Pediatric Blood & Cancer最新文献

{"title":"Silent Persistence: Genomic Characterization of Late Pulmonary Relapse in Osteosarcoma After a Decade of Remission","authors":"Oz Mordechai, Anat Ilivitzki, Eugene Vlodavsky, Myriam Ben-Arush, Sergey Postovsky","doi":"10.1002/pbc.32007","DOIUrl":"10.1002/pbc.32007","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964348","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":"Rare EWSR1::PBX1 Fusion-Positive Infant B-ALL With Coexisting KRAS Mutation: Sustained Molecular Remission Achieved With Standard Chemotherapy","authors":"Xiaoyong Chen, Zhiming Deng, Qiang Yao","doi":"10.1002/pbc.32022","DOIUrl":"10.1002/pbc.32022","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964421","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":"High Expression of NTRK1 in ETV6::RUNX1 Positive Acute Lymphoblastic Leukaemia Drives Factor Independence and Sensitivity to Larotrectinib","authors":"Luke Quinlan,&nbsp;Elyse C. Page,&nbsp;Jacqueline Rehn,&nbsp;Barbara J. McClure,&nbsp;Michael P. Osborn,&nbsp;Andrew S. Moore,&nbsp;Rishi S. Kotecha,&nbsp;Michelle J. Henderson,&nbsp;Michelle Martin,&nbsp;Frank Alvaro,&nbsp;David T. Yeung,&nbsp;Deborah L. White,&nbsp;Susan L. Heatley","doi":"10.1002/pbc.31983","DOIUrl":"10.1002/pbc.31983","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p><i>ETV6</i>::<i>RUNX1</i> is one of the most common recurrent genomic abnormalities in acute lymphoblastic leukaemia (ALL) and is associated with a good prognosis. High expression of <i>NTRK1</i>, encoding tropomyosin receptor kinase A (TrkA), confers a poor prognosis in other malignancies and may contribute to therapy resistance in patients with <i>ETV6::RUNX1</i> B-ALL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Method</h3>\u0000 \u0000 <p>Relapse-free survival was estimated by the Kaplan–Meier and log-rank analyses. <i>ETV6::RUNX1</i> and <i>NTRK1</i> were isolated from patient cDNA and transduced into IL-3–dependent Ba/F3 cells. Proliferation was assessed via CellTiter-Glo-2.0 with the addition of the TrkA agonist, nerve growth factor (NGF). Sensitivity to the TRK inhibitor larotrectinib was assessed via Annexin V/7AAD staining and flow cytometry. Changes in TrkA signalling through effectors, ERK and AKT, were evaluated by western blotting.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Patients with <i>ETV6::RUNX1</i> had increased <i>NTRK1</i> expression in comparison with other B-ALL cases (<i>p</i> &lt; 0.0001), with those overexpressing <i>NTRK1</i> exhibiting a trend towards increased relapse. in vitro experiments revealed that only Ba/F3 <i>ETV6::RUNX1</i>+<i>NTRK1</i> cells demonstrated IL-3 independence, indicative of leukaemic transformation (vs parental Ba/F3, <i>p</i> &lt; 0.0001). These cells were sensitive to larotrectinib (LD₅₀ 161 nM) and significantly decreased phosphorylation of ERK and AKT (vs NGF <i>p =</i> 0.0004; <i>p =</i> 0.007, respectively).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p><i>NTRK1</i> overexpression has not previously been reported in B-ALL and was associated with increased relapse in patients with <i>ETV6::RUNX1</i> and <i>NTKR1</i> overexpression. The in vitro success of larotrectinib treatment warrants further in vivo investigation and may be a viable therapeutic addition for patients with <i>ETV6::RUNX1</i> B-ALL and <i>NTRK1</i> overexpression.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964347","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":"Virtual Transition Assessments in Sickle Cell Disease Are Feasible and Available Transition Instruments May Highlight Different Components of the Transition Experience","authors":"Alexandra Boye-Doe,&nbsp;Samuel Wilson,&nbsp;Lindsey Akpuogu,&nbsp;Jane Little,&nbsp;Jacquelyn Baskin-Miller","doi":"10.1002/pbc.31963","DOIUrl":"10.1002/pbc.31963","url":null,"abstract":"<div>\u0000 \u0000 <p>There is increased morbidity and mortality during the transition to adult care in sickle cell disease. Standardized implementation of validated transition tools may improve readiness for transition (RFT). We tested the impact of telehealth on this process by comparing in-person or via telehealth administration of the provider-administered tool, TRxANSITION Index (TI), to the patient-reported tool, transition intervention program-RFT (TIP-RFT) in patients 15–21 years old. Scores between testing given in person or via telehealth did not differ (<i>p</i> &gt; 0.9, <i>N</i> = 20). TIP-RFT scores were up to 14% higher than TI. Our results indicate that telehealth transition tools are feasible and are acceptable to providers and participants. Our data also suggest that the more objective TI may be most sensitive in detecting transition needs; this awaits validation.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964354","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":"Effect of Pelvic External Beam Radiation Therapy on Lower Urinary Tract Function of Pediatric Patients","authors":"Michael H. Edwards,&nbsp;Andrew Nicklawsky,&nbsp;Gemma Beltran,&nbsp;Elizabeth Stein,&nbsp;Jennifer Pyrzanowski,&nbsp;Kyle Rove,&nbsp;Karlie Boone,&nbsp;Sarah Milgrom,&nbsp;Nicholas G. Cost","doi":"10.1002/pbc.31996","DOIUrl":"10.1002/pbc.31996","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Pediatric patients with pelvic solid tumors often receive multimodal treatment, but the long-term effects of this treatment on lower urinary tract (LUT) function are not well-described. This prospective cohort study (NCT05818462) investigates the effect of external beam radiation therapy (EBRT) on LUT function by comparing patient-reported outcome measures, non-invasive urodynamic studies, and post-void residuals (PVRs) amongst childhood cancer survivors (CCS).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>All subjects were CCS greater than 1 year off-therapy for a pediatric solid tumor. Subjects were divided into cohorts by whether they had received pelvic EBRT. Research subjects completed the Dysfunctional Voiding Scoring system (DVSS) survey, uroflow testing, and PVR bladder scan.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Thirty-two patients enrolled; half received pelvic EBRT (mean 30.0 Gy). There was an equal number of patients exposed to alkylators (69%) in each cohort. Radiation cohort was further off therapy (5.3 vs. 4.0 years). The pelvic radiation cohort was more likely to have abnormal uroflow patterns (81% vs. 56%, <i>p</i> = 0.12). The flow pattern distribution differed between groups (<i>p</i> = 0.03); the pelvic radiation cohort was more likely to have a “plateau”-shaped curve (44% vs. 6%) and abnormal PVR (odds ratio [OR] 9.0, <i>p</i> = 0.002). Exploratory conditional likelihood ratio models yielded potential associations between pelvic EBRT and LUTD, as well as a potential dose–response. There was no difference in DVSS scores between cohorts.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>EBRT appears to affect LUT function in CCS who received pelvic EBRT. DVSS does not appear useful as a screening tool in this population. Further study is needed to better appreciate the impact of EBRT on LUT function and develop more accurate screening tools.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964358","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":"HABIT Efficacy Trial Intervention Improves Elements of General and Disease-Specific Quality of Life in Youth With Sickle Cell Disease","authors":"Arlene Smaldone,&nbsp;Deepa Manwani,&nbsp;Banu Aygun,&nbsp;Abena Appiah-Kubi,&nbsp;Kim Smith-Whitley,&nbsp;Nancy S. Green","doi":"10.1002/pbc.31990","DOIUrl":"10.1002/pbc.31990","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Whether interventions to improve hydroxyurea adherence in youth with sickle cell disease (SCD) also improve health-related quality of life (HRQoL) has not been determined. We prospectively examined changes in generic and disease-specific HRQoL over a 12-month period in youth who participated in “Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT)” randomized controlled multi-site efficacy trial. The HABIT intervention was led by community health workers and augmented by tailored text message reminders.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Improvements in generic and disease-specific HRQoL were secondary HABIT outcomes. Intervention efficacy and sustainability were measured as changes in HRQoL from Months 0 to 9 and from Months 9 to 12 of the trial, respectively. Data were analyzed for within-group and between-group changes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Fifty youth, 24 assigned to intervention and 26 to the control group, mean age of 13.3 ± 1.9 years, participated in the trial. There were no differences between groups at study entry. At Month 0, total generic and disease-specific HRQoL scores were 71.2 ± 15.6 and 62.7 ± 19.0, respectively. At 9 months, compared to controls, the intervention group significantly improved generic HRQoL total score (<i>p</i> = 0.04) and two subscales (emotional, <i>p</i> = 0.03, and social, <i>p</i> = 0.01), and one disease-specific HRQoL subscale (treatment, <i>p</i> = 0.006). HRQoL improvements were not sustained at 12 months.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Findings of this study contribute to the evolving understanding of the impact of interventions to improve hydroxyurea adherence on HRQoL in youth with SCD. Further research directed to enhancing intervention sustainability is needed to maintain hydroxyurea adherence and HRQoL improvements to reduce health disparities for youth with SCD.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964375","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":"Child and Adolescent Cancer Communication Preferences for Treatment Decision Making: A Meta-Synthesis","authors":"Kimberly A. Pyke-Grimm,&nbsp;Katherine Patterson Kelly,&nbsp;Ginny L. Schulz","doi":"10.1002/pbc.31944","DOIUrl":"10.1002/pbc.31944","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Children and adolescents (C&amp;A) &lt;18 years of age report varying cancer treatment communication preferences. The aim of this qualitative meta-synthesis was to describe C&amp;A voices regarding their preferences for engagement in their treatment communication. This report shares their preferences specific to their involvement in cancer treatment decision-making (TDM).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Applying meta-aggregation methods, a systematic search was conducted in multiple databases with no limitation to the date range. Studies included participants &lt;18 years of age with childhood cancer using qualitative or mixed research methods, written in English. Findings were limited to C&amp;A self-reported findings.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 3213 articles were identified, with 55 articles included. A total of 156 findings related to preferences for TDM were extracted from 39 studies. Three synthesized findings were identified: (1) How I participate in decisions about my cancer treatment, (2) When I do not participate in decisions about my cancer treatment, and (3) Why I participate in decisions about my cancer treatment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>This meta-synthesis identified how, when, and why C&amp;A want to be involved in their cancer TDM. Their voices represent a range of preferences for involvement, from a more passive to more active role. Evidence-based practice recommendations were identified with a focus on meeting the C&amp;A communication preferences.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964377","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":"‘All I Had to Do Was Open My Mouth Wide’—A Qualitative Exploration of the Acceptability of Photobiomodulation for Oral Mucositis Management in Paediatric Supportive Care","authors":"Claudia Heggie,&nbsp;Amrit Chauhan,&nbsp;Kara A. Gray-Burrows,&nbsp;Peter F. Day,&nbsp;Bob Phillips","doi":"10.1002/pbc.31978","DOIUrl":"10.1002/pbc.31978","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Oral mucositis (OM) presents a common and debilitating side effect of chemotherapy for children and young people (CYP). Photobiomodulation is recommended for OM prevention in international guidance; however, the acceptability of photobiomodulation in paediatric cancer care is uncertain. This study explores this acceptability with CYP, their parents, and healthcare professionals (HCPs).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Semi-structured interviews with CYP/parent dyads and focus groups with HCPs were audio-recorded and professionally transcribed. Framework analysis was completed utilising the Theoretical Framework of Acceptability (TFA) using an initial deductive approach for theoretical rigour. Over-arching themes within and between constructs and participant groups were developed. Recruitment occurred alongside analysis until there was repetition of data and an absence of novel codes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Twenty-seven participants were interviewed. CYP were aged 8–15 years old; HCPs had diverse professional roles within paediatric oncology and dentistry. Over half of families and three quarters of HCPs had previous photobiomodulation experience. Data were coded for all seven TFA constructs. Four themes, consisting of multiple subthemes, were developed from 42 distinct codes: (i) positive attitudes towards photobiomodulation; (ii) importance of child-centredness and autonomy; (iii) lack of understanding of photobiomodulation treatment; (iv) perceived additional burden to healthcare teams.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Photobiomodulation for OM prevention is acceptable to CYP, their parents, and HCPs during cancer treatment. Exploration of the theoretical facets of this acceptability supports adaptation of services to overcome highlighted challenges to photobiomodulation acceptance. Photobiomodulation services should be designed to reduce burden on healthcare services, with resources developed to support CYP's autonomy, comprehension and self-efficacy with intra-oral treatment.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31978","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964300","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":"Unmet Needs in Pediatric Oncological Patients Eligible for Palliative Care","authors":"Sara Cerasi,&nbsp;Davide Leardini,&nbsp;Claudio Chiossi,&nbsp;Giovanna Locatelli,&nbsp;Deborah Masini,&nbsp;Maria Prisco,&nbsp;Cinzia Scalera,&nbsp;Monica Beccaro,&nbsp;Riccardo Masetti,&nbsp;Tamara Belotti","doi":"10.1002/pbc.32001","DOIUrl":"10.1002/pbc.32001","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964365","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":"Reply to: Comment on: Ototoxicity in Pediatric Hematopoietic Stem Cell Transplantation With High Dose Carboplatin: Identifying Modifiable Risk Factors Through the Inclusion of Younger Patients","authors":"Anne Spence,&nbsp;Miranda Camet,&nbsp;Susan S. Hayashi,&nbsp;Mary Claire McGlynn,&nbsp;Ningying Wu,&nbsp;Robert J. Hayashi","doi":"10.1002/pbc.32008","DOIUrl":"10.1002/pbc.32008","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964359","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}