Combination Chemotherapy, Autologous Stem Cell Transplant, and/or Radiation Therapy in Treating Young Patients With Extraocular Retinoblastoma
Condition: Extraocular Retinoblastoma
MeSH terms: Retinoblastoma
Intervention: Autologous Bone Marrow Transplantation; Autologous Hematopoietic Stem Cell Transplantation; Carboplatin; Cisplatin; Cyclophosphamide; Etoposide; Filgrastim; In Vitro-Treated Peripheral Blood Stem Cell Transplantation; Radiation Therapy; Thiotepa; Vincristine Sulfate
Intervention description: Undergo peripheral blood stem cell or bone marrow transplant; Undergo peripheral blood stem cell or bone marrow transplant; Given IV; Given IV; Given IV; Given IV; Given SC; Undergo peripheral blood stem cell or bone marrow transplant; Undergo radiotherapy; Given IV; Given IV
Intervention MeSH terms: Cyclophosphamide; Thiotepa; Cisplatin; Carboplatin; Etoposide; Vincristine; Etoposide phosphate; Podophyllotoxin; Lenograstim
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 60.0
Sex: All
Maximum age: 10 Years
Healthy volunteers: No

Summary:

      This phase III trial is studying the side effects and how well giving combination
      chemotherapy together with autologous stem cell transplant and/or radiation therapy works in
      treating young patients with extraocular retinoblastoma. Giving chemotherapy before an
      autologous stem cell transplant stops the growth of tumor cells by stopping them from
      dividing or killing them. After treatment, stem cells are collected from the patient's blood
      and/or bone marrow and stored. More chemotherapy is given to prepare the bone marrow for the
      stem cell transplant. The stem cells are then returned to the patient to replace the
      blood-forming cells that were destroyed by the chemotherapy. Radiation therapy uses high
      energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy
      and/or autologous stem cell transplant may kill any remaining tumor cells.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients must have histologic or cytologic verification of extra-ocular
             retinoblastoma; extra-ocular disease includes orbital disease, optic nerve involvement
             at the surgical margin, regional nodal disease, and/or overt distant metastatic
             disease (at sites such as bone, bone marrow, liver and/or the central nervous system);
             patients with trilateral retinoblastoma will also be included in this protocol

               -  Patients with a CNS lesion consistent with trilateral or stage 4b disease may be
                  enrolled without tissue confirmation if (1) unequivocal leptomeningeal disease is
                  present on brain or spine magnetic resonance imaging (MRI) scan and/or (2) the
                  primary tumor is at least 2 cm in diameter, predominantly solid, and demonstrates
                  enhancement on the post-gadolinium images; however, even in such cases surgery
                  should be given serious consideration

          -  Patients must have a performance status corresponding to Eastern Cooperative Oncology
             Group (ECOG) scores of 0, 1, or 2; use Karnofsky for patients > 16 years of age and
             Lansky for patients =< 16 years of age

          -  No prior chemotherapy or radiotherapy for the extra-ocular retinoblastoma may have
             been administered prior to entering this study; prior treatment (chemotherapy and/or
             radiation therapy) for intra-ocular retinoblastoma is permissible

          -  Peripheral absolute neutrophil count (ANC) >= 750/uL

               -  If the ANC and/or platelet count are not adequate, but due to bone marrow
                  metastatic disease, these criteria will be waived

          -  Platelet count >= 75,000/uL (transfusion independent)

               -  If the ANC and/or platelet count are not adequate, but due to bone marrow
                  metastatic disease, these criteria will be waived

          -  Creatinine clearance OR radioisotope glomerular filtration rate >= 70 mL/min/1.73 m^2
             or a serum creatinine based on age/gender as follows:

               -  0.4 mg/dL (1 month to < 6 months of age)

               -  0.5 mg/dL (6 months to < 1 year of age)

               -  0.6 mg/dL (1 years to < 2 years of age)

               -  0.8 mg/dL (2 years to < 6 years of age)

               -  1.0 mg/dL (6 years to < 10 years of age)

               -  1.2 mg/dL (10 years to < 13 years of age)

               -  1.5 mg/dL (male) or 1.4 mg/dL (female) (13 years to < 16 years of age)

               -  1.7 mg/dL (male) or 1.4 mg/dL (female) (>= 16 years of age)

          -  Total bilirubin =< 1.5 times upper limit of normal (ULN)

          -  Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or
             serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x
             upper limit of normal (ULN)

          -  All patients and/or their parents or legal guardians must sign a written informed
             consent

          -  All institutional, Food and Drug Administration (FDA), and National Cancer Institute
             (NCI) for human studies must be met