Pediatric Blood & Cancer最新文献

{"title":"Surgical Fertility Preservation can be Safely Performed Without Delaying Treatment for Children With Severe Aplastic Anemia.","authors":"Christopher J McCauley, Erin E Rowell, Monica M Laronda, Sara Zarnegar-Lumley","doi":"10.1002/pbc.32114","DOIUrl":"https://doi.org/10.1002/pbc.32114","url":null,"abstract":"<p><p>Pediatric patients with severe aplastic anemia (SAA) who undergo hematopoietic cell transplantation are at significantly increased risk of infertility. Pretransplant fertility preservation (FP) options for prepubertal children are surgical procedures: ovarian or testicular tissue cryopreservation (OTC and TTC). Bleeding and infection risk must be considered given profound cytopenias in these patients. We prospectively enrolled patients in a surgical FP clinical protocol at our institution from 2015 to 2023. We evaluated the approach and outcomes of all 14 patients with SAA who underwent surgical FP (TTC: n = 7; OTC: n = 7). No patients experienced a surgical complication related to FP. With our patient cohort, surgical FP was safely achieved utilizing a comprehensive standard of care pathway.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32114"},"PeriodicalIF":2.3,"publicationDate":"2025-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145337384","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":"Inflammatory Bowel Disease Following Allogeneic Hematopoietic Cell Transplant for Sickle Cell Disease.","authors":"Emily Rowland, Akshay Sharma, Samantha Paglinco, Ross M Maltz, Jeffrey E Deyo, Kiran Joglekar, John Brannon Alberty, Courtney Fitzhugh, Hemalatha G Rangarajan","doi":"10.1002/pbc.32121","DOIUrl":"https://doi.org/10.1002/pbc.32121","url":null,"abstract":"<p><p>Although graft-versus-host disease (GvHD) is an important cause of gastrointestinal (GI) complications after allogeneic hematopoietic stem cell transplantation (HCT), the non-specific symptoms make diagnosis challenging. We described three patients with sickle cell disease who developed inflammatory bowel disease (IBD) like manifestations 4-18 months post-HCT. All patients received peripheral blood stem cell grafts and sirolimus for GvHD prophylaxis. All had chronic diarrhea, and biopsies showed extensive colonic ulceration, non-necrotizing granulomas, with minimal histologic evidence of GvHD. Symptoms resolved promptly with IBD-directed therapy. Our report highlights the importance of considering alternative rare etiologies for GI complications such as IBD after HCT.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32121"},"PeriodicalIF":2.3,"publicationDate":"2025-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145337453","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":"Sickle Cell Disease-Related Stigma: Quantitative Assessment From India.","authors":"Bontha V Babu, Yogita Sharma, Parikipandla Sridevi, Shaily B Surti, Madhusmita Bal, Deepa Bhat, Jatin Sarmah, Manoranjan Ranjit, Rabindra K Jena","doi":"10.1002/pbc.32116","DOIUrl":"https://doi.org/10.1002/pbc.32116","url":null,"abstract":"<p><p>This multisite cross-sectional study quantitatively assessed stigma related to sickle cell disease (SCD) in India using the Indian Council of Medical Research-SCD Stigma Scale for India among 208 adult patients and 184 caregivers. Approximately 27% of patients and 41% of caregivers reported severe or very severe stigma. 'Familial & reproductive', 'illness burden' and 'perceived blame & social judgement' stigma domains contributed significantly. Ordinal logistic regression analysis identified pain episodes in patients (AOR = 1.199, p = 0.013) and caregiver gender (AOR = 0.300, p = 0.016) and income (AOR = 0.999, p = 0.048) as significant factors associated with stigma severity. The findings underscore a substantial psychosocial burden and highlight the need for culturally grounded, multilevel interventions integrated into SCD care programs to address stigma comprehensively.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32116"},"PeriodicalIF":2.3,"publicationDate":"2025-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145308401","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":"Longitudinal Patterns of Fatigue in Long-Term Survivors of Childhood and Adolescent Cancers: A Report From the Swiss Childhood Cancer Survivor Study.","authors":"Salome Christen, Luzius Mader, André O von Bueren, Eva Maria Tinner, Grit Sommer, Christina Schindera, Claudia E Kuehni, Katharina Roser, Gisela Michel","doi":"10.1002/pbc.32110","DOIUrl":"https://doi.org/10.1002/pbc.32110","url":null,"abstract":"<p><strong>Background: </strong>Fatigue negatively affects quality of life. We aimed to compare the prevalence of fatigue in survivors of childhood cancer with the Swiss general population, describe longitudinal patterns of fatigue, and identify characteristics associated with persistent fatigue in survivors.</p><p><strong>Procedure: </strong>In this cohort study, we used data from the Swiss Childhood Cancer Registry and the Swiss Childhood Cancer Survivor Study, including survivors (≥5 years since diagnosis; diagnosed between 1976 and 2015 at <20 years of age) aged ≥20 years at study entry, using data from the baseline and follow-up survey. A representative sample of the general population was used as a comparison group. Fatigue prevalence and fatigue severity were measured using the SF-36 vitality scale, from which we derived longitudinal patterns (no/low fatigue, late onset, improving, persistent). We used multivariable logistic regression to identify clinical, psychosocial and demographic characteristics associated with persistent fatigue.</p><p><strong>Results: </strong>Overall, 1846 survivors participated at baseline (52% male), and 684 survivors also participated at follow-up (median 9 years from baseline; 52% male). From the general population, 863 persons participated (42% male). Survivors had similar fatigue prevalence at baseline/follow-up (26%/29%) as the general population (26%). No/Low fatigue was experienced by 64%, late onset by 14%, improving by 7%, and persistent fatigue by 15% of survivors. More late effects, psychological distress, pain, and less time spent on moderate-intensity physical activity were associated with persistent fatigue.</p><p><strong>Conclusions: </strong>This study provides data on longitudinal patterns of fatigue in survivors and identifies factors associated with persistent fatigue that can be used to identify survivors at risk and as a target for interventions aimed at improving fatigue.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32110"},"PeriodicalIF":2.3,"publicationDate":"2025-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145308410","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 RISE: Development of a Poverty-Targeted Cash Support Intervention for Pediatric Cancer.","authors":"Colleen A Kelly, Morgan A Paul, Jennifer Kellett, Sunyu Kang, Anna Revette, Rahela Aziz-Bose, Leanne Duhaney, Puja J Umaretiya, Amy Lin, Erika Hanson, Kira Bona","doi":"10.1002/pbc.32103","DOIUrl":"https://doi.org/10.1002/pbc.32103","url":null,"abstract":"<p><strong>Background: </strong>Poverty is independently associated with relapse and death in childhood cancer despite highly standardized treatment. Prior data show that direct cash support interventions are feasible and improve child outcomes; however, no such interventions exist within pediatric oncology. To address this, we aimed to pilot and refine Pediatric RISE (Resource Intervention to Support Equity), a novel, direct cash support intervention in pediatric oncology.</p><p><strong>Procedure: </strong>This was a single-arm pilot study among low-income children with cancer at a single center. Participants received twice-monthly cash disbursements and optional benefits counseling for 3 months. Parent surveys and qualitative interviews evaluated acceptability, satisfaction, and barriers to utilization to inform refinement.</p><p><strong>Results: </strong>Families received all intended cash disbursements and used funds for essential resources with a reduction in household material hardship. Parents described RISE as useful and acceptable. There was no parent-reported loss or reduction of means-tested government benefits. Parents reported a need for larger disbursements and extended duration to mitigate treatment-associated income losses.</p><p><strong>Conclusion: </strong>RISE was acceptable and valuable, with appropriate mitigation of the risk of means-tested government benefit loss or reduction by intervention design. Parent feedback informed refinement, including an increase in cash disbursement dollar amount and duration. The refined RISE intervention is currently being evaluated in a multi-site, randomized signal-finding study.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32103"},"PeriodicalIF":2.3,"publicationDate":"2025-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293261","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":"A Phase I Trial of Nab-Paclitaxel in Combination With Gemcitabine for Relapsed/Refractory Pediatric Solid Tumors.","authors":"Jonathan Metts, Kevin Ginn, Zhulin He, Rohali Keesari, Jane C Cook, Stacy Senn, Melissa Schink, Samer Sansil, Hong Yin, Thomas Cash","doi":"10.1002/pbc.32102","DOIUrl":"https://doi.org/10.1002/pbc.32102","url":null,"abstract":"<p><strong>Background: </strong>The single-agent pediatric maximum tolerated dose (MTD) of nab-paclitaxel is significantly higher than adult dosing. We conducted a Phase I trial to establish the MTD/recommended Phase 2 dose (RP2D) of nab-paclitaxel with gemcitabine in children with relapsed/refractory solid tumors.</p><p><strong>Methods: </strong>Nab-paclitaxel was administered intravenously with fixed-dose gemcitabine on days 1, 8, and 15 of 28-day cycles. Three dose levels (DL) of nab-paclitaxel, 180, 210, and 240 mg/m², were evaluated using a rolling six design. Toxicity, nab-paclitaxel pharmacokinetics (PK), and radiologic responses were evaluated. Pretreatment tumor tissue was assessed for SPARC and CAV-1.</p><p><strong>Results: </strong>Twenty-four patients enrolled, 22 received therapy, and 20 were evaluable for dose-limiting toxicity (DLT): 17 during dose escalation and 3 in dose expansion at the MTD. Median age was 12.5 years. Diagnoses included osteosarcoma (n = 11), neuroblastoma (n = 4), and rhabdomyosarcoma (n = 4). At the starting dose level (gemcitabine 1000 mg/m², nab-paclitaxel 180 mg/m²), two of five patients experienced DLT, prompting an amendment lowering gemcitabine to 675 mg/m²/dose. Post-amendment, nab-paclitaxel 240 mg/m²/dose with gemcitabine 675 mg/m²/dose was identified as the MTD. Grade ≥3 hematologic toxicities were common. Two patients experienced a partial response (Wilms tumor and osteosarcoma), and PK exhibited linearity across DL. SPARC immunoreactivity was present in most tumors, while CAV-1 immunoreactivity was infrequent.</p><p><strong>Conclusions: </strong>The MTD/RP2D of nab-paclitaxel with gemcitabine in patients with relapsed/refractory pediatric solid tumors is nab-paclitaxel 240 mg/m²/dose and gemcitabine 675 mg/m²/dose on days 1, 8, and 15 of 28-day cycles; responses were limited in this patient population.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32102"},"PeriodicalIF":2.3,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293231","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":"Socioeconomic Status and Stroke Risk in Pediatric Sickle Cell Disease: A DISPLACE Study Secondary Analysis.","authors":"Jeffrey G Edwards, Daniel T Humphrey, Martina Mueller, Shannon Phillips, Alyssa Schlenz, Morohuntodun O Oni, Julie Kanter, Kira Bona, Natasha M Archer","doi":"10.1002/pbc.32112","DOIUrl":"https://doi.org/10.1002/pbc.32112","url":null,"abstract":"<p><strong>Background: </strong>Transcranial doppler (TCD) screening identifies children ages 2-16 years with sickle cell disease (SCD) at an increased risk for stroke. Previous studies have shown that individuals from households on public insurance with limited neighborhood opportunity have increased barriers to receiving adequate healthcare; however, it is unknown whether low socioeconomic status (SES) influences TCD results.</p><p><strong>Methods: </strong>We conducted a secondary analysis of the DISPLACE study data, which included children aged 2-6 years who had at least one TCD screen performed during their year of study enrollment, if space allowed. We utilized insurance type (public insurance vs. all other insurances) and overall childhood opportunity index (COI) group (Very Low vs. all other COI levels [Low, Medium, High, and Very High]) to proxy household- and neighborhood-level SES. We then examined the association between household- and neighborhood-level SES and conditional or abnormal TCD screening results.</p><p><strong>Results: </strong>The analytic cohort included 3124 children, with 1295 (41.5%) children living in Very Low COI neighborhoods and 2019 (64.6%) of households with public insurance. In the multinomial logistic regression model, only the lack of HU prescription remained significantly associated at p < 0.05 with abnormal TCD result (adjusted odds ratio = 19.01, p < 0.001).</p><p><strong>Conclusion: </strong>Lack of hydroxyurea (HU) prescription was the factor most strongly associated with abnormal TCD outcome. Low SES proxies were not independently associated with abnormal or conditional TCD results. Qualitative studies are needed to better understand why a child with SCD might not have been prescribed HU.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32112"},"PeriodicalIF":2.3,"publicationDate":"2025-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145286682","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":"Comment on: \"Long-Term Clinical Outcome and Quality of Life of Children, Adolescents, and Young Adults With Chordoma or Chondrosarcoma Treated With Pencil Beam Scanning Proton Therapy.\" Commendable Progress in Pediatric Proton Therapy, but Methodological Precision Remains Crucial.","authors":"Raza Ur Rehman, Ahmad Jabbar","doi":"10.1002/pbc.31981","DOIUrl":"https://doi.org/10.1002/pbc.31981","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31981"},"PeriodicalIF":2.3,"publicationDate":"2025-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145280951","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":"Diabetes Mellitus and Metabolic Profile in Long-Term Survivors of Wilms Tumor: A Nationwide Cohort Study With Sibling Controls.","authors":"Stine Høgsholt, Kirstine Stochholm, Esben Søndergaard, Peter Haubjerg Asdahl, Catherine Rechnitzer, Henrik Hasle","doi":"10.1002/pbc.32097","DOIUrl":"https://doi.org/10.1002/pbc.32097","url":null,"abstract":"<p><strong>Background: </strong>As survival after Wilms tumor has markedly improved, focus on potential long-term consequences is necessary. We investigated the metabolic profile among Danish survivors of Wilms tumor >20 years from cancer diagnosis.</p><p><strong>Methods: </strong>As of January 15, 2015, we established a cohort of 20-plus-year survivors of Wilms tumor identified through the national cancer registries. Siblings contributed as controls. Participation included clinical examination with anthropometric measurements, blood samples, blood pressure measurements, and a comprehensive health questionnaire. Using logistic regression, we identified risk factors for diabetes mellitus (DM).</p><p><strong>Results: </strong>With a response rate of 64%, we included 99 survivors of Wilms tumor with a median of 37 years since diagnosis (median age of 41, range: 24-70), and 38 sibling controls (median age of 41, range: 24-66). An increased prevalence of DM was found in the survivors as compared with their siblings (15% vs. 0%). A logistic regression analysis adjusted for age at follow-up showed that left-sided versus right-sided abdominal radiation therapy was a risk factor for the development of DM (odds ratio [OR] 7.9, 95% confidence interval [CI]: 1.4-44). A larger fraction of the survivors was in active treatment for hypertension compared to siblings (42% vs. 37%), but with no significant difference in body mass index, hip, or waist circumferences. Furthermore, Wilms tumor survivors were more likely to be in active treatment with lipid-lowering agents (8% vs. 3%).</p><p><strong>Conclusion: </strong>Left-sided abdominal radiation is a major risk factor for DM in long-term survivors of Wilms tumor. Patients may benefit from surveillance with increased focus on metabolic risk profile to decrease the risk of DM and cardiovascular events.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32097"},"PeriodicalIF":2.3,"publicationDate":"2025-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145280924","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":"Examining the Impact of Time-to-Treatment on Disease-Specific Survival in Pediatric Pilocytic Astrocytoma: A Multi-Institutional Retrospective Cohort Analysis.","authors":"Anisha Kohli, Kate S Woods, Blake Recupido, Avisya Mishra, Mitchell A Taylor, Molly E Kubesh, Aditya Sharma, Peter T Silberstein","doi":"10.1002/pbc.32026","DOIUrl":"https://doi.org/10.1002/pbc.32026","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32026"},"PeriodicalIF":2.3,"publicationDate":"2025-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145281004","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}