Diana Vargas, Luisa Chantada, Bruno Cuturi, Anaulina Silveira, Ángeles Rodríguez, Luján Guerrero, Lucia Díaz, Mariela Castiglioni, Fabiana Morosini, Carolina Pages, Elizabeth Simón, Luis Castillo
{"title":"Real-world implementation of North American and SIOP strategies for the treatment of Wilms tumor in Uruguay.","authors":"Diana Vargas, Luisa Chantada, Bruno Cuturi, Anaulina Silveira, Ángeles Rodríguez, Luján Guerrero, Lucia Díaz, Mariela Castiglioni, Fabiana Morosini, Carolina Pages, Elizabeth Simón, Luis Castillo","doi":"10.1080/08880018.2024.2345662","DOIUrl":"10.1080/08880018.2024.2345662","url":null,"abstract":"<p><p>Wilms tumor has been selected as an index tumor by the WHO Global Initiative for Childhood Cancer with the aim to improve cure rates worldwide. Nevertheless, there is a scarcity of published data on outcomes beyond those of the major cooperative groups. Therefore, we conducted a retrospective analysis including all patients with Wilms tumor treated at our referral center in Uruguay between 1995 and 2020. Treatment consisted of North American (NA) strategies in 23 cases (1995-2004), followed by the SIOP strategy in 35 cases thereafter. Staging included: I-II = 28, III = 7, IV = 14, and <i>V</i> = 9. There were no major surgical or medical complications; however, a delay in the administration of local radiotherapy was observed (median of 21 days after surgery). There were no cases of toxicity- or surgery-related deaths or treatment abandonment. Five-year probability of overall survival was 0.72 and 0.92 for the NA and SIOP groups, respectively. We conclude that outcomes were better for the SIOP strategy with no unexpected toxicities and high treatment compliance in both strategies. Timely implementation of radiotherapy was challenging.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141555316","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}
Acacia Bowden, Jeanette Zambito, Jinia El-Feghaly, Jeffrey R Andolina
{"title":"Adjuvant immune checkpoint inhibitor therapy may benefit pediatric patients with stage III melanoma and sentinel lymph node positivity: a case series.","authors":"Acacia Bowden, Jeanette Zambito, Jinia El-Feghaly, Jeffrey R Andolina","doi":"10.1080/08880018.2024.2350455","DOIUrl":"10.1080/08880018.2024.2350455","url":null,"abstract":"<p><p>Melanoma is the most common skin cancer in children. While the current literature establishes treatment protocols for adult-type melanoma, very few pediatric-specific studies exist, and children are often excluded from melanoma clinical trials<sup>2</sup>. We report a case series of 23 pediatric patients aged 2-20 years old diagnosed with melanoma at the University of Rochester Medical Center between 1/1/2011 and 1/1/2022. 9/23 patients were Stage III; all patients underwent wide local excision and 9 received adjuvant therapies. 2/23 (8.7%) patients had recurrence of their malignancy after therapy while 21/23 (91.3%) remained without disease progression; 1 patient died from unknown cause, but the rest are alive and currently without disease. All patients whose initial therapy included nivolumab in addition to wide local excision did not have recurrence or progression of their disease. This case series highlights trends in the presentation, treatment, and outcomes of pediatric melanoma; however, additional multi-center studies are needed to establish the clinical utility of such features in pediatric melanoma.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140862224","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}
Ariella Barhen, Paul A Martinez, Prithvi Sendi, Balagangadhar R Totapally
{"title":"Characteristics and complications of acute promyelocytic leukemia in children: an analysis of a national database.","authors":"Ariella Barhen, Paul A Martinez, Prithvi Sendi, Balagangadhar R Totapally","doi":"10.1080/08880018.2024.2352727","DOIUrl":"10.1080/08880018.2024.2352727","url":null,"abstract":"<p><p>Acute promyelocytic leukemia (APL) is an uncommon subtype of acute myelogenous leukemia (AML) that was previously one of the most fatal forms of acute leukemia. With advances in diagnosis and treatment, APL has become one of the most curable myeloid leukemias. The major reason for treatment failure in APL is early death after initiation of treatment. We performed a retrospective cross-sectional analysis of the Healthcare Cost and Utilization Project 2016 and 2019 Kids' Inpatient Database, with the diagnosis of APL or AML not in remission as defined by ICD-10-CM codes. We compared complications and outcomes associated with APL and AML (exclusive of APL) in hospitalized children in the U.S. and described yearly national incidence. The national incidence of APL was 2.2 cases per million children per year. Children with APL were more likely to have cardiopulmonary complications (OR 1.79; CI 1.20-2.67; <i>p</i> = 0.004), coagulation abnormalities or DIC (OR 7.75; CI 5.81-10.34; <i>p</i> < 0.001), pulmonary hemorrhage (OR 2.18; CI 1.49-3.17; <i>p</i> < 0.001), and intracranial hemorrhage (OR 10.82; CI 5.90-19.85; <i>p</i> < 0.001) and less likely to have infectious complications (OR 0.48; CI 0.34-0.67; <i>p</i> < 0.001) compared to children with AML. In-hospital mortality rates were similar in children with APL and AML (4.2% vs 2.6%; OR 1.62; CI 0.86-3.06; <i>p</i> = 0.13), while the median length of stay for children who died from APL was shorter compared to AML (2 (IQR: 1-7) versus 25 (IQR: 5-66) days; <i>p</i> < 0.05). Hemorrhagic complications occur more often, and infectious complications occur less often in hospitalized children with APL compared to AML.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141555313","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}
{"title":"The relationship between liver stiffness by two-dimensional shear wave elastography and iron overload status in transfusion-dependent patients.","authors":"Pimporn Puttawibul, Supika Kritsaneepaiboon, Thirachit Chotsampancharoen, Polathep Vichitkunakorn","doi":"10.1080/08880018.2024.2353900","DOIUrl":"10.1080/08880018.2024.2353900","url":null,"abstract":"<p><p>Increased liver stiffness (LS) can be result of increased liver iron concentration (LIC) which may not yet be reflected in the liver fibrotic status. The objective of our study was to examine relationship between hemochromatosis, LS, and serum ferritin level in transfusion-dependent patients. We recruited all 70 transfusion-dependent patients, whose median age was 15, referred for evaluating LIC status by magnetic resonance imaging (MRI) followed by two-dimensional ultrasonography shear wave elastography (2D-SWE). Thalassemia beta affected the majority of the patients. The optimal cut point for prediction of severe hemochromatosis using median SWE (kPa) and SWV (m/s) was ≥ 7.0 kPa and ≥ 1.54 m/s, respectively, with sensitivity of 0.76 (95% confidence interval [CI] 0.55, 0.91) and, specificity of 0.69 (95%CI 0.53, 0.82). When combing the optimal cut point of SWE (kPa) at ≥ 7.0 and serum ferritin ≥ 4123 ng/mL, the sensitivity increased to 0.84 (95%CI 0.64, 0.95) with specificity of 0.67 (95%CI 0.50, 0.80), positive predictive value (PPV) of 0.60 (95%CI 0.42, 0.76), and negative predictive value (NPV) of 0.88 (95%CI 0.71, 0.96). Simultaneous tests of 2D-SWE and serum ferritin for prediction of severe hemochromatosis showed the highest sensitivity of 84% (95%CI 0.64-0.95), as compared to 2D-SWE alone at 76% (95%CI 0.55, 0.91) or serum ferritin alone at 44% (95%CI 0.24-0.65). We recommend measuring both 2D-SWE and serum ferritin in short interval follow up patients. Adding 2D-SWE to management guideline will help in deciding for aggressive adjustment of iron chelating medication and increased awareness of patients having severe hemochromatosis.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559453","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}
Yessenia L Molina, Luis Blasco-Santana, Alejandro Sanz, Cristina Julia Blázquez Gómez, Josune Zubicaray, June Iriondo, Jesús González de Pablo, Julián Sevilla, Elena Sebastián
{"title":"Myelofibrosis associated with immune cytopenia in an infant: a diagnostic and therapeutic challenge.","authors":"Yessenia L Molina, Luis Blasco-Santana, Alejandro Sanz, Cristina Julia Blázquez Gómez, Josune Zubicaray, June Iriondo, Jesús González de Pablo, Julián Sevilla, Elena Sebastián","doi":"10.1080/08880018.2024.2350424","DOIUrl":"10.1080/08880018.2024.2350424","url":null,"abstract":"","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140870334","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}
{"title":"The Italian Consensus Conference on the role of rehabilitation for children and adolescents with leukemia, central nervous system tumors, and bone cancer, part 2: general principles for the rehabilitation treatment of motor function impairments.","authors":"Francesca Rossi, Stefano Botti, Mattia Morri, Sebastian Asaftei, Daniele Bertin, Simona Breggiè, Roberto Casalaz, Marta Cervo, Paola Ciullini, Monica Coppo, Annalisa Cornelli, Maria Esposito, Miriana Ferrarese, Marina Ghetti, Lucia Longo, Gabriella Naretto, Nicoletta Orsini, Daniele Panzeri, Chiara Pellegrini, Michela Peranzoni, Antonella Perna, Nadine Petit, Fabiola Picone, Gianna Pittorru, Debora Raffa, Veronica Recchiuti, Lucia Rizzato, Marina Sarzana, Raffaella Sensi, Franca Fagioli, Federica Ricci","doi":"10.1080/08880018.2024.2353360","DOIUrl":"10.1080/08880018.2024.2353360","url":null,"abstract":"<p><p>In Italy, 1400 children and 800 adolescents are diagnosed with cancer every year. About 80% of them can be cured but are at high risk of experiencing severe side effects, many of which respond to rehabilitation treatment. Due to the paucity of literature on this topic, the Italian Association of Pediatric Hematology and Oncology organized a Consensus Conference on the role of rehabilitation of motor impairments in children/adolescents affected by leukemia, central nervous system tumors, and bone cancer to state recommendations to improve clinical practice. This paper includes the consensus on the rehabilitation of children and adolescents with these cancers.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141564023","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}
{"title":"Co-transplantation of umbilical cord mesenchymal stem cells and peripheral blood stem cells in children and adolescents with refractory or relapsed severe aplastic anemia.","authors":"Fangfang Yuan, Minghui Li, Xudong Wei, Yuewen Fu","doi":"10.1080/08880018.2024.2324394","DOIUrl":"10.1080/08880018.2024.2324394","url":null,"abstract":"<p><p>To evaluate the co-transplantation efficacy of umbilical cord mesenchymal stem cells (UC-MSCs) and peripheral blood stem cells (PBSCs) as a novel approach for refractory or relapsed severe aplastic anemia (R/R SAA) in children and adolescents, thirty-two children and adolescents diagnosed with R/R SAA underwent a retrospective chart review. The patients were categorized into two groups based on the source of PBSCs: the matched sibling donor (MSD) group and the unrelated donor (UD) group. No adverse events related to UC-MSC infusion occurred in any of the patients. The median time for neutrophil engraftment was 13 days (range: 10-23 days), and for platelets, it was 15 days (range: 11-28 days). Acute GVHD of Grade I-II and moderate chronic GVHD were observed in 21.8 and 12.5% of cases, respectively. No statistically significant differences were found between the MSD and UD groups in terms of engraftment, GVHD, and complications, including infection and hemorrhagic cystitis. The median follow-up time was 38.6 months (range: 1.4-140.8 months). As of October 31, 2021, five patients had succumbed, while 27 (84.4%) survived. The 5-year OS rate showed no statistically significant difference between the MSD and UD groups (84.8 ± 10.0 <i>vs.</i> 82.4 ± 9.2%, <i>p</i> = 0.674). In conclusion, the application of UC-MSCs in the treatment of R/R SAA in PBSC transplantation is reliable and safe, they had no graft rejection, low incidence of severe GVHD which may have been contributed by the co-infusion of MSC.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140022292","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}
Jamie M Brown, Sherry Mathew, Maria Luisa Sulis, L Lee Dupuis, Jennifer Thackray
{"title":"Vomiting in children and adolescents receiving intravenous pegaspargase: a retrospective study.","authors":"Jamie M Brown, Sherry Mathew, Maria Luisa Sulis, L Lee Dupuis, Jennifer Thackray","doi":"10.1080/08880018.2024.2311886","DOIUrl":"10.1080/08880018.2024.2311886","url":null,"abstract":"<p><p>Due to an evidence gap, the emetogenicity of intravenous (IV) pegaspargase was unable to be included in the clinical practice guideline classifying chemotherapy emetogenicity in pediatric patients. This single-center, retrospective chart review describes the proportion of pediatric patients who did not vomit during the acute phase (complete response; CR) after receiving IV pegaspargase and provides an emetogenicity classification using a preexisting framework. Of 44 patients who received IV pegaspargase between 2011 and 2020, 13 received a serotonin receptor antagonist plus dexamethasone or palonosetron alone and all experienced a CR. We, therefore, recommend classifying IV pegaspargase as moderately emetogenic.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140060090","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}
Carol Rossetto, Kent Sepkowitz, Jill Ackerman, Rachel Corke, Nina J Pickett, Wini Cudjoe, Janet Eagan, Mini Kamboj, Richard J O'Reilly, Farid Boulad
{"title":"Management of a major varicella zoster exposure in a pediatric oncology population.","authors":"Carol Rossetto, Kent Sepkowitz, Jill Ackerman, Rachel Corke, Nina J Pickett, Wini Cudjoe, Janet Eagan, Mini Kamboj, Richard J O'Reilly, Farid Boulad","doi":"10.1080/08880018.2024.2315456","DOIUrl":"10.1080/08880018.2024.2315456","url":null,"abstract":"<p><p>Management of the exposure of pediatric oncology patients to varicella zoster virus (VZV) is controversial. We report the exposure of 56 patients to a single child with chicken pox at a pediatric cancer housing facility and describe our strategic approach for their management. We reviewed the immune and clinical status of 56 children with cancer receiving ongoing treatment at Memorial Sloan Kettering Cancer Center (MSK) who, while living at a pediatric cancer housing facility, were exposed to the index patient. The management of patients exposed included: (1) determination of immune status, (2) availability of vaccination history or VZV disease prophylaxis, (3) exposure status and subsequent isolation during the period of incubation, and (4) VZV disease prophylaxis. In addition to the 56 patients exposed to the index case, eight children with cancer treated at other facilities and 11 healthy siblings living in the facility were exposed. Of the 56 MSK patients, 21 were classified as immunosuppressed and received varicella zoster immune globulin (human), intravenous standard immune globulin, or acyclovir based on serostatus and immune function. The cohort was followed for 4 weeks after the exposure and no secondary infections were diagnosed. We performed a risk assessment and created a management plan to control and prevent further exposure and development of disease. No secondary cases developed. This strategic approach could serve as a model for the management of VZV exposure for other pediatric oncology centers.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11432746/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139913178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Nirmalya Roy Moulik, Arlene Harriss-Buchan, Giuseppe Saglio, Meinolf Suttorp
{"title":"Challenges in Management of Pediatric Chronic Myeloid Leukemia (pCML) in the Low-Middle Income Countries (LMICs): Insights from an International CML Foundation (iCMLf) Multi-National Survey.","authors":"Nirmalya Roy Moulik, Arlene Harriss-Buchan, Giuseppe Saglio, Meinolf Suttorp","doi":"10.1080/08880018.2023.2301404","DOIUrl":"10.1080/08880018.2023.2301404","url":null,"abstract":"<p><p>Despite being a rare disease, high-income countries (HICs) have formulated guidelines for management of pediatric CML (pCML). We conducted a survey amongst 24 physicians from 22 Low-Middle Income Countries (LMICs) to gather information regarding their practice and experience in treating children with pCML. Rarity of pCML, lack of adequate expertise, and setting-adapted guidelines along with limitations in diagnostic infrastructure as well as challenges in accessing tyrosine kinase inhibitors (TKIs) were identified as major barriers in optimum management of patients. For the first time in literature, our findings provide valuable insights into the multifaceted challenges faced in managing pCML in LMICs.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.7,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139477866","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}