Pediatric Blood & Cancer最新文献

{"title":"Development and Validation of a Prediction Model for Severe Pre-Engraftment Syndrome During the Treatment of Unrelated Umbilical Cord Blood Transplantation in Pediatric Patients.","authors":"Shiyuan Wang, Lipeng Liu, Yuanyuan Ren, Xia Chen, Yang Wan, Xiaolan Li, Wenyu Yang, Ye Guo, Xiaofan Zhu, Fang Liu","doi":"10.1002/pbc.31733","DOIUrl":"https://doi.org/10.1002/pbc.31733","url":null,"abstract":"<p><strong>Background: </strong>Pre-engraftment syndrome (PES) is a common immune response during the early stages of umbilical cord blood transplantation (UCBT), but the severity classification of PES remains uncertain.</p><p><strong>Objectives: </strong>To develop and validate a predictive model for PES severity in pediatric patients receiving UCBT.</p><p><strong>Study design: </strong>This retrospective study (2017-2025) included 123 pediatric patients (training cohort) and 41 external validation cases. Uni- and multivariable analysis was conducted to assess the potential parameters affecting PES severity. Based on the multivariable Cox model, the optimal model's performance was illustrated using a nomogram and evaluated through discrimination and calibration.</p><p><strong>Results: </strong>Infused CD34⁺ counts (hazard ratio [HR]: 1.249, 95% confidence interval [CI]: 1.037-1.503, p < 0.05), donor chimerism on day 7 (HR: 1.025, 95% CI: 1.010-1.040, p < 0.05), and serum IL-6 level on symptoms onset (HR: 1.101, 95% CI: 1.000-1.123, p < 0.05) were identified as independent risk factors. The predictive performance of this nomogram model was evaluated by C-index and AUC, with C-index of 0.754 (95% CI: 0.693-0.815) and 9-day and 14-day AUC of 0.757 (95% CI: 0.655-0.859) and 0.856 (95% CI: 0.699-0.981), respectively. High-risk patients (score ≥104.9) had higher 9-day severe PES incidence (65.4% vs. 11.4%, p < 0.05) and Grade II-IV/III-IV aGVHD rates (84.2%/59.9% vs. 48.2%/28.2%, p < 0.05). External validation in an independent cohort (n = 41) displayed a C-index of 0.821 (95% CI: 0.689-0.953) and 14-day AUC of 0.933 (95% CI: 0.873-0.993).</p><p><strong>Conclusion: </strong>This predictive model could predict the severity of PES in pediatric patients undergoing UCBT, thereby promoting preemptive treatment.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31733"},"PeriodicalIF":2.4,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144010285","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 Pediatric Living Donor Liver Transplantation for Acute Liver Failure Due to Non-transplant-Related Severe Sinusoidal Obstruction Syndrome After Adjuvant Chemotherapy for Solid Organ Malignancy.","authors":"Hiroki Hirao, Masaki Honda, Kaori Isono, Toru Goto, Kazuya Hirukawa, Yuto Sakurai, Yasuhiko Sugawara, Katsuyuki Sagishima, Tadashi Anan, Shirou Matsumoto, Taizo Hibi","doi":"10.1002/pbc.31751","DOIUrl":"https://doi.org/10.1002/pbc.31751","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31751"},"PeriodicalIF":2.4,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144027064","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":"Calling to Safeguard the Future of Classical Hematology.","authors":"Yves D Pastore, Victoria Price, Sara Israels, Anthony K C Chan, Mark Belletrutti, Aisha Bruce, Ewurabena Simpson, Lynette Bowes, Soumitra Tole, Roona Sinha, Josee Brossard, Caroline Malcomson, Laura Wheaton, Meera Rayar","doi":"10.1002/pbc.31740","DOIUrl":"https://doi.org/10.1002/pbc.31740","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31740"},"PeriodicalIF":2.4,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143974400","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":"Hypersensitivity to Recombinant Erwinia Asparaginase (JZP458) in Acute Lymphoblastic Leukemia and Management With Desensitization.","authors":"Jason Catanzaro, John van Doorninck, Mariana Castells","doi":"10.1002/pbc.31738","DOIUrl":"https://doi.org/10.1002/pbc.31738","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31738"},"PeriodicalIF":2.4,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144006946","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":"Venetoclax Alone or in Combination With Chemotherapy in Paediatric and Adolescent/Young Adult Patients With Relapsed/Refractory Acute Myeloid Leukaemia.","authors":"Seth E Karol, Seong L Khaw, C Michel Zwaan, Andre Baruchel, Henrique Bittencourt, Todd M Cooper, Christian Flotho, Christopher Fraser, Christopher J Forlenza, Kelly C Goldsmith, Margaret E Macy, Daniel A Morgenstern, Maureen M O'Brien, Arnaud Petit, David S Ziegler, Dirk Reinhardt, Joseph T Opferman, Jeffrey E Rubnitz, Maika Onishi, Diana R Dunshee, Fengjiao Dunbar, Deeksha Vishwamitra, Jeremy A Ross, Xin Chen, Kristina Unnebrink, Maja Kammerlander, Ahmed Hamed Salem, Tammy L Palenski, Gauri Sunkersett, Andrew E Place","doi":"10.1002/pbc.31714","DOIUrl":"https://doi.org/10.1002/pbc.31714","url":null,"abstract":"<p><strong>Background: </strong>Venetoclax is a potent, oral BCL-2 inhibitor approved as combination therapy for the treatment of adults with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy. This study evaluated the safety and preliminary efficacy of venetoclax alone or combined with chemotherapy in paediatric and adolescent/young adult patients with relapsed/refractory AML.</p><p><strong>Procedure: </strong>In this phase 1, open-label, two-part, multicentre study, paediatric and adolescent/young adult patients (<25 years of age) with relapsed/refractory AML were treated with venetoclax alone or in combination with hypomethylating agents or cytarabine. The study is registered with ClinicalTrials.gov, NCT03236857.</p><p><strong>Results: </strong>A total of 37 patients received treatment with either venetoclax as a monotherapy (n = 3) or in combination with decitabine (n = 5), azacitidine (n = 19), low-dose cytarabine (n = 1) or high-dose cytarabine (HDAC; n = 9). Febrile neutropenia (57%), hypokalaemia (38%), and thrombocytopenia (35% [thrombocytopenia, 19%; platelet count decreased, 16%]) were the most common grade 3/4 treatment-emergent adverse events. Across all venetoclax combinations, the overall response rate (ORR) was 24% (9/37), and the median duration of response was 2.6 months (95% CI, 0.5-7.9). Among the combinations, ORR was 44% with venetoclax plus HDAC and 21% with venetoclax plus azacitidine. In biomarker-evaluable patients, responses to venetoclax plus chemotherapy were observed in patients harbouring mutations across a range of functional classifications and heterogeneous BH3 family member dependencies.</p><p><strong>Conclusions: </strong>Venetoclax alone or combined with chemotherapy was well tolerated in paediatric and adolescent/young adult patients with relapsed/refractory AML, with promising, although transient, responses with venetoclax plus HDAC or azacitidine.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31714"},"PeriodicalIF":2.4,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144030905","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 Cure We Seek.","authors":"David Spencer Mangum","doi":"10.1002/pbc.31735","DOIUrl":"https://doi.org/10.1002/pbc.31735","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31735"},"PeriodicalIF":2.4,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144025581","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 Course of Pearson Syndrome.","authors":"Miki Murakoshi, Miho Ashiarai, Shinsuke Hirabayashi, Akira Nishimura, Rintaro Ono, Hiroki Yoshihara, Yosuke Hosoya, Atsushi Manabe, Miwa Ozawa, Daisuke Hasegawa","doi":"10.1002/pbc.31736","DOIUrl":"https://doi.org/10.1002/pbc.31736","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31736"},"PeriodicalIF":2.4,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144007013","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":"Incident Stroke in Pediatric Sickle Cell Anemia Despite Overall Improved Transcranial Doppler Velocity in a Ugandan Hydroxyurea Trial: Antecedent and Ongoing Risks.","authors":"Bill Wambaka, Amiirah Mpungu, Vincent Mboizi, Dennis Kalibbala, Grace Nambatya, Susan Murungi, Maxencia Kabatabaazi, Maria Nakafeero, Phillip Kasirye, Deogratias Munube, Ruth Namazzi, Richard Idro, Nancy S Green","doi":"10.1002/pbc.31722","DOIUrl":"https://doi.org/10.1002/pbc.31722","url":null,"abstract":"<p><strong>Introduction: </strong>Transcranial Doppler ultrasound (TCD) screening for primary stroke prevention in children with sickle cell anemia (SCA) was established in higher-resource regions, targeting interventions for highest velocity (\"abnormal\"). We sought to identify additional stroke risk factors in Uganda.</p><p><strong>Methods: </strong>We conducted a 30-month open-label single-arm Ugandan hydroxyurea trial, dose-escalated to maximum-tolerated dose, aimed to test brain protection for children aged 3-9 years with SCA. Study procedures included history, clinical stroke examination, and prospective TCD and laboratory assessments.</p><p><strong>Results: </strong>Overall, 264 children received study HU, mean age 5.6 ± 1.7, hemoglobin 7.8 ± 1.2 g/dL, fetal hemoglobin (HbF) 11.9% ± 8.1%, enrolment TCD mean TAMV 148.4 ± 29.3 cm/sec; 15 (5.7%) had abnormal TCD. The mean TAMV at trial completion was 131.9 ± SD 25.7 cm/sec. Four participants without abnormal enrolment TCD developed acute stroke within the initial 16 months (incidence 0.62 per 100 person-years): two had enrolment HbF ≤3.1%, two had low oxygen saturation (90%), and one had recurring severe anemia necessitating multiple transfusions. Apparent stroke precipitants were severe malaria, acute chest syndrome, recent pain crisis, or uncertain cause. At trial completion, eight additional participants had a higher risk TCD category than at enrolment.</p><p><strong>Conclusion: </strong>The effectiveness of TCD screening for stroke prevention may vary by region, as no participant with an incident stroke was at the highest risk. Antecedent and/or ongoing SCA-related risks of anemia, low HbF, hypoxemia, infections, and/or disease complications likely contributed to stroke despite trial HU. Results suggest that TCD alone may not fully identify the highest stroke risk in the region, and there is a need for primary stroke prevention from early childhood and continuous hydroxyurea therapy.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31722"},"PeriodicalIF":2.4,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144023125","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":"Baseline Characteristics of Parents Enrolled in Children's Oncology Group Study ACCL20N1CD: Financial Toxicity During Treatment for Pediatric Acute Lymphoblastic Leukemia in the United States.","authors":"Sheila Judge Santacroce, Melissa P Beauchemin, Olivia Ponce, Lingyun Ji, Subhash Ramakrishnan, Kira Bona, Yudy Muñeton-Castaño, Joanna M Robles, Sarah Vargas, Crystal De Los Santos, Beth Fisher, Sarah Alexander, Lillian Sung, Susan K Parsons","doi":"10.1002/pbc.31710","DOIUrl":"https://doi.org/10.1002/pbc.31710","url":null,"abstract":"<p><strong>Background: </strong>Costs to parents and associated financial distress may contribute to observed health disparities in pediatric oncology. We describe financial distress in a cohort of parents with children newly diagnosed with acute lymphoblastic leukemia (ALL) and identify factors contributing to high financial distress.</p><p><strong>Procedures: </strong>Settings were 28 Children's Oncology Group practices in the National Cancer Institute Community Oncology Research Program. We included English- or Spanish-speaking parents of children with newly diagnosed ALL. Analyses focused on baseline surveys completed by parents during their child's ALL induction therapy. Survey items asked about socio-demographics, household material hardships, financial burden, financial distress, and financial coping behaviors.</p><p><strong>Results: </strong>The cohort included 104 parents. Most were female (87; 83.7%), White (68; 65.4%), non-Hispanic (50; 48.1%), and paid for their child's care with Medicaid or CHIP (60; 57.7%). Financial burden totaled greater than 15% of monthly gross household income for half (54, 51.5%), and 45 (43%) indicated high financial distress (score 1-4). Prevalent coping behaviors with health implications included cutting back on groceries (62, 59.6%) or other necessities (61, 58.7%). Parents who had poverty-level income (p = 0.0009), paid with Medicaid or CHIP (OR 3.0 [CI: 1.26, 7.13], p = 0.01), were unemployed (OR 2.5 [CI: 1.1, 5.7], p = 0.04), or lived where more than 50% of residents had socioeconomic disadvantages (OR 3.0 [CI: 1.13, 8.05], p = .03) were more likely to indicate high financial distress than others.</p><p><strong>Conclusions: </strong>During their child's ALL induction therapy, sizeable proportions of parents exhibited high financial burdens, high levels of financial distress, and multiple financial coping behaviors with health implications.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31710"},"PeriodicalIF":2.4,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144005549","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":"Spiritual Communication Between Pediatric Oncologists, Caregivers, and Patients With Brain Tumors.","authors":"Alexandra K Superdock, Amy S Porter, Walter Spears, Justin N Baker, Jennifer W Mack, Erica C Kaye","doi":"10.1002/pbc.31721","DOIUrl":"https://doi.org/10.1002/pbc.31721","url":null,"abstract":"<p><strong>Background: </strong>Spiritual care is recognized as an essential component of standard care for children with cancer and their families. Oncologists lack training in navigating spirituality discussions in primary cancer care. The current landscape of spiritual dialog during clinical oncology encounters remains understudied.</p><p><strong>Procedures: </strong>This qualitative study described the frequency, context, and content of religious and spiritual communication between pediatric neuro-oncologists, caregivers, and patients with brain tumors during disease re-evaluation encounters. This study is part of the U-CHAT trial (NCT02846038), a prospective longitudinal investigation of real-time clinical communication across the trajectory of poor-prognosis cancer. Patients with brain tumors and their caregivers were eligible if their primary oncologist estimated ≤50% survival. All disease re-evaluation encounters for enrolled patients were audio-recorded and underwent rapid qualitative analysis to identify, summarize, and synthesize religious and spiritual communication.</p><p><strong>Results: </strong>Religious or spiritual references were identified in 23 (18%) of 129 total encounters. References usually comprised a single religious or spiritual term, with \"prayer\"-related language representing the majority of references. Caregivers introduced spiritual dialog more often than oncologists (65% vs. 35%). References were most often identified in the context of a discussion about an uncertain or uncontrollable future.</p><p><strong>Conclusions: </strong>Results suggest oncologists rarely integrate spiritual care into disease re-evaluation discussions. In light of recommendations from multiple consensus groups to integrate spiritual care into cancer care, future work should explore how generalist spiritual care training could better equip oncologists to attend to spiritual needs that arise along the cancer trajectory.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31721"},"PeriodicalIF":2.4,"publicationDate":"2025-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143976569","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}