The diffuse-type tenosynovial giant cell tumor (dt-TGCT) patient journey: a prospective multicenter study View Full Text


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Article Info

DATE

2021-04-29

AUTHORS

Nicholas M. Bernthal, Geert Spierenburg, John H. Healey, Emanuela Palmerini, Sebastian Bauer, Hans Gelderblom, Eric L. Staals, Julio Lopez-Bastida, Eva-Maria Fronk, Xin Ye, Petra Laeis, Michiel A. J. van de Sande

ABSTRACT

BackgroundTenosynovial giant cell tumor (TGCT) is a rare, locally aggressive neoplasm arising from the synovium of joints, bursae, and tendon sheaths affecting small and large joints. It represents a wide spectrum ranging from minimally symptomatic to massively debilitating. Most findings to date are mainly from small, retrospective case series, and thus the morbidity and actual impact of this rare disease remain to be elucidated. This study prospectively explores the management of TGCT in tertiary sarcoma centers.MethodsThe TGCT Observational Platform Project registry was a multinational, multicenter, prospective observational study involving 12 tertiary sarcoma centers in 7 European countries, and 2 US sites. This study enrolled for 2 years all consecutive ≥ 18 years old patients, with histologically diagnosed primary or recurrent cases of diffuse-type TGCT. Patient demographic and clinical characteristics were collected at baseline and every 6 months for 24 months. Quality of life questionnaires (PROMIS-PF and EQ-5D) were also administered at the same time-points. Here we report baseline patient characteristics.Results166 patients were enrolled between November 2016 and March 2019. Baseline characteristics were: mean age 44 years (mean age at disease onset: 39 years), 139/166 (83.7%) had prior treatment, 71/166 patients (42.8%) had ≥ 1 recurrence after treatment of their primary tumor, 76/136 (55.9%) visited a medical specialist ≥ 5 times, 66/116 (56.9%) missed work in the 24 months prior to baseline, and 17/166 (11.6%) changed employment status or retired prematurely due to disease burden. Prior treatment consisted of surgery (i.e., arthroscopic, open synovectomy) (128/166; 77.1%) and systemic treatments (52/166; 31.3%) with imatinib (19/52; 36.5%) or pexidartinib (27/52; 51.9%). Treatment strategies at baseline visits consisted mainly of watchful waiting (81/166; 48.8%), surgery (41/166; 24.7%), or targeted systemic therapy (37/166; 22.3%). Patients indicated for treatment reported more impairment compared to patients indicated for watchful waiting: worst stiffness NRS 5.16/3.44, worst pain NRS 6.13/5.03, PROMIS-PF 39.48/43.85, and EQ-5D VAS 66.54/71.85. ConclusionThis study confirms that diffuse-type TGCT can highly impact quality of life. A prospective observational registry in rare disease is feasible and can be a tool to collect curated-population reflective data in orphan diseases. Name of registry: Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project (TOPP).Trial registration number: NCT02948088.Date of registration: 10 October 2016.URL of Trial registry record: https://clinicaltrials.gov/ct2/show/NCT02948088?term=NCT02948088&draw=2. More... »

PAGES

191

Identifiers

URI

http://scigraph.springernature.com/pub.10.1186/s13023-021-01820-6

DOI

http://dx.doi.org/10.1186/s13023-021-01820-6

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1137619866

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/33926503


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23 schema:description BackgroundTenosynovial giant cell tumor (TGCT) is a rare, locally aggressive neoplasm arising from the synovium of joints, bursae, and tendon sheaths affecting small and large joints. It represents a wide spectrum ranging from minimally symptomatic to massively debilitating. Most findings to date are mainly from small, retrospective case series, and thus the morbidity and actual impact of this rare disease remain to be elucidated. This study prospectively explores the management of TGCT in tertiary sarcoma centers.MethodsThe TGCT Observational Platform Project registry was a multinational, multicenter, prospective observational study involving 12 tertiary sarcoma centers in 7 European countries, and 2 US sites. This study enrolled for 2 years all consecutive ≥ 18 years old patients, with histologically diagnosed primary or recurrent cases of diffuse-type TGCT. Patient demographic and clinical characteristics were collected at baseline and every 6 months for 24 months. Quality of life questionnaires (PROMIS-PF and EQ-5D) were also administered at the same time-points. Here we report baseline patient characteristics.Results166 patients were enrolled between November 2016 and March 2019. Baseline characteristics were: mean age 44 years (mean age at disease onset: 39 years), 139/166 (83.7%) had prior treatment, 71/166 patients (42.8%) had ≥ 1 recurrence after treatment of their primary tumor, 76/136 (55.9%) visited a medical specialist ≥ 5 times, 66/116 (56.9%) missed work in the 24 months prior to baseline, and 17/166 (11.6%) changed employment status or retired prematurely due to disease burden. Prior treatment consisted of surgery (i.e., arthroscopic, open synovectomy) (128/166; 77.1%) and systemic treatments (52/166; 31.3%) with imatinib (19/52; 36.5%) or pexidartinib (27/52; 51.9%). Treatment strategies at baseline visits consisted mainly of watchful waiting (81/166; 48.8%), surgery (41/166; 24.7%), or targeted systemic therapy (37/166; 22.3%). Patients indicated for treatment reported more impairment compared to patients indicated for watchful waiting: worst stiffness NRS 5.16/3.44, worst pain NRS 6.13/5.03, PROMIS-PF 39.48/43.85, and EQ-5D VAS 66.54/71.85. ConclusionThis study confirms that diffuse-type TGCT can highly impact quality of life. A prospective observational registry in rare disease is feasible and can be a tool to collect curated-population reflective data in orphan diseases. Name of registry: Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project (TOPP).Trial registration number: NCT02948088.Date of registration: 10 October 2016.URL of Trial registry record: https://clinicaltrials.gov/ct2/show/NCT02948088?term=NCT02948088&draw=2.
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29 schema:keywords ConclusionThis study
30 European countries
31 Life Questionnaire
32 Results166 patients
33 TGCT
34 US sites
35 actual impact
36 age 44
37 aggressive neoplasm
38 baseline
39 baseline characteristics
40 baseline patient characteristics
41 baseline visit
42 burden
43 bursa
44 case series
45 cases
46 cell tumors
47 center
48 characteristics
49 clinical characteristics
50 countries
51 data
52 date
53 disease
54 disease burden
55 employment status
56 findings
57 giant cell tumor
58 imatinib
59 impact
60 impairment
61 joints
62 journey
63 large joints
64 life
65 management
66 mean age 44
67 medical specialists
68 months
69 morbidity
70 more impairment
71 most findings
72 multicenter
73 multicenter study
74 neoplasms
75 observational registry
76 observational study
77 older patients
78 orphan disease
79 patient characteristics
80 patient journey
81 patients
82 pexidartinib
83 platform project
84 primary tumor
85 prior treatment
86 project
87 prospective multicenter study
88 prospective observational registry
89 prospective observational study
90 quality
91 quality of life
92 questionnaire
93 rare disease
94 records
95 recurrence
96 recurrent cases
97 reflective data
98 registration
99 registry
100 registry records
101 retrospective case series
102 sarcoma center
103 series
104 sheath
105 sites
106 specialists
107 spectra
108 status
109 strategies
110 study
111 surgery
112 synovium
113 synovium of joints
114 systemic therapy
115 systemic treatment
116 tendon sheath
117 terms
118 tertiary sarcoma centre
119 therapy
120 time
121 tool
122 treatment
123 treatment strategies
124 trial registry records
125 tumors
126 visits
127 waiting
128 watchful waiting
129 wide spectrum
130 work
131 year old patient
132 years
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