Treatment of Amyloidosis Associated Factor X Deficiency

1976 ◽  
Vol 35 (02) ◽  
pp. 377-381 ◽  
Author(s):  
Joel A. Spero ◽  
Jessica H. Lewis ◽  
Ute Hasiba ◽  
Lawrence D. Ellis
Keyword(s):  
Fresh Frozen Plasma ◽  
Factor Ix ◽  
Bleeding Complications ◽  
Primary Amyloidosis ◽  
Factor X ◽  
Associated Factor ◽  
Factor X Deficiency ◽  
Fresh Frozen ◽  
Frozen Plasma

SummaryThis is the tenth patient in thirteen years to be reported with the findings of an isolated factor X deficiency associated with primary amyloidosis. A favorable response to factor IX concentrate was manifested by temporary clinical and laboratory correction of her diathesis. This mode of treatment, therefore, provides an approach to therapy for bleeding complications in this group of patients who have previously failed to respond to fresh frozen plasma.

‘Do not Do’ Recommendations in Hemophilia

2020 ◽  
Vol 20 (3) ◽  
pp. 168-174
Author(s):  
Hortensia De la Corte-Rodriguez ◽  
E. Carlos Rodriguez-Merchan ◽  
M. Teresa Alvarez-Roman ◽  
Monica Martin-Salces ◽  
Victor Jimenez-Yuste
Keyword(s):  
Improve Patient Safety ◽  
Health Resources ◽  
Fresh Frozen Plasma ◽  
Factor Ix ◽  
Levels Of Care ◽  
Fresh Frozen ◽  
History Of ◽  
Improve Patient ◽  
Different Levels ◽  
Frozen Plasma

Background: It is important to discard those practices that do not add value. As a result, several initiatives have emerged. All of them try to improve patient safety and the use of health resources. Purpose: To present a compendium of "do not do recommendations" in the context of hemophilia. Methods: A review of the literature and current clinical guidelines has been made, based on the best evidence available to date. Results: The following 13 recommendations stand out: 1) Do not delay the administration of factor after trauma; 2) do not use fresh frozen plasma or cryoprecipitate; 3) do not use desmopressin in case of hematuria; 4) do not change the product in the first 50 prophylaxis exposures; 5) do not interrupt immunotolerance; 6) do not administer aspirin or NSAIDs; 7) do not administer intramuscular injections; 8) do not do routine radiographs of the joint in case of acute hemarthrosis; 9) Do not apply closed casts for fractures; 10) do not discourage the performance of physical activities; 11) do not deny surgery to a patient with an inhibitor; 12) do not perform instrumental deliveries in fetuses with hemophilia; 13) do not use factor IX (FIX) in patients with hemophilia B with inhibitor and a history of anaphylaxis after administration of FIX. Conclusions: The information mentioned previously can be useful in the management of hemophilia, from different levels of care. As far as we know, this is the first initiative of this type regarding hemophilia.

scholarly journals Geographic differences in hemophilia-associated AIDS incidence in Pennsylvania. By the Pennsylvania AIDS Surveillance Study Group

Blood ◽  
1987 ◽  
Vol 70 (4) ◽  
pp. 1208-1210 ◽  
Keyword(s):  
Blood Product ◽  
Fresh Frozen Plasma ◽  
Factor Ix ◽  
Antibody Prevalence ◽  
Product Usage ◽  
Fresh Frozen ◽  
Acquired Immunodeficiency ◽  
Immunodeficiency Syndrome ◽  
Hemophilia Treatment Centers ◽  
Frozen Plasma

A 1986 survey of seven hemophilia treatment centers in Pennsylvania (PA) has revealed that 22 hemophiliacs residing in PA have developed the acquired immunodeficiency syndrome (AIDS), representing 9.2% of the total 238 United States hemophiliac AIDS cases. These 22 included ten (45.5%) from western PA (W-PA), eleven (50.0%) from central PA (C-PA), and one (0.5%) from eastern PA (E-PA). The HIV antibody prevalence for these three geographic groups is comparable, with 84 of 178 (47.2%) of hemophiliacs in W-PA seropositive, 102 of 182 (56.0%) in C-PA seropositive, and 105 of 177 (59.3%) in E-PA seropositive. Blood product usage for these three areas is comparable: 47.8 X 10(3) (W-PA) v 43.9 (C-PA) v 53.3 (E-PA) units factor VIII concentrate per patient per year; 36.5 v 24.5 v 33.7 for factor IX concentrate; 8.4 v 4.7 v 7.7 for cryoprecipitate; and 1.3 v 2.7 v 1.0 for fresh frozen plasma, respectively. These data demonstrate a geographic variation in hemophilia AIDS incidence in PA, with a tenfold higher incidence in W- PA and C-PA than E-PA, which is unrelated to differences in HIV antibody prevalence, patient blood product usage, or inaccuracies in AIDS case reporting. Because of the greater than or equal to 5 year median latency between HIV infection and development of AIDS, the AIDS incidence will continue to change, but other factors appear to be operative in the development of AIDS in hemophiliacs.

scholarly journals Inherited Moderate Factor X Deficiency Presenting as Cardiac Tamponade

2019 ◽  
Vol 2019 ◽  
pp. 1-4
Author(s):  
Tamer Othman ◽  
Ayman Abdelkarim ◽  
Karen Huynh ◽  
An Uche ◽  
Jennifer Lee
Keyword(s):  
Cardiac Tamponade ◽  
Clinical Manifestations ◽  
Missense Variant ◽  
Fresh Frozen Plasma ◽  
Factor X ◽  
Pericardial Window ◽  
Extensive Experience ◽  
Management Guidelines ◽  
Factor X Deficiency ◽  
Fresh Frozen

Factor X deficiency is a rare bleeding disorder that varies in the severity of its clinical manifestations. The symptoms of this disorder can occur at any age, although most severe cases appear in childhood. The rarity of this condition has not allowed for the establishment of evidence‐based management guidelines, and thus, individuals afflicted with factor X deficiency are treated based on limited literature and the opinions of clinicians with extensive experience. In this case report, we discuss a unique presentation of a 38-year-old male who was found to have cardiac tamponade as a result of his newly diagnosed inherited moderate factor X deficiency. This was discovered by obtaining a factor X activity assay and confirmed with genetic testing which demonstrated a missense variant on the factor X gene on chromosome 13. His management involved correction of his factor X deficiency with fresh frozen plasma, a pericardiocentesis, and placement of a pericardial window. He has been asymptomatic and without hemorrhagic episodes for the 10 months following his discharge.

scholarly journals Geographic differences in hemophilia-associated AIDS incidence in Pennsylvania. By the Pennsylvania AIDS Surveillance Study Group

Blood ◽  
1987 ◽  
Vol 70 (4) ◽  
pp. 1208-1210 ◽  
Keyword(s):  
Blood Product ◽  
Fresh Frozen Plasma ◽  
Factor Ix ◽  
Antibody Prevalence ◽  
Product Usage ◽  
Fresh Frozen ◽  
Acquired Immunodeficiency ◽  
Immunodeficiency Syndrome ◽  
Hemophilia Treatment Centers ◽  
Frozen Plasma

Abstract A 1986 survey of seven hemophilia treatment centers in Pennsylvania (PA) has revealed that 22 hemophiliacs residing in PA have developed the acquired immunodeficiency syndrome (AIDS), representing 9.2% of the total 238 United States hemophiliac AIDS cases. These 22 included ten (45.5%) from western PA (W-PA), eleven (50.0%) from central PA (C-PA), and one (0.5%) from eastern PA (E-PA). The HIV antibody prevalence for these three geographic groups is comparable, with 84 of 178 (47.2%) of hemophiliacs in W-PA seropositive, 102 of 182 (56.0%) in C-PA seropositive, and 105 of 177 (59.3%) in E-PA seropositive. Blood product usage for these three areas is comparable: 47.8 X 10(3) (W-PA) v 43.9 (C-PA) v 53.3 (E-PA) units factor VIII concentrate per patient per year; 36.5 v 24.5 v 33.7 for factor IX concentrate; 8.4 v 4.7 v 7.7 for cryoprecipitate; and 1.3 v 2.7 v 1.0 for fresh frozen plasma, respectively. These data demonstrate a geographic variation in hemophilia AIDS incidence in PA, with a tenfold higher incidence in W- PA and C-PA than E-PA, which is unrelated to differences in HIV antibody prevalence, patient blood product usage, or inaccuracies in AIDS case reporting. Because of the greater than or equal to 5 year median latency between HIV infection and development of AIDS, the AIDS incidence will continue to change, but other factors appear to be operative in the development of AIDS in hemophiliacs.

Thrombin Generation in Newborn Plasma Is Critically Dependent on the Concentration of Prothrombin

1990 ◽  
Vol 63 (01) ◽  
pp. 027-030 ◽  
Author(s):  
Maureen Andrew ◽  
Barbara Schmidt ◽  
Lesley Mitchell ◽  
Bosco Paes ◽  
Frederick Ofosu
Keyword(s):  
Thrombin Generation ◽  
Fresh Frozen Plasma ◽  
Factor Ix ◽  
Blood Products ◽  
Platelet Concentrates ◽  
Term Infants ◽  
Cord Plasma ◽  
Coagulation Proteins ◽  
Fresh Frozen ◽  
Frozen Plasma

SummaryThe ability to generate thrombin is decreased and delayed in plasma from the healthy newborn infant compared to the adult. Only 30 to 50% of peak adult thrombin activity can be produced in neonatal plasma. To test whether this observation can be explained by the low neonatal levels of the contact or vitamin K dependent factors, we measured neonatal thrombin generation after raising the concentration of these factors to adult values. We also determined whether the addition of a variety of blood products to neonatal plasma improved thrombin generation. An amidolytic method was used to quantitate intrinsic (APTT) and extrinsic (PT) pathway thrombin generation in defibrinated pooled cord plasma from healthy term infants. Added individually, factors VII, IX, X or the contact factors (CF) failed to alter the rate or the total amount of thrombin generated in neonatal plasma. In contrast, the addition of prothrombin increased the total amount of thrombin generated to above adult values in both the APTT and the PT systems but did not alter the rate of thrombin generation. The rate of thrombin generation in cord plasma shortened after a combination of II, IX, X and CF was added to the APTT system or II, VII and X to the PT system. In both systems, the total amount of thrombin generated was linearly related to the initial prothrombin concentration. Each of fresh frozen plasma, cryoprecipitate, plasma from platelet concentrates, or factor IX concentrate (in amounts used therapeutically) caused an increase in the total amount of thrombin generated which was related to the increase in prothrombin concentration. Thus, the total amount of thrombin generated in newborn plasma is critically dependent on the prothrombin concentration whereas the rate at which thrombin is generated is dependent on the levels of many other coagulation proteins in combination.

Preprocedural Coagulation Studies in Pediatric Patients Undergoing Percutaneous Intervention for Appendiceal Abscesses

2015 ◽  
Vol 81 (9) ◽  
pp. 859-864 ◽  
Author(s):  
Sandra M. Farach ◽  
Paul D. Danielson ◽  
Nicole M. Chandler
Keyword(s):  
Bleeding Risk ◽  
International Normalized Ratio ◽  
Fresh Frozen Plasma ◽  
Percutaneous Intervention ◽  
Bleeding Complications ◽  
Poor Correlation ◽  
Appendiceal Abscess ◽  
Significant Difference ◽  
Fresh Frozen ◽  
Frozen Plasma

The literature reports poor correlation between coagulation screening and prediction of bleeding risk in children. Our aim is to determine whether there is a role for coagulation studies in children undergoing percutaneous intervention for appendiceal abscesses. A retrospective review of 1805 patients presenting with a diagnosis of appendicitis from September 2008 to September 2013 was performed. Patients presenting with appendiceal abscess who underwent percutaneous intervention were selected for further review (n = 131). A total of 76 patients (58%) had normal coagulation studies, whereas 55 (42%) had elevated values. An international normalized ratio ≥ 1.3 was found in 26 patients. Patients with normal coagulation values had an incidence of bleeding of 1.3 per cent. In the abnormal coagulation group, 8 patients received fresh frozen plasma before intervention, whereas 47 did not. There was one hematoma noted in each group with an incidence of bleeding of 3.6 per cent. The overall incidence of hematoma was 2.3 per cent with no significant difference in bleeding risk between the normal and abnormal coagulation groups. In conclusion, although many patients are found to have elevated coagulation studies, most do not have bleeding complications after intervention. There is poor correlation between coagulation screening and postprocedural outcomes evidenced by the low risk of bleeding.

Fresh Frozen Plasma (FFP) Usage and Appropriateness in Adult Medical in-Patients: A Retrospective Audit

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4375-4375
Author(s):  
Raghava Reddy Levaka Veera ◽  
Doron Schneider ◽  
Peter V. Pickens
Keyword(s):  
Vitamin K ◽  
Conflicts Of Interest ◽  
Fresh Frozen Plasma ◽  
Bleeding Complications ◽  
Medical Patients ◽  
Nursing Time ◽  
Inappropriate Use ◽  
Retrospective Audit ◽  
Fresh Frozen ◽  
Frozen Plasma

Abstract Abstract 4375 BACKGROUND: The use of Fresh Frozen Plasma (FFP) has increased considerably in recent years. In the USA there was 70% increase in the use of plasma in 10 years between 1991 and 2001. Around 3.9 million units were transfused in 2001 and 5.7 million units in 2009. FFP is often used inappropriately globally and studies evaluating FFP use in the US are scarce. We conducted a study to assess the trends of FFP use, its implications, appropriateness, and impact of FFP on International Normalized Ratio (INR) in hospitalized adult medical patients with an elevated INR. DESIGN AND METHODS: The study was a retrospective audit of all adult medical in-patients with an INR of 1.2 or higher who received FFP during a 3 month period from December 1, 2011 to February 28, 2012 admitted to Abington Memorial Hospital. A list of all patients who received FFP during the study period was obtained from the blood bank. Patients with major trauma, surgical, post-op, pediatric and obstetric/gynecological patients were excluded. Data was collected on demographics, indications of FFP, pre and post transfusion INR and effect of FFP on pre-transfusion INR. Partial thromboplastin time values (PTT) were not collected. RESULTS: A total of 479 units of FFP were transfused to 97 patients over 114 transfusion episodes. Of the 97 patients, 51 were male (52.6%) and 46 were female (47.3%) with a mean age of 70.4 yrs (median 73 yrs, range 19–95 yrs). Of the total 479 units of FFP, 233 units (48.6%) were given to patients who were not actively bleeding (n=53) with a mean pre-transfusion INR (pre-INR) of 2.82 (range 1.5 to >10, SD 1.6). Warfarin related coagulopathy was noted in 65 patients (67%) and 340 units of FFP (71%) were transfused to these patients. FFP units transfused as pre-procedural prophylaxis were 176 out of 479 (36.7%). Twenty transfusion episodes amounting to 53 units of FFP (11.1%) were given to patients who were not actively bleeding and had a pre-INR of <2. Six patients did not get the planned procedure done and received 25 units (5.2%) of FFP. Of 15 patients who received FFP as pre-procedure prophylaxis with INR <2, only one (6.6%) had normalized post-transfusion INR (post-INR), 5 (33.3%) had post-INR of <1.5 and none had any bleeding complications. Inappropriate use of FFP in this study was noted to be 23.7% (114/479 units of FFP). Mean improvement in INR per unit of FFP for all transfusion episodes (n=114) was 0.36, with mean pre-INR of 3.28 (range 1.5 to >10) and mean post-INR of 1.72 (range 1.2 to 4.2). Mean improvement in INR/FFP in patients with pre-INR <2 vs. those with pre-INR 2 or higher was 0.22 vs. 0.42 respectively (p=0.021). Mean improvement in INR/FFP was significantly higher in patients with warfarin related coagulopathy vs. in those secondary to other causes (0.46 vs. 0.33, p=0.001). Patients who received vitamin K concurrently with FFP had higher mean improvement in INR/FFP than those who did not receive vitamin K (0.45 vs. 0.17, p=0.001). It was also noted that timing of post-INR check was very heterogeneous. Two patients developed an allergic reaction needing stoppage of transfusion and further treatment. Approximately $6,840 were wasted in direct costs ($60 per unit) in just 3 months for only medical in-patients excluding indirect costs (estimated at $100 per unit= $11,400) such as technician, nursing time; transfusion sets, pre-medication, etc. CONCLUSION: Inappropriate use of FFP remains high even among medical in-patients. This study is consistent with previous studies in finding warfarin reversal as the major indication of FFP use and higher the pre-INR, higher was the improvement in INR. Especially, high percentage of use of FFP for pre-procedural prophylaxis with mild elevation of INR is alarming; therefore, studies evaluating restrictive vs. liberal use of FFP are needed. *ICU=Intensive care unit, PCU= Progressive care unit, ER= Emergency room, GMF= General medical floor. Disclosures: No relevant conflicts of interest to declare.

scholarly journals AIDS retrovirus antibodies in hemophiliacs treated with factor VIII or factor IX concentrates, cryoprecipitate, or fresh frozen plasma: prevalence, seroconversion rate, and clinical correlations

Blood ◽  
1986 ◽  
Vol 67 (3) ◽  
pp. 592-595 ◽  
Author(s):  
MV Ragni ◽  
GE Tegtmeier ◽  
JA Levy ◽  
LS Kaminsky ◽  
JH Lewis ◽  
...  
Keyword(s):  
Factor Viii ◽  
Seroconversion Rate ◽  
Fresh Frozen Plasma ◽  
Factor Ix ◽  
Human T Cell ◽  
Fresh Frozen ◽  
Acquired Immunodeficiency ◽  
Immunodeficiency Syndrome ◽  
Time To Onset ◽  
Frozen Plasma

Abstract Antibodies to the AIDS retrovirus, specifically to human T cell lymphotropic virus, type III, and AIDS-associated retrovirus, were detected with increasing prevalence in a population of 190 hemophiliacs from western Pennsylvania between 1981 and 1984: 7.7% in 1981, 20.0% in 1982, 45.5% in 1983, and 62.5% in 1984. The seropositive included approximately three fourths of those receiving factor VIII concentrate, nearly one third of those receiving factor IX concentrate, nearly one fifth of those receiving cryoprecipitate, and none of those receiving fresh frozen plasma. The seroconversion rate, determined on 43 seropositive hemophiliacs from this group who were serially sampled, was 0% in 1977, 4.7% in 1978, 4.9% in 1979, 2.6% in 1980, 10.5% in 1981, 52.9% in 1982, 87.5% in 1983, and 100% in 1984. Of 27 seropositive for three or more years (since 1982 or before), four (15%) have developed AIDS and seven (26%), diffuse lymphadenopathy (ARC); of 16 seropositive for less than three years, none has developed AIDS and three (19%) have developed ARC. The mean time from seroconversion to onset of ARC, 0.8 +/- 0.2 years (SEM), was shorter (P less than .001) than the time to onset of AIDS, 4.1 +/- 0.6 years. These findings confirm the widespread presence of AIDS retrovirus and support the association of these retroviruses with the acquired immunodeficiency syndrome and related conditions.

scholarly journals Successful use of BPL Factor X concentrate in a child with severe factor X deficiency

2013 ◽  
Vol 1 (2) ◽  
pp. 8-10 ◽  
Author(s):  
Kate Khair ◽  
Poornima Kumar ◽  
Mary Mathias ◽  
Jemma Efford ◽  
Ri Liesner
Keyword(s):  
Central Venous Access ◽  
Coagulation Factor ◽  
Venous Access ◽  
Fresh Frozen Plasma ◽  
Factor X ◽  
Post Dose ◽  
Thrombotic Risk ◽  
Factor X Deficiency ◽  
Fresh Frozen ◽  
The Right

Abstract Introduction: Severe factor X deficiency is a rare serious bleeding disorder historically treated with fresh frozen plasma (FFP) and more recently with prothrombin complex concentrate (PCC) which contains activated factors II, VII, IX and X. The infusion volume of PCC is smaller than FFP, but there is a risk of thromboembolic complications given the presence of activated forms of vitamin K-dependent factor concentrates when treating an isolated coagulation factor deficiency. Methods: We describe the case of a nine-year-old girl of consanguineous origin with co-existent congenital merosin deficient muscular dystrophy and severe factor X deficiency treated with twice-weekly PCC prophylaxis via an indwelling central venous access device (CVAD). Infusion occlusion of her fifth CVAD occurred 24-months post-insertion; thrombus within the right subclavian and brachiocephalic veins was seen on radiological imaging. She started peripheral treatment with BPL Factor X concentrate as infusion volumes were smaller and given her immobility further thrombotic risk was predicted to be reduced. A sixth CVAD was inserted seven months later and BPL Factor X prophylaxis was continued. Results:BPL Factor X concentrate was effective in maintaining trough levels of 13IU/ml 72-hours post-dose, with no intercurrent bleeding episodes or further problems in terms of occlusion of her portacath. Further radiological screening has not been undertaken. Conclusion: BPL Factor X has been shown to be a safe and effective alternative to PCC for treatment of severe factor X deficiency in this case.

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