DESCRIPTION
Hematopoietic stem cell transplantation (HSCT) refers to a procedure in which hematopoietic stem cells are infused to restore bone marrow function in individuals with cancer who receive bone-marrow-toxic doses of cytotoxic drugs. Hematopoietic stem cells may be obtained from the transplant recipient (i.e., autologous stem cell transplantation) or from a donor (i.e., allogeneic stem cell transplantation). They may be harvested from bone marrow, peripheral blood, or umbilical cord blood shortly after delivery of neonates. High-dose chemotherapy with hematopoietic stem cell transplantation has been investigated as a possible therapy in pediatric brain tumors, particularly those with disease considered high risk.
CNS Embryonal Tumors
Classification of brain tumors is based on both histopathologic characteristics of the tumor and location in the brain. CNS embryonal tumors are more common in children and are the most common brain tumor in childhood. Embryonal tumors of the central nervous system (CNS) include medulloblastoma, medulloepithelioma, supratentorial PNETs (pineoblastoma, cerebral neuroblastoma, ganglioneuroblastoma), ependymoblastoma, and atypical teratoid/rhabdoid tumor). Medulloblastomas account for 20% of all childhood CNS tumors. The other types of embryonal tumors are rare by comparison. Surgical resection is the mainstay of therapy with the goal being gross total resection with adjuvant radiation therapy, as medulloblastomas are very radiosensitive. In general, use of autologous hematopoietic cell transplantation in individuals with medulloblastoma who have not received radiation treatment has shown no survival benefit for those individuals considered to be at average risk (i.e., individual age greater than 3 years, without metastatic disease, and with total or near total surgical resection [less than 1.5cm2 residual tumor]).
Ependymoma Tumor
An ependymoma is a neuroepithelial tumor that arises from the ependymal lining cell of the ventricles and is, therefore, usually contiguous with the ventricular system. In children, the tumor typically arises intracranially, while in adults, a spinal cord location is more common. Ependymomas have access to the cerebrospinal fluid and may spread throughout the entire neuroaxis and are distinct from ependymoblastomas due to their more mature histologic differentiation. Initial treatment of ependymoma consists of maximal surgical resection followed by radiotherapy. Disease relapse is common, typically occurring at the site of origin. Treatment of recurrence is problematic as further surgical resection or radiation therapy is usually not possible. Given the poor response to conventional-dose chemotherapy, high-dose chemotherapy with autologous hematopoietic stem cell transplantation has been investigated as a possible salvage therapy.
POLICY
Autologous hematopoietic stem cell transplantation for the treatment of embryonal tumors of the central nervous system is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Allogenic stem cell transplant and tandem autologous for the treatment of embryonal tumors of the central nervous system is considered investigational.
Hematopoietic stem cell transplantation (i.e., autologous, tandem autologous and allogeneic) for the treatment of an ependymoma tumor is considered investigational.
MEDICAL APPROPRIATENESS
Autologous hematopoietic stem cell transplantation for the treatment of embryonal tumors of the central nervous system is considered medically appropriate if ANY ONE of the following criteria are met:
New diagnosis when ALL of the following are met:
Treatment is indicated for ANY ONE of the following:
Partial or complete response to induction chemotherapy
Stable disease after induction therapy
ABSENCE of average risk of recurrence in medulloblastoma as indicated by ALL of the following:
Age older than 3 years
No metastatic disease
Total or near total resection [less than 1.5 cm2 residual tumor]
Recurrent disease
IMPORTANT REMINDERS
Any specific products referenced in this policy are just examples and are intended for illustrative purposes only. It is not intended to be a recommendation of one product over another, and is not intended to represent a complete listing of all products available. These examples are contained in the parenthetical e.g. statement.
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the medical policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.
ADDITIONAL INFORMATION
The evidence to support hematopoietic stem cell transplantation for the treatment of ependymoma includes relatively small case series. The results of these studies do not report higher survival rates for individuals treated with HSCT compared with standard therapies. The evidence is insufficient to determine the effects of the technology on health outcomes.
SOURCES
American Society for Transplantation and Cellular Therapy (ASTCT). (2020, March). Indications for hematopoietic cell transplantation and immune effector cell therapy: Guidelines from the American Society for Transplantation and Cellular Therapy. Retrieved April 14, 2023 from http://www.astctjournal.org/.
BlueCross BlueShield Association. Evidence Positioning System. (2:2023). Hematopoietic cell transplantation for central nervous system embryonal tumors and ependymoma (8.01.28). April 13, 2023 https://www.evidencepositioningsystem.com/. (42 articles and/or guidelines reviewed)
Centers for Medicare & Medicaid Services. CMS.gov. NCD for stem cell transplantation (110.23). Retrieved October 24, 2016 from http://www.cms.gov.
National Cancer Institute. (2022, August). Childhood ependymoma treatment (PDQ®). Retrieved April 13, 2023 from http://www.cancer.gov.
National Cancer Institute. (2022, August).Childhood medulloblastoma and other central nervous system embryonal tumors treatment (PDQ®. Retrieved April 13, 2023 from http://www.cancer.gov.
National Comprehensive Cancer Network. (2023, March). NCCN Guidelines for the Treatment of Cancer (NCCN Guidelines®).Central nervous system cancers (V.1.2023). Retrieved April 13, 2023 from the National Comprehensive Cancer Network.
Raleigh, D., Tomlin, B., Buono, B., Roddy, E., Sear, K., Byer, L., et al. (2017). Survival after chemotherapy and stem cell transplant followed by delayed craniospinal irradiation is comparable to upfront craniospinal irradiation in pediatric embryonal brain tumor patients. Journal of Neuro-Oncology, 131 (2), 359-368. Abstract retrieved October 11, 2017 from PubMed database.
Sung, K., Lim, D., Lee, S., Yoo, K., Koo, H., Kim, J., et al. (2012). Tandem high-dose chemotherapy and autologous stem cell transplantation for anaplastic ependymoma in children younger than 3 years of age. Journal of Neurooncology 107 (2), 335-342. Abstract retrieved October 24, 2016 from PubMed database.
Zhang, M., Liu, C., Zhou, H., Wang, W., Wang, L., Shi, B., & Xue, X. (2022). Meta of classical chemotherapy compared with high-dose chemotherapy and autologous stem cell rescue in newly diagnosed medulloblastoma after radiotherapy. Medicine, 101 (30), e29372. (Level 2 evidence)
ORIGINAL EFFECTIVE DATE: 7/14/2012
MOST RECENT REVIEW DATE: 6/8/2023
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Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
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