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Activity of topotecan in retinoblastoma
Guillermo L Chantada1, Adriana C Fandiño, Sandra J Casak
1Department of Hematooncology, Hospital de Pediatria JP Garrahan, Buenos Aires, Argentina. gchantada@garrahan.gov.ar
Purpose:
To report our experience with topotecan in children with relapsed/refractory metastatic and intraocular retinoblastoma.
Patients And Methods:
Topotecan was administered intravenously as a 30-min infusion at a dose of 2 mg/m2/d for five consecutive days and repeated after three weeks. If obvious progression was detected by physical examination in patients with overt extraocular disease or if progressive disease was noted after fundoscopic examination in patients with intraocular disease, a second cycle was not administered. Response was evaluated at Week 6.
Results:
Nine patients (6 extraocular, 3 intraocular) were treated from November 1998 to March 2002. A total of 16 cycles were administered. In patients with extraocular disease, there were three partial responses, two cases of stable disease, and one case of progressive disease. Two patients with relapsed/resistant intraocular disease had partial response. allowing local therapy to be performed, and the third patient had progressive disease. The drug was well-tolerated. No patient developed fever or documented infections. No other serious toxicity was found.
Conclusion:
Topotecan is active in extraocular and relapsed/resistant intraocular retinoblastoma. The role of this drug in the treatment of retinoblastoma should be explored in further studies.
Insights
Topotecan shows activity in treating advanced retinoblastoma in children. This chemotherapy agent was well-tolerated in patients with extraocular and intraocular disease, warranting further investigation.
Area of Science:
- Pediatric Oncology
- Ophthalmology
- Medical Research
Background:
- Retinoblastoma is a pediatric eye cancer.
- Relapsed/refractory cases present treatment challenges.
- Intraocular and extraocular disease require distinct management strategies.
Purpose of the Study:
- To evaluate topotecan's efficacy and safety.
- To assess treatment outcomes in pediatric retinoblastoma.
- To explore topotecan for relapsed/refractory retinoblastoma.
Main Methods:
- Intravenous topotecan administered at 2 mg/m2/d for five days, repeated every three weeks.
- Treatment cycles adjusted based on disease progression.
- Response evaluated at six weeks.
Main Results:
- Nine patients (6 extraocular, 3 intraocular) received 16 cycles.
- Extraocular disease: 3 partial responses, 2 stable disease, 1 progressive disease.
- Intraocular disease: 2 partial responses, 1 progressive disease; drug well-tolerated.
Conclusions:
- Topotecan demonstrates activity in retinoblastoma.
- The drug is a potential option for relapsed/refractory cases.
- Further studies are recommended to define topotecan's role.
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