Background: Vitamin D deficiency and insufficiency have been associated with poorer health outcomes. Children with cancer are at high risk for Vitamin D deficiency and insufficiency. At our institution, we identified high variability in Vitamin D testing and supplementation in this population. Of those tested, 65% were Vitamin D deficient/insufficient. We conducted a quality improvement (QI) initiative with aim to improve Vitamin D testing and supplementation among children aged 2-18 years old with newly-diagnosed cancer to ≥ 80% over 6 months. Methods: An inter-professional team reviewed baseline data, then developed and implemented interventions using Plan-Do-Study-Act (PDSA) cycles. Barriers were identified using QI tools, including lack of automated triggers for testing and inconsistent supplementation criteria and follow-up testing post-supplementation. Interventions included an institutional Vitamin D guideline, clinical decision-making tree for Vitamin D deficiency, insufficiency and sufficiency, electronic medical record triggers, and automated testing options. Results: Pre-intervention: N=26 patients, four (15%) had baseline Vitamin D testing; two (8%) received appropriate supplementation. Post-intervention: N=33 patients; 32 (97%) had baseline Vitamin D testing; 33 (100%) received appropriate supplementation and completed follow-up testing timely (6-8 weeks post-supplementation). Change was sustained over 24 months. Conclusions: We achieved and sustained our aim for Vitamin D testing and supplementation in children with newly-diagnosed cancer through inter-professional collaboration of hematology/oncology, endocrinology, hospital medicine, pharmacy, nursing, and information technology. Future PDSA cycles will address patient compliance with Vitamin D supplementation and impact on patients’ Vitamin D levels.

Background It is important to ensure access to hydroxyurea (HU) for patients with sickle cell anemia (SCA) living in rural areas without easy access to experts in sickle cell management. The UAB Pediatric Sickle Cell program’s satellite clinics reduce the barrier of transportation to the university-based clinic. However, as compared to the university clinic, these satellite clinics do not offer immediate access to HU dosing laboratory results. Therefore, a nurse clinician calls families with HU dose adjustments after the completion of the clinic visit. This study evaluated the impact of telehealth dosing adjustments on HU laboratory and clinical response as compared to university-based patients. Methods A one year retrospective chart reviewed was performed to evaluate HU laboratory and clinical response based on clinic location and socioeconomic status for patients with SCA. We identified the number of clinic and acute care visits for one year and calculated the mean CBC and HbF values for each patient. Results We identified 107 academic center participants with SCA prescribed HU and 65 satellite clinic participants. We identified no difference in HbF, Hb, MCV, or ANC by clinic location. We also identified no difference in hospital admissions based on clinic location. Finally, mean socioeconomic indicators by zip code were lower in satellite clinic patients but not associated with a difference in HbF response. Conclusions The use of telehealth did not negatively impact laboratory response to HU. Future studies should identify novel approaches to improve access to HU among patients with SCA living in rural areas.

Background: We aimed to retrospectively investigate the role of neoadjuvant chemotherapy in low-risk patients with hepatoblastoma (HB) who underwent curative resection between February 2009 and December 2017. We also verified the feasibility of the risk stratification system to select the optimal patients for upfront resection. Procedure: We compared 5-year overall survival (OS) and event-free survival (EFS) between the upfront surgery (US) (n=26) and neoadjuvant chemotherapy (NC) (n=104) groups at three oncology centers in Beijing, China. To reduce the effect of covariate imbalances, propensity score matching (PSM) was used. We explored whether preoperative chemotherapy affected surgical outcomes and identified the risk factors for events and death, including resection margin status, PRETreatment EXTent of disease stages, age, sex, pathology classification, and α-fetoprotein levels. Results: The median follow-up period was 64 months (interquartile range 60–72). After PSM, 22 pairs of patients were identified and the patient characteristics were similar for all variables included in propensity score matching. In the US group, the 5-year EFS and OS rates were 81.8% and 86.3%, respectively. In the NC group, 5-year EFS and OS rates were 81.8% and 90.9%, respectively. No significant differences in EFS or OS were observed between the groups. Pathological classification was the only risk factor for death and disease progression, tumor recurrence, diagnosis of other malignant neoplasms, and death from any cause ( p=0.007 and p=0.032, respectively). Conclusion: Upfront resection can achieve long-term disease control in low-risk patients with resectable HB, thus reducing the cumulative toxicity of platinum-based chemotherapy drugs.

Background: V enetoclax is frequently used as salvage treatment in pediatric, adolescent, and young adult (AYA) patients with advanced hematologic malignancies. However, more robust data are needed from real-world studies to guide the safe and appropriate use of venetoclax in this population. Procedure: We retrospectively reviewed the medical records of all patients diagnosed with hematologic malignancies less than 30 years of age treated with venetoclax outside of clinical trials at the University of California San Francisco (UCSF) Benioff Children’s Hospitals from 2016 to 2022. Results: We identified 13 patients (AML , n= 8, B-ALL, n= 3, MDS, n= 2) aged 4 months to 27 years. A median of 3 prior lines of therapy were given (range 0 to 5). All patients received venetoclax in combination with either a hypomethylating agent or conventional chemotherapy. Three (23%) patients achieved a complete remission (CR); 2 (15%) achieved a partial remission (PR); 3 (23%) had stable disease (SD), and 5 (42%) had progressive disease. Median survival and time to progression from venetoclax initiation was 9 months (range 2.5 to 52 months), and 3 months (range 2 weeks to 7.5 months), respectively. Five patients (38%) developed life-threatening infections while receiving venetoclax, including bacteremia due to atypical organisms, invasive pulmonary infections with Aspergillus, cytomegalovirus (CMV) viremia, skin infections, encephalitis with bacterial brain abscesses. Conclusions: Venetoclax in combination with hypomethylating agents or cytotoxic chemotherapy was effective in a subset of pediatric/AYA patients with advanced hematologic malignancies, but was frequently associated with severe atypical infections, particularly in combination with cytotoxic chemotherapy.

Bone marrow necrosis (BMN) is a rare pathologic finding, but when encountered is most often associated with malignancy. In adults its presence correlates with an inferior prognosis, however in children the prognostic implication is unclear. We performed a retrospective review of 3,760 bone marrow specimens in patients ≤ 18 years over a ten-year period. BMN was identified in less than 1% of specimens and only in patients with leukemia, lymphoma or neuroblastoma. BMN contributed to a delay in diagnosis; however, advanced medical imaging may serve as a tool to localize non-necrotic areas for bone marrow sampling, facilitating an expedited diagnosis.

Background: There remains limited knowledge about the immune response to severe acute respiratory syndrome coronavirus 2 (SARS-COV2) vaccination in pediatric oncology patients, which is essential to provide counseling and risk adaptation in this vulnerable population. The goal of this study was to understand immunogenicity after vaccination in pediatric oncology patients and determine if certain clinical factors impacted response. Methods: Patients 0-25 years of age with a diagnosis of cancer and actively receiving therapy were enrolled on study. We excluded patients who were completely vaccinated prior to their cancer diagnosis. Blood samples were collected pre-vaccination, as well as 2, 4-6, and 8-12 weeks after vaccination. Healthy children who were fully vaccinated enrolled as controls. Clinical data and complete blood counts around time of vaccination were collected. To study B and T cell immunity, we measured neutralizing antibodies by enzyme-linked immunoassay and interferon gamma secretion by enzyme-linked immunospot, respectively. Results: 26 patients enrolled on study, for which 11 were evaluable oncology patients and 7 were healthy controls. Adequate B cell response was seen in 36.4% of patients and adequate T cell response in 77.8% of patients. Numbers were too small to detect differences based on malignancy type. There was no differences in immunity based on absolute lymphocyte count (ALC) or intensity of therapy. Conclusion: Pediatric oncology patients have a suboptimal immune response to SARS-COV2 vaccination. Booster doses will be imperative to provide optimal protection against COVID-19, however blood counts may not be a useful guide to optimize the time of administration.

Purpose Phase 1 study assessing the safety and toxicity of cabozantinib in combination with topotecan and cyclophosphamide for relapsed osteosarcoma and Ewing sarcoma. Methods Oral cabozantinib (25mg/m 2) was administered daily for 21 (dose level 1) or 14 (dose level -1B) days. Topotecan (0.75mg/m 2) and cyclophosphamide (250mg/m 2) were administered IV on days 1-5. A modified 3+3 design based upon first cycle dose-limiting toxicities (DLT) was used for dose escalation. Results Twelve patients with a median age of 15 (12.9-33.2) years were enrolled (7 with Ewing sarcoma; 5 with osteosarcoma); all were evaluable for toxicity. At dose level 1, three of six patients developed first cycle DLT: grade 3 epistaxis; grade 3 transaminitis; prolonged grade 2 thrombocytopenia. Six patients were enrolled on dose level -1B (interrupted cabozantinib, given days 8-21), with one first cycle DLT (grade 3 pneumothorax) observed. Of the 10 response evaluable patients, one had partial response (Ewing sarcoma), seven had stable disease, and two had progressive disease. Conclusions The recommended phase 2 doses and schedules for this combination are topotecan 0.75mg/m 2 IV days 1-5, cyclophosphamide 250mg/m 2 IV days 1-5, and cabozantinib 25mg/m 2 days 8-21. Non-concomitant administration of cabozantinib with cytotoxic therapy in this population has acceptable toxicity while allowing for potential disease control.

As we celebrate 2020 as the Year of the Nurse and the Midwife and recognize the Global Initiative for Childhood Cancer, members of the International Society of Pediatric Oncology (SIOP) Baseline Nursing Standards Taskforce would like to highlight advocacy efforts promoting the baseline nursing standards.1, 2 Your published article, An ethical imperative: safety and specialization as nursing priorities of WHO Global Initiative for Childhood Cancer(Pergert and colleagues) reveals the importance of ongoing efforts to support implementation of the Baseline Nursing Standards.3 Given that the majority of hospitals are not meeting the standards in low- and middle-income countries (LMIC), as well as some high-income countries (HIC),4, 5 advocacy initiatives are required to raise awareness of the need to meet these standards. During the COVID-19 pandemic, health facilities face new challenges in meeting the standards. To achieve the WHO global initiative’s goal to save one million children’s lives by 2030, it is important to continue efforts to address baseline nursing standards.Pediatric oncology as a subspecialty requires a nursing workforce with specialized education and clinical skills to achieve optimal patient outcomes. Knowledge itself is not enough if nurses lack the resources and support to practice or implement appropriate nursing care in their work settings. The six Baseline Nursing Standards focus on key elements essential to delivering quality and safe care (Table 1). Collectively, they serve as a framework and foundation for positive pediatric oncology nursing practice environments internationally.Advocacy efforts to disseminate the baseline standards are well established. To date, fourteen organizations have endorsed the Standards. Members of the SIOP PODC Nursing Working Group hosted a “Leadership and Advocacy Workshop: Disseminating the Baseline Nursing Standards” prior to the SIOP Conference in October 2017. Twenty-two pediatric hematology/oncology nurse leaders and four stakeholder-group representatives (parent, physicians, advocates) from 14 countries met and established goals and strategic priorities for advocacy of the standards. As a result, the Baseline Nursing Standards Advocacy Toolkit was developed and can be found on the SIOP Nursing Website https://siop-online.org/baseline-nursing-standards-advocacy-toolkit. The toolkit contains practical advocacy resources, including a PowerPoint presentation, an endorsement letter template, publications, podcasts, a social media campaign and examples of elevator speeches for each standard. Furthermore, the Standards have been featured in international presentations, such as a keynote presentation (S. Day) in SIOP Lyon, an award session and nursing abstract presentations at SIOP congresses and continental meetings.To reach the WHO target of doubling the global childhood cancer survival rate to 60%, achievement of baseline nursing standards for pediatric oncology must be prioritized and appropriately resourced by hospital administrators, governments and other stakeholders. Amid a global pandemic where nursing resources are stretched, creative ways to support and advocate for implementation of the standards is needed. In recognition of the recent publication by the Nurse Specialists of the Global Initiative for Childhood Cancer noting the baseline standards, now is the time to act and improve childhood and adolescent cancer outcomes through raising the standard of pediatric oncology nursing practice around the world.Linda Abramovitz, Rehana Punjwani, Glenn M. Afungchwi and Courtney Sullivan and the SIOP PODC Baseline Standards Nursing Task Force.A special thank you to Rachel Hollis for her commitment and ongoing advocacy efforts focused on the baseline nursing standards.ReferencesDay S, Hollis R, Challinor J, Bevilacqua G, Bosomprah E, SIOP PODC Nursing Working Group. Baseline standards for paediatric oncology nursing care in low to middle income countries: position statement of the SIOP PODC Nursing Working Group. Lancet Oncol. 2014; 15(7):681-682 PMID: 24872097.Day S, Challinor J, Hollis R, Abramovitz L, Hanaratri Y, Punjwani R. Paediatric Oncology nursing care in low-and middle-income countries: a need for baseline standards. Cancer Control. 2015;2015:111-116Pergert P, Sullivan CE, Adde M, et al. An ethical imperative: Safety and specialization as nursing priorities of WHO Global Initiative for Childhood Cancer. Pediatr Blood Cancer. 2019;e28143. https://doi.org/ 10.1002/pbc.28143Morrissey L, Lurvey M, Sullivan C, et al. Disparities in the delivery of pediatric oncology nursing care by country income classification: international survey results. Pediatr Blood Cancer. 2019;66(6):e27663.Sullivan CE, Morrissey L, Day SW, Chen Y, Shirey M, Landier W. Predictors of Hospitals’ Nonachievement of Baseline Nursing Standards for Pediatric Oncology. Cancer Nurs. 2019 Mar 29;

Abstract: Background. Doxorubicin chemotherapy drug , use is limited by it’s potential to cause cardiotoxicity. In resource poor settings, like Malawi, monitoring of doxorubicin cardiotoxicity is not routinely conducted in cancer patients and the incidence of doxorubicin cardiotoxicity is not known. Methods. Children aged 3 months to 18 years with cancer were prospectively enrolled from the paediatric oncology ward and followed up from January 2016 to June 2019. Transthoracic echocardiographic monitoring of left ventricular ejection fraction (LVEF) was done at baseline, one month, six months and a year after completion of therapy. Cardiotoxicity was defined as a decline in LVEF of ≥10% to a final value of <50%, and an overall incidence risk of developing cardiotoxicity was estimated. A one-way analysis of variance was conducted to compare baseline LVEF with that measured during follow up intervals. Findings. A total of 91 children were enrolled into the study, 74% (68/91) were male, and 67% (62/91) were aged 5 months to 14 years. Burkitt lymphoma was diagnosed in 41% (38/91) of the children. No one experienced cardiotoxicity during the study period. However, of 77 children who had at least one follow up, five children 6·54% (95% CI: 2.1-14.5) experienced a reduction in LVEF of >10%, though not to a final value of <50%. No deterioration of systolic function was found among 20 children who had completed follow up. (F= 2·43, p-value=0·07). Interpretation. In this cohort, there were no observed cardiotoxic events associated with doxorubicin administration as per pre-defined criterion

Some vascular anomalies can present with challenging hematologic aberrations. Kaposiform hemangioendothelioma (KHE) may be complicated with Kasabach-Merritt phenomenon (KMP) and stagnant blood flow in slow flow malformations can promote activation and consumption of coagulation factors which results in bleeding and clotting known as localized intravascular coagulopathy (LIC). These patients can experience significant morbidity secondary to pain due to thrombosis and are at higher risk of hematologic complications during surgical procedures. No standard of care has been established to prevent or manage these complications. This review focuses on the management of coagulopathy in children and adults with vascular anomalies.

Rhabdomyosarcomas (RMS) are the most common pediatric soft tissue sarcomas. High-risk and metastatic disease continues to be associated with very poor prognosis. RMS model systems that faithfully recapitulate the human disease and provide rapid, cost-efficient estimates of anti-tumor efficacy of candidate drugs are needed to facilitate drug development and personalized medicine approaches. Here, we present a new zebrafish-based xenotransplant model allowing for rapid and easily accessible drug screening using low numbers of viable tumor cells and relatively small amounts of water-soluble chemicals. Under optimized temperature conditions, embryonal RMS-xenografts were established in zebrafish embryos at 3 hours post fertilization (hpf). In proof-of-principle experiments, chemotherapy drugs with established clinical anti-RMS efficacy (vincristine, dactinomycin) and the MEK inhibitor trametinib were shown to significantly reduce the cross-sectional area of the tumors by 120 hpf. RMS xenograft models in zebrafish embryos henceforth could serve as a valuable addition to cell culture and mammalian models of RMS and represent a rapid and cost-effective solution for pre-clinical candidate drug testing.

Background To assess the outcomes of pediatric patients with Undifferentiated Embryonal Sarcoma of the Liver (UESL) and treatment including at least surgery and systemic chemotherapy. Methods This study included patients aged up to 21 years with a pathological diagnosis of UESL prospectively enrolled from 1995 to 2016 in three european trials focusing on the effects of surgical margins, preoperative chemotherapy, use of radiotherapy (RT) and chemotherapy. Results Out of 65 patients with a median age at diagnosis of 8.7 years (0.6-20.8), 15 had T2 tumors, and 1 had lymph node spread, 14 were Intergroup Rhabdomyosarcoma Study (IRS) I, 9 IRSII, 38 IRSIII, and 4 IRSIV. Twenty-eight upfront surgeries resulted in 5 operative spillages and 11 infiltrated surgical margins, whereas 37 delayed surgeries resulted in no spillages (P= 0.0119) and 3 infiltrated margins (P=0.0238). All patients received chemotherapy, including anthracyclines in 47. Radiotherapy was administered in 15 patients. With a median follow-up of 78.6 months, 5 year overall and event free survivals (EFS) were 90.1% (95%CI 79.2-95.5) and 89.1% (95%CI 78.4-94.6), respectively. Two out 4 local relapses had previous infiltrated margins and 2 out of 3 patients with metastatic relapses received reduced doses of alkylating agents. Infiltrated margins (P=0.1607), T2 stage (P=0.3870), use of RT (P= 0.8731), and anthracycline-based chemotherapy (P= 0.1181) were not correlated with EFS. Conclusions Neoadjuvant chemotherapy for pediatric patients with UESL increases the probability of complete surgical resection. The role of anthracyclines and radiotherapy for localized disease remains unclear. The use of alkylating agents is recommended.

BACKGROUND: Rhabdomyosarcoma (RMS) is an aggressive malignancy, and 20% of children present with metastases at diagnosis. Patients presenting with disseminated disease very occasionally have no clear evidence of a primary tumor mass. Since these patients have rarely been investigated, we report on a series of patients with RMS and unknown primary tumor site registered in the MTS 2008 protocol (October 2008 - December 2016) coordinated by the European pediatric Soft tissue sarcoma Study Group. METHODS: Patients were administered 9 cycles of induction chemotherapy, and 48 weeks of maintenance chemotherapy. Surgery and/or radiotherapy was planned after the first assessment of tumor response, and implemented after six cycles of chemotherapy. If feasible, radiotherapy to all sites of metastasis was recommended. RESULTS: We identified 10 patients with RMS and unknown primary site, most of them adolescents (median age 15.8 years, range 4.6-20.4). Nine had fusion-positive alveolar RMS. Multiple organ involvement was identified in 7 patients, 2 only had bone marrow disease, and 1 only had leptomeningeal dissemination. All patients were given chemotherapy, 4 were irradiated, and none had surgery. Three patients underwent allogeneic bone marrow transplantation. At the time of this analysis, only 2 patients are alive in complete remission: 1 had received radiotherapy; and 1 had a bone marrow transplant. CONCLUSIONS: RMS with unknown primary tumor occurs mainly in adolescents and is typically fusion-positive alveolar. Radiotherapy may be important, but survival is poor and patients should be offered enrollment in investigational trials.

INTRODUCTIONThe frequency of pediatric head and neck neoplasms ranges from approximately 2-15% of all pediatric cancers1. Skull base and calvarial neoplasms, including temporal bone and orbital masses are a subset of head and neck neoplasms occupying a number of neoplastic processes and categorically encompass several imaging patterns: singular dominant mass lesions with or without metastatic disease (e.g., rhabdomyosarcoma (RMS), chordoma), singular or multifocal metastatic disease (e.g., neuroblastoma), and multifocal disease due to systemic malignancy (e.g., leukemia, lymphoma, histiocytosis, etc.).While pathologies certainly vary between pediatric and adult patients, there remains significant overlap. As such, imaging protocols for skull base and calvarial neoplasms are similar in their general construct. However, optimized pediatric-specific protocols remain a must, as a retrofitted adult head and neck protocol is often ill equipped to offer quality, efficient and safe imaging (e.g., limiting radiation exposure) of the size-variable infant and pediatric patient. Moreover, sedation or general anesthesia is often required in the pediatric population in order to minimize motion artifact. Thus, optimization of imaging acquisition time is a very important technical consideration because it may decrease the necessity and duration of sedation/anesthesia and their potential risks in this vulnerable population. Standardized protocols for anatomic sub sites of the head and neck offer significant benefit in individual patient follow up on a local scale, and, on a broader scale, allow for collaborative understanding of imaging pathologies and innovative or benchmarked standardized treatment response assessment across institutions.In an effort to standardize protocols, this article offers minimum, pediatric specific anatomy-based initial and follow up imaging guidelines for pediatric malignancies of the orbits, calvarium, skull base and temporal bone. This manuscript was funded in part by the National Clinical Trials Network Operations Center Grant U10CA180886. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Abstract Background: Location of cancer care (LOC: pediatric versus adult center) impacts outcomes in adolescents and young adults (AYA) with some cancer types. Data on impact of LOC on survival in AYA with osteogenic sarcoma (OGS) and Ewing sarcoma (EWS) are limited. Objectives: To compare differences in demographics, disease/treatment characteristics, and survival in a population-based cohort of AYA with OGS or EWS treated at pediatric versus adult centers Methods: The IMPACT Cohort captured demographic, disease, and treatment data for all AYA (15-21 years old) diagnosed with OGS and EWS in Ontario, Canada between 1992-2012. Patients were linked to provincial administrative healthcare databases. Outcomes were compared between patients treated in pediatric versus adult centers using appropriate statistical methods. Results: 137 AYA were diagnosed with OGS (LOC: 47 pediatric, 90 adult) and 84 with EWS (LOC: 38 pediatric, 46 adult). AYA treated at pediatric centers were more likely to be enrolled in a clinical trial (OGS 55% vs 1%, [p<0.001]; EWS 53% vs 2%, [p<0.001]) and received higher cumulative chemotherapy doses. Five-year event-free survival (EFS ± Standard Error) in OGS and EWS were 47% ± 4 and 43% ± 5, respectively. In multivariable analysis, the impact of LOC (pediatric vs adult center) on EFS in OGS (adjusted hazard ratio [HR] 1.15, 95% CI 0.58-2.27, P=0.69) and EWS (adjusted HR 1.82, 95% CI 0.97-3.43, P=0.06) were not statistically significant. Conclusion: Outcomes did not differ by LOC suggesting that AYA with bone tumors can be treated at either pediatric or adult centers.

The incidence of bone marrow metastasis (BMM) in newly diagnosed Ewing sarcoma (ES) is variable across studies. An optimal staging strategy for detecting BMM is not defined. While BM biopsy and/or aspirate (BMBA) have been the gold standard, [F-18]fluorodeoxyglucose positron emission tomography (FDG-PET) to detect BM metastasis may decrease reliance on BMBA. We conducted a systematic review to assess incidence of BMM and the role of FDG-PET. We observed a pooled incidence by BMBA of BMM of 4.8% in all newly diagnosed ES patients and 17.5% among patients with metastatic disease. Only 1.2% of patients had BMM as their sole metastatic site. FDG-PET detection of BMM compared to BMBA demonstrated pooled 100% sensitivity and 96% specificity, positive predictive value of 75% and negative predictive value of 100%. In the era of FDG-PET imaging, omission of BMBA may be considered in patients with otherwise localized disease after initial staging studies.

BACKGROUND: Diagnostic mIBG (meta-iodobenzylguanidine) scans are an integral component of response assessment in children with high-risk neuroblastoma. The role of end of induction (EOI) Curie Scores (CS) was previously described in patients undergoing a single autologous hematopoietic cell transplant (AHCT) as consolidation therapy. OBJECTIVE: We now examine the prognostic significance of CS in patients randomized to tandem AHCT on the Children’s Oncology Group (COG) trial ANBL0532. STUDY DESIGN: A retrospective analysis of mIBG scans obtained from patients enrolled in COG ANBL0532 was performed. Evaluable patients had mIBG-avid, International Neuroblastoma Staging System (INSS) stage 4 disease, did not progress during induction therapy, consented to consolidation randomization, and received a tandem AHCT (n=80). Optimal CS cut points maximized the outcome difference (≤ vs >CS cut-off) according to the Youden index. RESULTS: For recipients of tandem AHCT, the optimal cut point at diagnosis was CS=12, with superior EFS from study enrollment for patients with CS<12 (3-year EFS 74.2±7.9%) vs CS>12 (59.2±7.1%) (p=0.002). At EOI, the optimal cut point was CS=0, with superior end-induction EFS for patients with CS=0 (72.9±6.4%) vs CS>0 (46.5±9.1%) (p=0.002). CONCLUSION: In the setting of tandem transplantation for children with high-risk neuroblastoma, Curie scores at diagnosis and end-induction may identify a more favorable patient group. Patients treated with tandem AHCT who exhibited a CS<12 at diagnosis or CS=0 at EOI had superior EFS compared to those with CS above these cut points.

Since the publication of the last Cellular Therapy and Stem Cell Transplant blueprint in 2013, Children’s Oncology Group cellular therapy-based trials for advanced the field and created new standards of care across a wide spectrum of pediatric cancer diagnoses. Key findings include that tandem autologous transplant improved survival for patients with neuroblastoma and atypical teratoid/rhabdoid brain tumors, one umbilical cord blood (UCB) donor was safer than two UCB donors, killer immunoglobulin receptor (KIR) mismatched donors did not improve survival for pediatric acute myeloid leukemia when in vivo T cell depletion is used and the depth of remission as measured by next-generation sequencing based minimal residual disease assessment pre-transplant was the best predictor of relapse for acute lymphoblastic leukemia. Plans for the next decade include optimizing donor selection for transplants for acute leukemia/myelodysplastic syndrome, using novel engineered cellular therapies to target a wide array of malignancies, and developing better treatments for cellular therapy toxicities such as viral infections and graft-vs-host disease.