Background: The Florida Association of Pediatric Tumor Programs (FAPTP) has used the Statewide Patient Information Reporting System (SPIRS) since 1980 to track all cases of pediatric cancer. We reviewed the last 40 years of SPIRS data to see how pediatric cancer care has evolved. Methods: We retrospectively analyzed the SPIRS data from 1980 through 2020 in 5-year increments, looking at numbers of new diagnoses, care delivery sites and trial enrollment in Children’s Oncology Group (COG) studies. Results: From 1981-2020 Florida’s population increased almost 88% while the pediatric population only grew 61%. New pediatric cancer diagnoses increased 326% to over 1,000 new cases/year with the median age increasing from 6 to 9. The percentage of patients treated at FAPTP centers grew from 30% to 57% with an annual percentage change (APC) of 10.3% (95% Confidence Interval [CI] of 0.6 to 20.9%) and those with known follow-up rose from 65% to 94%, an APC of 4.5% (95% CI of 3 to 6%). The rate of COG clinical trial enrollment decreased from 32% in 1981-1985 to 20% from 2016-2020, for an APC of 8.91% (95% CI of -13.3 to -4.3%). Conclusions: The striking increase in pediatric cancer cases in Florida over the last 40 years was out of proportion to the population growth. More patients received care at FAPTP centers but a lower percentage were enrolled on COG clinical trials. Improved access to care has not translated into a higher rates of trial enrollment, a deficit which merits further investigation and initiatives.

Acute graft versus host disease (aGvHD) remains one of the most serious complications, occurring in about 30-70%, of allogeneic hematopoietic stem cell transplantation (HSCT) recipients. While about 40-60% of these recipients will respond to steroids as first line, there is no consensus on second line agents. The management of steroid-refractory (SR) and steroid dependent (SD) GVHD after HSCT continues to be challenging. In the absence of clinical trials, treatment in most cases is based on individual physician or center experience. Herein, we present our institutional experience with the use of bortezomib, a first-generation reversible proteasome inhibitor, in SD aGvHD.

Background: Patients with late, occurring ≥18 months post-diagnosis, isolated central nervous relapse (iCNS-R) of B-acute lymphoblastic leukemia (ALL) have excellent outcomes with chemotherapy plus cranial radiotherapy, with 5-yr overall survival (OS) approaching 80% in POG 9412. Subsequent relapse and radiation-related morbidity remain the causes of treatment failure and long-term sequelae. COG AALL02P2 aimed to maintain outcomes in patients with late iCNS-R using intensified chemotherapy and a decrease in cranial irradiation from 1800 to 1200 cGy. Procedures: COG AALL02P2 enrolled 118 eligible patients with B-ALL and early iCNS-R who received intensified systemic therapy, triple intrathecal chemotherapy and 1200 cGy cranial irradiation delivered at 12 months, with maintenance chemotherapy continuing until104 weeks post-diagnosis. Results: The 3-yr event-free and overall survival (EFS) and OS were 64.3±4.5% and 79.6±3.8%, with 46.1% (18/39) of relapses including the CNS. Of the 112 patients who completed therapy, 78 received protocol-specified radiation. Study enrollment was closed after interim monitoring analysis showed inferior EFS compared to POG 9412. Patients with initial NCI standard risk classification fared better than high risk patients. Conclusions: COG AALL02P2 showed inferior EFS but similar OS compared to POG 9412. Limitations included a small sample size, more intensive prior therapies, and a significant number of patients (34/118, 29%) who did not receive protocol-directed radiation due to early relapse prior to 1 year or did not otherwise follow the treatment plan. New approaches are needed to improve outcome for these patients and determine the optimal timing and dose of cranial radiation in the treatment of iCNS-R.