Pediatric Hematology Oncology Journal最新文献

{"title":"Pediatric mature B-cell non-hodgkin lymphoma in India: A retrospective multicenter pooled analysis of treatment approaches and outcomes","authors":"Nirmalya Roy Moulik ,&nbsp;Sameer Bakhshi ,&nbsp;Shripad Banavali ,&nbsp;Venkatraman Radhakrishnan ,&nbsp;Amita Trehan ,&nbsp;Anshul Gupta ,&nbsp;Niharendu Ghara ,&nbsp;Rachna Seth ,&nbsp;Ramandeep Singh Arora","doi":"10.1016/j.phoj.2024.11.104","DOIUrl":"10.1016/j.phoj.2024.11.104","url":null,"abstract":"<div><h3>Background</h3><div>Published data on outcomes of pediatric mature B-cell non-Hodgkin lymphoma (B-NHL) from India is limited and difficult to interpret due to small sample size and non-uniform treatment protocols. This study aims to do a pooled analysis of published patient data from multiple centers across India to provide a clearer understanding of survival rates and treatment-related toxicities with respect to the treatment protocols in this population.</div></div><div><h3>Methods</h3><div>A pooled analysis was conducted of patient data from 505 children with mature B-NHL, including Burkitt lymphoma (n = 395), diffuse large B-cell lymphoma (DLBCL, n = 52), and other subtypes (n = 58), treated from 2000 to 2022 at seven major cancer centers in India. Outcomes assessed were grade 3/4 toxicities, toxic deaths, relapse/progression, and survival rates.</div></div><div><h3>Results</h3><div>Most patients (401/505) presented with advanced disease; bone marrow and CNS involvement were observed in 13.9 % and 6.9 % of cases, respectively. Treatment protocols primarily included LMB (n = 208), BFM (n = 191), and MCP (n = 61). Grade 3/4 toxicities were reported in 79.2 % of patients, with higher rates observed with LMB protocol (92.1 %) compared to BFM (70.8 %) and MCP (70.1 %) (p &lt; 0.001). Toxic death rates were similar across protocols. Overall survival (OS) and event-free survival (EFS) at a median follow-up of 17 months were 69.4 ± 2.2 % and 64.9 ± 2.2 %, respectively, with no significant differences in relapse/progression rates or stage-specific OS between protocols (p = 0.28 and 0.51).</div></div><div><h3>Conclusions</h3><div>This pooled analysis shows that although treatment-related toxicities differ by protocol, overall survival outcomes were similar across the LMB, BFM, and MCP regimens, despite being much lower than those reported from high income countries. Uniform standardized protocols may further improve outcomes for pediatric B-NHL in India.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 33-40"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143697553","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":"Ewing sarcoma of the hands and feet: Outcome and prognostic factors of a rare subsite in a low-middle income country","authors":"Shuvadeep Ganguly ,&nbsp;Archana Sasi ,&nbsp;Chitrakshi Nagpal ,&nbsp;Bivas Biswas ,&nbsp;Sandeep Agarwala ,&nbsp;Deepam Pushpam ,&nbsp;Ahitagni Biswas ,&nbsp;Venkatesan Sampath Kumar ,&nbsp;Love Kapoor ,&nbsp;Shah Alam Khan ,&nbsp;Vishesh Jain ,&nbsp;Sameer Bakhshi","doi":"10.1016/j.phoj.2025.02.004","DOIUrl":"10.1016/j.phoj.2025.02.004","url":null,"abstract":"<div><h3>Background</h3><div>The small bones of the hand and feet represent a rare site of Ewing sarcoma (ES) origin. This study presents a real-world dataset describing the clinical presentation, survival outcomes, and their determinants in this subsite.</div></div><div><h3>Methods</h3><div>This is a single-institutional retrospective study of patients with ES originating from the hands/feet (ES-HF), treated between 2003 and 2018. Clinical/demographic details and survival outcomes were retrieved from medical records. Descriptive statistics were used to summarize baseline characteristics. Prognostic factors for event-free survival (EFS) and overall survival (OS) were identified by Cox regression. The clinical features and outcomes were compared between ES-HF and ES-others (ES from other sites) in the cohort.</div></div><div><h3>Results</h3><div>Of 859 ES cases, 28 (3.2 %) patients had ES-HF including four ES-hands (0.5 %) and 24 ES-feet (2.8 %). The calcaneum was the most common site [11 of 28 cases; 39.3 %]. More than half of the patients (53.6 %) had metastatic disease at presentation. In comparison with ES-others, ES-HF had longer median symptom duration [12 versus 4 months; p &lt; 0.001] and smaller tumor diameter [5.5 versus 9 cm; p &lt; 0.001]. The median EFS and OS of the cohort were 30.5 and 39.13 months respectively. Only local therapy receipt was associated with improved EFS (multivariable HR:0.013; 95%CI:0.001–0.158; p &lt; 0.001) and OS (multivariable HR:0.028; 95 % CI:0.003–0.272; p = 0.002). Patients receiving radiotherapy alone had inferior OS compared to those receiving surgery alone. (HR: 9.22; 95 % CI: 1.12–76.31; p = 0.039)</div></div><div><h3>Conclusion</h3><div>ES-HF is a rare ES subsite. Although indolent, metastases are common at presentation. Meticulous local control can improve survival in both localized and metastatic disease for this subsite.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 24-32"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143683014","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":"Cefiderocol for severe bloodstream infection by multidrug-resistant Klebsiella pneumoniae in a pediatric patient with acute myeloid leukemia","authors":"Francesco De Leonardis ,&nbsp;Francesca Marasciulo ,&nbsp;Roberta Koronica ,&nbsp;Mariachiara Servedio ,&nbsp;Enza Pentassuglia ,&nbsp;Vittorio Greco Miani ,&nbsp;Lidia Dalfino ,&nbsp;Stefania Stolfa ,&nbsp;Nicola Santoro","doi":"10.1016/j.phoj.2025.02.003","DOIUrl":"10.1016/j.phoj.2025.02.003","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 57-58"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Challenges faced in screening for thalassemia and other hemoglobinopathies in a North Indian population","authors":"Manisha Kumar,&nbsp;Vani Kargwal,&nbsp;Rajeev Goel,&nbsp;Ekta Debnath,&nbsp;Seema Malhotra,&nbsp;Kirti Balyan,&nbsp;Mahrukh Zaidi,&nbsp;Reena Yadav","doi":"10.1016/j.phoj.2025.02.005","DOIUrl":"10.1016/j.phoj.2025.02.005","url":null,"abstract":"<div><h3>Background</h3><div>Hemoglobinopathies are a common group of inherited disorders contributing significantly to the global healthcare burden, particularly in low and middle-income countries (LMICs) such as India.</div></div><div><h3>Objective</h3><div>This prospective cross-sectional study aimed to investigate the epidemiological, clinical, and genetic aspects of hemoglobinopathies in the antenatal population attending a tertiary care hospital in Delhi.</div></div><div><h3>Material and method</h3><div>A total of 7077 antenatal women were investigated over four years, revealing an overall prevalence of Beta Thalassemia Trait (BTT) of 4.55%. The screening algorithm involved a complete blood count (CBC) and High Performance Liquid Chromatography (HPLC) for all the women, followed by mutation testing using Multiplex amplification refractory mutation system - polymerase chain reaction (ARMS PCR) for four common mutations: IVS1-5 (G &gt; C), CD41/42 -TTCT, CD8/9 +G, and Del 619 bp. Multiplex ligation dependent probe amplification assay (MLPA), ARMS PCR and Sanger sequencing were used to identify other mutations. In cases where abnormal High Peformance Liquid Chromatography (HPLC) results were found, further testing of the husband was conducted to guide counselling and decisions regarding prenatal testing.</div></div><div><h3>Results</h3><div>IVS1-5 (G &gt; C) was identified as the most common mutation. Hemoglobin E (HbE) with CD26 (G &gt; A) exhibited the highest hemoglobin and HbA2 values amongst all mutations screened for. The study underscores the challenges of using RBC indices to diagnose BTT, due to marked overlap with Nutritional Deficiency Anemia (NDA). Similar issues were observed with HbD and HbE due to their relatively high MCV and MCH values. An algorithm for antenatal screening and diagnosis for LMIC is proposed but, given these caveats, its value remains to be proven.</div></div><div><h3>Conclusion</h3><div>The study provides data on the relative distribution of hemoglobinopathy mutations in the antenatal population in North India, and revealed the problems of detecting BTT or hemoglobinopathy in this population with a CBC due to the widespread prevalence of NDA. It emphasized the challenges of targeted screening strategies and genetic counselling to effectively reduce the prevalence of thalassemia in North India.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 59-65"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current management of pulmonary relapse in Ewing sarcoma: A report from the Pediatric Surgical Oncology Research Collaborative","authors":"Audra J. Reiter ,&nbsp;Lynn Huang ,&nbsp;Jennifer H. Aldrink ,&nbsp;Brian T. Craig ,&nbsp;Andrew M. Davidoff ,&nbsp;Lindsay J. Talbot ,&nbsp;Jordan Coggins ,&nbsp;Jasmine Smith ,&nbsp;Katherine C. Bergus ,&nbsp;Taleen A. MacArthur ,&nbsp;Stephanie F. Polites ,&nbsp;Roshni Dasgupta ,&nbsp;Chloe Boehmer ,&nbsp;Joseph Brungardt ,&nbsp;Marcus M. Malek ,&nbsp;Hannah N. Rinehardt ,&nbsp;Zachary J. Kastenberg ,&nbsp;Cameron M. Arkin ,&nbsp;Antoine Gourmel ,&nbsp;Nelson Piche ,&nbsp;Timothy B. Lautz","doi":"10.1016/j.phoj.2024.11.105","DOIUrl":"10.1016/j.phoj.2024.11.105","url":null,"abstract":"<div><h3>Background</h3><div>Relapse occurs in 30–40 % of patients with localized Ewing sarcoma (EWS). Our objective was to describe the current management and outcomes of patients with initially localized EWS who experience first pulmonary relapse.</div></div><div><h3>Methods</h3><div>This multi-center retrospective cohort study included patients ≤22 years old with initially localized EWS treated from 2007 to 2020 at 19 Pediatric Surgical Oncology Research Collaborative institutions, who developed pulmonary relapse. Kaplan-Meier analysis was performed.</div></div><div><h3>Results</h3><div>Thirty-three patients with initially localized EWS developed pulmonary relapse at a median age of 17 (IQR 14; 20) years. Eleven (33 %) patients also had extra-pulmonary metastases (EPM) at relapse. Among the 22 (67 %) patients with pulmonary-only relapse, 10 (45 %) had solitary pulmonary nodules. Pulmonary metastasectomy was performed in 8/10 (80 %) patients with solitary pulmonary-only metastases, 5/12 (42 %) patients with multiple pulmonary-only metastases, and 2/11 (18 %) patients who also had EPM. Whole lung irradiation was administered in 7/10 (70 %) with solitary pulmonary-only metastases, 7/12 (58 %) with multiple pulmonary-only metastases, and 2/11 (18 %) with EPM. Rates of further pulmonary relapse/progression were similar between groups (p = 0.97). In Kaplan-Meier analysis, 3-year overall survival was 73 % with solitary pulmonary-only metastases, 40 % with multiple pulmonary-only metastases, and 23 % with EPM (p = 0.097).</div></div><div><h3>Conclusions</h3><div>While survival for patients with relapsed EWS is poor, the subset of patients with solitary relapse confined to the lung are often good candidates for pulmonary metastasectomy and have a non-statistically significant trend towards improved survival outcomes.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 20-23"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143683013","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":"Prevalence of vitamin D deficiency in pediatric patients with sickle cell disease: A systematic review","authors":"Thiago de Souza Vilela ,&nbsp;Mauro Fisberg ,&nbsp;Gerson Ferrari ,&nbsp;Josefina Aparecida Pellegrini Braga","doi":"10.1016/j.phoj.2025.02.001","DOIUrl":"10.1016/j.phoj.2025.02.001","url":null,"abstract":"<div><div>Hypovitaminosis D is significantly more prevalent in sickle cell disease (SCD) compared to healthy control patients. The absence of adequate vitamin D levels in patients with a chronic inflammatory condition may exacerbate inflammation, worsening the disease. Therefore, a systematic review was conducted analyzing articles from the Medline database of the National Institute of Medicine between April 2023 and November 2023. Inclusion criteria were observational studies in pediatric populations up to 20 years old, published in English. Intervention studies, clinical trials, abstracts, and reviews were excluded. The final number of selected articles was 18 of 85 listed. The overall frequency of vitamin D deficiency in the pediatric age group with SCD was 50.49 % (569 out of 1127), considering 20 ng/mL (50 nmol/L) as the cutoff for deficiency, the most commonly used reference level for vitamin D deficiency in the listed studies. The prevalence of vitamin D deficiency in this population appeared to be higher when compared to children and adolescents without SCD. Our review concluded that vitamin D deficiency is prevalent in the pediatric population with SCD, but future studies should confirm the implications of this finding.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 42-47"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143704653","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":"Thalassemia major due to compound heterozygous Codon 8/9 (+G) mutation with Dutch beta° thalassemia deletion","authors":"Dolat Singh Shekhawat ,&nbsp;Tanuja Rajial ,&nbsp;Siyaram Didel ,&nbsp;Abhishek Purohit ,&nbsp;Kuldeep Singh","doi":"10.1016/j.phoj.2024.11.120","DOIUrl":"10.1016/j.phoj.2024.11.120","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 48-50"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869652","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Management outcomes of South African children diagnosed with neuroblastoma in adolescence","authors":"Van Heerden Jaques ,&nbsp;Esterhuizen Tonya ,&nbsp;Du Plessis Jan ,&nbsp;Geel Jennifer ,&nbsp;Büchner Ané ,&nbsp;Van Zyl Anel ,&nbsp;Hendricks Marc ,&nbsp;Naidu Gita ,&nbsp;van Emmenes Barry ,&nbsp;Vaithilingum M ,&nbsp;Kruger Mariana","doi":"10.1016/j.phoj.2024.12.003","DOIUrl":"10.1016/j.phoj.2024.12.003","url":null,"abstract":"<div><h3>Background</h3><div>Neuroblastoma (NB) is an indolent disease in adolescents. Only 5 % of neuroblastoma diagnoses are made in adolescents and adults. Limited data exists on Africans of older age groups.</div></div><div><h3>Objectives</h3><div>We aimed to compare the characteristics and outcomes of South African children ≥10 years diagnosed with NB with international reports.</div></div><div><h3>Patients and methods</h3><div>Multicentric, retrospective data of South African patients (0–15 y) with NB diagnosed between January 2000 and December 2016 were analyzed. Clinical profiles and outcomes of adolescents (≥10 years) with NB were compared to those of the younger patients in our cohort. The outcomes of adolescents (≥10 years) with NB were compared with those reported in the global literature.</div></div><div><h3>Results</h3><div>Thirty adolescents with a male-to-female ratio of 1:0.6 were diagnosed with NB, constituting 6.5 % of all patients with NB. Metastatic disease at diagnosis was present in 24 (75 %), including lung metastases in 2 (6 %). Half of the patients presented with a raised lactate dehydrogenase (LDH) and/or ferritin and unfavorable pathology.</div><div>Post-induction remission rates were higher in the adolescent group (33 %) compared to the 18–59 months (24.8 %) and 5–9.9 years (29.7 %) cohorts but less than the &lt;18 months (47.2 %) cohort (p &lt; 0.001). The adolescent group had the poorest five-year overall survival (20.9 %) compared to the children &lt;18 months (39.0 %), 18–59 months (44.1 %), or 5–9.9 years old (63.5 %) (p &lt; 0.001).</div></div><div><h3>Conclusions</h3><div>Despite a favorable response rate to induction chemotherapy, the five-year overall survival of adolescents with NB is dismal in South Africa.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 9-16"},"PeriodicalIF":0.0,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143164846","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 autoimmune hemolytic anemia – The good, bad and ugly!","authors":"Shakambaree Prusty,&nbsp;Vandana Bharadwaj,&nbsp;Somdipa Pal,&nbsp;M., Jyothi,&nbsp;Sidharth Totadri,&nbsp;Anand Prakash","doi":"10.1016/j.phoj.2025.100526","DOIUrl":"10.1016/j.phoj.2025.100526","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 4","pages":"Article 100526"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145537308","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Unlocking Pandora’s box: Phenotypic diversity and genotypic correlation in pediatric rare anemias","authors":"Minal Soneji,&nbsp;Anwesha Singh,&nbsp;Ritika Khurana,&nbsp;Purva Kanvinde,&nbsp;Minnie Bodhanwala,&nbsp;Sangeeta Mudaliar","doi":"10.1016/j.phoj.2025.100527","DOIUrl":"10.1016/j.phoj.2025.100527","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 4","pages":"Article 100527"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145537309","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}