Pediatric Blood & Cancer最新文献

Introduction: Sickle cell disease (SCD) is frequently associated with cardiovascular complications, with cardiac involvement being evident in the electrocardiogram (ECG). This study aimed to systematically research the literature to investigate factors associated with ECG abnormalities in SCD patients.

Method: Five online databases of PubMed, Scopus, Web of Science, Embase, and Google Scholar were reviewed using a broad search strategy to identify original studies reporting factors associated with abnormal ECG findings among patients with SCD. Using Comprehensive Meta-Analysis software, various effect sizes-odds ratios (ORs), mean differences, and correlation coefficients-were analyzed, pooled with a random effects model, and visualized through forest plots.

Results: Data analysis from 23 studies covering 2943 SCD patients revealed increased odds of prolonged QTc interval (2.54, 95% CI = 1.34-4.83), ST segment elevation (9.23, 95% CI = 3.15-27.07), and ST segment depression (3.82, 95% CI = 1.99-7.33) during crises. Patients with moderate SCD severity had higher odds of having any ECG abnormalities compared to those with mild severity (2.80, 95% CI = 1.47-5.33). No significant associations were observed between moderate and mild severity for left ventricular hypertrophy, sinus arrhythmia, and sinus tachycardia, or between male and female for having any ECG abnormalities, left ventricular hypertrophy, and prolonged QTc interval. Additionally, mean hemoglobin level was 0.60 mg/dL lower (95% CI = -0.91 to 0.28) in those with prolonged QTc interval.

Conclusion: This study highlights the link between SCD status and severity, and hemoglobin, and ECG abnormalities. It emphasizes the need for cardiac monitoring, caution with QTc-prolonging medications, and routine ECG assessments to prevent cardiovascular complications in these high-risk patients.

A better understanding of positron emission tomography (PET) response in nodular lymphocyte-predominant Hodgkin lymphoma (nLPHL) is critical for incorporating PET into prospective trials. PET scans from Children's Oncology Group study AHOD03P1 for patients less than 22 years with low-risk nLPHL, treated with three cycles of doxorubicin, vincristine, prednisone, and cyclophosphamide chemotherapy, were retrospectively reviewed and assigned Deauville 5-point scale (5PS) scores. Five-year post-PET event-free survival was 90.1% (80% CI: 85.2%-93.4%) for PET-negative (5PS 1-3) and 66.7% (80% CI: 36.4%-85.0%) for PET-positive (5PS 4-5) patients. PET response after three cycles of low-dose chemotherapy is predictive of relapse risk for low-risk nLPHL.

Background: Children with sickle cell disease (SCD) require comprehensive care to prevent and treat serious and life-threatening complications and to access disease-specific treatment approaches that can improve outcomes. This study characterized barriers and facilitators to care for SCD in the context of the Conceptual Framework of Access to Care Model.

Methods: This qualitative descriptive study was conducted using semi-structured interviews with 27 patient/caregivers focused on sickle cell anemia (SCA; a subtype of SCD). Data were analyzed using directed content analysis with the model above as the initial coding framework.

Results: Themes were identified among healthcare system and patient/community-level factors. Healthcare system facilitators predominated themes, with a focus on the extent to which the healthcare services provided were a good match for the family and available and accommodating to patient and family needs. Additional facilitators at the patient/community level focused on whether patients and families could perceive and seek out, reach and pay for, and engage with healthcare. Barriers reflected the opposite experiences, with negative or challenging healthcare experiences and adverse social determinants of health interfering with access to care.

Conclusions: Barriers and facilitators were mapped to the Conceptual Framework of Access to Care Model, with facilitators playing a more substantial role than barriers in access to comprehensive care among children with SCA and their caregivers. A focus on optimizing facilitators at both the healthcare system and patient/family level may have a considerable impact on improving access to and engagement in care.

Background: In contrast to adult salivary gland tumors, our institutional guidelines utilize lower radiation doses, smaller target margins, and proton therapy (PT) to minimize radiation to children's developing skull base anatomy. Herein, we report outcomes of our pediatric management approach.

Procedures: We identified 31 pediatric patients with salivary gland tumors treated with PT at our institution between 2006 and 2023. Most common histologies were mucoepidermoid carcinoma (n = 14), acinic cell carcinoma (n = 5), and adenoid cystic carcinoma (n = 5). Eight patients with radiographic lymphadenopathy underwent neck dissection prior to presentation, with selective dissections involving <4 nodal levels in 7/8 cases. The gross tumor volume (GTV) encompassed the residual tumor and tumor bed. Clinical target volume (CTV1), defined as GTV + 1 cm margin, received 50.4 Gy. Prophylactic nodal irradiation was administered to three patients. CTV2 equaled the GTV and was treated to a median total dose of 64.8 Gy (range, 61.2-70.8). Toxicity was assessed with CTCAE Version 5.0.

Results: Median follow-up was 7.5 years (range, 1.2-15). Overall survival (OS) was 96%, and local control was 94%. There were no nodal recurrences. Acute toxicities were limited to mucositis requiring opioids and transient Grade 3 dermatitis. The most serious late toxicity involved the auditory system, including three patients requiring hearing aids, one with chronic otitis media, one with osteonecrosis of the mastoid, and one with external canal stenosis. There was neither Grade 4 toxicity nor second malignancy.

Conclusions: Conservative neck management and moderate-dose PT delivered to smaller volumes resulted in excellent long-term disease control and limited toxicity in children with common salivary gland tumors.

Background: Localized lymphoblastic lymphoma (LL) is rare in pediatric patients. The best treatment for patients with localized LL remains to be determined because of the rarity of the disease.

Methods: Between November 2004 and October 2019, 41 newly diagnosed patients up to 18 years of age with localized LL (Murphy stages I and II) were enrolled in the LLB-NHL03 trial. The treatment consisted of five phases: induction, consolidation, central nervous system prophylaxis, delayed intensification, and maintenance. The total duration of therapy was 24 months from the time of diagnosis.

Results: Of the 41 patients, six patients were excluded with a different diagnosis; therefore, 35 were included in the primary efficacy and safety analysis. The mean age at diagnosis was 9.2 years (range 2.1-16.1 years). Sixty-five percent of patients were male. Twenty-nine patients had a pre-B immunophenotype, and six had a pre-T immunophenotype. The head and neck area accounted for 66% of the primary sites. At a median follow-up of more than 10 years, the 3-year event-free survival rate [95% confidence interval] was 97.1% [81.4-99.6%], and the 3-year overall survival rate was 100%. Overall, patients tolerated the therapy well, and no treatment-related deaths were observed.

Conclusion: This is the largest clinical trial conducted exclusively in children with newly diagnosed localized stage LL. The outcomes of pediatric patients with localized LL treated with 2 years of acute lymphoblastic leukemia-type therapy, which is characterized by a relatively low anthracycline dose, exclusive use of prednisolone steroids, and fewer intrathecal therapies compared with previous studies, were excellent.

Clinical trial registration number: This trial was registered in the Japan Registry of Clinical Trials (jRCT): jRCTs041180131.

Background: Management of neonatal portal vein thrombosis (PVT), a relatively common type of pediatric deep vein thrombosis, is not completely standardized. Questions remain about the benefit of anticoagulation (ATC) therapy and about the optimal frequency and duration of doppler ultrasound (US) surveillance for liver complications such as portal hypertension and gastrointestinal bleeding. Current guidelines suggest reserving ATC only for occlusive PVT, highlighting a need for explicit grading of PVT in radiologic reports and a consensus approach to imaging and management.

Methods: To address these issues, we implemented an institutional Neonatal PVT Management Algorithm using plan-do-study-act (PDSA) methodology. We aimed to standardize screening tests, reduce unnecessary ATC, and optimize imaging checkpoints. A five-year retrospective review established baseline data, which we compared to outcomes five years post-implementation of the algorithm.

Results: The algorithm recommended ATC only for occlusive PVT and advised US imaging at Week 1, Month 1, Month 3, and Month 6 from diagnosis, with annual surveillance for unresolved or abnormal cases. Post-implementation analysis revealed improvements in radiologic documentation of PVT grading, a reduction in the use of ATC for subocclusive PVT, and a decrease in the median duration of ATC for all patients. Follow-up imaging adherence did not improve between the pre- and post-implementation periods.

Conclusions: The algorithm successfully enhanced documentation of PVT grading and reduced unnecessary ATC but highlighted persistent challenges in follow-up adherence, suggesting a need for further refinement in future PDSA cycles.