Desmoid tumor (DT; other synonymously used terms: Desmoid-type fibromatosis, aggressive fibromatosis) is a rare and locally aggressive monoclonal, fibroblastic proliferation characterised by a variable and often unpredictable clinical course. Previously surgery was the standard primary treatment modality; however, in recent years a paradigm shift towards a more conservative management has been introduced and an effort to harmonise the strategy amongst clinicians has been made. We present herein an evidence-based, joint global consensus guideline approach to the management of this disease focussing on: molecular genetics, indications for an active treatment, and available systemic therapeutic options. This paper follows a one-day consensus meeting held in Milan, Italy, in June 2018 under the auspices of the European Reference Network for rare solid adult cancers, EURACAN, the European Organisation for Research and Treatment of Cancer (EORTC) Soft Tissue and Bone Sarcoma Group (STBSG) as well as Sarcoma Patients Euro-Net (SPAEN) and The Desmoid tumour Research Foundation (DTRF). The meeting brought

The risk of non-SN metastasis with a low-volume metastasis in the SN is around 10-15 per cent, depending on the method of detection of SN involvement. This should be taken into account when assessing the risk of omission of axillary dissection after a positive SN biopsy yielding micrometastatic or immunohistochemically positive SNs.

While they account for one-fifth of new cancer cases, rare cancers are difficult to study. A higher than average degree of uncertainty should be accommodated for clinical as well as for population-based decision making. Rules of rational decision making in conditions of uncertainty should be rigorously followed and would need widely informative clinical trials. In principle, any piece of new evidence would need to be exploited in rare cancers. Methodologies to explicitly weigh and combine all the available evidence should be refined, and the Bayesian logic can be instrumental to this end. Likewise, Bayesian-design trials may help optimize the low number of patients liable to be enrolled in clinical studies on rare cancers, as well as adaptive trials in general, with their inherent potential of flexibility when properly applied. While clinical studies are the mainstay to test hypotheses, the potential of electronic patient records should be exploited to generate new hypotheses, to create external controls for future studies (when internal controls are unpractical), to study effectiveness of new treatments in real conditions. Framework study protocols in specific rare cancers to stepwisely test sets of new agents, as from the early post-phase I development stage, should be encouraged. Also the compassionate and the off-label settings should be exploited to generate new evidence, and flexible regulatory innovations such as adaptive licensing could convey new agents early to rare cancer patients, while generating evidence. Though validation of surrogate end points is problematic in rare cancers, the use of an updated notion of tumor response may be of great value in the single patient to optimize the use of therapies, all the more the new ones. Disease-based communities, involving clinicians and patients, should be regularly consulted by regulatory bodies when setting their policies on drug approval and reimbursement in specific rare cancers .

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Sixty-two patients with the short bowel syndrome (30-150 cm) were managed by continuous enteral nutrition (CEN) in the early adaptive phase. In all, 82 per cent were referrals from other units and 85 per cent of referrals had failure of one or more organ systems on admission. There were intra-abdominal abscesses in 41 per cent of patients and 37 per cent had an enterocutaneous fistula. The diet included polysaccharides, medium chain triglycerides and protein hydrolysates, mixed with a high-viscosity tapioca suspension. An elemental diet was used initially in 15 per cent of patients. Thirty-three patients had an interruption of the gastrointestinal tract by a temporary enterostomy. Chyme was re-infused into the distal intestine in 20 cases. 'Zero-time' was taken as the time of operation or, for referred patients treated conservatively, the date of admission. CEN was commenced at a mean of 14 days from zero-time. Total parenteral nutrition could be discontinued at a mean of 36 days and exclusive oral alimentation was resumed at a mean of 87 days. Patients with small bowel longer than 80 cm attained enteral autonomy earlier than patients with a shorter length. Mean faecal volume did not increase following institution of CEN, suggesting tolerance to the high-viscosity diet. In cases with re-infusion of enteric content, the distal circuit (length of distal small intestine 46 cm) was able to absorb 70 per cent of the volume re-infused (mean volume 2700 ml). Body weight and nutritional markers increased significantly during the course of CEN. This study suggests that enteral autonomy can be attained early in the short bowel syndrome, even under challenging conditions. Elemental formulae do not appear to offer a benefit over polymeric diets.