Pediatric Blood & Cancer最新文献

{"title":"A comparative in vivo study of hyperthermic intraperitoneal chemotherapy with cisplatin versus doxorubicin versus cisplatin plus doxorubicin for the treatment of intra-abdominally disseminated alveolar rhabdomyosarcoma in mice","authors":"Illya Martynov,&nbsp;Jens Gesche,&nbsp;Lajwanti Dhaka,&nbsp;Luca Tobi,&nbsp;Paul Hoyer,&nbsp;Guido Seitz","doi":"10.1002/pbc.31366","DOIUrl":"10.1002/pbc.31366","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Treatment options for advanced intra-abdominal pediatric rhabdomyosarcoma (RMS) with peritoneal sarcomatosis (PS) include cytoreductive surgery (CRS) and hyperthermic intraperitoneal chemotherapy (HIPEC). However, optimal dosages and combination regimens of drugs used for HIPEC are underexplored. We aimed to evaluate the efficacy of HIPEC with cisplatin, doxorubicin, and their combination in vivo.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We established PS/RMS mouse model by intraperitoneally injecting RH30 cells into NOD/LtSz-scid IL2Rγ-null mice. Two weeks post xenotransplantation, mice underwent a single HIPEC procedure at 42°C for 60 minutes. Treatment groups received cisplatin (50, 100, and 150 mg/m²) and doxorubicin (30, 45, and 60 mg/m²), administered alone or combined. The control group underwent an intraperitoneal lavage with isotonic saline. Peritoneal cancer index (PCI) was used to quantify the extent of peritoneal tumor spread. Tissue samples were evaluated regarding proliferation (Ki-67) and apoptosis (caspase 3).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Mice treated with cisplatin at 100 mg/m² (PCI of 3.875, <i>p</i> = .007) and 150 mg/m² (PCI of 4.556, <i>p</i> = .026), and doxorubicin at 30 mg/m² (PCI of 2.875, <i>p</i> &lt; .001) and 45 mg/m² (PCI of 4.143, <i>p</i> = .021) showed reduced PCI, with the combination of cisplatin 50 mg/m² and doxorubicin 30 mg/m² showing the most prominent effect (PCI of 3.333, <i>p</i> &lt; .001) compared to the control group (PCI of 8.615). Histologically, there was no difference in Ki-67 or caspase 3 expression among the groups.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>The cisplatin- and doxorubicin-based HIPEC significantly reduces peritoneal tumor dissemination in vivo. Further investigations are needed to explore the underlying molecular responses to optimize therapeutic strategies.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142392289","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychiatric disorders among survivors of childhood acute lymphoblastic leukemia in Denmark and Sweden","authors":"Gitte V. Sørensen,&nbsp;Hanna Mogensen,&nbsp;Anna S. Holmqvist,&nbsp;Line Kenborg,&nbsp;Camilla Pedersen,&nbsp;Thomas T. Nielsen,&nbsp;Mats Talbäck,&nbsp;Friederike Erdmann,&nbsp;Marianne Ifversen,&nbsp;Maria Feychting,&nbsp;Kjeld Schmiegelow,&nbsp;Mats M. Heyman,&nbsp;Jeanette F. Winther,&nbsp;Henrik Hasle,&nbsp;Line E. Frederiksen","doi":"10.1002/pbc.31361","DOIUrl":"10.1002/pbc.31361","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>The diagnosis and treatment of childhood acute lymphoblastic leukemia (ALL) may impact mental health. We investigated the long-term risk of psychiatric disorders among survivors of ALL in a population-based cohort study.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We identified patients diagnosed with ALL in Denmark and Sweden before age 20 during 1982–2008. Survivors of ALL (<i>n</i> = 2026), their siblings (<i>n</i> = 3027), and population comparison subjects (<i>n</i> = 9713) were followed for hospital contacts for psychiatric disorders from 5 years after ALL diagnosis (or corresponding index date) until 2017.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>By age 30, the absolute risk of psychiatric hospital contacts was 19.9% (95% confidence interval [CI]: 17.9–22.1) for ALL survivors, 18.5% (95% CI: 16.9–20.2) for siblings, and 18.3% (95% CI: 17.3–19.2) for population comparison subjects. Overall, survivors were at higher risk of any psychiatric disorders than siblings (hazard ratio [HR] = 1.25; 95% CI: 1.04–1.50), and population comparison subjects (HR = 1.20; 95% CI: 1.06–1.35). The subgroup of survivors (<i>n</i> = 332) who received a hematopoietic stem cell transplantation (HSCT) and/or had a relapse were at highest risk of psychiatric disorders (HR = 2.07; 95% CI: 1.26–3.41 compared to siblings; HR = 1.67; 95% CI: 1.25–2.23 compared to population comparison subjects).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>The overall absolute risk of psychiatric hospital contacts among ALL survivors was close to that in siblings and population comparison subjects. The modestly increased relative risk was mainly driven by the subgroup of survivors who received HSCT and/or had a relapse. Our findings are reassuring for the large subgroup of ALL survivors without HSCT or relapse, and provide novel insight on both absolute and relative risk of hospital contacts for psychiatric disorders.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31361","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142392339","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quality of life and late therapy effects in pediatric non-Hodgkin lymphoma survivors: Insights from a single-institution study","authors":"Azza Ayad,&nbsp;Reham Khedr,&nbsp;Asmaa Hamoda,&nbsp;Nahla Elnabarawy,&nbsp;Elhamy Rifky,&nbsp;Tamer Diab,&nbsp;Eman El Desouky,&nbsp;Leslie Lehmann,&nbsp;Alaa Elhaddad","doi":"10.1002/pbc.31357","DOIUrl":"10.1002/pbc.31357","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background and aim</h3>\u0000 \u0000 <p>The survival outcomes of pediatric patients with mature B-non-Hodgkin lymphoma (NHL) have improved due to advances in treatment. We aimed to assess the frequency and severity of late effects and their impact on quality of life among pediatric NHL survivors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patients and methods</h3>\u0000 \u0000 <p>This retrospective study included patients diagnosed with mature B-NHL at Children's Cancer Hospital of Egypt (CCHE) 57357 from January 2012 through December 2015. Patients received treatment according to the modified LMB 96 protocol. The minimum follow-up period was 5 years. Assessments for toxicity and quality of life were conducted at regular intervals during and after treatment. Patients were assessed for toxicity including pulmonary dysfunction, cardiac dysfunction, lipid profile abnormalities, endocrine dysfunction (thyroid function, vitamin D levels, growth curves), and cognitive function (intelligence quotient [IQ] level using Stanford–Binet Intelligence Scales—5th Edition, and quality of life (QoL) assessment (PedsQoL).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 273 patients were eligible, and 206 were evaluable. Median age was 5.45 (range: 2.4–18), with a male-to-female ratio 4:1. Pulmonary function abnormalities were detected in 119/203 (58.6%); most had mild dysfunction (72/119, 60.5%), while 17% had severe dysfunction. Cardiac toxicity occurred in 10% of the patients (<i>n</i> = 20). IQ testing showed that 52 patients had a low average IQ score, while 151 patients had either average or above average scores. The total mean QoL score was 99 ± 0.058 classified as “satisfactory.” However, significant impairment of the physical domain of quality of life was observed among group C patients compared to A/B (<i>p</i> = .033), older age at diagnosis (<i>p</i> = .042), and those with pulmonary dysfunction (<i>p</i> &lt; .001). Total score of quality of life was significantly impaired among patients with pulmonary dysfunction (<i>p</i> = .009), likewise older age at diagnosis (<i>p</i> = .017) and those with low average IQ scores (<i>p</i> = .033).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Childhood mature B-NHL survivors are at significant risk for late effects; pulmonary dysfunction and low average IQ that can subsequently impact QoL</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142381468","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Burden of SARS-CoV-2 infection prior to vaccine eligibility among immunocompromised children aged 1–11 years at a pediatric tertiary referral hospital in Toronto, Canada","authors":"Dara Petel,&nbsp;Mohsin Ali,&nbsp;James Wright,&nbsp;Aaron Campigotto,&nbsp;Michelle Science,&nbsp;Sumit Gupta,&nbsp;Shelly Bolotin","doi":"10.1002/pbc.31365","DOIUrl":"10.1002/pbc.31365","url":null,"abstract":"<p>SARS-CoV-2 seroprevalence reflects the efficacy of pandemic infection prevention and control measures. We performed anti-spike IgG serological testing on residual sera of children 1–11 years old at a tertiary care referral center between October and November 2021. Immunocompromised patients had the highest SARS-CoV-2 seroprevalence, at 40.5%, compared to 19.3% in non-immunocompromised patients. Targeted infection prevention and public health interventions are warranted for pediatric immunocompromised patients in future pandemics.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375718","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CHIEF: A retrospective self-control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis","authors":"Daniel Mashiach,&nbsp;Patrice Mead,&nbsp;Kendall Carneiro,&nbsp;Jemily Malvar,&nbsp;Susan Knight,&nbsp;Guy Young","doi":"10.1002/pbc.31351","DOIUrl":"10.1002/pbc.31351","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Hemophilia A (HA) is an X-linked bleeding disorder diagnosed by a deficiency in factor VIII (FVIII). For severe HA (SHA), prophylaxis clotting factor concentrates (CFC) has become the standard of care; however, it imparts a high treatment burden and typically results in an annualized bleeding rate (ABR) of 2–6. Emicizumab, a subcutaneously administered FVIII substitute, has become the de facto standard-of-care prophylaxis for children with SHA in many countries. Previous clinical trials of emicizumab have assessed ABR in patients greater than 12 years without inhibitors, and in children less than 12 years with inhibitors; however, there is little information published regarding the ABR of emicizumab compared to CFC in non-inhibitor SHA children.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Using a retrospective electronic medical record chart review, we conducted a self-control analysis of 15 patients less than 12 years of age during equivalent periods of CFC versus emicizumab prophylaxis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The mean ABR on CFC and emicizumab was 1.79 and 1.13 (<i>p</i> = .092), respectively, with a substantially decreased rate of joint bleeds (CFC 0.94; emicizumab 0.33; <i>p</i> = .001) and spontaneous bleeds (CFC 0.79; emicizumab 0.23; <i>p</i> = .008). No safety events were recorded for patients while administering emicizumab. The mean annual cost of CFC prophylaxis was $515,340 (SD $199,540), compared to $328,410 (SD $137,230) for emicizumab prophylaxis (<i>p</i> &lt; .001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Emicizumab resulted in an improved ABR compared to CFC, especially for joint and spontaneous bleeds, had fewer administration complications, and was substantially less expensive compared to CFC prophylaxis; however, more research is necessary for a complete understanding of the effect of emicizumab on joint health and muscle bleeds.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31351","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Extreme thrombocytosis negative for GATA1 mutation in an infant with trisomy 21","authors":"Tatiana Lestido,&nbsp;Kaitlin Ryan-Smith,&nbsp;Alice Werner,&nbsp;Linda Pegram","doi":"10.1002/pbc.31352","DOIUrl":"10.1002/pbc.31352","url":null,"abstract":"&lt;p&gt;Transient abnormal myelopoiesis (TAM) is characterized by a transient proliferation of abnormal myeloid cells in the bone marrow, leading to an excess of immature cells circulating in the peripheral blood.&lt;span&gt;&lt;sup&gt;1&lt;/sup&gt;&lt;/span&gt; TAM diagnosis is associated with the presence of &lt;i&gt;GATA1&lt;/i&gt; mutations, but other features such as blasts on peripheral blood smear, flow cytometry immunophenotype, or cytogenetics can be indicative of TAM. This patient's case is unusual due to the &lt;i&gt;GATA1&lt;/i&gt; mutation-negative status. The &lt;i&gt;GATA1&lt;/i&gt; gene encodes a transcription factor crucial for normal hematopoiesis, and mutations in this gene are associated with myeloid disorders including TAM.&lt;span&gt;&lt;sup&gt;2&lt;/sup&gt;&lt;/span&gt; TAM leads to a wide range of hematologic abnormalities and clinical complications, including hyperviscosity syndrome with potential for thrombosis, hydrops fetalis, pericardial effusion, respiratory distress, hypereosinophilia, pseudohyperkalemia, hyperbilirubinemia with liver failure, multiorgan failure, and potential for death.&lt;span&gt;&lt;sup&gt;1, 3-5&lt;/sup&gt;&lt;/span&gt; Patients with TAM require timely diagnosis as well as close follow-up, as 20%–30% of these patients subsequently develop myeloid leukemia associated with Down syndrome (ML-DS) before the age of 4 years.&lt;span&gt;&lt;sup&gt;6&lt;/sup&gt;&lt;/span&gt; &lt;i&gt;GATA1&lt;/i&gt; mutations have, to date, been discovered in nearly all patients with TAM and ML-DS.&lt;span&gt;&lt;sup&gt;7&lt;/sup&gt;&lt;/span&gt; The absence of a &lt;i&gt;GATA1&lt;/i&gt; mutation in this case raises the question of an alternative molecular mechanism contributing to the development of TAM and extreme thrombocytosis in this patient.&lt;/p&gt;&lt;p&gt;The patient is a 37-week gestation male delivered via cesarean section due to breech presentation. Birth weight was 2880 g. Promptly upon delivery, the neonate exhibited respiratory compromise necessitating urgent endotracheal intubation and conventional mechanical ventilation.&lt;/p&gt;&lt;p&gt;He was noted to have distinct facial dysmorphology consistent with trisomy 21. Initial complete blood count (CBC) showed a white blood cell (WBC) count of 25,100/mL with 21% blasts, hemoglobin of 16.7 g/dL, and a platelet count of 633,000/mL.&lt;/p&gt;&lt;p&gt;While the neonate's oxygen saturation exhibited some improvement following mechanical ventilation, his PaO&lt;sub&gt;2&lt;/sub&gt; remained low. A chest x-ray showed no acute cardiopulmonary process. A discrepancy between pre- and post-ductal saturations prompted an echocardiogram, which demonstrated elevated right ventricular pressures, with pulmonary pressures measuring at two-thirds the systemic level, and a patent ductus arteriosus (PDA) characterized by bidirectional flow.&lt;/p&gt;&lt;p&gt;In light of deteriorating respiratory function and severe pulmonary hypertension, the infant was switched to high-frequency jet ventilation (HFJV). Subsequently, the neonate was transferred to our tertiary care facility. Despite high-frequency jet ventilation, adjunctive inhaled nitric oxide therapy, and administration of corticosteroids at stress dosages, his ","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31352","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375720","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Roles of pediatric surgeons in palliative pediatric oncology.","authors":"Hau D Le, Sarah Braungart, Jaime Shalkow-Klincovstein, Nelson Piché","doi":"10.1002/pbc.31354","DOIUrl":"https://doi.org/10.1002/pbc.31354","url":null,"abstract":"<p><p>Pediatric surgeons engaged in oncology will inevitably treat patients receiving palliative care, but their role in this context is poorly described. This article identifies some of the challenges and opportunities of surgical involvement in pediatric oncology palliative care, underscoring how the surgeon's expertise can be exploited to significantly benefit children with cancer. Specific examples of skills (procedural, communication, and coordination) that surgeons can provide to the multidisciplinary palliative care teams are described and the importance of collaboration is highlighted.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31354"},"PeriodicalIF":2.4,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immune or inherited thrombocytopenia? A population-based cohort study on children and adolescents presenting with a low platelet count","authors":"Lauri-Matti Kulmala,&nbsp;Henri Aarnivala,&nbsp;Tytti Pokka,&nbsp;Anu Huurre,&nbsp;Liisa Järvelä,&nbsp;Sauli Palmu,&nbsp;Tuuli Pöyhönen,&nbsp;Riitta Niinimäki","doi":"10.1002/pbc.31363","DOIUrl":"10.1002/pbc.31363","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Thrombocytopenia is a common hematologic finding in children and adolescents. Immune thrombocytopenia (ITP) is the most common cause of this finding, but the differential diagnosis includes a growing list of genetic disorders. We aimed to report differences in phenotypes of patients with ITP, inherited platelet disorder (IPD)/primary immunodeficiency disorder (PID), and other causes, with a focus on differentiating ITP from inherited thrombocytopenia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>This retrospective, population-based observational cohort from 2006 to 2020 involved 506 Finnish children under 16 years of age presenting with isolated thrombocytopenia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of the 506 participants, 79.7% had ITP, 6.7% had IPD/PID, and 13.6% had other causes of thrombocytopenia. A platelet count of ≤12 × 10⁹/L best distinguished between ITP and other reasons with a sensitivity of 60% and a specificity of 80%. Among patients with the lowest platelet count of less than 10 × 10⁹/L, 95.9% had ITP, 3.3% had IPD/PID, and 0.8% had other causes. Severe bleeding events were reported in 20 patients (4.0%), but there were no cases of intracranial or fatal bleeding due to thrombocytopenia. Up to 50% of patients with a high suspicion of inherited thrombocytopenia remained without a specific diagnosis despite genetic testing.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>ITP remains the most common cause of thrombocytopenia. A platelet count of ≤12 × 10⁹/L often leads to an ITP diagnosis. Genetic disorders are rare but should be suspected in patients with persisting thrombocytopenia, especially with platelet counts constantly above 12 × 10⁹/L, a positive family history, or atypical clinical features.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31363","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375721","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding the 2020 pediatric cancer deficit: Insights from the National Childhood Cancer Registry","authors":"Jason Semprini,&nbsp;Erin M. Mobley","doi":"10.1002/pbc.31345","DOIUrl":"10.1002/pbc.31345","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375723","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pediatric oncology nutritional practices in high-income countries: A survey from the International Society of Paediatric Oncology (SIOP)","authors":"Erin Gordon,&nbsp;Mirjam van den Brink,&nbsp;Nina van der Linden,&nbsp;Karen Ringwald-Smith,&nbsp;Breeana Gardiner,&nbsp;Alexia J. Murphy-Alford","doi":"10.1002/pbc.31353","DOIUrl":"10.1002/pbc.31353","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Optimal nutrition in pediatric oncology can influence cancer-related outcomes. To establish an understanding of nutrition practice and perceptions of best practice, we queried nutrition providers practicing in pediatric oncology care centers in high-income countries.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>An electronic, multidisciplinary, cross-sectional survey of nutrition practices was conducted among pediatric oncology nutrition practitioners. Final analysis included 110 surveys from 71 unique institutions and included practitioners from Europe, the United States, Canada, Australia/New Zealand, South America, and the Middle East/Asia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The majority of institutions (97%) reported having dietitians; 72% had designated oncology dietitians. Approximately half of the practitioners (47%) reported feeling their institutions were inadequately staffed. The majority (78%) of institutions completed nutrition risk screening, but there was no consensus on specific screening practices. Half (50%) of the institutions that screened for nutrition risk did so in both inpatient and outpatient settings. The majority (80%) of institutions completed a nutrition assessment close to the time of diagnosis. Those that did not cite lack of staff and/or lack of time, lack of standardized approach, and consult only level of nutritional care as primary barriers. The most common topic of nutrition education provided to patients/families was nutrition-related symptom management (68%).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>While most institutions reported having pediatric oncology dietitians, we found a lack of standardized practice and perceived inadequate staffing. In addition, what providers perceived to be best practice did not always align with day-to-day clinical practice. Ongoing efforts are needed to develop evidence-based guidelines, including staffing recommendations, to support specialized care in this population.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142372478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}