Pediatric Blood & Cancer最新文献

Background: Precision in surgical documentation is crucial for minimizing communication discrepancies and preventing errors in patient care. Synoptic operative reports (SR) offer greater accuracy than narrative operative reports (NR); however, their adoption in pediatric surgical oncology remains limited. To enhance documentation practices, a standardized operative report was developed. This study assessed its effectiveness through a two-phase approach. Phase 1 retrospectively evaluated the completeness of NR for pediatric patients undergoing solid tumor resection at major pediatric cancer centers across diverse regions and resource settings. In Phase 2, following the implementation of SR at these institutions, we prospectively analyzed the completeness of operative documentation.

Methods: We conducted a multi-regional pre- and post-implementation study in five pediatric oncology reference centers. Eligible cases included primary malignant solid tumor resections. Pulmonary or other metastatic resections, benign tumors, and biopsies were excluded. Phase 1 consisted of a retrospective review of NR over 6 months in 2023. Phase 2 prospectively evaluated SR over 6 months post-implementation.

Results: A total of 165 operative reports were analyzed (73 NR, 92 SR). SR demonstrated significantly higher completeness in documenting key intraoperative elements, including completeness of resection, locoregional spread, tumor spillage, vascular involvement, lymph node sampling, and specimen naming. End-user surveys (n = 17) showed that 94% agreed SR captured key intraoperative details and improved documentation clarity. Seventy-seven percent agreed SR supported treatment decision-making, 71% agreed they enhanced communication during multidisciplinary team (MDT) meetings, and 82% agreed SR improved workflow efficiency. The mean System Usability Scale score was 81.1, reflecting excellent usability.

Conclusions: SR enhances completeness, data extraction, and communication compared to NR. Their integration into pediatric oncology practice may improve both documentation quality and multidisciplinary care.

Background: Despite the excellent outcomes achieved for pediatric mature B-cell non-Hodgkin lymphoma (MB-NHL) in high-income countries, outcomes remain very poor in low- and middle-income countries. High-dose methotrexate (HD-MTX), which is highly efficacious for the disease, is still not commonly used in low-resource settings due to the potential for treatment-associated toxicity. Though there have been more recent reports of successful use of HD-MTX in resource-limited settings, there are no reports to date regarding the methodology used to safely introduce HD-MTX in these environments. Subsequently, at our treatment center in northern Tanzania, we endeavored to utilize implementation science methodology to elucidate generalizable methods to allow for a safe introduction of HD-MTX in a resource-limited setting.

Procedure: The Active Implementation Frameworks were utilized as a starting point to generate an initial implementation plan. Intervention fit was assessed, and resource mapping and cost analysis were completed. Key stakeholders were identified and engaged. Novel educational strategies, decision support tools, and protocol fidelity monitoring systems were developed.

Results: Following completion of all planning and education activities, use of a new HD-MTX containing MB-NHL protocol began in October of 2024. As of June 2025, 27 cycles of HD-MTX have been given to a cumulative total of 10 patients. Few protocol deviations have occurred, and only one Grade III non-hematologic adverse event has been reported.

Conclusions: The use of implementation science methodology allowed a careful introduction of the use of HD-MTX for the treatment of pediatric MB-NHL in a resource-limited setting.

Background: Pediatric patients with extracranial solid tumors (ST) receiving chemotherapy are at an increased risk for Pneumocystis jirovecii pneumonia (PJP). However, evidence guiding prophylaxis practices in this population is limited. A PJP-related fatality at our institution highlighted inconsistent prescribing approaches and concerns about prophylaxis-related toxicity, prompting the development of a quality improvement (QI) initiative to standardize PJP prophylaxis for patients with ST.

Procedure: Our quality improvement (QI) initiative aimed to increase PJP prophylaxis initiation rates in pediatric patients (0-18 years) with newly diagnosed extracranial ST (excluding osteosarcoma) to over 90%, without associated chemotherapy delays or significant prophylaxis-related toxicity. The initiative was designed using the Model for Improvement with monthly Plan-Do-Study-Act cycles. Interventions included regular stakeholder engagement, education, and electronic health record (EHR)-integrated prophylaxis prescriptions. Balancing measures, including discontinuation rates of prophylaxis, related toxicity, and chemotherapy delays, were assessed through clinician surveys and chart review. Kotter's 8-Step Change Model secured engagement during planning and implementation of this 6-month (April-September 2024) initiative across inpatient and outpatient settings in a tertiary oncology center.

Results: PJP prophylaxis was initiated in 100% (n = 20) of eligible patients, an 88.7% relative improvement in coverage, compared to initiation rates of 53% in a historical cohort (n = 16/30). No chemotherapy delays or discontinuations of prophylaxis due to toxicity were reported.

Conclusion: A universal approach to PJP prophylaxis can be safely and effectively implemented in pediatric patients with ST without significant toxicity. Our experience highlights how change management models can effectively support the implementation of QI initiatives in pediatric oncology.

Background: Family rules and routines are modifiable behaviors that may help children cope with stress, yet their role in pediatric cancer remains understudied. This longitudinal study examined links between psychosocial risk, family rules and routines, and child emotional and behavioral health during the first year of pediatric cancer treatment. We hypothesized that family rules and routines would mediate the impact of psychosocial risk on child internalizing and externalizing symptoms.

Method: Eighty (N = 80) primary caregivers of youth ages 2-14 years (M = 7.9 years, SD = 3.9 years) with a new diagnosis of cancer reported demographics, psychosocial risk, frequency of engagement in family rules and routines, and child emotional and behavioral health symptoms across three time points after diagnosis: T1, 1-3 months; T2, 6-7 months; and T3, 12-13 months. Treatment intensity was abstracted from the medical record at T3. Longitudinal mediation models were fit to evaluate the impact of psychosocial risk at T1 on subsequent family rules and routines at T2, and child internalizing and externalizing symptoms at T3.

Results: Family rules and routines partially mediated relations between T1 psychosocial risk and T3 child externalizing symptoms, such that families at higher levels of psychosocial risk also exhibited lower engagement in family rules and routines, leading to more child externalizing symptoms. Family rules and routines did not mediate the association between T1 psychological risk and T3 child internalizing symptoms.

Conclusions: Psychosocial risk factors may disrupt family behaviors during cancer treatment, leading to more child externalizing concerns. Family based interventions should consider integrating support for family rules and routines during cancer treatment to mitigate behavioral risk.

Ongoing evidence indicates increased risk of sarcopenic obesity among children and young people (CYP) with acute lymphoblastic leukemia (ALL), often beginning early in treatment, persisting into survivorship. This review evaluates current literature on body composition in CYP with ALL during and after treatment. This study adhered to the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) guidelinesand was registered at the International Prospective Register of Systematic Reviews (PROSPERO) (CRD42023480732). Three databases (PubMed, MEDLINE (OVID), and CINAHL) were searched until March 2024. Studies with individuals aged 0-21 years with ALL during or after treatment were included. The Joanna Briggs Institute checklist was used to assess the bias risk. Of the 126 studies, 13 were included (eight cross-sectional and five prospective). Eight studies used dual-energy X-ray absorptiometry, three used bioelectrical impedance analysis, two used air-displacement plethysmography, and one applied the four-compartment model. Fat mass (FM) increased early (T2-T3 ≈ +1standard deviation score [SDS]), and remained elevated at treatment end, and was above reference at follow-up (T5 ≈ +0.7 SDS). Fat-free mass (FFM) declined during therapy (lowest at T4 ≈ -0.7 SDS) with partial recovery by T5 (confidence interval crossing 0). Body mass index was elevated in the ALL groups versus controls. Heterogeneity was substantial, reflecting variation in age, assessment timing, and methodology. Despite methodological limitations, this review demonstrates persistent increases in FM and a reduction in FFM during and after treatment. Large, international studies using standardized body composition methodologies and clinically relevant cut-offs are needed to define long-term risks.

Background: Quality of life (QOL) is impacted in children treated for leukemia. AALL0932 randomized reduction in vincristine/dexamethasone (VCR/DEX) pulses every 4 versus 12 weeks during maintenance in the average-risk subset of NCI standard risk B-ALL (NCI-SR AR B-ALL). We longitudinally assessed physical and emotional QOL, behavioral health, and school services by randomization.

Procedure: NCI-SR AR B-ALL English-speaking patients aged ≥4 years were evaluated at T1-T5 (∼2, 9, 18, 26 months [females treatment end], and 38 [males only] months from diagnosis) with parent-report. The Pediatric Quality of Life Inventory-4.0 and school services survey were administered longitudinally, and the Behavior Assessment Scale for Children-2 at therapy end.

Results: Data were obtained from 420 consented and randomized participants (mean 6.0±1.6 years, 45.7% female). Impairment among randomized participants at T2 and T4, respectively, was 11.3% and 12.5% for physical, and 12.2% and 9.8% for emotional function. In longitudinal models adjusting for race/ethnicity, time, and baseline impairment, pulse frequency was not associated with impairment (physical odds ratio [OR] = 0.9, 95% confidence interval [CI] = 0.5-1.8; emotional OR = 0.9, 95% CI = 0.5-1.7). T2 impairment was associated with increased risk of post-randomization impairment for physical (OR = 4.3, 95% CI: 1.9-9.9) and emotional (OR = 4.9, CI: 2.3-10.5) function. Approximately 73.8% reported one or more school-based service during treatment: special education/accommodations (67.6%) and physical/occupational therapy (8.8%). Depression (20%) and anxiety (19%) did not differ by pulse frequency.

Discussion: Children with NCI-SR AR B-ALL experience diminished QOL, despite reduced frequency of VCR/DEX maintenance pulses. Impairment begins early during ALL therapy and persists; interventions should commence early and continue throughout and after therapy.

Background: Childhood cancers remain a major cause of morbidity and mortality in low- and middle-income countries (LMICs), where nearly 90% of the world's children live. In 2018, the World Health Organization (WHO) and St Jude Children's Research Hospital launched the Global Initiative for Childhood Cancer (GICC) with the goal of increasing global survival to at least 60% by 2030. Achieving this requires a systematic approach to understanding country-level contexts to guide national planning.

Methods: The Pediatric Oncology Facility Integrated Local Evaluation (PrOFILE) tool was implemented in Cameroon in 2021 to evaluate the national context for childhood cancer care, alongside facility and financing assessments. This paper reports how the PrOFILE assessment informed planning and describes the translation of results into an actionable plan. Stakeholders used an impact-effort matrix to prioritize recommendations. A follow-up review was conducted in 2023 to document progress after two years. Diagnosis- and treatment-specific components of the assessment are being reported separately.

Results: The assessment and workshops yielded four overarching themes: (1) workforce development and training of physicians, nurses, supportive care staff, and palliative care providers; (2) strengthening diagnosis and treatment capacity including pathology, timeliness of solid tumor diagnosis, chemotherapy safety, and subspecialty access; (3) improving financial and social support systems including local fundraising and family support; and (4) enhancing patient outcomes through reduction of early deaths and upfront integration of palliative care. At two years, measurable progress had been achieved, particularly in workforce training, expansion of psychosocial and financial support, and integration of palliative care, although gaps remain in diagnostic capacity and government financing.

Conclusion: The PrOFILE tool facilitated a robust, data-driven prioritization process for childhood cancer care in Cameroon. Two years on, early gains are evident, though sustained efforts are required to address persisting diagnostic and financing barriers.

Introduction: This study examines iron overload and assesses the safety and efficacy of chelation therapy in patients with leukemia.

Methods: The medical records of 208 children with acute leukemia were retrospectively screened. The iron status at diagnosis, cumulative packed red blood cell (pRBC) volume, number of pRBC transfusions, and iron burden of the patients at the end of intravenous chemotherapy were compared between patients who were and were not given iron chelation treatment with deferasirox.

Results: A total of 193 patients with leukemia were enrolled in the study. The average age was 65.5 months, and 56% were male. The patients were grouped according to receiving chelation therapy. Forty-four patients (22.8%) received iron chelation therapy. High-risk patients needed significantly more chelation treatment (p < 0.001). The number of pRBC transfusions, cumulative pRBC volumes, and ferritin values at the beginning of maintenance therapy were significantly higher in patients who received chelation treatment (p < 0.001). The cut-off values for the predictivity of serum ferritin, cumulative pRBC volume, and number of pRBC transfusions in chelation need were determined as 1952 mcg/L, 145 mL/kg, and 12 times, respectively (0.933, 95% confidence interval [CI]: [0.887-0.964], p < 0.001; 0.942, 95% CI: [0.899-0.970], p < 0.001; 0.903, 95% CI: [0.853-0.941], p < 0.001, respectively). Deferasirox was effective and safe in reducing iron burden. Only mild-to-moderate adverse effects were observed.

Conclusion: Iron overload can develop in pediatric patients with leukemia and is associated with number of pRBC transfusions and cumulative pRBC transfusional volume.

Background: Families of children with cancer often incur substantial nonmedical costs for travel, lodging, and food when accessing specialized care. These costs can contribute to health-related financial strain, yet the relationship between distance to care and nonmedical costs remains poorly understood.

Procedure: We conducted a retrospective cohort study of families applying for support from Children's Cancer Partners of the Carolinas (CCPC) between August 2011 and March 2024. Eligible participants included families of children with cancer from North and South Carolina with complete data on zip code, treatment center, and reimbursed costs. Distance to the primary treatment center was calculated in miles and minutes. Outcomes included total nonmedical costs (USD) and the frequency, magnitude, and types of reimbursed costs.

Results: Among 1890 children, the mean age was 10.4 years (SD 5.4), most were male (56%), White (51%), and publicly insured (56%). The most common diagnosis was hematologic malignancy (49%). One-fifth (22%) lived in rural areas. Median nonmedical costs were $1094/family (interquartile range [IQR]: $320-2379), and represent costs of land travel, food, and leisure. Median travel distance was 37.8 miles [IQR: 21.3-71.4] or 46.9 min [IQR: 31.0-81.3] each way. After adjusting for potential confounders, each additional travel minute was associated with $9.14 higher nonmedical costs (95% confidence interval [CI]: 6.91-11.37), and each additional mile was associated with $8.85 higher costs (95% CI: 6.69-11.00).

Conclusions and relevance: Longer distance to cancer care is associated with increased total nonmedical costs. Additional resources may help reduce travel-related costs for families living far from pediatric cancer centers.