Morbidity and mortality in adults with idiopathic thrombocytopenic purpura

Abstract

To study outcomes of adults with idiopathic thrombocytopenic purpura (ITP), we performed a follow-up study in a cohort of 152 consecutive patients who were treated according to a well-defined algorithm. Long-term outcomes were determined relative to the response 2 years after diagnosis, because most (93%) patients who ultimately attained platelet counts above 30.0 × 109/L (30 000/μL) did so within this time frame. Complete follow-up for mortality could be studied in 99% of patients and for morbidity in 95% of patients, with a mean of 10.5 years. Within 2 years after diagnosis, 4 patients died, 2 were lost to follow-up, and 12 were reclassified as having secondary immune thrombocytopenia. Of the remaining 134 patients, 114 (85%) had obtained platelet counts above 30.0 × 109/L while all therapies had been discontinued. These patients had a long-term mortality risk equal to the general population. Twelve of 134 patients (9%), all with severe thrombocytopenia, had refractory disease and suffered a mortality risk of 4.2 (95% confidence interval, 1.7-10.0). Bleeding and infection equally contributed to the death of these patients. Another 8 patients (6%) had platelet counts above 30.0 × 109/L while on maintenance therapy. Similar to patients with refractory disease, these latter patients had considerably increased ITP-related hospital admissions, but mortality was only slightly higher than in the general population. In conclusion, most adults with ITP have a good outcome with infrequent hospital admissions and no excess mortality. The absence of gross morbidity and mortality in patients with moderate thrombocytopenia supports clinical practice refraining from further treatment.