Pediatric Blood & Cancer最新文献

{"title":"Feasibility of Atezolizumab in Combination With Chemotherapy for Children With Relapsed or Refractory Solid Tumors","authors":"Matthew E. Campbell,&nbsp;Sonja Stutzman,&nbsp;Sharon Primeaux,&nbsp;Deseray Sida,&nbsp;Minjae Lee,&nbsp;Avanthi T. Shah,&nbsp;Arhanti Sadanand,&nbsp;Elizabeth Sokol,&nbsp;Natalie B. Collins,&nbsp;Brian Turpin,&nbsp;Shoba Navai,&nbsp;Katie Albert,&nbsp;Theodore W. Laetsch,&nbsp;Dinesh Rakheja,&nbsp;Kenneth S. Chen,&nbsp;David E. Gerber,&nbsp;Andrew Y. Koh","doi":"10.1002/pbc.32046","DOIUrl":"10.1002/pbc.32046","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Combining immune checkpoint inhibitors (ICI) with chemotherapy may improve treatment response in children with solid tumors. We sought to determine the feasibility of combining vincristine, irinotecan, and temozolomide with the ICI atezolizumab in children with relapsed or refractory solid tumors (VITAS;).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Patients ≥6 months and ≤18 years old with a relapsed or refractory solid tumor, no prior ICI, and evaluable disease per RECIST v1.1 were eligible for the Phase I cohort (NCT04796012). Patients received atezolizumab 15 mg/kg on Day 1, vincristine 1.5 mg/m² on Day 1, irinotecan 50 mg/m² on Days 1–5, and temozolomide 100 mg/m² on Days 1–5 in 21-day cycles. The primary endpoint was the number of patients with dose-limiting toxicities (DLT) in the first two cycles of therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Six patients (median age: 14 years) with rhabdomyosarcoma (<i>n</i> = 3), osteosarcoma (<i>n</i> = 2), and Ewing sarcoma (<i>n</i> = 1) received therapy and were evaluable for toxicity. Patients received a median of seven (range: 2–20) cycles of treatment. No patients experienced a DLT. One patient experienced Grade 2 immune-related colitis. Four patients experienced Grade ≥3 adverse events (decreased neutrophil count, febrile neutropenia, weight loss, anorexia). One patient with rhabdomyosarcoma had a sustained partial response through 16 cycles. One patient with relapsed pulmonary osteosarcoma has ongoing stable disease through 20 cycles.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Atezolizumab combined with vincristine, irinotecan, and temozolomide was feasible and well tolerated in children with solid tumors. Efficacy of this regimen is now being assessed in relapsed and refractory rhabdomyosarcoma in an ongoing Phase II cohort.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32046","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040840","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":"Multidisciplinary Approach to the Diagnosis and Treatment of Ewing Sarcoma of the Kidney With an Extremely Rare FUS::ETV4 Genetic Rearrangement","authors":"Natalia D. Faseeva,&nbsp;Ivan M. Kharkov,&nbsp;Grigory A. Raskin,&nbsp;Ilya V. Sidorov,&nbsp;Aleksey A. Chernev,&nbsp;Konstantin F. Boyko,&nbsp;Natalia Y. Usman,&nbsp;Sergey V. Aleksandrov,&nbsp;Alexander A. Zakharenko,&nbsp;Ilya A. Paltyshev,&nbsp;Nikolay A. Vorobyov,&nbsp;Alexander E. Druy","doi":"10.1002/pbc.32054","DOIUrl":"10.1002/pbc.32054","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040918","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 Impact of Abnormal Uterine Bleeding on Adolescents’ Quality of Life: Results of the Adolescent Menstrual Bleeding Questionnaire","authors":"Aytül Temuroğlu,&nbsp;Esra Dişçi,&nbsp;Büşra Demir,&nbsp;Ayşe Özden","doi":"10.1002/pbc.32051","DOIUrl":"10.1002/pbc.32051","url":null,"abstract":"&lt;div&gt;\u0000 \u0000 \u0000 &lt;section&gt;\u0000 \u0000 &lt;h3&gt; Objective&lt;/h3&gt;\u0000 \u0000 &lt;p&gt;To determine the extent to which abnormal uterine bleeding affects the quality of life in adolescents.&lt;/p&gt;\u0000 &lt;/section&gt;\u0000 \u0000 &lt;section&gt;\u0000 \u0000 &lt;h3&gt; Materials and Methods&lt;/h3&gt;\u0000 \u0000 &lt;p&gt;The study included a total of 69 patients who presented to our clinic with menstrual bleeding lasting more than 7 days, occurring more frequently than every 21 days, and showing signs of increased blood loss within a cycle. Following the collection of detailed medical histories and physical examinations, initial diagnostic tests were performed, including a complete blood count, coagulation tests (prothrombin time, activated partial thromboplastin time, and fibrinogen), and iron parameters (serum iron, ferritin, and total iron-binding capacity). After obtaining informed consent, adolescents who presented to general pediatrics, pediatric endocrinology, and pediatric hematology clinics with complaints of abnormal uterine bleeding were asked to complete the Adolescent Menstrual Bleeding Questionnaire.&lt;/p&gt;\u0000 &lt;/section&gt;\u0000 \u0000 &lt;section&gt;\u0000 \u0000 &lt;h3&gt; Results&lt;/h3&gt;\u0000 \u0000 &lt;p&gt;The mean age of the 69 patients included in the study was 14.95 years (range: 11–18), and the mean age at menarche was 12.2 years (range: 9–16). Of the patients, 28.9% (&lt;i&gt;n&lt;/i&gt; = 20) had been admitted to the hospital for the first time due to bleeding, and 71.1% (&lt;i&gt;n&lt;/i&gt; = 49) had been admitted before and had used some treatments. During menstruation, 23.2% (&lt;i&gt;n&lt;/i&gt; = 16) of the patients reported severe pain, 43.5% (&lt;i&gt;n &lt;/i&gt;= 30) reported moderate pain, and 18.8% (&lt;i&gt;n&lt;/i&gt; = 13) reported mild pain. School absenteeism was observed in 36.2% (&lt;i&gt;n&lt;/i&gt; = 25) of the patients. Additionally, 63.8% (&lt;i&gt;n&lt;/i&gt; = 44) of the patients reported avoiding at least 1 day of family-related activities (such as household chores or shopping). The mean questionnaire score for patients with heavy bleeding was 33.9 ± 1.6, while those with light to moderate bleeding had a mean score of 27 ± 1.8 (&lt;i&gt;p&lt;/i&gt; = 0.005). As a result, 47.8% (&lt;i&gt;n&lt;/i&gt; = 33) of the patients scored below 30, and 52.2% (&lt;i&gt;n&lt;/i&gt; = 36) scored above 30. When patients scoring above and below 30 were compared, the need for adult diaper use, incidence of bleeding through outer clothing, school absenteeism, and level of activity avoidance were significantly higher in those scoring above 30. The highest score was observed in patients who had never received treatment [35.5 (±11.6) (11–61)] and the lowest score was observed in patients receiving iron and combined oral contraceptive pills (OCPs) treatment [28.6 (± 10.06) (10–44)] (&lt;i&gt;p&lt;/i&gt; = 0.058) Anxiety levels were rated at 10 points by 36.2% (&lt;i&gt;n&lt;/i&gt; = 25) of the patients. The mean anxiety score of patients with an aMBQ score &gt; 30 was 8.64 ± 2, and that of those with an aMBQ","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040850","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":"CD123-Targeted Therapy to Achieve Negative Measurable Residual Disease in Pediatric Blastic Plasmacytoid Dendritic Cell Neoplasm Prior to Transplantation","authors":"Flavia Gava,&nbsp;Luiz Fernando Bazzo Catto,&nbsp;Marilia Bazzo Catto,&nbsp;Ana Luiza Morais,&nbsp;Maristella Bergamo Francisco dos Reis,&nbsp;Thiago Eleuterio Gonçalves,&nbsp;Luiz Guilherme Darrigo Jr,&nbsp;Carlos Alberto Scrideli,&nbsp;Elvis Terci Valera","doi":"10.1002/pbc.32034","DOIUrl":"10.1002/pbc.32034","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040878","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":"Ifosfamide Neurotoxicity Revisited: Recurrence Triggered by Cyclophosphamide—A Rare Clinical Insight","authors":"Nishkala Uday Rao,&nbsp;Appaji L,&nbsp;Prakruthi S. Kaushik,&nbsp;Priyadarashani Mishra,&nbsp;Arun Kumar","doi":"10.1002/pbc.32048","DOIUrl":"10.1002/pbc.32048","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145033898","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 Blinatumomab Efficacy in RAM Immunophenotype/CBFA2T3::GLIS2 Fusion AML","authors":"Maya Ball-Burack,&nbsp;Andrew Menssen,&nbsp;Lisa Eidenschink Brodersen,&nbsp;Arun Nalla,&nbsp;Michael Loken,&nbsp;Laura Pardo,&nbsp;Ann Dahlberg,&nbsp;Chad Hudson,&nbsp;Soheil Meshinchi,&nbsp;Katherine Tarlock","doi":"10.1002/pbc.32035","DOIUrl":"10.1002/pbc.32035","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145033828","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":"Characterizing Difficulty and Life Disruption During B-Acute Lymphoblastic Leukemia Therapy From the Perspective of Parents: A Survey Study.","authors":"Kellee N Parker, Sarah W Alexander, Lisa M Jacola, Kimberly A Buff, Kyobin Hwang, Elham Hashemi, Gregory J Stoddard, Lindsay A Jibb","doi":"10.1002/pbc.32041","DOIUrl":"https://doi.org/10.1002/pbc.32041","url":null,"abstract":"<p><strong>Purpose: </strong>Children with B-acute lymphoblastic leukemia (B-ALL) treated in resource-intensive settings have a high likelihood of cure, but therapy is long, burdensome, and associated with substantial toxicities. Understanding parents' perceptions of the most disruptive and difficult aspects of B-ALL treatment is critical to future improvements in care. We aimed to understand which child side effects, chemotherapeutic agents, and aspects of leukemia care are rated difficult or disruptive by parents, and variations based on parent or child characteristics.</p><p><strong>Methods: </strong>Parents of children (1-19 years) currently or previously treated for B-ALL who are members of the Momcology pediatric cancer support and advocacy organization were invited to complete an online survey on difficult and disruptive aspects associated with their child's diagnosis. Data were analyzed descriptively, and inferential statistical tests evaluated characteristic-based differences.</p><p><strong>Results: </strong>Parents of 442 children completed the survey. Nausea/vomiting was the most commonly reported difficult side effect during pre-maintenance therapy (55.4%), followed by decreased energy (41.4%) and neuropathy (40.7%). Mood changes were the most difficult side effect during maintenance therapy (29.3%). The extent of difficulty associated with each side effect reported was high. Most parents (79.6%) rated oral corticosteroids as the most difficult chemotherapeutic agent. The components of care most difficult and disruptive were unplanned hospital visits (79.9%) and compromised immunity (76.9%). Parent-rated difficulties significantly varied by child age at diagnosis.</p><p><strong>Conclusions: </strong>Parents of children with B-ALL report substantial child and family difficulties that are directly attributable to leukemia treatment. These findings should inform areas of investigation for optimizing treatment regimens and supportive care.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32041"},"PeriodicalIF":2.3,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145023942","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":"Increased Plasma ST2 Levels Precede the Development of Severe Acute GvHD and Chronic GvHD After Pediatric Hematopoietic Stem Cell Transplantation","authors":"Nakisa Kamari-Kany,&nbsp;Sarah Weischendorff,&nbsp;Marianne Ifversen,&nbsp;Denise Elbæk Horan,&nbsp;Christian Enevold,&nbsp;Katrine Kielsen,&nbsp;Klaus Müller","doi":"10.1002/pbc.31995","DOIUrl":"10.1002/pbc.31995","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>The suppressor of tumorigenesis 2 (ST2) has emerged as one of the most promising biomarkers for predicting mortality of acute graft-versus-host disease (aGvHD) when measured at the onset of symptoms, but detailed time course studies are needed to understand the potential of ST2 as a risk marker of both aGvHD and chronic graft-versus-host disease (cGvHD), potentially allowing pre-emptive adjustment of immunosuppressive treatment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>We measured ST2 levels in 117 children undergoing standard hematopoietic stem cell transplantation (HSCT) before conditioning and at regular intervals post-HSCT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>ST2 levels were significantly increased from Day +7 in patients developing aGvHD of any grade (no GvHD: 23.6 ng/mL; Grade I: 31.9 ng/mL; Grade II: 33.2 ng/mL; Grade III–IV: 59.1 ng/mL; <i>p </i>&lt; 0.0001) and in patients developing aGvHD with visceral involvement (no GvHD: 23.6 ng/mL; skin only aGvHD: 24.9 ng/mL; GI and/or liver aGvHD: 59.8 ng/mL; <i>p </i>&lt; 0.0001). The association between ST2 levels and aGvHD Grade II–IV was confirmed in a multivariable logistic regression analysis, adjusting for diagnosis, conditioning regimen, and donor type (OR = 1.99 per doubling in ST2, 95% confidence interval [CI] = 1.40–2.92; <i>p</i> = 0.00025). Patients developing cGvHD had significantly higher ST2 levels before conditioning and from Day +21 to Day +180 (all <i>p </i>&lt; 0.05). ST2 levels at Day +90 were significantly associated with later development of cGvHD after adjusting for diagnosis, conditioning regimen, donor type, and prior aGvHD (HR = 2.1 per doubling in ST2, 95% CI = 1.51–2.91, <i>p </i>&lt; 0.0001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This study confirms ST2 as a biomarker reflecting risk of aGvHD and prognosis in pediatric allogeneic HSCT, and our findings indicate a role of ST2 as an early risk marker of cGvHD.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31995","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016084","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":"Successful Treatment and Prognostic Analysis of a Pediatric B-Cell Acute Lymphoblastic Leukemia With a Rare CREBBP::ZNF362 Fusion Gene","authors":"Meizhu Luo,&nbsp;Xiaoyong Chen,&nbsp;Xiaoying Fu,&nbsp;Zhenhu Lin,&nbsp;Qiang Yao","doi":"10.1002/pbc.32043","DOIUrl":"10.1002/pbc.32043","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145023950","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":"Moyamoya Syndrome in Patients With Down Syndrome and Acute Lymphoblastic Leukemia","authors":"Robert Lisac,&nbsp;Ryan J. Summers,&nbsp;D. John Bergsagel,&nbsp;Frank G. Keller,&nbsp;Bryan Philbrook,&nbsp;David Wrubel,&nbsp;Gary Woods,&nbsp;Sunil S. Raikar","doi":"10.1002/pbc.32045","DOIUrl":"10.1002/pbc.32045","url":null,"abstract":"<div>\u0000 \u0000 <p>Moyamoya syndrome (MMS) is a chronic vasculopathy characterized by progressive stenosis of intracerebral arteries, leading to an increased risk of stroke. Children with Down syndrome (DS) are at an increased risk of co-occurring medical conditions, including MMS and leukemia. We report four patients with the triad of DS, MMS, and acute lymphoblastic leukemia (ALL). Two patients had a diagnosis of MMS prior to developing ALL, whereas two were diagnosed with MMS during ALL therapy. This series highlights the need to remain cognizant of MMS in patients with DS and ALL, particularly in those with acute neurologic changes.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016020","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}