Mycophenolate Mofetil Therapy Is a Steroid Sparing In Childhood and Young Adult Chronic ITP and Evans Syndrome.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1441-1441
Author(s):  
Zaher Naji ◽  
Madhvi Rajpurkar ◽  
Sureyya Savasan ◽  
Roland Chu ◽  
Meera Chitlur ◽  
...  
Keyword(s):  
Mycophenolate Mofetil ◽  
Side Effects ◽  
Bleeding Complications ◽  
Severe Bleeding ◽  
Evans Syndrome ◽  
Corticosteroid Administration ◽  
Chronic Itp ◽  
Life Threatening ◽  
The Mean ◽  
Steroid Sparing

Abstract Abstract 1441 Chronic ITP and Evans syndrome are diseases characterized by autoantibody formation with resultant destruction of platelets or both platelets and red blood cells on neutrophils, respectively. Affected patients are at risk of life threatening bleeding complications and/or life threatening anemia. Conventional therapies are often ineffective or transiently effective and have significant toxicity. One of the most commonly used therapeutic strategies employs chronic corticosteroid administration with the attendant weight gain, hypertension, hyperglycemia, bone loss, infection risk, mood changes and protean other undesirable side effects. Mycophenolate Mofetil (MMF) is an immunosuppressive agent with a favorable side effect profile. It is converted to the active metabolite, mycophenolic acid, and interferes with purine metabolism in T-lymphocytes, effectively killing many of these cells and down-regulating autoimmune phenomena. With approval of our local IRB /HIC we retrospectively reviewed the charts of 11 chronic ITP/Evans syndrome patients who had received MMF, all such patients treated at a large urban Children's Hospital. Clinical variables included age, sex, duration of disease, steroid use, IVIG use, Anti-D use, platelet counts, hemoglobin concentrations and reticulocyte percentages. These data were analyzed using paired t-tests, one-sample t-test and descriptive statistics. The 11 patients ranged in age from 9–22 years old, with a mean age of 15 years. The mean time from diagnosis of disease was 41.8 months with a range of 6–95 months. There were 5 female subjects, 6 Evans syndrome patients and 5 with chronic ITP alone. The median platelet count over the 6 months prior to MMF was 70×109/L, (18-223), with a median of 90×109/L with (27-145) during the first 6 months of MMF therapy (p=0.4). In the Evans syndrome group, the mean hemoglobin prior to MMF was 10.9 g/dL, (9.1-14.6), with a mean of 12.1 g/dL, (6.9-16.7) on MMF (p=0.54). Similarly, the mean reticulocyte percentage was 2.7%, (0.5-14) prior to MMF, with a mean of 2.3%, (0.3-8.9) during MMF therapy. The mean total dose of steroids used in the 6 months prior to MMF was 84.2 mg/kg (prednisone equivalent), (11.2-170), compared to 62.2 mg/kg, (0-193.8) on MMF. 9 of 11 patients had reductions in steroid requirement by an average of 49.3% (p=0.0013). During the first 6 months of MMF both IVIG and anti-D usage decreased from total doses for the entire group of 17 and 3 doses to 8 and 1 dose, respectively. None of the patients experienced severe bleeding episodes or side effects more serious than a transient rash while on MMF. These data suggest that MMF may have a role as a steroid sparing therapy in the treatment of chronic ITP and Evans syndrome. Disclosures: Off Label Use: mycophenolate mofetil for ITP Evans syndrome.

scholarly journals Risks associated with oral deferiprone in the treatment of infratentorial superficial siderosis

2019 ◽  
Vol 267 (1) ◽  
pp. 239-243 ◽  
Author(s):  
Y. Sammaraiee ◽  
G. Banerjee ◽  
S. Farmer ◽  
B. Hylton ◽  
P. Cowley ◽  
...  
Keyword(s):  
Side Effects ◽  
Case Series ◽  
Iron Chelator ◽  
Superficial Siderosis ◽  
Neutropenic Sepsis ◽  
Favourable Safety ◽  
Favourable Safety Profile ◽  
Life Threatening ◽  
The Mean ◽  
Mean Time

Abstract Objective Deferiprone is an iron chelator that has recently been used to treat patients with infratentorial superficial siderosis (iSS). It is considered to have a generally favourable safety profile but concerns have been raised due to the risk of agranulocytosis. We aimed to evaluate the safety and tolerability of oral deferiprone as a treatment for patients with iSS. Methods We present a case series of 10 consecutive patients presenting with classical iSS treated with deferiprone. Results Ten patients were followed up for a mean period of 2.3 years (range 0.5–5.5 years). Four patients (40%) were withdrawn from treatment because of treatment-related side effects. The reasons for treatment discontinuation were neutropenic sepsis (n = 3) and fatigue (n = 1). In 2 out of the 3 cases of neutropenic sepsis, patients initially developed neutropenia without sepsis. The mean time to neutropenic sepsis following deferiprone was 1.2 years (range 0.3–2.5) with mean neutrophil count of 0.4 (range 0.3–0.5). Six patients (60%) reported no change in neurological function while on treatment, and four patients (40%) reported that their condition deteriorated. Conclusions Deferiprone was poorly tolerated, with 40% of patients withdrawing from treatment, most commonly due to neutropenic sepsis, after an average of 2 years on treatment. This study increases the number of reported cases of agranulocytosis in patients with iSS treated with deferiprone. Clinicians treating iSS patients with deferiprone should be aware that this drug has a potentially life-threatening side effect of neutropenic sepsis, and should ensure that appropriate haematological monitoring is in place.

The Administration of Fondaparinux in Strongly Suspected Heparin-Induced Thrombocytopenia (HIT): Further Evidence of Its Safety and Efficacy, and Need for a Controlled Randomized Study.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 1072-1072
Author(s):  
Franco Piovella ◽  
Stefano Barco ◽  
Marisa Barone ◽  
Chiara Beltrametti ◽  
Mara De Amici
Keyword(s):  
Unfractionated Heparin ◽  
Bleeding Complications ◽  
Controlled Study ◽  
Severe Bleeding ◽  
Minor Bleeding ◽  
Heparin Induced Thrombocytopenia ◽  
Safety And Efficacy ◽  
Coronary Syndrome ◽  
Platelet Number ◽  
The Mean

Abstract Abstract 1072 Poster Board I-94 Introduction: Heparin-induced thrombocytopenia (HIT), alone or associated with thrombosis (HITT), may develop during anticoagulant treatment with unfractionated heparin (UFH) or low-molecular weight heparin (LWMH). Fondaparinux is a selective inhibitor of coagulation factor Xa which apparently does not react with anti-PF4/heparin antibodies in in vitro testing. Methods: From january 2005 to december 2007 we treated 44 patients who had strong suspect of HIT (12 patients) or HITT (32 patients, of whom 12 had both DVT and PE). Of these, 30 patients were previously exposed to unfractionated heparin (UFH) for major cardiac surgery. The remaining patients were admitted to medical or general surgery wards. In the 32 patients who developed HITT, we applied therapeutic dosages of fondaparinux, i.e. 7.5 mg QD or lower, in accordance with their bleeding risk. The remaining patients were treated with prophylactic dosages of fondaparinux, i.e. 2.5 mg QD. Switch to warfarin was performed as soon as possible. The mean of our patients 4T's score was 6.2, corresponding to high risk patients; the mean of anticorpal optical density (GTI Enhanced®) was 1.4; the mean platelet number was 45 × 109/L. Results: All patients but four showed sustained normalization of the platelet number. All patients but four showed a significant reduction of their thromboembolic burden. No death related to severe bleeding was recorded. Two episodes of major bleeding were recorded in post-surgical patients (4,5%); 4 episodes of minor bleeding were recorded (9,1%). Four patients underwent dialysis during fondaparinux without bleeding complications. One patient developed acute coronary syndrome during treatment with fondaparinux. Nine patients did not survive (20,5%), with a key role of primitive diseases (i.e. sepsis) causing delay in the diagnostic process of HIT (four of them had a diagnosis of HIT with a mean delay of 7 days). In 16 patients submitted to therapeutic dosages of fondaparinux, anti-PF4/heparin antibody titers were followed over time showing a constant decrease: in one case antibody levels did not decrease up to 6 months after stopping both heparin and fondaparinux. Thirty patients were easily switched from fondaparinux to VKAs without problems in reaching the appropriate INR target. Conclusions: This report provides further evidence supporting the safety and efficacy of fondaparinux in the treatment of HIT and underlines the importance of an early diagnosis. However, it shows the need of a randomized controlled study between fondaparinux and a registered comparator drug. Disclosures: Piovella: GlaxoSmithKline: Honoraria, Speakers Bureau; Bayer: Honoraria, Speakers Bureau; Boehringer Ingelheim: Honoraria, Speakers Bureau. Off Label Use: fondaparinux in the treatment of heparin-induced thrombocytopenia.

scholarly journals Analysis of Using Low Dose 3 Factor PCC (Bebulin®) for INR Reversal in Patients with Warfarin Induced Severe/Life-Threatening Bleeding

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5043-5043 ◽  
Author(s):  
Sabeen F. Mekan ◽  
Lennart Logdberg ◽  
Arline Stein ◽  
Leah Nash ◽  
Jessica Cohen ◽  
...  
Keyword(s):  
Clinical Judgment ◽  
Low Dose ◽  
Skilled Nursing Facility ◽  
Severe Bleeding ◽  
Nursing Facility ◽  
Skilled Nursing ◽  
Off Label ◽  
Average Patient ◽  
Life Threatening ◽  
The Mean

Abstract Background: 3 Factor Prothrombin Complex Concentrates have been used off label with plasma products for effective and timely reversal of INR in patients on Warfarin presenting with severe bleeding. The exact dosage required for this purpose has yet to be determined. Methods: We conducted a retrospective review of all patients taking Warfarin who presented to our institution between January 1, 2013 to June 30, 2014 who received 3 Factor Prothrombin Complex Concentrate (PCC) Bebulin® for INR reversal for significant/lifethreatening bleeding. The dose of Bebulin was determined by clinical judgment by the blood bank and the treating physician in accordance with the patient’s weight, bleeding location and the presenting INR. Results: A total of 116 patients were treated. Mean age was 72 years. Forty-two (36.21%) of patients were female. Most common bleeding site was intracranial (n=61), followed by systemic hemorrhage (n=24), GI bleeding (n=16), trauma (n=10) and other bleed (n=5). Some patients were also on other anticoagulants besides Warfarin – 28 patients (24%) were on Aspirin and 9 (7.8%) on Plavix. The mean INR prior to Bebulin treatment was 3.62 which dropped to 2.05 at an average of 2.2 hours post-treatment. Mean level at 12 hour post-Bebulin dropped to 1.57. The average patient received 2 units of plasma and 1 unit of packed red cell transfusions. The average Bebulin dose administered was 959 IU (SD +/-465 IU) with average patient weight of 82.9 kg (SD +/- 27kg). Based on this the average Bebulin dose administered was 11.57 IU/kg. Eighty-eight patients (76%) were able to be discharged home or to a skilled nursing facility. Thirty-six patients (31.1%) required more than one dose of Bebulin®. Mean INR at presentation in this group of patients were not similar to the remainder of the group. Mean INR at average of 2.5 hours post-Bebulin in this group was 2.18 which dropped further to an average of 1.75 at 9 hours. The total average Bebulin dose incrementally administered was similar in both groups. Conclusion: Low dose Bebulin at doses of approximately 12 IU/kg with 2 Units of plasma was effective in timely reversal of INR in majority of patients who presented with Warfarin induced bleeding. Disclosures Off Label Use: Bebulin (3 Factor PCC) is approved for use in Hemophilia patients; we discuss using low dose for INR reversal in Warfarin associated bleeding.

Severe Bleeding Complications during Antiepileptic Treatment with Valproic Acid in Children: A Description of Four Cases.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2155-2155
Author(s):  
Elvira Cannizzaro ◽  
Manuela Albisetti ◽  
Eva Bergstraesser ◽  
Franziska Scherer ◽  
Bernhard Schmitt ◽  
...  
Keyword(s):  
Intensive Care ◽  
Side Effects ◽  
Von Willebrand Factor ◽  
Serum Levels ◽  
Bleeding Complications ◽  
Severe Bleeding ◽  
Intensive Care Treatment ◽  
Factor Concentrate ◽  
Von Willebrand ◽  
Willebrand Factor

Abstract Sodium valproat (VPA) is a commonly used antiepileptic drug in children. It is known to cause a variety of different haematological side effects, including thrombocytopenia, a decrease in plasma fibrinogen, von Willebrand factor (VWF)- and factor XIII, impaired platelet aggregation with ADP and Collagen and prolonged PFA-100 closure times. The mechanisms responsible for such a variety of haematological abnormalities are still poorly understood and controversy exists about their clinical relevance and the risk for significant bleeding. We report on 4 children with severe bleeding complications during VPA treatment that had been followed at our institution. A first child with a cerebral malformation and no history of bleeding developed a subdural haematoma during VPA treatment without trauma. Additional studies revealed decreased von Willebrand factor levels that persisted with episodes of severe epistaxis also after VPA was stopped. A second infant suffered from herpes encephalitis with generalised seizures and died from an intracranial bleeding a day after a VPA loading dose was administred. Laboratory studies revealed prolonged PFA100 closure times. A third child with a therapeutic VPA serum-levels developed asymptomatic mild thrombocytopenia (around 100 G/l); unexpectedly in this child a tonsillectomy was complicated with sever diffuse haemorrhage that required red cell transfusion and intensive care treatment. Finally, a forth child who had been on VPA therapy for years causing prolonged PFA100 closure times, developed a large spinal epidural haematoma during epidural anaesthesia three days after urological surgery. This child had received VWF containing factor concentrate preoperatively only. For the bleeding additional VWF containing factor concentrate was given, the catheter was removed and the child recovered completely. Paediatricians, neurologists and anesthesists should be aware of the haemostatic side effects of VPA that can lead to significant bleeding. Before starting VPA therapy, patients should be evaluated for the presence of a congenital bleeding disorder. Preoperatively for any surgical intervention and in case of the occurrence of bleeding symptoms the patients have to be further evaluated for a VPA-induced disorder of the plasmatic haemostasis or an impaired platelet function. Haemostatic side effects of VPA can occur at therapeutic VPA serum levels and are found at any time during the course of VPA treatment. VPA has to be used cautiously in severely ill children and in intensive care patients and the risk of bleeding of any surgical or anesthesiological procedure has to be evaluated. Treatment options for VPA induced haemostatic abnormalities include plasma derived factor concentrates that contain VWF and the use of antifibrinolytic agents. Further studies have to assess the feasibility of standard treatment with the vasopressin analog DDAVP for this group of patients taking into account their high risk for seizures.

scholarly journals The Clinical Application of Feiba Immuno in Factor VIII Inhibitor Patients

1977 ◽  
Author(s):  
K. Anderle
Keyword(s):  
Side Effects ◽  
Factor Viii ◽  
Case Reports ◽  
Bleeding Complications ◽  
Factor Viii Inhibitor ◽  
Acute Side Effects ◽  
Minor Surgery ◽  
Viii And Ix ◽  
Viii Inhibitor ◽  
The Mean

The aim of this report is to summarize the clinical and laboratory effect and possible side-effects arising in the use of FEIBA IMMUNO for treating Factor VIII and IX inhibitor patients.In the time between 22nd November 1974 and today FEIBA IMMUNO was supplied for 104 treatment periods. More than 70 patients in 22 hemophilia centers all over Europe were treated, 59 case reports from 19 of the centers were sent to IMMUNO AG for further evaluation.In 8 cases minor surgery was carried out, of which only one case was complicated by post-operative bleeding. In all other cases different bleeding complications were treated. The clinical effect was difficult to evaluate, but the impression was favourable.FEIBA’s effect on WBCT, the r-value of TEG, and on aPTT are statistically evaluated for two different mean dosages.Acute side-effects were allergic reactions in 5 cases and laboratory signs of DIC in 4 cases, two of which had received extremely high dosages. Calculation of the mean pre- and post-treatment values for platelets and fibrinogen did not show any differences.

The spectrum of Evans syndrome in adults: new insight into the disease based on the analysis of 68 cases

Blood ◽  
2009 ◽  
Vol 114 (15) ◽  
pp. 3167-3172 ◽  
Author(s):  
Marc Michel ◽  
Valérie Chanet ◽  
Agnès Dechartres ◽  
Anne-Sophie Morin ◽  
Jean-Charles Piette ◽  
...  
Keyword(s):  
Lymphoproliferative Disorders ◽  
Severe Bleeding ◽  
Evans Syndrome ◽  
Related Risk ◽  
Threatening Condition ◽  
Life Threatening ◽  
Underlying Disorder ◽  
Immune Neutropenia ◽  
Common Variable

Abstract Evans syndrome (ES) is a rare disease characterized by the simultaneous or sequential development of autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP) and/or immune neutropenia. To better describe the characteristics and outcome of ES in adults, a survey was initiated in 2005. The data from 68 patients (60% of them women) fulfilling strict inclusion criteria for ES are reported. The mean age at time of ITP and/or AIHA onset was 52 plus or minus 33 years, both cytopenias occurred simultaneously in 37 cases (54.5%). ES was considered as “primary” in 34 patients (50%) but was associated with an underlying disorder in half of the cases, including mainly systemic lupus, lymphoproliferative disorders, and common variable immunodeficiency. All patients were given corticosteroids, but 50 of them (73%) required at least one “second-line” treatment, including splenectomy(n = 19) and rituximab (n = 11). At time of analysis, after a mean follow-up of 4.8 years, only 22 patients (32%) were in remission off treatment; 16 (24%) had died. In elderly patients, the risk of cardiovascular manifestations related to AIHA seems to be higher than the ITP-related risk of severe bleeding. In conclusion, ES is a potentially life-threatening condition that may be associated with other underlying autoimmune or lymphoproliferative disorders.

scholarly journals Steroid-Sparing Agents in Giant Cell Arteritis

2019 ◽  
Vol 13 (1) ◽  
pp. 61-71
Author(s):  
Amol Sagdeo ◽  
Ayman Askari ◽  
Josh Dixey ◽  
Hana Morrissey ◽  
Patrick A. Ball
Keyword(s):  
Mycophenolate Mofetil ◽  
Side Effects ◽  
Giant Cell Arteritis ◽  
Giant Cell ◽  
Psychological Effects ◽  
Remission Induction ◽  
Remission Maintenance ◽  
Short And Long Term ◽  
Steroid Sparing

Background: Giant cell arteritis is the commonest form of medium-to-large vessel vasculitis, requiring long-term corticosteroid therapy. The short- and long-term side effects of corticosteroids are many, including weight gain, psychological effects, osteoporosis, cardiometabolic complications, and infections. Materials and Methods: Various agents used in place of or in combination with corticosteroids to reduce corticosteroid-related side effects were reviewed. However, considerable variation in practice was identified giving unclear guidance. This review included the most recent evidence on methotrexate, mycophenolate mofetil, azathioprine, cyclophosphamide, abatacept, and tocilizumab Results and Discussion: Also discussed are encouraging results with tocilizumab in GCA patients. Amongst the agents available for steroid-sparing effects, tocilizumab demonstrated the most robust data and is consequently recommended as the agent of choice for steroid-sparing, for remission induction, remission maintenance, and treating relapsing and refractory cases of GCA.

scholarly journals Toxic effects of immunosuppressive drugs: mechanisms and strategies for controlling them

1996 ◽  
Vol 42 (8) ◽  
pp. 1316-1321 ◽  
Author(s):  
L M Shaw ◽  
B Kaplan ◽  
D Kaufman
Keyword(s):  
Mycophenolate Mofetil ◽  
Clinical Practice ◽  
Side Effects ◽  
Immunosuppressive Drugs ◽  
New Drugs ◽  
Therapeutic Drug ◽  
Clinical Effects ◽  
Transplant Patients ◽  
Life Threatening ◽  
Definition Of

Abstract Since cyclosporine (CsA) was introduced into clinical practice in late 1983 to prevent rejection in transplant patients, there has been an almost explosive growth in the number and types of transplants and the number of transplant centers, an increase in the life expectancy of the transplanted organ, and substantial decreases in rates of acute rejection and life-threatening infections. Despite these successes, major improvements in immunosuppressive therapy are needed, especially a reduction in toxic side effects and a rigorous definition of the relation between drug concentration and clinical effects. Such improvements may be achievable with the incorporation of new drugs such as tacrolimus and mycophenolate mofetil into immunosuppression protocols and the development of rigorously defined therapeutic drug-monitoring programs.

scholarly journals Understanding the risks of total hip arthroplasty in patients with von Willebrand’s disease

2020 ◽  
Vol 28 (3) ◽  
pp. 230949902096024
Author(s):  
Andrew G. Yun ◽  
Marilena Qutami ◽  
Sean A. Fischer ◽  
Kory B. Dylan Pasko
Keyword(s):  
Total Hip Arthroplasty ◽  
Hip Arthroplasty ◽  
Direct Anterior Approach ◽  
Bleeding Complications ◽  
Severe Bleeding ◽  
Von Willebrand's Disease ◽  
Von Willebrand’S Disease ◽  
Total Hip ◽  
Estimated Blood Loss ◽  
The Mean

Purpose: Patients with von Willebrand’s disease (VWD) have either a qualitative or quantitative deficiency in a key clotting protein called von Willebrand’s factor. Type Ⅰ disease is the most common variant, but its clinical implications in total hip arthroplasty (THA) are unclear. Our purpose is to describe the perioperative impact of VWD in THA. Methods: We retrospectively reviewed a total of 17 primary THAs in 14 patients with type Ⅰ VWD performed between 2008 and 2019. Almost all cases (88%) received tranexamic acid, and most (59%) received DDAVP. All patients had a direct anterior approach (DAA) THA. Results: None of these cases required a blood transfusion. Mean estimated blood loss was 229 mL, and the mean hemoglobin dropped from 13.9 g/dL to 10.2 g/dL. There were no major bleeding complications. After a mean follow-up of 4 years, the mean hip disability and osteoarthritis outcome score, junior (HOOS, JR) was 79, and there were no reoperations or revisions for any cause. Conclusion: Patients with type Ⅰ VWD do not experience severe bleeding with routine chemoprophylaxis combined with DAA THA.

scholarly journals Idarucizumab (Praxbind®) for dabigatran reversal in patients undergoing heart transplantation: a cohort of ten patients

2021 ◽  
pp. FSO689
Author(s):  
Eran Kalmanovich ◽  
Pascal Battistella ◽  
Philippe Rouviere ◽  
Bernard Albat ◽  
Jean-Marc Frapier ◽  
...  
Keyword(s):  
Heart Transplantation ◽  
Emergency Surgery ◽  
Oral Anticoagulants ◽  
Bleeding Complications ◽  
Single Center Experience ◽  
Increased Risk ◽  
Life Threatening ◽  
The Mean ◽  
End Stage ◽  
Dabigatran Therapy

Background: Novel oral anticoagulants are used in atrial fibrillation. Idarucizumab has been approved for reversal of dabigatran in situations of life-threatening hemorrhage or emergency surgery. Objectives: We report a single center experience of ten patients on dabigatran therapy who were given idarucizumab prior to heart transplantation. Methods & results: The mean plasma concentration of dabigatran prior to reversal was 139 ± 89 ng/ml. Hemoglobin, hematocrit and platelet levels were decreased after surgery. Surgical procedures were successfully performed with no increased risk, especially regarding bleeding complications. All patients were alive after 90 days. Conclusion: Dabigatran reversal with idarucizumab in contexts of emergency surgery/urgent procedures is an attractive and safe option to be taken into consideration for patients with end stage heart disease awaiting transplantation and indication of anticoagulant therapy.

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