Although great strides have been made in the cure of many pediatric tumors, a subset of solid tumors remains for which the chance of survival is uniformly poor. The probability of achieving disease-free survival for children with newly diagnosed metastatic rhabdomyosarcoma, Ewing's / primitive neuroectodermal tumors (PNET) or high-grade glioma remains less than 25 percent, despite multi-modal conventional therapy.

Likewise, the survival from a variety of pediatric solid tumors and brain tumors such as meduloblastoma, which have proven resistant to or have recurred following conventional multi-modal therapy remains poor with less than 10 percent of patients achieving long-term survival.

Autologous bone marrow stem cell transplants — where the child's own bone marrow cells are used — have been utilized as the final stage of therapy for these pediatric patients with some encouraging improvement in survival. Nonetheless, at least half of the patients develop recurrent disease. A principal barrier to survival is the development of tumor resistance to standard chemotherapy drugs. Patients with recurrent lymphoma also benefit from autologous transplant.

Solid Tumor Protocol

The UCSF Children's Hospital Bone Marrow Transplant Program uses several different conditioning regimens for patients with solid tumors depending upon the type of tumor and clinical circumstances. They include using the following drugs:

• Topotecan, thiotepa and carboplantinum
• VP-16 (etoposide), melphalan and carboplantinum
• Cyclophosphamide (cytoxan), BCNU and VP-16

Eligibility

The following patients are eligible for this protocol:

• Patients with metastatic rhabdomyosarcoma or metastatic peripheral PNET in at least a partial remission following therapy
• Patients with refractory or recurrent brain tumors, soft tissue sarcomas, Wilms' tumor or germ cell tumors who have achieved a second or greater complete remission
• Patients with progressive or recurrent Hodgkin's or non-Hodgkin's lymphoma

Brain Tumor Protocol

Patients with high grade brain tumors, in particular those less than 3 years of age and those who progress after standard chemotherapy and radiation have a very poor survival rate, may be candidates for intensive chemotherapy and autologous stem cell transplant. The decision about using high dose chemotherapy and transplant is made on the individual patient's circumstances in consultation with the pediatric neuro-oncology team.

Reviewed by health care specialists at UCSF Children's Hospital.
Last updated October 15, 2009