Background: Intravenous immunoglobulin (IVIG) is the mainstay therapy in human immune deficiency states characterized by qualitative and quantitative reductions in B cells. In addition, however, there is widespread use of IVIG in a number of other areas, including neuroimmunologic, infectious, dermatologic, hematologic, autoimmune, inflammatory and idiopathic disorders. In many of these cases, there are little objective data to support the use.
Methods: We performed a review of more than 400 publications in PubMed using the key words 'intravenous immunoglobulin' and excluded publications that focused on immune deficiency, for which the indication for IVIG is already clear.
Results: For a number of off-label indications, there is significant evidence of efficacy and IVIG has become the standard of care for many clinical syndromes other than immune deficiency. In some conditions, however, the data have not been well controlled or randomized and are often limited to case reports that are difficult to interpret. Although the critical shortage of IVIG of the last decade is no longer an issue, IVIG is expensive and not without risk. The use of IVIG should be based not only on clinical data, but also, and especially, on the biological rationale for its use.
Conclusions: The appropriate use of IVIG is an important issue that is difficult to resolve, and will continue to challenge clinicians based on expense and potentially limited supply, including the intrinsic limitations of donor plasma. The establishment of national and international voluntary registries to report use of IVIG in disorders for which evidence is lacking would be a first step toward facilitating randomized, controlled clinical trials.