Unilateral Retinoblastoma
Conditions
Brief summary
This protocol provides guidelines for the management of non-metastatic unilateral retinoblastoma and introduces an innovative adjuvant therapy for higher risk patients based upon the results of the Grupo de America Latina de Oncologia Pediatrica (GALOP) I study. Conservative therapy will be not protocolized.
Detailed description
Patients with non-metastatic retinoblastoma undergoing enucleation will be staged using the American Joint Committee on Cancer (AJCC), version 8, Tumor, Node, Metastasis (TNM)- H system and the International Retinoblastoma Staging System (IRSS). IRSS stage I patients recognized as higher risk will be assigned for adjuvant therapy. Those with standard risk will not receive adjuvant therapy after enucleation. Higher risk patients are defined as those with pathological retrolaminar optic nerve invasion and-or any degree of scleral invasion (pT3b, pT3c, pT3d). Based on the results of the GALOP I protocol, they will receive a reduced dose adjuvant chemotherapy regimen with 3 cycles of alternating cyclophosphamide, vincristine and idarubicin alternating with another 3 cycles of carboplatin and etoposide. Six doses of intrathecal topotecan will be given. Patients presenting with severe buphthalmia (cT3c-cT3e) will receive neo-adjuvant therapy with the same intensive regimen but including a higher dose of carboplatin as per GALOP I protocol plus intrathecal topotecan and secondary enucleation followed by adjuvant chemotherapy for a total of 8 cycles. Stage II patients (pT4) will receive the same adjuvant regimen plus orbital radiotherapy (45 cGy).
Interventions
COMBINATION_PRODUCTCombination Chemotherapy plus Intrathecal Topotecan
Adjuvant chemotherapy with a reduced dose (compared to GALOP I protocol) of carboplatin
OTHERNo Adjuvant Therapy
Patients will not receive any adjuvant therapy after enucleation of the affected eye.
COMBINATION_PRODUCTHigher Dose Combination Chemotherapy plus Intrathecal Topotecan
GALOP I-based systemic adjuvant therapy plus intrathecal topotecan
COMBINATION_PRODUCTHigher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy
GALOP I-based systemic adjuvant therapy plus intrathecal topotecan and orbital radiotherapy (45 Gy) up to chiasm
Sponsors
Hospital JP Garrahan
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
0 Years to 6 Years
Healthy volunteers
No
Inclusion criteria
* Histological diagnosis of retinoblastoma confirmed at participating institutions * Ophthalmological diagnosis of unilateral retinoblastoma in patients undergoing conservative therapy. * No prior therapy for retinoblastoma * Lansky Performance Scale greater or equal to 50 * Normal organ function in those patients assigned for chemotherapy * Signed informed consent
Exclusion criteria
* Patients with unilateral retinoblastoma and germline mutations of the Rb1 gene or family history for retinoblastoma
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rate of extraocular relapses | 3 years | Number of participants experiencing extraocular relapse |
| Evaluation of number of patients experiencing acute, chronic and fatal toxicities | 5 years | Number of participants experiencing treatment-related adverse effects and mortality as measured by CTCAE v4.0 |
Countries
Argentina
Outcome results
None listed