Rachel Offenbacher, Lara Fabish, Alice Lee, Rui Yang, Bang Hoang, David S. Geller, Jana L. Fox, David M. Loeb, Alissa Baker
{"title":"Pravastatin for Radiation-Induced Fibrosis of the Extremity: A Single-Center Experience","authors":"Rachel Offenbacher, Lara Fabish, Alice Lee, Rui Yang, Bang Hoang, David S. Geller, Jana L. Fox, David M. Loeb, Alissa Baker","doi":"10.1002/pbc.32084","DOIUrl":"10.1002/pbc.32084","url":null,"abstract":"<div>\u0000 \u0000 <p>Radiation therapy (RT) is a mainstay of treatment for several sarcomas. Radiation-induced fibrosis (RIF) is a common side effect of RT with an incidence of approximately 80%. RIF is caused by excessive proliferation of myofibroblasts, which produce collagen and extracellular matrix components, coupled with a reduction of remodeling enzymes. Targeting this pathway with pravastatin to prevent and ameliorate RIF has been shown to improve outcomes in patients with head and neck cancer. We share our single-center experience using pravastatin to treat RIF involving the joints in children with sarcomas who developed RIF, as well as in a population administered prophylactically.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145192346","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":"Dual Use of Skeletal Muscle Index and Body Mass Index Measured at the Time of Diagnosis Predicts Prognosis in Children With Malignant Solid Tumors: A Single-Center Retrospective Study","authors":"Wataru Kudo, Keita Terui, Katsuhiro Nishimura, Daisuke Katsumi, Ryoya Furugane, Ayako Takenouchi, Shugo Komatsu, Satoru Oita, Yunosuke Kawaguchi, Tomoro Hishiki","doi":"10.1002/pbc.32082","DOIUrl":"10.1002/pbc.32082","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Sarcopenia, a progressive muscle-wasting disorder, is increasingly being recognized in childhood cancers. While skeletal muscle index (SMI) is commonly used to assess sarcopenia, we investigated the combined utility of SMI and body mass index (BMI) in predicting the prognosis of children with malignant solid tumors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The BMI and SMI of 119 children with different types of malignant solid tumors were analyzed. Computed tomography and height at the time of diagnosis were used to determine the SMI. SMI and BMI were standardized using reference values. Cluster and survival time analyses were conducted to identify subpopulations.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Cluster analysis performed using <i>z</i>-scores for SMI and BMI yielded four clusters: Cluster 1 had high <i>z</i>-scores for both indices; Cluster 2 had average <i>z</i>-scores for both indices; Cluster 3 had low <i>z</i>-scores for SMI and average <i>z</i>-scores for BMI; and Cluster 4 had low <i>z</i>-scores for both indices. Cluster classification was an independent prognostic factor for both event-free survival (hazard ratio 1.56; 95% confidence interval: 1.09–2.25) and overall survival (hazard ratio 1.70; 95% confidence interval: 1.10–2.64) after combining age and metastasis. Cluster 1 had low metastasis prevalence and a favorable prognosis, whereas Cluster 4 exhibited a poorer prognosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The potential prognoses of the four distinct patient clusters based on the <i>z</i>-scores for SMI and BMI at the time of diagnosis were determined. The dual use of SMI and BMI may facilitate the identification of children with malignant solid tumors who are at a high risk of poor outcomes.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145192369","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}
Prachi Sharma, Nivya George, Deokumar Srivastava, Eric J. Chow, Melissa A. Alderfer, Wendy Leisenring, Kristin A. Long, Anne E. Lown, Kevin C. Oeffinger, Lonnie K. Zeltzer, Gregory T. Armstrong, Kevin R. Krull, Tara M. Brinkman, David Buchbinder
{"title":"Psychosocial Health and Chronic Health Conditions Among Bereaved Siblings: A Report From the Childhood Cancer Survivor Study (CCSS)","authors":"Prachi Sharma, Nivya George, Deokumar Srivastava, Eric J. Chow, Melissa A. Alderfer, Wendy Leisenring, Kristin A. Long, Anne E. Lown, Kevin C. Oeffinger, Lonnie K. Zeltzer, Gregory T. Armstrong, Kevin R. Krull, Tara M. Brinkman, David Buchbinder","doi":"10.1002/pbc.32076","DOIUrl":"10.1002/pbc.32076","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To compare psychosocial health and chronic health conditions (CHCs) in bereaved and non-bereaved adult siblings impacted by childhood cancer and to identify predictors of emotional distress and health-related quality of life among bereaved siblings.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A total of 4558 adult siblings (733 bereaved; 3825 non-bereaved) of 5-year survivors of childhood cancer completed measures of emotional distress (Brief Symptom Inventory [BSI]-18) and health-related quality of life (Medical Outcomes Survey Short Form [SF]-36) and reported their social attainment milestones (i.e., educational attainment, employment, and marital status). CHCs’ burden was classified as none/low versus medium/high/very severe. Cancer-associated complications prior to the patient's death, sibling age at bereavement, and social attainment variables were examined as predictors of emotional distress and health-related quality of life among bereaved siblings using multivariable modified Poisson regression.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Bereaved siblings in this sample reported excellent psychosocial health long term (e.g., depressive symptoms 6.5%, somatization 4.4%, anxiety 3.5%). Bereaved siblings had an elevated risk of depression (relative risk [RR] 1.53; 1.10–2.13, <i>p</i> = 0.01), reduced social quality of life (RR 1.35; 1.00–1.82, <i>p</i> = 0.05), diminished educational attainment, and greater CHC burden than non-bereaved siblings. No differences were observed for other subscales or social attainment outcomes. Among bereaved siblings, risk factors for depression included male sex (RR 0.42; 0.19–0.93, <i>p</i> = 0.05), never being married (RR 3.02; 1.45–6.28, <i>p</i> = 0.05), and greater CHC burden (RR 2.42; 1.18–4.99, <i>p</i> = 0.05). Risk factors for poor social functioning included unemployment (RR 2.24; 1.12–4.45, <i>p</i> = 0.05) and never being married (RR 2.16; 1.22–3.82, <i>p</i> = 0.05).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Bereaved siblings report excellent psychosocial health long-term and demonstrate only a marginally elevated risk of experiencing symptoms of depression and poor social quality of life compared to non-bereaved siblings.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186507","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}
Zahra Hudda, Stella M. Davies, Adam Lane, Deborah E. Schiff, Eric J. Anderson, Parinda A. Mehta, Kasiani C. Myers, Nicholas J. Gloude
{"title":"Abatacept Prevents Severe Acute Graft-Versus-Host Disease Without Increasing Graft Failure Risk in Pediatric Bone Marrow Failure Syndromes","authors":"Zahra Hudda, Stella M. Davies, Adam Lane, Deborah E. Schiff, Eric J. Anderson, Parinda A. Mehta, Kasiani C. Myers, Nicholas J. Gloude","doi":"10.1002/pbc.32085","DOIUrl":"10.1002/pbc.32085","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Pediatric patients with inherited or acquired bone marrow failure syndromes (BMFS) often require an allogeneic hematopoietic stem cell transplant (HSCT) to cure the hematological manifestations. Amongst these, those without a matched sibling donor (MSD), are at increased risk for graft failure and are known to tolerate graft-versus-host disease (GVHD) poorly. Abatacept (ABA) is Food and Drug Administration (FDA)-approved for the prevention of acute GVHD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Our primary objective was to investigate the cumulative incidence (CI) of severe acute (Grade III–IV) GVHD in patients with BMFS undergoing a matched unrelated donor (MUD) or mismatched related or unrelated donor (MMRD/MMUD) HSCT with ABA added to standard GVHD prophylaxis of calcineurin inhibitor and methotrexate or mycophenolate mofetil, and evaluate the incidence of graft failure. Secondary outcomes included overall survival (OS) and acute and chronic GVHD-free graft failure-free overall survival (GFS) at 1 year.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Study Design</h3>\u0000 \u0000 <p>We compared the CI of severe acute GVHD and graft failure by Day 180 in patients who received ABA (<i>n</i> = 26), against a historical cohort of HSCT BMFS patients who did not receive ABA (<i>n</i> = 21) in a combined retrospective review from two centers.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>None of the patients in the ABA cohort experienced severe (Grade III–IV) acute GVHD compared to 14% in our historical cohort (CI 0% vs. 14%, <i>p</i> = 0.05). All patients in our ABA cohort successfully engrafted, with one patient experiencing poor graft function. In the historical cohort, one patient experienced primary graft failure. The rates of chronic GVHD were comparable between the historical and ABA groups, 28.6% versus 19.2% (<i>p</i> = 0.51). The OS (95.2% vs. 96.0%, <i>p</i> = 0.3) and GFS at 1 year (64.3% vs. 65.3%, <i>p</i> = 0.5) were similar in the ABA group compared to the historical group.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>ABA was well tolerated in patients with BMFS and prevented severe acute GVHD without increasing the incidence of graft failure. Rates of chronic GVHD and GFS at 1 year were similar, but analysis was limited due to sample size and variability in ABA dosing.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32085","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186502","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}
Julia Loureiro Sion, Daniele M. Celeste, Bernadete L. Liphaus, Marlene P. Garanito, Jorge D. A. Carneiro
{"title":"Cerebral Sinovenous Thrombosis: 20 Years of Experience in a Pediatric Tertiary Center","authors":"Julia Loureiro Sion, Daniele M. Celeste, Bernadete L. Liphaus, Marlene P. Garanito, Jorge D. A. Carneiro","doi":"10.1002/pbc.32083","DOIUrl":"10.1002/pbc.32083","url":null,"abstract":"<div>\u0000 \u0000 <p>Cerebral sinovenous thrombosis (CSVT) is rare in children and is related to risk factors, as dehydration and infections. This study analyzed 44 pediatric cases of CSVT from a 20-year registry. Risk factors were identified in 93%, infection being the most frequent. Thrombophilia was detected in eight patients. Most patients (91%) received anticoagulation: 78% low molecular weight heparin and 20% unfractionated heparin. Treatment duration exceeded current recommendations (median 21 weeks), as most cases were treated based on CHEST 2012 guidelines. One patient experienced major bleeding related to anticoagulation. All thrombi improved, with 49% achieving complete resolution. There were no recurrences and no fatalities linked to anticoagulation.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145177227","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}
Ashley C. Holland, Margit K. Mikkelsen, Soniya Pinto, Noah D. Sabin, Lu Wang, Bradley Muller, Raja B. Khan, Hiroto Inaba
{"title":"Cytarabine-Related Neurotoxicity in an Infant With Acute Lymphoblastic Leukemia","authors":"Ashley C. Holland, Margit K. Mikkelsen, Soniya Pinto, Noah D. Sabin, Lu Wang, Bradley Muller, Raja B. Khan, Hiroto Inaba","doi":"10.1002/pbc.32087","DOIUrl":"10.1002/pbc.32087","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145177234","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}
Broadman Joshua, Erin Barthelmess Goodell, Donna J. Curtis, Laura McLaughlin, Amanda Strommen, Katherine T. Lind
{"title":"Successful Use of Maribavir for Secondary CMV Prophylaxis in a Pediatric HSCT Patient","authors":"Broadman Joshua, Erin Barthelmess Goodell, Donna J. Curtis, Laura McLaughlin, Amanda Strommen, Katherine T. Lind","doi":"10.1002/pbc.32090","DOIUrl":"10.1002/pbc.32090","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150316","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":"Improving Implementation of the Psychosocial Standards of Care Together","authors":"Leandra Desjardins","doi":"10.1002/pbc.32088","DOIUrl":"10.1002/pbc.32088","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150308","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}
Allison L. Bartlett, Kristie N. Ramos, Jamen R. Bartlett, Kenneth S. Ramos, Nathan Luebbering, Kasiani C. Myers, Parinda A. Mehta, Stella M. Davies
{"title":"LINE-1 Retroelement Activation and Neuroinflammation in Persons With Fanconi Anemia","authors":"Allison L. Bartlett, Kristie N. Ramos, Jamen R. Bartlett, Kenneth S. Ramos, Nathan Luebbering, Kasiani C. Myers, Parinda A. Mehta, Stella M. Davies","doi":"10.1002/pbc.32081","DOIUrl":"10.1002/pbc.32081","url":null,"abstract":"<p>We describe the first immunohistochemical analysis performed on brain lesion biopsies from two young individuals with Fanconi anemia neuroinflammatory syndrome (FANS). We identified aberrant activation of the LINE-1 retrotransposon as a novel mechanism of neuroinflammation in individuals with hematological disorders. LINE-1 ORF-1 protein is upregulated in biopsies of inflammatory lesions compared to brain tissue from persons with FA without FANS, and shows activation of an innate immune response with increased STING and IFI6 expression. Awareness among hematologists is vital, as this condition can mimic an aggressive brain tumor both clinically and radiographically, and the proposed mechanism has meaningful therapeutic implications.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32081","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145150239","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}