Journal of Pediatric Hematology/Oncology最新文献

{"title":"Effect of Donor/Recipient ABO Mismatch on Outcome of Allogenic Hematopoietic Stem Cell Transplantation in Children: Single-center Study.","authors":"Asmaa Abdel-Raof El-Kaffas, Amany Mahmoud El-Barky, Eslam Elsayed Elhawary","doi":"10.1097/MPH.0000000000002958","DOIUrl":"10.1097/MPH.0000000000002958","url":null,"abstract":"<p><p>ABO blood group mismatch between donor and recipient is thought to be associated with several immunopharmacological complications but is not considered a major contraindication to allogeneic hematopoietic stem cell transplantation (HSCT). However, the impact of such a mismatch on overall survival, transplant-related mortality, graft-versus-host disease, and time to neutrophil and platelet engraftment seems to be conflicting. This retrospective cohort was carried out on children and adolescents who underwent allogenic HSCT between January 2016 and January 2023. ABO compatibility state was assessed and grouped into compatible, minor, major, and bidirectional incompatible groups. The effect of ABO compatibility on various transplantation outcomes was assessed. Forty-three children (25 males and 18 females) with different diagnoses were included. More than half of the patients had no ABO mismatch. Major ABO mismatch was found to be associated with a higher incidence of acute graft-versus-host-disease (aGVHD), whereas near significantly higher mortality was observed in the minor mismatch group. Otherwise, no association was found between ABO mismatch and platelet or neutrophil engraftment. ABO mismatch does not affect the overall survival or the posttransplant engraftment of patients undergoing allogeneic HSCT. aGVHD was observed to be the only factor affected by ABO compatibility.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e1-e9"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142622674","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":"Elevated Interleukin-6 Levels as a Potential Marker of Neonatal Morbidity in Full-term Infants With Polycythemia: A Prospective Study.","authors":"Reyhan Tamer, Şerife Suna Oğuz Ünal, Can Yilmaz Yozgat","doi":"10.1097/MPH.0000000000002968","DOIUrl":"10.1097/MPH.0000000000002968","url":null,"abstract":"<p><strong>Objective: </strong>To research and show that interleukin-6 (IL-6) and c-reactive protein (CRP), which can be used as infection markers, are also higher among newborns with polycythemia. The study took place in the neonatal intensive care unit of Zekai Tahir Burak Maternity Teaching and Research Hospital.</p><p><strong>Patients and methods: </strong>Infants with a gestational age of >37 weeks were included in the study. Infants with chorioamnionitis, perinatal asphyxia, and positive blood culture were excluded from the study. Blood samples were obtained six hours after the delivery from the peripheral vein of the infants for measurements of central hematocrit, blood culture, IL-6, and CRP. Infants with a venous hematocrit value of >65% were grouped as the \"polycythemia group,\" and the ones with a venous hematocrit value of <65% were designated as the \"control group.\" Observation of significantly higher levels of CRP and IL-6 among newborns admitted to the neonatal intensive care unit due to different causes (such as respiratory distress, hypoglycemia, and feeding intolerance), but significantly higher IL-6 levels in newborns with polycythemia.</p><p><strong>Results: </strong>Thirty-five newborns (18 infants in the polycythemia group and 17 infants in the control group) were enrolled in the study. The IL-6 values for the polycythemia group were higher than the upper normal limits (mean ± 2SD, 37.6 ± 55 vs 12 ± 5 pg/dL, respectively; P = 0.00). The IL-6 values of the polycythemia group were found to be higher than the IL-6 values of the control group, with a mean ± 2SD of 37.6 ± 55 vs 6.3 ± 3.4 pg/dL, respectively; this was significant ( P = 0.00). Although the CRP values of the polycythemia group were found to be slightly higher than those of the control group (a mean ± 2SD of 3.06 ± 4.07 vs 1.54 ± 2.21 mg/dL, respectively, P > 0.05), this was not significant.</p><p><strong>Conclusions: </strong>This study found a significant and robust statistical correlation between IL-6 and v. Hct values ( P = 0.01, rs = 0.641). Contrary to IL-6 levels, however, a meaningful relationship was not found between CRP and v.htc values ( P = 0.286; rs = 0.184).</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e15-e18"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142622681","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":"Management of Spontaneous Epidural Hematoma in the Setting of Vaso-occlusive Crisis Among Pediatric Patients With Sickle Cell Disease: A Case Series and Scoping Literature Review.","authors":"Rutvi Patel, Hailey Reisert, Margaret Keymakh, Eleni Drakou, Jessica Briggs, Joshua Cohen, Jimmy S Lee, Daniel Lax, Kaitlin Strumph, Mandana Behbahani, Amanda Baker, Andrew Kobets","doi":"10.1097/MPH.0000000000002975","DOIUrl":"10.1097/MPH.0000000000002975","url":null,"abstract":"<p><p>Spontaneous epidural hematoma (EDH) is a rare sickle cell disease (SCD) complication. We report 3 pediatric cases with SCD and spontaneous EDH and 1 with subgaleal hematomas in the setting of vaso-occlusive crises and elaborate on their presentation and management. Through a scoping review, we identified 71 additional cases reported from 1970 to 2024 and highlighted notable features. We highlight skull infarction as a prognostic factor associated with 88% lower odds of death relative to patients without skull infarction (P<0.01). We clarify optimal management strategies like early and aggressive treatment of disseminated intravascular coagulation (DIC), as 57% of patients with DIC died such that DIC was associated with 7.56 times higher odds of death (P=0.01).</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 1","pages":"38-46"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142907146","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":"Plasma Exchange and N-Acetylcysteine Therapy in a Case of Congenital Thrombotic Thrombocytopenic Purpura Presenting With Acute Renal Failure.","authors":"Çağri Coşkun, Tekin Aksu, Bora Gülhan, Ali Düzova, Şule Ünal","doi":"10.1097/MPH.0000000000002963","DOIUrl":"10.1097/MPH.0000000000002963","url":null,"abstract":"<p><p>Congenital thrombotic thrombocytopenic purpura (cTTP), which is associated with mutations in the gene for a disintegrin and metalloproteinase with a thrombospondin type 1 motif member 13 (ADAMTS13), is a chronic and lifelong disease. The clinical course is variable. Regularly using ADAMTS13-containing products such as fresh frozen plasma (FFP) for long-term prophylaxis is the most important treatment to prevent thrombotic microangiopathy (TMA) episodes. Here, we identified novel pathogenic mutations of ADAMTS13 in our patients who experienced severe acute renal failure. Infections can trigger acute hemolytic episodes, and if the initiation of FFP therapy is delayed, this leads to severe organ dysfunction, as in our case. We have shown that regular use of products containing ADAMTS13 can reverse TMA episodes and long-term morbidity and mortality. When severe acute renal failure occurs, daily plasma exchange and N-acetylcysteine (NAC) are useful.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 1","pages":"e65-e67"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142907206","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":"Like Father, Like Daughter: A Family With a Constitutional Thrombocytopenia Variant Due to a Novel Heterozygous Missense Mutation in GP1BA.","authors":"Jennifer Light, Gerardo Quezada","doi":"10.1097/MPH.0000000000002980","DOIUrl":"10.1097/MPH.0000000000002980","url":null,"abstract":"<p><p>Constitutional platelet disorders have become better understood since Bernard and Soulier first described a case in 1948. Their diagnosis can also be challenging due to overlap in clinical presentation and lab findings with platelet type von Willebrand. Bernard-Soulier syndrome is a disorder caused by GPIb receptor mutations that decrease its affinity for von Willebrand factor resulting in reduced platelet function and macrothrombocytopenia. It is associated with multiple genetic mutations in either GPIBA, GPIBB, GP9, or GP5. We present a case report of a family with a Bernard Soullier-like thrombocytopenia variant due to a heterozygous missense mutation in GPIBA.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 1","pages":"e62-e64"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142907143","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":"Contributions of Pediatric Hematology/Oncology to the Diagnosis, Treatment, and Cure of Acute Lymphoblastic Leukemia: Part 2a (Numbers 11 to 15).","authors":"Denis R Miller","doi":"10.1097/MPH.0000000000002960","DOIUrl":"10.1097/MPH.0000000000002960","url":null,"abstract":"<p><p>Pediatric hematology/oncology as a subspecialty has made major contributions to the diagnosis and treatment of acute lymphoblastic leukemia, the most common malignancy in the pediatric population. This impressive progress has yielded complete response rates of 98%, median durations of complete continuous remissions of over 5 years, and long-term leukemia-free survival and probable cure in 80% to 85% of patients. Sixty-five years ago, such data could only be imagined as future goals. This offering, part 2 of a planned trilogy, represents the second historical review from an admittedly elderly investigator, proud to have witnessed firsthand many of these advances. Part 2a contains numbers 11 to 15 and Part 2b consists of numbers 16 to 20. In consecutive order from the first 10: (11) more clues to the causation of ALL relating to in utero events and after birth, very early responses to infection; (12) the superiority of pediatric-inspired ALL trials for adolescents and young adults; (13) L-asparaginase; (14) chimeric antigen receptor T-cell therapy (CART); and (15) aggressive multiagent therapy for high-risk ALL introduced by BFM and CCG. My involvement with colleagues in some of these advances has provided a 65-year odyssey and its accompanying gratification and sense of accomplishment. Mostly, our goals have been achieved and have benefitted substantially our patients.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"31-37"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882404","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":"Successful Treatment Outcome of a Pediatric Malignant Germ Cell Tumor With an Extensive Cavo-atrial Tumor Thrombus Presenting as an Oncological Emergency.","authors":"Sai Shiva G, Prasanth Vr, Rajani Lilly Cheriyan, Ajay Sankar, Manjusha Nair, Binitha Rajeswari, Guruprasad Cs, Kalasekhar Vs, Baiju S Dharan, Priyakumari Thankamony","doi":"10.1097/MPH.0000000000002978","DOIUrl":"10.1097/MPH.0000000000002978","url":null,"abstract":"<p><strong>Background: </strong>Pediatric germ cell tumor (GCT) with extensive cavo-atrial tumor thrombus presenting as an oncologic emergency is extremely rare.</p><p><strong>Observation: </strong>A 13-month-old female child with sacrococcygeal GCT having inferior vena cava (IVC) and right atrial thrombus presenting with anasarca and respiratory distress is reported. Because of worsening symptoms post initiation of chemotherapy, tumor thrombus debulking from the right atrium and distal end of IVC was performed on day 9 on an emergency basis. Her symptoms improved, and after planned chemotherapy, tumor excision with coccygectomy could be done. The IVC thrombus remained inoperable due to sclerosis. She remains recurrent-free now 12 months post therapy.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e34-e37"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882421","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":"Eltrombopag for Treatment of Thrombocytopenia After Autologous Stem Cell Transplantation in Children: Single Center Experience.","authors":"Nihan Bayram, Yontem Yaman, Kursat Ozdilli, Serdar Nepesov, Isik Odaman Al, Murat Elli, Sema Anak","doi":"10.1097/MPH.0000000000002971","DOIUrl":"https://doi.org/10.1097/MPH.0000000000002971","url":null,"abstract":"<p><strong>Introduction: </strong>Thrombocytopenia is a common clinical problem in cancer patients undergoing high-dose chemotherapy and autologous hematopoietic stem cell transplantation (HSCT). It can occur as prolonged isolated thrombocytopenia (PIT) or secondary failure of platelet recovery (SFPR) and may cause potentially fatal bleeding. However, data on the treatment of post-transplant thrombocytopenia is still lacking.</p><p><strong>Methods: </strong>We reported our practices involving 15 pediatric patients who received eltrombopag (ELT) treatment for PIT and SFPR after autologous HSCT.</p><p><strong>Results: </strong>The overall response was 78.5% (11/14), with 1 patient excluded due to noncompliance. The 12 surviving patients' median follow up was 699 days (range: 167 to 2250 d).</p><p><strong>Conclusions: </strong>Our study indicates the efficacy and safety of ELT for treating PIT and SFPR after autologous HSCT in pediatric patients. However, more studies are needed to confirm these findings in children.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 1","pages":"e10-e14"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142907027","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":"Retrospective Comparison of Targeted Anticancer Drugs Predicted by the CNS-TAP Tool Versus Those Selected by a Molecularly Driven Tumor Board in Children With DIPG.","authors":"Holly J Roberts, Karthik Ravi, Bernard L Marini, Allison Schepers, Cassie Kline, Lindsay Kilburn, Michael Prados, Sara A Byron, Julie Sturza, Sabine Mueller, Carl Koschmann, Andrea T Franson","doi":"10.1097/MPH.0000000000002964","DOIUrl":"10.1097/MPH.0000000000002964","url":null,"abstract":"<p><p>The recent trial Pediatric Neuro-Oncology Consortium 003 (PNOC003) utilized a molecular tumor board to recommend personalized treatment regimens based on tumor sequencing results in children with DIPG. We separately developed the Central Nervous System Targeted Agent Prediction (CNS-TAP) tool, which numerically scores targeted anticancer agents using preclinical, clinical, and patient-specific data. We hypothesized that highly scored agents from CNS-TAP would overlap with the PNOC003 tumor board's recommendations. For each of the 28 participants, actionable genetic alterations were derived from PNOC003 genomic reports and input to CNS-TAP to identify the highest scoring agents. These agents were then compared with PNOC003 recommendations, with a resultant concordance percentage calculated. Overall, 38% of the total agents recommended by the tumor board were also selected by CNS-TAP, with higher concordance (63%) in a subanalysis including only targeted anticancer agents. Furthermore, nearly all patients (93%) had at least 1 drug chosen by both methods. We demonstrate overlap between agents recommended by CNS-TAP and PNOC003 tumor board, though this does not appear to improve survival. We do observe some discordance, highlighting strengths and limitations of each method. We propose that a combination of expert opinion and data-driven tools may improve targeted treatment recommendations for children with DIPG.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"19-30"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11676589/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142622702","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}

{"title":"Successful Development and Implementation of Communication Skills Simulation Training Within Pediatric Hematology-oncology Fellowship Education.","authors":"Kerri Becktell, Sarah Rumler, Richard L Tower","doi":"10.1097/MPH.0000000000002959","DOIUrl":"10.1097/MPH.0000000000002959","url":null,"abstract":"<p><p>Difficult discussions with patients and families are a primary component of pediatric oncology care. We report our experience in the development and implementation of a longitudinal simulation-based Communication Skills Curriculum within a pediatric hematology-oncology fellowship training program. A 6-session simulation curriculum was created for fellows to practice difficult oncology related discussions with a standardized patient (SP). This program was implemented in 2017 and continues presently. Throughout fellowship training the scenarios progress in content and scope. Each simulation was observed and recorded, and written and verbal feedback was provided. The Gap-Kalamazoo Communication Skills Assessment Form was used to assess communication skills in each session. A total of 15 fellows (100% of possible participants) have participated in this curriculum to date. Across all domains of communication skills assessed, the ratings given by the physician evaluators and self-evaluations improved over the course of the scenarios completed throughout fellowship training. This type of simulation-based communication skills curriculum within a pediatric hematology-oncology fellowship training program is feasible to implement. This training is useful to fellows beyond training in improving communication skills, which has the potential to directly benefit the future patients they care for.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"7-11"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142622713","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}