Long-term risk for haematologic disease in persistent, isolated mild thrombocytopenia

Individuals with persistent, isolated, mild thrombocytopenia (PIMT) had a much higher long-term risk of progressing towards immune thrombocytopenia (ITP) or haematologic neoplasia than healthy individuals. Furthermore, bleeding events and systemic autoimmunity were more frequently observed in individuals with PIMT than in healthy subjects.

Dr Nardeen Ayad (Massachusetts General Hospital, MA, USA) and colleagues studied the long-term risk of ITP and haematologic neoplasia in adults with PIMT of unknown aetiology over a course of >20 years [1]. The observational cohort study identified 91 patients from the Mass General Brigham research patient data registry who had platelet counts between 100–149 x 10⁹/L at ≥3 visitations between 1995 and 2004 and were diagnosed with PIMT of unknown aetiology. They were matched with 364 healthy subjects. The primary outcome of the study was the progression to haematologic disease.

After a median follow-up of 20.5 years, the 25-year cumulative incidence of haematologic disease among individuals with PIMT was 37.0%; the 25-year cumulative incidences of ITP and haematologic neoplasia were 26.0% and 15.7%, respectively. Compared with healthy controls and adjusted for age, the risk for haematologic disease was much higher in the PIMT group, with a sub-distribution HR of 19.0 (95% CI 8.4–43.0; P<0.001). The sub-distribution HRs for developing ITP and haematologic neoplasia were 71.1 (95% CI 9.4–538.7; P<0.001) and 10.3 (95% CI 3.8–28.0; P<0.001), respectively.

Dr Ayad mentioned that ISTH major/CRNM minor bleedings occurred in 26% of the patients in the PIMT arm over the course of follow-up and in 2% of the healthy control subjects. Furthermore, systemic autoimmunity was observed in 13% of the patients in the PIMT group and in 3% of the healthy individuals. Finally, 21% of the patients in the PIMT group and 6% of the healthy individuals died.

“We believe that our findings have significant implications for the initial diagnostic workup of patients with mild thrombocytopenia, as well as for the potential long-term surveillance and prognosis,” decided Dr Ayad.

1. Ayad N, et al. Long-Term Risk of Developing Immune Thrombocytopenia and Hematologic Neoplasia in Adults with Persistent, Isolated Mild Thrombocytopenia. Abstract 19, ASH 64^th Annual Meeting, 10–13 December 2022, New Orleans, LA, USA.

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Posted on 20 February 2023