Chordoma is a very rare tumor of bone that arises from remnants of the notochord within the clivus, spinal vertebrae, or sacrum; the most common site in children is the cranium.  The incidence in the United States is approximately one case per 1 million people per year, and only 5% of all chordomas occur in patients younger than 20 years.   Most pediatric patients have the classical or chondroid variant of chordoma, while the dedifferentiated variant is rare in children.  
Younger children appear to have a worse outlook than do older patients.       The survival rate in children and adolescents ranges from about 50% to 80% for cranial chordomas.    A retrospective literature review and review of institutional patients identified 682 patients with chordomas of the spine, with a median age of 57 years.  [Level of evidence: 3iiiA] Age younger than 18 years, location in sacral spine, dedifferentiated pathology, and chemotherapy were associated with a lower probability for progression-free survival (PFS). Young age (<18 years), old age (>65 years), bladder or bowel dysfunction at presentation, dedifferentiated pathology, recurrence or progression, and metastases were associated with a worse overall survival. Histopathology is also an important prognostic factor, with atypical or chondroid pathology having worse outcomes than classical pathology.  [Level of evidence: 3iiiA]
A multicenter, retrospective study identified 40 children with chordomas (median age, 12 years).  [Level of evidence: 3iiiA] Most of the patients had the histologically classical form of chordoma (45.5%), and the chordomas were mostly located at the skull base (72.5%). The overall survival (OS) rates were 66.6% at 5 years and 58.6% at 10 years. The PFS rates were 55.7% at 5 years and 52% at 10 years. Total resection correlated with a better outcome (P = .04 for OS and PFS, log-rank). Loss of BAF47 immunoexpression appeared to be a significant independent adverse prognostic factor (PFS, P = .033).
A retrospective analysis identified seven children with poorly differentiated chordomas.  [Level of evidence: 3iiA] The median survival of these patients was 9 months. All poorly differentiated chordomas showed loss of SMARCB1 expression by immunohistochemistry. Copy number profiles were derived from intensity measures of the methylation probes and indicated 22q losses affecting the SMARCB1 region in all poorly differentiated chordomas.
Patients usually present with pain, with or without neurologic deficits such as cranial or other nerve impairment. Diagnosis is straightforward when the typical physaliferous (soap bubble–bearing) cells are present. Differential diagnosis is sometimes difficult and includes dedifferentiated chordoma and chondrosarcoma. Childhood chordoma has been associated with tuberous sclerosis complex. 
Treatment options for childhood chordoma include the following:
Standard treatment includes surgery and external radiation therapy, often proton-beam radiation.   Surgery is not commonly curative in children and adolescents because of difficulty obtaining clear margins and the likelihood of the chordoma arising in the skull base, rather than in the sacrum, making them relatively inaccessible to complete surgical excision. However, if gross-total resection can be achieved, outcome is improved.  [Level of evidence: 3iiA]
The best results have been obtained using proton-beam therapy (charged-particle radiation therapy) because these tumors are relatively radiation resistant, and radiation-dose conformality with protons allows for higher tumor doses while sparing adjacent critical normal tissues.   ;   [Level of evidence: 3iiA];  [Level of evidence: 3iiiDiii]
There are only a few anecdotal reports of the use of cytotoxic chemotherapy after surgery alone or surgery plus radiation therapy. Treatment with ifosfamide/etoposide and vincristine/doxorubicin/cyclophosphamide has been reported with some success.   The role for chemotherapy in the treatment of this disease is uncertain.
Imatinib mesylate has been studied in adults with chordoma on the basis of the overexpression of PDGFR alpha, beta, and KIT in this disease.   Among 50 adults with chordoma treated with imatinib and evaluable by Response Evaluation Criteria In Solid Tumors (RECIST) guidelines, there was one partial response and 28 additional patients had stable disease at 6 months.  The low rate of RECIST responses and the potentially slow natural course of the disease complicate the assessment of the efficacy of imatinib for chordoma.  Other tyrosine kinase inhibitors and combinations involving kinase inhibitors have been studied in adults.    One multicenter French retrospective study reported five patients who had partial responses to treatment with either imatinib, sorafenib, or erlotinib, with a median progression-free survival of 36 months. 
Recurrences are usually local but can include distant metastases to lungs or bone.
Information about National Cancer Institute (NCI)–supported clinical trials can be found on the NCI website. For information about clinical trials sponsored by other organizations, refer to the ClinicalTrials.gov website.
The following is an example of a national and/or institutional clinical trial that is currently being conducted:
Patients with chordomas and SMARCB1 mutations may be offered treatment with tazemetostat on the APEC1621C (NCT03213665) treatment arm of this trial.
Tumor tissue from progressive or recurrent disease must be available for molecular characterization. Patients with tumors that have molecular variants addressed by treatment arms included in the trial will be offered treatment on Pediatric MATCH. Additional information can be obtained on the NCI website and ClinicalTrials.gov website.
Cancer in children and adolescents is rare, although the overall incidence of childhood cancer has been slowly increasing since 1975.  Referral to medical centers with multidisciplinary teams of cancer specialists experienced in treating cancers that occur in childhood and adolescence should be considered for children and adolescents with cancer. This multidisciplinary team approach incorporates the skills of the following health care professionals and others to ensure that children receive treatment, supportive care, and rehabilitation that will achieve optimal survival and quality of life:
(Refer to the PDQ Supportive and Palliative Care summaries for specific information about supportive care for children and adolescents with cancer.)
Guidelines for pediatric cancer centers and their role in the treatment of pediatric patients with cancer have been outlined by the American Academy of Pediatrics.  At these pediatric cancer centers, clinical trials are available for most types of cancer that occur in children and adolescents, and the opportunity to participate in these trials is offered to most patients and their families. Clinical trials for children and adolescents diagnosed with cancer are generally designed to compare potentially better therapy with therapy that is currently accepted as standard. Most of the progress made in identifying curative therapy for childhood cancers has been achieved through clinical trials. Information about ongoing clinical trials is available from the NCI website.
Dramatic improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2010, childhood cancer mortality decreased by more than 50%.  Childhood and adolescent cancer survivors require close monitoring because cancer therapy side effects may persist or develop months or years after treatment. (Refer to the PDQ summary on Late Effects of Treatment for Childhood Cancer for specific information about the incidence, type, and monitoring of late effects in childhood and adolescent cancer survivors.)
Childhood cancer is a rare disease, with about 15,000 cases diagnosed annually in the United States in individuals younger than 20 years.  The U.S. Rare Diseases Act of 2002 defines a rare disease as one that affects populations smaller than 200,000 persons. Therefore, all pediatric cancers are considered rare.
The designation of a rare tumor is not uniform among pediatric and adult groups. Adult rare cancers are defined as those with an annual incidence of fewer than six cases per 100,000 people, and they are estimated to account for up to 24% of all cancers diagnosed in the European Union and about 20% of all cancers diagnosed in the United States.   Also, the designation of a pediatric rare tumor is not uniform among international groups, as follows:
Most cancers within subgroup XI are either melanomas or thyroid cancer, with the remaining subgroup XI cancer types accounting for only 1.3% of cancers in children aged 0 to 14 years and 5.3% of cancers in adolescents aged 15 to 19 years.
These rare cancers are extremely challenging to study because of the low incidence of patients with any individual diagnosis, the predominance of rare cancers in the adolescent population, and the lack of clinical trials for adolescents with rare cancers.
The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.
Added text about the results of a multicenter, retrospective study that identified 40 children with chordomas (cited Beccaria et al. as reference 9 and level of evidence 3iiiA).
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This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of pediatric chordoma. It is intended as a resource to inform and assist clinicians who care for cancer patients. It does not provide formal guidelines or recommendations for making health care decisions.
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PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Chordoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/bone/hp/child-chordoma-treatment-pdq. Accessed <MM/DD/YYYY>.
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