Pediatric Blood & Cancer最新文献

Background

Immune thrombocytopenic purpura (ITP), which poses challenges in treatment response, is an autoimmune-mediated bleeding disorder with an extremely complex pathogenesis and unpredictable clinical progression. Dysregulation of apoptotic pathways may influence both the pathogenesis and prognosis of ITP. This study aimed to evaluate the expression patterns of the apoptotic protein insulin-like growth factor-binding protein 2 (IGFBP2) and the anti-apoptotic protein B-cell lymphoma 2 (BCL2) as potential predictive or prognostic biomarkers for disease progression in childhood ITP.

Patients and Methods

The expression levels of BCL2 and IGFBP2 were assessed in peripheral blood samples from 40 pediatric ITP patients and 30 age- and sex-matched healthy controls using enzyme-linked immunosorbent assays.

Results

Plasma levels of BCL2 and IGFBP2 were higher in ITP patients than in control subjects. Although the difference in IGFBP2 expression was not statistically significant (p = 0.910), BCL2 expression was significantly elevated (p < 0.001). Notably, chronic ITP patients had significantly lower levels of both IGFBP2 and BCL2 markers compared to patients who achieved spontaneous recovery (p < 0.001).

Conclusion

BCL2 and IGFBP2 appear to be promising noninvasive biomarkers for predicting disease outcomes in newly diagnosed ITP, emphasizing the need for validation in large-scale, multicenter longitudinal studies.

Introduction

Knowledge of the guidelines for treating people with bleeding disorders (PwBD) is vital for emergency department (ED) staff, as PwBD rely on the ED for treatment of acute bleeding episodes or injuries. Unfamiliarity with these guidelines can delay treatment. We aimed to improve the timely treatment of PwBD seeking emergency care by reducing the mean time-to-factor administration in the ED by 20%, from the baseline of 110 to 88 minutes.

Methods

Our quality improvement (QI) initiative (2019–2024) used Plan-Do-Study-Analyze cycles. Weekly electronic health record (EHR) reports tracked PwBD's time of arrival, admission, clotting factor concentrate ordering, and administration. Interventions targeted delays identified in the process.

Results

Baseline data showed a 110-mean time-time-to-factor. Our aim of reducing the time-to-factor by 20% from the baseline of 110 minutes was reached in May 2020. This improvement was exceeded and sustained throughout the project. At the conclusion, we achieved a 62% reduction in time-to-factor and a mean time-to-factor of 42 minutes.

Conclusion

By using an EHR tool, we were able to identify when during the ED visit process delays occurred, which helped us better target our interventions. Our educational-based interventions were adapted to reach our intended audiences while working around barriers related to the COVID-19 pandemic, such as high patient volumes and staff shortages. Our results show a decrease in time to factor in the ED and an increase in the PwBD receiving factor within 60 minutes of arrival time. This has been a sustainable improvement.

Introduction

Studies on the use of image-guided percutaneous biopsy for hepatoblastoma (HB), and recommendations put forth by the pediatric hepatic international tumor trial (PHITT), are limited. It is unknown if sufficient tissue can be obtained for trial enrollment as well as molecular profiling, which will likely play a key role in informing future treatment strategies.

Methods and Materials

Patients with HB who underwent percutaneous biopsy at initial diagnosis in interventional radiology (IR) over a 12-year period at a single center were included. Patient demographics, pretreatment extent of disease (PRETEXT) stage, tumor size, and procedure details were collected. Pathology reports and tumor genomic analysis, when performed, were assessed for specimen adequacy. Post-procedure records were assessed for hemoperitoneum.

Results

A total of 33 percutaneous biopsies were performed on 32 patients [17 female; median age 1.3 years (IQR: 0.7–2.5 years); median weight 10.5 kg (IQR: 7.4–12.7 kg)]. Most (n = 27) had a single liver lesion, and most (n = 18) were PRETEXT II. A total of 15 were positive for at least one annotation factor. Median longest tumor axis was 9.3 cm (IQR: 5.0–13.5 cm). A total of 16 patients had concurrent non-targeted liver biopsy, per PHITT recommendations. An 18-gauge instrument was most commonly used (n = 24, 73%) with a median of 8 cores (IQR: 6–12) obtained. There were no instances of hemoperitoneum.

Tissue was adequate for histologic diagnosis in 97% (n = 32), with histologic subtyping obtained in 94% (30/32). When available (n = 29), comparison with the subsequent surgical resection specimen showed subtype concordance in 15 (52%) patients and minor variations secondary to sampling or treatment effect in 14 patients. Molecular profiling was completed on 21/21 specimens (100%), with 19/21 (90%) showing potentially clinically significant variants, most commonly in CTNNB1 (16/21).

Conclusion

In this single-center study, percutaneous biopsy resulted in no serious adverse events, a high rate of diagnosis, and successful subtyping and molecular characterization of HB.

Background

Diffuse intrinsic pontine glioma (DIPG) is a challenging pediatric tumor that frequently progresses within the first year following local radiotherapy. However, several small studies have suggested that re-irradiation may improve quality of life and extend overall survival.

Patients and Methods

This retrospective study included 109 children who experienced disease progression ≥3 months after their initial radiotherapy, and subsequently received re-irradiation at a single institution. These patients were compared with a cohort of 60 children, meeting the same criteria, who were treated before adopting the re-irradiation policy and received only the best supportive care (BSC). Most of the re-irradiated children (94%) received first radiation dose as hypofractionation (39 Gy/13 fractions).

Results

The re-irradiation group demonstrated significantly higher overall survival (OS) rates after the first progression, with a 6-month OS of 42% (95% CI: 34%–53%) compared to 16% (95% CI: 8.9%–32%) in the BSC group (p < 0.001). Re-irradiation reduced the hazard of death by more than half (HR = 0.45, p < 0.001). Clinical response (p < 0.001) and radiological response (p = 0.016) were significant predictors of improved survival. While the time from initial radiotherapy to progression (p = 0.059) and higher re-irradiation doses (p = 0.074) were associated with improved OS, these factors did not reach statistical significance but may represent potential prognostic indicators.

Conclusion

Re-irradiation improved the OS in children with progression of DIPG and alleviated their signs and symptoms.

Background

Although psychosocial support for siblings of youth with cancer is a standard of care, what sibling supportive services should entail remains unclear. Given limited resources for sibling care, establishing clinical and research priorities may guide resource allocation toward supports perceived as holding the greatest potential benefit. The current study used a two-round, value-weighting approach to identify priorities for sibling support services.

Procedure

Participants were recruited from a group of sibling experts (clinicians, researchers, community program leaders, and adults who had a sibling with childhood cancer) invited to attend an international sibling summit. In Round 1, 27 participants provided feedback on a list of potential priorities for sibling psychosocial support. In Round 2, 30 participants completed a web-based value-weighting questionnaire indicating how they would allocate 100 units of hypothetical funding among various priorities and qualitatively described the rationale for their decisions.

Results

Funding allocations generally averaged out across participants, highlighting the need for investments across all domains of sibling support. Participants allocated the greatest proportion of hypothetical funding to community-based sibling supports, which they perceived as more accessible to siblings than hospital-based supports. Participants allocated a particularly high level of funding to sibling supports in local schools. Within sibling subpopulations, bereaved siblings, siblings during active cancer treatment, and siblings with more adverse social determinants of health were allocated the largest proportion of funds.

Conclusions

Sibling-focused researchers, clinicians, program leaders, and adult siblings endorse broad investments in sibling support. Investments in community-based supports particularly may improve access to sibling support services.

Background

Children treated for pediatric cancer are at risk for cognitive late effects, as well as impairments in sleep and physical activity. The aim of the present study was to examine the psychometric properties of clinical screening of child- and caregiver-reported cognitive functioning, sleep, and physical activity and the relationship between cognitive functioning and health behaviors within a pediatric oncology long-term follow-up clinic.

Procedure

The study included a retrospective chart review of 99 caregivers and 80 children (8–17 years old) who completed the Conners Short Form (parent only) and PROMIS cognitive functioning, sleep-related impairment, sleep disturbance, and physical activity scales at the child's annual long-term follow-up visit. Test statistics and T-tests were used to assess psychometrics of the PROMIS scales. Bivariate correlations were used to examine the relationship between cognitive function and health behaviors.

Results

The child- and parent-report, short-version PROMIS cognitive functioning, sleep, and physical activity scales demonstrated high internal consistency and inter-rater reliability. High convergent validity was observed between PROMIS cognitive functioning and Conners Short Form. Caregiver- and child-reported cognitive functioning and health behaviors were significantly related (p ≤ 0.042).

Conclusions

The PROMIS short-version scales are reliable and valid measures for screening cognitive function and health behaviors in pediatric oncology long-term follow-up care. Further research examining the predictive validity and longitudinal utility of the PROMIS scales in survivors is warranted. There was a positive association between cognitive functioning and health behaviors in survivors, warranting further investigation to inform potential targets of intervention in long-term follow-up care.