Pediatric Blood & Cancer最新文献

Background/objectives: Pain is a prevalent late effect of cancer that can disrupt critical developmental milestones during young adulthood. This study examined the relationship between pain and psychosocial outcomes in young adult (YA) survivors of cancer.

Methods: One hundred YA cancer survivors completed measures assessing pain, anxiety, depression, posttraumatic stress, and alcohol use at baseline and follow-up (2-4 weeks later).

Results: In multivariate regression analyses, higher pain predicted poorer outcomes across all domains at follow-up (p's <.05).

Conclusions: Pain predicts psychosocial outcomes in YA cancer survivors, underscoring the need for routine assessment of and early intervention for pain to support psychological well-being.

The pediatric hematology-oncology fellowship training curriculum has not substantially changed since its inception. The first year of training is clinically focused, and the second and third years are devoted to scholarship. However, this current structure leaves many fellows less competitive in the current job market, resulting in approximately one-third of all pediatric hematology/oncology fellowship graduates pursuing subspecialty fellowship training in niche fields such as stem cell transplant, neuro-oncology, and hemostasis/thrombosis. In this article, we propose an individualized PHO fellowship curriculum to better prepare the future PHO physician for their future career, potentially abrogating the need for additional subspecialty training.

Children with cancer are at-risk for infertility, yet most desire biological parenthood. Fertility preservation is available for pre- and postpubertal patients, with tissues typically banked for autologous use. Some survivors require third-party reproduction (TPR) but do not complete the Food and Drug Administration (FDA)-required donor eligibility steps when banking their reproductive materials. This article reviews medical and nonmedical factors associated with TPR, FDA regulatory requirements, risks of non-compliance, and recommendations for facilitation in the pediatric oncology setting. Challenges, opportunities, and clinical decision trees for discussing TPR during pediatric fertility consultations are presented, emphasizing collaboration with adult reproductive clinics to support survivors' long-term reproductive goals.

Background: Long-term survival for high-risk neuroblastoma has increased from 40% to 60% by optimizing chemotherapy, surgery, radiation therapy, and the addition of anti-GD2 antibody therapy. However, the high cost of this antibody presents access issues globally. This study evaluates the availability, financial coverage, and barriers to accessing these therapies across diverse healthcare settings.

Methods: An online survey collected data on demographics, treatment practices, financial coverage, regulatory status, and barriers to anti-GD2 therapy. Fisher's exact test was used to compare categorical variables between high-income countries (HICs) and low- and middle-income countries (LMICs). A retrospective cost analysis estimated the financial burden of anti-GD2 therapies using standardized dosing for a representative 3-year-old patient.

Results: Responses came from 100 facilities in 80 cities and 59 countries. Anti-GD2 therapy for frontline maintenance was available in 93% of HIC centers, but only 21% of LMIC centers; 65% of LMIC centers reported no access, while the remainder had limited or irregular availability (p < 0.0001, Fisher's exact test). When unavailable, isotretinoin was most often used alone. Financial coverage differed significantly: HICs relied on government or insurance funding, while LMICs depended on out-of-pocket or non-profit support. Cost analysis showed a full treatment course would cost approximately $192,750 for dinutuximab, $142,695 for dinutuximab beta, and $610,800 for naxitamab, highlighting the substantial financial burden these therapies impose.

Conclusion: Global access to anti-GD2 therapy is highly unequal. Strategic measures, including negotiations for reduced drug costs, research-based access, and WHO Essential Medicines List inclusion, could help address these disparities.

Neurocognitive deficits in adult survivors of childhood cancer are well established, but less is known about developmental disorders (DD) arising shortly after cancer diagnosis. Using 2016-2019 linked Ohio cancer registry and Medicaid data, we compared DD among 324 children with cancer and 606,913 cancer-free controls. Pre-diagnosis, DD prevalence was similar (16% vs. 16.9%). However, post-diagnosis, children with cancer had over twice the risk of DD (ARR 2.09, 95% CI 1.55-2.83) across developmental domains. These findings reveal that DD emerges soon after cancer diagnosis, potentially related to treatment and/or secondary toxicities and highlight the need for early screening and rehabilitative intervention.

Background: B-acute lymphoblastic leukemia (B-ALL) is the most common pediatric cancer, and while most children in high-resource settings are cured, therapy carries risks for long-term toxicities. Understanding parents' concerns about these late effects is essential to guide anticipatory support and inform evolving therapeutic approaches.

Procedure: In partnership with the national patient advocacy organization, Momcology, parents of children diagnosed with ALL between the ages 1 and 19 years completed an online survey. Parents selected up to five concerning potential late effects, rated the level of intensity of concern, and provided open-text responses. Quantitative analyses examined associations with child and parent factors, and qualitative responses were analyzed using directed content analysis.

Results: The survey was completed by 442 parents of children with B-ALL. The most frequently cited concerns were neurocognitive deficits (55%), secondary cancers (49%), mental health changes (36%), cardiotoxicity (31%), and infertility (30%). Concern for neurocognitive deficits persisted during and after therapy and was of greater concern among parents of younger children compared with older children (58% vs. 41%; p = 0.006). Concerns about neurocognitive effects correlated with concern for mental health and social changes (RR 1.86, 95% CI 1.40-2.47, RR 1.43, 1.03-1.98). Open-text responses for all late effects of concern indicated enduring worry and impacts on family anxiety, daily life, and children's future opportunities.

Conclusions: Parents of children with B-ALL report substantial, persistent concern about late effects, particularly neurocognitive impairment. Findings underscore the need for information and guidance for parents of children with ALL about late effects and the importance of integration of parent perspectives in defining ALL research priorities.

Background: Pediatric hematopoietic stem cell transplantation (HSCT) is a life-saving therapy for malignant and non-malignant hematologic diseases. However, access to this high-complexity treatment remains limited and uneven across the Andean subregion. This study aimed to evaluate the current status, capacity, and equity of pediatric HSCT programs in six Andean countries (Bolivia, Chile, Colombia, Ecuador, Peru, and Venezuela) within the framework of WHO Global Initiative of Childhood Cancer.

Methods: A mixed-methods, cross-sectional situational assessment was conducted between November 2024 and January 2025 under the coordination of the Andean Health Organization (ORAS-CONHU) and the Pan American Health Organization (PAHO). Standardized national and center-level surveys (34 and 150 items, respectively) were applied to Ministries of Health and HSCT centers to collect data on infrastructure, workforce, financing, quality management, and barriers to access. Quantitative data were analyzed descriptively and validated by national committees, complemented by qualitative interviews with key stakeholders.

Results: Twenty-seven HSCT centers were identified across the subregion, revealing major disparities in access and capacity. Chile reached 100% coverage of estimated transplant needs, whereas Colombia, Peru, and Venezuela achieved 80%, 29%, and 42.5%, respectively. Bolivia and Ecuador had the lowest coverage (1.6% and 24%). Only 28% of units were pediatric-exclusive, and less than two-thirds met full quality and training standards. High-cost medicines, limited infrastructure, and shortages of specialized personnel were identified as critical barriers, whereas regional collaboration and national policies emerged as key facilitators.

Conclusion: The study highlights significant inequities in pediatric HSCT access and capacity across the Andean subregion. Strengthening infrastructure, workforce training, financing mechanisms, and data systems, supported by sustained regional cooperation, is essential to achieve equitable, high-quality transplant care for all children.

Background: Blinatumomab has transformed the treatment of pediatric B-acute lymphoblastic leukemia (B-ALL). However, it presents distinct operational challenges for administration given it is delivered as a continuous infusion and has unique toxicities. The objective of this project was to develop, implement, and evaluate a standardized institutional protocol for blinatumomab administration to optimize safety, efficiency, and patient experience.

Procedure: This quality improvement (QI) initiative was conducted at The Hospital for Sick Children, Toronto, Canada. A multidisciplinary Blinatumomab Working Group developed harmonized standards across five domains: (1) caregiver education, (2) fever management, (3) nursing-led assessments, (4) infusion interruption management, and (5) use of 7-day infusion bags. Patients receiving blinatumomab for upfront therapy or for relapsed disease between July 2024 and August 2025 were included. Demographic and clinical data were extracted from the institutional data warehouse. Outcomes included rates of caregiver education completion, antibiotic use during blinatumomab initiation, and completion of nursing-led assessments.

Results: Fifty-three patients received standard of care blinatumomab. All eligible caregivers (100%) completed standardized education. The rate of empiric antibiotic use during blinatumomab initiation decreased from 68% to 22% following adoption of selective antibiotic initiation without increased readmission, or prolonged stay. Nursing assessment completion was 95% for inpatients and 81% for outpatients. Infusion interruptions were managed using a standardized, risk-based algorithm without observed safety events.

Conclusion: Implementation of multidisciplinary, standardized blinatumomab administration protocols was feasible and safe. These processes improved antibiotic stewardship, empowered nursing-led care, and reduced practice variability. This framework may inform best practices for the safe and efficient delivery of blinatumomab in other pediatric oncology centers.