CIC ‐rearranged sarcoma is a recently established, ultra‐rare, molecularly defined sarcoma subtype. We aimed to further characterise clinical features of CIC ‐rearranged sarcomas and explore clinical management including systemic treatments and outcomes. Methods A multi‐centre retrospective cohort study of patients diagnosed between 2014–2019. Results Eighteen patients were identified. The median age was 27 years (range 13–56), 10 patients were male (56%), 11 patients (61%) had localised disease and 7 patients had advanced (metastatic or unresectable) disease at diagnosis. Of 11 patients with localised disease at diagnosis, median overall survival (OS) was 40.6 months and the 1‐, 2‐ and 5‐year OS estimates were 82%, 64% and 34% respectively. Nine patients (82%) underwent surgery (all had R0 resections), 8 (73%) patients received radiotherapy to the primary site (median dose 57Gy in 28 fractions), and 8 (73%) patients received chemotherapy (predominantly Ewing‐based regimens). Metastases developed in 55% with a median time to recurrence of 10.5 months. In patients with advanced disease at diagnosis, median OS was 12.6 months (95% CI 5.1–20.1), 1‐year OS was 57%. Median progression‐free survival was 5.8 months (95% CI 4.5–7.2). Durable systemic therapy responses occurred infrequently with a median duration of systemic treatment response of 2.1 months. One durable complete response of metastatic disease to VDC/IE chemotherapy was seen. Responses to pazopanib ( n = 1) and pembrolizumab ( n = 1) were not seen. Conclusion In this series, CIC ‐rearranged sarcomas affected young adults and had a high incidence of presenting with, or developing, metastatic disease. The prognosis overall was poor. In advanced disease, durable systemic therapy responses were infrequent.
Background One of the most frequent complications of left ventricular assist device (LVAD) implantation is the development of right ventricular (RV) failure, which occurs in 10%-40% of patients. It is, therefore, essential to identify which LVAD candidates are at risk for RV failure and will require additional RV mechanical support with a biventricular assist device (BiVAD). However, the echocardiographic assessment of the RV is challenging due to its complex geometry and marked load dependence of its function indices. To our knowledge, there is no published evidence regarding which specific RV echocardiographic parameters should inform this decision in the paediatric population. Aims We sought to determine which pre-operative RV echocardiographic parameters best correlate with the need for a BiVAD, as opposed to LVAD alone, in a paediatric population undergoing VAD insertion at a tertiary care institution. Methods Retrospective review of the pre-operative echocardiograms of children (<18 years) with dilated cardiomyopathy undergoing elective VAD implantation at our institution, from November 2007 to December 2018. Preoperative quantitative and qualitative RV echocardiographic parameters described in the literature to be associated with RV failure after LVAD implantation in adults were collected. Moreover, qualitative RV function was independently assessed by three echocardiographers, blinded to the outcome of the patients. Results 89 patients were included, 39 (43.8%) males, median age = 1.7 years (IQR = 6.9), median weight = 11.6 kg (IQR = 13.8). 49 (55.1%) patients had an LVAD implanted, whereas 40 (44.9%) were deemed to need biventricular support. 45 (50.6%) patients received an EXCOR Berlin Heart, 16 (18%) a HeartWare HVAD and 28 (31.4%) a Levitronix Centrimag device. Requirement of BiVAD support was significantly more common in patients with moderate to severe RV impairment, as per expert assessment (OR = 2.864; 95% CI: 1.188–6.903, p=0.018), and tricuspid regurgitation > grade III (OR = 3.154; 95% CI: 1.124–8.850, p=0.025). All the other parameters collected – tricuspid annular plane systolic excursion (TAPSE), tricuspid regurgitant jet velocity, tricuspid regurgitation duration corrected for heart rate, RV tissue Doppler indices, RV fractional area change, and RV/LV diameter ratio– were not significantly different among groups. Conclusions In our paediatric population, expert assessment of RV function and degree of tricuspid regurgitation were strong predictors of RV failure among patients undergoing LVAD implantation, allowing for pre-emptive RVAD implantation and improving patient outcomes.
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