搜索结果：Journal of pediatric hematology/oncology

Research suggests that burnout and work-related quality of life are concerns for pediatric oncology professionals, with limited supportive resources. This study aimed to address this resource gap through an intervention promoting hope and targeting burnout and quality of life in this group. A single-arm pilot study of the intervention was conducted at Rady Children's Hospital San Diego. The intervention was delivered as a single-session workshop, followed by reinforcement through a mobile application. Feasibility was assessed through recruitment and retention metrics, application usage data, and informal participant acceptability feedback. Exploratory efficacy analyses were conducted on measures of hope and perceived control (intervention mechanisms), occupational burnout and professional quality of life (primary outcomes), and empathy (secondary outcome) at baseline and 1, 4, and 12 weeks post-intervention. Twenty participants were consented and 18 participated, with 13 (72%) completing 12-week surveys. A total of 15 participants (83%) opted to receive daily push notifications, but only 3 (20%) engaged in daily booster questions. Positive feedback suggested acceptability. Exploratory findings suggested changes in emotional exhaustion (baseline to 1 week, P = .02; baseline to 4 weeks, P = .01), a sub-construct of burnout, and secondary traumatic stress (baseline to 4 weeks, P = .01; baseline to 12-week scores, P = .01), a sub-construct of professional quality of life. The Hopetimize intervention was feasible for pediatric oncology health-care professionals, although daily application usage was limited. Exploratory findings suggested that a randomized controlled trial would be beneficial to delineate intervention effects on the proposed mechanisms and outcomes.

The impact of radiotherapy-induced lymphopenia on pediatric medulloblastoma outcomes remains debated, with some evidence suggesting a potential risk of tumor recurrence. This theory has not been explored in low-middle-income countries where other factors may have a greater influence on prognosis. We retrospectively reviewed the medical charts of all children 3 to 18 years of age, diagnosed with medulloblastoma at KHCC/Jordan between 2017 and 2023 and treated with upfront craniospinal irradiation (CSI). We reviewed their clinical and tumor characteristics, lymphocytes' count during and after radiotherapy, and outcome. We considered grade-3 lymphopenia (<0.2 to 0.5&#xd7;10 9 /L) as the cutoff number for survival analysis. Sixty-six patients were identified (59% male). The median age at diagnosis was 9 years (range: 4.8 to 17.9&#xa0;y). Thirty-eight tumors (58%) were subgrouped; most (n=15, 39%) were subgroup-4. Thirty-five patients (53%) with completely resected nonmetastatic tumors received 23.4&#xa0;Gy CSI with tumor bed boost. Twenty-nine patients (44%) received radiotherapy within 49 days from their tumor resection, and 91% finished radiotherapy within 51 days. Forty-one patients (62%) received dexamethasone during radiotherapy. Most patients (n=63, 96%) received vincristine during radiotherapy, and 3 received, in addition, carboplatin or etoposide. More than 50% of patients had grade &#x2265;3 lymphopenia starting from week 2 of radiotherapy and continued until week 6; however, this dropped to 6.1% at weeks 4 to 6 postradiation. With a median follow-up of 3.5 years (range: 0.9 to 7.6&#xa0;y), the 3-year RFS and OS were 75% and 86%, respectively. Gender, molecular subgroup, and lymphopenia grade &#x2265;3 at weeks 4, 5, or 4 to 6 weeks after radiotherapy, were not prognostic for PFS. Metastatic disease was associated with a nearly 4-fold increase in hazard of progression (HR 3.69, 95% CI: 0.978-13.915), with a trend of significance ( P -value 0.0539). In our cohort, grade &#x2265;3 lymphopenia during/after radiotherapy did not emerge as a significant prognostic factor for tumor recurrence. However, several confounding variables may have influenced this outcome. To precisely assess the impact of lymphopenia on pediatric medulloblastoma prognosis, larger-scale prospective studies with extended follow-up are required.

Chemotherapy for pediatric leukemia induces profound immunosuppression, resulting in waning protection against vaccine-preventable diseases. Despite high survival rates, standardized revaccination protocols for non-transplant survivors remain controversial. We aimed to assess the serologic immunity to measles, mumps, rubella, and varicella among pediatric leukemia survivors and to identify clinical and treatment-related determinants of immune persistence. In this retrospective cohort study, 192 pediatric leukemia survivors treated with non-HSCT chemotherapy at Phoenix Children's Hospital (2021 to 2023) underwent serologic testing for measles, mumps, rubella, and varicella IgG antibodies. Demographic, clinical, and immunization data were retrieved from institutional and state registries. Associations between patient characteristics and serologic immunity were analyzed using logistic regression. At a mean of 6.51 years following chemotherapy completion, the overall seropositivity rates were 37.0% for measles, 40.6% for mumps, 70.8% for rubella, and 24.5% for varicella. Survivors who received post-chemotherapy vaccination demonstrated significantly higher seropositivity for rubella (81.9% vs. 64.1%, P =0.004) and varicella (35.6% vs. 16.3%, P =0.005), with higher but non-significant seropositivity for measles and mumps. Low-risk B-cell acute lymphoblastic leukemia (ALL) patients exhibited higher immunity compared with high-risk patients, with significant differences observed for mumps (53.6% vs. 24.6%, P =0.0009) and rubella (81.2% vs. 66.7%, P =0.039). Moreover, a shorter interval from therapy completion was associated with improved rubella immunity (adjusted OR=0.753; 95% CI: 0.66-0.86). Post-chemotherapy revaccination was independently associated with increased odds of rubella (adjusted OR=4.89; 95% CI: 1.762-13.572) and varicella (adjusted OR=2.65; 95% CI: 1.124-6.26) immunity. Substantial attenuation of vaccine-derived immunity persists years after chemotherapy completion in pediatric leukemia survivors. Both treatment intensity and absence of post-chemotherapy revaccination contribute to impaired long-term humoral immunity. These findings support routine revaccinations for all non-transplant pediatric leukemia survivors and underscore the need for risk-adapted, individualized survivorship immunization strategies.

During the first months following diagnosis and treatment of childhood-onset craniopharyngioma, a substantial proportion of patients experience rapid and marked weight gain. This frequently progresses to severe hypothalamic obesity, which results from hypothalamic injury caused either by the tumor itself or by therapeutic interventions. Hypothalamic obesity should be regarded as one manifestation within the broader clinical spectrum of hypothalamic syndrome. Given the pivotal role of hypothalamic nuclei in maintaining physiological homeostasis, hypothalamic syndrome encompasses a wide range of disturbances, including hypothalamic-pituitary hormone deficiencies, disruption of circadian rhythms, impaired regulation of hunger, satiety, and thirst, as well as thermoregulatory dysfunction and cognitive, sleep-related, and psychosocial impairments. Consequently, affected individuals often develop persistent obesity, chronic fatigue, excessive daytime sleepiness, and mood disturbances, which may contribute to social withdrawal, academic challenges, reduced participation in daily activities, and impaired quality of life. Over time, these patients are at increased risk for metabolic syndrome, cardiovascular disease, sustained reductions in quality of life, and premature mortality. Historically, the management of hypothalamic syndrome has been challenging for both patients and clinicians, as conventional obesity treatments, including lifestyle modification, dietary interventions, and physical activity, have shown limited long-term efficacy. Pharmacological approaches have likewise been unsatisfactory, either due to insufficient effectiveness or unacceptable adverse effects leading to their withdrawal from clinical use. The therapeutic impact of central nervous system stimulants and glucagon-like peptide-1 receptor agonists in acquired hypothalamic obesity remains inconsistent and subject to ongoing debate. Recent findings from a randomized controlled trial provide, for the first time, encouraging evidence that setmelanotide, a melanocortin-4 receptor agonist, may substantially improve outcomes in patients with hypothalamic dysfunction associated with hypothalamic obesity. The emergence of a safe and effective pharmacological therapy that addresses not only metabolic abnormalities but also key psychosocial features, such as hyperphagia and overall quality of life, may represent a significant advancement in the management of this complex condition and offers the potential to meaningfully improve outcomes in this highly burdened and underserved patient population.

This study aims to evaluate the demographic, clinical, laboratory, and radiological characteristics of pediatric patients with posterior reversible encephalopathy syndrome (PRES) admitted to the pediatric intensive care unit (PICU) and to identify prognostic factors associated with mortality. This retrospective cohort study included 52 MRI-confirmed pediatric PRES cases managed in a tertiary-level PICU between January 2020 and January 2026. Demographic features, etiologies, clinical findings, severity scores (PRISM/PELOD), laboratory parameters, treatment modalities, MRI patterns, and outcomes were compared between survivors and non-survivors. Univariable logistic regression and receiver operating characteristic (ROC) curve analyses were performed to identify unadjusted clinical correlates of mortality; given the small number of deaths (n&#x2009;=&#x2009;8), a multivariable model was not fitted, and Firth's penalized logistic regression was used as a sensitivity analysis for the main associations. The study adhered to STROBE guidelines. The mean age was 120.0&#x2009;&#xb1;&#x2009;50.8&#xa0;months; 65.4% had hematologic-oncologic diagnoses. Overall mortality was 15.4% (8/52). Seizures occurred in 90.4% and hypertension in 88.5% of patients. MRI most frequently demonstrated occipital and parietal involvement (90.4% each). Non-survivors had significantly lower admission GCS scores (median 10.0 [IQR 8.0-12.5] vs 13.0 [12.0-15.0], p&#x2009;=&#x2009;0.012), and longer PICU length of stay (median 14.5&#xa0;days [IQR 9.2-26.5] vs 5.0&#xa0;days [3.0-12.2], p&#x2009;=&#x2009;0.016). GCS demonstrated an area under the curve (AUC) of 0.777 (95% CI 0.619-0.918) with optimal cutoff&#x2009;&#x2264;&#x2009;10 (sensitivity 62%, specificity 84%). In univariate logistic regression with Firth penalized correction, vasopressor requirement was strongly associated with mortality (unadjusted penalized OR&#x2009;=&#x2009;29.75, 95% CI 2.800-315.530, p&#x2009;=&#x2009;0.005); the wide confidence interval reflects near-complete separation (7/8 non-survivors vs 6/44 survivors required vasopressors). The Glasgow Coma Scale (GCS) score at admission was associated with survival; each one-unit increase in the GCS score was associated with a 27% lower unadjusted odds of death (Firth-penalized estimate OR 0.73, 95% CI 0.550-0.940, p&#x2009;=&#x2009;0.016). In this large single-center pediatric series, lower GCS score, vasopressor requirement, and prolonged PICU stay were associated with mortality in univariate analysis. Vasopressor requirement may reflect the degree of hemodynamic compromise and the severity of illness rather than serve as an early clinical warning. These exploratory findings require validation in larger multicenter cohorts before clinical application. &#x2022; PRES typically presents with acute hypertension, seizures, headaches, visual disturbances, and altered mental status. Radiologically, brain imaging reveals cerebral edema, which is more pronounced in the parietal and occipital regions. &#x2022; In univariate logistic regression with Firth penalized correction, vasopressor requirement was associated with mortality (OR&#x2009;=&#x2009;29.75, 95% CI 2.800-315.530, p&#x2009;=&#x2009;0.005). &#x2022; Non-survivors had lower admission GCS, and GCS&#x2009;&#x2264;&#x2009;10 predicted death (AUC 0.777, 95% CI 0.619-0.918; 84% specificity).

This retrospective observational study examined the timing and sequence of clinical events during terminal hospitalization in pediatric oncology patients with progressive, treatment-refractory disease. The study included 54 children with solid tumors who died as inpatients at a tertiary center in Turkey between 2015 and 2025. Clinical events were mapped from admission to death, including both the time from admission to event onset and the interval from event onset to death. The most common diagnoses were neuroblastoma, Ewing sarcoma, and osteosarcoma. Patients experienced a mean of 3.1 &#xb1; 1.9 clinical events during terminal hospitalization. The most frequent events were intubation (64.8%), hypotension (57.4%), sepsis (48.1%), and electrolyte imbalance (35.2%). Temporal analysis showed that sepsis and hypotension occurred relatively early, with median onset times of 15.5 and 15 days after admission, respectively, whereas acute renal failure emerged later, with a median onset of 49 days. New-onset hypotension, intubation, and acute renal failure were closely associated with imminent death, with median intervals from event onset to death of 6, 7, and 3.5 days, respectively. These findings suggest that terminal decline in pediatric oncology follows measurable temporal patterns, and objective events such as hypotension, intubation, and acute renal failure may help clinicians recognize irreversible deterioration earlier.

Constitutional mismatch repair deficiency is a rare pediatric cancer predisposition syndrome caused by mutations in mismatch repair genes. We present the case of a 10-year-old with family history of Lynch syndrome, diagnosed with hepatic flexure adenocarcinoma and duodenal adenoma. Germline testing revealed a novel pair of biallelic PMS2 mutations. She received neoadjuvant ipilimumab and nivolumab, followed by total abdominal colectomy and adjuvant CAPEOX (capecitabine, oxaliplatin). Pathology revealed stage IIIB adenocarcinoma with partial response to immunotherapy. This case highlights the use of immunotherapy and screening for pediatric CMMRD-associated colorectal adenocarcinoma in the setting of a novel PMS2 variant.

To evaluate the impact of cancer treatment on the health-related quality of life (HRQoL) of children and adolescents. HRQoL self-perception was assessed using the Pediatric Quality of Life Inventory 3.0 Cancer Module (PedsQL), applied to 103 children and adolescents aged 1 to 18 years, recruited from a hematology and oncology unit of a Brazil public hospital and a support house. Sociodemographic and health data were collected through structured questionnaires, and a dental examination was conducted to assess dental caries. Data were analyzed using SPSS, including descriptive and Chi-square tests. Most participants were male (54.4%), aged 1-7 years (55.3%), with a primary diagnosis of hematologic and lymphatic cancer (85.4%). Family income was up to one minimum wage (55.3%), and 70.9% of mothers had at least 8 years of education. Children aged 1-7 scored lower in "Procedural Anxiety" (45.14 &#xb1; 40.37) than adolescents (66.67 &#xb1; 36.30). Adolescents scored lower in "Nausea" (55.11 &#xb1; 30.06) and "Worry" (56.34 &#xb1; 38.69) than children. HRQoL scores were associated with age (p < 0.001), maternal education (p = 0.022), brushing frequency (p = 0.046), and decayed teeth (p = 0.001). In the final model, self-reported HRQoL among children and adolescents aged 8 to 18 years (OR = 4.28; 95% CI: 1.07-17.12) was associated with lower HRQoL. Anxiety, nausea, and worry negatively impact the health-related HRQoL of pediatric cancer patients, and sociodemographic factors, such as the child's age, also influence this perception, highlighting the importance of individualized and integrated approaches to care.

Transient aplastic crisis (TAC) is a known complication of hereditary spherocytosis (HS), typically triggered by parvovirus-B19. However, in children with HS, the occurrence, clinical course and predictors of TAC severity remain incompletely defined. We aimed to characterize the clinical and hematologic features of TAC and to identify factors associated with its severity. We conducted a single-center retrospective cohort study of children with HS, analyzing clinical presentation, laboratory parameters, and outcomes. Associations between baseline disease severity and TAC course were assessed using correlation analyses and multivariable logistic regression. We studied 123 children with HS of whom 61 (49.6%) experienced TAC (median age 6.6 years, interquartile range (IQR) 4.56-8.18). Pancytopenia was observed in 28 (50.9%) of 55 patients with complete data and was associated with a lower mean nadir hemoglobin level (5.3&#x2009;&#xb1;&#x2009;1.0 vs. 5.9&#x2009;&#xb1;&#x2009;0.8g/dL, p&#xa0;=&#x2009;0.009) and a higher transfusion requirement (median 2, IQR 1.8-2 vs. 1, IQR 1-2 units, p&#x2009;<&#x2009;0.001). Acute parvovirus-B19 infection was confirmed in 94.4% of those tested. Lower baseline hemoglobin (&#x3c1;&#x2009;= -&#x2009;0.26, p =&#x2009;0.039) and higher baseline reticulocyte percentage (&#x3c1;&#x2009;=&#x2009;0.52, p&#x2009;<&#x2009;0.001) correlated with greater transfusion requirements. Baseline HS characteristics were more severe in patients who developed pancytopenia during TAC. In multivariable analysis, higher baseline reticulocyte percentage and older age at crisis were independently associated with greater TAC severity. All the patients achieved complete hematologic recovery. TAC-related pancytopenia is common in children with HS. Baseline hemolytic severity is associated with a more severe clinical course of TAC, supporting risk-adapted monitoring and management during aplastic crisis. &#x2022; Transient aplastic crisis (TAC) is a recognized complication of hereditary spherocytosis, most commonly triggered by parvovirus-B19. &#x2022; TAC is classically described as pure red cell aplasia but may be accompanied by additional cytopenias. &#x2022; In the largest pediatric cohort to date, pancytopenia occurred in about half of TAC events and was associated with increased transfusion requirements. &#x2022; Baseline hemolytic severity predicted the clinical course of TAC, including pancytopenia and transfusion requirements, supporting risk-adapted monitoring.

Acute promyelocytic leukemia (APL) is a highly curable form of acute myeloid leukemia (AML) when treated early with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). Resource constraints in low- and middle-income countries (LMICs) may affect results. This study examined the clinical characteristics and outcomes of pediatric patients with APL receiving an ATRA-ATO-based regimen. The retrospective review included 1- to 16-year-olds diagnosed between January 2019 and December 2022. Morphology and PML-RARA detection by fluorescence in situ hybridization (FISH) confirmed the diagnosis. Patients were stratified by initial white cell count (WCC) (low-risk <10&#xd7;10 9 /L; high-risk &#x2265;10&#xd7;10 9 /L). Data were retrieved from electronic medical records. Event-free survival (EFS) rates were obtained using the Kaplan-Meier analysis. Fifty patients (mean age: 10.7&#xa0;y; 56% male) were evaluated. Low-risk was 32%, and high-risk was 68%. Thrombocytopenia and hypofibrinogenemia were common. All patients had PML-RARA FISH-positives, and 100% of evaluated patients attained morphologic and molecular remissions. Eleven (22%) patients died, mostly from hemorrhage. Abandonment and relapses were observed in 10% and 8% of patients, respectively. Two-year EFS was 60% (low-risk 81%; high-risk 50%). The ATRA-ATO regimen showed substantial remission rates, but early deaths remain a concern. Increased survival rates in resource-constrained environments require improved early detection, referral, and supportive care.

This single institutional analysis explored outcomes and factors associated with local control and survival in pediatric patients with nonrhabdomyosarcoma soft tissue sarcoma (NRSTS). Patients 18 years of age or younger diagnosed between October 1990 and November 2021 who received external beam radiotherapy (EBRT) were retrospectively identified. Overall survival (OS) and disease-free survival (DFS) were analyzed using Kaplan-Meier methods and univariate Cox models. Among 51 included patients, there were 33 extremity tumors (65%), with 80% T1/T2 and 90% M0. Surgery in 49/51 (96%) resulted in 32 (65%) R0 resections. All received EBRT, with brachytherapy or intraoperative radiotherapy boosts in 21/51 (41%). Thirty-four (66.7%) received chemotherapy. With 5.7 years median follow-up, 5-year OS and DFS were 70.7% and 67.9%, respectively. Positive margins were significantly associated with worse OS. Two-year local failure incidence was 18.1% for EBRT versus 5.3% for EBRT+boost (HR 3.67, P =0.24). Among 6 local failures, 5 received EBRT alone, including 3 with positive margins. Two treated with EBRT+boost had positive margins, neither with local recurrence. Multimodality treatment of pediatric NRSTS results in 5-year OS and DFS around 70%. Although complete resection remains essential, and the modern incidence of R1 resection has declined, IORT/brachytherapy boost may continue to have a role in selected patients with a high local recurrence risk.

Romiplostim, a thrombopoietin receptor agonist, is a second-line therapy for primary immune thrombocytopenia (ITP). This real-world observational study evaluated romiplostim safety and efficacy in 52 children with persistent/chronic ITP. Baseline and follow-up data were retrospectively collected from patients' files. Primary endpoints: platelet response rate and time to first response. Secondary endpoints: durable response rate, safety, and health-related quality of life (HRQoL) using (PedsQL 4.0). Romiplostim decreased significant bleeding frequency (from 76.9% to 34.6%, P =0.003), hospital admission rate (from 69.2% to 7.7%, P <0.001), and rescue medication use (from 65.4% to 11.5%, P <0.001). In all, 92.3% of patients achieved platelet response in a median of 1 month, and 80.8% achieved a durable response. The most frequent adverse events were headache and joint pain; no serious adverse events occurred. Platelet count was positively correlated with HRQoL in parent-proxy reports ( P <0.05). In linear regression univariate analysis, platelet count &#x2265;100&#xd7;10 9 /L was associated with better parent-proxy HRQoL ( P =0.004) but not after multivariate adjustment, while joint pain remained independently associated with lower parent-proxy HRQoL. Beyond confirming the efficacy and safety of romiplostim, this study supports the sequential use of thrombopoietin receptor agonists, as romiplostim was still effective in patients who did not respond to previous treatment with eltrombopag.

Many childhood and adolescent cancer survivors suffer from late effects that impair quality of life and life expectancy. However, only an estimated 5% of >&#x2009;47,000 survivors in Germany receive adequate long-term follow-up (LTFU) care. The LE-Na (Evaluation and implementation of multidisciplinary, standardized, guideline-based long-term follow-up care for adult survivors of childhood cancer in Germany) study aims to address this care gap. Our interim analysis compares three recruitment and information pathways and provides information on the feasibility and acceptance of a guideline-based psychosocial screening. Over the total study period of 5&#x2009;years, 5000 survivors without previous LTFU care are systematically invited to specialized LTFU clinics in Germany: (1) through the German Childhood Cancer Registry (GCCR), (2) during transition from pediatric to adult care, and (3) via media presence. Longitudinal sociodemographic, medical, and psychosocial data are documented in an online database. Follow-up intervals are based on therapy-associated risk for late effects: annually (high risk), biannually (medium risk), or every 5&#x2009;years (low risk). By the time of our interim analysis, 300 survivors with completed data documentation had been enrolled: 121 (40.3%) were recruited via GCCR invitations, 155 (51.7%) through a structured transition, and 24 (8%) via media presence. Nearly half (131/300; 43.7%) were at high risk for late effects. Almost all (299/300, 99.7%) completed psychosocial screening. A Distress-Thermometer score of &#x2265;&#x2009;5 was found in half of the participants (146/299; 48.8%), indicating relevant psychosocial distress. Through the different recruitment methods, survivors with a high risk for late effects were particularly encouraged to accept the LTFU care offer. Integrating a psychosocial screening is feasible and confirms survivors' distress. DRKS identifier: DRKS00033303.

Children with severe neutropenia are advised to avoid activities and public spaces where they may be exposed to potential sources of infection, due to the risk of neutropenic fever, an oncologic emergency. The influence of exposure to environmental infectious disease risk factors (IDRFs) on the incidence of bacteremia has been difficult to study. Lockdowns enforced during the COVID-19 pandemic resulted in widespread social distancing and were associated with reduced opportunities for exposure to such environmental risk factors. We performed a retrospective chart review to compare the incidence of fever and bloodstream infection in patients with chemotherapy-induced neutropenia before (March 15, 2019 to March 14, 2020) and during the COVID-19 pandemic (March 15, 2020 to March 14, 2021). Before COVID-19, there were 219 episodes of neutropenia, 79 episodes with fever, and 12 episodes with bacteremia. During COVID-19, there were 236 episodes of neutropenia, 80 episodes with fever, and 13 episodes with bacteremia. In this small retrospective study, we did not observe a statistically significant difference in the incidence of either febrile neutropenia or febrile neutropenic bloodstream infection across 2 years with differing levels of exposure to environmental IDRFs (febrile neutropenia: IRR=0.89, P=0.38; bloodstream infection: IRR=1.01, P=0.99). Further multicenter studies are needed to better define the risks of exposure to potential environmental sources of infection, which may ultimately impact provider guidance for children with severe neutropenia.

To alert clinicians to the risk of severe hypercalcemia arising from the ATRA-azole interaction and to highlight a rare diagnostic pitfall involving a false-positive cerebrospinal fluid (CSF) (1,3)-&#x3b2;-D-glucan (BDG) result. Concurrent use of all-trans retinoic acid (ATRA) and azole antifungals poses a risk of severe hypercalcemia in pediatric acute promyelocytic leukemia (APL). We performed a retrospective clinical and laboratory review of two 10-year-old patients with APL who developed drug-induced toxicity during induction therapy. Both patients developed severe hypercalcemia induced by ATRA combined with fluconazole or voriconazole, presenting with intractable vomiting and headache. Notably, one case was confounded by an elevated CSF (1,3)-&#x3b2;-D-glucan level (a false-positive laboratory artifact), which mimicked fungal meningitis and delayed the correct diagnosis. Clinicians must remain vigilant for ATRA-associated hypercalcemia during azole co-administration. Recognizing diagnostic mimics and implementing routine calcium monitoring are crucial to prevent misdiagnosis and severe toxicity.

Survivors of childhood, adolescent, and young adult (CAYA) cancer face lifelong risks of treatment-related late effects, emphasizing the need for innovative, person-centred survivorship care. The PanCareFollowUp (PCFU) Care intervention, based on person-centred care (PCC), prioritizes survivors' values and needs. This paper describes the evaluation of experiences of healthcare professionals' (HCPs) and supporting staff's experiences with the training package that was developed for implementation of PCC in the PCFU Care. To support PCC delivery, a training package, including a training presentation with voice-over and a workshop, was developed. Healthcare professionals (HCPs) and supporting staff from four European survivorship clinics evaluated the training package through questionnaires measuring readiness to apply PCC, knowledge, confidence, perceived utility, and tool-related experiences. Eighteen HCPs evaluated the training presentation. Readiness to apply PCC increased from 68% pre-training to 89% post-training, while confidence declined from 78% to 68%. Most participants reported improved knowledge, with 94% indicating at least some increase. Frameworks such as Ekman's pillars, the seven steps for a PCC consultation, and shared decision-making questions were rated highly useful. Twenty-eight HCPs evaluated the workshop; 82% found it useful and 82% reported an overall favourable impression. Participants valued open discussion, multidisciplinary perspectives, and survivor involvement. The workshop's short duration and more broadly, limited time, staff shortages, and digital infrastructure were identified as barriers to PCC implementation. This multi-country evaluation shows that even brief PCC training can enhance HCPs' readiness to deliver person-centred survivorship care. The findings highlight both the potential of concise educational interventions and the importance of delivery format and organizational context for the sustainable implementation of PCC across survivorship care settings.

Nonaccidental trauma (NAT) can present with bleeding symptoms. Judicious use of hematologic testing is recommended to evaluate for medical causes of bleeding, while acknowledging inherited bleeding disorders and NAT may be present concurrently. A retrospective chart review using the Pediatric Health Information System database identified pediatric patients <18 years of age with an ICD diagnostic code for NAT associated with admission. Using laboratory charge data, we identified the hematologic testing sent and the number of encounters with expanded hematologic testing (EHT), defined as testing beyond CBC, PT, PTT, Factor IX, and von Willebrand testing (and fibrinogen/d-dimer in cases of intracranial hemorrhage). In 9561 admissions meeting inclusion criteria, laboratory testing was sent in 91.9% of encounters and EHT in 35.9% of encounters. The most common EHT test being fibrinogen. EHT was associated with a significant increase in laboratory-associated charges. Only 69 children (0.7%) were later identified to have an underlying bleeding disorder, most commonly von Willebrand Disease. Factors associated with EHT included hematology consultation, young age, higher income, and private health insurance. Despite few patients diagnosed with underlying bleeding disorders, EHT was frequently obtained. Sociodemographic features may influence testing decisions. Adherence to guidelines for evaluation may help reduce disparities.

Anthracycline chemotherapy is associated with cardiotoxicity, underscoring the importance of minimizing modifiable cardiac risk factors in affected individuals. This study evaluated cardiac function, prevalence of metabolic syndrome and smoking, and awareness of prior cardiotoxic treatment among adult survivors of childhood cancer treated with anthracyclines at the pediatric oncology service in Iceland between 1981 and 2011 (n=89). Data were collected from medical records, self-reported questionnaires, and clinical assessments conducted between 2016 and 2019. Of 76 eligible survivors, 60 participated (29 females, 31 males; aged 20 to 48&#xa0;y). Two individuals were excluded before enrollment due to previously diagnosed heart failure. Left ventricular dilation was observed in 15 participants (25%), predominantly mild to moderate, with 1 severe case. The prevalence was lower after BSA indexing. Metabolic syndrome was present in 20% of participants, and 20% reported current smoking. Approximately half of the participants reported undergoing echocardiography within the past 10 years, while only 10% were aware that their cancer treatment could cause cardiotoxicity. These findings suggest that survivors treated with anthracyclines require systematic surveillance and targeted interventions to address modifiable cardiovascular risk factors. Increasing awareness of prior cardiotoxic cancer treatment among survivors and health care providers may facilitate cardiovascular risk reduction.

Rare diseases affect a small percentage of the population but collectively impact millions worldwide. In the Middle East, the challenges are intensified by regional factors such as high rates of consanguinity, sociocultural stigma, limited diagnostic capacity, and inadequate healthcare infrastructure. These challenges often lead to delayed diagnoses, restricted access to treatment, and poor quality of life for affected individuals and their families. The Rare Advocacy Council conducted two 1.5-hour virtual expert panels involving 14 regional and international stakeholders (5 clinicians, 4 patient advocates, and 5 international academic experts) to identify and prioritize the challenges of managing rare diseases in the Middle East region. Discussions were organized across four domains: disease recognition and diagnosis, the patient journey and continuum of care, access to timely diagnostics, and access to adequate treatment, followed by structured online voting (involving only clinicians and patient advocates; n&#x2009;=&#x2009;9), discussions focused on prioritization, and a descriptive follow-up survey to identify the most critical barriers and propose actionable solutions. Key challenges identified included the lack of national disease registries, limited public awareness, underrepresentation of patient voices in decision-making, fragmented multidisciplinary care, and restricted access to diagnostics and advanced therapies. Top priorities included developing national registries, enhancing media-driven education, strengthening collaboration among care providers, and improving treatment accessibility through policy reforms. Effective management of rare diseases in the Middle East requires a coordinated, patient-centered approach. Strengthening health system infrastructure, investing in education, and aligning policy with patient needs are essential for sustainable improvement. Collaborative action among policymakers, healthcare providers, and advocacy groups can significantly advance care delivery and improve outcomes for individuals living with rare diseases.