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Background Sarcomatous transformation of Paget's disease of bone is a very rare, highly fatal complication. Isolated cohort studies have characterized these findings many times. Still, little has been established concerning the effects of patient and tumor characteristics and treatment type on the oncologic prognosis when defined as local recurrence-free, metastatic disease-free, disease-specific, and overall survival.
Questions/Purposes (1) What are the oncologic and clinical characteristics of patients who develop and are diagnosed with Paget’s sarcoma? (2) What are the outcomes when examining variables such as sarcoma type, presentation at diagnosis and treatment in Paget’s sarcoma patients? (3) How have treatment and oncologic prognosis changed in recent decades?
Methods A literature review of (n=1,001) Paget’s sarcoma cases, retrieved from previous studies and public databases, and a retrospective analysis on a cohort of all adult patients with Paget’s disease of bone (n=1,872) admitted to two large academic centers between 1979 and 2019 was performed. Medical records and the Social Security Index assessed patient survival. Surgical reports were reviewed to determine margin characteristics and intervention type. Incidences of metastasis and recurrence were based on patient notes and biopsy report review. This two-part study describes the effects of patient and tumor characteristics and treatment type on oncologic prognosis.
Results Pooled data from our literature review demonstrated a 5-year overall survival rate of 7.9%, in which patient age was significantly associated with worse survival prognosis (p=0.026). Treatment with surgery and adjuvant therapy was significantly associated with improved overall survival (p=0.006) compared to surgery alone or non-surgical treatment. Examining overall trends historically, neither presenting age nor survival appear to have a significant association with the year of diagnosis, demonstrating an R2 of 0.003 (p=0.48) for age at diagnosis and 0.023 (p=0.39) for survival from 1960 and 2016. From our retrospective cohort study, 32 out of 1,872 Paget’s patients were determined to have Paget’s sarcoma, indicating a prevalence of 1.7% for our database. The 5-year local recurrence-free, metastatic disease-free, disease-specific, and overall survival rates were 12.5%, 15.6%, 12.5%, and 18.8%, respectively. Patient age was significantly associated with worse survival outcomes (p=0.005) in our institutions’ patient registry.
Conclusion Including over 1,000 Paget's sarcoma patients in our study greatly strengthens the associations between treatment type and survival. While patients undergoing multimodal therapy reported the greatest longevity, concerns remain for mobility and quality of life. Our cohort study indicates that few factors can effectively predict oncologic outcomes for Paget's sarcoma patients. Age is the strongest underlying factor for poor prognosis. Patient outcomes have not changed significantly in the last 80 years; our literature and cohort study highlights the need for increased clinician awareness and improved treatment modalities for Paget's sarcoma.
Level of Evidence Level III, retrospective comparative study and review of other level III studies.
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