5 TIWIKLY 5 Things I Wish I Knew Last Year (2015)

Total Page:16

File Type:pdf, Size:1020Kb

5 TIWIKLY 5 Things I Wish I Knew Last Year (2015) 5 TIWIKLY 5 Things I Wish I Knew Last Year (2015) Louis Kuritzky, MD University of Florida Family Medicine Residency Program Gainesville, Florida (352)-377-3193 Phone/FAX [email protected] Disclosure Louis Kuritzky, MD has NOTHING TO DISCLOSE In reference to the content of this presentation 1 Objectives • Identify potential therapeutic roles for tranexamic acid • Understand the limitations of AAT replacement therapy • Recognize the weaknesses of current data substantiating triglyceride treatment Disclosure Louis Kuritzky has nothing to disclose in reference to the content of this presentation. 2 5 TIWIKLY 2015 Score Card item # Knew Didn’t Know Keep Toss 1) Lung CA Screen Xray Burden 2) Melanoma Adhesive Patch Dx 3) Thermal Rx & Endothelial Fx 4) False + UDT Methadone 5) TRG Rx to ↓ CVD Risk 6) Warfarin & Dental Surgery 7) What is Tranexamic Acid 8) Menorrhagia & Factor VIII 9) A1AT & Emphysema 10) A1AT Replacement $$ 11) A1AT Efficacy 12) TRG & Pancreatitis How Low Dose is LDCT for Lung Cancer Screening? Arnold is a 62 year old man with a 80 p-y smoking history, who is considering participating in the currently approved protocol for lung cancer screening. He asks you: “It says ‘low dose’ CT. What does that mean?” You would best inform him that a LDCT is equivalent to a) A day in bright sunlight at the beach b)15-20 Chest PA x-rays c) 30-40 Chest PA x-rays d) 110-120 Chest PA x-rays 3 Lung CA Screening: Summary Recommendation “The USPSTF recommends annual screening for Lung CA with LDCT in adults aged 55-80 years who have a 30 p-y smoking Hx and currently smoke or have quit within the past 15 years.” Moyer VA, et al Ann Int Med online accessed 013-Dec-30 “Typical” Effective Doses from X-ray Radiographic Dose Equivalent Study (mSv) #CXR ChestPA 0.013 1 L-spine AP 0.44 30 Mammogram (4 view) 0.2 15 Dental Panorama 0.012 1 BE 5 350 CT L-spine 7 550 CT Abdomen 10 750 CT Chest 10 750 LDCT Chest 1.5 113 Adapted from Linet MS et al CA Cancer J Clin 2012;62:75-100 4 Evolution of Radiation Exposure 1980-2006 2006 per capita dose Other Sources < 0.14 mSv Medical 1980 per capita dose Sources 3.0 mSv Medical Sources Other Sources < 0.05 mSv 0.53 mSv Natural Sources 2.4 mSv Linet MSNatural et al Sources CA Cancer 2.4 mSv J Clin 2012;62:75-100 Linet MS et al CA Cancer J Clin 2012;62:75-100 Evolution of Radiation Exposure (mSv) 1980 2006 Natural Sources 2.4 2.4 Medical Sources 0.53 3.0 CT scans 0.03 1.47 Nuclear Medicine 0.1 0.77 Fluoroscopy 0.4 0.76 Other < 0.05 <0.14 Consumer products --- 0.13 Occupational --- 0.005 Nuclear Power --- 0.0005 TOTALLinet MS et al CA Cancer 3.0 J Clin mSv 2012;62:75-100 5.6 mSv Linet MS et al CA Cancer J Clin 2012;62:75-100 5 Estimated Radiation-Related Cancers from Repeated Screening Age X-ray related Test Frequency (years) CA/100,000 Lung LDCT Q1Y 50-70 230 ( ♂) 850 ( ♀) Coronary Ca++ Q1Y 45-70 40 ( ♂) 55-70 60 ( ♀) Mammography Q1Y <55 45-74 90 Q2Y >55 Linet MS et al CA Cancer J Clin 2012;62:75-100 First Glimpses into the Future From Your Dermatology Time Machine • Differentiation of melanoma from other pigmented skin lesions usually requires a tissue sample. Northwestern University (Chicago) has recently published data on a novel non-invasive methodology. It is ♦ Saliva testing for beta-microglobulin ♦ Adhesive patch testing for mRNA ♦ Ultraviolet light reflectometry ♦ Cutaneous DEXA scanning 6 Melanoma: Some Issues “The incidence of melanoma is ↑ at a rate of 3%-7%/yr for fair-skinned white populations, faster than any other major cancer.” Gerami P et al J Am Acad Dermatol 2014;71:237-44 Melanoma: Some Issues Invasive Depth 10-yr Survival Insitu 100% Breslowdepth < 1 mm >90% Breslowdepth > 4mm <50% Gerami P et al J Am Acad Dermatol 2014;71:237-44 7 Melanoma: Some Issues “While mortality from almost all preventable cancers has markedly decreased since 1975, melanoma-related mortality remains steady….” Gerami P et al J Am Acad Dermatol 2014;71:237-44 Melanoma: Some Issues “A number of studies have shown that early detection through a skin examination allows for Dx at an earlier and more curable state….In 1 study, screening skin examinations reduced mortality by 63%.” Gerami P et al J Am Acad Dermatol 2014;71:237-44 8 A Noninvasive Adhesive Patch Test for Pigmented Lesions • Study: Comparison DermTech adhesive patch vs Bx results (n=150) • Patch MOA: captures mRNA from CMIP & LINC00518 genes • Method: apply & remove patch, then Bx Gerami P et al J Am Acad Dermatol 2014;71:237-44 A Noninvasive Adhesive Patch Test for Pigmented Lesions Patch Bx Classifiction Classification Melanoma Nevus Melanoma 41 6 Nonmelanoma 1 16 Sensitivity 41/42 (97.6%) Specificity 16/22 (72.7%) Gerami P et al J Am Acad Dermatol 2014;71:237-44 9 What About Those False Positives? “Six of 22 cases with a histologic Dx of nevus had a molecular score consistent with a Dx of melanoma. Interestingly, among those 6 cases, 3 had a histologic reading of dysplastic nevus with severe atypia. Unfortunately, it is impossible to determine whether these 3 cases were truly false positives or actually early melanomas.” Gerami P et al J Am Acad Dermatol 2014;71:237-44 10 I Know I Have Risk Factors for CAD, but I HATE to Exercise…. • A 62 year old man with T2DM X 10 years, HTN, and dyslipidemia--all Rx appropriately--gives a ‘2 thumbs down’ to the suggestion of exercise. Which of the following interventions has been shown to improve endothelial function in persons with CAD risk factors? ♦ Magnesium Supplementation ♦ Thermal treatment (sauna) ♦ Ginseng tea ♦ Inversion boots (upside-down suspension) 11 Thermal Rx to Improve Endothelial Function • Study: Japanese men with and without CAD RF (n= 73) • Rx: 15 mins/d dry sauna (60 0 C) X 14 d • Outcome: %FMD (flow-mediated endothelium-dependent dilation) Imamura M, et al J Am Coll Cardiol 2001;38(4):1083-1088 Thermal Rx to Improve Endothelial Fx: Premises Ox-LDL DM HTN Smoking Endothelial Dysfunction Impaired FMD (Flow Mediated Vasodilation) Imamura M, et al J Am Coll Cardiol 2001;38(4):1083-1088 12 Thermal Rx to Improve Endothelial Function * 45% ↑ *emphasis added Imamura M, et al J Am Coll Cardiol 2001;38(4):1083-1088 Thermal Rx to Improve Endothelial Function Baseline PostRx p* p** Control CAD RF CAD RF %FMD 8.2 4.0 <0.0001 5.8 <0.001 %NTG 20.4 18.7 0.32 18.1 NS * vs control **vs preRx Imamura M, et al J Am Coll Cardiol 2001;38(4):1083-1088 13 Thermal Rx & Glucose: Unexpected Payoff? “A significant decrease in FPG concentration [99 mg/dL → 94 mg/dL, p < 0.05] after two weeks of sauna Rx was observed, consistent with the previous report using hot-tub therapy.” Imamura M, et al J Am Coll Cardiol 2001;38(4):1083-1088 An Unexpected UDT + for Methadone • A 56 y.o. house painter fell off a ladder fracturing 3 ribs. He has been prescribed tramadol for pain relief. A UDT comes back positive for methadone. The patient claims he has not taken any methadone. ♦ The pt is lying: FP methadone tests are very rare ♦ His tramadol (Ultram) causes a FP UDT ♦ The quetiapine (Seroquel) he takes for depression is causing a FP UDT for methadone ♦ Endogenous opioids induced by acute pain may cause a FP UDT for methadone 14 False Positive Methadone UDT Case Series of 10 “… 10 inpatients……suffering from mood or psychotic disorders who tested + for methadone. Since such false + methadone screenings may seriously affect the therapeutic relationship, this potential interaction is highly relevant for clinicians. Fischer M et al J Clin Psychiatr 2010;71(12):(LTE)1696-1696 False Positive Methadone UDT Case Series of 10 • All INPATIENTS: methadone access? • All denied methadone use • Gas chromatography-mass spectrometry re-test: methadone negative • All 10 pts Rx with quetiapine (Seroquel) Fischer M et al J Clin Psychiatr 2010;71(12):(LTE)1696-1696 15 False Positive Methadone UDT Case Series of 10 “…this report strongly suggests that positive methadone drug screenings be confirmed by a second method….especially in patients treated with quetiapine.” Fischer M et al J Clin Psychiatr 2010;71(12):(LTE)1696-1696 The Triglyceride Story: CVD Risk Reduction • Your new pt Jason W is an obese (BMI 32) 36 y.o. construction worker who requests a fibrate refill for Rx of ↑TRG (off-Rx = 380, on-Rx = 110). He denies pancreatitis. He drinks ‘a couple beers’ after work every day. He has no personal/FH of CVD, but was told by his since-retired GI Doc to take the fibrate to reduce his risk of CVD, which he has done for the last 3 years. He does not have HTN or T2DM. The fibrate causes no AEs. a) Refill the Rx as-is: success is success b) Increase the fibrate dose: his TRGs could be improved c) Decrease the fibrate dose: TRG ≤150 would be OK d) Decline to fill the Rx due to lack of outcomes evidence 16 Two Basic Clinical Questions • Has pharmacologic Rx of elevated triglycerides been PROVEN to improve CV outcomes? • Has pharmacologic Rx of triglycerides been PROVEN to reduce risk for pancreatitis? Two Basic Clinical Answers • ↓ CVD?: No RCT has demonstrated this 17 The Triglyceride Story: UpToDate 2014 CVD Risk Reduction with Rx: Uncertain “It is uncertain whether pharmacologic therapy targeted at reducing TRG levels will reduce CV risk.” Rosenson RS “Approach to the patient with hypertriglyceridemia” UpToDate accessed 9/2414 The Triglyceride Story: UpToDate 2014 Preventing Pancreatitis: No High Quality Evidence “Although high quality evidence is lacking, for primary prevention of pancreatitis, we and others suggest initiating pharmacologic therapy to reduce TRG when fasting concentration is >1000 mg/dL” Rosenson RS “Approach to the patient with hypertriglyceridemia”
Recommended publications
  • Thrombocytopenia
    © Copyright 2012 Oregon State University. All Rights Reserved Drug Use Research & Management Program Oregon State University, 500 Summer Street NE, E35 Salem, Oregon 97301-1079 Phone 503-947-5220 | Fax 503-947-2596 Drug Class Review: Thrombocytopenia Date of Review: January 2019 End Date of Literature Search: 11/05/2018 Purpose for Class Review: Treatments for thrombocytopenia are being reviewed for the first time, prompted by the recent approval of three new drugs; avatrombopag (Doptelet®), fostamatinib (Tavalisse™) and lusutrombopag (Mulpleta®). Research Questions: 1. What is the evidence for efficacy and harms of thrombocytopenia treatments (avatrombopag, eltrombopag, lusutrombopag, fostamatinib, and romiplostim)? 2. Is there any comparative evidence for therapies for thrombocytopenia pertaining to important outcomes such as mortality, bleeding rates, and platelet transfusions? 3. Is there any comparative evidence based on the harms outcomes of thrombocytopenia treatments? 4. Are there subpopulations of patients for which specific thrombocytopenia therapies may be more effective or associated with less harm? Conclusions: Three guidelines, six randomized clinical trials and five high-quality systematic reviews and meta-analyses met inclusion criteria for this review. There was insufficient direct comparative evidence between different therapies to treat thrombocytopenia. A majority of trials were small and of short duration. Guidelines recommend corticosteroids and intravenous immunoglobulin (IVIg) as first-line therapy for most adults with idiopathic thrombocytopenia (ITP). Thrombopoietin receptor agonists (TPOs) and the tyrosine kinase inhibitor, fostamatinib, are recommended as second-line treatments after failure of at least one other treatment.1–3 Avatrombopag and lusutrombopag are only approved for short-term use before procedures in patients with chronic liver failure.
    [Show full text]
  • Review Article
    SHOCK, Vol. 41, Supplement 1, pp. 44Y46, 2014 Review Article TRANEXAMIC ACID, FIBRINOGEN CONCENTRATE, AND PROTHROMBIN COMPLEX CONCENTRATE: DATA TO SUPPORT PREHOSPITAL USE? Herbert Scho¨chl,*† Christoph J. Schlimp,† and Marc Maegele‡ *AUVA Trauma Centre Salzburg, Academic Teaching Hospital of the Paracelsus Medical University Salzburg, Salzburg; and †Ludwig Boltzmann Institute for Experimental and Clinical and Traumatology, AUVA Research Centre, Vienna, Austria; and ‡Department of Trauma and Orthopedic Surgery, University of Witten/Herdecke, Cologne-Merheim Medical Center, Cologne, Germany Received 15 Sep 2013; first review completed 1 Oct 2013; accepted in final form 8 Nov 2013 ABSTRACT—Trauma-induced coagulopathy (TIC) occurs early after severe injury. TIC is associated with a substantial increase in bleeding rate, transfusion requirements, and a 4-fold higher mortality. Rapid surgical control of blood loss and early aggressive hemostatic therapy are essential steps in improving survival. Since the publication of the CRASH-2 study, early administration of tranexamic acid is considered as an integral step in trauma resuscitation protocols of severely injured patients in many trauma centers. However, the advantage of en route administration of tranexamic acid is not proven in prospective studies. Fibrinogen depletes early after severe trauma; therefore, it seems to be reasonable to maintain plasma fibrinogen as early as possible. The effect of prehospital fibrinogen concentrate administration on outcome in major trauma patients is the subject of an ongoing prospective investigation. The use of prothrombin complex concentrate is potentially helpful in patients anticoagulated with vitamin K antagonists who experience substantial trauma or traumatic brain injury. Beyond emergency reversal of vitamin K antagonists, safety data on prothrombin complex concentrate use in trauma are lacking.
    [Show full text]
  • December 4 Saturday
    ASH Meeting – Dec 5-8, 2020 CanVECTOR Oral & Poster Presentations Day / Time / Session Title Presenter Wednesday – December 2 December 2- 7:05 AM-8:30 AM The Immunohemostatic Response to Ed Pryzdial, PhD Infection Overview Program: Scientific Workshops @ ASH Session: The Immunohemostatic Response to Infection Friday – December 4 December 4 - 7:05 AM-8:05 AM Approach to Pregnancy and Delivery in Michelle Sholzberg, MDCM, FRCPC, MSc Women with Bleeding Disorders Program: Scientific Workshops @ ASH Session: Thrombosis and Bleeding in Pregnancy Prevention and Treatment of Venous Leslie Skeith, MD Thromboembolism in Pregnancy Saturday – December 5 December 5 - 7:00 AM-3:30 PM A Retrospective Cohort Study Evaluating the Kanza Naveed, Grace H Tang, MSc, McKenzie Safety and Efficacy of Peri-Partum Quevillon, RN, BScN, MN, Filomena Meffe, MD, MSc, Program: Oral and Poster Abstracts Tranexamic Acid for Women with Inherited Rachel Martin, MD, Jillian M Baker, MD, MSc, and Bleeding Disorders Michelle Sholzberg, MDCM, FRCPC, MSc Session: 322. Disorders of Coagulation or Fibrinolysis: Poster I Number: 863 Page 1 of 7 Day / Time / Session Title Presenter December 5 - 7:00 AM-3:30 PM A Retrospective Cohort Study Evaluating the Kanza Naveed, Grace H Tang, MSc, McKenzie Safety and Efficacy of Peri-Partum Quevillon, RN, BScN, MN, Filomena Meffe, MD, MSc, Program: Oral and Poster Abstracts Tranexamic Acid for Women with Inherited Rachel Martin, MD, Jillian M Baker, MD, MSc, and Bleeding Disorders Michelle Sholzberg, MDCM, FRCPC, MSc Session: 322. Disorders of Coagulation or Fibrinolysis: Poster I Number: 863 Frequency of Venous Thromboembolism in Mark Crowther, MD, David A.
    [Show full text]
  • Reversal of Anticoagulation in GI Bleeding
    Blood and Guts Reversal of anticoagulation in GI Bleeding John Hanley Consultant Haematologist Newcastle Hospitals NHS Trust [email protected] Healthy situation Haemostatic seesaw in a happy balance Clinical Thrombosis Atrial Fibrillation Deep Vein Thrombosis Pulmonary Embolus Cerebral Sinus Thrombosis Mesenteric Vein Thrombosis Arterial Embolus or Thrombosis Metallic Heart Valves Ventricular Assist Devices Antiphospholipid syndrome Anticoagulation Therapy Atrial Fibrillation Deep Vein Thrombosis Pulmonary Embolus ANTICOAGULANT Cerebral Sinus Thrombosis Mesenteric Vein Thrombosis DRUG Arterial Embolus or Thrombosis Metallic Heart Valves Ventricular Assist Devices Antiphospholipid syndrome Successful Anticoagulation Atrial Fibrillation Deep Vein Thrombosis Pulmonary Embolus Cerebral Sinus Thrombosis Mesenteric Vein Thrombosis Arterial Embolus or Thrombosis ANTICOAGULANT Metallic Heart Valves DRUG Ventricular Assist Devices Antiphospholipid syndrome Unsuccessful Anticoagulation Atrial Fibrillation Deep Vein Thrombosis Pulmonary Embolus Cerebral Sinus Thrombosis Mesenteric Vein Thrombosis Arterial Embolus or Thrombosis Metallic Heart Valves Ventricular Assist Devices Antiphospholipid syndrome ANTICOAGULANT DRUG Steady increase in numbers of patients receiving anticoagulation ≈1-2% of the UK population anti-coagulated AF 70% VTE 25% Other 5% 70000 60000 50000 40000 30000 20000 10000 0 1996 1998 2000 2002 2004 2006 2008 2010 2012 2014 Anticoagulants / Anti-platelets Unfractionated Heparin Low Molecular Weight Heparin Warfarin Other Vit
    [Show full text]
  • Reversal of Anticoagulation 14
    Table 6. Continued from page 4. References Continued from page 5. Primary 7. Kearon C. Hirsh J. Management of anticoagulation before and after elective surgery. New England Journal of Medicine. 336(21):1506-11, 1997. Anticoagulant Anticoagulation Reversal Anticoagulant Half-Life Excretion Therapeutic Anticoagulation Dose 8. Hanley JP. Warfarin reversal. Journal of Clinical Pathology. 57(11):1132-9, 2004 Nov. Action Monitoring Agents Mode 9. Holland L. Warkentin TE. Refaai M. Crowther MA. Johnston MA. Sarode R. Suboptimal effect of a three-factor prothrombin complex concen- trate (Profilnine-SD) in correcting supratherapeutic international normalized ratio due to warfarin overdose. Transfusion. 49(6):1171-7, 2009 Pediatrics Adults 10. Bauer KA. New anticoagulants. Hematology Am Soc Hematol Educ Program. 2006:450-6. Xa-Inhibitors 11. O'Connell NM. Perry DJ. Hodgson AJ. O'Shaughnessy DF. Laffan MA. Smith OP. Recombinant FVIIa in the management of uncontrolled hemorrhage. Transfusion. 43(12):1711-6,2003. Winter 2011 Fondaparinux: 17-21 Renal Anti-thrombin 0.15 mg/kg, daily, SC <50 kg: 5 mg, SC, Typically no rFVIIa 12. O'Connell KA, Wood JJ, Wise RP, Lozier JN, Braun MM. Thromboembolic adverse events after use of recombinant human coagulation factor (Arixtra) hours mediated inhibi- daily monitoring (NovoSeven) VIIa. JAMA. 2006 Jan 18;295(3):293-8. tion of factor Xa 50-100 kg: 7.5 mg, required SC, daily Anti-factor Xa 13. Riess HB. Meier-Hellmann A. Motsch J. Elias M. Kursten FW. Dempfle CE. Prothrombin complex concentrate (Octaplex) in patients requiring >100 kg: 10 mg daily (Calibrated with immediate reversal of oral anticoagulation.
    [Show full text]
  • A Randomized Study of 4-Factor Prothrombin Complex Concentrate and Authors: Jerrold H
    A Randomized Study of 4-Factor Prothrombin Complex Concentrate and Tranexamic Acid on Bleeding, Thrombin Generation, and Pharmacodynamics After Punch Biopsies in Rivaroxaban Treated Subjects With Supratherapeutic Drug Levels Authors: Jerrold H. Levy, MD1; Kenneth T. Moore, MS2; Matthew D. Neal, MD3; David Schneider, MD4; Victoria S. Marcsisin, MS2; Jay Ariyawansa, MS2; Jeffrey I. Weitz, MD5 1Duke University School of Medicine, Durham, North Carolina, United States; 2Janssen Pharmaceuticals, Janssen Scientific Affairs, Titusville, New Jersey, United States; 3University of Pittsburgh, Pittsburgh, Pennsylvania, United States; 4University of Vermont, Burlington, Vermont, United States; 7057 Figure 3 dra 1.4 5 This tagline is for informaon only; McMaster University and Thrombosis and Atherosclerosis Research Institute, Hamilton, Ontario, Canada DO NOT PRINT Figure 3. Time course of (A) prothrombin time (PT) and (B) endogenous thrombin Introduction Results potential (ETP) on mean absolute values change from baseline for treatment with 4F-PCC (Group A), TXA (Group B), and placebo (saline control) (Group C). • Oral factor Xa (FXa) inhibitors, including rivaroxaban, are replacing vitamin K • Of the 147 subjects enrolled (49 in each of the 3 cohorts), 145 (98.6%) completed A End of 4F-PCC/Placebo Infusion antagonists such as warfarin for many thromboembolic indications the study End of TXA/Placebo Infusion • Reversal of the anticoagulant effects of FXa inhibitors is important in the event of • Subject characteristics were comparable between the 3 cohorts (Table 1) clinically relevant bleeding 4F-PCC – The majority of subjects enrolled were male (70.1%) and white (66.0%), with a 25 2 TXA • Currently, there are no therapies approved for reversing oral FXa inhibitors, but median age of 28.0 years and a mean BMI of 25.0 kg/m Saline 4-factor prothrombin concentrate (4F-PCC) and tranexamic acid (TXA) have been suggested for this purpose lues, seconds Table 1.
    [Show full text]
  • Avatrombopag and Lusutrombopag For
    Journals Library Health Technology Assessment Volume 24 • Issue 51 • October 2020 ISSN 1366-5278 Avatrombopag and lusutrombopag for thrombocytopenia in people with chronic liver disease needing an elective procedure: a systematic review and cost-effectiveness analysis Nigel Armstrong, Nasuh Büyükkaramikli, Hannah Penton, Rob Riemsma, Pim Wetzelaer, Vanesa Huertas Carrera, Stephanie Swift, Thea Drachen, Heike Raatz, Steve Ryder, Dhwani Shah, Titas Buksnys, Gill Worthy, Steven Duffy, Maiwenn Al and Jos Kleijnen DOI 10.3310/hta24510 Avatrombopag and lusutrombopag for thrombocytopenia in people with chronic liver disease needing an elective procedure: a systematic review and cost-effectiveness analysis Nigel Armstrong ,1* Nasuh Büyükkaramikli ,2 Hannah Penton ,2 Rob Riemsma ,1 Pim Wetzelaer ,2 Vanesa Huertas Carrera ,1 Stephanie Swift ,1 Thea Drachen ,1 Heike Raatz ,1 Steve Ryder ,1 Dhwani Shah ,1 Titas Buksnys ,1 Gill Worthy ,1 Steven Duffy ,1 Maiwenn Al 2 and Jos Kleijnen 1 1Kleijnen Systematic Reviews, York, UK 2Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands *Corresponding author Declared competing interests of authors: Rob Riemsma is a member of the National Institute for Health Research Health Technology Assessment and Efficacy and Mechanism Evaluation Editorial Board. Published October 2020 DOI: 10.3310/hta24510 This report should be referenced as follows: Armstrong N, Büyükkaramikli N, Penton H, Riemsma R, Wetzelaer P, Huertas Carrera V, et al. Avatrombopag and lusutrombopag for thrombocytopenia in people with chronic liver disease needing an elective procedure: a systematic review and cost-effectiveness analysis. Health Technol Assess 2020;24(51). Health Technology Assessment is indexed and abstracted in Index Medicus/MEDLINE, Excerpta Medica/EMBASE, Science Citation Index Expanded (SciSearch®) and Current Contents®/ Clinical Medicine.
    [Show full text]
  • New Drug Evaluation Monograph Template
    © Copyright 2012 Oregon State University. All Rights Reserved Drug Use Research & Management Program Oregon State University, 500 Summer Street NE, E35 Salem, Oregon 97301-1079 Phone 503-947-5220 | Fax 503-947-2596 Drug Class Review: Thrombocytopenia Date of Review: January 2019 End Date of Literature Search: 11/05/2018 Purpose for Class Review: Treatments for thrombocytopenia are being reviewed for the first time, prompted by the recent approval of three new drugs; avatrombopag (Doptelet®), fostamatinib (Tavalisse™) and lusutrombopag (Mulpleta®). Research Questions: 1. What is the evidence for efficacy and harms of thrombocytopenia treatments (avatrombopag, eltrombopag, lusutrombopag, fostamatinib, and romiplostim)? 2. Is there any comparative evidence for therapies for thrombocytopenia pertaining to important outcomes such as mortality, bleeding rates, and platelet transfusions? 3. Is there any comparative evidence based on the harms outcomes of thrombocytopenia treatments? 4. Are there subpopulations of patients for which specific thrombocytopenia therapies may be more effective or associated with less harm? Conclusions: Three guidelines, six randomized clinical trials and five high-quality systematic reviews and meta-analyses met inclusion criteria for this review. There was insufficient direct comparative evidence between different therapies to treat thrombocytopenia. A majority of trials were small and of short duration. Guidelines recommend corticosteroids and intravenous immunoglobulin (IVIg) as first-line therapy for most adults with idiopathic thrombocytopenia (ITP). Thrombopoietin receptor agonists (TPOs) and the tyrosine kinase inhibitor, fostamatinib, are recommended as second-line treatments after failure of at least one other treatment.1–3 Avatrombopag and lusutrombopag are only approved for short-term use before procedures in patients with chronic liver failure.
    [Show full text]
  • Informationen Zur
    Kriterien zur Bestimmung der zweckmäßigen Vergleichstherapie und Recherche und Synopse der Evidenz zur Bestimmung der zweckmäßigen Vergleichstherapie nach § 35a SGB V Vorgang: 2017-B-074 Damoctocog alfa pegol Stand: Juni 2017 I. Zweckmäßige Vergleichstherapie: Kriterien gemäß 5. Kapitel § 6 VerfO G-BA Damoctocog alfa pegol_2017-B-074 [Behandlung und Prophylaxe der Hämophilie A] Kriterien gemäß 5. Kapitel § 6 VerfO Sofern als Vergleichstherapie eine Arzneimittelanwendung in Betracht kommt, muss das Siehe Tabelle „II. Zugelassene Arzneimittel im Anwendungsgebiet“ Arzneimittel grundsätzlich eine Zulassung für das Anwendungsgebiet haben. Sofern als Vergleichstherapie eine nicht- medikamentöse Behandlung in Betracht kommt, muss nicht angezeigt diese im Rahmen der GKV erbringbar sein. Beschlüsse/Bewertungen/Empfehlungen des - Richtlinie Ambulante Behandlung im Krankenhaus nach § 116b des Fünften Gemeinsamen Bundesausschusses zu im Buches Sozialgesetzbuch (SGB V) (Anlage 2, Nr. 2: Diagnostik und Versorgung Anwendungsgebiet zugelassenen Arzneimitteln/nicht- von Patienten mit Gerinnungsstörungen (Hämophilie)) medikamentösen Behandlungen - Beschluss zur Nutzenbewertung nach § 35a SGB V zum Wirkstoff Turoctocog alfa vom 3. Juli 2014 - Beschluss zur Nutzenbewertung nach § 35a SGB V zum Wirkstoff Simoctocog alfa vom 7. Mai 2015 - Beschluss zur Nutzenbewertung nach § 35a SGB V zum Wirkstoff Efmoroctocog alfa vom 16. Juni 2016 Die Vergleichstherapie soll nach dem allgemein anerkannten Stand der medizinischen Erkenntnisse zur Siehe systematische Literaturrecherche
    [Show full text]
  • Clinical Pharmacology of Aminocaproic and Tranexamic Acids
    J Clin Pathol: first published as 10.1136/jcp.s3-14.1.41 on 1 January 1980. Downloaded from J Clin Pathol, 33, Suppl (Roy Coll Path), 14, 41-47 Clinical pharmacology of aminocaproic and tranexamic acids INGA MARIE NILSSON From the Coagulation Laboratory, University ofLund, Allmdnna Sjukhuset, Malmo, Sweden In a systematic search for a substance with anti- arbitrary urokinase units by reference to a standard fibrinolytic properties Okamoto and his group in urokinase preparation.'0 11 Japan found that several mercapto- and amino- Intravenous administration of 10 g EACA or carbonic acids were active. Of these substances 100 mg EACA/kg bodyweight produces an initial epsilon-aminocaproic acid (EACA) had the strong- serum concentration of about 150 mg/100 ml which est antifibrinolytic effect.1 2 The Japanese workers falls to 3 5 mg/100 ml within 3-4 hours (Fig. 1). described it as a plasmin inhibitor in vitro and The biological half life was calculated to be about 77 useful in inhibiting proteolytic enzymes in vivo. They minutes. Andersson et al.8 found that about 70 % of gave EACA in a dose of 10-20 g a day by mouth or the dose given intravenously was excreted in the intravenously to over 100 patients and observed no urine within 24 hours (Fig. 2). McNicol et al.7 toxic effects. Their investigation did not include any found 80-100I% of the given dose in the urine metabolic studies. EACA has since been widely used within 4-6 hours. The EACA is thus concentrated and its mode of action and pharmacokinetics intensively studied.
    [Show full text]
  • Novel T 2019 Vol 3 2.0.Indd
    Novel Treatments in Haemophilia & Other Bleeding Disorders: A Periodic Review 2019 - Issue 2 Irish Haemophilia Society haemophilia.ie October 2019 Contents Foreword Page 03 Abbreviations Page 04 An update on novel treatments for haemophilia A Page 05 Replacement therapies Page 05 Non-replacement therapies Page 08 Gene therapy Page 11 A comment on gene therapy Page 14 An update on novel treatments in haemophilia B Page 15 Replacement therapies Page 15 Non-replacement therapies Page 16 Gene therapy Page 16 A comment on PEG: Same data, same discussion, different regulatory decisions Page 18 An update on novel therapies for inhibitor treatment Page 20 By-passing therapies Page 20 Non-replacement therapies Page 20 Gene therapy Page 22 An update on novel therapies for von Willebrand disease Page 22 An update on novel therapies for rare bleeding disorders Page 23 Disclaimer: This publication is produced by the European Haemophilia Consortium (EHC) primarily as an educational tool for National Member Organisations (NMOs). With the constantly changing therapeutic environment, it is our intention to publish updates on a periodic basis. The information contained, and the views expressed herein constitute the collective input of the EHC New Products Working Group. The EHC does not engage in medical practice and under no circumstances recommends particular treatment for specifi c individuals. The EHC makes no representation, express or implied, that drug doses or other treatment recommendations in this publication are correct. For these reasons it is strongly recommended that individuals seek the advice of a medical adviser and/or consult printed instructions provided by the pharmaceutical company before administering any of the drugs referred to in this publication.
    [Show full text]
  • Tranexamic Acid (4.2) These Highlights Do Not Include All the Information Needed to Use LYSTEDA Safely and Effectively
    HIGHLIGHTS OF PRESCRIBING INFORMATION Hypersensitivity to tranexamic acid (4.2) These highlights do not include all the information needed to use LYSTEDA safely and effectively. See full prescribing information for -----------------------WARNINGS AND PRECAUTIONS------------------------ LYSTEDA. Concomitant use of LYSTEDA with Factor IX complex concentrates, anti-inhibitor coagulant concentrates or all-trans retinoic acid (oral LYSTEDA™ (tranexamic acid) Tablets tretinoin) may increase the risk of thrombosis. (5.1) Initial U.S. Approval: 1986 Visual or ocular adverse effects may occur with LYSTEDA. Immediately discontinue use if visual or ocular symptoms occur. (5.1) ---------------------------------RECENT MAJOR CHANGES--------------------- In case of severe allergic reaction, discontinue LYSTEDA and seek Contraindications (4.1) 10/2013 immediate medical attention. (5.2) Warnings and Precautions (5.1) 10/2013 Cerebral edema and cerebral infarction may be caused by use of LYSTEDA in women with subarachnoid hemorrhage. (5.3) Ligneous conjunctivitis has been reported in patients taking tranexamic ----------------------------INDICATIONS AND USAGE--------------------------- acid. (5.4) LYSTEDA (tranexamic acid) Tablets is an antifibrinolytic indicated for the treatment of cyclic heavy menstrual bleeding. (1) ------------------------------ADVERSE REACTIONS------------------------------- Most common adverse reactions in clinical trials (≥ 5%, and more frequent in ----------------------DOSAGE AND ADMINISTRATION----------------------- LYSTEDA
    [Show full text]