ABSTRACT
Cis-retinoic acid has been used as maintenance therapy for treatment of advanced neuroblastoma in paediatric patients after BMT showing significant advantage in 3-year event-free survival than patients receiving no maintenance therapy. However, there is no data available about using Cis-retinoic acid during induction phase of chemotherapy. The Aim of this study is to evaluate the efficacy of Cis-retinoic acid when used in combination with conventional chemotherapy in the paediatric patient who is newly diagnosed with locally advanced Neuroblastoma. Seventeen newly diagnosed children with locally advanced Neuroblastoma who are candidate to receive chemotherapy also received oral Cis retinoic acid starting at a dose of 160 /m[2] day oral [day 2-15 of chemotherapy]. Patients received at least 6 cycles of OPEC/OJEC regimen. N- Myc was tested in 12 patients. Median follow up was 6 months. Median age 2.4 years. Male: Female ratio was 1.4:1. Most patients were stage III or IV [70%] with only 30% stage II. Abdominal mass was the presenting symptom in 88%. Maximum tolerated dose was 130mg/m[2]. Thirty percent of patients achieved complete remission, 40% had very good response short of complete remission, 12% had partial response, 18% had disease progression. Median disease free survival was 12 months [95% confidence interval: 10.6 - 13.4]. The observed toxicities were hypercalcemia; rash, elevated liver enzymes and hematological toxicities in the form of thrombocytopenia and neutropenia were the main dose limiting effect of Cis retinoic acid. Cis-retinoic acid at dose 130mg/m[2] is a well tolerated drug with chemotherapy. Response to treatment is better than historical control. Randomized phase III trial is warranted