Morbidity and mortality in adults with idiopathic thrombocytopenic purpura

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 wer e determined relative to the response 2 years after diagnosis, be cause mos t (93%) patients who ultimately attained platelet counts above 30.0 x 10(9) /L (30 000/muL) did so within this time frame. Complete follow-up for morta lity could be studied in 99% of patients and for morbidity in 95% of patien ts,with a mean of 10.5 years. Within 2 years after diagnosis, 4 patients di ed, 2 were lost to follow-up, and 12 were reclassified as having secondary immune thrombocytopenia. Of the remaining 134 patients, 114 (85%) had obtai ned platelet counts above 30.0 x 10(9)/L while all therapies had been disco ntinued. 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% confidenc e interval, 1.7-10.0). Bleeding and infection equally contributed to the de ath of these patients. Another 8 patients (6%) had platelet counts above 30 .0 x 10(9)/L while on maintenance therapy. Similar to patients with refract ory disease, these latter patients had considerably increased ITP-related h ospital admissions, but mortality was only slightly higher than in the gene ral population. In conclusion, most adults with ITP have a good outcome wit h infrequent hospital admissions and no excess mortality. The absence of gr oss morbidity and mortality in patients with moderate thrombocytopenia supp orts clinical practice refraining from further treatment. (Blood, 2001;97:2 549-2554) (C) 2001 by The American Society of Hematology.