Giant Cell Tumors
Conditions
Keywords
Tenosynovial Giant Cell Tumors, Disease registry, Routine clinical practice, Real World Evidence
Brief summary
TGCT is a rare disease that is difficult to manage, surgical resection is the primary treatment currently available. To date no disease registry exists and there is little data available detailing the management of patients with diffuse TGCT (d-TGCT), the burden of d-TGCT for patients (including pain, joint stiffness, swelling, reduced mobility and quality of life) or the economic impact of d-TGCT. This study aims to collect data by an observational disease registry involving no intervention to the patient or changes to investigators treatment decisions.
Detailed description
GCT is a rare, benign, but potentially locally aggressive and recurrent disease. Treatment pattern and treatment initiation, continuation or changes are solely at the discretion of the physician and the patient. There will be no attempt to influence the prescribing patterns of any individual treating physician. All medication will be prescribed in the usual standard of care and will not be provided by the study sponsor. Participation in the study will in no way influence payment or reimbursement for any treatment received by patients during the study. It is the responsibility of the investigator and his study staff to enter all relevant patient data required for this registry in the electronic Case Report Form (eCRF) and in the patients' medical records. Approximately 15 sites from up to 6 European Countries (France, Germany, Italy, The Netherlands, Spain and United Kingdom) and 4 sites from the US are planned to participate. The sites will be specialized sites that treat d-TGCT regularly.
Interventions
None listed
Sponsors
Optimapharm
Daiichi Sankyo
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Written informed consent for participation in the study (ICF) * Patients with d-TGCT (diagnosed histologically) confirmed naïve or recurrent case
Exclusion criteria
* As this is a non interventional study, no explicit
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean PROMIS Physical Function Score From Baseline Through 24 Months | Baseline up to end of observation period (approximately 2 years) | PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures that evaluates and monitors physical, mental, and social health. Physical function scores range from 0 (worst physical function) to 100 (best physical function, where higher scores indicate better clinical outcome. |
| Time Since Most Recent Tumor Recurrence Until Baseline in Patients With Diffuse TGCT (d-TGCT) | at Baseline | Time since most recent tumor recurrence was collected from information routinely recorded in the patient files / medical records. |
| Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT) | Baseline up to end of observation period (approximately 2 years) | Time from baseline to first tumor recurrence was collected from information routinely recorded in the patient files / medical records. |
| Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Baseline up to end of observation period (approximately 2 years) | The BPI short form is a self administered questionnaire that assesses severity of pain, impact of pain on daily function, location of pain, pain medications and amount of pain relief in the past 24 hours or the past week. The BPI pain severity and interference score ranges from 0 (pain does not interfere) to 10 (pain completely interferes), where lower scores indicate better clinical outcome. |
| Mean Worst Stiffness Score From Baseline Through 24 Months | Baseline up to end of observation period (approximately 2 years) | The Worst Stiffness numerical rating scale (NRS) is a one-item self-administered, patient-reported outcome questionnaire assessing the worst stiffness in the last 24 hours. The NRS for this item ranges from 0 (no stiffness) to 10 (stiffness as bad as you can imagine), where lower scores indicate better clinical outcome. |
| Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Baseline up to end of observation period (approximately 2 years) | The management plan received among the patients was collected from information routinely recorded in the patient files / medical records. |
| Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Baseline up to end of observation period (approximately 2 years) | The current TGCT treatment plan among the patients was collected from information routinely recorded in the patient files / medical records. |
| Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Baseline up to end of observation period (approximately 2 years) | Type of treatment plan received among the patients was collected from information routinely recorded in the patient files / medical records. |
| Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Baseline up to end of observation period (approximately 2 years) | The status of treatment plan among the patients was collected from information routinely recorded in the patient files / medical records. |
| Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | Baseline up to end of observation period (approximately 2 years) | Type of current TGCT treatment among the patients was collected from information routinely recorded in the patient files / medical records. |
| Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT) | at Baseline | Tumor severity classification was collected from information routinely recorded in the patient files / medical records. ). Tumor severity classification was based on MRI. In this classification scheme, moderate diffuse TGCT is characterized by intra- and/or extra-articular disease, without or with involvement of muscle/tendinous tissue/ligaments. Severe diffuse TGCT is characterized by intra- and extra-articular involvement and involvement of at least one of the 3 structures (muscle/tendinous tissue/ligaments). |
| Number of Cases With Complications Due to Surgery in Patients With Diffuse TGCT (d-TGCT) | at Baseline (prior to any d-TGCT therapy) | Complications due to surgery were collected from information routinely recorded in the patient files / medical records. |
| Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT) | at Baseline | Tumor recurrence was collected from information routinely recorded in the patient files / medical records. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | 13-24 months before baseline up to end of observation period (approximately 2 years) | Health resource utilization was assessed by mean number of general practitioner visits, specialist visits, and physical therapy sessions. In addition, the mean number of days in rehabilitation and the number of work days missed were also reported. |
| Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | 13-24 months before baseline up to end of observation period (approximately 2 years) | The median number of days in rehabilitation and the number of work days missed were also reported. |
| Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | At end of observation period (approximately 2 years post-baseline) | TGCT outcome status was collected from information routinely recorded in the patient files / medical records. |
| Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | Baseline up to end of observation period (approximately 2 years) | The EQ-5D-5L questionnaire measures the patient's quality of life (QoL) based on the 5 dimensions mobility, self care, usual activities, pain/discomfort, and anxiety/depression. The EQ-5D index score ranges from 0 (worst QoL) to 1 (best QoL), where lower scores indicate worse clinical outcome. The EQ-5D VAS reports patient's self rated health ranging from 0 (worst health) to 100 (best health you can imagine), where lower scores indicate worse clinical outcome. |
Countries
Austria, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States
Participant flow
Recruitment details
A total of 183 participants who met all eligibility criteria were enrolled in the study at 12 sites in Europe and the United States.
Pre-assignment details
All participants with diffuse TGCT (diagnosed histologically) with a confirmed primary diagnosis or recurrent case were enrolled.
Participants by arm
| Arm | Count |
|---|---|
| Tenosynovial Giant Cell Tumors All participants with diffuse tenosynovial giant cell tumors (d-TGCT) who were enrolled in the study. | 176 |
| Total | 176 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
| Overall Study | Lost to Follow-up | 8 |
| Overall Study | Other | 2 |
| Overall Study | Withdrawal by Subject | 4 |
Baseline characteristics
| Characteristic | Tenosynovial Giant Cell Tumors | — |
|---|---|---|
| Age, Continuous | 43.5 years STANDARD_DEVIATION 14.3 | — |
| Age, Customized ≥40 and <60 years | 69 Participants | — |
| Age, Customized <40 years | 78 Participants | — |
| Age, Customized ≥60 years | 29 Participants | — |
| Current symptoms Limited range of movement | 116 Participants | — |
| Current symptoms Pain | 138 Participants | — |
| Current symptoms Patient unable to judge | 0 Participants | — |
| Current symptoms Stiffness | 99 Participants | — |
| Current symptoms Swelling | 111 Participants | — |
| Most disturbing symptom Limited range of movement | 32 Participants | — |
| Most disturbing symptom Missing | 3 Participants | — |
| Most disturbing symptom Not applicable | 8 Participants | — |
| Most disturbing symptom Pain | 86 Participants | — |
| Most disturbing symptom Patient unable to judge | 16 Participants | — |
| Most disturbing symptom Stiffness | 13 Participants | — |
| Most disturbing symptom Swelling | 18 Participants | — |
| Number of participants with current symptoms 2 current symptoms | 47 Participants | — |
| Number of participants with current symptoms 3 current symptoms | 36 Participants | — |
| Number of participants with current symptoms 4 current symptoms | 60 Participants | — |
| Number of participants with current symptoms Unknown/missing | 33 Participants | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Region of Enrollment Austria | 10 participants | — |
| Region of Enrollment France | 4 participants | — |
| Region of Enrollment Germany | 12 participants | — |
| Region of Enrollment Italy | 38 participants | — |
| Region of Enrollment Netherlands | 61 participants | — |
| Region of Enrollment Spain | 14 participants | — |
| Region of Enrollment United Kingdom | 3 participants | — |
| Region of Enrollment United States | 34 participants | — |
| Sex: Female, Male Female | 108 Participants | — |
| Sex: Female, Male Male | 68 Participants | — |
| Tumor location/affected joint Ankle joint | 18 Participants | — |
| Tumor location/affected joint Elbow joint | 4 Participants | — |
| Tumor location/affected joint Foot | 7 Participants | — |
| Tumor location/affected joint Hand | 3 Participants | — |
| Tumor location/affected joint Hip | 12 Participants | — |
| Tumor location/affected joint Knee joint | 120 Participants | — |
| Tumor location/affected joint Shoulder joint | 8 Participants | — |
| Tumor location/affected joint Temporomandibular | 1 Participants | — |
| Tumor location/affected joint Wrist joint | 3 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 183 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months
The BPI short form is a self administered questionnaire that assesses severity of pain, impact of pain on daily function, location of pain, pain medications and amount of pain relief in the past 24 hours or the past week. The BPI pain severity and interference score ranges from 0 (pain does not interfere) to 10 (pain completely interferes), where lower scores indicate better clinical outcome.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Brief Pain Inventory Pain Severity and Pain Interference were assessed in participants with available data in the Full Analysis Set.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Baseline | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Severity | 3.35 score on a scale | Standard Deviation 2.36 |
| Baseline | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Interference | 2.97 score on a scale | Standard Deviation 2.65 |
| Within 1st Year | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Severity | 3.12 score on a scale | Standard Deviation 2.26 |
| Within 1st Year | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Interference | 2.94 score on a scale | Standard Deviation 2.56 |
| Within 2nd Year | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Severity | 3.15 score on a scale | Standard Deviation 2.22 |
| Within 2nd Year | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Interference | 2.80 score on a scale | Standard Deviation 2.44 |
| 18 Months | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Interference | 2.80 score on a scale | Standard Deviation 2.47 |
| 18 Months | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Severity | 3.05 score on a scale | Standard Deviation 2.3 |
| 24 Months | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Severity | 2.60 score on a scale | Standard Deviation 2.19 |
| 24 Months | Mean Brief Pain Inventory (BPI) Pain Severity and Interference Score From Baseline Through 24 Months | Brief Pain Inventory Pain Interference | 2.53 score on a scale | Standard Deviation 2.57 |
Primary
Mean PROMIS Physical Function Score From Baseline Through 24 Months
PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures that evaluates and monitors physical, mental, and social health. Physical function scores range from 0 (worst physical function) to 100 (best physical function, where higher scores indicate better clinical outcome.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: PROMIS physical function was assessed in participants with available data in the Full Analysis Set.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Baseline | Mean PROMIS Physical Function Score From Baseline Through 24 Months | 41.9 score on a scale | Standard Deviation 8.2 |
| Within 1st Year | Mean PROMIS Physical Function Score From Baseline Through 24 Months | 41.6 score on a scale | Standard Deviation 9.2 |
| Within 2nd Year | Mean PROMIS Physical Function Score From Baseline Through 24 Months | 43.2 score on a scale | Standard Deviation 9.3 |
| 18 Months | Mean PROMIS Physical Function Score From Baseline Through 24 Months | 42.2 score on a scale | Standard Deviation 8.8 |
| 24 Months | Mean PROMIS Physical Function Score From Baseline Through 24 Months | 43.0 score on a scale | Standard Deviation 9 |
Primary
Mean Worst Stiffness Score From Baseline Through 24 Months
The Worst Stiffness numerical rating scale (NRS) is a one-item self-administered, patient-reported outcome questionnaire assessing the worst stiffness in the last 24 hours. The NRS for this item ranges from 0 (no stiffness) to 10 (stiffness as bad as you can imagine), where lower scores indicate better clinical outcome.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Worst stiffness was assessed in participants with available data in the Full Analysis Set.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Baseline | Mean Worst Stiffness Score From Baseline Through 24 Months | 4.4 score on a scale | Standard Deviation 2.8 |
| Within 1st Year | Mean Worst Stiffness Score From Baseline Through 24 Months | 4.2 score on a scale | Standard Deviation 2.6 |
| Within 2nd Year | Mean Worst Stiffness Score From Baseline Through 24 Months | 4.2 score on a scale | Standard Deviation 2.8 |
| 18 Months | Mean Worst Stiffness Score From Baseline Through 24 Months | 4.2 score on a scale | Standard Deviation 2.7 |
| 24 Months | Mean Worst Stiffness Score From Baseline Through 24 Months | 3.9 score on a scale | Standard Deviation 2.7 |
Primary
Number of Cases With Complications Due to Surgery in Patients With Diffuse TGCT (d-TGCT)
Complications due to surgery were collected from information routinely recorded in the patient files / medical records.
Time frame: at Baseline (prior to any d-TGCT therapy)
Population: Complications were assessed among participants with documented surgeries.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Baseline | Number of Cases With Complications Due to Surgery in Patients With Diffuse TGCT (d-TGCT) | 9 surgical cases |
Primary
Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT)
The current TGCT treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: The current treatment plan (currently being treated or planned) was assessed in participants with no changes at any data collection point where the data of the previous reported data collection point is being reported in the Full Analysis Set.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Currently being treated or planned to be treated | 97 Participants |
| Baseline | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Wait and see | 79 Participants |
| Baseline | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Unknown/Missing | 0 Participants |
| Within 1st Year | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Currently being treated or planned to be treated | 85 Participants |
| Within 1st Year | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Wait and see | 77 Participants |
| Within 1st Year | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Unknown/Missing | 11 Participants |
| Within 2nd Year | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Wait and see | 105 Participants |
| Within 2nd Year | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Unknown/Missing | 3 Participants |
| Within 2nd Year | Number of Participants According to the Current TGCT Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Currently being treated or planned to be treated | 65 Participants |
Primary
Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT)
The management plan received among the patients was collected from information routinely recorded in the patient files / medical records.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Management plans were assessed in participants with available data in the Full Analysis Set. Participants may have be included in more than one category.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any changes to TGCT treatment (including also planned treatments) since the last visit | NA Participants |
| Baseline | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any prior or concomitant therapies for managing TGCT-related symptoms | 94 Participants |
| Baseline | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any changes to concomitant therapies for managing TGCT-related symptoms since the last visit | NA Participants |
| Within 1st Year | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any changes to TGCT treatment (including also planned treatments) since the last visit | 74 Participants |
| Within 1st Year | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any changes to concomitant therapies for managing TGCT-related symptoms since the last visit | 43 Participants |
| Within 1st Year | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any prior or concomitant therapies for managing TGCT-related symptoms | NA Participants |
| Within 2nd Year | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any changes to TGCT treatment (including also planned treatments) since the last visit | 61 Participants |
| Within 2nd Year | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any changes to concomitant therapies for managing TGCT-related symptoms since the last visit | 19 Participants |
| Within 2nd Year | Number of Participants According to the Management Plan in Patients With Diffuse TGCT (d-TGCT) | Any prior or concomitant therapies for managing TGCT-related symptoms | NA Participants |
Primary
Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)
The status of treatment plan among the patients was collected from information routinely recorded in the patient files / medical records.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Status of treatment plan was assessed in participants currently being treated or planned to be treated in the Full Analysis Set. Participants with different reported treatment plans during the period (e.g. participants who may have switched treatment plans) are counted as 'currently being treated or planned to be treated' in the corresponding period.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Current | 44 Participants |
| Baseline | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Planned | 50 Participants |
| Baseline | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Missing | 5 Participants |
| Within 1st Year | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Current | 50 Participants |
| Within 1st Year | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Planned | 9 Participants |
| Within 1st Year | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Missing | 2 Participants |
| Within 2nd Year | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Planned | 9 Participants |
| Within 2nd Year | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Missing | 0 Participants |
| Within 2nd Year | Number of Participants Based on Status of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Current | 23 Participants |
Primary
Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT)
Tumor severity classification was collected from information routinely recorded in the patient files / medical records. ). Tumor severity classification was based on MRI. In this classification scheme, moderate diffuse TGCT is characterized by intra- and/or extra-articular disease, without or with involvement of muscle/tendinous tissue/ligaments. Severe diffuse TGCT is characterized by intra- and extra-articular involvement and involvement of at least one of the 3 structures (muscle/tendinous tissue/ligaments).
Time frame: at Baseline
Population: Tumor severity was assessed in participants in the Full Analysis Set.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT) | Moderate diffuse | 66 Participants |
| Baseline | Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT) | Severe diffuse | 90 Participants |
| Baseline | Number of Participants Based on Tumor Severity Classification at Baseline Based on MRI in Patients With Diffuse TGCT (d-TGCT) | Not assessable | 20 Participants |
Primary
Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT)
Type of current TGCT treatment among the patients was collected from information routinely recorded in the patient files / medical records.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Status of treatment plan was assessed in participants currently being treated in the Full Analysis Set. Participants may have been included in more than one category.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | Systemic therapy | 37 Participants |
| Baseline | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | 90Yttrium therapy | 0 Participants |
| Baseline | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | Surgery | 7 Participants |
| Within 1st Year | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | Systemic therapy | 12 Participants |
| Within 1st Year | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | 90Yttrium therapy | 1 Participants |
| Within 1st Year | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | Surgery | 37 Participants |
| Within 2nd Year | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | 90Yttrium therapy | 0 Participants |
| Within 2nd Year | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | Surgery | 17 Participants |
| Within 2nd Year | Number of Participants Based on Type of Current TGCT Treatment in Patients With Diffuse TGCT (d-TGCT) | Systemic therapy | 8 Participants |
Primary
Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT)
Type of treatment plan received among the patients was collected from information routinely recorded in the patient files / medical records.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Type of treatment plans were assessed in participants with available data in the Full Analysis Set. Participants may have be included in more than one category.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | 90Yttrium therapy | 1 Participants |
| Baseline | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Unchanged therapy | 0 Participants |
| Baseline | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Surgery | 42 Participants |
| Baseline | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | No current or planned treatment | 79 Participants |
| Baseline | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Radiotherapy | 5 Participants |
| Baseline | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Future surgery required | 5 Participants |
| Baseline | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Systemic therapy | 48 Participants |
| Within 1st Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Future surgery required | 2 Participants |
| Within 1st Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | No current or planned treatment | 77 Participants |
| Within 1st Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Unchanged therapy | 36 Participants |
| Within 1st Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Systemic therapy | 16 Participants |
| Within 1st Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | 90Yttrium therapy | 1 Participants |
| Within 1st Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Surgery | 40 Participants |
| Within 1st Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Radiotherapy | 1 Participants |
| Within 2nd Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Unchanged therapy | 35 Participants |
| Within 2nd Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | 90Yttrium therapy | 0 Participants |
| Within 2nd Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Systemic therapy | 8 Participants |
| Within 2nd Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Surgery | 25 Participants |
| Within 2nd Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Future surgery required | 0 Participants |
| Within 2nd Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | No current or planned treatment | 105 Participants |
| Within 2nd Year | Number of Participants Based on Type of Treatment Plan in Patients With Diffuse TGCT (d-TGCT) | Radiotherapy | 0 Participants |
Primary
Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT)
Tumor recurrence was collected from information routinely recorded in the patient files / medical records.
Time frame: at Baseline
Population: Tumor recurrence was assessed in participants with available baseline data in the Full Analysis Set.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT) | 2 recurrences | 18 Participants |
| Baseline | Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT) | 3 recurrences and more | 19 Participants |
| Baseline | Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT) | Missing | 1 Participants |
| Baseline | Number of Participants With Tumor Recurrence at Baseline in Patients With Diffuse TGCT (d-TGCT) | 1 recurrence | 40 Participants |
Primary
Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT)
Time from baseline to first tumor recurrence was collected from information routinely recorded in the patient files / medical records.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Time from baseline to first tumor recurrence was assessed in participants with available data in the Full Analysis Set.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Baseline | Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT) | 8.4 months |
| Within 1st Year | Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT) | 19.0 months |
| Within 2nd Year | Time From Baseline to First Tumor Recurrence in Patients With Diffuse TGCT (d-TGCT) | 14.8 months |
Primary
Time Since Most Recent Tumor Recurrence Until Baseline in Patients With Diffuse TGCT (d-TGCT)
Time since most recent tumor recurrence was collected from information routinely recorded in the patient files / medical records.
Time frame: at Baseline
Population: Time since last recent tumor recurrence until baseline was assessed in participants with available baseline data in the Full Analysis Set.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Baseline | Time Since Most Recent Tumor Recurrence Until Baseline in Patients With Diffuse TGCT (d-TGCT) | 12.55 months |
Secondary
Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months
The EQ-5D-5L questionnaire measures the patient's quality of life (QoL) based on the 5 dimensions mobility, self care, usual activities, pain/discomfort, and anxiety/depression. The EQ-5D index score ranges from 0 (worst QoL) to 1 (best QoL), where lower scores indicate worse clinical outcome. The EQ-5D VAS reports patient's self rated health ranging from 0 (worst health) to 100 (best health you can imagine), where lower scores indicate worse clinical outcome.
Time frame: Baseline up to end of observation period (approximately 2 years)
Population: Quality of life assessed by EQ-5D index score was assessed in participants with available data in the Full Analysis Set.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Baseline | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D Index Score | 0.714 score on a scale | Standard Deviation 0.2 |
| Baseline | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D VAS | 69.3 score on a scale | Standard Deviation 20.95 |
| Within 1st Year | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D Index Score | 0.742 score on a scale | Standard Deviation 0.18 |
| Within 1st Year | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D VAS | 69.0 score on a scale | Standard Deviation 19.84 |
| Within 2nd Year | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D Index Score | 0.740 score on a scale | Standard Deviation 0.2 |
| Within 2nd Year | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D VAS | 73.0 score on a scale | Standard Deviation 18.23 |
| 18 Months | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D VAS | 69.8 score on a scale | Standard Deviation 20.53 |
| 18 Months | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D Index Score | 0.741 score on a scale | Standard Deviation 0.2 |
| 24 Months | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D Index Score | 0.746 score on a scale | Standard Deviation 0.21 |
| 24 Months | Mean EuroQol Questionnaire (EQ) of 5 Dimensions (5D) Index Score and EQ Visual Analog Scale Score From Baseline Through 24 Months | EQ-5D VAS | 71.7 score on a scale | Standard Deviation 20.74 |
Secondary
Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months
Health resource utilization was assessed by mean number of general practitioner visits, specialist visits, and physical therapy sessions. In addition, the mean number of days in rehabilitation and the number of work days missed were also reported.
Time frame: 13-24 months before baseline up to end of observation period (approximately 2 years)
Population: Health resource utilization was assessed in participants with available data in the Full Analysis Set.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Baseline | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Specialists visits | 7.0 visits | Standard Deviation 8.55 |
| Baseline | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | General practitioner visits | 3.9 visits | Standard Deviation 4.8 |
| Baseline | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Physical therapy sessions | 20.3 visits | Standard Deviation 22.93 |
| Within 1st Year | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Physical therapy sessions | 25.4 visits | Standard Deviation 27.96 |
| Within 1st Year | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | General practitioner visits | 3.0 visits | Standard Deviation 4.6 |
| Within 1st Year | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Specialists visits | 5.2 visits | Standard Deviation 5.89 |
| Within 2nd Year | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | General practitioner visits | 3.8 visits | Standard Deviation 4.03 |
| Within 2nd Year | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Specialists visits | 4.1 visits | Standard Deviation 3.76 |
| Within 2nd Year | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Physical therapy sessions | 17.7 visits | Standard Deviation 14.34 |
| 18 Months | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Specialists visits | 2.6 visits | Standard Deviation 2.8 |
| 18 Months | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | General practitioner visits | 3.3 visits | Standard Deviation 4.83 |
| 18 Months | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Physical therapy sessions | 33.1 visits | Standard Deviation 31.12 |
| 24 Months | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | General practitioner visits | 4.7 visits | Standard Deviation 5.71 |
| 24 Months | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Physical therapy sessions | 31.3 visits | Standard Deviation 32.44 |
| 24 Months | Mean Number of General Practitioner, Specialist, or Physical Therapy Visits to Assess the Health Resource Utilization in Patients With Diffuse TGCT Through 24 Months | Specialists visits | 5.1 visits | Standard Deviation 4.82 |
Secondary
Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months
The median number of days in rehabilitation and the number of work days missed were also reported.
Time frame: 13-24 months before baseline up to end of observation period (approximately 2 years)
Population: Number of days in rehabilitation was assessed in the Full Analysis Set. A participant could have documented days in rehabilitation for the 1st year visit as well as for the 2nd year visit which resulted in a sum greater than the single results per visit/year.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Baseline | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Days in rehabilitation | 15 days |
| Baseline | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Work days missed | 25.50 days |
| Within 1st Year | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Days in rehabilitation | 15 days |
| Within 1st Year | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Work days missed | 16 days |
| Within 2nd Year | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Days in rehabilitation | 12 days |
| Within 2nd Year | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Work days missed | 6 days |
| 18 Months | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Work days missed | 8 days |
| 18 Months | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Days in rehabilitation | 20 days |
| 24 Months | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Days in rehabilitation | 16 days |
| 24 Months | Median Number of Days in Rehabilitation and Work Days Missed in Patients With Diffuse TGCT Through 24 Months | Work days missed | 10 days |
Secondary
Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT)
TGCT outcome status was collected from information routinely recorded in the patient files / medical records.
Time frame: At end of observation period (approximately 2 years post-baseline)
Population: TGCT outcome status was assessed in participants with available data in the Full Analysis Set.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological residual disease (only pts with radiological residual disease): Watchful waiting | 84 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological residual disease (only pts with radiological residual disease): Awaiting surgery | 4 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | TCGT status at end of observational period: No evidence of disease (NED) | 39 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | TCGT status at end of observational period: Radiological residual disease | 107 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | TCGT status at end of observational period: Radiological recurrent disease | 20 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | TCGT status at end of observational period: Missing | 10 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | NED (only pts with NED): With complaints | 21 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | NED (only pts with NED): Without complaints | 18 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | NED (only pts with NED): Missing | 0 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological residual disease (only pts with radiological residual disease): Awaiting medication | 16 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological residual disease (only pts with radiological residual disease):Awaiting other treatment | 2 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological residual disease (only pts with radiological residual disease): Missing | 1 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological recurrent disease (only pts with radiological recurrent disease): Watchful waiting | 12 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological recurrent disease (only pts with radiological recurrent disease): Awaiting surgery | 6 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological recurrent disease (only pts with radiological recurrent disease): Awaiting medication | 1 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological recurrent disease (only pts with radiological recurrent): Awaiting other treatment | 1 Participants |
| Baseline | Overall TGCT Outcome Status in Patients With Diffuse TGCT (d-TGCT) | Radiological recurrent disease (only pts with radiological recurrent disease): Missing | 0 Participants |