Oral vinorelbine in young patients with desmoid-type fibromatosis

Autor: Laure Kornreich, Daniel Orbach, Nayla Nicolas, Hervé J Brisse, Pablo Berlanga, Anne-Sophie Defachelles, Ludovic Mansuy, Cécile Verite, Laure Saumet, Marie Karanian, Nadège Corradini
Rok vydání: 2023
Předmět:
Zdroj: Tumori Journal. :030089162311698
ISSN: 2038-2529
0300-8916
DOI: 10.1177/03008916231169806
Popis: Background: Desmoid-type fibromatosis are rare intermediate tumors in children and adolescents. Owing to local aggressiveness and relapse, systemic treatment for symptomatic advanced or progressive forms is recommended. Following promising results in adult patients, oral vinorelbine is investigated in young patients. Methods: A retrospective review of young patients (Results: From 2005 to 2020, 24 patients (median age 13.9 years [range, 1.0-23.0]) received oral vinorelbine. Median number of prior systemic lines of treatment was 1 (range, 0-2), mainly based on intravenous low dose methotrexate and vinblastine. Before vinorelbine initiation, all patients had a progressive disease: radiological for 19, radiological and clinical (pain) for three and only clinical for two. Oral vinorelbine was delivered for a median duration of 12 months (range, 1-42). The toxicity profile was favorable, with no grade 3-4 event. Overall response estimated on 23 evaluable patients according to RECIST 1.1 criteria was three partial responses (13%), 18 stabilization (78%) and two progressive disease (9%). Overall progression-free survival was 89.3% (95% confidential intervals 75.2-100) at 24 months. Four stable tumors according to standard RECIST criteria displayed a partial response with > 65% tumor volume reduction. Among 21 informative patients, the estimated fibrosis score decreased for 15 patients, was stable for four patients and increased for two patients. Conclusion: Oral vinorelbine seems to be effective to control advanced or progressive desmoid type fibromatosis in young patients, with a well-tolerated profile. These results support testing this drug as first-line alone or in combination to improve response rate while preserving quality of life.
Databáze: OpenAIRE
Externí odkaz: