ICHT followed by AuHCR in young patients with newly diagnosed sPNET appears to not only provide an improved EFS and OS for patients who typically have a poor prognosis, but also it successfully permitted deferral and elimination of radiation therapy in a significant proportion of patients.

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Abstract Background “Head Start” III, was a prospective clinical trial using intensive induction followed by myeloablative chemotherapy and autologous hematopoietic cell rescue (AuHCR) to either avoid or reduce the dose/volume of irradiation in young children with medulloblastoma. Methods Following surgery, patients received 5 cycles of induction followed by myeloablative chemotherapy using carboplatin, thiotepa, and etoposide with AuHCR. Irradiation was reserved for children >6 years old at diagnosis or with residual tumor post-induction. Results Between 2003 and 2009, 92 children <10 years old with medulloblastoma were enrolled. Five-year event-free survival (EFS) and overall survival (OS) rates (±SE) were 46 ± 5% and 62 ± 5% for all patients, 61 ± 8% and 77 ± 7% for localized medulloblastoma, and 35 ± 7% and 52 ± 7% for disseminated patients. Nodular/desmoplastic (ND) medulloblastoma patients had 5-year EFS and OS (±SE) rates of 89 ± 6% and 89 ± 6% compared with 26 ± 6% and 53 ± 7% for classic and 38 ± 13% and 46 ± 14% for large-cell/anaplastic (LCA) medulloblastoma, respectively. In multivariate Cox regression analysis, histology was the only significant independent predictor of EFS after adjusting for stage, extent of resection, regimen, age, and sex (P <0.0001). Five-year irradiation-free EFS was 78 ± 8% for ND and 21 ± 5% for classic/LCA medulloblastoma patients. Myelosuppression was the most common toxicity, with 2 toxic deaths. Twenty-four survivors completed neurocognitive evaluation at a mean of 4.9 years post-diagnosis. IQ and memory scores were within average range overall, whereas processing speed and adaptive functioning were low-average. Conclusion We report excellent survival and preservation of mean IQ and memory for young children with ND medulloblastoma using high-dose chemotherapy, with most patients surviving without irradiation.

Objective Childhood cancer metrics are currently primarily focused on survival rates and late effects of therapy. Our objectives were to design and test a metric that reflected overall quality and safety performance, across all cancer types, of an oncology–bone marrow transplant service line and to use the metric to drive improvement. Method The Cancer Care Index (CCI) aggregates adverse safety events and missed opportunities for best practices into a composite score that reflects overall program performance without regard to cancer type or patient outcome. Fifteen domains were selected in 3 areas as follows: (1) treatment-related quality and safety, (2) provision of a harm-free environment, and (3) psychosocial support. The CCI is the aggregate number of adverse events or missed opportunities to provide quality care in a given time frame. A lower CCI reflects better care and improved overall system performance. Multidisciplinary microsystem-based teams addressed specific aims for each domain. The CCI was widely followed by all team members, particularly frontline providers. Results The CCI was easy to calculate and deploy and well accepted by the staff. The annual CCI progressively decreased from 278 in 2012 to 160 in 2014, a 42% reduction. Improvements in care were realized across most index domains. Multiple new initiatives were successfully implemented. Conclusions The CCI is a useful metric to document performance improvement across a broad range of domains, regardless of cancer type. By the use of quality improvement science, progressive reduction in CCI has occurred over a 3-year period.