{"title":"Autologous Cord Blood Transplantation for Idiopathic Severe Aplastic Anemia.","authors":"Anselm Chi-Wai Lee","doi":"10.1097/MPH.0000000000003106","DOIUrl":"10.1097/MPH.0000000000003106","url":null,"abstract":"<p><p>Cord blood units are seldom used for autologous transplantation and selection criteria have not been defined. A 6-year-old male with severe aplastic anemia received an autologous cord blood transplantation containing total nucleated cells (TNC) 2.0×107/kg and CD34+0.32×105/kg. However, he lost the graft and was rescued with a second, haploidentical donor graft. Together with those reported in the literature, 15 children with aplastic anemia received autologous cord blood transplantation. Thirteen children succeeded but 2 failed. Successful transplantation can be predicted by TNC doses of >2.5×107/kg (12/12 vs. 1/3; P=0.029), but not by CD34+ dose or patient's age. Cord blood units with adequate TNC are valid products for autologous transplantation.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144883065","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}
Diana Wu, Megan R Williams, Akshay A Patwardhan, Yu Bi, Subodh Selukar, Jennifer L Pauley, John McCormick, Akshay Sharma
{"title":"Concomitant Azole Initiation Decreases Time to Stable Goal Sirolimus Concentrations in Pediatric Allogeneic Transplant Recipients.","authors":"Diana Wu, Megan R Williams, Akshay A Patwardhan, Yu Bi, Subodh Selukar, Jennifer L Pauley, John McCormick, Akshay Sharma","doi":"10.1097/MPH.0000000000003093","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003093","url":null,"abstract":"<p><p>Variability of sirolimus pharmacokinetics, often compounded by interactions with azole antifungal therapy, leads to concentrations outside the goal range and increased risk of related complications. We assessed the time to initial attainment of stable goal sirolimus concentrations in patients who either concomitantly initiated azole therapy and sirolimus (concomitant group) or initiated an azole after sirolimus initiation (delayed group). The median time to attainment of stable goal sirolimus concentrations was 8.0 days in the delayed group versus 6.0 days in the concomitant group. Patients in the concomitant group reached stable goal concentrations earlier than those in the delayed group (hazard ratio: 0.27, 95% CI: 0.14-0.5, P<0.001), after adjusting for age, sex, and race. The delayed group was estimated to have concentrations outside of goal range at almost 2 times the rate in the concomitant group (P<0.001), after adjusting for age, sex, and race. Thus, we conclude that concomitant initiation of sirolimus and azoles may facilitate the achievement of stable goal sirolimus concentrations sooner after allogeneic cell transplant.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821670","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}
Braghmandita W Indraswari, Eddy Supriyadi, Gertjan J L Kaspers, Mei N Sitaresmi
{"title":"Longitudinal Assessment of Health-Related Quality of Life in Childhood Acute Lymphoblastic Leukemia During Active Treatment in Indonesia.","authors":"Braghmandita W Indraswari, Eddy Supriyadi, Gertjan J L Kaspers, Mei N Sitaresmi","doi":"10.1097/MPH.0000000000003097","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003097","url":null,"abstract":"<p><p>Children with acute lymphoblastic leukemia (ALL) are at risk for poor health-related quality of life (HRQOL) due to the treatment and disease itself. This study presents a serial measurement of the HRQOL of children with ALL during cancer treatment and investigates the impact of demographic, socioeconomic, and medical patient characteristics on their HRQOL. A prospective HRQOL longitudinal study of children with ALL was conducted at an academic hospital between 2016 and 2020. HRQOL was measured using PedsQL 4.0 and PedsQL 3.0 at 3 treatment phases: induction, consolidation, and maintenance. The HRQOL of children 2 to 18 years of age was assessed using a proxy report, and children 5 to 18 years also reported themselves using patient self-report. The comparison scores between subsequent treatment phases of those interviewed in each time measurement were measured using a repeated measures ANOVA test and a post hoc analysis with the Bonferroni test. One hundred thirteen children 5 to 18 years of age and 221 parents participated. The mean age at diagnosis was 6.6±4.0 years. Children had standard-risk ALL (51%) and were boys (56%). The total score of HRQOL and most subscales significantly improved during treatment. Physical health, school functioning, procedural anxiety, and communication were most affected in early treatment. Although all scores improved over time, school functioning and communication remained lower than other subscales. The age classification impacted the improvement of most HRQOL subscales. In conclusion, the HRQOL improved during treatment. Interventions to maintain physical health and reduce procedural anxiety in early treatment are required. Improving health care providers' communication skills and facilitating hospital schooling will ameliorate HRQOL.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821672","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}
Clara Guilbault, Arnaud Bonnefoy, Georges-Étienne Rivard, Marie-Claude Pelland-Marcotte
{"title":"Inhibitor Development in Severe Congenital Factor V Deficiency.","authors":"Clara Guilbault, Arnaud Bonnefoy, Georges-Étienne Rivard, Marie-Claude Pelland-Marcotte","doi":"10.1097/MPH.0000000000003099","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003099","url":null,"abstract":"<p><p>We report on a 10-year-old female diagnosed with severe congenital FV deficiency who developed anti-FV inhibitory antibody 3 weeks following dental procedure hemorrhage treated with FFP. The patient presented a large spontaneous gluteal hematoma. Despite multiple FFP transfusions, FV levels remained critically low, prompting suspicion of FV inhibitor development, confirmed by ELISA. Bleeding control was achieved with recombinant factor VIIa. An immune tolerance induction protocol was initiated, and administration of FFP was continued. Factor V inhibitors became undetectable after 1 year and the patient had a favorable clinical evolution without further bleeding episodes. This case reports the successful eradication of FV inhibitor using FFP exposure and immunosuppressive therapy.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821671","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 ETV6::NCOA2 Fusion, a Recurrent Cytogenetic Abnormality in Childhood Leukemia With Defining Properties.","authors":"Zayan Safi, Mahdi Fakhouri, Carla Monsef, Nada Assaf","doi":"10.1097/MPH.0000000000003095","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003095","url":null,"abstract":"<p><p>t(8;12)(q13;p13)(ETV6::NCOA2) is a rare but recurrent cytogenetic abnormality in childhood leukemia with mixed myeloid/T-cell lineage. We hereby present the first pediatric B-ALL with ETV6::NCOA2. A 5-year-old boy presented with B-ALL residual disease at end-of-induction. He achieved complete remission after therapy intensification, but relapsed 2 months later. Karyotype at relapse showed 46,XY,del(6)(q21q23),t(8;12)(q13;p13),-9,+mar[17]. ETV6::NCOA2 was confirmed by FISH and RT-PCR. Next-generation sequencing revealed a pathogenic NRAS variant. The patient developed severe neutropenia, complicated by bacterial sepsis and death 10 months later. Unlike cases with mixed myeloid/T-cell phenotype, no NOTCH1 mutations were detected. Review of published cases suggests that the presence of additional cytogenetic abnormalities dictates adverse prognosis.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144789345","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}
Bahri Can Duran, Yilmaz Ay, Hidayet Imancli, Gurbuz Akcay
{"title":"The Effect of Iron Deficiency Anemia and Serum Ferritin Deficiency on Cognitive Functions in Children 1 to 5 Years Old.","authors":"Bahri Can Duran, Yilmaz Ay, Hidayet Imancli, Gurbuz Akcay","doi":"10.1097/MPH.0000000000003090","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003090","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to investigate the effects of iron deficiency anemia and serum ferritin deficiency on mental and motor functions in children 1 to 5 years of age and whether these effects could be improved by treatment.</p><p><strong>Materials and methods: </strong>The study was conducted between February 2021 and July 2021 and included a total of 79 children between 1 and 5 years old. Participants were divided into 3 groups according to their blood results: iron deficiency anemia (n = 19), nonanemic iron deficiency (n = 21), and control group (n = 39). Ankara Developmental Screening Inventory (ADSI) was administered to all participants before treatment. The case groups were subjected to 3-month iron treatment, and participants were reevaluated with ADSI.</p><p><strong>Results: </strong>The ADSI T-scores of the case groups were significantly lower than the control group. A significant increase in T-scores was observed in both case groups after treatment. In addition, positive and moderate correlation was observed between serum ferritin level and language-cognitive activity, fine-motor development, gross-motor development, and social skills-self-care, and, positive and strong correlation was observed between serum ferritin level and general development and T-scores.</p><p><strong>Conclusion: </strong>We observed that the decrease in serum ferritin levels, an early indicator of iron deficiency, negatively affected cognitive functions and that this effect could be partially corrected by treatment.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.8,"publicationDate":"2025-08-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144789344","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}
Pankaj Prasun, Jennifer R Suarez, Katelyn J Watkin
{"title":"Cobalamin E Disease: An Ultrarare Treatable Cause of Hemolytic Anemia in Infancy.","authors":"Pankaj Prasun, Jennifer R Suarez, Katelyn J Watkin","doi":"10.1097/MPH.0000000000003072","DOIUrl":"10.1097/MPH.0000000000003072","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"321-323"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475742","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}
Rachel Hill, Lindsey Thomas, David Farbo, Kenneth Heym
{"title":"Exocrine Pancreatic Insufficiency Following Asparaginase-induced Pancreatitis in Pediatric Acute Lymphoblastic Leukemia Patients: A Case Series.","authors":"Rachel Hill, Lindsey Thomas, David Farbo, Kenneth Heym","doi":"10.1097/MPH.0000000000003058","DOIUrl":"10.1097/MPH.0000000000003058","url":null,"abstract":"<p><p>Asparaginase is vital for the treatment of pediatric acute lymphoblastic leukemia (ALL), but it can cause numerous adverse effects, including asparaginase-associated pancreatitis (AAP). Exocrine pancreatic insufficiency (EPI) has been reported following pancreatitis but has not yet been reported in the setting of pediatric leukemia. If undiagnosed, EPI can lead to nutritional deterioration. This series describes 3 cases of severe, necrotizing AAP followed by EPI of variable duration. The purpose of this case series is to document these findings and increase awareness about the association between these conditions to facilitate timely identification and intervention, when warranted.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e265-e268"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248391","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":"Methemoglobinemia Induced by Food During Chemotherapy in a Boy With Acute Lymphoblastic Leukemia and Literature Review.","authors":"Yanqing Wang, Xiuping Chen, Chanjuan Tong, Bin Lin, Yongmin Tang, Meixin Fang","doi":"10.1097/MPH.0000000000003061","DOIUrl":"10.1097/MPH.0000000000003061","url":null,"abstract":"<p><p>Methemoglobinemia, a rare and potentially life-threatening condition in pediatric hematology-oncology patients, requires accurate cause identification for effective treatment. We report a case of a 7-year-old boy with acute lymphoblastic leukemia who developed methemoglobinemia during chemotherapy. Initial vitamin C treatment was ineffective, requiring methylene blue. A detailed dietary history revealed the consumption of nitrate-rich vegetables and rapeseed oil, suggesting these as potential triggers. Our literature review highlights the diverse etiologies of methemoglobinemia in this patient population, emphasizing the need for heightened clinical vigilance, and careful differentiation of possible causes to guide appropriate management.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 6","pages":"e273-e277"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144698891","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}
Amr Elgehiny, Alexander Hsu, David McCall, Amber Gibson, Branko Cuglievan, Cesar Nunez, Miriam B Garcia, Luz Castellanos, Alexander Funck, Shehla Razvi
{"title":"Severe Hypertriglyceridemia Secondary to Long-acting Asparaginase in Pediatric Patients With Acute Lymphoblastic Leukemia.","authors":"Amr Elgehiny, Alexander Hsu, David McCall, Amber Gibson, Branko Cuglievan, Cesar Nunez, Miriam B Garcia, Luz Castellanos, Alexander Funck, Shehla Razvi","doi":"10.1097/MPH.0000000000003077","DOIUrl":"10.1097/MPH.0000000000003077","url":null,"abstract":"<p><p>Pegylated asparaginase is now standard in US treatment protocols for acute lymphoblastic leukemia (ALL). However, they are associated with significant side effects, including severe hypertriglyceridemia. In this case series, we report 8 patients with severe (triglyceride >1000 mg/dL) hypertriglyceridemia after receiving long-acting asparaginase for ALL and describe their clinical course. The 8 patients included 3 females and 5 males (aged 2 to 14 y; median=12 y); 7 were Hispanic and 1 was Middle Eastern. The median time from dose to peak hypertriglyceridemia was 17 days and to resolution was 25 days. Presentations included isolated hypertriglyceridemia, pseudohyponatremia, hypoglycemia, and lipemia interfering with complete blood count results. Median length of hospitalization was 3.5 days. Management included hydration, a low-fat diet, omega-3 supplements, fenofibrates, statins, and levocarnitine. An insulin drip was used in 2 patients in the intensive care unit. Asparaginase treatment continued per protocol after triglyceride levels were <1000 mg/dL. In conclusion, severe hypertriglyceridemia can occur after long-acting asparaginase and is typically asymptomatic and transient, not requiring a pause or modification in treatment. We recommend monitoring for hypertriglyceridemia closely in patients with risk factors who are resuming long-acting asparaginase therapy after triglyceride levels fall <1000 mg/dL.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e215-e221"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144497382","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}