Pediatric Blood & Cancer最新文献

Introduction: The TEDDI trial tested the feasibility and reproducibility of deep-inspiration breath-hold (DIBH) in pediatric patients referred for radiotherapy. This report presents final results, including patient-reported outcomes (PRO) and dosimetric comparison of DIBH and free-breathing (FB).

Patients and methods: Pediatric patients able to perform three sequential breath-holds and potentially requiring thoracic or upper abdominal radiotherapy were recruited. DIBH training was during staging or planning computed tomography (CT) scanning, using external gating with an external marker and visual coaching. Each patient underwent planning CT in both DIBH and FB, generating two radiotherapy plans. DIBH was selected if it resulted in a lower overall dose to organs at risk. At two centers, patients evaluated their DIBH experience during training. Those treated in DIBH also completed three daily questions and extended questionnaires at the start, midpoint, and end of treatment, using yes/no and five-point Likert scales.

Results: Twenty-five patients (12 females/13 males, median age 15 years, range: 9-17 years) were enrolled across three centers. Eight received photon radiotherapy, five in DIBH. Of 13 eligible patients, 11 rated DIBH training, with 10 selecting "Really good" or "Good." Patients treated in DIBH reported feeling safe and comfortable. Dosimetric analysis showed clear heart and lung dose reductions with DIBH. FB patients had similar doses across both plans.

Conclusion: The TEDDI trial demonstrated the feasibility and safety of DIBH in pediatric radiotherapy. High compliance with the procedure and favorable dosimetric outcomes support the use of DIBH to reduce long-term toxicity risks in this population.

Background: Proton beam therapy (PBT) offers dosimetric advantages in pediatric radiotherapy by reducing radiation exposure to healthy tissues. However, broad implementation raises important ethical, logistical, and equity considerations, especially in settings with limited access to advanced technologies.

Procedure: This position paper by the Paediatric Radiation Oncology Society (PROS) reviews the clinical rationale for selecting radiotherapy modalities in children, with a specific focus on ethical utilization of PBT. The document synthesizes evidence on the benefits and limitations of modern photon and proton therapies, explores disparities in access, outlines the role of local pediatric radiation oncologists, and addresses the impact of industry-driven messaging on family decision-making.

Results: While PBT offers clinical benefit in selecting pediatric cancers, high-quality photon-based treatments such as IMRT and VMAT remain effective and widely accessible alternatives. The referral and evaluation process for PBT is complex and may introduce treatment delays. Travel and financial burdens can exacerbate disparities. PROS underscores the critical role of local pediatric radiation oncologists in counseling families, and calls for balanced, evidence-based communication from providers and industry stakeholders.

Conclusions: PROS advocates for an ethical, equitable, and evidence-informed approach to radiotherapy modality selection in children, reflecting holistic considerations beyond dosimetry. Proton therapy should be pursued when clinically indicated and accessible without compromising timely care. Investment in a comprehensive radiation therapy infrastructure, including professional education, collaborative research, and responsible public messaging, is essential to optimize pediatric cancer outcomes globally.

Background: Over 90% of US children with cancer are treated at Children's Oncology Group (COG) centers, which seek to maximize enrollment in therapeutic and biobanking studies. Rare cancers have demonstrated lower than expected COG enrollment. We evaluated trends in COG rare cancer enrollment compared to US incidence from Surveillance, Epidemiology, and End Results (SEER) registries, examining the impact of COG therapeutic trials and Project:EveryChild, a cancer biobank/registry.

Procedure: COG and SEER data from 2002 to 2020 were queried for US patients younger than 18 years old with adrenocortical carcinoma (ACC), nasopharyngeal carcinoma (NPC), retinoblastoma (RB), thyroid carcinoma, and melanoma. We compared demographic data between COG and SEER, extrapolating incidence for each cancer to analyze trends in COG enrollment.

Results: Patient characteristics, including age, sex, and race, were similar between COG (n = 2184) and SEER (n = 5514). COG enrollment for rare cancers remained low (11%). Initiating Project:EveryChild did not increase enrollment (12% pre- vs. 8% post-Project:EveryChild; p < 0.01). For cancers with available therapeutic trials (ACC, NPC, and RB), COG enrollment was higher during trial accrual (40%) than when no trial was open (12%; p < 0.01). Patient geography and income did not appear as barriers to COG enrollment.

Conclusions: Although children with rare cancers enrolled in COG studies reflect the US population, enrollment in the COG registry/biospecimen repository continues to be limited in the absence of therapeutic trials, impacting data and biospecimens available to inform therapeutic trial development. Expansion of therapeutic trials or free molecular testing through the Molecular Characterization Initiative may increase data and biospecimens for these rare cancers.

Introduction: Elevated visceral fat is associated with poor cardiovascular health but is not well characterized in survivors of childhood cancer. We examined central adiposity and associated risk factors in a pediatric acute lymphoblastic leukemia (ALL) survivorship cohort.

Methods: Visceral adipose tissue (VAT) mass and estimated waist circumference (WC) were extracted from dual energy x-ray absorptiometry (DXA) scans on 70 survivors of pediatric ALL >10 years from diagnosis. Waist-to-height ratios (WHtRs), a body shape index (ABSI), ABSI z scores, and descriptive statistics were calculated to examine central adiposity. We tested sensitivity/specificity of WHtR at established thresholds for identifying VAT ≥85th percentile (%le).

Results: VAT z scores were shifted positively relative to population norms with 25.7% ≥ 85th %le. Mean WHtR was 0.55 ± 0.06 with 82.9% above the "take action" threshold of 0.5. A WHtR ≥0.59 had a sensitivity of 90.2% (95% CI 82.0-98.4) and specificity of 68.4% (95% CI 47.5-89.3) for identifying individuals with VAT ≥85th %le. The mean ABSI z score was 1.88 ± 0.85; higher in women, in high risk ALL, and post-cranial radiation (p = 0.01-0.02). The ABSI z scores for 94.3% of survivors fell in the highest quintile of population values.

Conclusion: Nearly the entire cohort of long-term survivors of pediatric ALL have an elevated WC relative to height, weight, and population norms, regardless of their body mass index (BMI) or visceral fat. This suggests that a broader screening approach, which considers waist indices, may be better able to detect those at increased cardiometabolic risk. Evaluation and confirmation in a larger prospective cohort is indicated.