BLOOD, 15 MARCH 2002 VOLUME 99, NUMBER 6
AVOIDANCE OF SPLENECTOMY WITH ANTI-D IN ITP
able to discontinue treatment and that some patients were still improving and achieving near normal platelet counts up to 38 months after diagnosis.
It is possible that a referral bias resulted in a disproportionate number of patients with less severe ITP entering the trial. However, 28 of 31 patients eligible for the study were enrolled; 15 had platelet counts below 15 3 109/L (15 000/mL) at study entry, and 14 had previously received both steroids and IVIG.
One of the disadvantages of this approach is that patients were seen more frequently for outpatient visits and often had lower platelet counts than if they had undergone successful splenectomy. In addition, patients with marginal platelet counts in the range of 30 3 109/L to 50 3 109/L (30 000-50 000/mL), although off treat- ment, could potentially have periodic crises. These crises might require additional treatment, including hospitalization, all of which could have been relieved by early splenectomy, if it was successful. However, no ITP-related hospitalizations occurred during the course of the study, and there were no episodes of serious bleeding symptoms in the 64 patient years of follow-up in this study; nor were there significant adverse events or decrease in quality of life. Furthermore, many patients avoided both surgery and the toxicity of long-term steroids by following this protocol. The rudimentary cost analysis in Table 3 shows that if a substantial proportion of patients can eventually discontinue treatment without undergoing splenectomy, estimated previously to be 47%,23 the use of intermit- tent IV anti-D, even for many months, may be cost-effective in comparison to all patients undergoing splenectomy.
Predicting which patients will improve over time would further improve the cost-effectiveness and give patients a better idea of what they could expect if they do not undergo early splenectomy. Patients with higher platelet counts at study entry, in particular those with platelet counts above 14 3 109/L (14 000/mL), appear to be more likely to improve. No other variables—ie, glycocalicin
levels, sCD16 levels, or blood type—were predictive, although the sample sizes were small.
Overall, this study proposes that splenectomy, which is only successful in 60% to 70% of patients,9-11 may not be immediately necessary in all adults with recently diagnosed ITP who do not go into remission with initial steroids. It suggests that most adults with ITP have a tendency for their platelet counts to improve over time. It also shows that IV anti-D is a safe and effective treatment in approximately two thirds of Rh1, nonsplenectomized adults with ITP and can be used as a steroid-sparing agent. There is no reason to believe that anti-D is curative or that it affected the natural history of the ITP in these adults. Rather, it appeared to give patients time to improve on their own.
ITP is clearly a heterogeneous disease. Better understanding of the pathology of this disease and identification of good predictors of response to splenectomy and other therapies would help to define treatment strategies that would be most appropriate for individual patients.
We thank Howard Fleit at the State University of New York at Stony Brook for performing the sCD16 assays, Ginette Lanoix for technical assistance, and all the physicians and their staff whose patients participated in this study: Drs Bernard Bernhardt, Edward Amorosi, Roy Berger, Ellin Berman, Laurence Bilsky, Terry Davies, Richard Furman, Gerard Hellman, Gary Horbar, Alexandra Ikeguchi, Robert Jacobson, Shirley Levine, Neal Lewin, Stuart Lewis, Eva Levitan, William Lipera, Taibor Moskowitz, Vijay Roy, Eduardo Saponara, William Solomon, Richard Taubman, Zella Zeigler, and Robert Zielinski.
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