Pediatric Blood & Cancer最新文献

{"title":"Revisiting the Incidence of Hypercalcemia in Contemporary Diagnoses of Pediatric Patients With Solid Tumors.","authors":"Felicia Y Ho, Dajana Alku, Chalinee Monsereenusorn, Nan Chen, Wendy B London, Allison F O'Neill","doi":"10.1002/pbc.32044","DOIUrl":"https://doi.org/10.1002/pbc.32044","url":null,"abstract":"<p><strong>Background: </strong>Hypercalcemia is rarely observed in pediatric patients with cancer. However, rare cases of renal insufficiency and osteoporosis have been reported as severe consequences. Published literature focuses predominantly on hypercalcemia in pediatric hematologic malignancies, precluding causative associations with solid tumor disease histology or tumor characteristics.</p><p><strong>Methods: </strong>In this retrospective cohort analysis, we identified 39 patients (aged < 21 years) diagnosed and hospitalized with a solid tumor and prolonged hypercalcemia over the span of 25 years at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center. Hypercalcemia was defined as a sustained serum calcium of at least 11.5 mg/dL for more than 1 week. Disease characteristics, clinical symptoms, management, and outcome were analyzed.</p><p><strong>Results: </strong>The median age for patients in this cohort was 2.3 years, and the majority of patients had a diagnosis of neuroblastoma, rhabdoid tumor, hepatoblastoma, or rhabdomyosarcoma. The 39 patients experienced 45 episodes of hypercalcemia, with a median peak serum calcium level (corrected for albumin) of 13.1 mg/dL. The median time from tumor diagnosis to a hypercalcemic episode was 5.4 months, suggesting that disease-directed therapy, tumor progression, or organ dysfunction may have contributed. Peak ionized calcium was not significantly associated with bone or bone marrow involvement, nor with metastatic disease.</p><p><strong>Conclusions: </strong>This work describes the largest retrospective series of pediatric patients with solid tumors and hypercalcemia to date. Hypercalcemia was most frequently reported for a small subset of diseases, suggesting that disease histology, biology, and the intensive therapeutic interventions required to treat these diseases may be contributing to hypercalcemic risk.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32044"},"PeriodicalIF":2.3,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069952","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":"Mast Cell Leukemia With Myelodysplastic Syndrome With a KIT D816V Mutation and a GATA2 Germline Mutation in a Child: Case Report and Literature Review.","authors":"Fangyuan Zheng, Mingming Ding, Miao Wang, Aidong Lu, Yueping Jia, Huimin Zeng, Leping Zhang","doi":"10.1002/pbc.31953","DOIUrl":"https://doi.org/10.1002/pbc.31953","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31953"},"PeriodicalIF":2.3,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065337","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":"Dabrafenib as a Therapeutic Option for Chemotherapy-Refractory Pediatric Juvenile Xanthogranuloma.","authors":"Mengxuan Wang, Jian Ge, Honghao Ma, Tianyou Wang, Zhigang Li, Rui Zhang","doi":"10.1002/pbc.32053","DOIUrl":"https://doi.org/10.1002/pbc.32053","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32053"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065221","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":"Lower Neighborhood Socioeconomic Status Is Associated With Intensive Care Unit Admission at Initial Presentation in Children With Acute Leukemia.","authors":"Chynna M Swift, Earl Chism, Katherine Lin, Aly Cortella, Tanushree Banerjee, Jacklyn Sanchez Alvarez, Lena E Winestone","doi":"10.1002/pbc.32049","DOIUrl":"https://doi.org/10.1002/pbc.32049","url":null,"abstract":"<p><strong>Background: </strong>Lower socioeconomic status (SES) and minority race and ethnicity are associated with lower overall survival in pediatric leukemia. Higher acuity at initial presentation is associated with mortality during induction chemotherapy. We hypothesized that the mechanism underlying these associations is differences in access to healthcare and that patients who resided in lower SES neighborhoods were more likely to have higher illness acuity at initial presentation compared to patients in higher SES neighborhoods.</p><p><strong>Procedure: </strong>This retrospective cohort study included patients aged 0-21 diagnosed with acute leukemia and treated at a single institution from 2012 to 2022. Neighborhood SES (nSES) data from the California Neighborhoods Data System were used as a measure of access to care. Laboratory abnormalities indicative of illness acuity and intensive care unit (ICU) admission in the first 72 h of leukemia presentation were collected. Multivariable regression was used to evaluate the association between ICU admission and nSES.</p><p><strong>Results: </strong>Of 147 patients, 25% required ICU admission within the first 72 h of leukemia presentation. After adjusting for race, ethnicity, insurance, and leukemia risk group, patients in the lowest and middle nSES tertiles were 4.0 times more likely to require ICU admission compared to patients in the highest tertile (confidence interval [CI]: 1.8-9.1).</p><p><strong>Conclusions: </strong>Children living in lower SES neighborhoods are significantly more likely to require ICU admission at the time of leukemia diagnosis compared to children in higher SES neighborhoods. Interventions focused on improving healthcare access may improve outcomes for groups that have been economically and socially disadvantaged.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32049"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065292","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":"Social Participation Among Long-Term Survivors of Childhood and Adolescent Cancer.","authors":"Clarissa E Schilstra, Sarah J Ellis, Andrew Sedger, Mary A Burns, Kimberley Miller, Claire E Wakefield, Richard J Cohn, Suncica Lah, Ursula M Sansom-Daly, Antoinette C Anazodo, Joanna E Fardell","doi":"10.1002/pbc.32042","DOIUrl":"https://doi.org/10.1002/pbc.32042","url":null,"abstract":"<p><strong>Background: </strong>The impact of cancer treatment on the social participation of childhood and adolescent cancer survivors remains understudied. Social participation is an important determinant of long-term social outcomes. Therefore, this study aimed to describe the proportion of a cohort of Australian childhood and adolescent long-term cancer survivors experiencing social participation challenges post-treatment.</p><p><strong>Methods: </strong>Survivors aged 8-25 years (1-10 years post-treatment) were recruited from a childhood cancer survivorship clinic. Self-report surveys assessed clinical/demographic characteristics, and perceived levels of social participation before/after cancer treatment. Survivors were invited to an optional, semi-structured interview about the impact of their cancer experience on their social participation.</p><p><strong>Results: </strong>A total of 52 children/adolescents (M_age = 11.9 ± 2.7 years) and 21 young adults (M_age = 20.5 ± 2.1 years) participated. Approximately 16%-36% of children/adolescents and 14%-43% of young adults perceived a decrease in social participation post-treatment (most children/adolescents did not report on pre-diagnosis participation). In interviews, survivors discussed physical and mental health challenges that occurred during their cancer treatment that affected their social participation, with limited mention of challenges after treatment.</p><p><strong>Conclusion: </strong>A subset of survivors of childhood and adolescent cancer experiences social participation challenges. However, qualitatively, survivors' descriptions of social challenges centered on their experiences during treatment, rather than after. This suggests that even in the absence of current challenges, some survivors perceive a lasting impact of their cancer experience on their social participation. These findings highlight need for larger, longitudinal mixed-methods studies involving tailored, evidence-based measures of social participation to document changes in the prevalence and course of social participation challenges for post-treatment survivors.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32042"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145064804","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 Children With Intermediate-Risk Extracranial Malignant Germ Cell Tumors by Response Status at the End of Induction Chemotherapy: A Report From the Children's Oncology Group.","authors":"Adriana Fonseca, Doojduen Villaluna, Mark Krailo, Thomas A Olson, Farzana Pashankar, Marcio H Malogolowkin, James F Amatruda, Deborah F Billmire, Frederick J Rescorla, Jonathan H Ross, Bryan J Dicken, Carlos Rodriguez-Galindo, Lindsay Frazier, Furqan Shaikh","doi":"10.1002/pbc.32033","DOIUrl":"https://doi.org/10.1002/pbc.32033","url":null,"abstract":"<p><strong>Background: </strong>In many pediatric malignant germ cell tumor (MGCT) protocols, patients who do not achieve a complete response (CR; normal tumor markers, no radiological/histological residual) after three cisplatin-etoposide-bleomycin (PEb) cycles receive two to three \"consolidation\" cycles of the same regimen. Evidence for this practice is limited, and historical outcomes for non‑CR patients remain poor.</p><p><strong>Methods: </strong>We retrospectively reviewed patients diagnosed with intermediate-risk MGCT, enrolled in AGCT0132, between 2003 and 2011. All patients received three cycles of PEb and underwent response assessment at the end of induction. Patients not in CR were prescribed three additional cycles of PEb as consolidation. We compared event-free survival (EFS) and overall survival (OS) for patients who did and did not receive consolidation.</p><p><strong>Results: </strong>Among 210 patients enrolled, two patients were excluded from analyses: one without complete information, and one had rapid progressive disease (PD) and did not complete induction therapy. Out of 208 patients, 193 had a CR after three cycles of induction chemotherapy, and their post-induction 4-year EFS and OS were 93% and 99%. Fifteen patients were not in CR at the end of the first three cycles. Twelve received consolidated chemotherapy as mandated per protocol, and their 4-year EFS and OS were 61% and 65%, respectively. Three patients were deemed to have progressive disease at the end of induction and received second-line therapy.</p><p><strong>Conclusion: </strong>Children with MGCTs who did not achieve CR after the first three cycles of chemotherapy had an inferior outcome compared to those with a CR, despite receiving additional cycles of PEb chemotherapy. We conclude that consolidation is of unclear benefit. We suggest that pediatric MGCT patients who fail to achieve a CR after standard induction chemotherapy may receive a salvage regimen with different agents rather than consolidation with more of the same chemotherapy.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32033"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065341","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":"Veno-Occlusive Disease in Very Young Children: A Profile of High Morbidity With Low Mortality.","authors":"Miki Nishitani, Kyle Hebert, Donna Neuberg, Trisha Rahman, Leslie Lehmann, Christine N Duncan","doi":"10.1002/pbc.32038","DOIUrl":"https://doi.org/10.1002/pbc.32038","url":null,"abstract":"<p><strong>Background/objectives: </strong>Younger age is a recognized risk factor for veno-occlusive disease (VOD), yet no prior studies have focused on diagnostic criteria and outcomes in very young children. This retrospective study compared overall survival (OS) in very young children with and without a clinical diagnosis of VOD, in addition to comparing OS for those with clinical VOD versus those who retrospectively fulfilled VOD criteria.</p><p><strong>Design/methods: </strong>Included were patients who received their first hematopoietic stem cell transplant (HCT) for any indication at less than 4 years of age between January 1, 2016 and October 31, 2021. Three pediatric VOD criteria were retrospectively applied to subjects.</p><p><strong>Results: </strong>Of 150 HCT recipients less than 4 years old, 18 were clinically diagnosed with VOD at a median of 14 days. More patients with VOD were admitted to the ICU [79% vs. 15%, p < 0.001]. Of those with VOD, 78% required peritoneal drainage, 17% pleural effusion drainage, and 56% respiratory failure. All patients with VOD survived to discharge and to Day +100. In allo-recipients, OS at Day +365, cumulative incidence of Grade II-IV acute graft-versus-host disease (aGVHD) at Day +100, and chronic graft-versus-host disease (cGVHD) at Day +365 were similar between those with and without VOD. Median length of stay was longer in those with VOD [60.5 vs. 38 days, p < 0.001]. More patients retrospectively met criteria for VOD than were clinically diagnosed.</p><p><strong>Conclusion: </strong>While VOD in very young children can be highly morbid, they have excellent early survival. Retrospective evaluation demonstrates that only some patients who fulfilled VOD criteria were clinically diagnosed.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32038"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065026","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":"Large-Bore Vacuum Suction Biopsy of Pediatric Soft Tissue Tumors: A 5-Year Retrospective Analysis of Safety and Effectiveness.","authors":"Abhinav R Balu, Shankar Rajeswaran, Nitin R Wadhwani, Ian Gelarden, Elizabeth Sokol, Joseph R Ness, Darshan E Variyam, Timothy Lautz, Joe B Baker","doi":"10.1002/pbc.32037","DOIUrl":"https://doi.org/10.1002/pbc.32037","url":null,"abstract":"<p><strong>Introduction: </strong>Percutaneous large-bore vacuum suction biopsy (LBVSB) is an innovative alternative to open surgical biopsy and minimally invasive image-guided percutaneous core needle biopsy (PCNB) for pediatric soft tissue tumors. This study reviews our experience using this modality.</p><p><strong>Methods: </strong>A retrospective review of all children undergoing LBVSB between 2019 and 2025 was performed. Operative notes, pathology reports, and clinical follow-ups were analyzed to determine tissue adequacy and diagnosis/prognosis.</p><p><strong>Results: </strong>Fifty-five children underwent a total of 59 ultrasound-guided LBVSBs. The average age of patients was 10.5 years, but ranged from 4 months to 20 years. Biopsy was performed most often with a 10 g needle (56% of cases). The median number of samples was six (interquartile range [IQR]: 4-8), and individual core sizes ranged from 0.2 to 2.7 cm, with a median diameter of 0.3 cm (IQR: 0.2-0.4). Pathologic evaluation of the biopsy specimens accurately confirmed the final diagnosis in 54/55 cases (98% accuracy). Repeat surgical biopsy was necessary for two patients, one to more accurately characterize the tumor (Hodgkin lymphoma), and another due to persistent nodal swelling following a diagnostic biopsy (lymphoid tissue with fibrosis). Tissue samples were adequate for obtaining immunohistochemistry and molecular fusion transcripts in all cases where these were deemed necessary to make the final diagnosis. The median procedure time was 24 min (IQR: 20-30.5 min). There were two hemorrhagic complications directly associated with the biopsy procedures, and one complication associated with general anesthesia administration.</p><p><strong>Conclusion: </strong>Our experience demonstrates that image-guided percutaneous LBVSB is an accurate and efficient way to biopsy pediatric soft tissue tumors and seems to be quite safe based on our small cohort. LBVSB samples were adequate in all our patients, allowing for immunohistochemistry and fusion analysis when necessary. There were only two complications directly related to the procedure, and a repeat biopsy was only required in two cases. LBVSB can be considered as an initial diagnostic tool for certain pediatric soft tissue tumors.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32037"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065198","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":"Microphotopheresis for the Treatment of Graft-Versus-Host Disease in Pediatric Patients.","authors":"Kumukova Irina, Kurnikova Elena, Garloeva Julia, Trakhtman Pavel, Balashov Dmitry, Shelikhova Larisa, Skvorcova Julia","doi":"10.1002/pbc.32039","DOIUrl":"https://doi.org/10.1002/pbc.32039","url":null,"abstract":"<p><strong>Introduction: </strong>Extracorporeal photopheresis (ECP) is an attractive treatment for graft-versus-host disease (GVHD) following allogeneic hematopoietic stem cell transplantation due to the lack of systemic immunosuppression. Modifications of ECP without leukapheresis are being actively studied in clinical practice around the world. A low-volume method of extracorporeal photochemotherapy-micro-ECP-has been developed at the Rogachev Federal Scientific and Clinical Centre of Pediatric Hematology, Oncology and Immunology. We present the results of using micro-ECP to treat acute and chronic GVHD.</p><p><strong>Patients and methods: </strong>We analyzed the treatment outcomes of 9 cases of acute GVHD and 15 cases of chronic GVHD using micro-ECP for the period from April 2021 to March 2024. The cellular product was obtained by collecting 20-30 mL of whole blood followed by photochemical treatment. The processed autologous cell product was administered to the patient.</p><p><strong>Results: </strong>The overall response rate in patients diagnosed with acute GVHD was 44%, while the organ-specific response rate was 56% for skin and 40% for gastrointestinal. The steroid-sparing effect for acute GVHD was 89%. In the treatment of chronic GVHD, the overall response rate was 73.3%. The organ-specific response rate was 71% for skin, 67% for gastrointestinal, 67% for lung, 100% for eyes, and 100% for liver chronic GVHD. The steroid-sparing effect for chronic GVHD was 63%.</p><p><strong>Conclusion: </strong>In the treatment of acute and chronic GVHD, micro-ECP has demonstrated promising efficacy. Although the method remains experimental, it is already apparent that micro-ECP constitutes an attractive alternative to standard ECP when leukapheresis is not a viable option.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32039"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145065340","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 Salvage Treatment With Afatinib and Anti-PD-1 Combined Chemotherapy in a Pediatric Patient With Relapsed Cellular-Type Congenital Mesoblastic Nephroma Harboring an EGFR-KDD Mutation.","authors":"Jian Li, Yan Ma, Shaohua Zhang, Lulu He, Chunlei Zhou, Jianfeng Zhou, Tao Li, Li Zhou","doi":"10.1002/pbc.32066","DOIUrl":"https://doi.org/10.1002/pbc.32066","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32066"},"PeriodicalIF":2.3,"publicationDate":"2025-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145064800","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}