Abstract
Purpose :
Outcomes for 8,000 children newly diagnosed with retinoblastoma each year depend where they live (globally 70% die, with resources and knowledge >95% survive). Retinoblastoma complexities (rarity, laterality, multifocality, genetic predisposition, global geographic distribution) confound conventional clinical trials impairing quality evidence for care. We hypothesize that full view by the circle of care, applied to clinical trials of innovative simple therapies (such as sustained-release episcleral topotecan) will improve both conduct of clinical trials and outcomes for children.
Methods :
DEPICT HEALTH, a novel healthcare communication resource that provides visual representation of the child’s complex timeline of care, was evaluated for quality and efficiency of communication across the circle of care, including parents/patients. With parents/patients as partners, we entered clinical details of de-identified patients into a DEMO version of DEPICT HEALTH. Two children with retinoblastoma resistant to conventional therapy in only remaining eyes were treated with sustained-release episcleral topotecan (the first in the world for this therapy). Toxicity and efficacy were evaluated. DEPICT HEALTH supported sharing of retinoblastoma-specific details across the circle of care.
Results :
Feedback from clinicians and families directed technical, usability, and utility improvements in DEPICT HEALTH. After 28/46 days respectively of sustained episcleral topotecan, all multiple small volume tumors disappeared in both patients; no toxicity was observed. At 133 days Patient One showed no activity in the pre-study 30+ small tumors but recurrence in the large calcified tumor, which was treated with a ruthenium plaque. Patient Two had no tumor activity at 68 days. DEPICT HEALTH provided point-of-care treatment data before study treatment and through follow-up. A Phase I-II clinical trial is in development to target small volume retinoblastoma with DEPICT HEALTH providing the pre- and post-intervention data.
Conclusions :
If preliminary results are reproducible, sustained episcleral chemotherapy may be game changing to improve health outcomes of patients with retinoblastoma. Clinical trials with DEPICT HEALTH providing life-long retinoblastoma-specific data for study by artificial intelligence, may achieve quality evidence to support care of children with retinoblastoma.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.