Identification of Patient Needs and Preferences in Pigmented Villonodular Synovitis (PVNS) Using a Qualitative Online Bulletin Board Study

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Abstract

Introduction

Pigmented villonodular synovitis (PVNS), also known as giant-cell tumour of the tendon sheath (GCTT), is a rare, benign proliferative tumour affecting the inner lining of synovial joints and tendon sheets. Information on treatment needs of PVNS patients to inform drug development is currently scarce. We conducted an exploratory qualitative study with PVNS patients to generate insights into the objective and emotional aspects related to their medical journey and experiences of living with this disease.

Methods

A 4-day study using an online bulletin board (OBB), an asynchronous, online qualitative research platform, was conducted with patients recruited via physician referral who underwent screening questions to ensure eligibility for the study and willingness to participate. The discussion was moderated, was structured and allowed open answers in response to other participants’ posts.

Results

Eleven patients (4 from the USA, 4 from the UK and 3 from Canada; 45% female), aged 28–57 years, suffering from PVNS for 2–27 years participated in the study. Key patient insights from the study were: (1) pain was the topmost, spontaneous thought that the participants associated with PVNS, constituting a significant emotional and psychological burden; (2) surgery (arthroscopy) did not completely ameliorate symptoms associated with PVNS, as the relapse rate was high in these patients; (3) PVNS has a substantial negative financial impact on patients, their families and the healthcare system; (4) orthopaedic specialists/surgeons predominantly managed PVNS, as surgery is currently the only therapeutic option.

Conclusion

PVNS patients expressed an urgent need for a medical drug treatment, which can reduce pain, avoid relapses and provide an alternative to surgery, the current standard of care.

Electronic Supplementary Material

The online version of this article (10.1007/s12325-020-01364-4) contains supplementary material, which is available to authorized users.

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Most cited references 35

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Qualitative research methods: when to use them and how to judge them.

M S Kirkman, K Hammarberg, S de Lacey (2016)

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Current Systemic Treatment Options for Tenosynovial Giant Cell Tumor/Pigmented Villonodular Synovitis: Targeting the CSF1/CSF1R Axis.

Philippe Cassier, Mehdi Brahmi, Armelle Vinceneux (2016)

Adequate surgical resection remains the treatment of choice for tenosyovial giant cell tumor (TGCT). However, diffuse type TGCT (D-TGCT) is more difficult to resect and has a higher rate of recurrence (up to 50 %), which is often multiple. D-TGCT is rarely lethal and only rare cases of metastases have been described. Nevertheless, patients might have a significant decline in their quality of life due to multiple operations, which may sometimes result in a partial loss of function of the affected joint and may also be associated with perioperative morbidity and secondary arthrosis. As of today, no systemic treatment is approved for this rare disease. The aims of systemic therapy in the context of a non-lethal tumor are to reduce surgical morbidity and to preserve function and patient quality of life. Because TGCT is associated with characteristic cytogenetic abnormalities resulting in the overexpression of CSF1, systemic therapies targeting the CSF1/CSF1R axis (imatinib, nilotinib, emactuzumab, and PLX3397) have been tested in patients with locally advanced or relapsed D-TGCT. The more recent and more specific CSF1R inhibitors have shown a very interesting clinical activity with acceptable toxicity in early phase trials. These results will need to be confirmed in larger, ideally randomized, trials. But the high rate of clinical and functional improvement seen in some patients with advanced D-TGCT, often after multiple operations, suggests that these inhibitors will likely have a role in the management of patients with an inoperable disease; the definition of "inoperable TGCT" still requires refinement to reach a consensus. Another point that will need to be addressed is that of "the optimal duration of therapy" for these patients. Indeed, we and others have observed often prolonged clinical benefit and symptomatic relief even after treatment was stopped, with both monoclonal antibodies and tyrosine kinase inhibitors. Responses were observed very early on with emactuzumab and PLX3397, and patients experienced significant symptom improvement within a few weeks of starting therapy (2-4 weeks). Another possible application of CSF1R inhibitors could be used either as a preoperative or postoperative therapy for patients with operable TGCT. However, we currently lack sufficient follow-up to adequately address these questions which will each require specific trial designs. Overall, the striking clinical activity of CSF1R specific inhibitors in TGCT has created great enthusiasm among clinicians, and further development of these agents is clearly medically needed. Nevertheless, further investigations are necessary to validate those treatments and assess how to best incorporate them among other treatment modalities into the overall therapeutic strategy for a given patient.

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Increasing Patient Involvement in Drug Development.

Lisa Cone, Rahul Sasane, Maria M Lowe … (2016)

To ensure the creation of treatments that maximize value at the lowest cost, all aspects of the health care system need to align with patient needs and preferences. Despite growing efforts to engage patients in research and regulatory activities, the pharmaceutical industry has yet to maximize patient involvement in the drug development process.

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Author and article information

Contributors

Nigel S. Cook:

ORCID: http://orcid.org/0000-0001-8211-7613

pallapotu.vikrant@novartis.com

Journal

Journal ID (nlm-ta): Adv Ther

Journal ID (iso-abbrev): Adv Ther

Title: Advances in Therapy

Publisher: Springer Healthcare (Cheshire )

ISSN (Print): 0741-238X

ISSN (Electronic): 1865-8652

Publication date (Electronic): 11 May 2020

Publication date PMC-release: 11 May 2020

Publication date (Print): 2020

Volume: 37

Issue: 6

Pages: 2813-2828

Affiliations

[1 ]GRID grid.419481.1, ISNI 0000 0001 1515 9979, Novartis Pharma AG, ; Basel, Switzerland

[2 ]GRID grid.464975.d, ISNI 0000 0004 0405 8189, Novartis Healthcare Pvt. Ltd., ; Hyderabad, India

[3 ]Ipsos SA, Basel, Switzerland

Author information

Nigel S. Cook http://orcid.org/0000-0001-8211-7613

Article

Publisher ID: 1364

DOI: 10.1007/s12325-020-01364-4

PMC ID: 7467432

PubMed ID: 32394210

SO-VID: 85026fc1-0868-499f-b6ce-f60bc9531d6b

License:

Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/.

History

Date received : 24 March 2020

Funding

Funded by: Novartis AG

Custom metadata

Keywords: gctt,giant-cell tumour of the tendon sheath,online bulletin board,patient-based evidence,patient perspective,pigmented villonodular synovitis,pvns,qualitative research,rare disease,rheumatology

Data availability:

Keywords: gctt, giant-cell tumour of the tendon sheath, online bulletin board, patient-based evidence, patient perspective, pigmented villonodular synovitis, pvns, qualitative research, rare disease, rheumatology

Identification of Patient Needs and Preferences in Pigmented Villonodular Synovitis (PVNS) Using a Qualitative Online Bulletin Board Study

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Abstract

Introduction

Methods

Results

Conclusion

Electronic Supplementary Material

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Resource Identification

Most cited references 35

Qualitative research methods: when to use them and how to judge them.

Current Systemic Treatment Options for Tenosynovial Giant Cell Tumor/Pigmented Villonodular Synovitis: Targeting the CSF1/CSF1R Axis.

Increasing Patient Involvement in Drug Development.

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