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The growth and development of children with β-thalassemia major one year after allogeneic hematopoietic stem cell transplantation.
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-11-30 Epub Date: 2024-11-26 DOI: 10.21037/tp-24-202
Hongfeng Luo, Yun Lin, Caiyun Kuang

Background: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has proven to be an effective curative intervention for children with β-thalassemia major (β-TM). They are susceptible to growth disorders due various factors. The primary objective of this study was to investigate the growth and development of children with β-TM both before and after undergoing allo-HSCT during a one-year follow-up period.

Methods: According to the international reference standards for child growth established by the World Health Organization in 2006, participants were divided into two groups based on their Z scores: weight-for-age Z scores (WAZ), height-for-age Z scores (HAZ), and body mass index-for-age Z scores (BAZ) before allo-HSCT. The first group included children with normal nutritional levels (-2≤ Z scores ≤2), while the second group consisted of children with malnutrition (Z scores <-2 or >2). Measurements of weight, height, and body mass index (BMI) were recorded at six time points: prior to allo-HSCT (T0), and 1 month (T1), 3 months (T3), 6 months (T6), 9 months (T9), and 12 months (T12) following the procedure.

Results: Among the 56 children diagnosed with β-TM, 25% were malnourished prior to HSCT. In the group with normal nutrition, mean WAZ (P=0.04) and HAZ (P<0.001) values were significantly higher at T12 compared to T0; however, mean BAZ was similar at T12 and T0 (P=0.61). In contrast, in the malnutrition group, no significant differences were observed in mean WAZ (P=0.12), HAZ (P=0.27), or BAZ (P=0.75) values between T12 and T0. Notably, the incidence of increased HAZ was significantly more prevalent in children aged seven years or younger than in those older than seven years (P=0.01).

Conclusions: Children diagnosed with β-TM who had normal nutritional levels at the time of allo-HSCT showed significant growth improvements within one year of follow-up compared to their pre-transplantation state. Additionally, the age at which allo-HSCT was performed may substantially influence post-transplantation growth rates. Regular monitoring of growth and development from the time of diagnosis is crucial to ensure optimal nutritional status at the time of allo-HSCT, thereby enhancing the prognosis for these children after transplantation.

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引用次数: 0
Biliary atresia with ectopic thyroid in the porta hepatis: a case report and literature review.
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-11-30 Epub Date: 2024-11-26 DOI: 10.21037/tp-24-307
Li Zhao, Linsheng Zhao, Xiaoli Hu, Zhiru Wang, Jianghua Zhan

Background: Ectopic thyroid is a rare disease, with ectopic thyroid in the porta hepatis being extremely rare. We present a rare case involving an infant with biliary atresia (BA) wherein ectopic thyroid tissue was incidentally discovered in the porta hepatis after the Kasai operation.

Case description: A 2-month-old boy initially presented with jaundice, manifesting as yellow skin and sclera, on the first day following birth. After receiving treatment at a local healthcare facility, jaundice partially subsided, but the color of his stool became lighter 15 days before admission to Tianjin Children's Hospital. Upon B-ultrasound examination at our outpatient clinic, the patient showed bilateral oblique hernia, bilateral testicular hydrocele, and umbilical hernia, and he was admitted to the Department of Gastroenterology at Tianjin Children's Hospital for further treatment of unexplained jaundice. Laparotomy was performed after a comprehensive evaluation, during which we observed the gallbladder to be small and poorly developed. Cholangiography was then performed, revealing visualization of the gallbladder and common bile duct but not the common hepatic duct or right and left hepatic ducts. Intraoperative frozen section examination of wedge-shaped liver tissue indicated findings consistent with extrahepatic biliary obstruction. The Kasai operation was subsequently performed, and an examination of the gallbladder and a fibrous plaque from the porta hepatis after the operation revealed the presence of ectopic thyroid tissues.

Conclusions: Ectopic thyroid in the porta hepatis is extremely rare. We reviewed literature from 1960 to 2022 and identified six relevant case reports of ectopic thyroid in the porta hepatis. To our knowledge, this report constitutes the first report of BA complicated by ectopic thyroid in the porta hepatis.

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引用次数: 0
Efficacy and safety of spleen aminopeptide oral solution for children with allergic rhinitis and adenoid hypertrophy: a randomised trial. 脾氨肽口服溶液治疗过敏性鼻炎和腺样体肥大患儿的有效性和安全性:随机试验。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-28 DOI: 10.21037/tp-24-203
Yue Huang, Meng Zhao, Chen Lin, Fenghua Qin, Qi Li, Bo Duan, Wenxia Chen, Zhengmin Xu

Background: The spleen aminopeptide oral solution (SAOS) is a solution containing a mixture of polypeptide amino acids and polynucleotides derived from healthy bovine spleen. This study aimed to establish a randomized controlled clinical trial to evaluate the efficacy and safety of SAOS for children with allergic rhinitis (AR) and adenoid hypertrophy (AH).

Methods: Children with AR and AH who visited the Otolaryngology Department of the Children's Hospital Affiliated with Fudan University between June 2022 and April 2023 were included. The primary outcome was the adenoid-to-nasopharynx (A/N) ratio, and the secondary outcomes included nasal symptom score, AH score, and medication score. The primary outcome was evaluated before treatment (T0), after 1 month of treatment (T1), and after 2 months of treatment (T2). Blood and urine samples were collected at T0 and T2 for safety evaluation.

Results: A total of 78 patients were included, with 36 in the control group and 42 in the SAOS group. As the duration of medication increased, there was a significant decrease in nasal symptom scores (sneezing: F=52.806, P<0.001; runny nose: F=28.802, P<0.001; nasal itching: F=101.272, P<0.001; nasal congestion: F=83.349, P<0.001). Significant improvements in mouth breathing (Z=-2.650, P=0.008) and restless sleep (Z=-2.759, P=0.006) were observed in the SAOS group compared to the control group at T2. Additionally, the difference in scores between T2 and T0 was significantly lower in the SAOS group compared to the control group (Z=-2.299, P=0.02).

Conclusions: As an adjuvant therapy for 8 weeks, SAOS could significantly reduce the size of adenoids and improve clinical symptoms associated with AH.

Trial registration: The study was registered on the Chinese Clinical Trial Registry website (https://www.chictr.org.cn/) (registration number: ChiCTR2200056763).

背景:脾脏氨肽口服溶液(SAOS)是一种含有从健康牛脾脏中提取的多肽氨基酸和多核苷酸混合物的溶液。本研究旨在建立一项随机对照临床试验,以评估 SAOS 对过敏性鼻炎(AR)和腺样体肥大(AH)患儿的疗效和安全性:方法:纳入2022年6月至2023年4月期间在复旦大学附属儿童医院耳鼻喉科就诊的过敏性鼻炎和腺样体肥大患儿。主要结果为腺样体与鼻咽部(A/N)比率,次要结果包括鼻部症状评分、AH评分和用药评分。主要结果在治疗前(T0)、治疗 1 个月后(T1)和治疗 2 个月后(T2)进行评估。在治疗前(T0)和治疗后(T1)及治疗 2 个月后(T2)采集血液和尿液样本进行安全性评估:共纳入 78 名患者,其中对照组 36 人,SAOS 组 42 人。随着用药时间的延长,鼻部症状评分显著下降(打喷嚏:F=52.806,PC=0.001;打喷嚏:F=52.806,PC=0.001):F=52.806,PC结论:作为8周的辅助治疗,SAOS可明显缩小腺样体体积,改善与AH相关的临床症状:该研究已在中国临床试验注册中心网站(https://www.chictr.org.cn/)注册(注册号:ChiCTR2200056763)。
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引用次数: 0
A brief clinical genetics review: stepwise diagnostic processes of a monogenic disorder-hypertriglyceridemia. 临床遗传学简要回顾:单基因疾病--高甘油三酯血症的逐步诊断过程。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-23 DOI: 10.21037/tp-24-131
Masako Ueda

The completion of the Human Genome Project and tremendous advances in automated high-throughput genetic analysis technologies have enabled explosive progress in the field of genetics, which resulted in countless discoveries of novel genes and pathways. Many phenotype- or disease-associated single nucleotide polymorphisms (SNPs) with a high statistical significance have been identified through numerous genome-wide association studies (GWAS), and various polygenic risk scoring (PRS) schemes have been proposed to identify individuals with a high risk for a certain trait or disorder. Meanwhile, medical education in genetics has lagged far behind, leaving many physicians and healthcare providers unprepared in the genomic era. Thus, there is an urgent need to educate physicians and healthcare providers with basic knowledge and skills in genetics. To facilitate this, some basic terminologies and concepts are discussed in this review. In addition, some important considerations in delineating and incorporating clinical genetic testing in the diagnosis and management of a monogenic disorder are illustrated in a stepwise fashion. Furthermore, the effects of disease-associated SNPs represented by a PRS scheme clearly demonstrated that even the phenotypes of a monogenic disorder due to the same pathogenic variant in family members are modulated by the polygenic background. In human genetics, despite these explosive advancements, we are still far from clearly deciphering the interplay of gene variants to effect unique characteristics in an individual. In addition, sophisticated genome or gene directed therapies are being investigated for numerous disorders. Therefore, evolution in the field of genetics is likely to continue into the foreseeable future. In the meantime, much emphasis should be placed on educating physicians and healthcare professionals to be well-versed and skillful in the clinical use of genetics so that they can fully embrace the new era of precision medicine.

人类基因组计划的完成以及自动化高通量遗传分析技术的巨大进步,使遗传学领域取得了爆炸性进展,发现了无数新基因和新途径。通过大量的全基因组关联研究(GWAS),发现了许多与表型或疾病相关的单核苷酸多态性(SNPs),这些单核苷酸多态性具有很高的统计学意义,人们还提出了各种多基因风险评分(PRS)方案,以确定某种性状或疾病的高风险个体。与此同时,遗传学方面的医学教育却远远滞后,导致许多医生和医疗服务提供者在基因组时代毫无准备。因此,迫切需要教育医生和医疗服务提供者掌握遗传学的基本知识和技能。为此,本综述讨论了一些基本术语和概念。此外,本综述还以循序渐进的方式说明了在诊断和管理单基因遗传性疾病时,在界定和纳入临床基因检测方面的一些重要注意事项。此外,以 PRS 方案为代表的疾病相关 SNPs 的影响清楚地表明,即使单基因遗传疾病的表型是由家族成员中的同一致病变体引起的,也会受到多基因背景的影响。在人类遗传学领域,尽管取得了这些爆炸性的进展,但我们仍远未清楚地解读基因变异对个体独特特征的影响。此外,针对许多疾病的复杂基因组或基因导向疗法也在研究之中。因此,在可预见的未来,遗传学领域的发展仍将继续。在此期间,我们应重视对医生和医疗保健专业人员的教育,使他们精通并熟练掌握遗传学的临床应用,从而全面迎接精准医疗的新时代。
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引用次数: 0
Histiocytic necrotizing lymphadenitis with autoimmune encephalitis in a child: a case report. 儿童组织细胞坏死性淋巴结炎伴自身免疫性脑炎:病例报告。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-28 DOI: 10.21037/tp-24-380
Yiyun Zhou, Sainan Shu

Background: Histiocytic necrotizing lymphadenitis (HNL) is a rare benign self-limiting inflammatory disease that mainly affects young adults; however, the exact pathogenesis is unknown. A four-year-old child who was diagnosed with HNL combined with autoimmune encephalitis (AE) was admitted to Tongji Hospital of Huazhong University of Science and Technology. This study aimed to describe the clinical characteristics, laboratory tests, imaging findings, and treatment outcomes of this pediatric patient with HNL + AE.

Case description: A 4-year-old male child was admitted to our hospital after presenting with a neck mass persisting for over two months and a fever lasting five days. The initial symptom was fever accompanied by lymph node enlargement. The patient was diagnosed with HNL; however, the patient did not respond significantly to treatment, and he continued to have lymph node enlargement and intermittent fever. Some six months later, the patient developed neurological symptoms, including decreased voluntary activity, impaired speech, and reduced appetite. Subsequent serum testing yielded positive results for contactin-associated protein-2 (CASPR2), leading to a diagnosis of CASPR2 antibody-associated encephalitis. The final diagnosis was HNL + AE. The patient's condition improved after the corresponding treatment was administered. Subsequent follow-up examinations indicated no recurrence.

Conclusions: This is the first documented case of HNL + AE in a pediatric patient exhibiting typical symptoms of fever, lymph node swelling, and pain, accompanied by acute neurologic symptoms, and an extended disease course. This report provides a reference for the diagnosis of HNL + AE in the future.

背景:组织细胞坏死性淋巴结炎(HNL)是一种罕见的良性自限性炎症性疾病,主要累及青壮年,但确切的发病机制尚不清楚。华中科技大学同济医学院附属同济医院收治了一名被诊断为 HNL 合并自身免疫性脑炎(AE)的四岁儿童。本研究旨在描述这名 HNL + AE 儿童患者的临床特征、实验室检查、影像学检查结果和治疗效果:一名 4 岁男童因颈部肿块持续两个多月、发热 5 天而入院。最初的症状是发热并伴有淋巴结肿大。患者被诊断为 HNL,但治疗效果不佳,淋巴结肿大和间歇性发热持续存在。大约六个月后,患者出现了神经系统症状,包括自主活动减少、言语障碍和食欲减退。随后的血清检测结果显示接触素相关蛋白-2(CASPR2)呈阳性,诊断为 CASPR2 抗体相关性脑炎。最终诊断为 HNL + AE。在接受相应治疗后,患者的病情有所好转。结论:这是首例记录在案的 HNL + AE 儿童患者,表现出典型的发热、淋巴结肿大和疼痛症状,伴有急性神经系统症状,病程较长。本报告为今后诊断 HNL + AE 提供了参考。
{"title":"Histiocytic necrotizing lymphadenitis with autoimmune encephalitis in a child: a case report.","authors":"Yiyun Zhou, Sainan Shu","doi":"10.21037/tp-24-380","DOIUrl":"https://doi.org/10.21037/tp-24-380","url":null,"abstract":"<p><strong>Background: </strong>Histiocytic necrotizing lymphadenitis (HNL) is a rare benign self-limiting inflammatory disease that mainly affects young adults; however, the exact pathogenesis is unknown. A four-year-old child who was diagnosed with HNL combined with autoimmune encephalitis (AE) was admitted to Tongji Hospital of Huazhong University of Science and Technology. This study aimed to describe the clinical characteristics, laboratory tests, imaging findings, and treatment outcomes of this pediatric patient with HNL + AE.</p><p><strong>Case description: </strong>A 4-year-old male child was admitted to our hospital after presenting with a neck mass persisting for over two months and a fever lasting five days. The initial symptom was fever accompanied by lymph node enlargement. The patient was diagnosed with HNL; however, the patient did not respond significantly to treatment, and he continued to have lymph node enlargement and intermittent fever. Some six months later, the patient developed neurological symptoms, including decreased voluntary activity, impaired speech, and reduced appetite. Subsequent serum testing yielded positive results for contactin-associated protein-2 (CASPR2), leading to a diagnosis of CASPR2 antibody-associated encephalitis. The final diagnosis was HNL + AE. The patient's condition improved after the corresponding treatment was administered. Subsequent follow-up examinations indicated no recurrence.</p><p><strong>Conclusions: </strong>This is the first documented case of HNL + AE in a pediatric patient exhibiting typical symptoms of fever, lymph node swelling, and pain, accompanied by acute neurologic symptoms, and an extended disease course. This report provides a reference for the diagnosis of HNL + AE in the future.</p>","PeriodicalId":23294,"journal":{"name":"Translational pediatrics","volume":"13 10","pages":"1892-1898"},"PeriodicalIF":1.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11543126/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142628085","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}
引用次数: 0
The effectiveness of therapist-led family-centered language intervention for children with language delay. 以治疗师为主导、以家庭为中心的语言干预对语言发育迟缓儿童的有效性。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-28 DOI: 10.21037/tp-24-225
Qiongli Fan, Xiumei Yu, Wang Cheng, Lisha Su, Yuping Zhang, Quanxing Liu, Zhifeng Wu

Background: China's language therapist shortage and intergenerational caregiving trends underscore the need for family-based language training, yet such interventions are underutilized and family roles undervalued. This study aims to investigate the effect of a therapist-led family-centered intervention on children with language delay.

Methods: The study comprised 134 children, comprising 59 girls and 75 boys, aged between 2 and 5 years (39.92±10.23 months) who presented with language delay. These participants were selected from the pediatric rehabilitation ward of the Second Affiliated Hospital of Army Medical University from July 2021 and July 2023. They were divided into a control group (n=70) receiving language therapy led by a language therapist, and an observation group (n=64) receiving therapist-led family-centered language intervention in conjunction with the treatment provided to the control group. Demographic characteristics were analyzed. The Gesell and Sign-significant (S-S) were used for assessments before and after the intervention.

Results: No significant demographic differences were found between the groups. Both groups demonstrated significant improvements in language development following a 3-month intervention as assessed by the Gesell assessment. The control group showed an increase from 52.97±4.79 to 65.97±3.48 (P<0.001), while the observation group improved from 53.53±4.06 to 71.98±4.51 (P<0.001). Additionally, prior to the intervention, differences were observed between the control group and the observation group in S-S assessments, particularly in symbolic form and instructional content (P=0.04 and P<0.001) as well as foundational research topics (P=0.04 and P<0.001). Following the intervention, the observation group demonstrated more significant advancements in language development compared to control group as evidenced by higher Gesell scores (18.45±5.72 vs. 13.00±6.16, P<0.001), S-S assessments in symbolic form and instructional content (1.11±1.55 vs. 0.53±1.57, P=0.03), and foundational research topics (1.42±1.88 vs. 0.64±2.14, P=0.02). Based on the Gesell assessment, there was a significant improvement in personal-social skills for both groups of children following intervention (all P<0.05), with the observation group demonstrating a more pronounced enhancement compared to the control group (4.73±4.07 vs. 2.20±3.68, P<0.001).

Conclusions: Therapist-led family-centered interventions enhance language development, with parents playing a significant role in mitigating skip-generation caregiving and fostering greater parent-child interactions.

背景:中国语言治疗师的短缺和代际照料的趋势凸显了以家庭为基础的语言培训的必要性,然而此类干预措施却未得到充分利用,家庭的作用也未得到重视。本研究旨在探讨以治疗师为主导、以家庭为中心的干预措施对语言发育迟缓儿童的影响:研究对象包括 134 名 2 至 5 岁(39.92±10.23 个月)的语言发育迟缓儿童,其中包括 59 名女孩和 75 名男孩。这些参与者选自 2021 年 7 月至 2023 年 7 月陆军军医大学第二附属医院儿科康复病房。他们被分为对照组(70人)和观察组(64人),对照组接受语言治疗师指导的语言治疗,观察组在对照组治疗的基础上接受治疗师指导的以家庭为中心的语言干预。对人口统计学特征进行了分析。干预前后采用格赛尔(Gesell)和手势(S-S)进行评估:结果:两组之间没有发现明显的人口统计学差异。通过格赛尔评估,两组儿童在接受为期 3 个月的干预后,语言发展均有明显改善。对照组从 52.97±4.79 提高到 65.97±3.48(Pvs.13.00±6.16,Pvs.0.53±1.57,P=0.03),基础研究课题(1.42±1.88 vs. 0.64±2.14,P=0.02)也有所提高。根据格塞尔评估,两组儿童的个人社交能力在干预后都有显著提高(所有 Pvs 均为 2.20±3.68,P=0.03):以治疗师为主导、以家庭为中心的干预措施能促进语言发展,而父母在减轻跳代照料和促进亲子互动方面发挥着重要作用。
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引用次数: 0
Successful treatment of a 4-year-old girl with pure malignant rhabdoid tumor of the bladder: a case report. 成功治疗一名患有膀胱纯恶性横纹肌瘤的 4 岁女孩:病例报告。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-23 DOI: 10.21037/tp-24-255
Jian Li, Jiajia Bao, Tianjiao Hu, Qian Zhao, Jianfeng Zhou, Li Zhou, Tao Li

Background: Extracranial malignant rhabdoid tumors (eMRTs) are rare, highly aggressive neoplasms, predominantly arising in the kidneys, with bladder involvement being extremely uncommon. Optimal management strategies, particularly bladder-preserving approaches, are not well-established due to the rarity of these cases.

Case description: We report the case of a 4-year-old girl who presented with persistent gross hematuria. Imaging revealed a large bladder mass and histopathological examination confirmed the diagnosis of an extrarenal rhabdoid tumor with loss of SMARCB1 expression. The patient underwent a multimodal treatment approach, including bladder-preserving surgery, 12 cycles of high-dose MRTK-2020 neoadjuvant chemotherapy [comprising actinomycin D, vincristine, doxorubicin, cyclophosphamide (AVDC), ifosfamide, carboplatin, and etoposide (ICE)], followed by adjuvant radiotherapy. Remarkably, the patient achieved complete remission after two cycles of chemotherapy and remained in continuous remission with no evidence of disease at the 15-month follow-up.

Conclusions: This case underscores the diagnostic challenges of malignant rhabdoid tumors (MRTs) in bladder pathologies and highlights the significance of considering such tumors in differential diagnoses. Despite the limited evidence base, bladder-preserving surgeries, when feasible, should be considered and accompanied by adjuvant therapies for optimal outcomes. This case illustrates the potential for successful treatment of pure MRTs of the bladder using a combination of surgery, chemotherapy, and radiotherapy.

背景:颅外恶性横纹肌瘤(eMRTs)是一种罕见的高侵袭性肿瘤,主要发生在肾脏,膀胱受累极为罕见。由于这些病例的罕见性,最佳治疗策略,尤其是保留膀胱的方法,尚未得到很好的确立:我们报告了一例 4 岁女孩的病例,她出现持续性毛细血尿。影像学检查发现了一个巨大的膀胱肿块,组织病理学检查确诊为肾外横纹肌瘤,SMARCB1表达缺失。患者接受了多模式治疗,包括膀胱保留手术、12个周期的大剂量MRTK-2020新辅助化疗(包括放线菌素D、长春新碱、多柔比星、环磷酰胺(AVDC)、伊福酰胺、卡铂和依托泊苷(ICE)),随后接受了辅助放疗。值得注意的是,患者在接受两个周期的化疗后病情得到完全缓解,并且在 15 个月的随访中病情持续缓解,没有任何疾病迹象:本病例凸显了膀胱病变中恶性横纹肌瘤(MRTs)的诊断难题,并强调了在鉴别诊断中考虑此类肿瘤的重要性。尽管证据基础有限,但在可行的情况下,应考虑进行膀胱保留手术,并辅以辅助治疗,以获得最佳疗效。本病例说明了采用手术、化疗和放疗相结合的方法成功治疗膀胱单纯 MRT 的可能性。
{"title":"Successful treatment of a 4-year-old girl with pure malignant rhabdoid tumor of the bladder: a case report.","authors":"Jian Li, Jiajia Bao, Tianjiao Hu, Qian Zhao, Jianfeng Zhou, Li Zhou, Tao Li","doi":"10.21037/tp-24-255","DOIUrl":"https://doi.org/10.21037/tp-24-255","url":null,"abstract":"<p><strong>Background: </strong>Extracranial malignant rhabdoid tumors (eMRTs) are rare, highly aggressive neoplasms, predominantly arising in the kidneys, with bladder involvement being extremely uncommon. Optimal management strategies, particularly bladder-preserving approaches, are not well-established due to the rarity of these cases.</p><p><strong>Case description: </strong>We report the case of a 4-year-old girl who presented with persistent gross hematuria. Imaging revealed a large bladder mass and histopathological examination confirmed the diagnosis of an extrarenal rhabdoid tumor with loss of <i>SMARCB1</i> expression. The patient underwent a multimodal treatment approach, including bladder-preserving surgery, 12 cycles of high-dose MRTK-2020 neoadjuvant chemotherapy [comprising actinomycin D, vincristine, doxorubicin, cyclophosphamide (AVDC), ifosfamide, carboplatin, and etoposide (ICE)], followed by adjuvant radiotherapy. Remarkably, the patient achieved complete remission after two cycles of chemotherapy and remained in continuous remission with no evidence of disease at the 15-month follow-up.</p><p><strong>Conclusions: </strong>This case underscores the diagnostic challenges of malignant rhabdoid tumors (MRTs) in bladder pathologies and highlights the significance of considering such tumors in differential diagnoses. Despite the limited evidence base, bladder-preserving surgeries, when feasible, should be considered and accompanied by adjuvant therapies for optimal outcomes. This case illustrates the potential for successful treatment of pure MRTs of the bladder using a combination of surgery, chemotherapy, and radiotherapy.</p>","PeriodicalId":23294,"journal":{"name":"Translational pediatrics","volume":"13 10","pages":"1879-1884"},"PeriodicalIF":1.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11543121/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142628757","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}
引用次数: 0
Efficacy of core biopsies for diagnosing inflammatory myofibroblastic tumors in pediatric patients: case series from a single tertiary referral center. 核心活检对诊断儿科炎性肌成纤维细胞瘤的疗效:来自一家三级转诊中心的病例系列。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-28 DOI: 10.21037/tp-24-239
Ugo Maria Pierucci, Irene Paraboschi, Carlotta Ardenghi, Camilla Viglio, Giorgio Giuseppe Orlando Selvaggio, Giulia Lanfranchi, Michela Casanova, Paola Collini, Marta Barisella, Marcello Napolitano, Anna Camporesi, Gloria Pelizzo

Background: Inflammatory myofibroblastic tumors (IMTs) are rare, often non-metastasizing neoplasms characterized by fibro/myofibroblastic spindle cells with varying infiltrates of plasma cells, lymphocytes, and/or eosinophils. Despite their generally indolent nature, IMTs can exhibit locally aggressive behavior and a significant tendency for local recurrence, making complete surgical resection the standard treatment approach. Accurate diagnosis can be challenging due to the overlap in imaging features with more aggressive tumors, necessitating preoperative biopsies to enable differential diagnosis and guide treatment decisions. The complexity of distinguishing IMTs from other malignancies underscores the importance of biopsy in establishing an accurate diagnosis and planning appropriate management strategies.

Case description: This study presents the cases of four pediatric patients (three males, one female) diagnosed with IMT, involving tumors located in the lung (one case), bladder (one case), and liver (two cases). Initial minimally invasive biopsies, including a US-guided tru-cut core biopsy and a percutaneous core biopsy in one case, as well as endoscopic core biopsies in two other cases, yielded inconclusive results. These initial procedures failed to provide definitive diagnostic information, necessitating the use of more precise diagnostic techniques to achieve a definitive histological diagnosis of IMT.

Conclusions: The findings indicate that when initial biopsy results are inconclusive in cases suspected to be IMT, more precise diagnostic procedures may be necessary to secure a definitive diagnosis. This highlights the need for careful consideration of alternative biopsy methods to ensure accurate identification and effective management of IMT in pediatric patients.

背景:炎性肌纤维母细胞瘤(IMTs)是一种罕见的非转移性肿瘤,以纤维/肌纤维母细胞纺锤形细胞为特征,伴有不同程度的浆细胞、淋巴细胞和/或嗜酸性粒细胞浸润。尽管 IMT 通常具有不扩散的特性,但它会表现出局部侵袭性和明显的局部复发倾向,因此完全手术切除是标准的治疗方法。由于与侵袭性更强的肿瘤在影像学特征上存在重叠,因此准确诊断可能具有挑战性,因此有必要进行术前活检,以进行鉴别诊断并指导治疗决策。IMT与其他恶性肿瘤鉴别的复杂性凸显了活检在确定准确诊断和规划适当治疗策略方面的重要性:本研究介绍了四例被诊断为IMT的儿科患者(三男一女)的病例,肿瘤分别位于肺部(一例)、膀胱(一例)和肝脏(两例)。最初的微创活检,包括美国引导下的真切核心活检和一个病例的经皮核心活检,以及另外两个病例的内窥镜核心活检,均未得出结论。这些初始程序未能提供明确的诊断信息,因此有必要使用更精确的诊断技术,以获得IMT的明确组织学诊断:结论:研究结果表明,当怀疑是内皮内瘤变的病例的初步活检结果不确定时,可能需要采用更精确的诊断程序才能获得明确诊断。这凸显了仔细考虑其他活检方法的必要性,以确保准确识别和有效管理儿童患者的内皮内瘤变。
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引用次数: 0
The indications and safety of rituximab for the treatment of pediatric autoimmune diseases: a single-center retrospective study. 利妥昔单抗治疗小儿自身免疫性疾病的适应症和安全性:一项单中心回顾性研究。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-28 DOI: 10.21037/tp-24-233
Mohammed A Alsubaie, Abdulrahman B Bahkali, Saeed A Alhudaifi, Majed T Osaylan, Abdulaziz M Alghamdi, Mohammed Nashawi, Faris A Althubaiti

Background: Autoimmune diseases in children pose therapeutic challenges due to their refractory nature and the associated morbidity. Rituximab (RTX), a monoclonal antibody targeting CD20, has emerged as a promising steroid-sparing therapy for various autoimmune disorders by depleting B cells. However, its indications and safety in pediatric populations in our region remain insufficiently studied. This study aims to review the indications and safety of RTX in treating pediatric autoimmune diseases within a single-center setting.

Methods: A retrospective study was conducted on pediatric patients aged 18 years or younger who received RTX for different autoimmune diseases between January 2013 and March 2023 at King Abdulaziz University Hospital, Saudi Arabia. Data on demographics, indications, infusion details, adverse events, and concurrent medications were collected and analyzed.

Results: Twenty-two patients were included, with nephrotic syndrome and systemic lupus erythematosus (SLE) being the most common indications for RTX. The mean age at diagnosis and first RTX infusion was 8 and 9 years, respectively. The most commonly used protocol involved administering 2 infusions of 375 mg/m2 each, given 2 weeks apart. RTX was commonly used as a second-line treatment following corticosteroids. Infusion-related adverse events occurred in 31.8% of patients, ranging from mild reactions such as chest tightness, fever, and headache to severe reactions such as anaphylaxis. No infectious related adverse events were observed.

Conclusions: This study highlights both the varied indications for which RTX was prescribed and the need for vigilance concerning infusion-related adverse events. It underscores the importance of close monitoring and appropriate management to ensure the safety of RTX therapy in pediatric patients. Further research is warranted to optimize treatment strategies and enhance patient outcomes in this population.

背景:儿童自身免疫性疾病因其难治性和相关的发病率给治疗带来了挑战。利妥昔单抗(RTX)是一种靶向 CD20 的单克隆抗体,可通过消耗 B 细胞来治疗各种自身免疫性疾病,是一种很有前景的节省类固醇疗法。然而,在我们地区,对其在儿科人群中的适应症和安全性的研究仍然不足。本研究旨在回顾RTX在单中心环境下治疗小儿自身免疫性疾病的适应症和安全性:方法:本研究对沙特阿拉伯阿卜杜勒阿齐兹国王大学医院在 2013 年 1 月至 2023 年 3 月期间接受 RTX 治疗不同自身免疫性疾病的 18 岁或以下儿童患者进行了回顾性研究。研究收集并分析了有关人口统计学、适应症、输注细节、不良事件和并发症药物的数据:共纳入22名患者,肾病综合征和系统性红斑狼疮(SLE)是RTX最常见的适应症。确诊和首次输注 RTX 的平均年龄分别为 8 岁和 9 岁。最常用的方案是输注 2 次,每次 375 毫克/平方米,每次间隔 2 周。RTX通常作为皮质类固醇之后的二线治疗方法。31.8%的患者发生了输液相关不良反应,轻者如胸闷、发热和头痛,重者如过敏性休克。未观察到与感染相关的不良事件:本研究强调了 RTX 的各种适应症,以及对输液相关不良事件保持警惕的必要性。它强调了密切监测和适当管理对确保儿童患者 RTX 治疗安全性的重要性。我们有必要开展进一步研究,以优化治疗策略,提高这一人群的治疗效果。
{"title":"The indications and safety of rituximab for the treatment of pediatric autoimmune diseases: a single-center retrospective study.","authors":"Mohammed A Alsubaie, Abdulrahman B Bahkali, Saeed A Alhudaifi, Majed T Osaylan, Abdulaziz M Alghamdi, Mohammed Nashawi, Faris A Althubaiti","doi":"10.21037/tp-24-233","DOIUrl":"https://doi.org/10.21037/tp-24-233","url":null,"abstract":"<p><strong>Background: </strong>Autoimmune diseases in children pose therapeutic challenges due to their refractory nature and the associated morbidity. Rituximab (RTX), a monoclonal antibody targeting CD20, has emerged as a promising steroid-sparing therapy for various autoimmune disorders by depleting B cells. However, its indications and safety in pediatric populations in our region remain insufficiently studied. This study aims to review the indications and safety of RTX in treating pediatric autoimmune diseases within a single-center setting.</p><p><strong>Methods: </strong>A retrospective study was conducted on pediatric patients aged 18 years or younger who received RTX for different autoimmune diseases between January 2013 and March 2023 at King Abdulaziz University Hospital, Saudi Arabia. Data on demographics, indications, infusion details, adverse events, and concurrent medications were collected and analyzed.</p><p><strong>Results: </strong>Twenty-two patients were included, with nephrotic syndrome and systemic lupus erythematosus (SLE) being the most common indications for RTX. The mean age at diagnosis and first RTX infusion was 8 and 9 years, respectively. The most commonly used protocol involved administering 2 infusions of 375 mg/m<sup>2</sup> each, given 2 weeks apart. RTX was commonly used as a second-line treatment following corticosteroids. Infusion-related adverse events occurred in 31.8% of patients, ranging from mild reactions such as chest tightness, fever, and headache to severe reactions such as anaphylaxis. No infectious related adverse events were observed.</p><p><strong>Conclusions: </strong>This study highlights both the varied indications for which RTX was prescribed and the need for vigilance concerning infusion-related adverse events. It underscores the importance of close monitoring and appropriate management to ensure the safety of RTX therapy in pediatric patients. Further research is warranted to optimize treatment strategies and enhance patient outcomes in this population.</p>","PeriodicalId":23294,"journal":{"name":"Translational pediatrics","volume":"13 10","pages":"1696-1702"},"PeriodicalIF":1.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11543116/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142628770","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}
引用次数: 0
Improvements in Children's Oncology Group neuroblastoma risk stratification through a change in age cut-off and use of INRGSS. 通过改变年龄分界线和使用 INRGSS,改进儿童肿瘤组织神经母细胞瘤风险分层。
IF 1.5 4区 医学 Q2 PEDIATRICS Pub Date : 2024-10-01 Epub Date: 2024-10-28 DOI: 10.21037/tp-24-319
Wendy B London, Hannah Bousquet, Meredith S Irwin, Michael D Hogarty, Susan L Cohn
{"title":"Improvements in Children's Oncology Group neuroblastoma risk stratification through a change in age cut-off and use of INRGSS.","authors":"Wendy B London, Hannah Bousquet, Meredith S Irwin, Michael D Hogarty, Susan L Cohn","doi":"10.21037/tp-24-319","DOIUrl":"https://doi.org/10.21037/tp-24-319","url":null,"abstract":"","PeriodicalId":23294,"journal":{"name":"Translational pediatrics","volume":"13 10","pages":"1899-1901"},"PeriodicalIF":1.5,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11543114/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142628224","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}
引用次数: 0
期刊
Translational pediatrics
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