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The efficacy of Allium ampeloprasum L. in reducing neutrophil recovery time in childhood cancer with febrile neutropenia: a randomized, double-blind, placebo-controlled trial. 一项随机、双盲、安慰剂对照试验:薤白对减少儿童癌症伴发热性中性粒细胞减少的中性粒细胞恢复时间的疗效。
Pub Date : 2023-10-15 eCollection Date: 2023-01-01
Mohammadreza Bordbar, Mehdi Vahidifar, Mohammad Mehdi Zarshenas, Sezaneh Haghpanah, Soheila Zareifar, Mahdi Shahriari, Omid Reza Zekavat, Mehran Karimi, Gholamreza Fathpour, Arman Zargaran, Nader Shakibazad

Introduction: Febrile neutropenia is a serious complication of cancer chemotherapy that can result in delays in treatment. This study evaluates the efficacy of A. ampeloprasum L. at neutrophil recovery time in children with chemotherapy-associated febrile neutropenia.

Methods: This single-center, parallel-group, double-blind, randomized clinical trial was conducted at an oncology hospital. Patients selected among childhood cancers with febrile neutropenia. Overall, 97 febrile neutropenic children were enrolled. The intervention group (n=49) was given A. ampeloprasum L. in capsules (500 mg twice daily) for seven days plus supportive care. The control group (n=48) was treated similarly with supportive care and placebo capsules. Total white blood cell (WBC) and absolute neutrophil counts (ANC) were checked daily and neutrophil recovery time in both groups was compared.

Results: Patients in the intervention group experienced shorter neutrophil recovery compared to the control group (4.02 ± 2.32 days vs. 6.38 ± 2.80 days, respectively, P less than 0.001). The intervention group was discharged from the hospital earlier than the control group with a mean of two days, but it did not reach statistical significance (P=0.133). Mean WBC and ANC were not significantly different in the two groups. Herbal medicine was well tolerated, and no adverse effect was reported.

Conclusions: A fresh, lyophilized extract from deciduous leaves of A. ampeloprasum L. can effectively shorten the ANC recovery time leading to an earlier release from the hospital. The trial was registered in the Iranian Registry of Clinical Trials with registration No. IRCT2015051615666N2 (http://www.irct.ir/).

发热性中性粒细胞减少症是癌症化疗的严重并发症,可导致治疗延误。本研究评价黑藤对化疗相关发热性中性粒细胞减少症患儿中性粒细胞恢复时间的影响。方法:在某肿瘤医院进行单中心、平行组、双盲、随机临床试验。儿童癌症伴发热性中性粒细胞减少症患者。共纳入97例发热性中性粒细胞减少症患儿。干预组(n=49)给予蛇耳草胶囊(500 mg,每日2次),连用7天,并给予支持治疗。对照组(n=48)同样给予支持治疗和安慰剂胶囊。每天检测总白细胞(WBC)和绝对中性粒细胞计数(ANC),比较两组中性粒细胞恢复时间。结果:干预组患者中性粒细胞恢复时间较对照组短(分别为4.02±2.32天∶6.38±2.80天,P < 0.001)。干预组比对照组平均提前2天出院,但差异无统计学意义(P=0.133)。两组患者平均WBC、ANC差异无统计学意义。草药耐受性良好,无不良反应报道。结论:新鲜、冻干的蛇梨落叶提取物能有效缩短ANC的恢复时间,提前出院。该试验已在伊朗临床试验登记处注册,注册号为:IRCT2015051615666N2 (http://www.irct.ir/)。
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引用次数: 0
Intracranial disease in pediatric Hodgkin lymphoma-case report and review of literature. 儿童霍奇金淋巴瘤颅内病变病例报告及文献复习。
Pub Date : 2023-10-15 eCollection Date: 2023-01-01
Nidhi Dhariwal, Nirmalya Roy Moulik, Vasudeva Bhat, Vasundhara Smriti, Sangeeta Kakoti, Sayak Choudhury, Epari Sridhar, Sumeet Gujral, Chetan Dhamne, Sneha Shah, Gaurav Narula, Shripad Banavali

Central nervous system (CNS) involvement in Hodgkin lymphoma (HL) is an extremely rare presentation with dismal outcomes according to reported literature. An 8-year-old girl presented to us with complaints of on-off fever, right cervical swelling and bilateral ptosis. Positron emission tomography (PET) showed intracranial extra-axial soft tissue masses in right infero-lateral temporal lobe, sella and bilateral parasellar region along with cervical, mediastinal, axillary, abdominal and inguino-pelvic nodes, liver lesions and extensive marrow lesions involving the axial and appendicular skeleton. Histopathology of the cervical lymph node revealed a diagnosis of classical Hodgkin lymphoma. Child received 2 cycles of OEPA and 4 cycles of COPP followed by radiotherapy to bulky cervical lymph nodes and intracranial lesion. The child has been disease-free for 44 months with no neurological sequalae. Intracranial spread is rare in Hodgkin lymphoma and is associated with inferior outcomes. Due to its rarity, there are no specific treatment guidelines for this entity. The choice of ideal chemotherapeutic agents and role of whole-brain radiotherapy needs further evaluation.

根据文献报道,中枢神经系统(CNS)累及霍奇金淋巴瘤(HL)是一种极其罕见的表现,其预后令人沮丧。一名8岁女孩向我们提出的投诉,时断时续发烧,右颈椎肿胀和双侧上睑下垂。正电子发射断层扫描(PET)显示,颅内轴外软组织肿块位于右侧颞叶下外侧、鞍座和双侧鞍旁区,伴颈椎、纵隔、腋窝、腹部和腹股沟-骨盆淋巴结,肝脏病变和广泛的骨髓病变累及轴向和阑尾骨骼。颈部淋巴结组织病理学诊断为经典霍奇金淋巴瘤。患儿接受2个周期的OEPA和4个周期的COPP治疗,并对颈部肿大淋巴结和颅内病变进行放疗。这名儿童已无病44个月,无神经系统后遗症。颅内扩散在霍奇金淋巴瘤中是罕见的,并且与较差的预后相关。由于它的罕见性,没有针对这种实体的具体治疗指南。理想化疗药物的选择及全脑放疗的作用有待进一步探讨。
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引用次数: 0
A randomized controlled trial to explore the safety and efficacy of irradiated buffy-coat granulocytes in pediatric patients with febrile neutropenia. 一项随机对照试验,探讨辐照白大衣粒细胞治疗小儿发热性中性粒细胞减少症的安全性和有效性。
Pub Date : 2023-10-15 eCollection Date: 2023-01-01
Mohanaraj Ramachandran, Aditya Kumar Gupta, Jagdish Prasad Meena, Ashish Datt Upadhyay, Poonam Coshic, Rakesh Lodha, Rachna Seth

Background: Transfusion of granulocytes obtained by apheresis is beneficial in febrile neutropenia (FN) but expensive and time-consuming. Buffy-coat-derived granulocytes could be an alternative. We studied the efficacy and safety of the administration of irradiated buffy-coat-derived granulocytes along with the standard of care in pediatric high-risk (HR) FN.

Methods: Sixty children ≤18 years with malignancy and chemotherapy-induced HR FN were randomized to either the granulocyte transfusion (GT) arm which received irradiated buffy-coat derived granulocyte transfusion along with the standard treatment or the standard treatment (ST) arm.

Results: Baseline characteristics, day-to-defervescence, antibiotic duration, hospital stay, and mortality were comparable between the groups. A significant difference was seen in days to achieve absolute neutrophil count (ANC) >500/mm3 in the 2 groups: 4.5 days (3-6.5) in the GT arm v/s 8 days (4-11) in the ST arm (P=0.01).

Conclusion: Buffy-coat-derived granulocyte transfusion was safe and led to early hematological recovery but was not associated with survival benefits. Future studies with earlier initiation in the intended dose could be undertaken to generate more evidence.

背景:输血采血获得的粒细胞对发热性中性粒细胞减少症(FN)是有益的,但昂贵且耗时。灰褐色的粒细胞可能是另一种选择。我们研究了在儿童高危FN (HR)治疗标准下,使用辐照的灰褐色粒细胞的疗效和安全性。方法:60例≤18岁的恶性肿瘤伴化疗引起的HR FN患儿,随机分为粒细胞输注组(GT)和标准治疗组(ST)。GT组接受辐照的白皮源性粒细胞输注,并给予标准治疗。结果:两组之间的基线特征、日至热、抗生素持续时间、住院时间和死亡率具有可比性。两组达到绝对中性粒细胞计数(ANC) >500/mm3的天数有显著差异:GT组为4.5天(3-6.5),ST组为8天(4-11)(P=0.01)。结论:buffy -coat来源的粒细胞输注是安全的,可导致早期血液学恢复,但与生存益处无关。未来的研究可以更早地开始预期剂量,以产生更多的证据。
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引用次数: 0
Therapeutic benefits of prophetic medicine remedies in treating hematological diseases (A review article). 预言性药物治疗血液病的疗效(综述文章)。
Pub Date : 2023-08-15 eCollection Date: 2023-01-01
Ghazi H Mogharbel, Ahmad S Badawi, Amal Yaseen Zaman, Mehrevan M Abd Elmoniem, Ibrahim M Abdel-Rahman, Mariam Eid Alenazi, Fareed Akbar Shah, Mohamed Abdelnaem Aly, Syed Nazar Imam, Nusaybah Eid Alenazi, Salah Mohamed El Sayed

Hematological disorders are common medical ailments constituting an important cause of morbidity and mortality worldwide, which may be managed efficiently using different prophetic medicine remedies as adjuvants to current therapeutics. Prophetic medicine includes the body of knowledge about medicine that has been derived from the deeds, customs (sunnah), ahadith (sayings), actions, and agreements of Prophet Muhammad, peace be upon him. This review article aims at exploring the magnitude of therapeutic benefits of prophetic medicine remedies as adjuvant treatments to many different types of hematological disorders. Herein, we reviewed many published research studies throughout the literature to delineate the potential therapeutic benefits of prophetic remedies on hematological disorders. Several types of hematological disorders may benefit from prophetic medicine remedies that are rich in natural antioxidants that combat oxidative stress-induced harm e.g. nigella sativa, oral honey, camel milk and urine, Ajwa date fruits, olive oil, Zamzam water and figs. Many prophetic medicine remedies were reported to decrease the hematological cytotoxicity effects induced by different chemicals and are beneficial in treating anemias e.g. iron deficiency anemia, sickle cell anemia, thalassemia, coagulopathies and hematological malignancies as leukemia and myeloma. These remedies treat or alleviate the different hematological disorders using different mechanisms e.g. modulating the immune function, treating deficiencies of different substances, protecting against toxins-induced cytotoxicity, decreasing platelets aggregation, suppressing clotting factors activation, exerting antineoplastic effects (enhancing cancer cells cytotoxicity) and inhibiting angiogenesis. Prophetic medicine remedies exert clinically significant therapeutic benefits for treating COVID-19 pandemic, anemia, thrombosis, thalassemia and blood cancers without inducing toxicity or side effects.

血液病是常见的医学疾病,是全球发病率和死亡率的重要原因,使用不同的预言性药物作为当前治疗方法的佐剂可以有效地治疗。预言医学包括从先知穆罕默德的行为、习俗(sunnah)、格言(ahadith)、行动和协议中获得的关于医学的知识体系,愿他平安。这篇综述文章旨在探索预言性药物作为多种不同类型血液病的辅助治疗的治疗益处。在此,我们回顾了文献中许多已发表的研究,以描述预言疗法对血液病的潜在治疗益处。几种类型的血液病可能受益于富含天然抗氧化剂的预言性药物治疗,这些抗氧化剂可以对抗氧化应激引起的伤害,如黑穗藻、口服蜂蜜、骆驼奶和尿液、Ajwa枣果、橄榄油、Zamzam水和无花果。据报道,许多预言性药物可以降低不同化学物质诱导的血液细胞毒性作用,并有益于治疗贫血,如缺铁性贫血、镰状细胞性贫血、地中海贫血、凝血障碍和血液系统恶性肿瘤,如白血病和骨髓瘤。这些药物使用不同的机制治疗或减轻不同的血液系统疾病,例如调节免疫功能、治疗不同物质的缺陷、保护免受毒素诱导的细胞毒性、减少血小板聚集、抑制凝血因子激活、,发挥抗肿瘤作用(增强癌症细胞的细胞毒性)和抑制血管生成。预防性药物在治疗新冠肺炎大流行、贫血、血栓形成、地中海贫血和血癌方面发挥了临床显著的治疗作用,而不会产生毒性或副作用。
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引用次数: 0
Hematological parameters and their predictive value for assessing disease severity in laboratory-confirmed COVID-19 patients: a retrospective study. 血液学参数及其对实验室确诊新冠肺炎患者疾病严重程度评估的预测价值:一项回顾性研究。
Pub Date : 2023-08-15 eCollection Date: 2023-01-01
Mezgebu Alemayehu Awoke, Ayinshet Adane, Belete Assefa, Solomon Getawa, Gebrehiwot Lema Legese, Mekonen Yimer

Background: The coronavirus disease 19 (COVID-19) infection has spread globally and caused a substantial amount of mortality and morbidity. Early detection of severe infections will improve care and reduce deaths. The use of hematological parameters in predicting COVID-19 disease severity, patient outcomes, and early risk stratification is limited. Therefore, the study was aimed at determining hematological parameters and their predictive value for assessing disease severity in laboratory-confirmed COVID-19 patients in Northwest Ethiopia.

Methods: A retrospective cross-sectional study was conducted at the University of Gondar comprehensive specialized hospital and Tibebe Ghion comprehensive specialized referral hospital on 253 patients diagnosed with COVID-19 and admitted between March 2021 and February 2022. Data were extracted, and entered into Epi-data 4.2.0.0, and analyzed using SPSS version 25 software. Hematological parameters were provided as the median and interquartile range (IQR). Categorical variables were represented by their frequency, and the χ2 test was applied to compare observed results with expected results. The receiver-operating curve (ROC) was used to establish the predictive value of hematological parameters for COVID-19 severity. A p-value < 0.05 was considered statistically significant.

Results: On a total of 253 patients, there were 43.87% severe cases, with a mortality rate of 26.9%. The ROC analysis showed the optimal cutoff values for hematological parameters were ANC (3370), lymphocyte (680), NLR (9.34), PLR (290.77), platelets (332,000), and WBCs (4390.65). The area under the curve (AUC) values for NLR (0.679) and ANC (0.631) were high, with the highest sensitivity and specificity, and could potentially be used to predict COVID-19 severity.

Conclusion: This study proved that high NLR and high ANC have prognostic value for assessing disease severity in COVID-19. Thus, assessing and considering these hematological parameters when triaging COVID-19 patients may prevent complications and improve the patient's outcome.

背景:冠状病毒疾病19(新冠肺炎)感染已在全球范围内传播,并导致大量死亡和发病。早期发现严重感染将改善护理并减少死亡。血液学参数在预测新冠肺炎疾病严重程度、患者结局和早期风险分层方面的应用有限。因此该研究旨在确定埃塞俄比亚西北部实验室确诊的新冠肺炎患者的血液学参数及其对评估疾病严重程度的预测价值新冠肺炎,于2021年3月至2022年2月入院。提取数据,输入Epi数据4.2.0.0,并使用SPSS 25版软件进行分析。血液学参数以中位数和四分位间距(IQR)的形式提供。分类变量用其频率表示,并应用χ2检验将观察结果与预期结果进行比较。受试者工作曲线(ROC)用于确定血液学参数对新冠肺炎严重程度的预测值。p值<0.05被认为具有统计学意义。结果:在总共253名患者中,有43.87%的重症病例,死亡率为26.9%。ROC分析显示,血液学参数的最佳截止值为ANC(3370)、淋巴细胞(680)、NLR(9.34)、PLR(290.77)、血小板(332000)和WBCs(4390.65)。NLR(0.679)和ANC(0.631)的曲线下面积(AUC)值较高,具有最高的敏感性和特异性,并有可能用于预测新冠肺炎的严重程度。结论:本研究证明高NLR和高ANC对评估新冠肺炎疾病严重程度具有预后价值。因此,在对新冠肺炎患者进行试验时,评估和考虑这些血液学参数可以预防并发症并改善患者的预后。
{"title":"Hematological parameters and their predictive value for assessing disease severity in laboratory-confirmed COVID-19 patients: a retrospective study.","authors":"Mezgebu Alemayehu Awoke, Ayinshet Adane, Belete Assefa, Solomon Getawa, Gebrehiwot Lema Legese, Mekonen Yimer","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>The coronavirus disease 19 (COVID-19) infection has spread globally and caused a substantial amount of mortality and morbidity. Early detection of severe infections will improve care and reduce deaths. The use of hematological parameters in predicting COVID-19 disease severity, patient outcomes, and early risk stratification is limited. Therefore, the study was aimed at determining hematological parameters and their predictive value for assessing disease severity in laboratory-confirmed COVID-19 patients in Northwest Ethiopia.</p><p><strong>Methods: </strong>A retrospective cross-sectional study was conducted at the University of Gondar comprehensive specialized hospital and Tibebe Ghion comprehensive specialized referral hospital on 253 patients diagnosed with COVID-19 and admitted between March 2021 and February 2022. Data were extracted, and entered into Epi-data 4.2.0.0, and analyzed using SPSS version 25 software. Hematological parameters were provided as the median and interquartile range (IQR). Categorical variables were represented by their frequency, and the χ<sup>2</sup> test was applied to compare observed results with expected results. The receiver-operating curve (ROC) was used to establish the predictive value of hematological parameters for COVID-19 severity. A <i>p</i>-value < 0.05 was considered statistically significant.</p><p><strong>Results: </strong>On a total of 253 patients, there were 43.87% severe cases, with a mortality rate of 26.9%. The ROC analysis showed the optimal cutoff values for hematological parameters were ANC (3370), lymphocyte (680), NLR (9.34), PLR (290.77), platelets (332,000), and WBCs (4390.65). The area under the curve (AUC) values for NLR (0.679) and ANC (0.631) were high, with the highest sensitivity and specificity, and could potentially be used to predict COVID-19 severity.</p><p><strong>Conclusion: </strong>This study proved that high NLR and high ANC have prognostic value for assessing disease severity in COVID-19. Thus, assessing and considering these hematological parameters when triaging COVID-19 patients may prevent complications and improve the patient's outcome.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"13 4","pages":"117-129"},"PeriodicalIF":0.0,"publicationDate":"2023-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10509465/pdf/ajbr0013-0117.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41097508","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Coagulopathy in the absence of overt DIC in postoperative neurosurgical patients is a strong predictor of poor outcome. 神经外科术后患者在没有明显DIC的情况下出现凝血障碍是预后不良的有力预测因素。
Pub Date : 2023-08-15 eCollection Date: 2023-01-01
Pooja Sharma, Richa Gupta, Satendra Sharma, Mrinalini Kotru, Gurubachan Singh, Meera Sikka

Objectives: To assess the prevalence of coagulopathy in postoperative neurosurgical patients and correlate it with the outcome.

Materials and method: This longitudinal study was conducted in a tertiary care hospital in the Department of Pathology and Neurosurgery. Ethical approval was taken from the Institutional Ethical Committee - Human Research. Seventy-two (72) participants were recruited within 48 hours of surgery after obtaining consent. Complete clinical and surgical details were recorded. A 6.5 mL venous sample was collected and dispensed in two separate vials. The EDTA sample was run within 2 hours of collection on an automated hematology analyzer to obtain complete blood counts, including platelet count. The citrated sample was run on a fully automated coagulometer to determine PT, APTT, plasma fibrinogen, FVIII assay, and D-dimer levels. Subjects with a DIC-ISTH score of 5 or more were excluded. Coagulopathy was defined as three or more coagulation parameters deranged in a patient. All patients were followed up for the outcome. The outcome was correlated with coagulopathy, and a p-value less than 0.05 was considered statistically significant.

Results: The study found that the number of hemostatic parameters deranged correlated with outcome (P < 0.001). The proportion of patients with coagulopathy was 32/72 (44.4%), while those without coagulopathy were 40/72 (55.6%). Of patients with coagulopathy, 87.5% (28/32) had an adverse outcome, while 12.5% (4/32) had a favorable outcome. The difference was found to be statistically significant (P < 0.001).

Conclusions: Coagulopathy, defined as the derangement of three or more parameters, is a predictor of poor outcomes in postoperative neurosurgical patients. This timely recognition of coagulopathy can help triage patients requiring appropriate blood products, significantly reducing morbidity and mortality associated with postoperative neurosurgical patients.

目的:评估神经外科术后患者凝血障碍的患病率,并将其与预后联系起来。材料和方法:这项纵向研究在病理和神经外科的一家三级护理医院进行。伦理批准来自人类研究机构伦理委员会。七十二(72)名参与者在获得同意后的48小时内被招募。记录完整的临床和手术细节。采集6.5 mL静脉样本,并将其分装在两个单独的小瓶中。EDTA样品在自动血液分析仪上采集后2小时内运行,以获得完整的血液计数,包括血小板计数。柠檬酸盐样品在全自动凝血仪上运行,以测定PT、APTT、血浆纤维蛋白原、FVIII测定和D-二聚体水平。DIC-ISTH评分为5分或以上的受试者被排除在外。凝血障碍被定义为患者的三个或三个以上凝血参数紊乱。对所有患者进行了随访。结果与凝血障碍相关,p值小于0.05被认为具有统计学意义。结果:止血参数紊乱的次数与结果相关(P<0.001)。有凝血障碍的患者比例为32/72(44.4%),无凝血障碍的为40/72(55.6%)。在凝血障碍患者中,87.5%(28/32)有不良结果,12.5%(4/32)有良好结果。结论:凝血障碍,定义为三个或三个以上参数的紊乱,是神经外科术后患者不良预后的预测因素。这种对凝血障碍的及时识别可以帮助对需要适当血液制品的患者进行分类,显著降低神经外科术后患者的发病率和死亡率。
{"title":"Coagulopathy in the absence of overt DIC in postoperative neurosurgical patients is a strong predictor of poor outcome.","authors":"Pooja Sharma,&nbsp;Richa Gupta,&nbsp;Satendra Sharma,&nbsp;Mrinalini Kotru,&nbsp;Gurubachan Singh,&nbsp;Meera Sikka","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Objectives: </strong>To assess the prevalence of coagulopathy in postoperative neurosurgical patients and correlate it with the outcome.</p><p><strong>Materials and method: </strong>This longitudinal study was conducted in a tertiary care hospital in the Department of Pathology and Neurosurgery. Ethical approval was taken from the Institutional Ethical Committee - Human Research. Seventy-two (72) participants were recruited within 48 hours of surgery after obtaining consent. Complete clinical and surgical details were recorded. A 6.5 mL venous sample was collected and dispensed in two separate vials. The EDTA sample was run within 2 hours of collection on an automated hematology analyzer to obtain complete blood counts, including platelet count. The citrated sample was run on a fully automated coagulometer to determine PT, APTT, plasma fibrinogen, FVIII assay, and D-dimer levels. Subjects with a DIC-ISTH score of 5 or more were excluded. Coagulopathy was defined as three or more coagulation parameters deranged in a patient. All patients were followed up for the outcome. The outcome was correlated with coagulopathy, and a <i>p</i>-value less than 0.05 was considered statistically significant.</p><p><strong>Results: </strong>The study found that the number of hemostatic parameters deranged correlated with outcome (P < 0.001). The proportion of patients with coagulopathy was 32/72 (44.4%), while those without coagulopathy were 40/72 (55.6%). Of patients with coagulopathy, 87.5% (28/32) had an adverse outcome, while 12.5% (4/32) had a favorable outcome. The difference was found to be statistically significant (P < 0.001).</p><p><strong>Conclusions: </strong>Coagulopathy, defined as the derangement of three or more parameters, is a predictor of poor outcomes in postoperative neurosurgical patients. This timely recognition of coagulopathy can help triage patients requiring appropriate blood products, significantly reducing morbidity and mortality associated with postoperative neurosurgical patients.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"13 4","pages":"110-116"},"PeriodicalIF":0.0,"publicationDate":"2023-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10509467/pdf/ajbr0013-0110.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41096125","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment of sickle cell disease - options and perspective. 镰状细胞病的治疗--选择与展望。
Pub Date : 2023-04-15 eCollection Date: 2023-01-01
Loubna Abdel-Hadi, Yendry Ventura Carmenate, Yandy Marx Castillo-Aleman, Samira Sheikh, Aya Zakaria, John Phillips

Sickle Cell Disease (SCD) is one of the most inherited hematologic diseases affecting humans. Clinically, there is a progressive multiorgan failure and increased mortality in severe cases. The highest prevalence is in West Africa, India, the Mediterranean region, and Middle East countries. Hydroxyurea was the primary drug available for SCD and remains first-line therapy for patients with SCD. Three additional drug therapies, L-glutamine, Voxelotor, and Crizanlizumab, have been approved as adjunctive agents. However, none of these treatments are curative. Effective cell-based therapies are available, such as red blood cell (RBC) exchange and the only curative therapy is hematopoietic stem cell transplantation (HSCT). Gene-editing now shows promise in treating SCD and the β-thalassemias. Recent clinical trials have proven that this therapeutic strategy is effective, however costly. Despite the availability of safe and effective drug treatments, questions focusing on the overall value of these drugs exist in light of rising healthcare costs including hospitalizations and medical interventions. Herein, we report a cost-effective evaluation that can guide future efforts in making decisions towards HSCT as cell therapy treatment in SCD patients.

镰状细胞病(SCD)是影响人类的遗传性血液病之一。在临床上,SCD 会导致进行性多器官功能衰竭,严重病例的死亡率会升高。西非、印度、地中海地区和中东国家发病率最高。羟基脲是治疗 SCD 的主要药物,目前仍是 SCD 患者的一线治疗药物。另外三种药物疗法,即 L-谷氨酰胺、Voxelotor 和 Crizanlizumab,已被批准作为辅助药物。然而,这些疗法都不能治愈疾病。目前已有有效的细胞疗法,如红细胞(RBC)交换,而唯一能治愈的疗法是造血干细胞移植(HSCT)。目前,基因编辑技术在治疗 SCD 和 β-地中海贫血症方面大有可为。最近的临床试验证明,这种治疗策略是有效的,但成本高昂。尽管有了安全有效的药物治疗方法,但鉴于住院和医疗干预等医疗费用不断上涨,人们对这些药物的总体价值仍存在疑问。在此,我们报告了一项成本效益评估,该评估可指导未来将造血干细胞移植作为细胞疗法治疗 SCD 患者的决策。
{"title":"Treatment of sickle cell disease - options and perspective.","authors":"Loubna Abdel-Hadi, Yendry Ventura Carmenate, Yandy Marx Castillo-Aleman, Samira Sheikh, Aya Zakaria, John Phillips","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Sickle Cell Disease (SCD) is one of the most inherited hematologic diseases affecting humans. Clinically, there is a progressive multiorgan failure and increased mortality in severe cases. The highest prevalence is in West Africa, India, the Mediterranean region, and Middle East countries. Hydroxyurea was the primary drug available for SCD and remains first-line therapy for patients with SCD. Three additional drug therapies, L-glutamine, Voxelotor, and Crizanlizumab, have been approved as adjunctive agents. However, none of these treatments are curative. Effective cell-based therapies are available, such as red blood cell (RBC) exchange and the only curative therapy is hematopoietic stem cell transplantation (HSCT). Gene-editing now shows promise in treating SCD and the β-thalassemias. Recent clinical trials have proven that this therapeutic strategy is effective, however costly. Despite the availability of safe and effective drug treatments, questions focusing on the overall value of these drugs exist in light of rising healthcare costs including hospitalizations and medical interventions. Herein, we report a cost-effective evaluation that can guide future efforts in making decisions towards HSCT as cell therapy treatment in SCD patients.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"13 2","pages":"61-70"},"PeriodicalIF":0.0,"publicationDate":"2023-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10195315/pdf/ajbr0013-0061.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9858420","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Prognostic relevance of NPM1, CEBPA, and FLT3 mutations in cytogenetically normal adult AML patients. 细胞遗传学正常的成年AML患者NPM1、CEBPA和FLT3突变与预后的相关性
Pub Date : 2023-02-15 eCollection Date: 2023-01-01
Aparna Ningombam, Deepak Verma, Rajive Kumar, Jay Singh, M Shadab Ali, Avanish Kumar Pandey, Inder Singh, Sameer Bakhshi, Atul Sharma, Deepam Pushpam, Jayanth Kumar Palanichamy, Pranay Tanwar, Amar Ranjan Singh, Anita Chopra

Background: Acute myeloid leukemia with normal cytogenetics (CN-AML) is the largest group of AML patients with very heterogenous patient outcomes. The revised World Health Organization classification of the hematolymphoid tumours, 2022, has incorporated AML with Nucleophosphmin1 (NPM1) and CCAAT/enhancer binding protein-alpha (CEBPA) mutations as distinct entities. Despite the existing evidence of the prognostic relevance of FMS-like tyrosine kinase-3 internal tandem duplication (FLT3-ITD) in AML, it has not been included in the revised classification.

Method: In this prospective study, we determined the prevalence of NPM1, CEBPA, and FLT3 gene mutations in 151 de novo CN-AML adult patients (age ≥18 years) in a tertiary care hospital in north India. Additionally, the prognostic relevance of these mutations was also evaluated.

Results: NPM1, FLT3-ITD, and CEBPA mutations were found in 33.11%, 23.84%, and 15.77% of CN-AML patients, respectively. CEBPA mutations were found at 3 domains: transactivation domain 1 (TAD1) in 10 (6.62%), transactivation domain 2 (TAD2) in 5 (3.31%), and basic leucine zipper domain (bZIP) in 11 (7.82%) patients. Patients with NPM1 mutation had better clinical remission rate (CR) (P=0.003), event-free survival (P=0.0014), and overall survival (OS) (P=0.0017). However, FLT3-ITD and CEBPA mutations did not show any association with CR (P=0.404 and 0.92, respectively). Biallelic CEBPA mutations were found in 12 (7.95%) patients and were associated with better OS (P=0.043).

Conclusions: These findings indicate that NPM1 and CEBPA mutations can be precisely used for risk stratification in CN-AML patients.

背景:细胞遗传学正常的急性髓系白血病(CN-AML)是最大的AML患者群体,患者预后非常异质性。修订后的世界卫生组织(World Health Organization) 2022年血淋巴肿瘤分类将AML与核磷脂1 (NPM1)和CCAAT/增强子结合蛋白α (CEBPA)突变合并为不同的实体。尽管已有证据表明fms样酪氨酸激酶-3内部串联重复(FLT3-ITD)与AML的预后相关,但尚未将其纳入修订后的分类。方法:在这项前瞻性研究中,我们测定了印度北部一家三级医院151例新生CN-AML成年患者(年龄≥18岁)中NPM1、CEBPA和FLT3基因突变的患病率。此外,还评估了这些突变的预后相关性。结果:NPM1、FLT3-ITD和CEBPA分别在33.11%、23.84%和15.77%的CN-AML患者中存在突变。CEBPA在3个结构域发生突变:反活化结构域1 (TAD1) 10例(6.62%),反活化结构域2 (TAD2) 5例(3.31%),碱性亮氨酸拉链结构域(bZIP) 11例(7.82%)。NPM1突变患者具有更好的临床缓解率(CR) (P=0.003)、无事件生存期(P=0.0014)和总生存期(OS) (P=0.0017)。然而,FLT3-ITD和CEBPA突变与CR没有任何关联(P分别为0.404和0.92)。在12例(7.95%)患者中发现双等位基因CEBPA突变,并与较好的OS相关(P=0.043)。结论:这些发现表明NPM1和CEBPA突变可以精确地用于CN-AML患者的风险分层。
{"title":"Prognostic relevance of <i>NPM1, CEBPA,</i> and <i>FLT3</i> mutations in cytogenetically normal adult AML patients.","authors":"Aparna Ningombam, Deepak Verma, Rajive Kumar, Jay Singh, M Shadab Ali, Avanish Kumar Pandey, Inder Singh, Sameer Bakhshi, Atul Sharma, Deepam Pushpam, Jayanth Kumar Palanichamy, Pranay Tanwar, Amar Ranjan Singh, Anita Chopra","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Acute myeloid leukemia with normal cytogenetics (CN-AML) is the largest group of AML patients with very heterogenous patient outcomes. The revised World Health Organization classification of the hematolymphoid tumours, 2022, has incorporated AML with Nucleophosphmin1 (<i>NPM1</i>) and CCAAT/enhancer binding protein-alpha (<i>CEBPA</i>) mutations as distinct entities. Despite the existing evidence of the prognostic relevance of FMS-like tyrosine kinase-3 internal tandem duplication (<i>FLT3-ITD</i>) in AML, it has not been included in the revised classification.</p><p><strong>Method: </strong>In this prospective study, we determined the prevalence of <i>NPM1, CEBPA,</i> and <i>FLT3</i> gene mutations in 151 de novo CN-AML adult patients (age ≥18 years) in a tertiary care hospital in north India. Additionally, the prognostic relevance of these mutations was also evaluated.</p><p><strong>Results: </strong><i>NPM1, FLT3-ITD,</i> and <i>CEBPA</i> mutations were found in 33.11%, 23.84%, and 15.77% of CN-AML patients, respectively. <i>CEBPA</i> mutations were found at 3 domains: transactivation domain 1 (TAD1) in 10 (6.62%), transactivation domain 2 (TAD2) in 5 (3.31%), and basic leucine zipper domain (bZIP) in 11 (7.82%) patients. Patients with <i>NPM1</i> mutation had better clinical remission rate (CR) (P=0.003), event-free survival (P=0.0014), and overall survival (OS) (P=0.0017). However, <i>FLT3-ITD</i> and <i>CEBPA mutations</i> did not show any association with CR (P=0.404 and 0.92, respectively). Biallelic <i>CEBPA</i> mutations were found in 12 (7.95%) patients and were associated with better OS (P=0.043).</p><p><strong>Conclusions: </strong>These findings indicate that <i>NPM1</i> and <i>CEBPA</i> mutations can be precisely used for risk stratification in CN-AML patients.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"13 1","pages":"28-43"},"PeriodicalIF":0.0,"publicationDate":"2023-02-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10017593/pdf/ajbr0013-0028.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9750771","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Inherited thrombophilia: undetected comorbidity complicating COVID-19 infection. 遗传性血栓病:未发现的COVID-19感染合并症
Pub Date : 2023-01-01
Divya Khatana, Poonam Rani, Swati Jain, Richa Gupta, Ashish Goel, Mrinalini Kotru

The relation between the severity of COVID-19 and coexisting undiagnosed underlying thrombophilic conditions is not yet established. It may be possible that undiagnosed thrombophilia exaggerates an already pro-thrombotic state in COVID-19 patients and may be responsible for severe disease in absence of any known co-morbidity. The aim was to analyze the association of underlying thrombophilia with the severity of COVID-19 infection in post-COVID patients after a minimum of 6 weeks of recovery and to compare thrombophilia profile in severe versus non-severe COVID-19 patients. Forty severe and 40 non-severe COVID patients at least 6 weeks post recovery were selected for thrombophilia profile and complete blood count evaluation. The data were analyzed using Stata software, USA; version 13. The Chi-square test and Student's t-test were used to compare proportions and mean respectively. A total of 14/80 (17.5%) were positive for the thrombophilia screen. Protein C deficiency was noted in 6/40 (15%) severe COVID patients but not in the non-severe group. The Protein S deficiency was seen in 7/40 (17.5%) severe patients and only 1 patient was deficient in the non-severe group (2.5%). The mean Protein C and Protein S levels of severe and non-severe COVID patients were statistically significant (P-0.002) and (P-0.007) respectively. The difference in mean anti-COVID IgG antibody titer of severe and non-severe COVID patients was also statistically significant (P-0.0001). To Conclude, Protein C & S deficiencies were the commonest abnormalities detected in severe COVID patients. Positive thrombophilia profile and higher titers of anti-IgG COVID-19 antibodies were seen in a significant number of patients who had suffered from Severe COVID-19 than in non-severe infection, even after 6 weeks of recovery. Thereby, suggesting that underlying thrombophilia might have affected the severity of the disease.

COVID-19的严重程度与共存的未确诊的潜在血栓性疾病之间的关系尚未确定。在没有任何已知合并症的情况下,未确诊的血栓性疾病可能会夸大COVID-19患者已经存在的血栓形成前状态,并可能导致严重疾病。目的是分析在恢复至少6周后的COVID-19患者中潜在的血栓形成与COVID-19感染严重程度的关系,并比较严重和非严重COVID-19患者的血栓形成情况。选择康复后至少6周的重症和非重症COVID患者各40例,进行血栓倾向分析和全血细胞计数评估。数据分析采用美国Stata软件;13个版本。分别采用卡方检验和学生t检验比较比例和平均值。共有14/80(17.5%)的血栓形成筛查呈阳性。6/40(15%)重症COVID患者存在蛋白C缺乏症,但在非重症组中没有。重度组7/40(17.5%)出现蛋白S缺乏,非重度组仅有1例(2.5%)出现蛋白S缺乏。重症和非重症患者的平均蛋白C和蛋白S水平分别有统计学意义(P-0.002)和(P-0.007)。重症和非重症患者抗-COVID IgG抗体滴度的差异也有统计学意义(P-0.0001)。总之,蛋白C和S缺陷是重症COVID患者中最常见的异常。即使在康复6周后,与非严重感染的患者相比,严重COVID-19患者的血栓倾向阳性和抗- igg COVID-19抗体滴度更高。因此,表明潜在的血栓形成可能影响了疾病的严重程度。
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引用次数: 0
The effect of aging on the epidemiology of blood transfusions in North Khorasan province, Iran. 年龄对伊朗北呼罗珊省输血流行病学的影响。
Pub Date : 2023-01-01
Hamed Mortazavi, Akbar Mohammadi, Elham Sharifian, Nima Firouzeh, Alex van Belkum, Hamed Ghasemzadeh-Moghaddam

Purpose: Additional knowledge on the epidemiology and recipients of blood transfusions will help health-care managers to estimate the future needs. The study was performed to define the blood transfusion rate based on gender, sex, and clinical features of patients receiving blood products in all hospitals of the North Khorasan province of Iran.

Methods: Data on blood transfusion implementation were extracted from blood bank documents. The data for all patients who received at least one blood product were collected from March 2018 to March 2019.

Results: Among blood transfused patients, the highest transfusion rate was for packed red blood cells (PRBC) (47.7%). The two other most frequently used products were fresh frizzed plasma (FFP) (27.2%) and platelets (PLT) (21.9%). The patients in the age group of 51-80 years received the majority of PRBCs and FFPs. Patients aged 21-40 and 61-70 yrs had the highest transfusion rates for PLT. Elderly female patients (57.4%) received more blood products than their male counterparts. The highest blood transfusion rates were among patients with neoplasms, anemia, gastrointestinal bleeding, and gastric diseases.

Conclusion: The primary Iranian blood recipients were elderly patients. Population aging is associated with an increase in the number of blood recipients and simultaneously declines the blood donors pool. It highlights the need for optimizing the use of blood in hospitals and having better strategies for overcoming the shortage of blood.

目的:关于流行病学和输血接受者的更多知识将有助于卫生保健管理人员估计未来的需求。该研究旨在根据伊朗北呼罗珊省所有医院接受血液制品的患者的性别、性别和临床特征确定输血率。方法:从血库资料中提取输血执行情况资料。收集了2018年3月至2019年3月期间接受至少一种血液制品的所有患者的数据。结果:输血患者中,填充红细胞(PRBC)输血率最高(47.7%)。另外两种最常使用的产品是新鲜卷曲血浆(FFP)(27.2%)和血小板(PLT)(21.9%)。51 ~ 80岁的患者接受了大部分的红细胞和FFPs。21-40岁和61-70岁的PLT患者输血率最高。老年女性患者(57.4%)比男性患者接受更多的血液制品。输血率最高的是肿瘤、贫血、胃肠道出血和胃部疾病患者。结论:伊朗血主要受血者为老年患者。人口老龄化与接受血液的人数增加有关,同时减少了献血者的数量。它强调需要优化医院血液的使用,并制定更好的战略来克服血液短缺。
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引用次数: 0
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American journal of blood research
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