Midostaurin in Advanced Systemic Mastocytosis

Chandesris, Marie-Olivia; Damaj, Gandhi; Canioni, Danielle; Brouzes, Chantal; Lhermitte, Ludovic; Hanssens, Katia; Frenzel, Laurent; Cherquaoui, Zoubair; Durieu, I.; Durupt, S.; Gyan, Emmanuel; Beyne-Rauzy, O.; Launay, David; Faure, Cyril; Hamidou, M.; Besnard, Sophie; Diouf, Momar; Schiffmann, A.; Niault, Mathilde; Jeandel, Pierre-Yves; Ranta, Dana; Gressin, Rémy; Chantepie, Sylvain; Barète, Stéphane; Dubreuil, Patrice; Bourget, Philippe; Lortholary, Olivier; Hermine, Olivier

doi:10.1056/nejmc1515403

Cited by 59 publications

(84 citation statements)

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“…Cladribine (2CdA) is often recommended as first-line therapy in patients with advanced SM with multi-organ involvement and slow progression (82–85). A forthcoming new standard of therapy in advanced SM is midostaurin (PKC412) (86–88). For ASM/MCL patients with rapid progression and those who are resistant against 2CdA or midostaurin, poly-chemotherapy (protocols otherwise used for high-risk AML) is usually recommended (23,64,65).…”

Section: First Line Therapy In Patients With Advanced Smmentioning

confidence: 99%

“…In addition, in contrasting to other KIT-targeting drugs, midostaurin also inhibits IgE-dependent release of histamine (92,93). Finally, midostaurin has been reported to be efficacious in patients with advanced SM, including ASM and MCL (86–88). In particular, data from the global trial of midostaurin in advanced SM indicate that the drug exhibits high response rates and durable activity (86).…”

Section: New Treatment Options For Patients With Advanced Smmentioning

confidence: 99%

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Advances in the Classification and Treatment of Mastocytosis: Current Status and Outlook toward the Future

et al. 2017

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Mastocytosis is a term used to denote a heterogeneous group of conditions defined by expansion and accumulation of clonal (neoplastic) tissue mast cells in various organs. The classification of the World Health Organization (WHO) divides the disease into cutaneous mastocytosis (CM), systemic mastocytosis (SM), and localized mast cell tumors. Based on histomorphologic criteria, clinical parameters, and organ involvement, SM is further divided into indolent SM (ISM) and advanced SM variants, including aggressive SM (ASM) and mast cell leukemia (MCL). The clinical impact and prognostic value of this classification has been confirmed in numerous studies, and its basic concept remains valid. However, refinements have recently been proposed by the consensus group, the WHO, and the European Competence Network on Mastocytosis (ECNM). In addition, new treatment options are available for patients with advanced SM, including allogeneic hematopoietic stem cell transplantation and multi-kinase inhibitors directed against KIT D816V and other key signalling molecules. Our current article provides an overview of recent advances in the field of mastocytosis, with emphasis on classification, prognostication, and emerging new treatment options in advanced SM.

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Section: First Line Therapy In Patients With Advanced Smmentioning

confidence: 99%

Section: New Treatment Options For Patients With Advanced Smmentioning

confidence: 99%

Advances in the Classification and Treatment of Mastocytosis: Current Status and Outlook toward the Future

et al. 2017

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“…The histone deacetylase inhibitors SAHA epigenetically silences c-KIT followed by major mast cell apoptosis, with a correlation between cell death and systemic mastocytosis disease severity, with cell death more pronounced in the case of aggressive disease. In another study, midostaurin, an inhibitor of tyrosine kinases, has shown to target c-KIT mutants associated with mastocytosis and seems to reduce the risk of death, so indeed new targeted therapies are being developed for the treatment of this condition (60). In specific relation to flushing, this is often treated with an H 1 -histaminereceptor antagonist such as hydroxyzine or non-sedating antihistamines such as cetirizine or fexofenadine, with expert opinion endorsing the daytime use of non-sedating antihistamines and nighttime use of sedating ones (61).…”

Section: Mastocytosismentioning

confidence: 99%

MANAGEMENT OF ENDOCRINE DISEASE: Flushing: current concepts

Huguet

Grossman

2017

European Journal of Endocrinology

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Objective: Flushing can be defined as a sensation of warmth accompanied by erythema that most commonly is seen on the face and which occurs in episodic attacks. Such a problem can be clinically problematic, since many conditions and drugs can be related to flushing, and while often there appears to be no underlying organic disease, it is important to exclude disorders since they may be life-threatening conditions. Design and methods: We performed a search in MEDLINE using the terms 'flushing' in combination with 'carcinoid syndrome', 'pheochromocytoma', 'mastocytosis', 'menopausal hot flush' and 'treatment'. European and American guidelines relating to neuroendocrine tumours, mastocytosis and menopause were reviewed. Results: In this review, we discuss the main causes of flushing and propose an algorithm based on pathogenesis, which can be used to guide the clinical evaluation process. We also review recent significant developments in the assessment and treatment of the carcinoid syndrome and menopausal hot flushes, which should guide the clinical practice regarding this common but sometimes confusing condition. Conclusions: When evaluating flushing, a precise systematic approach is needed to exclude potentially serious underlying causes, although despite this, the cause of the disorder is not always found. If symptoms are not progressive, the patient should be advised about its apparently benign nature in order to avoid unnecessary studies or initiating treatments of minimal benefit.

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“…The only curative option is an allogeneic stem cell transplant (alloSCT), however most patients do not have a sustained remission post‐transplant (Valent et al , ; Ustun et al , ). In the era of midostaurin, remission after alloSCT may be achieved depending on tolerance and associated toxicity (Chandesris et al , ; Gotlib et al , ; Ustun et al , ; Gallogly et al , ).…”

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confidence: 99%

“…Midostaurin has demonstrated activity against KIT D816V in patients with ASM (Chandesris et al , ; Gotlib et al , ; Gallogly et al , ). In a multicentre phase II study, 57% of patients with systemic mastocytosis treated with midostaurin had a >50% reduction in bone marrow mast cells, as well as normalization of cytopenias, liver function abnormalities and hypoalbuminaemia (Chandesris et al , ).…”

mentioning

confidence: 99%