Standing Council on Health Criteria for the clinical use of intravenous immunoglobulin in Australia Second Edition July 2012 Criteria for the clinical use of intravenous immunoglobulin in Australia Second Edition July 2012 Disclaimer: Important information about this document This document is not a clinical practice guideline. It intends only to provide information about criteria for accessing intravenous immunoglobulin funded under the National Blood Arrangements. Any advice relating to other forms of treatment relevant to the conditions in The Criteria for the clinical use of intravenous immunoglobulin in Australia (the Criteria) have not been subject to a systematic review and should not be relied upon to guide treatment. Patients and doctors should not use this document as a substitute for expert medical guidance and advice. The relevance and appropriateness of information in this document depends, amongst other things, on an accurate diagnosis, the severity of the condition being properly ascertained, the individual response to diagnostic tests and therapies, and other relevant circumstances in each case. The Criteria was first published in 2007 after a systematic review that was completed in mid-2006. Another systematic review, of a limited number of indications, was then undertaken in 2010-11 to update the Criteria. This review resulted in the addition of a small number of indications, removal of a small number of indications and a rewording of a limited number of indications. Inclusion of indications in the Criteria is based where possible upon systematic review of the evidence. In the absence of published evidence, information and access criteria are based on clinical advice provided to the development group by clinical colleges, clinical societies and individual experts. Each person involved in developing this document, and their employer where they are involved as an employee, or the organisation they represent where they are involved in a representative capacity, expressly disclaims and accepts no responsibility for any consequences arising from relying upon the information or recommendations contained herein. II This document was prepared under the auspices of the Jurisdictional Blood Committee for and on behalf of the Standing Council on Health (formerly the Australian Health Ministers’ Conference). The Jurisdictional Blood Committee would like to acknowledge and thank those who contributed to the compilation of the first and second editions of the document. In particular, we would like to thank the members of the original IVIg Working Party and the National IVIg Criteria Review Working Group. We would also like to thank all individuals and organisations that provided submissions to the 2010–11 Criteria Review. Principal clinical advisers were Associate Professor John Gibson, Associate Professor Andrew Kornberg and Associate Professor Sean Riminton. Clinical representatives from; the Australian Society of Clinical Immunology and Allergy, the Australian & New Zealand Association of Neurology, the Haematology Society of Australia and New Zealand and the Australian Red Cross Blood Service Transfusion Medicine Team also contributed to this review. Many other clinical experts gave generously of their time and expertise. All contributions are gratefully acknowledged. It is intended that this document will be updated periodically. This document and its updates will be available on the National Blood Authority website at www.nba.gov.au. This document may be cited as Jurisdictional Blood Committee, for and on behalf of the Australian Health Ministers’ Conference. Criteria for the clinical use of intravenous immunoglobulin in Australia. Second Edition. Canberra: Commonwealth of Australia, 2012. Criteria for the clinical use of intravenous immunoglobulin in Australia III ISBN 978-0-9872519-0-9 © Commonwealth of Australia, National Blood Authority 2012 Save for the content within these criteria supplied by third parties, the National Blood Authority logo, the Commonwealth Coat of Arms, and any material protected by a trade mark, the National Blood Authority has applied the Creative Commons Attribution 3.0 Australia Licence http://creativecommons.org/licenses/by-nc- sa/3.0/au/ Attribution-Noncommercial-No Derivatives CC BY-NC-ND. This licence allows redistribution of the work in its current form only. You are allowed to download and share the work as long as you credit the original creator/s (and any other nominated parties). You cannot change the material in any way and you cannot use it commercially. IV Contents Foreword 2 Structure of this document 3 Maintaining the currency of this document 4 National IVIg Criteria Review Working Group 4 Further Information 5 Index of conditions 6 Executive summary 11 Access to intravenous immunoglobulin in Australia 11 Production and safety 12 Rationale for developing criteria for intravenous immunoglobulin use 12 Conditions considered for IVIg therapy 13 1. Introduction 16 National Blood Agreement 16 Governance arrangements for the supply and management of blood products in Australia 17 Availability of intravenous immunoglobulin in Australia 19 Intravenous Immunoglobulin Standing Offer 21 2. Production of normal immunoglobulins 24 Plasma fractionation 24 Product safety and regulation 25 Virus and prion removal 27 Intravenous immunoglobulin products in Australia 28 Criteria for the clinical use of intravenous immunoglobulin in Australia V 3. Intravenous immunoglobulin supply and demand 30 International 30 Domestic 30 World trends 33 IVIg demand management 34 4. Development and maintenance of the Criteria 36 Development of first edition 36 2008 clarification process 37 2010–11 Criteria Review 37 Assessment of evidence 39 Condition proforma 41 Replacement and immunomodulation therapy 42 Dosing 44 Research priorities 44 Maintenance 44 5. Conditions for which IVIg has an established therapeutic role 46 6. Conditions for which IVIg has an emerging therapeutic role 104 7. Conditions for which IVIg use is in exceptional circumstances only 184 8. Conditions for which IVIg use is not supported 214 Acronyms and abbreviations 218 Glossary of terms 222 Bibliography 225 VI Appendix A: Funding policy statement 228 Policy principles 228 Funding for IVIg 228 Ongoing review of funded conditions 229 Appendix B: National Stewardship Expectations 230 Appendix C: Development of the Criteria for the clinical use of IVIg in Australia (First Edition) 233 National workshop 233 Systematic reviews 234 Jurisdictional Blood Committee IVIg Working Party 235 Development and consultation 237 Sources of information and assessment 241 Contributors 243 Finalisation and approval 243 Appendix D: 2010–11 Criteria Review 244 National IVIg Criteria Review Working Group 244 Formal submission process 246 Review of submissions 247 Systematic review 248 Development of revised wording 249 Consensus process 250 Public consultation 251 Finalisation and approval 252 Appendix E: Alphabetical index of conditions 253 Index 262 Criteria for the clinical use of intravenous immunoglobulin in Australia VII List of figures Figure 1 Plasma sent to CSL Biotherapies Australia for fractionation 31 Figure 2 IVIg grams issued in Australia 32 Figure 3 Australian IVIg issued in g/1000 head of population 33 List of tables Table 1 Level of evidence categories 40 Table 2 Conditions for which IVIg has an established therapeutic role as immunoglobulin-replacement therapy 46 Table 3 Conditions for which IVIg has an established therapeutic role as immunomodulation therapy 47 Table 4 Conditions for which IVIg has an emerging therapeutic role as immunoglobulin replacement therapy 105 Table 5 Conditions for which IVIg has an emerging therapeutic role as immunomodulation therapy 105 Table 6 Conditions for which IVIg is used as immunoglobulin replacement therapy in exceptional circumstances 185 Table 7 Conditions for which IVIg is used in immunomodulation therapy in exceptional circumstances 186 Table 8 Conditions for which IVIg use is not supported 214 VIII Foreword Foreword This document is to assist clinicians and transfusion medicine professionals identify the conditions and circumstances for which the use of intravenous immunoglobulin (IVIg) is able to be accessed under the National Blood Arrangements. IVIg is a precious biological product and, as such, its use should be consistent with the evidence base and prescribed for the treatment of patients who are likely to benefit from IVIg therapy and for whom there are no safe and effective alternative treatments. The growth in demand for IVIg prompted action by Australian governments to ensure it is reserved for use in those patients with the greatest need. There is not an exact alignment between Therapeutic Goods Administration registered indications and the indications listed in the Criteria. Where safe and effective alternative therapies are available, the alternative product should be used in preference to IVIg. In addition, IVIg is used to treat a growing number of diseases where immunomodulation or immunoglobulin replacement therapy is of benefit but the treatment indication does not have regulatory approval. Some of these uses of IVIg have a foundation in the medical literature and others are supported by clinical consensus but have a less conclusive basis in evidence. The Criteria is based on evidence identified through systematic reviews of the literature and the opinions of clinical experts. In conjunction with government policy, this publication may be used to identify those conditions and