Publications

Authors:

Mannelli, Francesco; Crupi, Francesca; Zanotti, Roberta; Pagano, Livio; Rapezzi, Davide; Tanasi, Ilaria; Criscuolo, Marianna; Bonifacio, Massimiliano; Fresa, Alberto; Guglielmelli, Paola; Vannucchi, Alessandro M

Title:

The clinical experience of compassionate use program for avapritinib: implications for drug positioning in the therapeutic scenario of systemic mastocytosis

Year:

2023

Type of item:

Articolo in Rivista

Tipologia ANVUR:

Articolo su rivista

Language:

Inglese

Referee:

No

Name of journal:

THERAPEUTIC ADVANCES IN HEMATOLOGY

ISSN of journal:

2040-6207

N° Volume:

14

Page numbers:

1-14227

Keyword:

avapritinib; case report; cytoreductive treatment; systemic mastocytosis

Short description of contents:

In systemic mastocytosis, cytoreductive treatment is indicated for advanced systemic mastocytosis (AdvSM) variants. The treatment scenario is rapidly diversifying especially with the introduction of KIT tyrosine kinase inhibitors. Avapritinib is a second-generation potent and selective inhibitor of the mutant KIT D816V that, based on the results of pivotal clinical trials, was approved for the treatment of adults with AdvSM by the regulatory agencies US FDA and EMA. The present article reports the experience of treating SM patients with avapritinib in an Italian compassionate use program. The data from our case series confirm the drug as being active after multiple lines of treatment allowing rapid achievement of profound responses, making it also an effective bridging strategy to allogeneic transplant in eligible patients. However, the anticipated wider use of avapritinib in the near future will require careful monitoring of side effects, especially in heavily pretreated patients.

Product ID:

139896

Handle IRIS:

11562/1127400

Last Modified:

October 31, 2024

Bibliographic citation:

Mannelli, Francesco; Crupi, Francesca; Zanotti, Roberta; Pagano, Livio; Rapezzi, Davide; Tanasi, Ilaria; Criscuolo, Marianna; Bonifacio, Massimiliano; Fresa, Alberto; Guglielmelli, Paola; Vannucchi, Alessandro M, The clinical experience of compassionate use program for avapritinib: implications for drug positioning in the therapeutic scenario of systemic mastocytosis «THERAPEUTIC ADVANCES IN HEMATOLOGY» , vol. 14 , 2023 , pp. 1-14227

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