Verification of surface-guided radiation therapy (SGRT) alignment for proton breast and chest wall patients by comparison to CT-on-rails and kV-2D alignment.

BACKGROUND: Surface-guided radiation therapy (SGRT) systems have been widely installed and utilized on linear accelerators. However, the use of SGRT with proton therapy is still a newly developing field, and published reports are currently very limited. PURPOSE: To assess the clinical application and alignment agreement of SGRT with CT-on-rails (CTOR) and kV-2D image-guided radiation therapy (IGRT) for breast treatment using proton therapy. METHODS: Four patients receiving breast or chest wall treatment with proton therapy were the subjects of this study. Patient #1's IGRT modalities were a combination of kV-2D and CTOR. CTOR was the only imaging modality for patients #2 and #3, and kV-2D was the only imaging modality for patient #4. The patients' respiratory motions were assessed using a 2-min surface position recorded by the SGRT system during treatment. SGRT offsets reported after IGRT shifts were recorded for each fraction of treatment. The agreement between SGRT and either kV-2D or CTOR was evaluated. RESULTS: The respiratory motion amplitude was <4 mm in translation and <2.0° in rotation for all patients. The mean and maximum amplitude of SGRT offsets after application of IGRT shifts were ≤(2.6 mm, 1.6° ) and (6.8 mm, 4.5° ) relative to kV-2D-based IGRT; ≤(3.0 mm, 2.6° ) and (5.0 mm, 4.7° ) relative to CTOR-based IGRT without breast tissue inflammation. For patient #3, breast inflammation was observed for the last three fractions of treatment, and the maximum SGRT offsets post CTOR shifts were up to (14.0 mm, 5.2° ). CONCLUSIONS: Due to the overall agreement between SGRT and IGRT within reasonable tolerance, SGRT has the potential to serve as a valuable auxiliary IGRT tool for proton breast treatment and may improve the efficiency of proton breast treatment.

Mental health disorders are more common in patients with Hodgkin lymphoma and may negatively impact overall survival.

BACKGROUND: Long-term mental health outcomes were characterized in patients who were diagnosed with Hodgkin lymphoma (HL), and risk factors for the development of mental health disorders were identified. METHODS: Patients who were diagnosed with HL between 1997 and 2014 were identified in the Utah Cancer Registry. Each patient was matched with up to five individuals from a general population cohort identified within the Utah Population Database, a unique source of linked records that includes patient and demographic data. RESULTS: In total, 795 patients who had HL were matched with 3575 individuals from the general population. Compared with the general population, patients who had HL had a higher risk of any mental health diagnosis (hazard ratio, 1.77; 95% confidence interval, 1.57-2.00). Patients with HL had higher risks of anxiety, depression, substance-related disorders, and suicide and intentional self-inflicted injuries compared with the general population. The main risk factor associated with an increased risk of being diagnosed with mental health disorders was undergoing hematopoietic stem cell transplantation, with a hazard ratio of 2.06 (95% confidence interval, 1.53-2.76). The diagnosis of any mental health disorder among patients with HL was associated with a detrimental impact on overall survival; the 10-year overall survival rate was 70% in patients who had a mental health diagnosis compared with 86% in those patients without a mental health diagnosis (p < .0001). CONCLUSIONS: Patients who had HL had an increased risk of various mental health disorders compared with a matched general population. The current data illustrate the importance of attention to mental health in HL survivorship, particularly for patients who undergo therapy with hematopoietic stem cell transplantation.

NCCN Guidelines® Insights: Gastrointestinal Stromal Tumors, Version 2.2022.

Gastrointestinal stromal tumors (GIST) are the most common type of soft tissue sarcoma that occur throughout the gastrointestinal tract. Most of these tumors are caused by oncogenic activating mutations in the KIT or PDGFRA genes. The NCCN Guidelines for GIST provide recommendations for the diagnosis, evaluation, treatment, and follow-up of patients with these tumors. These NCCN Guidelines Insights summarize the panel discussion behind recent important updates to the guidelines, including revised systemic therapy options for unresectable, progressive, or metastatic GIST based on mutational status, and updated recommendations for the management of GIST that develop resistance to specific tyrosine kinase inhibitors.

Soft Tissue Sarcoma, Version 2.2022, NCCN Clinical Practice Guidelines in Oncology.

Soft tissue sarcomas (STS) are rare malignancies of mesenchymal cell origin that display a heterogenous mix of clinical and pathologic characteristics. STS can develop from fat, muscle, nerves, blood vessels, and other connective tissues. The evaluation and treatment of patients with STS requires a multidisciplinary team with demonstrated expertise in the management of these tumors. The complete NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Soft Tissue Sarcoma provide recommendations for the diagnosis, evaluation, and treatment of extremity/superficial trunk/head and neck STS, as well as retroperitoneal/intra-abdominal STS, desmoid tumors, and rhabdomyosarcoma. This portion of the NCCN Guidelines discusses general principles for the diagnosis and treatment of retroperitoneal/intra-abdominal STS, outlines treatment recommendations, and reviews the evidence to support the guidelines recommendations.

Factors Associated with Nodal Pathologic Complete Response Among Breast Cancer Patients Treated with Neoadjuvant Chemotherapy: Results of CALGB 40601 (HER2+) and 40603 (Triple-Negative) (Alliance).

BACKGROUND: De-escalation of axillary surgery after neoadjuvant chemotherapy (NAC) requires careful patient selection. We seek to determine predictors of nodal pathologic complete response (ypN0) among patients treated on CALGB 40601 or 40603, which tested NAC regimens in HER2+ and triple-negative breast cancer (TNBC), respectively. PATIENTS AND METHODS: A total of 760 patients with stage II-III HER2+ or TNBC were analyzed. Those who had axillary surgery before NAC (N = 122), or who had missing pretreatment clinical nodal status (cN) (N = 58) or ypN status (N = 41) were excluded. The proportion of patients with ypN0 disease was estimated for those with and without breast pathologic complete response (pCR) according to pretreatment nodal status. RESULTS: In 539 patients, the overall ypN0 rate was 76.3% (411/539) to 93.2% (245/263) in patients with breast pCR and 60.1% (166/276) with residual breast disease (RD) (P < 0.0001). For patients who were cN0 pretreatment, the ypN0 rate was 88.8% (214/241), 96.3% (104/108) with breast pCR, and 82.7% (110/133) with RD. For patients who were cN1, 66.2% (157/237) converted to ypN0, 91.7% (111/121) with breast pCR and 39.7% (46/116) with RD. For patients who were cN2/3, 65.6% (40/61) converted to ypN0, 88.2% (30/34) with breast pCR and 37.0% (10/27) with RD. On multivariable analysis, only pretreatment clinical nodal status and breast pCR/RD were associated with ypN0 status (both P < 0.0001). CONCLUSIONS: Breast pCR and pretreatment nodal status are predictive of ypN0 axillary nodal involvement, with < 5% residual nodal disease among cN0 patients who experience breast pCR. These findings support the incorporation of axillary surgery de-escalation strategies into NAC trials.

The use and efficacy of chemotherapy and radiotherapy in children and adults with pilocytic astrocytoma.

PURPOSE: The aim of this study was to understand the use of chemotherapy (CMT) and radiotherapy (RT) in pilocytic astrocytoma (PA) and their impact on overall survival (OS). METHODS: Data from the National Cancer Database (NCDB) for patients with non-metastatic WHO grade I PA from 2004 to 2014 were analyzed. Pearson's chi-squared test and multivariate logistic regression analyses were performed to assess the distribution of demographic, clinical, and treatment factors. Inverse probability of treatment weighting (IPTW) was used to account for differences in baseline characteristics. Kaplan-Meier analyses and doubly-robust estimation with multivariate Cox proportional hazards modeling were used to analyze OS. RESULTS: Of 3865 patients analyzed, 294 received CMT (7.6%), 233 received RT (6.0%), and 42 (1.1%) received both. On multivariate analyses, decreasing extent of surgical resection was associated with receipt of both CMT and RT. Brainstem tumors were associated with RT, optic nerve tumors were associated with CMT. Cerebellar tumors were inversely associated with both CMT and RT. Younger age was associated with receipt of CMT; conversely, older age was associated with receipt of RT. After IPTW, receipt of CMT and/or RT were associated with an OS decrement compared with matched patients treated with surgery alone or observation (HR 3.29, p < 0.01). CONCLUSIONS: This is the largest study to date to examine patterns of care and resultant OS outcomes in PA. We identified patient characteristics associated with receipt of CMT and RT. After propensity score matching, receipt of CMT and/or RT was associated with decreased OS.

Acute lymphoblastic leukemia.

The survival of patients with acute lymphoblastic leukemia (ALL) has improved significantly with the use of intensive multimodality treatment regimens including chemotherapy, high-dose chemotherapy and stem cell rescue, and radiation therapy when indicated. This report summarizes the treatment strategies, especially radiation therapy in the Children's Oncology Group for children with ALL. Currently, radiation therapy is only indicated for children with high-risk CNS involvement at diagnosis or relapse, testicular relapse and as part of the conditioning regimen for hematopoietic stem cell transplantation. Future research strategies regarding the indications for and dosages of radiation therapy and novel radiation techniques are discussed.

Gliomas, germ cell tumors, and craniopharyngioma.

This report summarizes the current multimodality treatment approaches for children with low- and high-grade gliomas, germinoma, and nongerminomatous germ cell tumors, and craniopharyngiomas used in the Children's Oncology Group (COG) and the International Society of Pediatric Oncology (SIOP). Treatment recommendations are provided in the context of historical approaches regarding the roles of surgery, radiation, and chemotherapy. Future research strategies for these tumors in both COG and SIOP are also discussed.

NCCN Guidelines Insights: Soft Tissue Sarcoma, Version 1.2021.

The NCCN Guidelines for Soft Tissue Sarcoma provide recommendations for the diagnosis, evaluation, treatment, and follow-up for patients with soft tissue sarcomas. These NCCN Guidelines Insights summarize the panel discussion behind recent important updates to the guidelines, including the development of a separate and distinct guideline for gastrointestinal stromal tumors (GISTs); reconception of the management of desmoid tumors; inclusion of further recommendations for the diagnosis and management of extremity/body wall, head/neck sarcomas, and retroperitoneal sarcomas; modification and addition of systemic therapy regimens for sarcoma subtypes; and revision of the principles of radiation therapy for soft tissue sarcomas.

The radiation dose-response of non-retroperitoneal soft tissue sarcoma with positive margins: An NCDB analysis.

OBJECTIVES: Positive margins can increase the risk of local recurrence of soft tissue sarcomas (STS). Utilizing a national registry, we investigated patterns of care and overall survival (OS) of patients with margin-positive non-retroperitoneal STS who received preoperative radiation therapy, adjuvant radiation therapy, or both. METHODS: Adult patients with non-retroperitoneal STS who underwent resection and RT from 2004 to 2015 were included. Kaplan-Meier, log-rank analysis, and Cox regression analysis were performed. RESULTS: We identified 5726 patients. Most had a tumor size >5 cm (60%), grade 3 disease (67%), and microscopically positive margins (57%). Compared to ≤50.4 Gy, a dose of 66 to 69.99 Gy was associated with decreased risk of death on multivariate analysis (HR 0.69, 95%; CI, 0.50-0.94). Receipt of a boost was associated with decreased risk of death on univariate analysis (HR 0.54, 95%; CI, 0.29-0.99). In patients with grade 2 to 3 tumors without the gross disease, there was an OS benefit associated with a boost on multivariate analysis (HR 0.39, 95%; CI, 0.16-0.97). CONCLUSION: This analysis appears to show an OS benefit of dose escalation to 66 to 69 Gy for margin-positive non-retroperitoneal STS. A Postoperative boost is associated with higher OS in grade 2 to 3 STS without the gross disease.

The value of high-dose radiotherapy in intracranial ependymoma.

BACKGROUND: We sought to evaluate the impact of adjuvant radiotherapy dose on overall survival (OS) after surgical resection for localized intracranial ependymoma. PROCEDURE: The National Cancer Database (NCDB) was queried from 2004 to 2015 for patients of all ages with intracranial WHO grade II to III ependymoma treated with surgery and 4500 to 7000 cGy of adjuvant radiotherapy. Pearson χ2 test and multivariate logistic regression analyses were used to assess clinicodemographic factors and patterns of care. After propensity-score matching, OS was assessed with Kaplan-Meier analyses and doubly robust estimation with multivariate Cox proportional hazards modeling. RESULTS: Of the 1153 patients meeting criteria, 529 (46%) received ≤ 5400 cGy and 624 (54%) received > 5400 cGy. At a median follow-up of 54.5 months, an OS benefit was observed for > 5400 cGy in pediatric patients aged 2-18 years (hazard ratio [HR] 0.53; 95% confidence interval [CI] 0.28-0.99, P = 0.047). No OS difference was found between ≤ 5400 cGy and > 5400 cGy in pediatric patients aged < 2 years (P = 0.819) or in adults (P = 0.180). Increasing age, WHO grade III, subtotal resection, and receipt of chemotherapy portended worse OS. Age 2 to 18 years, WHO III grade, supratentorial location, and receipt of chemotherapy were associated with receiving > 5400 cGy. CONCLUSION: Adjuvant radiotherapy dose > 5400 cGy was associated with improved OS for children aged 2-18 years with WHO grade II-III intracranial ependymoma. No OS benefit was found with > 5400 cGy in adults or children less than two years of age.

Is radiation indicated for young women with early stage, node-negative breast cancer after mastectomy? A multi-institution, retrospective review.

The role of post-mastectomy radiation for women with node negative, early stage disease is not well-defined. The purpose of this study is to more clearly define a subset of women who are ≤40 years of age with T1-T2, node negative breast cancer who may benefit from post-mastectomy radiation. Using tumor registries at two institutions, we identified 219 women ≤40 years of age with T1-T2, node negative breast cancer treated with mastectomy. Of these 219 patients, 38 received post-mastectomy radiation and 181 did not. Kaplan-Meier methods and cox proportional-hazards regression models were employed for statistical analysis. There were no locoregional failures in the women receiving post mastectomy radiation, which lead to a nonsignificant increase in freedom from locoregional recurrence (P=.08). For women not receiving post-mastectomy radiation, freedom from locoregional recurrence was 94.7% and 89.7% at 5- and 10-years. Lymphovascular space invasion (LVSI) was the only factor predictive of locoregional recurrence. For women without LVSI, freedom from locoregional recurrence was 96.0% and 93.3% at 5- and 10-years respectively. For women with LVSI who did not receive post mastectomy radiation, freedom from locoregional recurrence was 89.1% at 5-years. There were no failures in the women with LVSI who received post mastectomy radiation. For women ≤40 years of age with T1-2, node negative breast cancer treated with mastectomy and no post-mastectomy radiation, locoregional control is excellent in the absence of LVSI, regardless of other risk factors. In the presence of LVSI (regardless of other risk factors), the risk of locoregional recurrence is high and appears to be decreased with post-mastectomy radiation.

Disparities in care for elderly women with endometrial cancer adversely effects survival.

PURPOSE: Elderly women with endometrial cancer are at increased risk of local recurrence and cancer-specific death compared to younger women. We sought to investigate adjuvant radiotherapy (RT) practice patterns and effects on survival in elderly women with endometrial cancer. METHODS: Women from the National Cancer Data Base (NCDB) with FIGO IA grade 3 to FIGO IVA endometrial cancer diagnosed from 2004-2013 were included. Chi square analysis was used to compare the elderly (80+) and non-elderly women (18-79) and women who received RT and those that did not. Univariate and multivariate logistic regression were used to determine predictors of receipt of oncologic surgery and adjuvant RT. Univariate and multivariate Cox survival analyses were performed to examine the effect of radiotherapy on survival. Propensity score matching and shared frailty analysis were done in the elderly cohort. RESULTS: We identified 48,871 women for analysis. Rates of oncologic surgery were higher in the women 80+ compared with rates of adjuvant RT (95% versus 34%). Rates of RT receipt were higher in non-elderly women (48% versus 34%, p<0.001). Age over 80 was a negative predictive factor (OR 0.62, p<0.001) for receipt of adjuvant RT and oncologic surgery (OR 0.81, p=0.03). Adjuvant RT was associated with a decreased risk of death in elderly (HR 0.79, p<0.001) and non-elderly women (HR 0.77, p<0.001). CONCLUSION: Endometrial cancer patients over age 80 have similar rates of oncologic surgery as younger women but are significantly less likely to receive adjuvant RT, and this negatively impacts their survival.

Hodgkin Lymphoma Version 1.2017, NCCN Clinical Practice Guidelines in Oncology.

This portion of the NCCN Guidelines for Hodgkin lymphoma (HL) focuses on the management of classical HL. Current management of classical HL involves initial treatment with chemotherapy or combined modality therapy followed by restaging with PET/CT to assess treatment response using the Deauville criteria (5-point scale). The introduction of less toxic and more effective regimens has significantly advanced HL cure rates. However, long-term follow-up after completion of treatment is essential to determine potential long-term effects.

The Addition of Adjuvant Chemotherapy to Radiation in Early-Stage High-Risk Endometrial Cancer: Survival Outcomes and Patterns of Care.

OBJECTIVE: Early-stage high-risk endometrial cancer (HREC) treated with adjuvant radiotherapy (aRT) alone has been associated with an increased risk of distant relapse. The addition of chemotherapy to radiotherapy (aCRT) may benefit overall survival (OS). We investigated the patterns-of-care and OS benefit of aCRT in HREC by analyzing a large national registry. METHODS: Our query was limited to patients with the International Federation of Gynecology and Obstetrics stage IB and II HREC with either papillary serous, clear cell, or grade 3 adenocarcinoma, diagnosed between 2004 and 2012. Logistic and Cox regression analyses were utilized to identify predictors of aCRT use and OS, respectively. Survival analysis was performed with Kaplan Meier and log-rank methods. Propensity score matching was employed to decrease the potential influence of selection bias. RESULTS: A total of 11,746 patients were identified for analysis with 8206 (69.9%) receiving aCRT, and 3540 (30.1%) received aRT. Predictors of aCRT included International Federation of Gynecology and Obstetrics stage II (odds ratio [OR], 1.39; 95% confidence interval [CI], 1.22-1.57), papillary serous (OR, 9.44; 95% CI, 8.22-10.85) or clear cell (OR, 3.21; 95% CI, 2.59-3.97) histology, lymph nodes removed (OR, 1.48; 95% CI, 1.31-1.69), and receipt of brachytherapy alone (OR, 1.55; 95% CI, 1.36-1.78). Estimated 5-year OS was 75.2% for patients receiving aRT only and 79.2% for those receiving aCRT (P < 0.001). When compared with aRT, aCRT was associated with improved OS on multivariate (hazard ratio, 0.78; 95% CI, 0.61-0.99) analysis. A univariate shared-frailty Cox regression after propensity score matching revealed persistence of the OS benefit with aCRT (hazard ratio, 0.74; 95% CI, 0.65-0.84). CONCLUSIONS: The addition of adjuvant chemotherapy to radiation in HREC is associated with improved OS. Multiple demographic and clinical factors significantly influence the choice of adjuvant therapy in this setting.

Post-mastectomy Radiotherapy for pT3N0 Breast Cancers: A Retrospective, Multi-Institution Review.

The role of post-mastectomy radiotherapy for pT3N0 breast cancers remains undefined. The purpose of this study was to report institutional outcomes for women with pT3N0 breast cancers treated with and without post-mastectomy radiotherapy. We collected data from two large tumor registries on pT3N0 breast cancers diagnosed between 1985 and 2014. Kaplan-Meier estimates were used to analyze freedom from local-regional recurrence (FFLR), relapse free survival, and overall survival. This analysis identified 93 women with pT3N0 breast cancers. Of these, 53 received post-mastectomy radiotherapy and 40 did not. Median follow-up was 6.2 years and 5.3 years in the non-post-mastectomy radiotherapy and post-mastectomy radiotherapy cohorts, respectively. Women not undergoing post-mastectomy radiotherapy were more likely to be diagnosed in the 1980s and 1990s and were less likely to receive systemic therapies than women receiving post-mastectomy radiotherapy (p < 0.05). There was a trend toward increased FFLR in the women receiving post-mastectomy radiotherapy (p = 0.15). FFLR in the post-mastectomy radiotherapy cohort was 98% at both 5 and 10 years. For women not receiving post-mastectomy radiotherapy, FFLR was 88% at both 5 and 10 years. Women not receiving post-mastectomy radiotherapy in our study had an isolated local-regional failure rate of 12% at 10 years, despite receiving inferior systemic treatment by current standards. Local-regional control after post-mastectomy radiotherapy for pT3N0 breast cancers was excellent. Further research is needed to define post-mastectomy radiotherapy indications for this patient population when receiving chemotherapy and endocrine therapy in line with current guidelines.

Chemoradiation versus chemotherapy or radiation alone in stage III endometrial cancer: Patterns of care and impact on overall survival.

PURPOSE: We aimed to investigate the patterns-of-care and overall survival (OS) benefit of aCRT versus adjuvant monotherapy (aMT), defined as either chemotherapy or radiation alone, utilizing a large national registry of patients. PATIENTS AND METHODS: Adult patients with stage III endometrial adenocarcinoma diagnosed from 2004 to 2013 were included. Logistic and Cox regression modeling was used to identify factors predictive of receipt of aCRT and OS, respectively. Survival analysis was performed with Kaplan Meier and log-rank analysis. Propensity score matching and sensitivity analysis was performed to address selection bias and presence of potential confounding variables. RESULTS: A total of 21,027 patients were identified: 11,435 (54.4%) patients received aMT, while 9592 (45.6%) received aCRT. Utilization of aCRT increased over the study period (p<0.01). Factors predictive of receiving aCRT include private insurance (OR: 1.67, 95% CI: 1.30-2.14), Medicare (OR: 1.33, 95% CI: 1.01-1.75), FIGO stage IIIC disease (OR: 1.36, 95% CI: 1.19-1.54), lymphovascular space invasion (OR: 1.14, 95% CI: 1.03-1.27), and lymph node surgery performed (OR: 1.42, 95% CI: 1.15-1.74). Median survival in years for aCRT, RT, and CT was 10.3, 7.1, and 5.6, respectively (p<0.001). Compared to aMT, aCRT was associated with a decrease risk of death on multivariate analysis (HR: 0.62, 95% CI: 0.56-0.70). The benefit of aCRT over aMT persisted after propensity score matching. CONCLUSION: The use of aCRT for stage III endometrial cancer is increasing. Multiple clinical and demographic factors were predictive of aCRT use. When compared to chemotherapy or radiation alone, aCRT is associated with an OS benefit.

Brachytherapy improves survival in primary vaginal cancer.

PURPOSE: Prospective, randomized data does not exist to guide treatment in primary vaginal cancer (PVC). We evaluated the impact of brachytherapy on survival in women with PVC. METHODS AND MATERIALS: Women who received radiotherapy for PVC were identified using the Surveillance, Epidemiology, and End Result database. Two retrospective cohorts were created; women who received external beam radiotherapy (EBRT) alone and those who received brachytherapy (alone or in combination of EBRT). Nearest-neighbor propensity score matching was used to balance the groups according to measured covariates. Cox proportional hazard regression modeling was used to estimate the effect of receipt of brachytherapy on survival. RESULTS: Two thousand five hundred seventeen vaginal cancer patients were identified. Squamous cell carcinoma made up 75% of tumors. Median overall survival (OS) for patients receiving EBRT alone was 3.6years (95% CI, 3.0-4.2years) versus 6.1years (95% CI 5.2-7.2years) for patients receiving brachytherapy (p=<0.001). Cox proportional hazard model revealed decrease risk of death among patients that received brachytherapy in the matched cohort (HR 0.77; 95% CI 0.68-0.86). Brachytherapy reduced risk of death among patients in all stage groups. No patient demographic or tumor variables favored the use of EBRT alone. Brachytherapy was associated with a decreased risk of death for all FIGO stages. Brachytherapy benefited patients with squamous cell carcinoma (HR 0.80; 95% CI 0.70-0.92) and adenocarcinoma (HR 0.69; 95% CI 0.49-0.95). Tumors larger than 5cm had the greatest benefit from brachytherapy (HR 0.68; 95% CI 0.50-0.91). CONCLUSIONS: Brachytherapy should be encouraged for all suitable patients with PVC.

Hodgkin lymphoma, version 2.2015.

Hodgkin lymphoma (HL) is an uncommon malignancy involving lymph nodes and the lymphatic system. Classical Hodgkin lymphoma (CHL) and nodular lymphocyte-predominant Hodgkin lymphoma are the 2 main types of HL. CHL accounts for most HL diagnosed in the Western countries. Chemotherapy or combined modality therapy, followed by restaging with PET/CT to assess treatment response using the Deauville criteria (5-point scale), is the standard initial treatment for patients with newly diagnosed CHL. Brentuximab vedotin, a CD30-directed antibody-drug conjugate, has produced encouraging results in the treatment of relapsed or refractory disease. The potential long-term effects of treatment remain an important consideration, and long-term follow-up is essential after completion of treatment.

Long-term life expectancy for children with ependymoma and medulloblastoma.

OBJECTIVES: There is a paucity of long-term follow-up data for children with intracranial ependymoma (IE) and medulloblastoma (MB). What happens to these children 20, 30, or 40 years after diagnosis? Do they have potential for a normal lifespan? The purpose of this study was to ascertain the long-term survival potential in children with MB or IE who have survived 5 years from diagnosis. METHODS: A retrospective analysis was conducted using the SEER Program. Children (ages 0-19 years) from 1973 to 2011 with a diagnosis of MB or IE were identified. A cohort was created of potentially cured patients who survived 5 years from diagnosis. Cox proportional hazards models and Kaplan-Meier estimates were utilized to analyze long-term survival. RESULTS: We identified 876 patients with MB and 474 patients with IE who were alive 5 years from diagnosis. Patients with MB had a 30-year overall survival (OS) and cancer-specific survival (CSS) of 70.2% and 80.1%, respectively. Patients with IE had a 30-year OS and CSS of 57.3% and 68.8%, respectively. When comparing MB with IE, MB had improved CSS (P = 0.04) and trended toward increased OS (P = 0.10). CONCLUSIONS: A significant number of deaths due to disease occur for several decades after treatment for both IE and MB. Despite this, the potential for long-term survival exists in 5-year survivors of both histologies. If alive at 5 years from diagnosis, patients with MB tend to have a lower risk of death from disease compared to those with IE.