Platinum-Based Regimens Are Active in Advanced Pediatric-Type Rhabdomyosarcoma in Adults and Depending on HMGB1 Expression

• Published in: International journal of molecular sciences Vol. 24; no. 1; p. 856
• Main Authors: Hindi, Nadia, Carrillo-García, Jaime, Blanco-Alcaina, Elena, Renshaw, Marta, Luna, Pablo, Durán, José, Jiménez, Natalia, Sancho, Pilar, Ramos, Rafael, Moura, David S, Martín-Broto, Javier
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI 03.01.2023; MDPI AG
• Subjects: Adult; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; BOMP-EPI; Child; cisplatin; Cisplatin - therapeutic use; Etoposide - therapeutic use; Female; HMGA2; HMGB1; HMGB1 Protein - metabolism; HMGB2; Humans; Male; Neoplasm Recurrence, Local - drug therapy; Retrospective Studies; rhabdomyosarcoma; Rhabdomyosarcoma - drug therapy; Rhabdomyosarcoma - pathology; Rhabdomyosarcoma, Embryonal - drug therapy; Rhabdomyosarcoma, Embryonal - metabolism; Vincristine - therapeutic use; HMGB2; HMGA2; BOMP-EPI; HMGB1; cisplatin; rhabdomyosarcoma
• ISSN: 1422-0067; 1661-6596; 1422-0067
• DOI: 10.3390/ijms24010856

Rhabdomyosarcoma (RMS) in adults is a rare and aggressive disease, which lacks standard therapies for relapsed or advanced disease. This retrospective study aimed to describe the activity of BOMP-EPI (bleomycin, vincristine, methotrexate and cisplatin alternating with etoposide, cisplatin and ifosfamide), an alternative platinum-based regimen, in adult patients with relapsed/metastatic RMS. In the study, 10 patients with RMS with a median age at diagnosis of 20.8 years and a female/male distribution of 6/4 received a mean of 2.5 cycles of BOMP-EPI. The best RECIST response was a complete response in 1/10 (10%) patients, a partial response in 5/10 (50%), stable disease in 3/10 (30%) and progression in 1/10 (10%). With a median follow-up in the alive patients from the start of therapy of 30.5 months (15.7-258), all patients progressed with a median progression-free survival of 8.47 months (95% CI 8.1-8.8), and 7/10 patients died with a median overall survival of 24.7 months (95% CI 13.7-35.6). BOMP-EPI was an active chemotherapy regimen in adults with pediatric-type metastatic RMS, with outcomes in terms of survival that seem superior to what was expected for this poor-prognosis population. Low HMGB1 expression level was identified as a predictive factor of better response to this treatment.