Identification and assessment of health-related quality of life issues in patients with sporadic desmoid-type fibromatosis: a literature review and focus group study

Milea J M Timbergen, Lonneke V van de Poll-Franse, Dirk J Grünhagen, Winette T van der Graaf, Stefan Sleijfer, Cornelis Verhoef, Olga Husson, Milea J M Timbergen, Lonneke V van de Poll-Franse, Dirk J Grünhagen, Winette T van der Graaf, Stefan Sleijfer, Cornelis Verhoef, Olga Husson

Abstract

Purpose: Sporadic desmoid-type fibromatosis (DTF) is a rare, chronic, non-metastasising, disease of the soft tissues. It is characterised by local invasive and unpredictable growth behaviour and a high propensity of local recurrence after surgery thereby often having a great impact on health-related quality of life (HRQL). This study aims to review currently used HRQL measures and to asses HRQL issues among DTF patients.

Methods: A mixed methods methodology was used consisting of (1) a systematic literature review, according to the PRISMA guidelines (2009), using search terms related to sporadic DTF and HRQL in commonly used databases (e.g. Embase, Medline Ovid, Web of science, Cochrane Central, Psyc Info, and Google scholar), to provide an overview of measures previously used to evaluate HRQL among DTF patients; (2) focus groups to gain insight into HRQL issues experienced by DTF patients.

Results: The search strategy identified thirteen articles reporting HRQL measures using a wide variety of cancer-specific HRQL tools, functional scores, symptom scales (e.g. NRS), and single-item outcomes (e.g. pain and functional impairment). No DTF-specific HRQL tool was found. Qualitative analysis of three focus groups (6 males, 9 females) showed that participants emphasised the negative impact of DTF and/or its treatment on several HRQL domains. Six themes were identified: (1) diagnosis, (2) treatment, (3) follow-up and recurrence, (4) physical domain, (5) psychological and emotional domain, and (6) social domain.

Conclusion: A DTF-specific HRQL tool and consensus regarding the preferred measurement tool among DTF patients is lacking. Our study indicates that HRQL of DTF patients was negatively affected in several domains. A DTF-specific HRQL measure could improve our understanding of short- and long-term effects and, ideally, can be used in both clinic and for research purposes.

Keywords: Desmoid-type fibromatosis; Focus group; Health-related quality of life; Literature review.

Conflict of interest statement

Conflict of interest

The authors declare that they have no conflict of interest.

Ethical approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. Approval from the Medical Ethics Committee of Erasmus MC in Rotterdam, the Netherlands was obtained for this study (file number MEC-2017-269).

Informed consent

Informed consent was obtained from all individual participants included in the study.

Figures

Fig. 1
Fig. 1
Focus group guideline

References

    1. World Health Organization (2016). International statistical classification of diseases and related health problems, 10th revision, volume 3.
    1. van Broekhoven DL, et al. Time trends in the incidence and treatment of extra-abdominal and abdominal aggressive fibromatosis: A population-based study. Annals of Surgical Oncology. 2015;22:2817–2823. doi: 10.1245/s10434-015-4632-y.
    1. De Marchis ML, et al. Desmoid tumors in familial adenomatous polyposis. Anticancer Research. 2017;37:33573336.
    1. Lips DJ, et al. The role of APC and beta-catenin in the aetiology of aggressive fibromatosis (desmoid tumors) European Journal of Surgical Oncology. 2009;35:3–10. doi: 10.1016/j.ejso.2008.07.003.
    1. Reitamo JJ, et al. The desmoid syndrome. New aspects in the cause, pathogenesis and treatment of the desmoid tumor. The American Journal of Surgery. 1986;151:230–237. doi: 10.1016/0002-9610(86)90076-0.
    1. Mullen JT, et al. β-catenin mutation status and outcomes in sporadic desmoid tumors. The Oncologist. 2013;18:1043–1049. doi: 10.1634/theoncologist.2012-0449.
    1. van Broekhoven DL, et al. Prognostic value of CTNNB1 gene mutation in primary sporadic aggressive fibromatosis. Annals of Surgical Oncology. 2015;22:1464–1470. doi: 10.1245/s10434-014-4156-x.
    1. Fiore M, et al. Desmoid-type fibromatosis and pregnancy: A multi-institutional analysis of recurrence and obstetric risk. Annals of Surgery. 2014;259:973–978. doi: 10.1097/SLA.0000000000000224.
    1. Sleijfer S. Management of aggressive fibromatosis: Can we unravel the maze of treatment options? European Journal of Cancer. 2009;45:2928–2929. doi: 10.1016/j.ejca.2009.09.012.
    1. Colombo C, et al. Sporadic extra abdominal wall desmoid-type fibromatosis: Surgical resection can be safely limited to a minority of patients. European Journal of Cancer. 2015;51:186192. doi: 10.1016/j.ejca.2014.11.019.
    1. Salas S, et al. Prognostic factors influencing progression-free survival determined from a series of sporadic desmoid tumors: A wait-and-see policy according to tumor presentation. Journal of Clinical Oncology. 2011;29:3553–3558. doi: 10.1200/JCO.2010.33.5489.
    1. Al-Jazrawe M, et al. Optimal therapy for desmoid tumors: Current options and challenges for the future. Expert Review of Anticancer Therapy. 2015;15:1443–1458. doi: 10.1586/14737140.2015.1096203.
    1. Penel N, et al. Surgical versus non-surgical approach in primary desmoid-type fibromatosis patients: A nationwide prospective cohort from the French Sarcoma Group. European Journal of Cancer. 2017;83:125–131. doi: 10.1016/j.ejca.2017.06.017.
    1. van Broekhoven DL, et al. Tailored Beta-catenin mutational approach in extra-abdominal sporadic desmoid tumor patients without therapeutic intervention. BMC Cancer. 2016;16:686. doi: 10.1186/s12885-016-2704-4.
    1. Landelijke Richtlijn Wekedelen Tumoren, versie 2.0 (2011). Oncoline, richtlijnen oncologische zorg; 2011.
    1. Bonvalot S, et al. Spontaneous regression of primary abdominal wall desmoid tumors: More common than previously thought. Annals of Surgical Oncology. 2013;20:4096–4102. doi: 10.1245/s10434-013-3197-x.
    1. Peng PD, et al. Management and recurrence patterns of desmoids tumors: A multi-institutional analysis of 211 patients. Annals of Surgical Oncology. 2012;19:4036–4042. doi: 10.1245/s10434-012-2634-6.
    1. Kasper B, et al. Imatinib induces sustained progression arrest in RECIST progressive desmoid tumours: Final results of a phase II study of the German Interdisciplinary Sarcoma Group (GISG) European Journal of Cancer. 2017;76:60–67. doi: 10.1016/j.ejca.2017.02.001.
    1. Bertani E, et al. Desmoid tumors of the anterior abdominal wall: Results from a monocentric surgical experience and review of the literature. Annals of Surgical Oncology. 2009;16:1642–1649. doi: 10.1245/s10434-009-0439-z.
    1. Kasper B, et al. An update on the management of sporadic desmoid-type fibromatosis: A European Consensus Initiative between Sarcoma PAtients EuroNet (SPAEN) and European Organization for Research and Treatment of Cancer (EORTC)/Soft Tissue and Bone Sarcoma Group (STBSG) Annals of Oncology. 2017;28:2399–2408. doi: 10.1093/annonc/mdx323.
    1. Marshall S, et al. Impact of patient-reported outcome measures on routine practice: A structured review. Journal of Evaluation in Clinical Practice. 2006;12:559–568. doi: 10.1111/j.1365-2753.2006.00650.x.
    1. Moher D, et al. Preferred reporting items for systematic reviews and meta-analyses: The PRISMA statement. International Journal of Surgery. 2010;8:336–341. doi: 10.1016/j.ijsu.2010.02.007.
    1. Beaton DE, et al. Measuring the whole or the parts? Validity, reliability, and responsiveness of the disabilities of the arm, shoulder and hand outcome measure in different regions of the upper extremity. Journal of Hand Therapy. 2001;14:128–146. doi: 10.1016/S0894-1130(01)80043-0.
    1. Dhinsa BS, et al. The outcome of resection of the distal ulna for tumour without soft-tissue or prosthetic reconstruction. The Bone & Joint Journal. 2014;96:1392–1395. doi: 10.1302/0301-620X.96B10.33664.
    1. Hudak PL, et al. Development of an upper extremity outcome measure: The DASH (disabilities of the arm, shoulder and hand) [corrected]. The Upper Extremity Collaborative Group (UECG) American Journal of Industrial Medicine. 1996;29:602–608. doi: 10.1002/(SICI)1097-0274(199606)29:6<602::AID-AJIM4>;2-L.
    1. Engelhardt TO, et al. About the self-reported quality of life after amputation of the hand in patients with upper extremity tumors. Handchirurgie Mikrochirurgie Plastische Chirurgie. 2008;40:23–30. doi: 10.1055/s-2007-989452.
    1. Tang F, et al. Large desmoid-type fibromatosis of the shoulder girdle: Operative approach selection and clinic outcome. International Orthopaedics. 2015;39:363–369. doi: 10.1007/s00264-014-2660-z.
    1. Gibbons CL, et al. Function after subtotal scapulectomy for neoplasm of bone and soft tissue. The Journal of Bone and Joint Surgery. 1998;80:38–42. doi: 10.1302/0301-620X.80B1.8183.
    1. Enneking WF, et al. A system for the functional evaluation of reconstructive procedures after surgical treatment of tumors of the musculoskeletal system. Clinical Orthopaedics and Related Research. 1993;286:241–246.
    1. Goubier JN, et al. Desmoid tumors and brachial plexus. Chirurgie de la Main. 2003;22:203–206. doi: 10.1016/S1297-3203(03)00058-1.
    1. Aaronson NK, et al. The European Organization for Research and Treatment of Cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. Journal of the National Cancer Institute. 1993;85:365–376. doi: 10.1093/jnci/85.5.365.
    1. Kummar S, et al. Clinical activity of the gamma-secretase inhibitor PF-03084014 in adults with desmoid tumors (aggressive fibromatosis) Journal of Clinical Oncology. 2017;35:1561–1569. doi: 10.1200/JCO.2016.71.1994.
    1. Cleeland CS, et al. Assessing symptom distress in cancer patients: The M.D. Anderson Symptom Inventory. Cancer. 2000;89:1634–1646. doi: 10.1002/1097-0142(20001001)89:7<1634::AID-CNCR29>;2-V.
    1. Catton CN, et al. Aggressive fibromatosis: Optimisation of local management with a retrospective failure analysis. Radiotherapy and Oncology. 1995;34:17–22. doi: 10.1016/0167-8140(94)01483-J.
    1. Johnstone PA, et al. Sarcomas of the hand and foot: Analysis of local control and functional result with combined modality therapy in extremity preservation. International Journal of Radiation Oncology Biology Physics. 1994;29:735–745. doi: 10.1016/0360-3016(94)90561-4.
    1. Hjermstad MJ, et al. Studies comparing Numerical Rating Scales, Verbal Rating Scales, and Visual Analogue Scales for assessment of pain intensity in adults: A systematic literature review. Journal of Pain and Symptom Management. 2011;41:1073–1093. doi: 10.1016/j.jpainsymman.2010.08.016.
    1. Ghanouni P, et al. Magnetic resonance-guided focused ultrasound treatment of extra-abdominal desmoid tumors: A retrospective multicenter study. European Radiology. 2017;27:732–740. doi: 10.1007/s00330-016-4376-5.
    1. Fiore M, et al. Activity of toremifene in sporadic desmoid-type fibromatosis. Journal of Clinical Oncology. 2011;29:10033. doi: 10.1200/jco.2011.29.15_suppl.10033.
    1. Davis AM, et al. Development of a measure of physical function for patients with bone and soft tissue sarcoma. Quality of Life Research. 1996;5:508–516. doi: 10.1007/BF00540024.
    1. Willeumier JJ, et al. Cross-cultural adaptation, translation, and validation of the toronto extremity salvage score for extremity bone and soft tissue tumor patients in Netherlands. Sarcoma. 2017;2017:6197525. doi: 10.1155/2017/6197525.
    1. Stoeckle E, et al. A critical analysis of treatment strategies in desmoid tumours: A review of a series of 106 cases. European Journal of Surgical Oncology. 2009;35:129–134. doi: 10.1016/j.ejso.2008.06.1495.
    1. Al-Otaibi ML, et al. Low-dose chemotherapy for extra-abdominal desmoid tumor. Saudi Medical Journal. 2008;29:1730–1734.
    1. Hoos A, et al. Desmoid tumors of the head and neck: A clinical study of a rare entity. Head and Neck. 2000;22:814–821. doi: 10.1002/1097-0347(200012)22:8<814::AID-HED11>;2-#.
    1. Tausch AP, Menold N. Methodological aspects of focus groups in health research: Results of qualitative interviews with focus group moderators. Global Qualitative Nursing Research. 2016;3:2333393616630466. doi: 10.1177/2333393616630466.
    1. The Desmoid Tumor Research Foundation (2017). DTRF patient registry.
    1. Paty J, et al. Prospective development of a patient reported outcomes (PRO) tool in desmoid tumors: A novel clinical trial endpoint. Journal of Clinical Oncology. 2017;35:11022. doi: 10.1200/JCO.2017.35.15_suppl.11022.
    1. Johnson, C., et al. Guidelines for developing questionnaire modules. On Behalf of EORTC Quality of Life Group 2011.

Source: PubMed

Sponsorit ja yhteistyökumppanit

Sairaudet

Huumeiden interventiot

3
Tilaa