Desmoid-type fibromatosis
Conditions
Interventions
Sponsors
UMC Utrecht
Eligibility
Inclusion criteria
Inclusion criteria: - Patients > 18 years - Histological evidence of DTF - Patients with failure of active surveillance* for their present manifestation of DTF - Desmoid tumor must be targetable with MR-HIFU device - Desmoid tumor must be visible on pre-treatment MR-imaging - Patient is able to fit in the MRI gantry - Capable to understand and sign informed consent *failure of active surveillance due to tumor growth and/or new or worsening symptoms
Exclusion criteria
Exclusion criteria: - Personal or family history of familial adenomatous polyposis (FAP) - Intra-abdominal tumor localization - Patients with a tumor greater than 10 centimeters - Patients who have undergone prior active treatment (systemic therapy, radiotherapy, prior ablation) for the present manifestation of DTF - Patients with recurrent disease within 12 months after treatment for their prior desmoid tumor - Patients weighing more than 140 kilograms - Pregnancy - Contra indications to MRI, MRI contrast agents or sedation - Unavoidable critical structures or dense tissues in target area* - Any other condition, which in the opinion of the investigators, would put the patient at increased risk or otherwise make the patients unsuitable for this study *as judged by the operator. e.g.: nerve bundles, skin, extensive scarring, non-targeted bones, air (e.g. hollow viscera), (external) fixation device, surgical clips
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Analysis of efficacy: MR-HIFU will be considered as an effective treatment modality for DTF patients if a success is observed in >3 of the 13 patients. If a success is observed in =3 of the 13 DTF patients, it is concluded that MR-HIFU is not an effective treatment strategy in these patients, and should not be further investigated. Otherwise, this study will conclude that this treatment strategy is effective and sufficiently promising, and warrants further investigation in this patient population. The primary outcome will be presented in absolute numbers and proportions. | — |
Secondary
| Measure | Time frame |
|---|---|
| Presence of non-perfused volume on MRI The amount of non-perfused volume will be calculated as a percentage of the total targeted volume. This amount will be reported as median percentage with corresponding IQR. Change in tumor volume Change in total and vital tumor volume will be reported as median percentage with corresponding IQR. Results will be plotted in a waterfall plot. Response rate The response rate will be calculated as the number of patients with PD, SD, PR, and CR 12 months after completion of treatment divided by the total number of patients treated with MR-HIFU. Response rate will be presented in absolute numbers and proportions. Safety of MR-HIFU treatment Safety will be measured by the number of patients who experienced grade III-IV toxicity. This number will be described as an absolute number and proportion of all the included patients. Change in symptoms A mean score or median score will be calculated with standard deviation (SD) or IQR of the PGIS and PGIC. Change in pain scores For patients with painful tumors, defined as a score of at least 4 out of 10 on the NRS, the worst and average daily NRS pain scores will be compared before and at 3, 6 and 12 months after completion of treatment. A decrease in worst and average pain scores of = 2 points will be considered as pain reduction after MR-HIFU treatment. Median decrease with corresponding IQR will be reported. Decrease of at least 2 points between baseline and follow-up NRS pain scores will be tested using a paired t-test or an exact one-sided Wilcoxon test. Health-related Quality of Life. The mean score or median score per scale of the EORTC QLQ-C30 questionnaire (symptom scales, single item scales, global health scale, and functioning scales) will be calculated with the standard deviation (SD) or interquartile range (IQR). Additionally, a summary score will be calculated based on all scales except financial difficulties and the global quality of life score, to reflect overall quali | — |
Contacts
Public ContactAnne-Rose Schut
University Medical Center Utrecht
Outcome results
None listed