Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors

Ann & Robert H Lurie Children's Hospital of Chicago

Status and phase

Unknown

Phase 2

Phase 1

Conditions

Germ Cell Tumor

Soft Tissue Sarcoma

Hodgkin's Disease

Ewing's Sarcoma

Hepatoblastoma

Treatments

Drug: High-Dose Chemotherapy with Tandem PBSC Rescue.

Study type

Interventional

Funder types

Other

Identifiers

NCT00179816

BMT 0499 Solid

Details and patient eligibility

About

This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors.

Full description

Significant advances have been made in recent years in the treatment of solid tumors of childhood. However, much of the improvement in survival has been made in low stage and localized disease. Of significance is the fact that the improvements have come in up-front remission rates without translation into significantly high event-free survival(EFS) or overall survival (OS). This is despite the fact that these tumors as a whole are largely chemotherapy responsive.

Recent advances in the understanding of the biology of hematopoeitic stem cells have driven the design of treatment regimens that allow for dose intensification without unacceptable hematologic toxicity. Protocol development has focused on active agents that have a broad range between hematologic and non-hematologic toxicities. This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors. This study utilizes PBSC to limit the risk of tumor cell contamination while retaining prompt hematologic recovery from these highly intensified treatments.

Enrollment

12 estimated patients

Sex

All

Ages

Under 21 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

Malignant Diseases:
- Ewing's sarcoma/PNET:
  - CR1 - Metastatic disease at diagnosis, tumor volume > 100 ml, pelvic bone primary
  - CR2 - Locally recurrent disease
- Soft tissue sarcoma
  - CR1 - Metastatic disease at diagnosis or locally advanced disease where local control is suboptimal (i.e., inability to provide radiation therapy due to extent of disease).
  - CR2 - Locally recurrent disease (VGPR2 acceptable)
- Hepatoblastoma:
  - VGPR1 - Patients with metastatic disease at diagnosis who have a persistently elevated alpha FP, or unresectable primary as a way of converting to resectable.
  - CR2/VGPR2
- Hodgkin's Disease:
  - VGPR1 - Progression on primary therapy/Refractory disease
  - CR2/VGPR2
- Germ Cell Tumor:
  - CR2/VGPR2 - recurrent disease
- Wilms Tumor:
  - CR2/VGPR2 - recurrent disease
IRB approved signed written informed consent by patient and/or their legally authorized guardian.
Patients 21 years of age or younger at initial diagnosis, with older patients considered individually for primary pediatric disease diagnosis.
Adequate central venous access (double lumen CVL or 2 single lumen PCVC).
Adequate PBSC harvests with a minimum of 2.0 x 108 MNC/kg available for each PBSC rescue.
Organ Function:
- Platelets > 50,000/ml
- SGOT < 10 x upper limits of normal
- Creatinine < 1.5 x normal baseline
- Normal cardiac function in accordance with institutional policies
- Normal pulmonary function in accordance with institutional policies.
Physiologic status:
- No active infections
- Adequate performance status as measured by Karnofsky (> 70%) or Lansky scale (> 60%) as appropriate for age.
Bone Marrow Status
- No evidence of morphologic involvement with tumor at the time of transplant

Off Study Criteria:

Severe toxicity. Contact the Study Coordinator immediately and complete Adverse Reaction Form.
Disease progression or relapse prior to PBSC #1 or between PBSC rescue # 1 and #2.
Inability to collect adequate numbers of PBSC for successful transplantation.
Patient or parent/guardian refusal to remain on study.