Pediatric Blood & Cancer最新文献

Objective: To explore the preliminary effectiveness and implementation outcomes (feasibility, acceptability, cost) of an intervention (eMaP), which provides electronic psychosocial screening and support for parents of children and adolescents with cancer (0-18 years).

Methods: This single-site, pilot implementation-effectiveness study, integrated routine screening into standard care for parents of children 12-16 weeks post diagnosis. Parents completed an online distress screening questionnaire, and those scoring high in distress received additional support from a social worker. Feasibility was measured using study uptake and completion rates, while costs were calculated based on social workers' time. Qualitative interviews provided insights into acceptability.

Results: Out of 66 parents approached, 57 (86%) agreed to participate, indicating strong interest. Preliminary results showed that parents with concerning initial distress levels, who received clinical social worker support, experienced significant reductions in distress and support needs, and improvements in quality of life from baseline to T2 (8 weeks later) (all p ≤ 0.01). Routine monthly screening was considered feasible and valuable by both parents and social workers. Parents appreciated distress screening as a means of increasing awareness of their needs and valued the additional psychosocial support provided when indicated, while social workers found it useful for capturing clinical information often missed in standard care.

Conclusion: The eMaP intervention was feasible and acceptable to both parents and staff, showing promise to improve parents' psychosocial outcomes. The study suggests the need for refinements of the intervention and explorations of effectiveness in improving parents' outcomes in future studies.

Background: Diarrhea is a common complication among pediatric hematopoietic stem cell transplantation (HCT) recipients. Although many of the cases are secondary to graft-versus-host disease (GVHD), stool microbiological studies are often performed to evaluate an underlying infectious etiology. The aim of this study was to assess the frequency and utility of stool studies in children who have undergone HCT.

Methods: Demographics, clinical characteristics, and stool study results (viral, parasitic, and bacterial) of all patients who underwent HCT at a large, academic, freestanding children's hospital between January 2006 and December 2023 were obtained. Statistical analysis conducted included t tests, chi-square, and linear regression.

Results: Overall, 1381 HCT recipients (9.2 ± 6.6 years) were included. Altogether, 6509 stool studies were obtained among 741 (54%) patients. Salmonella, Shigella, Yersinia, Campylobacter, and Escherichia coli (SSYCE) studies were sent on 363 (26%, 2252 studies) patients with 1 (0.04%) positive result. Clostridium difficile was sent on 706 (51%, 2055 studies) patients, with 156 positive studies (7.6%). Stool ova and parasite testing was sent on 143 (10%, 242 studies) patients, with two positive results (0.8%). Viral studies were sent on 638 (46%, 1960 studies) patients, with 107 positive studies (5.5%).

Conclusions: While testing for Clostridium difficile and enteric viruses may have value in the work-up of pediatric HCT patients, SSYCE and O&P studies hold little to no value. Clinical practices surrounding routine stool microbiological studies should be reconsidered.

Since 2000, centers across Central America have shared treatment guidelines for Wilms tumor, using histology (anaplasia present or absent) and tumor stage to stratify patients into low-, intermediate-, and high-risk groups. Weekly virtual tumor board meetings involving local and international experts were held to ensure consistent treatment assignments. We analyzed data from 367 children with unilateral tumors treated per these guidelines. Five-year abandonment-sensitive event-free and overall survival estimates were: low risk 82% ± 3.8% and 86% ± 3.6%, intermediate risk 50% ± 3.4% and 60% ± 3.4%, and high risk 36% ± 7.6% and 45% ± 7.9%. Survival outcomes were suboptimal, primarily due to advanced disease in fragile children at presentation and abandonment of treatment.

After 75 years of clinical use of folic acid antagonists such as methotrexate, relevant pharmacological data currently important for the effective and safe use of methotrexate were reviewed to see if it is possible to improve outcomes. Specifically, to improve how high-dose methotrexate (HD-MTX) can be given safely, what doses of MTX (methotrexate) are adequate to achieve therapeutic levels, and what is the appropriate folinic acid (FA) dose for effective rescue. This review is based on 50 years of personal experience with the use of HD-MTX in published literature. Many pharmacologic studies were performed over 50 years ago, but are still relevant and stand up to scrutiny today. What should be considered HD-MTX and how it can be given safely and effectively without late toxicity are presented. The variables responsible for effective folinic acid rescue, especially the doses of MTX and folinic acid and the time to start of rescue, are discussed. Understanding these highlighted aspects of therapy could help to prevent acute toxicity, improve treatment results, and prevent late effects.

Background: Little progress has been made in determining prognostic factors for patients with high-grade mature B-cell non-Hodgkin lymphoma (HG B-NHL). Based on the important role of lymphocytes in cancer progression, this study aimed to explore the effect of peripheral blood lymphocytes on the prognosis of pediatric HG B-NHL.

Methods: Patients aged less than 18 years with newly diagnosed HG B-NHL were enrolled. Peripheral blood lymphocyte subset levels were detected at diagnosis, and their optimal cutoff values were determined according to event-free survival (EFS).

Results: In total, 206 patients were enrolled. The 5-year EFS and overall survival (OS) rates of the whole group were 92.1% ± 1.9% and 96.6% ± 1.3%, respectively. The 5-year EFS rate was worse in patients with a low relative CD4⁺ T-cell count (87.2% vs. 97.0%, p = 0.008), high relative CD8⁺ T-cell count (79.1% vs. 93.4%, p = 0.03), low CD4/CD8 ratio (80.5% vs. 94.2%, p = 0.01), and low B-cell count (80.0% vs. 93.4%, p = 0.02) at diagnosis than their counterparts. Cox multivariate analysis identified low relative CD4⁺ T-cell (HR = 4.91) and B-cell (HR = 3.87) counts at diagnosis as independent adverse prognostic factors. Patients with simultaneously low levels of CD4⁺ T and B cells had the worst outcomes in the entire cohort, with a 5-year EFS rate of 60.0%.

Conclusion: Low relative CD4⁺ T-cell and B-cell counts at diagnosis are associated with poor prognosis in children and adolescents with HG B-NHL in the real world.

Background: Vascular anomalies (VA) are rare developmental disorders due to somatic variants in intracellular growth signaling pathways. Although genetic evaluation is considered the standard of care for optimizing management, the frequency of variants of uncertain significance (VUS) in VA and their clinical implications are not defined.

Methods: Medical records were reviewed on all patients seen in our VA clinic from January 2014 to August 2024 with vascular malformations or related disorders who had undergone genetic testing. The year and patient age at the time of genetic testing, whether testing was done on peripheral blood or involved tissue, and results were noted. Laboratory vendors were queried about policies for reclassifying VUS and selective re-evaluations of VUS were requested.

Results: In all, 154 patients underwent single gene or gene panel evaluations, with the number of patients tested increasing from zero in 2014 to 39 in 2023; 21 patients had testing done during the first 7 months of 2024. VUS were reported in 22 patients (14%). Many laboratory vendors maintain a policy of revisiting these nonpathogenic variants only when requested by the treating physician. Unsolicited reclassification was reported in a single patient whose VUS was found to be a normal variant. No changes were identified in six other patients whose VUS were re-examined.

Conclusions: VUS are not uncommon in VA. Reclassification may be possible as more variant-specific data become available and more testing is performed. Centers need to be aware that reclassification efforts are not automatic and should be considered where changes in therapy might result.

Adolescents and young adults (AYAs) with cancer face unique demands, which result in barriers to psychosocial/behavioral trial retention. Theory-informed and patient-engaged strategies have the potential to increase retention. The purpose of this manuscript is to illustrate how to partner with AYAs and leverage the Theoretical Domains Framework to identify barriers to retention and behavior change techniques (BCTs) targeting these barriers to create retention strategies. Exemplar strategies developed using this approach and implemented in our trial of an adherence-promotion intervention are discussed, and a roadmap is included for teams interested in developing similar strategies to meet their unique needs.

Background: Glucose-6-phosphate dehydrogenase (G6PD) deficiency is a red cell enzymopathy in which exposure to oxidative stressors, such as drugs or fava bean ingestion, can trigger acute hemolytic episodes (AHEs). This study aimed to compare the clinical characteristics of fava bean-induced hemolysis (FBIH) with non-fava bean-induced hemolysis (NFBIH) in children with G6PD deficiency in a high-prevalence setting.

Methods: A prospective cohort study was conducted at a region referral hospital in Oman. Hospital records of children hospitalized for AHE due to G6PD deficiency over a 3-year period were analyzed. Participants were categorized into FBIH and NFBIH groups based on the documented precipitating factor.

Results: Among the 236 recruited cases, 51.6% AHEs were attributed to FBIH. Children with FIBH were younger, more likely to present with abdominal pain, and had greater severity of hemolysis upon admission (hemoglobin: 4.8 vs. 6.7 g/dL; p < 0.001). Lab markers such as serum ferritin, blood urea, lactate dehydrogenase, and alkaline phosphatase were significantly elevated in FBIH. The least squares regression model demonstrated a strong link between various predictor variables and hemoglobin levels, explaining about 76.6% of the variance in the study cohort.

Conclusion: Children with FBIH experience more severe hemolytic episodes compared to those with NFBIH. Our statistical model identified clinical and laboratory parameters potentially useful in early risk stratification during AHEs. Culturally sensitive dietary education of patients and caregivers is necessary, particularly in regions where fava beans are a dietary staple. The potential influence of specific G6PD genotypes within the NFBIH group merits future investigation.