TY - JOUR
T1 - Clinical characteristics and outcomes of adult alveolar rhabdomyosarcoma patients on first-line systemic therapies
T2 - A single-institution cohort
AU - Nakazawa, Michael S.
AU - Livingston, J. Andrew
AU - Zarzour, Maria A.
AU - Bishop, Andrew J.
AU - Ratan, Ravin
AU - Ludwig, Joseph A.
AU - Araujo, Dejka M.
AU - Somaiah, Neeta
AU - Ravi, Vinod
AU - Nassif, Elise F.
AU - Roland, Christina L.
AU - Lazar, Alexander J.
AU - Guadagnolo, B. Ashleigh
AU - Harrison, Douglas J.
AU - Benjamin, Robert S.
AU - Patel, Shreyaskumar R.
AU - Conley, Anthony P.
N1 - Publisher Copyright:
© The Author(s) 2024.
PY - 2024/1/1
Y1 - 2024/1/1
N2 - Background: Rhabdomyosarcomas are the most common soft tissue sarcoma in children, and pediatric alveolar rhabdomyosarcoma (ARMS) prognosis has improved based on cooperative studies. However, in adults, ARMS is significantly rarer, has poorer outcomes, and currently lacks optimal treatment strategies. Objective: This study aimed to evaluate the clinical outcome of an adult ARMS population with different front-line systemic chemotherapies and determine if any chemotherapy regimen is associated with improved survival. Materials and methods: This is a retrospective study of histologically confirmed fusion-positive ARMS patients over 18 years of age, who were treated at MD Anderson Cancer Center (MDACC) from 2004 to 2021 and received systemic chemotherapy. Descriptive clinical statistics were performed, including staging, front-line chemotherapy, multimodal therapy usage, response rates, and survival analyses. Results: 49 ARMS patients who received upfront chemotherapy were identified. Locoregional treatments included radiotherapy (RT) alone (29%, n = 14), surgery alone (10%, n = 5), or both (45%, n = 22). Median overall survival (OS) for the entire cohort was 3.6 years, and the overall response rate to systemic therapy was 89%. No chemotherapy regimen showed OS benefit, specifically analyzing the pediatric-based vincristine, actinomycin-D, cyclophosphamide (VAC) or adult-based vincristine, doxorubicin, ifosfamide (VDI) regimens, even when controlled for other clinical risk factors. Conclusion: In this single-center contemporary series, adult ARMS patient outcomes remain poor. There was no statistically significant OS difference in patients who did or did not receive adult or pediatric based ARMS regimens, although a high overall response rate to chemotherapy was seen across the entire cohort. Based on these observations, further randomized prospective studies are necessary to delineate which frontline chemotherapy regimen is most beneficial in this rare adult cancer.
AB - Background: Rhabdomyosarcomas are the most common soft tissue sarcoma in children, and pediatric alveolar rhabdomyosarcoma (ARMS) prognosis has improved based on cooperative studies. However, in adults, ARMS is significantly rarer, has poorer outcomes, and currently lacks optimal treatment strategies. Objective: This study aimed to evaluate the clinical outcome of an adult ARMS population with different front-line systemic chemotherapies and determine if any chemotherapy regimen is associated with improved survival. Materials and methods: This is a retrospective study of histologically confirmed fusion-positive ARMS patients over 18 years of age, who were treated at MD Anderson Cancer Center (MDACC) from 2004 to 2021 and received systemic chemotherapy. Descriptive clinical statistics were performed, including staging, front-line chemotherapy, multimodal therapy usage, response rates, and survival analyses. Results: 49 ARMS patients who received upfront chemotherapy were identified. Locoregional treatments included radiotherapy (RT) alone (29%, n = 14), surgery alone (10%, n = 5), or both (45%, n = 22). Median overall survival (OS) for the entire cohort was 3.6 years, and the overall response rate to systemic therapy was 89%. No chemotherapy regimen showed OS benefit, specifically analyzing the pediatric-based vincristine, actinomycin-D, cyclophosphamide (VAC) or adult-based vincristine, doxorubicin, ifosfamide (VDI) regimens, even when controlled for other clinical risk factors. Conclusion: In this single-center contemporary series, adult ARMS patient outcomes remain poor. There was no statistically significant OS difference in patients who did or did not receive adult or pediatric based ARMS regimens, although a high overall response rate to chemotherapy was seen across the entire cohort. Based on these observations, further randomized prospective studies are necessary to delineate which frontline chemotherapy regimen is most beneficial in this rare adult cancer.
KW - Sarcoma
KW - alveolar rhabdomyosarcoma
KW - chemotherapy
KW - soft tissue sarcoma
UR - http://www.scopus.com/inward/record.url?scp=85200405244&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85200405244&partnerID=8YFLogxK
U2 - 10.1177/20363613241271669
DO - 10.1177/20363613241271669
M3 - Article
C2 - 39105190
AN - SCOPUS:85200405244
SN - 2036-3605
VL - 16
JO - Rare Tumors
JF - Rare Tumors
ER -