Intro: 131I-MIBG is a highly active form of tumor-targeted radiotherapy in patients with relapsed neuroblastoma, with response rates of 30% to 40%. Several reports of second malignant neoplasm (SMN) in patients after treatment with 131I-MIBG suggest the possibility of increased risk of secondary malignancy, particularly myelodysplasia and leukemia. Incidence of and risk factors for SMN after 131I-MIBG have not been defined.
Methods: This is a multi-institutional retrospective review of patients with neuroblastoma treated with 131I-MIBG therapy at four institutions between March 1, 1984 and March 1, 2014. A competing risks approach was used to calculate the cumulative incidence of SMN from time of first exposure to 131I-MIBG. Competing risks regression was used to identify potential risk factors for secondary malignancy.
Results: The analytical cohort included 644 patients treated with 131I-MIBG. The cumulative incidence of SMN was 7.6% (90% CI 4.8-11.9%) and 14.3% (90% CI 9.1-22.1%) at five and ten years from first 131I-MIBG, respectively. No increase in SMN risk was found with increased number of 131I-MIBG treatments or higher cumulative activity per kilogram of 131I-MIBG received. An increased risk of SMN was found in patients who had bone disease at the time of first 131I-MIBG therapy. In a multivariate analysis, patients with relapsed/progressive disease had significantly lower risk of SMN (Subdistribution Hazard Ratio 0.3, 95% CI, 0.1-0.8, p=0.023) compared to patients with persistent/refractory.
Conclusion: The cumulative risk of SMN after 131I-MIBG therapy for patients with relapsed or refractory neuroblastoma is similar to the published incidence for high-risk neuroblastoma overall. We found no dose-dependent increase in SMN risk. As the number of patients treated with 131I-MIBG earlier after diagnosis, and length of follow up time from 131I-MIBG therapy increase, it will be important to reassess this risk.