Pediatric Blood & Cancer最新文献

Purpose: Early-Phase clinical trials in pediatric oncology are designed to test drug safety and feasibility, offering minimal direct therapeutic benefit (5%-10%) while carrying a risk of toxicity, death, and worsened symptom burden. Pediatric palliative care (PPC) provides supportive care to patients and families enrolling in Phase 1 trials; however, oncologists inconsistently refer patients for PPC. This study aimed to explore potential benefits and ideal timepoints for integrating PPC for pediatric oncology patients participating in Phase 1 trials.

Methods: Semi-Structured qualitative interviews were conducted with parents of patients enrolling in Phase 1 trials at two academic cancer centers. Interviews were audio-recorded, transcribed, and analyzed using inductive content analysis.

Results: Parents of 31 patients were interviewed. Approximately 61% of parents (n = 19) were familiar with PPC, of whom a majority (68%, n = 13) defined PPC to mean end-of-life/hospice. Fewer than one third (30%, n = 9) described the evolution of this definition to include comfort, quality of life, and extra support. Most parents (97%, n = 30) supported early integration of PPC, with 87% (n = 27) identifying enrollment on a Phase 1 study as an ideal timepoint for PPC referral. Parents described PPC as helpful with symptom management, care coordination, and general support and advocated for clinicians to introduce PPC by emphasizing these benefits.

Conclusions: Parents identified enrollment in a Phase 1 study as an ideal timepoint for PPC referral, highlighting benefits from PPC subspecialty support. Future research will assess the impact of the implementation of clinical interventions to promote PPC consultation in pediatric cancer Phase 1 trial populations.

Purpose: To investigate the patterns of recurrence/metastasis and the clinical value of radiotherapy in local control for pediatric pancreatoblastoma.

Materials and methods: A retrospective analysis was conducted on 14 pediatric patients with pathologically confirmed pancreatoblastoma treated at our institution from June 2017 to June 2024. Clinical data, including baseline characteristics, surgical approaches, pathological staging, adjuvant therapies (chemotherapy/radiotherapy), recurrence/metastasis patterns, and subsequent interventions, were systematically collected. The impact of radiotherapy on local control was evaluated, with survival analysis performed using Kaplan-Meier methods, and prognostic factors analyzed via log-rank tests and Cox regression models.

Results: The median age of the entire cohort was 7 years (range, 3-13 years), with 4 cases of pancreatic head tumors and 10 cases of pancreatic body/tail tumors. At initial diagnosis, 57.1% (8/14) presented with regional lymph node metastasis, and 57.1% (8/14) had distant metastasis. The R0 resection rate during the first surgery was 57.1% (8/14), while R1/R2 resections accounted for 28.6% (4/14); 2 did not undergo surgery. With a median follow-up of 31 months, the overall survival rate was 78.6% (11/14). The recurrence/metastasis rate was 64.2% (9/14), with predominant patterns including tumor bed recurrence (3/9, 33.3%), regional lymph node metastasis (3/9, 66.7%), and liver metastasis (5/9, 55.6%). Multimodal therapies encompassed chemotherapy, secondary surgery, liver transplantation, and radiotherapy for metastatic lesions. In the radiotherapy group, the 1-year and 2-year local control rates were 100% and 88%, respectively. Log-rank test and Cox analysis identified failure to achieve R0 resection and regional lymph node metastasis as independent prognostic factors for inferior overall survival (P < 0.05). Other factors-including age, gender, presence of initial metastasis, initial liver/lung metastasis, number of recurrence/metastasis events, and radiotherapy-showed no significant correlation with overall survival.

Conclusion: Regional lymph node metastasis and failure to achieve R0 resection are critical prognostic factors affecting long-term survival in pancreatoblastoma patients. Adjuvant radiotherapy significantly improves local control rates and may enhance survival outcomes in patients with positive margins or lymph node metastasis by strengthening local disease control, warranting further validation in prospective studies.

Background: Adolescents with cancer have unique communication needs from other patients with cancer and adolescents without cancer. Electronic health record (EHR) patient portals offer opportunities for communication and health management, but pose challenges in balancing transparency, privacy, and health management. The 21st Century Cures Act amplified these challenges by increasing the availability of health records in patient portals. To inform portal use and policies, we analyzed advice from adolescents with cancer, their parents, and oncology clinicians regarding portal access and use.

Methods: We purposefully sampled adolescents aged 12-17 with cancer and their parents from large academic pediatric oncology centers and used convenience and snowball sampling to recruit clinicians from a national professional group. We conducted semi-structured interviews, descriptively coded transcripts to generate preliminary codes, categorized themes, and applied them across transcripts.

Results: We interviewed 38 adolescent-parent dyads and 53 clinicians. Interviews revealed agreement and tensions among stakeholders. Participants recommended a standard introduction of the portal, portal modifications to improve users' experiences, and encouraging portal use for health management and communication while maintaining patient access to clinicians. Adolescents and parents suggested clinicians modify notes to have a more hopeful tone. Clinicians favored delayed release of results, while parents preferred immediate release. Adolescents and clinicians encouraged adolescent control over parental proxy access, while parents favored default parental access.

Conclusion: This qualitative study provides actionable recommendations for improving portal use, policy, and design for adolescents with cancer. Implementation of these findings and future research can optimize adolescent and caregiver engagement with the portal and portal usability.

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.

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: The survival outcomes among children with acute myeloid leukemia (AML) in low- and middle-income countries are still poor despite adopting modern treatment regimens from developed countries. The study aimed to identify additional potential determinant factors for relapse and death among children with AML in Thailand.

Methods: In all, data from 282 children newly diagnosed with AML between 2015 and 2019 across Thailand were retrospectively reviewed. Data, including initial white blood cell numbers, genetic analysis, post-induction minimal residual disease (MRD), hematopoietic stem cell transplantation, and supportive care, were analyzed.

Results: The probability of 5-year, event-free survival, overall survival, and cumulative incidence of relapse were 40.5%, 42.3%, and 47.4%, respectively. The risk of death was significantly increased among patients stratified as high-risk AML with an adjusted hazard ratio (HR) of 1.8 (95% confidence interval [CI]: 1.1-2.9, p = 0.01). The accessibility to MRD was significantly associated with the risk of death with an adjusted HR of 1.7 (95% CI: 1.1-2.5, p = 0.01). Patients with low-risk AML did carry a significant risk for both death, with an adjusted HR of 1.8 (95% CI: 1.1-3.0, p = 0.02), and relapse, with an adjusted HR of 2.6 (95% CI: 1.5-4.7, p < 0.001) for initial WBC greater than 100,000/mm³.

Conclusion: Elevated initial WBC numbers and accessibility to MRD could be considered additional risk factors for unfavorable outcomes in childhood AML.