Pediatric Hematology and Oncology最新文献_第4页

Temporal trends in pediatric cancer mortality: rare cancers lag behind more common cancers. 儿科癌症死亡率的时间趋势：罕见癌症落后于常见癌症。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2025-02-01 Epub Date: 2024-10-24 DOI: 10.1080/08880018.2024.2413643

Brian R Englum, Shalini Sahoo, Theodore W Laetsch, Gregory M Tiao, Minerva Mayorga-Carlin, Hilary Hayssen, Yelena Yesha, John D Sorkin, Brajesh K Lal

{"title":"Temporal trends in pediatric cancer mortality: rare cancers lag behind more common cancers.","authors":"Brian R Englum, Shalini Sahoo, Theodore W Laetsch, Gregory M Tiao, Minerva Mayorga-Carlin, Hilary Hayssen, Yelena Yesha, John D Sorkin, Brajesh K Lal","doi":"10.1080/08880018.2024.2413643","DOIUrl":"10.1080/08880018.2024.2413643","url":null,"abstract":"Temporal trends demonstrate improved survival for many types of common pediatric cancer. Studies have not examined improvement in very rare pediatric cancers or compared these improvements to more common cancers. In this cohort study of the Surveillance, Epidemiology, and End Results (SEER) registry, we examined patients from 1975 to 2016 who were 0-19 years of age at the time of diagnosis. Cancers were grouped by decade of diagnosis and 3 cancer frequency groups: Common, Intermediate, and Rare. Trends in mortality across decades and by cancer frequency were compared using Kaplan-Meier curves and adjusted Cox proportional hazards models. A total of 50,222 patients were available for analysis, with the top 10 cancers grouped as Common (67%), 13 cancers grouped with Intermediate (24%), and 37 cancers as Rare (9%). Rare cancers had higher rates of children who were older and Black. 5-year survival increased from 63% to 86% across all cancers from the 1970s to the 2010s. The hazard ratio (HR) for mortality decreased from the reference point of 1 in the 1970s to 0.27 (95% CI: 0.25-0.30) in the 2010s in Common cancers, while the HR only dropped to 0.60 (0.49-0.73) over that same period for rare cancers. Pediatric oncology patients have experienced dramatic improvement in mortality since the 1970s, with mortality falling by nearly 75% in common cancers. Unfortunately, rare pediatric cancers continue to lag behind more common and therefore better studied cancers, highlighting the need for a renewed focus on research efforts for children with these rare diseases.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"1-13"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Procedural sedation performed by pediatric critical care physicians for children undergoing daily radiation therapy is effective and safe. 儿科重症监护医师对接受日常放射治疗的儿童进行程序性镇静是有效和安全的。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2025-02-01 Epub Date: 2024-12-13 DOI: 10.1080/08880018.2024.2436496

Nicole M Batista, Maxwell Corrigan, J Gene Chen

{"title":"Procedural sedation performed by pediatric critical care physicians for children undergoing daily radiation therapy is effective and safe.","authors":"Nicole M Batista, Maxwell Corrigan, J Gene Chen","doi":"10.1080/08880018.2024.2436496","DOIUrl":"10.1080/08880018.2024.2436496","url":null,"abstract":"Radiation therapy targets tumor tissue and requires children to lay still, often necessitating sedation. Historically anesthesiologists provided procedural sedation, but pediatric critical care physicians now regularly administer sedation outside the operating room. Procedural sedation for radiation poses unique challenges. The objective was to evaluate the success and assess complications of repeated sedations for radiation performed by pediatric critical care physicians. We performed a single-center, retrospective case series of children who received procedural sedation for radiation therapy by PICU physicians. The primary outcome was success, defined as completion of radiation treatment. Secondary outcomes included type of medication, dosing, tolerance, and complications requiring intervention. In our sample, 55 patients underwent 1174 sedation instances (mean 19.8 per patient). Patients had a mean age of 4.7 years (SD3.4), and weight of 20.2 kg (SD11.9). All patients had an ASA of 2 or 3. All patients had either a brain tumor or a non-mediastinal solid tumor. The success rate was 99.8%. The mean duration of sedation was 30.7 min (SD12.4). All sedations included propofol as a first agent with a mean bolus 3.3 mg/kg (SD1.4) and drip rate 148.7 mcg/kg/min (SD39.7). 4.4% of sedations required a second agent medication. There was no significant effect of repeated sedation with regards to the medication amount received (p = 0.97). Laryngospasm occurred during 0.2% of sedations. No patients required bag-mask ventilation, intubation, or chest compressions; no patients died during sedation. Pediatric critical care physicians can perform procedural sedation for radiation therapy successfully.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"54-62"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822469","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Impact of socioeconomic factors on time to diagnosis of childhood cancer. 社会经济因素对儿童癌症诊断时间的影响。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2025-02-01 Epub Date: 2024-11-29 DOI: 10.1080/08880018.2024.2434876

Chloé Goncalves, Jérémie Rouger, Isabelle Pellier, Jean-Jacques Parienti, Julien Lejeune, Audrey Grain, Julien Rod, Virginie Gandemer, Fanny Delehaye

{"title":"Impact of socioeconomic factors on time to diagnosis of childhood cancer.","authors":"Chloé Goncalves, Jérémie Rouger, Isabelle Pellier, Jean-Jacques Parienti, Julien Lejeune, Audrey Grain, Julien Rod, Virginie Gandemer, Fanny Delehaye","doi":"10.1080/08880018.2024.2434876","DOIUrl":"10.1080/08880018.2024.2434876","url":null,"abstract":"In adults, there is a link between socioeconomic status (SES) and cancer prognosis, notably due to increased time to diagnosis (TTD) in deprived population leading to the dissemination of the disease. In children, such an association has not been clearly reported. The objective of our study was to assess the impact of SES on TTD of childhood cancer and its potential consequences on cancer prognosis. We carried out a multicenter retrospective study based on the LOGAFTER multi centric database. We studied SES at the individual level (parental professions, family structure) and the ecological level (EDI score, travel time by car). We assessed the factors potentially associated with an increased TTD with a Cox regression model, and we illustrated TTD by categories by using Kaplan-Meier curves. 854 children were included. The median time to diagnosis was 28 days [12;64]. TTD differed significantly according to the type of tumor. An usual care pathway did not impact TTD. However, an initial management by professionals not usually involved in the specific childhood cancer context increased TTD. None of the SES ecological variables were strictly associated with an impact on TTD, and a trend was noted for single-parent families (increased TTD, p = 0.057). In our cohort, TTD did not impact on the vital and relapse status. In this study, the impact of SES on TTD in children on both the individual and ecological levels was not clear. However, we noted some keys at the individual scale that require further investigation to explain potential associations.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"37-53"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142751428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Long-Term Survey of Japanese Children with Recurrent Nephroblastoma: A Report from Japan Children's Cancer Group. 日本复发性肾母细胞瘤患儿的长期调查：日本儿童癌症小组的报告。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2025-02-01 Epub Date: 2024-11-05 DOI: 10.1080/08880018.2024.2423207

Hiroshi Yagasaki, Yoshiki Katsumi, Miwako Nozaki, Satoshi Hamanoue, Hiroaki Fukuzawa, Koji Fukumoto, Shinji Mochizuki, Shuichiro Uehara, Takaharu Oue, Tsugumichi Koshinaga

{"title":"Long-Term Survey of Japanese Children with Recurrent Nephroblastoma: A Report from Japan Children's Cancer Group.","authors":"Hiroshi Yagasaki, Yoshiki Katsumi, Miwako Nozaki, Satoshi Hamanoue, Hiroaki Fukuzawa, Koji Fukumoto, Shinji Mochizuki, Shuichiro Uehara, Takaharu Oue, Tsugumichi Koshinaga","doi":"10.1080/08880018.2024.2423207","DOIUrl":"10.1080/08880018.2024.2423207","url":null,"abstract":"In prospective Japanese studies of pediatric renal tumors, 5-year event-free survival and overall survival (OS) for patients with nephroblastoma ranges from 75-90% and 89-97%, respectively. However, treatments strategies for recurrent nephroblastoma in Japanese patients remain unclear. This retrospective study aimed to inform the development of treatment strategies by analyzing the long-term results and side effects of salvage therapies for recurrent nephroblastoma in Japan. A questionnaire survey involving 41 institutions (74 patients) collected clinical data on recurrent cases reported to the Renal Tumor Committee of the Japan Children's Cancer Group. Survey forms from 54 cases were evaluated. Median time to recurrence was 9.5 months among 51 patients without underlying disorders. Recurrence occurred at lung-only in 18 patients and at other sites in 33. The 5-year OS for all 51 patients was 70.6%, with recurrent disease causing death in 15 patients and one patient dying from treatment-related complications. Patients with lung-only recurrence had higher 5-year OS rates than those with other-site recurrence. Initial chemotherapy intensity also affected prognosis, with lower intensity associated with higher 5-year OS. In 17 survivors with lung-only recurrence, the most frequent treatment approach combined chemotherapy, surgery and radiotherapy. Conventional chemotherapy included platinum-containing regimens and/or Regimen I-based treatment containing cyclophosphamide and etoposide. Salvage therapies showed remarkable effectiveness for patients with lung-only recurrence or low intensity of the initial chemotherapy, highlighting the need to standardize prospective studies for post-recurrence treatment and identify risks of late complications for long-term survivors.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"14-26"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142582737","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Current approaches in development and implementation of medical education strategies among pediatric hematology/oncology fellowship programs. 当前儿科血液学/肿瘤学奖学金项目中医学教育策略的发展和实施方法。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2025-02-01 Epub Date: 2024-12-08 DOI: 10.1080/08880018.2024.2432272

Samantha Scanlon, Kristina Dzara, Rebecca Ronsley

{"title":"Current approaches in development and implementation of medical education strategies among pediatric hematology/oncology fellowship programs.","authors":"Samantha Scanlon, Kristina Dzara, Rebecca Ronsley","doi":"10.1080/08880018.2024.2432272","DOIUrl":"10.1080/08880018.2024.2432272","url":null,"abstract":"Application of evidenced-based practices is often lacking in medical education. Increased recognition that methods of knowledge delivery based in relevant learning theories are more effective has led to improved ability to meet the needs of adult learners. We designed a study to assess approaches to education for Pediatric Hematology/Oncology (PHO) trainees and identify opportunities for incorporation of evidence-based practices. A national survey was shared with program directors (PDs) of PHO programs to investigate current trends in education design. Respondents were contacted via email, the survey was distributed by REDCap, and data were collected from April-July 2023. Quantitative data were analyzed descriptively and free-text responses using directed content analysis. Of the 77 eligible participants, 46 completed the survey (59.74% response rate), representing various geographic regions and class sizes. Respondents reported a wide range of familiarity with adult learning theory (range 1-5, median 3) and self-regulated learning (range 1-5, median 2). While programs employed active learning strategies to varying degrees, the lecture format was the most used method of education delivery. PDs recognized the need for development in several domains, including incorporation of educational frameworks, support for structure and content of didactics, and implementation of feedback related to education sessions. Differences in approaches were described, revealing a wide range of familiarity with and application of specific educational theories, as well as inconsistent efforts to consider principles of adult learning theory and self-regulated learning. These data demonstrate opportunities for targeted education and curriculum development.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"27-36"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142795080","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Soluble cytotoxic T-lymphocyte antigen-4 (sCTLA-4) in children with immune cytopenia: relation to disease activity. 免疫细胞减少症患儿的可溶性细胞毒性 T 淋巴细胞抗原-4（sCTLA-4）：与疾病活动的关系。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-11-01 Epub Date: 2024-10-07 DOI: 10.1080/08880018.2024.2409852

Sara Mostafa Makkeyah, Nayera Hazaa El-Sherif, Mona Fathey Hasan, Marwa Gamal Ibrahim, Nihal Hussien Aly

{"title":"Soluble cytotoxic T-lymphocyte antigen-4 (sCTLA-4) in children with immune cytopenia: relation to disease activity.","authors":"Sara Mostafa Makkeyah, Nayera Hazaa El-Sherif, Mona Fathey Hasan, Marwa Gamal Ibrahim, Nihal Hussien Aly","doi":"10.1080/08880018.2024.2409852","DOIUrl":"10.1080/08880018.2024.2409852","url":null,"abstract":"Cytotoxic T-lymphocyte associated antigen-4 (CTLA-4) is a costimulatory receptor exhibiting a potent inhibitory signal on antigen-activated immune responses. A soluble form, sCTLA-4, has been identified and was found to be increased in several autoimmune diseases. We aimed to evaluate serum levels of sCTLA-4 in different immune cytopenias, and to determine its possible relation to the disease activity. We measured serum levels of sCTLA-4 in 47 patients with immune cytopenias and compared them to 47 age- and sex-matched healthy controls. sCTLA-4 levels were significantly higher in patients with immune cytopenias compared to healthy controls (p < 0.001), however, levels were comparable between different groups of immune cytopenias (p = 0.084). Serum sCTLA-4 inversely correlated with age at diagnosis and hemoglobin level (p = 0.048, and p = 0.039 respectively), while it directly correlated with disease duration (p = 0.023) as well as markers of hemolysis including reticulocyte count, serum LDH and indirect bilirubin (p = 0.025; p = 0.019; p = 0.004 respectively). In the AIHA group, serum sCTLA-4 levels were significantly lower in patients in remission compared to patients with active disease (p = 0.026). Children with immune cytopenia exhibit significantly higher levels of circulating sCTLA-4 which correlated with disease activity, yet the prognostic significance and its use to tailor treatment regimen require additional studies.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"611-619"},"PeriodicalIF":1.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142381454","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

The relationship between circulating tumor cells in peripheral blood and clinical characteristics of pediatric neuroblastoma and prognostic evaluation. 小儿神经母细胞瘤外周血循环肿瘤细胞与临床特征的关系及预后评估

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-11-01 Epub Date: 2024-09-27 DOI: 10.1080/08880018.2024.2408559

Junhua Tuo, Zhi Zhao, Xiaoning Ma, Zhengsheng Liu, Baogang Yang, Meng Zhang, Xuan He

{"title":"The relationship between circulating tumor cells in peripheral blood and clinical characteristics of pediatric neuroblastoma and prognostic evaluation.","authors":"Junhua Tuo, Zhi Zhao, Xiaoning Ma, Zhengsheng Liu, Baogang Yang, Meng Zhang, Xuan He","doi":"10.1080/08880018.2024.2408559","DOIUrl":"10.1080/08880018.2024.2408559","url":null,"abstract":"This study investigates the correlation between circulating tumor cells (CTCs) in peripheral blood and the clinical characteristics and prognosis of advanced pediatric neuroblastoma (NB). We conducted a retrospective analysis of 144 children with advanced NB who underwent comprehensive treatment. Detailed clinical data were collected, and CTCs were detected using a negative enrichment method combined with immunofluorescence technology. Prognostic evaluation criteria and cutoff values for CTCs were established using ROC curve analysis. Univariate and Cox multivariate regression analyses identified independent risk factors impacting prognosis. Patients were categorized into high and low-expression groups based on optimal cutoff values determined with X-tile software. The high expression group had a significantly higher incidence of disease progression (p < 0.001), maximum tumor diameter ≥10 cm (p = 0.004), undifferentiated subtype (p = 0.034), and stage IV disease (p = 0.007) compared to the low expression group. CTCs were notably higher in patients with progression compared to those with mitigation (p < 0.001), in those with maximum tumor diameter ≥10 cm compared to <10 cm (p < 0.001), and in stage IV compared to stage III patients (p = 0.036). The AUC values for maximum tumor diameter, degree of differentiation, and tumor stage were 0.703, 0.669, 0.574, and 0.598, respectively. The detection of CTCs provides significant insights into the clinical characteristics and prognosis of advanced pediatric NB, highlighting its potential as a prognostic tool.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"596-610"},"PeriodicalIF":1.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142351379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Long-Term Survival and Immune Reconstitution of Donor-Derived Chimeric Antigen Receptor T-Cell Therapy for Childhood Molecular Relapse of B-Cell Acute Lymphoblastic Leukemia After Allogeneic Hematopoietic Stem Cell Transplantation. 捐献者衍生嵌合抗原受体 T 细胞疗法治疗异基因造血干细胞移植后儿童 B 细胞急性淋巴细胞白血病分子复发的长期生存和免疫重建。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-11-01 Epub Date: 2024-10-03 DOI: 10.1080/08880018.2024.2408535

Guan-Hua Hu, Ying-Xi Zuo, Pan Suo, Lu Bai, Xiao-Hui Zhang, Yu Wang, Yi-Fei Cheng, Xiao-Jun Huang

{"title":"Long-Term Survival and Immune Reconstitution of Donor-Derived Chimeric Antigen Receptor T-Cell Therapy for Childhood Molecular Relapse of B-Cell Acute Lymphoblastic Leukemia After Allogeneic Hematopoietic Stem Cell Transplantation.","authors":"Guan-Hua Hu, Ying-Xi Zuo, Pan Suo, Lu Bai, Xiao-Hui Zhang, Yu Wang, Yi-Fei Cheng, Xiao-Jun Huang","doi":"10.1080/08880018.2024.2408535","DOIUrl":"10.1080/08880018.2024.2408535","url":null,"abstract":"Measurable residual disease (MRD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an independent risk factor for relapse in patients with acute lymphoblastic leukemia (ALL). This study aimed to assess the efficacy, safety, and immune reconstitution of chimeric antigen receptor T-cell (CAR-T) therapy in patients with molecular relapse after allo-HSCT. Eleven patients with molecular relapse of B-cell-ALL who underwent CAR-T therapy after allo-HSCT were enrolled. The rate of MRD negativity after a month of CAR-T infusion was 81.8%. Patients who bridged to second-HSCT after CAR-T therapy (n = 3) showed a trend of higher 3-year leukemia-free survival and 3-year overall survival than those who did not (n = 8; 100% vs. 75.0%; 95% CI, 45.0-104.9%; p = 0.370). No treatment-related mortalities were observed. Among patients who did not bridge to second-HSCT and remained in complete remission until the last follow-up (n = 6), five of them had not recovered normal immunoglobulin concentrations with a median follow-up of 43 months. CAR-T therapy may be a safe and effective treatment strategy to improve survival after allo-HSCT; however, the problem of prolonged hypogammaglobulinemia in patients who do not bridge to second-HSCT is worth noting.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"583-595"},"PeriodicalIF":1.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142366088","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Assessment of cancer predisposition syndromes in children with leukemia and solid tumors: germline-genomic profiling and clinical features in a series of cases. 评估白血病和实体瘤患儿的癌症易感综合征：一系列病例的种系基因组分析和临床特征。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-11-01 Epub Date: 2024-10-12 DOI: 10.1080/08880018.2024.2411321

Minu Singh, Prateek Bhatia, Pankaj Sharma, Amita Trehan, Richa Jain

{"title":"Assessment of cancer predisposition syndromes in children with leukemia and solid tumors: germline-genomic profiling and clinical features in a series of cases.","authors":"Minu Singh, Prateek Bhatia, Pankaj Sharma, Amita Trehan, Richa Jain","doi":"10.1080/08880018.2024.2411321","DOIUrl":"10.1080/08880018.2024.2411321","url":null,"abstract":"Cancer predisposition syndromes (CPS) are a group of genetic disorders that increase the risk of various cancers. Identifying CPS has a significant impact on the treatment plan, screening and follow-up strategy, and genetic counseling of the family. However, in children, it goes underdiagnosed in most clinical setups, especially in low- and middle-income (LMIC) countries. In the present study, we screened 60 pediatric oncology patients for a possible CPS based on pre-defined selection criteria. Six patients met the criteria, three of whom had hematological malignancy, while the remaining three had sarcoma. Whole exome sequencing was performed in the selected patients to confirm the diagnosis. Germline mutation in CPS-related genes was discovered in five of six cases, including novel mutations discovered in two. An adverse outcome was observed in all five patients with underlying cancer predisposition syndrome, with three having relapsed and two having progressive disease. Our study reflects a prevalence of 10% underlying CPS in a limited cohort of patient based on the phenotype-genotype approach in our cohort. Using pre-defined clinical selection criteria, screening can be directed to a high-risk patient cohort with high-pick up rate for CPS. The selection criteria could be utilized in any LMIC-based clinical setup for pediatric cancer patients who may benefit from modification of treatment as well as genetic counseling.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"620-632"},"PeriodicalIF":1.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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 Comparative Analysis of Low Dose Grafalon^® Versus Thymoglobuline^® as Serotherapy in Hematopoietic Stem Cell Transplant in Pediatric and Young Adult Population. 小剂量格拉法隆®与胸腺球蛋白®在儿童和青少年造血干细胞移植中作为血清疗法的比较分析》（A Comparative Analysis of Low Dose Grafalon® Versus Thymoglobuline® as Serotherapy in Hematopoietic Stem Cell Transplant in Pediatric and Young Adult Population）。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-11-01 Epub Date: 2024-09-23 DOI: 10.1080/08880018.2024.2398523

Garima Nirmal, Gaurav Kharya, Ravi Shankar, Saksham Singh, Subhasish Paul, Mohit Choudhary, Vaibhav Chadha, Kamol Iskandarov, Sayitov Begali, Atish Bakane, Goutomi Chatterjee

{"title":"A Comparative Analysis of Low Dose Grafalon® Versus Thymoglobuline® as Serotherapy in Hematopoietic Stem Cell Transplant in Pediatric and Young Adult Population.","authors":"Garima Nirmal, Gaurav Kharya, Ravi Shankar, Saksham Singh, Subhasish Paul, Mohit Choudhary, Vaibhav Chadha, Kamol Iskandarov, Sayitov Begali, Atish Bakane, Goutomi Chatterjee","doi":"10.1080/08880018.2024.2398523","DOIUrl":"10.1080/08880018.2024.2398523","url":null,"abstract":"Anti-thymocyte globulin (ATG) forms an essential component of conditioning in hematopoietic stem cell transplantation (HSCT). Due to the shift of donor preference to alternate donors, reliance on rabbit-ATG (rATG) has increased. Two different forms of rATG (Thymoglobuline® and Grafalon®) are available for clinical use but data to support the use of one over the other is sparse. We retrospectively analyzed data of 144 patients who underwent allogenic-HSCT for benign hematological conditions at our center, from August 2019 to August 2023. Of these, 87 received Grafalon® and 57 received Thymoglobuline®. The majority (77.7%) underwent HSCT for hemoglobinopathies and all received pre-transplant immunosuppression. Engraftment kinetics was similar in 2 cohorts. Six patients had primary graft failure (PGF). There was no difference in the incidence of PGF stratified by serotherapy. Overall survival(OS) for the cohort was 74.9%. Kaplan-Meier estimate of OSand EFSwas significantly better in Grafalon® group than Thymoglobuline® (84.4 ± 0.04% vs 64.1% ±0.065%) (p-value= 0.04%) and (84.4 ± 0.04% and 61.2%±0.065% (p-value = 0.01)). Extensive chronic GVHD was (14%) higher in Thymoglobuline® group and (2.3%) in Grafalon®. Immune reconstitution at day + 100 was not statistically different between the two groups. On univariate analysis, Thymoglobuline® serotherapy (OR (95% CI) =4.665 (1.2-18.04))was associated with increased risk of acute grade III-IV GvHD. In our study, Grafalon® tended to have better OS, decreased incidence of acute grade III-IV GvHD, and extensive cGVHD. There was no difference in engraftment kinetics, PGF, and immune reconstitution between 2 cohorts of serotherapy.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"557-571"},"PeriodicalIF":1.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292896","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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