Anthem Blue Cross Connecticut TRANS.00027 Hematopoietic Stem Cell Transplantation for Pediatric Solid Tumors Form

This document addresses hematopoietic stem cell transplantation for pediatric solid tumors including neuroblastoma, primitive neuroectodermal tumors (PNETs) of the central nervous system, ependymoma, pineoblastoma, Ewing sarcoma, Wilms’ tumor, osteosarcoma, retinoblastoma, and rhabdomyosarcoma. These types of solid tumors generally develop in children; however, some may also present in adulthood.

Note: For additional information and criteria for umbilical cord transplantation, see:

• TRANS.00016 Umbilical Cord Blood Progenitor Cell Collection, Storage and Transplantation

Position Statement

Neuroblastoma

Medically Necessary:

An autologous hematopoietic stem cell transplantation is considered medically necessary as the initial treatment for high-risk neuroblastoma.

A planned autologous tandem* hematopoietic stem cell transplantation is considered medically necessary as the initial treatment for high-risk neuroblastoma.

An autologous hematopoietic stem cell transplantation is considered medically necessary as a treatment for primary refractory or recurrent neuroblastoma in individuals who have not previously undergone treatment with hematopoietic stem cell transplantation.

A repeat autologous hematopoietic stem cell transplantation due to primary graft failure or failure to engraft is considered medically necessary.

Hematopoietic stem cell harvesting** for an anticipated but unscheduled transplant is considered medically necessary in individuals with neuroblastoma who meet the criteria above when the treating physician documents that a future transplant is likely.

*Tandem transplantation refers to a planned infusion (transplant) of previously harvested hematopoietic stem cells with a repeat hematopoietic stem cell infusion (transplant) that is performed within 6 months of the initial transplant. This is distinguished from a repeat transplantation requested or performed more than 6 months after the first transplant, and is used as salvage therapy after failure of initial transplantation or relapsed disease.

**Hematopoietic stem cell harvesting does not include the transplant procedure.

Investigational and Not Medically Necessary:

An autologous hematopoietic stem cell transplantation is considered investigational and not medically necessary for individuals who do not meet the above criteria.

An allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplantation is considered investigational and not medically necessary as a treatment of neuroblastoma.

A planned tandem allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplantation as a treatment of neuroblastoma is considered investigational and not medically necessary.

A second or repeat autologous hematopoietic stem cell transplantation due to persistent, progressive or relapsed disease is considered investigational and not medically necessary.

Hematopoietic stem cell harvesting for a future but unscheduled transplant is considered investigational and not medically necessary when the criteria above are not met.

Primitive Neuroectodermal Tumors (PNETs) of the Central Nervous System, Ependymoma and Pineoblastoma

Medically Necessary:

An autologous hematopoietic stem cell transplantation with or without associated radiotherapy, is considered medically necessary for the treatment of PNETs (such as medulloblastoma), arising in the central nervous system, ependymoma or pineoblastoma.

A repeat autologous hematopoietic stem cell transplantation due to primary graft failure or failure to engraft is considered medically necessary.

Hematopoietic stem cell harvesting for an anticipated but unscheduled transplant is considered medically necessary in individuals with PNET, ependymoma or pineoblastoma who meet the criteria above when the treating physician documents that a future transplant is likely.

Investigational and Not Medically Necessary:

An allogeneic (ablative or non-myeloablative [mini transplant]) hematopoietic stem cell transplantation is considered investigational and not medically necessary for the treatment of PNETs (such as medulloblastoma), arising in the central nervous system, ependymoma or pineoblastoma.

A planned tandem allogeneic or autologous hematopoietic stem cell transplantation is considered investigational and not medically necessary for the treatment of PNETs (such as medulloblastoma), arising in the central nervous system, ependymoma or pineoblastoma.

A second or repeat autologous hematopoietic stem cell transplantation due to persistent, progressive or relapsed disease is considered investigational and not medically necessary.

Hematopoietic stem cell harvesting for a future but unscheduled transplant is considered investigational and not medically necessary when the criteria above are not met.

Other High-Risk Solid Tumors of Childhood (Ewing Sarcoma, Wilms’ Tumor, Osteosarcoma, Retinoblastoma, and Rhabdomyosarcoma)

Medically Necessary:

An autologous hematopoietic stem cell transplantation is considered medically necessary as a treatment for Ewing sarcoma (including extraosseous Ewing, peripheral neuroepithelioma and Askin's tumor).

A syngeneic allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplantation is considered medically necessary as a treatment for Ewing sarcoma (including extraosseous Ewing, peripheral neuroepithelioma and Askin's tumor).

A repeat autologous or allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplantation due to primary graft failure or failure to engraft is considered medically necessary.

Hematopoietic stem cell harvesting for an anticipated but unscheduled transplant is considered medically necessary in individuals with Ewing sarcoma who meet the criteria above when the treating physician documents that a future transplant is likely.

An autologous hematopoietic stem cell transplantation is considered medically necessary as a treatment for stage IVa and stage IVb retinoblastoma.

Investigational and Not Medically Necessary:

An allogeneic (ablative or non-myeloablative [mini transplant]) or autologous hematopoietic stem cell transplantation is considered investigational and not medically necessary for all other pediatric solid tumors, including but not limited to: Wilms’ tumor (nephroblastoma), osteosarcoma, and rhabdomyosarcoma.

An autologous hematopoietic stem cell transplantation is considered investigational and not medically necessary for retinoblastoma other than stage IVa or stage IVb.

An allogeneic (ablative or non-myeloablative [mini transplant]) is considered investigational and not medically necessary for retinoblastoma.

An allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplantation is considered investigational and not medically necessary as a treatment of all high risk pediatric solid tumors relapsing after prior therapy with high-dose chemotherapy and autologous hematopoietic stem cell transplantation.

A planned tandem allogeneic or autologous hematopoietic stem cell transplantation is considered investigational and not medically necessary as a treatment of all high risk pediatric solid tumors of childhood.

A second or repeat autologous or allogeneic (ablative or non-myeloablative) hematopoietic stem cell transplantation due to persistent, progressive or relapsed disease is considered investigational and not medically necessary.

Hematopoietic stem cell harvesting for a future but unscheduled transplant is considered investigational and not medically necessary when the criteria above are not met.