Pediatric Blood & Cancer最新文献

{"title":"Hypoalbuminemia and Nutritional Status in Children with Cancer.","authors":"Thomas W McLean, Natasha S Shah, Ronald D Barr, Janet A Tooze","doi":"10.1002/pbc.31450","DOIUrl":"https://doi.org/10.1002/pbc.31450","url":null,"abstract":"<p><strong>Background: </strong>In children with cancer, poor nutritional status adversely affects outcomes. Hypoalbuminemia is common in pediatric oncology patients, and in some groups is associated with inferior survival rates. We sought to determine if serum albumin associates with body mass index (BMI) percentile and if combining serum albumin and BMI is associated with survival.</p><p><strong>Methods: </strong>We performed a single institution, retrospective review of pediatric oncology patients and collected data regarding baseline BMI, serum albumin, and survival outcome. Combining baseline BMI and serum albumin, we classified patients' nutritional status as adequately nourished, mildly/moderately depleted, and severely depleted.</p><p><strong>Results: </strong>In a cohort of 490 pediatric oncology patients, hypoalbuminemia prevalence was 49%. Serum albumin did not associate with BMI percentile for age. Overall, those defined as severely depleted had an increased risk of relapse or death at 3 and 6 months from chemotherapy initiation compared with those defined as adequately nourished (hazard ratio [HR] = 2.37, 95% CI 1.29-4.37 at 3 months, p = 0.006; HR = 1.77, CI 1.11-2.82 at 6 months, p = 0.017). Statistical analyses suggest the inferior survival in those deemed severely depleted was primarily driven by hypoalbuminemia rather than BMI.</p><p><strong>Conclusions: </strong>In this cohort of pediatric oncology patients, serum albumin did not correlate with BMI. Severe hypoalbuminemia is an adverse prognostic factor. Baseline BMI had a minimal impact on relapse-free survival and overall survival, independently or in combination with hypoalbuminemia.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31450"},"PeriodicalIF":2.4,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142682167","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":"Implications of Implementing Children's Oncology Group Risk Stratification to Patients With Rhabdomyosarcoma Treated on European Paediatric Soft Tissue Sarcoma Study Group Clinical Trial.","authors":"Gianni Bisogno, Veronique Minard-Colin, Josephine Haduong, Ilaria Zanetti, Andrea Ferrari, Julia Chisholm, Christine M Heske, Raquel Hladun, Meriel Jenney, Johannes Hendrikus Maria Merks, Rajkumar Venkatramani","doi":"10.1002/pbc.31436","DOIUrl":"https://doi.org/10.1002/pbc.31436","url":null,"abstract":"<p><strong>Background: </strong>Prognostic factors are crucial in tailoring treatments for patients with rhabdomyosarcoma (RMS). The European paediatric Soft tissue sarcoma Study Group (EpSSG) and the Children's Oncology Group (COG) employ similar prognostic factors, but utilize them differently resulting in diverse stratification systems. This diversity may result in dissimilar treatment approaches for comparable patients and hinder the comparison of clinical trial results.</p><p><strong>Procedure: </strong>We reclassified 1993 patients enrolled in the EpSSG RMS 2005 and MTS 2008 studies based on the risk stratification used in current EpSSG and COG trials, and compared the type and cumulative doses of chemotherapy recommended to the different risk groups. Alkylating agents were compared using the cyclophosphamide equivalent dose formula. Metastatic RMS with high-risk features were excluded because no standard recommended treatment exists.</p><p><strong>Results: </strong>Patients were variably distributed across EpSSG and COG risk stratifications. Notably, 34.2% of EpSSG standard-risk patients fell into three different COG risk groups (very low, low, and intermediate), and 66.8% of the total population, classified as standard, high, and very high risk by EpSSG, would all be considered intermediate risk by COG. Consequently, only 57.3% of the study population would receive comparable intensive chemotherapy under both EpSSG and COG protocols. Disparities emerged, with 16.5% undergoing more intensive and 17.2% receiving less intensive treatment in COG protocols compared to EpSSG studies.</p><p><strong>Conclusions: </strong>Our study shows the complexities of the current RMS risk stratification systems, emphasizing the need for a global consensus. A unified approach would reduce the risk of disparate treatments for similar patients and facilitate more straightforward cross-study comparisons.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31436"},"PeriodicalIF":2.4,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142682307","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":"Systemic Inflammatory Response Post Alemtuzumab and Low-Dose Total Body Irradiation in Pediatric Patients With Sickle Cell Disease: A Case Series.","authors":"Rozalyn Chok, Katherine Girgulis, Robert S Nickel, Gregory M T Guilcher","doi":"10.1002/pbc.31448","DOIUrl":"https://doi.org/10.1002/pbc.31448","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31448"},"PeriodicalIF":2.4,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142682255","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":"A Rare t(X;21)(p11;q22) in Childhood Acute Myeloid Leukemia: Case Report and Literature Review.","authors":"Yiwen Du, Kun Yang, Yuqian Tang, Ruixia Fan, Yuping Gong","doi":"10.1002/pbc.31457","DOIUrl":"10.1002/pbc.31457","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31457"},"PeriodicalIF":2.4,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667489","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":"Pediatric Acute B-Lymphoblastic Leukemia Presenting as Hypereosinophilia With Lung Involvement and Elevated Immunoglobulin E Levels.","authors":"Aida Zeckanovic, Nastassja Scheidegger, Seraina Prader, Leo Thanikkel, Magdeldin Elgizouli, Nicole Bodmer","doi":"10.1002/pbc.31461","DOIUrl":"10.1002/pbc.31461","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31461"},"PeriodicalIF":2.4,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667835","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":"Centralized Investigator Review of Radiological and Functional Imaging Reports in Real-World Oncology Studies: The SACHA-France Experience.","authors":"Pablo Berlanga, Isabelle Aerts, Nadège Corradini, Lee Aymar Ndounga-Diakou, Natacha Entz-Werle, Stéphane Ducassou, Nicolas André, François Sevrin, Pascal Chastagner, Chloe Puiseux, Morgane Cleirec, Dominique Plantaz, Emilie De Carli, Marion Gambart, Camille Khanfar, Sandrine Thouvenin, Arnaud Petit, Sébastien Klein, Claire Briandet, Frédéric Millot, Claire Pluchart, Yves Reguerre, Pascale Schneider, Jill Serre, Carine Halfon-Domenech, Liana Carausu, Christophe Piguet, Laure Saumet, Joy Benadiba, Samuel Abbou, Salim Laghouati, Birgit Geoerger, Gilles Vassal","doi":"10.1002/pbc.31449","DOIUrl":"10.1002/pbc.31449","url":null,"abstract":"<p><p>SACHA-France (NCT04477681) is a prospective real-world study that collects clinical safety and efficacy data of novel anticancer therapies prescribed off-label or on compassionate use to patients <25 years. From March 2020 until February 2024, 640 patients with solid tumors or lymphomas were included, with 176 (28%) reported objective tumor responses. Centralized medical monitoring of local radiological/functional imaging reports by the SACHA coordinating investigator led to response modification in 45 out of 176 cases (26%), highlighting the relevance of the medical review of study data. We suggest this pragmatic approach for improving clinical trial data when centralized radiological review is not performed.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31449"},"PeriodicalIF":2.4,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667824","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":"Direct Parenchymal Injection of Indocyanine Green for Identification and Resection of an Osteosarcoma Pulmonary Metastasis.","authors":"Hemal Kodikara, Andrew Laing","doi":"10.1002/pbc.31447","DOIUrl":"10.1002/pbc.31447","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31447"},"PeriodicalIF":2.4,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667828","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":"Disseminated DIG-DIA With a Rare FMR1-RAF1 Fusion With Good Response to Targeted Therapy.","authors":"Laurence Reeves-Breton, Laurence Allain, Sayanthen Sathyakumar, Emily Boyer, Louis Crevier, Sebastien Perreault, Maxime Richer, Catherine Bourget, Panagiota Giannakouros, William LeGallou, Damien Faury, Julie Laflamme, Cynthia Hawkins, Eric Bouffet, Nada Jabado, Hallie Coltin, Samuele Renzi","doi":"10.1002/pbc.31454","DOIUrl":"10.1002/pbc.31454","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31454"},"PeriodicalIF":2.4,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667831","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":"Mitapivat for Acquired Pyruvate Kinase Deficiency.","authors":"Hanny Al-Samkari","doi":"10.1002/pbc.31440","DOIUrl":"https://doi.org/10.1002/pbc.31440","url":null,"abstract":"<p><p>Pyruvate kinase (PK) activation is emerging as a promising treatment modality for numerous congenital hemolytic anemias of diverse pathophysiology, and one agent, mitapivat, is already licensed to treat patients with congenital PK deficiency. However, PK deficiency may also be acquired in the setting of clonal myeloid disorders and other pathologies, where it may result in severe hemolytic anemia and remains without known therapies. This case report describes the novel application of mitapivat therapy in a patient with acquired PK deficiency causing red cell transfusion dependence, liberating the patient from transfusions and resulting in marked improvement in symptoms and quality of life.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31440"},"PeriodicalIF":2.4,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625671","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":"Comment on: Access to Legacy-Oriented Interventions at End of Life for Pediatric Oncology Patients: A Decedent Cohort Review.","authors":"Eman Zahra","doi":"10.1002/pbc.31438","DOIUrl":"https://doi.org/10.1002/pbc.31438","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31438"},"PeriodicalIF":2.4,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625643","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}