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Observational Study
. 2022 Dec;126(8):1520-1532.
doi: 10.1002/jso.27067. Epub 2022 Aug 25.

A prospective real-world study of the diffuse-type tenosynovial giant cell tumor patient journey: A 2-year observational analysis

Nicholas M Bernthal  1 , John H Healey  2 , Emanuela Palmerini  3 , Sebastian Bauer  4 , Hendrik Schreuder  5 , Andreas Leithner  6 , Javier Martin-Broto  7 , Francois Gouin  8 , Julio Lopez-Bastida  9 , Hans Gelderblom  10 , Eric L Staals  11 , Zachary D Burke  1 , Erik J Geiger  1 , Geert Spierenburg  12 , Petra Laeis  13 , Elisabeth Beyerlein  14 , Xin Ye  15 , Michiel van de Sande  12
Affiliations
Observational Study

A prospective real-world study of the diffuse-type tenosynovial giant cell tumor patient journey: A 2-year observational analysis

Nicholas M Bernthal et al. J Surg Oncol. 2022 Dec.

Abstract

Background and objectives: Diffuse-tenosynovial giant cell tumor (D-TGCT) is a rare, locally aggressive, typically benign neoplasm affecting mainly large joints, representing a wide clinical spectrum. We provide a picture of the treatment journey of D-TGCT patients as a 2-year observational follow-up.

Methods: The TGCT Observational Platform Project registry was a multinational, multicenter, prospective observational study at tertiary sarcoma centers spanning seven European countries and two US sites. Histologically confirmed D-TGCT patients were categorized as either those who remained on initial treatment strategy (determined at baseline visit) or those who changed treatment strategy with specific changes documented (e.g., systemic treatment to surgery) at the 1-year and/or 2-year follow-up visits.

Results: A total of 176 patients were assessed, mean diagnosis age was 38.4 (SD ± 14.6) years; most patients had a knee tumor (120/176, 68.2%). For the 2-year observation period, most patients (75.5%) remained on the baseline treatment strategy throughout, 54/79 patients (68.4%) remained no treatment, 30/45 patients (66.7%) remained systemic treatment, 39/39 patients (100%) remained surgery. Those who changed treatment strategy utilized multimodal treatment options.

Conclusions: This is the first prospectively collected analysis to describe D-TGCT patient treatments over an extended follow-up and demonstrates the need for multidisciplinary teams to determine an optimal treatment strategy.

Keywords: TGCT observational platform project (TOPP); diffuse-tenosynovial giant cell tumor (D-TGCT); pexidartinib; prospective; real-world.

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Conflict of interest statement

NMB reports consulting fees from Daiichi Sankyo, Zimmer Biomet, and Onkos Surgical. JHH reports consulting fees from Daiichi Sankyo and Stryker; payment for expert testimony from Martin, Magnuson, McCarthy & Kenney; leadership or fiduciary role in Clinical Orthopaedics and Related Research, Musculoskeletal Transplant Foundation, and MIB Agents (osteosarcoma). EP reports consulting fees for advisory board from Amgen, Daiichi Sankyo, Lilly, Eusa Pharma, Deciphera Pharmaceuticals; drugs for preclinical research from Bristol‐Myers Squibb, Pfizer, PharmaMar; medical writing support for preclinical research from PharmaMar; and support for attending meetings and/or travel from Lilly, PharmaMar, and Takeda. SB reports research funding from Incyte to institution; consulting fees from Adcendo, Bayer, Blueprint Medicine, Boehringer Ingelheim, Cogent, Daiichi Sankyo, Deciphera, Exelixis, GSK, Novartis; honoraria from BluePrint Medicine and Deciphera; unpaid participation on Data Safety Monitoring Board or Advisory Board for Novartis, and unpaid leadership or fiduciary role in Deutsche Sarkomstiftung. HS declares no competing interests. AL reports institution education grants from Johnson and Johnson, Alphamed, Medacta and Implantec. JMB reports grants or contracts from, honoraria from, and participation on a Data Safety Monitoring Board or Advisory board for Amgen, Asofarma, Bayer, GSK, Lilly, Novartis, PharmaMar, Roche, Tecnofarma; consulting fees, payment for expert testimony, and support for attending meetings and/or travel from PharmaMar. FG reports consulting fees from Amgen, stock ownership in and co‐founder of Atlanthera, leadership or fiduciary role in NetSarc and French Sarcoma Group. JLB declares no competing interests. HG declares no competing interests. ELS reports participation on a Data Safety Monitoring Board or Advisory Board for Daiichi Sankyo and Deciphera. ZB declares no competing interests. EG reports honoraria and medical writing support from Daiichi Sankyo. GS reports research funding to his institution (LUMC) from Daiichi Sankyo outside the submitted work. PL, EB, and XY are employees for Daiichi Sankyo. MAJvdS reports research funding to his institution from Daiichi Sankyo and Carbofix; consulting fees to institution from AmMax; participation on Data Safety Monitoring Board or Advisory Board and consulting fees for emectuzumab; and leadership or fiduciary role in EMSOS, Dutch Sarcoma Group (DSG), and Euro Ewing Consortium.

Figures

Figure 1
Figure 1
Study design. *Additional data collection points may occur at any time the patient visits the site, even if it is outside this schedule
Figure 2
Figure 2
Patient eligibility. APS, all‐documented patient set; BAS, baseline analysis set; FAS, full analysis set; ICF, informed consent form
Figure 3
Figure 3
Breakdown of patient treatment plans at baseline (full analysis set)
Figure 4
Figure 4
Treatment strategy during 2‐year observation period with no current/planned treatment at baseline Solid lines indicate patients who remained on the same treatment strategy. Dotted lines indicate patients who changed treatment strategy
Figure 5
Figure 5
Treatment strategy during 2‐year observation period with no current/planned treatment at baseline Solid lines indicate patients who remained on the same treatment strategy. Dotted lines indicate patients who changed treatment strategy. *Surgery (n = 5) and future surgery (n = 1). †Seven were classified as unknown treatment. ‡Two were classified as unknown treatment
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