- Email: [email protected]
Intravenous Immunoglobulin: Prevention and Treatment of Disease
Intravenous Immunoglobulin: Prevention and Treatment oC Disease SUMMARY OF THE NIH CONSENSUS DEVELOPMENT CONFERENCE
MMUNOGLOBULINS are disease-fighting proteins, or antibodies, that are produced by the immune system. People with certain immune system deficiencies may not produce immunoglobuIins and are therefore susceptible to a variety of infections. Since 1952, immunoglobulin preparations made from human blood have been available to boister the immune system. When first developed, they were administered intramuscularly (injected into a muscle) in order to prevent or treat the variety of infections that can occur in these patients. In the last decade, seven preparations of immunoglobulin made to be injected intravenously (into the vein) have become available in the United States. Use of intravenous immunoglobulin (lVIG) has rapidly increased in the past several years as a result of improvements in its preparation and the unexpected benefits it showed in the treatment of certain diseases. However, important questions regarding its use still remain. In an effort to make recommendations regarding the safe and effective use of lVIG, the National Institutes of Health (NIH) held a Consensus Development Canference on Intravenaus Immunoglobulin: Prevention and Treatment of Disease, May 21-23, 1990. A 13-member panel developed a consensus statement after considering scientific presentations and discussions from several physicians, scientists, health care professionals, and others. The following are the panel's conclusions. IVIG is a safe and effective replacement therapy for people with primary immunodeficiencies, ie, immune deficiencies not due to other diseases, in which lack af production of antibodies against eammon infeetious agents can be demonstrated. The usefulness of IVIG in pediatrie acquired immune deficiency syndrome (AIDS) has not yet heen documented, but it will be evaluated at the eonclusion of two angoing NIH-supported clinical triais. Currently available data are insufficient to support the use of IVIG to prevent late onset infections in low-birth-weight premature infants. !ts use in
I
Transfusion Medicine Reviews, Vol
v, No 3 (July).
1991: pp 171-172
treatment of neonatal infections also is not supported by available data. IVIG can significantly reduce the number af infeetions that occur in eertain patients with chronic lymphocytic leukemia. Although its use has no effect on long-term survival, IVIG ean decrease the amount of time these patients spend in the hospital or canvalescing. In immunosuppressed patients who have received bone marrow transplants, IVIG is useful in preventing and treating certain infeetions and may help in preventing graft-versus-host disease, in which transplanted cells attack the tissues of the recipient. A rapid increase in the bIood platelet levels of children with acute immune thrombocytopenic purpura (lTP) , an immune disorder marked by blood platelet destruction, can occur with the use of IVIG. IVIG is not as effective in the treatment of adults with ITP, although it can increase the number of platelets in the blood. It is most useful in adults in special situations requiring an acute increase in platelet count, such as immediately before surgery. IVIG in conjunction with aspirin should be the standard of care for preventing damage to the coronary arteries in children with Kawasaki syndrome, a condition of unknown cause marked by acute high fever, rash, and conjunctivitis. Clinical triais in the use of IVIG are warranted in certain diseases of the nervous system such as Guillain-Bam~ syndrome, an inflammation of the nervous system that causes paralysis, and in intractabIe seizure disorder. All IVIG preparations are safe and effective in treating the conditions for which they are licensed; however, the efficacy of various preparations in treating other conditions remains to be established. Given the large number of conditions for which IVIG may have potential value, the prescribing physician should be aware of the demonstrated efficacy of each preparation. Effective regimens have heen developed for primary immunodeficiencies and secondary immuno171
172
deficiencies, ie, immune deficiencies resultingfrom other diseases such as chronic lymphocytic leukemia, as well as for ITP and Kawasaki syndrome. However, optimal dosages and treatment schedules still need to be established for patients who may benefit from IVIG therapy. The risks of IVIG therapy are minima!. Adverse events, which are rare, can often be alleviated by reducing the rate or volume of infusion. Appropriate means should be developed to monitor IVIG preparations for the potential presence of new or unexpected disease-causing agents, such as the recently identified hepatitis-C virus. The panel identified the following areas for fu-
INTRAVENOUS IMMUNOGLOBULIN
ture research: (1) discem how IVIG works in the body; (2) when possible, compare the effectiveness of IVIG preparations; (3) design more specifically targeted immunoglobulin preparations; (4) determine cost-effectiveness and effect on quality of life for patients receiving IVIG; and (5) examine long-term effectiveness of IVIG. Free single copies of the complete NIH Consensus Statement on Intravenous Immunoglobulin: Prevention and Treatment of Disease may be ordered from the Office of Medical Applications of Research, National Institutes of Health, Building l, Room 260, 9000 Rockville Pike, Bethesda, MD 20892, or phone 301-496-1143.
MMUNOGLOBULINS are disease-fighting proteins, or antibodies, that are produced by the immune system. People with certain immune system deficiencies may not produce immunoglobuIins and are therefore susceptible to a variety of infections. Since 1952, immunoglobulin preparations made from human blood have been available to boister the immune system. When first developed, they were administered intramuscularly (injected into a muscle) in order to prevent or treat the variety of infections that can occur in these patients. In the last decade, seven preparations of immunoglobulin made to be injected intravenously (into the vein) have become available in the United States. Use of intravenous immunoglobulin (lVIG) has rapidly increased in the past several years as a result of improvements in its preparation and the unexpected benefits it showed in the treatment of certain diseases. However, important questions regarding its use still remain. In an effort to make recommendations regarding the safe and effective use of lVIG, the National Institutes of Health (NIH) held a Consensus Development Canference on Intravenaus Immunoglobulin: Prevention and Treatment of Disease, May 21-23, 1990. A 13-member panel developed a consensus statement after considering scientific presentations and discussions from several physicians, scientists, health care professionals, and others. The following are the panel's conclusions. IVIG is a safe and effective replacement therapy for people with primary immunodeficiencies, ie, immune deficiencies not due to other diseases, in which lack af production of antibodies against eammon infeetious agents can be demonstrated. The usefulness of IVIG in pediatrie acquired immune deficiency syndrome (AIDS) has not yet heen documented, but it will be evaluated at the eonclusion of two angoing NIH-supported clinical triais. Currently available data are insufficient to support the use of IVIG to prevent late onset infections in low-birth-weight premature infants. !ts use in
I
Transfusion Medicine Reviews, Vol
v, No 3 (July).
1991: pp 171-172
treatment of neonatal infections also is not supported by available data. IVIG can significantly reduce the number af infeetions that occur in eertain patients with chronic lymphocytic leukemia. Although its use has no effect on long-term survival, IVIG ean decrease the amount of time these patients spend in the hospital or canvalescing. In immunosuppressed patients who have received bone marrow transplants, IVIG is useful in preventing and treating certain infeetions and may help in preventing graft-versus-host disease, in which transplanted cells attack the tissues of the recipient. A rapid increase in the bIood platelet levels of children with acute immune thrombocytopenic purpura (lTP) , an immune disorder marked by blood platelet destruction, can occur with the use of IVIG. IVIG is not as effective in the treatment of adults with ITP, although it can increase the number of platelets in the blood. It is most useful in adults in special situations requiring an acute increase in platelet count, such as immediately before surgery. IVIG in conjunction with aspirin should be the standard of care for preventing damage to the coronary arteries in children with Kawasaki syndrome, a condition of unknown cause marked by acute high fever, rash, and conjunctivitis. Clinical triais in the use of IVIG are warranted in certain diseases of the nervous system such as Guillain-Bam~ syndrome, an inflammation of the nervous system that causes paralysis, and in intractabIe seizure disorder. All IVIG preparations are safe and effective in treating the conditions for which they are licensed; however, the efficacy of various preparations in treating other conditions remains to be established. Given the large number of conditions for which IVIG may have potential value, the prescribing physician should be aware of the demonstrated efficacy of each preparation. Effective regimens have heen developed for primary immunodeficiencies and secondary immuno171
172
deficiencies, ie, immune deficiencies resultingfrom other diseases such as chronic lymphocytic leukemia, as well as for ITP and Kawasaki syndrome. However, optimal dosages and treatment schedules still need to be established for patients who may benefit from IVIG therapy. The risks of IVIG therapy are minima!. Adverse events, which are rare, can often be alleviated by reducing the rate or volume of infusion. Appropriate means should be developed to monitor IVIG preparations for the potential presence of new or unexpected disease-causing agents, such as the recently identified hepatitis-C virus. The panel identified the following areas for fu-
INTRAVENOUS IMMUNOGLOBULIN
ture research: (1) discem how IVIG works in the body; (2) when possible, compare the effectiveness of IVIG preparations; (3) design more specifically targeted immunoglobulin preparations; (4) determine cost-effectiveness and effect on quality of life for patients receiving IVIG; and (5) examine long-term effectiveness of IVIG. Free single copies of the complete NIH Consensus Statement on Intravenous Immunoglobulin: Prevention and Treatment of Disease may be ordered from the Office of Medical Applications of Research, National Institutes of Health, Building l, Room 260, 9000 Rockville Pike, Bethesda, MD 20892, or phone 301-496-1143.