Know Cancer

forgot password

Double Dose Intensive Chemo-Radiotherapy With Peripheral Blood Progenitor Cell Rescue for Children With Advanced Stage Neuroblastoma and Sarcomas

Phase 2
19 Years
Not Enrolling
Neuroblastoma, Ewings Sarcoma, Non-rhabdomyosarcoma Soft Tissue Sarcoma

Thank you

Trial Information

Double Dose Intensive Chemo-Radiotherapy With Peripheral Blood Progenitor Cell Rescue for Children With Advanced Stage Neuroblastoma and Sarcomas

- The treatment is separated into three stages: 1) Initial treatment-chemotherapy and
stem cell collection; 2) Treatment targeted directly to the main tumor-surgery and
radiation therapy; 3) Intensified treatment: two stem cell transplants.

- Patients will receive intensive treatment with a combination of seven drugs which will
be given every 3 weeks, or as soon as the patient's blood counts are within safe
limits. A total of 5 or 6 courses of chemotherapy will be given. The third and fourth
course will be followed by stem cell collections and the last course will be followed
by bone marrow harvest in preparation for transplant.

- The first treatment involves a high-dose of Cisplatin intravenously once a day for 5
days and VP-16 intravenously on days 2,3 and 4 of the treatment.

- The second course of treatment involves cyclophosphamide intravenously on day 1 and day
2, adriamycin intravenously continuously over 48 hours, and vincristine intravenously
on day 1.

- The third course of treatment involves ifosfamide daily on days 1-5 and VP-16 on days
1-3. Mesna will also be given to prevent bladder irritation.

- During the fourth course patients will receive, carboplatin intravenously on days 1 and
2 and VP-16 on days 1,2 and 3 of treatment.

- The fifth course of treatment will be identical to the second course.

- G-CSF (granulocyte colony-stimulating factor) stimulates bone marrow to produce more
white blood cells and will be given subcutaneously until the patients' white blood cell
counts are at an acceptable level. This may require 10-14 days of G-CSF treatment.

- After the first two treatments, and after the last course of treatment, patients will
be evaluated for response. If significant tumor is still present in the bone marrow
before the third treatment, stem cell collection will be delayed. If the bone marrow
still shows disease after the next treatment, this plan of treatment will be stopped.

- Patients may undergo surgery after the last treatment to remove the tumor and to
evaluate the remaining disease. After recovery, radiation therapy may be performed to
the main tumor area.

- Patients will receive transplantations of stem cells twice during this study. After
each preparative treatment, half of the patient's stored stem cells will be given
intravenously. Until the stem cells restore safe levels of blood cells, the patient
will be hospitalized (3-4 weeks or longer).

- The first treatment will begin with high doses of carboplatin and VP-16 daily for 3
consecutive days followed by cyclophosphamide on the fourth and fifth days.

- Between 4-6 weeks after the start of the first course of high dose chemotherapy a
second course consisting of melphalan and total body irradiation (TBI) will be given.
Melphalan will be given daily for 3 consecutive days followed by TBI twice a day for 3

- After each of the two high dose treatments outlined above, the stem cells that were
previously harvested from the patient will be given back to them through a central

- Stem cells will be collected on 3 separate occasions following each of two cycles of
chemotherapy. They will then be processed using a technique that attempts to separate
stem cells from other cells, including any cancer cells that may be present.

- Bone marrow will also be harvested prior to the first transplant and will be used if
blood counts do not recover after the stem cells are given.

Inclusion Criteria:

- Previously untreated neuroblastoma Stage D > 365 days

- Previously untreated neuroblastoma Stage C at age > 365 days with n-myc amplification

- Previously untreated metastatic Ewings sarcoma/PNET

- Previously untreated non-rhabdomyosarcoma soft tissue sarcoma

- Confirmation of neuroblastoma at time of diagnosis by histologic specimen or with
bone marrow specimen showing solid tumor. Confirmation of sarcoma by histologic
specimen with evidence of metastatic disease be imaging study

- Patient 19 years of age or younger

- ANC > 1000

- Platelet count > 75,000

- SGOT < 2.5 x ULN

- Normal serum creatinine levels for age

Exclusion Criteria:

- Previous malignancy requiring nonsurgical treatment or bone marrow transplant.

- Relapsed disease

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 toxicity and feasibility of double dose chemo-radiotherapy with blood progenitor cell rescue in this patient population.

Outcome Time Frame:

5 years

Safety Issue:


Principal Investigator

Lisa Diller, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute


United States: Institutional Review Board

Study ID:




Start Date:

January 1996

Completion Date:

February 2009

Related Keywords:

  • Neuroblastoma
  • Ewings Sarcoma
  • Non-rhabdomyosarcoma Soft Tissue Sarcoma
  • Stem cell transplant
  • High-risk pediatric tumors
  • Neuroblastoma
  • Sarcoma, Ewing's
  • Neuroectodermal Tumors, Primitive, Peripheral
  • Sarcoma



Dana-Farber Cancer Institute Boston, Massachusetts  02115