Advanced mast cell disease: An Italian Hematological Multicenter experience

Livio Pagano, Caterina Giovanna Valentini, Morena Caira, Michela Rondoni, Maria Teresa Van Lint, Anna Candoni, Bernardino Allione, Chiara Cattaneo, Laura Marbello, Cecilia Caramatti, Enrico Maria Pogliani, Emilio Iannitto, Fiorina Giona, Felicetto Ferrara, Rosangela Invernizzi, Rosa Fanci, Monia Lunghi, Luana Fianchi, Grazia Sanpaolo, Pietro Maria StefaniAlessandro Pulsoni, Giovanni Martinelli, Giuseppe Leone, Pellegrino Musto

Research output: Contribution to journalArticle

Abstract

The aim of the study is to evaluate clinical features, treatments and outcome of patients with systemic mast cell disease (MCD) who arrived to the attention of hematologists. A retrospective study was conducted over 1995-2006 in patients admitted in 18 Italian hematological divisions. Twenty-four cases of advanced MCD were collected: 12 aggressive SM (50%), 8 mast cell leukemia (33%), 4 SM with associated clonal non-mast cell-lineage hematologic disease (17%). Spleen and liver were the principal extramedullary organ involved. The c-kit point mutation D816V was found in 13/18 patients in which molecular biology studies were performed (72%). Treatments were very heterogeneous: on the whole Imatinib was administered in 17 patients, α-Interferon in 8, 2-CdA in 3; 2 patients underwent allogeneic hematopoietic stem cell transplantation. The overall response rate to Imatinib, the most frequently employed drugs, was of 29%, registering one complete remission and four partial remission; all responsive patients did not present D816V c-kit mutation. Overall three patients (12%) died for progression of disease. We conclude that MCD is characterized by severe mediator-related symptoms but with a moderate mortality rate. D816V c-kit mutation is frequent and associated with resistance against Imatinib. Because of the rarity of these forms, an effective standard of care is lacking. More data are needed to find new and successful therapeutic strategies.

Original languageEnglish
Pages (from-to)483-488
Number of pages6
JournalInternational Journal of Hematology
Volume88
Issue number5
DOIs
Publication statusPublished - Dec 2009

Fingerprint

Mastocytosis
Mast-Cell Leukemia
Systemic Mastocytosis
Mutation
Hematologic Diseases
Hematopoietic Stem Cell Transplantation
Cell Lineage
Standard of Care
Point Mutation
Interferons
Disease Progression
Molecular Biology
Spleen
Retrospective Studies
Mortality
Liver
Therapeutics

Keywords

  • Mast cell disease
  • Therapy
  • Tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Hematology

Cite this

Advanced mast cell disease : An Italian Hematological Multicenter experience. / Pagano, Livio; Valentini, Caterina Giovanna; Caira, Morena; Rondoni, Michela; Van Lint, Maria Teresa; Candoni, Anna; Allione, Bernardino; Cattaneo, Chiara; Marbello, Laura; Caramatti, Cecilia; Pogliani, Enrico Maria; Iannitto, Emilio; Giona, Fiorina; Ferrara, Felicetto; Invernizzi, Rosangela; Fanci, Rosa; Lunghi, Monia; Fianchi, Luana; Sanpaolo, Grazia; Stefani, Pietro Maria; Pulsoni, Alessandro; Martinelli, Giovanni; Leone, Giuseppe; Musto, Pellegrino.

In: International Journal of Hematology, Vol. 88, No. 5, 12.2009, p. 483-488.

Research output: Contribution to journalArticle

Pagano, L, Valentini, CG, Caira, M, Rondoni, M, Van Lint, MT, Candoni, A, Allione, B, Cattaneo, C, Marbello, L, Caramatti, C, Pogliani, EM, Iannitto, E, Giona, F, Ferrara, F, Invernizzi, R, Fanci, R, Lunghi, M, Fianchi, L, Sanpaolo, G, Stefani, PM, Pulsoni, A, Martinelli, G, Leone, G & Musto, P 2009, 'Advanced mast cell disease: An Italian Hematological Multicenter experience', International Journal of Hematology, vol. 88, no. 5, pp. 483-488. https://doi.org/10.1007/s12185-008-0166-4
Pagano, Livio ; Valentini, Caterina Giovanna ; Caira, Morena ; Rondoni, Michela ; Van Lint, Maria Teresa ; Candoni, Anna ; Allione, Bernardino ; Cattaneo, Chiara ; Marbello, Laura ; Caramatti, Cecilia ; Pogliani, Enrico Maria ; Iannitto, Emilio ; Giona, Fiorina ; Ferrara, Felicetto ; Invernizzi, Rosangela ; Fanci, Rosa ; Lunghi, Monia ; Fianchi, Luana ; Sanpaolo, Grazia ; Stefani, Pietro Maria ; Pulsoni, Alessandro ; Martinelli, Giovanni ; Leone, Giuseppe ; Musto, Pellegrino. / Advanced mast cell disease : An Italian Hematological Multicenter experience. In: International Journal of Hematology. 2009 ; Vol. 88, No. 5. pp. 483-488.
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AU - Candoni, Anna

AU - Allione, Bernardino

AU - Cattaneo, Chiara

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AU - Caramatti, Cecilia

AU - Pogliani, Enrico Maria

AU - Iannitto, Emilio

AU - Giona, Fiorina

AU - Ferrara, Felicetto

AU - Invernizzi, Rosangela

AU - Fanci, Rosa

AU - Lunghi, Monia

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AU - Sanpaolo, Grazia

AU - Stefani, Pietro Maria

AU - Pulsoni, Alessandro

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