Pediatric Blood & Cancer最新文献

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Feasibility of transcranial Doppler to evaluate vasculopathy among survivors of childhood brain tumors exposed to cranial radiation therapy 经颅多普勒评估接受头颅放射治疗的儿童脑肿瘤幸存者血管病变的可行性。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-20 DOI: 10.1002/pbc.31392
Daniel C. Bowers, Mark D. Johnson
{"title":"Feasibility of transcranial Doppler to evaluate vasculopathy among survivors of childhood brain tumors exposed to cranial radiation therapy","authors":"Daniel C. Bowers,&nbsp;Mark D. Johnson","doi":"10.1002/pbc.31392","DOIUrl":"10.1002/pbc.31392","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>The ability of transcranial Doppler (TCD) to detect asymptomatic cerebrovascular disease among childhood brain tumor survivors following exposure to cranial radiation therapy has not been established.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Survivors of childhood brain tumors, more than 3 years since diagnosis and exposed to greater than 30 Gy cranial radiation, underwent a history and physical exam, laboratory biomarkers of cerebrovascular disease (cholesterol, high-density lipoprotein cholesterol (HDL), low-density lipoprotein cholesterol (LDL), high-sensitivity CRP, hemoglobin A1C, apoprotein A, and apoprotein B), and a TCD evaluation of their cerebral arteries.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>In all 165 cerebral arteries from 13 patients (medulloblastoma = 10; germ cell tumor = 3; females = 5; mean age at diagnosis = 8.0 years; mean age at time of study = 20.9 years) were examined. Twenty-eight of 165 (17%) were considered abnormal by pre-specified criteria. Total 114 cerebral arteries from 13 patients were assessed for greater than 50% stenosis velocities. Arteries most likely to be considered abnormal included the distal bilateral vertebral arteries (right 38%, left 30%), basilar artery 30%, bilateral siphon internal carotid arteries (right 30%, left 23%), bilateral middle cerebral arteries (23% bilaterally), and bilateral anterior cerebral arteries (7% bilaterally). Two vessels had mean flow velocities consistent with <span></span><math>\u0000 <semantics>\u0000 <mo>≥</mo>\u0000 <annotation>$ ge $</annotation>\u0000 </semantics></math>50% stenosis (1.8%). No vessels were found to have greater than 80% stenosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>TCD may be a useful and practical tool to examine asymptomatic cerebrovascular disease among childhood brain tumor survivors after exposure to cranial radiation therapy. Posterior circulation vessels appear to have the highest burden of disease in this group of brain tumor survivors, a majority of whom had medulloblastoma.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31392","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472044","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}
引用次数: 0
How you play the game 如何玩游戏
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-18 DOI: 10.1002/pbc.31405
Andrea Watson
{"title":"How you play the game","authors":"Andrea Watson","doi":"10.1002/pbc.31405","DOIUrl":"10.1002/pbc.31405","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472046","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}
引用次数: 0
Reply to: Comment on: Perinatal risk factors and neurocognitive outcomes in children and adolescents with sickle cell disease 答复评论:患有镰状细胞病的儿童和青少年的围产期风险因素和神经认知结果。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-18 DOI: 10.1002/pbc.31391
Jennifer Longoria, Andrew Heitzer, Jane Hankins
{"title":"Reply to: Comment on: Perinatal risk factors and neurocognitive outcomes in children and adolescents with sickle cell disease","authors":"Jennifer Longoria,&nbsp;Andrew Heitzer,&nbsp;Jane Hankins","doi":"10.1002/pbc.31391","DOIUrl":"10.1002/pbc.31391","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472058","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}
引用次数: 0
Comment on: Perinatal risk factors and neurocognitive outcomes in children and adolescents with sickle cell disease 评论:患有镰状细胞病的儿童和青少年的围产期风险因素和神经认知结果。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-18 DOI: 10.1002/pbc.31318
Wei-Zhen Tang, Wen-Xin Deng, Tai-Hang Liu
{"title":"Comment on: Perinatal risk factors and neurocognitive outcomes in children and adolescents with sickle cell disease","authors":"Wei-Zhen Tang,&nbsp;Wen-Xin Deng,&nbsp;Tai-Hang Liu","doi":"10.1002/pbc.31318","DOIUrl":"10.1002/pbc.31318","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472038","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}
引用次数: 0
Correction to “Delivery of re-irradiation and complex palliative radiotherapy using proton therapy in pediatric cancer patients” 对 "利用质子疗法为儿科癌症患者提供再照射和复合姑息放疗 "的更正。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-18 DOI: 10.1002/pbc.31403
{"title":"Correction to “Delivery of re-irradiation and complex palliative radiotherapy using proton therapy in pediatric cancer patients”","authors":"","doi":"10.1002/pbc.31403","DOIUrl":"10.1002/pbc.31403","url":null,"abstract":"<p>Berlin E, Eisenberg R, Hill-Kayser C, et al. Delivery of re-irradiation and complex palliative radiotherapy using proton therapy in pediatric cancer patients. <i>Pediatr Blood Cancer</i>. 2023;70:e30708. doi:10.1002/pbc.30708</p><p>Eva Berlin and Rachel Eisenberg should be listed as co-first authors and equal contribution.</p><p>The author byline should read:</p><p>This should have read “Eva Berlin*, Rachel Eisenberg*, Christine Hill-Kayser, Robert A. Lustig, Goldie Kurtz, Elizabeth Cummings, Michael LaRiviere *Co-first authors”</p><p>We apologize for this error.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31403","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472040","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}
引用次数: 0
Upfront immunotherapy for synchronous high-grade glioma and B-lymphoma in a pediatric patient with CMMRD syndrome 对一名患有CMMRD综合征的儿科患者进行同步高级别胶质瘤和B淋巴瘤的前期免疫治疗。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-18 DOI: 10.1002/pbc.31399
Thérèse De Vanssay, Nour El Riachy, Caroline Donze, Romain Appay, Didier Scavarda, Benoit Testud, Nicolas Andre, Gabriel Revon-Rivière
{"title":"Upfront immunotherapy for synchronous high-grade glioma and B-lymphoma in a pediatric patient with CMMRD syndrome","authors":"Thérèse De Vanssay,&nbsp;Nour El Riachy,&nbsp;Caroline Donze,&nbsp;Romain Appay,&nbsp;Didier Scavarda,&nbsp;Benoit Testud,&nbsp;Nicolas Andre,&nbsp;Gabriel Revon-Rivière","doi":"10.1002/pbc.31399","DOIUrl":"10.1002/pbc.31399","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472062","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}
引用次数: 0
Prevent Acute Chest Syndrome checklist (PACScheck): A quality improvement initiative to reduce acute chest syndrome 预防急性胸部综合征检查表(PACScheck):减少急性胸部综合征的质量改进措施。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-17 DOI: 10.1002/pbc.31378
Kerry Morrone, Kaitlin Strumph, Catherine Pisacano, Jessica Briggs, Rachelle Zipper, Bhaumik B. Patel, Susanna Chang, Wen-Ling Kyon, Kristen Ronca, Miranda Abyazi, Geoffrey Cheng, Leya Schwartz, Jennifer De Los Santos, Janine Keenan, Marina Reznik, Deepa Manwani, Michael L. Rinke
{"title":"Prevent Acute Chest Syndrome checklist (PACScheck): A quality improvement initiative to reduce acute chest syndrome","authors":"Kerry Morrone,&nbsp;Kaitlin Strumph,&nbsp;Catherine Pisacano,&nbsp;Jessica Briggs,&nbsp;Rachelle Zipper,&nbsp;Bhaumik B. Patel,&nbsp;Susanna Chang,&nbsp;Wen-Ling Kyon,&nbsp;Kristen Ronca,&nbsp;Miranda Abyazi,&nbsp;Geoffrey Cheng,&nbsp;Leya Schwartz,&nbsp;Jennifer De Los Santos,&nbsp;Janine Keenan,&nbsp;Marina Reznik,&nbsp;Deepa Manwani,&nbsp;Michael L. Rinke","doi":"10.1002/pbc.31378","DOIUrl":"10.1002/pbc.31378","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Acute chest syndrome (ACS) is a life-threatening complication of sickle cell disease (SCD). The Prevent Acute Chest Syndrome checklist (PACScheck) was created to drive appropriate ordering of opioids, incentive spirometry (IS), intravenous fluids (IVF), evaluation of oxygen desaturation, and bronchodilator use.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Decrease the development of ACS by 5% in a hospitalized pediatric SCD population.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A multidisciplinary team conducted a quality improvement (QI) project between April 2020 and August 2021 on an inpatient pediatric hematology unit. At-risk hospitalizations were patients with SCD who did not have ACS upon hospital admission. PACScheck was implemented and weekly run charts assessed documentation. Process control (p) charts, geometric control (g) charts, and chi-square tests assessed checklist process measures pre- and post-PACScheck. G chart assessed the number of encounters between ACS events.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 483 at-risk hospitalizations were identified in the 12 months prior and 363 during the study period. A g chart demonstrated that fewer encounters developed ACS during PACScheck. A p chart demonstrated that IS documentation increased during PACScheck. A run chart of PACScheck documentation demonstrated a median of 100% documentation at least once per hospitalization during the last six months of the intervention.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Development of ACS can be reduced by implementing a best-practices checklist (PACScheck) on an inpatient pediatric hematology unit with a multidisciplinary team.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472054","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}
引用次数: 0
Oral mucositis assessment in pediatric and adolescent oncological patients: A systematic review 儿童和青少年肿瘤患者的口腔黏膜炎评估:系统综述。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-17 DOI: 10.1002/pbc.31388
Júlia Breda Soares, Amanda de Farias Gabriel, Laura Borges Kirschnick, Vinicius Coelho Carrard, Marina Curra, Lauren Frenzel Schuch, Marco Antonio Trevizani Martins, Manoela Domingues Martins
{"title":"Oral mucositis assessment in pediatric and adolescent oncological patients: A systematic review","authors":"Júlia Breda Soares,&nbsp;Amanda de Farias Gabriel,&nbsp;Laura Borges Kirschnick,&nbsp;Vinicius Coelho Carrard,&nbsp;Marina Curra,&nbsp;Lauren Frenzel Schuch,&nbsp;Marco Antonio Trevizani Martins,&nbsp;Manoela Domingues Martins","doi":"10.1002/pbc.31388","DOIUrl":"10.1002/pbc.31388","url":null,"abstract":"<p>Oral mucositis (OM) is a prevalent acute adverse effect of various cancer treatments. Accurate assessment of OM is vital for effective prevention and treatment strategies. However, a lack of validated pediatric instruments for evaluating OM can lead to unreliable data, and hinder interventional and epidemiological research. This study aims to evaluate the methods used for assessing OM in pediatric oncology patients. A systematic review of four databases and a manual search yielded 113 articles. Nine different scales were identified, with the World Health Organization (WHO) scale being the most commonly used (61.9%). The Children's International Mucositis Evaluation Scale (ChIMES) was used in 7.9% of the studies. Of the 8155 pediatric patients evaluated, 47.7% had both hematological malignancies and malignant solid tumors, while 46% had solely hematological malignancies. Despite the prevalence of the WHO scale, it lacks pediatric-specific criteria. Future OM research should incorporate validated tools like ChIMES for improved pediatric assessment.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472049","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}
引用次数: 0
Auer rod-positive acute leukemia with predominantly lymphoid immunophenotype: Report on 11 cases and review of literature 以淋巴免疫表型为主的奥氏杆阳性急性白血病:11 个病例的报告和文献综述。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-16 DOI: 10.1002/pbc.31394
Ondrej Hrusak, Jitka Stancikova, Elena Vodickova, Tereza Podolska, Anja Möricke, Andishe Attarbaschi, Michael Dworzak, Zuzana Sestakova, Peter Svec, Marketa Kubricanova Zaliova, Iveta Janotova, Ondrej Zapletal, Ester Mejstrikova, Jan Stary
{"title":"Auer rod-positive acute leukemia with predominantly lymphoid immunophenotype: Report on 11 cases and review of literature","authors":"Ondrej Hrusak,&nbsp;Jitka Stancikova,&nbsp;Elena Vodickova,&nbsp;Tereza Podolska,&nbsp;Anja Möricke,&nbsp;Andishe Attarbaschi,&nbsp;Michael Dworzak,&nbsp;Zuzana Sestakova,&nbsp;Peter Svec,&nbsp;Marketa Kubricanova Zaliova,&nbsp;Iveta Janotova,&nbsp;Ondrej Zapletal,&nbsp;Ester Mejstrikova,&nbsp;Jan Stary","doi":"10.1002/pbc.31394","DOIUrl":"10.1002/pbc.31394","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Auer rods (AuRs) are prominent intracellular structures found almost exclusively in myeloid cell malignancies, such as acute myeloid leukemia (AML), chronic and juvenile myelomonocytic leukemia and myelodysplastic syndrome. Extremely rare AuRs have been reported in patients with acute lymphoblastic leukemia (ALL) or among ambiguous lineage leukemia patients with a dominantly lymphoblastic immunophenotype.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>We report diagnostic and follow-up data of an international cohort of 11 children suffering from leukemias with AuRs and with significant presence of T and myeloid markers, majority of whom categorized as early T-cell precursor (ETP, <i>n</i> = 7); or T-ALL (ETP status unknown, <i>n</i> = 2), ALAL (acute leukemia of ambiguous lineage, <i>n</i> = 1), and AML reclassified from ALAL (<i>n</i> = 1). We described other diagnostic details and treatment types and responses. Moreover, we summarize previously published data.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among the four patients who started and remained on ALL-type therapy, all were in the first complete remission, whereas both patients who started and remained on AML-type therapy relapsed and died. Of the patients who followed either a combined ALL/AML protocol (Interfant 06) or who switched from one of the two types of therapy to the other, one patient died, and the remaining four were in first complete remission at the most recent follow-up. We also searched for similar cases in the literature and found only three additional children with nonmyeloid leukemia and AuRs and 10 adults with this type of leukemia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Briefly, ALL- or combined ALL/AML-type therapy may be effective for treating AuR-positive leukemia patients with a lymphoid immunophenotype.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31394","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472029","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}
引用次数: 0
Pediatric patients with von Hippel–Lindau and hemangioblastomas treated successfully with belzutifan 用贝珠替凡治疗冯-希佩尔-林道和血管母细胞瘤的小儿患者获得成功。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2024-10-16 DOI: 10.1002/pbc.31371
Emily Duan, Michael Robinson, Charles Davis, Sumit Pruthi, Christina Shin, Marisa Lewis, Julian A. Martinez-Agosto, Michael B. Gorin, Brian M. Shuch, Debra L. Friedman, Vivian Y. Chang
{"title":"Pediatric patients with von Hippel–Lindau and hemangioblastomas treated successfully with belzutifan","authors":"Emily Duan,&nbsp;Michael Robinson,&nbsp;Charles Davis,&nbsp;Sumit Pruthi,&nbsp;Christina Shin,&nbsp;Marisa Lewis,&nbsp;Julian A. Martinez-Agosto,&nbsp;Michael B. Gorin,&nbsp;Brian M. Shuch,&nbsp;Debra L. Friedman,&nbsp;Vivian Y. Chang","doi":"10.1002/pbc.31371","DOIUrl":"10.1002/pbc.31371","url":null,"abstract":"<p>Hemangioblastoma is the most common tumor associated with von Hippel–Lindau (VHL), and are a leading cause of mortality. We present five pediatric patients with VHL-associated hemangioblastomas treated with belzutifan, a hypoxia-inducible factor 2a (HIF2a) inhibitor. Three patients were started on belzutifan due to vision loss from progressive retinal hemangioblastomas. Within one year of treatment, all three patients had improvement in hemangioblastoma size and visual acuity. For patients with intracranial lesions, belzutifan resulted in an improvement in neurologic symptoms and hemangioblastoma size. Four patients experienced grade 1–2 anemia and two patients required a dose reduction. Our report suggests that belzutifan can be an effective therapy for pediatric, adolescent, and young adult patients with VHL-associated hemangioblastomas.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31371","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472053","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}
引用次数: 0
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