Pediatric Blood & Cancer最新文献

Abstracts.

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-21 DOI: 10.1002/pbc.31444

引用次数: 0

Unraveling Juvenile Hyaline Fibromatosis: A Case of Novel ANTXR2 Mutations Associated with Subcutaneous Masses and Hydronephrosis.

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-20 DOI: 10.1002/pbc.31497

Shu-Di Zhang, Yuan Fang, Lian Chen

引用次数: 0

Modified G-CSF/ATG-Based Haploidentical Transplantation Protocol in Pediatric Primary Hemophagocytic Lymphohistiocytosis: A Long-Term Follow-Up Single-Center Experience. 基于 G-CSF/ATG 的小儿原发性嗜血细胞淋巴组织细胞增多症单倍体移植改良方案：单中心长期随访经验。

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-20 DOI: 10.1002/pbc.31495

Juan Xiao, Xingcheng Yang, Nanhai Wu, Shifen Fan, Zhouyang Liu, Fan Jiang, Jiao Chen, Jia Wei, Yuan Sun

{"title":"Modified G-CSF/ATG-Based Haploidentical Transplantation Protocol in Pediatric Primary Hemophagocytic Lymphohistiocytosis: A Long-Term Follow-Up Single-Center Experience.","authors":"Juan Xiao, Xingcheng Yang, Nanhai Wu, Shifen Fan, Zhouyang Liu, Fan Jiang, Jiao Chen, Jia Wei, Yuan Sun","doi":"10.1002/pbc.31495","DOIUrl":"https://doi.org/10.1002/pbc.31495","url":null,"abstract":"Background: Primary hemophagocytic lymphohistiocytosis (HLH) is a hyperinflammatory syndrome caused by immune dysregulation. Hematopoietic stem cell transplantation (HSCT) represents the only option for long-term cure for primary HLH. However, only around 25% of patients have a fully HLA-matched donor.Methods: In this retrospective study, we analyzed 42 pediatric patients with primary HLH who underwent haplo-SCT using the modified granulocyte colony-stimulating factor (G-CSF)/antithymocyte globulin (ATG)-based protocol. The conditioning regimen included 300-600 mg/m2 etoposide (VP16), along with low doses of busulfan (Bu) (0.8-1.2 mg/kg every 6 hours on Days -8 to -6), cyclophosphamide (Cy) (10 mg/kg/day on Days -4 to -3), fludarabine (Flu) (30 mg/m2/day on Days -5 to -3), and ATG (8-9 mg/kg total dose on Days -5 to -2) to reduce complications.Results: All 42 patients achieved successful engraftment. Following a median follow-up period of 48.7 months, 32 of the 42 patients remained alive and disease free. The 2-year overall survival (OS) rate was 78.4%, and the 5-year OS rate was 73.7%. The 2-year failure-free survival (FFS) rate was 71.3%, and the 5-year FFS rate was 66.5%. Patients who achieved complete remission at the time of HSCT showed better OS (p < 0.05). The incidence of Grade III-IV acute graft-versus-host disease (GVHD) was 26.2%, and severe chronic GVHD was observed in 11.9% of patients. Thrombotic microangiopathy occurred in 13 patients, and veno-occlusive disease in two patients.Conclusions: This modified G-CSF/ATG-based haploidentical protocol demonstrates significant potential for pediatric patients with primary HLH, exhibiting commendable effectiveness and safety.","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31495"},"PeriodicalIF":2.4,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142864956","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

Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321.

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-19 DOI: 10.1002/pbc.31490

James I Geller, Lindsay A Renfro, Paul E Grundy, Elizabeth J Perlman, John A Kalapurakal, Peter F Ehrlich, Jackie Biegel, Vicki Huff, Anne B Warwick, Arnold Paulino, Elizabeth A Mullen, Najat C Daw, Fredric A Hoffer, Zelig Tochner, Kenneth Gow, Eric Gratias, Deborah A Ward, James R Anderson, Conrad V Fernandez, Jeffrey S Dome

{"title":"Rhabdoid Tumor of the Kidney and Soft Tissues: Results from National Wilms Tumor Study-5 and Children's Oncology Group Study AREN0321.","authors":"James I Geller, Lindsay A Renfro, Paul E Grundy, Elizabeth J Perlman, John A Kalapurakal, Peter F Ehrlich, Jackie Biegel, Vicki Huff, Anne B Warwick, Arnold Paulino, Elizabeth A Mullen, Najat C Daw, Fredric A Hoffer, Zelig Tochner, Kenneth Gow, Eric Gratias, Deborah A Ward, James R Anderson, Conrad V Fernandez, Jeffrey S Dome","doi":"10.1002/pbc.31490","DOIUrl":"https://doi.org/10.1002/pbc.31490","url":null,"abstract":"Purpose: National Wilms Tumor Study-5 (NWTS-5) and AREN0321 evaluated the outcomes of children with rhabdoid tumor of the kidney (RTK) and malignant rhabdoid tumor of soft tissues (MRT).Patients and methods: Eligible patients with RTK were enrolled prospectively on NWTS-5 (1995-2002) and treated with carboplatin and etoposide alternating with cyclophosphamide (Regimen RTK). Patients with RTK or MRT were enrolled on AREN0321 (2005-2012) and received vincristine, doxorubicin, and cyclophosphamide alternating with carboplatin, cyclophosphamide, and etoposide (Regimens UH-1 or dose-reduced Revised UH-1). We report event-free survival (EFS) and overall survival (OS) from each study.Results: Thirty patients received Regimen RTK on NWTS-5; on AREN0321, 20 received UH-1 and 19 received Revised UH-1. Patient and disease characteristics were statistically similar between studies. Patients on AREN0321 had significantly improved EFS and OS compared to those on NWTS-5 (4-year EFS = 23.1% vs. 16.7%; p = 0.020; 4-year OS = 30.6% vs. 20.0%; p = 0.014), mostly driven by patients with Stage I/II disease (p = 0.05). Median time to an event was 3.6 months on NWTS-5 compared to 7.2 months on AREN0321. There were no differences in EFS or OS by revised versus original Regimen UH-1 on AREN0321, or by renal versus extra-renal primary disease when the studies were pooled.Conclusions: The more intensive treatment regimen used on AREN0321 improved EFS and OS overall, a result driven by patients with Stage I/II disease. Despite this improvement, outcomes for patients with rhabdoid tumor remain unsatisfactory and there is a need for novel therapeutic strategies.","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31490"},"PeriodicalIF":2.4,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142864958","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

A Unique Case of a Pediatric Patient with Six Childhood Cancers in Association with a Germline TP53 Gene Pathogenic Variant. 一个独特的小儿患者病例：六种儿童癌症与种系 TP53 基因致病性变异有关。

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-19 DOI: 10.1002/pbc.31487

Shaelene Standing, David Malkin, Donna L Johnston

引用次数: 0

Treatment of a Pediatric Patient with Core Binding Factor Acute Myeloid Leukemia Post-Cytotoxic Therapy With Cytarabine, Daunorubicin, and Gemtuzumab Followed by Stem Cell Transplantation. 用胞磷胆碱、多诺比星和吉妥珠单抗对一名核心结合因子急性髓性白血病小儿患者进行细胞毒治疗后再进行干细胞移植。

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-19 DOI: 10.1002/pbc.31489

Irtiza N Sheikh, Destiny Uwaezuoke, Olayinka Okeleji, Andres Quesada, Cynthia E Herzog, Dristhi Ragoonanan, Priti Tewari, Demetrios Petropoulos, David McCall, Cesar Nunez, Michael Roth, Branko Cuglievan, Sajad Khazal

引用次数: 0

Cancer Stage Geospatial Analysis to Optimize Educational Activities in Pediatric Oncology and Hematology: A Retrospective Cohort Study.

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-18 DOI: 10.1002/pbc.31494

Monika Barełkowska, Barbara Więckowska, Martyna Borowczyk, Katarzyna Derwich

{"title":"Cancer Stage Geospatial Analysis to Optimize Educational Activities in Pediatric Oncology and Hematology: A Retrospective Cohort Study.","authors":"Monika Barełkowska, Barbara Więckowska, Martyna Borowczyk, Katarzyna Derwich","doi":"10.1002/pbc.31494","DOIUrl":"https://doi.org/10.1002/pbc.31494","url":null,"abstract":"Background: Enhancing therapy outcomes in pediatric oncology and hematology relies on robust epidemiological surveillance. This study aimed to estimate cancer stage-related survival in pediatric patients with lymphoma and solid tumors by assessing changes over time and identifying spatial clustering of patients diagnosed at advanced stages.Procedure: This retrospective observational cohort study included pediatric cancer patients treated in a single Greater Poland center, constituting 9% of Polish children from 2004 to 2017. The incidence, cancer stage, and patient coordinates were analyzed. The follow-up period ranged from 5 to 18 years. Survival differences across tumor stages were evaluated using Kaplan‒Meier curves, log-rank tests, and trend analysis. Geographical analysis was performed with Kulldorff's scan statistics and the Bernoulli model.Results: Among 1094 diagnosed patients, 511 with lymphoma and solid tumors were eligible. There was a decreasing trend in advanced-stage diagnoses (p = 0.0001), with a nearly twofold increase in low-stage diagnoses (OR = 1.98 [1.22; 3.24], p = 0.0061) from 2009-2011 to 2015-2017. Hazard ratios for neuroblastoma and sarcoma patients were more than fourfold greater, while survival differences were not significant for patients with nephroblastoma, germ cell tumors, or lymphoma.Conclusions: This study shows cancer stage-related survival dynamics. A substantial decrease in advanced-stage diagnoses over time emphasizes improved early detection. Geographical analysis pinpointed clusters with prevalent late-stage diagnoses, offering a practical tool for targeted educational interventions. The study underscores the pivotal impact of cancer stage on survival outcomes, emphasizing the need for ongoing surveillance and tailored interventions to further optimize pediatric oncology and hematology care.","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31494"},"PeriodicalIF":2.4,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854991","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

Coagulation Dysfunction Caused by a De Novo Mutation of Gene SLC37A4.

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-18 DOI: 10.1002/pbc.31488

Mauro Guariento, Nicola Martinelli, Laura Pezzoli, Federica Bortolotti, Elisa Bonetti, Ada Zaccaron, Matteo Chinello, Virginia Vitale, Giulia Caddeo, Vincenza Pezzella, Maria Pia Esposto, Chiara Guardo, Simone Cesaro

引用次数: 0

Return-to-School Experiences of Adolescents After Allogeneic Hematopoietic Cell Transplant: A Qualitative Interview Study of Transplant Recipients.

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-17 DOI: 10.1002/pbc.31481

Olivia A Do, Timothy J D Ohlsen, Kelly J Shipman, Sheri A Ballard, Kari M Jenssen, K Scott Baker, Abby R Rosenberg, Krysta S Barton, Neel S Bhatt

{"title":"Return-to-School Experiences of Adolescents After Allogeneic Hematopoietic Cell Transplant: A Qualitative Interview Study of Transplant Recipients.","authors":"Olivia A Do, Timothy J D Ohlsen, Kelly J Shipman, Sheri A Ballard, Kari M Jenssen, K Scott Baker, Abby R Rosenberg, Krysta S Barton, Neel S Bhatt","doi":"10.1002/pbc.31481","DOIUrl":"https://doi.org/10.1002/pbc.31481","url":null,"abstract":"Background: Returning to school after allogeneic hematopoietic cell transplant (HCT) can improve quality of life and promote positive adjustment. However, this process may be challenging, and there is a limited understanding of school-aged children and adolescents' perspectives on this process.Methods: We conducted semi-structured interviews over video with pediatric recipients of HCT (10-18 years of age at HCT; 1-7 years post HCT) who were treated at our institution and had returned to in-person school post HCT. We performed a thematic network analysis focused on exploring salient challenges regarding the return-to-school process post HCT and potential areas for improvement.Results: We interviewed 16 participants (mean age 13.8 years at HCT). Four themes emerged: (i) challenges of returning to school, (ii) keys for a successful return-to-school experience, (iii) overall perceptions of the process, and (iv) recommendations for improvement. HCT recipients described several social/emotional, physical, and academic challenges while returning to school and cited strong sources of support as critical to a successful transition. Recommendations for a better transition process included the following: (a) fostering peer support, (b) establishing social connections, (c) providing mental health support, (d) identifying a go-to point of contact for issues, and (e) maintaining academic support.Conclusions: Our findings highlight perspectives from school-aged recipients of HCT regarding gaps in support and areas for improvement to facilitate successful return to school after HCT. Additional assistance throughout the process may optimize academic and social reintegration and support recovery after HCT.","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31481"},"PeriodicalIF":2.4,"publicationDate":"2024-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142847334","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

Screening and Impact of Neuropathic Pain in Young Individuals With Sickle Cell Disease. 镰状细胞病年轻患者神经性疼痛的筛查及其影响

IF 2.4 3区医学

Pediatric Blood & Cancer Pub Date : 2024-12-17 DOI: 10.1002/pbc.31493

Amira Adly, Maha Mohammed, Ahmed Aboata, Ghada Wassif, Marwa Nassef, Sara Makkeyah

{"title":"Screening and Impact of Neuropathic Pain in Young Individuals With Sickle Cell Disease.","authors":"Amira Adly, Maha Mohammed, Ahmed Aboata, Ghada Wassif, Marwa Nassef, Sara Makkeyah","doi":"10.1002/pbc.31493","DOIUrl":"https://doi.org/10.1002/pbc.31493","url":null,"abstract":"Objective: To assess the frequency of neuropathic pain (NP) and its impact in young patients with sickle cell disease (SCD).Methods: We used the ID-Pain (ID-P) questionnaire and a bedside clinical sensory testing (CST) as screening tools for NP and performed sensory nerve conduction study (SNCS) for all the participants. The impact of pain was assessed using Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaires and Pediatric Quality of Life Inventory (PedsQL) SCD module.Results: The mean age was 12.4 years. The ID-P questionnaire revealed that 20.5% of patients likely had NP, while 10 (34%) of the participants had abnormal CST, and only two patients (4.5%) had abnormal SNCS. On the PROMIS pain scores, the mean pain intensity score was 7.9 ± 2.0. Pain interference was the most affected domain in both patients and parent proxy reports (mean ± SD T-score 63.4 ± 6.6 and 65.2 ± 6.6, respectively) which was worse than other domains including pain quality-sensory and affective, and pain behavior (median T-scores of 47.8, 63.0, and 56.2, respectively). The mean global health-related quality of life (HRQoL) score of the children was 50.3 ± 12.1 indicating poor quality of life.Conclusion: We report the utility of simple tools like ID-P questionnaire and CST for screening of NP in young individuals with SCD.","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31493"},"PeriodicalIF":2.4,"publicationDate":"2024-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142847337","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