Pediatric Hematology Oncology Journal最新文献

{"title":"An uncommon inborn error of immunity in an adolescent with Hodgkin lymphoma and bronchiectasis","authors":"Sangeetha Ramdas ,&nbsp;Sidharth Totadri ,&nbsp;Priyanka Medhi ,&nbsp;Elanthenral Sigamani ,&nbsp;Arun Kumar Arunachalam ,&nbsp;Leni Grace Mathew","doi":"10.1016/j.phoj.2024.03.008","DOIUrl":"10.1016/j.phoj.2024.03.008","url":null,"abstract":"<div><h3>Background</h3><p>Individuals with inborn errors of immunity (IEI) have an increased risk of developing malignancies compared to their peers. We report a case of Hodgkin <u>l</u>ymphoma in an adolescent with CD27 deficiency.</p></div><div><h3>Case report</h3><p>A 15-year-old girl presented with cervical swelling and breathlessness for 3 days. Her past history was remarkable, with a history of recurrent respiratory infections. On examination, she had grade 2 clubbing, bilateral cervical lymphadenopathy, hepatosplenomegaly, and bilateral coarse crepitations. Biopsy showed overlapping immunomorphological features of classic Hodgkin lymphoma (HL), with features intermediate between diffuse large B-cell lymphoma and HL. A staging PET-CT revealed a stage III disease and bronchiectatic changes in bilateral lungs. The serum immunoglobulin levels showed hypogammaglobulinemia. Next generation sequencing demonstrated a homozygous missense variant in the CD27 gene (c.319C&gt;T; p.Arg107Cys). She was treated with ABVD/COPDac chemotherapy along with supportive care. She is currently 16 months post-treatment.</p></div><div><h3>Conclusion</h3><p>CD 27 deficiency is a rare IEI with a common variable immunodeficiency phenotype and a high propensity to develop lymphomas. Clinical suspicion, early detection, and management are warranted to prevent complications and mortality.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 125-128"},"PeriodicalIF":0.0,"publicationDate":"2024-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000202/pdfft?md5=e50cbebf6fa8609f912f426c85a76377&pid=1-s2.0-S2468124524000202-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140275089","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emergency management of a 13-year-old patient with primary mediastinal B cell lymphoma: Extracorporeal membrane oxygenation and superior vena cava stent prior to chemotherapy","authors":"Salvador R. Maffei ,&nbsp;Gary Stapleton ,&nbsp;Katherine Doane ,&nbsp;James Thomas ,&nbsp;M Hossein Tcharmtchi ,&nbsp;Julienne Brackett ,&nbsp;Saleh Bhar","doi":"10.1016/j.phoj.2024.03.010","DOIUrl":"https://doi.org/10.1016/j.phoj.2024.03.010","url":null,"abstract":"<div><h3>Background</h3><p>Primary mediastinal B cell lymphoma (PMBCL) is a rare type of non-Hodgkin's lymphoma, and prompt diagnosis and initiation of chemotherapy are necessary to limit compression of cardiovascular structures.</p></div><div><h3>Case report</h3><p>A 13-year-old patient was diagnosed with PMBCL, resulting in acute hypoxemic respiratory failure and cardiogenic shock with clinical pericardial tamponade. Chemotherapy was initiated after veno-arterial extracorporeal membrane oxygenation and subsequent endovascular stenting of the superior vena cava (SVC) to optimize cardiac output due to malignant SVC syndrome.</p></div><div><h3>Conclusion</h3><p>We discuss the challenges of diagnosis and emergency management of pediatric patients with PMBCL and review the existing relevant literature for SVC syndrome secondary to PMBCL.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 3","pages":"Pages 129-132"},"PeriodicalIF":0.0,"publicationDate":"2024-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000299/pdfft?md5=3055232a31419eafdc7570941430d2f9&pid=1-s2.0-S2468124524000299-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140621924","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient-related factors and outcome of retinoblastoma in Calabar, Nigeria: A single-center, retrospective study","authors":"Elizabeth D. Nkanga ,&nbsp;Anthony C. Nlemadim ,&nbsp;Mattan Arazi ,&nbsp;Dennis G. Nkanga ,&nbsp;Roseline E. Duke ,&nbsp;Ido D. Fabian ,&nbsp;Essemfon D. Nkanga ,&nbsp;Friday A. Odey ,&nbsp;Martin M. Meremikwu","doi":"10.1016/j.phoj.2024.03.007","DOIUrl":"10.1016/j.phoj.2024.03.007","url":null,"abstract":"<div><h3>Background</h3><p>Retinoblastoma is associated with mortality in resource-poor nations due to disparities and poor access to treatment. The aim was to determine the relationships between patient-related factors and clinical outcomes of retinoblastoma in a tertiary hospital in Nigeria.</p></div><div><h3>Material and methods</h3><p>It was a retrospective study of all children diagnosed and treated for retinoblastoma from January 2017 through December 2022. Information obtained from their records included bio-socioeconomic data, symptoms, lag time from initial symptoms, staging, treatment, and survival outcome.</p></div><div><h3>Results</h3><p>Fifty-three patients, aged 6–88 months on first hospital presentation were recruited. There were 29 (54.7%) females. Twenty (37.7%) patients died. The majority were the last child of their parents (62.3%) with a low socioeconomic class (86.8%) and lived in rural areas (50.9%). The median (interquartile) age at diagnosis [24 (18–36) months, <em>p</em> = 0.005] and lag time [13 (6–20) months, <em>p</em> = 0.274] were lower in those who survived than in those who died. The prevalence of bilateral disease (20.8%, <em>p</em> <strong>=</strong> 0.002), brain metastasis (22.6%, <em>p</em> <strong>&lt;</strong> 0.001), stage IV disease (18.9%, <em>p</em> <strong>=</strong> 0.01) and relapse (34%, <em>p</em> <strong>&lt;</strong> 0.001) was higher among the patients who died. The median (interquartile) overall survival (OS) was 22 (11.8–32.2) months with a 1-year OS of 63%. Treatment with only chemotherapy [HR 4.76 (95% CI: 1.7–13.1)], incomplete chemotherapy [HR 5.61 (95% CI: 1.3–24.7)], relapse [HR 5.98 (95% CI: 1.4–25.9)] and eye surgery delayed after 3 chemotherapy cycles [HR 8.22 (95% CI: 1.1–62.2)] were predictors of mortality.</p></div><div><h3>Conclusion</h3><p>Most patients with retinoblastoma arrived at our treatment center approximately 14 months following the first symptom. Most (84.9%) presented with proptosis. The majority were of a low social class (86.8%), had a secondary level of education only (47.2%), and lived in rural areas (50.9%). The 3-year overall survival was 29%.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 87-94"},"PeriodicalIF":0.0,"publicationDate":"2024-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000196/pdfft?md5=e4c830646754795f877ed0907e3dc1e1&pid=1-s2.0-S2468124524000196-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140275035","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pediatric inflammatory multisystem syndrome in a patient with Hodgkin lymphoma: A case report","authors":"Carlos Julian Díaz-Torres ,&nbsp;Irina Katia Cano Flores ,&nbsp;Julio A. Poterico","doi":"10.1016/j.phoj.2024.03.009","DOIUrl":"10.1016/j.phoj.2024.03.009","url":null,"abstract":"<div><h3>Background</h3><p>The inflammatory state of Hodgkin lymphoma can generate increases in inflammatory markers that can sometimes be attributed to an infection in these immunosuppressed patients. During the COVID-19 pandemic, the diagnosis and treatment of patients with hematological malignancies was complicated by the presence of SARS-CoV2. It could range from an asymptomatic presentation to a multisystem inflammatory syndrome known as the Pediatric inflammatory multisystem syndrome (PIMS).</p></div><div><h3>Case report</h3><p>We present the case of a girl who, during the COVID-19 pandemic, was treated at our institution for lymphadenopathy and persistent fever. The febrile syndrome was attributed to intercurrent infection; however, despite antimicrobial coverage, fever persisted, and inflammatory markers increased. Although SARS-CoV2 was not detected in the samples, PIMS was clinically suspected. Treatment with human immunoglobulin and acetylsalicylic acid was administered. An improvement in the inflammatory parameters was observed, which allowed ruling out an opportunistic infection and initiating chemotherapy.</p></div><div><h3>Conclusion</h3><p>A patient diagnosed with lymphoma and presenting with PIMS could have a synergic interaction of both conditions, resulting in an adverse outcome.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 95-100"},"PeriodicalIF":0.0,"publicationDate":"2024-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000214/pdfft?md5=fcc97a4d35714b17da085869ada28f48&pid=1-s2.0-S2468124524000214-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140274894","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A unique case series of pediatric autoimmune hemolytic anemia","authors":"Sanghamitra Ray ,&nbsp;Anuradha Rai ,&nbsp;Manish Kumar ,&nbsp;Diganta Saikia","doi":"10.1016/j.phoj.2024.03.002","DOIUrl":"10.1016/j.phoj.2024.03.002","url":null,"abstract":"<div><h3>Background</h3><p>Autoimmune hemolytic anemia (AIHA) is a group of hematological disorders where there is autoantibody mediated destruction of red blood cells. It can be life threatening if not appropriately treated. Early diagnosis and work up and timely multipronged management is the key to success.</p></div><div><h3>Case report</h3><p>We report four cases of diverse etiology - one each of post SARS-CoV-2 AIHA, idiopathic AIHA, drug-induced AIHA and common variable immunodeficiency (CVID) associated Evans syndrome. All were treated with steroids as the first line agent while the child with CVID required additional immunosuppressive therapy.</p></div><div><h3>Conclusion</h3><p>This case series re-emphasizes the need to look for diverse etiologies in AIHA. The role of whole exome sequencing is discussed for a definitive diagnosis if accessible in selected cases.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 78-81"},"PeriodicalIF":0.0,"publicationDate":"2024-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000147/pdfft?md5=8a162d2e563334b3cbd5712070d71b6e&pid=1-s2.0-S2468124524000147-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140279321","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Isosexual precocious pseudopuberty during mitotane treatment in a child with adrenocortical carcinoma: A case report","authors":"Maria Riedmeier ,&nbsp;Sonir Antonini ,&nbsp;Clemens Benoit ,&nbsp;Cheri L. Deal ,&nbsp;Fassnacht Martin ,&nbsp;Bonald C. Figueiredo ,&nbsp;Elmas Nazli Gonc ,&nbsp;Christoph Härtel ,&nbsp;Jan Idkowiak ,&nbsp;Max Kurlbaum ,&nbsp;Ronald de Krijger ,&nbsp;Raul C. Ribeiro ,&nbsp;Jaydira del Rivero ,&nbsp;Paul-Gerhardt Schlegel ,&nbsp;Lester D.R. Thompson ,&nbsp;Bilgehan Yalcin ,&nbsp;Verena Wiegering","doi":"10.1016/j.phoj.2024.03.005","DOIUrl":"10.1016/j.phoj.2024.03.005","url":null,"abstract":"<div><h3>Background</h3><p>Mitotane is employed as adjuvant therapy in managing adrenocortical carcinoma in pediatric patients. While various adverse effects, such as estrogen-like manifestations, are well-documented in adults, there is limited knowledge regarding pediatric-specific toxicity. This report details an uncommon case of isosexual precocious pseudopuberty induced during childhood due to the estrogen-like effects of mitotane.</p></div><div><h3>Case report</h3><p>A 2.8-year-old female diagnosed with adrenocortical carcinoma (pT4 pN0 M0) underwent adjuvant treatment with mitotane and cytotoxic chemotherapy following incomplete resection (tumor stage III). Approximately eight months into mitotane treatment, she exhibited signs of puberty (Tanner stage 2), including progressive breast development, uterine enlargement, vaginal discharge, and an advancement of bone age by nearly two years. Gonadotrophin-dependent puberty and endogenous estrogen production were ruled out. The precocious pseudopuberty was attributed to previously reported estrogen-like effects of mitotane therapy. Subsequent administration of the aromatase inhibitor anastrozole in combination with mitotane led to a reduction in clinical signs of puberty.</p></div><div><h3>Conclusion</h3><p>Monitoring for estrogen-like effects of mitotane is crucial, particularly in pre-pubertal children, to avert potentially irreversible changes associated with precocious pseudopuberty. Aromatase inhibitors may serve as a prompt therapeutic option, enabling the continuation of mitotane treatment.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 74-77"},"PeriodicalIF":0.0,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000172/pdfft?md5=bb12fece58d29e51f39003b70aca4599&pid=1-s2.0-S2468124524000172-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140276979","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An unusual association of deletion of SMARCB1 in a patient with intracranial yolk sac tumor: A case-report","authors":"A. Gupte ,&nbsp;E. Al-Antary ,&nbsp;K. Regling ,&nbsp;W.J. Kupsky ,&nbsp;D. Altinok ,&nbsp;C. Koschmann ,&nbsp;S. Camelo-Piragua ,&nbsp;K. Bhambhani","doi":"10.1016/j.phoj.2024.03.004","DOIUrl":"10.1016/j.phoj.2024.03.004","url":null,"abstract":"<div><h3>Background</h3><p>Deletion of <em>SMARCB1</em>/loss of INI is a well-known association in atypical rhabdoid teratoid tumors (ATRT) in the brain, rhabdoid tumors in the kidney, and less common tumors, including sinonasal INI1 deficient carcinoma, gastric undifferentiated carcinoma, undifferentiated uterine sarcomas, and poorly differentiated chordomas.</p></div><div><h3>Case report</h3><p>We describe homozygous deletion of the <em>SMARCB1</em> gene in a patient diagnosed with pineal yolk sac tumor, which is a rare entity. The association highlights the importance of INI1 staining when the clinical course is not progressing as expected and raises a critical management question: should this rare entity be treated aggressively, like ATRT, versus the conventional approach to intracranial yolk sac tumor?</p></div><div><h3>Conclusion</h3><p>This case highlights the importance of INI1 staining in pediatric primitive central nervous system tumors as some germ cell markers are expressed in rhabdoid tumors at the stem cell level, implicating the germ cell origin of ATRT, which can complicate the diagnosis.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 82-86"},"PeriodicalIF":0.0,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000160/pdfft?md5=e5ff084ee88c8ecda39db3215871a0a5&pid=1-s2.0-S2468124524000160-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140280739","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mitapivat: New dawn in pyruvate kinase deficiency and beyond","authors":"Ritika Khurana,&nbsp;Sangeeta Mudaliar","doi":"10.1016/j.phoj.2024.03.003","DOIUrl":"10.1016/j.phoj.2024.03.003","url":null,"abstract":"<div><p>Mitapivat is the first in class oral allosteric activator of pyruvate kinase enzyme, leading to increased ATP production. Since red blood cells (RBC) rely on anaerobic metabolism, converting phosphoenolpyruvate to pyruvate in Embden– Meyerhof glycolytic pathway is the most important step for ATP production. Deficiency of ATP in patients with pyruvate kinase deficiency (PKD) leads to the destruction of RBCs. In hemoglobinopathies, including thalassemia and sickle cell disease, increased stress and utilization leads to rapid depletion of ATP resources.</p><p>Phase II DRIVE PK study was the first randomized controlled trial that showed benefits in adult patients without regular transfusion requirement in regards to a rise in hemoglobin ≥1.0 g/dl and improvement in other parameters of hemolysis even with a low 50 mg twice daily dose. Minor adverse effects, including headache, insomnia, and nausea were reported.</p><p>Subsequent adult studies like ACTIVATE III (non-transfusion-dependent) and ACTIVATE III – T (transfusion-dependent) in patients with PKD showed sustained hemoglobin response in 16/40 (40%) patients. It was tolerated well, and the adverse effect profile was similar to the previous study except for hypertriglyceridemia and hypertension in two patients.</p><p>Phase I/II trials on patients with thalassemia and sickle cell anemia have also shown promising results in reducing transfusion burden and other disease-related co-morbidities, paving the way for further studies.</p><p>Mitapivat appears to be a safe, well-tolerated, and effective drug for PKD and other RBC pathologies in adults. Results of ongoing pediatric studies in these settings are awaited to reveal its safety profile in children.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 2","pages":"Pages 101-104"},"PeriodicalIF":0.0,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000159/pdfft?md5=c658ca6de7604bd64b802b581d8c3232&pid=1-s2.0-S2468124524000159-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140281149","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rare red cell enzymopathies in the Indian population: A comprehensive review","authors":"","doi":"10.1016/j.phoj.2024.03.006","DOIUrl":"10.1016/j.phoj.2024.03.006","url":null,"abstract":"<div><p>Red blood cell enzyme deficiencies are a rare category of hemolytic anaemia that typically present in children with varying degrees of hemolysis, indirect hyperbilirubinemia and splenomegaly. Glucose 6-phosphate dehydrogenase (G6PD) is the most common enzyme deficiency, followed by pyruvate kinase deficiency (PKD). This article aims to understand the pathophysiology of the rare enzymopathies due to deficiencies in the Embden<strong>-</strong>Meyerhoff pathway, glutathione metabolism, hexose monophosphate shunt and nucleotide metabolism pathway, and to study the incidence, clinical symptoms, diagnostic strategy, and management of enzyme deficiencies in the Indian patients. Most red blood cell (RBC) enzyme deficiencies are inherited in an autosomal recessive fashion except for G6PD, and phosphoglycerate kinase deficiency which has X-linked inheritance. Presentation depends on the representation of the enzyme on different tissues of the body, hence some enzyme deficiencies may present with neurological or muscular manifestations. In patients with suspected hemolytic anaemia, complete blood count and peripheral smear help in differentiating from hemoglobinopathies, etc. To clinch the diagnosis enzyme levels and genetic testing may be required. These tests guide antenatal diagnosis in subsequent pregnancies. Management of RBC enzymopathies depends on the predominant symptoms and severity of hemolysis. Patients with marked hemolysis require regular blood transfusions and hence appropriate chelation therapy. Hematopoeitic stem cell transplant is attempted in patients with severe spectrum with variable results. Newer drugs, including mitapivat have proven to be beneficial in adults with PKD and ongoing trials in children are showing promising results.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 4","pages":"Pages 235-243"},"PeriodicalIF":0.0,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000184/pdfft?md5=e76d42fbc8beeb1adcf50858df264ab4&pid=1-s2.0-S2468124524000184-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140278347","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Peg-asparaginase associated toxicities in children with acute lymphoblastic leukemia: A single-center cross-sectional study","authors":"Sameh Awwad ,&nbsp;Rawan Abu Alnasr ,&nbsp;Fahad Almanjomi ,&nbsp;Murtada Al Sultan ,&nbsp;Jude Howaidi ,&nbsp;Mohammed Almotairi ,&nbsp;Issam AlFayyad","doi":"10.1016/j.phoj.2024.03.001","DOIUrl":"https://doi.org/10.1016/j.phoj.2024.03.001","url":null,"abstract":"<div><h3>Background</h3><p>Leukemia is the most prevalent type of cancer affecting children, representing approximately 35% of cancer diagnoses in Saudi Arabia. Notably, significant improvement in the overall survival and cure rates for pediatrics with acute lymphoblastic leukemia (ALL) has been attributed to adding asparaginase to the chemotherapy regimen. However, the administration of these agents may lead to a multifactorial range of toxicities that often alter treatment outcomes.</p></div><div><h3>Objective</h3><p>The study aimed to characterize the prevalence of common toxicities related to polyethylene glycol (PEG) asparaginase in children aged 0–14 years diagnosed with ALL. Additionally, it assessed the types of toxicities associated with intravenous (IV) and intramuscular (IM) administration of Peg-asparaginase.</p></div><div><h3>Method</h3><p>It was an observational retrospective cross-sectional study. Data was extracted from the hospital's electronic health record (EPIC). Using EPIC, we reviewed medical charts of all pediatric patients below 14 years old diagnosed with ALL who received at least one dose of PEG-asparaginase between January 2020 and March 2023. The toxicity grading was based on the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE).</p></div><div><h3>Results</h3><p>One hundred and ninety-one patients with ALL were included in the study. Overall, 79 toxicity episodes were experienced by the study patients. Anaphylaxis/hypersensitivity (36.7%) and hepatotoxicity (31.6%) were the most prevalent toxicities reported, followed by pancreatitis and hyperglycemia (12.7% each). According to the CTCAE grading, approximately 70% of toxicities were categorized as Grade 3 and 4. Notably, 60% of the events occurred during the induction and consolidation phase of therapy. Interestingly, there was no significant difference in rates of toxicities between patients receiving IV or IM PEG-asparaginase.</p></div><div><h3>Conclusion</h3><p>The distribution of toxicities highlighted in our study aligns with findings from previously published studies. Furthermore, multivariate analysis indicated that patients with high-risk ALL and T-ALL were more likely to develop toxicities compared to other forms of ALL.</p></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"9 1","pages":"Pages 54-62"},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2468124524000135/pdfft?md5=a3f1f110aeb21e3069f8ad8db17052a8&pid=1-s2.0-S2468124524000135-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140135164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}