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Blood, 1 May 2001, Vol. 97, No. 9, pp. 2549-2554
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Morbidity and mortality in adults with idiopathic
thrombocytopenic purpura
Johanna E. A. Portielje,
Rudi G. J. Westendorp,
Hanneke
C. Kluin-Nelemans, and
Anneke Brand
From the Departments of Internal Medicine, Clinical
Epidemiology, and Hematology, Leiden University Medical Center, The
Netherlands.
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.

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