Hematopoietic stem cell transplantation (HSCT) is often the only curative treatment option for patients suffering from various types of malignant diseases and some non-cancerous conditions. Nevertheless, it is associated with a high risk of complications leading to transplant-related mortality and long-term morbidity. An increasing number of therapeutic and prevention strategies have been developed over the last few years to tackle the complications arising in patients receiving an HSCT. These strategies have been mainly carried out in adults and some are now being translated into children. In this manuscript, we review the recent advancements in the development and implementation of treatment options for post-HSCT non-infectious complications in pediatric patients with leukemia and other non-malignant conditions, with a special attention on the new agents available within clinical trials. We focused on the following conditions: graft failure, prevention of relapse and early interventions after detection of minimal residual disease positivity following HSCT in acute lymphoblastic and myeloid leukemia, chronic graft versus host disease, non-infectious pulmonary complications, and complications of endothelial origin.

IntroductionEligibility criteria safeguard trial integrity and safety, yet restrictive requirements frequently limit patient access and generalizability. While initiatives exist to simplify criteria in adult populations, evidence supporting their use and justification within pediatric oncology remains undefined.MethodsSixty-four protocols from 48 ITCC phase I–II trials (2011–2020) were evaluated. Protocol, literature analysis, and regulatory standards review was performed. Eligibility criteria were quantified and categorized by frequency: High (appeared in >66% of protocols), average (33%–66%), and low (<33%). For each high-frequency and relevant criteria identified in the literature review, the scientific justification provided within the trial protocol was assessed.ResultsAnalysis revealed a median of 28 criteria per protocol (range 12–49), with a significant increase between 2011–2016 and 2017–2020 (p=0.05). Substantial heterogeneity was observed in organ function thresholds and washout periods, irrespective of investigational agent characteristics. Repetition was prevalent; 69% of trials repeated similar criteria in both inclusion and exclusion sections. Notably, a median of only 7% (range 0%–62%) of high-frequency and relevant criteria possessed scientific justification within the protocol, with several exclusion categories lacking justification entirely.ConclusionPediatric early-phase trial eligibility criteria are increasingly complex, heterogeneous and predominantly lacking justification. We recommend implementing evidence-based, medically relevant requirements to optimize patient accrual and representation. Harmonizing these standards is essential to maintain safety while ensuring broader access to innovative therapies in pediatric drug development.

Introduction: Healthcare delivery is increasingly challenged by rising demand, escalating costs, and the need for efficiency. Contributing factors such as the nursing workforce crisis, fragmented and excessive clinical data, outdated IT infrastructures, and the growing complexity of care exacerbate the risk of medical errors, treatment delays, and staff burnout. To address these challenges, we evaluated AMIT, a novel digital platform designed to support nursing practice in pediatric oncology. Methods: AMIT is a web-based platform developed in 2022 and implemented in Q4 2023 in the pediatric oncology day care unit. It integrates data from 5 hospital IT systems, applying predefined rules based on local practice to highlight only clinically relevant information requiring intervention or safety event prevention. In addition, analytic and diagnostic alerts were generated. Primary outcomes included prevention of safety events, reduction in urgent PICU referrals, and shorter patient journey times; the secondary outcome was nursing satisfaction, assessed by questionnaire. Results: Following implementation, 61 safety events, including 2 potentially fatal cases, were identified and addressed. The incidence of urgent PICU referrals decreased by 78%, timely antibiotic administration (≤3 h) improved by 47%, and patient journey times for electrolytes and transfusions decreased by 58% (104 to 44 min). Nurse surveys indicated 100% satisfaction with workflow integration. Conclusions: AMIT improved patient safety, accelerated treatment, and streamlined nursing workflow, underscoring the value of rule-based digital platforms in clinical care. Future integration of large language models will enable automated translation of guidelines into validated clinical rules and support feedback loops to identify patient-specific deterioration patterns, fostering more harmonized and timely interventions across institutions.