Journal of Pediatric Hematology/Oncology最新文献_第5页

A Neonate Presenting With Large Abdominal Mass: A Rare Case of Congenital Leukemia. 新生儿腹部大肿块：罕见的先天性白血病病例。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2025-01-08 DOI: 10.1097/MPH.0000000000002991

Bablu K Gaur, Chirag Varshney, Rupa R Singh, Prashansa Soneja

引用次数: 0

Second Primary Neoplasms in Pediatric Cancer Survivors With Single Institution Experience From Turkey.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2025-01-20 DOI: 10.1097/MPH.0000000000003001

Hamiyet Hekimci Özdemir, Sena TürkYilmaz, Eda Ataseven, Gülcihan Özek, Serap Aksoylar, Serra Arun Kamer, Mehmet Kantar

{"title":"Second Primary Neoplasms in Pediatric Cancer Survivors With Single Institution Experience From Turkey.","authors":"Hamiyet Hekimci Özdemir, Sena TürkYilmaz, Eda Ataseven, Gülcihan Özek, Serap Aksoylar, Serra Arun Kamer, Mehmet Kantar","doi":"10.1097/MPH.0000000000003001","DOIUrl":"10.1097/MPH.0000000000003001","url":null,"abstract":"Background: This study aims to establish the characteristics of second primary neoplasms (SPNs) and the long-term follow-up status of a tertiary pediatric oncology center.Methods: Records of 1799 patients followed up in the pediatric oncology division between January 1981 and December 2022 were evaluated retrospectively.Results: Thirty-four (1.9%) cases of secondary neoplasms were identified throughout 42 years. The 5-year and 10-year cumulative incidence was 1% and 4%, respectively. The 3 most common SPNs were thyroid carcinomas (TC), central nervous system (CNS) tumors, and leukemias. The shortest median latent period of SPN detection was 15.5 (2 to 35) months in secondary leukemias, whereas 8 (0 to 17) years in all SPNs. Secondary solid tumors that occurred within the radiation field were TC and meningiomas with a 5.5 (3 to 12) and 16 (6 to 22) years latency period, respectively. Ten patients died; the median death time from the diagnosis of SPN was 10 months in all secondary leukemias and 3.5 months in CNS tumors. The 5-year overall survival was 91%, with a median follow-up time of 13.1 years in all patients with SPN.Conclusions: Considering the SPN-inducing effects of radiotherapy and chemotherapy, patient-protective improvements in treatment protocols are required. Multidisciplinary and long-term follow-up is essential even in adulthood because of the long latency period of some SPN occurring in pediatric cancer survivors.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"99-107"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033351","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}

引用次数: 0

Life-threatening Lymphatic Malformation With Somatic Activating NRAS Mutation Successfully Treated With Trametinib: A Case Study. 曲美替尼成功治疗具有体细胞活化NRAS突变的危及生命的淋巴畸形：一个案例研究。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2025-01-03 DOI: 10.1097/MPH.0000000000002990

Lindsay Zumwalt, Haley Schluterman, Anish Ray, Kenneth Heym

{"title":"Life-threatening Lymphatic Malformation With Somatic Activating NRAS Mutation Successfully Treated With Trametinib: A Case Study.","authors":"Lindsay Zumwalt, Haley Schluterman, Anish Ray, Kenneth Heym","doi":"10.1097/MPH.0000000000002990","DOIUrl":"10.1097/MPH.0000000000002990","url":null,"abstract":"Kaposiform lymphangiomatosis (KLA) is a rare and aggressive subtype of complex lymphatic anomalies (CLA), characterized by abnormal lymphatic proliferation leading to distinct clinical manifestations. Despite the complexity of this condition, there is no established standard therapy, and treatment options such as sclerotherapy, laser therapy, and surgery remain variably effective and are limited to symptom management rather than curative. Sirolimus, an mTOR pathway inhibitor, has shown promise as a primary therapy, particularly in patients without a driver mutation. However, in some instances, the genetic landscape of KLA has revealed somatic mutations in the RAS-MAPK pathway, most notably the NRAS variant (c.182A>G, p.Q61R), representing a potential therapeutic target. We present a case of a 4-year-old male who presented with pericardial and pleural effusion s without notable coagulopathy found to harbor an NRAS p.Gln61Arg gene mutation, diagnosed through next-generation sequencing (NGS) analysis. Initial therapy with sirolimus failed to provide optimal benefit with persistent pleural effusion. Subsequent treatment with the MEK inhibitor trametinib led to significant clinical improvement, evidenced by the resolution of effusions and removal of the chest tube. In the short term, no significant adverse effect was reported. Our findings underscore the value of genomic profiling in guiding personalized treatment strategies for rare and complex diseases presenting like KLA. This case highlights the potential of targeted therapies, such as trametinib, in improving clinical outcomes for patients with disease with activating NRAS variants, emphasizing the importance of ongoing research to validate and expand these therapeutic approaches in the management of vascular anomalies.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e104-e106"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142962299","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}

引用次数: 0

Pediatric Borderline Ovarian Tumors: A Retrospective Study of 15 Cases at a Single Institution. 儿童交界性卵巢肿瘤：对同一机构15例病例的回顾性研究。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2025-01-13 DOI: 10.1097/MPH.0000000000002998

Bretton Laboret, Marinda G Scrushy, Samir Pandya, Joseph T Murphy

{"title":"Pediatric Borderline Ovarian Tumors: A Retrospective Study of 15 Cases at a Single Institution.","authors":"Bretton Laboret, Marinda G Scrushy, Samir Pandya, Joseph T Murphy","doi":"10.1097/MPH.0000000000002998","DOIUrl":"10.1097/MPH.0000000000002998","url":null,"abstract":"Borderline ovarian tumors (BOTs) are rare in pediatric populations and typically follow an indolent clinical course with few reported recurrences. Consequently, guidelines for pediatric BOT management are minimal. We retrospectively examined the management of 15 adolescent patients who underwent BOT resection at our institution over 14 years, with a specific focus on recurrence. Data collected include age, symptoms, tumor characteristics, laboratory markers, surgical management, staging, and follow-up. Fifteen patients with BOTs (median age: 16 y) presented with abdominal pain (67%), or distention (33%). Cancer antigen-125 marker was elevated in 10/13 patients. There were 11 (73%) tumors with serous and 4 (23%) with mucinous histology. Most received fertility-preserving surgery (93%) and disease stage was 1A in 7 (47%), 1B/1C in 5 (33%), and stage 2B or higher in 3 (20%) patients. Additional staging procedures, including peritoneal washings (73%), omentectomy (53%), and peritoneal biopsy (47%), varied in use. Four (27%) patients recurred, with 1 case of benign tumor, 1 BOT, and 2 serous carcinomas. Median patient follow-up was 45 months. BOTs can be successfully treated with fertility-preserving surgery but demonstrate a non-negligible rate of recurrence. We recommend surgical staging and posttreatment surveillance for all patients with BOT.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e77-e82"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142971386","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}

引用次数: 0

Unusual Presentation of a Pediatric Burkitt's Lymphoma With Infiltration of the Interatrial Septum. 小儿伯基特淋巴瘤伴房间隔浸润的不寻常表现。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2024-12-24 DOI: 10.1097/MPH.0000000000002984

Gaser Abdelmohsen, Aliaa Alamri, Ali Algiraigri, Mohamed Elnakeeb, Khadijah Maghrabi

{"title":"Unusual Presentation of a Pediatric Burkitt's Lymphoma With Infiltration of the Interatrial Septum.","authors":"Gaser Abdelmohsen, Aliaa Alamri, Ali Algiraigri, Mohamed Elnakeeb, Khadijah Maghrabi","doi":"10.1097/MPH.0000000000002984","DOIUrl":"10.1097/MPH.0000000000002984","url":null,"abstract":"Background: Burkitt's lymphoma (BL) is the most common subtype of non-Hodgkin lymphoma in children, typically presenting with extranodal masses in areas such as the abdomen, face, and neck. Cardiac involvement in BL is extremely rare, especially in pediatric patients, and has significant implications for diagnosis, management, and prognosis.Case presentation: A 6-year-old male patient with Burkitt's lymphoma, presenting with an uncommon infiltration of the interatrial septum, is described. The patient initially presented with a painful right facial mass, bilateral periorbital edema, and a history of fever, weight loss, and lower limb pain. Imaging revealed extensive disease, including a right masticator muscle mass, mandibular infiltration, and multiple soft tissue and skeletal lesions, along with significant thickening of the interatrial septum. A diagnosis of Stage III Burkitt's lymphoma was confirmed through biopsy and immunohistochemical analysis. The patient was treated according to the ANHL1131 protocol, including Rituximab and multi-agent chemotherapy. Remarkably, the cardiac infiltration showed substantial regression on follow-up echocardiography after chemotherapy.Conclusions: This case underscores the importance of a comprehensive cardiac evaluation in pediatric patients with Burkitt's lymphoma, particularly when presenting with atypical manifestations such as interatrial septum involvement. The findings demonstrate that systemic chemotherapy can effectively treat cardiac infiltration, even in complex cases with extensive disease. This report highlights the need for awareness of rare cardiac presentations in pediatric lymphoma and the critical role of advanced imaging modalities in diagnosis and management. Continuous monitoring and early implementation of cardioprotective strategies are essential to optimize outcomes in such cases.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e121-e124"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882425","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}

引用次数: 0

A 5-Year Single-center Experience on the Use of Emicizumab Prophylaxis in Children With Severe Hemophilia A With and Without Factor VIII Inhibitors.

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-03-01 Epub Date: 2025-01-14 DOI: 10.1097/MPH.0000000000002997

Esraa Mohamed Ahmed Hasan, Jayashree Motwani

{"title":"A 5-Year Single-center Experience on the Use of Emicizumab Prophylaxis in Children With Severe Hemophilia A With and Without Factor VIII Inhibitors.","authors":"Esraa Mohamed Ahmed Hasan, Jayashree Motwani","doi":"10.1097/MPH.0000000000002997","DOIUrl":"10.1097/MPH.0000000000002997","url":null,"abstract":"Objective: Emicizumab promotes efficacious hemostasis in persons with hemophilia A persons with hemophilia A with and without inhibitors. Primary analyses of real-world data and clinical trials have shown emicizumab efficacy and safety; however, long-term data are limited.Methods: This retrospective study was conducted to assess real-world long-term outcomes of pediatric patients on emicizumab in our hemophilia center between the period of February 2018 and September 2023. Relevant demographic and clinical data were gathered.Results: Seventy-eight patients were enrolled. Previously untreated patients and minimally treated patients accounted for 14.1% and 10.3% of our recruited patients, respectively. One of 5 patients with active inhibitors experienced a single recombinant activated factor VII-treated bleeding episode while on emicizumab prophylaxis. Twenty-eight (28/78) patients underwent surgical/dental procedures without bleeding complications except for 2 patients (7.1%). Thirty-three patients experienced 62 factor-treated bleeding episodes with only 11 imaging-confirmed joint/muscle bleeding episodes among 8 patients. No major safety concerns were reported in the study and emicizumab was discontinued in 5 patients (2 for antidrug antibody development, 2 for joint bleeding, and 1 for non-compliance).Conclusion: Emicizumab prophylaxis was well tolerated with no new safety concerns, and 45 patients (57.7%) of our cohort exhibited zero-treated bleeds, which was comparable to other published experiences.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e83-e89"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033326","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}

引用次数: 0

Characterization of Factor 8 Gene Variants in Patients With Hemophilia A. A 型血友病患者中因子 8 基因变异的特征。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-01-01 Epub Date: 2024-11-18 DOI: 10.1097/MPH.0000000000002962

Asdaf Alamri, Omaima Alharrasi, Aisha Alkhayate, Murtadha Al-Khabori, Abdulhakim AlRawas, Yasser Wali

引用次数: 0

Time to Resolution of Severe Anemia in Young Children With Iron Deficiency. 缺铁儿童严重贫血的解决时间。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-01-01 Epub Date: 2024-11-29 DOI: 10.1097/MPH.0000000000002979

John Leister, Faye L Norby, Alexander A Boucher

{"title":"Time to Resolution of Severe Anemia in Young Children With Iron Deficiency.","authors":"John Leister, Faye L Norby, Alexander A Boucher","doi":"10.1097/MPH.0000000000002979","DOIUrl":"10.1097/MPH.0000000000002979","url":null,"abstract":"Iron deficiency anemia in children remains a pervasive problem. Prolonged iron deficiency anemia (IDA) is linked to adverse neurodevelopmental outcomes, but the duration of severe IDA (hemoglobin <7 g/dL) in children is poorly studied, particularly for racial/ethnic minority groups. We reviewed the electronic medical records of 92 children (1 to 5 y old) with IDA within a major metropolitan health care system. Duration of anemia, hemoglobin at diagnosis and nadir, age, sex, race/ethnicity, and the use of intravenous interventions were analyzed by Kaplan-Meier curves, Cox regression, and logistic regression. The majority of the included children were Asian (72.8%). Anemia resolution was documented in 68% of cases, and 47% of cases received intravenous intervention. Iron repletion was only confirmed in 37% of cases (n=34), leaving many children with unclear resolution at risk for recurrence. Caucasian children had anemia resolution faster than Black or Asian children, and the latter groups were also less likely to reach hemoglobin normalization. Children with intravenous interventions were more likely to have documented resolution than those with only oral treatment. Those receiving intravenous interventions were more likely to be followed through anemia resolution, although treatment standardization was lacking, and confirmation of iron storage repletion was rarely checked. Future studies should emphasize the importance of ensuring iron storage replacement and potentially utilize time-to-anemia resolution data to determine optimal hemoglobin values for intravenous iron as a first-line intervention.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e38-e43"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142786171","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}

引用次数: 0

Improving Sickle Cell Patient Appointment Compliance in a Hospital-based Pediatric Ophthalmology Clinic. 改善镰状细胞患者的预约依从性在医院为基础的儿童眼科诊所。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-01-01 Epub Date: 2024-11-08 DOI: 10.1097/MPH.0000000000002970

Emily Louie, Anthony Tang, Amie Patel, Ragha Srinivasan, Shiva Bohn, Matthew Wilson

{"title":"Improving Sickle Cell Patient Appointment Compliance in a Hospital-based Pediatric Ophthalmology Clinic.","authors":"Emily Louie, Anthony Tang, Amie Patel, Ragha Srinivasan, Shiva Bohn, Matthew Wilson","doi":"10.1097/MPH.0000000000002970","DOIUrl":"https://doi.org/10.1097/MPH.0000000000002970","url":null,"abstract":"This quality improvement initiative aimed to reduce the no-show rate at a hospital-based tertiary sickle cell ophthalmology clinic. Missed appointments place a significant burden on the healthcare system, resulting in prolonged waiting times and underutilized clinical resources that impact the quality of care provided. Individuals with sickle cell disease commonly require multiple appointments to address the myriads of comorbidities associated with their disease. Nevertheless, the sickle cell ophthalmology clinic experienced an alarmingly high no-show rate of 49% from June to September of 2021 despite offering same-day appointments with the hematology clinic. To address this issue, we conducted a study in which we compared the baseline no-show rate with the rate after relocating both clinics to the same floor, aiming to overcome patient-related barriers. Following 3.5 months of the clinics sharing the same floor, the no-show rate decreased to 39%, reflecting a 10% improvement from the baseline rate. In summary, co-locating two related clinics can alleviate patient burdens, foster communication between multidisciplinary specialties, and contribute to an overall improvement in the quality of care and treatment provided.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 1","pages":"e44-e47"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142907043","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}

引用次数: 0

Pediatric T-lymphoblastic Leukemia With an Entirely Mature Immunophenotype: A Prompt and Challenging Diagnosis. 具有完全成熟免疫表型的儿童t淋巴细胞白血病：一个及时和具有挑战性的诊断。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-01-01 Epub Date: 2024-12-31 DOI: 10.1097/MPH.0000000000002967

Jinjun Cheng, Yannan Wang, Shunyou Gong, Birte Wistinghausen, Shana Jacobs, Reuven J Schore, Keri Toner

引用次数: 0