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Peripheral Blood Stem Cell Transplantation in Children With High-Risk or Recurrent Solid Tumors

35 Years
Not Enrolling
High Risk Solid Tumors, Recurrent Solid Tumors

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Trial Information

Peripheral Blood Stem Cell Transplantation in Children With High-Risk or Recurrent Solid Tumors

The optimal treatment for refractory (disease won't go away with standard treatment) or
relapsed (disease comes back after going away) solid tumors is unknown. Recent studies have
shown some benefit to tandem peripheral blood stem cell transplantation (PBSCT). In a tandem
transplant, two transplants are done, one after the other. To do a PBSCT, stem cells
("mother cells" that can become any other type of cell) are first collected from the
patient's circulating blood. The patient then undergoes high-dose chemotherapy called the
preparative regimen. The preparative regimen destroys not only the tumor cells, but it also
destroys all of the normal blood making cells. The collected cells are then given back to
the patient to "rescue" the patient from the devastating effects of the preparative regimen.
By using a stem cell rescue will are able to give much higher doses of chemotherapy than we
would be able to give without the stem cell rescue. To make sure that all of the tumor
cells are destroyed, patients in this study will undergo two separate transplants using two
different preparative regimens. The preparative regimens will use the best agents that
have been found to work against recurrent and refractory solid tumors: Busulfan, Thiotepa,
Cyclophosphamide and Melphalan.

Inclusion Criteria:

1. Patients diagnosed with high risk recurrent or refractory solid tumors following
initial chemotherapy

2. Tumor must be sensitive to chemotherapy (maximum 8 cycles) and/or radiation defined
as a greater than 50% reduction in size of the primary and/or metastatic sites.

3. Patients who are under 35 years of age.

4. Patients with a life expectancy of at least 8 weeks and performance status (Karnofsky
or Lansky score) of at least 70%.

5. Patients who are acceptable candidates for peripheral blood stem cell transplantation
based on their pre-transplant evaluation.

Exclusion Criteria:

1. Patients will not be excluded based on sex, race.

2. Patients with central nervous system tumors are not eligible for this protocol.

3. Patients have significant functional deficits in major organs which would interfere
with successful outcome following PBSCT.

4. Patients who have been treated for infections must have appropriate responses as
documented by negative cultures and/or a normal radiographic examination.

5. Patients may not have active CNS disease or marrow involvement with the tumor at the
time of transplant.

6. Patients with disease progression after tandem PBSC #1 will not be eligible for
tandem PBSC #2.

7. Patients will be excluded if they are women of childbearing potential who are
currently pregnant (HCG+) or who are not practicing adequate contraception.

8. Patients who have had a previous stem cell transplant.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To determine the feasibility and toxicity of tandem high dose chemotherapy and peripheral blood stem cells rescue in patients with high-risk or recurrent solid tumors.

Outcome Time Frame:

1 year after last patient is enrolled

Safety Issue:


Principal Investigator

Kuang-Yueh Chiang, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Emory University


United States: Institutional Review Board

Study ID:




Start Date:

May 2003

Completion Date:

July 2011

Related Keywords:

  • High Risk Solid Tumors
  • Recurrent Solid Tumors
  • malignancy
  • solid tumor
  • recurrent
  • high risk
  • Neoplasms



Emory UniversityAtlanta, Georgia  30322
Children's Healthcare of AtlantaAtlanta, Georgia  30342