Rhabdomyosarcoma
Conditions
Keywords
pediatric rhabdomyosarcoma, proton radiation therapy
Brief summary
The main purpose of this study is to see if using proton beam radiation therapy instead of photon beam radiation therapy can reduce side effects from radiation treatment for rhabdomyosarcoma. Photon beam radiation is the standard type of radiation for treating most rhabdomyosarcoma and many other types of cancer. Photon beam radiation enters the body and passes through healthy tissue, encounters the tumor, then leaves the body through healthy tissue. A beam of proton radiation enters the body and passes through healthy tissue, encounters tumor, but then stops. This means that less healthy tissue is affected by proton beam radiation than by photon beam radiation.
Detailed description
* A special device is made for each participant to help them hold still during the treatment. This may be in the form of a mask or a custom made foam cradle depending on the area to be treated. * Radiation treatments will be given once per day, 5 days a week for a total of 4 to 6 weeks, depending upon how much total dose the tumor requires. * Participants will be seen once per week by their radiation doctor to monitor health and record any side effects from treatment. * After the radiation treatments are completed, participants will be required to undergo further tests and evaluations for several years following treatment.
Interventions
RADIATIONProton Beam Radiation
Once per day, 5 days a week for a total of 4 to 6 weeks.
Sponsors
M.D. Anderson Cancer Center
Brigham and Women's Hospital
Boston Children's Hospital
Dana-Farber Cancer Institute
National Institutes of Health (NIH)
National Cancer Institute (NCI)
Massachusetts General Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 30 Years
Healthy volunteers
No
Inclusion criteria
* Patients with biopsy proven newly diagnosed rhabdomyosarcoma. * Patients less than or equal to 30 years of age. * Patients must be treated with a standardly accepted chemotherapy regimen. * May not have metastatic disease unless aged 2-10 with embryonal histology. * Must be willing to receive follow-up care for a minimum of five years after treatment at MGH and annual visits unless it is too difficult to return to MGH for follow-up care. In that event, they must be willing to have their outside medical information released to us to track the results. * Timing of radiation must be according to the IRB protocol upon which the patient is treated within either 35 days of last chemotherapy or surgery.
Exclusion criteria
* Life expectancy of less than 2 years. * Co-morbidities that would make the use of radiation too toxic to deliver safely, such as serious local injury or collagen vascular disease. * Patients who are pregnant * Previous treatment with radiation therapy.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cumulative Incidence (Estimated Percent of Participants) With Grade 3 or Higher Toxicity in Follow-up | 5 years from the start of radiation treatment | Cumulative incidence of participants who experienced a toxicity defined as either grade 3, 4, or 5 after the completion of radiation therapy in pediatric patients with pediatric rhabdomyosarcomas. Late toxicity is scored on a yearly follow-up basis for at least five years. Late toxicities will be graded according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0. Cumulative incidence is shown at 5 years follow-up. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Who Experienced an Acute Toxicity by Grade | 3 months from radiation treatment | Percentage of participants who experienced an acute toxicity following completion of radiation treatment by grade. Acute toxicity will be scored at weekly status check visits by the radiation oncology nurse or doctor. In addition, acute toxicity will be scored at least one time within the 3 months following completion of radiation therapy. The recommended evaluation time for acute toxicity after radiotherapy was 6 weeks following completion, but may be at any time during the 3 months after treatment. Acute toxicities are graded according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0. Toxicities are graded on a scale of 1 to 5. A higher grade indicates a worse outcome with 1 being mild, 4 being life-threatening, and 5 being death. |
| Dosimetric Comparison | 4 years from the start of radiation treatment | Comparison of dose distribution to tumor and surrounding normal structures using dose volume histograms (DVH) generated from the proton plan used to treat the patient and the photon plan generated for comparison purposes. Percent of normal tissue spared was calculated using the following equation: (100 x (mean x-ray dose - mean proton dose))/mean X-ray dose. No measure of dispersion was calculated for these estimates. The data is no longer available. |
| Local Control | 5 years from the start of radiation therapy | Estimated survival probability (defined as being free of a local recurrence) was calculated using Grays formula for competing risks |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Proton Beam Radiation Proton Beam Radiation
Proton Beam Radiation: Once per day, 5 days a week for a total of 4 to 6 weeks. | 114 |
| Total | 114 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Patient found ineligible upon chart review | 1 |
Baseline characteristics
| Characteristic | Proton Beam Radiation |
|---|---|
| Age, Continuous | 4.3 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 7 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 19 Participants |
| Race (NIH/OMB) White | 86 Participants |
| Region of Enrollment United States | 114 participants |
| Sex: Female, Male Female | 51 Participants |
| Sex: Female, Male Male | 63 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 28 / 114 |
| other Total, other adverse events | 113 / 114 |
| serious Total, serious adverse events | 6 / 114 |
Outcome results
Primary
Cumulative Incidence (Estimated Percent of Participants) With Grade 3 or Higher Toxicity in Follow-up
Cumulative incidence of participants who experienced a toxicity defined as either grade 3, 4, or 5 after the completion of radiation therapy in pediatric patients with pediatric rhabdomyosarcomas. Late toxicity is scored on a yearly follow-up basis for at least five years. Late toxicities will be graded according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0. Cumulative incidence is shown at 5 years follow-up.
Time frame: 5 years from the start of radiation treatment
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Proton Beam Radiation | Cumulative Incidence (Estimated Percent of Participants) With Grade 3 or Higher Toxicity in Follow-up | 10.9 Percentage of patients |
95% CI: [5.7, 18]
Secondary
Dosimetric Comparison
Comparison of dose distribution to tumor and surrounding normal structures using dose volume histograms (DVH) generated from the proton plan used to treat the patient and the photon plan generated for comparison purposes. Percent of normal tissue spared was calculated using the following equation: (100 x (mean x-ray dose - mean proton dose))/mean X-ray dose. No measure of dispersion was calculated for these estimates. The data is no longer available.
Time frame: 4 years from the start of radiation treatment
Population: Patients who had DVH's generated from proton plans for comparison
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Proton Beam Radiation | Dosimetric Comparison | Hypothalamus | 88 Percentage of normal tissue spared of RT |
| Proton Beam Radiation | Dosimetric Comparison | Pituitary | 73 Percentage of normal tissue spared of RT |
| Proton Beam Radiation | Dosimetric Comparison | Lens (Contra) | 100 Percentage of normal tissue spared of RT |
| Proton Beam Radiation | Dosimetric Comparison | Maxilla | 42 Percentage of normal tissue spared of RT |
p-value: <0.01t-test, 2 sided
p-value: <0.01t-test, 2 sided
p-value: <0.01t-test, 2 sided
p-value: 0.02t-test, 2 sided
Secondary
Local Control
Estimated survival probability (defined as being free of a local recurrence) was calculated using Grays formula for competing risks
Time frame: 5 years from the start of radiation therapy
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Proton Beam Radiation | Local Control | 80.9 percent probability |
95% CI: [73, 87.7]
Secondary
Percentage of Participants Who Experienced an Acute Toxicity by Grade
Percentage of participants who experienced an acute toxicity following completion of radiation treatment by grade. Acute toxicity will be scored at weekly status check visits by the radiation oncology nurse or doctor. In addition, acute toxicity will be scored at least one time within the 3 months following completion of radiation therapy. The recommended evaluation time for acute toxicity after radiotherapy was 6 weeks following completion, but may be at any time during the 3 months after treatment. Acute toxicities are graded according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0. Toxicities are graded on a scale of 1 to 5. A higher grade indicates a worse outcome with 1 being mild, 4 being life-threatening, and 5 being death.
Time frame: 3 months from radiation treatment
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Proton Beam Radiation | Percentage of Participants Who Experienced an Acute Toxicity by Grade | Grade 1 | 110 Participants |
| Proton Beam Radiation | Percentage of Participants Who Experienced an Acute Toxicity by Grade | Grade 2 | 92 Participants |
| Proton Beam Radiation | Percentage of Participants Who Experienced an Acute Toxicity by Grade | Grade 3 | 30 Participants |
| Proton Beam Radiation | Percentage of Participants Who Experienced an Acute Toxicity by Grade | Grade 4 | 5 Participants |
| Proton Beam Radiation | Percentage of Participants Who Experienced an Acute Toxicity by Grade | Grade 5 | 0 Participants |