Treatment of
immune thrombocytopenic purpura (
ITP), the most common
bleeding disorder of childhood, is a controversial subject for most practitioners. Diagnosis and management of
ITP has historically been based primarily on expert opinion rather than on evidence. Due to a paucity of carefully conducted clinical trials in children, the management of
ITP varies widely, ranging from observation only, to aggressive management with
intravenous immunoglobulin (
IVIG), intravenous
anti-D rhesus (Rh)0
immunoglobulin (IV RhIG),
corticosteroids, and
splenectomy. To address the controversies, the American Society of Hematology (ASH) and the British Society for Hematology (BSH) have developed
ITP practice guidelines. These guidelines, based on expert opinion, differ in their recommendations for treatment. The ASH guidelines favor
therapy based on a low platelet count, and the more current BSH guidelines recommend a more conservative 'wait and watch' approach. In addition to treating children with severe
bleeding symptoms, there is a tendency (not evidence based) to treat early in order to prevent a life-threatening
bleeding episode, including
intracerebral hemorrhage.
Corticosteroids are a highly effective
therapy, inexpensive, and can usually increase the platelet count within hours to days. However, chronic or prolonged use is associated with toxicity. In the US, based on the knowledge of known toxicities of
corticosteroids, as well as the efficacy of alternative treatments (IV RhIG,
IVIG), many pediatricians prefer to treat with
IVIG and IV RhIG, reserving
corticosteroid treatment for serious
bleeding or refractory disease. However, in the UK, for the most part,
corticosteroids are used as first-line
therapy in children with
ITP.
Splenectomy is rarely indicated in children except for those with life-threatening
bleeding and chronic, severe
ITP with impairment of quality of life. For children who develop chronic or refractory
ITP, immunosuppressive drugs and/or
chemotherapy agents may offer some promise. However, the long-term effects of these drugs in children are unknown and they should not be considered unless there is unequivocal evidence that the patient is refractory to IV RhIG,
IVIG, and
corticosteroids. To date, virtually all of the randomized clinical trials conducted in children with
ITP have focused on platelet counts as the sole outcome measure. Only carefully designed, multicenter, randomized clinical trials comparing the effects of different treatment modalities in terms of
bleeding, quality of life, adverse effects, and treatment-related costs will be able to address the controversies surrounding childhood
ITP treatment and allow management of this condition to be based on scientific data rather than treatment philosophy.