160
July 1975 The Journal of P E D I A T R I C S
Idiopathic thrombocytopenic p u r p u r a - t o treat or not to treat?
CONSCIENTIOUS READERS of pediatric journals may feel that they have come full circle regarding the therapy of idiopathic thrombocytopenic purpura in childhood. The medical progress article by Lusher and Zuelzer' in THE JOURNAL in 1966 reported a series o f 152 children with ITP (with neonatal thrombocytopenia excluded) in which there were no deaths directly attributable to thrombocytopenia. In their summary, the authors of this article indicated that the administration of corticosteroid hormones failed to shorten the course of the disease or hasten restoration of normal platelet counts and indicated that steroid therapy was not required for this illness in children. This particular article followed quite closely upon a brief comment by Schulman'-' in another periodical in which, while admitting that administration of steroids did not reduce the incidence of chronic ITP, he stated that it did elevate the platelet count in approximately 40% o f children and recommended that these drugs be used during the initial period of the illness, since most deaths occurred during this period. Thus two well-qualified pediatric hematologists recorded sharply different opinions within a two-year period. The practicing pediatrician and the hematologist alike might well be confused as to whether or not corticosteroids are truly indicated for this entity. Review of many series of patients with ITP almost uniformly reveals the following: 1. Most, but not all, deaths occur during the first several weeks after the onset of the illness. For example, Choi and McClure ~ recorded a death seven or more weeks after the onset of the illness, Walker and Walker ~ record a death six weeks after onset, and Lammi and Lovric ~ have recorded a death two years following the onset of the illness. 2. Evaluation of virtually all series of ITP has resulted in agreement that the administration of steroids does not reduce the small number of patients who develop chronic ITP and require splenectomy. 3. In all series in which patients treated with steroids have been compared with untreated patients, the evidence
Vol. 87, No. 1, pp. 160-161
indicated that the total duration of the acute illness is not altered by administration of steroids. The article in this issue by Simons and colleagues also indicates that administration of steroids does not change the total duration o f the illness and probably does not reduce the number of patients whose disease becomes chronic. They have, however, added a new criterion for evaluation of results and one which deserves comment. Utilizing serial platelet counts, they were abte to determine that a rise of more than 25,000 platelets from the baseline occurred more rapidly in patients treated with a corticosteroid and that patients achieved a value of 50,000 thrombocytes per cubic millimeter more rapidly with therapy than without it. Since a platelet count above 50,000 is usually considered to be above the dangerous range and results in only mild purpura, the results of this study deserve careful consideration. Their results seem statistically significant and must be weighed against the possible hazards to the patients of corticosteroid administration. The risks of such administration in children are considerably less, particularly when they are utilized for only two or three weeks, than the risks in adults. One should determine, if possible, that the child does not have tuberculosis; in addition we might have some hesitancy in utilizing a corticosteroid in the midst of an epidemic of chickenpox, but most pediatricians would concur that such short-term administration is virtually without morbidity or mortality. There is agreement that long-term therapy is never indicated and may even contribute to prolongation of the thrombocytopenia. If the advice in the current article is followed, we have not truly come full circle, from therapy to no therapy and back to therapy, but have rather reached a middle ground, as actually suggested by Lusher and Zuelzer, 1 in which only some patients with thrombocytopenia are treated on the basis of statistical probability of risk. Since most children who die do so within the first month of the illness, it is likely that many, but not all, of the infrequent deaths from ITP may be prevented by such a regimen. It is also evident from
Volume 87 Number 1
these and m a n y other studies that patients with only modest degrees of thrombocytopenia do not require corticosteroids therapy and that the course of their illness is not altered by administration of these drugs. Last, the diagnosis of ITP must b e firmly established prior to administration of steroids since corticosteroids alone may induce a remission in patients with thrombocytopenia associated with acute leukemia and may mask the diagnosis of this disease for a considerable time. To paraphrase the Ecclesiastes: "A time to treat, a time to refrain from treating." Arthur E. MeElfresh, M.D. Cardinal Glennon Memorial Hospital for Children 1465 S. Grand Blvd. St. Louis, Mo. 63104
Editor's column
16 1
REFERENCES
1. Lusher JM, and Zuelzer WW: Idiopathic thrombocytopepAc purpura in childhood, J PEDIATR68:971, 1966. 2. Schulman I: Diagnosis and treatment: management of idiopathic thrombocytopenic purpura, Pediatrics 33:979, 1964. 3. Choi SI, and McClure PD: Idiopathic thrombocytopenic purpura in childhood, Can Med Assoc J 97:562, 1967. 4. Walker JH, and Walker W: Idiopathic thrombocytopenic purpura in childhood, Arch Dis Child 36:649, 1961. 5. Lammi AT, and Lovric VA: Idiopathic thrombocytopenic purpura: an epidemiologic study, J PEDIATR83:31, 1973.
July 1975 The Journal of P E D I A T R I C S
Idiopathic thrombocytopenic p u r p u r a - t o treat or not to treat?
CONSCIENTIOUS READERS of pediatric journals may feel that they have come full circle regarding the therapy of idiopathic thrombocytopenic purpura in childhood. The medical progress article by Lusher and Zuelzer' in THE JOURNAL in 1966 reported a series o f 152 children with ITP (with neonatal thrombocytopenia excluded) in which there were no deaths directly attributable to thrombocytopenia. In their summary, the authors of this article indicated that the administration of corticosteroid hormones failed to shorten the course of the disease or hasten restoration of normal platelet counts and indicated that steroid therapy was not required for this illness in children. This particular article followed quite closely upon a brief comment by Schulman'-' in another periodical in which, while admitting that administration of steroids did not reduce the incidence of chronic ITP, he stated that it did elevate the platelet count in approximately 40% o f children and recommended that these drugs be used during the initial period of the illness, since most deaths occurred during this period. Thus two well-qualified pediatric hematologists recorded sharply different opinions within a two-year period. The practicing pediatrician and the hematologist alike might well be confused as to whether or not corticosteroids are truly indicated for this entity. Review of many series of patients with ITP almost uniformly reveals the following: 1. Most, but not all, deaths occur during the first several weeks after the onset of the illness. For example, Choi and McClure ~ recorded a death seven or more weeks after the onset of the illness, Walker and Walker ~ record a death six weeks after onset, and Lammi and Lovric ~ have recorded a death two years following the onset of the illness. 2. Evaluation of virtually all series of ITP has resulted in agreement that the administration of steroids does not reduce the small number of patients who develop chronic ITP and require splenectomy. 3. In all series in which patients treated with steroids have been compared with untreated patients, the evidence
Vol. 87, No. 1, pp. 160-161
indicated that the total duration of the acute illness is not altered by administration of steroids. The article in this issue by Simons and colleagues also indicates that administration of steroids does not change the total duration o f the illness and probably does not reduce the number of patients whose disease becomes chronic. They have, however, added a new criterion for evaluation of results and one which deserves comment. Utilizing serial platelet counts, they were abte to determine that a rise of more than 25,000 platelets from the baseline occurred more rapidly in patients treated with a corticosteroid and that patients achieved a value of 50,000 thrombocytes per cubic millimeter more rapidly with therapy than without it. Since a platelet count above 50,000 is usually considered to be above the dangerous range and results in only mild purpura, the results of this study deserve careful consideration. Their results seem statistically significant and must be weighed against the possible hazards to the patients of corticosteroid administration. The risks of such administration in children are considerably less, particularly when they are utilized for only two or three weeks, than the risks in adults. One should determine, if possible, that the child does not have tuberculosis; in addition we might have some hesitancy in utilizing a corticosteroid in the midst of an epidemic of chickenpox, but most pediatricians would concur that such short-term administration is virtually without morbidity or mortality. There is agreement that long-term therapy is never indicated and may even contribute to prolongation of the thrombocytopenia. If the advice in the current article is followed, we have not truly come full circle, from therapy to no therapy and back to therapy, but have rather reached a middle ground, as actually suggested by Lusher and Zuelzer, 1 in which only some patients with thrombocytopenia are treated on the basis of statistical probability of risk. Since most children who die do so within the first month of the illness, it is likely that many, but not all, of the infrequent deaths from ITP may be prevented by such a regimen. It is also evident from
Volume 87 Number 1
these and m a n y other studies that patients with only modest degrees of thrombocytopenia do not require corticosteroids therapy and that the course of their illness is not altered by administration of these drugs. Last, the diagnosis of ITP must b e firmly established prior to administration of steroids since corticosteroids alone may induce a remission in patients with thrombocytopenia associated with acute leukemia and may mask the diagnosis of this disease for a considerable time. To paraphrase the Ecclesiastes: "A time to treat, a time to refrain from treating." Arthur E. MeElfresh, M.D. Cardinal Glennon Memorial Hospital for Children 1465 S. Grand Blvd. St. Louis, Mo. 63104
Editor's column
16 1
REFERENCES
1. Lusher JM, and Zuelzer WW: Idiopathic thrombocytopepAc purpura in childhood, J PEDIATR68:971, 1966. 2. Schulman I: Diagnosis and treatment: management of idiopathic thrombocytopenic purpura, Pediatrics 33:979, 1964. 3. Choi SI, and McClure PD: Idiopathic thrombocytopenic purpura in childhood, Can Med Assoc J 97:562, 1967. 4. Walker JH, and Walker W: Idiopathic thrombocytopenic purpura in childhood, Arch Dis Child 36:649, 1961. 5. Lammi AT, and Lovric VA: Idiopathic thrombocytopenic purpura: an epidemiologic study, J PEDIATR83:31, 1973.
