Locoregional Control and Survival in Children, Adolescents, and Young Adults With Localized Head and Neck Alveolar Rhabdomyosarcoma-The French Experience.

Roxane Machavoine (1) , Sylvie Helfre (2) , Valérie Bernier (3) , Stéphanie Bolle (4) , Julie Leseur (5) , Nadège Corradini (6) , Angélique Rome (7) , Anne-Sophie Defachelles (8) , Sophie Deneuve (9) , Sophie Bernard , Pierre Fayoux (10) , Richard Nicollas (7) , Michel Mondain (11) , Romain Luscan (1) , Françoise Denoyelle (1) , François Simon (1) , Natacha Kadlub (1) , Frédéric Kolb (4) , Jean-François Honart (12) , Daniel Orbach (4) , Véronique Minard-Colin (13) , Antoine Moya-Plana (4) , Vincent Couloigner (1)

1 Hôpital Necker - Enfants Malades [AP-HP]
2 Institut Curie [Paris]
3 UNICANCER/ICL - Institut de Cancérologie de Lorraine - Alexis Vautrin [Nancy]
4 IGR - Institut Gustave Roussy
5 CRLCC - CRLCC Eugène Marquis
6 IHOPe - Institut d'hématologie et d'oncologie pédiatrique [CHU - HCL]
7 TIMONE - Hôpital de la Timone [CHU - APHM]
8 UNICANCER/Lille - Centre Régional de Lutte contre le Cancer Oscar Lambret [Lille]
9 Centre Léon Bérard [Lyon]
10 METRICS - Evaluation des technologies de santé et des pratiques médicales - ULR 2694
11 CHU Montpellier
12 Département de chirurgie plastique [Gustave Roussy]
13 ITIC - Immunologie anti-tumorale et immunothérapie des cancers

Introduction: The head and neck (HN) are the most frequent sites of pediatric rhabdomyosarcoma (RMS). Alveolar RMS (ARMS) represents ~20% of all RMS cases and frequently spread to lymph nodes (LNs). The aim was to report locoregional control, event-free survival (EFS), and overall survival (OS), according to clinical and pathological features, LN staging, and treatment modalities. Methods: The study included all patients prospectively enrolled in EpSSG RMS 2005 study under 21 years of age with localized HN ARMS and diagnosed between 2005 and 2016 in France. Medical data including imaging, surgical report, and radiation therapy planes were analyzed. Results: Forty-eight patients (median age 6 years; range 4 months−21 years), corresponding to 30 parameningeal and 18 non-parameningeal ARMS, were included. There were 33 boys (69%). Tumor locations included the following: orbit (n = 7) among which four cases had bone erosion, paranasal sinuses and nasal cavity (n = 16), deep facial spaces (n = 10), nasolabial fold (n = 8), and other non-parameningeal HN sites (n = 7). A fusion transcript of PAX3-FOXO1 or PAX7-FOXO1 was expressed in 33 of the 45 cases (73%) with molecular analysis. At diagnosis, 10 patients had primary resection of the primary tumor (PRPT) (none with microscopic complete resection) and 9 had LN staging. After induction chemotherapy, 26 patients (54%) had secondary resection of the primary tumor (SRPT) and 13 patients (27%) had cervical LN dissection. A total of 43 patients (90%) were treated with radiation therapy. With a median follow-up of 7 years (range 2–13 years), 5-year OS and EFS were 78% (95% CI, 63–88%) and 66% (95% CI, 51–78%), respectively. We observed 16 events (10 deaths): 4 local, 4 regional, 1 local and regional, and 7 metastatic. In univariate analysis, OS was only superior for patients under 10 years of age (p = 0.002), while FOXO1-negative ARMS, SRPT for parameningeal ARMS, and LN surgery were associated with significantly better EFS. Conclusion: Our study confirms a better outcome for fusion-negative ARMS and ARMS in children under 10 years. Moreover, LN surgery and SRPT of parameningeal tumor may improve EFS of ARMS. Larger studies are needed to confirm our findings.

Mots clés: alveolar rhabdomyosarcoma (ARMS); head and neck neoplasm; children; neck dissection; survival

Domaines: Sciences du Vivant [q-bio]

Roxane Machavoine, Sylvie Helfre, Valérie Bernier, Stéphanie Bolle, Julie Leseur, et al.. Locoregional Control and Survival in Children, Adolescents, and Young Adults With Localized Head and Neck Alveolar Rhabdomyosarcoma-The French Experience.. Frontiers in Pediatrics, 2022, Frontiers in Pediatrics, 9, pp.783754. ⟨10.3389/fped.2021.783754⟩. ⟨hal-04572907⟩