible for the pathogenesis of the disease.

Acknowledgments. Work supported by European LeukemiaNet, AIRC, AIL, PRIN, University of Bologna and BolognAIL.

Citation Format: Cristina Papayannidis, Mariachiara Abbenante, Sarah Parisi, Stefania Paolini, Ilaria Iacobucci, Emanuela Ottaviani, Anna Ferrari, Viviana Guadagnuolo, Chiara Sartor, Simona Soverini, Caterina De Benedittis, Carmen Baldazzi, Michele Baccarani, Michele Cavo, Giovanni Martinelli. PKC412 (Midostaurin) is safe and highly effective in Systemic Mastocytosis patients: the Bologna experience. [abstract]. In: Proceedings of the 104th Annual Meeting of the American Association for Cancer Research; 2013 Apr 6-10; Washington, DC. Philadelphia (PA): AACR; Cancer Res 2013;73(8 Suppl):Abstract nr 2082. doi:10.1158/1538-7445.AM2013-2082

  • ©2013 American Association for Cancer Research
Back to top
Cancer Research: 73 (8 Supplement)
April 2013
Volume 73, Issue 8 Supplement
  • Table of Contents
  • Index by Author

Sign up for alerts

Article Alerts
Sign In to Email Alerts with your Email Address
Email Article

Thank you for sharing this Cancer Research article.

NOTE: We request your email address only to inform the recipient that it was you who recommended this article, and that it is not junk mail. We do not retain these email addresses.

Enter multiple addresses on separate lines or separate them with commas.
Abstract 2082: PKC412 (Midostaurin) is safe and highly effective in Systemic Mastocytosis patients: the Bologna experience.
(Your Name) has forwarded a page to you from Cancer Research
(Your Name) thought you would be interested in this article in Cancer Research.
Citation Tools
Abstract 2082: PKC412 (Midostaurin) is safe and highly effective in Systemic Mastocytosis patients: the Bologna experience.
Cristina Papayannidis, Mariachiara Abbenante, Sarah Parisi, Stefania Paolini, Ilaria Iacobucci, Emanuela Ottaviani, Anna Ferrari, Viviana Guadagnuolo, Chiara Sartor, Simona Soverini, Caterina De Benedittis, Carmen Baldazzi, Michele Baccarani, Michele Cavo and Giovanni Martinelli
Cancer Res April 15 2013 (73) (8 Supplement) 2082; DOI: 10.1158/1538-7445.AM2013-2082

Citation Manager Formats

  • BibTeX
  • Bookends
  • EasyBib
  • EndNote (tagged)
  • EndNote 8 (xml)
  • Medlars
  • Mendeley
  • Papers
  • RefWorks Tagged
  • Ref Manager
  • RIS
  • Zotero
Share
Abstract 2082: PKC412 (Midostaurin) is safe and highly effective in Systemic Mastocytosis patients: the Bologna experience.
Cristina Papayannidis, Mariachiara Abbenante, Sarah Parisi, Stefania Paolini, Ilaria Iacobucci, Emanuela Ottaviani, Anna Ferrari, Viviana Guadagnuolo, Chiara Sartor, Simona Soverini, Caterina De Benedittis, Carmen Baldazzi, Michele Baccarani, Michele Cavo and Giovanni Martinelli
Cancer Res April 15 2013 (73) (8 Supplement) 2082; DOI: 10.1158/1538-7445.AM2013-2082
del.icio.us logo Digg logo Reddit logo Twitter logo CiteULike logo Facebook logo Google logo Mendeley logo
  • Tweet Widget
  • Facebook Like
  • Google Plus One

Jump to section

  • Article
  • Info & Metrics
Advertisement

Related Articles

Cited By...

More in this TOC Section

Experimental and Molecular Therapeutics

  • Abstract SY14-03: Anaplastic lymphoma kinase (ALK): Normal biology and role in hematopoietic malignancies
  • Abstract SY18-01: Targeting the p53-MDM2 interaction for cancer therapy
  • Abstract SSY01-04: Discovery and validation of genome-wide genetic signatures of chemotherapy susceptibility: A translational model
Show more 3

Poster Presentations - Proffered Abstracts

  • Abstract SY14-03: Anaplastic lymphoma kinase (ALK): Normal biology and role in hematopoietic malignancies
  • Abstract SY18-01: Targeting the p53-MDM2 interaction for cancer therapy
  • Abstract SSY01-04: Discovery and validation of genome-wide genetic signatures of chemotherapy susceptibility: A translational model
Show more 3

Poster Presentations - Inhibition of Kinase Signaling 2

  • Abstract 5653: Novel targets mediating resistance to EGFR/c-Met tyrosine kinase inhibitors in NSCLC.
  • Abstract 2089: NT113, a potent and selective pan-ErbB inhibitor, is efficacious in vivo and has high brain penetrance.
  • Abstract 2081: Evaluation of Src as a therapeutic target in biliary tract cancer using bosutinib, a c-Src/Abl kinase inhibitor.
Show more 3
  • Home
  • Alerts
  • Feedback
Facebook  Twitter  LinkedIn  YouTube  RSS

Articles

  • Online First
  • Current Issue
  • Past Issues
  • Meeting Abstracts

Info for

  • Authors
  • Subscribers
  • Advertisers
  • Librarians
  • Reviewers

About Cancer Research

  • About the Journal
  • Editorial Board
  • Permissions
  • Submit a Manuscript
AACR logo

Copyright © 2018 by the American Association for Cancer Research.

Cancer Research Online ISSN: 1538-7445
Cancer Research Print ISSN: 0008-5472
Journal of Cancer Research ISSN: 0099-7013
American Journal of Cancer ISSN: 0099-7374

Advertisement