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Evaluation of Thromboembolic Events in Patients Hospitalized in the Palliative Care Unit: A Single Center Experience. 姑息治疗病房住院患者血栓栓塞事件的评估:单中心经验。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-27 DOI: 10.1097/MPH.0000000000003091
Zeliha Güzelküçük, Hatice Dilara Aydoğdu, Ganime Ayar, Namik Yaşar Özbek

Objectives: Although thromboembolic events are less common in children compared with adults, complications related to thromboembolism are becoming more frequent due to improved care for children with chronic illnesses. The aim of the study was to evaluate thromboembolic events in pediatric patients hospitalized in palliative care unit (PCU).

Materials and methods: The files of the patients who were hospitalized in PCU of our hospital between January 2020 and January 2023 were retrospectively analyzed. A total of 1002 hospitalizations of 449 patients were examined.

Results: The median age was 6.1 years, F/M: 214/235. We found that 83 (18.4%) patients had thrombosis. In most patients with thrombosis, the primary diseases were malignancy and trauma. During hospitalization, the median time for detecting thrombosis was 26 days (range: 1 to 180 d). We treated 67 out of 83 thrombosis patients with low-molecular-weight heparin (LMWH) for an average of 26 days, ranging from 1 to 188 days. Unfractionated heparin was used in 2 patients, while thrombolytic therapy was used in 2 patients with massive pulmonary embolism and middle cerebral artery thrombosis. None of the thromboembolic events resulted in fatality.

Conclusions: This study revealed that the frequency of thrombosis in pediatric patients hospitalized in PCU was ∼1/5, which is significantly higher than the rates observed in childhood. Catheter-related thrombosis was more frequently observed among patients with thrombosis in the PCU, with malignancy and trauma representing the most common indications for hospitalization. Our findings suggest that certain patient groups should be evaluated for prophylactic anticoagulation.

目的:虽然与成人相比,血栓栓塞事件在儿童中不太常见,但由于对慢性疾病儿童的护理改善,与血栓栓塞相关的并发症正变得越来越频繁。该研究的目的是评估在姑息治疗病房(PCU)住院的儿科患者的血栓栓塞事件。材料与方法:回顾性分析2020年1月至2023年1月在我院PCU住院的患者资料。共检查了449例住院患者的1002例。结果:中位年龄6.1岁,F/M: 214/235。83例(18.4%)患者有血栓形成。在大多数血栓患者中,原发疾病是恶性肿瘤和创伤。住院期间,检测血栓形成的中位时间为26天(范围:1 ~ 180 d)。83例血栓患者中有67例使用低分子肝素(LMWH)治疗,平均疗程为26天,从1天到188天不等。2例患者采用未分割肝素治疗,2例合并大量肺栓塞及大脑中动脉血栓患者采用溶栓治疗。所有血栓栓塞事件均未导致死亡。结论:本研究显示,在PCU住院的儿童患者中,血栓形成的频率为1/5,明显高于儿童期的发生率。导管相关性血栓形成在PCU血栓患者中更为常见,恶性肿瘤和创伤是最常见的住院指征。我们的研究结果表明,某些患者群体应该评估预防性抗凝治疗。
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引用次数: 0
The Incidence and Burden of Peripheral Neuropathy in Pediatric Oncology Patients Receiving Levofloxacin and Vincristine Compared With Vincristine Alone. 小儿肿瘤患者左氧氟沙星联合长春新碱与单用长春新碱比较周围神经病变的发生率和负担
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-25 DOI: 10.1097/MPH.0000000000003103
Prerna Kumar, Molly Meister, Ramkumar Narendran, Kejin Lee

Levofloxacin is the first line antibiotic used to prevent infection in pediatric oncology patients with prolonged and profound neutropenia. These patients often receive vincristine, a critical component of therapy for leukemia and for many solid tumors. Both levofloxacin and vincristine are known to cause peripheral neuropathy (PN); however, the cumulative effect of both drugs on PN remains unknown. The objective of this study was to evaluate the incidence and extent of PN in pediatric oncology patients receiving vincristine and levofloxacin (VL) compared with vincristine alone (VA). In this retrospective cohort study, manual chart review was performed to extract demographic and clinical data for newly diagnosed patients treated at our institution between 2015 and 2020. Patients who received vincristine alone (VA) were compared with patients who received vincristine and levofloxacin (VL). There was no significant difference in the incidence of peripheral neuropathy between the 2 groups; however, the cohort exposed to vincristine and levofloxacin (VL) experienced significantly more peripheral neuropathy symptoms, had more affected categories of peripheral neuropathy, and received additional treatment for their neuropathy symptoms. Exposure to levofloxacin in addition to vincristine therapy was associated with an increased burden of peripheral neuropathy in this pediatric oncology cohort.

左氧氟沙星是预防儿童肿瘤患者长期和深度中性粒细胞减少感染的一线抗生素。这些患者通常接受长春新碱治疗,这是白血病和许多实体瘤治疗的关键成分。已知左氧氟沙星和长春新碱都会引起周围神经病变(PN);然而,两种药物对PN的累积效应尚不清楚。本研究的目的是评估小儿肿瘤患者接受长春新碱和左氧氟沙星(VL)治疗与单独长春新碱(VA)治疗的PN发生率和程度。在这项回顾性队列研究中,对2015年至2020年期间在我院治疗的新诊断患者进行手工图表审查,以提取人口学和临床数据。将单独接受长春新碱(VA)治疗的患者与接受长春新碱和左氧氟沙星(VL)治疗的患者进行比较。两组患者周围神经病变发生率比较,差异无统计学意义;然而,暴露于长春新碱和左氧氟沙星(VL)的队列明显经历了更多的周围神经病变症状,有更多的周围神经病变的影响类别,并接受了额外的神经病变症状治疗。在这个儿科肿瘤学队列中,除了长春新碱治疗外,暴露于左氧氟沙星与周围神经病变负担增加有关。
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引用次数: 0
Epigenetic Aging in Childhood Cancer Survivors. 儿童癌症幸存者的表观遗传衰老。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-25 DOI: 10.1097/MPH.0000000000003104
Prerna Kumar, Beth Speckhart, Yanzhi Wang, Kristen Dotson, Susan Gaitros, Brinda Mehta

Background: Pediatric cancer survivors remain at risk for numerous late effects of therapy and have shown signs of advanced aging. The specific mechanisms at play are unclear but epigenetic modulation may play a role.

Methods: This cross-sectional study included 20 pediatric cancer survivors who received intensive chemotherapy with or without radiation and were a minimum of 5 years from treatment and 20 healthy biological siblings. DNA methylation patterns were analyzed from peripheral blood samples to determine epigenetic age (the difference between biological age and chronological age). Paired t test analysis or the Wilcoxon signed-rank test was used to compare results between survivors and siblings.

Results: The childhood cancer survivor cohort consisted of 12 males and 8 females; the comparative sibling cohort consisted of 8 males and 12 females. Mean chronological age was 15.2±6.28 years for survivors and 16.4±8.31 years for siblings (mean±SD). Survivors demonstrated increased epigenetic age compared with siblings (1.38±3.71 vs. -0.03±3.12 y (mean±SD), mean difference 1.41, [0.30 to 2.52], P=0.016, n=20 pairs). Patients who additionally received photon radiation demonstrated a more notable increase in epigenetic age compared with siblings (3.33±4.13 vs. 0.78±3.04 y (mean±SD), mean difference 2.55, [0.75 to 4.35], P=0.012, n=8 pairs).

Conclusions: Childhood cancer survivors demonstrate increased epigenetic age compared with their healthy siblings. Exposure to radiation was associated with further increased epigenetic age. Epigenetic modulation through DNA methylation may be a potential mechanism contributing to the aging process in childhood cancer survivors.

背景:儿童癌症幸存者仍然存在许多治疗后期效应的风险,并显示出高龄的迹象。具体的机制尚不清楚,但表观遗传调节可能起作用。方法:这项横断面研究包括20名接受强化化疗(含或不含放疗)且治疗至少5年的儿童癌症幸存者和20名健康的生物学兄弟姐妹。从外周血样本中分析DNA甲基化模式,以确定表观遗传年龄(生物年龄与实足年龄之间的差异)。配对t检验分析或Wilcoxon符号秩检验用于比较幸存者和兄弟姐妹之间的结果。结果:儿童癌症幸存者队列包括12名男性和8名女性;比较兄弟姐妹队列包括8名男性和12名女性。幸存者的平均实足年龄为15.2±6.28岁,兄弟姐妹的平均实足年龄为16.4±8.31岁(平均±SD)。与兄弟姐妹相比,幸存者表现出表观遗传年龄增加(1.38±3.71比-0.03±3.12 y (mean±SD),平均差异1.41,[0.30至2.52],P=0.016, n=20对)。与兄弟姐妹相比,额外接受光子辐射的患者表观遗传年龄更显着增加(3.33±4.13比0.78±3.04 y (mean±SD),平均差2.55,[0.75 ~ 4.35],P=0.012, n=8对)。结论:与健康的兄弟姐妹相比,儿童癌症幸存者表现出表观遗传年龄的增加。暴露于辐射与表观遗传年龄的进一步增加有关。通过DNA甲基化的表观遗传调节可能是促进儿童癌症幸存者衰老过程的潜在机制。
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引用次数: 0
Rare Pancreatitis-Related Complications of L-Asparaginase in Pediatric Acute Lymphoblastic Leukemia: A Case With Pseudocyst and Panniculitis and Literature Review. 小儿急性淋巴细胞白血病中l -天冬酰胺酶罕见的胰腺炎相关并发症:假性囊肿和泛膜炎1例并文献复习。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-25 DOI: 10.1097/MPH.0000000000003110
Fatma B Kurtipek, Çağri Damar, Elif Gökçek, Belgin Gülhan, Müjdem N Azili, Neşe Yarali

Background: L-asparaginase is an essential chemotherapeutic agent for treating pediatric acute lymphoblastic leukemia (ALL), but its use is commonly linked to severe complications, including pancreatitis. Pancreatic pseudocysts and panniculitis are a rare complication of L-asparaginase-induced pancreatitis.

Case presentation: We report the case of a 20-month-old girl diagnosed with pre-B ALL who developed pancreatitis, complicated by pseudocyst formation and panniculitis, following L-asparaginase therapy. The patient had clinical deterioration and the development of a mechanical ventilation requirement; septic shock was initially suspected. However, due to the appearance of cutaneous findings a few days later, radiologic investigations were performed, revealing the formation of intra-abdominal pseudocysts and subcutaneous fat necrosis. Consequently, a delayed diagnosis of pancreatitis was made, which was managed conservatively with a multidisciplinary approach. Eventually, the pseudocyst resolved with close clinical and radiologic monitoring.

Discussion: Pancreatic pseudocyst formation in pediatric oncology patients is uncommon but necessitates prompt recognition and an individualized treatment strategy. Panniculitis secondary to pancreatitis is even rarer and may serve as an early cutaneous clue to underlying pancreatic pathology. Conservative management of pseudocysts, supported by interventional radiology and close follow-up, can be effective in select cases, reducing the risk associated with surgical intervention.

Conclusion: This case underscores the importance of heightened clinical suspicion for pancreatic complications in pediatric patients receiving L-asparaginase. A multidisciplinary team approach is essential for effective diagnosis and treatment. Additional research is warranted to refine treatment guidelines for L-asparaginase-induced pancreatitis and its complications.

背景:l -天冬酰胺酶是治疗儿童急性淋巴细胞白血病(ALL)必不可少的化疗药物,但其使用通常与严重并发症有关,包括胰腺炎。胰腺假性囊肿和胰膜炎是l -天冬酰胺酶引起的胰腺炎的罕见并发症。病例介绍:我们报告一个20个月大的女孩诊断为b前ALL谁发展胰腺炎,并发假性囊肿形成和胰腺炎,l -天冬酰胺酶治疗后。患者出现临床恶化,需要机械通气;最初怀疑为感染性休克。然而,由于几天后出现皮肤发现,影像学检查显示腹腔内假性囊肿和皮下脂肪坏死的形成。因此,胰腺炎的延迟诊断,这是保守管理与多学科的方法。最终,假性囊肿在密切的临床和放射学监测下消退。讨论:胰腺假性囊肿形成在儿科肿瘤患者中并不常见,但需要及时识别和个性化的治疗策略。继发于胰腺炎的胰膜炎更为罕见,可作为潜在胰腺病理的早期皮肤线索。在介入放射学和密切随访的支持下,假性囊肿的保守治疗在特定病例中是有效的,降低了与手术干预相关的风险。结论:本病例强调了对接受l -天冬酰胺酶治疗的儿科患者胰腺并发症的高度临床怀疑的重要性。多学科团队的方法对于有效的诊断和治疗至关重要。需要进一步的研究来完善l -天冬酰胺酶诱导的胰腺炎及其并发症的治疗指南。
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引用次数: 0
Comparing Tolerability and Toxicity of Calaspargase-Pegol and Pegaspargase in Pediatric Leukemia Patients. 比较天冬氨酸- pegol和天冬氨酸酶治疗儿童白血病的耐受性和毒性。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-22 DOI: 10.1097/MPH.0000000000003098
Jinjoo Kang, Srivarshini Kanukollu, Angelica Bevinetto, Rosamarie Corless, Arlene Redner

As of December 2022, calaspargase-pegol is the standard long-acting asparaginase formulation for patients younger than 22 years of age with acute leukemia. This 3-year retrospective evaluation compared real-world safety and efficacy of calaspargase-pegol versus pegaspargase in pediatric patients. Incidence and severity of hypersensitivity reactions, silent inactivation, and common asparaginase-associated adverse effects were collected. A total of 103 patients were included, of whom 50 received calaspargase-pegol and 58 received pegaspargase. Similar rates of symptomatic hypersensitivity reactions (20% vs. 10.3%; P=0.16) and silent inactivation (16% vs. 5.2%; P=0.18) were seen with calaspargase-pegol versus pegaspargase, respectively. However, 13 patients in the calaspargase-pegol group required conversion to short-acting asparaginase compared with 6 patients in the pegaspargase group (26% vs. 10.3%; P=0.03). Most adverse effects were comparable between the 2 groups, except for a lower rate of hyperbilirubinemia with calaspargase-pegol compared with pegaspargase (34% vs. 58.6%; P=0.01). Overall, no significant differences were found between the formulations in terms of rate of hypersensitivity reactions and silent inactivation. However, calaspargase-pegol did require more conversions to short-acting asparaginase therapy. Additional monitoring and preventative measures should be utilized to prevent calaspargase-pegol-associated adverse effects.

截至2022年12月,calaspargas -pegol是22岁以下急性白血病患者的标准长效天冬酰胺酶制剂。这项为期3年的回顾性评估比较了calaspargas -pegol与pegaspargase在儿科患者中的实际安全性和有效性。收集过敏反应的发生率和严重程度、无症状失活和常见的天冬酰胺酶相关不良反应。共纳入103例患者,其中50例接受calaspargas -pegol治疗,58例接受pegaspargase治疗。calaspargas -pegol与pegaspargase的症状性超敏反应发生率相似(20% vs. 10.3%, P=0.16)和无症状失活(16% vs. 5.2%, P=0.18)。然而,calaspargas -pegol组有13例患者需要转换为短效天冬酰胺酶,而pegaspargase组有6例患者需要转换为短效天冬酰胺酶(26% vs. 10.3%; P=0.03)。除了与pegaspargase相比,calaspargas -pegol组高胆红素血症发生率较低(34% vs. 58.6%; P=0.01),两组间大多数不良反应具有可比性。总体而言,在过敏反应和沉默失活率方面,配方之间没有发现显着差异。然而,calaspargas -pegol确实需要更多的转换到短效天冬酰胺酶治疗。应采用额外的监测和预防措施,以防止与calaspargas - pegols相关的不良反应。
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引用次数: 0
Comparing Iron Prophylaxis Strategies in Infants: Is Sucrosomial Iron a Better Alternative? 比较婴儿铁预防策略:是一个更好的选择吗?
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-20 DOI: 10.1097/MPH.0000000000003111
Sultan Okur Acar, Neryal Tahta

Iron deficiency and iron deficiency anemia (IDA) are prevalent during infancy. Timely iron prophylaxis can prevent associated irreversible complications. As part of the "Iron Like Turkey" program initiated in April 2004, iron prophylaxis is administered from the fourth month of life for at least 5 months. This study aims to evaluate the implementation of iron prophylaxis in our country and compare the effectiveness of different iron preparations used in prophylaxis. Between January 2021 and January 2024, the iron prophylaxis status of 248 healthy, full-term, breastfed infants 9 to 15 months old who were referred to the Pediatric Hematology outpatient clinic of Manisa City Hospital for various reasons was assessed. Iron prophylaxis was administered to 204 (82.3%) infants, of whom 92 (44.1%) had irregular usage. Among those receiving iron prophylaxis, 110 (53.9%) used Fe+3, while 94 (46.1%) used Sucrosomial Iron (SI). No infants received Fe+2 for prophylaxis. Iron deficiency anemia (IDA) was present in 142 (57.3%) cases. No significant difference was found between Fe+3and Sucrosomial Iron in terms of adherence and duration of prophylaxis (P>0.05). However, hemoglobin (Hb), mean corpuscular volume (MCV), ferritin, and transferrin saturation levels were significantly lower in the Sucrosomial Iron group compared with the Fe+3 group (P<0.05). Iron prophylaxis is an easy and effective method to prevent IDA. Both mothers during pregnancy and infants should receive prophylaxis on time and for an adequate duration. Further research is needed to evaluate the efficacy of different iron formulations used in prophylaxis.

缺铁和缺铁性贫血(IDA)在婴儿期很普遍。及时的铁预防可以预防相关的不可逆并发症。作为2004年4月启动的“像铁一样的火鸡”计划的一部分,从出生后第四个月开始进行铁预防,至少持续5个月。本研究旨在评价我国铁预防的实施情况,并比较不同铁制剂用于预防的效果。在2021年1月至2024年1月期间,对248名因各种原因转诊至马尼萨市医院儿科血液学门诊的健康足月母乳喂养9至15个月婴儿的铁预防状况进行了评估。204例(82.3%)婴儿接受铁预防治疗,其中92例(44.1%)使用不规律。在接受铁预防治疗的患者中,110例(53.9%)使用铁+3,94例(46.1%)使用上染色体铁(SI)。没有婴儿接受铁+2预防。缺铁性贫血142例(57.3%)。铁+3和上染色体铁在预防依从性和持续时间方面无显著差异(P < 0.05)。然而,与铁+3组相比,铁组的血红蛋白(Hb)、平均红细胞体积(MCV)、铁蛋白和转铁蛋白饱和度水平显著降低
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引用次数: 0
Iron and Vitamin B12 Levels in School-Age Children. 学龄儿童的铁和维生素B12水平。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-20 DOI: 10.1097/MPH.0000000000003100
Isıl Culha Hosceylan, Tunç Kallemoglu, Mine Basibuyuk, Nalan Karabayir

Objective: Iron and vitamin B12 deficiencies remain widespread among school-age children, contributing to growth and developmental issues. This study aimed to investigate iron and vitamin B12 levels in school-age children.

Materials and methods: A cross-sectional study was conducted at a Family Medicine Unit in Istanbul. Hemogram, serum iron, total iron-binding capacity, ferritin, and vitamin B12 levels of 6 to 18-year-old children under regular follow-up were retrospectively evaluated.

Results: The study included 234 children (115 boys, 119 girls; mean age: 9.9±3.2 y). Anemia prevalence was 9.4%, significantly higher in girls (13.4%) than boys (5.2%). Iron deficiency anemia was detected in 3.4% without significant gender or age variation. Ferritin levels were below 12 ng/mL in 56.3% of cases, with a significant difference between genders. Mean vitamin B12 level was 261.5±162.9 pg/mL (boys: 217.3±173.9; girls: 275.2±151.1). Vitamin B12 deficiency affected 40.6% of children, particularly boys and those age 12 to 18 years of age (P<0.05). No correlation was found between vitamin B12 levels and other parameters except hemoglobin.

Conclusion: While anemia is more common in school-age girls, vitamin B12 deficiency has been found to be more common in adolescent boys. Regular monitoring of anemia, iron and vitamin B12 levels in school-age children is critical for a healthy future.

目的:铁和维生素B12缺乏在学龄儿童中仍然普遍存在,导致生长和发育问题。这项研究旨在调查学龄儿童的铁和维生素B12水平。材料和方法:在伊斯坦布尔的一家家庭医学单位进行了一项横断面研究。回顾性评价6 ~ 18岁儿童定期随访的血象、血清铁、总铁结合力、铁蛋白和维生素B12水平。结果:共纳入234例儿童,其中男孩115例,女孩119例,平均年龄9.9±3.2岁。贫血患病率为9.4%,女孩(13.4%)明显高于男孩(5.2%)。缺铁性贫血3.4%,无明显性别和年龄差异。56.3%的病例铁蛋白水平低于12 ng/mL,性别差异显著。平均维生素B12水平为261.5±162.9 pg/mL(男孩:217.3±173.9;女孩:275.2±151.1)。维生素B12缺乏症影响了40.6%的儿童,特别是男孩和12至18岁的儿童(结论:虽然贫血在学龄女孩中更常见,但维生素B12缺乏症在青春期男孩中更常见。定期监测学龄儿童的贫血、铁和维生素B12水平对健康的未来至关重要。
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引用次数: 0
Palliative Chemotherapy is Well Tolerated in a Patient With Metastatic Colorectal Cancer Due to Bloom Syndrome With a Novel BLM Mutation. 一种新型BLM突变的布卢姆综合征导致的转移性结直肠癌患者姑息化疗耐受良好。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-20 DOI: 10.1097/MPH.0000000000003108
Jacqueline Baca, Katherine Anderson, Thomas Lahiri, Amelia Sybenga, Timothy Higgins, Jessica L Heath

Background: Bloom syndrome is an autosomal recessive condition of genomic instability caused by increased sister-chromatid exchange, which results in a predisposition to a variety of cancers at a young age. The molecular alterations in Bloom Syndrome predisposing to chromosomal instability alter the expected response to and toxicities of chemotherapy in patients with this condition.

Observations: We report a 16-year-old patient with previously undiagnosed Bloom syndrome who presented with metastatic mucinous adenocarcinoma and tolerated palliative chemotherapy, initially with modified FOLFOX and subsequently with FOLFIRI, both at 50% dose reduction, without significant toxicity.

Conclusions: Dose modified multiagent chemotherapy was well tolerated in an adolescent patient with Bloom Syndrome and metastatic colorectal cancer.

背景:Bloom综合征是一种常染色体隐性遗传病,由姐妹染色单体交换增加引起的基因组不稳定性,导致年轻时易患多种癌症。易导致染色体不稳定的Bloom综合征的分子改变改变了患者对化疗的预期反应和毒性。观察:我们报告了一名16岁的未确诊Bloom综合征患者,他表现为转移性粘液腺癌,并耐受姑息性化疗,最初使用改良FOLFOX,随后使用FOLFIRI,剂量均减少50%,无明显毒性。结论:一名青少年布卢姆综合征合并转移性结直肠癌患者的剂量调整多药化疗耐受良好。
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引用次数: 0
The Top 50 Most-Cited Studies of Extended Half-Life Factors in Hemophilia. 血友病延长半衰期因子被引用最多的前50项研究。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-20 DOI: 10.1097/MPH.0000000000003101
Ayşe Şimşek

Objective: In recent years, one of the most significant advancements in hemophilia treatment has been the introduction of extended half-life (EHL) factors. This study aims to identify the 50 most influential articles on EHL factor use in hemophilia treatment and analyze their key characteristics.

Methods: A comprehensive literature search was conducted without restrictions on publication date, country, or language using the Boolean operator "OR." A total of 665 documents published between 2005 and 2024 were identified from the Web of Science (WOS) Core Collection as of December 3, 2024. Articles were ranked in descending order of citation count.

Results: The 50 most-cited articles were published between 2008 and 2020, with the most productive years being 2016 and 2019, contributing 25 articles. These studies accumulated 4130 citations, averaging 82.6 citations per article. The most-cited paper, published in 2014, received 388 citations, and had the highest citation density (35.27 citations per year). The 50 articles encompassed 6 study types: reviews, phase II to III clinical trials, experimental/ex vivo studies, prospective and retrospective observational studies, and questionnaire-based studies. Review articles were the most prevalent (22 articles), while the most-cited study was a phase II to III clinical trial. No systematic reviews, meta-analyses, or randomized controlled trials were among the top-cited papers.

Conclusion: The literature on EHL factors in hemophilia has expanded over the past 2 decades, with notable growth between 2016 and 2019. Haemophilia was the most prominent journal in this field.

目的:近年来,血友病治疗最显著的进步之一是延长半衰期(EHL)因子的引入。本研究旨在找出50篇最具影响力的EHL因子在血友病治疗中的应用,并分析其主要特征。方法:使用布尔运算符“or”进行全面的文献检索,不受出版日期、国家或语言的限制。截至2024年12月3日,从Web of Science (WOS)核心馆藏中共鉴定出2005年至2024年间发表的665篇文献。文章按引用次数降序排列。结果:被引次数最多的50篇文章发表在2008年至2020年之间,其中被引次数最多的是2016年和2019年,贡献了25篇文章。这些研究累计被引用4130次,平均每篇文章被引用82.6次。被引次数最多的论文发表于2014年,被引次数为388次/年,被引密度最高,为35.27次/年。这50篇文章包括6种研究类型:综述、II至III期临床试验、实验/离体研究、前瞻性和回顾性观察性研究以及基于问卷的研究。评论文章是最普遍的(22篇),而被引用最多的研究是一项II至III期临床试验。被引最多的论文中没有系统评价、荟萃分析或随机对照试验。结论:关于血友病EHL因子的文献在过去20年中不断扩大,2016年至2019年期间增长显著。血友病是这一领域最著名的期刊。
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引用次数: 0
Standardized Neurologic Assessment Documentation for Daily Nelarabine Administration: A Bedside Questionnaire. 每日给药奈拉滨的标准化神经系统评估文件:床边问卷。
IF 0.8 4区 医学 Q4 HEMATOLOGY Pub Date : 2025-08-14 DOI: 10.1097/MPH.0000000000003109
Andrew Wahba, Anil George, Eric S Schafer, Alexandra Stevens, Mary C Shapiro

Nelarabine is an essential component in the treatment of pediatric T-cell lymphoblastic malignancies. Rare high-grade neurologic toxicities have been reported, but nelarabine discontinuation when early symptoms arise mitigates poor outcomes. Currently, no standard assessment of neurotoxicity exists. Our patient safety initiative implemented a novel 7-question neurologic assessment tool performed daily during nelarabine courses, which prompted early identification of neurotoxicity and allowed for appropriate interventions. Investigating 31 patients over 18 months demonstrated improved documentation from 65.9% to 97.2% (Δ+31.3%). Nine doses were omitted due to discovered neurotoxicity. Tool implementation advanced our ability to optimize patient care during nelarabine administration.

奈拉拉滨是治疗儿童t淋巴细胞恶性肿瘤的重要成分。罕见的高度神经毒性已被报道,但早期症状出现时停用奈拉宾可减轻不良后果。目前,尚无标准的神经毒性评估方法。我们的患者安全倡议实施了一种新颖的7个问题的神经系统评估工具,每天在奈拉滨疗程中进行,这有助于早期识别神经毒性并允许适当的干预。对31例患者18个月的调查显示,病历记录从65.9%提高到97.2% (Δ+31.3%)。由于发现神经毒性,9剂被省略。工具的实施提高了我们在奈拉滨给药期间优化患者护理的能力。
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Journal of Pediatric Hematology/Oncology
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