bluuebunny/crossref_metadata_2025_split huggingface dataset:
Risk of Venous Thromboembolism in Hospitalized Patients with Sickle Cell Disease
Authors: Taylor Joshua , Anghel Alexandra , Corsi Daniel J , Carrier Marc , Tinmouth Alan , Saidenberg Elianna 
Published: October 2019

Abstract
Background: Patients with sickle cell disease (SCD) are at an increased risk of developing venous thromboembolism (VTE). However, the underlying risk of VTE complication during hospitalization is unclear in this patient population. We sought to report the incidence of VTE and its associated risk factors in hospitalized SCD patients.
Patients/Methods: A retrospective cohort study of SCD patients requiring hospitalization was undertaken at a tertiary care center. Incidence ratios of VTE per hospitalization for different risk factors (Thromboprophylaxis use, central venous catheter (CVC), past history of VTE, surgery during hospitalization) were assessed. Univariate, age adjusted and multivariate Poisson models were estimated accounting for the repeated hospitalizations per patients.
Results: A total of 101 patients with at least one hospitalization were included in the study. The mean of number of admissions per patients was 8.9. Overall, 17 out of 896 (1.9%) admissions were complicated by VTE. The incidence of VTE varied by risk factors, from 0.8% in patient without CVC to 6.7% among patients admitted with previous history of VTE. Age adjusted and multivariate Poisson models for incidence rate ratios of VTE per hospitalization among patients with SCD for different risk factors are depicted in Table 1.
Conclusion: The risk of VTE seems low in hospitalized SCD. A prior history of VTE and a hospitalization for surgery might be associated with higher risk of VTE complication. Future studies assessing these risk factors to tailor thromboprophylaxis regimens are needed.

Disclosures
No relevant conflicts of interest to declare.
Are Bleeding Scores Predicting Severity and Outcome in Hemophilia and Rare Bleeding Disorders?
Authors: Shahriari Mahdi , Karimi Mehran 
Published: October 2019

Abstract

Background: Hemophilia is an inherited severe bleeding disorder, Factors VIII and IX deficiencies have X linked inheritance. Most rare bleeding disorders (RBD) have autosomal recessive inheritance. The ISTH-BAT is a valuable research tool that is applicable to clinical practice and scores patients' bleeding symptoms from 0 to 4. ISTH-BAT has the potential to avoid unwanted laboratory testing, predict the risk of bleeding, describe symptom severity and inform treatment.
Aims: To assess the utility of ISTH-BAT in our bleeding disorder population, compare the bleeding score (BS) in adult and pediatric groups and investigate its association with plasma factor levels.Von Wilebrand disease and thrombasthenia was excluded from this study. One hundred cases of epistaxis whose coagulation workups were within normal limits were enrolled in the control group.
Methods: This is an observational analytical study. The ISTH-BAT was used to calculate bleeding scores (BS) in a group of hemophilic patients and healthy controls. Ethics approval and informed consents were secured prior to the study.
Results: A total of 100 patients, (38 hemophilia A; FVIII deficiency,22hemophilia B; FIX deficiency, 10 hemophilia C; factor XI deficiency, 10 factor VII deficiency, 8 factor X deficiency, 6 factor V deficiency, 5 Afibrinogenemia, Factor I deficiency) and 100 controls were analyzed using the ISTH BAT. Mean BAT score in hemophilia A, B and C were 12.8±8.2; 11.6±8.6; and 7.5 ±5.52respectively.While in RBD were and 7.97±5.56. BAT score in 30 controls was 5.35±4.48. Bleeding Scores were significantly higher in hemophilia A and B patients as compared to controls (P < 0.05) but it was not significant when BAT Scores of hemophilia C patients compared with controls (P=0.35). ANOVA test revealed BAT Score were significantly different among the mild, moderate and severe hemophilia A and B in both adult and pediatric patients but there was no difference in Hemophilia C patients. BAT scores were linked to hematomas; minor wound bleeding in hemophilia A, B patients in the pediatric group while more linked with epistaxis bleeding after dental extraction and surgical interventions in the adult group. Mean BAT Scores of rare bleeding disorders was significantly higher than control group (10.97± 5.65; compared with 5.35±4.48; P<0.05). BAT Score of 10 cases of factor VII deficiency and 5 cases of Afibrinogenemia were higher than other case of RBD, and control group (14.35 ± 7.87; and 13.54±5.56 compared with 5.35±4.48; P< 0.001 and P<0.05respectively).
Conclusions: Our data revealed that the ISTH-BAT can help diagnose the bleeding condition in hemophilia and RBD patients and can be considered a predictor for the bleeding risk or severity. This will decrease need of subspecialty coagulation tests; it ultimately improvesdecision making and the clinical management of patients.

Disclosures
No relevant conflicts of interest to declare.
Clinical Features and Morbidities of Hb H Disease in Taiwan
Authors: Lu Meng Yao , Kuo Ming Chung , Wang Shih Chung , Chen Shih Hsiang , Ko Bor Sheng , Chang Cheng-Shyong , Tang Jih-Luh 
Published: October 2019

Abstract
Introduction Patients with non-transfusion-dependent thalassemia experience a wide array of clinical complications despite their independence from frequent, regular red blood cell transfusions. They have the higher incidence of osteoporosis, extramedullary hematopoeisis (EMH), hypogonadism, cholelithiasis, thromboembolic disease, pulmonary hypertension, silent cerebral ischemia, and leg ulcers. Thalassemia is highly prevalent in Taiwan and Hb H disease is predominant. But limited data are available about clinical features and morbidities. Here, we studied clinical features and morbidities in Taiwanese patients with Hb H disease.
Methods & Results We collected 90 patients with Hb H disease in three hospitals since 2014 Nov till 2016 July. Male to female were 43/59. The mean age was 33.1 years ( from 0.5 to 92.3 years). Two cases died of pulmonary hypertension and old age at 31 years old and 87 years old. Alfa-globin gene genotype studies were done in 44 cases. The (- -(SEA)) type of α(0)-thalassemia mutation was detected in all patients. Twenty- four (57.1%) cases were deletional (α(3.7)/ α(4.2)/unknown 19/4/1) and 20 (42.9%) were nondeletional (CS/RS 18/2) type. The mean of Hemoglobin (Hb) and serum ferritin level were 8.7 g/dL and 730 ng/mL. We also revealed the positive correlation between age and serum ferritin level. The liver iron concentration (LIC) were 6.694 mg Fe/g dw (n=35). The Hb, ferritin and LIC level were not different between deletional and non- deletional groups. They received the transfusion management : 1 with regular transfusion ≦ 6 weeks interval, 5 with irregular transfusion ≧ 6 weeks interval, 27 with occasional transfusion and 57 without transfusion. Fifteen cases received splenectomy. There were significantly higher prevalence for transfusion frequency and splenectomy in non-deletional group. The prevalence of morbidities were 16/79 for cholelithiasis, 12/90 for thromboembolic event, 4/90 for heart failure symptoms ( 2 for pulmonary hypertension), 5/90 for arrhythmia, 3/90 for bone fracture, 5/20 for osteoporosis and 0 for renal stone. There were non-significantly higher prevalence for morbidities in non-deletional group.
Discussion & Conclusion The study provides the clinical features and the prevalence of morbidities in Hb H disease in Taiwan. Surprisingly, the prevalence of thromboembolic event and pulmonary hypertension are overlooked in our routine Hb H disease care. We need to schedule close and careful clinical follow up of Hb H patients as they get older, they get some morbidities or they are non-deletional genotype.

Disclosures
No relevant conflicts of interest to declare.
Use of the Platelet Indices for Differential Diagnosis of Pediatric Immune Thrombocytopenic Purpura ( ITP )
Authors: Nakadate Hisaya , Ishiguro Akira , Matsumoto Kimikazu 
Published: October 2019

Abstract
Introduction
The prognosis of pediatric immune thrombocytopenic purpura (ITP) has a favorable prognosis, but it is difficult to predict the course of this disease at the time of diagnosis. Recent advances of automated blood cell analyzer enable to measure platelet indices such as mean platelet volume (MPV), platelet size deviation width (PDW) and platelet-large cell ratio (P-LCR). These have brought about some information for thrombocytopenia. We studied the significance of such platelet indices in the differential diagnosis of immune thrombocytopenic purpura (ITP) and their ability to discriminate the clinical courses of ITP.
Methods
Ninety-three patients were enrolled this study at our institution from 2004 to 2006. Their ages ranged from 1 to 17 years (media 4.3 years). Of 93, patients were divided into two goups for analysis, according to the diagnosis of ITP. 34 were diagnosed as ITP and 59 were diagnosed various blood diseases other than ITP. In these 59 non-ITP patients, five were with thrombocytopenia (platelet count < 100.0 x 109/L), the other without thrombocytopenia. Of 34 ITP patients, 29 were acute subtype and 5 were chronic. The Sysmex-XE2100 automated blood cell analyzer (Sysmex, Kobe, Japan) was used to measure the blood parameters, including the platelet indices.
Results
According to MPV, PDW and P-LCR, there were no significant differences between ITP patients and non-ITP. In children with ITP, there were significant inverse correlations between the platelet count and evaluated parameters, MPV (R2=0.376), PDW (R2=0.26) and P-LCR(R2=0.338). But no significant correlations were found between the platelet count and these evaluated platelet indices in non-ITP. Also, we have found all platelet indices were significantly higher in chronic ITP than in acute ITP. In particular, PDW (9.910 vs 13.080) and P-LCR (21.071 vs 33.220) showed marked differences between the two groups.
Conclusions
ITP is considered as a result of hyper-destruction of platelets. Increase of platelet count is regarded as decrease of destruction of platelets. Our data suggested that platelet indices, MPV, PDW and P-LCR showed the useful information for the degree of platelet destruction and the ability to predict the clinical courses of ITP.

Disclosures
No relevant conflicts of interest to declare.
Primary Thrombophilia in Mexico XII: Miscarriages Are More Frequent in Persons with the Sticky Platelet Syndrome (SPS)
Authors: Ruiz-Delgado Guillermo J , Cantero-Fortiz Yahveth , Méndez-Huerta Mariana , León-González Mónica , León-Peña Andrés A. , Nuñez-Cortés Ana Karen , Ruiz-Arguelles Guillermo J. 
Published: October 2019

Abstract
Background: The sticky platelet syndrome (SPS) is an inherited condition which leads into arterial and venous thrombosis. There is scant information about the association between the SPS and obstetric complications.
Objective: To assess the relationship of the SPS and fetal loss in a single institution.
Material and methods: The obstetric history of all the consecutive female patients prospectively studied along a 324 month period, in a single institution with a history of thrombosis and a clinical marker of primary thrombophilia was reviewed.
Results: Between 1989 and 2016, 268 consecutive patients with a clinical marker of primary thrombophilia and a history of arterial or venous thrombosis were studied; of these, 108 were female patients. Within this subset, 77 (71%) had been pregnant at some moment. Twenty eight of these 77 patients (37%) had had an abortion and 24 out of these (86%) were found to have the SPS. On the other hand, in a subset of 73 female patients with the SPS who had been pregnant, 32% had miscarriages (14 had one abortion, 5 two abortions and 4 three or more abortions). These figures are significantly higher than the prevalence of abortions in the Mexican general population of pregnant women, which is 12-13% (chi square = 7.47; p = 0.0063). Accordingly, the relative risk of having a miscarriage is 2.66 times higher in female patients with the SPS than in the general population (p = 0.0014 ).
Conclusion: In México, female patients with the SPS experience significantly more miscarriages than the general population. Since the treatment of the SPS is simple and effective and could in turn prevent adverse obstetric outcomes, its investigation in women studied because obstetric complications may be useful and deserves further research.

Disclosures
No relevant conflicts of interest to declare.
Therapeutic Plasma Exchange for Thrombotic Microangiopathy
Authors: Ilhan Osman , Atilla Erden , Ataca Atilla Pinar , Civriz Bozdag Sinem , Kurt Yuksel Meltem , Toprak Selami Kocak , Topcuoglu Pervin , Akan Hamdi , Demirer Taner , Beksac Meral , Arslan Onder , Ozcan Muhit , Gurman Gunhan 
Published: October 2019

Abstract
Background: Thrombotic microangiopathy (TMA) is a syndrome characterized by thrombocytopenia, microangiopathic hemolytic anemia, neurologic abnormalities, fever and renal dysfunction. Therapeutic Plasma Exchange (TPE) is a standart treatment approach for the patients. Our aim is to determine the clinical characteristics and outcome of patients with TMA treated with TPE in our center.
Patients and Methods: We retrospectively evaluated 46 patients who have been diagnosed as TMA at Ankara University Department of Hematology between 2007 and 2015. Patients were treated with TPE (Frensenius Kabi AG, Homburg, Germany) until the normalization of laboratory parameters. The plasma exchanged was 1.5 times the predicted plasma volume with fresh frozen plasma for the first procedure, and usually 1.0 time the predicted volume thereafter until remission.
Results: 22M/24F was included in the study with a median age of 55 (range, 18-8310 of 46 patients (22%) were consultated from intensive care units, 7/46 (15%) from emergency unit and 6/46 (13%) from nephrology unit. The most common presenting symptom was purpura in 52%, followed by neurological disturbance 48%, renal function abnormality in 43% and fever in 28% of patients. At diagnosis the median hemoglobin (g/dl), leucocyte count (10^9/L) and thrombocyte count (10^9/L) were as follows: 9.4, 11.4 and 58.6. Median time period of procedure was 99 minutes (range, 64-313). 5/46 (11%) patients had femoral catheters and central venous catheters were the access for the rest of patients. None of the patients had severe adverse events during procedures. 21 patients achieved complete response (46%) after 2-40 sessions and 2 of them were died during follow-up. Responders were diagnosed mostly with infectious related TMA. 21 patients (46%) who had progressive disease died within 30 days after diagnosis. 2 non-responder patients had diagnosis of TTP and treated with succesfully with Rituximab (Table 1).
Conclusions: TPE is safe treatement modality in patients with TMA however there is still high mortality rate.

Disclosures
No relevant conflicts of interest to declare.
Treatment with Plasma Exchange Followed By Plasma Infusion for Thrombotic Thrombocytopenic Purpura Associated with SjöGren's Syndrome
Authors: Sun Xingfu , Lu Hua 
Published: October 2019

Abstract
Abstract: Thrombotic thrombocytopenic purpura (TTP) is rarely associated with Sjögren's syndrome (SS) with only 12 previously reported cases in the literature. All of them were women. A case of a previously healthy 64-year-old Chinese male presenting with microangiopathic hemolytic anemia, otherwise unexplained thrombocytopenia, and central nervous system involvement is reported. At the beginning the patient's family refused plasma exchange (PE) therapy due to cost consideration, later plasma infusion (PI) was initiated along with methylprednisolone pulse therapy. Significant clinical and laboratory response was achieved. However, recurrence happened after discontinuing the treatment for only three weeks without consulting his doctor. Then the patient received another two cycles of therapy of PE with again an excellent clinical and laboratory response. The patient was discharged in a state of remission on methylprednisolone and thymopolypeptides. In order to prevent further complications, prophylactic infusions of plasma was performed per month. The patient remained asymptomatic thereafter. Autoimmune workup revealed positive antinuclear antibodies and Anti-SSA. Lip biopsy revealed findings consistent with SS. TTP with SS tends to be recurrence. PE along with immunosuppressive therapy followed by prophylactic PI can prevent long-term organ complications in these cases.

Disclosures
No relevant conflicts of interest to declare.
Highly Procoagulant Extracellular Vesicles in Amniotic Fluid
Authors: Thaler Johannes , Hell Lena , Wisgrill Lukas , Spittler Andreas , Schwameis Michael , Jilma Bernd , Altevogt Peter , Ay Cihan , Pabinger Ingrid 
Published: October 2019

Abstract
Background: The pathomechanisms underlying disseminated intravascular coagulation (DIC) following amniotic fluid (AF) embolism remain to be fully elucidated. Highly procoagulant phosphatidylserine (PS)- and tissue factor (TF) expressing extracellular vesicles (EVs) might play a central role.
Objective: To perform extensive analyses of the procoagulant properties of AF with a panel of functional coagulation assays and flow cytometry to investigate the pathogenesis of AF induced DIC.
Methods: A prothrombinase assay, an EV-TF dependent factor Xa (FXa) generation assay, a modified thrombin- and fibrin-generation assay, a whole blood clotting model and flow cytometry were applied in AF- and control plasma.
Results: Phosphatidylserine expression was 21-fold increased in AF compared to plasma. Factor Xa generation was extremely high when TF-expressing EVs from AF were co-incubated with recombinant FVIIa. In the thrombin- and fibrin generation assay AF-derived EVs strongly activated the blood coagulation cascade via PS and TF. In a whole blood clotting model AF-derived TF-expressing EVs significantly shortened the clotting time from 734 ± 139 seconds in the presence- to 232 ± 139 seconds in the absence of an anti-TF antibody. The contact activation pathway via factor FXII was not affected. Applying flow cytometry, a sub-population of PS- and TF co-expressing EVs was clearly identified in AF.
Conclusions: We thoroughly investigated the effect of AF on blood coagulation and found that PS+ and TF+ EVs determine its procoagulant potential. Taken together our data further delineate the pathomechanisms underlying AF-induced coagulopathy, which could improve diagnostic- and treatment modalities.

Disclosures
No relevant conflicts of interest to declare.
Real World Data Analysis of Coagulation Factor IX Costs in Specific Patients with Hemophilia B Switching from Standard Half-Life to Extended Half-Life Products
Authors: Tortella Bartholomew J. , Fogarty Patrick F. , Alvir José , McDonald Margaret , Spurden Dean , Pleil Andreas M. 
Published: October 2019

Abstract
Introduction: Hemophilia B, an X-linked genetic disease characterized by low clotting factor IX (FIX) levels, leads to spontaneous and traumatic bleeding in affected individuals. Intravenous FIX replenishment is administered to maintain adequate levels. The recent introduction of an extended half-life (EHL) FIX replacement product provided the opportunity to compare costs of care associated with treatment with an EHL product versus a standard half-life (SHL) product.
Methods: The Truven Marketscan US claims database (Oct 2010 - Apr 2016) was used to identify factor concentrate charges for patients who had claims data for at least 3 months prior to and after switching from SHL to EHL products. Additionally, available data for up to 12 months pre- and post-switch on these identified patients were analyzed.
Results: Thirteen patients, ranging from 3-64 years of age (median 18) were included in the analyses. Factor concentrate costs were higher after switch from SHL to EHL in each of the time periods examined (Table). Median costs were used since they were less influenced by isolated outliers and were similar to means in most cases. The median costs were 238% higher in the 3 months just after switching to the EHL product compared to the 3 months just prior to switching from the SHL product. Median costs were $51,881, $79,654, $75,695 and $61,515 in the 12-10, 9-7, 6-4 and 3-1 months prior to switch respectively compared to $173,515, $147,061, $149,971 and $207,973 in the corresponding quarterly month intervals post switch.
Conclusion: This analysis of real world administrative data following individual patients through the switch from SHL to EHL products continuum suggests that switching from an SHL to an EHL product is associated with increased median factor costs throughout the 12 months following the switch, and that further analysis with larger numbers of patients should be explored.

Disclosures
Tortella: Pfizer Inc: Employment. Fogarty:Pfizer Inc: Employment. Alvir:Pfizer Inc: Employment. McDonald:Pfizer Inc: Employment. Spurden:Pfizer Inc: Employment. Pleil:Pfizer Inc: Employment.
Defining Haemostatic Safety Zones for the Practice of Sport in Patients with Severe Haemophilia By Thromboelastography
Authors: Fernandez-Bello Ihosvany , Alvarez-Román MT , de la Corte-Rodríguez H , Butta N , Hernández-Moreno AL , Rivas Muñoz S , Martín-Salces M , Rodriguez-Merchan EC , Canales Albendea Miguel , Jiménez-Yuste V 
Published: October 2019

Abstract
Introduction: Sport may comprise variable physical activity associated bleeding risk(BRisk) in haemophilia. We aimed to determine value of global thromboelastography (TEG) profile for tailoring prophylaxis in patients younger than 18 years old (YO) with severe haemophilia A (SHA) subjected to activities with different BRisk.
Methods: Patients with SHA, younger than 18 YO and on prophylaxis at least 12 months before inclusion were invited to participate. Patients signed informed consent before inclusion. Samples before prophylactic dosing were collected. Factor VIII (FVIII) and kaolin-activated TEG profile were determined. Physical activity (PhA) and annual joint bleeding rate (AJBR) were recorded retrospectively. BRisk was categorized following the US-National Haemophilia Foundation guidelines (1= safe; 1.5= safe-moderate risk; 2= moderate risk; 2.5= moderate-dangerous risk; 3= dangerous risk of bleeding).
Results: 26 patients, median age (min-max) 10.3 (6.8-14.3) YO were included. Sixty one percent of patients had BRisk= 1.0; 7.7% had BRisk= 2.0 and 2.5; 23,1% had BRisk= 3. In the last 12 months, haemarthrosis were presented in 33% of patients with BRisk=3.0 and 13% in those with BRisk= 1.0. Interesting, patients with BRisk= 2.0 or 2.5 did not present any joint bleeding in the previous year. Median levels of FVIII was 0.5 (0.0-1.9) IU/dl. There were no differences on trough FVIII levels between patients with or without joint bleeds (FVIII= 0.3 [0.0-1.9] and 1.1[0.2-4.4] IU/dl respectively, p=0.515) in the year before. There was no correlation between FVIII trough plasma levels and the AJBR (p=0.416). TEG trough profiles were similar in patients with and without haemarthrosis. However, patients with AJBR=0 presented a clear correlation between trough TEG parameters and BRisk (BRisk vs. k: r=-0,439; p= 0.047 and BRisk vs. alpha angle: r= 0.478; p= 0.025) so that, the higher the exercise-associated BRisk the higher procoagulant state needed to keep a joint-bleeding-free state. This correlation was not found for FVIII.
Conclusions: Kaolin-activated TEG assay may help to define safety zones to minimize bleedings in response to PhA in patients < 18 YO with SHA and may guide prophylaxis tailoring depending on BRisk of PhA.

Disclosures
No relevant conflicts of interest to declare.