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. 2024 Dec;13(23):e70420.
doi: 10.1002/cam4.70420.

Therapy and Outcomes of Patients with Relapsed Nonmetastatic Rhabdomyosarcoma: A Report from the French Society of Pediatric Oncology Malignant Mesenchymal Tumor Committee

François Sevrin  1 , Emilie Bogart  2 , Daniel Orbach  3 , Marie Cécile Le Deley  2 , Pablo Berlanga  4 , Valérie Bernier  5 , Nadège Corradini  6 , Cindy Fayard  7 , Florent Guerin  8 , Sarah Jannier  9 , Marie Karanian  10 , Stéphanie Proust  11 , Angélique Rome  12 , Cécile Verité  13 , Véronique Minard-Colin  4 , Anne-Sophie Defachelles  1
Affiliations

Therapy and Outcomes of Patients with Relapsed Nonmetastatic Rhabdomyosarcoma: A Report from the French Society of Pediatric Oncology Malignant Mesenchymal Tumor Committee

François Sevrin et al. Cancer Med. 2024 Dec.

Abstract

Background: The prognosis for patients with relapse of localized rhabdomyosarcoma (RMS) remains poor, with limited evidence for optimal second-line therapy. This study describes the management and outcomes of relapsed RMS patients in France.

Methods: We retrospectively reviewed all nonmetastatic RMS patients enrolled in France in the RMS 2005 study who relapsed between 2006 and 2019 after achieving complete local control, defined as complete remission or stable residue ≥ 6 months after treatment completion. Data were extracted from the RMS 2005 database and medical records.

Results: Ninety-five patients relapsed at a median age of 6.0 years (range: 1.0-27.0). The median time from diagnosis to relapse was 17.5 months (range: 7.4-82.0). Most patients had embryonal RMS (65.3%) and local/locoregional relapses (71.6%). The first relapse treatment included chemotherapy (all except two patients), radiotherapy (52.6%), and surgery (48.4%). Second-line chemotherapy yielded a 58.5% objective response rate after 3 ± 1 cycles. Fifty-five patients achieved second complete remission. With a median follow-up of 7.2 years from the first relapse (range: 0.3-11.3), 5-year progression-free survival was 26% (95% CI: 18-36), and 5-year overall survival was 35% (95% CI: 25-45). Importantly, no patient survived relapse without receiving locoregional treatment (surgery and/or radiotherapy).

Conclusion: This study confirmed the inconsistencies in therapy and the poor prognosis for relapsed RMS but highlighted the potential for long-term survival in patients who received surgery and/or radiotherapy, emphasizing the crucial role of achieving local control in improving outcomes at relapse.

Keywords: outcome; prognosis; relapse; rhabdomyosarcoma; salvage therapy; second‐line therapy.

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Conflict of interest statement

The authors have nothing to report.

Daniel Orbach reports doing consultant work for the French Larotrectinib transparency committee (consultancy agreement signed with the institution). An independent translational research project conducted by him is partially supported by Bayer (investigation‐supported research). He also reports having consultant activity for Lilly, Merk, Bayer Healthcare, Sanofi, Hoffman La Roche, Novartis, and Eusapharm. Veronique Minard‐Colin reports having consultant activity for Roche (AdBoard; no personal benefits). The other authors have no conflicts of interest to disclose.

Figures

FIGURE 1
FIGURE 1
Kaplan–Meier survival curves. Progression‐free survival (PFS) (A) and overall survival (OS) (B) from the first relapse in all 95 patients included in the study according to the type of recurrence. PFS (C) and OS (D) from the first relapse in 68 patients with nonmetastatic relapse according to the time interval to relapse.
See this image and copyright information in PMC

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