Quality of Life of Patients With Desmoid-type Fibromatosis

NCT04289077 | Completed

• 1 other identifier: MEC-2019-0816
• Study Type: observational
• Target: 156
• Locations: 2 countries
• Sites: 4

Brief Summary

Rationale: Desmoid-type fibromatosis (DTF) is a rare, histologically benign, soft tissue tumour. Although incapable of metastasizing, the clinical course is unpredictable and can be aggressive because of local invasive growth. Various treatments are available including; surgical resection, radiotherapy, hormonal therapy and chemotherapy. Nowadays there is a trend towards a more conservative strategy with a wait and see policy because of high recurrence rates after surgical resection. Health-related quality of life (HRQL) is a corner stone in treatment choice and can be used during the disease for monitoring the impact of the disease on physical, psychological and social level. Additionally, HRQL can be in important endpoint for future clinical trials. Today, no HRQL-tools are available which capture the needs of DTF patients. The hypothesis is that patients with DTF have issues on several HRQL domains including physical, social and emotional well-being. For this reason we developed a list of items based on previous research. This study aims to evaluate HRQL issues experienced by DTF patients. Purpose: Multi-centre, cross-sectional, observational study to measure HRQL of DTF patients and to evaluate the prevalence of the experienced problems.

Conditions

Desmoid Tumor; Desmoid-type Fibromatosis

Keywords

Health-related quality of life; Quality of life

Outcome Measures

Primary Outcomes (3)

• EORTC QLQ-C30 questionnaire (European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core-30; version 3.0)

  Each question will be scored: not at all (1), a little (2), quite a bit (3), very much (4). The scores of the EORTC-QLQ30 will be calculated using a Likert scale from 1-4. The scoring manual of the EORTC will be followed. After linear transformation, all scales and single item measures range in score from 0-100. Scores for each scale will be reported as mean (SD), or as median (IQR). A higher score on the functional scales and global quality of life means better functioning and HRQoL, whereas a higher score on the symptom scales means more complaints. The EORTC QLQ-C30 summary score will be calculated using the mean scores of the function scales and the reversed mean scores of the symptom scales and represented as the mean of the combined 13 QLQ-C30 scale scores (financial impact and global health status excluded). A higher summary score represented a better outcome.

  Participants have to fill out the questionnaire once and have to complete the questionnaire within 3 months after the invitation. A reminder for participation will be send one month after the initial invitation.

• DTF-QoL questionnaire (Desmoid-type fibromatosis Quality of Life Questionnaire)

  The scores for each item of the DTF-QoL will be calculated using a Likert scale from 1-4. The scores of each item will be calculated as mean ± SD, or as median (IQR). These scores (median or mean) will be reported per item.

  Participants have to fill out the questionnaire once and have to complete the questionnaire within 3 months after the invitation. A reminder for participation will be send one month after the initial invitation.

• EQ-5D-5L questionnaire (EuroQol five-dimensional questionnaire; version 1.0)

  Measures health-related quality of life on five dimensions of health: mobility, self-care, usual activities, pain-discomfort, and anxiety/depression. Additionally two questions aim to rank a patients' health (scale 0-100, with 100 representing a better health). Each of the five dimensions can be divided into five levels of perceived problems: no problems (level 1), slight problems (level 2), moderate problems (level 3), severe problems (level 4) and extreme problems (level). Health states can be converted into single index values. The outcomes will be reported as frequency (proportion) of reported problems for each level and for each dimension. The Visual Analogue Scale (VAS) data will be presented as a mean value (SD). In case of skewed data, median values and IQR will be used.

  Participants have to fill out the questionnaire once and have to complete the questionnaire within 3 months after the invitation. A reminder for participation will be send one month after the initial invitation.

Secondary Outcomes (4)

• EORTC QLQ-C30 questionnaire (European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core-30; version 3.0)

  Participants have to fill out the questionnaire once and have to complete the questionnaire within 3 months after the invitation. A reminder for participation will be send one month after the initial invitation.

• Baseline questionnaire (version 1.0)

  Participants have to fill out the questionnaire once and have to complete the questionnaire within 3 months after the invitation. A reminder for participation will be send one month after the initial invitation.

• Healt care utilization questionnaire (version 1.0)

  Participants have to fill out the questionnaire once and have to complete the questionnaire within 3 months after the invitation. A reminder for participation will be send one month after the initial invitation.

• Decision making questionnaire (version 1.0)

  Participants have to fill out the questionnaire once and have to complete the questionnaire within 3 months after the invitation. A reminder for participation will be send one month after the initial invitation.

Study Arms (1)

Patients with histopathological proven DTF

Other: Questionnaires

Interventions

Questionnaires OTHER

\- Baseline questionnaire, version 1.0, October 29 2019, (Dutch) - Health care utilization, version 1.0, October 29 2019, (Dutch) - Decision-making, version 1.0, October 29 2019, (Dutch) - EORTC QLQ-C30, version 3.0, (Dutch) - DTF-QoL, version 1.0, October

Patients with histopathological proven DTF

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients, aged ≥18 years, diagnosed with a histopathological proven DTF, regardless of disease stage and received treatment(s) who have received their last check-up of their DTF in one of the participating hospitals the hospital between October 2014 and July 2020.

You may qualify if:

• Aged 18 years and older
• Histopathological proven DTF, regardless of disease stage or treatment.
• Diagnosed between January 1990 and July 2020, with a visit to the hospital for their DTF (between October 2014 and July 2020)
• Sufficient Dutch language skills
• Competent to complete a questionnaire
• Written informed consent

You may not qualify if:

• Familial adenomatous polyposis (FAP)

Sponsors & Collaborators

• Erasmus Medical Center: lead
• Radboud University Medical Center: collaborator
• The Netherlands Cancer Institute: collaborator
• Royal Marsden NHS Foundation Trust: collaborator

Study Sites (4)

3. Antoni van Leeuwenhoek Ziekenhuis (AVL) / The Netherlands Cancer Instituut , Amsterdam, The Netherlands

Amsterdam, 1066 CX, Netherlands

Location

Radboud University Medical Center (MC), Nijmegen, The Netherlands

Nijmegen, 6525 GA, Netherlands

Location

Erasmus MC Cancer Institute

Rotterdam, 3015 GD, Netherlands

Location

Royal Marsden NHS Foundation Trust

London, SW3 6JJ, United Kingdom

Location

Related Publications (15)

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• Crago AM, Denton B, Salas S, Dufresne A, Mezhir JJ, Hameed M, Gonen M, Singer S, Brennan MF. A prognostic nomogram for prediction of recurrence in desmoid fibromatosis. Ann Surg. 2013 Aug;258(2):347-53. doi: 10.1097/SLA.0b013e31828c8a30.

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• Gronchi A, Colombo C, Le Pechoux C, Dei Tos AP, Le Cesne A, Marrari A, Penel N, Grignani G, Blay JY, Casali PG, Stoeckle E, Gherlinzoni F, Meeus P, Mussi C, Gouin F, Duffaud F, Fiore M, Bonvalot S; ISG and FSG. Sporadic desmoid-type fibromatosis: a stepwise approach to a non-metastasising neoplasm--a position paper from the Italian and the French Sarcoma Group. Ann Oncol. 2014 Mar;25(3):578-583. doi: 10.1093/annonc/mdt485. Epub 2013 Dec 9.

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• Husson O, Younger E, Dunlop A, Dean L, Strauss DC, Benson C, Hayes AJ, Miah A, van Houdt W, Zaidi S, Smith M, Williams J, Jones RL, van der Graaf WTA. Desmoid fibromatosis through the patients' eyes: time to change the focus and organisation of care? Support Care Cancer. 2019 Mar;27(3):965-980. doi: 10.1007/s00520-018-4386-8. Epub 2018 Aug 28.

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• Bonvalot S, Ternes N, Fiore M, Bitsakou G, Colombo C, Honore C, Marrari A, Le Cesne A, Perrone F, Dunant A, Gronchi A. Spontaneous regression of primary abdominal wall desmoid tumors: more common than previously thought. Ann Surg Oncol. 2013 Dec;20(13):4096-102. doi: 10.1245/s10434-013-3197-x. Epub 2013 Sep 20.

  PMID: 24052312 BACKGROUND

• Penel N, Le Cesne A, Bonvalot S, Giraud A, Bompas E, Rios M, Salas S, Isambert N, Boudou-Rouquette P, Honore C, Italiano A, Ray-Coquard I, Piperno-Neumann S, Gouin F, Bertucci F, Ryckewaert T, Kurtz JE, Ducimetiere F, Coindre JM, Blay JY. Surgical versus non-surgical approach in primary desmoid-type fibromatosis patients: A nationwide prospective cohort from the French Sarcoma Group. Eur J Cancer. 2017 Sep;83:125-131. doi: 10.1016/j.ejca.2017.06.017. Epub 2017 Jul 20.

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• Timbergen MJM, van de Poll-Franse LV, Grunhagen DJ, van der Graaf WT, Sleijfer S, Verhoef C, Husson O. Identification and assessment of health-related quality of life issues in patients with sporadic desmoid-type fibromatosis: a literature review and focus group study. Qual Life Res. 2018 Dec;27(12):3097-3111. doi: 10.1007/s11136-018-1931-3. Epub 2018 Jul 16.

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MeSH Terms

Conditions

Desmoid Tumors

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Fibroma; Neoplasms, Fibrous Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• C. Verhoef, MD, PhD

  Erasmus Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. dr. C. Verhoef, MD, PhD

Study Record Dates

• First Submitted: February 24, 2020
• First Posted: February 28, 2020
• Study Start: August 6, 2020
• Primary Completion: February 1, 2021
• Study Completion: February 1, 2021
• Last Updated: September 7, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

NL (3); GB (1)