Characteristics and treatment of acute myeloid neoplasms with cutaneous involvement in infants up to 6 months of age: A retrospective study.

BACKGROUND: Myeloid neoplasms account for 50% of cases of pediatric leukemias in infants. Approximately 25%-50% of patients with newborn leukemia have cutaneous extramedullary disease (EMD). In less than 10% of patients, aleukemic leukemia cutis or isolated extramedullary disease with cutaneous involvement (cEMD) occurs when skin lesions appear prior to bone marrow involvement and systemic symptoms. Interestingly, in acute myeloid leukemia with cutaneous EMD (AML-cEMD) and cEMD, spontaneous remissions have been reported. METHOD: This is a multicentric retrospective cohort study aiming to describe characteristics, treatment, and outcome of infants with either cEMD or presence of cutaneous disease with involvement of the bone marrow (AML-cEMD). This study included patients born between 1990 and 2018 from Italy, the Netherlands, Switzerland, and the United States, diagnosed between 0 and 6 months of life with cEMD or AML-cEMD. Descriptive statistics, Fisher's exact test, Kaplan-Meier method, and log rank test were applied. RESULTS: The cohort consisted of n = 50 patients, including 42 AML-cEMD and eight cEMD patients. The most common genetic mutation found was a KMT2A rearrangement (n = 26, 52%). Overall 5-year event-free survival (EFS) and overall survival (OS) were 66% [confidence interval (CI): 51-78] and 75% [CI: 60-85], respectively. In two patients, complete spontaneous remission occurred without any therapy. Central nervous system (CNS) involvement was found in 25% of cEMD patients. No difference in outcomes was observed between the AML-cEMD and cEMD groups, but none of the latter patients included in the study died. KMT2A rearrangements were not associated with poorer prognosis. CONCLUSION: In the largest cohort to date, our study describes the characteristics of infants with cutaneous involvement of myeloid neoplasms including cytomolecular findings and survival rates. Further prospective biologic and clinical studies of these infants with myeloid neoplasms will be required to individualize therapy for this rare patient population.

PASTEC - a prospective, single-center, randomized, cross-over trial of pure physical versus physical plus attentional training in children with cancer.

Despite recent improvements in survival rates in children with cancer, long-term toxicities remain a major concern. Physical activity could reduce the impact of long-term sequelae, notably in neuropsychological and physical areas. We report of a randomized trial of pure physical versus physical/attentional training in pediatric oncology patients. Twenty-two patients aged 6-18 y.o. were included, irrespective of their clinical diagnosis or treatment status, stratified by age and randomized 1:1 into pure physical vs. physical/attentional activity arms, with a cross-over at study midpoint. Neurological, motor and neuropsychological assessments were performed at inclusion, start, crossover and end of the program. Feasibility, defined as > 80% patients attending > 80% of sessions, was the primary endpoint. Secondary outcomes were improvements in neuropsychological and motor performance tests. While 68% of patients attended more than 80% of sessions during the pre-crossover phase of the study, this dropped to 36% post-crossover. Our study therefore failed to meet our primary endpoint. Nonetheless, significant improvements in anxiety (p<0.001), emotional control (p = 0.04), organization skills (p = 0.03), as well as motor deficit scores (p = 0.04) were observed. We noted no significant difference between the pure physical and the physical/attentional training arms, or when analyzing subgroups by age or sequence of intervention. We conclude that physical activity has a positive impact on anxiety, emotional and organizational aspects as well as motor deficits. Attendance dropped during the course of the study and motivational interventions should be included in future studies or equivalent programs.Supplemental data for this article is available online at https://doi.org/10.1080/08880018.2021.1994677 .

Adverse Events and Associated Factors During Intrahospital Transport of Newborn Infants.

OBJECTIVE: To determine the frequency, type, and severity of adverse events (AEs) during intrahospital transport of newborn infants and to identify associated factors. STUDY DESIGN: We conducted a prospective observational study in a tertiary care academic neonatal unit. All patients hospitalized in the neonatal unit and undergoing intrahospital transport between June 1, 2015, and May 31, 2017 were included. Transports from other hospitals and the delivery room were not included. RESULTS: Data from 990 intrahospital transports performed in 293 newborn infants were analyzed. The median postnatal age at transport was 13 days (Q1-Q3, 5-44). Adverse events occurred in 25% of transports (248/990) and were mainly related to instability of cardiovascular and respiratory systems, agitation, and temperature control. Adverse events were associated with no harm in 207 transports (207/990, 21%), mild harm in 37 transports (37/990, 4%), and moderate harm in 4 transports (4/990, 0.4%). There was no severe or lethal adverse event. Hemodynamic support with catecholamines, the presence of a central venous catheter, and a longer duration of transport were independent predictors for the occurrence of adverse events during transport. CONCLUSIONS: Intrahospital transports of newborns are associated with a substantial proportion of adverse events of low-to-moderate severity. Our data have implications to inform clinical practice, for benchmarking and quality improvement initiatives, and for the development of specific guidelines.

Intra-hospital transport of newborn infants dataset.

This article presents a dataset on intra-hospital transport of newborn infants. We collected prospectively data from patients hospitalized between 1.6.2015 and 31.5.2017 at the tertiary care neonatal unit of the University Hospital of Lausanne, Switzerland. An intra-hospital transport was defined as a transport for a diagnostic or a therapeutic intervention outside the neonatal unit, but within the hospital. Healthcare professionals present during the transport collected data in a case report form. We obtained additional data from electronic medical charts and through the clinical information system Metavision®. We recorded information on patients' demographics and clinical characteristics, transports (indication, date, duration, destination, number and type of staff involved, medical devices and treatments), adverse events and interventions. Heart rate, peripheral oxygen saturation and fraction of inspired oxygen were recorded within 5 min before and after the transport, with an additional measure during transport for patients that had continuous monitoring of vital signs. This dataset will be of use to clinicians, researchers and policy makers, to inform clinical practice, for benchmarking, and for the development of future guidelines. These data have been further analyzed and interpreted in the article "Adverse events and associated factors during intra-hospital transport of newborn infants" (Delacrétaz et al, 2021).