NT-proBNP provides prognostic information beyond a conventional risk marker model in patients with ACHD and can reliably exclude the risk of death and heart failure. Elevated levels of NT-proBNP, high-sensitive troponin-T, and growth-differentiation factor 15 identify patients at highest risk of cardiovascular events. These biomarkers therefore may play an important role in the monitoring and management of patients with ACHD.

Objective: To assess tumor behavior and the efficacy of active surveillance (AS) in patients with desmoid-type fibromatosis (DTF). Summary of Background Data: AS is recommended as initial management for DTF patients. Prospective data regarding the results of AS are lacking. Methods: In this multicenter prospective cohort study (NTR4714), adult patients with non-intraabdominal DTF were followed during an initial AS approach for 3 years. Tumor behavior was evaluated according to Response Evaluation Criteria in Solid Tumors. Cumulative incidence of the start of an active treatment and progression-free survival (PFS) were calculated using the Kaplan-Meier method. Factors predictive for start of active treatment were assessed by Cox regression analyses. Results: A total of 105 patients started with AS. Median tumor size at baseline was 4.1cm (interquartile range 3.0–6.6). Fifty-seven patients had a T41A CTNNB1 mutation; 14 patients a S45F CTNNB1 mutation. At 3 years, cumulative incidence of the start of active treatment was 30% (95% confidence interval [CI] 21–39) and PFS was 58% (95% CI 49–69). Median time to start active treatment and PFS were not reached at a median follow-up of 33.7 months. During AS, 32% of patients had stable disease, 28% regressed, and 40% demonstrated initial progression. Larger tumor size (≥5 cm; hazard ratio = 2.38 [95% CI 1.15–4.90]) and S45F mutation (hazard ratio = 6.24 [95% CI 1.92–20.30]) were associated with the start of active treatment. Conclusions: The majority DTF patients undergoing AS do not need an active treatment and experience stable or regressive disease, even after initial progression. Knowledge about the natural behavior of DTF will help to tailor the follow-up schedule to the individual patient.

Background: This study evaluates the results of the active surveillance (AS) approach in adult patients with desmoid-type fibromatosis (DTF) because AS is advocated as a front-line approach for DTF in the European consensus guidelines. Methods: A systematic literature search was conducted (December 19th, 2019, updated on April 14th, 2020. Studies describing the outcomes of the AS approach were included. The PRISMA guidelines were used. Results: Twenty-five articles were included for data retrieval. Forty-two percent of reported patients (1480 of 3527 patients) received AS, the majority were women and the majority had a primary tumour. The median age at diagnosis ranged from 28 to 59 years. Common tumour sites were the extremities/girdles (n Z 273), the abdominal wall (n Z 253) and the trunk (n Z 153). The median reported percentage of progressive disease, stable disease and partial response was 20% (interquartile range [IQR]: 13e35%), 59% (IQR: 37e69%) and 19% (IQR 3e23%), respectively. In 640 patients, the outcome was not specified. The median reported percentage of shifting to an active form of treatment was 29%, most commonly to systemic treatment (n Z 195) and surgery (n Z 107). The reported median follow-up time ranged between 8 and 73 months. The reported median time to progression and/or initiation of the subgroup shifting from AS to 'active' therapy ranged from 6.3 months to 19.7 months. Conclusion:The majority of patients undergoing AS have either stable disease or a partial response, and about one-third of patients shift to an active form of treatment. Selecting

Desmoid-type fibromatosis (DTF) is a rare, soft-tissue tumour. These tumours do not metastasize, but their local aggressive tumour growth and unpredictable behaviour can have a significant impact on health-related quality of life (HRQoL). Little is known about which DTF patients are particularly affected by an impaired HRQoL. The objectives of this study were to assess HRQoL among different groups of DTF patients and to investigate which socio-demographic and clinical characteristics were associated with DTF-specific HRQoL. A cross-sectional study was conducted among DTF patients from the United Kingdom and the Netherlands. HRQoL was assessed using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30), accompanied by the DTF-QoL to assess DTF-specific HRQoL. The scores were compared amongst subgroups, based on the socio-demographic and clinical characteristics of DTF patients. Multiple linear regression analyses with a backward elimination were conducted to identify the factors associated with DTF-specific HRQoL. A total of 235 DTF patients completed the questionnaires. Female patients, patients with more than two comorbidities, or patients who received treatment other than only active surveillance (AS) or surgery scored significantly worse on the subscales of both the EORTC QLQ-C30 and DTF-QoL. Patients that were ≥ 40 years scored significantly worse on the physical functioning scale of the EORTC QLQ-C30, while younger patients (18–39 years) scored significantly worse on several DTF-QoL subscales. Differences in the DTF-QoL subscales were found for tumour location, time since diagnosis and the presence of recurrent disease. Furthermore, treatments other than AS or surgery only, female sex, younger age and the presence of comorbidities were most frequently associated with worse scores on the DTF-QoL subscales. This study showed that (DTF-specific) HRQoL differs between groups of DTF patients. Awareness of these HRQoL differences could help to provide better, personalised care that is tailored to the needs of a specific subgroup.

Sporadic desmoid-type fibromatosis (DTF) is a rare soft tissue tumour with an unpredictable clinical course. These tumours are incapable of metastasising, but their local aggressive tumour growth and tendency to recur locally can result in a substantial symptom burden. Measuring the impact of DTF on health-related quality of life (HRQoL) can be challenging due to the variable clinical presentation of the disease. Therefore, a HRQoL instrument assessing DTF-specific issues is needed. The QUALIFIED study aims to (1) pre-test a previously developed DTF-specific HRQoL tool (the DTF-QoL); (2) evaluate prevalence of HRQoL issues in adult DTF patients; and (3) identify subgroups at risk of impaired HRQoL. This study (NCT04289077) is an international, multicentre, cross-sectional, observational cohort study. Patients ≥ 18 years with sporadic DTF from the Netherlands and the United Kingdom will be invited to complete a set of questionnaires specifically composed for this patient group. Questionnaires will be completed using PROFILES (Patient Reported Outcomes Following Initial treatment and Long-term Evaluation of Survivorship). Analyses will include testing the psychometric properties of the DTF-QoL and evaluating the prevalence of HRQoL issues using the DTF-QoL, EORTC QOL-C30 and EQ-5D-5L, among other questionnaires. This study will provide insight into HRQoL issues experienced by patients with DTF. Awareness of these issues and the implementation of the DTF-QoL in research and clinical practice can help to improve overall HRQoL and to provide personalised care.