Pediatric Blood & Cancer最新文献

Introduction: The COVID-19 pandemic onset had a global debilitating impact on individuals and on burgeoning careers. In 2021, the Children's Oncology Group Young Investigators Committee, Young SIOP (International Society of Paediatric Oncology) Network, and Young SIOPE (European Society for Paediatric Oncology) co-sponsored a survey to explore the impacts of the first year of the pandemic on early-career pediatric oncology professionals with respect to working practices, productivity, professional and career development, personal wellbeing, and changing childcare needs.

Methods: The survey comprised demographic, multiple-choice, and free-text questions, and was distributed via email and social media with English, French, and Spanish versions available. Descriptive statistics and chi-square tests were used to compare quantitative data by self-designated gender and country of origin. Qualitative data were described using content analysis.

Results: Professionals (N = 499, 26.3% male, 77.2% MDs) in 48 countries (77.6% high income) responded in English (79.4%), Spanish (12.4%), and French (8.2%). Respondents had difficulty obtaining and keeping jobs (26.9%), worsened overall academic productivity (50.7%, with higher rates among bench scientists, p < 0.01), and decreased career opportunities (40.9%). Childcare challenges impacted 56.7% of respondents and was felt more negatively among women (p = 0.008) and in high-income settings (p < 0.0001). Qualitative data (n = 300) highlighted these differences were often attributable to diminished professional/personal boundaries and impacted their personal wellbeing.

Conclusion: The COVID-19 pandemic significantly impacted early-career academic and clinical professionals working in pediatric oncology, with unique challenges noted among those with childcare responsibilities. Career disruptions that resulted from the pandemic should be considered and mitigated by governing bodies and hiring institutions.

Children and adolescents and young adults (CAYAs) with sickle cell disease (SCD) suffer from recurrent acute pain episodes that result in hospitalization and confer substantial morbidity. Pharmacologic pain control is often inadequate, thus non-pharmacologic modalities are needed. Osteopathic manipulative treatment (OMT) is a potentially valuable non-pharmacologic addition to pain management. We describe eight patients hospitalized with SCD pain who received OMT. OMT was safe, feasible, and a promising adjunctive option for management of SCD pain.

Introduction: The death of a child has a tremendous impact on parents' lives. The experience of parents who have lost a child to cancer may differ from other bereavement experiences, including other childhood and adulthood causes of death, because of the uncertainty of the prognosis, the aggressive treatment, and the potential for regret about treatment decisions. Bereavement care remains scarce, and effective interventions to meet the diverse needs of parents have not been defined.

Objective: To provide insights on bereaved parents' experiences, their needs, and wishes of support following the loss of their child to cancer.

Methods: We conducted 18 qualitative, in-depth, semi-structured interviews with 23 bereaved parents (seven fathers, 16 mothers), and used reflexive thematic analysis to analyze the data.

Results: Parents received both informal and professional support. Regarding informal support, parents expressed a high level of ambivalence rooted in grief illiteracy. Parents also recognized their own struggles to express what forms of informal support they would have liked to receive. Support provided by healthcare professionals, institutions, and organizations involved in the children's care or in bereavement was inconsistent due to personnel time constraints, interpersonal relationships, or disruptions due to the COVID-19 pandemic. These factors could result in parents not having access to certain forms of support or not receiving long-term support.

Conclusion: Improving grief literacy may strengthen informal support and make discussions of grief and death less taboo. Institutional policies, training, and networking may help to ensure that support provided by healthcare professionals, institutions, and organizations is less vulnerable to inconsistencies.

Background: Tumor invasion of the spinal canal is detected radiographically in approximately 15% of patients with newly diagnosed neuroblastoma (NB). The optimal clinical approach to maintain excellent survival outcomes while minimizing long-term sequelae is yet to be defined.

Methods: Patients with intermediate-risk neuroblastoma (IR-NB) and radiographically identified intraspinal tumors who were treated on the Children's Oncology Group study ANBL0531 were studied prospectively to evaluate neurologic outcomes related to cord compression. Patients were defined as being symptomatic versus asymptomatic based on reporting of neurologic motor deficits at diagnosis. Patient characteristics, tumor biology, chemotherapy treatment, surgical interventions, and neurologic and disease outcomes are reported.

Results: Of the 92 patients with intraspinal tumors, 42 (46%) were symptomatic and most (73%) had complete resolution of symptoms. Age, degree of motor deficit, and duration of symptoms at diagnosis were not associated with complete resolution. While symptomatic patients were more likely to undergo upfront laminectomy, laminectomy was not associated with improvement of motor symptoms. Administration of additional chemotherapy beyond initial treatment assigned per protocol to achieve the treatment end point was not associated with achieving symptom resolution.

Conclusion: Patients presenting with motor deficits due to intraspinal tumor had excellent survival and favorable neurologic outcomes, with the majority reporting complete resolution of motor symptoms regardless of severity and duration of symptoms at diagnosis or neurosurgical intervention. Prompt diagnosis and initiation of first-line chemotherapy treatment remain priority, while neurosurgical intervention should be reserved for patients with rapid neurologic deterioration. Biology-based therapy and tumor response should continue to be used to maintain favorable outcomes.

Background: Spinal cord compression (SCC) is a severe complication in pediatric patients with relapsed, progressing, or refractory solid malignancies. This study aims to report the presentation, treatment, and role of proactive radiation therapy in these patients.

Methods: This retrospective study reviewed 28 patients with relapsed, progressing, or refractory solid tumors who were referred for radiation therapy consultation due to potential (impending) or actual spinal cord compression (pSCC and aSCC, respectively) over a 12-year period. Collected data included disease characteristics, details of SCC events, management approaches, and patient outcomes.

Results: Among the 28 patients, 34 SCC events were identified (18 pSCC, 16 aSCC), with neuroblastoma being the most frequent diagnosis (46.4%). No significant differences were noted between pSCC and aSCC groups in pre-event imaging follow-up, age distribution, or malignancy status at SCC presentation. However, aSCC patients exhibited significantly more symptoms at diagnosis. Both groups received SCC-targeted therapy, with no significant differences in functional outcomes, event-free survival (EFS), or overall survival (OS). Pain assessments post treatment showed comparable improvements in both groups.

Conclusions: Proactive radiotherapy for pSCC did not yield superior outcomes compared to reactive treatment for aSCC. Given the limited benefits observed, proactive RT should be considered on an individual basis, balancing potential QoL improvements against treatment burdens. Further research in larger cohorts is necessary to refine therapeutic strategies for SCC in pediatric oncology.

Background: Adult survivors of unilateral, nonmetastatic, non-syndromic Wilms tumor (WT) treated with whole abdomen radiation therapy (WART) are at risk for impaired kidney function. The impact of bias and accuracy on estimated glomerular filtration rate (eGFR) among adult survivors of WT has not been well documented.

Procedure: We clinically evaluated male and female WT survivors with creatinine and cystatin C, calculated eGFR using the Chronic Kidney Disease-Epidemiology equations with and without cystatin C, and measured ^99mTc diethylenetriamine pentaacetic acid (DTPA) plasma clearance. WT survivors treated with unilateral nephrectomy (UN), non-nephrotoxic chemotherapy (NNC) and WART or treated with UN, no radiation therapy, and NNC were enrolled. Correlations between ^99mTc DTPA clearance and eGFR were calculated. Bias and the percentage of eGFR calculations that differed from the ^99mTc DTPA clearance by 10% or less (P₁₀) or 30% or less (P₃₀) (accuracy) were calculated.

Results: Among female WT survivors, none of the eGFR calculations was statistically significantly correlated with ^99mTc DTPA clearance. Among both unirradiated and WART-treated male WT survivors, ^99mTc DTPA clearance correlated well with eGFR calculations that included creatinine. eGFR calculations that included creatinine were positively biased among female participants compared to ^99mTc DTPA clearance, and no P₃₀ was greater than 90% among either irradiated males or females.

Conclusions: Among female survivors of unilateral, nonmetastatic, non-syndromic WT who have undergone UN, eGFR is poorly correlated with, is positively biased, and lacks sufficient accuracy, compared to ^99mTc DTPA clearance.

Background: Individuals with sickle cell disease (SCD) experience significant healthcare disparities; however, there is little known on the impact of psychosocial stressors and neighborhood disadvantage on preventive and acute care utilization in this population.

Procedure: A retrospective data collection was performed for all patients cared for at a comprehensive pediatric sickle cell center in the Midwest who had also completed the Psychosocial Assessment Tool (PAT), a validated caregiver-reported measurement of family psychosocial risk, from September 2021 through December 2022. Patient age, payor, primary language, frequency of acute and missed preventive care visits, as well as Area Deprivation Index (ADI) and Childhood Opportunity Index (COI) scores were collected for 256 patients.

Results: The average state ADI was 6.3 (±2.9), and the majority of patients were in the Very Low or Low COI categories. Total PAT score (p = 0.003), state ADI (p < 0.001), and state COI (p < 0.001) were all significantly correlated with missed SCD clinic visits, with increased odds of a missed visit with increasing neighborhood disadvantage (OR 1.22, p < 0.001). The odds of acute care were also increased in those with higher family psychosocial risk (OR 1.76, p < 0.011), though this was not seen with ADI or COI, suggesting an additional effect of family stressors and resilience on acute care utilization.

Conclusions: This highlights the importance of regular, universal social and psychological risk screening, as well as inclusion of psychosocial team members in SCD programs to identify and readily address risk factors that impact child outcomes in a minoritized chronic disease population.

Neuroblastoma is one of the most common tumors in young children, arising from the adrenal medulla or paraspinal sympathetic ganglia. We describe primitive round cell tumors presenting in three patients less than 1.5 years old, with striking clinical and pathologic similarities to neuroblastoma. Unlike neuroblastoma, however, these primitive tumors did not show specific histologic or immunophenotypic evidence of neuroblastic differentiation, and harbored a MN1::ZNF341 fusion. All patients progressed through neuroblastoma therapy and ultimately died of disease. These highly aggressive tumors mimicking neuroblastoma appear to be a novel and distinctive entity in need of further characterization.

We report two neurocutaneous melanocytosis (NCM) patients who required ventriculoperitoneal shunt placement and subsequently developed intraperitoneal melanoma. These patients with NCM are at an increased risk for developing NRAS-associated melanomas in the central nervous system, which in turn may lead to symptomatic hydrocephalus requiring cerebrospinal fluid diversion. Due to the rarity of NCM, current knowledge on disease progression and appropriate management is limited. Ongoing studies aiming to better understand this condition and inform its clinical management may help to identify risk factors for developing more severe complications.