title : Intravenous Immunoglobulins in Clinical Practice author : Lee, Martin L. publisher : Informa Healthcare isbn10 | asin : 0824798813 print isbn13 : 9780824798819 ebook isbn13 : 9780585157924 language : English subject Immunoglobulins Therapeutic use, Intravenous therapy, Immunoglobulins, Intravenous therapeutic use. publication date : 1997 lcc : RM282.I44I586 1997eb ddc : 615/.37 subject : Immunoglobulins Therapeutic use, Intravenous therapy, Immunoglobulins, Intravenous therapeutic use. Page i Intravenous Immunoglobulins in Clinical Practice Edited By Martin L. Lee School of Public Health University of California Los Angeles, California Vibeke Strand Stanford University San Francisco, California MARCEL DEKKER, INC. NEW YORK BASEL HONG KONG Page ii Library of Congress Cataloging-in-Publication Data Intravenous immunoglobulins in clinical practice / edited by Martin L. Lee, Vibeke Strand. p. cm. Includes index. ISBN 0-8247-9881-3 (hardcover : alk. paper) 1. ImmunoglobulinsTherapeutic use. 2. Intravenous therapy. I. Lee, Martin L. II Strand, Vibeke. [DNLM: 1. Immunoglobulins, Intravenoustherapeutic use. QW 601 I616 1997] RM282.I44I586 1997 615'.37dc21 DNLM/DLC for Library of Congress 97-25515 CIP The publisher offers discounts on this book when ordered in bulk quantities. For more information, write to Special Sales/Professional Marketing at the address below. This book is printed on acid-free paper. Copyright © 1997 by Marcel Dekker, Inc. All Rights Reserved. Neither this book nor any part may be reproduced or transmitted in any form or by any means, electronic or mechanical, including photocopying, microfilming, and recording, or by any information storage and retrieval system, without permission in writing from the publisher. MARCEL DEKKER, INC. 270 Madison Avenue, New York, New York 10016 http://www.dekker.com Current printing (last digit): 10 9 8 7 6 5 4 3 2 1 PRINTED IN THE UNITED STATES OF AMERICA Page iii PREFACE In the 1940s Cohn and colleagues developed a relatively straightforward chemical process for fractionating human blood into many of its significant component proteins, thus enabling the production of the first immunoglobulin concentrates (although suitable for intramuscular use only). In the following decade, Bruton and others recognized the genetic basis of various types of primary immunodeficiency syndromes and further characterized them. These two discoveries allowed for the regular treatment of patients using replacement infusions of human immunoglobulins and the concomitant improvement in quality of life and, ultimately, survival. Subsequently, specific immunoglobulin preparations were produced for treatment of or prophylaxis against specific pathogens such as hepatitis B, polio, tetanus, and pertussis. All of these so-called hyperimmune globulins were administered by the intramuscular route. It became quite clear that this means of administration was not adequate for both the provider and the patient. Injections were quite painful; doses were limited in size and frequency; muscle proteases degraded much of the infused immune globulins; and the remaining protein reached the circulation only after significant delay. Attempts to inject material directly into the vasculature proved to be dangerous, and occasionally catastrophic, apparently as a result of the IgG aggregates that formed as part of the fractionation process. Subsequent developments employing first partial enzyme digestion (using proteases such as pepsin and papain) and then improvements in the fractionation process allowed for the ultimate production of true intravenous immunoglobulin (IVIG) concentrates. Since the late 1970s when these concentrates became widely available, their use has grown exponentially. The serendipitous discovery by Imbach, Barandun, and colleagues in 1980 that IVIG could reverse the autoimmune thrombocytopenia in a young patient with severe chronic ITP and secondary hypogammaglobulinemia opened another avenue of applications: the treatment of autoimmune diseases. Our goal in compiling this volume was to summarize critically the large array of clinical literature available on the use of IVIG preparations. Indeed, a review of MEDLINE citations since 1980 showed more than 1800 entries. Much of the work over the past several years has involved controlled clinical trials, putting research in this area on a firm, scientific footing. This is the focus of our book. In recent years, studies have shown that IVIG may be useful in treating various primary and secondary immunodeficiencies. With regard to the latter, successful trials have been conducted in AIDS patients, premature neonates, individuals with multiple myeloma and chronic lymphocytic leukemia, bone marrow and liver transplantees, patients after high-risk (for infection) abdominal surgeries, and thermal burn victims. Page iv A vast literature has also developed on the prophylaxis and treatment of numerous autoimmune diseases. Although the mechanisms of action of IVIG are incompletely understood, the range of successful applications is remarkable. Nonetheless, the number of large-scale controlled studies in this area remains small. This is changing, particularly with the recent publication by van der Meché and colleagues of a successful trial of IVIG in the treatment of acute Guillain- Barré syndrome. In this book, many of the leading authorities on clinical applications of IVIG in their respective fields of medical research discuss work done to date. We sincerely believe that the reviews contained herein are comprehensive, but recognize the explosive growth of this literature. This volume will serve as a good overview for both clinician and researcher wishing to survey current information available on the clinical use of IVIG. We are grateful to so many people for their invaluable assistance and support with this project. We want to offer our sincere gratitude to the contributors to this book. Their efforts clearly demonstrated a commitment to furthering knowledge about this important therapeutic agent. We also wish to express our appreciation to Ms. Shirley Sutjiadi for providing invaluable administrative assistance in organizing this volume, and Dr. Ed Gomperts and Dr. Gordon Bray for providing many of the resources needed to complete our effort. And, of course, we owe our families a large debt of gratitude. M. L. would like to thank his wife, Marilyn, and his two sons, Eliot and Danny, for their love and support. V. S. appreciates all the encouragement and understanding her husband, Jack, provided. MARTIN L. LEE VIBEKE STRAND Page v CONTENTS Preface iii Contributors ix I. Overview 1. Pharmacokinetics of Intravenous Immunoglobulin Preparations Andreas Morell 1 2. Pharmacoeconomics of Intravenous Immunoglobulin Martin L. Lee and Vibeke Strand 19 3. Proposed Mechanisms for the Efficacy of Intravenous Immunoglobulin Treatment Vibeke Strand 23 4. Production and Properties of Intravenous Immunogloblins John A. Hooper 37 5. Nonviral Side Effects of Intravenous Immunoglobulins Mario Dicato, C. Duhem, and F. Ries 57 6. Viral Safety of IVIG Peng Lee Yap 67 7. Alternative Methods for the Administration of Intravenous Immunoglobulins Martin L. Lee 107 II. Infectious Disease Applications 8. IVIG in Bone Marrow Transplantation Maurice J. Wolin and Robert Peter Gale 113 9. Use of Intravenous Immunoglobulins for the Prevention and Treatment of Viral Infections in Solid Organ Transplantation Jeffrey A. DesJardin and David R. Snydman 119 Page vi 10. Intravenous Immunoglobulin Use in the Newborn Infant: Treatment and Prevention of Infection Rajam S. Ramamurthy 135 11. Use of Intravenous Immunoglobulins in High-Risk Surgical Procedures and in Posttrauma Patients Giorgio Zanetti and Michel-Pierre Glauser 151 12. Intravenous Gammaglobulin Regimen for HIV-Infected Children: Infection Prophylaxis and Immunomodulation Arye Rubinstein 159 13. Use of Intravenous Immune Globulin in Adults with HIV Disease David J. Rechtman 167 14. Treatment of Primary Immunodeficiency Diseases with Gammaglobulin Richard I. Schiff 175 15. Intravenous Immunoglobulin Treatment for IgG Subclass Deficiency Thomas F. Smith 193 16. Prevention of Infections in B-Cell Lymphoproliferative Diseases Helen Griffiths and Helen Chapel 203 17. Etiology and Prevention of Infection Following Thermal Injury Khan Z. Shirani, George M. Vaughan, Albert T. McManus, Arthur D. Mason, Jr., and Basil A. Pruitt, Jr. 225 18. Prevention and Treatment of Viral Infection Martha M. Eibl and Hermann M. Wolf 243 19. Intravenous Immunoglobulin Therapy of Neonates with Nonpolio Enteroviral Infections Harry L. Keyserling 257 20. Treatment of Chronic Fatigue Syndrome Andrew R. Lloyd and Denis Wakefield 267 III. Autoimmune Disease Applications: Pediatric 21. Intravenous Gammaglobulin Therapy for Autoimmune Thrombocytopenic Purpura, Neutropenia, and Hemolytic Anemia James B. Bussel 275 Page vii 22. Use of IVIG in Kawasaki Syndrome Marian E. Melish 293 23. Juvenile Rheumatoid Arthritis Thomas A. Griffin and Edward H. Giannini 309 24. Intravenously Administered Gammaglobulin for the Prevention or Modulation of Insulin-Dependent Diabetes Mellitus John M Dwyer and Stephen Colagiuri 317 IV. Autoimmune Disease Applications: Adult 25. Advances in the Treatment of Alloimmune-Mediated Platelet Disorders with Intravenous Immunoglobulin Thomas S. Kickler 327 26. Guillain-Barré Syndrome Frans G. A. van der Meché and Pieter A. van Doorn 337 27. Chronic Inflammatory Demyelinating Polyneuropathy Pieter A. van Doorn and Frans G. A. van der Meché 349 28. Intravenous Immunoglobulin in the Management of Myasthenia Gravis David Grob 363 29. Multiple Sclerosis Anat Achiron 381 30. Polymyositis/Dermatomyositis Lori B. Tucker and Earl D. Silverman 399 31. Use of Intravenous Immunoglobulin in Therapy of Rheumatoid Arthritis David E. Yocum 409 32. Treatment of Systemic Lupus Erythematosus with Pooled Human Intravenous Immunoglobulin Stanley C. Jordan 415 33. Intravenous Immunoglobulin Therapy of Systemic Necrotizing Vasculitis Leonard H. Calabrese 425 34. Lambert-Eaton Myasthenic Syndrome John Newsom-Davis 431 Page viii 35. Intravenous Gammaglobulin in the Treatment of Recurrent Pregnancy Loss Ann L. Parke 439 36. Intravenous Immunoglobulin and Other Autoimmune Diseases Martin L. Lee 447 37. Intravenous Immunoglobulin Therapy in Idiopathic Inflammatory Bowel Diseases Douglas S. Levine 451 V. Hyperimmunoglobulins 38. Development of Hyperimmune Immunoglobulins William J. Landsperger and Roger Lundblad 467 Index 503 Page ix CONTRIBUTORS Anat Achiron, MD., Ph.D. Director, Multiple Sclerosis Center, Sheba Medical Center, Tel-Hashomer, Israel James B. Bussel, M.D. Associate Professor, Department of Pediatrics, Division of Hematology/Oncology, The New York Hospital-Cornell Medical Center, New York, New York Leonard H. Calabrese, D.O. Vice Chairman and Head of Clinical Immunology, Department of Rheumatic and Immunologic Disease, Cleveland Clinic Foundation, Cleveland, Ohio Helen Chapel, M.D., M.R.C.P., F.R.C.Path. Consultant Immunologist and Senior Clinical Lecturer, Department of Immunology, Oxford Radcliffe Hospital, Oxford, England Stephen Colagiuri, M.D. The University of New South Wales, Sydney, Australia Mario Dicato, M.D. Central Hospital of Luxembourg, Luxembourg, Belgium Jeffrey A. DesJardin, M.D. Department of Geographic Medicine and Infectious Diseases, New England Medical Center and Tufts University School of Medicine, Boston, Massachusetts C. Duhem, M.D. Central Hospital of Luxembourg, Luxembourg, Belgium John M Dwyer, M.D., B.S., F.R.A.C.P., Ph.D. Professor, Department of Medicine, The University of New South Wales, Sydney, Australia Martha M. Eibl, M.D. Professor, Institute of Immunology, University of Vienna, Vienna, Austria Robert Peter Gale, M.D., Ph.D., F.A.C.P. Corporate Director, Blood Cell and Bone Marrow Transplantation, Salick Health Care, Inc., Los Angeles, California Edward H. Giannini, M.Sc. Dr. P.H. Professor, William S. Rowe Division of Rheumatology, Department of Pediatrics, Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio [...]... BM, Kauffman R Pharmacokinetics and effectiveness of intravenous immunoglobulins in neonates J Pediatr 1988; 112:325326 33 Weisman LE, Fischer GW, Hemming VG, et al Pharmacokinetics of intravenous immunoglobulin (Sandoglobulin) in neonates Pediatr Infect Dis 1986; 5(suppl 3):185188 34 Weisman LE, Fischer GW, Marinelli P, et al Pharmacokinetics of intravenous immunoglobulin in neonates Vox Sang 1989;... Wedgwood RJ, et al Survival of antigen-specific antibody following administration of intravenous immunoglobulin in patients with primary immunodeficiency diseases Monogr Allergy 1988; 23:225235 22 Eibl M Intravenous immunoglobulins: Clinical and experimental studies In: Alving BM, Finlayson JS, eds Immunoglobulins: Characteristics and Uses of Intravenous Preparations Page 17 Bethesda: U.S Dept Health... weight neonates Pediatr Infect Dis J 1989; 8:759763 38 Kyllonen KS, Clapp DW, Kliegman RM, et al Dosage of intravenously administered immune globulin and dosing intervals required to maintain target levels of immunoglobulin G in low birth weight infants J Pediatr 1989; 115:10131016 39 Kinney J, Mundorf L, Gleason C, et al Efficacy and pharmacokinetics of intravenous immune globulin administered to high-risk... low-dose intravenous immunoglobulin in hypogammaglobulinemia and chronic lung disease Lancet 1987; 2:10751077 26 Leen CLS, Yap PL, McClelland DBL Increase of serum immunoglobulin level into the normal range in primary hypogammaglobulinemia by dosage individualization of intravenous immunoglobulin Vox Sang 1986; 51:278286 27 NIH Consensus Development Conference 1990 Intravenous immunoglobulin, prevention... of single and multiple infusion of 5S intravenous immunoglobulin Vox Sang 1995; 68:58 12 Barandun S, Castel V, Makula MF, et al Clinical tolerance and catabolism of plasmin-treated γ-globulin for intravenous application Vox Sang 1975; 28:157175 13 Janeway CA, Merler E, Rosen FS, et al Intravenous gamma-globulin Metabolism of gamma-globulin fragments in normal and agammaglobulinemic persons N Engl J... IgG, IgG subclasses, and type specific antibodies in immunodeficient patients Immunol Invest 1991; 20:193198 20 Ochs HD, Morell A, Skvaril F, et al Survival of IgG subclasses following administration of intravenous gammaglobulin in patients with primary immunodeficiency diseases In: Morell A, Nydegger UE, eds Clinical use of intravenous immunoglobulins New York: Academic Press, 1986:7785 21 Fischer... B Clinical use of a new pH 4.25 intravenous immunoglobulin preparation (Gamimune-N) J Infect 1987; 15(suppl 1):2937 18 Mankarious S, Lee M, Fischer S, et al The half-lives of IgG subclasses and specific antibodies in patients with primary immunodeficiency who are receiving intravenously administered immunoglobulin J Lab Clin Med 1988; 112:634640 19 Lee ML, Mankarious S, Ochs H, et al The pharmacokinetics... the catabolism of immunoglobulins Lancet 1966; 2:10871093 29 Weiner AS The half-life of passively acquired antibody globulin molecules in infants J Exp Med 1951; 94:213221 30 Wilson CB Immunologic basis for increased susceptibility of the neonate to infection J Pediatr 1986; 108:112 31 Chirico G, Rondini G, Plebani A, et al Intravenous gammaglobulin therapy for prophylaxis of infection in high risk neonates... TA, Terry WD Familial hypercatabolic hypoproteinemia A disorder of endogenous catabolism of albumin and immunoglobulin J Clin Invest 1990; 86:20932098 4 Morell A, Terry WD, Waldmann TA Metabolic properties of IgG subclasses in man J Clin Invest 1970; 49:673680 5 Schiff RI Intravenous immunoglobulins for treatment of antibody deficiencies In: Good RA, Lindenlaub E, eds The Nature, Cellular, and Biochemical... levels Extrapolation of the final slope -b1 to the ordinate and intercept C0 are explained in the text Both the α and β phases may be influenced by intrinsic IgG synthesis of the patient and by extrinsic carryover IgG from previous IVIG infusions (see Refs 5,24) Page 4 Table 1 Pharmacokinetics of Normal IgG in Normal Individuals (mean values ± 1 SD) Total IgG Fraction (%) of intravascular pool catabolized . Immunoglobulins Therapeutic use, Intravenous therapy, Immunoglobulins, Intravenous therapeutic use. Page i Intravenous Immunoglobulins in Clinical Practice Edited By Martin L. Lee School of Public. title : Intravenous Immunoglobulins in Clinical Practice author : Lee, Martin L. publisher : Informa Healthcare isbn10 | asin : 0824798813 print isbn13 : 9780824798819 ebook. California MARCEL DEKKER, INC. NEW YORK BASEL HONG KONG Page ii Library of Congress Cataloging -in- Publication Data Intravenous immunoglobulins in clinical practice / edited by Martin L. Lee, Vibeke