Pubblicazioni

Autori:

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

Titolo:

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

Anno:

2023

Tipologia prodotto:

Articolo in Rivista

Tipologia ANVUR:

Articolo su rivista

Lingua:

Inglese

Referee:

No

Nome rivista:

THERAPEUTIC ADVANCES IN HEMATOLOGY

ISSN Rivista:

2040-6207

N° Volume:

14

Intervallo pagine:

1-14227

Parole chiave:

avapritinib; case report; cytoreductive treatment; systemic mastocytosis

Breve descrizione dei contenuti:

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.

Id prodotto:

139896

Handle IRIS:

11562/1127400

ultima modifica:

31 ottobre 2024

Citazione bibliografica:

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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