Pediatric Blood & Cancer最新文献

{"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":"https://doi.org/10.1002/pbc.32085","url":null,"abstract":"<p><strong>Background: </strong>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><p><strong>Objectives: </strong>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><p><strong>Study design: </strong>We compared the CI of severe acute GVHD and graft failure by Day 180 in patients who received ABA (n = 26), against a historical cohort of HSCT BMFS patients who did not receive ABA (n = 21) in a combined retrospective review from two centers.</p><p><strong>Results: </strong>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%, p = 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% (p = 0.51). The OS (95.2% vs. 96.0%, p = 0.3) and GFS at 1 year (64.3% vs. 65.3%, p = 0.5) were similar in the ABA group compared to the historical group.</p><p><strong>Conclusion: </strong>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>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32085"},"PeriodicalIF":2.3,"publicationDate":"2025-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"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":"https://doi.org/10.1002/pbc.32083","url":null,"abstract":"<p><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>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32083"},"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}

{"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":"https://doi.org/10.1002/pbc.32087","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32087"},"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}

{"title":"A Case of Pigmented Mesoblastic Nephroma With Melanocytic Features.","authors":"Amine Bouachba, Gessica Lobascio, Philippe Drabent, Pascale Philippe Chomette, Gaelle Pierron, Dominique Berrebi","doi":"10.1002/pbc.32078","DOIUrl":"https://doi.org/10.1002/pbc.32078","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32078"},"PeriodicalIF":2.3,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145177216","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":"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":"https://doi.org/10.1002/pbc.32090","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32090"},"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":"https://doi.org/10.1002/pbc.32088","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32088"},"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}

{"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":"https://doi.org/10.1002/pbc.32081","url":null,"abstract":"<p><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":" ","pages":"e32081"},"PeriodicalIF":2.3,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Safety and Efficacy of Ruxolitinib in an Infant With Familial Hemophagocytic Syndrome Type 3: A Bridging Therapy Toward Hematopoietic Cell Transplantation and Treatment of Post-Transplant Complications, Including Sinusoidal Obstruction Syndrome.","authors":"Kazuki Takahashi, Shinsuke Hirabayashi, Masahiro Ueki, Masataka Hasegawa, Hiroki Ohata, Shunichiro Takezaki, Yukayo Terashita, Yuko Cho, Atsushi Manabe","doi":"10.1002/pbc.32073","DOIUrl":"https://doi.org/10.1002/pbc.32073","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32073"},"PeriodicalIF":2.3,"publicationDate":"2025-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145138143","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":"Outcome of Premature Infants Treated With Chemotherapy for Neuroblastoma in France.","authors":"Dana Al Khalifa, Pascal Chastagner, Gudrun Schleiermacher, Julien Lejeune","doi":"10.1002/pbc.32027","DOIUrl":"https://doi.org/10.1002/pbc.32027","url":null,"abstract":"<p><p>Neuroblastoma is the most common extracranial solid tumor in children, and congenital forms account for 5% of all neuroblastoma cases. In utero diagnosis is very rare, and the progression leading to premature delivery for urgent treatment is exceptional. In some of these cases, chemotherapy is required after birth, given tumor progression that threatens the functional prognosis (dumbbell neuroblastoma), or even survival (Stage MS). However, the optimal doses of chemotherapy in premature newborns are not known. We present a retrospective cohort of six patients treated in French pediatric oncology centers in order to analyze treatment modalities, and report on their tolerance (five Grade 4 hematological toxicity, three septicemias) as well as the outcome of these newborns (two deaths, four remissions).</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32027"},"PeriodicalIF":2.3,"publicationDate":"2025-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145138169","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":"Acute Myeloid Leukemia With KAT6A Deletion in a 7-Week-Old.","authors":"Ingrid S Tam, Savio S T Nguyen, Andrea Hawrysh, Christopher S Pharis, Paul R D'Alessandro","doi":"10.1002/pbc.32077","DOIUrl":"https://doi.org/10.1002/pbc.32077","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32077"},"PeriodicalIF":2.3,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125650","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}