Immune thrombocytopenia in adults: A single-centre review of demographics, clinical features and treatment outcomes.

OBJECTIVES: Primary immune thrombocytopenia (ITP) is a bleeding disorder characterised by an isolated thrombocytopenia in the absence of an alternative diagnosis. The condition is highly heterogeneous with some patients requiring multiple of therapy before achieving response. In this study, we collected data on a large cohort of primary ITP patients with the objective of identifying variables which may predict treatment requirements. METHODS: We collected data on 379 patients, 275 with a confirmed diagnosis of primary ITP included demographics, baseline laboratory results and treatments. These were compared against treatment responses and lines of therapy. RESULTS: Patients who presented with a platelet count of <30 × 109 /L or bleeding symptoms were observed to require more subsequent lines of therapy (P-value <0.001). 32% of patients (n = 87) received no treatment, and these patients had a significantly higher median count compared to those with required >2 lines of therapy (P-value <0.001). Superior response rates were demonstrated with thrombopoietin receptor agonists when compared with other agents irrespective of baseline characteristics. CONCLUSIONS: Platelet counts at diagnosis are a potentially strong predictive indicator of subsequent lines of therapy. Patients with bleeding symptoms at diagnosis were more likely to have lower median platelets counts.

The utility of ADAMTS13 in differentiating TTP from other acute thrombotic microangiopathies: results from the UK TTP Registry.

Thrombotic microangiopathies (TMAs) are frequently difficult to differentiate clinically, and measurement of ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) remains vital in thrombotic thrombocytopenic purpura (TTP) diagnosis. We retrospectively reviewed cases referred for ADAMTS13 testing, using UK TTP Registry screening data. Of a total 810 cases, 350 were confirmed as TTP. The 460 non-TTP cases comprised secondary TMAs (24·57%) and haemolytic uraemic syndrome (HUS) (27·17% aHUS, 2·83% Shiga-like toxin-producing E. coli [STEC]-HUS); the remainder were TMAs with no clear association, not TMAs, or had no confirmed diagnosis. ADAMTS13 levels were significantly lower in TTP than STEC-HUS, aHUS and other TMAs. TTP patients had significantly lower platelet count (15 × 10(9) /l; range 0-96) than aHUS (57 × 10(9) /l; range 13-145, P < 0·0001) or STEC-HUS (35 × 10(9) /l; range 14-106, P < 0·0001); they also had lower creatinine levels (92 µmol/l; range 43-374) than aHUS (255 µmol/l; range 23-941, P < 0·0001) and STEC-HUS (324 µmol/l; range 117-639, P < 0·0001). However, 12/34 (35·3%) aHUS patients had a platelet count <30 × 10(9) /l and 26/150 (17·3%) of TTP patients had a platelet count >30 × 10(9) /l; 23/150 (15·3%) of TTP patients had a creatinine level >150 µmol/l. This study highlights the wide variety of TMA presentations, and confirms the utility of ADAMTS13 testing in TTP diagnosis.