Neurofibromatosis Type 1, Sarcoma
Conditions
Keywords
adult neurofibrosarcoma, childhood neurofibrosarcoma, metastatic childhood soft tissue sarcoma, nonmetastatic childhood soft tissue sarcoma, stage IV adult soft tissue sarcoma, neurofibromatosis type 1
Brief summary
RATIONALE: Drugs used in chemotherapy, such as doxorubicin, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill tumor cells. Giving combination chemotherapy with or without radiation therapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving combination chemotherapy after surgery may kill any tumor cells that remain after surgery. PURPOSE: This phase II trial is studying how well combination chemotherapy works in treating patients with stage III or stage IV malignant peripheral nerve sheath tumors.
Detailed description
OBJECTIVES: Primary * Determine the clinical response rate (complete and partial) in patients with sporadic or neurofibromatosis type 1 (NF1)-associated high-grade stage III or IV malignant peripheral nerve sheath tumors (MPNSTs) after treatment with 4 courses of chemotherapy comprising doxorubicin hydrochloride and ifosfamide (IA) followed by etoposide and ifosfamide (IE). Secondary * Evaluate the utility of fludeoxyglucose F18 positron emission tomography (\^18FDG-PET) and automated MRI volumetric tumor analysis as tools to assess response to treatment. * Correlate response evaluation by 2-dimensional WHO criteria, 1-dimensional RECIST criteria, \^18FDG-PET, and volumetric MRI with percent necrosis in tumor specimens from patients who undergo surgery for local control after chemotherapy. * Evaluate the response of plexiform neurofibroma(s) (if present) to chemotherapy using WHO criteria and automated volumetric MRI analysis. * Evaluate the molecular biology of sporadic and NF1-associated MPNSTs by performing a detailed pathologic analysis of tumor samples with the goal to analyze if markers can be identified that predict for response to chemotherapy or outcome. * Construct a tissue microarray from submitted tumor samples, that will be used in the future to identify novel targets for treatment of MPNSTs. * Assess if a serum biomarker can be identified, that predicts for the presence of a MPNST versus benign plexiform neurofibroma. * Increase the knowledge of the epidemiology and clinical presentation of NF1-associated MPNSTs. OUTLINE: This is a multicenter study. Patients are stratified according to type of malignant peripheral nerve sheath tumor (MPNST) (sporadic MPNST vs neurofibromatosis type 1 \[NF1\]-associated MPNST). Patients receive 1 of 2 treatment regimens depending on the location of the MPNST and tumor response to chemotherapy. * Chemotherapy and local control by radiotherapy and surgery: Patients receive doxorubicin hydrochloride and ifosfamide (IA) chemotherapy comprising doxorubicin hydrochloride IV over 15 minutes on days 1 and 2 and ifosfamide IV over 1 hour on days 1-5. Treatment repeats every 21 days for 2 courses in the absence of unacceptable toxicity. Patients then receive etoposide and ifosfamide (IE) chemotherapy comprising etoposide IV over 1 hour and ifosfamide IV over 1 hour on days 1-5. Treatment repeats every 21 days for 2 courses in the absence of disease progression or unacceptable toxicity. Patients also receive filgrastim (G-CSF) subcutaneously (SC) after each chemotherapy course beginning on day 6 or 7 and continuing until blood counts recover or pegfilgrastim SC once on day 6 or 7. After recovery from chemotherapy, patients undergo radiotherapy and receive 2 more courses of IE during radiotherapy followed by 2 more courses of IA after completion of radiotherapy. Some patients may then undergo surgery. * Chemotherapy and local control by surgery: Patients receive 2 courses of IA followed by 2 courses of IE as above. After recovery from chemotherapy, patients undergo surgery. After recovery from surgery, patients receive 2 more courses of IA followed by 2 more courses of IE in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed periodically for up to 5 years. PROJECTED ACCRUAL: A total of 74 patients will be accrued for this study.
Interventions
DRUGetoposide
Given IV
DRUGifosfamide
Given IV
PROCEDUREconventional surgery
Patients undergo surgery
RADIATIONradiation therapy
Patients undergo radiotherapy
BIOLOGICALfilgrastim
Given subcutaneously
DRUGdoxorubicin hydrochloride
Given IV
Sponsors
National Cancer Institute (NCI)
Sarcoma Alliance for Research through Collaboration
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
DISEASE CHARACTERISTICS: * Newly diagnosed sporadic or neurofibromatosis type 1 (NF1)-associated high-grade malignant peripheral nerve sheath tumors (MPNSTs) * Stage III or stage IV (metastatic) disease * Measurable disease, defined as at least 1 tumor that is measurable in 2 dimensions on CT scan or MRI PATIENT CHARACTERISTICS: * Ejection fraction normal by echocardiogram or MUGA * Serum creatinine normal for age OR creatinine clearance \> 60 mL/min * SGPT \< 5 times upper limit of normal (ULN) * Bilirubin \< 2.5 times ULN * Absolute neutrophil count ≥ 1,500/mm\^3\* * Hemoglobin ≥ 9.0 g/dL\* * Platelet count ≥ 100,000/mm\^3\* * ECOG performance status 0-2 * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception during and for 6 months after completion of study treatment NOTE: \* Unsupported PRIOR CONCURRENT THERAPY: * No prior chemotherapy for MPNST * Prior surgical resection of MPNST allowed provided residual or recurrent measurable disease is present * Recovered from toxic effects of all prior therapy * At least 3 weeks since prior chemotherapy or biologic therapy for treatment of a plexiform neurofibroma, optical pathway tumor, or other NF1-associated tumor (in patients with NF1) * At least 6 weeks since prior radiotherapy for treatment of a plexiform neurofibroma, optical pathway tumor, or other NF1-associated tumor (in patients with NF1) * At least 4 weeks since prior radiotherapy to the area involved by MPNST * No other concurrent growth factors (e.g., sargramostim \[GM-CSF\] or interleukin-11) * Concurrent epoetin alfa allowed
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Response Rate (Complete Response and Partial Response) | After 4 Cycles (1 cycle=21 days) | WHO criteria was used to determine responses due to the nonspherical shape of most MPNST. Complete Response (CR), Disappearance of all target lesions; Partial response (PR), \>=50% decrease of target lesions. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Utility of Fludeoxyglucose F18 Positron Emission Tomography (18FDG-PET) and Automated MRI Volumetric Tumor Analysis to Assess Response to Treatment | After 4 cycles | Evaluate the utility of fludeoxyglucose F18 positron emission tomography (18FDG-PET) and automated MRI volumetric tumor analysis as tools to assess response to treatment. |
| Response Evaluation Using WHO, RECIST, 18 FDG-PET and Volumetric MRI With Percent Necrosis in Tumor Specimens | After 4 cycles | Correlate response evaluation using WHO, RECIST, 18 FDG-PET and volumetric MRI with percent necrosis in tumor specimens from patients who undergo surgery for local control after chemotherapy. |
| Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | After 4 cycles | Evaluate the molecular biology of sporadic and NF1-associated MPNSTs by performing a detailed pathologic analysis of tumor samples with the goal to analyze if markers can be identified that predict for response to chemotherapy or outcome. |
| Response of Plexiform Neurofibroma to Neoadjuvant Chemotherapy Using Volumetric MRI Analysis | After 4 Cycles (1 cycle=21 days) | Evaluate the response of plexiformneurofibroma (if present) to neoadjuvant chemotherapy using WHO criteria and volumetric MRI analysis as a tool for response assessment |
| Identify the Number of Participants With a Serum Biomarker to Predict the Presence of MPNST Versus Benign Plexiform Neurofibroma | After 4 cycles | Assess if a serum biomarker can be identified that predicts for the presence of a MPNST versus benign plexiform neurofibroma. |
| Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | After 4 cycles | Increase the knowledge of the epidemiology and clinical presentation of NF1-associated MPNSTs. |
| Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | After 4 cycles | Construct a tissue microarray from submitted tumor samples that will be used in the future to identify novel targets for treatment of MPNSTs. The tissue microarray looked at various gene deletions and amplifications. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| NF1 MPNST 2 cycles of ifosfamide + doxorubicin ('IA') followed by 2 cycles of ifosfamide + etoposide ('IE') prior to local control measures (surgery and/or radiation therapy).
Local control with surgery and/or radiation will commence after recovery from toxicities. Patients, who undergo surgery only, will receive 2 more cycles of 'IA' followed by 2 cycles of 'IE' beginning after recovery from surgery. Patients, who receive radiation therapy in addition to surgery, will receive 2 cycles of 'IE' during radiation treatment, as doxorubicin cannot be concurrently administered with radiation therapy, and 2 cycles of 'IA' after completion of radiation treatment.
1 cycle = 21 days Doxo = Doxorubicin 37.5 mg/m2/dose IV over 15 minutes on days 1, 2 Ifos = Ifosfamide 1,800 mg/m2/dose IV over 60 minutes on days 1, 2, 3, 4, 5 Etop = Etoposide 100 mg/m2/dose IV over 60 minutes on days 1, 2, 3, 4, 5 | 34 |
| Sporadic MPNST 2 cycles of ifosfamide + doxorubicin ('IA') followed by 2 cycles of ifosfamide + etoposide ('IE') prior to local control measures (surgery and/or radiation therapy).
Local control with surgery and/or radiation will commence after recovery from toxicities. Patients, who undergo surgery only, will receive 2 more cycles of 'IA' followed by 2 cycles of 'IE' beginning after recovery from surgery. Patients, who receive radiation therapy in addition to surgery, will receive 2 cycles of 'IE' during radiation treatment, as doxorubicin cannot be concurrently administered with radiation therapy, and 2 cycles of 'IA' after completion of radiation treatment.
1 cycle = 21 days Doxo = Doxorubicin 37.5 mg/m2/dose IV over 15 minutes on days 1, 2 Ifos = Ifosfamide 1,800 mg/m2/dose IV over 60 minutes on days 1, 2, 3, 4, 5 Etop = Etoposide 100 mg/m2/dose IV over 60 minutes on days 1, 2, 3, 4, 5 | 14 |
| Total | 48 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Disease Progression | 5 | 1 |
| Overall Study | Other | 5 | 2 |
| Overall Study | Physician Decision | 8 | 3 |
| Overall Study | Withdrawal by Subject | 4 | 2 |
Baseline characteristics
| Characteristic | NF1 MPNST | Sporadic MPNST | Total |
|---|---|---|---|
| Age, Continuous | 33 years | 40 years | 36.5 years |
| Sex/Gender, Customized Female | 12 Participants | 5 Participants | 17 Participants |
| Sex/Gender, Customized Male | 22 Participants | 9 Participants | 31 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 3 / 48 |
| serious Total, serious adverse events | 13 / 48 |
Outcome results
Primary
Number of Participants With Response Rate (Complete Response and Partial Response)
WHO criteria was used to determine responses due to the nonspherical shape of most MPNST. Complete Response (CR), Disappearance of all target lesions; Partial response (PR), \>=50% decrease of target lesions.
Time frame: After 4 Cycles (1 cycle=21 days)
Population: 37/48 patients total were evaluable for response.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| NF1 MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Partial response | 5 Participants |
| NF1 MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Complete response | 0 Participants |
| NF1 MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Stable Disease | 20 Participants |
| NF1 MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Progressive Disease | 3 Participants |
| Sporadic MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Progressive Disease | 1 Participants |
| Sporadic MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Partial response | 4 Participants |
| Sporadic MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Stable Disease | 4 Participants |
| Sporadic MPNST | Number of Participants With Response Rate (Complete Response and Partial Response) | Complete response | 0 Participants |
Secondary
Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue
Construct a tissue microarray from submitted tumor samples that will be used in the future to identify novel targets for treatment of MPNSTs. The tissue microarray looked at various gene deletions and amplifications.
Time frame: After 4 cycles
Population: The number of participants vary in the rows from overall number analyzed, due to the tissue that was available for testing.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | EGFR (7p12) amplification | 3 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | TOPO2A (17q21-q22) amplification | 5 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | Her2/Neu (17q11-q12) amplification | 2 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | Cyclin D1 (11q13) amplification | 1 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | c-MYC (8q24) amplification | 5 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | N-MYC (2p24) amplification | 3 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | NF1 (17q11) deletion | 7 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | p16 (9p21) deletion | 10 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | RB (13q14) deletion | 1 participants |
| NF1 MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | p53 (17q13) deletion | 6 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | p16 (9p21) deletion | 3 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | EGFR (7p12) amplification | 1 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | N-MYC (2p24) amplification | 1 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | TOPO2A (17q21-q22) amplification | 2 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | p53 (17q13) deletion | 2 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | Her2/Neu (17q11-q12) amplification | 0 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | NF1 (17q11) deletion | 0 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | Cyclin D1 (11q13) amplification | 2 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | RB (13q14) deletion | 2 participants |
| Sporadic MPNST | Construct Tissue Microarray to Identify Novel Targets for Treatment for the Number of Participants With Available Tissue | c-MYC (8q24) amplification | 0 participants |
Secondary
Identify the Number of Participants With a Serum Biomarker to Predict the Presence of MPNST Versus Benign Plexiform Neurofibroma
Assess if a serum biomarker can be identified that predicts for the presence of a MPNST versus benign plexiform neurofibroma.
Time frame: After 4 cycles
Population: Data looked at response evaluable patients with MPNST and focused on the TOPO2A gene being amplified.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| NF1 MPNST | Identify the Number of Participants With a Serum Biomarker to Predict the Presence of MPNST Versus Benign Plexiform Neurofibroma | NF1 patients with gene amplified | 3 Participants |
| NF1 MPNST | Identify the Number of Participants With a Serum Biomarker to Predict the Presence of MPNST Versus Benign Plexiform Neurofibroma | Sporadic patients with gene amplified | 2 Participants |
Secondary
Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response
Evaluate the molecular biology of sporadic and NF1-associated MPNSTs by performing a detailed pathologic analysis of tumor samples with the goal to analyze if markers can be identified that predict for response to chemotherapy or outcome.
Time frame: After 4 cycles
Population: The rows are among three different categories: Histologic Variant, Cellularity and Necrosis.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Conventional | 15 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Perineural | 1 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Epithelioid | 0 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Divergent | 1 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Mixed histology | 9 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Low Cellularity | 0 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Moderate Cellularity | 5 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | High Cellularity | 21 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis- absent | 5 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis 1-10% | 8 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis 10-50% | 10 Participants |
| NF1 MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis >50% | 3 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis 10-50% | 5 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Conventional | 7 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Moderate Cellularity | 4 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Perineural | 0 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis 1-10% | 2 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Epithelioid | 2 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | High Cellularity | 7 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Divergent | 0 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis >50% | 3 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Histologic Variant- Mixed histology | 2 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Necrosis- absent | 1 Participants |
| Sporadic MPNST | Perform Pathologic Analysis of Tumor Samples to Analyze the Number of Participants With Markers as Predictors of Response | Low Cellularity | 0 Participants |
Secondary
Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs.
Increase the knowledge of the epidemiology and clinical presentation of NF1-associated MPNSTs.
Time frame: After 4 cycles
Population: Clinical evaluation of patients with NF1 was performed at trial enrollment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | ≥10 subcutaneous neurofibromas | 10 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | 6 or more CAL | 17 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Intertriginous freckling | 19 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Neurofibromas | 26 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Plexiform neurofibroma | 12 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Paraspinal neurofibromas | 12 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | ≥10 cutaneous neurofibromas | 13 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Optic glioma | 1 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Glioma | 1 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Scoliosis | 5 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Intellectual delay | 8 Participants |
| NF1 MPNST | Provide Epidemiology and Clinical Presentation of the Number of Participants With NF1-associated MPNSTs. | Hypertension | 8 Participants |
Secondary
Response Evaluation Using WHO, RECIST, 18 FDG-PET and Volumetric MRI With Percent Necrosis in Tumor Specimens
Correlate response evaluation using WHO, RECIST, 18 FDG-PET and volumetric MRI with percent necrosis in tumor specimens from patients who undergo surgery for local control after chemotherapy.
Time frame: After 4 cycles
Population: 33 patients were evaluable for response after cycle 4, response evaluation using WHO and RECIST was performed. Response evaluation was not assessed with 18 FDG-PET and volumetric MRI.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| NF1 MPNST | Response Evaluation Using WHO, RECIST, 18 FDG-PET and Volumetric MRI With Percent Necrosis in Tumor Specimens | Responses in agreement | 29 Participants |
| NF1 MPNST | Response Evaluation Using WHO, RECIST, 18 FDG-PET and Volumetric MRI With Percent Necrosis in Tumor Specimens | Responses were not in agreement | 4 Participants |
Secondary
Response of Plexiform Neurofibroma to Neoadjuvant Chemotherapy Using Volumetric MRI Analysis
Evaluate the response of plexiformneurofibroma (if present) to neoadjuvant chemotherapy using WHO criteria and volumetric MRI analysis as a tool for response assessment
Time frame: After 4 Cycles (1 cycle=21 days)
Population: MRI imaging of the MPNST and plexiform neurofibroma component was not sufficient to allow for volumetric analysis over time. Reasons include differences in imaging technique over time and incomplete coverage of the entire tumor.
Secondary
Utility of Fludeoxyglucose F18 Positron Emission Tomography (18FDG-PET) and Automated MRI Volumetric Tumor Analysis to Assess Response to Treatment
Evaluate the utility of fludeoxyglucose F18 positron emission tomography (18FDG-PET) and automated MRI volumetric tumor analysis as tools to assess response to treatment.
Time frame: After 4 cycles
Population: This assessment involved 18FDG-PET and MRI imaging of the MPNST and plexiform neurofibroma component. Due to technical issues with MRI imaging, data was not reliably collected from any study participant to allow for meaningful analysis.