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

被引：1

作者：

Mannelli, Francesco ^{[1
]}

Crupi, Francesca ^{[2
,3
]}

Zanotti, Roberta ^{[4
,5
]}

Pagano, Livio ^{[6
,7
]}

Rapezzi, Davide ^{[8
]}

Tanasi, Ilaria ^{[4
,5
]}

Criscuolo, Marianna ^{[6
]}

Bonifacio, Massimiliano ^{[4
,5
]}

Fresa, Alberto ^{[6
,7
]}

Guglielmelli, Paola ^{[2
,3
]}

Vannucchi, Alessandro M. ^{[2
,3
]}

机构：

[1] Univ Firenze, Ctr Ric & Innovaz Malattie Mieloproliferat, SOD Ematol, AOU Careggi, Largo Brambilla 3, I-50134 Florence, Italy

[2] Azienda Osped Univ Careggi, Ctr Ric & Innovaz Malattie Mieloproliferat, SOD Ematol, Florence, Italy

[3] Univ, Denothe Excellence Ctr, Dipartimento Med Sperimentale & Clin, Florence, Italy

[4] Univ Integrata Verona, Azienda Osped, Unita Ematol, Dipartimento Med, Verona, Italy

[5] Univ Integrata Verona, Azienda Osped, Grp Interdisciplinare Studio Mastocitosi, Verona, Italy

[6] Fdn Policlin Univ A Gemelli IRCCS, Dipartimento Diagnost Immagini, Radioterapia Oncolog Ematol, Rome, Italy

[7] Univ Cattolica Sacro Cuore, Dipartimento Sci Radiolog Ematolog, Sez Ematol, Rome, Italy

[8] Osped S Croce & Carle, Unita Ematol, Cuneo, Italy

来源：

THERAPEUTIC ADVANCES IN HEMATOLOGY | 2023年 / 14卷

关键词：

avapritinib; case report; cytoreductive treatment; systemic mastocytosis; HEALTH-ORGANIZATION CLASSIFICATION; PATHOGENESIS; DIAGNOSIS; NEOPLASMS; MUTATION; NETWORK;

D O I：

10.1177/20406207231205643

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页数：14

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