Pediatric Blood & Cancer最新文献

{"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":"10.1002/pbc.32073","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"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,&nbsp;Pascal Chastagner,&nbsp;Gudrun Schleiermacher,&nbsp;Julien Lejeune","doi":"10.1002/pbc.32027","DOIUrl":"10.1002/pbc.32027","url":null,"abstract":"<div>\u0000 \u0000 <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>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"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,&nbsp;Savio S. T. Nguyen,&nbsp;Andrea Hawrysh,&nbsp;Christopher S. Pharis,&nbsp;Paul R. D'Alessandro","doi":"10.1002/pbc.32077","DOIUrl":"10.1002/pbc.32077","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"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}

{"title":"Hemorrhagic Cystitis Following Hematopoietic Stem Cell Transplantation in Children: A Single Pediatric Center Experience","authors":"Pauline Mazilier,&nbsp;Laurence Dedeken,&nbsp;Eleonore Powis,&nbsp;Pierluigi Calò,&nbsp;Stéphane Luyckx,&nbsp;Laure Kornreich,&nbsp;Safiatou Diallo,&nbsp;Adriano Salaroli,&nbsp;Christine Devalck","doi":"10.1002/pbc.32071","DOIUrl":"10.1002/pbc.32071","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Hemorrhagic cystitis (HC) is a frequent and potentially severe complication following hematopoietic stem cell transplantation (HSCT) in children. It significantly affects the quality of life and prolongs hospitalization. Despite its frequency, no standardized management guidelines exist. This study aimed to describe the characteristics, risk factors, and treatments of HC in a pediatric HSCT population.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted a retrospective single-center study including all pediatric patients who underwent allogeneic HSCT between January 2007 and December 2022. HC was defined and graded according to the European Conference on Infections in Leukaemia (ECIL) guidelines. We analyzed risk factors, BK virus (BKV) viruria, treatments, and outcomes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>HC occurred in 24 patients (14.7%), with a median onset at 27.5 days post-HSCT. Acute graft-versus-host disease (aGVHD) was significantly associated with HC occurrence (45.8% vs. 19.4%, <i>p</i> = 0.01), particularly in its severe forms (<i>p</i> = 0.007). BKV was detected in 66.7% of HC cases. Viral load in urine was not significantly associated with HC severity or duration. All patients received supportive care; cidofovir was used in 79.2%, with increasing frequency in higher grade HC. Severe cases required multimodal management, including intravesical installations, hyperbaric oxygen therapy (HBO; 37.5%), and arterial embolization (all Grade 4 cases). No deaths were directly attributed to HC.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>HC remains a significant and morbid complication after pediatric HSCT. Our findings highlight the need for early identification of high-risk patients and access to timely, multidisciplinary interventions. Multicenter prospective studies are essential to standardize care and improve outcomes in this vulnerable population.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32071","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125590","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":"Sintilimab Bevacizumab as Salvage Therapy in Pediatric Thymic Lymphoepithelioma-Like Carcinoma","authors":"Tianjiao Hu,&nbsp;Jian Li,&nbsp;Yao Sun,&nbsp;Lulu He,&nbsp;Xiaojun Zhang,&nbsp;Jin Xu,&nbsp;Tao Li,&nbsp;Li Zhou","doi":"10.1002/pbc.32079","DOIUrl":"10.1002/pbc.32079","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125562","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":"Post-Transplant Cyclophosphamide for Graft-Versus-Host Disease Prophylaxis in Pediatric Stem Cell Transplantation","authors":"Aaron E. Fan,&nbsp;Wilson Andres Vasconez,&nbsp;Ramya Ramakrishnan,&nbsp;Priti Tewari,&nbsp;Amanda Olson,&nbsp;Elizabeth J. Shpall,&nbsp;Irtiza N. Sheikh","doi":"10.1002/pbc.32061","DOIUrl":"10.1002/pbc.32061","url":null,"abstract":"<p>Graft-Versus-Host disease (GVHD) is a leading contributor to morbidity and mortality following stem cell transplantation (SCT). Current GVHD prevention methods utilize a combination of immunosuppressive drugs to prevent injury to host tissues. Post-Transplant cyclophosphamide (PTCy) for GVHD prophylaxis initially was shown to be effective in the adult haploidentical SCT setting and later in matched donor SCTs. Implementation of PTCy has increased the utilization of alternative donors in curative SCT. In pediatrics, PTCy for GVHD prophylaxis has recently demonstrated safety and efficacy. However, its widespread use still lags compared with adult use due to variability between institutions and the lack of prospective pediatric trials. In this review, we summarize the pathophysiology of GVHD and PTCy and synthesize studies in adults and children of PTCy for GVHD prophylaxis, which support its use. We focus on using PTCy in haploidentical, matched donor, and umbilical cord blood transplantation settings, and discuss the current and future role of PTCy in pediatrics. Our goal is to inform pediatric SCT physicians regarding the use of PTCy to help reduce morbidity and mortality following transplantation.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32061","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125601","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":"Pediatric Hematology/Oncology Fellowship Match Trends in the United States: Analysis From 2008 to 2025","authors":"Saman K. Hashmi,&nbsp;Fair Berg,&nbsp;Margit K. Mikkelsen,&nbsp;Nidhi Bhatt,&nbsp;Hiroto Inaba,&nbsp;Daniel C. Moreira","doi":"10.1002/pbc.32070","DOIUrl":"10.1002/pbc.32070","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>There is limited research characterizing the supply of pediatric hematology/oncology (PHO) specialists through fellowship training programs. This study examines trends in the PHO match, with a focus on applicant numbers, match outcomes, and representation.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We analyzed data from the National Resident Matching Program for PHO fellowships between 2008 and 2025. Match rates for programs and applicants were analyzed, and Pearson's coefficient was used to correlate continuous variables and time.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>During the study period, the number of available PHO fellowship positions increased from 129 to 194. The number of applicants rose between 2008 and 2016, but declined thereafter through 2025. From 2008 to 2025, the applicant-to-position ratio declined from 1.16 to 0.80 (<i>r</i> = −0.72, <i>p</i> = 0.0008). The overall number of PHO fellowship programs increased from 54 to 77. Between 2008 and 2025, the percentage of unfilled programs increased (<i>r</i> = 0.57, <i>p</i> = 0.01), while the match rate for applicants increased from 87.8% in 2008 to 97.9% in 2025 (<i>r</i> = 0.89, <i>p</i> &lt; 0.0001). US allopathic graduates remained the largest group among matched applicants. The fraction of US allopathic graduates and US international medical graduates (IMGs) matching into PHO has been stable, while the fraction of US osteopathic graduates has overall increased, and that of non-US IMGs has decreased.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Over the past two decades, the applicant-to-position ratio declined in PHO fellowships in the United States, largely secondary to an increase in the number of positions. This growing disparity highlights a misalignment between training capacity and applicant interest, raising concerns about the sustainability of PHO fellowship programs and future workforce planning.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125596","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":"Outcomes of Infant Medulloblastoma Treated With Standard-Dose Chemotherapy—A Single-Institution Experience From a Low-Middle-Income Country","authors":"Gnanamani Senguttuvan,&nbsp;Leenu Lizbeth Joseph,&nbsp;Rikki Rorima John,&nbsp;Geeta Chacko,&nbsp;Ranjani Jayachandran,&nbsp;Krishna Prabhu,&nbsp;Ranjit K. Moorthy,&nbsp;Baylis Vivek Joseph,&nbsp;Rajesh Balakrishnan,&nbsp;Patricia Sebastin,&nbsp;Leni G. Mathew","doi":"10.1002/pbc.32056","DOIUrl":"10.1002/pbc.32056","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Infant medulloblastoma (iMBL) is predominantly desmoplastic nodular (DN) or medulloblastoma with extensive nodularity (MBEN) histology, sonic hedgehog (SHH) driven. Intensified chemotherapy (intraventricular chemotherapy or high-dose chemotherapy with autologous stem cell rescue) is recommended to compensate for the omission of radiation. This study aims to analyze the outcomes of iMBL treated without treatment intensification.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This retrospective study was performed on 28 patients with iMBL (children with medulloblastoma &lt;3 years at diagnosis or 3–4 years with DN/MBEN histology) treated between January 2004 and April 2024 in a tertiary care institute in southern India.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The median age at diagnosis and duration of symptoms were 24.5 months and 1 month, respectively. Vomiting (82%) and ataxia (71.4%) were the common presentations. Ventriculoperitoneal shunt or endoscopic third ventriculostomy was done in 57% of patients. Twenty-four patients (∼86%) underwent gross/near-total resection. The most common surgical complication was cerebellar mutism (<i>n</i> = 6, 21%). Half of the cohort had nonmetastatic disease. Sixteen patients had DN/MBEN histology. Only 68% (<i>n</i> = 19) opted to continue postoperative treatment. Chemotherapy protocols used were baby SFOP, HIT SKK, and modified Packer. One fourth (<i>n</i> = 7) received radiation (DN/MBEN = 3; other histology = 4) as a part of their initial treatment. There was no treatment-related mortality. The 5-year event-free survival (EFS) of patients who received postoperative chemotherapy ± RT was 70% (those with DN/MBEN were 82.5% and non-DN/MBEN were 35.7%). The 5-year EFS of patients with DN/MBEN histology who received only chemotherapy (<i>n</i> = 9) was 88.9%.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Managing medulloblastoma in infants is challenging in LMIC, as <b>a</b> significant proportion of families opt against postoperative treatment. Although traditionally considered high risk, good survival can be achieved with non-intensified therapy without an increase in treatment-related mortality, especially in patients with DN/MBEN histology.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145114025","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 Recurrent Spinal Arteriovenous Malformations: Genetic Testing and Novel Therapeutic Approaches","authors":"Valentina Roa Forster,&nbsp;Mildred C. Franco-Liñan,&nbsp;John Ragheb,&nbsp;Ziad Khatib,&nbsp;Jefry Biehler","doi":"10.1002/pbc.32055","DOIUrl":"10.1002/pbc.32055","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125656","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":"Parent and Participant Perspectives on Research Biopsies in Pediatric Solid Malignancies","authors":"Meaghann S. Weaver,&nbsp;Liza-Marie Johnson,&nbsp;Jami S. Gattuso,&nbsp;Mary Caples,&nbsp;Alberto Pappo,&nbsp;Samira Deshpande,&nbsp;Belinda Mandrell","doi":"10.1002/pbc.32059","DOIUrl":"10.1002/pbc.32059","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Purpose</h3>\u0000 \u0000 <p>Federal agencies recently released draft recommendations for research biopsies with special considerations for children. Little is known about parent or patient perspectives on research biopsy practices. The purpose of this study was to quantify acceptance or non-acceptance of a research biopsy, to explore the decisional reasons, and to measure post-procedure decisional congruence or regret.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patients and Methods</h3>\u0000 \u0000 <p>Participants aged 2–21 years diagnosed with a recurrent or progressive solid tumor amenable to biopsy (based on safety review team) were eligible. This paper reports findings from a pre- and post-questionnaire to describe their acceptance or non-acceptance of the research biopsy procedure.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>All approached candidates agreed to participate (<i>n</i> = 8). Reasons for participation in the research biopsy included altruism, personal benefit, convenience of a concurrent procedure, and diagnostic goals. The average distress score for the research biopsy pre-procedure was 2.4 (1 = not distressed, 5 = very distressed). Participants endorsed a high level of decisional congruence and a low level of regret post-biopsy using a validated Decisional Regret Instrument. Participants reported that the biopsy was the right decision (average 4.3/5-point scale) and they would choose the same again (average 4.3/5). Participants reported low regret (average 1.7/5) and low perception of harm associated with the research biopsy decision (average 1.3/5).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Parents may consider research biopsies acceptable under certain circumstances and may maintain decisional congruence post-procedure. National guidelines and ethical considerations can help navigate the complexities of research biopsies in children. Best practice-informed consent processes are necessary due to the existence of therapeutic misconceptions.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145092295","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}